Pharma and biotech news, week of Sep 21 – Sep 27, 2026

The U.S. FDA has approved Merck and Eisai's dual oral regimen of WELIREG plus LENVIMA for adult patients with advanced clear cell renal cell carcinoma (ccRCC) following a PD-1/PD-L1 inhibitor. Approval was based on the Phase 3 LITESPARK-011 trial, where the combination reduced the risk of disease progression or death by 26% compared to cabozantinib. Detailed results will be presented at the ESMO Congress 2026.AI summary

Health Canada has approved Rhythm Pharmaceuticals' IMCIVREE® (setmelanotide) for weight management in adult and pediatric patients aged 4 years and older with acquired hypothalamic obesity (HO). The decision was backed by data from the pivotal Phase 3 TRANSCEND trial. Rhythm plans to focus on supporting patient access to the drug across Canada.AI summary

Japan's Ministry of Health, Labor and Welfare has approved Merck's KEYTRUDA QLEX (KEYJECT) for subcutaneous injection across all indications approved for intravenous KEYTRUDA in Japan. The approval is based on Phase 3 trial data showing comparable pharmacokinetic exposure and consistent overall response rates between the formulations. KEYJECT can be administered by a healthcare provider in one to two minutes.AI summary

The U.S. FDA has approved Mirum Pharmaceuticals and Incyte's Atebrioz (zilurgisertib) to reduce new heterotopic ossification volume in patients aged 12 and older with fibrodysplasia ossificans progressiva. In the trial, mean total new lesion volume decreased by 3.2 cm³ with zilurgisertib versus a 24.6 cm³ increase with placebo at 24 weeks. U.S. commercial availability is expected in October 2026.AI summary

The FDA has approved Elevar Therapeutics' Lyrfigtu (lirafugratinib) as a second-line treatment for cholangiocarcinoma with FGFR2 fusion or rearrangement. In the Phase 1/2 ReFocus trial, Lyrfigtu achieved a confirmed objective response rate of 46% and a median duration of response of 11.8 months. Elevar expects the drug to be available in the U.S. by Q4 2026.AI summary

The FDA approved Teva Pharmaceuticals, Inc.'s biologics license application for Degevma (denosumab-adet) injection. It is approved as a biosimilar to and interchangeable with US-Xgeva for multiple myeloma, bone metastases from solid tumors, giant cell tumor of bone, and refractory hypercalcemia of malignancy.AI summary

The FDA approved AbbVie Inc.'s new drug application for Juvmo (tavapadon) tablets in 0.25 mg, 1 mg, 5 mg, 10 mg, and 15 mg strengths. The approval provides for the treatment of Parkinson’s disease in adults. PDUFA goal date: 2026-07-26.AI summary

Daiichi Sankyo and Merck have voluntarily withdrawn the Biologics License Application seeking accelerated approval for ifinatamab deruxtecan in adult patients with extensive-stage small cell lung cancer. The decision follows FDA discussions indicating that data, including results from the Phase 2 IDeate-Lung01 trial, do not meet requirements for accelerated approval. Patient enrollment continues in the Phase 3 IDeate-Lung02 trial.AI summary

The FDA approved Eli Lilly and Company's new biologics license application for Onswik (insulin efsitora alfa-gobe) injection. The product is approved as an adjunct to diet and exercise to improve glycemic control in adults with type 2 diabetes mellitus.AI summary

AstraZeneca's Trixeo Aerosphere has been approved in the European Union as a maintenance treatment for asthma in patients aged 12 and older. The approval was based on Phase 3 KALOS and LOGOS trials, where Trixeo demonstrated statistically significant improvements in lung function and reductions in severe exacerbations versus dual-combination comparators. Trixeo is the first single-inhaler triple-combination therapy approved in the EU for this patient population.AI summary

The U.S. FDA has approved a U.S. product label update for Merck's WINREVAIR (sotatercept-csrk) based on data from the Phase 3 HYPERION trial. The update incorporates efficacy and safety data showing WINREVAIR added to background therapy reduced the risk of clinical worsening events by 76% in adults newly diagnosed with pulmonary arterial hypertension compared to placebo. The expanded label provides clinicians with meaningful data on utilizing WINREVAIR earlier in the treatment journey.AI summary

Lantheus received final FDA approval for BRAVNETSA (lutetium Lu 177 dotatate) via the Abbreviated New Drug Application pathway. It is indicated for adult patients with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors. Lantheus is focusing on a commercial launch to support supply and patient access.AI summary

The European Commission has granted marketing authorisation under exceptional circumstances for NEZGLYAL (leriglitazone) to treat male cALD patients aged 2-12 years with Gadolinium-negative brain lesions. The approval, based on Phase 2/3 NEXUS study data, makes it the first approved pharmacological treatment for cALD in the EU. A first European launch is expected in Germany by the end of 2026.AI summary

The US FDA has granted Priority Review to AstraZeneca's sBLA for Imfinzi in combination with neoadjuvant enfortumab vedotin for muscle-invasive bladder cancer patients ineligible for or declining cisplatin-based chemotherapy. The application is based on VOLGA Phase III trial results showing statistically significant improvements in event-free and overall survival. The PDUFA regulatory decision date is anticipated in the fourth quarter of 2026.AI summary

aTyr Pharma has aligned with the U.S. FDA on the protocol for a Phase 3 study evaluating efzofitimod in pulmonary sarcoidosis patients with restrictive lung disease. The planned global trial will enroll up to approximately 372 patients, using change in forced vital capacity at week 48 as the primary endpoint. Study-related activities are expected to begin in the fourth quarter of 2026.AI summary

Gossamer Bio has submitted a New Drug Application to the U.S. FDA for seralutinib to treat pulmonary arterial hypertension. The submission is supported by data from the Phase 3 PROSERA study and the Phase 2 TORREY study. Gossamer anticipates the FDA will determine whether to accept the NDA for filing later this year.AI summary

The FDA recommended that Compass Therapeutics conduct a new trial demonstrating a survival benefit before submitting a Biologics License Application for tovecimig in advanced biliary tract cancer. Compass does not believe a new trial is warranted based on Phase 2/3 COMPANION-002 study data. The company intends to further engage with the FDA as it continues preparing its BLA submission.AI summary

The U.S. FDA cleared Prime Medicine's Investigational New Drug application for PM647, an in vivo Prime Editor for Alpha-1 Antitrypsin Deficiency. The clearance enables the company to initiate a Phase 1/2 trial evaluating ascending doses of the one-time infusion in adults with AATD. Initial clinical data from the trial is expected in 2027.AI summary

The U.S. FDA accepted Pharming's supplemental New Drug Application (sNDA) for lower doses of Joenja (leniolisib) to treat APDS in children aged 4 and older weighing 13 kg to 27 kg. The application received Priority Review supported by Phase III trial data showing improved lymphadenopathy and naive B cells. The FDA set a PDUFA target action date of January 30, 2027.AI summary

Elevar Therapeutics announced that the FDA granted conditional acceptance of LYRFIGTU as the brand name for lirafugratinib. The drug is under FDA Priority Review for advanced/metastatic cholangiocarcinoma harboring FGFR2 fusion or other rearrangement, with a PDUFA date of September 25, 2026. Elevar is currently preparing for a potential commercial launch pending FDA approval.AI summary

Kodiak Sciences announced that it will report topline results from the Phase 3 DAYBREAK study on September 28, 2026. The study evaluates Zenkuda and KSI-501 for the treatment of patients with wet age-related macular degeneration. The company will host a webcast to present the data.AI summary

Alexion announced 52-week open-label extension data from the Phase III PREVAIL trial of gefurulimab in adults with generalised myasthenia gravis (gMG). Results showed sustained clinical improvements, including an MG-ADL score change of -5.3, with no reported meningococcal infections. Alexion will present these and other gMG data across 15 presentations at upcoming medical meetings.AI summary

Milestone Pharmaceuticals has enrolled its first Canadian participant in the Phase 3 ReVeRA-301 trial evaluating etripamil nasal spray for atrial fibrillation with rapid ventricular rate (AFib-RVR). The trial is testing a self-administered 70 mg repeat-dose regimen outside of emergency settings. The expansion advances the multinational study toward its target of approximately 150 patient events.AI summary

Ionis Pharmaceuticals and Roche announced positive prespecified interim results from the Phase 3 IMAgINATION study evaluating sefaxersen in adults with primary IgA nephropathy. Sefaxersen met its primary endpoint by demonstrating statistically significant and clinically meaningful reductions in proteinuria compared to placebo at 37 weeks. Interim data will be presented at an upcoming medical congress and shared with health authorities.AI summary

Vistagen reported preliminary positive data from the open-label extension portion of its PALISADE-4 Phase 3 study of fasedienol nasal spray for social anxiety disorder. Treatment up to six times daily for up to 12 months was well-tolerated, demonstrating exploratory improvements in social anxiety scores over four months. Vistagen is preparing to meet with the FDA during the current quarter to discuss a proposed new registrational Phase 3 clinical trial.AI summary

Ionis Pharmaceuticals and Otsuka announced positive topline results from the Phase 3 FUSION trial of ulefnersen in patients with FUS-ALS. The study met its primary endpoint measuring functional impairment and survival compared to placebo (p=0.0005). Otsuka plans to discuss the results with global health authorities to explore potential expedited regulatory submission pathways.AI summary

Amgen announced positive topline Phase 3 OASIZ 301 study results for dazodalibep in adults with moderate-to-severe systemic Sjögren's disease. The trial met its primary endpoint, demonstrating statistically significant and clinically meaningful improvement in disease activity at Week 48. Amgen plans to present detailed data at an upcoming medical meeting, with another Phase 3 trial expected to complete in Q4 2026.AI summary

Celldex reported positive topline results from its Phase 3 EMBARQ-CSU1 and EMBARQ-CSU2 trials evaluating barzolvolimab in patients with chronic spontaneous urticaria. Both studies met their primary endpoint at Week 12 along with all key secondary endpoints, with barzolvolimab demonstrating a favorable safety profile through 24 weeks. Treatment will continue through 52 weeks, and the company plans to submit a BLA to the FDA in 2027.AI summary

Johnson & Johnson announced positive topline results from the pivotal Phase 3 Study 451 evaluating CAPLYTA (lumateperone) for manic episodes in adults with bipolar I disorder. The trial met its primary endpoint with a statistically significant 4.8-point reduction in YMRS total score versus placebo at Week 3. Data analysis is currently underway for a second completed pivotal Phase 3 study.AI summary

Beacon Therapeutics announced positive topline results from the pivotal Phase 2/3 VISTA trial evaluating laru-zova for X-linked retinitis pigmentosa. The trial met its primary endpoint, showing a statistically significant proportion of high and low dose participants achieved a ≥15-letter visual acuity improvement at Month 12 versus control. Beacon plans to initiate a rolling BLA submission later this year.AI summary

Johnson & Johnson announced long-term follow-up data from cohort A of the Phase 2 CARTITUDE-2 study evaluating CARVYKTI in relapsed or refractory multiple myeloma. At five years, 50% of patients remained alive and progression-free after a single infusion without maintenance therapy. Findings were presented at the International Myeloma Society Annual Meeting.AI summary

Kyverna Therapeutics announced positive one-year topline data from its KYSA-8 trial of miv-cel in stiff person syndrome and longer-term follow-up from the Phase 2 KYSA-6 trial in generalized myasthenia gravis. The treatment demonstrated sustained clinical improvements with a well-tolerated safety profile. Kyverna remains on track to complete its rolling BLA submission for stiff person syndrome in the fourth quarter of 2026.AI summary

REGENXBIO announced positive three-year follow-up data from the Phase II ALTITUDE study of surabgene lomparvovec in diabetic retinopathy. At Dose Level 3, 60% of participants achieved a >2-step improvement on the DRSS without additional treatment, and no intraocular inflammation was observed. Sura-vec is currently being evaluated in the Phase IIb/III NAAVIGATE trial.AI summary

Acadia Pharmaceuticals announced topline Phase 2 results from its RADIANT trial evaluating remlifanserin for Alzheimer’s disease psychosis. The 60 mg dose narrowly missed the primary SAPS-H+D endpoint (p=0.0603) but achieved nominal significance on the key secondary CGI-S-ADP endpoint (p=0.0077) with a favorable safety profile. Acadia plans to continue enrolling Phase 3 studies while dropping the 30 mg dose.AI summary

Verastem Oncology announced that abstracts from the Phase 2 RAMP 201 trial of avutometinib plus defactinib in recurrent low-grade serous ovarian cancer (LGSOC) were selected for presentations at the IGCS 2026 Annual Global Meeting. The presentations include a late-breaking oral presentation on molecular profiling and tumor biomarker analysis, as well as a seminal oral presentation of the Phase 2 RAMP 201J study in Japanese patients.AI summary

Oruka Therapeutics announced positive Week 28 data from its ongoing Phase 2a EVERLAST-A trial evaluating ORKA-001 in moderate-to-severe plaque psoriasis. Response rates deepened without dosing after Week 4, with PASI 100 reaching 71.4% and PASI 90 reaching 87.3%. Full 52-week data for EVERLAST-A and 16-week Phase 2b EVERLAST-B data are expected in late 2026.AI summary

Pharming announced positive Phase II topline data for leniolisib in primary immunodeficiencies with immune dysregulation. Results showed clinical improvements including a mean 26.4% spleen volume reduction and a favorable safety profile in 13 subjects. Additional data will be presented at ESID 2026, and Pharming expects to report separate Phase II CVID results in Q4 2026.AI summary

Vertex Pharmaceuticals announced positive results from the Phase 2b AMPLIFIED study of inaxaplin in APOL1-mediated kidney disease. Treatment led to Week 13 UACR reductions of 42.7% in participants with modest proteinuria and 17.3% in those with type 2 diabetes. Vertex completed enrollment in the Phase 2/3 AMPLITUDE trial, with interim analysis data expected in early 2027.AI summary

Adicet Bio announced it will report clinical data from a Phase 1 study evaluating prula-cel for systemic lupus erythematosus with or without lupus nephritis on September 28, 2026. The company will host a live webcast presentation at 8:00 a.m. ET to discuss the results. An archived replay will be available on the company's website after the event.AI summary

Ollin Biosciences announced an upcoming presentation of results from its completed head-to-head JADE trial evaluating OLN324 versus faricimab at the EURETINA 2026 Congress. In the trial, OLN324 demonstrated faster and greater anatomic outcomes as well as numerically greater vision gains versus faricimab in DME and wAMD patients. Global Phase 3 trials in DME and wAMD are planned to initiate in Q4 2026.AI summary

Orca Bio announced the publication of long-term retrospective data evaluating TREGZI in patients with hematologic malignancies. Data from a Phase 1b trial showed TREGZI achieved an 83% three-year overall survival rate compared to 66% for a registry cohort receiving PTCy. The published findings support the therapeutic value of high-precision graft engineering platforms in blood cancer.AI summary

Alkermes announced positive topline results from a phase 1 proof-of-concept study evaluating ALKS 7290 in adults with ADHD and healthy volunteers. The drug was generally well tolerated, demonstrating dose-dependent, clinically meaningful improvements in ADHD symptom scores at day 14. An ongoing phase 2 study is currently enrolling, with data expected in 2027.AI summary

Johnson & Johnson announced that TREMFYA (guselkumab) met its primary and major secondary endpoints in the Phase 4 STAR study evaluating biologic-naïve adults with active psoriatic arthritis and axial involvement. At Week 24, TREMFYA demonstrated significant improvement in axial symptoms and a reduction in inflammation by MRI assessment compared to placebo. Detailed efficacy and safety results will be presented at an upcoming scientific congress.AI summary

Sarepta Therapeutics announced it will present new clinical and preclinical data from its Duchenne muscular dystrophy portfolio at the 31st Annual Congress of the World Muscle Society. Presentations include a late-breaking poster on delandistrogene moxeparvovec in ambulatory patients aged 8–12 years, along with data on golodirsen and casimersen. Abstracts and presentations will be published on Sarepta's website following the congress.AI summary

Merck announced topline results from the pivotal Phase 2b/3 BRUNELLO trial evaluating remigromig in adults with diabetic macular edema. At 52 weeks, both doses of remigromig demonstrated non-inferiority to active control ranibizumab for mean change in best-corrected visual acuity. The year one results will be presented at the AAO meeting and discussed with regulatory authorities.AI summary

Vistagen presented positive exploratory data at Psych Congress 2026 on fasedienol nasal spray for very severe social anxiety disorder. Subpopulation analyses from Phase 3 and Phase 2a trials showed statistically significant improvements in anxiety symptoms during a public speaking challenge compared to placebo. These findings will help inform Vistagen's ongoing evaluation of fasedienol.AI summary

Sarepta Therapeutics announced it will present seven posters featuring new data from its neuromuscular portfolio at the 27th Annual Neuromuscular Study Group Scientific Meeting. The presentations include initial Phase 4 ENDURE study results and real-world safety data for delandistrogene moxeparvovec in Duchenne muscular dystrophy. Additionally, Sarepta will share interim Phase 1/2 findings for SRP-1003 and SRP-1001.AI summary

Mineralys Therapeutics announced that data from its Transform-HTN open-label extension trial of lorundrostat will be presented at the American Heart Association Scientific Sessions 2026. The late-breaking science presentation will highlight long-term efficacy and safety data in uncontrolled hypertension. The ongoing trial evaluates participants who completed eligible parent studies to assess automated office systolic blood pressure changes.AI summary

Roivant announced topline results from a proof-of-concept trial evaluating IMVT-1402 (imeroprubart) in cutaneous lupus erythematosus (CLE). The study did not achieve statistical significance on its primary endpoint of percent change in CLASI-A score at Week 12. Immunovant plans to stop development of IMVT-1402 in CLE while continuing development across other autoimmune diseases.AI summary

Immunovant reported topline results from a proof-of-concept trial of IMVT-1402 (imeroprubart) in cutaneous lupus erythematosus (CLE). The study failed to achieve statistical significance on its primary endpoint of percent change in CLASI-A score at Week 12. Consequently, Immunovant plans to stop developing IMVT-1402 for CLE while advancing development across other autoimmune diseases.AI summary

Incyte announced that data from across its Inflammation and Autoimmunity portfolio will be presented at the EADV Congress 2026. The presentations will showcase findings on ruxolitinib cream and povorcitinib across conditions including atopic dermatitis, hidradenitis suppurativa, prurigo nodularis, and vitiligo. The company aims to demonstrate its commitment to advancing innovation in immune-mediated skin diseases.AI summary

Viking Therapeutics announced positive top-line results from a maintenance study of GLP-1/GIP receptor dual agonist VK2735 for obesity. Subjects transitioned to less frequent dosing maintained up to 97% of weight loss with every-other-week regimens and up to 90% with monthly regimens. Viking plans to explore oral maintenance dosing regimens in an upcoming part 2 of the study.AI summary

Telix Pharmaceuticals has signed a strategic agreement to lead a merger with ITM Isotope Technologies Munich SE, a radioisotope producer with 2025 revenue of US$273 million. Telix will pay US$1.65 billion upfront, including about US$1.25 billion in Telix Shares, plus up to US$700 million in contingent consideration tied to ITM-11 regulatory approvals and sales. The transaction is subject to Telix Shareholder approval and is expected to close by the end of FY 2026.AI summary

Viking Therapeutics announced the pricing of upsized concurrent public offerings of 7,857,143 shares of common stock at $35.00 per share and $225.0 million of 2.00% convertible senior notes due 2032. The offerings are scheduled to settle on September 25, 2026. Net proceeds will fund the clinical development, advancement, and commercialization of VK2735, the clinical advancement of VK3019, and general corporate purposes.AI summary

Viking Therapeutics announced proposed public offerings of $200.0 million of common stock and $200.0 million aggregate principal amount of convertible senior notes due 2032. The company intends to use the net proceeds for the clinical development, advancement, and commercialization of its VK2735 program, the development of its VK3019 program, and general corporate purposes.AI summary

Biotechnology company. Total offering $50M; first sale 2026-09-08. Form D is filed up to 15 days after the first sale.

Lexeo Therapeutics has agreed to acquire Mantle Therapeutics for up to $21.3 million, including $8.3 million upfront, and entered into three research collaborations to expand its Friedreich ataxia pipeline. The deals add multiple non-gene therapy and CNS-targeted programs to complement its lead candidate, LX2006. Lexeo expects to provide a program prioritization update in early 2027 and submit an IND for its next FA candidate in 2027.AI summary

Biotechnology company. Total offering $40M; first sale 2026-09-18. Form D is filed up to 15 days after the first sale.

Biotechnology company. Total offering $10M; first sale 2026-09-07. Form D is filed up to 15 days after the first sale.

NVIDIA has joined a global coalition including Google DeepMind and EMBL-EBI to release predicted 3D structures for protein complexes of over 2,800 viruses. The dataset was generated using AlphaFold2 optimized with NVIDIA BioNeMo Inference Runtime and is openly available through the AlphaFold Database. NVIDIA is also releasing the BioNeMo Structure Prediction Pipeline to allow researchers to predict 3D structures for their own targets.AI summary

Zymeworks Inc. has completed its previously announced acquisition of Theravance Biopharma, making Theravance part of the company. The transaction supports Zymeworks' strategy to build a revenue-generating biotechnology business combining R&D with commercial and royalty-based cash flows. Zymeworks will host a conference call on September 28, 2026, to discuss final transaction details.AI summary

PTC Therapeutics has completed its agreement with Sangamo Therapeutics to acquire ST-920, a BLA-stage AAV gene therapy for Fabry disease. A rolling BLA submission to the FDA for accelerated approval of the therapy is expected to be finished in Q4 2026.AI summary

Cullinan Therapeutics outlined its fourth quarter 2026 milestones across its immunology and oncology T cell engager portfolio. The company plans to share data updates for velinotamig in November, as well as data updates for CLN-978 and CLN-049 in December. Cullinan also plans to initiate a potentially registrational Phase 2 study for CLN-049 following a recent meeting with the FDA.AI summary

Breye Therapeutics announced the publication of pre-clinical and Phase 1b data for its lead asset, danegaptide, in Science Translational Medicine. The study demonstrated that the oral small molecule protects the inner blood-retina barrier in non-proliferative diabetic retinopathy. Breye is planning a Phase 2 proof-of-concept trial in NPDR set to begin in 2027.AI summary

Eli Lilly and Company has broken ground on a new $6.5 billion active pharmaceutical ingredient manufacturing facility in Houston, Texas. The site will produce Foundayo (orforglipron), a GLP-1 receptor agonist approved for weight management, alongside other small-molecule medicines and advanced therapeutics. The project will create more than 600 high-wage jobs and includes $12.5 million to support workforce training with San Jacinto College.AI summary