Pharma and biotech news, week of Sep 7 – Sep 13, 2026

The FDA has approved Scholar Rock's ISEMBYLD (apitegromab-mstn) for adults and pediatric patients aged two and older with spinal muscular atrophy who are receiving SMN2-targeted treatment. In the Phase 3 SAPPHIRE trial, the drug demonstrated a statistically significant 2.2-point HFMSE improvement in motor function at 10 mg/kg compared to placebo. Scholar Rock has initiated the U.S. commercial launch, with product shipments expected in the coming days.AI summary

The FDA has approved Pharming's sNDA for Joenja (leniolisib) to treat children aged 4 to 11 years with APDS weighing at least 27 kg. Supported by Phase III trial data showing improved lymphadenopathy and B cell levels, Joenja is the first FDA-approved therapy for this pediatric group. Pharming expects the new doses to be available in the U.S. in October.AI summary

The FDA approved Telix Pharmaceuticals (US) Inc.'s new drug application for Pixclara (floretyrosine F 18) injection. The application provides for using Pixclara with positron emission tomography (PET) to differentiate recurrent or progressive glioma from treatment-related change in adults and pediatric patients 1 month of age and older. PDUFA goal date: 2026-09-11.AI summary

Japan’s Ministry of Health, Labour and Welfare has approved a prefilled syringe presentation of GSK's Shingrix for the prevention of shingles. The ready-to-use presentation replaces a two-vial format that required manual combination prior to administration. The approval does not involve any change in dosing or indication.AI summary

The FDA approved a supplemental new drug application from Biofrontera Inc. for Ameluz (aminolevulinic acid HCl) topical gel, 10%. The approval provides for a new indication to treat superficial basal cell carcinoma (sBCC). PDUFA goal date: 2026-09-28.AI summary

The U.S. FDA accepted Intellia Therapeutics' Biologics License Application for Lonvoguran Ziclumeran (lonvo-z) for the treatment of hereditary angioedema. The FDA granted the application Priority Review and set a PDUFA target action date of March 10, 2027. Intellia noted that the FDA is not currently planning to hold an advisory committee meeting.AI summary

Taiho Oncology, Taiho Pharmaceutical, and Cullinan Therapeutics announced results from the Phase 3 REZILIENT3 trial evaluating zipalertinib plus chemotherapy in first-line EGFR exon 20 insertion mutation-positive NSCLC. The combination demonstrated a statistically significant median progression-free survival benefit of 6.0 months compared to chemotherapy alone (14.5 vs. 8.5 months). The companies plan to discuss these results with health authorities.AI summary

Hansoh Pharmaceutical and licensor GSK presented Phase III ARTEMIS-008 trial data showing risvutatug rezetecan (Ris-Rez) reduced the risk of death by 54% compared to topotecan in relapsed small cell lung cancer patients. Patients receiving Ris-Rez achieved a median overall survival of 18.5 months versus 10.3 months with topotecan. GSK is advancing a global clinical development program for Ris-Rez, including the Phase III EMBOLD SCLC-301 trial.AI summary

AstraZeneca announced that its Phase III SERENA-4 trial evaluating Etcamah in combination with palbociclib did not meet its primary endpoint of statistically significant progression-free survival in upfront 1st-line ER-positive, HER2-negative advanced breast cancer. A numerical improvement in progression-free survival was observed, with no new safety concerns identified. Data from the trial will be shared in due course.AI summary

Cullinan Therapeutics will host a virtual event on September 14, 2026, to showcase Phase 3 REZILIENT3 trial results of zipalertinib plus chemotherapy. The trial, evaluating first-line EGFR exon 20 insertion mutation-positive NSCLC, previously met its primary endpoint of progression-free survival at a planned interim analysis. The data will be presented at the IASLC 2026 World Conference on Lung Cancer.AI summary

Rhythm Pharmaceuticals announced new presentations showcasing data for setmelanotide at the European Society for Paediatric Endocrinology Meeting. Real-world French data showed reductions in BMI and hunger for up to 18 months in 30 paediatric patients with acquired hypothalamic obesity. Additionally, a post-hoc analysis from the Phase 3 TRANSCEND trial demonstrated improvements across multiple composite metabolic index scores.AI summary

Minoryx Therapeutics and Neuraxpharm completed enrollment in the global Phase 3 CALYX trial, which enrolled 41 adult male patients with cerebral Adrenoleukodystrophy. The trial evaluates leriglitazone (NEZGLYAL), with top-line results expected in early 2028. Data from CALYX are intended to support future label expansion in the EU and enable an NDA filing in the US.AI summary

Amgen announced that its Phase 3 DeLLphi-305 study met its primary endpoint of overall survival at a pre-specified interim analysis. IMDELLTRA in combination with AstraZeneca's Imfinzi demonstrated a statistically significant improvement in overall survival compared to Imfinzi alone as first-line maintenance for extensive stage small cell lung cancer. Amgen plans to present detailed data at an upcoming medical congress and share results with regulatory authorities.AI summary

Johnson & Johnson announced that 24 abstracts across its neuropsychiatry portfolio will be presented at the 2026 Psych Congress Annual Meeting. The presentations include new pivotal Phase 3 efficacy and safety data for Caplyta in bipolar mania, along with additional data for Caplyta, Spravato, and seltorexant across mood disorders. The company continues to advance clinical and real-world evidence across its neuropsychiatry pipeline.AI summary

Vor Bio has completed enrollment in its global Phase 3 UPSTREAM MG trial evaluating telitacicept for generalized myasthenia gravis. Topline results for the 24-week primary endpoint are expected in the first half of 2027. The company also plans to initiate the global Phase 3 UPSTREAM oMG trial in ocular myasthenia gravis, expecting first patient dosing in the first half of 2027.AI summary

AstraZeneca presented full Phase III results from the OBERON and TITANIA trials showing tozorakimab significantly reduced moderate and severe COPD exacerbations by up to 34% in former smokers and 30% overall. The FDA accepted a Biologics License Application for tozorakimab under Priority Review. A PDUFA target decision date is expected during the first quarter of 2027.AI summary

Novartis announced that its global Phase III HARBOR trial of delpacibart etedesiran (del-desiran) in myotonic dystrophy type 1 did not meet its primary endpoint of video hand opening time versus placebo. The study did show evidence of clinical activity across secondary endpoints and exploratory analyses, with safety consistent with past data. Novartis will evaluate the complete dataset and consult health authorities to decide on future development.AI summary

Ascendis Pharma announced it will present new data from its rare disease portfolio at ESPE 2026. Highlights include first sentinel cohort data from the pivotal reACHin Trial of TransCon CNP in infants with achondroplasia aged 0 to <2 years. The company will also present posters on hypoparathyroidism and the Phase 3 trial design for lonapegsomatropin.AI summary

Legend Biotech announced it will present updated data for CARVYKTI at the 23rd International Myeloma Society Annual Meeting. The presentations will feature updated long-term (five-year or more) follow-up data from Cohort A of the Phase 2 CARTITUDE-2 study. Additionally, real-world evidence and prospective post-authorization safety study findings in relapsed or refractory multiple myeloma will be shared.AI summary

Ascendis Pharma presented Week 52 data from the open-label sentinel cohort of the Phase 2 reACHin trial evaluating once-weekly TransCon CNP in infants with achondroplasia. Treatment provided stable or improved foramen magnum stenosis scores and increased linear growth, with an ACH-specific supine length Z-score improvement of +0.42. TransCon CNP was generally well tolerated, supporting the 100 μg/kg/week dose.AI summary

Structure Therapeutics reported positive clinical results for its oral small molecule candidates ACCG-2671 and aleniglipron. ACCG-2671 demonstrated a ~6-day half-life and up to 3.3% weight loss in a Phase 1/2a SAD trial, with MAD dosing now initiated. In the Phase 2b ACCESS extension, aleniglipron achieved up to 16.2% body weight loss at 72 weeks.AI summary

Spyre Therapeutics reported positive 12-week induction data from Part A of the Phase 2 SKYLINE trial evaluating SPY003 for moderately-to-severely active ulcerative colitis. SPY003 met its primary endpoint with a statistically significant 10.0-point reduction in Robart's Histopathology Index score and was well tolerated. Topline induction data from Part B, evaluating monotherapies and pairwise combinations, are expected in 2027.AI summary

Inhibrx Biosciences announced primary endpoint results from the randomized Phase 2 HexAgon study evaluating INBRX-106 in combination with pembrolizumab for first-line HNSCC. The combination achieved a confirmed objective response rate of 48.3% and an interim median progression-free survival of 9.6 months compared to 26.5% and 4.9 months for pembrolizumab alone. Inhibrx plans to expand the study by approximately 50 HPV+ patients to support a potential accelerated approval pathway.AI summary

Roivant announced positive top-line results from the Phase 2 PHocus trial evaluating mosliciguat in patients with pulmonary hypertension associated with interstitial lung disease. The study met its primary endpoint, showing a statistically significant -56.3% placebo-adjusted reduction in pulmonary vascular resistance at Week 16. Following these results, Roivant has initiated the global Phase 3 PHrontier study.AI summary

AirNexis Therapeutics announced Phase 2b study results where its dual PDE3/4 inhibitor AN01 met its primary endpoint in COPD patients. Both tested doses delivered statistically significant improvements in lung function versus placebo at Week 4, including in patients on dual bronchodilator therapy. AirNexis is preparing to initiate its Phase 2 program in 2027.AI summary

Lyell Immunopharma completed the technology transfer for LYL273 to its LyFE Manufacturing Center following FDA review. The Phase 1/2 clinical trial in relapsed/refractory metastatic colorectal cancer continues to enroll patients to determine the recommended Phase 2 dose. Additional clinical data and an End-of-Phase 1 meeting with the FDA are now expected in 2027.AI summary

Opus Genetics announced positive preliminary results from Cohort 1 of its Phase 1/2 trial evaluating OPGx-BEST1 for BEST1-related retinal diseases. All five participants demonstrated clinically meaningful improvements in visual function with no serious adverse events. Following a successful FDA meeting, the company expects Cohort 2 dosing to complete in Q4 2026 and plans Phase 3 trial dosing in 2027.AI summary

Dyne Therapeutics announced it will present additional one-year clinical data from the Phase 1/2 ACHIEVE trial of z-basivarsen in myotonic dystrophy type 1 at upcoming medical meetings. The presentations will compare data from a pooled dose group against a matched natural history cohort. Additionally, Dyne will share baseline vHOT values for the registrational expansion cohort, with topline data expected in Q1 2027.AI summary

Evotec SE's subsidiary, Just – Evotec Biologics, has initiated a Phase 1 clinical trial evaluating JST-018. The investigational monoclonal antibody cocktail targets orthopoxviruses under the U.S. Department of War's Accelerated Antibodies Program. JST-018 is being developed as a prophylactic therapy to protect warfighters from biological threats.AI summary

Johnson & Johnson will present new data for RYBREVANT and RYBREVANT FASPRO in EGFR-mutated non-small cell lung cancer at WCLC 2026. Presentations include final overall survival results from the Phase 3 PAPILLON study evaluating first-line RYBREVANT plus chemotherapy. Additionally, updated findings and longer follow-up data from Phase 2 studies of RYBREVANT FASPRO plus LAZCLUZE will be presented.AI summary

Johnson & Johnson announced it will present over 35 abstracts detailing data from its multiple myeloma portfolio at the 2026 International Myeloma Society Annual Meeting. The presentations cover approved and pipeline therapies across various disease stages, including long-term follow-up and Phase 3 analyses. Featured drugs include CARVYKTI, TECVAYLI, TALVEY, DARZALEX FASPRO, and the investigational trispecific antibody ramantamig.AI summary

United Therapeutics Corporation announced it will enter into an Accelerated Share Repurchase (ASR) agreement with Citibank, N.A. to buy back approximately $477.6 million of its common stock. This utilizes the remainder of its previously announced $2.0 billion repurchase authorization. The final settlement of the ASR agreement is expected to be completed in the fourth quarter of 2026.AI summary

Korsana Biosciences has completed its merger with Cyclerion Therapeutics alongside a private placement of $380 million. The combined company holds approximately $475 million in cash, expected to fund operations into 2029. Korsana plans to advance lead program KRSA-028 toward the clinic, with Phase 1 healthy volunteer data expected in mid-2027.AI summary

Axogen announced the pricing of an underwritten public offering of 4,910,000 shares of common stock at $42.50 per share. The gross proceeds are expected to be approximately $208.7 million, with underwriters granted a 30-day option to buy up to 736,500 additional shares. Axogen plans to use net proceeds to fund its acquisition of BioCircuit Technologies and for general corporate purposes.AI summary

Axogen has entered into a definitive agreement to acquire BioCircuit Technologies, the developer of NerveTape, for $200 million in cash. The deal adds the first FDA-approved device for sutureless peripheral nerve repair to Axogen's portfolio. The transaction is expected to close in the fourth quarter of 2026, subject to customary closing conditions.AI summary

Biotechnology company. Total offering $500M; first sale 2026-08-26. Form D is filed up to 15 days after the first sale.

Biotechnology company. Total offering $67M; first sale 2026-08-14. Form D is filed up to 15 days after the first sale.

Pharmaceuticals company. Total offering $50M; first sale 2026-08-31. Form D is filed up to 15 days after the first sale.

Kura Oncology announced the launch of Caspian Therapeutics to advance small-molecule menin inhibitors for diabetes and cardiometabolic diseases. Caspian completed a $50 million financing to fund IND-enabling development and initial clinical proof-of-concept for lead compound KO-7246. Kura retains approximately half ownership of Caspian while remaining focused on its oncology pipeline.AI summary

Biotechnology company. Total offering $51M; first sale 2026-08-10. Form D is filed up to 15 days after the first sale.

Biotechnology company. Total offering $30M; first sale 2026-08-24. Form D is filed up to 15 days after the first sale.

Biotechnology company. Total offering $15M; first sale 2026-08-27. Form D is filed up to 15 days after the first sale.

MannKind and Rose Pharma have entered into a worldwide licensing and collaboration agreement to develop ROSE-010 Technosphere for weight management. Under the deal, Rose Pharma receives an exclusive global license to MannKind's Technosphere technology, while MannKind receives an equity interest in Rose Pharma, a board seat, and potential royalties. MannKind will provide pharmaceutical development and manufacturing support through the completion of a Phase 1b clinical study.AI summary

Google DeepMind introduced AlphaGenome Atlas, an accessible platform containing predictions for the molecular impact of 9 billion single-nucleotide variants across the human genome. The resource incorporates the AlphaGenome Variant Impact score to help researchers score, rank, and interpret genetic variants for rare disease research and target identification. The dataset is currently available for academic use via a web portal, API, and Google Antigravity.AI summary