As Bridgebio Pharma Inc. awaits word from the U.S. FDA regarding the small-molecule transthyretin (TTR) stabilizer acoramidis for TTR-mediated amyloidosis cardiomyopathy, the firm closely followed this week’s headline about another spinout with news that the Palo Alto, Calif.-based company has signed a joint research pact with Hitgen Ltd., of Chengdu, China.
Regeneron Pharmaceuticals Inc. suffered an unsurprising hitch in the form of a complete response letter (CRL) related to linvoseltamab, a bispecific antibody designed to bridge B-cell maturation antigen on multiple myeloma cells containing CD3-expressing T cells, for relapsed/refractory multiple myeloma.
A week after the U.S. FDA spurned Lykos Therapeutics Inc.’s bid for approval of a therapy in post-traumatic stress disorder (PTSD), shares of Alzamend Neuro Inc. (NASDQ:ALZN) zoomed upward on more news in the indication with a next-generation, cocrystal lithium compound AL-001. Atlanta-based Alzamend’s stock closed Aug. 19 at $5.85, up $3.82, or 188%.
Label comparisons began promptly with the accelerated U.S. FDA clearance of Gilead Sciences Inc.’s oral peroxisome proliferator-activated receptor (PPAR)-delta drug, Livdelzi (seladelpar), for primary biliary cholangitis (PBC). The space includes Ipsen Pharma SA’s dual PPAR alpha/delta agonist, Iqirvo (elafibranor), licensed from Genfit SA and cleared in June 2024, as well as Ocaliva (obeticholic acid), the first-in-class farnesoid X receptor agonist from Intercept Pharmaceuticals Inc., greenlighted for PBC in May 2016.
Wall Street promptly started speculating about the product’s odds in the graft-vs.-host disease (GVHD) marketplace shortly after Syndax Pharmaceuticals Inc. and Incyte Corp. scored U.S. FDA approval – well ahead of the Aug. 29 PDUFA date – of Niktimvo (axatilimab), an anti-CSF-1R antibody for the treatment of chronic disease after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs.).
With favorable data from the phase III trial testing izokibep, a small protein therapeutic designed to inhibit IL-17A, in hidradenitis suppurativa (HS), Acelyrin Inc. CEO Mina Kim said the firm will “look at all the options and do what’s best for the program.” The phase III trial hit its primary endpoint of Hidradenitis Suppurativa Clinical Response 75 at 12 weeks. At the same time, the Los Angeles-based firm made known a refocused pipeline strategy that prioritizes lonigutamab in thyroid eye disease (TED) and is projected to extend its cash runway.
Aiming to offer an advantage over Dupixent (dupilumab, Regeneron Pharmaceuticals Inc.), dermatology-focused Galderma Holding SA won clearance from the U.S. FDA for Nemluvio (nemolizumab) as a prefilled pen providing a subcutaneous injection to treat adults with prurigo nodularis (PN).
Speculation about potentially wider meaning among developers of psychedelic drugs was reignited after the complete response letter (CRL) from the U.S. FDA to Lykos Therapeutics Inc. for midomafetaminecapsules, which the company wants to use in combination with psychological intervention to treat post-traumatic stress disorder (PTSD). CEO Amy Emerson called the FDA move “deeply disappointing,” adding that San Jose, Calif.-based Lykos plans to request a meeting with the FDA to ask for reconsideration.
Novartis AG CEO Vas Narasimhan during the company’s July 18 second-quarter earnings call said his firm was “still in the midst of completing” the buyout disclosed in February of Morphosys AG, of Munich, for €2.7 billion (then US$2.9 billion), which brought worldwide rights to pelabresib, the small-molecule BET inhibitor for myelofibrosis.
Just ahead of the Aug. 13 PDUFA date, Citius Pharmaceuticals Inc. won the U.S. FDA’s go-ahead for Lymphir (denileukin diftitox-cxdl), a new immunotherapy for relapsed/refractory (r/r) cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy. The drug is Cranford, N.J.-based Citius’ first approved therapy and the only CTCL treatment that targets the interleukin-2 receptor found on malignant T cells and regulatory T cells. It’s also the first green light given by the FDA for r/r CTCL since 2018.
