Makary Resigns, BMS Bets $15B on China R&D, and Regenxbio Wins Phase 3 in Duchenne – This Week in Biotech #100
The FDA loses its commissioner amid mounting instability, BMS makes the biggest pharma-China deal of 2026, and a clean gene therapy dataset gives Sarepta's Elevidys its first real competition.
Welcome back to This Week in Biotech by Biotech Blueprint, edition 100, covering biotech and biopharma news from May 8th to 14th, 2026.
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VIDEO SUMMARY
THIS WEEK’S KEY TAKEAWAYS 🔑
Three large stories defined the week. Bristol Myers Squibb committed up to $15.2B to license 13 early stage assets from China's Hengrui Pharma, betting that external discovery at Chinese R&D speed is the fastest path to pipeline. On the same day, FDA Commissioner Marty Makary resigned after weeks of pressure, adding a new layer of regulatory unpredictability to an already unstable agency. And on Thursday, Regenxbio posted a clean Phase 3 win in Duchenne muscular dystrophy, landing in the middle of Sarepta’s ongoing Elevidys safety crisis.
The BMS-Hengrui deal is really about the China R&D speed differential. BMS executives explicitly cited that clinical trial filing timelines in China run 50-70% faster than the rest of the world, and they structured the deal accordingly. $600M upfront, another $350M in fixed payments over 30 months, four Hengrui oncology and hematology assets ex-China, four BMS immunology assets going the other direction, and five jointly discovered programs. The specific molecular targets weren’t disclosed. Chinese discovery engines are now treated as outsourceable infrastructure by large pharma, not competitive risk. Pfizer’s Kailera deal, Merck’s Terns acquisition, and now this are all expressions of the same logic.
Makary’s resignation is the story with the most immediate practical consequences for drug developers. His tenure produced unpredictable rejections, including Replimune’s second FDA rejection in late April that cost the company 60% of its workforce, high profile leadership departures including CDER director George Tidmarsh and oncology chief Richard Pazdur, who joined and left within three weeks, and sustained public industry frustration. Acting commissioner Kyle Diamantas comes from the food side of the FDA with no drug review track record. For companies midway through regulatory review, the risk is silence because Type B meetings might stall through summer while a permanent successor is found.
Regenxbio’s RGX-202 result is the cleanest dataset of the week. In 30 boys with Duchenne muscular dystrophy, a single dose of the gene therapy drove mean microdystrophin expression of 71.1%, with 93% of patients crossing the 10% threshold that served as the primary endpoint. More importantly, the trial formally bridged biomarker to function: microdystrophin expression at week 12 was statistically significantly correlated with functional improvement at one year on a standardized motor assessment. That biomarker-to-function link is what prior Duchenne gene therapy submissions have been missing, and it's what gives this dataset a credible accelerated approval path rather than just scientific interest. Sarepta’s Elevidys currently carries a boxed warning for liver injury after three patient deaths and lost its platform technology designation. RGX-202 gives prescribers a real second option for the first time.
BIOTECH/PHARMA NEWS 🧬
🔹 Bristol Myers Squibb announced a global strategic collaboration with Hengrui Pharma on May 12 covering 13 early stage programs across oncology, hematology, and immunology. BMS pays $600M upfront, $175M on the first anniversary, and a contingent $175M in 2028, with total deal value reaching $15.2B if all milestones are hit. BMS gets exclusive ex-China rights to four Hengrui oncology and hematology assets. Hengrui gets four BMS immunology assets inside Greater China, and five programs will be jointly discovered with Hengrui leading early clinical work. Specific targets were not disclosed. The deal closes Q3 2026 pending antitrust clearance. This is the largest single pharma-China licensing deal of 2026 and the clearest signal yet that big pharma has stopped treating Chinese R&D as a competitive threat and started treating it as a supply chain.
🔹 FDA Commissioner Marty Makary resigned on May 12, with Deputy Commissioner for Food Kyle Diamantas named acting head. The immediate trigger was reportedly a dispute with the White House over fruit-flavored vape approvals, but the structural pressure had been building for months: CDER director George Tidmarsh departed, Richard Pazdur joined CDER and left within three weeks, and a string of high profile rejections including Replimune’s second complete response letter generated sustained industry pushback. The Biotechnology Innovation Organization issued a same day statement calling for organizational stability. Diamantas has no drug review background, and a permanent successor has not been named. For companies in active regulatory review, the near term risk is delays in Type B meeting feedback and PDUFA decisions while the commissioner's office transitions. Industry executives have reportedly floated Pazdur as a permanent replacement, though his recent departure complicates that path.
🔹 REGENXBIO reported positive pivotal Phase 3 data for RGX-202, an AAV8-delivered microdystrophin gene therapy for Duchenne muscular dystrophy, on May 14. In 30 ambulatory boys, 93% achieved microdystrophin expression above 10% at week 12, with mean expression of 71.1% across the full cohort. The headline number that matters most for the regulatory path: microdystrophin expression at week 12 was statistically significantly correlated with functional improvement on the North Star Ambulatory Assessment at one year, the first time a Duchenne gene therapy has formally bridged biomarker to function in a registration dataset. Two serious adverse events were reported, a case of subacute myocarditis that resolved and an asymptomatic liver enzyme elevation that normalized. REGENXBIO is pursuing accelerated approval with a 2027 commercial launch target. Sarepta’s Elevidys carries a boxed warning for serious liver injury after three patient deaths and is approved only for ambulatory patients aged 4 and older. RGX-202’s safety profile to date is the commercial differentiator.
🔹 Isomorphic Labs, the Alphabet-backed DeepMind spinout focused on AI drug design, closed a $2.1B Series B on May 12 led by Thrive Capital, with participation from Alphabet, GV, Temasek, and both the UK Sovereign AI Fund and Abu Dhabi’s MGX. This is the largest AI biotech private round on record. The capital funds continued development of the company’s AI drug design engine and its own therapeutic pipeline, with first-in-human trials targeted for late 2026. Isomorphic retains active partnerships with Novartis, Lilly, and J&J. This is no longer a company positioning itself as a service layer for pharma. It’s funded to be an integrated drug discoverer and developer. Whether AI-designed molecules clear Phase 1 this year is the credibility test for the entire category.
🔹 argenx received FDA approval on May 8 expanding Vyvgart and Vyvgart Hytrulo to all adult patients with generalized myasthenia gravis, including antibody negative patients who previously had no on label biologic options. The Phase 3 ADAPT SERON trial in 119 antibody-negative patients hit its primary endpoint with p=0.0068. argenx reported Q1 product net sales of $1.3B, up 63% year-over-year, its seventeenth consecutive quarter of growth. The label expansion removes the antibody status gating step that had been slowing prescribing decisions in community neurology. Vyvgart is now the only treatment approved for the full generalized myasthenia gravis patient population.
🔹 Italy’s Angelini Pharma agreed to acquire Catalyst Pharmaceuticals for $31.50 per share in cash, roughly $4.1B and a 28% premium, giving Angelini its first US foothold through Catalyst’s rare neurology portfolio: FIRDAPSE, the only FDA-approved treatment for Lambert-Eaton myasthenic syndrome, AGAMREE, an alternative steroid for Duchenne, and US rights to FYCOMPA for partial-onset seizures. This is the second European mid cap pharma company to buy a US-marketed rare neurology asset in as many weeks, after Chiesi’s KalVista deal, and the pattern likely reflects urgency to build US-marketed inventory ahead of Section 232 pharma tariffs taking effect July 31.
WATCHLIST 👀
ASCO 2026 Plenary, May 31: Daraxonrasib LBA5 from Revolution Medicines (Phase 3 RASolute 302, 2L metastatic PDAC, OS 13.2m vs 6.7m, HR 0.40). Final analysis and additional subgroup detail. Plenary slot is the highest-profile oncology platform of 2026.
FDA permanent commissioner nomination: Trump has named Diamantas only as acting. Pazdur was floated by industry but already departed. Watch for nomination signals from HHS and Senate HELP Committee
REGENXBIO BLA submission for RGX-202: Accelerated approval pathway flagged. FDA surrogate endpoint discussion is the gating step. Likely Q3 2026 filing.
EHA 2026 Congress, Stockholm, June 11-14: First clinical data from Ajax Therapeutics’s AJ1-11095 (Type II JAK2 inhibitor for myelofibrosis), the asset Lilly is acquiring for up to $2.3B (closing expected H2 2026)
Cytokinetics aficamten NDA filing timeline: Investor day or topline regulatory commentary at HFSA expected; ACACIA-HCM full data presentation at upcoming cardiology meeting.
BMS-Hengrui assets disclosed at closing: Specific molecular targets, modality breakdown, and any GLP-1 or ADC programs included in the 13 asset portfolio
Q2 2026 earnings season, late July-early August: First quarter under MFN drug pricing implementation; first commentary on Section 232 pharma tariff impact (effective July 31)
Have a great rest of your week and thanks for reading Biotech Blueprint!
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