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2.3500+0.1800 (+8.29%)
As of 12:03PM EDT. Market open.
Nice Robust Volume too!
These 2 Penny Stocks Are Poised for a Big Rally, Says Oppenheimer
TipRanks
Tue, August 9, 2022 at 5:39 PM
Gamida Cell (GMDA)
The first potentially high-yielding penny stock we’ll look at is Gamida Cell, a clinical-stage biopharma researcher with a focus on cell therapy treatments for severe blood disorders and blood cancers. The company features a proprietary research platform using nicotinamide to expand on various natural cell types, especially natural killer (NK) cells while maintaining their potency. Moving from this platform, the company has developed omidubicel, a new drug under investigation as a treatment for hematological malignancies.
Omidubicel has completed Phase 3 testing in that area – blood cancers – and the company has progressed to the Biologics License Application to the FDA. The next regulatory step is review from the agency, with a PDUFA date of January 30, 2023. According to the company, ‘If approved, omidubicel will be the first allogeneic advanced stem cell therapy donor source for patients with blood cancers in need of a stem cell transplant.’ Gamida also has a Phase 1/2 study ongoing, investigating omidubicel as a treatment for severe aplastic anemia.
Also on the clinical track is Gamida’s second drug candidate, GDA-201. The drug is being evaluated in the treatment of non-Hodgkin Lymphoma. A clinical hold from last fall was removed earlier this year, and Gamida is proceeding with Phase 1/2 clinical trials.
Based on the potential of the company's drug candidates, and its $2.09 share price, Oppenheimer analyst Mark Breidenbach thinks that now is the time to get in on the action.
Breidenbach sees the likely FDA approval of omidubicel as the main catalyst for this stock, writing: “We remain confident in omidubicel’s regulatory approval based on its strong Phase 3 data... Relative to unmanipulated cord blood, omidubicel demonstrated faster neutrophil engraftment and platelet recovery— translating to significant reductions in serious infections and hospitalization time. We believe omidubicel could represent an attractive new option due to its reliable procurement speed (~30 days) and near-universal HLA compatibility... In preparation for the potential launch, Gamida has engaged with >45 high-volume US transplant centers and has begun to proactively educate payers to help ensure coverage shortly after approval."
To this end, Breidenbach puts an Outperform (i.e. Buy) rating on Gamida shares, and his $15 price target suggests potential for a whopping 618% upside in the next 12 months. (To watch Breidenbach’s track record, click here)
Gamida Cell Announces the Date of Its Second Quarter 2022 Financial Results and Webcast
https://finance.yahoo.com/news/gamida-cell-announces-date-second-120000917.html
BOSTON, August 08, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, today announced that the company will host a conference call and live audio webcast on Monday, August 15, 2022, at 8:00 a.m. ET to review its second quarter 2022 financial results and provide an update on the company.
To access the conference call, please register here and be advised to do so at least 10 minutes prior to joining the call. A live webcast of the conference call can be accessed in the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com. A replay of the webcast will be available approximately two hours after the event, for approximately 30 days.
Yes, I thought so as well.
I guess we just need to look at it as a chance to accumulate more cheap shares!
Murocman
Gamida stock rises 13% as FDA grants priority review to stem cell therapy omidubicel
Aug. 01, 2022 7:21 AM ETGamida Cell Ltd. (GMDA)
By: Ravikash, SA News Editor
The U.S. Food and Drug Administration (FDA) granted priority review to Gamida Cell's (NASDAQ:GMDA) application seeking approval of stem cell therapy omidubicel to treat patients with blood cancers in need of an allogenic hematopoietic stem cell transplant.
The FDA accepted the company's Biologics License Application (BLA) and is expected to make a decision by Jan. 30, 2023.
Under priority review, the FDA's goal is to take action within six months,
compared to 10 months under standard review.
Gamida said the FDA is not planning to hold an Advisory Committee
meeting as part of the BLA review.
The company noted that the BLA was backed by data from a phase 3 trial.
Upon FDA approval, omidubicel will be manufactured at the Gamida's manufacturing facility in Israel, the company added.
Gamida Cell Announces FDA Acceptance of Biologics License Application for Omidubicel with Priority Review
https://finance.yahoo.com/news/gamida-cell-announces-fda-acceptance-110000411.html
- If approved, omidubicel will be the first allogeneic advanced stem cell therapy donor source for patients with blood cancers in need of a stem cell transplant -
- PDUFA target action date is January 30, 2023 -
- Company to host conference call on BLA acceptance and commercial opportunity today at 8:00 a.m. ET -
BOSTON, August 01, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, announced today that the U.S. Food and Drug Administration (FDA) has accepted for filing the Company’s Biologics License Application (BLA) for omidubicel for the treatment of patients with blood cancers in need of an allogenic hematopoietic stem cell transplant. Omidubicel is a first-in-class, advanced NAM-enabled stem cell therapy candidate with breakthrough and orphan drug designations.
The FDA granted Priority Review for the BLA and has set a Prescription Drug User Fee Act (PDUFA) target action date of January 30, 2023. The FDA grants Priority Review to product applications that, if approved, would provide significant improvements in the safety or effectiveness of the treatment, diagnosis, or prevention of serious conditions when compared to standard applications. At this time, the FDA has indicated that it is not planning an advisory committee meeting as part of the BLA review.
"The FDA’s acceptance of our BLA with Priority Review signifies a critical milestone in our mission to deliver a new stem cell therapy option for patients in need of a donor for an allogeneic stem cell transplant," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We are encouraged by the positive and sustained follow-up results from patients participating in the Phase 3 trial of omidubicel, including a positive overall survival trend one-year out from treatment. These results provide promising rationale that, if approved, omidubicel could become a treatment of choice for patients in need of an allo-HSCT transplant. We look forward to working with the FDA throughout the review process to bring omidubicel to patients as quickly as possible."
Upon FDA approval, omidubicel will be manufactured at the Gamida Cell owned manufacturing facility in Israel. This is a newly constructed, state-of-the-art, modular facility which allows for additional capacity to be added to address growing demand. Batches from this facility were used to support the BLA for omidubicel and the facility is currently manufacturing clinical batches.
The omidubicel BLA is supported by the statistically significant results from Gamida Cell’s pivotal Phase 3 study, the results of which were published in Blood, the official journal of the American Society of Hematology. Results for the study’s primary endpoint, the median time to neutrophil engraftment in patients with hematologic malignancies undergoing allogeneic bone marrow transplant with omidubicel compared to standard umbilical cord blood (UCB), demonstrated a median time to neutrophil engraftment of 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p < 0.001). The secondary endpoints of this Phase 3 study were all achieved and were statistically significant. These secondary endpoints were platelet engraftment, the rate of infection, and days alive and out of hospital. Omidubicel was generally well tolerated in the Phase 3 study.
The full Blood manuscript is available here: https://ashpublications.org/blood/article/doi/10.1182/blood.2021011719/476235/Omidubicel-Versus-Standard-Myeloablative-Umbilical.
In 2019, approximately 8,000 patients who were 12 years old and up with hematologic malignancies underwent an allogeneic stem cell transplant in the United States.1 Unfortunately, it is estimated that another 1,200 patients were eligible for transplant but could not find a donor source.2 If approved, omidubicel has the potential to improve outcomes for patients based on transplanter feedback and to potentially increase access for patients to get to transplant. If approved, omidubicel has the potential to treat approximately 2,000 – 2,500 patients each year in the U.S.
Conference Call Information
Gamida Cell will host a conference call today, August 1, 2022, at 8:00 a.m. ET to discuss this update. To access the conference call, please register here and please be advised to do so at least 10 minutes prior to joining the call. A live webcast of the conference call can be accessed in the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com. A replay of the webcast will be available approximately two hours after the event, for approximately 30 days.
About Omidubicel
Omidubicel is an advanced cell therapy candidate developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment in comparison to standard umbilical cord blood in an international, multi-center, randomized Phase 3 study (NCT0273029) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.
Omidubicel is an investigational stem cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit https://www.gamida-cell.com.
About NAM Technology
Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.
About Gamida Cell
Gamida Cell is pioneering a diverse immunotherapy pipeline of potentially curative cell therapy candidates for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapy candidates with potential to redefine standards of care. These include omidubicel, an investigational product with potential as a life-saving alternative for patients in need of bone marrow transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.
Cautionary Note Regarding Forward Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of initiation and progress of, and data reported from, the clinical trials of Gamida Cell’s product candidates (including omidubicel), regulatory filings submitted to the FDA (including the potential timing of the FDA’s review of the BLA for omidubicel), commercialization planning efforts, and the potentially life-saving or curative therapeutic and commercial potential of Gamida Cell’s product candidates (including omidubicel), and Gamida Cell’s expectations for the expected clinical development milestones set forth herein. Any statement describing Gamida Cell’s goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions, including those related to the impact that the COVID-19 pandemic could have on our business, and including the scope, progress and expansion of Gamida Cell’s clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such product candidates. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cell’s Quarterly Report on Form 10-Q, filed with the Securities and Exchange Commission (SEC) on May 12, 2022, as amended, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cell’s actual results could differ materially and adversely from those anticipated or implied thereby. Although Gamida Cell’s forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Gamida Cell. As a result, you are cautioned not to rely on these forward-looking statements.
1CIBMTR 2019 – allogeneic transplants in patients 12+ years with hematological malignancies.
2Gamida Cell market research
View source version on businesswire.com: https://www.businesswire.com/news/home/20220801005265/en/
Contacts
For investors:
Courtney Turiano
Stern Investor Relations, Inc.
Courtney.Turiano@sternir.com
1-212-362-1200
For media:
Heather DiVecchia
Chief of Staff
Heather@gamida-cell.com
1-617-892-9083
Yeah I read it and it seems like sometimes BLA approval grants the ability to market and other times there is still a PDUFA approval needed. That’s what I was confused about in Gamida’s case.
At any rate, this morning’s PR answered that question.
Good news and I would hope this candidate would be about as close as a sure thing as there is for approval!
Will definitely be accumulating more on any dips.
Murocman
Read this:
https://www.nuventra.com/resources/blog/regulatory-differences-between-an-nda-bla/#:~:text=To%20formally%20request%20approval%20to,to%20traditional%20small%20molecule%20drugs.
What are the Regulatory Differences Between an NDA and BLA?
by Scientific Writing Team
To formally request approval to market a new drug in the United States, Sponsors must submit either a New Drug Application (NDA) or a Biologics License Application (BLA) to the FDA. As their names suggest, BLAs relate to biological products while NDAs generally pertain to traditional small molecule drugs. While they share the same goal of obtaining marketing approval, they also vary slightly in content and scope. In this post, we explore the similarities and differences between the two marketing applications, as well as special considerations based on recent regulatory changes.
What are New Drug Applications (NDA) & Biologics License Applications (BLA)?
An NDA is an application to permit the sale and marketing of a new drug in the United States. A traditional NDA consists of data and information about the drug as gained from both nonclinical and clinical studies, as well as a summary of formulation development and manufacturing processes, and proposed labeling information to be included in the drug’s packaging.
In general, an NDA should contain enough data for the FDA to determine if the drug is safe and effective for its proposed use, if the benefits of taking the drug outweigh the risks, and if the drug product is manufactured in a way that preserves its identity, strength, quality, and purity. Drugs that are approved via an NDA pathway are regulated under Section 505 of the Food, Drug, & Cosmetics (FD&C) Act.
A BLA is a request to introduce, or deliver for introduction, a biological product into interstate commerce. Like an NDA, a BLA should include all information about the biological product that was gained over the development process and should demonstrate the biologic’s safety, purity, and potency. The BLA also contains the proposed labeling information to be included in the drug’s packaging. Per the Biologics and Price Competition and Innovation (BPCI) Act of 2009, as of March 23rd, 2020, all biological products must be approved through the BLA pathway, and therefore will be licensed under Section 351 of the Public Health Service (PHS) Act, in addition to being regulated by the FD&C Act.
What is a Biological Product?
Biological products are a subset of drugs defined by Section 351 of the PHS Act as a “virus, therapeutic serum, toxin, antitoxin, vaccine, blood, blood component or derivative, allergenic product, or analogous product, … applicable to the prevention, treatment, or cure of a disease or condition of human beings.” The definition was later amended by the BPCI Act of 2009 and the Further Consolidated Appropriations Act of 2020 to include proteins (excluding peptides). Because biological products are typically derived from living systems, their large, complex structures are often difficult to characterize. This is a key distinction from traditional drug molecules, which are chemically synthesized and structurally both simpler and smaller in size.
The manufacturing process for biological products is also more complicated, due to genetic variability in the source material. Because of this, it is critical that BLAs contain a thorough description of product development and relevant manufacturing procedures, as well as all steps taken to ensure that the final biological product performs consistently across batches.
Key Differences Between BLAs & NDAs
While BLAs and NDAs serve the same purpose of gaining approval to market a drug in the United States, they differ slightly in terms of their application content and submission requirements. Regarding approval criteria, NDAs must fulfill three conditions:
The drug is safe and effective for the proposed use and that the benefits outweigh the risks
The labeling is appropriate and contains all necessary information about the drug
Manufacturing methods preserve the drug’s identity, strength, quality, and purity
Similarly, contents of a BLA should establish that the biological product is safe and potent; however, because biological products are processed from living material, BLA content must also demonstrate purity specifically in terms of showing that the final product does not contain extraneous material.
Due to the complexities of manufacturing biological products, a pre-license inspection of the facility is generally required before a BLA is approved. Pre-approval inspections sometimes also take place during an NDA review, but are typically conducted based on risk assessment by the Agency.
Once a BLA is approved, the Sponsor is granted a license for the biological product, which permits its introduction into interstate commerce per Section 351 of the PHS Act. This licensing process is not a part of the NDA, as drugs that are approved by NDA are regulated only by the FD&C Act, and not the PHS Act.
Until very recently, certain biological products could be approved under an NDA rather than a BLA. However, according to the Biologics Price Competition and Innovation Act (further discussed below) this is no longer the case, and all biological product approvals now occur through a BLA.
Regulatory Agencies
There are two Centers within the FDA that are responsible for the review and approval of drug marketing applications and general regulatory oversight: the Center for Drug Evaluation and Research (CDER) and the Center for Biologics Evaluation and Research (CBER). While all conventional drug products (i.e., small molecules) are regulated by CDER, biological products can be regulated by either CDER or CBER, depending on the product’s classification as discussed above.
The majority of BLA submissions are assigned to CBER; however, BLAs for certain biological product categories are reviewed by CDER instead. These product categories include monoclonal antibodies for in vivo use, most proteins for therapeutic use (e.g., cytokines, enzymes, and other novel proteins except those assigned to CBER, such as vaccines and blood products), immunomodulators, and growth factors. Regardless of the category, NDAs for all drug products fall under the jurisdiction of CDER.
Key Similarities Between BLA & NDA
Like an NDA, a BLA is submitted to the FDA in order to market a new drug in the US. As they share the same goal of obtaining marketing approval, NDAs and BLAs are similar in that both must contain enough information to demonstrate the efficacy and safety of the drug, as well as demonstrate an ideal risk:benefit ratio, in order to be successful. Additionally, many of the same regulations apply to NDAs and BLAs, including labeling and advertising rules, accelerated approval pathways, pediatric study requirements, and PDUFA fees.
Regardless of whether a Sponsor is submitting an NDA or BLA, the same pre-marketing regulations apply. This includes initial filing of an IND and subsequent maintenance of the IND throughout the drug development program until the marketing application is submitted. The structure and contents of the IND do not differ between drugs and biological products.
Biologics Price Competition and Innovation Act
It is important to note that on March 23, 2020, the Biologics Price Competition and Innovation (BPCI) Act went into effect. Along with introducing an abbreviated approval pathway for highly similar biological products (i.e., biosimilars), this act mandated that moving forward, all biological products must be submitted for marketing approval through a BLA, and not an NDA. For biological products that had been previously approved via NDA (e.g., protein products), the approved marketing application will be “deemed to be a license” (i.e., serve as an approved BLA) for the biological product under Section 351 of the PHS Act.
Conclusions
NDAs and BLAs are the two types of applications that are submitted in order to market a new drug in the United States. While they are both submitted to gain FDA drug approval, they differ in terms of product categories, approval criteria, and certain regulations. Nuventra consultants are widely experienced in the preparation and submission of both BLAs and NDAs for numerous drugs and indications.
I always get BLA’s and NDA’s mixed up.
If the BLA is approved, the therapeutic is approved, subject to any restrictions, limitations or required follow ups with the FDA, correct?
Murocman
Gamida Cell (GMDA) Investor Presentation- Slideshow
Jun. 24, 2022 2:24 PM ETGamida Cell Ltd. (GMDA)
The following slide deck was published by Gamida Cell Ltd. in conjunction with this event.
https://seekingalpha.com/article/4520221-gamida-cell-gmda-investor-presentation-slideshow
Gamida Cell Ltd.'s (NASDAQ:GMDA) Shift From Loss To Profit
By Simply Wall St
PublishedJune 15, 2022
https://simplywall.st/stocks/us/pharmaceuticals-biotech/nasdaq-gmda/gamida-cell/news/gamida-cell-ltds-nasdaqgmda-shift-from-loss-to-profit?utm_medium=article&utm_source=robinhood
We feel now is a pretty good time to analyse Gamida Cell Ltd.'s (NASDAQ:GMDA) business as it appears the company may be on the cusp of a considerable accomplishment. Gamida Cell Ltd., a clinical-stage biopharmaceutical company, develops cell therapies to cure blood cancers and serious hematologic diseases. With the latest financial year loss of US$90m and a trailing-twelve-month loss of US$91m, the US$110m market-cap company amplified its loss by moving further away from its breakeven target. As path to profitability is the topic on Gamida Cell's investors mind, we've decided to gauge market sentiment. We've put together a brief outline of industry analyst expectations for the company, its year of breakeven and its implied growth rate.
View our latest analysis for Gamida Cell
Consensus from 6 of the American Biotechs analysts is that Gamida Cell is on the verge of breakeven. They expect the company to post a final loss in 2023, before turning a profit of US$8.4m in 2024. The company is therefore projected to breakeven around 2 years from now. In order to meet this breakeven date, we calculated the rate at which the company must grow year-on-year. It turns out an average annual growth rate of 66% is expected, which is extremely buoyant. Should the business grow at a slower rate, it will become profitable at a later date than expected.
earnings-per-share-growth
NasdaqGM:GMDA Earnings Per Share Growth June 15th 2022
We're not going to go through company-specific developments for Gamida Cell given that this is a high-level summary, however, bear in mind that generally biotechs, depending on the stage of product development, have irregular periods of cash flow. This means that a high growth rate is not unusual, especially if the company is currently in an investment period.
Before we wrap up, there’s one issue worth mentioning. Gamida Cell currently has a debt-to-equity ratio of over 2x. Generally, the rule of thumb is debt shouldn’t exceed 40% of your equity, which in this case, the company has significantly overshot. Note that a higher debt obligation increases the risk in investing in the loss-making company.
Next Steps:
This article is not intended to be a comprehensive analysis on Gamida Cell, so if you are interested in understanding the company at a deeper level, take a look at Gamida Cell's company page on Simply Wall St. We've also compiled a list of relevant factors you should look at:
Valuation: What is Gamida Cell worth today? Has the future growth potential already been factored into the price? The intrinsic value infographic in our free research report helps visualize whether Gamida Cell is currently mispriced by the market.
Management Team: An experienced management team on the helm increases our confidence in the business – take a look at who sits on Gamida Cell’s board and the CEO’s background.
Gamida Cell to Present Corporate Highlights at the JMP Securities Life Sciences Conference
https://finance.yahoo.com/news/gamida-cell-present-corporate-highlights-110000806.html%
BOSTON, June 13, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, announces that company management will present its corporate highlights at the JMP Securities Life Sciences Conference, June 16, 2022 with a presentation at 2:00 p.m. ET in New York, NY.
Management will discuss 2022 catalysts and potential milestones including the U.S. market opportunity for omidubicel upon potential U.S. Food and Drug Administration (FDA) approval, accelerating the development of its first-in-class NAM-enabled natural killer (NK) cell therapy candidate, GDA-201, as a potential new approach for patients with follicular and diffuse large B-cell lymphomas, and expansion of its NAM-enabled cell therapy pipeline with multiple next-generation, genetically engineered NK cells.
A webcast of the event will be available on the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com, and will be available for at least 14 days following the event.
The Company also announces the resignation of Ofer Gonen from its Board of Directors, effective June 9, 2022. Mr. Gonen will be joining MediWound Ltd. as chief executive officer effective June 30, 2022.
https://finance.yahoo.com/news/gamida-cell-appoints-ivan-m-110000306.html
Gamida Cell Appoints Ivan M. Borrello, M.D., Expert in Immuno-Oncology, Cell Therapies, and Bone Marrow Transplant to Board of Directors
https://finance.yahoo.com/news/gamida-cell-appoints-ivan-m-110000306.html
BOSTON, June 10, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, announces the appointment of Ivan M. Borrello, M.D. to its Board of Directors, effective June 9, 2022. Dr. Borrello is an Associate Professor of Oncology at the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins and a renowned physician and author who has made major contributions to better the understanding of immunotherapies and the treatment of hematologic malignancies as well as bone marrow transplant. He will also be joining Gamida Cell’s Science and Technology Committee.
The Company also announces the resignation of Ofer Gonen from its Board of Directors, effective June 9, 2022. Mr. Gonen will be joining MediWound Ltd. as chief executive officer effective June 30, 2022.
"I am excited to have Ivan join our Board of Directors. As a distinguished physician in hematologic malignancies, cellular therapeutics, and immunotherapies, Ivan has significantly contributed to the progress in the clinical oncology field," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "His deep knowledge and clinical experience in immune-based therapies, most notably in establishing the first adoptive T-cell clinical trials at Johns Hopkins, will continue to support Gamida Cell as we advance our pipeline of NAM-enabled cell therapy candidates for patients with blood cancers and other serious blood disorders. In addition, on behalf of the entire Board of Directors, I want to thank Ofer for his longstanding service to Gamida Cell. We wish him every success going forward."
"It is a privilege to join Gamida Cell’s Board of Directors, as the company leverages its truly innovative NAM-technology to develop potentially curative cell therapy candidates," said Dr. Borrello. "I believe the Company's novel technology holds tremendous promise, which is supported by remarkable clinical data, coupled with their deep expertise in oncology and the development of cellular therapy candidates. I look forward to supporting Gamida Cell as it continues to advance its growing pipeline of cell therapy candidates for patients with solid tumor and blood cancers and other serious blood diseases."
Dr. Borrello’s clinical research interest is focused on developing immune-based therapies for the treatment of multiple myeloma. His laboratory research has focused on the development of a novel approach of adoptive T-cell therapy utilizing marrow infiltrating lymphocytes (MILs) as a more tumor-specific T-cell approach. He has held multiple appointments at Johns Hopkins University, including Instructor, Immunotherapy and Hematopoiesis, Johns Hopkins Oncology Center from 1999 to 2000, and Assistant Professor, Immunotherapy and Hematopoiesis, Hematologic Malignancies, Johns Hopkins Oncology Center, from 2001 to 2008. Dr. Borrello is also the director of the myeloma program and medical director of the Cell Therapy Lab. Dr. Borrello received his medical degree from the Medical College of Virginia and completed his residency at the University of Chicago and fellowship at Johns Hopkins.
Gamida Cell to Present Corporate Highlights at the Jefferies Healthcare Conference
Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, announces that company management will present its corporate highlights at the Jefferies Healthcare Conference, June 8, 2022 with a presentation at 11:00 a.m. ET in New York, NY.
Management will discuss 2022 catalysts and potential milestones including the U.S. market opportunity for omidubicel upon potential U.S. Food and Drug Administration approval, accelerating the development of its first-in-class NAM-enabled natural killer (NK) cell therapy candidate, GDA-201, as a potential new approach for patients with follicular and diffuse large B-cell lymphomas, and expansion of its NAM-enabled cell therapy pipeline with multiple next-generation, genetically engineered NK cells.
A webcast of the event will be available on the “Investors & Media” section of Gamida Cell’s website at www.gamida-cell.com, and will be available for at least 14 days following the event.
Omidubicel, if approved, has the potential to improve outcomes for patients based on transplanter feedback and to potentially increase access for patients to get to transplant. Omidubicel, if approved, has the potential to treat approximately 2,000 – 2,500 patients each year in the U.S.
Gamida Cell Completes Rolling Biologics License Application Submission to the FDA for Omidubicel
https://finance.yahoo.com/news/gamida-cell-completes-rolling-biologics-110000831.htmlGMDA
- Omidubicel is a first-in-class, advanced NAM-enabled stem cell therapy candidate being evaluated as the first potential allogeneic advanced cell therapy donor source for patients with blood cancers in need of a transplant –
- Omidubicel has Orphan Drug Designation and Breakthrough Therapy Designation -
BOSTON, June 02, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, today announced completion of the rolling Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) for omidubicel for the treatment of patients with blood cancers in need of an allogenic hematopoietic stem cell transplant.
"The BLA submission marks an important milestone for both Gamida and the transplant community, as omidubicel has the potential to be the first approved advanced cell therapy product for allogeneic stem cell transplantation," said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. "Completion of this BLA submission is a key inflection point in our mission to deliver a new treatment option for patients with blood cancers. We look forward to working closely with the FDA to bring this potentially important therapy to patients."
The FDA has 60 days to determine whether the BLA for omidubicel is acceptable for filing. The omidubicel BLA is supported by the statistically significant results from Gamida Cell’s pivotal Phase 3 study, the results of which were published in Blood, the official journal of the American Society of Hematology. For the study’s primary endpoint, the median time to neutrophil engraftment in patients with hematologic malignancies undergoing allogeneic bone marrow transplant receiving omidubicel compared to standard umbilical cord blood (UCB), the median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p < 0.001).
In key secondary endpoints of this Phase 3 study: platelet engraftment was significantly accelerated [55 percent of patients randomized to omidubicel achieving platelet engraftment by day 42, compared to 35 percent for the comparator (p = 0.028)]; the rate of infection was significantly reduced [cumulative incidence of first grade 2 or grade 3 bacterial or invasive fungal infection for patients randomized to omidubicel of 37 percent, compared to 57 percent for the comparator (p = 0.03)]; and hospitalization in the first 100 days after transplant was significantly reduced [median number of days alive and out of hospital for patients randomized to omidubicel of 61 days, compared to 48 days for the comparator (p = 0.005)]. Omidubicel was generally well tolerated in the Phase 3 study.
The full Blood manuscript is available here: https://ashpublications.org/blood/article/doi/10.1182/blood.2021011719/476235/Omidubicel-Versus-Standard-Myeloablative-Umbilical.
About Omidubicel
Omidubicel is an advanced cell therapy candidate developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment in comparison to standard umbilical cord blood in an international, multi-center, randomized Phase 3 study (NCT0273029) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.
Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit https://www.gamida-cell.com.
M arket Opportunity
In 2019, approximately 8,000 patients who were 12 years old and up with hematologic malignancies underwent an allogeneic stem cell transplant.1 Unfortunately, it is estimated that another 1,200 patients were eligible for transplant but could not find a donor source.2 Omidubicel, if approved, has the potential to improve outcomes for patients based on transplanter feedback and to potentially increase access for patients to get to transplant. Omidubicel, if approved, has the potential to treat approximately 2,000 – 2,500 patients each year in the U.S.
About NAM Technology
Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.
Gamida Cell Announces Opening to Enrollment of Company-Sponsored Phase 1/2 Study of NK Cell Therapy Candidate GDA-201
https://finance.yahoo.com/news/gamida-cell-announces-opening-enrollment-110000783.html
BOSTON, June 01, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapy candidates for patients with hematologic and solid cancers and other serious diseases, today announced the activation of the initial clinical sites to screen and enroll patients in the company-sponsored Phase 1/2 study evaluating a cryopreserved formulation of GDA-201, a readily available cell therapy candidate for the treatment of follicular and diffuse large B cell lymphomas (NCT05296525). On April 26, 2022, Gamida had announced that the U.S. Food and Drug Administration (FDA) cleared its investigational new drug (IND) application and removed the clinical hold for a cryopreserved formulation of GDA-201.
"We are excited to be screening patients for enrollment in our company-sponsored Phase 1/2 clinical study of our novel, cryopreserved formulation of GDA-201, which has the potential to address the significant unmet need that exists for patients with follicular and diffuse large B cell lymphomas having relapsed or refractory disease," said Ronit Simantov, M.D., chief medical and scientific officer of Gamida Cell. "As described in previously announced clinical data, an investigator-sponsored (IS) study evaluating the fresh formulation of GDA-201 demonstrated encouraging results in heavily pretreated patients with lymphoma. With the initiation of enrollment, we look forward to dosing the first patient in our clinical study of the novel cryopreserved formulation of GDA-201."
GDA-201 leverages Gamida Cell’s proprietary NAM technology platform to expand the number and functionality of NK cells to direct tumor cell killing properties and antibody-dependent cellular cytotoxicity (ADCC). In an investigator-sponsored Phase 1/2 study in patients with relapsed or refractory lymphoma, treatment with the fresh formulation of GDA-201 with rituximab demonstrated significant clinical activity. Of the 19 patients with non-Hodgkin lymphoma (NHL), 13 complete responses and one partial response were observed, with an overall response rate of 74% and a complete response rate of 68%. The most common Grade 3/4 adverse events were thrombocytopenia, hypertension, neutropenia, febrile neutropenia, and anemia. At the December 2021 Annual Meeting of American Society of Hematology, two-year follow-up data were reported on outcomes and cytokine biomarkers associated with survival. The data demonstrated a median duration of response of 16 months (range 5-36 months) and an overall survival at two years of 78% (95% CI, 51%–91%). In the IS study, GDA-201 was well-tolerated and no dose-limiting toxicities were observed in 19 patients with NHL and 16 patients with multiple myeloma.
The study of the cryopreserved formulation of GDA-201 is currently open to enrollment at Henry Ford Health (Detroit, MI) and the Masonic Cancer Center at the University of Minnesota; additional sites will be added in the coming months and updated in Clinicaltrials.gov (NCT05296525). The Phase 1 portion of the study is designed to evaluate the safety of GDA-201 in patients with follicular lymphoma (FL), diffuse large B-cell lymphoma (DLBCL)/high grade B-cell lymphoma (HGBCL), marginal zone lymphoma or mantle cell lymphoma. The Phase 2 expansion phase is designed to evaluate the safety and efficacy of GDA-201 in two patient cohorts, FL and DLBCL/HGBCL. The study will include patients who have relapsed or refractory lymphoma after at least two prior treatments, which may include CAR-T or stem cell transplant.
About NAM Technology
Our NAM-enabling technology, supported by positive Phase 3 data, is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.
About GDA-201
Gamida Cell applied the capabilities of its nicotinamide (NAM)-enabled cell expansion technology to develop GDA-201, an innate NK cell immunotherapy candidate for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical trial results. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, GDA-201 improves antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells. There are approximately 40,000 patients with relapsed/refractory lymphoma in the US and EU, which is the patient population that will be studied in the GDA-201 Phase 1/2 clinical trial.
For more information about GDA-201, please visit https://www.gamida-cell.com. For more information on the Phase 1/2 clinical trial of GDA-201, please visit www.clinicaltrials.gov.
GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
Gamida Cell to Present Corporate Highlights at the H.C. Wainwright Global Life Sciences Conference
May 17 2022 - 04:30PM
Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with hematologic and solid cancers and other serious diseases, today announced that company management will present at the upcoming H.C. Wainwright Global Life Sciences Conference. A pre-recorded presentation will become available to registered conference attendees on May 24, 2022 at 7:00 a.m. ET. Management will discuss 2022 catalysts and potential milestones including the U.S. launch opportunity for omidubicel upon potential U.S. Food and Drug Administration approval, accelerating the development of its first-in-class NAM-enabled natural killer (NK) cell therapy candidate, GDA-201, as a potential new approach for patients with follicular and diffuse large B-cell lymphomas, and expansion of its NAM-enabled cell therapy pipeline with multiple next-generation, genetically engineered NK cells.
A webcast of the presentation will be available on the “Investors & Media” section of Gamida Cell’s website at www.gamida-cell.com, and will be available for at least 14 days following the event.
Gamida Cell Ltd. (GMDA) Management on Q1 2022 Results - Earnings Call Transcript
May 10, 2022 11:26 AM ETGamida Cell Ltd. (GMDA)
Gamida Cell Ltd. (NASDAQ:GMDA) Q1 2022 Results Conference Call May 10, 2022 8:00 AM ET
Company Participants
Heather DiVecchia - Chief of Staff
Michele Korfin - Chief Operating Officer and Chief Commercial Officer
Ronit Simantov - Chief Medical Officer and Chief Scientific Officer
Shai Lankry - Chief Financial Officer
Conference Call Participants
Eric Musonza - Evercore
Edward Tenthoff - Piper Sandler
Gilbert Blum - Needham and Company
Matthew Cross - Alliance Global Partners
Operator
Ladies and gentlemen, thank you for standing by. Welcome to Gamida Cell’s Conference Call for the First Quarter 2022 Financial Results. My name is Carmen and I will be your Operator for today’s call. Please be advised that this call is being recorded at Gamida Cell’s request.
Now, I would like to introduce your host for today’s conference, Heather DiVecchia, Gamida Cell’s Chief of Staff. Please go ahead.
Heather DiVecchia
Thank you, Carmen and good morning, everyone. Welcome to today’s call during which we will provide an update on the Company and review our financial results for the first quarter of 2022. Earlier this morning, we issued a press release summarizing our financial results and progress across the Company, which is available on our website at website at www.gamidacell.com.
Please note that unfortunately, our Chief Executive Officer, Julian Adams is unable to join us this morning due to contracting COVID-19. Company operations are unaffected and Julian has advised us that his symptoms are improving, and he expects to recover shortly.
With that here with me on our call today are Michele Korfin, our Chief Operating Officer and Chief Commercial Officer; Ronit Simantov, our Chief Medical Officer and Chief Scientific Officer; and Shai Lankry, Chief Financial Officer.
During this call, we may make Forward-Looking Statements about our future expectations and plans, including in respect to the timing of initiation and progress of, and data reported from the clinical trials of our product candidates, anticipated regulatory filings, including the submission of the BLA for Omidubicel to the FDA, commercialization planning efforts, the potentially life-saving or curative therapeutic and commercial potential of Gamida Cell’s product candidate, including GDA-201 and Omidubicel, and our expectations regarding our projected cash to be used for operating activities and cash runway.
Our actual results may differ materially from what we project today due to a number of important factors, including the impacts of COVID-19 pandemic on our operations, the scope, progress and expansion of our clinical trials and cost impact thereof, clinical, scientific, regulatory and technical developments and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics, and the endeavor of building a business around such product candidates, as well as those considerations described in the Risk Factors section of our most recent annual report on Form 10-K and other filings that we make with the SEC from time-to-time.
These forward-looking statements represent our views only as of today, and we caution you that we may not update them in the future, whether as a result of new information, fewer future events or otherwise.
Now I would like to turn the call over to Michelle.
Michele Korfin
Thank you, Heather, and thank you to everyone for joining us this morning. Gamida Cell had a productive quarter as we made important progress across all areas of our Company. We continue to build momentum as we head into several inflection points expected this year, most near-term being the full BLA submission for Omidubicel, which we are on track to complete in the second quarter of this year.
Additionally, our GDA-201 program advanced marked by the recent clearance by FDA of our IND and removal of the clinical hold for the cryopreserved formulation. We are excited to have reached this milestone and are now moving forward with our plans to initiate a company sponsored Phase I/II study in patients with follicular and diffuse large B-cell lymphomas, which Ronit will provide additional detail on.
Beyond GDA-201, which is our lead program in our expanding NAM enabled NK cell pipeline. We are also looking forward to announcing a new product candidate from our genetically engineered NAM enabled NK cell constructs later this year. We recently announced preclinical data at the ISCT conference supporting the development of GDA-301 and GDA-601.
It is an exciting time in Gamida Cell’s development and we are focused on advancing our pipeline of potentially curative cell therapies. Our continued commitment is demonstrated by the new and recent data on Omidubicel that we presented this quarter, focused on Omidubicel’s, encouraging clinical data and our health economic efforts, which we will comment on later on in the call.
With all we have accomplished only this past quarter, but also in the last few months, we are poised to be a leader in the development of NAM-enabled cell therapies with two promising and important clinical programs and a robust pipeline of genetically modified NK cell product candidates in preclinical development.
The progress we achieved this quarter continues to add to the important foundation that we have established. This strong foundation is due to our dedicated employees and their continued focus on patients and our vision of advancing therapies to help to address unmet needs.
With this, I will now turn the call over to Ronit.
Ronit Simantov
Thanks, Michelle. And good morning everyone. Thank you for joining us on the call this morning. Let me start with our lead program Omidubicel, which has breakthrough therapy designation as well as orphan drug status and to potential to be the first FDA-approved cell therapy for stem cell transplant.
Earlier this year, we were excited to initiate the rolling BLA submission for Omidubicel starting with our non-clinical module, followed by the submission of our clinical module. I’m pleased that we are currently on track to complete the full BLA submission in the second quarter of this year, moving us closer to bringing this important potential therapy to patients. Our BLA for Omidubicel is supported by strong Phase III results, which met all primary and secondary endpoints.
This past quarter, we announced the presentation of new data at the Transplantation & Cellular Therapy or TCT meeting. As we have continued to add to the body of evidence demonstrating the potential of Omidubicel to address unmet needs in patients undergoing allogeneic stem cell transplantation. Patients often have serious infections after transplant due to the slow recovery of their immune system, following treatment.
In a presentation that received a TCT best abstract award, Dr. Paul Szabolcs presented data from our Phase III randomized trial of Omidubicel, showing rapid recovery of abroad repertoire of immune cells, which was associated with reduced infection rates in Omidubicel treated patients.
In another oral presentation at TCT, Dr. [indiscernible] presented the final results of our Phase III study, including patient follow-up of over one year after transplant. The results showed that the early engrossment and lower infection rates previously observed in patients treated with Omidubicel were accompanied by longer-term clinical benefits, including a reduction in transplant related mortality and a trend towards an increase in overall survival, with no increase in relapsed or graft versus host disease, compared to transplantation with standard umbilical cord blood.
In addition, among our six posters at TCT, we presented 10-year follow-up data of patients treated in the Omidubicel clinical development program, demonstrating long-term sustainable hematopoiesis and immune confidence, with one case of secondary graft failure and one case of severe chronic GVHD among the 22 patients in the cohort.
We also presented an analysis of resource utilization in the Phase III study, demonstrating that faster hematopoietic recovery and lower rates of infections in patients transplant with Omidubicel were associated with significantly shorter length of hospital stay, reduced admissions to intensive care unit settings and lower healthcare resource utilization, compared to control.
Another poster outlined a quantitative analysis of well-established measures of patient health related quality of life, in a randomized Phase III study. The analysis demonstrated that Omidubicel was associated with meaningfully greater preservation or improvement of quality of life.
Tying together clinical data in the Phase III study to important outcomes from a patient focused perspective. Overall, we have continued to develop a robust set of clinical scientific translational and quality of life data demonstrating the potential clinical benefit of transplantation with Omidubicel.
Now turning to our NK pipeline, I will start with an update on GDA-201, we are proceeding with our plans to initiate a study of GDA-201 in patients with non-Hodgkin lymphoma. Although there have been recent advances for patients with lymphoma, we recognize that there is still an unmet need for patients with relapse and refractory disease.
Given the responses observed in the investigator led study at the University of Minnesota using the fresh formulation of GDA-201. We have developed the cryopreserved formulation, which allows us to perform a multicenter study.
We are very pleased to receive FDA clearance of our IMD for GDA-201, removing the clinical hold and allowing us to move forward with the study. We are proceeding with the operational activities at several sites, and we anticipate conducting formal site initiation visits and opening sites for enrollment this quarter.
Turning to our research and development activities, we are continuing to advance the preclinical data for our gene edited NK cell pipeline aimed at hematologic malignancies and solid tumors. We recently presented two posters at the International Society for Cell Gene Therapy or IFCP meeting in San Francisco. One poster presented new preclinical data on GDA-301, our CISH knockout membrane bound IL-15 expressing NK cell. Our poster showed cytotoxicity in potency of GDA-301 in multiple tumor cell line.
The second poster detailed preclinical data demonstrating cytotoxicity of GDA-601, our CD-38 knockout anti CD-38 CAR expressing NAM NK cells against multiple myeloma cell lines. We look forward to presenting more data at scientific meetings this year.
We plan to continue to conduct preclinical proof of concept studies for these genetically modified NK therapeutic candidates and by the end of 2022, we plan to select a pipeline candidate for IND enabling studies.
With that, I will turn the call over to Michele to now talk more about Omidubicel and commercial preparation. Michele.
Michele Korfin
Thank you, Ronit. I will now comment on our advancements with our CMC module and our launch readiness for Omidubicel. Earlier this year, we initiated our rolling BLA submission for Omidubicel, and we are nearing completion of the rolling BLA, which is on track for completion in the second quarter of this year.
In the first quarter of this year, we reached an important milestone with the FDA’s acknowledgement of the analytical comparability from our planned Gamida Cell owned commercial facility in Israel, as compared to the manufacturing facility used for the Phase III study. This allowed us to move forward with completion of the remaining production modules in our facility in Israel to support the BLA.
Our cross functional team in Israel, including operations quality R&D and regulatory are progressing well on the CMC module in preparation for completing that module and the BLA submission in the second quarter. We are not only preparing for the BLA submission from an operations perspective, but also preparing for launch readiness.
Our facility in Israel is modular and upon FDA approval of Omidubicel, we will have the ability to add additional cores as demand grows from Omidubicel. We are confident we could support the launch demand requirements from our facility, not only from a production, but also a supply chain standpoint.
The team is diligently working to ensure our manufacturing reliability, chain of identity and chain of custody for our commercial process to ensure a positive transplant center and positive patient experience.
Transitioning to launch readiness, the Gamida team recognizes the unmet need that Omidubicel may address for patients upon FDA approval. We are focused on our launch readiness to ensure upon FDA approval that patients could have access to Omidubicel. For patients with hematologic malignancies that are deemed eligible for an allergenic stem cell transplant, the procedure may be their best chance for a potential cure.
There are two key opportunities that Omidubicel may address for patients upon FDA approval. The first being potentially improving outcomes as compared to other donor sources based on transplant or feedback and the second potentially increasing access
In the United States are approximately 8,000 patients above the age of 12 with hematologic malignancies who undergo an allogeneic stem cell transplant each year. For potentially improving outcomes, we have conducted extensive market research over the last two years, and the insights are consistent and clear.
Transplanters see important opportunities for Omidubicel to potentially improve outcomes based on their experience with other donor sources. This is due to the strength of our clinical data, the ability to provide patients with pre-defined number of cells and the ability to provide Omidubicel within approximately one month of patient identification.
The majority of the patients in the United States are still receiving their graft from an unrelated donor and that could take on average, at least two to three months to align the patient and the donor. This timing for unrelated donors unfortunately puts the patient at risk for relapse.
Unfortunately, there are approximately 1,200 patients each year who are ages 12 and up with hematologic malignancies, who are deemed eligible for allogeneic stem cell transplant but cannot find an appropriate donor.
In terms of potentially increasing access for patients, unfortunately, there is racial disparity in the U.S. in regards to access to allogeneic stem cell transplants. If you are non-Caucasian and do not have access to a family member donor, you have a very low likelihood of finding a match in the public database. For example, patients who are African American may have less than a 20% chance of finding a match.
Omidubicel has a less stringent matching criteria, and we demonstrated our ability to match racially and ethnically diverse patients in our Phase III study, approximately 40% of the patients in our study were non-Caucasian.
And a study recently highlighted in a Poster Presentation at TCT, we leveraged available registry data and population modeling to project the potential impact of Omidubicel on racial and ethnic disparities.
In the model population increases in Omidubicel use in eligible patients were associated with potentially higher proportions of patients undergoing transplant and overall potentially improved outcomes with improvements being greater among racial minorities.
Taken together we anticipate that these two opportunities combined pending FDA approval, both improving outcomes based on transplant or feedback and increasing access may result in Omidubicel capturing approximately 20% to 25% of the addressable market once we reach peak market share. So upon FDA approval, this would equate to approximately 2,000 to 2,500 patients treated each year in the U.S. alone with Omidubicel.
In regards to reaching transplant centers in the U.S., the transplant centers that perform allogeneic stem cell transplants are extremely concentrated. For reference in the U.S., there are approximately 200 transplant centers that perform allogeneic stem cell transplants, 70 of those centers conduct approximately 80% of the transplants, allowing for an optimized approach to commercialization by initially targeting those centers.
Another important aspect of our launch is engaging with payers to ensure that they understand the potential value of Omidubicel upon FDA approval. Payer conversations are underway with national and key regional payers and we continue to hear consistent encouraging feedback on the overall value proposition of Omidubicel, including the strength of the clinical data and the health economic data we have published to-date.
We are also encouraged by their feedback on both the coverage and reimbursement approach they anticipate taking with Omidubicel upon FDA approval. We are excited to continue to hear positive feedback across all stakeholders and are extremely encouraged and driven by the potential of Omidubicel. We look forward to continuing to provide updates on our commercial preparations.
I will now turn the call over to Shai to review our financial results. Shai.
Shai Lankry
Thank you, Michele. And good morning, everyone. Today, I will summarize our financial results for the first quarter of 2022. As of March 31, 2022, our total cash position was approximately $70 million compared to $96 million as of December 31, 2021.
Research and development expenses for the quarter were $11.3 million, compared to $11.4 million in the first quarter of last year. The decrease was primarily due to a $1.1 million decrease in Omidubicel cell and GDA-201 clinical studies activities, offset by an increase of $1 million in boarding our scientific capabilities in talent.
Commercial expenses for the quarter were $3.9 million, compared to $4.2 million in the first quarter of 2021. The decrease was mainly due to reducing our near-term commercial readiness expenses, as we are assessing alternatives for the commercialization of Omidubicel, including potential U.S. or global partnership.
General and administrative expenses were $4.1 million in the first quarter of 2022, compared to $3.5 million for the same period in 2021. The increase was mainly due to a $0.5 million increase in headcount and related expenses.
Finance expenses net were $0.9 million for the first quarter of 2022, compared to $0.1 million for the same period last year. The increase was primarily due to a $0.6 million increase in interest expenses from our convertible node.
Net loss for the first quarter of 2022 was $20.2 million, compared to a net loss of $19.2 million in the first quarter of 2021. We expect cash used for ongoing operating activities for the entirety of this year to range from $65 million to $70 million.
We anticipate our current total cash position will support our ongoing operating activities into mid 2023. This cash run way guidance is based on our current operational plans and exclude any additional funding that may be received or business development activities that may be undertaken.
With that, I will turn the call back over to Michele.
Michele Korfin
Thank you, Shai. As we look ahead to the rest of the year, we are uniquely poised to deliver on our mission of developing potentially curative cell therapies for patients with blood cancers and other serious diseases.
We look forward to our full BLA submission for Omidubicel expected in the second quarter of this year and the initiation of our Phase I/II multicenter Gamida Cell sponsored study for the cryopreserve formulation of GDA-201 in patients with follicular and diffuse large B cell lymphomas this year.
We are excited for the opportunity to continue to leverage our unique NAM-enabled platform across a broad range of cell therapy candidates, and we look forward to providing updates throughout the year.
Now we will open the call for questions, Carmen.
Question-And-Answer Session
Operator
Thank you. [Operator Instructions] Your first question comes from John Miller with Evercore. Please go ahead.
Eric Musonza
Hi, this is Eric Musonza calling in for Jonathan Miller. Just had two quick questions on GDA-20,1 with trial sites opening in second quarter, when can we expect the first patient in and what sort of dynamics do you see around enrollment like do you expect COVID or summer travel to impact that? And then I have one follow-up.
Heather DiVecchia
Excellent. Thank you, Eric. Thanks for joining the call. I will turn to Ronit to address both of those. Thank you.
Ronit Simantov
Thanks Eric. So, in terms of opening sites, so sites will open this quarter and patients will need to be recruited and screened appropriately before the first patient is treated. So it usually takes several weeks for that to happen until a patient is actually treated with GDA-201.
But you know there is a lot of interest among the investigators for this study. There is an unmet need. These are patients who are relapsed or refractory, and clearly need additional therapies and are engaged in enrolling in a clinical trial. And so with the high need for these patients and the fact that they are ill with very serious cancer. We believe that enrollment will be robust for these patients.
I will make one more point about this portion of the study. It is a Phase I portion of the study. And so as this is a Phase I where toxicities are being evaluated patient-by-patient, we will be evaluating toxicity in each patient before proceeding with enrollment to the next patient. So we can carefully gauge patient enrollment as we move forward in the next few months and evaluate dose limiting toxicities. Once we evaluate that, then we can enroll patients more concurrently.
Eric Musonza
Got it. Very helpful. And just one more question. Do you expect any differences in enrollment timeline between follicular and the DLBCL cohorts?
Ronit Simantov
At this point, we don’t anticipate a difference between those two cohorts, but that is something that we can gauge as we move along. We certainly have the ability to analyze them separately, if need be, if there is a discrepancy we can manage that within the confines of the clinical trial design. But at this point, we don’t anticipate that there will be a difference because our investigators have expressed that patients with both histologies are in need of new therapies.
Eric Musonza
Great. Thank you.
Heather DiVecchia
Thank you Eric.
Operator
Your next question comes from Ted Tenthoff with Piper Sandler. Please go ahead.
Edward Tenthoff
Great, thank you and good morning everyone. I’m curious, what is sort of going on or what the latest is with respect to commercial prep for Omidubicel. I know, you are considering potential strategic alternatives, but also really just trying to get a sense for as you are finishing up the BLA, what might be the most likely scenario for marketing.
Michele Korfin
Excellent. Good, good morning, Ted. And thank you for joining us. I will go ahead and, and take that question. So in regards to commercial preparation, we have accomplished some critical milestones to-date.
So first off was really honing the commercial insights to understand first off the overall opportunity, but second of all, what could Omidubicel potential be for that opportunity. So, we have over the last couple of years, and most recently we have reiterated or relooked at some of these market insights.
We recognize the two key opportunities from Omidubicel. One is the ability to improve outcomes as compared to other donor sources, and this is based on transplant or feedback. And then the second is increasing access.
In terms of improving outcomes, some of the key insights that transplantor share with us is that the strength of the clinical data from our Phase III study, both the efficacy and the safety that Ronit has presented also the ability of having pre-defined number of cells.
And then finally the turnaround time in the United States, the majority of patients currently are still getting their donor source from an unrelated donor. And on average, we are hearing that takes at least two to three months to align the donor in the patient.
And as you know, with acute leukemia as an aggressive lymphomas that puts the patient at risk for relapse. So with Omidubicel being approximately one month from time of patient identification in the Phase III study to return of Omidubicel that is a positive feature.
And then switching to the increasing access or improving access, unfortunately in the United States, we do see many patients who are deemed eligible for transplant that cannot find the donor. The latest data that we have assessed is approximately 1,200 patients each year and that may end up growing. And unfortunately, it is also a 1situation of racial disparity. If you are non-Caucasian in the U.S. and don’t have access to a family member, it is incredibly difficult to find a match.
So to summarize, based on the encouragement of those market insights, in regards to the opportunity for Omidubicel, we have hired our commercial leadership team and we also have the operations and supply chain leadership team in place. So this way that that core group of leaders could continue to assess not only the opportunity, but most importantly the launch readiness.
And that is moving in parallel to our assessment of potential strategic alternatives. We feel very, very strongly that upon FDA approval, we do need to make sure patients have access to Omidubicel so we have got our leadership team in place as we are also in parallel looking at potential strategic alternatives.
Edward Tenthoff
Okay and would those alternatives include both the United States and overseas because this feels to me like a market that is appropriately sized for a biotech company to launch, especially since you guys have so much familiarity with the product, with the treatment group. So I just want to get a little more color on that. Thanks.
Michele Korfin
Yes. Thank you Ted. So we are assessing strategic opportunities globally, so including the U.S., but also ex-U.S., we have conducted initial commercial assessments in Europe and in Asia specifically in Japan and are very encouraged by the commercial opportunity from a Omidubicel in those areas. So, we are, as we discuss strategic alternatives for potential launch of Omidubicel looking globally.
Edward Tenthoff
Great. Thank you very much.
Heather DiVecchia
Thank you Ted.
Operator
Your next question comes from Gil Blum with Needham and Company. Please go ahead.
Gilbert Blum
Good morning everyone and thanks for taking my question. Maybe a broader one, considering recent the result in donated NK cells in the space, I’m just curious how encouraged you are seeing that a company sponsored study could lead to some very interesting early results and how do you think that translates for GDA-201? Thank you.
Heather DiVecchia
Excellent. good morning Gil and thank you for joining us and for the question. Let me turn to Ronit to address Gil’s question.
Ronit Simantov
Sure. Thanks Gil. So, there have been some encouraging results that we have seen from other product, NK cells type product recently and overall, those are actually really encouraging because it sets the stage for NK cells. And in particular, some of the results we have seen are related to high doses delivered of NK cells with results seen with greater response seen at the higher doses.
And that actually sets us up quite well because we are able to deliver with GDA-201 high doses of NK cells, high numbers of NK cells that are extremely functional and retain their killing capacity, because the NAM technology that we have used to expand and maintain the stemness of stem cells actually expand and maintains the NK-ness or the queuing potential of NK cells. So, we actually think it sets us up quite nicely for our Phase I/II study.
Our study is designed to evaluate the safety of the cryopreserved formulation at first and then move right into a formal efficacy evaluation. The entire study is un-blinded - open label study, and we will be able to see as we go what the potential for activity is with this formulation. So, we are excited to start enrolling patients and seeing results.
Gilbert Blum
Thank you. Thank you Ronit that was very helpful. Maybe also on a quick financial question. So, on the SG&A spend, there was a line that mentioned that, there are some cost savings due to consideration of strategic options for commercialization. Just help me to understand, does that mean that the company is cutting back on commercial preparedness launch, or just help me to understand that? Thank you.
Michele Korfin
Thank you, Gill. So, I will start with sort of the strategic aspect and I will turn to Shai for any additions on the financial side. So, we were fortunate last year to hire some incredibly strong commercial leaders. Linda Stamler to lead marketing and account management, [indiscernible] to lead market access. Those two individuals and their teams were able to conduct a lot of the critical market insights and beginning of launch preparation last year.
So then that allowed us to use this past quarter more to evaluate the insights and less OpEx associated with having to gather additional insights and also less hiring. We were able to hire our core group of commercial and operation leaders last year. So, that is sort of the high-level strategic aspects of it. And let me turn to Shai, if there is anything to add from the financial side.
Shai Lankry
Hi Gil, good morning. Thank you for the question. The way we see it, Gamida Cell as a company, continue to diligently manage our cash position. And as such, we announced back in January, this year, that we are assessing some strategic alternative for launching Omidubicel as our vision is to bring Omidubicel to patient.
Yes, we did prioritized, the key activities on not only on the commercial side, but across all the business to make sure, we are not jeopardizing our launch activities. And as I mentioned, and Michele mentioned before making sure each patient will have access to Omidubicel upon the approval.
Gilbert Blum
Alright. Thank you both for taking our questions this morning.
A - Heather DiVecchia
Thank you Gil.
Operator
Your next question comes from Jason Butler with JMP Securities. Please go ahead.
Unidentified Analyst
Hi. So its [Ronan] (Ph) for Jason. Thanks for taking our questions. I want to follow-up on the partnering questions. I guess, can you give a little more detail maybe on the level of interest that you are seeing. Do you think you can have a partnership in pot in place before, a potential approval maybe around year end? And can you remind us what structure you prefer for the U.S. Maybe a co-promote? Thanks.
Michele Korfin
Thank you. Good morning. And thank you for joining us. I will go ahead and take both of the questions. So we won’t comment on specifics in regards to our assessment at this point in time. But I do want to reiterate something very important that that Shai had said earlier.
And that is the fact that although we are assessing strategic alternatives for launch, we also do have the leadership team in place on both the commercial, the medical affairs, the quality and the operations side to assure that patients have access to Omidubicel upon FDA approval.
So when you asked about sort of the latter part of the question in terms of timing, we do have the strategy in place, the plans in place to assure access to patients for Omidubicel upon FDA approval.
At this point in time, as we indicated earlier this year, we are assessing strategic alternatives, but also continuing to do our work internally around assessing the opportunity and assessing what would be needed for launch planning.
Unidentified Analyst
Okay, great. That is helpful. Thank you. Then for the four NK candidates beyond GDA-201, are you going to take only one into the IND enabling studies or potentially multiple candidates, and then what happens to the other candidates if you know that don’t go into IND enabling studies this year, do they go on hold? Are you going to continue to refine them pre clinically? Thanks.
Heather DiVecchia
Excellent. Thank you very much. I will turn to Ronit for to address that question.
Ronit Simantov
Thanks Jason. One at a time. So we will take the lead candidate by the end of this year and, do the appropriate IND enabling studies and continue to develop, based on data of course, the additional candidates and as move those forward as we are able.
Unidentified Analyst
Great. Thank you very much.
Heather DiVecchia
Thank you.
Operator
Your next question comes from Mark Breidenbach with Oppenheimer. Please go ahead.
Unidentified Analyst
Hi, this is [Jacqueline] (Ph) for Mark, and thanks for taking our questions. So can you please remind us what dose levels will be tested in the dose escalation from the GDA-201. And if there are any differences in lympho depletion conditions to cytokine support or rituximab dosing?
Heather DiVecchia
Thank you for joining us Jacqueline. I will turn to Ronit to address both the dosing question and the question around the lympho depletion and rituximab in the protocol.
Ronit Simantov
Absolutely. So the design of the study, the design of the administration or the treatment design is based on the Minnesota study using the fresh formulation. And so we will be testing doses that are similar to the ones used in the Minnesota study and the administration of lympho depleting chemotherapy, rituximab, and IL2 are also very similar if not identical to what was done in the Minnesota study. So I don’t think we have come out yet with the exact dose levels. But they are guided completely by the previous data in Minnesota.
Unidentified Analyst
And when do you expect to commence manufacturing runs for GDA-201 for the Phase I trial?
Heather DiVecchia
Yes, actually a last one Ronit, if you could also take that please.
Ronit Simantov
Of course, I’m happy to take that. So, manufacturing we are building - because, in the Phase I trial, the GDA-201 is produced ahead of time and not matched for patients, but basically readily available. We are building our inventory already and expect to have the product available for patients as soon as the first dosing is required.
Unidentified Analyst
Great. thanks for detailed questions.
Ronit Simantov
Thank you Jacqueline.
Heather DiVecchia
Thank you.
Operator
Your last question comes from Matthew Cross with Alliance Global Partners. Please go ahead.
Matthew Cross
Hi all good morning. Best wishes to Julian for a speedy recovery. I had two quick questions related to the Phase I/II for GDA-201, and kind of following up on your comments that are neat about the scheduling for lymphodepleting chemotherapy and antibody use. I guess considering this internal study will be using the cryopreserve formulation, not the fresh, just wanted to understand any the kind of special considerations here. I guess for the study compared to the prior one, it sounded like the schedule will be more or less the same or based off of the prior study. But given that you are not baking in time for name expansion with the cryopreserved, just wanted to understand whether that scheduling would still look similar and maybe to kind of recap any modifications that would have to be made to this study that were needed to remedy the FDA’s concerns around donor eligibility requirements. And anything that may play into which sites are able to participate in this study, given the combination of those donor eligibility requirement changes and the cryopreservation use here versus what was done previously? Thanks.
Heather DiVecchia
Excellent. Thank you Matt, thank you for joining us and thank you for the well wishes, we will pass them along, thank you. And I will turn to Ronit to answer Matt’s questions in regards to the Phase I/II.
Ronit Simantov
Sure. I’m happy to answer that, and I will take the donor eligibility piece first. So, the donor eligibility procedures and the assay qualifications, we needed to make sure that those are compliance with FDA requirements.
And so we replaced our donor testing laboratory with a Cleo certified lab in the U.S. and we have now made sure that all appropriate tests are licensed and cleared and that eligibility requirement has been met.
So there has been no change in the design of the study based on the quote - the hold itself was not require any changes in the design of the study, the protocol, the sites, or anything that had to do with the clinical trial itself. It really was confined to the eligibility of the product. So that was that piece.
Now in terms of cryopreserve formulation, so that it is great that you that you picked up on that. So in the fresh formulation study, patients had to have a donor available that donor had to be recruited and if reasons had to be done and then the product had to be produced over time.
So because we have a readily available product, now it will be at the site when the patient is enrolled and so that may save some time in terms of getting the patients up and treated because they will have product there, they don’t have to wait for the donor to get already get the donation and do all that.
And then procedurally, there will be a following process, the product will be there, it will be frozen, and it will be thought at the bedside to provide the patient with the product when they need it.
Matthew Cross
Perfect. Okay super helpful. And I just had one quick follow-up, which was around the sizing. It looked like per what you have drawn up on clinicaltrials.gov that there is kind of a target enrollment of about a hundred patients. So I guess I know you are not commenting at this point on the actual dose levels that, that you may step through, but just wanted to confirm whether that total sizing was based around kind of an expectation internally around the number of Phase I cohorts that you may look at, if it will be ultimately still driven by, the standard kind of safety evaluations as you step through doses, or maybe just kind of an expectation for the Phase II population for that portion of the study. Just wanted to get a little bit of an understanding around the assumptions for that sizing. Thanks.
Ronit Simantov
Yes. Absolutely. So, that number, which is put in clinicaltrials.gov and which is incorporated into the protocol itself incorporates, there is some flexibility there because you don’t know for sure how many patients you are going to end up enrolling in Phase I.
It is a standard 3x3 design where, if there is a dose limiting toxicity observed in the dose level numbers are expanded to include more patients. And so this is the maximum size based on the possible expansion of cohorts in the Phase I portion.
And then it also includes the Phase II portion where we are going to be evaluating separately, the patients with lymphoma, from the patients with the aggressive lymphomas and each of those sort of Phase II portions has its own, steps and analysis surrounding it. But they are relatively small cohorts to allow us to evaluate rather quickly efficacy results and then make decisions based on those early efficacy results that we see.
Matthew Cross
Understood. Okay. Thanks again for the clarity. I appreciate it.
Heather DiVecchia
Thank you Matt.
Ronit Simantov
Thanks so much Matthew.
Operator
Thank you. And this concludes our Q&A session. I will turn the call back to Michele Korfin for her final remarks.
Michele Korfin
Thank you, Carmen. Thank you very much for joining us for the call today. And we look forward to keeping you all updated on our future milestones. Have a nice day. Thank you, Carmen. That will conclude our call.
Operator
Thank you, ladies and gentlemen for participating. And you may now disconnect.
Gamida Cell GAAP EPS of -$0.34 in-line
May 10, 2022 7:13 AM ETGamida Cell Ltd. (GMDA)
By: Niloofer Shaikh, SA News Editor
Gamida Cell press release (NASDAQ:GMDA): Q1 GAAP EPS of -$0.34 in-line.
The company expects that its current cash and cash equivalents of $70M will support the company’s ongoing operating activities into mid 2023.
This cash runaway guidance is based on the company’s current operational plans and excludes any additional funding and any business development activities that may be undertaken.
Gamida Cell Reports First Quarter 2022 Financial Results and Provides Company Update
https://finance.yahoo.com/news/gamida-cell-reports-first-quarter-110000541.html
- On track for full BLA submission of omidubicel in the second quarter of 2022 -
- Planning to open sites for enrollment in second quarter of 2022 for Phase 1/2 study of GDA-201 in patients with follicular and diffuse large B-cell lymphomas -
- Presented new preclinical data supporting potential of NAM-enabled, genetically modified NK pipeline cell therapy candidates GDA-301 and GDA-601 -
- Finished first quarter of 2022 with approximately $70 million in cash; sufficient cash to fund the company’s operations into mid-2023 -
- Company to host conference call at 8:00 a.m. ET today -
BOSTON, May 10, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with hematologic and solid cancers and other serious diseases, today provided a business update and reported financial results for the quarter ended March 31, 2022. Net loss for the first quarter of 2022 was $20.2 million, compared to a net loss of $19.2 million in the first quarter of 2021. As of March 31, 2022, Gamida Cell had total cash and cash equivalents of $69.7 million.
During the past quarter, Gamida Cell:
Progressed omidubicel, a potentially life-saving cell therapy candidate for patients with blood cancers in need of stem cell transplant, towards full Biologics License Application (BLA) submission to the U.S. Food and Drug Administration (FDA) in the second quarter of this year.
Planning to open sites for enrollment in second quarter of 2022 for Phase 1/2 study of lead natural killer (NK) cell therapy candidate, GDA-201, for patients with follicular and diffuse large B-cell lymphomas. The FDA recently cleared the company’s Investigational New Drug (IND) application and removed the clinical hold on the program, allowing Gamida Cell to move forward with the planned Phase 1/2 study with the cryopreserved formulation.
Continued development of the company’s NAM-enabled NK cell pipeline, including genetically modified product candidates GDA-301, GDA-401, GDA-501 and GDA-601, which focus on solid-tumor and hematological cancers. These product candidates utilize CAR, membrane bound- and CRISPR-mediated technologies to increase the NK cell targeting, potency and persistence against hematologic malignancies and solid tumors.
"We have made significant strides advancing our pipeline and demonstrating the potential benefit of our NAM-enabled cell therapy candidates for patients with blood cancers and other serious blood," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "And this starts with the recent milestone of the clearance of our IND for the cryopreserved formulation of GDA-201 by removal of the clinical hold. We are proceeding with operational activities in second quarter at multiple sites for our planned Phase 1/2 study, and are on track to advance this novel cell therapy candidate to the clinic this year. We were also pleased to present important data on omidubicel, supporting its potential long term clinical benefit, as we approach the full BLA submission for omidubicel during the second quarter of this year."
First Quarter and Recent Developments
Omidubicel: Advanced Cell Therapy
BLA submission: Following the receipt of positive Type B meeting correspondence from the FDA confirming that analytical comparability has been established between product made at Gamida Cell’s wholly-owned commercial manufacturing facility and the product that was manufactured for the Phase 3 study, Gamida Cell initiated a rolling BLA submission for omidubicel in February 2022. The company is on-track to complete the BLA submission in the second quarter of 2022. In parallel with the BLA submission, Gamida Cell is assessing alternatives for the commercialization of omidubicel, including potential U.S. or global partnerships.
New data presented at the 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings (TCT): In April 2022, Gamida Cell announced two oral and six poster presentations that focused on omidubicel’s positive Phase 3 clinical data, Gamida Cell’s health economic efforts, and transcriptional and metabolic profiling for our lead NK candidate, GDA-201. These presentations continued to add to the totality of evidence supporting omidubicel as a potential allogeneic hematopoietic stem cell transplant and our developments with our NK candidates. Highlights from these presentations included:
A presentation which received TCT’s Best Abstract Award entitled "Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel is Associated with Enhanced Circulatory Plasmacytoid Dendritic Cells (pDC), NK Cells and CD4+ T Cells with Lower Rates of Severe Infections Compared to Standard Umbilical Cord Blood Transplantation." This presentation detailed that Allo-HSCT with omidubicel demonstrated rapid hematopoietic recovery, reduced rates of infections and no increase in acute or chronic GvHD rates compared with standard UCB, with no unexpected adverse events attributable to ex vivo expansion.
An oral presentation titled "Allogeneic Hematopoietic Stem Cell (allo-HSCT) Transplant with Omidubicel Demonstrates Sustained Clinical Improvement Versus Standard Myeloablative Umbilical Cord Blood Transplantation (UCBT): Final Results of a Phase III Randomized, Multicenter Study." The data showed that the advantages of early engraftment and lower infections with omidubicel translated into long term clinical benefits in the first-year post-transplant, as demonstrated by reduction in non-relapse mortality, and no increase in relapse or GvHD rate compared to Umbilical Cord Blood Transplantation (UCBT). Additionally, there was a continued trend toward improved OS in favor of the omidubicel arm over time (73% vs 60%).
A health economic study titled "Projected Impact of Omidubicel on Racial and Ethnic Disparities in Allogeneic Hematopoietic Cell Transplant (allo-HCT) Access and Outcomes for Patients with Hematologic Malignancies in the US." The study, which assessed the projected impact of omidubicel on racial and ethnic health disparities in a projection model, showed that, if approved, broad access to omidubicel was projected to decrease time to allo-HCT and improve allo-HCT outcomes overall, with the greatest improvements among racial and ethnic groups least served by current graft sources.
GDA-201: NAM-Enabled NK Cell Therapy
IND cleared and clinical hold removed for Phase 1/2 Study with cryopreserved formulation of GDA-201: Gamida Cell recently announced that FDA cleared its IND application and removed the clinical hold for a cryopreserved formulation of GDA-201. The company is now proceeding with operational activities at several clinical trial sites, and is on track to initiate a company-sponsored Phase 1/2 clinical study in patients with follicular and diffuse large B-cell lymphomas in 2022.
NAM-Enabled NK Cell Pipeline Expansion
Progressed NAM-enabled genetically modified NK pipeline: Gamida Cell continues to progress its NAM-enabled genetically modified NK pipeline, which utilizes CAR, membrane bound- and CRISPR-mediated technologies to increase targeting, potency and persistence against hematologic malignancies and solid tumors. The company plans to conduct preclinical proof of concept studies for these genetically modified NK therapeutic targets and to select a product candidate for IND enabling studies by the end of 2022. These therapeutic targets include:
GDA-301: Knockout of CISH (cytokine inducible SH2 containing protein) in NK cells using CRISPR/Cas9 in combination with a membrane-bound IL-15/IL-15Ra;
GDA-401: A development candidate with an undisclosed target.
GDA-501: Anti HER2 CAR-engineered NK cells to target solid tumors expressing HER2, based on a single-chain variable fragment of the widely used humanized monoclonal antibody trastuzumab; and
GDA-601: CRISPR Knockout of CD38 on NK cells combined with anti CD38 CAR. CD38 is an established immunotherapeutic target in multiple myeloma, but its expression on NK cells and its further induction during ex vivo NK cell expansion represents a barrier to the development of an anti CD38 CAR-NK cell therapy. Gamida Cell is advancing this program in collaboration with the Dana-Farber Cancer Institute to study the in vitro cytotoxicity of GDA-601 in fresh samples from multiple myeloma patients.
Presented preclinical data from product candidates at the International Society for Cell & Gene Therapy (ISCT) 2022: Gamida Cell recently presented new preclinical data for GDA-301 and GDA-601 that continued to demonstrate the potential of these product candidates:
A poster titled "GDA-301: Engineered NAM-NK Cells via CISH Knockout and Membrane-Bound IL-15 Expression Increases Cytotoxicity Against Malignancies," detailed that GDA-301 produces enhanced potency and persistence with combined genetic manipulation of CISH gene editing and the engineered expression of membrane-bound IL-15 for targeting hematologic malignancies and solid tumors.
In a poster titled "GDA-601: NAM-NK Cells With CD38 Knockout Expresses Enhanced CD38 Chimeric Antigen Receptor and Targets Multiple Myeloma Cells With Increased Cytotoxicity," it was shown that GDA-601 displays superior antitumoral responses against multiple myeloma cells and represents a promising adoptive cell therapeutic strategy.
First Quarter 2022 Financial Results
Research and development expenses were $11.3 million in the first quarter of 2022, compared to $11.4 million in the same quarter in 2021. The decrease was primarily due to a $1.1 million decrease in omidubicel and GDA 201 clinical study activities, offset by an increase of $1.0 million in broadening the company’s scientific capabilities and talent.
Commercial expenses were $3.9 million in the first quarter of 2022, compared to $4.2 million in the first quarter of 2021. The decrease was attributable mainly to reducing the company’s near-term commercial readiness expenses, as it is assessing alternatives for the commercialization of omidubicel, including potential U.S. or global partnerships.
General and administrative expenses were $4.1 million in the first quarter of 2022, compared to $3.5 million in the same period in 2021. The increase was mainly due to a $0.5 million increase in headcount and related expenses.
Finance expenses, net, were $0.9 million in the first quarter of 2022, compared to $0.1 million in the same period in 2021. The increase was primarily due to a $0.6 million increase in interest expenses from convertible notes.
Net loss was $20.2 million in the first quarter of 2022, compared to a net loss of $19.2 million in the first quarter of 2021.
2022 Financial Guidance
Gamida Cell expects that its current cash and cash equivalents will support the company’s ongoing operating activities into mid 2023. This cash runaway guidance is based on the company’s current operational plans and excludes any additional funding and any business development activities that may be undertaken.
Expected Milestones in 2022
Omidubicel
Completion of full BLA submission to the FDA in the second quarter of 2022
GDA-201
Initiation of a company-sponsored Phase 1/2 clinical study with the cryopreserved formulation in follicular and diffuse large B-cell lymphomas
NK cell pipeline expansion
Conduct preclinical proof of concept studies of the NAM-enabled, genetically modified NK therapeutic targets
Select pipeline candidate for IND-enabling studies
Conference Call Information
Gamida Cell will host a conference call today, May 10, 2022, at 8:00 a.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com. To participate in the live call, please dial 866-930-5560 (domestic) or 409-216-0605 (international) and refer to conference ID number 3344029. A replay of the webcast will be available approximately two hours after the event, for approximately 30 days.
About Omidubicel
Omidubicel is an advanced cell therapy candidate under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the U.S. FDA and has also received Orphan Drug Designation in the U.S. and EU. Gamida Cell has completed an international, multi-center, randomized Phase 3 study (NCT0273029) evaluating the safety and efficacy of omidubicel in patients with hematologic malignancies undergoing allogeneic bone marrow transplant compared to a comparator group of patients who received a standard umbilical cord blood transplant. That study achieved its primary endpoint, demonstrating a highly statistically significant reduction in time to neutrophil engraftment, a key milestone in a patient’s recovery from a stem cell transplant. The Phase 3 study also achieved its secondary endpoints of reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. Gamida Cell initiated a rolling BLA submission for omidubicel in the first quarter of 2022 with full BLA submission on track for the second quarter of 2022. In 2019, approximately 8,000 patients who were 12 years old and up with hematologic malignancies underwent an allogeneic stem cell transplant. Unfortunately it is estimated that another 1,200 patients were eligible for transplant but could not find a donor source. Omidubicel has the opportunity, upon FDA approval to improve outcomes for patients based on transplanter feedback and increase access for patients to get to transplant. Omidubicel has the potential to treat approximately 2000 – 2500 patients each year in the U.S. For more information about omidubicel, please visit https://www.gamida-cell.com.
Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
About GDA-201
Gamida Cell applied the capabilities of its nicotinamide (NAM)-enabled cell expansion technology to develop GDA-201, an innate NK cell immunotherapy candidate for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical trial results. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, GDA-201 improves antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells. There are approximately 40,000 patients with relapsed/refractory lymphoma in the E.U.5 and U.S. which is the patient population that will be studied in the GDA-201 Phase 1/2 clinical trial.
For more information about GDA-201, please visit https://www.gamida-cell.com. For more information on the Phase 1/2 clinical trial of GDA-201, please visit www.clinicaltrials.gov.
GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
About NAM Technology
Our NAM-enabling technology, supported by positive Phase 3 data, is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (Nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.
About Gamida Cell
Gamida Cell is pioneering a diverse immunotherapy pipeline of potentially curative cell therapies for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapies with potential to redefine standards of care. These include omidubicel, an investigational product with potential as a life-saving alternative for patients in need of bone marrow transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.
Cautionary Note Regarding Forward Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of initiation and progress of, and data reported from, the clinical trials of Gamida Cell’s product candidates (including GDA-201), anticipated regulatory filings (including the timing of submission of the BLA for omidubicel to the FDA), commercialization planning efforts, and the potentially life-saving or curative therapeutic and commercial potential of Gamida Cell’s product candidates (including GDA-201 and omidubicel), and Gamida Cell’s expectations for the expected clinical development milestones set forth herein. Any statement describing Gamida Cell’s goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions, including those related to the impact that the COVID-19 pandemic could have on our business, and including the scope, progress and expansion of Gamida Cell’s clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such product candidates. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cell’s Annual Report on Form 10-K, filed with the Securities and Exchange Commission (SEC) on March 24, 2022, as amended, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cell’s actual results could differ materially and adversely from those anticipated or implied thereby. Although Gamida Cell’s forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Gamida Cell. As a result, you are cautioned not to rely on these forward-looking statements.
CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited)
U.S. dollars in thousands (except share and per share data)
March 31,
December 31,
2022
2021
ASSETS
CURRENT ASSETS:
Cash and cash equivalents
$
42,057
$
55,892
Marketable securities
27,661
40,034
Prepaid expenses and other current assets
2,994
2,688
Total current assets
72,712
98,614
NON-CURRENT ASSETS:
Restricted deposits
3,893
3,961
Property, plant and equipment, net
37,533
35,180
Operating lease right-of-use assets
6,722
7,236
Severance pay fund
2,046
2,148
Other long-term assets
1,632
1,647
Total non-current assets
51,826
50,172
Total assets
$
124,538
$
148,786
CONDENSED CONSOLIDATED BALANCE SHEETS (Unaudited)
U.S. dollars in thousands (except share and per share data)
March 31,
December 31,
2022
2021
LIABILITIES AND SHARHOLDERS' EQUITY
CURRENT LIABILITIES:
Trade payables
$
4,346
$
8,272
Employees and payroll accruals
4,119
4,957
Operating lease liabilities
2,596
2,699
Accrued interest of convertible senior notes
551
1,640
Accrued expenses and other current liabilities
8,866
7,865
Total current liabilities
20,478
25,433
NON-CURRENT LIABILITIES:
Convertible senior notes, net
71,607
71,417
Accrued severance pay
2,327
2,396
Long-term operating lease liabilities
5,142
5,603
Total non-current liabilities
79,076
79,416
CONTINGENT LIABILITIES AND COMMITMENTS
SHAREHOLDERS' EQUITY:
Share capital -
169
169
*
-
Additional paid-in capital
382,495
381,225
Accumulated deficit
(357,680)
(337,457)
Total shareholders' equity
24,984
43,937
Total liabilities and shareholders' equity
$
124,538
$
148,786
* Represents an amount lower than $1.
CONDENSED CONSOLIDATED STATEMENTS OF OPERATIONS (Unaudited)
U.S. dollars in thousands (except share and per share data)
Three months ended
March 31,
2022
2021
Research and development expenses, net
$
11,305
$
11,360
Commercial expenses
3,879
4,231
General and administrative expenses
4,139
3,513
Total operating loss
19,323
19,104
Financial expenses, net
900
82
Loss
20,223
19,186
Net loss per share attributable to ordinary shareholders, basic and diluted
$
0.34
$
0.32
Weighted average number of shares used in computing net loss per share
attributable to ordinary shareholders, basic and diluted
59,474,366
59,122,973
CONDENSED CONSOLIDATED STATEMENTS OF CASH FLOWS (Unaudited)
U.S. dollars in thousands (except share and per share data)
Three months ended
March 31,
2022
2021
Cash flows from operating activities:
Loss
$
(20,223)
$
(19,186)
Adjustments to reconcile loss to net cash used in operating activities:
Depreciation of property, plant and equipment
112
98
Financing expense (income), net
(1,172)
220
Share-based compensation
1,194
913
Amortization of issuance costs
191
323
Operating lease right-of-use assets
562
516
Operating lease liabilities
(613)
(929)
Accrued severance pay, net
33
-
Increase in prepaid expenses and other assets
(889)
(515)
Increase (decrease) in trade payables
(3,927)
907
Decrease in accrued expenses and current liabilities
(996)
(3,192)
Net cash used in operating activities
(25,728)
(20,845)
Cash flows from investing activities:
Purchase of property, plant and equipment
(723)
(2,806)
Purchase of marketable securities
(2,086)
-
Proceeds from maturity of marketable securities
14,126
-
Proceeds from restricted deposits
500
-
Net cash provided by (used in) investing activities
$
11,817
$
(2,806)
Cash flows from financing activities:
Proceeds from exercise of options
76
502
Proceeds from issuance of convertible senior notes, net
-
70,777
Net cash provided by financing activities
76
71,279
Increase (decrease) in cash and cash equivalents
(13,835)
47,628
Cash and cash equivalents at beginning of period
55,892
127,170
Cash and cash equivalents at end of period
$ 42,057
$ 174,798
View source version on businesswire.com: https://www.businesswire.com/news/home/20220510005438/en/
Contacts
For investors:
Courtney Turiano
Stern Investor Relations, Inc.
Courtney.Turiano@sternir.com
1-212-362-1200
For media:
Rhiannon Jeselonis
Ten Bridge Communications
rhiannon@tenbridgecommunications.com
1-978-417-1946
Gamida Cell Presents New Data from NAM-Enabled Genetically Modified Natural Killer (NK) Pipeline at International Society for Cell & Gene Therapy 2022
https://finance.yahoo.com/news/gamida-cell-presents-data-nam-110000072.html
Poster selected for inclusion in conference’s Elevator Pitch Session: GDA-301 produces enhanced potency and persistence with combined genetic manipulation of CISH gene editing and the engineered expression of membrane-bound IL-15 for targeting hematologic malignancies and solid tumors
GDA-601 generates promising immunotherapeutic potential to target multiple myeloma cells
Company plans to select a genetically modified NK cell therapy candidate for IND enabling study by the end of 2022
BOSTON, May 05, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with solid and hematological cancers and other serious diseases, today will share preclinical data at the International Society for Cell & Gene Therapy (ISCT) 2022, being held in San Francisco, CA, May 4-7, 2022 on GDA-301 and GDA-601, two product candidates in the Company’s NAM-enabled genetically modified natural killer (NK) pipeline.
"The preclinical data generated from our expanding pipeline of NAM-enabled cell therapies is already showing signs of meaningful potential as a future approach to fighting cancer," said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. "With evidence of enhanced cytotoxicity demonstrated across hematologic cancers and solid tumors with these diverse, genetically modified NK cell immunotherapy programs, we look forward to continuing our progress toward opening new frontiers in cancer immunotherapy."
GDA-301 is an investigational genetically modified NAM-NK cell therapy candidate aimed at targeting hematologic malignancies and solid tumors. The poster (#501), titled "GDA-301: Engineered NAM-NK Cells via CISH Knockout and Membrane-Bound IL-15 Expression Increases Cytotoxicity Against Malignancies," demonstrated that after six hours of co-culture with a chronic myelogenous leukemia (K562) or multiple myeloma (RPMI) cell line, GDA-301, a combined genetic manipulation of CISH gene editing and the engineered expression of mb IL-15, showed increased cytotoxicity compared with control NAM-NK cells. Additional in vitro assays showed elevation of degranulation marker CD107a, and intracellular proinflammatory cytokines interferon-? and tumor necrosis factor-a, suggesting increased potency of GDA-301 compared with control. The potency and cytotoxicity data suggest that GDA-301 represents a novel potential immunotherapeutic targeting hematologic malignancies as well as solid tumors.
The poster on GDA-301 was selected for presentation at the conference’s Elevator Pitch Session 2 on Thursday, May 5, 2022 at 6:00 p.m. EST/3:00 p.m. PST – 7:00 p.m. EST/4:00 p.m. PST.
GDA-301 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
GDA-601 is an investigational genetically engineered NAM-NK cell therapy candidate designed to target multiple myeloma (MM) cells. The poster (#517), titled "GDA-601: NAM-NK Cells With CD38 Knockout Expresses Enhanced CD38 Chimeric Antigen Receptor and Targets Multiple Myeloma Cells With Increased Cytotoxicity," showed that in vitro killing assays performed six hours after co-culture of GDA-601 with a MM (RPMI) cell line showed increased cytotoxicity compared with control NAM-NK cells. Fratricide attributable to CD38 antigen was effectively eliminated with GDA-601. There was a significant enhancement of potency against CD38-positive MM cells demonstrated by elevation of the degranulation marker CD107a and intracellular proinflammatory cytokines interferon-? and tumor necrosis factor-a in vitro. These results suggest that GDA-601 displays superior antitumoral responses against MM cells and represent a promising adoptive cell therapeutic strategy against MM.
Both posters will be presented on Thursday, May 5, 2022 at Poster Session 2, at 5:45 p.m. EST/2:45 p.m. PST – 7:15 p.m. EST/4:15 p.m. PST.
GDA-601 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
For more information, please visit isctglobal.org.
About NAM Technology
Our NAM-enabling technology, supported by positive Phase 3 data, is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (Nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.
About Gamida Cell
Gamida Cell is pioneering a diverse immunotherapy pipeline of potentially curative cell therapies for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapies with potential to redefine standards of care. These include omidubicel, an investigational product candidate with potential as a life-saving alternative for patients in need of bone marrow transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.
Cautionary Note Regarding Forward Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of initiation and progress of, and data reported from, the clinical trials of Gamida Cell’s product candidates (including GDA-201), anticipated regulatory filings and the potentially life-saving or curative therapeutic and commercial potential of omidubicel. Any statement describing Gamida Cell’s goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions, including those related to the impact that the COVID-19 pandemic could have on our business, and including the scope, progress and expansion of Gamida Cell’s clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such product candidates. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cell’s Annual Report on Form 10-K, filed with the Securities and Exchange Commission (SEC) on March 24, 2022, as amended, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cell’s actual results could differ materially and adversely from those anticipated or implied thereby. Although Gamida Cell’s forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Gamida Cell. As a result, you are cautioned not to rely on these forward-looking statements.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220505005255/en/
Contacts
For investors:
Courtney Turiano
Stern Investor Relations, Inc.
courtney.turiano@sternir.com
1-212-362-1200
For media:
Rhiannon Jeselonis
Ten Bridge Communications
rhiannon@tenbridgecommunications.com
1-978-417-1946
PR Newswire
Gamida Cell Announces the Date of Its First Quarter 2022 Financial Results and Webcast
https://finance.yahoo.com/news/gamida-cell-announces-date-first-120000108.html
BOSTON, May 03, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious blood diseases, today announced that the company will host a conference call and live audio webcast on Tuesday, May 10, 2022, at 8:00 a.m. ET to review its first quarter 2022 financial results and provide an update on the company.
The webcast will be available on the "Investors & Media" section of the Gamida Cell website at www.gamida-cell.com. To participate in the live call, please dial 866-930-5560 (domestic) or 409-216-0605 (international) and refer to conference ID number 3344029. A replay of the webcast will be available approximately two hours after the event, for approximately 30 days.
About Gamida Cell
Gamida Cell is pioneering a diverse immunotherapy pipeline of potentially curative cell therapies for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary expansion platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapies with potential to redefine standards of care. These include omidubicel, an investigational product candidate with potential as a life-saving alternative for patients in need of bone marrow transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.
Gamida Cell stock falls a day after soaring on FDA lifting clinical hold on GDA-201
Apr. 27, 2022 12:07 PM ETGamida Cell Ltd. (GMDA)
By: Anuron Mitra, SA News Editor
Shares of Gamida Cell (NASDAQ:GMDA) have fallen 8% to $2.48 in morning trade on Wednesday, a day after gaining as much as 16%.
GMDA on April 26 said the U.S. Food and Drug Administration (FDA) had cleared its new drug application and lifted the clinical hold for a cryopreserved formulation of GDA-201, the company's cell therapy candidate for the treatment of patients with follicular and diffuse large B cell lymphomas.
GMDA stock had soared 32% in Tuesday premarket trading on the announcement, and had risen as much as 16% to $3.27 in the opening 10 minutes of trade. However, it then gave up all the gains through the day and ended 4.6% lower as investors priced in the news.
Separately, on Wednesday, GMDA presented updated one-year post-transplant follow up data from a phase 3 study of its cell therapy omidubicel, which is under development as a potential stem cell transplant for patients with blood cancers.
The data showed sustained clinical benefits in the first-year post-transplant with omidubicel, as demonstrated by significant reduction in infectious complications.
The data also showed a reduction in non-relapse mortality and no significant increase in relapse rates with omidubicel vs. standard umbilical cord blood transplantation (UCBT).
The company said there was a continued trend toward improved overall survival in favor of the omidubicel arm (73%), compared to UCBT (60%) over time.
Gamida Cell (GMDA) concluded that HSCT with omidubicel results in rapid hematopoietic recovery, reduced rates of infections and no increase in GvHD rates compared to standard UCB.
GMDA presented the data at the 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings that was held from April 23-26.
In February, the company began a rolling submission of its biologics license application with the U.S. Food and Drug Administration for omidubicel.
Today's premarket activity indicates shorts
are playing their 'tricks'. Pedro was spot on!
Yesterday trade started at +35% ended -6%
May today be the exact opposite!
Gamida Cell Presents Updated One-Year Post-Transplant Follow Up Data from Phase 3 Study of Omidubicel at 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings
https://finance.yahoo.com/news/gamida-cell-presents-updated-one-110000904.html
Omidubicel is a first-in-class, advanced NAM-enabled stem cell therapy candidate with breakthrough and orphan drug designations being evaluated as the first potential allogeneic advanced cell therapy donor source for patients with blood cancers in need of a transplant
Updated data from oral presentation demonstrates overall survival trend due to early engraftment and lower infections with omidubicel
Concludes that HSCT with omidubicel results in rapid hematopoietic recovery, reduced rates of infections and no increase in GvHD rates compared with standard UCB
BOSTON, April 27, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with solid and hematological cancers and other serious diseases, today announced updated one-year post-transplant data presented on omidubicel at the 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings (TCT), being held in Salt Lake City, UT, April 23-26, 2022.
In an oral presentation titled "Allogeneic Hematopoietic Stem Cell (allo-HSCT) Transplant with Omidubicel Demonstrates Sustained Clinical Improvement Versus Standard Myeloablative Umbilical Cord Blood Transplantation (UCBT): Final Results of a Phase III Randomized, Multicenter Study," Mitchell Horwitz, M.D., Professor of Medicine, Duke Cancer Institute, shared one-year post-transplant follow up data from the omidubicel Phase 3 trial. The data showed sustained clinical benefits in the first-year post-transplant with omidubicel, as demonstrated by significant reduction in infectious complications. Results also showed reduction in non-relapse mortality and no significant increase in relapse rates with omidubicel, compared to UCBT (23% vs. 18%). It was concluded that HSCT with omidubicel results in rapid hematopoietic recovery, reduced rates of infections and no increase in GvHD rates compared with standard UCB. There was a continued trend toward improved OS in favor of the omidubicel arm over time (73% vs. 60%). The overall and sustained clinical benefit of omidubicel makes it an important addition to the options for allogeneic HSCT.
"In allo-HSCT, early engraftment and lower infections are the key predictors of long-term success for patients," said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell, "We are encouraged by the continuous positive and sustained results from patients involved in the Phase 3 trial of omidubicel, now one-year out from treatment. These results provide promising rationale that omidubicel could become a compelling treatment option for patients in need of an allo-HSCT transplant."
Gamida Cell initiated a rolling Biologics License Application (BLA) submission for omidubicel in the first quarter of 2022 and is on-track to complete submission of all modules of the BLA in the second quarter of 2022.
In total, Gamida Cell is presenting two oral and six poster presentations at TCT 2022, including an oral presentation that was selected as a TCT Best Abstract. All poster presentations are publicly available at www.ASTCT.org.
About Omidubicel
Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant for patients with hematologic malignancies (blood cancers), for which it has been granted Breakthrough Status and orphan drug designation by the FDA. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). For more information on clinical trials of omidubicel, please visit the Gamida Cell website.
Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
About NAM Technology
Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we are able to enhance, expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. This allows us to administer a therapeutic dose of cells that may help cancer patients live longer better lives.
About Gamida Cell
Gamida Cell is pioneering a proprietary NAM-enabled immunotherapy pipeline of diverse potentially curative cell therapies for patients with solid tumor and blood cancers and other serious blood diseases. We apply a proprietary platform leveraging the properties of NAM to allogeneic cell sources including umbilical cord blood-derived cells and NK cells to create therapies with potential to redefine standards of care. These include omidubicel, an investigational product with potential as a life-saving alternative for patients in need of transplant, and a line of modified and unmodified NAM-enabled NK cells targeted at solid tumor and hematological malignancies. For additional information on Gamida Cell, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, Twitter, Facebook or Instagram at @GamidaCellTx.
Cautionary Note Regarding Forward Looking Statements
This press release contains forward-looking statements as that term is defined in the Private Securities Litigation Reform Act of 1995, including with respect to timing of initiation and progress of, and data reported from, the clinical trials of Gamida Cell’s product candidates (including GDA-201), anticipated regulatory filings and the potentially life-saving or curative therapeutic and commercial potential of omidubicel. Any statement describing Gamida Cell’s goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to a number of risks, uncertainties and assumptions, including those related to the impact that the COVID-19 pandemic could have on our business, and including the scope, progress and expansion of Gamida Cell’s clinical trials and ramifications for the cost thereof; clinical, scientific, regulatory and technical developments; and those inherent in the process of developing and commercializing product candidates that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such product candidates. In light of these risks and uncertainties, and other risks and uncertainties that are described in the Risk Factors section and other sections of Gamida Cell’s Annual Report on Form 10-K, filed with the Securities and Exchange Commission (SEC) on March 24, 2022, as amended, and other filings that Gamida Cell makes with the SEC from time to time (which are available at http://www.sec.gov), the events and circumstances discussed in such forward-looking statements may not occur, and Gamida Cell’s actual results could differ materially and adversely from those anticipated or implied thereby. Although Gamida Cell’s forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Gamida Cell. As a result, you are cautioned not to rely on these forward-looking statements.
View source version on businesswire.com: https://www.businesswire.com/news/home/20220427005019/en/
Contacts
For investors:
Courtney Turiano
Stern Investor Relations, Inc.
courtney.turiano@sternir.com
1-212-362-1200
For media:
Rhiannon Jeselonis
Ten Bridge Communications
rhiannon@tenbridgecommunications.com
1-978-417-1946
Alliance Global Partners Thinks Gamida Cell’s Stock is Going to Recover
April 26 2022 - 08:46AM
In a report released yesterday, Matthew Cross from Alliance Global Partners maintained a Buy rating on Gamida Cell (GMDA – Research Report), with a price target of $11.00.
BIOS ended even worse:
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Bios take a blow, GMDA included in spite
of its (relative) good news today.
How about DOWN 7%. Pedro is right......the shorts have taken complete control of this stock with 7% short interest. With news like this, and even better news coming this one should be up a in the 7-8$ range by now, but yet everyday it is driven down and down.
We will see in a couple of months what happens when the BIG news is released, but I don't hold out too much hope for this to rise more than a few bucks, and then driven down again====such a shame really since this is the real deal.
GDA-201 is good news, however 'Omidubicel
BLA submission is on track to be completed
in the second quarter of 2022' is much more
important to move the needle north!
GDA is still early stage. I will not be too surprised
to see GMDA EOD at +5-8% at most.
(Hope to be proved wrong!)
It's getting to the point where we cannot trade anymore. The "Wolf-Pack" has already started to "short-attack" this company (during premarket trading).
Gamida Cell Announces FDA Clearance of IND and Removal of Clinical Hold for NK Cell Therapy Candidate GDA-201
https://finance.yahoo.com/news/gamida-cell-announces-fda-clearance-110000105.html
Company advancing plans to begin Phase 1/2 study in patients with follicular and diffuse large B-cell lymphomas
BOSTON, April 26, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with hematologic and solid cancers and other serious diseases, today announced that the U.S. Food and Drug Administration (FDA) cleared its investigational new drug (IND) application and removed the clinical hold for a cryopreserved formulation of GDA-201. GDA-201 is an off-the-shelf cell therapy candidate for the treatment of patients with follicular and diffuse large B cell lymphomas. Gamida Cell expects to initiate a company-sponsored Phase 1/2 clinical study in patients with follicular and diffuse large B-cell lymphomas in 2022.
"FDA clearance of our IND for the cryopreserved formulation of GDA-201 represents a significant milestone for the company and reflects our team’s expertise in the development of NAM-enabled cell therapies," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "Previously announced data from an investigator-sponsored (IS) study evaluating the fresh formulation of GDA-201 demonstrated durable complete responses in heavily pretreated patients with relapsed or refractory lymphoma. We are pleased to advance our plans to begin the company sponsored Phase 1/2 study and progress our novel cryopreserved formulation of GDA-201 with objective to address the unmet need that exists for patients with follicular and diffuse large B cell lymphomas."
GDA-201 leverages Gamida Cell’s proprietary NAM technology platform to expand the number and functionality of NK cells to direct tumor cell killing properties and antibody-dependent cellular cytotoxicity (ADCC). In an investigator-sponsored Phase 1/2 study in patients with relapsed or refractory lymphoma, treatment with the fresh formulation of GDA-201 with rituximab demonstrated significant clinical activity. Of the 19 patients with non-Hodgkin lymphoma (NHL) , 13 complete responses and one partial response were observed, with an overall response rate of 74% and a complete response rate of 68%. At the December 2021 Annual Meeting of American Society of Hematology, two-year follow-up data were reported on outcomes and cytokine biomarkers associated with survival. The data demonstrated a median duration of response of 16 months (range 5-36 months) and an overall survival at two years of 78% (95% CI, 51%–91%). In the IS study, GDA-201 was well-tolerated and no dose-limiting toxicities were observed in 19 patients with NHL and 16 patients with multiple myeloma. The most common Grade 3/4 adverse events were thrombocytopenia, hypertension, neutropenia, febrile neutropenia, and anemia. There was no incidents of cytokine release syndrome (CRS), neurotoxic events, GvHD or marrow aplasia.
Gamida Cell Presents Updated Omidubicel Data During Best Abstract Award Session at 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings
https://finance.yahoo.com/news/gamida-cell-presents-updated-omidubicel-000000045.html
Omidubicel is a first-in-class, advanced NAM-enabled stem cell therapy candidate with breakthrough and orphan drug designations being evaluated as the first potential allogeneic advanced cell therapy donor source for patients with blood cancers in need of a transplant
Oral presentation includes updated analysis demonstrating enhanced circulatory pDC, NK cell and CD4+ T cell recovery with omidubicel
Rapid immune cell recovery results in reduced rates of infection; no increase in GvHD rates compared with standard umbilical cord transplantation
Omidubicel BLA submission is on track to be completed in the second quarter of 2022
BOSTON, April 26, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with solid and hematological cancers and other serious diseases, today announced that updated infection data on omidubicel in comparison to umbilical cord blood transplantation (UCB), was shared in an oral presentation at the 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings (TCT), being held in Salt Lake City, UT, April 23-26, 2022.
The presentation, which received a TCT Best Abstract Award, titled "Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel is Associated with Enhanced Circulatory Plasmacytoid Dendritic Cells (pDC), NK Cells and CD4+ T Cells with Lower Rates of Severe Infections Compared to Standard Umbilical Cord Blood Transplantation," was presented by Paul Szabolcs, M.D., Division of Blood and Marrow Transplantation and Cellular Therapy, UPMC Children’s Hospital of Pittsburgh, Pittsburg, PA. The data from a sub-study of the Phase 3 randomized trial of omidubicel showed early and enhanced recovery of variety of immune cells, including circulatory dendritic cell subtypes, NK cells and CD4+ T cells within the first 28 days and sustained B-cell recovery from Day 28 onwards, and such immune recovery was associated with lower rates of severe infection. The data from an additional analyses of CD4+ subsets, T-cell receptor repertoire diversity and recent thymic emigrants support the long-term durability and functionality of the omidubicel graft.
"These data provide mechanistic support for the reduced infection rates seen with omidubicel-treated patients in the Phase 3 study, and clear demonstration of the benefits of omidubicel over standard cord blood transplantation, which can be associated with immune and hematopoietic recovery challenges that are devastating to patients," said Ronit Simantov, M.D., Chief Medical Officer of Gamida Cell. "As we advance omidubicel through the FDA review process, these data demonstrate that the therapy has the potential to change the outlook for patients suffering from blood cancer, who all too often struggle with post-transplant recovery complications."
Gamida Cell initiated a rolling Biologics License Application (BLA) submission for omidubicel in the first quarter of 2022 and is on-track to complete submission of all modules of the BLA in the second quarter of 2022.
In total, Gamida Cell is presenting two oral and six poster presentations at TCT 2022. All poster presentations are publicly available at www.ASTCT.org.
Gamida Cell Announces Results of New Health Economic and Outcome Study Reporting Improved Health Equity and Health Outcomes With Omidubicel at 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings
April 25 2022
Omidubicel is a first-in-class, advanced NAM-enabled stem cell therapy candidate with breakthrough and orphan drug designations being evaluated as the first potential allogeneic advanced cell therapy donor source for patients with blood cancers in need of a transplant
Study highlights increases in omidubicel use in eligible patients were associated with higher proportions of patients undergoing allo-HCT and overall improved outcomes, with improvements being greater among racial minorities
Only 20% of Black cancer patients can find a matched unrelated donor through donor registries, limiting access to disease-altering allogeneic hematopoietic stem cell transplants
Omidubicel BLA submission on track to be completed in second quarter of 2022
Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with solid and hematological cancers and other serious diseases, today announced the results of a new study demonstrating the potential impact of access to omidubicel on health disparities in allogeneic hematopoietic stem cell transplant in a poster presentation at the 2022 Transplantation & Cellular Therapy Meetings of ASTCT and CIBMTR Tandem Meetings (TCT), being held in Salt Lake City, UT, April 23-26, 2022.
The study, titled “Projected Impact of Omidubicel on Racial and Ethnic Disparities in Allogeneic Hematopoietic Cell Transplant Access and Outcomes for Patients with Hematologic Malignancies in the US,” leveraged a decision-tree model to project allo-HCT access and clinical outcomes in a hypothetical population of 10,000 allo-HCT–eligible patients in the U.S. with hematologic malignancies without an available match-related donor. The study concluded that broad use of omidubicel will extend access for allo-HCT-eligible patients, decrease time to transplant and improve clinical outcomes, notably among racial and ethnic groups with worse status quo outcomes. Projected increases in one-year overall survival ranged (with 20% omidubicel use among allo-HCT–eligible patients) from 2.5% for whites patients to 6.3% for Black patients. The study also concluded that higher levels of modeled omidubicel uptake were associated with greater improvements in clinical outcomes and greater reductions in racial disparities.
Previous studies indicate that non-white patients have a lower likelihood of finding an appropriate match in the U.S. public donor registries, with Black patients have a 16-20% chance of finding an appropriate match. Given that an allogeneic stem cell transplant is intended as a curative option, if patients cannot find an appropriate match they will not have access to allogeneic stem cell transplant, a potentially curative treatment. The Phase 3 study of omidubicel demonstrated the ability of the therapy to be used as a donor source for racially and ethnically diverse patients with 40% of patients enrolled in the study being non-white.
“Today, minority groups comprise only about 30% of all allogeneic hematopoietic stem cell transplant transplants, indicating that lack of access to a matched donor is a significant barrier to treatment in the current landscape,” said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. “This study is encouraging in that it projects that broad access to omidubicel has the potential to open up allo-HSCT as an effective treatment for more patients and address the barriers that have contributed to this alarming health disparity. These data are particularly encouraging as we continue to advance our rolling BLA submission to the FDA and move closer to bringing the therapy to more patients in need.”
Gamida Cell initiated a rolling Biologics License Application (BLA) submission for omidubicel in the first quarter of 2022 and is on-track to complete submission of all modules of the BLA in the second quarter of 2022.
In addition to this poster, two oral presentations and four additional poster presentations on omidubicel and a poster presentation on GDA-201, the company’s leading NK cell therapy program, will be shared during the conference. All poster presentations will be publicly available at www.ASTCT.org. Details below:
Title (Oral Presentation): Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel is Associated with Enhanced Circulatory Plasmacytoid Dendritic Cells (pDC), NK Cells and CD4+ T Cells with Lower Rates of Severe Infections Compared to Standard Umbilical Cord Blood Transplantation (Part of Best Abstract Award Session)
Presenting Author: Paul Szabolcs, M.D., Division of Blood and Marrow Transplantation and Cellular Therapy, UPMC Children’s Hospital of Pittsburgh, Pittsburg, PA
Session Title: Tandem Meetings Best Abstracts Session in the Scientific Track
Session Date / Time: Monday, April 25, 2022, 6:00 PM - 6:15 PM MT, SPCC, Ballroom D
Title (Oral Presentation): Allogeneic HSCT with Omidubicel demonstrates sustained clinical improvement versus standard myeloablative UCBT: Final results of a Phase III randomized multicenter study
Presenting Author: Mitchell Horwitz, M.D., Professor of Medicine, Duke Cancer Institute
Session Title: Oral Abstract - Session L - Consider the Source: Stem Cell Grafts and Donors
Session Date / Time: Tuesday, April 26, 2022, 3:15 PM – 3:30 PM MT
Title: (Poster): Allogeneic Hematopoietic Stem Cell Transplantation (Allo-HSCT) With Omidubicel: Long-Term Follow-Up From A Single Center (ASH Encore)
Lead Author: Chenyu Lin, M.D., Department of Medicine, Division of Hematologic Malignancies and Cellular Therapy, Duke University Medical Center, Durham, NC
Title (Poster): Total Costs of Care and Complication Rates Among Patients with Hematologic Malignancies Who Receive Allogeneic Hematopoietic Cell Transplants in the US
Lead Author: Richard Maziarz, M.D., Professor of Medicine, Medical Director Adult Blood and Marrow Stem Cell Transplant Program, Oregon Health and Science University, Portland, OR
Title (Poster): Hospitalization and Healthcare Resource Use of Omidubicel Vs Umbilical Cord Blood (UCB) for Hematological Malignancies in a Global Randomized Phase III Clinical Trial Setting
Lead Author: Navneet Majhail, M.D., Taussig Cancer Institute, Department of Hematology and Oncology, Cleveland Clinic, Cleveland, OH
Title (Poster): HRQOL following transplantation with omidubicel versus UCB in patients with hematologic malignancies: Results from a Phase III randomized , multicenter study
Lead Author: Mitchell Horwitz, M.D., Professor of Medicine, Duke Cancer Institute
Title (Poster): Transcriptional and Metabolic Profiling of Nicotinamide-Enhanced Natural Killer (NAM-NK) Cells (GDA-201)
Lead Author: Dima Yackoubov, Scientist, Gamida Cell
About Omidubicel
Omidubicel is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell transplant for patients with hematologic malignancies (blood cancers), for which it has been granted Breakthrough Status and orphan drug designation by the FDA. Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia (NCT03173937). For more information on clinical trials of omidubicel, please visit the Gamida Cell website.
Omidubicel is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.
About NAM Technology
Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we are able to enhance, expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. This allows us to administer a therapeutic dose of cells that may help cancer patients live longer better lives.
Gamida Cell to Present Corporate Highlights and Participate in Panel Discussion at the Needham Healthcare Conference
https://finance.yahoo.com/news/gamida-cell-present-corporate-highlights-120000400.html
BOSTON, April 07, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with solid and hematological cancers and other serious diseases, announced that company management will present at the upcoming 21st Annual Needham Virtual Healthcare Conference on April 12, 2022 at 1:30 p.m. EDT. Management will discuss 2022 catalysts and potential milestones including executing its U.S. commercial strategy for the launch of the first allogenic stem cell therapy upon U.S. Food and Drug Administration approval, accelerating the development of its first-in-class NAM-enabled natural killer (NK) cell therapy, GDA-201, as a new approach for patients with follicular and diffuse large B-cell lymphomas, and expansion of its NAM-enabled cell therapy pipeline with multiple next-generation, genetically engineered NK cells.
Additionally, Julian Adams, Ph.D., chief executive officer of Gamida Cell, will participate in a live panel discussion titled "Company Perspectives: Companies Discussing Key Features and Differentiators in the NK Cellular Therapeutics Space," on April 14, 2022 at 11:00 a.m. EDT.
The webcast of the presentation will be available on the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com, and will be available for at least 14 days following the event.
About NAM Technology
Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM, we are able to expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. This allows us to administer a therapeutic dose of cells that may help cancer patients live longer better lives.
Gamida Cell Ltd. (NASDAQ:GMDA)
https://finance.yahoo.com/news/10-best-biotech-stocks-under-141250414.html
Number of Hedge Fund Holders: 12
Share Price as of March 28: $4.1265
Headquartered in Jerusalem, Israel, Gamida Cell Ltd. (NASDAQ:GMDA) develops cell therapies for blood cancers, lymphoma, multiple myeloma, and hematologic diseases. It is one of the best cheap biotech stocks to invest in, priced at $4.1265.
On January 19, H.C. Wainwright analyst Vernon Bernardino reiterated a Buy rating on Gamida Cell Ltd. (NASDAQ:GMDA) with a $22 price target. This came in light of the company announcing its successful Type B meeting with the FDA and plans to initiate in the first half of 2022 a rolling Biologics License Application submission for omidubicel, a cell therapy for blood cancer. He calls Gamida Cell Ltd. (NASDAQ:GMDA) a top pick for 2022.
Among the hedge funds tracked by Insider Monkey, 12 funds were bullish on Gamida Cell Ltd. (NASDAQ:GMDA), with combined stakes worth $13.1 million. Rock Springs Capital Management is the biggest shareholder of the company, with 2.15 million shares worth $5.4 million.
Spire Wealth Management Buys Shares of 55,125 Gamida Cell Ltd. (NASDAQ:GMDA)
Posted by Stephan Byrd on Mar 22nd, 2022
https://www.tickerreport.com/banking-finance/8564111/spire-wealth-management-buys-shares-of-55125-gamida-cell-ltd-nasdaqgmda.html
Gamida Cell logoSpire Wealth Management bought a new stake in Gamida Cell Ltd. (NASDAQ:GMDA – Get Rating) during the fourth quarter, according to its most recent filing with the Securities and Exchange Commission (SEC). The fund bought 55,125 shares of the company’s stock, valued at approximately $140,000.
Other institutional investors and hedge funds also recently added to or reduced their stakes in the company. Bank of New York Mellon Corp purchased a new position in Gamida Cell in the third quarter valued at about $39,000. Guild Investment Management Inc. purchased a new position in Gamida Cell in the third quarter valued at about $71,000. Phoenix Holdings Ltd. purchased a new position in Gamida Cell in the third quarter valued at about $114,000. Cubist Systematic Strategies LLC boosted its stake in Gamida Cell by 54.5% in the third quarter. Cubist Systematic Strategies LLC now owns 33,523 shares of the company’s stock valued at $131,000 after acquiring an additional 11,821 shares during the last quarter. Finally, Jane Street Group LLC purchased a new position in Gamida Cell in the third quarter valued at about $143,000. Hedge funds and other institutional investors own 53.43% of the company’s stock.
Gamida Cell Ltd. (NASDAQ:GMDA) Given Consensus Rating of "Buy" by Brokerages
https://www.marketbeat.com/instant-alerts/nasdaq-gmda-consensus-analyst-rating-2022-03-2-3/
MONDAY, MARCH 21, 2022 | MARKETBEAT
Gamida Cell logoGamida Cell Ltd. (NASDAQ:GMDA - Get Rating) has been given an average recommendation of "Buy" by the seven brokerages that are covering the stock, Marketbeat Ratings reports. Seven research analysts have rated the stock with a buy recommendation. The average 1 year price objective among brokerages that have updated their coverage on the stock in the last year is $13.39.
Several equities research analysts recently issued reports on the stock. Zacks Investment Research raised shares of Gamida Cell from a "hold" rating to a "buy" rating and set a $3.75 price objective for the company in a research note on Thursday, February 3rd. HC Wainwright reaffirmed a "buy" rating and set a $22.00 price objective on shares of Gamida Cell in a report on Wednesday, March 16th. Finally, JMP Securities reissued a "buy" rating and set a $17.00 target price on shares of Gamida Cell in a research report on Tuesday, February 1st.
Several hedge funds and other institutional investors have recently added to or reduced their stakes in GMDA. Stonepine Capital Management LLC increased its stake in Gamida Cell by 288.8% during the third quarter. Stonepine Capital Management LLC now owns 1,724,201 shares of the company's stock valued at $6,759,000 after acquiring an additional 1,280,694 shares during the period. Altshuler Shaham Ltd lifted its position in Gamida Cell by 93,785.0% in the third quarter. Altshuler Shaham Ltd now owns 499,468 shares of the company's stock worth $1,968,000 after purchasing an additional 498,936 shares during the period. Marshall Wace LLP acquired a new stake in Gamida Cell during the third quarter worth $1,463,000. Renaissance Technologies LLC increased its holdings in Gamida Cell by 153.3% in the 3rd quarter. Renaissance Technologies LLC now owns 531,184 shares of the company's stock valued at $2,082,000 after buying an additional 321,484 shares during the period. Finally, Yahav Achim Ve Achayot Provident Funds Management Co Ltd. bought a new stake in Gamida Cell in the 3rd quarter valued at $686,000. Institutional investors and hedge funds own 53.43% of the company's stock.
Gamida Cell Ltd. (GMDA) Q4 2021 Earnings Call Transcript
By Motley Fool Transcribing - Mar 15, 2022 at 12:01PM
Gamida Cell Ltd. ( GMDA -2.10% )
Q4 2021 Earnings Call
Mar 15, 2022, 8:00 a.m. ET
Contents:
Prepared Remarks
Questions and Answers
Call Participants
Prepared Remarks:
Operator
Ladies and gentlemen, thank you for standing by. Welcome to Gamida Cell's conference call for the fourth quarter and full year 2021 financial results. My name is Howard and I'll be your operator for today's call. Please be advised that this call is being recorded at Gamida Cell's request.
Now, I would like to introduce your host for today's conference, Heather DiVecchia, Gamida Cell's chief of staff. Please go ahead.
Heather DiVecchia -- Chief of Staff
Thank you, Howard, and good morning, everyone. Welcome to today's call, during which we will provide an update on the company and review our financial results for the fourth quarter and full year of 2021. Earlier this morning, we issued a press release summarizing our financial results and progress across the company, which is available on our website at website at www.gamidacell.com. Here with me on our call today are Julian Adams, chief executive officer; Ronit Simantov, our chief medical officer and chief scientific officer; Michele Korfin, our chief operating officer and chief commercial officer; and Shai Lankry, chief financial officer.
Julian Adams -- Chief Executive Officer
Thank you, Heather, and thanks to everyone for joining us this morning. Before I begin, I want to take a moment to acknowledge the major events in the Ukraine, that are impacting the world globally over the past few weeks. While we don't have any operations in the Ukraine or Russia and expect minimal impact to our operations, as global citizens, our thoughts are with all those affected. At Gamida Cell, we are incredibly proud to be part of the significant advancements occurring in the field of cell therapy.
As it continues to grow and progress -- progress toward cures for patients in need. 2021 was an important year for us as we progressed our two clinical stage NAM-enabled cell therapy programs, Omidubicel and GDA-201. Both of which hold significant potential to benefit patients suffering from hematologic malignancies and serious blood disorders. Additionally, we further expanded our NAM-enabled platforms with the addition of genetically modified NAM-enabled NK cell constructs, allowing us to leverage our expertise in NK cells to potentially treat various hematologic malignancies and solid tumors.
I will start today's call by commenting on our lead program, Omidubicel, which has breakthrough therapy designation and the potential to be the first FDA-approved cell therapy for stem cell transplant. Throughout 2021, we announced several data presentations which contribute to the evidence for Omidubicel's potential efficacy, and also the positive health economic impact of Omidubicel, with data demonstrating a reduction in healthcare resource utilization. As Ronit will describe later on in this call, this reduction in healthcare resource utilization is very important for patients, hospitals, and payers. The robust data that we have generated in support of the potential for Omidubicel led us to our most recent and exciting milestone last month, namely the initiation of the rolling BLA for Omidubicel.
With the submission of our non-clinical module following the receipt -- the recent receipt of positive CMC focused Type B meeting correspondence from the FDA. In this correspondence, the FDA confirmed analytical comparability between our wholly owned commercial manufacturing facility in Israel to the Omidubicel batches that were produced at the clinical manufacturing sites for the Phase three study. This month, we also submitted our clinical module to the FDA. We are on track to submit the remaining modules and the complete -- and complete the full BLA submission by the end of the second quarter.
We are thrilled to have [Audio gap] reached this inflection point and look forward to working with the FDA to bring Omidubicel to patients as soon as possible. Beyond on Omidubicel, we are also developing our NAM-enabled NK pipeline, which is led by GDA-201. GDA-201 leverages our proprietary NAM technology platform to expand natural killer cells to enhance their functionality, direct tumor cell killing properties, an antibody-dependent cellular cytotoxicity or ADCC. We are highly encouraged by the potential of GDA-201, which has produced truly remarkable results in a phase one investigator sponsored study, where we have seen very high and complete durable responses in both follicular lymphoma and diffuse large B-cell lymphoma.
In September 2021, we submitted an IND application to the FDA for a phase one two trial with a cryopreserved formulation of GDA-201 in patients with diffuse large B-cell lymphoma and follicular lymphoma. Following this submission, we were placed on clinical hold in November of 21, prior to patients being dose since the FDA had questions about donor eligibility procedures and assay qualification. We are actively working with the FDA to address their comments to enable an IND acceptance and study initiation. This year, we plan to initiate dosing in this phase one/two study.
Moving to our newest product candidates, a genetically modified NAM NK enabled cell therapy programs. Last year, we were excited to establish a broad pipeline of NK product candidates that utilize CAR and CRISPR mediated strategies to increase targeting potency and persistence against hematologic malignancies and solid tumors. Robust preclinical evidence gives us the confidence that these new NAM-enabled NK product candidates hold promise as potentially curative therapies for both hematologic cancers and solid tumors. Furthermore, we announced a research collaboration with the Dana-Farber Cancer Institute for GDA-601, a CD38 CRISPR knockout combined with a CD38 CAR NK cell construct that has demonstrated promising preclinical results against multiple myeloma cell lines.
We are excited to leverage the expertise of researchers at Dana-Farber to study the in vitro NK cell killing activity of GDA-601 in multiple myeloma. Throughout 2022, we plan to execute preclinical proof of concept studies for these genetically modified NK therapeutic targets, and by the end of 2022, select a pipeline candidate for IND enabling studies. With the progress we achieved in 2021, we are encouraged about what the future holds for Gamida Cell in '22. And of course, this wouldn't be possible without our employees, whose dedication is focused on advancing therapies to help address unmet needs for patients.
I am grateful for and proud of their continued determination and focus on patients, which has brought us to where we are today. Before turning the call over to Ronit, I would like to take a moment to congratulate her on her recent promotion. Now, assuming the role of not only chief medical -- medical officer but also chief scientific officer, we at Gamida thank you all for your contributions thus far, and look forward to your continued work toward the vision with your expanded role. Ronit?
Ronit Simantov -- Chief Medical Officer
Thanks, Julian, and good morning, everyone. Thank you for joining us on our call. In 2021, we had the opportunity to present a broad range of scientific and clinical data on Omidubicel. Tying together, free clinical and translational analyzes with clinical data, patient experience, long-term outcomes, and value to the healthcare system.
In pre-clinical presentations in 2021, we demonstrated that our nicotinamide or NAM platform, generates a unique cellular phenotype through modulation of metabolic pathways leading to highly functional and potent stem cells. In translational data, we showed that patients treated with Omidubicel had robust functional reconstitution of T and B cell subsets, dendritic cells and natural killer cells in the days and weeks following transplant. The translational data provided mechanistic support for the clinical results of our global phase three randomized trial. The results of which were published in October 2021, demonstrating rapid somatic poetic recovery, significantly decreased infections and shorter duration of hospitalization for patients transplanted with Omidubicel.
Of note, the patients in our clinical trial were in critical need of allogeneic transplant for hematologic malignancies, with few or no alternatives available. Looking back at the totality of our experience with Omidubicel, we presented follow-up data from over 10 years of clinical studies demonstrating long-term sustainable [Inaudible] and immune competence in patients. We then went on to show that Omidubicel could decrease the cost to the healthcare system, as patients treated with Omidubicel had reduced healthcare resource utilization, including blood transfusions, outpatient procedures, and time in intensive care units. At next month's transplantation and cellular therapy meetings in Salt Lake City, we have eight abstracts accepted and planned to report new data focused on patient outcomes and health related quality of life.
Taken together, we have continued to add to the body of evidence supporting the mechanism of action, clinical activity, patient experience, and value of Omidubicel as a potential life-saving therapy. Moreover, we are confident in the scientific and clinical package we have assembled, and our efforts to make Omidubicel available to patients in need of a hematopoietic stem cell transplant. We also made considerable progress in our NAM-based natural killer cell platform. We presented preclinical data characterizing the properties in GDA-201.
We showed that NAM mediates set of cellular processes, expanding NK cells while down regulating differentiation, cellular stress, and exhaustion pathways that are typically activated in culture. GDA-201 cells are characterized by a rejuvenated NK phenotype similar to cytokine induced memory like NK cells, and were shown to be highly cytotoxic in in vitro and in vivo assays. These findings were supported by the clinical data reported in the phase one investigator sponsored study at the University of Minnesota. At ASH, we presented two-year follow-up data in patients with non-Hodgkin lymphoma, showing an overall response rate of 74%, including 13 out of 14 complete responses with a median duration of response of 16 months.
78% overall survival and toxicities in line with those reported previously for GDA-201. We've now developed a cryopreserved formulation and plan to initiate a multi-center company sponsored study this year in patients with follicular lymphoma and diffuse large B-cell lymphoma. In addition, we reported that we have expanded upon our NAM platform to develop genetically modified NK cells, GDA-301, GDA-501 and GDA-601, each of which is proceeding with preclinical proof of concept studies as we advance toward IND enabling studies. Overall, 2021 was a tremendously productive year in research and development and clinical research.
We look toward continuing to validate the therapeutic potential of our product candidates. I will now turn the call over to Michele, who will talk more about our launch readiness for Omidubicel. Michelle?
Michele Korfin -- Chief Operating Officer and Chief Commercial Officer
Thank you, Ronit, and good morning, everyone. This year has started off strong already, as last month we initiated our rolling BLA submission for Omidubicel. We continue to make excellent progress advancing our Gamida owned manufacturing facility in Israel. The FDA acknowledgment of the analytical comparability from our planned Gamida commercial facility as compared to the facility used for the phase three study was an important milestone as we work toward advancing Omidubicel.
The commercial opportunity for Omidubicel is extremely encouraging. Based on our continued assessment of market insights, and we continue to focus on our commercial preparation. In parallel with our rolling BLA submission, we are additionally assessing strategic alternatives for the potential commercialization of Omidubicel, including potentially partnerships to expand the reach of Omidubicel. Omidubicel has the potential to be the first FDA-approved advanced cell therapy product for allogeneic stem cell transplant in patients with hematology -- hematologic malignancies.
For patients with hematologic malignancies that are deemed eligible for an allergenic stem cell transplant, the procedure is their best chance for a potential cure. In the U.S., there are approximately 8,000 patients above the age of 12, with hematologic malignancies who undergo an allergenic stem cell transplant each year. Unfortunately, though, there are approximately 1,200 patients each year who are also aged 12 and up with hematologic malignancies who are deemed eligible for an allergenic stem cell transplant but cannot find an appropriate donor. Unfortunately, we observe racial disparity in the U.S.
in regards to access to allogeneic stem cell transplants. Few are non-caucasian and do not have access to a family member donor, we have a very low likelihood of finding a match in the public database. For example, patients who are African-American may only have about a 20% chance of finding a match. For patients in need of a stem cell transplant, this is often their only chance for a cure.
We are diligently working to advance Omidubicel and to expand access to patients. Based on encouraging clinical data and the less stringent matching criteria, transplanters feedback indicates that Omidubicel has the potential to improve outcomes for allogeneic stem cell transplant patients compared to other donor sources, and expand access for patients who cannot find a suitable donor. In the U.S., there are approximately 200 transplant centers that perform allogeneic stem cell transplants. 70 of those centers conduct about 80% of the transplants, and our medical team continues to engage with those top 70 centers through one on one meetings and at medical conferences.
We also continue to assess market insights from transplanters in the U.S. Based on extensive market research. We see the potential for Omidubicel to treat approximately 2,000 to 2,500 patients per year upon reaching peak market share. This equates to approximately 20% to 25% market share of the addressable population.
Our market access team is also actively engaging in meetings with national and regional commercial payers, and the feedback continues to be very encouraging. Payers recognize the overall value proposition, including the strength of the Omidubicel clinical data and the health economic data we have published to date. In summary, we are excited by the potential of Omidubicel to be the first FDA-approved cell therapy for allogeneic stem cell transplant. And we are also encouraged by the clinical data and feedback from physicians and payers.
I will now turn the call over to Shai, to review our financial results.
Shai Lankry -- Chief Commercial officer
Thank you, Michele, and good morning, everyone. Today, I will summarize our financial results for the full year of 2021. As of December 31, 2021, our total cash position was approximately $96 million, compared to $127.2 million at December 31st of last year. Research and development expenses for the year were $50.2 million, compared to $38.9 million for the same period in 2020.
The increase was primarily due to a $5.4 million increase in Omidubicel commercial manufacturing readiness activities and the advancements of our NK programs, as well as an increase of $5.9 million in broadening our scientific capabilities and talent. Commercial expenses in 2021 were $20 million, compared to $8.9 million in 2020. The increase was mainly due to a $6.5 million increase in commercial readiness expenses and $4.6 million increase in headcount within our commercial organization. Going forward, we anticipate reducing our near-term commercial readiness expenses as we are assessing alternative for the commercialization of Omidubicel including potential U.S.
or global partnership. General administrative expenses rose $17 million in 2021, compared to $13.2 million in 2020. The increase was mainly due to a $2.6 million increase in professional services expenses, and a $1.2 million increase in headcount and related expenses. Finance expenses net were $2.6 million in 2021, compared to $0.6 million in 2020.
The increase was primarily due to a $4.4 million interest expenses in our convertible notes offset by a $2.1 million capitalization and other non-cash income and $0.2 million increase in interest income from cash management. Net loss in 2021 was $89.8 million compared to a net loss of $61.6 million in 2020. We continue to expect cash use for ongoing operating activity this year to range from $60 million to $70 million. We anticipate the recurring total cash position will support our ongoing operating activities into mid-2023.
This cash one way guidance is based on our current operational plan and excludes any additional funding that may be received or business development activities that may be undertaken. I will now turn the call back over to Julian.
Julian Adams -- Chief Executive Officer
Thank you, Shai. As we look ahead into what we have set out to accomplish in this year, 2022. I believe we are well-positioned to deliver on our mission of developing potentially curative cell therapies for patients with blood cancers, solid tumors, and other serious blood disorders. We look toward our full BLA submission for Omidubicel, expected in the second quarter of this year.
And the planned initiation of our phase one/two multi-center Gamida's cell sponsored study for GDA-201 and non-Hodgkin lymphoma. We are excited for the opportunity to continue leveraging our unique NAM-enabled platform across a broad range of cell therapies. And I look forward to providing updates throughout the year. Now we will open the call for questions.
Operator?
Questions & Answers:
Operator
[Operator instructions] Our first question or comment comes from the line of Jonathan Miller from Evercore ISI. Your line is open.
Jonathan Miller -- Evercore ISI -- Analyst
Hey, guys. Thanks for taking my questions. First, maybe on GDA-201. It feels like the language here seems a little bit similar to last update.
And I just wondered if you had any additional color on your recent interactions with the agency, and what your current thoughts are and ability to get that IND [Inaudible]?. And then maybe secondly, I'm happy to see the runway guidance reinstated, but just wanted to get some clarity. How much commercial prep is included in the current runway guidance -- and maybe how much clinical work for GDA-201 is included in that guidance?
Julian Adams -- Chief Executive Officer
So let me begin by addressing the first part of your question. We haven't had any direct communication with the FDA. The FDA provided absolute clarity on what issues we needed to resolve. Most of those issues are resolved and we are getting ready to resubmit the amendment to the IND, and we'll be announcing when the IND is accepted.
Regarding runway guidance, let me turn it over to Michelle and Shai to talk about launch readiness and use of cash forge support of GDA-201. Michelle?
Michele Korfin -- Chief Operating Officer and Chief Commercial Officer
Thank you, Julian, and good morning, Jonathan. So Jonathan, I'll start and then I'll turn to Shai for the additional financials. So in regards to runway guidance, so for 2021, we were able to complete a lot of the key milestones for commercial preparation that led to the increase that Shai discussed for opex. So some of those included, the market insights such as quantitative assessments from transplanters and feedback from payers.
In addition of very, very important milestone for us in 2021, was the readiness of Gamida Cell Assist, although Gamida Cell sits within the commercial organization. It plays a critical role in starting the chain of identity and monitoring the chain of identity and chain of custody. Although Omidubicel has less stringent requirement, it is still an individualized therapy and hence we need to demonstrate chain of identity chain of custody. That key work for Gamida Cell Assist, including building the team, developing the processes, and establishing the IT infrastructure was all completed in 2021.
So what that allowed us to do in 2022 as we are assessing our strategic alternatives or potential strategic alternatives for launch. It allowed us to decrease the spend in regards to some of the commercial preparation because of the fact that so much had been done in '21. So let me turn to Shai to give us the actual financial details. And then Jonathan, we'll see if you have any follow-up questions.
Shai Lankry -- Chief Commercial officer
Thank you, Michelle. So, Jonathan, for your question, I would divide the answer into two. As Michele [Inaudible] for the commercial. We did an internal robust process of prioritization our expense in 2022 in order to meet the cash one way guidance.
And I can tell you that all the critical activity will continue to be advanced. And in addition, as we mentioned in -- in the previous comments, we did reduce some of the spending, which are less critical for the next few months. As for the R&D GDA-201 in particular, I can assure you that we continue to advance the GDA-201, and we did -- as part of that -- as part of the prioritization, we didn't reduce any of the GDA-201. We continue to advance this program, including the potential initiation of trials later this year and first patient in.
Jonathan Miller -- Evercore ISI -- Analyst
Thanks so much, guys.
Julian Adams -- Chief Executive Officer
Thank you, Jon.
Operator
Thank you. Our next question or comment comes from the line of Ted Tenthoff from Piper Sandler. Your line is open.
Ted Tenthoff -- Piper Sandler -- Analyst
Great. Thank you very much, and good morning, everybody. So I want to dig in a little bit more to the considerations with respect to what we do best on marketing. To me, it seems like the greatest value would be retained if this is a therapy that you take to market yourself.
But can you give it a little bit more just in terms of how you're assessing this? Could this be overseas deal where you keep the U.S.? Could this be a global deal? Give us maybe a sense for how and what kinds of options you're considering. Thanks.
Julian Adams -- Chief Executive Officer
So, Ted, thank you for your question and good morning. As was mentioned in the script, we're assessing our strategic alternatives for Omidubicel, and that's driven by our desire to see as many patients as possible, gain access to Omidubicel. So we are discussing regional deals, potential regional deals, as well as potentially U.S. deals with partners that may have a unique interest in the hematology-oncology franchise and that pairs up with their portfolio, and already has significant infrastructure to be able to deliver on Omidubicel to as many patients as possible.
Our involvement will always be very intimate, because we are the manufacturer and as you know in cell therapies, the manufacturing facilities is critical to supporting any kind of commercial product. So we haven't made any choices yet. And we're starting and certainly once the BLA is fully filed and accepted, that will open up avenues for conversations with interested parties.
Ted Tenthoff -- Piper Sandler -- Analyst
Yup. Very helpful, truly. And I appreciate that additional color.
Julian Adams -- Chief Executive Officer
My pleasure.
Operator
Thank you. Our next question or comment comes from the line of Jason Butler from JMP Securities. Your line is open.
Jason Butler -- JMP Securities -- Analyst
Hi, thanks for taking the question. Just another on Omidubicel. Can you just talk about plans for additional data presentations this year, including from the open label extension to extended -- expanded access program? Thank you.
Julian Adams -- Chief Executive Officer
So thank you, Jason, for your question. Ronit mentioned during the prepared remarks, that we have eight abstract TCT, but maybe Ronit, you could perhaps highlight other venues where we might present the maturing dataset for Omidubicel.
Ronit Simantov -- Chief Medical Officer
Absolutely, and thank you, Jason, for your question. So a TCT, as I mentioned, we'll be focusing on long-term follow up information, as well as patient reported outcomes, health related quality of life, additional immune reconstitution data and some preclinical data as well, and some information on the value and cost of care. In terms of the expanded access program, we are continuing to recruit patients for the expanded access program have not made a definitive decision about what the appropriate time would be to present that growing cohort of patients. But when there is a, I think, critical amount of information that's worth presenting, we certainly will do that either by the end of the year at a hematological meeting or maybe next year at the transplant meeting.
Jason Butler -- JMP Securities -- Analyst
Great. Thank you.
Operator
Thank you. Our next question or comment comes from the line of Gil Blum from Needham & Company. Your line is open.
Gil Blum -- Needham and Company -- Analyst
Good morning, everyone, and thanks for taking our questions. So this one's for Michelle. From your discussions with physicians, what feedback are you been getting regarding their interest in an FDA-approved transplant versus what they've been doing to date? The focus here being whether they have an opinion on something that is FDA-approved and the feedback that you've heard so far.
Michele Korfin -- Chief Operating Officer and Chief Commercial Officer
Thank you, Gil, and good morning. We -- we've done a lot of work speaking to physicians and what -- what is encouraging for them about Omidubicel, which falls into the category of an FDA-approved therapy is the following. So, the first off, Omidubicel will take on the responsibility around chain of identity and chain of custody from the time of the patient selection from Omidubicel all the way through the return of the therapy to the center. So that's an important responsibility, and that's something that comes with being an FDA-approved therapy.
The other aspect is around the Omidubicel itself. So the NAM technology allows us to not only expand but enhance the cells. And some of the challenges associated with using other donor sources is to have an adequate number of cells. Does the donor potentially have some co-morbidities that could potentially be affecting the donor ability? So the fact with Omidubicel that we will be an FDA regulated therapy with sort of a defined threshold around release criteria, that that is viewed as a positive.
I will say consistently the feedback we hear from U.S. transplant ERS is that when they see the Omidubicel clinical data, they see Omidubicel opportunity for their patients falling into two key categories. The first from the physician's perpection -- perspective, is the ability to improve outcomes as compared to donor sources that they're currently using. And then the second that very critical one of those patients who were deemed eligible for transplant but can't find a match, they see a very important opportunity for Omidubicel, given the fact we have a less stringent matching criteria, we demonstrated in our clinical study we had approximately 40% of patients that were non-caucasian in our clinical study.
So those two key opportunities really do resonate consistently when we speak to physicians.
Julian Adams -- Chief Executive Officer
And if I may add, Michelle. There's no question that having a quality assured product with specifications and an FDA label clearly gives us the opportunity to engage with physicians and educate, and over time, this is an exercise in behavioral economics, if you will. If physicians find patients that are suitable for Omidubicel and have a good experience that will go a long way to encouraging them to continue to prescribe Omidubicel. So we think that an FDA label for the first ever sponsored randomized phase three study is a significant game changer for the field of -- hematopoietic stem cell transplant.
Gil Blum -- Needham and Company -- Analyst
All right. Thank you, Michele, and thank you, Julian, for taking our question.
Michele Korfin -- Chief Operating Officer and Chief Commercial Officer
Thank you, Gil.
Julian Adams -- Chief Executive Officer
Thank you, Gil.
Operator
[Operator instructions] Our next question or comment comes from the line of Matthew Cross from Alliance Global. Your line is open.
Matthew Cross -- Alliance Global Partners -- Analyst
Hi, good morning, and thanks for taking a couple of questions for me. Both on the NK cell side. One, I wanted to ask regarding the cryopreservation status for GDA-201. I know you'd previously tested Omidubicel as both a cryopreserved and non-cryopreserved product.
Since you're finalizing these -- these IND requirements with the FDA for 201, I was just hoping to get a recap on the clinical supply chain for that asset in terms of cold chain and what's required for that product in particular, given continuing supply issues at a broad level.
Julian Adams -- Chief Executive Officer
So let -- let me take that on. The team has worked very diligently through 2021 to perfect and optimize the cryopreservation. We now have product and stability with many months of stability, maintain that liquid nitrogen. So the goal of this is to have an off the shelf product to enable a multi-center phase one/two study, and be able to recruit patients with a -- single treatment with GDA-201.
So all of those activities have been attended to, and we're just finalizing the amendment to the IND to resubmit to the FDA and begin the clinical study in 2022.
Matthew Cross -- Alliance Global Partners -- Analyst
Got it. OK. Thanks, Julian, appreciate the confirmation there. And then you had stated that you're looking to select a candidate for IND enabling studies this year out of genetically modified NK cell program.
But just wanted to confirm whether I'm taking that statement overly, literally. Are you looking at advancing one of these four programs to [Inaudible] IND this year and kind of tabling the rest for a bit? Or all of these making progress, and maybe just one will become a frontrunner during the year? Thanks.
Julian Adams -- Chief Executive Officer
So, Matt, thank you for your very important question. So right now, we have three candidates that are progressing. It's all data driven, and my comments about GDA-601 further allowed us to expand our collaboration with Dana-Farber to look at multiple myeloma cell lines, as well as fresh patient isolates, who are patients who have been heavily pretreated. So the idea is progress all three, but select one candidate for IND enabling studies, and then subsequently continue to make progress on the other candidates.
Again, it'll all be data driven, and we'll select the candidate with the most compelling data going forward.
Matthew Cross -- Alliance Global Partners -- Analyst
Great. OK. Thanks for the clarification there as well. I appreciate all the answers.
Operator
Thank you. I'm showing no additional in the queue -- additional questions in the queue at this time. I'd like to turn the conference back over to Julian Adams for any closing remarks.
Julian Adams -- Chief Executive Officer
Thank you, operator, and thank you everyone for joining us on today's call. We look forward to engaging with you, and have a good day, everyone, and see you next quarter.
Operator
[Operator signoff]
Duration: 39 minutes
Call participants:
Heather DiVecchia -- Chief of Staff
Julian Adams -- Chief Executive Officer
Ronit Simantov -- Chief Medical Officer
Michele Korfin -- Chief Operating Officer and Chief Commercial Officer
Shai Lankry -- Chief Commercial officer
Jonathan Miller -- Evercore ISI -- Analyst
Ted Tenthoff -- Piper Sandler -- Analyst
Jason Butler -- JMP Securities -- Analyst
Gil Blum -- Needham and Company -- Analyst
Matthew Cross -- Alliance Global Partners -- Analyst
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Gamida Cell Reports Fourth Quarter and Full Year 2021 Financial Results and Provides Company Update
https://finance.yahoo.com/news/gamida-cell-reports-fourth-quarter-110000407.html
- Initiated rolling BLA submission for omidubicel in the first quarter of 2022 with full BLA submission on track for first half of 2022 -
- Finished fourth quarter of 2021 with approximately $96 million in cash; sufficient cash to fund the company’s operations into mid-2023 -
- Company to host conference call at 8:00 a.m. ET today -
BOSTON, March 15, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, today provided a business update and reported financial results for the year and quarter ended December 31, 2021. Net loss for 2021 was $89.8 million, compared to a net loss of $61.6 million in 2020. As of December 31, 2021, Gamida Cell had total cash and cash equivalents of $95.9 million.
During the past quarter and into 2022, Gamida Cell:
Continued to advance omidubicel, a potentially life-saving cell therapy treatment for patients with blood cancers in need of stem cell transplant. In the first quarter of 2022, Gamida Cell initiated a rolling Biologics License Application (BLA) submission for omidubicel following the receipt of positive Type B meeting correspondence from the U.S. Food and Drug Administration (FDA).
Progressed activities to address the FDA’s clinical hold on the Investigational New Drug (IND) application for GDA-201, which was imposed based on FDA questions about donor eligibility procedures and assay qualification prior to the initiation of the study in patients with follicular and diffuse large B-cell lymphomas.
Advanced the company’s NAM-enabled natural killer (NK) cell pipeline, including targets GDA-301, GDA-501 and GDA-601, which focus on solid-tumor and hematological cancers. These targets utilize CAR, membrane bound- and CRISPR-mediated technologies to increase targeting, potency and persistence against hematologic malignancies and solid tumors.
"Throughout 2021, Gamida Cell made meaningful progress advancing our broad immunotherapy pipeline of potentially curative cell therapies for patients with solid tumor and blood cancers and other serious blood diseases, resulting in the recent initiation of our rolling BLA submission for omidubicel, which we expect to complete in the second quarter of this year. Additionally, we are continuing to work diligently to respond to the FDA regarding our IND for GDA-201, and expect to initiate our Phase 1/2 study in patients with follicular and diffuse large B-cell lymphomas once the clinical hold has been removed," said Julian Adams, Ph.D., chief executive officer of Gamida Cell. "Looking ahead in 2022, we are continuing to progress our genetically modified NK cell immunotherapy programs leveraging CAR- and CRISPR-mediated technologies focused on addressing unmet needs for patients with hematologic malignancies and solid tumors and we will select a product candidate for IND-enabling studies by the end of the year."
Fourth Quarter and Recent Developments
Omidubicel: Advanced Cell Therapy
BLA submission: Following the receipt of positive Type B meeting correspondence from the FDA confirming that analytical comparability has been established between Gamida Cell’s wholly-owned commercial manufacturing facility and the product that was manufactured for the Phase 3 study, Gamida Cell initiated a rolling BLA submission for omidubicel in the first quarter of 2022. As part of the rolling BLA, Gamida Cell submitted the nonclinical and clinical modules of the omidubicel BLA and is on-track to complete submission of all remaining modules of the BLA by the first half of 2022. In parallel with the BLA submission, the company is assessing alternatives for the commercialization of omidubicel, including potential U.S. or global partnerships.
New data presented at ASH: At the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition in December 2021, Gamida Cell presented clinical updates and a health economic analysis on hospital resource utilization.
Data collected from a subset of patients in the omidubicel Phase 3 trial showed that, in addition to more rapid short-term hematopoietic recovery, omidubicel-treated patients had more rapid recovery of a wide variety of immune cells including CD4+ T cells, B cells, monocytes, NK cells, and dendritic cells than the control arm. The robust recovery of a broad range of the immune system correlated with and supported clinical data showing fewer severe bacterial, fungal, and viral infections in patients treated with omidubicel.
Resource utilization data during the first 100 days after transplant showed that omidubicel-treated patients had significantly shorter durations of hospitalization, intensive care unit time, consultant visits, procedures, and transfusions than the control arm. These data provide further evidence of the clinical benefit associated with the more rapid hematopoietic recovery in patients treated with omidubicel and the corresponding reduction in healthcare resource utilization.
An analysis of outcomes of patients with hematologic malignancies treated with omidubicel over a 10-year period showed long-term sustained bone marrow function and immune recovery, with a 10-year overall survival of 48%. These data provide further support for the long-term clinical benefit of omidubicel with long-lasting hematopoietic recovery.
GDA-201: NAM-Enabled NK Cell Therapy
IND for Phase 1/2 Study: Gamida Cell is working diligently to address the clinical hold on the IND for a Phase 1/2 study of GDA-201. Gamida Cell expects to initiate a company-sponsored Phase 1/2 clinical study in patients with follicular and diffuse large B-cell lymphomas in 2022.
New data presented at ASH: Gamida Cell presented 2-year survival and correlation data with cytokine IL7 at the ASH Annual Meeting and Exposition in December 2021. This analysis provided longer follow-up in the investigator-led study of GDA-201 in patients with non-Hodgkin lymphoma and demonstrated an overall survival rate of 78% at two years with a median duration of response of 16 months.
NAM-Enabled NK Cell Pipeline Expansion
Advanced NAM-enabled genetically modified NK pipeline: Gamida Cell continues to progress its NAM-enabled genetically modified NK pipeline, which utilizes CAR, membrane bound- and CRISPR-mediated technologies to increase targeting, potency and persistence against hematologic malignancies and solid tumors. The company plans to execute preclinical proof of concept studies for these genetically modified NK therapeutic targets and to select a product candidate for IND enabling studies by the end of 2022. These therapeutic targets include:
GDA-301: Knockout of CISH (cytokine inducible SH2 containing protein) in NK cells using CRISPR/Cas9 in combination with a membrane-bound IL-15/IL-15Ra;
GDA-501: anti HER2 CAR-engineered NK cells to target solid tumors expressing HER2, based on a single-chain variable fragment of the widely used humanized monoclonal antibody trastuzumab; and
GDA-601: CRISPR Knockout of CD38 on NK cells combined with anti CD38 CAR. CD38 is an established immunotherapeutic target in multiple myeloma, but its expression on NK cells and its further induction during ex vivo NK cell expansion represents a barrier to the development of an anti CD38 CAR-NK cell therapy. Gamida Cell is advancing this program with a collaboration with the Dana-Farber Cancer Institute to study the in vitro cytotoxicity of GDA-601 in fresh samples from multiple myeloma patients.
GDA-401: A development candidate with the target still undisclosed.
Full Year 2021 Financial Results
Research and development expenses were $50.2 million in 2021, compared to $38.9 million in 2020. The increase was primarily due to a $5.4 million increase in omidubicel commercial manufacturing readiness activities and advancing the NK programs, as well as an increase of $5.9 million in broadening the company scientific capabilities and talent.
Commercial expenses in 2021 were $20.0 million, compared to $8.9 million in 2020. The increase was attributable mainly to a $6.5 million increase in commercial readiness expenses and a $4.6 million increase in headcount within the commercial organization. Going forward, the company anticipates reducing its near-term commercial readiness expenses, as it is assessing alternatives for the commercialization of omidubicel, including potential U.S. or global partnerships.
General and administrative expenses were $17.0 million in 2021, compared to $13.2 million in 2020. The increase was mainly due to a $2.6 million increase in professional services expenses and a $1.2 million increase in headcount and related expenses.
Finance expenses, net, were $2.6 million for 2021, compared to $0.6 million for 2020. The increase was primarily due to a $4.4 million interest expenses from convertible notes, offset by a $2.1 million capitalization and other non-cash expenses, and a $0.3 million increase in interest income from cash management.
Net loss for 2021 was $89.8 million, compared to a net loss of $61.6 million in 2020.
2022 Financial Guidance
Gamida Cell expects cash used for ongoing operating activities in 2022 to range from $60 million to $70 million.
Gamida Cell expects that its current cash and cash equivalents will support the company’s ongoing operating activities into mid 2023. This cash runaway guidance is based on the company’s current operational plans and excludes any additional funding and any business development activities that may be undertaken.
Expected Milestones in 2022
Omidubicel
Completion of full BLA submission to the FDA in the first half of 2022
GDA-201
Initiation of a company-sponsored Phase 1/2 clinical study in follicular and diffuse large B-cell lymphomas
NK cell pipeline expansion
Establish preclinical proof of concept studies of the NAM-enabled, genetically modified NK therapeutic targets
Select pipeline candidate for IND-enabling studies
Conference Call Information
Gamida Cell will host a conference call today, March 15, 2022, at 8:00 a.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com. To participate in the live call, please dial 866-930-5560 (domestic) or 409-216-0605 (international) and refer to conference ID number 1696742. A recording of the webcast will be available approximately two hours after the event, for approximately 30 days.
Gamida Cell to Present Corporate Highlights at the Oppenheimer 32nd Annual Healthcare Conference
https://finance.yahoo.com/news/gamida-cell-present-corporate-highlights-210000499.html
BOSTON, March 10, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious blood diseases, today announced that company management will present its corporate highlights at the upcoming Oppenheimer 32nd Annual Healthcare Conference on March 16, 2022 at 2:00 p.m. ET.
The webcast will be available on the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com, and will be available for at least 14 days following the event.
Gamida Cell Announces the Date of Its Fourth Quarter and Full Year 2021 Financial Results and Webcast
https://finance.yahoo.com/news/gamida-cell-announces-date-fourth-130000729.html
Tue, March 8, 2022, 3:00 PM
BOSTON, March 08, 2022--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for blood cancers and serious blood diseases, today announced that the company will host a conference call and live audio webcast on Tuesday, March 15, 2022, at 8:00 a.m. ET to review its fourth quarter and full year 2021 financial results and provide an update on the company.
The webcast will be available on the "Investors & Media" section of the Gamida Cell website at www.gamida-cell.com. To participate in the live call, please dial 866-930-5560 (domestic) or 409-216-0605 (international) and refer to conference ID number 1696742. A replay of the webcast will be available approximately two hours after the event, for approximately 30 days.
3.6900+0.3300 (+9.8214%)
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Nice! Robust volume too!
Gamida Cell surges 22% following rolling BLA submission for omidubicel
Feb. 09, 2022 3:05 PM ETGamida Cell Ltd. (GMDA)
By: Jonathan Block, SA News Editor
Shares of Gamida Cell (GMDA +22.3%) have surged higher after the company today said it started a rolling submission of a Biologics License Application ("BLA") for omidubicel, a treatment for patients with blood cancers in need of stem cell transplant.
The company is aiming to complete the submission by Q2.
Although he rates Gamida Cell as a hold, Seeking Alpha contributor Bret Jensen says the company has a favorable risk/reward profile.
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