Taking weight off with a glucagon-like peptide-1 (GLP-1) receptor agonist and keeping it off once the drug has been discontinued has been a tall order but Response Pharmaceuticals Inc. is seeing progress. Top-line results from its randomized, double-blind, placebo-controlled, parallel-group phase II study of RDX-002, in those who had already wrapped up a GLP-1 receptor agonist course for reducing obesity, were positive.
Novartis AG’s monoclonal antibody, ianalumab, has notched back-to-back wins, one in treating Sjögren’s disease and the other for primary immune thrombocytopenia (ITP). In Sjögren’s, which has no U.S. FDA-approved treatment, the phase III Neptunus-1 and Neptunus-2 studies are the first phase III trials to prompt statically significant reductions in adults with the autoimmune disease. In ITP, a disease that has yet to see a cure, top-line data of a phase III study of ianalumab combined with eltrombopag stretched to the time to treatment failure compared to placebo, the primary endpoint showing the maintenance of safe platelet levels.
Agios Pharmaceuticals Inc. said the patients’ deaths tied to anemia therapy Pyrukynd (mitapivat) that were announced in a securities analyst’s Aug. 4 note to investors hasn’t changed the drug’s already established benefit-risk profile on the drug’s U.S. prescribing information.
With the U.S. FDA’s approval of Lenz Therapeutics Inc.’s Vizz (aceclidine ophthalmic solution), there is a third eye drop on the market for treating presbyopia. Vizz, however, is the first aceclidine-based eye drop for adults.
Soligenix Inc.’s phase IIa study of SGX-945 (dusquetide) for treating oral ulcers in those with Behçet's disease showed biological efficacy in the proof-of-concept study. The positive results propelled the company’s stock (NASDAQ:SNGX) a hefty 134% higher on July 31, with shares closing at $2.93 each.
Cardiff Oncology Inc.’s mid-stage and ongoing clinical trial of its PLK1 inhibitor in colorectal cancer produced promising data but the company’s stock lost a quarter of its value on July 30 because of second-quarter losses. The randomized phase II study of onvansertib combined with a standard-of-care treatment in first-line, RAS-mutated metastatic colorectal cancer produced a 49% confirmed objective response rate at the 30-mg dose level compared to 30% in the control arm.
With reduced sales and operating expectations for the rest of the year, Novo Nordisk A/S is on the receiving end of a huge stock drop. For a big pharma, where the stock drops are usually more modest, the shares (NASDAQ:NVO) fell off a cliff on July 29, closing 21.8% downward to $53.93 each, the lowest price per share in the past 12 months. Lower U.S. sales of semaglutide blockbusters Wegovy and Ozempic for treating obesity and diabetes are at the heart of the plunge. In May, the Danish company had expected 13% to 21% sales growth but now is looking at only 8% to 14%.
After Recognify Life Sciences Inc.’s phase IIb failure in treating the cognitive impairment in those with schizophrenia, the indication is no closer to a U.S. FDA approved therapy. The placebo-controlled study of inidascamine missed its primary endpoint to improve cognition, joining a list of companies that have tried and failed to find a successful treatment.
Apnimed Inc.’s lead oral therapy for obstructive sleep apnea has delivered a second round of positive phase III data, bringing a potential U.S. FDA filing closer. Top-line results taken from the second pivotal late-stage study show AD-109 (aroxybutynin 2.5mg/atomoxetine 75 mg) hit its primary endpoint with clinically meaningful and statistically significant drops in airway obstruction at 26 weeks.
In a 10-1 vote, the U.S. FDA’s Psychopharmacologic Drugs Advisory Committee said Otsuka Pharmaceutical Co. Ltd.’s studies of its combination post-traumatic stress disorder (PTSD) treatment did not show enough efficacy. Had the adcom felt otherwise, the path may have been smoother for a new treatment in an indication that hasn’t seen regulatory advancement in decades. The committee’s conclusion, however, is not binding on the FDA, which will make its own decision whether to approve.
