After an up-and-down day – mostly up, toward the end – during which the phrase “totality of the data” got air time aplenty, shares of PTC Therapeutics Inc. (NASDAQ:PTCT) closed at $34.07, a rise of $5.66, or almost 20%, on word of top-line data from Study 041 with Translarna (ataluren) in nonsense mutation Duchenne muscular dystrophy (DMD).
New data from the phase I study of Ultimovacs ASA’s lead candidate showed positive two-year overall survival data for the cancer vaccine. UV-1, combined with the checkpoint inhibitor Keytruda (pembrolizumab, Merck & Co. Inc.) for treating malignant melanoma, demonstrated a 24-month survival rate of 73% in all 30 patients in the study.
Shares in Addex Therapeutics Ltd. (Zurich:ADXN) dropped 48% June 17 on news that the company had terminated a pivotal phase IIb/III study of dipraglurant as a potential treatment for dyskinesia in Parkinson’s disease.
From 20 years of research on metabolic change as a result of salt intake, Karen Duggan discovered in 2003 that a naturally occurring molecule in the human body, native vasoactive intestinal peptide (VIP), was capable of reversing fibrosis caused by hypertension and other chronic diseases such as diabetes. From that discovery, Vectus Biosystems Ltd. was founded, and the company has developed a new class of mimetic drug candidates and a drug library based on VIP.
Alzheimer’s experts have suggested that future research may involve combination therapies after yet another trial failure involving a drug targeting amyloid – in this case Roche Holding AG’s crenezumab. Originally discovered by Swiss biotech AC Immune SA, of Lausanne, crenezumab failed to slow or prevent cognitive decline in people with a certain mutation that causes early onset in a closely watched trial.
A day after announcing it would pump $120 million into a Michigan manufacturing facility for Paxlovid (nirmatrelvir/ritonavir), Pfizer Inc. produced new data it plans to use in an U.S. NDA submission for treating COVID-19. While some data for the oral antiviral fell short of statistical significance, other companies are not far behind Pfizer in their drive for FDA acceptance using similar approaches.
Daiichi Sankyo Co. Ltd.’s quizartinib met the primary endpoint of overall survival in the pivotal phase III Quantum-First study, which tested the addition of quizartinib to chemotherapy vs. chemotherapy alone for adults with newly diagnosed FLT3-ITD-positive acute myeloid leukemia (AML).
Hua Medicine Ltd. presented findings from two phase III trials of its first-in-class dual-acting glucokinase activator dorzagliatin that show the drug significantly improved early phase insulin secretion and glucose sensitivity in patients with type 2 diabetes, thereby restoring glucose homeostasis to treat the underlying cause of diabetes.
With top line results from its major phase III RECON study in hand, Axogen Inc. is hoping to expand adoption of its allograft nerve repair product Avance. RECON was designed as a non-inferiority study comparing Avance to nerve cuffs (manufactured conduits) which are commercially available. The study met its primary endpoint for the return of sensory function as measured by static two-point discrimination, and the safety profile was consistent with previously published data.
Sanofi SA and GSK plc were beat to market during the first wave of COVID-19 by vaccines from upstarts such as Biontech SE and Moderna Inc. – but the French and U.K. vaccine specialists are gaining traction in the race to develop booster shots against newer variants.