Ultragenyx, Alumis look ahead after late-stage losses

Ultragenyx and Alumis each lost more than 40% on late-stage readouts for key critical pipeline programs this week with results that leave both biotechs assessing next steps for the assets. Among positive readouts, Moderna and Merck & Co. released landmark data that marked a win for neoantigen vaccines, mRNA, and melanoma patients.

Ultragenyx Pharmaceutical Inc. (NASDAQ:RARE) lost 45% of its value Thursday after apazunersen (GTX102) failed to meet the primary Bayley-4 cognitive raw score primary endpoint in Angelman syndrome. The antisense oligonucleotide, which is delivered via intrathecal administration, also missed the key secondary endpoint of net Multidomain Responder Index response in Angelman syndrome patients...