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MGTA raises cash
Magenta announces private placement worth $86.4M
May 12, 2021 11:11 AM ETMagenta Therapeutics, Inc. (MGTA)By: Jonathan M Block, SA News Editor
Magenta Therapeutics (NASDAQ:MGTA) has announced a private placement of 9.6M shares priced at $9/share for expected gross proceeds of $86.4M.
Institutional investors include Deep Track Capital, TCG X, Great Point Partners, OrbiMed, and Janus Henderson Investors
The closing is expected on May 14.
Magenta shares are up 3.6% to $12.143 in morning trading.
Bladerunner
MGTA surges on preliminary data from cell mobilization study
Magenta posts positive preliminary results from mid-stage multiple myeloma study
May 12, 2021 10:23 AM ETMagenta Therapeutics, Inc. (MGTA)By: Aakash Babu, SA News Editor
Magenta Therapeutics (MGTA +0.8%) announces positive preliminary results from its Phase 2 clinical trial of MGTA-145 plus plerixafor in patients with multiple myeloma.
The investigator-initiated, 25-patient Phase 2 open-label clinical trial is designed to evaluate the ability of the drug combination to mobilize and collect stem cells for autologous stem cell transplant in patients with multiple myeloma.
All patients (10/10) met the primary endpoint of mobilization and collection of 2 million CD34+ stem cells per kg in up to two days of same-day mobilization and apheresis.
All transplanted patients (6/6) successfully engrafted, with median recovery of neutrophils after 12 days and platelets after 17 days, which are within transplant expectations in multiple myeloma.
The trial continues to enroll patients and Magenta expects to report additional data at the EHA Congress, as well as at the American Society of Clinical Oncology (ASCO) Annual Meeting, to be held in June.
Bladerunner
CRIS jumps (65%) after showing impressive results in hematological cancers
Curis jumps after blood cancer trial indicates impressive results
May 12, 2021 11:29 AM ETCuris, Inc. (CRIS)By: Dulan Lokuwithana, SA News
Curis (CRIS +58.7%) is surging after announcing updated data from its ongoing open-label, single-arm, Phase 1/2 study of CA-4948 in patients with acute myeloid leukemia (AML) or high-risk myelodysplastic syndromes (MDS).
As of data cut off on February 8, 2021, in 15 patients (8 MDS and 7 AML), four objective responses were recorded with signs of hematologic recovery.
The included one complete response (CR), one complete remission with incomplete hematologic recovery (CRi) with negative minimal residual disease, and two bone marrow CRs, the company said.
The two abstracts with updated data have been accepted for oral and poster presentation at the upcoming European Hematology Association 2021 Virtual Congress (EHA).
CA-4948 is an IRAK4 kinase inhibitor that is believed to be involved in toll-like receptor (TLR) and interleukin-1 receptor (IL-1R) signaling pathways.
TLR and IL-1R pathways are frequently dysregulated in patients with AML and MDS.
Rigel Pharmaceuticals (RIGL +16.5%) is also rising in conjunction. The company has a partnership with BerGenBio for BGB324 (bemcentinib), a first-in-class selective AXL kinase inhibitor.
BGB324 is undergoing Phase 1/2 studies as a single agent for relapsed AML and MDS. and in combination with erlotinib (Tarceva) in advanced (EGFR-positive) non-small-cell lung carcinoma.
Bladerunner
Blade, Thanks. I did get some FSTX yesterday, but then quickly wimped out today after seeing the broader market selling off. Only 300 shares, and I managed to break even, but I've become a nervous nellie when it comes to bio stocks, and stocks in general. After a great 2020 and Q1-21 I don't want to give back those gains. The broader market looks overbought anyway, so I'm sitting with over 50% in cash and T-Bills. The volatility in biotech is just too much for my frazzled nerves, lol. Still interesting to follow them though :o)
MYOV announces end of fiscal year results. Hard to imagine a more positive announcement. Stock remains cheap, IMO.
GlobeNewswire
Myovant Sciences Announces Corporate Updates and Financial Results for Fourth Fiscal Quarter and Fiscal Year Ended March 31, 2021
Myovant Sciences, Inc.
Tue, May 11, 2021, 3:58 AM·19 min read
MYOV
+1.96%
Fourth fiscal quarter 2020 total revenues of $24.6 million; net product revenue from sales of ORGOVYX in the U.S. of $3.6 million
FDA review of New Drug Application for relugolix combination tablet for uterine fibroids remains on track for a decision by June 1, 2021 target action date; U.S. launch expected in June 2021, if approved
Remain on track to submit U.S. regulatory filing for endometriosis in the second quarter of calendar year 2021
European Commission decision on uterine fibroids Marketing Authorization Application remains on track for mid-calendar year 2021; Gedeon Richter to launch and commercialize, if approved
European Medicines Agency validated Marketing Authorization Application for relugolix for the treatment of advanced prostate cancer
Reported positive data for relugolix combination therapy from Phase 3 SPIRIT extension study in women with endometriosis and from Phase 3 LIBERTY randomized withdrawal study in women with uterine fibroids
Company remains well-capitalized with cash, cash equivalents, marketable securities and committed funding of $726.2 million as of March 31, 2021
BASEL, Switzerland, May 11, 2021 (GLOBE NEWSWIRE) -- Myovant Sciences (NYSE: MYOV), a healthcare company focused on redefining care for women and for men, today announced corporate updates and financial results for the fourth fiscal quarter and fiscal year ended March 31, 2021.
“The ORGOVYX launch is off to a strong start and its differentiated clinical profile has the potential to redefine care for men with advanced prostate cancer. ORGOVYX demand accelerated over the course of the quarter, reflecting our ongoing efforts to educate urologists and medical oncologists about ORGOVYX while improving access and reimbursement for patients. In partnership with Pfizer, we continue to execute on our long-term goal of establishing ORGOVYX as the new standard of care androgen deprivation therapy,” said David Marek, Chief Executive Officer of Myovant Sciences, Inc. “I am also pleased with the progress we have made in preparing for the U.S. launch of relugolix combination tablet in women with uterine fibroids, which is expected this June. In addition, we have advanced relugolix combination tablet toward a U.S. regulatory submission in endometriosis and our European regulatory submission for relugolix monotherapy for the treatment of advanced prostate cancer was validated by the European Medicines Agency.”
Fourth Fiscal Quarter 2020 and Recent Corporate Updates
ORGOVYX
ORGOVYX was launched in the U.S. and authorized specialty distribution channels were fully stocked in early January 2021. Fourth fiscal quarter 2020 net product revenues for ORGOVYX in the U.S. were $3.6 million.
More than 800 treatment centers have prescribed ORGOVYX to over 2,000 patients, estimated through April 30, 2021.
Through April 30, 2021, Myovant achieved 43% commercial coverage and 51% Medicare Part D coverage for ORGOVYX. Myovant continues to engage in coverage negotiations with key commercial and Medicare Part D payors and remains on track to achieve its goal of broad coverage at the end of calendar year 2021.
Relugolix Monotherapy
On March 29, 2021, Myovant announced that the European Medicines Agency (EMA) validated its Marketing Authorization Application (MAA) for relugolix for the treatment of advanced prostate cancer. The validation of the application confirmed that the submission is sufficiently complete for the EMA to begin the review process.
Relugolix Combination Tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg)
Uterine Fibroids
On March 24, 2021, Myovant and Pfizer announced positive safety and efficacy data from the Phase 3 LIBERTY randomized withdrawal study in women with uterine fibroids. The study met its primary endpoint and all three key secondary endpoints. Bone mineral density was maintained through two years in the subset of women continuously treated with relugolix combination therapy (N = 31). The incidence of adverse events over one additional year of treatment was consistent with those observed in prior studies, with no new safety signals observed.
Endometriosis
On January 26, 2021, Myovant and Pfizer announced that the Phase 3 SPIRIT long-term extension study in women with endometriosis reported clinically meaningful reductions in dysmenorrhea (menstrual pain) and non-menstrual pelvic pain over one year with minimal and stable bone mineral density loss. The data are consistent with the efficacy and safety profile observed through 24 weeks in the Phase 3 SPIRIT 1 and SPIRIT 2 studies. These results will be included in the regulatory submission to the U.S. Food and Drug Administration (FDA) for relugolix combination tablet for the treatment of women with endometriosis, expected in the second quarter of calendar year 2021.
Prevention of Pregnancy
On April 12, 2021, Myovant and Pfizer announced that the first patient was dosed in the Phase 3 SERENE study evaluating the contraceptive efficacy of relugolix combination tablet in healthy women ages 18-35 years who are at risk for pregnancy. The SERENE study is designed to enroll 900 sexually active, healthy women ages 18-35 years with presumed normal fertility. The primary efficacy endpoint is the at-risk Pearl Index, defined as the number of on-treatment pregnancies per 100 women-years of treatment. Safety data will also be collected during the study. Results of the SERENE study could support a potential indication of pregnancy prevention for women treated with relugolix combination tablet, if approved.
Executive Appointments
On January 4, 2021, Myovant announced the appointment of David Marek as Chief Executive Officer of Myovant Sciences, Inc. Concurrent with this appointment, Mr. Marek was also appointed Principal Executive Officer of Myovant Sciences Ltd. and a member of its Board of Directors.
On April 5, 2021, Myovant announced the appointment of Lauren Merendino as Chief Commercial Officer of Myovant Sciences, Inc. Ms. Merendino is also a member of Myovant’s Executive Committee.
Expected Upcoming Milestones
FDA decision for relugolix combination tablet for the treatment of uterine fibroids is expected by the June 1, 2021 target action date. If approved, Myovant and Pfizer expect to launch in the U.S. in June 2021. Upon FDA approval, Myovant will receive a $100.0 million regulatory milestone payment from Pfizer.
U.S. regulatory submission to the FDA for relugolix combination tablet for the treatment of women with endometriosis-associated pain is expected in the second quarter of calendar year 2021.
Pfizer’s decision regarding its exclusive option to acquire development and commercialization rights to relugolix in oncology outside of the U.S. and Canada (excluding certain Asian markets) is expected in mid-calendar year 2021. If Pfizer exercises this option, Myovant will receive a $50.0 million payment and will be eligible to receive double-digit royalties on net sales.
European Commission (EC) decision on the uterine fibroids MAA is expected in mid-calendar year 2021. If approved, this launch will be executed by Gedeon Richter Plc. (Richter), Myovant’s commercialization partner for relugolix combination tablet for the uterine fibroids and endometriosis indications in Europe and certain other international markets.
MAA submission to the EMA for relugolix combination tablet for the treatment of women with endometriosis-associated pain is expected in calendar year 2021. Richter will be the MAA sponsor.
EC decision on the advanced prostate cancer MAA is expected in calendar year 2022.
Fourth Fiscal Quarter and Fiscal Year Ended March 31, 2021 Financial Summary
Total revenues for the three months and year ended March 31, 2021, were $24.6 million and $59.3 million, respectively. There were no revenues recorded in the comparable prior year periods.
Product revenue, net from sales of ORGOVYX in the U.S. for the three months and year ended March 31, 2021 were $3.6 million.
Collaboration revenue for the three months and year ended March 31, 2021 was $21.0 million and $22.4 million, respectively, and represents partial amortization of the upfront payment received from Pfizer pursuant to the Pfizer Collaboration and License Agreement.
License and milestone revenue for the year ended March 31, 2021 was $33.3 million and represents the partial recognition of revenue associated with the $40.0 million upfront payment and a $10.0 million regulatory milestone payment received from Richter under the Richter Development and Commercialization Agreement.
Cost of product revenue for the three months and year ended March 31, 2021, was $0.3 million related to the cost of goods sold and royalty expense payable to Takeda pursuant to the Takeda License Agreement. There were no such expenses for the comparable prior year periods.
Collaboration expense to Pfizer for the three months and year ended March 31, 2021, was $1.7 million, reflecting Pfizer’s 50% share of net profits from sales of ORGOVYX in the U.S., pursuant to the Pfizer Collaboration and License Agreement. There were no such expenses for the comparable prior year periods.
Research and development (R&D) expenses for the three months ended March 31, 2021, were $21.6 million compared to $41.7 million for the comparable prior year period. R&D expenses for the year ended March 31, 2021, were $136.7 million compared to $192.6 million for the prior fiscal year. The decrease in R&D expenses reflects a reduction in clinical study costs as a result of the wind down of Myovant’s Phase 3 LIBERTY, HERO, and SPIRIT studies and cost share reimbursements from Pfizer for certain R&D expenses in the fiscal 2020 periods. This decrease was partially offset primarily by an increase in personnel expenses, mainly driven by the continued expansion of Myovant’s medical affairs organization to support the U.S. commercial launch of ORGOVYX and the potential U.S. commercial launches of relugolix combination tablet for the women’s health indications, if approved, as well as regulatory expenses and incremental spend on new relugolix development programs.
Selling, general and administrative (SG&A) expenses for the three months ended March 31, 2021, were $78.0 million compared to $22.4 million for the comparable prior year period. SG&A expenses in the year ended March 31, 2021, were $181.4 million compared to $82.3 million for the prior fiscal year. The increase was primarily due to higher expenses related to commercial activities to support the ORGOVYX U.S. launch and commercial readiness activities for the potential U.S. launch of relugolix combination tablet, higher personnel-related costs primarily due to the hiring of Myovant’s commercial operations, marketing, and market access teams, as well as the oncology sales force, higher share-based compensation expense, and general overhead expenses to support Myovant’s organizational growth. Share-based compensation expense for the three months and year ended March 31, 2021 includes incremental expense of $25.7 million related to the acceleration, modification, and remeasurement of our former Principal Executive Officer’s outstanding equity awards. SG&A expenses for the year ended March 31, 2020 included $10.2 million in share-based compensation expense related to the accelerated vesting of certain equity awards as well as a $3.6 million capital tax accrual.
Interest expense was $3.5 million for the three months ended March 31, 2021, compared to $1.4 million for the comparable prior year period. Interest expense was $10.4 million for the year ended March 31, 2021, compared to $12.7 million for the prior fiscal year. The decrease in interest expense, despite higher outstanding loan balances, was primarily driven by the significantly lower interest rates associated with the Sumitomo Dainippon Pharma Loan Agreement as compared to Myovant’s previously outstanding debt obligations, which were repaid in December 2019.
There was no loss on extinguishment of debt for the year ended March 31, 2021. For the year ended March 31, 2020, Myovant recorded a $4.9 million loss resulting from the early repayment of Myovant’s previously outstanding debt obligations in December 2019.
Interest income for the three months ended March 31, 2021, was less than $0.1 million compared to $0.2 million for the comparable prior year period. Interest income for the year ended March 31, 2021, was $0.2 million compared to $2.6 million for the prior fiscal year. The decrease was primarily due to decreases in interest rates.
Foreign exchange loss (gain) for the three months ended March 31, 2021, was a loss of less than $0.1 million compared to a gain of $0.5 million for the comparable prior year period. Foreign exchange gain for the year ended March 31, 2021, was $16.2 million compared to $1.6 million for the prior fiscal year. The increase for the year ended March 31, 2021 was primarily due to a larger foreign currency exchange gain on Myovant’s outstanding balance under the Sumitomo Dainippon Pharma Loan Agreement during the year ended March 31, 2021 compared to the prior year.
Net loss for the three months ended March 31, 2021, was $81.4 million compared to $64.9 million for the comparable prior year period. Net loss for the year ended March 31, 2021, was $255.1 million compared to $289.0 million for the prior fiscal year. On a per common share basis, net loss was $0.89 and $0.73 for the three months ended March 31, 2021 and 2020, respectively, and $2.83 and $3.37 for the year ended March 31, 2021 and 2020, respectively.
Capital resources: Cash, cash equivalents, marketable securities, and amounts available under the Sumitomo Dainippon Pharma Loan Agreement totaled $726.2 million as of March 31, 2021, and consisted of $684.9 million of cash, cash equivalents, and marketable securities and $41.3 million of available borrowing capacity under the Sumitomo Dainippon Pharma Loan Agreement.
Conference Call
As previously announced, Myovant will hold a webcast and conference call at 8:30 a.m. Eastern Time (5:30 a.m. Pacific Time) today, May 11, 2021, to discuss corporate updates and financial results for its fourth fiscal quarter and fiscal year ended March 31, 2021. Investors and the general public may access a live webcast of the call by visiting the investor relations page of Myovant’s website at investors.myovant.com. Institutional investors and analysts may also participate in the conference call by dialing 1-800-532-3746 in the U.S. or +1-470-495-9166 from outside the U.S. The webcast will be archived on Myovant’s Investor Relations website following the call.
About Relugolix
Relugolix is a once-daily, oral gonadotropin-releasing hormone (GnRH) receptor antagonist that reduces testicular testosterone, a hormone known to stimulate the growth of prostate cancer, and ovarian estradiol, a hormone known to stimulate the growth of uterine fibroids and endometriosis. Relugolix monotherapy (120 mg) is FDA-approved as ORGOVYX™ for the treatment of adult patients with advanced prostate cancer and is under regulatory review in Europe for the treatment of men with advanced prostate cancer. Relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg) is under regulatory review in the U.S. and Europe for women with uterine fibroids, has completed Phase 3 registration-enabling studies for women with endometriosis, and is being assessed for contraceptive efficacy in healthy women ages 18-35 years who are at risk for pregnancy.
About Myovant Sciences
Myovant Sciences (Myovant) aspires to redefine care for women and for men through purpose-driven science, empowering medicines, and transformative advocacy. ORGOVYX™ (relugolix) was approved by the FDA in 2020 as the first and only oral gonadotropin-releasing hormone (GnRH) receptor antagonist for the treatment of adult patients with advanced prostate cancer, and relugolix is also under regulatory review in Europe for men with advanced prostate cancer. Myovant’s lead product candidate, relugolix combination tablet (relugolix 40 mg, estradiol 1.0 mg, and norethindrone acetate 0.5 mg), is under regulatory review in the U.S. and Europe for women with uterine fibroids, has completed Phase 3 registration-enabling studies for women with endometriosis, and is being assessed for contraceptive efficacy in healthy women ages 18-35 years who are at risk for pregnancy. Myovant is also developing MVT-602, an oligopeptide kisspeptin-1 receptor agonist, which has completed a Phase 2a study for female infertility as part of assisted reproduction. Sumitovant Biopharma, Ltd., a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd., is Myovant’s majority shareholder. For more information, please visit Myovant’s website at www.myovant.com. Follow @Myovant on Twitter and LinkedIn.
About Sumitomo Dainippon Pharma Co., Ltd.
Sumitomo Dainippon Pharma is among the top-ten listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including the U.S., Japan, China, and the European Union. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at https://www.ds-pharma.com.
About Sumitovant Biopharma Ltd.
Sumitovant is a global biopharmaceutical company with offices in New York City and London. Sumitovant is the majority shareholder of Myovant, and wholly owns Urovant Sciences, Enzyvant Therapeutics, Spirovant Sciences, and Altavant Sciences. Sumitovant’s pipeline is comprised of commercialized and investigational medicines across a range of disease areas targeting high unmet need. Sumitovant is a wholly owned subsidiary of Sumitomo Dainippon Pharma. For further information about Sumitovant, please visit https://www.sumitovant.com.
Bladerunner
gfp,
re: FSTX
My cancer docs think bi-specific tetravalent technology could be a big thing in oncology. The financing was really crappy and I'm pissed. But I remain long. If I had any cash I would be a buyer here.
Bladerunner
Blade, Just curious if you see this as possibly a good buying opportunity for FSTX, with the stock trading down at/near the $7 stock offering price.
Thanks.
Looks like this financing more than doubles the shares outstanding, but that is coming off of tiny base, and the company should now have plenty of cash for a while. Thanks for any insights.
ONCT announces two abstracts for presentation at (virtual) ASCO
Oncternal Therapeutics Announces Presentation of Two Abstracts at ASCO 2021 Virtual Meeting
Oncternal Therapeutics
Wed, April 28, 2021, 7:00 AM
ONCT
+1.72%
SAN DIEGO, April 28, 2021 (GLOBE NEWSWIRE) -- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today announced the presentation of two abstracts at the American Society of Clinical Oncology (ASCO) 2021 Annual Meeting being held virtually from June 4-8, 2021.
Oral Presentation:
Abstract Number for Publication: 11500
Abstract Title: TK216 for relapsed/refractory Ewing sarcoma: Interim phase 1/2 results
Session Title: Sarcoma
Session Date and Time: June 4, 2021 from 1:30 pm - 4:30 pm Eastern Time
Poster Presentation:
Abstract Number for Publication: 7556
Abstract Title: Phase 1/2 study of cirmtuzumab and ibrutinib in mantle cell lymphoma (MCL) or chronic lymphocytic leukemia (CLL)
Poster Session: Hematologic Malignancies — Lymphoma and Chronic Lymphocytic Leukemia
Session Date and Time: June 7, 2021 at 11:30 am Eastern Time
About TK216
TK216 is an investigational, potentially first-in-class, targeted small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins including fusion proteins. Tumorigenic fusion proteins involving the EWS protein and an ETS protein can be found in most cases of Ewing sarcoma. ETS-related translocations or overexpression are also found in many other tumors such as prostate cancer and acute myeloid leukemia (AML). TK216 was developed based on discoveries in the laboratory of Jeffrey Toretsky, M.D., at Georgetown Lombardi Comprehensive Cancer Center, who discovered inhibitors of EWS-FLI1 using a novel chemical screening assay. In preclinical models, TK216 was observed to bind to EWS-FLI1, blocking the interaction between this fusion protein and other transcriptome proteins such as RNA helicase A, leading to tumor cell apoptosis and inhibiting tumor growth in animal models. The U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation, Orphan Drug Designation and Fast Track Status to TK216 for the treatment of Ewing sarcoma. TK216 is an investigational medication that has not been approved by the FDA for any indication.
About Cirmtuzumab
Cirmtuzumab is an investigational, potentially first-in-class monoclonal antibody targeting ROR1, or Receptor tyrosine kinase-like Orphan Receptor 1. Cirmtuzumab is currently being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of MCL or CLL, in a collaboration with the University of California San Diego (UC San Diego) School of Medicine and the California Institute for Regenerative Medicine (CIRM). In addition, Oncternal is supporting two investigator-sponsored studies being conducted at the UC San Diego School of Medicine: (i) a Phase 1b clinical trial of cirmtuzumab in combination with paclitaxel for the treatment of women with HER2-negative metastatic or locally advanced, unresectable breast cancer, and (ii) a Phase 2 clinical trial of cirmtuzumab in combination with venetoclax, a Bcl-2 inhibitor, in patients with relapsed/refractory CLL.
ROR1 is a potentially attractive target for cancer therapy because it is an onco-embryonic antigen – not usually expressed on adult cells, and its expression confers a survival and fitness advantage when reactivated and expressed by tumor cells. Researchers at the UC San Diego School of Medicine discovered that targeting a critical epitope on ROR1 was key to specifically targeting ROR1 expressing tumors. This led to the development of cirmtuzumab, that binds this critical epitope of ROR1, which is highly expressed on many different cancers but not on normal tissues. Preclinical data showed that when cirmtuzumab bound to ROR1, it blocked Wnt5a signaling, inhibited tumor cell proliferation, migration and survival, and induced differentiation of the tumor cells. The FDA has granted Orphan Drug Designations to cirmtuzumab for the treatment of MCL and CLL/small lymphocytic lymphoma. Cirmtuzumab is in clinical development and has not been approved by the FDA for any indication.
About Oncternal Therapeutics
Oncternal Therapeutics is a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies for the treatment of cancers with critical unmet medical need. Oncternal focuses drug development on promising yet untapped biological pathways implicated in cancer generation or progression. The clinical pipeline includes cirmtuzumab, an investigational monoclonal antibody designed to inhibit the ROR1 pathway, a type I tyrosine kinase-like orphan receptor, that is being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of patients with mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL) and in investigator-sponsored, Phase 1b clinical trial in combination with paclitaxel for the treatment of women with HER2-negative metastatic or locally advanced, unresectable breast cancer, as well as a Phase 2 clinical trial of cirmtuzumab in combination with venetoclax, a Bcl-2 inhibitor, in patients with relapsed/refractory CLL. We are also developing a chimeric antigen receptor T cell (CAR-T) therapy that targets ROR1, which is currently in preclinical development as a potential treatment for hematologic cancers and solid tumors. The clinical pipeline also includes TK216, an investigational targeted small-molecule inhibitor of the ETS family of oncoproteins, that is being evaluated in a Phase 1/2 clinical trial for patients with Ewing sarcoma alone and in combination with vincristine chemotherapy. More information is available at https://oncternal.com.
Bladerunner
Blade, Thanks. The daily swings in these bio stocks is enough to send a person to the medicine cabinet for some Tagamet or Prozac lol. They are interesting to follow but I've always had trouble owning them for any length of time.
You're right about Dew, he's the bio genius. I remember also being impressed with Biomaven's knowledge, and many of the other posters over there have excellent insights. I think you have probably made the most money at it though :o) It's amazing you haven't gone into semi-retirement and become super cautious/risk averse. My own past misadventures have made me leery of taking undue risk.
KZIA in-licenses global rights to cancer drug from Evotec
April 19, 2021 5:36 AM
KAZIA LICENSES GLOBAL RIGHTS TO EVT801, A NOVEL, FIRST-IN-CLASS, CLINIC-READY, VEGFR3 INHIBITOR, FROM EVOTEC SE
PR Newswire
SYDNEY , April 19, 2021 /PRNewswire/ -- Kazia Therapeutics Limited (ASX: KZA; NASDAQ: KZIA), an oncology-focused drug development company, is pleased to announce that it has entered into a worldwide exclusive licensing agreement and a master services agreement with Evotec SE (FRA: EVT), a leading European drug discovery and development company, for EVT801, a small-molecule, first-in-class oncology drug candidate. Kazia expects to launch a phase I clinical trial of EVT801 in CY2021.
Kazia Therapeutics Limited Logo (PRNewsfoto/Kazia Therapeutics Ltd)
Key Points
Evotec has granted Kazia an exclusive global worldwide license to develop, manufacture, and commercialise EVT801 in all territories and indications.
Under the terms of the agreement, Kazia will pay an immediate upfront of €1 million (AU$ 1.6 million), contingent milestones of up to €308 million (AU$ 480 million) related to achievement of clinical, regulatory, and commercial outcomes over the lifetime of the drug, and a tiered single-digit royalty on net sales.
Evotec is a leading drug discovery and development company, headquartered in Hamburg, Germany , and listed on the Frankfurt Stock Exchange.
EVT801 is a small-molecule inhibitor of VEGFR3. Its primary activity is to inhibit lymphangiogenesis, the formation of new lymphatic vessels around a growing tumour. By doing so, EVT801 is expected to starve the tumour of vital nutrients and to reduce metastasis. EVT801 also has marked activity on the immune system within the tumour and may therefore enhance the activity of immuno-oncology therapies.
Kazia and Evotec have also entered into a master services agreement, under which the two companies will collaborate closely on the further development of EVT801.
EVT801 was originally discovered by Sanofi (NASDAQ: SNY), the largest pharmaceutical company in France and among the five largest in the world and was developed through a partnership between Sanofi and Evotec.
Kazia expects to launch a phase I clinical trial in CY2021. The initial exploratory indications for EVT801 include renal cell carcinoma (kidney cancer), hepatocellular carcinoma (liver cancer), and soft tissue sarcoma.
Kazia CEO, Dr James Garner , commented, "We are delighted to add this tremendously exciting new compound to the Kazia pipeline. Evotec have done first-class work in the early development of EVT801, and the preclinical data package is exceptionally strong. We intend to fast track a phase I clinical trial of the drug, which we expect to commence in CY2021."
He added, "As we have built Kazia over the past five years, our strategy has been to assemble a portfolio of world-class development candidates through in-licensing. The EVT801 transaction is wholly consistent with that strategy. We have demonstrated, through the paxalisib program, our ability to add value to a development candidate, and we intend to similarly accelerate EVT801 via a rich and innovative development program."
Evotec CEO, Dr Werner Lanthaler , commented, "we are very pleased to partner with Kazia for this promising asset, for which we have high hopes. Our corporate strategy does not provide for Evotec to take EVT801 through clinical trials itself, so we have sought to identify a partner who can do justice to the drug's potential. We recognise Kazia's track record and look forward to working together to make EVT801 available to patients and clinicians."
EVT801
EVT801 is a small molecule inhibitor of vascular endothelial growth factor receptor 3 (VEGFR3). It is orally available, and so can be administered to patients by mouth.
For more than two decades, one of the most successful approaches in the treatment of cancer has been to target angiogenesis, the formation of new blood vessels. Drugs which inhibit angiogenesis, such as Avastin® (bevacizumab), starve the growing tumour of nutrients. However, inhibiting angiogenesis also results in hypoxia (low levels of oxygen) around the tumour, and this is thought to generate resistance to treatment. Almost all cancers treated with current anti-angiogenic drugs will eventually develop resistance.
An alternative approach, which may avoid this problem, is to target lymphangiogenesis, which is the formation of new lymphatic vessels. Doing so achieves many of the same objectives as targeting angiogenesis but may avoid the problem of resistance induced by hypoxia. Moreover, the lymphatic system is a common route by which tumours spread (metastasise) throughout the body, and so inhibiting lymphangiogenesis may help to limit the ability of the tumour to spread.
In recent years, several new drug candidates have attempted to inhibit lymphangiogenesis. For example, Nexavar® (sorafenib) inhibits several forms of VEGFR, as well as other targets, and is approved for the treatment of renal cell carcinoma and hepatocellular carcinoma. Several drugs described as angiokinase inhibitors are in development, and some of these inhibit VEGFR3. However, each of these drugs has multiple targets, leading in many cases to significant side effects. The distinguishing feature of EVT801 is a high degree of specificity for VEGFR3, which should allow it to minimise toxicity.
In addition, EVT801 has shown powerful evidence in the laboratory of an ability to change the balance of immune cells within the tumour. Many tumours are resistant to the newest generation of immuno-oncology therapies because they do not contain the right immune cells for the drugs to act upon. It is hoped that administration of EVT801 may help to sensitise these tumours to immuno-oncology therapies such as Keytruda® (pembrolizumab) and Opdivo® (nivolumab) and thereby extend their use.
Kazia expects to explore all these potential uses of EVT801 during the clinical program. The initial focus will be on a phase I study, which is expected to be conducted at one or more leading hospitals in France and to commence in CY2021.
Master Services Agreement
In parallel with the license agreement, Kazia and Evotec have entered into a Master Services Agreement (MSA), under which they will collaborate on the further development of EVT801. Kazia intends to utilise Evotec's substantial capabilities and expertise in research, clinical trial management, biomarker development, and manufacturing, to expedite the development of EVT801.
Investor Conference Call
Kazia is pleased to invite investors to attend a conference call to further discuss the EVT801 in-licensing.
The call will be held on Tuesday 20 April 2021 at 8:00am , Sydney time (AET), which is 6pm on Monday 19 April in New York (ET) and 3pm on Monday 19 April in San Francisco (PT).
Participants will need to pre-register for the call via the following link:
Registration Link: https://s1.c-conf.com/diamondpass/10013602-bric5f.html
Click the 'Register Now' button and follow the prompts to complete pre-registration. You will then receive a calendar invite with dial in numbers, a passcode and a PIN to dial into the conference call.
About Kazia Therapeutics Limited
Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA) is an oncology-focused drug development company, based in Sydney, Australia .
Our lead program is paxalisib, a brain-penetrant inhibitor of the PI3K / Akt / mTOR pathway, which is being developed to treat glioblastoma, the most common and most aggressive form of primary brain cancer in adults. Licensed from Genentech in late 2016, paxalisib commenced recruitment to GBM AGILE, a pivotal study in glioblastoma, in January 2021 . Seven additional studies are active in other forms of brain cancer. Paxalisib was granted Orphan Drug Designation for glioblastoma by the US FDA in February 2018 , and Fast Track Designation for glioblastoma by the US FDA in August 2020 . In addition, paxalisib was granted Rare Pediatric Disease Designation and Orphan Designation by the US FDA for DIPG in August 2020 .
Kazia is also developing EVT801, a small-molecule inhibitor of VEGFR3, which was licensed from Evotec SE in April 2021 . Preclinical data has shown EVT801 to be active against a broad range of tumour types and has provided compelling evidence of synergy with immuno-onocology agents. A phase I study is expected to begin in CY2021.
For more information, please visit www.kaziatherapeutics.com or follow us on Twitter @KaziaTx.
About Evotec SE
Evotec is a drug discovery alliance and development partnership company focused on rapidly progressing innovative product approaches with leading pharmaceutical and biotechnology companies, academics, patient advocacy groups and venture capitalists.
We operate worldwide and our more than 3,500 employees provide the highest quality stand-alone and integrated drug discovery and development solutions. We cover all activities from target-to-clinic to meet the industry's need for innovation and efficiency in drug discovery and development (EVT Execute). The Company has established a unique position by assembling top-class scientific experts and integrating state-of-the-art technologies as well as substantial experience and expertise in key therapeutic areas including neuronal diseases, diabetes and complications of diabetes, pain and inflammation, oncology, infectious diseases, respiratory diseases, fibrosis, rare diseases and women's health.
On this basis, Evotec has built a broad and deep pipeline of more than 100 co-owned product opportunities at clinical, pre-clinical and discovery stages (EVT Innovate). Evotec has established multiple long-term alliances with partners including Bayer, Boehringer Ingelheim, Bristol Myers Squibb, CHDI, Novartis, Novo Nordisk, Pfizer, Sanofi, Takeda, UCB and others. For additional information please go to www.evotec.com and follow us on Twitter @Evotec.
Bladerunner
gfp, I have no hard and fast rules for investing in biotech stocks. I recommend that before you invest in a any biotech that you consult with "Dew Diligence,' the most knowledgeable person in all of biotech and pharmaceuticals. He is brilliant at sniffing out scams in biotech, and he is especially adept at deciphering PR's and CC's for their "hidden" meanings. He can help keep you out of trouble in biotech and that should be your first requirement. (I'm not saying that you can't make money on scam biotechs, but, at least, you ought to know what you're getting into.)
I don't invest in medical device companies. I'm not interested in companies with drugs that are "enhanced" versions of chemotherapeutics. I don't invest in cancer vaccine companies. I'm extremely skeptical of small-cap biotechs that are tackling AD and Parkinson's, but I readily admit that those stocks can take off on the slightest good news.
Biotech is out of favor now, so I think there are some real bargains out there.
Bladerunner
Blade, Thanks. The volatility of these bio stocks is what gets me. I have trouble not taking profits when they appear, even though that could mean missing out on a great longer term stock.
I was wondering if there are some general ground rules that you use, for example selling 1/2 of a position if you get a double? And do you set a stop loss under your positions? Bio stocks mainly move on the clinical progress and news flow, and you look for well funded companies, which is a great parameter, but do you have certain hard/fast rules, or is it a more flexible approach? Any bio sub-sectors that you always avoid, etc?
Thanks for any insights.
gfp, I sold all my PRVB around $12.50. I had 100,000 shares with an average cost basis around $16 and change, so I took a big loss. I just thought there were better places for my money and I could use the tax loss.
Small and micro-cap biotechs are definitely out of favor now and I don't know when that will turn around, if ever.
I'm still holding ONCT and KZIA. My cancer docs love FSTX (needs cash) and MYOV (gotten very cheap). We all love MGTA. Cancer docs also like CRIS a lot. One doc likes MEIP and another one likes TRIL. I own a medium sized position in TRIL. I sold all of my MEIP a long time ago, but may consider re-entering.
I personally like CRVS. Cancer docs are lukewarm on it.
Besides FSTX, the big, big sleeper is BPTH. With a market cap around $45 million, it seems way undervalued. Of course, I said that when it was $7.
I think you should look at MGTA, FSTX and BPTH. All are very cheap and all have explosive upside.
Bladerunner
Blade, Just curious what your strategy is with PRVB in light of the recent developments? I see they have a meeting with the FDA on May 27, which hopefully will clarify things. At these lower levels the stock looks interesting if there can be a timely resolution to the PK/PD issues. But looks like a lot of uncertainty until there is some clarification.
Thanks for any insights.
KZIA also looks like it might be at a good re-entry point, as does ONCT. Any thoughts on these? Thanks.
Fwiw I luckily bailed on all the bio stocks back in Feb/Mar, along with other previously hot/trendy sectors, but some of these are now starting to look cheap. My strategy for the stock allocation is mainly the broad index ETFs (S+P 500), but now those are also looking over-extended. Buy/hold has definitely become more difficult in this market..
PFE has an interest in TRIL, says "Seeking Alpha"
https://seekingalpha.com/article/4419037-trillium-therapeutics-cd47-lead-evokes-pfizer-interest-tril?mail_subject=tril-trillium-therapeutics-cd47-lead-evokes-pfizer-s-interest&utm_campaign=rta-stock-article&utm_content=link-2&utm_medium=email&utm_source=seeking_alpha
Bladerunner
Conix, I'm not a chartist, so you'll have to tell me what these mean.
Bladerunner
Yikes, looks like trouble for PRVB.
I thought something seemed amiss recently with the downward price action, so sold my position last week. Charts aren't especially useful with bio stocks, but sometimes news can leak, or legitimate concerns that investors have can erode the stock price, so I always take the chart into consideration. Oh well, biotech is full of surprises, and that aspect will never change -
>>> Provention Bio Provides Regulatory Update on Biologics License Application for Teplizumab for the Delay or Prevention of Clinical Type 1 Diabetes in At-Risk Individuals
Yahoo Finance
April 8, 2021
https://finance.yahoo.com/news/provention-bio-provides-regulatory-biologics-200100870.html
- Conference call and webcast to be held today at 5:00 p.m. Eastern Time
RED BANK, N.J., April 8, 2021 /PRNewswire/ -- Provention Bio, Inc. (Nasdaq: PRVB), a biopharmaceutical company dedicated to intercepting and preventing immune-mediated disease, today announced that the Company received a notification on April 2, 2021 from the U.S. Food and Drug Administration (FDA), stating that, as part of its ongoing review of the Company's Biologic License Application (BLA) for teplizumab for the delay or prevention of clinical type 1 diabetes, the FDA has identified deficiencies that preclude discussion of labeling and post-marketing requirements/commitments at this time. The FDA stated in the correspondence that the notification does not reflect a final decision on the information under review.
Additionally, during an informal discussion on April 2, 2021 regarding the agenda for the upcoming Advisory Committee meeting scheduled for May 27, 2021, the FDA informed the Company that it had completed its review of the data and analysis submitted by the Company for its single, low-dose pharmacokinetic/pharmacodynamic (PK/PD) bridging study conducted in healthy volunteers. This study evaluated the PK/PD comparability of drug product originating from drug substance manufactured by AGC Biologics, which the Company plans to use for commercialization, and drug product originating from historic drug substance manufactured by Eli Lilly used for the TN-10 study submitted for the teplizumab BLA. The FDA indicated that based on the data it has reviewed to date, the Agency's position is that the PK profiles of the two drug products evaluated in the PK/PD bridging study were not comparable and that additional data would be required before the FDA's considerations could be satisfied. As a follow up, today, the FDA stated to the Company that it is willing to discuss these issues concurrently with its ongoing review.
The FDA intends to continue the review of clinical data submitted in the BLA and to conduct the Advisory Committee meeting, scheduled on May 27, 2021.
"While we believe the FDA's initial feedback will likely result in a delay in timelines within which teplizumab has the potential to be approved by FDA and be made available for at-risk T1D patients, we believe in the comparability of the drug product produced by our partner AGC biologics with Eli Lilly manufactured product. We look forward to working closely with the Agency to address its additional data requirement, so we can deliver teplizumab to patients as soon as possible," said Ashleigh Palmer, CEO and Co-Founder, Provention Bio. "Additionally, we remain enthusiastic about the clinical efficacy and safety data submitted in connection with the BLA in support of teplizumab's potential to address the high unmet needs of at-risk T1D patients and look forward to meeting with the FDA's Advisory Committee and hearing from patients, KOLs and other key stakeholders next month."
Conference Call and Webcast Information:
Provention Bio will discuss these business updates via conference call today at 5:00 pm ET. To access the call, please dial 1-877-870-4263 (domestic) or 1-412-317-0790 (international) ten minutes prior to the start time and ask to be connected to the "Provention Bio Call". An audio webcast will also be available on the "Events and Webcasts" page of the Investors section of the Company's website, www.proventionbio.com. An archived webcast will be available on the Company's website approximately two hours after the conference call.
About Teplizumab (PRV-031):
Teplizumab is an investigational anti-CD3 monoclonal antibody (mAb) with a filed BLA under Priority Review by the FDA for the delay or prevention of clinical type 1 diabetes (T1D) in at-risk individuals. More than 800 patients have received teplizumab in multiple clinical studies involving more than 1,000 subjects. In previous studies of newly diagnosed patients, teplizumab consistently demonstrated the ability to preserve beta-cell function, a measure of endogenous insulin production. It correspondingly reduced the need for exogenous insulin use. Teplizumab has been granted Breakthrough Therapy Designation by the FDA and PRIME designation by the European Medicines Administration. Provention is currently also evaluating teplizumab in patients with newly diagnosed insulin-dependent T1D (the Phase 3 PROTECT study).
About Provention Bio, Inc.:
Provention Bio, Inc. (Nasdaq: PRVB) is a biopharmaceutical company focused on advancing the development of investigational therapies that may intercept and prevent debilitating and life-threatening immune-mediated diseases. The Biologics License Application (BLA) for teplizumab, its lead investigational drug candidate, for the delay or prevention of clinical type 1 diabetes in at-risk individuals has been filed by the U.S. Food and Drug Administration (FDA). The Company's pipeline includes additional clinical-stage product candidates that have demonstrated in pre-clinical or clinical studies proof-of-mechanism and/or proof-of-concept in other autoimmune diseases, including celiac disease and lupus. Visit www.ProventionBio.com for more information and follow us on Twitter: @ProventionBio.
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Kazia also looking good, close to 15 today. Here's an analyst report covering the recent licensing deal for China -
https://kza.irmau.com/irm/PDF/3daa28a3-bff3-44dc-a95d-a89c0c01789b/BellPotterKaziaTherapeuticsChinaOutLicenseDeal
H. C. Wainwright raises price target on BPTH to $12. I've been buying more between $7.10 and $7.20.
--Analyst Actions: HC Wainwright Adjusts Price Target on Bio-Path Holdings to $12 From $10, Maintains Buy Rating
BY MT Newswires
— 11:11 AM ET 04/06/2021
11:11 AM EDT, 04/06/2021 (MT Newswires) -- (MT Newswires covers equity, commodity and economic research from major banks and research firms in North America, Asia and Europe. Research providers may contact us here: https://www.mtnewswires.com/contact-us)
Price: 7.40, Change: -0.23, Percent Change: -3.01
Bladerunner
Oppenheimer initiates coverage of ONCT with an "outperform" rating & a $14 price target.
--Analyst Actions: Oppenheimer Starts Oncternal Therapeutics at Outperform with $14 Price Target
BY MT Newswires
— 9:31 AM ET 04/07/2021
09:31 AM EDT, 04/07/2021 (MT Newswires) -- (MT Newswires covers equity, commodity and economic research from major banks and research firms in North America, Asia and Europe. Research providers may contact us here: https://www.mtnewswires.com/contact-us)
Price: 9.06, Change: +0.40, Percent Change: +4.62
Bladerunner
ONCT up 20% today on no news that I'm aware of. Here is the 1-year chart:
https://finance.yahoo.com/quote/ONCT?p=ONCT&.tsrc=fin-srch
ONCT is a favorite of many SI Biotech Contest portfolios.
Bladerunner
>>> Innovation Pharma Completes Interim Safety Data Review—DMC Approves Increased Dosing Frequency in Phase 2 Clinical Trial of Brilacidin in Hospitalized COVID-19 Patients
Innovation Pharmaceuticals Inc.
April 5, 2021
https://finance.yahoo.com/news/innovation-pharma-completes-interim-safety-113000256.html
Study drug dosing regimen to increase to 5 days (from 3 days) based on DMC recommendation
Enrollment has reached 25 percent in the Company’s ~120-patient Phase 2 COVID-19 clinical trial
WAKEFIELD, Mass., April 05, 2021 (GLOBE NEWSWIRE) -- Innovation Pharmaceuticals (OTCQB:IPIX) (“the Company”), a clinical stage biopharmaceutical company, today announced that an independent Data Monitoring Committee (DMC) completed its scheduled review of interim safety data in the Company’s randomized, double-blind, placebo-controlled Phase 2 clinical trial of Brilacidin for treatment of moderate-to-severe COVID-19 in hospitalized patients (see NCT04784897).
Upon reaching 25 percent enrollment (30 subjects), recruitment was paused and a pre-specified unblinded safety data review and evaluation was conducted by the DMC. Following their review, the DMC recommended increasing the dosing regimen of Brilacidin from 3 days to 5 days of treatment, as intended per the protocol, which the Company has since implemented. Recruitment for the 5-day treatment regimen has begun. Efficacy data was not reviewed by the DMC and remains blinded.
“We are thrilled with the recommendation of the DMC pertaining to the safety profile of Brilacidin, a drug that has repeatedly been shown in lab studies by virology experts as a top candidate for fighting infectious and resilient coronaviruses, including SARS-CoV-2, the pathogen responsible for COVID-19,” said Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals. “There is a sense of urgency to develop novel pan-coronavirus therapeutics for COVID-19, particularly given the growing spread of variants worldwide, including P1 and B.1.1.7, which appear to be contributing to cases of infection cropping up in patients who have already been vaccinated. With its unique mechanism of action to directly disrupt the novel coronavirus, we believe Brilacidin possesses potential to address these worrisome reminders that SARS-CoV-2 isn’t going anywhere. More broadly, Brilacidin’s antiviral potency also appears to extend beyond coronaviruses to other types of viruses, based on independent ongoing laboratory research, further supporting Brilacidin’s broad spectrum antiviral potential.”
The clinical rationale for consideration of expanding treatment duration to 5 days, from the initial dosing regimen of 3 days, is to provide a longer duration of systemic Brilacidin exposure at a level that can strongly suppress SARS-CoV-2 virus replication, and associated symptoms, and thus maximize therapeutic benefits to hospitalized patients with moderate or severe COVID-19. The expanded dosing regimen has particular value in that a 5-day dosing is the recommended initial treatment duration for Gilead Sciences’ Veklury® (remdesivir), the only FDA-approved antiviral drug for treatment of COVID-19. Trial data for Veklury could serve as a benchmark in evaluating Brilacidin outcomes.
The Company aims to expedite enrollment in the coming weeks toward study completion and release of topline results. Two sites, in addition to the 9 currently active and recruiting patients, have been initiated, with a further two sites planned to initiate within the next week.
Innovation Pharma is developing Brilacidin, a defensin-mimetic, under Fast Track designation from the U.S. Food and Drug Administration (FDA).
Alerts
Sign-up for Innovation Pharmaceuticals email alerts is available at:
http://www.ipharminc.com/email-alerts/
About Innovation Pharmaceuticals
Innovation Pharmaceuticals Inc. (IPIX) is a clinical stage biopharmaceutical company developing a world-class portfolio of innovative therapies addressing multiple areas of unmet medical need, including inflammatory diseases, cancer, infectious diseases, and dermatologic diseases.
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BPTH announces successful completion of safety cohort in a Phase II in AML.
(I thought the early results in this small study were very impressive, although not emphasized in the PR header.)
Stock up 36% in pre-Market.
April 5, 2021 7:00 AM
BIO-PATH HOLDINGS ANNOUNCES SUCCESSFUL COMPLETION OF SAFETY COHORT OF TRIPLE COMBINATION OF PREXIGEBERSEN, DECITABINE AND VENETOCLAX IN STAGE 2 OF PHASE 2 CLINICAL TRIAL IN ACUTE MYELOID LEUKEMIA
HOUSTON, April 05, 2021 (GLOBE NEWSWIRE) -- Bio-Path Holdings, Inc., (NASDAQ:BPTH), a biotechnology company leveraging its proprietary DNAbilize ® antisense RNAi nanoparticle technology to develop a portfolio of targeted nucleic acid cancer drugs, today announced the successful completion of the safety run-in of the Stage 2 of the Phase 2 clinical study of prexigebersen (BP1001), a liposomal Grb2 antisense, for the treatment of acute myeloid leukemia (AML), in combination with frontline therapies, decitabine and venetoclax, in acute myeloid leukemia (AML) patients. The safety run-in of Stage 2 of the Phase 2 clinical trial was comprised of six evaluable patients who were treated with the triple combination of prexigebersen, decitabine and venetoclax.
“We are particularly pleased with the clean side effect profile and lack of toxicity shown in this segment of the study, as our Phase 2 efficacy segment will include de novo fragile AML patients for whom drug side effect profiles are particularly important. We are also very encouraged by the efficacy signals shown in this dataset, with five of six evaluable relapsed, refractory and newly diagnosed AML patients demonstrating clinical activity. These positive signals give us further confidence in the potential for this program in these late-stage and compromised patients,” stated Peter H. Nielsen, Chief Executive Officer of Bio-Path Holdings.
“We look forward to advancing this Phase 2 study, as we believe its unique design provides us with several definable registration pathways. We believe that prexigebersen, with its promising efficacy and safety profile, has the potential to be an ideal combination candidate with frontline therapies,” concluded Mr. Nielsen.
In the safety run-in, six evaluable patients were treated with the combination of prexigebersen, decitabine and venetoclax. These patients included four relapsed/refractory AML patients, and two newly diagnosed AML patients. In the preliminary safety data review, five of the patients (83%) responded to treatment, including four (67%) achieving complete response (CR) and one (17%) complete response with incomplete hematologic recovery (CRi). CR rates to combination treatment with decitabine and venetoclax for relapsed/refractory AML patients is 42-52% 1,2 and 0-39% 1,2 for relapsed/refractory secondary AML patients. Response rates to frontline treatment decitabine and venetoclax for newly diagnosed AML patients is 62-71% 3 ,4 . These preliminary data showed the treatment was well-tolerated and there were no dose limiting toxicities attributed to prexigebersen. Three patients remained on treatment for more than one cycle.
Stage 2 of the Phase 2 clinical trial has three treatment cohorts, which the Company believes provides for several potential regulatory pathways. The first two cohorts will treat patients with the triple combination of prexigebersen, decitabine and venetoclax. The first cohort includes newly diagnosed AML patients and the second cohort includes relapsed/refractory AML patients. Finally, the third cohort treats relapsed/refractory AML patients who are venetoclax resistant or intolerant with the two-drug combination of prexigebersen and decitabine.
The Phase 2 clinical trial continues with 21 patients currently enrolled across all three cohorts. Enrollment of 19 patients in each cohort should enable a data review to determine if there is a comparative increase in efficacy versus the decitabine and venetoclax combination therapy sufficient to support petitioning the FDA for approval to switch to breakthrough therapy for accelerated approval. The Phase 2 trial will be conducted at up to ten clinical sites in the U.S. For more information on the Phase 2 study, visit www.clinicaltrials.gov .
Bladerunner
>>> Humanigen Reports Positive Phase 3 Topline Results Demonstrating That Lenzilumab™ Improves Survival Without Need for Mechanical Ventilation in Hospitalized Patients With COVID-19
Yahoo Finance
March 29, 2021
https://finance.yahoo.com/news/humanigen-reports-positive-phase-3-110000526.html
Lenzilumab improved the relative likelihood of survival without need for invasive mechanical ventilation (IMV) by 54%, achieving the primary endpoint of the Phase 3 study
Clinical improvement was observed over and above other treatments, including steroids and/or remdesivir
Humanigen, Inc. (Nasdaq: HGEN) ("Humanigen"), a clinical-stage biopharmaceutical company focused on preventing and treating an immune hyper-response called ‘cytokine storm’ with its lead drug candidate, lenzilumab, today announced positive topline results from its Phase 3 clinical trial evaluating the efficacy and safety of lenzilumab in patients hospitalized with COVID-19. Trial results showed that patients who received lenzilumab and other treatments, including steroids and/or remdesivir, had a 54% greater relative likelihood of survival without the need for IMV compared with patients receiving placebo and other treatments. These results are statistically significant.
This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20210329005301/en/
"The results from our Phase 3 clinical trial with lenzilumab treatment were associated with better outcomes in hospitalized hypoxic COVID-19 patients who had not yet progressed to the point of requiring IMV," said Cameron Durrant, MD, MBA, Chief Executive Officer, Humanigen. "Additionally, the trial incorporated a diverse population with various comorbidities, most commonly a body mass index above 30, which is representative of a real-world, high-risk population. Our next step is to submit an application for Emergency Use Authorization (EUA) to the Food and Drug Administration (FDA) as soon as possible. We are also sharing these results with US governmental agencies and other authorities worldwide."
"Mayo Clinic is pleased to have been part of the investigation of lenzilumab from the earliest days of the development program in COVID-19 and are excited by these data," said Andrew Badley, MD, Professor of Infectious Diseases, and Professor and Chair of the Department of Molecular Medicine at Mayo Clinic. "If lenzilumab is authorized for emergency use by FDA, and based on our clinical trial experience to date, it may then be considered a part of our treatment armamentarium for newly hospitalized patients with COVID-19."
Study results demonstrate that lenzilumab significantly improved patient outcomes. The study achieved its primary endpoint of ventilator-free survival measured through day 28 following treatment (HR: 1.54; 95%CI: 1.03-2.33, p=0.0365). Ventilator-free survival is a validated and reliable measure used in studies that evaluate respiratory distress.1 The Kaplan-Meier estimate for IMV and/or death was 15.6% (95%CI: 11.5-21.0) in the lenzilumab arm versus 22.1% (95%CI: 17.4-27.9) in the placebo arm, representing a 54% improvement in the relative likelihood of survival without the need for IMV. Although this study was not powered to demonstrate a difference in mortality, a favorable trend in mortality was observed: 9.6% (95%CI: 6.4-14.2) in the lenzilumab arm compared with 13.9% (95%CI: 10.1-19.0) in the placebo arm (HR: 1.39; 95%CI: 0.82-2.39; p=0.2287). Approximately 88% of patients received dexamethasone (or other steroids), 62% received remdesivir, and 57% received both, balanced across both arms of the study. Serious adverse events (SAEs) were balanced in both study arms and the SAE profile was similar to that previously documented in prior lenzilumab studies. In this study, lenzilumab appeared to be safe and well-tolerated; no new SAEs were identified, and none were attributed to lenzilumab.
"The data strongly suggest that lenzilumab improved outcomes for hospitalized patients with COVID-19 pneumonia," said Zelalem Temesgen, MD, Professor of Medicine at Mayo Clinic and Principal Investigator of the Phase 3 trial. "The dosing regimen used in this study was specifically designed for hospitalized patients with COVID-19 pneumonia as a potential foundational therapy. Lenzilumab could make the difference between going on a ventilator, which reduces one’s chance of survival, and leaving the hospital alive."
"It is impressive to see lenzilumab achieve this milestone. At Emory University, a key center in the National Institutes of Health (NIH) ACTIV-5 study, which is currently enrolling, we are hopeful that lenzilumab will emerge as a valuable therapy for newly hospitalized patients. We believe there may be future opportunities to study lenzilumab in even larger trials, and further explore lenzilumab’s impact on mortality rates," added Vincent Marconi, MD, Professor of Medicine at Emory University School of Medicine.
About the Lenzilumab Phase 3 Study
This study was a randomized, double-blind, placebo-controlled, multi-center Phase 3 trial for the treatment and prevention of serious and potentially fatal outcomes in patients who were hospitalized with COVID-19 pneumonia. The primary objective was to assess whether lenzilumab, in addition to other treatments, which included dexamethasone (or other steroids) and/or remdesivir, could alleviate the immune-mediated cytokine release syndrome (CRS) and improve ventilator-free survival. Ventilator-free survival is a composite endpoint of time to death and time to IMV, which is a robust measure that is less prone to favor a treatment with discordant effects on survival or days free of ventilation.1 The trial enrolled 520 patients in 29 sites in the US and Brazil who were at least 18 years of age; experienced blood oxygen saturation (SpO2) of less than or equal to 94%; or required low-flow supplemental oxygen, or high-flow oxygen support, or non-invasive positive pressure ventilation (NIPPV); and were hospitalized but did not require IMV. Following enrollment, subjects were randomized to receive three infusions of either lenzilumab or placebo, each infusion separated by eight hours over a 24-hour period with other treatments. The primary endpoint was the difference between lenzilumab treatment and placebo treatment in ventilator-free survival through 28 days following treatment. Key secondary endpoints, also measured through 28 days, included ventilator-free days, duration of ICU stay, incidence of invasive mechanical ventilation, extracorporeal membrane oxygenation (ECMO), and/or death, time to death, all-cause mortality, and time to recovery. Results of the trial are planned to be submitted for potential publication in a peer-reviewed journal.
About Humanigen, Inc.
Humanigen, Inc. is developing its portfolio of clinical and pre-clinical therapies for the treatment of cancers and infectious diseases via its novel, cutting-edge GM-CSF neutralization and gene-knockout platforms. Humanigen’s immediate focus is to prevent or minimize cytokine release syndrome that precedes severe lung dysfunction in hospitalized and hypoxic patients with COVID-19 pneumonia. Humanigen is also working to create next-generation combinatory gene-edited CAR-T therapies using strategies to improve efficacy while employing GM-CSF gene knockout technologies to control toxicity. In addition, Humanigen is developing its own portfolio of proprietary first-in-class EphA3-CAR-T for various solid cancers and EMR1-CAR-T for various eosinophilic disorders. Humanigen is also exploring the effectiveness of its GM-CSF neutralization technologies (either through the use of lenzilumab as a neutralizing antibody or through GM-CSF gene knockout) in combination with other CAR-T, bispecific or natural killer (NK) T-cell-engaging immunotherapy treatments to break the efficacy/toxicity linkage, including to prevent and/or treat Graft versus Host Disease (GvHD) in patients undergoing allogeneic hematopoietic stem cell transplantation (HSCT). Additionally, Humanigen and Kite, a Gilead Company, are evaluating lenzilumab in combination with Yescarta® (axicabtagene ciloleucel) in patients with relapsed or refractory large B-cell lymphoma in a clinical collaboration. For more information, visit www.humanigen.com and follow Humanigen on LinkedIn, Twitter, and Facebook.
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KZIA licenses Paxalisib to Simcere for Greater China
https://kza.irmau.com/irm/PDF/4d768237-e0f5-4b62-8f07-96257d93c124/KazialicensespaxalisibtoSimcereinGreaterChina
Bladerunner
>>> Surface Oncology Reports Financial Results and Corporate Highlights for Fourth Quarter and Full Year 2020
Yahoo Finance
Surface Oncology, Inc.
March 9, 2021
https://finance.yahoo.com/news/surface-oncology-reports-financial-results-120100357.html
New Collaboration with Merck Will Evaluate SRF388, Targeting IL-27, in Combination with KEYTRUDA® (pembrolizumab) in Patients with Solid Tumors
CAMBRIDGE, Mass., March 09, 2021 (GLOBE NEWSWIRE) -- Surface Oncology (Nasdaq: SURF), a clinical-stage immuno-oncology company developing next-generation therapies that target the tumor microenvironment, today reported financial results and corporate highlights for the fourth quarter and full year 2020, as well as anticipated 2021 corporate milestones.
“The fourth quarter of 2020 was transformational for Surface. During this quarter, we provided encouraging clinical data from our lead candidates and validated our preclinical discovery capabilities with a second major outlicense agreement that provided the company with substantial financial flexibility for several years,” said Rob Ross, M.D., incoming chief executive officer. “Today, we are also announcing another clinical trial collaboration further enabling rapid assessment of our lead product candidates (in this case SRF388) in combination with pembrolizumab, focusing on patients with liver and kidney cancers. As we look forward into 2021, we are targeting ASCO in June to share additional clinical data on SRF617 and SRF388, and we will work with our partner, GSK, to advance SRF813 into the clinic.”
Recent Corporate Highlights:
On March 9, 2021, Surface announced a clinical trial collaboration with Merck to evaluate the safety and efficacy of combining Surface’s SRF388, an investigational antibody therapy targeting IL-27, with Merck’s KEYTRUDA® (pembrolizumab), the first anti-PD-1 therapy approved in the United States. This combination will be studied as a component of the first-in-human Phase 1 study of SRF388 and will be evaluated in patients with solid tumors, with a focus on patients with liver cancer and kidney cancer.
In December, Surface announced an agreement for GlaxoSmithKline (GSK) to exclusively license worldwide development and commercial rights to Surface Oncology’s preclinical program SRF813, a fully human IgG1 antibody targeting PVRIG (also known as CD112R), an inhibitory protein expressed on natural killer cells (NK cells) and T cells. Under the terms of the agreement, GSK made an $85 million upfront payment in December 2020. In addition, Surface Oncology may receive up to an additional $730 million in future milestone payments, as well as be eligible to receive tiered royalties on global net sales.
In November, Surface announced that both of its lead clinical programs, SRF617 (targeting CD39) and SRF388 (targeting IL-27), have achieved predefined criteria for advancement into combination and expansion stages of the ongoing Phase 1 trials. These criteria include acceptable safety profiles at biologically relevant doses, as well as demonstration of target engagement and meaningful pharmacodynamic activity in the ongoing Phase 1 trials.
Effective April 1, 2021, Rob Ross, M.D., who has served as chief medical officer at Surface Oncology since 2016, will become the company’s president and chief executive officer and will also be appointed to the board of directors. Rob will succeed current CEO Jeff Goater, who will assume the role of chairman of the Surface Oncology board of directors.
Selected Anticipated Near-term Corporate Milestones:
Preclinical data presentations for SRF617 and SRF388 at the American Association for Cancer Research (AACR) Virtual Annual Meeting in April.
Targeting the American Society of Clinical Oncology (ASCO) Virtual Annual Meeting in June for detailed clinical data presentations for SRF617 and SRF388.
Investigational new drug (IND) filing for SRF813 anticipated in 2021.
Financial Results:
As of December 31, 2020, cash, cash equivalents and marketable securities were $175.1 million, compared to $105.2 million on December 31, 2019.
Revenue recognized in the fourth quarter ended December 31, 2020 was $87.6 million, compared to revenue of less than $1.0 million for the same period in 2019. The increase was a result of the $85 million upfront payment received in the fourth quarter 2020 from GSK. Revenue recognized in the full year ended December 31, 2020 was $126.2 million, compared to $15.4 million for the same period in 2019. The increase was a result of the $85 million upfront payment received in the fourth quarter 2020 from GSK, as well as the expiration of the final Novartis option purchase period in January 2020 and the corresponding recognition of the remaining deferred revenue under the agreement.
Research and development (R&D) expenses were $10.7 million for the fourth quarter ended December 31, 2020, compared to $11.7 million for the same period in 2019. R&D expenses were $41.0 million for the full year ended December 31, 2020, compared to $52.1 million for the same period in 2019. This decrease was primarily driven by a reduction in expenses associated with contract manufacturing and other IND-enabling activities, as a result of the SRF617 and SRF388 IND filings in 2019, offset by an increase in spend on the SRF617 and SRF388 Phase 1 clinical trials, which began in 2020. R&D expenses included $2.8 million in stock-based compensation expense for the full year ended December 31, 2020.
General and administrative (G&A) expenses were $8.9 million for the fourth quarter ended December 31, 2020, compared to $5.1 million for the same period in 2019. G&A expenses were $23.6 million for the full year ended December 31, 2020, compared to $20.6 million for the same period in 2019. This increase was primarily due to increased consulting costs related to the GSK Agreement, as well as increased stock-based compensation expense and bonus achieved in 2020. G&A expenses included $4.9 million in stock-based compensation expense for the full year ended December 31, 2020.
For the fourth quarter ended December 31, 2020, net income was $67.3 million, or basic net income per share attributable to common stockholders of $1.66, and diluted net income per share attributable to common stockholders of $1.56. Net loss was $16.0 million for the same period in 2019, or basic and diluted net loss per share attributable to common stockholders of $0.57. For the full year ended December 31, 2020 net income was $59.3 million, or basic net income per share attributable to common stockholders of $1.67, and diluted net income per share attributable to common stockholders of $1.57. Net loss was $54.8 million for the same period in 2019, or basic and diluted net loss per share attributable to common stockholders of $1.97.
Financial Outlook:
Based upon our current operating plan, Surface continues to project cash runway sufficient through 2023.
About Surface Oncology:
Surface Oncology is an immuno-oncology company developing next-generation antibody therapies focused on the tumor microenvironment. Its pipeline includes two wholly-owned clinical-stage programs targeting CD39 (SRF617) and IL-27 (SRF388), as well as a preclinical program focused on depleting regulatory T cells via targeting CCR8 (SRF114). In addition, Surface has two partnerships with major pharmaceutical companies: a collaboration with Novartis targeting CD73 (NZV930; Phase 1) and a collaboration with GlaxoSmithKline targeting PVRIG (SRF813; preclinical). Surface’s novel cancer immunotherapies are designed to achieve a clinically meaningful and sustained anti-tumor response and may be used alone or in combination with other therapies. For more information, please visit www.surfaceoncology.com.
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Curis Reports Fourth Quarter and Year-End 2020 Financial Results
Tue March 16, 2021 4:01 PM|PR Newswire|About: CRIS
- Presented CA-4948 Phase 1 data showing broad clinical activity in patients with R/R AML and MDS and Phase 1 data in patients with R/R non-Hodgkin's lymphoma (NHL) showing durable and dose-dependent reductions in tumor burden -
- Raised $169.6M of gross proceeds in December 2020 public offering; extending cash runway into 2024 -
- Multiple data readouts expected in 2021 from CA-4948 and anti-VISTA antibody CI-8993 clinical trials -
- Management to host conference call today at 4:30 p.m. ET -
PR Newswire
LEXINGTON, Mass., March 16, 2021 /PRNewswire/ -- Curis, Inc. (CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, today reported its financial results for the fourth quarter and year ended December 31, 2020.
"2020 was a transformative year for Curis, as we made significant progress in our mission to develop the next generation of targeted cancer therapies that meaningfully improve and extend the lives of patients. Despite the difficulties and uncertainty brought about by the ongoing coronavirus pandemic, we significantly advanced and expanded each program in our clinical pipeline, headlined by the very encouraging data from our Phase 1 trials of lead asset, CA-4948, presented in December in conjunction with ASH," said James Dentzer, President and Chief Executive Officer of Curis. "We look forward to providing additional updates on our IRAK4 program throughout the year, with clinical data readouts from our ongoing studies, including the CA-4948 monotherapy study in AML/MDS and the CA-4948/ibrutinib combination study for patients with R/R hematologic malignancies that we initiated in early 2021. We also continue to enroll patients and bring additional trial sites online in our Phase 1a/1b trial of CI-8993, our first-in-class monoclonal anti-VISTA antibody for the treatment of patients with R/R solid tumors, and look forward to providing preliminary data from this exciting study later this year."
Mr. Dentzer continued, "2020 was also a pivotal year on the corporate side for Curis. Through the execution of several key financings and partnerships, we have the resources needed to advance our programs through their next data catalysts, while also providing us the ability to invest efficiently in our pipeline of first-in-class cancer therapeutics. We are excited about the opportunities stemming from our Q4 signing of the CRADA with the NCI in addition to the recently announced Phase 2 IST of CA-4948 for the treatment of anemia in patients with lower-risk MDS led by Dr. Uwe Platzbecker at Universität Leipzig. These new partnerships provide powerful validation of our IRAK4 platform and allow us to leverage the resources of premier research organizations to significantly expand the reach of our clinical and preclinical programs."
Fourth Quarter 2020 and Recent Operational Highlights
Precision oncology, CA-4948 (IRAK4 Inhibitor; Aurigene collaboration):
• In December 2020, Curis announced positive preliminary data from its ongoing Phase 1 study of CA-4948 monotherapy in patients with R/R AML and high-risk MDS, including marrow blast reductions observed in all evaluable patients and 2 of 6 evaluable patients experiencing a marrow complete response. Curis continues to enroll patients, is currently enrolling in the 500mg BID dose cohort of the study and expects to report additional data in mid-year 2021.
• In December 2020, Curis provided updated preliminary data from its ongoing Phase 1 study of CA-4948 showing durable and dose-dependent reductions in tumor burden in patients with R/R NHL and announced the recommended Phase 2 dose, in addition to the identification of two potentially predictive biomarkers demonstrating target engagement and potential for patient enrichment, in an oral presentation at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition.
• In February 2021, Curis announced the dosing of the first patient in its Phase 1 dose-escalation and expansion study of CA-4948, an IRAK4 kinase inhibitor, and the BTK inhibitor, ibrutinib, for the treatment of patients with relapsed or refractory hematologic malignancies. In preclinical models, CA-4948 demonstrated synergistic anti-cancer activity when combined with a potent BTK inhibitor such as ibrutinib.
• Approximately 18 patients will be enrolled in the dose-escalation portion and will receive starting dose and escalation doses that have been observed to be safe and effective, combined with ibrutinib doses appropriate for their respective NHL subtype.
• The primary endpoints of Part 1 will be determination of maximum tolerated dose (MTD), and the recommended Phase 2 dose (RP2D).
• Part 2 of the study will enroll patients across a basket of four cohorts:
• Marginal zone lymphoma (MZL)
• Activated B-cell subtype of Diffuse Large B-cell Lymphoma (ABC-DLBCL)
• Primary central nervous system lymphoma (PCNSL)
• NHL with adaptive ibrutinib resistance.
• An interim futility analysis will be conducted after approximately 15-20 patients are enrolled in each cohort.
• Primary endpoints of Part 2 will be complete response or objective response rate and duration of response.
• Curis expects to report initial data from the study in the fourth quarter of 2021.
• In February 2021, Curis announced the initiation of the investigator-sponsored Phase 2 LUCAS trial of CA-4948 for the treatment of anemia in patients with very low, low, or intermediate-risk MDS. The trial is expected to start recruitment in the second quarter of 2021 and is expected to enroll 84 patients across two cohorts:
• Cohort A: Erythropoiesis stimulating agent (ESA) refractory/intolerant patients
• Cohort B: ESA naïve patients with transfusion dependence (min. 20 patients) or transfusion independence (min. 20 patients)
Patients in both cohorts will receive 300mg CA-4948 twice-daily (BID) for 21 days in at least four repeating cycles lasting 28 days each.
• The primary endpoint of the study is to evaluate the proportion of patients that develop an erythroid response (HI-E) according to IWG 2018 criteria.
Immuno (IMGN)-oncology, CI-8993 (anti-VISTA antibody; ImmuNext collaboration):
• In November 2020, Curis published trial design details from its ongoing Phase 1a/1b dose-escalation study of its first-in-class monoclonal anti-VISTA antibody for the treatment of R/R solid tumors.
• Curis continues to enroll patients in the study and expects to report initial safety and efficacy data in the second half of 2021.
Corporate:
• In December 2020, Curis closed an underwritten public offering of 29,500,000 shares of its common stock, including the exercise in full by the underwriters of their option to purchase up to an additional 3,847,826 shares, raising gross proceeds of approximately $169.6 million before deducting underwriting discounts and commissions and offering expenses.
• In November 2020, Curis entered into a CRADA with the NCI. Under the CRADA, Curis will collaborate with the NCI Experimental Therapeutics Program (NExT) and the NCI Cancer Therapy Evaluation Program to conduct non-clinical and clinical studies of Curis' proprietary compound, CA-4948, an IRAK4 kinase inhibitor that acts as a Toll-like Receptor (TLR) suppressor, as an anti-cancer agent.
Upcoming 2021 Planned Milestones
• Report additional clinical data from the Phase 1 study of CA-4948 in patients with AML and high-risk MDS, including patients with spliceosome mutations that encode oncogenic IRAK4-L in mid-year 2021.
• Report additional clinical biomarker data gathered in the Phase 1 study of CA-4948 in patients with R/R NHL in mid-year 2021.
• Announce initial safety and efficacy data from the ongoing Phase 1 study of CA-4948 in combination with ibrutinib in patients with R/R NHL in the second half of 2021.
• Report initial safety and efficacy data from the ongoing Phase 1a/1b dose-escalation study of CI-8993 for the treatment of R/R solid tumors in the second half of 2021.
Full Year and Fourth Quarter 2020 Financial Results
For the year ended December 31, 2020, Curis reported a net loss of $29.9 million, or $0.61 per share on both a basic and diluted basis, as compared to a net loss of $32.1 million, or $0.97 per share on both a basic and diluted basis in 2019. For the fourth quarter of 2020, Curis reported a net loss of $7.5 million or $0.11 per share on both a basic and diluted basis, as compared to a net loss of $8.6 million, or $0.26 per share on both a basic and diluted basis for the same period in 2019.
Revenues for the year ended December 31, 2020, were $10.8 million as compared to $10.0 million for the same period in 2019. Revenues for both periods comprise primarily royalty revenues recorded on Genentech and Roche's net sales of Erivedge®. Revenues for the fourth quarters of 2020 and 2019 were $3.0 million and $3.3 million, respectively.
Operating expenses for the year ended December 31, 2020 were $35.7 million as compared to $34.4 million for the same period in 2019. Operating expenses for the fourth quarter of 2020 were $9.3 million, as compared to $10.6 million for the same period in 2019, and comprised the following:
Costs of Royalty Revenues. Costs of royalty revenues, primarily amounts due to third-party university patent licensors in connection with Genentech and Roche's Erivedge net sales, were $0.5 million for the years ended December 31, 2020 and 2019. Cost of royalty revenues were $0.2 million for the fourth quarter of 2020 and 2019.
Research and Development Expenses. Research and development expenses were $23.1 million for the year ended December 31, 2020, as compared to $22.3 million for the same period in 2019. Research and development expenses were $5.6 million for the fourth quarter of 2020 as compared to $7.5 million for the same period in 2019. The decrease was primarily due to a decrease in clinical and manufacturing costs related to CA-170 and fimepinostat.
General and Administrative Expenses. General and administrative expenses were $12.1 million for the year ended December 31, 2020, as compared to $11.6 million for the same period in 2019. General and administrative expenses were $3.5 million for the fourth quarter of 2020, as compared to $3.0 million for the same period in 2019. The increase was primarily due to an increase in personnel related costs.
Other Expense, Net. Net other expense was $5.0 million for the year ended December 31, 2020, as compared to $7.8 million for the same period in 2019. For the fourth quarter of 2020 and 2019, net other expense was $1.2 million and $1.3 million, respectively. Net other expense primarily consisted of imputed interest expense related to future royalty payments.
As of December 31, 2020, Curis's cash, cash equivalents and investments totaled $183.1 million, and there were approximately 91.5 million shares of common stock outstanding. Curis expects that its existing cash, cash equivalents and investments should enable it to maintain its planned operations into 2024.
Conference Call Information
Curis management will host a conference call today, March 16, 2021, at 4:30 p.m. ET, to discuss these financial results, as well as provide a corporate update.
To access the live conference call, please dial 1-888-346-6389 from the United States or 1-412-317-5252 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed on the Curis website at www.curis.com in the Investors section.
About Curis, Inc.
Curis is a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer. In 2015, Curis entered into a collaboration with Aurigene in the areas of immuno-oncology and precision oncology. As part of this collaboration, Curis has exclusive licenses to oral small molecule antagonists of immune checkpoints including the VISTA/PDL1 antagonist CA-170, and the TIM3/PDL1 antagonist CA-327, as well as the IRAK4 kinase inhibitor, CA-4948. CA-4948 is currently undergoing testing in a Phase 1 in patients with non-Hodgkin's lymphoma both as a monotherapy and in combination the with BTK inhibitor ibrutinib. Curis is also evaluating CA-4948 in a Phase 1 trial in patients with acute myeloid leukemia and myelodysplastic syndromes. In addition, Curis is engaged in a collaboration with ImmuNext for development of CI-8993, a monoclonal anti-VISTA antibody, which is currently undergoing testing in a Phase 1a/1b trial in patients with solid tumors. Curis is also party to a collaboration with Genentech, a member of the Roche Group, under which Genentech and Roche are commercializing Erivedge® for the treatment of advanced basal cell carcinoma. For more information, visit Curis' website at www.curis.com.
Bladerunner
>>> Oncternal Therapeutics Provides Business Update and Announces Fourth Quarter and Full Year 2020 Financial Results
Yahoo Finance
Oncternal Therapeutics
March 11, 2021
https://finance.yahoo.com/news/oncternal-therapeutics-provides-business-announces-210100905.html
Interim Phase 1/2 results for cirmtuzumab with ibrutinib in MCL presented at ASH in December 2020 compare favorably to historical single-agent ibrutinib data (47% CR vs. 20% CR historical single-agent ibrutinib)
Accelerating development of ROR1-targeting CAR-T cell therapies
Two durable complete responses in patients with metastatic relapsed/refractory Ewing sarcoma treated with TK216 in ongoing Phase 1/2 clinical trial
Appointed Edwina Baskin-Bey, M.D., as Acting Chief Medical Officer
Management to host webcast today at 5:00 pm ET
SAN DIEGO, March 11, 2021 (GLOBE NEWSWIRE) -- Oncternal Therapeutics, Inc. (Nasdaq: ONCT), a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies, today provided a business update and reported fourth quarter and full year 2020 financial results.
“In 2021, we are advancing a deep pipeline of differentiated oncology assets. We have now initiated early-stage work in ROR1-targeting immunotherapies, including CAR-T and CAR-NK cell therapies, while moving forward the later-stage clinical development of cirmtuzumab, an antibody targeting ROR1, in MCL, which has generated encouraging data in this difficult to treat cancer. At the same time, we continue to evaluate TK216, an ETS inhibitor which has generated promising results in Ewing sarcoma,” said James Breitmeyer, M.D., Ph.D., Oncternal’s President and CEO. “We have also strengthened our balance sheet by raising $125 million in 2020, which provides us with the runway to advance our promising programs into 2023. We have several key data read-outs pending in the second quarter this year.”
Recent Highlights
In January 2021, we announced an agreement with Lentigen Technology, Inc., a wholly-owned subsidiary of Miltenyi Biotec B.V. & Co. KG, to manufacture lentiviral vectors to support Oncternal’s investigational ROR1-targeting CAR-T cell therapy program.
In January 2021, we announced a research and development collaboration with Karolinska Institutet in Stockholm, Sweden, to advance novel ROR1-targeting CAR-T and CAR-NK cell therapies from the laboratory into the clinic.
In December 2020, we announced an interim clinical data update from the ongoing Phase 1/2 clinical trial of cirmtuzumab, an investigational anti-ROR1 monoclonal antibody, in combination with ibrutinib in patients with mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL) at the American Society of Hematology 2020 Virtual Annual Meeting. Best objective response rate of 87%, including complete response (CR) rate of 47%, was reported for 15 evaluable patients with relapsed/refractory (r/r) MCL. Median progression-free survival (PFS) was not reached for patients with MCL, with the 95% confidence interval above 17.5 months, after a median follow-up of 12.1 months. The median PFS was not reached for patients with treatment-naïve CLL (n=19) after a median follow-up of 16.6 months, and median PFS was 29.5 months for patients with r/r CLL (n=30) after a median follow-up of 17.1 months. The combination of cirmtuzumab and ibrutinib was well tolerated in this trial.
In November 2020, we announced an interim clinical data update from the ongoing Phase 1/2 clinical trial evaluating TK216, an investigational, potentially first-in-class, targeted small-molecule inhibitor of the E26 transformation-specific (ETS) family of oncoproteins, in patients with r/r Ewing sarcoma at the Connective Tissue Oncology Society 2020 Virtual Annual Meeting. The reported disease control rate (CR, partial response or stable disease) was 43%, including two patients with durable complete responses that were ongoing at over 1.5 years and 8 months on treatment.
In October 2020, we announced that the U.S. Food and Drug Administration granted Rare Pediatric Disease designation for TK216 for the treatment of Ewing sarcoma.
In November and December 2020, we raised an aggregate of approximately $109 million in gross proceeds from two underwritten offerings.
Expected Upcoming Milestones
Cirmtuzumab (ROR1 antibody) programs
Clinical data update for patients with MCL and CLL treated with cirmtuzumab plus ibrutinib in the ongoing Phase 1/2 study in Q2 2021
Clinical data update for patients with HER2-negative breast cancer in the ongoing Phase 1b study in Q2 2021
Preclinical data in additional ROR1 expressing tumors in Q2 2021
ROR1 CAR-T program
First-in-human dosing in China in the second half of 2021
TK216 (ETS inhibitor) program
Clinical data for patients with Ewing sarcoma treated in the ongoing Phase 1/2 expansion cohort in Q2 2021
Preclinical data in additional ETS-driven tumors in Q2 2021
Fourth Quarter and Full Year 2020 Financial Results
Our grant revenue was $1.6 million for the fourth quarter ended December 31, 2020. Our grant revenue is derived from a subaward under a grant from the California Institute for Regenerative Medicine (CIRM) to UC San Diego, which was awarded to advance our Phase 1/2 clinical trial evaluating cirmtuzumab in combination with ibrutinib for the treatment of patients with MCL or CLL. For the full year 2020, grant revenue was $3.4 million.
Our total operating expenses for the fourth quarter ended December 31, 2020 were $4.4 million. Research and development expenses for the quarter totaled $3.0 million, and general and administrative expenses for the quarter totaled $1.5 million. Net loss for the fourth quarter was $2.6 million, or a loss of $0.09 per share, basic and diluted. For the full year 2020, total operating expenses were $20.9 million. Net loss for the full year 2020 was $17.2 million, or a loss of $0.85 per share, basic and diluted.
As of December 31, 2020, we had $116.7 million in cash and cash equivalents. We believe these funds will be sufficient to fund our operations into 2023. As of December 31, 2020, we had approximately 48.8 million shares of common stock outstanding.
Management Webcast
As previously announced, Oncternal will host a webcast today, March 11, 2020, at 5:00 p.m. ET. The live webcast will be available online and may be accessed from the “Investors” page of the company website at http://investor.oncternal.com/. A replay of the webcast will be available beginning approximately one hour after the conclusion of the call and will remain available for at least 30 days thereafter.
About Oncternal Therapeutics
Oncternal Therapeutics is a clinical-stage biopharmaceutical company focused on the development of novel oncology therapies for the treatment of cancers with critical unmet medical need. Oncternal focuses drug development on promising yet untapped biological pathways implicated in cancer generation or progression. The clinical pipeline includes cirmtuzumab, an investigational monoclonal antibody designed to inhibit the ROR1 pathway, a type I tyrosine kinase-like orphan receptor, that is being evaluated in a Phase 1/2 clinical trial in combination with ibrutinib for the treatment of patients with mantle cell lymphoma (MCL) and chronic lymphocytic leukemia (CLL) and in an investigator-sponsored, Phase 1b clinical trial in combination with paclitaxel for the treatment of women with HER2-negative metastatic or locally advanced, unresectable breast cancer. The clinical pipeline also includes TK216, an investigational targeted small-molecule inhibitor of the ETS family of oncoproteins, that is being evaluated in a Phase 1 clinical trial for patients with Ewing sarcoma alone and in combination with vincristine chemotherapy. In addition, Oncternal has a program utilizing the cirmtuzumab antibody backbone to develop a CAR-T therapy that targets ROR1, which is currently in preclinical development as a potential treatment for hematologic cancers and solid tumors. More information is available at www.oncternal.com.
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Bladerunner, Midastouch, Just wondering if you have any opinions on Enlivex Therapeutics (ENLV)? Thanks
>>> Has Israel just found the cure for Covid?
Experimental Covid drug cures 30 out of 30 moderate to severe cases in Phase I clinical trial at Israeli hospital. Second new drug also shows promise.
Israel 21c
By Abigail Klein Leichman
FEBRUARY 7, 2021
https://www.israel21c.org/has-israel-just-found-the-cure-for-covid/
Even with Israel’s world-leading rollout of Covid-19 vaccinations, drugs to treat Covid patients are in desperate need across the world.
Two such drugs developed in Israel show great promise in clinical trials: EXO-CD24 and Allocetra.
EXO-CD24, an experimental inhaled medication developed at Tel Aviv Sourasky Medical Center, cured all 30 moderate-to-severe cases in a Phase I clinical trial.
Developed over the past six months at the hospital, EXOCD24 stops the “cytokine storm” – where the immune system goes out of control and starts attacking healthy cells – that occurs in the lungs of 5-7% of Covid-19 patients.
“To date, the preparation has been tried with great success on 30 severe patients, in 29 of whom the medical condition improved within two to three days and most of them were discharged home within three to five days. The 30th patient also recovered but after a longer time,” the hospital reports.
“The drug is based on exosomes, [vesicles] that are released from the cell membrane and used for intercellular communication. We enrich the exosomes with 24CD protein. This protein is expressed on the surface of the cell and has a known and important role in regulating the immune system,” explained Dr. Shiran Shapira, director of the laboratory of Prof. Nadir Arber, who has been researching the CD24 protein for over two decades.
“The preparation is given by inhalation, once a day, for only a few minutes, for five days,” Shapira said.
She said the experimental treatment has two unique characteristics. The first is that it inhibits the over-secretion of cytokines. The second is that it is delivered directly to the lungs and therefore has no systemic side effects that injected or oral drugs can cause.
“Even if the vaccines perform their function, and even if no new mutations are produced then still in one way or another the corona will remain with us,” said Arber, director of the medical center’s Integrated Cancer Prevention Center. “To this end, we have developed a unique drug, EXO-CD24.”
Arber added that this advanced preparation “can be produced quickly and efficiently and at a very low cost in every pharmaceutical facility in the country, and in a short time globally.”
Prof. Ronni Gamzu, CEO of the medical center, said, “Prof. Arber’s results for first-phase research were excellent and gave us all confidence in the method he has been researching [here] for many years. I personally assisted him in further obtaining the approvals from the Ministry of Health for further research.”
Allocetra
Meanwhile, Enlivex Therapeutics last week reported positive results from a multi-center Phase II clinical trial of its experimental Covid-19 immunotherapy drug Allocetra in severe and critical Covid-19 patients.
We reported in October that five Covid-19 intensive care patients were discharged from Hadassah University Medical Center in Jerusalem after treatment with Allocetra.
Nine severe and seven critical Covid-19 patients were treated with Allocetra in the Phase II clinical trial. Fourteen of them recovered and were discharged from the hospital after an average of 5.3 days.
The Phase II trial originally was expected to enroll 24 patients but was “completed early in support of anticipated accelerated regulatory filings of the trial’s positive safety and efficacy data,” Enlivex reported.
Altogether, 19 out of 21 Phase II and Phase Ib Allocetra trial patients recovered and were discharged from the hospital after an average of 5.6 days. Most of the patients in both studies had pre-existing risk factors such as male gender, obesity and hypertension.
“The results we have seen from the 12 Covid-19 patients treated to date with Allocetra are exciting,” said Prof. Vernon van Heerden, head of the General Intensive Care Unit at Hadassah and the lead investigator of both clinical trials.
“The Phase II patients who have been discharged from the hospital are currently healthy. We believe that these compelling results have demonstrated the safety and efficacy of Allocetra in these complicated patients, highlighting the potential of Enlivex’s product candidate to benefit severe and critical Covid-19 patients as well as others suffering from cytokine storms and organ dysfunctions across various clinical indications.”
Allocetra is based on the research of Enlivex chief scientific and medical officer Dr. Dror Mevorach, head of internal medicine and of one of Hadassah’s coronavirus wards. It works by restoring balance to the immune system.
Mevorach said Allocetra “may have utility as a safe and efficacious treatment … regardless of the specific coronavirus mutation that afflicted the patients, and across different life-threatening, high mortality clinical indications with high unmet medical needs.”
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A bullish case for MGTA (from Motley Fool).
Magenta Is Ready to Paint Your Portfolio Green
This clinical-stage biotech is looking to make stem cell transplantation faster, easier, and safer for both donors and patients.
Patrick Bafuma
(Fuma102)
Feb 27, 2021 at 8:08AM
For patients with certain blood cancers, a stem cell transplant can often be a curative treatment. However, it is also a complex process riddled with severe toxicities and high rates of relapse or failure. These issues are at least part of the reason why only about 60% of cancer patients, a third of genetic disease patients, and 5% of autoimmune patients who are eligible to receive a stem cell transplant actually receive one. It's not hard to imagine that should a safer, more efficient process exist, the market could potentially expand. Enter Magenta Therapeutics (NASDAQ:MGTA).
The clinical-stage biotech's lead compound, MGTA-145, has the potential to revolutionize the current standard-of-care transplant process for both patients and donors, making it faster and safer for all parties.
Focusing on donors
When you donate stem cells, on average it takes five or more days to collect them. This is an inefficient process for a donor because it requires daily injections of medication called G-CSF, which boosts the release of stem cells from bone marrow into the bloodstream for collection. These daily injections cause significant bone pain and a handful of other side effects. To make matters worse, about 15% of donors will need multiple collection attempts, which mean further daily G-CSF injections. Perhaps it's not surprising that about 50% of registered donors decline to donate when they are called to do so.
Patients receiving a stem cell transplant, meanwhile, first have to undergo G-CSF treatment to boost their stem cells' release from bone marrow, then undergo chemo and radiation to try to get rid of any bad or cancerous cells originating in the bone marrow before they get their transplant. That's a lot of steps, a lot of time, and a lot of potential side effects.
Magenta is working to streamline this process, and MGTA-145 has the potential to become the first medicine approved for this mobilization of stem cells in both donors as well as transplant recipients. (While G-CSF is commonly utilized by clinicians off-label for the mobilization of donor stem cells, the drug is actually not explicitly approved for that usage.) Phase 1 studies demonstrated a threefold increase in the number of stem cells collected from healthy donors when treated with MGTA-145 compared with G-CSF, and amazingly, 88% of donors infused with MGTA-145 were able to have their stem cells collected that same day, compared with zero for the G-CSF group. Lastly, there was only a 1% rate of grade 2 to grade 4 side effects with MGTA-145; that rate is 38% for G-CSF.
Data from two phase 2 trials are expected in the second half of 2021. One trial is looking at MGTA-145's effect on stem cell mobilization and collection, as well as the ever-important progression-free survival rate, in cases of multiple myeloma. The other is examining MGTA-145's effect on stem cell mobilization, collection, and disease outcomes for acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and myelodysplastic syndrome (MDS).
How green can Magenta get?
So far, MGTA-145 appears faster, safer, and requires fewer collections from donors than the current standard of care, with no competition on the horizon. So what's the addressable market look like?
One place to start measuring is with Sanofi's (NASDAQ:SNY) plerixafor, which is currently given to approximately half of patients who undergo stem cell treatment for certain blood cancers. That drug had sales of $200 million in 2019.
However, because MGTA-145 makes donation less of a hassle, it's no stretch to imagine that the drug could work as a catalyst to increase the number of stem cell transplants performed annually. Management estimates that they can grow the market from today's about 90,000 worldwide to more than 175,000. Magenta has also begun to explore MGTA-145's utility in the gene therapy market, having already partnered with Beam therapeutics (NASDAQ:BEAM), bluebird bio (NASDAQ:BLUE), and AVROBIO (NASDAQ:AVRO) to more efficiently mobilize stem cells for their gene therapies. Partnered phase 2 trials are already under way.
Should future MGTA-145 trials continue to be positive, which I think is likely, $200 million would likely be just the beginning of the addressable market. It could easily be more than double that when you account for the potential increase in donors and procedures, as well as the gene therapy market. With a current market cap of about $500 million, and other promising candidates in the pipeline, Magenta is well positioned to add some green to healthcare investors' portfolios over the coming years -- not to mention making a difference for tens of thousands of patients and donors.
This article represents the opinion of the writer, who may disagree with the “official” recommendation position of a Motley Fool premium advisory service. We’re motley! Questioning an investing thesis -- even one of our own -- helps us all think critically about investing and make decisions that help us become smarter, happier, and richer.
Patrick Bafuma owns shares of Bluebird Bio. The Motley Fool owns shares of and recommends Bluebird Bio. The Motley Fool has a disclosure policy.
Bladerunner
gfp, I'm watching GMTX, but haven't pulled the trigger. Sorry to hear about your parents.
Bladerunner
Blade, I saw your post on GMTX and wondered if you decided to invest? Also saw the bio newsletter post over on Dew's board (link below), and the author mentions 4 stocks involved in the dry macular space. Things looked great until seeing that 20% of the dry macular patients had flipped to 'wet' during treatment (compared to 1% in the placebo group).
Not sure if GMTX's drug will do better, but 20% of patients going wet is no laughing matter since the wet form is devastating. My mom had it, and after several years of Avastin and then Lucentis injections, she ultimately lost most of her vison over a 3 day period. She went from 20/80 to basically blind, what a nightmare. My dad had the dry form and luckily it stayed dry.
With the total lack of current treatments (other than vitamins/minerals), the GMTX drug could be a blockbuster if it can improve on that 20% risk aspect. I think that other drug was from Apellis Pharma -
https://mattbiotech.substack.com/p/4-the-geographic-atrophy-issue-apls
https://investorshub.advfn.com/boards/read_msg.aspx?message_id=161872601
Blade, >> BPTH <<
Thanks, sounds interesting -
>>> Bio-Path Quadruples on Receipt of Cancer-Drug Tech Patent
Bio-Path shares as much as quadrupled. The company received a patent that builds on earlier ones protecting the platform tech for its nanoparticle drugs.
DAN WEIL
FEB 10, 2021
https://www.thestreet.com/investing/bio-path-shares-up-on-patent-for-cancer-drug-technology?puc=yahoo&cm_ven=YAHOO
Bio-Path Holdings (BPTH) - shares as much as quadrupled on Wednesday after the biotechnology company said it received a patent for its cancer-treatment technology.
Bio-Path recently traded at $16.75, compared with its Tuesday close at $5.20. It has traded on Wednesday at as much as a 52-week high $24.34. It was trading below $3 in mid-March.
The technology receiving the patent is DNAbilize liposomal delivery and antisense technology, used to develop a portfolio of targeted nucleic acid cancer drugs.
The patent is titled "P-ethoxy nucleic acids for liposomal formulation," Bio-Path said.
“The new patent builds on earlier patents granted that protect the platform technology for DNAbilize, the company’s novel RNAi nanoparticle drugs.”
The U.S. Patent and Trademark Office also has sent notification for a patent related to the company’s lead product candidate, prexigebersen, Bio-Path said.
“Our innovative DNAbilize platform improves upon the drawbacks of traditional approaches, which are limited by the toxicity induced by either the DNA backbone or the lipid delivery,” Chief Executive Peter Nielsen said in a statement.
“DNAbilize overcomes these challenges by combining a neutral charge P-ethoxy DNA backbone with a neutral charge liposome.”
Further, “The result is a high payload liposome with DNA safely delivered inside non-toxic cell membrane-like molecules, allowing us to deliver antisense DNA in high doses to target cells through the blood and lymphatic system with no evidence of toxicity in patients in clinical trials to date, in contrast to other lipid delivery technologies with dose limiting toxicities,” he said.
The P-ethoxy nucleic acids patent “is the third patent in our family of platform intellectual property and offers expanded defense of our DNAbilize platform technology,” Nielsen said.
<<<
gfp, I sold all my HGEN (nice profit) way too early.
I have a small position in TRIL.
I've established a decent-sized position in BPTH.
Bladerunner
Blade, Yes, nice moves for KZIA and MGTA, and ONCT is also looking good.
Btw, are you still following HGEN? It looks interesting for a possible run-up ahead of their Phase 3 topline data in March. CYDY is their main competitor in the Covid 'cytokine storm' space, and the CYDY Phase 3 data announcement is now long overdue (trial ended mid-Jan). CYDY has shady management, and in addition to HGEN's own Phase 3 data, HGEN could get an added boost if the CYDY trial disappoints.
TA/charts are not generally as useful with bio stocks, but the HGEN chart looks like an impending breakout.
Also, just curious if you are still in TRIL? Thanks.
KZIA was up 20% today on 4X average daily volume.
MGTA was up 17% today on 3X average daily volume.
Two of my largest positions.
No news on either one that I'm aware of.
Bladerunner
P.S. ONCT has been moving up nicely.
ONCT up 9% hits 52-week high
Bladerunner
Blade, >> CRVS <<
It figures I would buy right before the financing announcement, lol. But promising stocks often bounce back quickly after a financing. Better to have the company adequately funded than to worry about a temporary dip in the share price.
Btw, that 'Psychedelic Medicine' sector idea is working out pretty well so far, and my basket of stocks already up 4-5%. Since the ETF is not available in the US yet, I put $500 each into 8 of the ETF's main holdings, for a total investment of $4000. So not huge either way, but I wanted to participate in the emerging sector. These stocks have started to move up with the recent introduction of the ETF. Personally I'm not thrilled with the idea of legalizing recreational use of cannabis and psychedelics, but investment-wise it has become a bonafide sector -
>>> The First Psychedelic's ETF is Here, Should You Invest?
February 11, 2021
by Neal Farmer
https://www.investorsobserver.com/news/featured/the-first-psychedelics-etf-is-here-should-you-invest
The world’s very first psychedelic exchange-traded fund began trading on Wednesday on the NEO Exchange in Toronto. The Horizons Psychedelic Stock Index ETF (TSX:PSYK) tracks the North American Psychedelics Index. The development marks a major milestone for the growing interest in the use of these drugs as potential mental health treatment options. The investment world has taken notice and has led to a few major companies attempting to capitalize on the fast-growing industry.
What’s in the ETF?
The Kevin O’Leary backed MindMed (MMEDF) is one of the 17 companies included in the ETF. MindMed has seen its shares soar since September as many are expecting it to follow the outlook for cannabis legalization. MindMed currently trades on the NEO Exchange and the over-the-counter market in the United States but has applied to be uplisted on the NASDAQ. Others such as Seelos Therapeutics (SEEL) and Nova Mentis Life Science (NOVA) have seen share prices jump in this time as well.
The 17 companies included in the Horizons Psychedelic Stock Index ETF are focused on using psychedelics to treat mental-health problems such as depression, anxiety, post-traumatic stress disorder (PTSD), and others. The introduction of many companies to such a hot corner of the investing market was bound to lead to at least one ETF eventually. The evolution of people’s opinions on cannabis leading it to be legalized for recreational use in many states and legal for medicinal purposes in the vast majority has partly led to bullish analysis on psychedelics for mental health treatment.
Case for Psychedelic Investment
The progression mindset about cannabis and its potentially beneficial attributes has led to some enthusiasm bleeding over to psychedelics but the biggest recent factor that led to share prices rising was the legalization of MDMA (i.e. ecstasy) in Oregon and Washington D.C. at licensed facilities. Other psychedelic substances were approved at licensed facilities for mental health purposes in these states with similar proposals in California, Vermont, and Iowa. This development along with some expectations that President Biden will advocate for the medicinal legalization due to mental health struggles in the U.S. along with an opioid crisis, has many wanting to invest now while its just taking off. Early legalization efforts and a massive mental health market are promising signs for this industry, but if cannabis is its most comparable product, it may take longer than most expect.
Case Against Psychedelic Investment
While a large majority of states have voted to legalize cannabis for medicinal use, only 11 have legalized for recreational use. The bigger problem for psychedelic medicinal use is that it took a long time for cannabis to get where it is now and it's still not fully legalized. Even the states that have approved it have many towns and cities that have banned retail stores. Additionally, cannabis stocks experienced a nice surge once they began trading but have been struggling since early 2019. Investors looking to jump on before this rocket takes off might want to consider that Oregon legalizing something isn't exactly a guarantee that the rest of the country jumping on board any time soon.
There were a lot of social factors that kept cannabis from being legalized even just for medicinal purposes for a long time. It seems arrogant to think psychedelics won't have similar problems. Getting over the preconception that these drugs aren't just for use at rave parties will be particularly difficult for a large segment of the population. Even still, some believe these drugs will disrupt a $70 billion market for mental health treatment that has only gotten more attention in recent years due to the nation’s various struggles especially now coming out of the coronavirus pandemic.
<<<
CRVS announces pricing of public offering
Close this content, you can also use the Escape key at anytime
GlobeNewswire
Corvus Pharmaceuticals Announces Pricing of Public Offering of Common Stock
Corvus Pharmaceuticals, Inc.
Fri, February 12, 2021, 6:00 AM
More content below
CRVS
-10.4036%
BURLINGAME, Calif., Feb. 12, 2021 (GLOBE NEWSWIRE) -- Corvus Pharmaceuticals, Inc. (NASDAQ:CRVS), a clinical-stage biopharmaceutical company, today announced the pricing of an underwritten public offering of 8,571,429 shares of its common stock at a price to the public of $3.50 per share, for gross proceeds of $30.0 million before deducting estimated underwriting discounts and commissions and estimated offering expenses payable by Corvus. All of the shares are being offered by Corvus. In addition, Corvus has granted the underwriters of the offering a 30-day option to purchase up to an additional 1,285,714 shares of common stock at the public offering price, less underwriting discounts and commissions.
Corvus currently expects to use the net proceeds from this offering to fund its Phase 3 clinical trial of CPI-006 and development of its other product candidates, with any remaining proceeds for working capital and general corporate purposes.
The offering is expected to close on or about February 17, 2021, subject to satisfaction of customary closing conditions.
Cantor Fitzgerald & Co. and H.C. Wainwright & Co. are acting as joint book-running managers for the offering.
A shelf registration statement on Form S-3 relating to the securities being sold in this offering was declared effective by the Securities and Exchange Commission on March 19, 2020. The offering of these securities is being made only by means of a prospectus forming a part of the effective registration statement. A preliminary prospectus supplement and accompanying prospectus relating to the offering have been filed with the SEC and a final prospectus supplement and accompanying prospectus relating to the offering will be filed with the SEC and will be available on the SEC’s website at www.sec.gov. A copy of the final prospectus supplement and accompanying prospectus relating to the offering, when available, may be obtained from: Cantor Fitzgerald & Co., Attention: Capital Markets, 499 Park Avenue, 6th Floor, New York, NY 10022 or by email at prospectus@cantor.com or H.C. Wainwright & Co., LLC, 430 Park Avenue, 3rd Floor, New York, NY 10022, by telephone at (646) 975-6996 or by email at placements@hcwco.com.
Bladerunner
Gfp,
I sold KURA quite a while ago. I should have held longer, Another screw-up.
Bladerunner
Blade, Thanks. Also, looks like KURA has been doing well. Any thoughts on them? TIA.
Btw, I recently set up a board to follow the emerging 'Psychedelic Medicine' sector (link below). Back in 2019 I followed the saga of Spravato / Esketamine (the mirror image isomer of Ketamine), which received FDA approval for treatment resistant depression. It's become obvious that these substances are to be made legal (for better or worse), so the search by investors for early stage companies is on. An ETF just came out covering the sector (Horizons Psychedelic Stock Index ETF), but so far is only available in Canada -
https://investorshub.advfn.com/Psychedelic-Medicine-Sector-37972/
MGTA is one of my largest positions. If everything works out, MGTA wull transform the way transplant medicine is done.
Bladerunner
Blade, Any thoughts on MGTA? The stock has had a good move in the last several months. I'm not up on the stem cell area, but looks like some advanced science. I remember you had mentioned MGTA a while back -
>>> Magenta Therapeutics, Inc. (MGTA), a clinical-stage biotechnology company, develops novel medicines to extend the curative power of stem cell transplant, gene therapy, genome editing, and cell therapy to patients. The company is developing C100, C200, and C300 targeted antibody-drug conjugates for transplant conditioning; MGTA-145, a novel stem cell mobilization product candidate to control stem cell mobilization; MGTA-456, an allogeneic stem cell therapy to control stem cell growth; E478, a small molecule aryl hydrocarbon receptor antagonist for the expansion of gene-modified stem cells; and G100, an antibody-drug conjugate program to prevent acute graft and host diseases. It has a has a research and clinical collaboration agreement with AVROBIO, Inc.to evaluate targeted antibody-drug conjugate as a conditioning regimen for lentiviral gene therapies; and clinical trial collaboration with bluebird bio, Inc. to evaluate the utility of MGTA-145, in combination with plerixafor, for mobilization and collection of stem cells in adults and adolescents with sickle cell disease. The company was formerly known as HSCTCo Therapeutics, Inc. and changed its name to Magenta Therapeutics, Inc. in February 2016. Magenta Therapeutics, Inc. was founded in 2015 and is headquartered in Cambridge, Massachusetts. <<<
Blade, >> CRDF <<
Thanks. I replaced LIFE with CRDF on the list. It's great having a knowledgeable guy like you around :o)
It's going to take a while to get even moderately up to speed on these stocks. Bio is the hardest sector there is, and no matter how much research one does, it never seems to be enough.
For stock exposure, I mostly am using broad index ETFs, and sector ETFs have largely replaced the role that individual stocks once played in the portfolio. Still, it's nice to have some promising stocks to follow. I use strict position limits, so you don't have to worry about me misusing your stock ideas to 'bet the farm' :o)
gfp,
LIFE is only on the list because of the Redditors. Fundamentally, I don't really like it.
CRDF has results out today and my cancer docs really like the results. I don't own any right now, but may get back in, if I have some cash.
Cardiff Oncology Presents Phase 2 mCRPC Trial Data Showing a Two-Fold Increase in Efficacy with an Optimized Onvansertib Dosing Schedule
Thu, February 11, 2021, 5:00 A
CRDF
+9.85%
- Two-fold increase (29% to 63%) in disease control rate at 12 weeks (the trial's primary efficacy endpoint) seen in patients receiving onvansertib for 14 days vs. 5 days in a 21-day cycle
- 75% (6/8) of evaluable patients in the optimized dosing cohort had stable disease upon radiographic scan at 12 weeks
- All patients in the optimized dosing cohort achieving the primary efficacy endpoint remain on treatment
- Trial on track to meet prespecified criteria for success on its primary efficacy endpoint, with a 35% disease control rate at 12 weeks in evaluable patients across all three cohorts. Patients eligible for the trial have two consecutive rises in PSA levels, indicating initial resistance to Zytiga® (abiraterone).
SAN DIEGO, Feb. 11, 2021 /PRNewswire/ -- Cardiff Oncology, Inc. (Nasdaq: CRDF), a clinical-stage biotechnology company developing drugs to treat cancers with the greatest medical need for new treatment options, including KRAS-mutated colorectal cancer, pancreatic cancer, castrate-resistant prostate cancer and leukemias, today announced that updated data from its Phase 2 metastatic castrate-resistant prostate cancer (mCRPC) trial were featured in a virtual oral poster presentation at the American Society of Clinical Oncology Genitourinary Cancers Symposium (ASCO-GU). The ongoing Phase 2 trial evaluates the all-oral combination of onvansertib, abiraterone and prednisone in patients showing initial abiraterone resistance, as defined by two consecutive rises in prostate-specific antigen (PSA) levels.
Cardiff Oncology is a clinical-stage biotechnology company and our mission is to develop new treatment options for cancer patients in indications with the greatest medical need. Our goal is to overcome resistance, improve response to treatment and increase overall survival. Our investigational drug, onvansertib, a first-in-class, third-generation Polo-like Kinase 1 (PLK1) inhibitor, is being evaluated in combination with standard-of-care chemotherapy and targeted therapeutics.
We are assessing tumor genomics and using our expertise in biomarker technology to rapidly evaluate patient response to treatment.
Cardiff Oncology is a clinical-stage biotechnology company and our mission is to develop new treatment options for cancer patients in indications with the greatest medical need. Our goal is to overcome resistance, improve response to treatment and increase overall survival.
Our investigational drug, onvansertib, a first-in-class, third-generation Polo-like Kinase 1 (PLK1) inhibitor, is being evaluated in combination with standard-of-care chemotherapy and targeted therapeutics. We are assessing tumor genomics and using our expertise in biomarker technology to rapidly evaluate patient response to treatment.
Newly presented data from the Phase 2 mCRPC trial showed that increasing the number of days of treatment with onvansertib from 5 to 14 in a 21-day cycle was associated with a greater than two-fold increase (29% to 63%) in disease control rate (DCR; defined by lack of PSA progression) at 12 weeks, the trial's primary efficacy endpoint. Six of eight (75%) evaluable patients receiving onvansertib for 14 of 21 days per cycle had stable disease upon radiographic scan at 12 weeks and five of these patients remain on treatment to-date. Across all cohorts, the DCR at 12 weeks is 35% (13/37), indicating the trial is on track to meet the stated criteria for success on its primary efficacy endpoint (30% DCR at 12 weeks).
"The preliminary data presented at ASCO-GU support a clinically meaningful onvansertib exposure effect," said David Einstein, M.D., attending physician at Beth Israel Deaconess Medical Center and principal investigator of the onvansertib mCRPC Phase 2 trial. "In the first eight patients treated to-date on Arm C, we are excited to see an increase in DCR with greater time on onvansertib, without excessive toxicity. Together with the clinically meaningful rates of disease control, and duration of disease control, we are seeing across all cohorts, these data demonstrate onvansertib's potential to address a critical unmet need for patients with abiraterone-resistant mCRPC."
"As data from this trial continue to emerge, we are very pleased to see increased efficacy with an optimized dosing schedule that is both well tolerated and increases the number of days a patient receives onvansertib in combination with abiraterone by nearly 3-fold" said Mark Erlander, Ph.D., chief executive officer of Cardiff Oncology. "These data suggest that the synergy demonstrated between onvansertib and abiraterone in pre-clinical models is being observed clinically. The trial's biomarker analyses are also promising, as the identification of mutations associated with response to the combination of onvansertib and abiraterone may enable more efficient design of future clinical studies and the identification of patients most likely to benefit from this combination."
Key data and conclusions from the ASCO-GU presentation include:
Efficacy:
The optimized dosing schedule of cohort C shows a greater than two-fold improvement in disease control rate compared to cohorts A and B
75% (6/8) of evaluable patients in cohort C had radiographic SD at 12 weeks, compared to 53% (9/17) in cohort A, 42% (5/12) in cohort B and 54% (20/37) across all cohorts
All cohort C patients achieving the primary efficacy endpoint remain on treatment
35% (13/37) of evaluable patients across all cohorts (A-C) achieved the primary efficacy endpoint of disease control at 12 weeks
Efficacy was observed in patients harboring androgen receptor (AR) alterations associated with abiraterone resistance across all 3 arms
Biomarker:
Circulating tumor DNA (ctDNA) analysis revealed differences in baseline genomic profiles of patients achieving SD at 12 weeks vs. those progressing at or before 12 weeks
Mutations present exclusively in patients with SD at 12 weeks were associated with cell cycle and DNA repair pathways that may result in increased efficacy of the onvansertib-abiraterone combination
Safety:
Data show that the combination of onvansertib and abiraterone is well tolerated across the three different dosing schedules of cohorts A-C:
The virtual poster, A Phase 2 Study of the Polo-like Kinase 1 (PLK1) Inhibitor Onvansertib in Combination with Abiraterone and Prednisone in Patients with Metastatic Castration-Resistant Prostate Cancer (mCRPC), is available on the "Scientific Presentations" section of the Cardiff Oncology website at https://cardiffoncology.com/scientific-presentations/.
About the Phase 2 Trial of Onvansertib in Metastatic Castration-Resistant Prostate Cancer
This trial is a Phase 2 open-label study of onvansertib in combination with abiraterone and prednisone, all administered orally, in patients with metastatic castration-resistant prostate cancer showing signs of early progressive disease (demonstrated by two rising prostate-specific antigen values separated by at least one week with no or minimal symptoms) while on Zytiga®/prednisone therapy. The primary efficacy endpoint is the proportion of patients achieving disease control after 12 weeks of study treatment, as defined by a lack of prostate-specific antigen (PSA), radiographic, or symptomatic progression. The trial is being conducted by Beth Israel Deaconess Medical Center (BIDMC), Dana-Farber Cancer Institute (Dana-Farber), and Massachusetts General Hospital Cancer Center (MGH). David Einstein, M.D., Genitourinary Oncology Program at BIDMC, is the principal investigator for the trial. For more information on the trial, please visit https://www.clinicaltrials.gov/ct2/show/NCT03414034.
About Cardiff Oncology, Inc.
Cardiff Oncology is a clinical-stage biotechnology company with the singular mission of developing new treatment options for cancer patients in indications with the greatest medical need. Our goal is to overcome resistance, improve response to treatment and increase overall survival. We are developing onvansertib, a first-in-class, third-generation Polo-like Kinase 1 (PLK1) inhibitor, in combination with standard-of-care chemotherapy and targeted therapeutics. Our clinical development programs incorporate tumor genomics and biomarker technology to enable assessment of patient response to treatment. We have three clinical programs that have demonstrated the safety and efficacy of onvansertib: a Phase 1b/2 study of onvansertib in combination with FOLFIRI/Avastin® (bevacizumab) in KRAS-mutated metastatic colorectal cancer (mCRC); a Phase 2 study of onvansertib in combination with Zytiga® (abiraterone)/prednisone in metastatic castration-resistant prostate cancer (mCRPC); and a Phase 2 study of onvansertib in combination with decitabine in relapsed or refractory acute myeloid leukemia (AML). A new Phase 2 trial of onvansertib in combination with nanoliposomal irinotecan, leucovorin and fluorouracil for the second-line treatment of patients with metastatic pancreatic ductal adenocarcinoma (PDAC) is planned for initiation in the first half of 2021. For more information, please visit https://www.cardiffoncology.com.
Bladerunner
Blade, >> MYOV <<
I see MYOV has a relatively huge $745 mil cash position, so a good sign. I noticed you have 3 UK based biotechs (and one Australian) among your current favorites, so you must be an 'Anglophile' :o) I like the UK also, and have been there several times on vacation.
Btw, I decided to put my recent IPIX profits into your current bio picks. It was only $10 K, but it's a start, and I spread it evenly among -
CRVS
CRIS
FSTX
LIFE - waiting for a pullback
MYOV
ONCT
PRVB
I already had similar size positions in HGEN and KZIA, so counting IPIX that makes 10 stocks. So a lot to get up to speed on. These bios take forever to research, but I'll start with the basic info and go from there. I also have a modest investment in the ARK Genomic Revolution ETF (ARKG), which is the easy path to take :o)
Let me know when you have some additions/deletions to your fav lists,
Thanks
Name | Symbol | % Assets |
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Newmont Corp | NEM | 12.36% |
Barrick Gold Corp | ABX.TO | 11.02% |
Franco-Nevada Corp | FNV.TO | 7.17% |
Newcrest Mining Ltd | NCM.AX | 5.15% |
Agnico Eagle Mines Ltd | AEM.TO | 4.96% |
Wheaton Precious Metals Corp | WPM.TO | 4.95% |
Anglogold Ashanti Ltd ADR | AU.JO | 4.14% |
Kirkland Lake Gold Ltd | KL.TO | 4.07% |
Royal Gold Inc | RGLD | 3.70% |
Kinross Gold Corp | K.TO | 3.11% |
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Kinross Gold Corp | K.TO | 6.00% |
Northern Star Resources Ltd | NST.AX | 6.00% |
Sibanye-Stillwater ADR | SBGL.JO | 5.99% |
Pan American Silver Corp | PAAS.TO | 5.49% |
Gold Fields Ltd ADR | GFI.JO | 4.60% |
Yamana Gold Inc | YRI.TO | 4.40% |
Evolution Mining Ltd | EVN.AX | 4.00% |
B2Gold Corp | BTO.TO | 3.73% |
Buenaventura Mining Co Inc ADR | BVN | 2.99% |
Saracen Mineral Holdings Ltd | SAR.AX | 2.95% |
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