M&A, Deals, Partnerships:
BioMarin (SF) Buys Alesta for $275M to Enter Rare Bone Disease
Acquiring Alesta Therapeutics for $275M upfront plus up to $215M in milestones to gain ALE1, an oral small molecule for hypophosphatasia (a rare genetic bone disease), setting up a challenge to AstraZeneca’s enzyme therapy. ALE1 is in a Phase 1/2a trial; Alesta will spin out its non-ALE1 assets before the deal closes, expected in Q3 2026.
Amgen (LA) Terminates $500M+ TScan Crohn’s Collaboration
Amgen exercised its right to end the 2023 Crohn’s disease target-discovery pact with TScan following a review of its portfolio and research priorities, wiping out more than $500M in potential milestones and royalties for the partner. The termination takes effect November 10, 2026, and comes without penalty to Amgen.
Ambros (SD) Goes Public via Werewolf Reverse Merger with $150M Raise
The Vivek Ramaswamy-cofounded chronic pain biotech is going public through a reverse merger with cash-strapped Werewolf Therapeutics, with Ambros shareholders taking 71.7% of the combined company. An oversubscribed, concurrent $150M private placement will fund late-stage development of neridronate for complex regional pain syndrome type 1, a rare condition with no approved treatments.
Chai Discovery (SF) Signs Bristol Myers Squibb for AI Antibody Discovery
Bristol Myers Squibb will tap Chai’s AI molecular folding and design models to discover therapeutic antibody candidates across its portfolio, adding to Chai’s roster of pharma partners that already includes Lilly, Pfizer, and Novartis. Financial terms were not disclosed; the deal deepens Chai’s push to build an AI-powered, continuously learning drug-discovery engine.
FDA Approvals:
Ultragenyx (SF) Wins First-Ever Gene Therapy Approval for Genglycos
The FDA granted accelerated approval to Genglycos (DTX401), a one-time AAV8 gene therapy and the first-ever treatment designed to target the underlying cause of glycogen storage disease type Ia, in patients eight and older. It is the company’s first gene therapy approval, arriving four days ahead of the August 23 decision date; continued approval is contingent on confirmatory data.
Other Interesting News:
Capricor’s (SD) Duchenne Cell Therapy Gets PDUFA Date Pushed to November
The FDA extended the target action date for Deramiocel, its cell therapy for Duchenne muscular dystrophy cardiomyopathy, by three months to November 22, 2026, after classifying a new data amendment as a major amendment. The amendment adds 24-month HOPE-3 open-label extension data supporting a refined indication focused on upper limb function.
Spruce (SF) Clears FDA Talks, Keeps Q4 BLA Filing on Track
Positive pre-BLA meetings keep the company on track to file a BLA in Q4 2026 for TA-ERT, an enzyme replacement therapy for the ultra-rare Sanfilippo syndrome type B (MPS IIIB). The FDA aligned on manufacturing comparability strategy and BLA content, clearing a hurdle that had previously pushed the filing from Q1 to Q4.
Genentech (SF) to Invest $750M in New Fill-Finish Manufacturing Capacity
The Roche subsidiary will invest about $750M in new device fill-finish capabilities at its Hillsboro, Oregon campus, expanding capacity for pre-filled syringes and autoinjectors to support its future pipeline. The project is expected to create roughly 250 manufacturing jobs, with commercial operations slated to begin in 2031.
Erasca (SD) Lands FDA Fast Track for Pan-RAS Molecular Glue in Pancreatic Cancer
The FDA granted Fast Track designation to ERAS-0015, an oral pan-RAS molecular glue, for metastatic pancreatic adenocarcinoma, following encouraging Phase 1 activity that included 2L+ KRAS G12X pancreatic cancer. The designation can enable more frequent FDA interactions and eligibility for accelerated/priority review; three potentially registration-enabling trials are planned across pancreatic and lung cancers, with more data due in H1 2027.
Pheast’s (SF) CEO Defends Radical R&D Transparency as Rivals Circle
CEO Paris Panayiotopoulos says he has no regrets about the cancer-immunotherapy biotech’s unusually open approach to sharing its science, arguing the benefits outweigh the risk of tipping off competitors. The stance is notable as the macrophage-checkpoint field heats up and rivals monitor Pheast’s progress — a bet that openness builds credibility and talent faster than secrecy.
