Mirum Pharmaceuticals, Inc.
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Latest From Mirum Pharmaceuticals, Inc.
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.
Ipsen's oral ileal bile acid transport inhibitor Bylvay is now approved for pruritus in patients with Alagille syndrome and progressive familial intrahepatic cholestaisis. However, the biggest opportunity is a third indication – biliary atresia – which could contribute about half of the €800m peak sales predicted by analysts at Jefferies.
AstraZeneca and CANbridge obtain new approvals for their rare disease drugs in China, where a new domestic mRNA vaccine is also being made available amid a new wave of COVID infections. Meanwhile, the CEOs of pharma and other companies pay visits, showing China remains high on their list of global priorities.
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.
- Specialty Pharmaceuticals
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