Pharma and biotech news, week of Aug 17 – Aug 23, 2026

The FDA has approved Regeneron's Pasatru (garetosmab-grts) to reduce new heterotopic ossification lesions and flare-ups in adults with fibrodysplasia ossificans progressiva. Approval was based on Phase 3 OPTIMA trial data showing a 90% or greater reduction in new lesions at 56 weeks compared to placebo. A European regulatory submission is currently under review, and a pediatric Phase 3 trial is planned for later this year.AI summary

Ultragenyx received FDA approval for Genglycos, a biologic (CBER). Application BLA125858. PDUFA goal date was 2026-08-23.

Milestone Pharmaceuticals has enrolled the first patient in the pivotal Phase 3 ReVeRA-301 trial evaluating etripamil nasal spray for atrial fibrillation with rapid ventricular rate (AFib-RVR). The study will assess a 70 mg repeat-dose regimen in approximately 150 patients, with a primary endpoint of ventricular rate reduction within 30 minutes. Milestone plans to use the data to support a single-study supplemental New Drug Application registration pathway.AI summary

Taiho Oncology and Cullinan Therapeutics announced that Phase 3 REZILIENT3 trial results will be presented at the IASLC 2026 World Conference on Lung Cancer. The study met its primary endpoint of progression-free survival for zipalertinib plus chemotherapy in first-line EGFR exon 20 insertion mutation NSCLC. Full abstract details will be available on the conference website under embargo policies.AI summary

Gossamer Bio has entered into a private placement agreement to raise up to approximately $250 million. The financing includes $150 million of committed capital, with $25 million funded upfront and $125 million contingent on FDA acceptance of the seralutinib NDA. Net proceeds will fund operations into 2028 and support advancing seralutinib through potential FDA approval in PAH.AI summary

Scholar Rock updated regulatory progress for apitegromab in spinal muscular atrophy across three regions. Under FDA guidance, the company removed Catalent Indiana LLC as a commercial fill-finish facility from its U.S. BLA and transitioned to an alternate facility, keeping review on track for a September 30, 2026 PDUFA date. Scholar Rock also withdrew its European MAA to resubmit with the alternate facility, and aligned with Japan's PMDA to submit a JNDA by year-end 2026 without domestic clinical data.AI summary

BioNTech will present new lung cancer clinical data at the IASLC 2026 World Conference on Lung Cancer in Seoul, September 12-15, 2026. A late breaking oral presentation will detail first global data from the Phase 1/2 trial (NCT06892548) of pumitamig combined with the B7H3-targeting ADC elfetabart drozuntecan in advanced or metastatic SCLC and NSCLC. Updated overall survival data from stage 1 of the Phase 3 PRESERVE-003 trial of gotistobart will also be presented, and its pivotal stage 2 is ongoing.AI summary

Ionis Pharmaceuticals announced it will present new clinical data at the European Society of Cardiology Congress 2026. Presentations include one-year long-term extension data for TRYNGOLZA in severe hypertriglyceridemia, Phase 1 single-dose 12-month data for ION775, and Phase 3 CARDIO-TTRansform trial data for eplontersen in ATTR-CM.AI summary

Biotechnology company. Total offering $70M; first sale 2026-05-26. Form D is filed up to 15 days after the first sale.

Biotechnology company. Total offering $30M; first sale 2026-08-10. Form D is filed up to 15 days after the first sale.

Biotechnology company. Total offering $25M; first sale 2026-04-30. Form D is filed up to 15 days after the first sale.

Pharmaceuticals company. Total offering $21M; first sale 2026-08-13. Form D is filed up to 15 days after the first sale.

Biotechnology company. Total offering $12M; first sale 2026-08-17. Form D is filed up to 15 days after the first sale.

Biotechnology company. Total offering $10M; first sale 2026-08-14. Form D is filed up to 15 days after the first sale.