Is PTC Therapeutics on the Road to Recovery?

PTC Therapeutics Inc. (NASDAQ: PTCT) saw its shares jump late Friday morning on positive news on its nonsense mutation Duchenne muscular dystrophy (nmDMD) treatment. The company announced that the National Institute for Health and Care Excellence (NICE) has recommended Translarna (ataluren) for ambulatory patients aged five years and older with nmDMD in connection with a Managed Access Agreement with NHS England.

It is worth noting that this provision of patient access is subject to the finalization of the NICE draft guidance, which the agency expects in May of 2016. This could be the first step in PTC’s recovery, as the stock has been crushed over the past year.

This company cratered in February after it announced that it received a Refuse to File letter from the U.S. Food and Drug Administration (FDA) regarding its New Drug Application (NDA) for Translarna. The FDA said in the letter that the application was not sufficiently complete to permit a substantive review. PTC first learned of the Refuse to File decision via this letter and is reviewing its content to determine the appropriate next steps.

Paul Lenihan, chief executive of Action Duchenne, commented on the NICE recommendation:

This is an important day for the Duchenne community, which has been working hard to make this ground-breaking drug available to boys with nonsense mutation Duchenne muscular dystrophy. We are delighted by this positive recommendation and NICE’s recognition that Translarna is an important new medicine for patients. This decision is a hugely encouraging sign that both NICE and NHS England have listened to the patient community, bringing hope to each and every parent and patient fighting DMD.

For some background: Translarna is an oral, first-in-class, protein restoration therapy for the treatment of nmDMD. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy.

Shares of PTC were trading up 42.5% at $8.88 on Friday in the noon hour, with a consensus analyst price target of $27.40 and a 52-week trading range of $5.27 to $74.40.

Contact [email protected] for any questions or corrections.