It’s a good day to be an investor in shares of PTC Therapeutics, Inc. (NASDAQ:PTCT), after the the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) released highlights from its November meeting, including the decision to renew the conditional marketing authorization for PTCT’s Translarna in nonsense mutation DMD. Given mixed clinical data to date, CHMP has requested that PTCT conduct an additional clinical trial to establish full benefit/risk to support full marketing approval.
PTC Therapeutics shares reacted to the news, jumping neary 80% to $10.75 in pre-market trading Friday.
“We are pleased with this outcome which took into account all available data for Translarna,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “This decision reflects the benefit that Translarna is having for patients suffering from nonsense mutation Duchenne muscular dystrophy.”
“Translarna has shown clinically meaningful benefits for patients,” said Eugenio Mercuri, M.D., Professor of Pediatric Neurology at the Catholic University, Rome, Italy. “Duchenne is a devastating disease with a progressive loss of function. Maintaining function is of the utmost importance to patients.”
“The consistency of Translarna’s benefit shown across key endpoints is impressive for a dystrophin replacement therapy,” said Craig McDonald, M.D., Professor of Pediatrics and Chair of the Department of Physical Medicine & Rehabilitation at University of California. “I am encouraged for the DMD community by the CHMP’s recommendation.”
“For boys with Duchenne, every day matters and functional loss cannot be regained. Patients need access to innovative new therapies like Translarna,” stated Filippo Buccella, founder of the Italian Parent Project, a patient advocacy group for Duchenne Muscular Dystrophy.
On the ratings front, PTCT has been the subject of a number of recent research reports. In a report issued on November 3, Cowen analyst Ritu Baral reiterated a Hold rating on PTCT. Separately, on November 2, Barclays’ Geoff Meacham reiterated a Buy rating on the stock and has a price target of $20.
According to TipRanks.com, which ranks over 7,500 financial analysts and bloggers to gauge the performance of their past recommendations, Ritu Baral and Geoff Meacham have a yearly average return of 3.7% and 15.3% respectively. Baral has a success rate of 40% and is ranked #671 out of 4200 analysts, while Meacham has a success rate of 62% and is ranked #200.
Overall, 6 research analysts have assigned a Hold rating and one research analyst has given a a Buy rating to the stock. When considering if perhaps the stock is under or overvalued, the average price target is $17.50 which is 191.7% above where the stock closed yesterday.
PTC Therapeutics, Inc. engages in the discovery, development, and commercialization of orally administered, small molecule therapeutics targeting an area of RNA biology. Its preclinical and discovery programs are focused on the development of new treatments for multiple therapeutic areas, including neuromuscular disease, oncology and infectious disease. It product, candidate ataluren, is used for the treatment of patients with genetic disorders that arise from a type of genetic mutation known as a nonsense mutation.