A Deuterated Ruxolitinib Derivative Among 14 Other Recent Notable Molecules
Here’s a preview of two molecules acquired in January 2023 among 14 mentioned in this article:
deuruxolitinib (CTP-543)
This molecule, a deuterated version of JAK1/2 inhibitor ruxolitinib (Opzelura, Incyte), has received Breakthrough Therapy designation from the FDA and is being acquired by Sun Pharma via Concert in a $0.576B transaction. The oral molecule is in Ph. III for alopecia areata, or autoimmune hair loss, and demonstrated remarkable efficacy over 24 weeks at 8-12 mg BID, with a comparable safety profile and dose to ruxolotinib (15-20 mg BID). While deuterium incorporation can modulate drug metabolism, it is not clear how it scientifically differentiates from ruxolitinib or other JAK inhibitors beyond a longer patent life enabling development for this new indication.
saridegib (IPI-926)
This SMO inhibitor derived from the natural product cyclopamine, one of the first known SMO inhibitors, was passed from BridgeBio company PellePharm to Sol-Gel Technologies for ~$75M. The hedgehog signaling pathway inhibitor is being studied in Gorlin syndrome and basal cell carcinomas as a topical agent to mitigate systemic toxicities.
In our full update of small molecules in the news we also cover recent:
(B/M)illion-Dollar Molecules: recent M&A’s, IPOs, and other fundraising
Molecules in Motion: clinical trial updates and FDA filings
Where Are They Now? updates on past Drug Hunter Molecules of the Month
Potential Upcoming Approvals: recent NDA filings and upcoming PDUFA dates
Read about all 14 molecules in the full Premium article here.
