Today, a brief rundown of news from Aspen Neuroscience, as well as updates from Aldeyra Therapeutics, Rhythm Pharmaceuticals and Mestag Therapeutics that you may have missed.
Aspen Neuroscience said Wednesday its experimental stem cell-derived therapy for Parkinson’s disease showed improvement in symptoms and quality of life in an early-stage trial. Sasineprocel is a type of personalized cell therapy that Aspen has tested in eight people with Parkinson's, with half receiving either a high or low dose. At 12 months, participants receiving a low dose had an average time of 2.1 hours keeping symptoms in check, compared with 2.4 hours in the high dose group. Low-dose participants saw an improvement in symptoms measured by a self-reported questionnaire by 51.6%, compared to 28.5% for the high-dose group. Aspen said there were no serious surgical adverse events and no severe graft-induced dyskinesia. Phase 3 testing for sasineprocel will start later this year. — Delilah Alvarado
Aldeyra Therapeutics lost more than 70% of its market value Tuesday after disclosing that the Food and Drug Administration rejected an approval request from the Massachusetts-based biotechnology company. According to Aldeyra, the FDA does not believe the company has gathered enough evidence from “well-controlled investigations” to conclude that its experimental medicine, “reproxalap,” is an effective treatment for dry eye disease. The agency also took issue with the clinical data package submitted, explaining in its rejection letter how the “inconsistency of study results raises serious concerns about the reliability and meaningfulness” of any positive findings. Aldeyra said the letter mentioned no problems with the drug’s safety or manufacturing, nor did it recommend conducting additional trials. Rather, agency staff advised the company to probe deeper into why certain studies failed and what populations or conditions might reproxalap be effective in. The company said it plans to quickly request a “Type A” meeting with the FDA. It also noted that it does not think label negotiations during the drug’s review were completed, based on label drafts provided by the FDA in December and again in March. — Jacob Bell
Rhythm Pharmaceuticals on Monday reported that all four substudies in a late-stage clinical trial focused on patients with certain kinds of rare, genetically-driven obesities had failed. To participate in the trial, a patient must have select genetic variants in “MC4R,” a neural pathway that regulates hunger, energy levels and satiety. Once enrolled, patients were given either a placebo or Rhythm’s setmelanotide, a drug initially approved in 2020 as Imcivree for several other uncommon forms of obesity. CEO David Meeker said in a statement that, while not the results Rhythm had hope for, his team remains “encouraged by compelling signals.” The announcement is a “modest disappointment,” according to TD Cowen analyst Phil Nadeau, who wrote in a client note that investor expectations were already low both in terms of the trial succeeding and the drug’s commercial opportunity for these indications. Dennis Ding of Jefferies, meanwhile, called the findings “surprising” but argued they don’t fundamentally impact Rhythm from an investment standpoint. — Jacob Bell
U.K. biotechnology startup Mestag Therapeutics said Wednesday it raised $40 million in additional venture financing to bring its portfolio of fibroblast drugs for cancer and immune diseases into the clinic. Its backers include SV Health Investors, Johnson & Johnson's venture arm JJDC, Forbion, GV and Northpond Ventures. Mestag’s lead program, codenamed "MST-0312," is a bispecific antibody it theorizes can provoke an immune response in solid tumors that have been difficult to treat with immunotherapy. An early clinical study is expected to start later this year. — Gwendolyn Wu