On November 10, 2025 Galecto, Inc. ("Galecto") reported the completion of the acquisition of Damora Therapeutics, Inc. ("Damora"), a privately held biotechnology company advancing a pipeline of antibody therapeutics aimed at transforming the treatment of mutant calreticulin- (mutCALR)-driven Myeloproliferative Neoplasms (MPNs), including Essential Thrombocythemia (ET) and Myelofibrosis (MF). The acquisition gives the combined company a pipeline covering a broad spectrum of hematological cancers and leverages the deep expertise of both teams, and positions Galecto to advance Damora’s complementary assets for people living with blood cancers, with the goal of developing safer, more effective, and more convenient disease-modifying therapies.

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A concurrent private placement of Series C non-voting convertible preferred stock was led by Fairmount with participation from the following institutional investors: Viking Global Investors, Venrock Healthcare Capital Partners, Commodore Capital, Janus Henderson Investors, Wellington Management, RA Capital Management, TCGX, Forbion, BB Biotech, Blackstone Multi-Asset Investing, Perceptive Advisors, Vestal Point Capital, Balyasny Asset Management, Andreessen Horowitz (a16z Bio + Health), and a leading life sciences investment firm. The private placement resulted in gross proceeds to Galecto of approximately $284.9 million and is expected to fund operations into 2029, enabling advancement of Damora’s lead program, DMR-001, through key Phase 1 proof-of-concept data expected in 2027, as well as pipeline programs, DMR-002 and DMR-003, into Phase 1 studies.

MPNs are a group of rare, chronic blood cancers characterized by the abnormal production of blood cells in the bone marrow. MutCALRs drive uncontrolled proliferation and disease progression in 25% of ET and 35% of MF cases. There are approximately 42,000 patients in the United States living with mutCALR-driven MPNs.

Damora is the sixth company launched based on assets developed by Paragon Therapeutics. DMR-001 is a potentially best-in-class anti-mutCALR monoclonal antibody demonstrated to have enhanced activity in Type 1 and Type 2 mutCALR-driven preclinical models, with approximately 10-fold greater potency against Type 2 mutCALR-driven cell proliferation, as compared to internal data generated using a reference molecule in clinical development, supporting its potential to address the full spectrum of mutCALR mutations in both ET and MF. DMR-001 is engineered with validated half-life extension technology to enable infrequent low-volume, subcutaneous dosing. An IND submission for DMR-001, with anticipated first-in-human administration via subcutaneous administration, is expected to occur in mid-2026.

"The acquisition of Damora marks a pivotal milestone for Galecto as we evolve our focus toward advancing Damora’s highly differentiated mutCALR portfolio, which addresses significant unmet need across the full spectrum of blood cancers driven by mutCALR," said Dr. Hans Schambye, President and CEO of Galecto. "This pipeline, led by DMR-001, is designed to overcome the limitations of current candidates in early clinical development and has the potential to establish a new standard of care in the treatment of ET and MF. With a strong balance sheet, we believe we are well-positioned to drive these programs rapidly into the clinic and ultimately deliver meaningful benefit to patients."

"The immediate access to public capital markets and the strengthened financial position achieved through this acquisition and concurrent private placement provides momentum to accelerate the development of our highly specialized portfolio," added Peter Harwin, incoming Galecto Board Member and Managing Member at Fairmount. "Our team is eager to leverage Galecto’s infrastructure to rapidly transition DMR-001 from IND-enabling studies to first-in-human trials by mid-2026, ensuring these assets, focused on superior efficacy and convenience, reach patients as quickly as possible."

In addition, Galecto is excited to combine these newly acquired complementary pipeline assets with its investigational candidate GB3226, a dual ENL-YEATS and FLT3 inhibitor for multiple genetic subsets of acute myeloid leukemia (AML). As previously disclosed, GB3226 will be featured in two poster presentations at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition, taking place December 6 – 9, 2025, in Orlando, Florida. Galecto also announced that it received constructive feedback from the U.S. Food and Drug Administration (FDA) in response to its pre-Investigational New Drug (pre-IND) submission and plans to submit an IND application in the first quarter of 2026 to enable future clinical evaluation of GB3226 in AML.

Management & Organization

In conjunction with the transaction, Galecto Board Members will include:

•
Carl Goldfischer, MD (current Chair)

•
Jayson Dalls, MD (current)

•
Amit Munshi (current)

•
Hans Schambye, MD, PhD (current)

•
Peter Harwin, Managing Member, Fairmount

•
Chris Cain, PhD, Director of Research, Fairmount

•
Julianne Bruno, Growth Partner, Fairmount

About the Acquisition and Financing Transaction

The acquisition was approved by the Board of Directors of Galecto and the Board of Directors and stockholders of Damora. The closings of the transactions are not subject to the approval of Galecto’s stockholders. On an as-converted basis and after accounting for these transactions (and without giving effect to any beneficial ownership limitations), the total number of shares of Galecto common stock will be 61,998,882.

Lucid Capital Markets is serving as financial advisor to the Board of Directors of Galecto. Mintz, Levin, Cohn, Ferris, Glovsky and Popeo, P.C., is serving as legal counsel to Galecto. Wedbush PacGrow is serving as financial advisor to Damora. Jefferies, Leerink Partners, UBS, and LifeSci are serving as the placement agents to Damora, and Gibson, Dunn & Crutcher LLP is serving as legal counsel to Damora.

Webcast Details

The company will host a webcast on November 10, at 8:00 a.m. ET to discuss the transaction as well as Damora’s approach and pipeline assets. To access the call, please dial 1-646-357-8766 (International) or 1-888-880-3330 (toll-free). To access the live webcast, please go to View Source A replay of the webcast presentation will be temporarily archived on the Investors section of Galecto’s website following the presentation.

(Press release, Galecto Biotech, NOV 10, 2025, View Source [SID1234659713])

On November 10, 2025 FibroGen, Inc. (NASDAQ: FGEN) reported financial results for the third quarter 2025 and provided an update on the company’s recent developments.

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"With the completion of the transformative sale of our FibroGen China operations, we have significantly simplified our capital structure and extended our cash runway into 2028. Notably, we initiated the Phase 2 monotherapy trial of FG-3246 and its companion diagnostic FG-3180, with an interim analysis anticipated in the second half of 2026," said Thane Wettig, Chief Executive Officer, FibroGen. "We remain on track to submit the Phase 3 protocol for roxadustat in LR-MDS in the fourth quarter of 2025 and continue to assess its development options. We are excited to continue building on the strong momentum we have gained throughout this year."

Recent Developments and Key Highlights of Third Quarter 2025:

Completed the sale of FibroGen China to AstraZeneca for a total consideration of approximately $220 million, consisting of $85 million in enterprise value and approximately $135 million in net cash held in China.
Successfully repaid term loan to Morgan Stanley Tactical Value, further simplifying the Company’s capital structure
FibroGen maintains its rights to roxadustat in the U.S. and in all markets outside of China, South Korea, and those licensed to Astellas.
Initiated the Phase 2 monotherapy dose optimization study of FG-3246 in mCRPC. The trial will also evaluate FG-3180 as a companion PET imaging agent and a potential predictive patient selection biomarker.
Reached agreement with the FDA on important design elements for the pivotal Phase 3 trial for roxadustat for the treatment of anemia in patients with LR-MDS and high transfusion burden. FibroGen continues to assess both internal development or partnership opportunities for this program.
Upcoming Milestones:

FG-3246 (CD46 Targeting ADC) and FG-3180 (CD46 Targeting PET Imaging Agent)

Topline results from the investigator-sponsored Phase 1b/2 study, conducted by UCSF, of FG-3246 in combination with enzalutamide in patients with mCRPC are expected to be presented at a medical conference in the first quarter of 2026.
Interim results from the Phase 2 monotherapy trial are expected in the second half of 2026. The trial will also assess the diagnostic performance of FG-3180 to determine the potential correlation between CD46 expression and response to FG-3246.
Roxadustat

FibroGen remains on track to file the pivotal Phase 3 clinical trial protocol for roxadustat for the treatment of anemia in patients with LR-MDS and high transfusion burden in the fourth quarter of 2025.
Financial:

Total revenue from continuing operations for the third quarter of 2025 was $1.1 million, as compared to $0.1 million for the third quarter of 2024.
Net loss from continuing operations for the third quarter of 2025 was $13.1 million, or $3.25 loss per basic and diluted share, compared to a loss of $48.3 million, or $12.01 loss per basic and diluted share, one year ago.
On September 30, 2025, FibroGen reported cash, cash equivalents, accounts receivable, and investments of $121.1 million.
The Company expects its cash, cash equivalents, accounts receivable, and investments to be sufficient to fund operating plans into 2028.
Conference Call and Webcast Presentation
The FibroGen management team will host a conference call and webcast presentation to discuss the financial results and provide a business update. A live Q&A session will follow the brief presentation. Interested parties may access a live audio webcast of the conference call here. To access the call by phone, please register here, and you will be provided with dial in details. A replay of the webcast will also be available for a limited time on the Events & Presentations page on FibroGen’s website.

About FG-3246
FG-3246 (FOR46) is a potential first-in-class fully human antibody-drug conjugate (ADC), exclusively in-licensed from Fortis Therapeutics, and is being developed by FibroGen for metastatic castration-resistant prostate cancer and potentially other tumor types. FG-3246 binds to an epitope of CD46, a cell receptor target, that induces internalization upon antibody binding, is present at high levels in prostate cancer and other tumor types and demonstrates very limited expression in most normal tissues. FG-3246 is comprised of an anti-CD46 antibody, YS5, linked to the anti-mitotic agent, MMAE, which is a clinically and commercially validated ADC payload. FG-3246 has demonstrated anti-tumor activity in both preclinical and clinical studies.

FG-3246 is currently in an ongoing Phase 1b/2 study being conducted at UCSF as an investigator-sponsored trial to evaluate FG-3246 in combination with enzalutamide. An additional investigator-sponsored radiopharmaceutical marker trial using a zirconium-89 positron emission tomography (PET) tracer for CD46 that utilizes the YS5 antibody is also underway at UCSF. The Phase 2 monotherapy dose optimization trial for FG-3246 in metastatic castration-resistant prostate cancer has been initiated. FG-3246 is an investigational drug and not approved for marketing by any regulatory authority.

About Roxadustat
Roxadustat, an oral medication, is the first in a new class of medicines comprising HIF-PH inhibitors that promote erythropoiesis, or red blood cell production, through increased endogenous production of erythropoietin, improved iron absorption and mobilization, and downregulation of hepcidin.

Roxadustat is approved in China, Europe, Japan, and numerous other countries for the treatment of anemia of CKD in adult patients on dialysis (DD) and not on dialysis (NDD). FibroGen has the sole rights to roxadustat in the United States, Canada, Mexico, and in all markets not held by AstraZeneca or licensed to Astellas. Astellas and FibroGen are collaborating on the commercialization of roxadustat for the treatment of anemia in territories including Japan, Europe, Turkey, Russia, and the Commonwealth of Independent States, the Middle East, and South Africa.

(Press release, FibroGen, NOV 10, 2025, View Source [SID1234659712])

On November 10, 2025 CRISPR Therapeutics (Nasdaq: CRSP), a biopharmaceutical company focused on creating transformative gene-based medicines for serious diseases, reported financial results for the third quarter ended September 30, 2025.

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"This has been another strong quarter of execution and progress across our portfolio," said Samarth Kulkarni, Ph.D., Chairman and Chief Executive Officer of CRISPR Therapeutics. "CASGEVY momentum continues to build globally, reflecting growing patient engagement and clinical advancement. Enrollment has been completed in two global Phase 3 pediatric studies, and dosing is on track to complete this quarter. Additionally, positive Phase 1 data for CTX310 presented in a late-breaking presentation at the American Heart Association Scientific Sessions and published in The New England Journal of Medicine, highlight the breadth and potential of our platform to address serious cardiovascular disease. We continue to advance our broader pipeline, including dosing the first patient in the Phase 2 clinical trial of SRSD107 and unveiling our novel SyNTase editing platform with CTX460, highlighting continued innovation and expansion of our therapeutic toolkit. With strong execution and growing momentum across our programs, CRISPR Therapeutics is well positioned to lead the next wave of gene editing innovation and deliver potentially transformative therapies to patients."

Recent Highlights and Outlook

Hemoglobinopathies and CASGEVY (exagamglogene autotemcel [exa-cel])

CASGEVY is a non-viral, ex vivo, CRISPR/Cas9 gene-edited cell therapy for eligible patients with sickle cell disease (SCD) or transfusion-dependent beta thalassemia (TDT), designed to eliminate both vaso-occlusive crises (VOCs) and transfusion requirements. CASGEVY is approved in the U.S., Great Britain, the EU, the Kingdom of Saudi Arabia (KSA), the Kingdom of Bahrain (Bahrain), Qatar, Canada, Switzerland and the United Arab Emirates (UAE) for the treatment of both SCD and TDT. Across these markets, there are more than 60,000 eligible patients in these countries, including approximately 37,000 in North America and Europe and more than 23,000 in the Middle East. With a well-established treatment pathway and a growing number of patients progressing through each stage, CASGEVY has strong momentum heading into late 2025 and beyond.

In September, a reimbursement agreement was reached in Italy for patients with SCD and TDT. Italy represents the largest population of individuals with TDT in Europe, with approximately 5,000 people aged 12 years and older with TDT and approximately 2,300 with SCD.

Globally, since launch through September 30th, 2025, approximately 165 patients with SCD or TDT have completed their first cell collection, including 50 people in the third quarter. 39 patients have received infusions of CASGEVY, including 10 infused in the third quarter of 2025. Nearly 300 patients have been referred by their physicians to an authorized treatment center (ATC) to begin the treatment process. This includes 110 cell collections in the first nine months of 2025, double the total for all of 2024.

Enrollment of children 5 to 11 years of age with SCD or TDT in two global Phase 3 studies of CASGEVY has been completed, and dosing is expected to be completed this quarter. Initial data from these studies will be presented at the American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting on December 6th, 2025.

The number of ATCs initiating patients continues to increase in the U.S., Europe, and the Middle East. Through the end of September, 25 ATCs had initiated more than 5 patients, with at least one ATC in each region initiating 20 or more patients.

Momentum continues to build through the final months of 2025. With continued uptake and reimbursement progress across major regions, Vertex expects a clear line of sight to over $100 million in total CASGEVY revenue this year with significant growth expected in 2026.

In Vivo Liver Editing

CRISPR Therapeutics is advancing a pipeline of in vivo gene editing candidates addressing major unmet needs in cardiovascular, metabolic and rare diseases using its proprietary, de-risked lipid nanoparticle (LNP) delivery platform.

CTX310, targeting ANGPTL3, is in an ongoing Phase 1 clinical trial in patients with homozygous familial hypercholesterolemia (HoFH), severe hypertriglyceridemia (SHTG), heterozygous familial hypercholesterolemia (HeFH), or mixed dyslipidemias. Phase 1 data were presented in a late-breaking session at the American Heart Association (AHA) Scientific Sessions and published simultaneously in The New England Journal of Medicine (NEJM). Results from the Phase 1 clinical trial highlight the potential of CTX310 to safely and durably lower both triglycerides (TG) and low-density lipoprotein (LDL) following a single-course IV administration. CRISPR Therapeutics is advancing CTX310 into Phase 1b clinical trials, prioritizing development in sHTG and mixed dyslipidemia.

CTX320 is in an ongoing Phase 1 clinical trial targeting the LPA gene in patients with elevated lipoprotein(a) [Lp(a)], a genetically determined risk factor associated with an increased incidence of major adverse cardiovascular events (MACE). Elevated Lp(a) levels affect up to 20% of the global population and remain unaddressed by current therapies. The Company plans to provide an update in the first half of 2026.

CRISPR Therapeutics continues to advance its preclinical in vivo programs: CTX460, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD); CTX340, targeting AGT for the treatment of refractory hypertension; and CTX450, targeting ALAS1 for the treatment of acute hepatic porphyria (AHP).

CTX460 is the first investigational candidate using the Company’s novel SyNTase editing platform, unveiled in October. SyNTase is designed to enable precise, in vivo gene correction, and represents an important expansion of CRISPR Therapeutics’ toolkit. Preclinical data presented at the European Society of Gene and Cell Therapy (ESGCT) Annual Congress demonstrated >90% mRNA correction, a 5-fold increase in total AAT levels, and >99% serum M-AAT:Z-AAT ratio in AATD disease models. These findings provide preclinical proof-of-concept for precise, single-dose in vivo gene correction using the SyNTase editing platform and support the potential best-in-class profile of CTX460. CRISPR Therapeutics expects to initiate a clinical trial of CTX460 in mid-2026.

Preclinical data from CTX340 were presented in a late-breaking poster presentation at the American Heart Association (AHA) Scientific Sessions. In a spontaneous hypertensive rat model, CTX340 showed >70% liver editing and mean arterial pressure reduction of 53 mmHg compared to vehicle that was durable throughout the study (~8.5 months). Furthermore, in non-human primates, CTX340 showed greater than 90% AGT reduction with a two-dose regimen showing the additive effects of repeat dosing and enabling dose titration. CTX340 was well tolerated with no hypotension or hypokalemia observed. CTX340 is currently in IND/CTA-enabling studies.

Autoimmune Disease and Immuno-Oncology

CTX112, targeting CD19, is being developed for autoimmune disease and hematologic malignancies and has received Regenerative Medicine Advanced Therapy (RMAT) designation from the U.S. Food and Drug Administration (FDA) for the treatment of relapsed or refractory follicular lymphoma and marginal zone lymphoma. A Phase 1 clinical trial in autoimmune disease is underway in systemic lupus erythematosus (SLE), systemic sclerosis and inflammatory myositis. In parallel, a Phase 1 clinical trial of CTX112 in relapsed or refractory B-cell malignancies is ongoing. The Company plans to provide a broad update on CTX112 by year-end.

CTX131, targeting CD70, was previously in development for both solid tumors and hematologic malignancies. While the Phase 1 data are encouraging, the Company has strategically redirected resources away from this program to advance other programs with the greatest potential for long-term value creation.

CRISPR Therapeutics is leveraging its expertise and proprietary lipid nanoparticle (LNP) delivery platform, mRNA, and conjugation capabilities to advance an in vivo CAR T platform with the ability to address autoimmune disease and oncology.

CRISPR Therapeutics’ autoimmune disease and immuno-oncology programs are supported by a wholly-owned, GMP manufacturing facility located in Framingham, Massachusetts, which provides end-to-end production capabilities for its cell therapy portfolio and supports both clinical and future commercial supply.

siRNA

In September, CRISPR Therapeutics and its partner Sirius Therapeutics announced that the first patient was dosed in Europe in the Phase 2 clinical trial of SRSD107, a long-acting Factor XI (FXI) small interfering RNA (siRNA) for thromboembolic disorders. The trial is evaluating the safety and efficacy of SRSD107 in preventing venous thromboembolism (VTE) following total knee arthroplasty (TKA) and will inform dose selection for future pivotal trials. SRSD107 has the potential to be a best-in-class FXI inhibitor, showing deep reductions in FXI via semi-annual subcutaneous injection. SRSD107 is being co-developed by CRISPR Therapeutics and Sirius Therapeutics as part of a broader strategic collaboration to advance RNA-based medicines.

CRISPR Therapeutics and Sirius Therapeutics have expanded the Phase 2 clinical trial with additional centers in Europe. SRSD107 is being developed for a range of thromboembolic and clotting-related indications, including arterial fibrillation (AF), cancer-associated thrombosis (CAT), chronic kidney disease (CKD), peripheral vascular disease (PVD), chronic coronary artery disease (CAD), ischemic stroke and VTE.

Regenerative Medicine

CRISPR Therapeutics continues to advance its regenerative medicine efforts for Type 1 diabetes (T1D). Beyond CTX211, the Company is developing next-generation programs that leverage induced pluripotent stem cell (iPSC) derived, allogeneic, gene-edited, beta islet cell precursors. These approaches aim to achieve insulin independence in T1D patients without requiring chronic immunosuppression. The Company expects to provide an update this year.

Upcoming Events

The Company will participate in the following events in November:

Guggenheim 2nd Annual Healthcare Innovation Conference
Date: Wednesday, November 12, 2025
Time: 11:30 a.m. ET
Jefferies Global Healthcare Conference
Date: Wednesday, November 19, 2025
Time: 1:00 p.m. GMT

Third Quarter 2025 Financial Results

Cash Position: Cash, cash equivalents, and marketable securities were $1,944.1 million as of September 30, 2025, compared to $1,903.8 million as of December 31, 2024. The increase in cash was primarily driven by proceeds from the issuance of common shares, option exercise activity and interest income, offset by operating expenses, as well as the $25.0 million upfront cash payment made as part of the Sirius Agreement.

R&D Expenses: R&D expenses were $58.9 million for the third quarter of 2025, compared to $82.2 million for the third quarter of 2024. The decrease in R&D expense was primarily driven by a decrease in variable external research and manufacturing costs, as well as a decrease in employee-related expenses, including stock-based compensation expenses.

G&A Expenses: General and administrative expenses were $16.9 million for the third quarter of 2025, compared to $17.4 million for the third quarter of 2024.

Collaboration Expense: Collaboration expense, net, was $57.1 million for the third quarter of 2025, compared to $11.2 million for the third quarter of 2024. In the third quarter of 2024, we exercised our option to defer specified costs under the CASGEVY program in excess of the deferral limit of $110.3 million under the A&R Vertex JDCA, as amended. The increase in collaboration expense, net, was primarily attributable to the timing of when we reached the deferral limit in 2024, as no such limit was applicable in 2025.

Net Loss: Net loss was $106.4 million for the third quarter of 2025, compared to a net loss of $85.9 million for the third quarter of 2024.

About CASGEVY (exagamglogene autotemcel [exa-cel])
CASGEVY is a non-viral, ex vivo CRISPR/Cas9 gene-edited cell therapy for eligible patients with SCD or TDT, in which a patient’s own hematopoietic stem and progenitor cells are edited at the erythroid specific enhancer region of the BCL11A gene. This edit results in the production of high levels of fetal hemoglobin (HbF; hemoglobin F) in red blood cells. HbF is the form of the oxygen-carrying hemoglobin that is naturally present during fetal development, which then switches to the adult form of hemoglobin after birth. CASGEVY has been shown to reduce or eliminate VOCs for patients with SCD and transfusion requirements for patients with TDT. CASGEVY is approved for eligible SCD and TDT patients 12 years and older by multiple regulatory bodies around the world.

About the CRISPR Therapeutics – Vertex Collaboration for CASGEVY
CRISPR Therapeutics and Vertex established a strategic research collaboration in 2015 to discover and develop therapies using CRISPR/Cas9 technology to address the underlying genetic causes of human disease. CASGEVY is the first approved therapy to emerge from this collaboration. Under an amended collaboration agreement, Vertex leads global development, manufacturing, and commercialization of CASGEVY and shares program costs and profits worldwide 60/40 with CRISPR Therapeutics. Vertex is the manufacturer and exclusive license holder of CASGEVY.

About CTX112
CTX112 is a wholly-owned, allogeneic chimeric antigen receptor (CAR) T cell therapy product candidate targeting Cluster of Differentiation 19 (CD19), in development for both autoimmune and immuno-oncology indications. CTX112 is being investigated in ongoing clinical trials in adult patients with systemic lupus erythematosus, systemic sclerosis, and inflammatory myositis and in adult patients with relapsed or refractory B-cell malignancies.

About In Vivo Programs
CRISPR Therapeutics has established a proprietary lipid nanoparticle (LNP) delivery platform to enable gene editing in the liver using both CRISPR/Cas9 and its novel, proprietary SyNTase editing technologies. The Company’s in vivo portfolio includes its lead investigational programs, CTX310 (directed towards angiopoietin-related protein 3 (ANGPTL3)) and CTX320 (directed towards LPA, the gene encoding apolipoprotein(a) (apo(a)), a major component of lipoprotein(a) [Lp(a)]). Both are validated therapeutic targets for cardiovascular disease. CTX310 and CTX320 are in ongoing clinical trials in patients with heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, mixed dyslipidemias, or severe hypertriglyceridemia, and in patients with elevated lipoprotein(a), respectively. In addition, the Company’s research and preclinical development candidates include: CTX460, targeting SERPINA1 for the treatment of alpha-1 antitrypsin deficiency (AATD); CTX340, targeting AGT for the treatment of refractory hypertension; and CTX450, targeting ALAS1 for the treatment of acute hepatic porphyria (AHP).

About SRSD107
SRSD107 is a novel double-stranded small interfering ribonucleic acid (siRNA). SRSD107 specifically targets the human coagulation factor XI (FXI) mRNA and inhibits FXI protein expression, thereby blocking the intrinsic coagulation pathway and promoting anticoagulant/anti-thrombotic effects. SRSD107 is being co-developed by CRISPR Therapeutics and Sirius Therapeutics as part of a strategic collaboration to advance innovative treatments for cardiovascular and clotting-related diseases.

(Press release, CRISPR Therapeutics, NOV 10, 2025, View Source [SID1234659711])

On November 10, 2025 Compugen Ltd. (Nasdaq: CGEN) (TASE: CGEN) a clinical-stage cancer immunotherapy company and a pioneer in predictive computational target discovery powered by AI/ML, reported financial results for the third quarter of 2025 and provided a corporate update.

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"I am thrilled to lead Compugen as our strong fundamentals and differentiated science gain clinical momentum," said Eran Ophir, Ph.D., President and CEO of Compugen. "Recent data at ESMO (Free ESMO Whitepaper) from our partner AstraZeneca and others reinforce our long-held view that not all anti-TIGIT antibodies are the same, and that the antibody-Fc format matters. This is because Fc reduced anti-TIGIT programs, like our fully owned anti-TIGIT, COM902, and AstraZeneca’s anti-PD-1/TIGIT bispecific rilvegostomig, preserve beneficial T cells and avoid depletion of peripheral T-regs in contrast to Fc-active anti-TIGITs and therefore have the potential for improved efficacy and safety profile. At ESMO (Free ESMO Whitepaper), AstraZeneca presented data from ARTEMIDE-01 showing rilvegostomig was well tolerated with promising efficacy confirming its potential in checkpoint naïve NSCLC with notable low rate of treatment related discontinuation supporting differentiation of the Fc-reduced format. AstraZeneca also presented data from TROPION-PanTumor03, evaluating the combination of rilvegostomig and Datroway, which showed promising efficacy and manageable safety underscoring the potential of next-generation IO bispecific plus ADCs. In addition, AstraZeneca announced it expects to launch its eleventh Phase 3 trial for rilvegostomig."

Dr. Ophir continued, "We presented pooled Phase 1 data at ESMO (Free ESMO Whitepaper) 2025 showing that COM701, our Fc-reduced anti-PVRIG antibody, is well tolerated as monotherapy and in combination and delivers durable responses in heavily pretreated platinum resistant ovarian cancer patients, with a median progression-free survival of 10.5 months in patients who derived clinical benefit. The data also showed that COM701 biology is differentiated, reflected in responses seen across PD-L1 expression levels. This data is important as it characterized responding patients, guiding the design of our ongoing blinded and randomized MAIA-ovarian platform trial evaluating single agent COM701 maintenance therapy in platinum sensitive ovarian cancer. Sites have been activated in the U.S., Israel and France, and we now estimate interim analysis in Q1 2027. We are also advancing GS-0321, a potential first-in-class anti-IL18BP antibody licensed to Gilead and presented the trial design at SITC (Free SITC Whitepaper) last week."

Dr. Ophir concluded, "Our solid financial position with cash runway expected into the third quarter of 2027 enables us to advance our differentiated IO pipeline and leverage our AI/ML powered computational discovery platform Unigen to discover novel ways to activate the immune system against cancer. I am confident in our fully owned programs, strengthened by validating partnerships with AstraZeneca and Gilead, which together offer over $1 billion in potential milestones and royalties."

Third Quarter 2025 Financial Highlights
Cash: As of September 30, 2025, Compugen had approximately $86.1 million in cash, cash equivalents, short-term bank deposits, and investment in marketable securities.

Compugen expects that its cash and cash-related balances will be sufficient to fund its operating plans into the third quarter of 2027. This does not include any cash inflows. The Company has no debt.

During October 2025, subsequent to the financial results for the quarter ended September 30, 2025, a total of approximately 0.8 million shares were sold through the Company’s ATM facility contributing net proceeds of approximately $1.6 million.

Revenue: Compugen reported approximately $1.9 million in revenues for the third quarter ended September 30, 2025, compared to approximately $17.1 million in revenues for the comparable period in 2024. The revenues reported in the third quarters of 2025 and 2024 reflect recognition of portions of both the upfront payment and the IND milestone payment from the license agreement with Gilead.

R&D expenses for the third quarter of 2025 were approximately $5.8 million compared to approximately $6.3 million for the comparable period in 2024.

G&A expenses were approximately $2.2 million for the third quarter of 2025 and $2.6 million for the comparable period in 2024.

Net loss for the third quarter of 2025 was approximately $6.98 million, or $0.07 per basic and diluted share, compared with a net profit of approximately $1.28 million, or $0.01 per basic and diluted share, in the third quarter of 2024.

Full financial tables are included below

Conference Call and Webcast Information
The Company will hold a conference call today, November 10, 2025, at 8:30 AM ET to review its third quarter 2025 results. To access the conference call by telephone, please dial 1-866-744-5399 from the United States, or +972-3-918-0644 internationally. The call will also be available via live webcast through Compugen’s website, located at the following link. Following the live audio webcast, a replay will be available on the Company’s website.

(Press release, Compugen, NOV 10, 2025, View Source [SID1234659710])

On November 10, 2025 Cogent Biosciences, Inc. (Nasdaq: COGT), a biotechnology company focused on developing precision therapies for genetically defined diseases, reported positive data from its Phase 3 PEAK trial of bezuclastinib plus sunitinib in patients with imatinib-resistant or intolerant Gastrointestinal Stromal Tumors (GIST). The combination reached a median progression free survival (mPFS) of 16.5 months compared to sunitinib monotherapy, which reached a mPFS of 9.2 months (HR=0.50, CI: 0.39-0.65; p<0.0001). In addition, the combination of bezuclastinib with sunitinib resulted in a 46% objective response rate (ORR) compared to 26% with sunitinib monotherapy (p<0.0001). Based on these data, Cogent is on track to submit a new drug application (NDA) to the U.S. Food and Drug Administration (FDA) for bezuclastinib in GIST in the first half of 2026. Cogent also plans to present detailed results from the PEAK trial at an upcoming scientific conference in the first half of 2026.

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"It is a historic day for Cogent Biosciences and the GIST patient community," said Andrew Robbins, Cogent’s President and Chief Executive Officer. "We are extremely excited to announce positive results from the Phase 3 PEAK trial of bezuclastinib plus sunitinib, which have far surpassed our expectations for the activity of this combination in patients with imatinib-resistant or intolerant GIST. With these incredible results, including a greater than seven-month improvement on mPFS – reducing the rate of progression or death by half – the bezuclastinib combination is poised to become the new standard of care for treatment of second-line GIST patients. We are pleased to have an existing Expanded Access Program available to GIST patients who have an urgency to access this novel treatment immediately and look forward to partnering with regulatory agencies to make this combination broadly available to patients as soon as possible."

"The results from the PEAK trial are truly transformative and practice changing," said Neeta Somaiah, M.D., Professor and Department Chair, Department of Sarcoma Medical Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX. "Following regulatory approval, I expect the bezuclastinib combination to be rapidly adopted as the new standard of care treatment for the majority of patients in the second-line GIST setting."

"Imatinib-resistant or intolerant GIST patients have waited nearly 20 years for a new second-line treatment option. The remarkable results of the PEAK study suggest that wait has come to an end," said Sara Rothschild, Executive Director, The Life Raft Group. "Like so many in the GIST community, we’ve actively followed this trial with real anticipation. On behalf of GIST patients around the world, we share our excitement for the hope that the bezuclastinib combination may bring these patients and their families."

PEAK Phase 3 Trial Results

PEAK is a global, randomized Phase 3 clinical trial evaluating bezuclastinib in combination with sunitinib vs. sunitinib monotherapy in patients with imatinib-resistant or intolerant GIST. In the top-line results, as of the cutoff date, September 30, 2025, the bezuclastinib combination demonstrated a substantial and highly statistically significant clinical benefit on the primary endpoint of progression free survival (PFS), reducing risk of disease progression or death compared to the current standard of care by 50% (hazard ratio of 0.50, 95% CI: 0.39 – 0.65). mPFS, as assessed by blinded independent central review, was 16.5 months for the bezuclastinib combination vs. 9.2 months for sunitinib monotherapy. Additionally, the bezuclastinib combination demonstrated an unprecedented ORR in imatinib-resistant patients, with 46% of patients treated with the bezuclastinib combination achieving an objective response compared to 26% of patients treated with sunitinib. At the time of this analysis, data for overall survival remains immature.

Based on these data, and the number of ongoing patients receiving treatment on the bezuclastinib arm, the estimated mean duration of treatment for the bezuclastinib combination is projected to exceed 19 months.

Safety Data

As of the data cutoff, the bezuclastinib combination was generally well tolerated, and no unique risks were observed with the novel combination when compared to the known safety profile of sunitinib. The most commonly reported Grade 3+ treatment emergent adverse events in either arm (bezuclastinib combination vs. sunitinib) included: Hypertension (29.4% vs. 27.4%), Neutropenia (15.2% vs. 15.4%), ALT/AST increased (10.8% vs. 1.4%), Anemia (9.3% vs. 4.8%) and Diarrhea (7.8% vs. 7.2%). 7.4% of patients on the bezuclastinib combination and 3.8% of patients on sunitinib monotherapy discontinued study treatment(s) due to treatment related adverse events. Hepatic adverse events were predominantly transient and manageable lab abnormalities; the majority of which were low grade, non-serious, reversible and asymptomatic. In the combination arm, ALT/AST elevations led to bezuclastinib dose reductions in 12.7% of patients with only 3 subjects (1.5%) discontinuing bezuclastinib for ALT/AST elevations. All Grade 3 ALT/AST elevations resolved, and no Grade 4 elevations were reported across the study.

Complete analysis of the Phase 3 PEAK data is ongoing, and Cogent plans to present detailed results at a major medical conference in the first half of 2026.

Anticipated Upcoming Milestones

Announce top-line results from the pivotal APEX trial in December 2025. APEX is a registration-directed, global, open-label trial in patients with Advanced Systemic Mastocytosis (AdvSM)
Present multiple bezuclastinib presentations at the 67th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), including two oral presentations from the pivotal SUMMIT trial in NonAdvanced Systemic Mastocytosis (NonAdvSM) patients
Present initial data from Cogent’s novel JAK2 V617F inhibitor at ASH (Free ASH Whitepaper), showcasing its best-in-class potential
Submit Cogent’s first NDA for bezuclastinib in NonAdvSM patients by the end of 2025
Submit NDA for bezuclastinib in imatinib-resistant or intolerant GIST patients in the first half of 2026
Webcast Information
Cogent will host a live webcast today, November 10, 2025 at 8:00 a.m. ET to discuss these results from PEAK with participation from Neeta Somaiah, M.D., Professor and Department Chair, Department of Sarcoma Medical Oncology, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, Houston, TX. The live event can be accessed on the Investors & Media page of Cogent’s website at investors.cogentbio.com. A replay of the webcast will be available approximately two hours after the completion of the event and will be archived for up to 30 days.

(Press release, Cogent Biosciences, NOV 10, 2025, View Source [SID1234659709])