On December 15, 2025 GRAIL, Inc. (Nasdaq: GRAL), a healthcare company whose mission is to detect cancer early when it can be cured, reported that company management will present at the 44th Annual J.P. Morgan Healthcare Conference in San Francisco on Monday, Jan. 12 at 7:30 a.m. PT.

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Live and replay webcasts may be accessed in the investor relations section of GRAIL’s website at investors.grail.com. The webcast will be archived and available for reply for at least 30 days after the event.

(Press release, Grail, DEC 15, 2025, View Source [SID1234661436])

On December 15, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported strategic leadership changes to accelerate its next phase of growth. Michael Kauffman, M.D., Ph.D., currently lead director of the Board, has been appointed as the president of development and will join the Company’s executive leadership team, while John Johnson, a board member since 2020, has been appointed as chairman of the board of directors succeeding Dr. Kauffman. Dr. Kauffman will remain on the Board but will no longer serve as lead director, or on the audit committee, or compensation committee of the Board upon transition to his new role. As part of these changes, Matthew Ros, chief operating officer, will be departing from the organization as the Company streamlines its operational structure and transitions his responsibilities across the executive team.

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"2025 has been a year of significant accomplishments where we advanced key clinical trials and launched an important new treatment for people living with a specific type of LGSOC, a rare ovarian cancer that is persistent and highly recurrent. We expect to enter 2026 from a position of strength and in that regard, I’m pleased to announce that John will assume the position of Chairman of the Board and bring his decades of corporate strategy and oncology commercialization to the role," said Dan Paterson, president and chief executive officer of Verastem Oncology. "The appointment of Michael as President of Development not only brings in a depth of experience in advancing novel agents from early development all the way through successful commercial launch, but also underscores our commitment to our R&D program and specifically the importance of VS-7375, and the anticipated positive impact we believe this potential best-in-class treatment may have on patients around the world."

Mr. Paterson added, "We thank Matt for his contributions to the initial success of the commercial launch of AVMAPKI FAKZYNJA CO-PACK and establishing a strong organizational foundation this year."

"I am honored to continue the leadership Michael has established over the past decade as Lead Director. Verastem is at a pivotal moment with the initial successful launch of AVMAPKI FAKZYNJA CO-PACK, which has provided a benefit to women where previously there were no FDA-approved treatments specifically for their disease. I look forward to working closely with Dan, Michael, and the rest of the Board to support the Company’s commercial and clinical development plans," said John Johnson, chairman of the board.

"After more than a decade on the Board, I am thrilled to join the executive team and dedicate myself full time to what I believe is a once-in-a-lifetime opportunity with VS-7375," said Michael Kauffman, M.D., Ph.D. "This potential best-in-class KRAS G12D dual ON/OFF inhibitor could transform outcomes for patients with currently limited options, and I am excited to bring my scientific expertise and proven track record of successful drug development and commercialization to the organization at this critical time."

RAMP 301

The Company also announced today that it has completed the additional patient enrollment for RAMP 301, its international Phase 3 confirmatory trial in recurrent LGSOC. Following a pre-planned interim analysis (IA), the Independent Data Monitoring Committee recommended a modest one-time increase of 29 patients across KRAS mutation status, based on the total enrollment achieved in October. The Company remains blinded to the IA results.

RAMP 301 is evaluating the combination of avutometinib plus defactinib versus standard chemotherapy for patients with recurrent LGSOC with and without a KRAS mutation. The trial will serve as a confirmatory study for the initial indication and has the potential to expand the indication regardless of KRAS mutation status. The Company expects to report a topline read-out of the primary endpoint in mid-2027.

Biographies for John Johnson and Michael Kauffman, M.D., Ph.D.

John Johnson

John Johnson is a recognized biopharma executive leader in the industry with more than three decades of experience across corporate strategy, operations, investing, clinical development, and oncology drug commercialization. He most recently served as Executive Chairman at Applied Therapeutics, a company focused on developing transformative treatments for rare disease. Mr. Johnson also previously was the Chief Executive Officer of Reaction Biology, a global Contract Research Organization. Prior to that he was the Chief Executive Officer of Stonebridge Biopharma prior to its merger with Xeris Biopharma. Mr. Johnson has held executive management roles at leading global corporations, including Johnson & Johnson, where he spent the majority of his career and served as the Company Group Chairman of Biopharmaceuticals within Johnson & Johnson. He was responsible for Johnson & Johnson Biotechnology, Immunology, and Oncology commercial businesses. Mr. Johnson also served as President of Eli Lilly & Company’s Worldwide Oncology unit, following the company’s 2008 acquisition of Imclone Systems, Inc., where he served as Chief Executive Officer and a member of Imclone’s Board of Directors.

Mr. Johnson has served on 19 boards and presently serves on the boards of Reaction Biology, Axogen (AXGN), Xeris Pharmaceuticals (XERS), and Verastem Oncology (VSTM). He served on two private equity backed company boards through successful exits. He has also served as a member of the board of directors of Pharmaceutical Research and Manufacturers of America (PhRMA) and as a member of the Health Section Governing Board of Biotechnology Industry Organization (BIO).

Michael Kauffman, M.D., Ph.D.

As a Lead Director of Verastem’s Board of Directors since June 2016, Dr. Kauffman has a deep understanding of the Company’s strategy, clinical development plans, and operations. Previously, Dr. Kauffman served as the Chief Executive Officer of Nereid Therapeutics. He was co-founder and Chief Executive Officer of Karyopharm, where he guided that company’s transition from a discovery stage biotechnology company to a commercial stage organization and the global approvals of XPOVIO. Prior to joining Karyopharm, Dr. Kauffman was Chief Medical Officer of Onyx Pharma, where he led the development of Kyprolis following the Onyx acquisition of Proteolix , where he served as board member and then Chief Medical Officer. Previously, Dr. Kauffman was President and Chief Executive Officer of EPIX Pharmaceuticals (previously Predix Pharmaceuticals.). Before that, he was the leader of the Velcade development program at Millennium Pharmaceuticals. He also held a number of senior positions at Millennium Predictive Medicine and Biogen.

Dr. Kauffman received his M.D. and Ph.D. from Johns Hopkins Medical School, trained in Internal Medicine at Beth Israel (Deaconess) Medical Center and in Rheumatology at Massachusetts General Hospital, and is board certified in Internal Medicine.

About AVMAPKI and FAKZYNJA Combination Therapy

AVMAPKI (avutometinib) inhibits MEK kinase activity while also blocking the compensatory reactivation of MEK by upstream RAF. RAF and MEK proteins are regulators of the RAS/RAF/MEK/ERK (MAPK) pathway. Blocking RAF and/or MEK activates FAK, a key mediator of drug resistance. FAKZYNJA (defactinib) is a FAK inhibitor and together, the avutometinib and defactinib combination was designed to provide a more complete blockade of the signaling that drives the growth and drug resistance of RAS/MAPK pathway-dependent tumors.

The U.S. Food and Drug Administration (FDA) approved AVMAPKI FAKZYNJA CO-PACK (avutometinib capsules; defactinib tablets) for the treatment of adult patients with KRAS-mutated recurrent LGSOC who have received prior systemic therapy on May 8, 2025. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial. Verastem is conducting RAMP 301 (GOG-3097/ENGOT-ov81/GTG-UK) (NCT06072781), an international Phase 3 confirmatory trial evaluating the combination of avutometinib and defactinib versus standard chemotherapy or hormonal therapy for the treatment of recurrent low-grade serous ovarian cancer (LGSOC) with and without a KRAS mutation. Verastem is also evaluating avutometinib plus defactinib with standard-of-care chemotherapy as a potential treatment in the first line for patients with advanced pancreatic cancer (RAMP 205; NCT05669482) and advanced KRAS G12C mutant non-small cell lung cancer (RAMP 203; NCT05074810). Avutometinib and defactinib are not approved by the FDA or any other regulatory authority, either in combination or with other therapies, for any of these investigative uses. Neither avutometinib nor defactinib are approved by the FDA or any other regulatory authority on a stand-alone basis for any use.

AVMAPKI FAKZYNJA CO-PACK U.S. Indication

Indication

AVMAPKI FAKZYNJA CO-PACK is indicated for the treatment of adult patients with KRAS-mutated recurrent low-grade serous ovarian cancer (LGSOC) who have received prior systemic therapy.

This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Important Safety Information

Warnings and Precautions

Ocular Toxicities: Ocular toxicities, including visual impairment and vitreoretinal disorders, occurred. Perform comprehensive ophthalmic evaluation at baseline, prior to cycle 2, every three cycles thereafter, and as clinically indicated. Withhold AVMAPKI FAKZYNJA CO-PACK for ocular toxicities until improvement at the same or reduced dose. Permanently discontinue AVMAPKI FAKZYNJA CO-PACK for any grade 4 toxicity.
Serious Skin Toxicities: Skin toxicities, including photosensitivity and severe cutaneous adverse reactions (SCARSs) occurred. Adhere to concomitant medications. Monitor for skin toxicities and interrupt, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity, tolerability and duration.
Hepatotoxicity: Monitor liver function tests prior to each cycle, on day 15 of the first 4 cycles, and as clinically indicated. Withhold, reduce or discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and persistence of abnormality.
Rhabdomyolysis: Monitor creatine phosphokinase prior to the start of each cycle, on day 15 of the first four cycles, and as clinically indicated. If increased CPK occurs, evaluate patients for rhabdomyolysis or other causes. Withhold, reduce or permanently discontinue AVMAPKI FAKZYNJA CO-PACK based on severity and duration of the adverse reaction.
Embryo-Fetal Toxicity: AVMAPKI FAKZYNJA CO-PACK can cause fetal harm. Advise patients of the potential risk to a fetus and to use effective contraception.
Adverse Reactions

The most common (≥ 25%) adverse reactions, including laboratory abnormalities, were increased creatine phosphokinase, nausea, fatigue, increased aspartate aminotransferase, rash, diarrhea, musculoskeletal pain, edema, decreased hemoglobin, increased alanine aminotransferase, vomiting, increased blood bilirubin, increased triglycerides, decreased lymphocyte count, abdominal pain, dyspepsia, dermatitis acneiform, vitreoretinal disorders, increased alkaline phosphatase, stomatitis, pruritus, visual impairment, decreased platelet count, constipation, dry skin, dyspnea, cough, urinary tract infection, and decreased neutrophil count.

Drug Interactions

Strong and moderate CYP3A4 inhibitors: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Strong and moderate CYP3A4 inducers: Avoid concomitant use with AVMAPKI FAKZYNJA CO-PACK.
Warfarin: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with warfarin and use an alternative to warfarin.
Gastric acid reducing agents: Avoid concomitant use of AVMAPKI FAKZYNJA CO-PACK with proton pump inhibitors (PPIs) or H2 receptor antagonists. If use of an acid-reducing agent cannot be avoided, administer FAKZYNJA 2 hours before or 2 hours after the administration of a locally acting antacid.
Use in Specific Populations

Lactation: Advise not to breastfeed.
Fertility: May impair fertility in males and females.
Click here for full Prescribing Information.

(Press release, Verastem, DEC 15, 2025, View Source [SID1234661434])

On December 15, 2025 XOMA Royalty Corporation ("XOMA Royalty") (NASDAQ: XOMA), the biotech royalty aggregator, reported it has entered into an agreement to acquire Generation Bio Co. ("Generation Bio") (NASDAQ: GBIO) for a cash price of $4.2913 per share at the closing of the merger. Generation Bio stockholders also will receive one non-transferable contingent value right ("CVR") per share that entitles holders to receive potential payments of a pro rata portion of:

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100% of the amount by which net cash at closing, as finally determined pursuant to the CVR agreement, exceeds $29 million;
either 90% or 100% of any savings realized by XOMA Royalty on the Company’s Cambridge office lease obligations, subject to the timing of resolution of the lease obligations;
a share of any proceeds from Generation Bio’s existing license agreement with Moderna, which includes potential development and commercial milestones and royalties on commercial sales, calculated on a sliding scale delivering up to 90% of such payments to CVR holders; and
a share of payments from any out license or sale of the Generation Bio ctLNP delivery platform, calculated on a sliding scale delivering up to 70% of such payments to CVR holders
following the closing.

Following a thorough review process conducted with the assistance of its legal and financial advisors, Generation Bio’s board of directors has determined that the acquisition by XOMA Royalty is in the best interests of all Generation Bio stockholders and has unanimously approved the Merger Agreement.

Terms
Pursuant and subject to the terms of the Merger Agreement, a wholly owned subsidiary of XOMA Royalty will commence a tender offer (the "Offer") within 15 business days, to acquire all outstanding shares of Generation Bio common stock. Closing of the Offer is subject to certain conditions, including the tender of Generation Bio common stock representing at least a majority of the total number of outstanding shares and other customary closing conditions. Immediately following the closing of the tender offer, Generation Bio will merge with a subsidiary of XOMA Royalty, and all remaining shares not tendered in the offer, other than appraisal shares, will be converted into the right to receive the same cash and CVR consideration per share as is provided in the tender offer.

Generation Bio stockholders in possession of approximately 15% of Generation Bio common stock have signed support agreements under which such stockholders agreed to tender their shares in the Offer and support the merger. The acquisition is expected to close in February 2026.

Advisors
XOMA Royalty was represented by Gibson, Dunn & Crutcher LLP. TD Cowen served as financial advisor, and Wilmer Cutler Pickering Hale and Dorr LLP served as legal counsel, to Generation Bio.

(Press release, Xoma, DEC 15, 2025, View Source [SID1234661433])

On December 15, 2025 RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, reported positive in vivo results, indicating that opaganib combined with venetoclax reduces Chronic Lymphocytic Leukemia (CLL) cells by half compared to controls, and further demonstrates opaganib’s potential as an add-on therapy to venetoclax in venetoclax-resistant CLL.

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"Understanding mechanisms of resistance to targeted therapies such as the BCL-2 inhibitor venetoclax is essential to improve current treatment strategies and may provide key insights to personalize treatment for chronic lymphocytic leukemia (CLL) patients" said Romina Gamberale, PhD, Independent Researcher at CONICET from the Institute of Experimental Medicine (IMEX, CONICET-National Academy of Medicine) in Buenos Aires, Argentina, who led the study. "Our previous ex vivo work has shown that sphingosine kinase 2 (SPHK2) is overexpressed in venetoclax-resistant CLL cells and that inhibiting SPHK2 may reduce T-cell-induced resistance and resensitize previously resistant cells. The results of this in vivo study in mice indicate that adding opaganib, a potent SPHK2 inhibitor, to venetoclax reduced CLL cell counts by 50% compared to controls, showing that opaganib may have a significant role to play in mitigating BCL-2 inhibitor resistance."

Dr. Mark Levitt, Chief Scientific Officer at RedHill said: "Venetoclax is a key CLL therapy and finding ways to maintain its effectiveness, and to reduce the potential for resistance-related treatment failure, could represent a breakthrough in the ability to treat CLL patients. This promising data supports the hypothesis that opaganib, as a potent inhibitor of SPHK2, provides a potential route to maintaining venetoclax effects in treating CLL. Opaganib has shown potential as add-on therapy in several preclinical oncology models and is currently undergoing a Phase 2 clinical trial in combination with darolutamide in advanced prostate cancer. This new data now adds CLL to the list of potential cancer indications where opaganib has shown potential to bring additive therapeutic value."

Opaganib has a safety and tolerability profile shown in more than 470 clinical trials / expanded access participants. It targets multiple oncology, virology, inflammation, medical countermeasures, diabetes and obesity indications, with several U.S. government partnerships, including BARDA funding, in place.

Approved by the FDA in 2016, venetoclax (Venclexta and Venclyxto, Abbvie / Genentech), is a first-in-class BCL-2 inhibitor that has become a mainstay of CLL therapy, achieving sales of approximately $2.5 billion in 2024. Venetoclax works by blocking a protein called BCL-2, which is often overproduced in certain cancer cells and prevents the process of apoptosis (programmed cell death) – helping to keep the cancer cells alive and growing. By binding to, and inhibiting, the BCL-2 protein, venetoclax enables the cancer cells to undergo apoptosis and die.

About Chronic Lymphocytic Leukemia (CLL)

CLL is a slow-growing blood and bone marrow cancer that affects a type of white blood cell called lymphocytes. It is the most common type of leukemia in adults and has a highly variable clinical course. It is generally not considered to be curable.

About Opaganib (ABC294640)

Opaganib is a first-in-class, proprietary investigational host-directed and potentially broad-acting orally administered drug with anticancer, anti-inflammatory and antiviral activity. Opaganib is targeted at multiple potential oncology, radioprotection, viral, inflammatory, and gastrointestinal indications, including several cancers, diabetes and obesity-related disorders, gastrointestinal acute radiation syndrome (GI-ARS), COVID-19, Ebola and other viruses as part of pandemic preparedness. Opaganib has also shown positive preclinical results in renal fibrosis.

Opaganib has received orphan-drug designations from the FDA in cholangiocarcinoma and neuroblastoma. It is currently undergoing a Phase 2 clinical trial in combination with darolutamide in advanced prostate cancer and has previously undergone studies in advanced cholangiocarcinoma (Phase 2a). Opaganib also has a Phase 1 chemoradiotherapy study protocol ready for FDA-IND submission.

Opaganib is thought to work through the inhibition of multiple pathways, the induction of autophagy and apoptosis, and disruption of viral replication, through simultaneous inhibition of three sphingolipid-metabolizing enzymes in human cells (SPHK2, DES1 and GCS).

Several U.S. government medical countermeasures and pandemic preparedness programs have selected opaganib for evaluation for multiple indications, including GI-ARS, Ebola virus disease and others. Funding bodies include the Radiation and Nuclear Countermeasures Program (RNCP), led by the National Institute of Allergy and Infectious Diseases (NIAID), part of the U.S. government Department of Health & Human Services’ National Institutes of Health, and the Administration for Strategic Preparedness and Response’s (ASPR) Center for Biomedical Advanced Research and Development Authority (BARDA).

Opaganib has demonstrated antiviral activity against SARS-CoV-2, multiple variants, and several other viruses, such as Influenza A and Ebola. Opaganib delivered a statistically significant increase in survival time when given at 150 mg/kg twice a day (BID) in a United States Army Medical Research Institute of Infectious Diseases (USAMRIID) in vivo Ebola virus study, making it the first host-directed molecule to show activity in Ebola virus disease. Opaganib also recently demonstrated a distinct synergistic effect when combined individually with remdesivir (Veklury, Gilead Sciences Inc.), significantly improving potency while maintaining cell viability, in a U.S. Army-funded and conducted in vitro Ebola virus study.

Being host-targeted, and based on data accumulated to date, opaganib is expected to maintain effect against emerging viral variants. In prespecified analyses of Phase 2/3 clinical data in hospitalized patients with moderate to severe COVID-19, oral opaganib demonstrated improved viral RNA clearance, faster time to recovery and significant mortality reduction in key patient subpopulations versus placebo on top of standard of care. Opaganib has demonstrated its safety and tolerability profile in more than 470 people in multiple clinical studies and expanded access use. Data from the opaganib global Phase 2/3 study was published in Microorganisms.

(Press release, RedHill Biopharma, DEC 15, 2025, View Source [SID1234661428])

On December 15, 2025 Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, reported the publication of the Precision-T Phase 3 study results of its lead investigational allogeneic T-cell immunotherapy, Orca-T, in Blood, a journal of the American Society of Hematology (ASH) (Free ASH Whitepaper). The study compared Orca-T to a conventional allogeneic hematopoietic stem cell transplant (alloHSCT) in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). These results were first presented on April 2, 2025, at the 51st Annual Meeting of The EBMT in Florence, Italy, and further analyses were presented at the 67th ASH (Free ASH Whitepaper) Annual Meeting on December 6, 2025.

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In the randomized Precision-T study, Orca-T plus single-agent tacrolimus (TAC) demonstrated a significant improvement in the primary endpoint of survival free from moderate-to-severe chronic graft versus host disease (cGFS) compared to alloHSCT plus tacrolimus/methotrexate (TAC/MTX). The rate for patients who received Orca-T was 78% (95% CI: 65%, 87%) compared to 38.4% (95% CI: 26%, 51%) for patients who received an alloHSCT (HR 0.26; p<0.00001), an improvement driven by a reduction in moderate-to-severe chronic graft versus host disease (cGvHD) and fewer patient deaths.

"Today, treating patients with serious blood cancers using conventional allogeneic stem cell transplants often forces a difficult risk–benefit trade-off, as clinicians work to cure the disease while avoiding life-threatening complications like GvHD," said lead author Everett Meyer, M.D., Ph.D., hematologist and associate professor of medicine in Blood and Marrow Transplantation and Cellular Therapy at Stanford Medicine. "The Precision-T results showed that Orca-T can meaningfully shift that balance, delivering improved GvHD-free survival alongside less toxicity, including fewer serious infections and lower non-relapse mortality. Orca-T has the potential to become an important new therapy for patients and a valuable new option for the providers who care for them."

In the study, all patients (n=187) with a median age of 43.6 years (range 19-65 years) were randomized 1:1 to Orca-T plus TAC or alloHSCT plus TAC/MTX. Patients across both groups received myeloablative conditioning and used a related or unrelated matched donor. Patients had a median follow-up time of 11.4 months (range 0.2-24.3 months) across both arms. Additional results from the Precision-T study at one year include:

A secondary endpoint of cumulative incidence of moderate-to-severe cGvHD was 12.6% (95% CI: 5%, 23%) and 44.0% (95% CI: 31%, 56%) in the Orca-T and alloHSCT arms, respectively (HR 0.19; p<0.00002).
The overall survival (OS), another secondary endpoint, was 93.7% (95% CI: 86%, 97%) in the Orca-T arm and 83.2% (95% CI: 73%, 90%) in the alloHSCT arm (HR 0.49; p=0.11823).
A secondary endpoint of GvHD and relapse-free survival (GRFS) was 63.1% and 30.9% with Orca-T and alloHSCT (p<0.001 in a post hoc analysis), respectively.
The cumulative incidence of non-relapse mortality (NRM) was 3.4% (95% CI: 0.9%, 8.8%) for Orca-T versus 13.2% (95% CI: 6.8%, 21.6%) for alloHSCT (HR 0.27 [95% CI: 0.08, 0.93]; p=0.03 in a post hoc analysis).

Additional safety findings were consistent with previous studies. The cumulative incidence for grade III or IV acute GvHD at day +180 was reduced with Orca-T with 6.2% (95% CI: 2.3, 12.9) versus 16.5% (95% CI: 9.4, 25.3) with alloHSCT (HR 0.37 [95% CI: 0.13, 1.02]; p=0.044 in a post hoc analysis). Grade 3+ infections were less common with Orca-T, with a one-year estimated incidence of 8.4% (95% CI: 3.6%, 16%) for Orca-T and 16.1% (95% CI: 9.2%, 25%) for alloHSCT.

An exploratory endpoint evaluated health-related quality of life (HRQoL) and hospitalization patterns and found Orca-T delivered marked improvements over conventional alloHSCT. In the data presented at ASH (Free ASH Whitepaper) 2025, patients receiving Orca-T experienced faster recovery to, and higher improvement above, baseline HRQoL, fewer ICU stays, lower likelihood of rehospitalization and higher rehospitalization-free survival, suggesting better early post-treatment recovery and a lower burden of GvHD symptoms.

Rehospitalizations due to adverse events occurred less frequently among Orca-T recipients (27.3% [24] vs. 45.7% [43]), with fewer total hospitalization days per patient (30.6 vs. 40.8). Rehospitalization-free survival at 18 months was also significantly improved with Orca-T, reaching 66.4% (95% CI: 54.0, 76.2) compared to 33.8% (95% CI: 18.5, 49.9) for conventional alloHSCT (p=0.0096 in a post hoc analysis; HR 0.53 [0.32, 0.86]).

"As the first Treg-based immunotherapy to show improved outcomes for patients with acute leukemias and myelodysplastic syndrome compared with a conventional transplant, Orca-T has the potential to become a new standard of care," said Nate Fernhoff, Ph.D., co-founder and chief executive officer at Orca Bio. "The Phase 3 results published in Blood underscore our ability to potentially redefine how blood cancers are treated today. We look forward to continuing to work closely with the FDA on the review of our application with the goal of making Orca-T available to patients who need it."

The safety and efficacy of Orca-T have not been determined by any regulatory authority. Orca-T is currently being evaluated under Priority Review by the U.S. Food and Drug Administration (FDA) with a Prescription Drug User Fee Act (PDUFA) target action date of April 6, 2026.

About Precision-T
Precision-T (NCT05316701) is a randomized, open-label multi-center study that evaluated the safety, efficacy and tolerability of Orca Bio’s lead investigational allogeneic T-cell immunotherapy, Orca-T, compared to conventional allogeneic hematopoietic stem cell transplant (alloHSCT). Orca Bio received guidance from the U.S. Food and Drug Administration on the design of Precision-T, which evaluated Orca-T in patients with acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL), high-risk myelodysplastic syndrome (MDS) and mixed-phenotype acute leukemia (MPAL). There are 19 leading treatment centers participating in the trial, which enrolled 187 patients across the U.S.

About Orca-T
Orca-T is an investigational allogeneic T-cell immunotherapy under evaluation for the treatment of multiple hematologic malignancies including acute leukemias and myelodysplastic syndromes. Orca-T is composed of highly purified regulatory T-cells, hematopoietic stem cells and conventional T-cells derived from either related or unrelated matched donors. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation for the prevention of graft versus host disease or death in patients eligible for hematopoietic stem cell transplant from the U.S. Food and Drug Administration (FDA). The Biologics License Application (BLA) for Orca-T is currently under Priority Review with the FDA with a Prescription Drug User Fee Act (PDUFA) target action date of April 6, 2026.

(Press release, Orca Bio, DEC 15, 2025, https://orcabio.com/orca-bio-announces-positive-results-from-the-pivotal-phase-3-study-of-investigational-orca-t-compared-to-allogeneic-stem-cell-transplant-for-the-treatment-of-hematologic-malignancies-2/?utm_source=rss&utm_medium=rss&utm_campaign=orca-bio-announces-positive-results-from-the-pivotal-phase-3-study-of-investigational-orca-t-compared-to-allogeneic-stem-cell-transplant-for-the-treatment-of-hematologic-malignancies-2 [SID1234661426])

(Press release, Orca Bio, DEC 15, 2025, https://orcabio.com/orca-bio-announces-positive-results-from-the-pivotal-phase-3-study-of-investigational-orca-t-compared-to-allogeneic-stem-cell-transplant-for-the-treatment-of-hematologic-malignancies-2/?utm_source=rss&utm_medium=rss&utm_campaign=orca-bio-announces-positive-results-from-the-pivotal-phase-3-study-of-investigational-orca-t-compared-to-allogeneic-stem-cell-transplant-for-the-treatment-of-hematologic-malignancies-2 [SID1234661426])