Million-Dollar Molecules: A Dual TYK2/JAK1 Inhibitor Licensed to Biohaven, an Oral IRAK4 Degrader Exclusively Licensed by Gilead, a Ph. I AT2R Inhibitor Acquired by Eli Lilly, and more
This article highlights some recent million-dollar molecules, including: Read about more million-dollar molecules from recent M&A’s, IPOs, and other fundraising from March 2023 below. Where structures are not disclosed, we show some examples from potentially related patents to illustrate what the chemical matter might look like. Want updates like this straight to your inbox? Sign…
Negative Readouts: Updates on an NMDAR PAM, an MK2 inhibitor, and a Porcupine inhibitor
This article discusses scientifically interesting molecules that have unfortunately had negative recent readouts. Aptinyx’s therapeutic approach of targeting positive allosteric modulators (PAMs) of NMDA receptors has hit another setback in Ph. II for NYX-458. The oral, twice-daily MK2 inhibitor from Aclaris Therapeutics has results from its Ph. IIa for the treatment of moderate-to-severe hidradenitis suppurativa…
AACR Orlando 2023: New Drug Candidates
The AACR 2023 first disclosures presented in the Chemistry in Cancer Research’s “New Drugs on the Horizon” sessions are always fascinating. This article contains the structures of all of the novel small molecule first disclosures presented at AACR in the session, along with notes on their significance, and another molecule disclosed in the poster session.…
Merck’s Oral PCSK9 Macrocyclic Peptide Efficiacious in Ph. IIb, Roche’s RNA Splicing Modifier Continues to Work 4 Years into Ph. III for SMA, and 9 More Molecules in Motion
Merck’s oral PCSK9 inhibitor is a surprisingly bioavailable and orally effective macrocyclic peptide with picomolar activity. It recently demonstrated significant LDL cholesterol reduction in Ph. IIb, paving the way for it to enter Ph. III later this year. Roche’s oral, SMN2-directed RNA splicing modifier and first molecule of its kind to be FDA-approved in 2020…
Recent & Upcoming FDA Approvals: A Gamma Secretase Inhibitor Outside of Alzheimer’s, a PI3Ki Outside of Oncology, and More!
Pfizer spinout, SpringWorks Therapeutics, was granted priority review by the FDA for its oral gamma secretase inhibitor for the treatment of soft-tissue desmoid tumors, which has no FDA-approved therapies. Meanwhile, Pharming Groups’ oral PI3Kδ inhibitor (licensed from Novartis) received the first and only FDA-approval for the treatment of the rare, activated phosphoinositide 3-kinase delta syndrome,…
Merck’s Oral PCSK9 Inhibitor and Nurix’s BTK Degrader Among ACS First Disclosures
The First Disclosures section of the 2023 ACS Spring Meeting in Indianapolis, organized by GSK’s Nikki Goodwin, covers three novel small molecules and an ADC this year. The molecules include: More notes on each of the molecules and why they matter appear below. This page will continue to be updated after the conference. MK-0616, Merck’s…