Sept. 29 Quick Takes: Mirum wins first approval for rare liver disease

In receiving the first FDA approval for a therapy to treat cholestatic pruritus in Alagille syndrome patients, Mirum Pharmaceuticals Inc. (NASDAQ:MIRM) gains a rare pediatric disease priority review voucher. The newly approved therapy, Livmarli maralixibat, is a minimally absorbed ileal bile acid transporter (IBAT) inhibitor. According to the company, 2,000-2,500 children in the U.S. are affected by the rare genetic disease.

Sanofi (Euronext:SAN; NASDAQ:SNY) said the positive serology data from the Phase I/II study of its mRNA-based COVID-19 vaccine candidate co-developed with Translate Bio Inc. supports the pharma’s broader ambitions in the modality, but did not indicate a path forward for the program. Across all three doses tested, the unmodified COVID-19 mRNA vaccine induced neutralizing antibody responses in 91-100% of the study participants two weeks after a second injection. On the heels of it $3.2 billion takeout of Translate in August, Sanofi said it was transitioning the newly acquired platform to modified mRNA, and plans to bring its first modified mRNA candidate to the clinic in 2022 for influenza. The marketed COVID-19 mRNA vaccines from Moderna Inc. (NASDAQ:MRNA) and Pfizer Inc. (NYSE:PFE) and BioNTech SE (NASDAQ:BNTX) use modified nucleosides; in June, CureVac N.V. (NASDAQ:CVAC) reported disappointing results for its unmodified COVID-19 mRNA vaccine candidate. ...