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Decision-gradeTopline na resulta

Beam Therapeutics: topline data ng BEAM-302 para sa AATD; pivotal cohort sa H2 2026

Na-update na Phase 1/2 topline data para sa BEAM-302 sa AATD ay nagpakita ng malakas na pagtaas ng AAT at isang mahusay na tinatanggap na profile, na humantong sa pagpili ng 60 mg bilang optimal biological dose. Ang feedback ng FDA ay sumusuporta sa accelerated approval pathway, na may pivotal cohort na ~50 pasyente na binalak na simulan sa ikalawang kalahati ng 2026 upang suportahan ang hinaharap na BLA.

Mahahalagang fact

Na-file
May 7, 2026, 11:06 AM
Kaganapan
Topline na resulta
Direksyon
Positibo
Kumpanya
BEAMBeam Therapeutics
Asset
BEAM-302
Pinagmulan
SEC 8-K
Kumpiyansa
May source

Sipi mula sa source

EX-99.1 2 beam-ex99_1.htm EX-99.1 EX-99.1 Exhibit 99.1 Beam Therapeutics Reports First Quarter 2026 Financial Results and Recent Business Updates Recent BEAM-302 Topline Data in Alpha-1 Antitrypsin Deficiency (AATD) Demonstrate Strong Single-dose Safety and Efficacy Profile, with 60 mg Selected as Optimal Biological Dose; Global Pivotal Cohort Expected to Initiate in Second Half of 2026 Data from Phase 1/2 BEACON Clinical Trial of Risto-cel in Sickle Cell Disease Published in April 1 Issue of the New England Journal of Medicine; U.S. Biologics License Application (BLA) Submission Expected as Early as Year-End 2026 Investigational New Drug (IND) Application for BEAM-304 in PKU and Data from BEAM-301 in GSDIa Anticipated in 2026 Ended First Quarter 2026 with $1.2 Billion in Cash, Cash

SEC 8-K

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