China’s National Medical Products Administration (NMPA) has accepted for review Hightide Therapeutics Inc.’s NDA for HTD-1801 for type 2 diabetes, marking the Shenzhen-based company’s first NDA submission and a major step toward commercialization.
The strife-marked Duchenne muscular dystrophy (DMD) space drew forth another outspoken political figure in the shape of Sen. Ron Johnson (R-Wisc.), who said he was “enraged” by the U.S. FDA’s refusal to consider PTC Therapeutics Inc.’s Translarna (ataluren) for the treatment of nonsense mutation disease.
On the heels of China’s approval of Sino Biopharmaceutical Ltd.’s rovadicitinib, Sanofi SA is now inlicensing the first-in-class dual JAK/ROCK inhibitor in a deal worth more than $1.4 billion.
Rapport Therapeutics Inc. added $20 million to its cash runway for its lead phase III oral seizure drug, RAP-219, through a potential $328 million license deal signed with Tenacia Biotechnology Co. Ltd.
Fast on the heels of the U.S. FDA’s go-ahead regarding the expanded use of Wellcovorin (leucovorin) tablets for cerebral folate deficiency in adult and pediatric patients with a confirmed variant in the folate receptor 1 gene – some of whom bear “autistic features” as part of their condition – the American Academy of Pediatrics advised against giving the drug to children with autism spectrum disorder.
Ipsen SA withdrew Tazverik (tazemetostat) from the U.S. market after evidence emerged of secondary hematological malignancies in an ongoing phase Ib/III study in follicular lymphoma.
The failure of the Persevera study, sponsored by Roche Holding AG’s Genentech unit, disrupts advancement of giredestrant in combination with palbociclib (Ibrance, Pfizer Inc.) as a first-line treatment for ER-positive, HER2-negative, locally advanced or metastatic breast cancer. For competitor Olema Pharmaceuticals Inc., which has palazestrant for the same indication, missing the phase III Persevera primary endpoint of progression-free survival translated to a 25.8% stock slide (NASDAQ:OLMA) to a $16 close on March 9.
Any lingering disappointment in the delay for Xenon Pharmaceuticals Inc.’s readout of the phase III X-Tole2 study testing azetukalner in focal onset seizures (FOS) appeared thoroughly extinguished as the company’s KV7 potassium channel opener yielded better-than-expected data, even besting earlier phase IIb findings and positioning the drug for an NDA submission later this year.
Dysregulation of the mechanistic target of rapamycin (mTOR) signaling pathway is frequently observed across a wide range of cancers and contributes to uncontrolled cell proliferation, enhanced cellular metabolism, and tumor progression.
On the heels of China’s approval of Sino Biopharmaceutical Ltd.’s rovadicitinib, Sanofi SA is now inlicensing the first-in-class dual JAK/ROCK inhibitor in a deal worth more than $1.4 billion.
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