Pipeline Therapeutics Inc., which received U.S. FDA approval to move into a phase Ib/IIa study of PIPE-307 in relapsing-remitting multiple sclerosis (RRMS) last year, will be advancing the oral, small-molecule muscarinic M1 receptor antagonist in collaboration with Janssen Pharmaceutica NV in an agreement that could be worth more than $1 billion.
Acute thrombosis, including heart attack and stroke, is a leading cause of mortality worldwide. Yet only a small fraction of patients can be treated with current therapeutic or surgical interventions. Enter Basking Biosciences Inc., a 2019 startup aimed at developing a short-acting, fast-onset thrombolytic drug alongside a reversal agent for treating acute ischemic stroke.
A few weeks after Gossamer Bio Inc. said it was pausing enrollment in a phase Ib/II study of CNS-penetrant BTK inhibitor GB-5121 in relapsed/refractory CNS lymphoma, citing the drug’s risk/benefit profile observed to date and a prioritization of resources, the company is dropping the drug’s development entirely.
A frustrating lack of detail left analysts with little to do but speculate on the odds for a 2023 U.S. FDA approval of Ascendis Pharma A/S’s Transcon PTH (palopegteriparatide), a parathyroid hormone prodrug for hypoparathyroidism, after the company disclosed a letter from the agency citing unspecified deficiencies that preclude further discussions about labeling and postmarketing requirements.
More than a decade ago, three scientists were part of a team at G1 Therapeutics Inc. that led to the now-approved CDK4/6 inhibitor Cosela (trilaciclib). The same work also led to findings showing CDK2 as a promising target for cancers that developed resistance to CDK4/6 inhibition.
Shares of Biomea Fusion Inc. (NASDAQ:BMEA) rocketed up 89% to close at $29.30 March 28 after the company reported early cohort data from its Covalent-111 phase I/II trial, showing treatment with the lowest dose of menin inhibitor BMF-219 reduced median A1c levels in patients with type 2 diabetes by 1% at only four weeks.
Preparing for next month’s launch of the first U.S. FDA-approved therapy for activated phosphoinositide 3-kinase delta syndrome (APDS), a rare disease only identified 10 years ago, Pharming Group NV disclosed pricing for the newly branded Joenja (leniolisib) of $750 per tablet, or $547,500 per year.
Pharming Group NV’s stock skyrocketed March 24 on news that the U.S. FDA approved Joenja (leniolisib) to treat activated phosphoinositide 3-kinase delta syndrome (APDS) for those ages 12 and older. The nod, which came a few days ahead of the March 29 PDUFA date, sent shares (NASDAQ:PHAR) up 33%, or $3.69, to end the day at $14.96.
In Alzheimer’s, the amyloid beta hypothesis has proved most persistent in terms of drug development efforts to date, but aggregation of other pathogenic factors – phosphorylated tau (p-tau), APOE4, TREM2 and alpha-synuclein, for example – have also emerged as hallmarks of the disease. It’s that aggregation that seven-year-old Truebinding Inc. aims to target with its lead program, TB-006, a monoclonal antibody against galectin-3.
Shares of Reata Pharmaceuticals Inc. tanked unexpectedly Feb. 27, a day before the U.S. FDA is set to make a decision on the NDA seeking approval of once-daily Nrf2 activator omaveloxolone for Friedreich’s ataxia, on reports that Billy Dunn, the director of the FDA’s Office of Neuroscience, was stepping down from his position.
