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What’s in Today’s Brief? (May 15th Preview)
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Regulatory actions for Prader-Willi rare disease drug candidates
The FDA has placed a full clinical hold on Aardvark Therapeutics’ ARD-101 Prader-Willi syndrome program, pausing both the ongoing Phase 3 study and the associated open-label extension. The hold follows a cardiac safety signal observed in healthy volunteers receiving higher doses than those used in other studies. Aardvark said it will unblind trial data to support an FDA review of the combined efficacy and safety dataset and to determine next steps for ARD-101. The company had dosed 68 people in the randomized Phase 3 trial and 19 in an extension before the pause. Separately, the FDA’s hold affects all ARD-101 studies, and earlier actions by Aardvark had already paused related work after the initial voluntary safety pause in March. The timing is critical for funding planning as the company’s cash runway is now extended to mid-2027. Investors reacted sharply to the hold, with Aardvark’s shares falling materially after the FDA action—underscoring the centrality of the company’s most advanced asset to both clinical and financing strategy.
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FDA approval for oncology in mantle cell lymphoma
BeOne Medicines said the FDA granted accelerated approval for its drug Beqalzi (sonrotoclax) in relapsed or refractory mantle cell lymphoma, positioning the company to challenge incumbents including AbbVie and Roche’s Venclexta. The regulatory action adds a new late-line option in a space where timing and mechanism differences can influence treatment sequencing. The approval comes as BeOne seeks to establish momentum for additional clinical programs built around its BCL2-targeting approach. For investors and clinicians, the key near-term issue is how BeOne’s label translates to real-world adoption versus existing regimens and whether confirmatory study plans align with FDA expectations. The accelerated path also highlights how the FDA continues to use interim efficacy signals in aggressive hematologic malignancies when the unmet need is high and trials can move quickly.
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Duchenne gene therapy pathway toward FDA submission
Regenxbio said its Duchenne muscular dystrophy gene therapy RGX-202 met the bar in a pivotal trial, producing sufficiently high levels of a miniaturized muscle protein and paving the way for an FDA submission. The company framed the result as meeting criteria consistent with an accelerated approval pathway. The update arrives after a period of market pressure tied to earlier clinical uncertainties and setbacks for the platform’s FDA prospects. Regenxbio’s next steps now focus on submission timing and addressing safety and confirmatory requirements expected for gene therapies in DMD. For the field, the readout reinforces that durable microdystrophin expression remains the key translational marker under discussion across competing DMD gene therapy programs.
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In vivo CAR-T platform financing expansion
CREATE Medicines raised a $122 million Series B to advance its in vivo CAR-T platform across autoimmune disease and oncology, pushing programs including CRT-402 (CD19 CAR T for autoimmune disease) and an oncology-directed asset targeting CD19 and BCMA combinations. The company said it has already dosed more than 50 patients across in vivo CAR clinical programs. The financing is co-led by existing investors and includes participation from Alexandria Venture Investments and others, with stated proceeds aimed at extending runway for clinical execution and internal manufacturing investment. CREATE emphasized repeat dosing potential and the ability to run “one-day manufacturing” via its mRNA-LNP approach delivering CAR instructions in the body. For the competitive landscape, the round underscores growing capital support for next-generation cellular therapies that seek to reduce logistical bottlenecks seen in conventional ex vivo CAR-T.
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Big Pharma partnership with Hengrui covering 13 programs
Bristol Myers Squibb and Hengrui Pharma signed a collaboration and license agreement covering 13 early-stage programs spanning oncology/hematology and immunology, valued at up to $15.2 billion. The partnership structure gives BMS exclusive rights to Hengrui-origin candidates outside Hengrui’s China/Hong Kong/Macau territory, while Hengrui receives rights to BMS-origin assets in those areas. BMS will pay up to $950 million over two years, including a $600 million upfront payment plus anniversary milestones. Hengrui is set to oversee early clinical development to accelerate proof-of-concept, with BMS retaining options to co-develop and commercialize select assets depending on milestones. The deal is notable for combining BMS’s global development and regulatory capabilities with Hengrui’s early development engine, reinforcing how large biopharma is continuing to expand discovery and pipeline coverage through multi-asset platform partnerships.
...and 5 more selected Biotech stories in today’s full edition — or archive.
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