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.
Sangamo Therapeutics Inc. put pen to paper on a would-be $1.9 billion-plus deal with Genentech, a unit of Roche AG, to develop intravenously administered genomic drugs for neurodegenerative conditions.
Closely watched-for phase I data from Elevation Oncology Inc. in the Claudin 18.2 space sent shares of the Boston-based firm (NASDAQ:ELEV) down 63%, or $1.35, to close Aug. 6 at 78 cents. Elevation provided initial data from the dose-escalation portion of the ongoing experiment with antibody-drug conjugate (ADC) EO-3021 in patients with advanced, unresectable or metastatic solid tumors likely to express Claudin 18.2, including gastric, gastroesophageal junction (GEJ), pancreatic or esophageal cancers.
In the wake of Sage Therapeutics Inc.’s recent phase II fizzle with SAGE-324 in essential tremor, Wall Street is waiting for better news in the indication, which has racked up failures across several developers. Among those finding favor lately is Praxis Precision Medicines Inc. with Nav-targeting ulixacaltamide, also known as PRAX-628, which Oppenheimer analyst Francois Brisebois said “warrants particular attention.”
As the Sept. 21 PDUFA date looms for arimoclomol from Zevra Therapeutics Inc. in Niemann-Pick type C (NPC), the U.S. FDA’s newly formed Genetic Metabolic Diseases Advisory Committee (GeMDAC) decided in favor of the drug.
Investors wanted more from ALX Oncology Holdings Inc.’s phase II study testing evorpacept (evo) combination therapy in gastric tumors, and the stock (NASDAQ:ALXO) tumbled 36% or $1.77 to close Aug. 1 at $3.04.
Pepgen Inc. fell slightly short of its phase II dystrophin goal with PGN-EDO51 for patients with Duchenne muscular dystrophy (DMD) whose mutations are amenable to an exon 51-skipping approach, but Wall Street reacted in a big way, sending the Boston-based firm’s stock (NASDAQ:PEPG) down 33%, or $5.55, to close July 31 at $11.43.
Succeeding where others have failed in an indication with high unmet need, Celldex Therapeutics Inc. disclosed top-line data from the phase II trial with barzolvolimab (barzo) against two common forms of chronic inducible urticaria (CIndU): cold urticaria (ColdU) and symptomatic dermographism (SD).
Another failure with allosteric tyrosine kinase 2 (TYK2) inhibitor VTX-958 put an end to internal work with the compound, and shares of Ventyx Biosciences Inc. (NASDAQ:VTYX) fell July 29 to $2.24, down 71 cents or 24%, after the firm disclosed results from the phase II, 109-subject trial in moderately to severely active Crohn’s disease (CD).
Biogen Inc. and partner Eisai Co. Ltd. said the EMA’s Committee for Medicinal Products for Human Use has adopted a negative opinion on the marketing bid for lecanemab in early Alzheimer’s disease (AD) and mild AD. The humanized anti-soluble aggregated amyloid-beta monoclonal antibody is approved in the U.S., Japan, China, South Korea, Hong Kong and Israel, and is being sold in the U.S., where it’s branded Leqembi, as well as Japan and China. Eisai, of Tokyo, will ask the CHMP to re-examine the matter.
