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:
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.
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.