Atlantic Healthcare's drug fails late-stage test; Translate Bio breaks out early CF data
→ Cambridge, UK-based Atlantic Healthcare disclosed that the late-stage program for its experimental drug, alicaforsen enema, has failed in patients with pouchitis, a progressive disease characterized by inflammation, ulceration and increasingly uncontrolled, frequent and urgent emptying of the bowel (up to 10-20 times a day and night). “Although we are disappointed that the Phase 3 trial did not achieve statistical significance, we believe the percentage of patients achieving remission in stool frequency and the endoscopic response observed in a number of subpopulations of patients could be noteworthy,” said chief Toby Wilson Waterworth. “Having consulted with key opinion leaders and regulatory advisors, we now plan to meet with the U.S. Food and Drug Administration and European Medicines Agency to discuss a pathway to regulatory approval.”
→ mRNA therapy developer Translate Bio $TBIO unveiled some early data from a 12-patient trial on its experimental inhaled therapy, MRT5005, for patients with cystic fibrosis (CF) on Wednesday. CF occurs when the cystic fibrosis transmembrane conductance regulator (CFTR) protein is either not made correctly, or not made at all, and is characterized by a build-up of thick sticky mucus in the lungs, digestive system and other organs. While there was variability seen percent predicted forced expiratory volume in 1 second (ppFEV1) in patients from day to day — some improvements support a CFTR-related mechanism and may suggest that MRT5005 can enable the production of functional protein, said Steven Rowe, the principal investigator of the Phase I/II study. “If future data confirms this early positive signal, MRT5005 has the potential to provide a clinically meaningful benefit for people with CF independent of CFTR genotype.”
→ Investment firm EQT is taking a majority stake in North Dakota-based Aldevron, a global supplier of plasmid DNA used in gene therapies, as well as proteins, antibodies and mRNA. The company’s founders, management and TA Associates will retain a minority stake.