Pipeline
BioMarin no longer expects to hit its $4 billion revenue target by 2027, citing various market factors such as impending competition for achondroplasia therapy Voxzogo and the divestment of the gene therapy Roctavian.
Intellia earlier this year reported a similar grade 4 liver enzyme elevation associated with the gene therapy nexiguran ziclumeran, though analysts at BMO Capital Markets at the time brushed it off as a “non-concern.”
The discontinued CAR T therapy bbT369 came to Regeneron when the pharma bought all of 2seventy bio’s pipeline assets for $5 million upfront in January 2024.
Two patients experienced grade 3 liver enzyme elevations that were deemed related to Terns’ investigational obesity pill TERN-601.
Despite announcing a broad pivot to siRNA earlier this year, Sarepta is following through with an investigational gene therapy: its limb-girdle muscular dystrophy candidate. But the treatment’s path forward, analysts say, is highly uncertain.
The decision to stop the Phase IIb study was driven by “strategic business reasons,” according to a federal clinical trials database.
Moving forward, Innate will focus on the clinical development of its antibody-drug conjugate IPH4502, the lymphoma candidate lacutamab and the AstraZeneca-partnered monalizumab.
Analysts at BMO Capital Markets expect the lack of other exon-44-skiping therapies to facilitate a “smooth” approval process for Avidity’s del-zota.
Truist Securities called pumitamig’s data on Monday “very reassuring,” given the consistency between its performance in Chinese and global patient populations.
With two late-stage programs set to read out in the next 48 months, Biogen is translating its wealth of experience in multiple sclerosis to lupus—developing a pipeline BMO Capital Markets analysts called “thoughtful.”
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