The U.S. FDA has approved the priority sNDA for a new first-line maintenance therapy in extensive-stage small-cell lung cancer from Jazz Pharmaceuticals plc. Zepzelca (lurbinectedin) can now be combined with Tecentriq (atezolizumab, Roche AG) or atezolizumab and hyaluronidase-tqjs (Tecentriq Hybreza, Roche) as a maintenance therapy for adults who haven’t progressed after first-line induction therapy with atezolizumab, carboplatin and etoposide.
A U.S. FDA complete response letter (CRL) citing manufacturing concerns has stepped into the way of a rare genetic disorder treatment. Fortress Biotech Inc. and its subsidiary, Cyprium Therapeutics Inc., said the letter in response to the NDA for CUTX-101, an injectable formulation of copper histidinate, noted cGMP deficiencies at the facility where the Menkes disease treatment is manufactured.
Anaptysbio Inc. said it intends to split into two publicly traded companies – one for developing a pipeline, the other for managing royalties – each with its own business model. The royalty company will hold and manage the rights to big-earning Jemperli (dostarlimab) royalties from collaborator GSK plc, along with imsidolimab milestones and royalties from Vanda Pharmaceuticals Inc.
Enanta Pharmaceuticals Inc.’s phase IIb study of zelicapavir missed its primary endpoint in treating respiratory syncytial virus (RSV) but mined enough positive results to get the company to talk about advancing the once-daily oral treatment into further, larger studies.
Eli Lilly and Co.’s oral selective estrogen receptor degrader (SERD) imlunestrant has been approved by the U.S. FDA for treating late-stage breast cancer patients. Branded Inluriyo, the second-line treatment is for treating adults with estrogen receptor-positive, human epidermal growth factor receptor-2-negative estrogen receptor 1-mutant advanced or metastatic breast cancer.
The winding regulatory road for the BLA to Capricor Therapeutics Inc.’s cell therapy for Duchenne muscular dystrophy has more clarity. Out of a recent type A meeting between Capricor and the U.S. FDA, prompted by a complete response letter in July regarding lead asset CAP-1002 (deramiocel), the two are in agreement about a path to potential approval.
After a phase III stumble, Acadia Pharmaceuticals Inc. will drop development of ACP-101, intranasal carbetocin, to treat hyperphagia in patients with the rare genetic disorder Prader-Willi syndrome. Top-line data from the 12-week, double-blind, randomized phase III study missed its primary endpoint by not producing a statistically significant improvement over placebo.
The first new U.S. FDA-approved therapeutic option for PTSD in more than 20 years will have to wait. A supplemental NDA seeking approval of Rexulti (brexpiprazole) combined with sertraline, filed by one of Japan’s biggest pharmas, Otsuka Pharmaceutical Co. Ltd., received a complete response letter (CRL) from the agency.
MBX Biosciences Inc. posted positive top-line phase II study data in treating chronic hypoparathyroidism, positioning itself to take on a U.S. FDA-approved therapy for the rare endocrine disease. MBX’s Avail study of canvuparatide hit its primary endpoint in treating adults, demonstrating statistical significance at week 12 and positive six-month results from the open-label extension study. MBX said it is poised to begin a phase III study of once-weekly canvuparatide next year.
Shape Therapeutics Inc. could bring in as much as $1.2 billion in a new option and license deal with Vectory Therapeutics BV. It’s another collaboration for both companies that are known for working with large and small pharmas. Vectory is getting the exclusive option to evaluate Shape’s brain-penetrating adeno-associated virus capsid, SHP-DB1, against three targets, including mHTT, TDP-43 for Huntington’s disease and phosphorylated tau for Alzheimer’s disease.
