The U.S. FDA has approved UCB SA’s Kygevvi (doxecitine and doxribtimine), the first treatment for the ultra-rare, genetic and life-threatening mitochondrial disease thymidine kinase 2 deficiency. The approval comes as the company reaps a reward of rebuilding and reorganizing that it began little more than a year ago.
Early stage data from Terns Pharmaceuticals Inc.’s lead candidate showed a large reduction in the number of leukemia cells in those with previously treated chronic myeloid leukemia. Results from the ongoing phase I Cardinal study of TERN-701, an oral, allosteric BCR-ABL tyrosine kinase inhibitor, were “unprecedented,” according to Terns.
Boehringer Ingelheim GmbH has licensed an unnamed small-molecule preclinical candidate from Kyowa Kirin Co. Ltd. in the autoimmune disease space in a deal worth up to €640 million (US$739 million).
Only two days after Bridgebio Pharma Inc. impressed investors with data from BBP-418 in limb-girdle muscular dystrophy type 2I/R9, the company was back at it again, this time reporting positive top-line results from its global phase III study of encaleret in autosomal dominant hypocalcemia type 1, a genetic form of hypoparathyroidism.
Sovargen Co. Ltd. inked a $550 million license deal with Angelini Pharma SpA, granting Angelini development and commercialization rights to SVG-105, a novel antisense oligonucleotide drug candidate in preclinical development as a potential treatment for intractable epilepsy.
Based on positive phase III study results, Metis Techbio is planning to file an NDA for its AI-derived orally disintegrating tablet drug candidate for pseudobulbar affect, MTS-004, in China next year.
Maplight Therapeutics Inc.’s pricing of a $258.9 million financing this week revived the debate over whether targeting the M1 as well as the M4 muscarinic receptor – as Bristol Myers Squibb Co. does with U.S. FDA-approved Cobenfy (xanomeline and trospium chloride) for schizophrenia – is a better strategy than going after M4 alone.
Based on positive phase III study results, Metis Techbio is planning to file an NDA for its AI-derived orally disintegrating tablet drug candidate for pseudobulbar affect, MTS-004, in China next year.
Tango Therapeutics Inc. plans a pivotal study next year in second-line MTAP-deleted pancreatic ductal adenocarcinoma based on positive phase I/II data with next-generation MTA-cooperative PRMT5 inhibitor vopimetostat (TNG-462) in patients with tumors of that type.
Ena Respiratory Pty Ltd. raised an AU$34 million (US$22.4 million) series B round to advance INNA-051, its nasal spray for symptomatic viral respiratory infections, to phase II trials. New investors in the Melbourne-headquartered company include the Gates Foundation and Flu Lab. Existing investors Brandon Capital, Uniseed and Stoic Venture Capital also participated in the round.
RSS
