The BioPharm Brief: Setbacks, Submissions, and a $1.3B Deal

Welcome to The BioPharm Brief, your daily snapshot of developments shaping the biopharmaceutical industry. Here’s what’s making headlines today.

Ultragenyx is facing a major setback in Angelman syndrome. The company’s apazunersen failed to meet the primary endpoint in the Phase 3 Aspire study, which evaluated the antisense oligonucleotide in children and adolescents with the rare neurodevelopmental disorder. The trial also missed a key secondary endpoint. The result is a significant blow to a program that had been an important part of Ultragenyx’s pipeline.

uniQure is moving AMT-130 into the regulatory review process for Huntington’s disease. The company submitted a Biologics License Application to the FDA seeking accelerated approval for ifezuntirgene inilparvovec, formerly known as AMT-130. uniQure also submitted a Marketing Authorisation Application to the UK’s Medicines and Healthcare products Regulatory Agency. The filings mark a major step for the investigational gene therapy, which is designed to address the underlying disease process in Huntington’s disease.

And HUTCHMED has struck a deal with GSK for its KRAS-EGFR therapy HMPL-A830. The agreement gives GSK rights outside Greater China and includes a $110 million upfront payment and up to $1.185 billion in additional development, regulatory, and commercial milestones. HUTCHMED will lead the global Phase 1 program, while GSK will take over subsequent development and commercialization outside the retained territories.

That’s The BioPharm Brief for today. For analysis and expert insights, please visit BioPharmInternational.com.

Key takeaways:

• Ultragenyx’s apazunersen failed to meet the primary and key secondary endpoints in phase 3 testing for Angelman syndrome.
• uniQure has submitted US and UK regulatory applications for AMT-130, its investigational gene therapy for Huntington’s disease.
• HUTCHMED licensed its KRAS-EGFR antibody-drug conjugate HMPL-A830 to GSK in a deal worth up to $1.295 billion.