SOUTH SAN FRANCISCO (dpa-AFX) - In a holiday-shortened week, the biotech sector saw a wave of FDA approvals, strategic acquisitions, and key clinical trial readouts - alongside several setbacks that shaped the sector's outlook.
- Regulatory approvals led the week, with multiple FDA, Japanese and Chinese clearances across several therapeutic areas - in addition to a key BLA resubmission and a funding update.
- A few regulatory setbacks also emerged, including a request for an additional Phase 3 trial by the FDA and a complete response letter delaying a major multiple sclerosis program.
- BioMarin and Sanofi both made acquisition pacts, opening their wallets to expand their portfolios. Repare Therapeutics has agreed with Gilead Sciences on a deal.
- Altimmune, Dogwood Therapeutics, NeuroSense, Lexaria, and Edgewise Therapeutics announced positive clinical results.
- Biohaven disappointed investors as it failed to meet the primary endpoint in a study.
Let's unpack the specifics.
FDA Approvals & Rejections
Galectin Secures $10Mln Credit Line, FDA provides Guidance On Regulatory Pathway
Galectin Therapeutics Inc. (GALT) has received a written response from the FDA regarding its regulatory pathway for belapectin, the company's investigational therapy for NASH cirrhosis. Alongside the update, Galectin secured an additional $10 million line of credit from Richard E. Uihlein, providing sufficient funding to cover expected expenditures through March 2027.
GALT closed Wednesday's (Dec.24, 2025) trading at $4.03, up 1 %.
Genentech's Lunsumio VELO Gains FDA Nod for Follicular Lymphoma
Genentech, a Roche company (RHHBY.OB), has won FDA approval for Lunsumio VELO (mosunetuzumab-axgb), a subcutaneous CD20xCD3 bispecific therapy for relapsed or refractory follicular lymphoma. The new formulation reduces administration time from hours to about one minute, offering patients faster, more convenient treatment.
RHHBY.OB closed Wednesday's trading at $52.53, down 0.11%.
PTC Therapeutics Secures Approval for Sephience in PKU In Japan
PTC Therapeutics Inc.'s (PTCT) Sephience (sepiapterin), an oral therapy for children and adults with phenylketonuria, has received approval in Japan. The decision was based on data from a Phase 3 APHENITY trial and its extension study, which showed clinically meaningful and sustained reductions in blood phenylalanine levels across a broad patient population. The drug is already approved in the U.S. and Europe.
PTCT closed Wednesday's trading at $78.37, up 0.86%.
Incyte Receives Approvals for Two Drugs in Japan
Incyte Biosciences (INCY) has received approval from Japan's Ministry of Health for two of its drugs - Minjuvi (tafasitamab) in combination with rituximab and lenalidomide for relapsed/refractory follicular lymphoma, and Zynyz (retifanlimab), with chemotherapy, for first-line treatment of advanced anal cancer.
INCY closed Wednesday's trading at $100.44, down 0.47 %.
Savara Resubmits BLA for Molbreevi in Autoimmune PAP
Savara Inc. (SVRA) resubmitted its Biologics License Application to the FDA for Molbreevi, a therapy for autoimmune pulmonary alveolar proteinosis. The filing includes Fujifilm Biotechnologies as the drug substance manufacturer, with a request for priority review. The U.S. regulatory agency refused to accept the MOLBREEVI BLA submitted in March 2025, citing that the application was incomplete for a substantive review and requested additional data on Chemistry, Manufacturing, and Controls.
SVRA closed Wednesday's trading at $6.40, down 0.62 %.
KalVista's Ekterly Approved in Japan for Hereditary Angioedema
KalVista Pharmaceuticals, Inc. (KALV) announced Japanese approval of Ekterly (sebetralstat), the first and only oral on-demand treatment for hereditary angioedema. The therapy will be commercialized in Japan by partner Kaken Pharmaceutical. EKTERLY is also approved in the United States, European Union, United Kingdom and Switzerland for the treatment of acute attacks of hereditary angioedema (HAE) in people 12 years of age and older.
KALV closed Wednesday's trading at $15.79, up 0.25 %.
Edwards Lifescience Secures FDA Approval for SAPIEN M3 Mitral Valve
Edwards Lifesciences (EW) has secured FDA approval for its SAPIEN M3 transcatheter mitral valve replacement system, the first transseptal option for patients with symptomatic moderate-to-severe or severe mitral regurgitation. The device reflects evidence supporting use in patients unsuitable for surgery or TEER, including those with mitral annular calcification. The system previously earned CE Mark in April 2025, becoming the first transfemoral TMVR platform in Europe.
EW closed Wednesday's trading at $86.29, up 0.07 %.
Reviva's NDA Filing for Brilaroxazine Faces Delay
Reviva Pharmaceuticals (RVPH) has been asked to conduct a second Phase 3 trial to generate additional efficacy and safety data prior to NDA submission of brilaroxazine for the treatment of schizophrenia. As a result, the filing is now expected no earlier than 2027, despite positive results from the completed RECOVER study, which showed efficacy and favourable safety.
RVPH closed Wednesday's trading at $0.27, down 13.48%.
Agios Wins FDA Approval for AQVESME in Thalassemia
Agios Pharmaceuticals, Inc. (AGIO) received FDA approval for AQVESME (mitapivat) for anaemia in adults with alpha-or beta-thalassemia. The decision was based on results from the Phase 3 ENERGIZE and ENERGIZE-T trials, which showed significant improvements in haemoglobin and quality-of-life measures. AQVESME will be distributed under an FDA-mandated REMS program due to liver injury risks observed in a small number of patients. The active ingredient in AQVESME is the same as that in the company's marketed drug for pyruvate kinase deficiency, PYRUKYND.
AGIO closed Wednesday's trading at $29.17, up 18.63%.
Dupixent Approved in Japan for Children with Asthma
Regeneron Pharmaceuticals (REGN) and Sanofi (SNY) have received approval in Japan for Dupixent (dupilumab) in treating children aged 6-11 with severe or refractory asthma. The update is supported by a global Phase 3 program and marks the first and only biologic shown to improve lung function in the pediatric group. Dupixent already holds multiple approvals in the U.S. and other markets across type 2 inflammatory diseases.
REGN closed Wednesday's trading at $783.71, down 0.19 %.
Zai Lab Secures Approval for Cobenfy in Schizophrenia in China
Zai Lab Limited (ZLAB) has gained NMPA clearance in China for Cobenfy (xanomeline and trospium chloride) for adults with schizophrenia. The therapy is the first novel mechanism option in more than 70 years and is now included in national treatment guidelines. The decision is supported by data from a Phase 2 PK study in China, the Phase 3 ZL-2701-001 trial, and three global EMERGENT studies, and builds on Cobenfy's earlier U.S. authorisation in 2024.
ZLAB closed Wednesday's trading at $18.58, up 6.29%.
FDA Denies Approval For Sanofi's Tolbrutinib
Sanofi (SNY) has received a complete response letter from the FDA for its tolebrutinib filing in non-relapsing secondary progressive MS. The US regulatory agency has sought additional data before reconsideration. The decision delays potential approval despite a prior breakthrough therapy designation, highlighting the drug's promise in addressing disability progression in MS.
SNY closed Wednesday's trading at $48.36, up 0.08%.
Omeros Wins FDA Nod for YARTEMLEA in TA-TMA
Omeros Corporation (OMER) announced that the FDA has approved YARTEMLEA (narsoplimab-wuug) for the treatment of transplant-associated thrombotic microangiopathy (TA-TMA) in adults and children aged two years and older. The approval follows an earlier FDA Complete Response Letter, with resubmission supported by Phase 2 and expanded access data showing improvements in response and survival rates.
OMER closed Wednesday's trading at $15.36, up 75.54 %.
Deal or No Deal
BioMarin To Acquire Amicus Therapeutics for Around $4.8 Bln BioMarin Pharmaceutical Inc. (BMRN) has agreed to acquire Amicus Therapeutics (FOLD) for $14.50 per share in cash - valued around $4.8 billion in total. The deal is expected to close in the second quarter of 2026.
BMRN closed Wednesday's trading at $60.16, down 1.60 %.
Sanofi to Commence Cash Tender Offer to Acquire Dynavax
Sanofi (SNY, SAN.PA) has agreed to acquire Dynavax Technologies (DVAX), a vaccines company with a marketed adult hepatitis B vaccine and a differentiated shingles vaccine candidate. Sanofi will commence a cash tender offer to acquire all outstanding shares of Dynavax for $15.50 per share in cash, reflecting a total equity value of approximately $2.2 billion.
SNY closed Wednesday's trading at $48.36, up 0.08%.
Repare Therapeutics to Sell Polymerase Theta ATPase Inhibitor to Gilead
Repare Therapeutics Inc. (RPTX), a clinical-stage precision oncology company, has entered into a definitive agreement with Gilead Sciences, Inc. (GILD) for the acquisition of its polymerase theta ATPase inhibitor, RP-3467. Under the terms of the agreement, Repare will receive up to $30 million in total consideration, including a $25 million upfront payment and an additional $5 million milestone payment tied to technology transfer activities.
RPTX closed Wednesday's trading at $2.55, up 17.51%.
Clinical Trials - Breakthrough and Setbacks
Altimmune's Pemvidutide Improved Fibrosis Markers and Weight Loss In Phase 2 Trial
Altimmune Inc.'s (ALT) investigational dual glucagon/GLP-1 receptor agonist, Pemvidutide, achieved statistically significant improvements in non-invasive markers of liver fibrosis and sustained weight loss at 48 weeks in its Phase 2b IMPACT trial in patients with metabolic dysfunction-associated steatohepatitis. Key findings highlighted improvements in fibrosis markers, liver health, and weight loss, along with a favorable safety profile.
ALT closed Wednesday's trading at $3.98, down 0.50%.
NeuroSense Revealed Favourable Safety Analysis Data of PrimeC in Alzheimer's Trial
NeuroSense Therapeutics Ltd. (NRSN) reported the completion of the safety analysis and favourable data from its phase 2 study of PrimeC in Alzheimer's disease, dubbed NST-AD-001. Safety analysis showed good tolerability, with no serious adverse events or new safety concerns.
NRSN closed Wednesday's trading at $0.81, down 2.68 %.
Dogwood's Halneuron Showed Evidence of Pain Relief in Phase 2b Trial
Dogwood Therapeutics Inc.'s (DWTX) ongoing Phase 2b study of Halneuron in chemotherapy-induced neuropathic pain yielded positive interim results, with treated patients demonstrating clear separation from placebo in pain improvement assessments.
DWTX closed Wednesday's trading at $4.38, up 12.88%.
Lexaria Aces Phase 1b GLP-1-H24-4 Study
Lexaria Bioscience Corp.'s (LEXX) Phase 1b, 12-week chronic study testing DehydraTECH-CBD, DehydraTECH-semaglutide and a combination of DehydraTECH-CBD with DehydraTECH-semaglutide in overweight, obese, pre-diabetic and/or type-2 diabetic patients achieved the safety and tolerability-based final primary endpoint. The three study arms were compared to the Rybelsus control study arm. The company expects to release additional non-primary (secondary/exploratory) endpoint results next week.
LEXX closed Wednesday's trading at $0.56, down 9.68%.
Edgewise To Report full 12-week Part D Data of CIRRUS-HCM Trial In Q2
Edgewise Therapeutics Inc. (EWTX) has completed Parts B and C of its Phase 2 CIRRUS-HCM trial evaluating EDG-7500 in hypertrophic cardiomyopathy, alongside favourable interim safety results from the ongoing Part D study.
Interim data from the ongoing Part D study showed EDG-7500 was generally well tolerated, with no clinically meaningful reductions in left ventricular ejection fraction and no atrial fibrillation events detected under continuous monitoring. These findings highlight EDG-7500's differentiated safety profile compared to cardiac myosin inhibitors. The full 12-week Part D readout is expected in the second quarter of 2026, with phase 3 trials planned for initiation by year-end.
EWTX closed Wednesday's trading at $27.29, up 25.50%.
Foresee Pharma Reported Positive Topline Results From Phase 3 Caspian Trial
Foresee Pharmaceuticals (6576.TWO) announced positive topline results from the Phase 3 Caspian trial evaluating the efficacy and safety of FP-001 42 mg, an investigational, sustained-release GnRH agonist, administered every six months in children with Central Precocious Puberty.
At Week 24, the proportion of patients achieving serum luteinizing hormone suppression to less than 4 mIU/mL at 60 minutes following a GnRHa stimulation test was 94%, which was statistically significant and exceeded the prespecified success criterion as reported by the firm.
Biohaven's Phase 2 Study of BHV-7000 In MDD Fails to Meet Key Goal
Biohaven (BHVN) reported that a Phase 2 proof-of-concept study evaluating BHV-7000 for the treatment of major depressive disorder did not meet its primary endpoint, a reduction of depressive symptoms as measured by change in the Montgomery Åsberg Depression Rating Scale over six weeks compared with placebo.
Additional analyses are ongoing, and the company plans to present the results at an upcoming scientific meeting.
BHVN closed Wednesday's trading at $10.81, up 2.95%.
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