Cancer of the uterus is the most common gynecological malignancy in the U.S., with over 60,000 diagnoses per year, where incidence and mortality have increased through the years. About 30%-40% of patients with high-grade disease harbor mutations in the PPP2R1A gene, which encodes the primary subunit of protein phosphatase 2A, with hotspots being P179R and S256F.
The BioWorld Cancer Index reversed its early year losses, rebounding from a 4.74% decline at the end of the first quarter (Q1) to finish Q2 up 12.78%. The Dow Jones Industrial Average followed a similar pattern, dropping to -4.41% by the end of April before recovering to a 3.64% gain by Q2’s end. Meanwhile, the Nasdaq Biotechnology Index, down 5.37% at the end of May, narrowed its losses to close June down just 1.92%.
One year after the FDA’s nod, the EMA is following on and recommending conditional approval of Madrigal Pharmaceuticals Inc.’s Rezdiffra (resmetirom) as the first drug in Europe for treating noncirrhotic metabolic dysfunction-associated steatohepatitis.
The biggest M&A activity of the year arrived in the form of Merck KGaA’s earlier-disclosed agreement to buy Springworks Therapeutics Inc. for $47 per share in cash, which represents an equity value of about $3.9 billion and an enterprise value of $3.4 billion, based on Stamford, Conn.-based Springworks’ cash in the bank as of the end of last year.
Snagging its second U.S. FDA approval since being spun out of Pfizer Inc. in 2017, Springworks Therapeutics Inc. is aiming to position Gomekli (mirdametinib), cleared for neurofibromatosis type 1-associated plexiform neurofibromas (NF1-PN) not amenable to complete resection, as a first-in-class drug for adults and potentially best-in-class option for children. The priority approval, which came after market close Feb. 11, more than two weeks ahead of the Feb. 28 PDUFA date, marks the second MEK1/2 inhibitor to hit the market for NF1-PN, a rare, genetic condition.
Rappta Therapeutics Oy has signed an exclusive global license agreement with Springworks Therapeutics Inc. for RPT-04402, a first-in-class molecular glue of specific protein phosphatase 2A (PP2A) complexes. Springworks will be responsible for global development and commercialization of RPT-04402, now named SW-3431, and expects to file an IND application by year-end.
Genetic alterations in FAT1, YAP1 or WWTR1 genes are commonly seen in head and neck squamous cell carcinoma (HNSCC) patients. Targeting Hippo and MAPK pathways in combination has proven effective in preclinical models of HNSCC.
Researchers from Springworks Therapeutics Inc. and affiliated organizations presented the discovery and preclinical characterization of a novel covalent and irreversible YAP/TAZ-TEAD inhibitor, SWTX-143, being developed for the treatment of mesothelioma.
Springworks Therapeutics Inc.’s nirogacestat became the first drug indicated specifically for desmoid tumors, as well as the first gamma-secretase inhibitor to win marketing approval, winning a U.S. FDA nod on the anticipated PDUFA date of Nov. 27. Branded Ogsiveo, nirogacestat has breakthrough therapy, fast track and orphan designations.
