Billion-Dollar Molecules: M4 Modulators, GLP-1 Biased Agonists, and an SSTR2 Radiotherapy

Today, ozanimod is a likely megablockbuster drug approved for ulcerative colitis and multiple sclerosis. But before it became the first S1P modulator approved in ulcerative colitis, it received a scary, headline-making “ Refuse to File ” letter from the FDA due to insufficient preclinical characterization of an active metabolite RP112273 [...]

In June, we saw several FDA approvals for treatments targeting primary biliary cholangitis and COPD. The industry witnessed multibillion- and multimillion-dollar deals across various therapeutic areas. Advances were made in obesity therapeutics, with novel targets and clinical readouts for GLP-1 agonists and NLRP3 inhibitors. Alkermes and Takeda reported results for OX2R agonists in narcolepsy, while an antimalarial drug showed promise in PCOS. Despite these advancements, there were clinical trial failures for key molecules. Here's a recap of the most notable news highlights from June 2024!

Amgen has acquired ChemoCentryx in a deal that includes lead molecule avacopan (Tavneos), a first-in-class orally bioavailable small molecule complement 5a receptor (C5aR) antagonist approved as an adjunctive treatment to standard of care therapy for adults with severe active anti-neutrophil cytoplasmic autoantibody (ANCA)-associated [...]

Companies inked notable high-profile transactions in March 2024, including AbbVie's purchase of Landos Biopharma for $137.5M, AstraZeneca's $2.4B acquisition of Fusion Pharmaceuticals, and its subsequent deal to buy Amolyt for up to $1.05B. The FDA approved the first treatment for MASH. In parallel, expectations are high for the approval of Xcovery's ALK inhibitor, ensartinib. Praxis made headlines with PRAX-628, a next-generation oral NaV modulator, which showed anti-seizure efficacy in a Ph. IIa trial. Read below for more details on these and other drug discovery news stories from March 2024

In May 2024, several significant transactions worth millions and billions of dollars took place to advance next-generation neuroplastogens, KRAS G12C inhibitors, Pompe disease treatments, and molecular glues. We also highlight positive clinical readouts for hot targets such as tetrameric transthyretin (TTR) stabilizers, Hedgehog pathway inhibitors, plasma kallikrein inhibitors, dipeptidyl peptidase 1 (DPP-1) inhibitors, and PCSK9 inhibitors. Here's a recap of the most notable news highlights from May 2024!