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.
Nektar Therapeutics Inc. President and CEO Howard Robin didn’t mince words during a call with investors after market close Feb. 23 to disclose top-line data from a phase II study testing rezpegaldesleukin (rezpeg) in systemic lupus erythematosus, which fell short of partner Eli Lilly and Co.’s criteria for advancing to phase III and raised uncertainty as to how the big pharma might proceed in other indications such as atopic dermatitis (AD).
Fresh off an end-of-year IPO, Coya Therapeutics Inc. is gearing up for clinical testing with its lead Treg-enhancing biologic in neurodegenerative disease, aiming to build on a wealth of academic-generated data highlighting the potential of Treg therapy to attack the neuroinflammation underlying diseases such as amyotrophic lateral sclerosis and Alzheimer’s disease.
Updated MRI results from Clene Inc.’s phase II Visionary-MS trial testing gold nanocrystal therapy CNM-Au8 bolsters the company’s premise that targeting energy metabolism could protect neuronal function in patients with multiple sclerosis and lays the groundwork for a phase III trial as soon as a strategic partner is found.
Adhering to the recommendation of a mixed advisory committee vote, the U.S. FDA cleared use of GSK plc’s daprodustat as the first oral hypoxia-inducible factor prolyl hydroxylase (HIF-PHI) inhibitor for treating anemia in patients with chronic kidney disease (CKD) who are on dialysis. For CKD patients not on dialysis, the agency determined the drug’s safety has not been established.
Winning the race to market with the first oral selective estrogen receptor degrader (SERD) for breast cancer is Menarini Group’s elacestrant, which gained U.S. FDA approval for use as second- and third-line therapy in patients with ER-positive/HER2-negative advanced or metastatic disease with the ESR1 mutation. It marks the first therapy approved specifically targeting ESR1, found in up to 40% of patients with ER-positive/HER2-negative disease.
Mantle cell lymphoma (MCL) patients developing resistance to existing BTK inhibitors now have a new treatment option, with the U.S. FDA’s accelerated approval of Jaypirca (pirtobrutinib) from Eli Lilly and Co.’s oncology unit, Loxo@Lilly. It is indicated for use in adults with relapsed or refractory disease who have received at least two lines of systemic therapy, including a BTK inhibitor.
