Watch PTC Therapeutics ahead of FDA panel meeting
Updated with FDA staff comments on September 26, 2017
The U.S. Food and Drug Administration has scheduled an advisory panel meeting for next week to review PTC Therapeutics’ (PTCT) ataluren for the treatment of Duchenne muscular dystrophy.
Commenting ahead of the meeting, Citi analyst Joel Beatty said that U.S. Congressman Collin Peterson is expected to voice his support for the drug.
The analyst argued that PTC Therapeutics could be worth about $59 per share if ataluren, which is marketed with the trade name #Translarna, is approved in the U.S. and about $8 per share if it is not.
PTC Therapeutics’ lead product candidate ataluren is a novel, orally administered small-molecule compound for the treatment of patients with genetic disorders due to a nonsense mutation.
Ataluren is in clinical development for the treatment of Duchenne muscular dystrophy caused by a nonsense mutation, or nmDMD.
The European Medicines Agency has designated ataluren as an orphan medicinal product and the FDA has granted orphan drug designation to ataluren for the treatment of nmDMD.
Back in June, PTC Therapeutics announced that the FDA had notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28 to review the new drug application, or NDA, for ataluren.
The company’s NDA submission was granted standard review by the FDA on March 6, with the FDA setting a Prescription Drug User Fee, or PDUFA, goal date of October 24 for completion of its review of the ataluren NDA.
Earlier this month, Citi analyst Joel Beatty said he had found comments online from U.S. Congressman Collin Peterson that voice support for approval of PTC Therapeutics’ DMD treatment.
The document appeared to have been posted in response to the FDA’s request for comments regarding its advisory panel meeting scheduled for September 28, and is written in the form of a speech to be given at the meeting, Beatty told investors in a research note.
The analyst confirmed with the Congressman’s office that a legislative assistant plans to read the letter on behalf of Representative Peterson at the advisory meeting.
The comments mentioned a constituent with DMD who will be in the audience and for whom “access to ataluren has allowed him to live and thrive well into his 20s with no side effects,” Beatty cited the statement as saying.
The analyst believes political support could play a role in the FDA’s review of ataluren.
Back in June, Beatty had told investors that he was increasing his probability of approval to 25% from 20% following the advisory meeting announcement, but said he still viewed U.S. approval at less than 50/50.
The analyst also pointed out that he believes PTC Therapeutics is worth about $59 per share if Translarna is approved in the U.S. and about $8 per share if Translarna does not receive a U.S. approval.
JPMorgan analyst Anupam #Rama says he remains “somewhat cautious” on shares of PTC Therapeutics (PTCT) with the FDA’s Peripheral and Central Nervous Systems Drugs Advisory Committee convening on September 28 to review the company’s Translarna for the treatment of non-sense mutation Duchenne muscular dystrophy.
Translarna has an FDA action date of October 24. The analyst cites the drug’s “mixed” Phase 2 and Phase 3 data sets and prior refuse-to-file letter for his “somewhat cautious” stance.
For Sarepta Therapeutics (SRPT), Rama sees a net positive read-through from a favorable panel, on the potential for an accelerated path forward for golodirsen in Exon 53 DMD.
Further, a negative panel is likely to be viewed as Translarna-specific, playing out as a net neutral for Sarepta, the analyst tells investors in a research note. He does not see “major fundamental downside risk” for Sarepta shares going into PTC’s panel. Rama keeps a Neutral rating on PTC
FDA STAFF COMMENTS
In the briefing documents, FDA staff wrote: “The Office of Clinical Pharmacology has reviewed the information contained in NDA 200896. The OCP review team does not consider the exposure-response analyses and in vitro data as supportive evidence of effectiveness. See Link for the complete comments and response!
In Monday’s trading, shares of PTC Therapeutics are up 11 cents to $18.64.
Since June 6, the day PTC reported the FDA’s tentative scheduling of the advisory committee meeting, the stock is up 34%.
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