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What’s in Today’s Brief? (May 13th Preview)
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Big pharma and China collaboration deal
Bristol Myers Squibb and Hengrui Pharma have agreed to a broad global drug-discovery pact spanning 13 programs across oncology, hematology and immunology, with BMS paying $600 million upfront. The partners framed the arrangement as an effort to accelerate discovery through Hengrui’s R&D capabilities while using BMS for select assets and joint development. The deal structure also reflects the growing role of China-based R&D in global pipeline buildouts: Hengrui will hold exclusive rights in mainland China, Hong Kong and Macau, while BMS will retain rights for the rest of the world. Multiple assets are slated for advancement toward clinical entry, with the overall value potentially topping $15 billion depending on progress and commercial outcomes. For biotech companies watching deal dynamics, the transaction signals that mega-partnerships with sizable upfront consideration remain active even as companies prioritize reinvestment into earlier-stage programs. It also underscores Big Pharma’s continued focus on adding geographically complementary discovery capacity rather than relying only on internal R&D throughput. Separately, the same transaction has been reported as including a mix of Hengrui-sourced assets, BMS-sourced assets and jointly discovered candidates, positioning the agreement as both a portfolio expansion and a discovery platform collaboration.
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FDA commissioner exit amid regulatory uncertainty
FDA Commissioner Marty Makary has resigned, with Kyle Diamantas, the agency’s top food official, stepping in as acting commissioner. The change ends Makary’s tenure that began in late 2024 and became marked by turnover in senior roles, shifting guidance expectations and debate over the agency’s approach to speeding reviews. The reporting around the departure emphasized uncertainty inside the regulator: analysts cited concerns about predictability for drugmakers, alongside delayed reviews and abrupt guidance reversals. Under Makary, the FDA also backed initiatives tied to AI use in reviews and efforts intended to compress timelines for certain development programs. For biopharma stakeholders, the immediate operational question is how quickly leadership transitions translate into regulatory continuity on ongoing decisions and trial-review expectations. Because FDA policy shifts can reverberate across clinical timelines, the leadership handoff adds another moving piece to an already active regulatory environment. The leadership change also signals continued internal pressure around organizational stability at the FDA, even as the agency maintains ongoing workstreams in drug and biologics review, safety oversight and trial authorization pathways.
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FDA gene therapy review path for autoimmune CAR-T
Kyverna Therapeutics has begun a rolling submission to the FDA for miv-cel, after the agency requested additional natural history analysis. The company said the added work is intended to support review of what it characterizes as the first autoimmune CAR-T program to reach the market. The update places emphasis on the FDA’s focus on evidence quality beyond standard trial datasets, particularly for autoimmune indications where historical clinical context may affect benefit-risk framing. Rolling submissions allow the agency and applicant to review modules as they are prepared rather than waiting for a complete package. For developers of cell therapies, the move highlights how “data readiness” requirements can extend natural-history and retrospective evidence collection even after key clinical milestones. It also illustrates the continuing importance of regulatory engagement during late-stage development for cell and gene therapy candidates. Investors and competitors will watch whether the expanded natural-history work delays timelines or instead improves alignment on endpoints and comparators.
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FDA approval: first therapy for ultra-rare NRG1 fusion cholangiocarcinoma
Partner Therapeutics’ Bizengri has received FDA approval for advanced cholangiocarcinoma driven by NRG1 gene fusions, marking the first approved therapy for this ultra-rare genetic driver. Partner said it acquired U.S. rights from Merus in 2024. The approval expands options for patients with a molecularly defined subset where treatment has historically been limited. For companies active in precision oncology, the decision reinforces demand for therapies targeting validated oncogenic fusions and other rare molecular events. The FDA approval also adds to the pipeline momentum around bispecific antibodies and other targeted biologics for genetically stratified populations. Follow-on commercialization strategy and further label expansions will be the next key datapoints for investors. Clinicians and payers will likely focus on testing workflows and the accuracy of companion diagnostics or FDA-authorized assays needed to identify eligible patients for NRG1 fusion status.
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Clinical signal: CRISPR-modified CD33-deleted stem cell transplant in AML/MDS
A Phase I/II multicenter trial led by Washington University School of Medicine reported that a CRISPR-Cas9 CD33-deleted allogeneic hematopoietic cell transplant can reduce recurrence risk in high-relapse acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). The study, published in Nature Medicine, used gemtuzumab ozogamicin as maintenance after transplant. The trial enrolled 30 adult patients at high risk of relapse across 15 sites in the U.S. and Canada. Investigators reported that all patients achieved engraftment by day 28 and said outcomes were similar to standard stem cell transplantation, supporting the CRISPR-edited approach as a feasible strategy in myeloid disease where CAR-T is limited by on-target toxicity. John DiPersio, MD, PhD, the study’s corresponding author, said the results support future combination efforts with CD33-targeted immunotherapies, including CAR-T, to expand treatment options for aggressive myeloid cancers. For hematology programs, the work adds another example of gene editing being used to modify donor cell properties for immune safety while keeping transplant efficacy. Subsequent follow-up on relapse, durability and toxicity will determine whether the strategy becomes a platform for next-generation allogeneic gene-edited transplants.
...and 5 more selected Biotech stories in today’s full edition — or archive.
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