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What’s in Today’s Brief? (December 8th Preview)
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Dyne Duchenne win — readies accelerated FDA bid
Dyne Therapeutics reported pivotal-study success for its exon‑51 skipping Duchenne muscular dystrophy candidate and signaled plans to seek accelerated FDA approval. In a registrational expansion cohort, treated patients reached adjusted dystrophin expression of 5.46% of normal after six months, substantially higher than historical comparators, and showed functional improvements across multiple endpoints. The company reported a favorable safety profile with mostly mild-to-moderate events and no related serious TEAEs in the registrational group. Dyne plans a rolling submission for accelerated approval in 2026 and is building commercial and CMC capacity ahead of a potential 2027 launch.
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Mirum shells out $620M: snaps up Bluejay's HDV antibody
Mirum Pharmaceuticals agreed to acquire Bluejay Therapeutics for $620 million in cash and stock to gain brelovitug, a monoclonal antibody in Phase 3 targeting hepatitis D virus (HDV). Bluejay’s Phase 2 readout showed complete HDV RNA responses and liver enzyme improvements; Mirum expects top-line Phase 3 data in the second half of next year and is targeting a regulatory filing and commercial launch in 2027. The deal includes up to $200 million in sales‑based milestones and a concurrent private placement to raise roughly $200 million to support development and commercialization.
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FDA heightens CAR‑T bar: new therapies must prove superiority
The FDA’s biologics chief signaled a policy shift requiring new CAR‑T cancer therapies to demonstrate superiority over existing treatments to secure approval. The position, articulated in a recent journal article, raises the evidentiary bar for developers pursuing CAR‑T approvals and could reshape trial design expectations, pushing sponsors toward randomized or active‑comparator studies. Companies and investors should anticipate longer, potentially more costly registrational programs as regulators push for comparative benefit rather than non‑inferiority or single‑arm efficacy readouts.
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BioNTech CTLA‑4 shows survival gain — tolerability concerns linger
BioNTech and partner OncoC4 reported Phase 3 survival data for an anti‑CTLA‑4 antibody in lung cancer that more than halved the risk of death, delivering a significant efficacy signal. Subsequent analyses and regulatory reviews, however, flagged a challenging tolerability profile with immune‑related adverse events that complicate the drug's clinical utility and commercial positioning. Analysts noted the efficacy is compelling but cautioned that toxicity management will be central to labeling, uptake and potential combination strategies.
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Structure posts competitive weight loss — nausea rates raise flags
Structure Therapeutics disclosed mid‑stage results for its oral GLP‑1 candidate showing substantial placebo‑adjusted weight loss that rivals injectable competitors. In one trial, a 120‑mg dose produced ~12.1% weight loss at 36 weeks; higher titration to 240 mg yielded ~14.2% in the ongoing study. The data were tempered by high rates of gastrointestinal adverse events—nausea in about 65% and vomiting in 32% at some doses—prompting questions about tolerability and commercial differentiation versus peptide injectables. The market reacted briskly, with biotech equities linked to oral GLP‑1 programs moving sharply on the readouts.
...and 5 more selected Biotech stories in today’s full edition.
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