ROYALTY PHARMA

BRAIN Biotech AG forms growth segment BRAINBiocatalysts by combining BioProducts and BioScience Zwingenberg. Targets include EUR 100 million revenue, 15% adjusted EBITDA margin, and high R&D ratio within five years. The company aims to become a top 10 innovative enzyme player with profitable growth and positive cash flows.

BRAIN Biotech AG launches BRAINBiocatalysts to drive profitable growth, aiming for EUR 100 million revenue and 15% adjusted EBITDA margin in five years. Strategic collaborations enhance liquidity and earnings, with CEO Adriaan Moelker highlighting production and research bundling opportunities. CFO Michael Schneiders emphasizes strengthened valuation through sustainable growth initiatives.

Novocure's electrical field-emitting device in Phase 3 trial showed extended survival in pancreatic cancer patients when used with chemo; Merus licensed U.S. rights of its cancer drug zenocutuzumab to Partner Therapeutics; FDA to decide on Cytokinetics' heart drug aficamten approval by Sept. 26, 2025; FDA investigates blood cancer cases post-Bluebird bio's Skysona treatment; Fate Therapeutics' CEO Scott Wolchko to retire, with Bob Valamehr taking over in 2025.

Abingworth seeks to raise up to $1.5 billion for biopharma clinical development in return for royalties, aiming to support up to eight late-stage trials. This follows earlier deals with Gilead and Teva, where Abingworth funded clinical studies in exchange for fixed payments and royalties on sales.

FDA approved Revuforj (revumenib), a menin inhibitor developed by Syndax, for treating relapsed or refractory acute leukemia with a KMT2A gene translocation in adults and pediatric patients. The approval was based on the AUGMENT-101 trial results, with 110 and 160 mg tablets expected to be available in November. Syndax also entered a $350 million royalty funding agreement for Niktimvo with Royalty Pharma.

Syndax Pharmaceuticals' price target increased by H.C. Wainwright to $51.00 following FDA approval of Revuforj for acute leukemia. The drug, a breakthrough menin inhibitor, showed a 21% complete response rate in trials. Despite stock volatility, Syndax holds more cash than debt, though it remains unprofitable.

FDA approves Syndax’s Revuforj as the first menin inhibitor for relapsed or refractory acute leukaemia, based on Phase 1/2 AUGMENT-101 trial data showing 21% CR/CRh rate. Revuforj, available in 110mg and 160mg tablets, is also being evaluated for AML with NPM1 mutations and has received orphan drug, breakthrough therapy, and fast track designations.

Syndax Pharmaceuticals announced positive Phase 2 trial results for revumenib in treating relapsed or refractory mutant NPM1 acute myeloid leukemia, with a 23% complete remission rate and a 47% overall response rate in heavily pre-treated patients. The company plans to file a supplemental New Drug Application in 2025 and present further data at the American Society of Hematology Annual Meeting in December 2024.

AstraZeneca and Amgen's Tezspire showed efficacy in reducing nasal congestion and nasal polyps in a Phase 3 trial for chronic rhinosinusitis. Jazz Pharmaceuticals plans to start an early-stage study for narcolepsy treatment JZP441 in 2024. Geron secured $250 million, potentially $375 million, for its myelofibrosis drug Rytelo. Aditum Bio and Leads Biolabs formed Oblenio Bio to develop a T cell engager for autoimmune diseases. Gilead Sciences halted Trodelvy development in second-line non-small cell lung cancer, resulting in a $1.8 billion write-down. Avid Bioservices is being taken private by GHO Capital Partners and Ampersand Capital Partners in a $1.1 billion deal.

Syndax Pharmaceuticals and Royalty Pharma announce a $350 million synthetic royalty funding agreement for Niktimvo (axatilimab-csfr), expected to fund Syndax through profitability. Syndax retains profit participation and upside potential, positioning them for successful launches of first-in-class medicines. Royalty Pharma sees Niktimvo's potential in treating chronic graft-versus-host disease (GVHD).