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Thank you rwwine!
I am pretty sure sp will increase, although some bumps
will still occur down the road.
It seems to me that $GMDA management are adopting the
AVIS slogan=We (now) try harder!
Enjoy your weekend!
No big difference. Dead money.
A great pity!
Vericel Announces Publication of Positive Results from Phase 3 Debride and Protect (DETECT) Study of Thermal Burn Patients Treated with NexoBrid
https://finance.yahoo.com/news/vericel-announces-publication-positive-results-123000746.html
Data published in the Journal of Burn Care & Research show treatment with NexoBrid resulted in early complete eschar removal in more than 90% of treated patients, and reduced surgery when compared to Gel Vehicle and standard of care
Results demonstrate that NexoBrid is safe and well-tolerated without deleterious effects on wound closure and scarring
CAMBRIDGE, Mass., Sept. 28, 2023 (GLOBE NEWSWIRE) -- Vericel Corporation (NASDAQ:VCEL), a leader in advanced therapies for the sports medicine and severe burn care markets, today announced the recent publication of results from the Phase 3 DETECT study assessing the safety and efficacy of NexoBrid® (anacaulase-bcdb) in the Journal of Burn Care & Research.
“These data demonstrate the ability of NexoBrid to provide patients with severe thermal burns safe and effective enzymatic eschar removal that is superior to current debridement methods,” said Dr. Jon Hopper, Chief Medical Officer of Vericel. “We are pleased that NexoBrid is now available to U.S. burn surgeons to treat severe burn patients and we believe the publication of these results, coupled with real-world clinical experience, will help establish NexoBrid as the new standard of care for eschar removal.”
The three-arm DETECT study consisted of 175 adult patients with deep thermal burns covering 3-30% of total body surface area, who were randomized to NexoBrid, surgical or non-surgical standard of care (SOC), or placebo Gel Vehicle (GV) treatment arms in a 3:3:1 ratio. The primary endpoint was complete eschar removal at the end of the debridement phase and secondary outcomes were the need for surgery and time to complete eschar removal. Key findings include:
More than 93% of the patients treated with NexoBrid achieved complete eschar removal following one application of NexoBrid compared with 4% in the GV arm (P<0.0001);
Surgical excision was lower in the NexoBrid arm when compared to the SOC group (4% vs. 72%; P<0.001);
The estimated median time to complete eschar removal was 1.02 and 3.83 days for the NexoBrid and SOC treatment arms, respectively (P<0.0001); and
NexoBrid appeared safe and well tolerated without deleterious effects on wound closure and scarring.
Data from this study ultimately served as the foundation for the FDA approval of NexoBrid for commercial use in the U.S.
About NexoBrid
NexoBrid (anacaulase-bcdb) is a botanical drug product containing proteolytic enzymes indicated for the removal of eschar in adults with deep partial- and/or full-thickness thermal burns. To learn more about NexoBrid, please visit www.NexoBrid-US.com.
About Vericel Corporation
Vericel is a leader in advanced therapies for sports medicine and severe burn care. The Company manufactures and markets two cell therapy products and one specialty biologic product in the United States. MACI® (autologous cultured chondrocytes on porcine collagen membrane) is an autologous cellularized scaffold product indicated for the repair of symptomatic, single or multiple full-thickness cartilage defects of the knee with or without bone involvement in adults. Epicel® (cultured epidermal autografts) is a permanent skin replacement for the treatment of patients with deep dermal or full thickness burns greater than or equal to 30% of total body surface area. Vericel also holds an exclusive license for North American rights to NexoBrid® (anacaulase-bcdb), a biological orphan product containing proteolytic enzymes, which is indicated for the removal of eschar in adults with deep partial-thickness and/or full-thickness burns. For more information, please visit www.vcel.com.
Epicel® and MACI® are registered trademarks of Vericel Corporation. NexoBrid® is a registered trademark of MediWound Ltd. and is used under license to Vericel Corporation. © 2023 Vericel Corporation. All rights reserved.
Forward Looking Statements
This press release contains forward-looking statements. Forward-looking statements are subject to risks and uncertainties such as those described in Vericel’s periodic reports on file with the Securities and Exchange Commission. Actual results may differ materially from anticipated results.
Investor Contact:
Eric Burns
ir@vcel.com
+1 (734) 418-4411
Media Contact:
Julie Downs
jdowns@vcel.com
Gamida Cell to Present at Society for Immunotherapy of Cancer's (SITC) 38th Annual Meeting
https://finance.yahoo.com/news/gamida-cell-present-society-immunotherapy-120000968.html
BOSTON, September 28, 2023--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), a cell therapy pioneer working to turn cells into powerful therapeutics, today announced that it will be presenting at the Society for Immunotherapy of Cancer’s 38th Annual Meeting (SITC 2023) taking place in San Diego, CA and virtually November 1-5, 2023.
Details about the poster presentations are as follows:
Title: GDA-201, nicotinamide (NAM) expanded NK cells derived from peripheral apheresis, show unique culture kinetics and increased expansion
Abstract number: 401
Presenter: Avner Yeffet, Ph.D.
Time: Friday, November 3, 2023, 9:00 a.m. – 7:00 p.m. PDT
Location: Exhibit Halls A and B1
Title: Significant myeloid and dendritic cellular enrichment of omidubicel graft suggests fast homeostatic proliferation of lymphoid populations
Abstract number: 336
Presenter: Dima Yackoubov
Time: Saturday, November 4, 2023, 9:00 a.m. – 8:30 p.m. PDT
Location: Exhibit Halls A and B1
About GDA-201
GDA-201 is an intrinsic NK cell therapy candidate being investigated for the treatment of hematologic malignancies. A multicenter Phase 1 study of GDA-201 for the treatment of non-Hodgkin lymphoma is ongoing (NCT05296525). Results are expected in Q1 2024.
GDA-201 is an investigational cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority.
Omisirge™ (omidubicel-onlv) Indication
Omisirge is a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from cord blood indicated for use in adults and pediatric patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.
Important Safety Information for Omisirge
BOXED WARNING: INFUSION REACTIONS, GRAFT VERSUS HOST DISEASE, ENGRAFTMENT SYNDROME, AND GRAFT FAILURE
Infusion reactions may be fatal. Monitor patients during infusion and discontinue for severe reactions. Use is contraindicated in patients with known allergy to dimethyl sulfoxide (DMSO), Dextran 40, gentamicin, human serum albumin or bovine material.
Graft-versus-Host Disease may be fatal. Administration of immunosuppressive therapy may decrease the risk of GvHD.
Engraftment syndrome may be fatal. Treat engraftment syndrome promptly with corticosteroids.
Graft failure may be fatal. Monitor patients for laboratory evidence of hematopoietic recovery.
Contraindications
OMISIRGE is contraindicated in patients with known hypersensitivity to dimethyl sulfoxide (DMSO), Dextran 40, gentamicin, human serum albumin, or bovine products.
Warnings and Precautions
Hypersensitivity Reactions
Allergic reactions may occur with the infusion of OMISIRGE. Reactions include bronchospasm, wheezing, angioedema, pruritis and hives. Serious hypersensitivity reactions, including anaphylaxis, may be due to DMSO, residual gentamicin, Dextran 40, human serum albumin (HSA) and bovine material in OMISIRGE. OMISIRGE may contain residual antibiotics if the cord blood donor was exposed to antibiotics in utero. Patients with a history of allergic reactions to antibiotics should be monitored for allergic reactions following OMISIRGE administration.
Infusion Reactions
Infusion reactions occurred following OMISIRGE infusion, including hypertension, mucosal inflammation, dysphagia, dyspnea, vomiting, and gastrointestinal toxicity. Premedication with antipyretics, histamine antagonists, and corticosteroids may reduce the incidence and intensity of infusion reactions. In patients transplanted with OMISIRGE in clinical trials, 47% (55/117) patients had an infusion reaction of any severity. Grade 3-4 infusion reactions were reported in 15% (18/117) patients. Infusion reactions may begin within minutes of the start of infusion of OMISIRGE, although symptoms may continue to intensify and not peak for several hours after the completion of the infusion. Monitor patients for signs and symptoms of infusion reactions during and after OMISIRGE administration. When a reaction occurs, pause the infusion and institute supportive care as needed.
Graft-versus-Host Disease
Acute and chronic GvHD, including life-threatening and fatal cases, occurred following treatment with OMISIRGE. In patients transplanted with OMISIRGE Grade II-IV acute GvHD was reported in 58% (68/117). Grade III-IV acute GvHD was reported in 17% (20/117). Chronic GvHD occurred in 35% (41/117) of patients. Acute GvHD manifests as maculopapular rash, gastrointestinal symptoms, and elevated bilirubin. Patients treated with OMISIRGE should receive immunosuppressive drugs to decrease the risk of GvHD, be monitored for signs and symptoms of GvHD, and treated if GvHD develops.
Engraftment Syndrome
Engraftment syndrome may occur because OMISIRGE is derived from umbilical cord blood. Monitor patients for unexplained fever, rash, hypoxemia, weight gain, and pulmonary infiltrates in the peri-engraftment period. Treat with corticosteroids as soon as engraftment syndrome is recognized to ameliorate symptoms. If untreated, engraftment syndrome may progress to multiorgan failure and death.
Graft Failure
Primary graft failure occurred in 3% (4/117) of patients in OMISIRGE clinical trials. Primary graft failure, which may be fatal, is defined as failure to achieve an absolute neutrophil count greater than 500 per microliter blood by Day 42 after transplantation. Immunologic rejection is the primary cause of graft failure. Monitor patients for laboratory evidence of hematopoietic recovery.
Malignancies of Donor Origin
Two patients treated with OMISIRGE developed post-transplant lymphoproliferative disorder (PTLD) in the second-year post-transplant. PTLD manifests as a lymphoma-like disease favoring non-nodal sites. PTLD is usually fatal if not treated. The etiology is thought to be donor lymphoid cells transformed by Epstein-Barr virus (EBV). Serial monitoring of blood for EBV DNA may be warranted in patients with persistent cytopenias. One patient treated with OMISIRGE developed a donor-cell derived myelodysplastic syndrome (MDS) during the fourth-year post-transplant. The natural history is presumed to be the same as that for de novo MDS. Monitor life-long for secondary malignancies. If a secondary malignancy occurs, contact Gamida Cell at (844) 477-7478.
Transmission of Serious Infections
Transmission of infectious disease may occur because OMISIRGE is derived from umbilical cord blood. Disease may be caused by known or unknown infectious agents. Donors are screened for increased risk of infection, clinical evidence of sepsis, and communicable disease risks associated with xenotransplantation. Maternal and infant donor blood is tested for evidence of donor infection. See full Prescribing Information, Warnings and Precautions, Transmission of Serious Infections for list of testing performed. OMISIRGE is tested for sterility, endotoxin, and mycoplasma. There may be an effect on the reliability of the sterility test results if the cord blood donor was exposed to antibiotics in utero. Product manufacturing includes bovine-derived reagents. All animal-derived reagents are tested for animal viruses, bacteria, fungi, and mycoplasma before use. These measures do not eliminate the risk of transmitting these or other transmissible infectious diseases and disease agents. Test results may be found on the container label and/or in accompanying records. If final sterility results are not available at the time of use, Quality Assurance will communicate any positive results from sterility testing to the physician. Report the occurrence of transmitted infection to Gamida Cell at (844) 477-7478.
Transmission of Rare Genetic Diseases
OMISIRGE may transmit rare genetic diseases involving the hematopoietic system because it is derived from umbilical cord blood. Cord blood donors have been screened to exclude donors with sickle cell anemia, and anemias due to abnormalities in hemoglobins C, D, and E. Because of the age of the donor at the time cord blood collection takes place, the ability to exclude rare genetic diseases is severely limited.
ADVERSE REACTIONS
The most common adverse reactions (incidence > 20%) are infections, GvHD, and infusion reaction.
Please see full Prescribing Information, including Boxed Warning.
About Gamida Cell
Gamida Cell is a cell therapy pioneer working to turn cells into powerful therapeutics. The company’s proprietary nicotinamide (NAM) technology leverages the properties of NAM to enhance and expand cells, creating allogeneic cell therapy products and candidates that are potentially curative for patients with hematologic malignancies. These include Omisirge™ (omidubicel-onlv), an FDA-approved nicotinamide modified allogeneic hematopoietic progenitor cell therapy, and GDA-201, an intrinsic NK cell therapy candidate being investigated for the treatment of hematologic malignancies. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, X, Facebook or Instagram.
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 the potentially life-saving or curative therapeutic and commercial potential of Omisirge™ (omidubicel-onlv), and the Company’s cell therapy candidate, GDA-201. 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 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. 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 August 14, 2023, and other filings that Gamida Cell makes with the SEC from time to time (which are available at 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.
OMISIRGE™ is a trademark of Gamida Cell Inc.
© 2023 Gamida Cell Inc. All Rights Reserved
View source version on businesswire.com: https://www.businesswire.com/news/home/20230927063152/en/
Contacts
Media Contact:
Dan Boyle
Orangefiery
media@orangefiery.com
1-818-209-1692
Investor Contact:
Chuck Padala
LifeSci Advisors
Chuck@lifesciadvisors.com
1-646-627-8390
PR Newswire
BioLineRx Announces Encouraging Data from Pilot Phase of Phase 2 Combination Clinical Trial with Motixafortide in First-Line Pancreatic Cancer (PDAC)
https://finance.yahoo.com/news/biolinerx-announces-encouraging-data-pilot-110000312.html
- 6 of 11 Patients in the Pilot Phase Experienced a Partial Response, with 4 Confirmed; One Patient Experienced Resolution of the Hepatic Metastatic Lesion -
- Data Presented at AACR Special Conference on Pancreatic Cancer -
- Multi-Center, Randomized Phase 2 Study Currently Enrolling -
TEL AVIV, Israel, Sept. 28, 2023 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today announced encouraging data from the single-arm pilot phase of the investigator-initiated CheMo4METPANC Phase 2 combination clinical trial evaluating the company's CXCR4 inhibitor motixafortide, the PD-1 inhibitor cemiplimab, and standard of care chemotherapies gemcitabine and nab-paclitaxel, versus gemcitabine and nab-paclitaxel alone, in first-line pancreatic cancer (PDAC).
The data were published in an online abstract as part of the American Association of Cancer Research (AACR) Special Conference on Pancreatic Cancer taking place in Boston, Massachusetts from September 27-30, 2023. An oral presentation of the data will take place later today, September 28, 2023.
The pilot phase of the Phase 2 study enrolled 11 patients with metastatic pancreatic cancer. As of May 2023, 6 patients (55%) experienced a partial response (PR) of which 4 (36%) were confirmed PRs with one patient experiencing resolution of the hepatic (liver) metastatic lesion. Three patients (27%) experienced stable disease, resulting in a disease control rate of 82%. These findings compare favorably to historic partial response and disease control rates of 23% and 48%, respectively, reported with the current standard of care, the chemotherapy combination gemcitabine and nab-paclitaxel.
"These initial data from the pilot phase of this ongoing Phase 2 study give us hope that motixafortide could potentially serve as the backbone of a new treatment regimen for PDAC, which is among the most difficult cancers to treat," said Philip Serlin, Chief Executive Officer of BioLineRx Ltd. "We are deeply committed to this important collaboration with Columbia University investigators and eagerly look forward to the data from the randomized phase of the trial."
Based on these pilot data, earlier this year, the CheMo4METPANC Phase 2 trial was amended to become a randomized study, with planned enrollment increasing from 30 to 102 patients. The trial, sponsored by Columbia University, is the first large, multi-center, randomized study evaluating motixafortide with a PD-1 inhibitor and first-line PDAC chemotherapies. A poster of the amended clinical trial design was presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting, held June 2-6 in Chicago, Illinois (see abstract).
Pancreatic ductal adenocarcinoma (PDAC) is the most common type of pancreatic cancer and is expected to be the second leading cause of cancer-related death in the U.S. by 2023. Because it is typically diagnosed at later stages, greater than 80 percent of pancreatic cancer is inoperable. Most pancreatic cancer is incurable and unfortunately newer immunotherapy approaches, while beneficial against other solid tumor types, have had limited efficacy in pancreatic cancer due to immunosuppressive pathways.
An earlier single-arm, Phase 2a clinical trial (COMBAT/KEYNOTE-202) and pre-clinical studies evaluating motixafortide in combination with PD-1 immunotherapies and chemotherapies in PDAC have been promising, suggesting the ability of motixafortide to support an immune response.
Presentation at AACR Special Conference in Cancer Research: Pancreatic Cancer
Westin Copley Place, Boston Massachusetts
Plenary Session Details
Title: CheMo4METPANC: Combination Chemotherapy (gemcitabine and nab-paclitaxel), chemokine (C-X-C) Motif receptor 4 inhibitor (motixafortide), and immune checkpoint blockade (cemiplimab) in METastatic treatment-naïve PANCreatic adenocarcinoma
Presenter: Gulam A. Manji, MD, PhD, Columbia University Irving Medical Center/New York Presbyterian, New York, N.Y.
Session: Plenary Session 3: Clinical Updates
Date: Thursday, September 28, 2023
Time: 2:30-4:40 pm EDT
About CheMo4METPANC Phase 2 Clinical Trial
The multi-center CheMo4METPANC Phase 2 clinical trial is a randomized, investigator-initiated clinical trial in first line metastatic pancreatic cancer. Sponsored by Columbia University, the study is evaluating the combination of CXCR4 inhibitor motixafortide, PD-1 inhibitor cemiplimab, and standard of care chemotherapies gemcitabine and nab-paclitaxel, versus gemcitabine and nab-paclitaxel, alone in 102 patients. The trial's primary endpoint is progression free survival (PFS). Secondary objectives include safety, response rate, disease control rate, duration of clinical benefit and overall survival.
About Pancreatic Cancer
Pancreatic cancer has a low rate of early diagnosis and a poor prognosis. In the United States in 2023, an estimated 64,000 adults will be diagnosed with the disease, which accounts for approximately 3% of all cancers in the U.S. and about 7% of all cancer deaths. Worldwide, an estimated 496,000 people were diagnosed with the disease in 2020. In the U.S., if the cancer is detected at an early stage when surgical removal of the tumor is possible, the 5-year relative survival rate is 44%. About 12% of people are initially diagnosed at this stage. If the cancer has spread to surrounding tissues or organs, the 5-year relative survival rate is 15%. For the 52% of patients who are initially diagnosed with metastatic cancer, the 5-year relative survival rate is 3%. In particular, hepatic (liver) metastases are a critical risk factor driving poor prognoses for patients with metastatic PDAC. These data highlight the need for the development of new therapeutic options.
About Motixafortide in Cancer Immunotherapy
Motixafortide inhibits CXCR4, a chemokine receptor and a well validated therapeutic target that is over-expressed in many human cancers including pancreatic ductal adenocarcinoma (PDAC). Motixafortide leverages the expression of the CXCR4 receptor on different immune cells and potentiates the immune system against the tumor. Among CXCR4-expressing immune cells, some exhibit anti-tumoral activity, such as effector T cells and some exhibit pro-tumoral activity and support tumor growth. By blocking the CXCR4 receptor, motixafortide was shown in a Phase 2 study in pancreatic cancer patients to enhance anti-tumoral activity and to ameliorate the pro-tumoral activities by modulating the effector/suppressor cell ratio towards a proinflammatory profile.
About BioLineRx
BioLineRx Ltd. (NASDAQ/TASE: BLRX) is a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases. The company's first approved product is APHEXDA™ (motixafortide) with an indication in the U.S. for stem cell mobilization for autologous transplantation in multiple myeloma. BioLineRx is advancing a pipeline of investigational medicines for patients with sickle cell disease, pancreatic cancer, and other solid tumors. Headquartered in Israel, and with operations in the U.S., the company is driving innovative therapeutics with end-to-end expertise in development and commercialization, ensuring life-changing discoveries move beyond the bench to the bedside.
Learn more about who we are, what we do, and how we do it at www.biolinerx.com, or on Twitter and LinkedIn.
Forward Looking Statement
Various statements in this release concerning BioLineRx's future expectations constitute "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include words such as "anticipates," "believes," "could," "estimates," "expects," "intends," "may," "plans," "potential," "predicts," "projects," "should," "will," and "would," and describe opinions about future events. These include statements regarding
management's expectations, beliefs and intentions regarding, among other things, the potential benefits of APHEXDA, the timing and execution of the launch of APHEXDA and the plans and objectives of management for future operations and expectations and commercial potential of motixafortide, as well as its potential investigational uses. These forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause the actual results, performance or achievements of BioLineRx to be materially different from any future results, performance or achievements expressed or implied by such forward-looking statements. Factors that could cause BioLineRx's actual results to differ materially from those expressed or implied in such forward-looking statements include, but are not limited to: the initiation, timing, progress and results of BioLineRx's preclinical studies, clinical trials and other therapeutic candidate development efforts; BioLineRx's ability to advance its therapeutic candidates into clinical trials or to successfully complete its preclinical studies or clinical trials; whether the clinical trial results for APHEXDA will be predictive of real-world results; BioLineRx's receipt of regulatory approvals for its therapeutic candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of BioLineRx's therapeutic candidates, including the degree and pace of market uptake of APHEXDA for the mobilization of hematopoietic stem cells for autologous transplantation in multiple myeloma patients; whether access to APHEXDA is achieved in a commercially viable manner and whether APHEXDA receives adequate reimbursement from third-party payors; BioLineRx's ability to establish and maintain corporate collaborations; BioLineRx's ability to integrate new therapeutic candidates and new personnel; the interpretation of the properties and characteristics of BioLineRx's therapeutic candidates and of the results obtained with its therapeutic candidates in preclinical studies or clinical trials; the implementation of BioLineRx's business model and strategic plans for its business and therapeutic candidates; the scope of protection BioLineRx is able to establish and maintain for intellectual property rights covering its therapeutic candidates and its ability to operate its business without infringing the intellectual property rights of others; estimates of BioLineRx's expenses, future revenues, capital requirements and its needs for and ability to access sufficient additional financing, including any unexpected costs or delays in the commercial launch of APHEXDA; risks related to changes in healthcare laws, rules and regulations in the United States or elsewhere; competitive companies, technologies and BioLineRx's industry; statements as to the impact of the political and security situation in Israel on BioLineRx's business; and the impact of the COVID-19 pandemic and the Russian invasion of Ukraine, which may exacerbate the magnitude of the factors discussed above. These and other factors are more fully discussed in the "Risk Factors" section of BioLineRx's most recent annual report on Form 20-F filed with the Securities and Exchange Commission on March 22, 2023. In addition, any forward-looking statements represent BioLineRx's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. BioLineRx does not assume any obligation to update any forward-looking statements unless required by law.
Contacts:
United States
John Lacey
BioLineRx
IR@biolinerx.com
Israel
Moran Meir
LifeSci Advisors, LLC
moran@lifesciadvisors.com
ASCO Cancer.Net. Cancer.Net Editorial Board Approval March 2023.
Cision
Cision
View original content:https://www.prnewswire.com/news-releases/biolinerx-announces-encouraging-data-from-pilot-phase-of-phase-2-combination-clinical-trial-with-motixafortide-in-first-line-pancreatic-cancer-pdac-301941126.html
SOURCE BioLineRx Ltd
Business Wire
First Patient Receives Gam
Curtains down as was expected. Poor ALS families,
hopes shattered!
Miracles have indeed occured in this part of the world,
however it occurred many many years ago.
Zero chance of FDA approval in spite of the ALS lobby!
BrainStorm Cell Therapeutics Provides Update on FDA Advisory Committee Meeting to Review NurOwn for the Treatment of ALS
https://finance.yahoo.com/news/brainstorm-cell-therapeutics-provides-fda-024800252.html
NEW YORK, Sept. 27, 2023 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced the outcome of the U.S. Food and Drug Administration's Cellular, Tissue and Gene Therapies Advisory Committee meeting to review the Biologics License Application (BLA) for NurOwn®, an investigational mesenchymal stem cell therapy for the treatment of amyotrophic lateral sclerosis (ALS).
Today the Committee voted that NurOwn did not demonstrate substantial evidence of effectiveness for treatment of mild to moderate ALS.
"The Committee's vote was a sad outcome for the ALS community, who have too few options to help manage this merciless and deadly disease," said Stacy Lindborg, PhD, co-CEO of BrainStorm. "We firmly believe that the totality of data presented for NurOwn today provide a compelling case for approval, with clinical evidence in those with less advanced disease supported by strong and consistent biomarker data that are predictive of clinical response. We truly did our best to make the NurOwn data clear to the FDA Advisory Committee. Unfortunately, had more time and opportunity been allowed, many remaining questions posed by Advisory Committee members could have been sufficiently addressed."
Chaim Lebovits, President and Chief Executive Officer of BrainStorm, added: "The discussion in today's Advisory Committee meeting, and the heartrending testimony of those living with ALS and their loved ones, underscores not only the need for regulatory flexibility but also for continuing research in the field. The people of BrainStorm will do everything in our power to fulfill the obligation we deeply feel we owe to the ALS community, and in the coming weeks we will explore all options available to us."
About NurOwn®
The NurOwn® technology platform (autologous MSC-NTF cells) represents a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are harvested from each person with ALS and are manufactured using an innovative and proprietary process to secrete neurotrophic factors to target specific neurodegenerative diseases. The lead program for NurOwn is for the treatment of ALS, which is under FDA review. BrainStorm's long-term commitment to ALS is demonstrated in preclinical research and a series of clinical studies, all of which have been published in peer-reviewed journals.
The Phase 3 pivotal trial of NurOwn did not reach statistical significance on the primary or secondary endpoints, likely due to a "floor effect," which confounds measurement of disease progression in patients with more advanced disease. A thorough analysis of NurOwn Phase 3 data shows evidence of clinically meaningful effectiveness in ALS participants who have not progressed to advanced levels of disease progression. In a pre-specified group of participants with an ALSFRS-R score ³35, there was a larger treatment effect across all endpoints with NurOwn compared to placebo, which aligned with historical trials and the study power assumptions and resulted in a statistically significant difference on a key endpoint (change from baseline in ALSFRS-R). Additionally, a post-hoc sensitivity analysis of patients across threshold of >26 through =35 on the ALSFRS-R highlighted that NurOwn-treated patients retain, on average, two points of function more compared to placebo–clinically meaningful preservation and important for quality of life for a person living with ALS and their loved ones.
NurOwn's clinical program also included most robust cerebrospinal fluid (CSF) biomarker study ever done in ALS, strong and consistent biomarker data, which are predictive of clinical response in the trial, span pathways that are important to ALS (neuroinflammation, neurodegeneration, neuroprotection), and align with NurOwn's mechanism of action. Biomarker data in all trial participants showed consistent biological patterns of NurOwn reducing markers of inflammation and neurodegeneration and increasing neuroprotective markers relative to placebo. Biomarker patterns were consistent across all NurOwn-participants, including in those with Advanced ALS disease where clinical scales, such as the ALS Functional Rating Scale, have demonstrated measurement challenges. Three CSF biomarkers were predictive of clinical outcomes in NurOwn-treated participants–NfL, galectin-1, latency associated peptide of TGF-beta1 (LAP or TGF-b).
The NurOwn clinical program has generated valuable insights into the pathology of ALS, as well as disease progression and treatment. Since the initial Phase 3 readout, BrainStorm has shared the full dataset through rigorous peer-reviewed analysis, including: quantification of Floor Effect, which had been noted but never before explored in depth; evaluation of multiple pre-specified biomarkers, collected at seven different points across 20 weeks during the trial, allowing a longitudinal view; and analysis of genetic data, which represents one of the first ALS trials to prospectively invoke pharmacogenomic analysis of clinical outcome, offering great promise for the development of future treatments for ALS.
BrainStorm previously announced the FDA's intention to hold an ADCOM meeting to review NurOwn for the treatment of ALS. BrainStorm filed a BLA for NurOwn on September 9, 2022, and received a Refusal to File (RTF) letter from FDA on November 8, 2022. Following a Type A meeting and subsequent discussions with the FDA BrainStorm requested that CBER utilize the FDA's "File Over Protest" procedure, which offers the shortest amount of time to complete the regulatory process. The BLA was filed over protest, allowing completion of the regulatory process in the shortest time possible, and active review resumed on February 7, 2023.
About BrainStorm Cell Therapeutics Inc.
BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. BrainStorm holds the rights to clinical development and commercialization of the NurOwn® technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug designation status from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm has completed a Phase 3 pivotal trial in ALS (NCT03280056); this trial investigated the safety and efficacy of repeat-administration of autologous MSC-NTF cells and was supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). BrainStorm completed under an investigational new drug application a Phase 2 open-label multicenter trial (NCT03799718) of autologous MSC-NTF cells in progressive MS and was supported by a grant from the National MS Society (NMSS).
Notice Regarding Forward-Looking Statements
This press release contains "forward-looking statements" that are subject to substantial risks and uncertainties, including statements regarding the upcoming ADCOM meeting related to NurOwn, the timing of a PDUFA action date for the BLA for NurOwn, the clinical development of NurOwn as a therapy for the treatment of ALS, the future availability of NurOwn to patients, and the future success of BrainStorm. All statements, other than statements of historical fact, contained in this press release are forward-looking statements. Forward-looking statements contained in this press release may be identified by the use of words such as "anticipate," "believe," "contemplate," "could," "estimate," "expect," "intend," "seek," "may," "might," "plan," "potential," "predict," "project," "target," "aim," "should," "will" "would," or the negative of these words or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements are based on BrainStorm's current expectations and are subject to inherent uncertainties, risks and assumptions that are difficult to predict. These potential risks and uncertainties include, without limitation, management's ability to successfully achieve its goals, BrainStorm's ability to raise additional capital,
BrainStorm's ability to continue as a going concern, prospects for future regulatory approval of NurOwn, whether BrainStorm's future interactions with the FDA will have productive outcomes, and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations, and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance, or achievements.
CONTACTS
Investor Relations:
John Mullaly
LifeSci Advisors, LLC
Phone: +1 617-429-3548
jmullaly@lifesciadvisors.com
Media:
Lisa Guiterman
Phone: +1 202-330-3431
lisa.guiterman@gmail.com
Cision
Cision
View original content:https://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-provides-update-on-fda-advisory-committee-meeting-to-review-nurown-for-the-treatment-of-als-301941169.html
SOURCE BrainStorm Cell Therapeutics Inc.
First Patient Receives Gamida Cell's Omisirge™ (omidubicel-onlv)
https://finance.yahoo.com/news/first-patient-receives-gamida-cells-203000857.html
Omisirge is the only allogeneic stem cell therapy approved by the U.S. Food and Drug Administration (FDA) on the basis of a global randomized Phase 3 trial
Rapid onboarding of transplant centers and significant payer coverage demonstrate strong launch progress
BOSTON, September 27, 2023--(BUSINESS WIRE)--Gamida Cell Ltd. (Nasdaq: GMDA), a cell therapy pioneer working to turn cells into powerful therapeutics, today announced that the first patient has received a stem cell transplant with Omisirge (omidubicel-onlv).
"This is a significant milestone for Gamida Cell, advancing our mission of delivering potentially curative therapies to patients with cancer," said Abbey Jenkins, President and Chief Executive Officer of Gamida Cell. "This patient will be the first of many who have new hope for a cure thanks to the availability of Omisirge as a new stem cell transplant donor source. This is why we do the work that we do – to make a difference for people with cancer."
Omisirge was approved by the U.S. FDA in April 2023 for use in adults and pediatric patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.
Gamida Cell has already exceeded its 2023 launch goals, with 15 transplant centers onboarded across the United States and confirmed coverage with payers that cover 90% of commercial lives. Gamida Cell is actively engaged with more than 90% of the top 70 transplant centers, which conduct approximately 80% of transplants. An increasing number of patients are being enrolled in Gamida Cell Assist®, which signals a transplanter’s intent to use Omisirge as the donor source.
"The launch of Omisirge is progressing very well in terms of payer coverage, transplant center onboarding and transplanter interest in using Omisirge as a donor source," said Michele Korfin, Chief Operating and Chief Commercial Officer of Gamida Cell. "We recognize the importance of making sure eligible patients can access Omisirge. To appropriately manage our cash, we launched with a limited investment and field footprint. The positive launch progress and strong interest from transplant centers now warrant expanding that investment and the team from four to eight account managers by the start of 2024. We are encouraged by transplanter feedback that Omisirge may both increase the number of patients able to access an appropriate donor source and address some limitations of other donor sources."
Approximately 8,000 stem cell transplants are performed in the U.S. each year in patients with hematologic malignancies1 and another 1,700 patients are estimated to be eligible for transplant but unable to find a donor.2 The ability to find a donor is historically more challenging for racially and ethnically diverse populations than for patients who are white.3 Gamida Cell market analyses indicate that Omisirge has the ability to capture approximately 20% of allogeneic stem cell transplant market share by ~2028.
Omisirge Indication
Omisirge is a nicotinamide modified allogeneic hematopoietic progenitor cell therapy derived from cord blood indicated for use in adults and pediatric patients 12 years and older with hematologic malignancies who are planned for umbilical cord blood transplantation following myeloablative conditioning to reduce the time to neutrophil recovery and the incidence of infection.
Important Safety Information for Omisirge
BOXED WARNING: INFUSION REACTIONS, GRAFT VERSUS HOST DISEASE, ENGRAFTMENT SYNDROME, AND GRAFT FAILURE
Infusion reactions may be fatal. Monitor patients during infusion and discontinue for severe reactions. Use is contraindicated in patients with known allergy to dimethyl sulfoxide (DMSO), Dextran 40, gentamicin, human serum albumin or bovine material.
Graft-versus-Host Disease may be fatal. Administration of immunosuppressive therapy may decrease the risk of GvHD.
Engraftment syndrome may be fatal. Treat engraftment syndrome promptly with corticosteroids.
Graft failure may be fatal. Monitor patients for laboratory evidence of hematopoietic recovery.
Contraindications
OMISIRGE is contraindicated in patients with known hypersensitivity to dimethyl sulfoxide (DMSO), Dextran 40, gentamicin, human serum albumin, or bovine products.
Warnings and Precautions
Hypersensitivity Reactions
Allergic reactions may occur with the infusion of OMISIRGE. Reactions include bronchospasm, wheezing, angioedema, pruritis and hives. Serious hypersensitivity reactions, including anaphylaxis, may be due to DMSO, residual gentamicin, Dextran 40, human serum albumin (HSA) and bovine material in OMISIRGE. OMISIRGE may contain residual antibiotics if the cord blood donor was exposed to antibiotics in utero. Patients with a history of allergic reactions to antibiotics should be monitored for allergic reactions following OMISIRGE administration.
Infusion Reactions
Infusion reactions occurred following OMISIRGE infusion, including hypertension, mucosal inflammation, dysphagia, dyspnea, vomiting, and gastrointestinal toxicity. Premedication with antipyretics, histamine antagonists, and corticosteroids may reduce the incidence and intensity of infusion reactions. In patients transplanted with OMISIRGE in clinical trials, 47% (55/117) patients had an infusion reaction of any severity. Grade 3-4 infusion reactions were reported in 15% (18/117) patients. Infusion reactions may begin within minutes of the start of infusion of OMISIRGE, although symptoms may continue to intensify and not peak for several hours after the completion of the infusion. Monitor patients for signs and symptoms of infusion reactions during and after OMISIRGE administration. When a reaction occurs, pause the infusion and institute supportive care as needed.
Graft-versus-Host Disease
Acute and chronic GvHD, including life-threatening and fatal cases, occurred following treatment with OMISIRGE. In patients transplanted with OMISIRGE Grade II-IV acute GvHD was reported in 58% (68/117). Grade III- IV acute GvHD was reported in 17% (20/117). Chronic GvHD occurred in 35% (41/117) of patients. Acute GvHD manifests as maculopapular rash, gastrointestinal symptoms, and elevated bilirubin. Patients treated with OMISIRGE should receive immunosuppressive drugs to decrease the risk of GvHD, be monitored for signs and symptoms of GvHD, and treated if GvHD develops.
Engraftment Syndrome
Engraftment syndrome may occur because OMISIRGE is derived from umbilical cord blood. Monitor patients for unexplained fever, rash, hypoxemia, weight gain, and pulmonary infiltrates in the peri-engraftment period. Treat with corticosteroids as soon as engraftment syndrome is recognized to ameliorate symptoms. If untreated, engraftment syndrome may progress to multiorgan failure and death.
Graft Failure
Primary graft failure occurred in 3% (4/117) of patients in OMISIRGE clinical trials. Primary graft failure, which may be fatal, is defined as failure to achieve an absolute neutrophil count greater than 500 per microliter blood by Day 42 after transplantation. Immunologic rejection is the primary cause of graft failure. Monitor patients for laboratory evidence of hematopoietic recovery.
Malignancies of Donor Origin
Two patients treated with OMISIRGE developed post-transplant lymphoproliferative disorder (PTLD) in the second-year post-transplant. PTLD manifests as a lymphoma-like disease favoring non-nodal sites. PTLD is usually fatal if not treated. The etiology is thought to be donor lymphoid cells transformed by Epstein-Barr virus (EBV). Serial monitoring of blood for EBV DNA may be warranted in patients with persistent cytopenias. One patient treated with OMISIRGE developed a donor-cell derived myelodysplastic syndrome (MDS) during the fourth-year post-transplant. The natural history is presumed to be the same as that for de novo MDS. Monitor life-long for secondary malignancies. If a secondary malignancy occurs, contact Gamida Cell at (844) 477-7478.
Transmission of Serious Infections
Transmission of infectious disease may occur because OMISIRGE is derived from umbilical cord blood. Disease may be caused by known or unknown infectious agents. Donors are screened for increased risk of infection, clinical evidence of sepsis, and communicable disease risks associated with xenotransplantation. Maternal and infant donor blood is tested for evidence of donor infection. See full Prescribing Information, Warnings and Precautions, Transmission of Serious Infections for list of testing performed. OMISIRGE is tested for sterility, endotoxin, and mycoplasma. There may be an effect on the reliability of the sterility test results if the cord blood donor was exposed to antibiotics in utero. Product manufacturing includes bovine-derived reagents. All animal-derived reagents are tested for animal viruses, bacteria, fungi, and mycoplasma before use. These measures do not eliminate the risk of transmitting these or other transmissible infectious diseases and disease agents. Test results may be found on the container label and/or in accompanying records. If final sterility results are not available at the time of use, Quality Assurance will communicate any positive results from sterility testing to the physician. Report the occurrence of transmitted infection to Gamida Cell at (844) 477-7478.
Transmission of Rare Genetic Diseases
OMISIRGE may transmit rare genetic diseases involving the hematopoietic system because it is derived from umbilical cord blood. Cord blood donors have been screened to exclude donors with sickle cell anemia, and anemias due to abnormalities in hemoglobins C, D, and E. Because of the age of the donor at the time cord blood collection takes place, the ability to exclude rare genetic diseases is severely limited.
ADVERSE REACTIONS
The most common adverse reactions (incidence > 20%) are infections, GvHD, and infusion reaction.
Please see full Prescribing Information, including Boxed Warning.
About Gamida Cell
Gamida Cell is a cell therapy pioneer working to turn cells into powerful therapeutics. The company’s proprietary nicotinamide (NAM) technology leverages the properties of NAM to enhance and expand cells, creating allogeneic cell therapy products and candidates that are potentially curative for patients with hematologic malignancies. These include Omisirge™ (omidubicel-onlv), an FDA-approved nicotinamide modified allogeneic hematopoietic progenitor cell therapy, and GDA-201, an intrinsic NK cell therapy candidate being investigated for the treatment of hematologic malignancies. For additional information, please visit www.gamida-cell.com or follow Gamida Cell on LinkedIn, X, Facebook or Instagram.
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 the potentially life-saving or curative therapeutic and commercial potential of Omisirge™ (omidubicel-onlv). 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 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. 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 August 14, 2023, and other filings that Gamida Cell makes with the SEC from time to time (which are available at 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.
Omisirge is a trademark of Gamida Cell Inc.
____________________
1Data are of transplants performed from January 1–December 31, 2020. These data were reported to the Center for International Blood and Marrow Transplant Research® (CIBMTR) as of February 4, 2021.
2Gamida Cell market research.
3Be The Match® website (accessed 5/30/23); IT-Ideation Department, February 2021 (ethnic background %).
View source version on businesswire.com: https://www.businesswire.com/news/home/20230927409567/en/
Contacts
Media Contact:
Dan Boyle
Orangefiery
media@orangefiery.com
1-818-209-1692
Investor Contact:
Chuck Padala
LifeSci Advisors
Chuck@lifesciadvisors.com
1-646-627-8390
Casterra Delivers First Shipment of Castor Seeds to Africa
https://finance.yahoo.com/news/casterra-delivers-first-shipment-castor-110000179.html
This first shipment, valued at approximately US$1 million, is part of a framework agreement with a world-leading oil and gas company
REHOVOT, Israel, Sept. 27, 2023 /PRNewswire/ -- Casterra Ag Ltd. ("Casterra"), an integrated castor cultivation solution company and a subsidiary of Evogene Ltd. ("Evogene") (Nasdaq: EVGN) (TASE: EVGN), today announced the shipment of its first batch of high-yield, high-oil castor seeds from Brazil and Zambia to its destination in Africa. This shipment, valued at US$1 million, is the first step in meeting US$11.3M purchase orders from a world-leading oil and gas company, which Casterra received in the second quarter of 2023.
Casterra's premium castor seeds are developed using Evogene's GeneRator AI tech-engine and undergo rigorous testing to meet industry standards. These seeds exhibit high-level cleanliness, successfully passed phytosanitary inspection, and have a germination percentage rate of 90%, exceeding the industry acceptance criteria benchmark.
Casterra CEO Eyal Ronen said: "We are pleased to initiate shipments under this framework agreement with a leading oil and gas company, demonstrating the feasibility of shipping large amounts of seeds, even across continents. Casterra anticipates it will supply the remainder of these seed's orders in the coming months."
About Casterra Ag Ltd.:
Casterra is engaged in developing and commercializing high-yielding castor bean seeds as a cost-competitive, sustainable, second-generation feedstock for the growing biofuel market. It has built its castor genetic assets based on a broad collection of over 300 castor lines from over 40 different geographic and climatic regions. As part of its development process, Casterra applies advanced breeding methods utilizing Evogene's GeneRator AI tech-engine, enabling the use of cutting-edge plant genomics tools and agro-technique expertise to enable efficient and sustainable industrial-scale production of the castor bean.
For more information, please visit http://www.casterra.co
Market price reflects a 10%
positive news at most.
BrainStorm Cell Therapeutics Stock Trading Halted Today
https://finance.yahoo.com/news/brainstorm-cell-therapeutics-stock-trading-110000718.html
FDA advisory committee (ADCOM) meeting to review NurOwn® for ALS
NEW YORK, Sept. 27, 2023 /PRNewswire/ -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leading developer of adult stem cell therapeutics for neurodegenerative diseases, today announced that Nasdaq has halted trading of the Company's common stock.
The U.S. Food and Drug Administration's (FDA) Cellular, Tissue and Gene Therapies Advisory Committee is meeting today to review BrainStorm's Biologics License Application (BLA) for NurOwn® for the treatment of amyotrophic lateral sclerosis (ALS). The ADCOM meeting is scheduled to take place 10:00 am to 6:00 pm ET.
The FDA has posted briefing materials, agendas and webcast information for the meeting that can be accessed on the Agency's website here. The online web conference meeting can be viewed through this link.
BrainStorm's BLA for NurOwn® has a Prescription Drug User Fee Act (PDUFA) action date targeted to occur by December 8, 2023.
Can-Fite Novel Approach for the Treatment of Pancreatic Carcinoma with Namodenoson Receives Appreciation of the American Association of Cancer Research (AACR)
https://finance.yahoo.com/news/fite-novel-approach-treatment-pancreatic-110000987.html
Namodenoson robustly inhibits pancreatic carcinoma growth in pre-clinical studies, via inhibition of KRAS and Wnt pathways
Poster summarizing the inhibitory effect of Namodenoson in pancreatic carcinoma is presented today at the AACR conference
Exploratory Phase II pancreatic cancer study protocol has been developed and is to be submitted to ethical committees in Israel and the U.S.
PETACH TIKVA, Israel, September 27, 2023--(BUSINESS WIRE)--Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncology, inflammatory and liver diseases, today announced that its study titled "Namodenoson Inhibits the Growth of Pancreatic Carcinoma via De-regulation of the Wnt/ß-catenin Signaling Pathway" has been accepted for a poster presentation at the AACR Special Conference on Pancreatic Cancer, held from September 27-30, 2023, in Boston, Massachusetts.
The pre-clinical studies presented in the poster used advanced pancreatic carcinoma patient cells that were treated with Namodenoson both in tissue cultures and in in vivo studies. Namodenoson treatment resulted in a significant growth inhibition and de-regulation of two signal transduction pathways, the Wnt and the KRAS, both of which are responsible for the etiology and pathology of this devastating disease.
Can-Fite completed the development of an exploratory Phase II protocol in patients with pancreatic carcinoma who have failed first line treatment and plans to shortly submit it for approval to medical sites in Israel and the U.S. Safety and efficacy endpoints including objective response, progression-free survival, duration of response, disease control, and overall survival will be monitored.
"The robust inhibition of pancreatic carcinoma growth both in vitro and in vivo, together with the definitive mechanism of action is a strong basis for evaluating Namodenoson in patients with pancreatic carcinoma where chemotherapeutic drugs have a very limited effect. We are very happy that the AACR granted us the opportunity to present these encouraging data and to discuss the very important results with the leading global experts in this field," stated Prof. Pnina Fishman, CSO & Executive Chairman of Can-Fite.
About Pancreatic Cancer
The highest incidence rates for pancreatic cancer are in Asia, Europe, and North America. According to the American Society of Clinical Oncology (ASCO), in 2020, an estimated 496,000 people were diagnosed with pancreatic cancer globally and an estimated 466,000 died from the disease. The 5-year survival rate for people with pancreatic cancer in the U.S. is 11%. Acumen Research estimates the global pancreatic cancer therapeutics market was valued at approximately $3.6 billion in 2021 and is projected to grow to approximately $6.6 billion by 2030.
About Namodenoson
Namodenoson was evaluated in Phase 2 trials for two indications, as a second line treatment for hepatocellular carcinoma (HCC), and as a treatment for non-alcoholic fatty liver disease (NAFLD) and non-alcoholic steatohepatitis (NASH). It is currently in a Phase 2b trial for steatotic liver disease and a pivotal Phase 3 for HCC. Namodenoson is a small orally bioavailable drug that binds with high affinity and selectivity to the A3 adenosine receptor (A3AR). A3AR is highly expressed in diseased cells whereas low expression is found in normal cells. This differential effect accounts for the excellent safety profile of the drug.
About Can-Fite BioPharma Ltd.
Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF) is an advanced clinical stage drug development Company with a platform technology that is designed to address multi-billion dollar markets in the treatment of cancer, liver, and inflammatory disease. The Company’s lead drug candidate, Piclidenoson recently reported topline results in a Phase III trial for psoriasis and is expected to commence a pivotal Phase III. Can-Fite’s cancer and liver drug, Namodenoson, is being evaluated in a Phase IIb trial for the treatment of steatotic liver disease (SLD), a Phase III pivotal trial for hepatocellular carcinoma (HCC), and the Company is planning a Phase IIa study in pancreatic cancer. Namodenoson has been granted Orphan Drug Designation in the U.S. and Europe and Fast Track Designation as a second line treatment for HCC by the U.S. Food and Drug Administration. Namodenoson has also shown proof of concept to potentially treat other cancers including colon, prostate, and melanoma. CF602, the Company’s third drug candidate, has shown efficacy in the treatment of erectile dysfunction. These drugs have an excellent safety profile with experience in over 1,600 patients in clinical studies to date. For more information please visit: www.can-fite.com.
Forward-Looking Statements
This press release may contain forward-looking statements, about Can-Fite’s expectations, beliefs or intentions regarding, among other things, its product development efforts, business, financial condition, results of operations, strategies or prospects. All statements in this communication, other than those relating to historical facts, are "forward looking statements". Forward-looking statements can be identified by the use of forward-looking words such as "believe," "expect," "intend," "plan," "may," "should" or "anticipate" or their negatives or other variations of these words or other comparable words or by the fact that these statements do not relate strictly to historical or current matters. Forward-looking statements relate to anticipated or expected events, activities, trends or results as of the date they are made. Because forward-looking statements relate to matters that have not yet occurred, these statements are inherently subject to known and unknown risks, uncertainties and other factors that may cause Can-Fite’s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Important factors that could cause actual results, performance or achievements to differ materially from those anticipated in these forward-looking statements include, among other things, our history of losses and needs for additional capital to fund our operations and our inability to obtain additional capital on acceptable terms, or at all; uncertainties of cash flows and inability to meet working capital needs; the initiation, timing, progress and results of our preclinical studies, clinical trials and other product candidate development efforts; our ability to advance our product candidates into clinical trials or to successfully complete our preclinical studies or clinical trials; our receipt of regulatory approvals for our product candidates, and the timing of other regulatory filings and approvals; the clinical development, commercialization and market acceptance of our product candidates; our ability to establish and maintain strategic partnerships and other corporate collaborations; the implementation of our business model and strategic plans for our business and product candidates; the scope of protection we are able to establish and maintain for intellectual property rights covering our product candidates and our ability to operate our business without infringing the intellectual property rights of others; competitive companies, technologies and our industry; risks related to the COVID-19 pandemic and the Russian invasion of Ukraine; risks related to not satisfying the continued listing requirements of NYSE American; and statements as to the impact of the political and security situation in Israel on our business. More information on these risks, uncertainties and other factors is included from time to time in the "Risk Factors" section of Can-Fite’s Annual Report on Form 20-F filed with the SEC on March 30, 2023 and other public reports filed with the SEC and in its periodic filings with the TASE. Existing and prospective investors are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. Can-Fite undertakes no obligation to publicly update or review any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by any applicable securities laws.
View source version on businesswire.com: https://www.businesswire.com/news/home/20230927971749/en/
Contacts
Can-Fite BioPharma
Motti Farbstein
info@canfite.com
+972-3-9241114
0.9765+0.0774 (+8.6086%)
As of 12:59PM EDT. Market open.
Rosy cheeks!
MediWound Secures Additional U.S. Department of Defense Funding to Advance NexoBrid® Development for the U.S. Army
https://finance.yahoo.com/news/mediwound-secures-additional-u-department-110000204.html
R&D project budget increased to $6.5Million to advance NexoBrid as a non-surgical field care solution
YAVNE, Israel, Sept. 26, 2023 (GLOBE NEWSWIRE) -- MediWound Ltd. (Nasdaq: MDWD), a fully-integrated biopharmaceutical company focused on next-generation enzymatic therapeutics for tissue repair, today announced that the U.S. Department of Defense (DoD), through the Medical Technology Enterprise Consortium (MTEC), has awarded MediWound additional funding to develop NexoBrid® as a non-surgical solution for field-care burn treatment for the U.S. Army (the “MTEC Research Project Award”). The $6.5 million project budget will advance the development of a new, temperature stable formulation of NexoBrid, positioning it as the first-line non-surgical solution for treating severe burn injuries in pre-hospital settings.
“We are honored to further our collaboration with the U.S. Department of Defense. Our shared vision of enhancing treatment outcomes for traumatic burns on the battlefield will ensure NexoBrid's availability for military use,” said Ofer Gonen, Chief Executive Officer of MediWound. “This additional funding reinforces NexoBrid's standing as the non-surgical, temperature stable, user-friendly, and highly effective solution for eschar removal, emphasizing its game-changing potential in early severe burn treatment protocols.”
The MTEC Research Project Award was granted by the DoD’s U.S. Army Medical Research and Development Command (USAMRDC) and funded by the Defense Health Agency through MTEC, a biomedical technology consortium working to advance innovative medical solutions to keep military personnel healthy and fully operational. In alignment with this mission, it's vital to have field solutions for severe burn treatments that are both easy-to-use and effective. Such solutions should be applicable immediately post-injury and demand minimal preparation and training.
About U.S. Army Medical Research and Development Command (USAMRDC)
The U.S. Army Medical Research and Development Command is the Army’s medical materiel developer, with responsibility for medical research, development, and acquisition. USAMRDC produces medical solutions for the battlefield with a focus on various areas of biomedical research, including military infectious diseases, combat casualty care, military operational medicine, medical chemical, and biological defense https://mrdc.health.mil/.
RDHL should perhaps r/s 500,000/1, thus
ensuring no need for an additional r/s for
at least 3 months!
Sigh!
Brainstorm falls as FDA posts briefing docs on ALS therapy
08:45 AM | Brainstorm Cell Therapeutics Inc. (BCLI)
By: Dulan Lokuwithana, SA News Editor
Even if BCLI obtains approval, the next immediate
problem will be if insurance companies will pay.
Thank you Scott, just visiting the board periodically
to see what's new, enjoy a chuckle and move on.
Remember the controller? Whatever happened to him i wonder?
Whatever happened to BP who tried to destroy PSTI via a hostile takeover?
Alas, too many unanswered questions, but surely next week will the good
news be out!
This week it's an either make
or break for $BCLI.
Fingers crossed.
You are too generous rwwine!
I opt for a straight F.
Very disappointing indeed!
Very happy to see one very
optimistic GMDA investor!
Teaser is my middle name!
BrainsWay Broadens Access to Deep TMS™ for U.S. Military Service Members Through Expanded Partnership with Katie’s Way Plus
https://finance.yahoo.com/news/brainsway-broadens-access-deep-tms-113000032.html
Provider of Mental Health Services with Focus on Military Community Orders 10 New Deep TMS™ Systems
BURLINGTON, Mass. and JERUSALEM, Sept. 20, 2023 (GLOBE NEWSWIRE) -- BrainsWay Ltd. (NASDAQ & TASE: BWAY) (“BrainsWay” or the “Company”), a global leader in advanced noninvasive neurostimulation treatments for mental health disorders, today announced that its longstanding partner, Katie’s Way Plus, which provides comprehensive mental health services tailored to the unique needs of active duty military members, veterans, and their families, has ordered an additional 10 Deep Transcranial Magnetic Stimulation (Deep TMS™) systems, which will more than double its existing offering.
“This is yet another demonstration of our strategic focus on larger enterprise customers across the U.S.,” said Eric Hirt, VP U.S. Sales of BrainsWay. “The strategic collaboration between BrainsWay and Katie's Way Plus is poised to make a substantial impact on addressing the critical and unique mental health issues faced by military personnel by providing world-class care to those who have served their country and are in need of advanced solutions to address their specialized conditions.”
“We are thrilled to support Katie's Way Plus in its vital mission to provide focused care to U.S. service members, veterans, and their families,” said Hadar Levy, Chief Executive Officer of BrainsWay. “BrainsWay's advanced Deep TMS technology has demonstrated remarkable clinical efficacy in treating depression, and we are confident that this expansion will bring hope and healing to many U.S. service members.”
“Katie's Way was founded in memory of my daughter, Katie, who tragically lost her battle with depression,” said Jeff Mathis, Founder of Katie’s Way. “Our goal is to ensure that no other family needs to endure such pain. These additional BrainsWay systems will allow us to extend our reach, offer hope, and ultimately save lives.”
Depression is a contributing factor to suicide, which is a particularly pressing concern within the military community. September is Suicide Prevention Month, which helps raise awareness to the fact that 22 U.S. military veterans take their lives each dayi.
BrainsWay Deep TMS is delivered via a unique treatment coil housed in a cushioned, patient and provider-friendly helmet. The patient is awake, alert, and engaged during the treatment session, and each session only takes less than 30 minutes from start to finish. For some patients, a shorter three-minute intermittent theta burst (iTBS) protocol is also an option. Most candidates for Deep TMS depression treatment have already failed multiple courses of medication and talk therapy, and in a recently published post-marketing analysis of close to 1,400 patients, those patients who had received 30 or more treatments of Deep TMS demonstrated an 82% response rate and a 65% remission rateii.
About BrainsWay
BrainsWay is a global leader in advanced noninvasive neurostimulation treatments for mental health disorders. The Company is boldly advancing neuroscience with its proprietary Deep Transcranial Magnetic Stimulation (Deep TMS™) platform technology to improve health and transform lives. BrainsWay is the first and only TMS company to obtain three FDA-cleared indications backed by pivotal clinical studies demonstrating clinically proven efficacy. Current indications include major depressive disorder (including reduction of anxiety symptoms, commonly referred to as anxious depression), obsessive-compulsive disorder, and smoking addiction. The Company is dedicated to leading through superior science and building on its unparalleled body of clinical evidence. Additional clinical trials of Deep TMS in various psychiatric, neurological, and addiction disorders are underway. Founded in 2003, with offices in Burlington, MA and Jerusalem, Israel, BrainsWay is committed to increasing global awareness of and broad access to Deep TMS. For the latest news and information about BrainsWay, please visit www.brainsway.com.
About Katie’s Way
Katie's Way TMS is a trailblazing mental health care provider with a heartfelt commitment to improving the lives of active duty military service members, veterans, and their families. Founded by Jeff Mathis, our mission is deeply rooted in a profound personal loss. Jeff's daughter, Katie, tragically lost her battle with depression, inspiring him to establish Katie's Way with a resolute determination to prevent others from experiencing a similar tragedy. At Katie's Way TMS, our focus is clear: to provide specialized care, hope, and healing to those who have selflessly served our nation. We understand the unique challenges that military members, veterans, and their families face when it comes to mental health, and we are dedicated to addressing these challenges head-on. With unwavering commitment, Katie's Way TMS strives to make a meaningful impact by providing accessible, evidence-based care to individuals who have sacrificed so much for our country. Our journey is driven by the memory of Katie and the hope that no other family will have to endure the pain of losing a loved one to depression. Katie’s Way currently operates 5 treatment facilities in the United States, and will expand to 12-15 clinics by early 2024. To learn more, please visit www.katieswayplus.com.
BioLineRx Announces Acceptance of Oral Presentation on Pilot Phase Data from Phase 2
Combination Clinical Trial with Motixafortide in First-Line PDAC at AACR Special Conference
on Pancreatic Cancer.
Abstracts to be Published on American Association of Cancer Research (AACR)
Virtual Meeting Platform at 5:30 pm EDT on Wednesday, September 27, 2023
Oral Presentation on Thursday, September 28, 2023, in Boston, Massachusetts
https://finance.yahoo.com/news/biolinerx-announces-acceptance-oral-presentation-110000805.html
BioLineRx Announces Acceptance of Oral Presentation on Pilot Phase Data from Phase 2 Combination Clinical Trial with Motixafortide in First-Line PDAC at AACR Special Conference on Pancreatic Cancer
https://finance.yahoo.com/news/biolinerx-announces-acceptance-oral-presentation-110000805.html
Abstracts to be Published on American Association of Cancer Research (AACR)
Virtual Meeting Platform at 5:30 pm EDT on Wednesday, September 27, 2023
Oral Presentation on Thursday, September 28, 2023, in Boston, Massachusetts
TEL AVIV, Israel, Sept. 19, 2023 /PRNewswire/ -- BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases, today announced that pilot phase data from an investigator-initiated, open-label, multicenter Phase 2 clinical trial with motixafortide in first-line pancreatic ductal adenocarcinoma (PDAC) will be presented at the American Association for Cancer Research (AACR) Special Conference on Pancreatic Cancer taking place in Boston, Massachusetts, September 27-30, 2023. The Phase 2 clinical trial is designed to evaluate the company's CXCR4 inhibitor motixafortide in combination with PD-1 inhibitor cemiplimab and standard of care chemotherapies gemcitabine and nab-paclitaxel, versus gemcitabine and nab-paclitaxel alone, in first-line pancreatic cancer (PDAC).
Sponsored by Columbia University, the single-arm pilot phase of the Phase 2 trial focused on the safety of the drug combination. Additionally, based on the original protocol, if =3 of the planned 10 patients within the pilot phase experienced a partial response (PR) by RECIST criteria within 16 weeks, the combination would be considered promising and an expansion cohort of an additional 30 patients would be initiated for enrollment. Earlier this year, following a review of the pilot phase data, the trial was amended to become a randomized study, with planned enrollment increasing from 30 to 102 patients. A poster of the amended clinical trial design was presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting, held June 2-6 in Chicago, Illinois (see abstract).
Presentation at AACR Special Conference in Cancer Research: Pancreatic Cancer
Westin Copley Place, Boston Massachusetts
Plenary Session Details
Title: CheMo4METPANC: Combination Chemotherapy (gemcitabine and nab-paclitaxel), chemokine (C-X-C) Motif receptor 4 inhibitor (motixafortide), and immune checkpoint blockade (cemiplimab) in METastatic treatment-naïve PANCreatic adenocarcinoma
Presenter: Gulam A. Manji, MD, PhD, Columbia University Irving Medical Center/New York Presbyterian, New York, N.Y.
Session: Plenary Session 3: Clinical Updates
Date: Thursday, September 28, 2023
Time: 2:30-4:40 pm EDT
About Pancreatic Cancer
Pancreatic cancer has a low rate of early diagnosis and a poor prognosis. In the United States in 2023, an estimated 64,000 adults will be diagnosed with the disease, which accounts for approximately 3% of all cancers in the U.S. and about 7% of all cancer deaths. Worldwide, an estimated 496,000 people were diagnosed with the disease in 2020. In the U.S., if the cancer is detected at an early stage when surgical removal of the tumor is possible, the 5-year relative survival rate is 44%. About 12% of people are initially diagnosed at this stage. If the cancer has spread to surrounding tissues or organs, the 5-year relative survival rate is 15%. For the 52% of people who are initially diagnosed with metastatic cancer, the 5-year relative survival rate is 3%. These data highlight the need for the development of new therapeutic options.
About Motixafortide in Cancer Immunotherapy
Motixafortide inhibits CXCR4, a chemokine receptor and a well validated therapeutic target that is over-expressed in many human cancers including pancreatic ductal adenocarcinoma (PDAC). Motixafortide leverages the expression of the CXCR4 receptor on different immune cells and potentiates the immune system against the tumor. Among CXCR4-expressing immune cells, some exhibit anti-tumoral activity, such as effector T cells and some exhibit pro-tumoral activity and support tumor growth. By blocking the CXCR4 receptor, motixafortide was shown in a Phase 2 study in pancreatic cancer patients to enhance anti-tumoral activity and to ameliorate the pro-tumoral activities by modulating the effector/suppressor cell ratio towards a proinflammatory profile.
About BioLineRx
BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX) is a commercial stage biopharmaceutical company pursuing life-changing therapies in oncology and rare diseases. The company's first approved product is APHEXDA™ (motixafortide) with an indication in the U.S. for stem cell mobilization for autologous transplantation in multiple myeloma. BioLineRx is advancing a pipeline of investigational medicines for patients with sickle cell disease, pancreatic cancer, and other solid tumors. Headquartered in Israel, and with commercial operations in the U.S., the company is driving innovative therapeutics with end-to-end expertise in development and commercialization, ensuring life-changing discoveries move beyond the bench to the bedside.
Learn more about who we are, what we do, and how we do it at www.biolinerx.com, or on Twitter and LinkedIn.
Bottom-Line Summary
BLRX has a drug approved, full stop. This is a big landmark for any company, and their drug definitely has a role to play in multiple myeloma. Exactly how big a role remains a question, since there's generic plerixafor competition now (even if plerixafor is marginally worse than motixafortide), and fewer patients need immediate autologous transplants. This stock is worth a close watch, since if they can bounce off this approval into decent sales relatively quickly, then the current market cap of $120 million is basically a joke.
Assessing BioLineRx As A Buy After Drug Approval
Sep. 18, 2023 1:28 AM ETBioLineRx Ltd. (BLRX)
Summary
BioLineRx has received approval for their stem cell mobilizing compound, motixafortide, for certain forms of blood cancer.
The compound has shown impressive results in mobilizing stem cells in patients, with a high success rate in optimal stem cell collection.
However, there are concerns about competition from generic versions of a similar drug and the decreasing need for immediate autologous transplants in patients with multiple myeloma.
Topline Summary
BioLineRx (NASDAQ:BLRX) is a biotech company focused mainly on developing a stem cell mobilizing compound to help patients with certain forms of blood cancer. Their exciting news is that they've received a first approval for their compound, but there is reason for caution moving forward, given that their setting of choice continues to be questioned in the literature. There's also generic competition, although BLRX has attempted to get in front of this with a post-hoc analysis suggesting better benefits with their drug. Long story short: this stock is sitting at a bargain price if they're able to capitalize decently on their recent approval.
Pipeline Overview
Motixafortide
The main compound being developed by BLRX is motixafortide, an inhibitor of CXCR4. This target has numerous potential benefits for patients with cancer and blood diseases, but the most advanced in BLRX's pipeline is as a stimulator of blood stem cell production and mobilization.
Why? Transplant of blood stem cells, either from the patients themselves (autologous) or from a donor (allogeneic), has been a core piece of managing blood cancers like leukemia and lymphoma for decades. In patients with multiple myeloma, autologous stem cell transplantation was for a long time one of the best options for patients.
That is, it's one of the best options if you're able to get the body to move stem cells into the blood so you can collect them. And a large number of patients are unable to present enough of these stem cells in the blood with standard procedures. Addition of the CXCR4 inhibitor plerixafor and the growth factor G-CSF (filgrastim) has improved this rate of mobilization, but many patients continue to struggle with getting the optimal number of stem cells, which can negatively impact how well they do post-transplant.
Motixafortide is a more potent CXCR4 inhibitor that was assessed in the phase 3 GENESIS trial, part 2 of which compared motixafortide plus filgrastim versus placebo plus filgrastim. This study showed impressive mobilization of stem cells with motixafortide, with 96.3% of patients achieving optimal stem cell collection by day 3, compared with 36.5% in the placebo group. Moreover, optimal collection was achieved after just one round of apheresis in 88.8% of patients with motixafortide.
Outcomes post-transplant, like engraftment of cells and graft durability, were similar between the two arms, suggesting that motixafortide did not negatively alter the stem cells that were collected. Almost 2 years since that presentation, motixafortide was approved on September 11, 2023, in combination with filgrastim to help mobilize hematopoietic stem cells in patients with multiple myeloma.
At ASH 2022, the company presented an indirect comparison of motixafortide versus plerixafor that suggested motixafortide could achieve optimal stem cell mobilization in fewer rounds of treatment, with concomitant potential benefit in terms of cost overall.
BLRX is continuing to develop motixafortide in different applications, mainly in sickle cell disease, but few developments have been announced in this direction.
More recently, the company announced that an investigator-initiated, randomized, phase 2 trial to investigate motixafortide plus cemiplimab plus gemcitabine/nab-paclitaxel in patients with advanced pancreatic cancer, hoping to build on results they observed in the COMBAT study in the second-line setting.
Financial Overview
As of their Q2 2023 filing, BLRX held $10.1 million in cash and equivalents, with another $22.7 million in short-term bank deposits. This was set against a $9.9 million operating loss, most of which went to expansion of their sales capabilities in anticipation of their approval. There was an unusual $7.7 million in non-operating income loss, as well, related to the revaluation of outstanding warrants, so the net loss reached $18.5 million for the quarter, compared with $7.4 million in the same fiscal quarter 2022.
Assuming this net loss was anomalous, BLRX has approximately 3 quarters of cash and current assets on hand to fund operations from the end of Q2. That gives them approximately 7 months to get a launch off the ground.
Strengths and Risks
A drug approval is the holy grail for developmental biotechs, and now BLRX gets to begin the transition into a commercial entity. That is indisputable, and now the company is going to be able to gain traction.
The only question, then, is how much traction? There are 2 red flags that come to mind for motixafortide. First, the GENESIS trial did not include plerixafor as a comparator, and their attempts to compare the cost/benefit of the 2 agents in a post-hoc analysis underscores how important a question it is to answer whether the new drug is as good or better than the old.
You cannot do this convincingly by these kinds of analyses. So it will remain a question whether a clinician should use plerixafor or motixafortide. A future head-to-head study could definitively answer the question, which would be a boon because the cost-effectiveness of plerixafor has also been called into question.
Now that plerixafor has multiple generic versions launched, BLRX may have an uphill battle.
The other potential red flag is that novel therapies have continuously undermined the role of upfront stem cell transplant for patients with multiple myeloma. Most recently, the DETERMINATION trial demonstrated that stem cell transplant offered no overall survival benefit after RVD therapy compared with RVD alone.
Long story short: newer, better therapies are delaying and preventing the need for a costly stem cell transplant in more patients than ever, which could tighten the pool of patients that could potentially benefit from motixafortide. Of course, it is approved to mobilize stem cells for storage in case of a future transplant, and a lot of patients may opt for that strategy.
But it's uncertainty, meaning that motixafortide is very much not a slam dunk as a commercial product. We'll have to watch carefully how BLRX handles the rollout.
That said, all of these risks cannot trump that motixafortide has impressive phase 3 data and drug approval. Some analysts on Seeking Alpha have proposed that BLRX is a strong buyout candidate, but that is very much speculative. Cash is another issue BLRX will need to solve in the near term. Hopefully, they can launch motixafortide sales before they need to do an equity raise.
Bottom-Line Summary
BLRX has a drug approved, full stop. This is a big landmark for any company, and their drug definitely has a role to play in multiple myeloma. Exactly how big a role remains a question, since there's generic plerixafor competition now (even if plerixafor is marginally worse than motixafortide), and fewer patients need immediate autologous transplants. This stock is worth a close watch, since if they can bounce off this approval into decent sales relatively quickly, then the current market cap of $120 million is basically a joke.
RedHill Announces FDA sNDA Approval for Talicia®
https://finance.yahoo.com/news/redhill-announces-fda-snda-approval-110000396.html
Mon, September 18, 2023 at 2:00 PM GMT+3
FDA approves supplemental new drug application (sNDA) for Talicia, changing dosing to a simplified three times daily (TID) regimen, which may support increased patient adherence and treatment outcomes
Talicia is the leading branded first-line therapy prescribed by U.S. gastroenterologists[1] for eradication of H. pylori - a bacterial infection that affects approximately 35% of the U.S. adult population[2]
RALEIGH, N.C. and TEL-AVIV, Israel, Sept. 18, 2023 /PRNewswire/ -- RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, announced the U.S. Food and Drug Administration's (FDA) approval of its supplemental new drug application (sNDA) for Talicia[3], allowing a change to a more flexible three times daily (TID), taken at least 4 hours apart with food, dosing regimen for H. pylori eradication. This differs from the previously approved dosing regimen (Q8H), which required dosing every eight hours with food, by enabling patients to follow a convenient "breakfast, lunch and dinner" dosing routine, which may support increased patient adherence and optimize the potential for successful H. pylori eradication.
"H. pylori treatment can be challenging for patients as most regimens require different pills to be taken multiple times per day. However, it is clear that simplified regimens promote improved patient adherence and should be a key factor when considering choice of H. pylori eradication therapy," said Colin W. Howden, MD, Professor Emeritus, University of Tennessee College of Medicine. "This new dosing regimen further supports the value of Talicia as an empirically prescribed first-line therapy for H. pylori eradication. Talicia's favorable efficacy, tolerability, and resistance profile, as well as being the only all-in-one formulation available, provides potential advantages over clarithromycin-based regimens for most patients."
"Talicia is unique in that it is the only FDA-approved rifabutin-based therapy for the eradication of H. pylori. Both its components and formulation are optimized to provide patients with the necessary medications for successful H. pylori eradication," said Dr. June Almenoff, MD, Ph.D., RedHill's Chief Medical Officer. "RedHill is committed to advancing GI and infectious disease management through patient-focused innovation. Through our successful collaboration with Certara, utilizing their Simcyp™ Simulator for physiologically based pharmacokinetic (PBPK) modeling, we have demonstrated therapeutic equivalence between TID and Q8H dosing, enabling us to provide what we believe is a more flexible Talicia regimen that we believe will be beneficial for the patient experience."
About H. pylori infection
H. pylori is a bacterial infection that affects approximately 35% of the U.S. population, with an estimated two million patients treated annually[4]. Worldwide, more than 50% of the population has H. pylori infection, which is classified by the WHO as a Group 1 carcinogen. It remains the strongest known risk factor for gastric cancer[5] and a major risk factor for peptic ulcer disease[6] and gastric mucosa-associated lymphoid tissue (MALT) lymphoma[7]. More than 27,000 Americans are diagnosed with gastric cancer annually[8]. Eradication of H. pylori is becoming increasingly difficult, with current therapies failing in approximately 25-40% of patients who remain H. pylori-positive due to high resistance of H. pylori to antibiotics – especially clarithromycin – which is still commonly used in standard combination therapies2.
About Talicia
Talicia is a novel, fixed-dose, all-in-one oral capsule combination of two antibiotics (amoxicillin and rifabutin) and a proton pump inhibitor (PPI) (omeprazole), approved by the U.S. FDA for the treatment of H. pylori infection in adults.
Talicia is the only low-dose rifabutin-based therapy approved for the treatment of H. pylori infection and is designed to address H. pylori's high resistance to other antibiotics. The high rates of H. pylori resistance to clarithromycin have led to significant rates of treatment failure with clarithromycin-based therapies and are a strong public health concern, as highlighted by the ACG, FDA and the World Health Organization (WHO) in recent years.
In the pivotal Phase 3 study, Talicia demonstrated 84% eradication of H. pylori infection in the intent-to-treat (ITT) group vs. 58% in the active comparator arm (p<0.0001). Minimal to zero resistance to rifabutin, a key component of Talicia, was detected in RedHill's pivotal Phase 3 study. Further, in an analysis of data from this study, it was observed that subjects who were confirmed adherent[9] to their therapy had response rates of 90.3% in the Talicia arm vs. 64.7% in the active comparator arm[10]. To reduce the development of drug-resistant bacteria and maintain the effectiveness of Talicia and other antibacterial drugs, Talicia should be used only to treat or prevent infections that are proven or strongly suspected to be caused by bacteria.
Talicia is eligible for a total of eight years of U.S. market exclusivity under its Qualified Infectious Disease Product (QIDP) designation and is also covered by U.S. patents which extend patent protection until 2034 with additional patents and applications pending and granted in various territories worldwide.
TALICIA: IMPORTANT SAFETY INFORMATION
Tell your healthcare provider about all of the medicines you take, including prescription or non-prescription medications or herbal supplements before starting Talicia. Talicia may affect the way other medicines work, and other medicines may affect the way Talicia works. Do not start any new medications while taking Talicia without first speaking with your healthcare provider.
You should not take Talicia if you are known to be sensitive to any of the components of Talicia (omeprazole, amoxicillin, rifabutin), penicillins, proton pump inhibitors or rifamycins.
You should not take Talicia if you are taking rilpivirine-containing products, delavirdine or voriconazole.
Before you take Talicia, tell your healthcare provider about all of your medical conditions, including if you:
Are pregnant or plan to become pregnant. Talicia may harm your unborn baby. Tell your healthcare provider if you become pregnant or think you may be pregnant during your treatment with Talicia.
Have severe kidney disease or liver disease.
When taking Talicia, do not crush or chew capsules. Do not take Talicia with alcohol.
Call your healthcare provider immediately if while taking Talicia you develop:
New rash or other skin changes, muscle or joint pains, swelling of any area of the body, severe flu-like symptoms, difficulty breathing, fever, blood in your urine, increased or decreased urination, drowsiness, confusion, nausea, vomiting, ongoing stomach pain, bloody diarrhea, or if diarrhea continues after therapy is completed, weight gain or changes in your eyesight.
What are the common side effects of Talicia?
The most common side effects of Talicia are diarrhea, headache, nausea, stomach pain, rash, indigestion, mouth or throat pain, vomiting, and vaginal yeast infection. Call your healthcare professional for medical advice about side effects.
Tell your healthcare provider if you experience tiredness, weakness, achiness, headaches, dizziness, depression, increased sensitivity to light, or pain when taking a deep breath.
Talicia may reduce the effectiveness of oral or other forms of hormonal birth-control. You should use an additional non-hormonal highly effective method of birth control while taking Talicia.
You may experience a brown-orange discoloration of your urine or tears while taking Talicia.
The information here is not comprehensive. Talk to your healthcare provider to learn more.
APPROVED USE FOR TALICIA
TALICIA is indicated for the treatment of Helicobacter pylori infection in adults.
Click here for the full Prescribing Information for TALICIA.
You are encouraged to report Adverse Reactions to RedHill Biopharma Inc. at 1-833-ADRHILL (1-833-237-4455) or the FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.
4.7200+0.3900 (+9.0069%)
As of 11:17AM EDT. Market open.
Momentum continues:
Volume 378,206
Avg. Volume 51,124
Bon chance! It will not be a smooth ride,
but long term you stand to gain imo.
Summary
BioLineRx reported its second piece of material good news in as many weeks with FDA approval of Aphexda for stem cell mobilization in multiple myeloma patients. The approval of the drug delivers additional credibility to BioLineRx’ other motixafortide programs and provides a tailwind to commercialization efforts in Asia. We expect to see IIA approval for the Asia deal in the next few months and could see first ex-US revenues shortly after. Other highlights for the company include the start of the Columbia University-sponsored Phase II PDAC trial, Washington University’s work on sickle cell disease and efforts advancing AGI-134 in solid tumors. The US commercialization team has been hard at work preparing for Aphexda’s approval and could have first sales in the next few weeks. Based on the approval of Aphexda, we increase our valuation to $7.00 per share reflecting an increase in the probability of regulatory success to 100%.