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What’s in Today’s Brief? (November 3rd Preview)
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FDA approval expands PARP inhibitor use in HRR-mutant mHSPC
The FDA approved talazoparib (Talzenna) in combination with enzalutamide (Xtandi) for adults with metastatic hormone sensitive prostate cancer carrying homologous recombination repair (HRR) gene mutations. The approval covers patients with HRR mutations including ATM, ATR, BRCA1, BRCA2, PALB2 and others, with eligibility determined prospectively via tumor tissue or circulating tumor DNA-based next-generation sequencing. The decision was based on results from the randomized, double-blind, placebo-controlled TALAPRO-3 trial. In 599 patients, the primary endpoint—investigator-assessed radiographic progression-free survival—was not reached in the talazoparib-plus-enzalutamide arm versus 45.8 months in the placebo arm (hazard ratio 0.48; P<.0001), with overall survival data immature at the time of analysis. Regulators cited consistent benefit across BRCA and non-BRCA HRR mutations. The approval gives a new combination option for patients with newly diagnosed mHSPC with HRR gene alterations, a group facing aggressive disease and limited treatment choices.
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uniQure’s Huntington’s gene therapy enters formal FDA review for May 1
The FDA accepted uniQure’s Biologics License Application (BLA) for ifezuntirgene inilparvovec (AMT-130) under Priority Review, setting a May 1 action date. The company disclosed that the agency is “tentatively planning” a future advisory committee discussion, likely through the Cellular, Tissue, and Gene Therapies Advisory Committee, after reviewers took the application for formal review. uniQure previously faced regulatory resistance, and the current acceptance represents a reversal of earlier opposition that intensified through the current year. The gene therapy uses uniQure’s gene-silencing miQURE platform with a microRNA designed to silence the huntingtin gene and reduce the potentially toxic exon 1 protein fragment. Despite the regulatory step forward, shares fell after the announcement, reflecting investor scrutiny of late-stage durability and safety signals, including questions about how long-term data compare with earlier, shorter follow-up snapshots. uniQure will now work through the BLA review process toward a decision that could define a first disease-modifying option for Huntington’s disease.
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AbbVie and Brise lock in up to $757m for preclinical migraine programs
Brise Pharmaceuticals disclosed a licensing agreement with AbbVie covering two preclinical migraine programs, with potential payments of up to $757 million. The companies signed the deal in 2024 but have only announced it publicly this week. Under the terms, Brise is responsible for discovery while AbbVie leads global development and commercialization. Brise said it has already achieved several near-term milestones, and the agreement includes development, regulatory, commercial and sales milestones, with no upfront payment disclosed. Brise’s most advanced asset referenced in the disclosure is BR005, an oral small molecule in Phase II/III clinical trials for acute migraine. The partnership adds major Big Pharma validation to Brise’s migraine platform and reinforces AbbVie’s investment focus in neurology.
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Lonza expands U.S. CDMO footprint via Merck KGaA network sale
Lonza signed a definitive agreement to acquire five Merck KGaA CDMO manufacturing sites in the U.S., expanding Lonza’s footprint from four to nine sites and more than doubling its U.S. presence. The transaction also adds about 1,100 employees and is positioned to strengthen Lonza’s end-to-end offering, including clinical and commercial development and manufacturing capabilities. Lonza said the acquired network will support customers across areas including antibody-drug conjugates and other bioconjugates, viral vectors for cell and gene therapies, and advanced drug-delivery technologies. Lonza will acquire the assets and related businesses at a total enterprise valuation in the low- to mid-triple-digit millions of Swiss francs. The announcement complements Lonza’s ongoing investment in manufacturing readiness in Vacaville, California, and a recently announced commercial spray-drying facility in Bend, Oregon—signaling continued capacity buildout ahead of demand for complex modalities in the U.S. market.
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New Fabry enzyme replacement licensing deal for HM15421/GC1134A
BioMarin struck a deal to license HM15421/GC1134A (LA-GLA), a long-acting enzyme replacement therapy candidate designed for Fabry disease. The program combines Hanmi’s long-acting drug technology with GC Biopharma’s enzyme replacement expertise to extend residence time using a recombinant alpha-galactosidase A approach. BioMarin received worldwide rights, while GC and Hanmi retained commercialization rights in Korea and responsibility for regulatory approval efforts. The companies also remain eligible for development and commercial milestone payments and tiered royalties, with milestones projected to reach $405 million split between the South Korean partners. BioMarin said the candidate is in Phase I/II testing (NCT06858397) and is being developed as a subcutaneous injection. The agreement adds to BioMarin’s Fabry-focused portfolio following its acquisition of Amicus earlier this year.
...and 5 more selected Biotech stories in today’s full edition — or archive.
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