Travere Looks Ahead To Phase III In Homocystinuria
Opportunity Bolstered By Competitor’s Recent HCU Failure
The latest data from a Phase I/II study shows that pegtibatinase can yield a rapid, sustained reduction in homocysteine, the protein behind the underlying cause of the rare disease.
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Deal Watch: Sangamo Inks Significant Option Deal With Prevail After Losing Tie-Ups With Novartis, Biogen
Plus deals involving Novartis/DTx, Neurogene/Neoleukin, Travere/Mirum, Sanofi/Scribe, Korro Bio/Frequency, MaxCyte/viTToria and more.
Travere’s drug, approved as Filspari for immunoglobulin A nephropathy, missed its eGFR primary endpoints in focal segmental glomerulosclerosis but showed meaningful improvement in proteinuria.
Public Company Edition: With the XBI up more than 9% in April, a few follow-on offerings –$23m to support Gamida Cell’s small product launch and $500m to fund Vaxcyte’s large clinical trial program – have made it to market. Also, China’s CytoMed launched the sixth biopharma IPO in the US this year.