In January, a Wall Street analyst predicted the U.S. FDA’s rejection of Eli Lilly and Co.’s application seeking accelerated approval of amyloid beta-targeting Alzheimer’s candidate, donanemab, would be a “mere footnote” in the drug’s development, a forecast confirmed in the wake of positive top-line phase III data showing donanemab significantly slowed cognitive and functional decline in people with early symptomatic disease.
It’s not often that getting an NDA rejected by the U.S. FDA is cause for a company’s stock to rise. But in the case of Ascendis Pharma A/S, the agency’s anticipated complete response letter (CRL) for Transcon PTH (palopegteriparatide) offered some much-needed clarity and a potentially optimistic time frame for an NDA resubmission seeking approval of the once-daily hormone replacement therapy for hypoparathyroidism, news that sent the company’s shares (NASDAQ:ASND) up 24%, or $16.78, to close May 1 at $86.74.
The data are early and from only seven evaluable patients, but results from In8bio Inc.’s phase I study of gamma-delta T-cell therapy INB-100 in leukemia patients who have undergone haploidentical hematopoietic stem cell transplant, presented at the European Society for Blood and Marrow Transplantation meeting, were compelling enough to drive the company’s shares (NASDAQ:INAB) up 183% April 24.
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.
