Ptc Therapeutics Stock Price, News & Analysis

PTC Therapeutics announced the approval of inducement grants to ten new employees on Nov. 20, 2024. The grants include 18,815 non-statutory stock options and 17,995 restricted stock units (RSUs). The stock options have an exercise price of $41.96 per share and a 10-year term, vesting over four years with 25% vesting on the first anniversary and 6.25% quarterly thereafter. The RSUs vest over four years with 25% vesting annually. These grants were approved under Nasdaq Listing Rule 5635(c)(4) as part of new hire compensation packages.

PTC Therapeutics (NASDAQ: PTCT) has received FDA accelerated approval for KEBILIDI™, the first-ever gene therapy directly administered to the brain for treating AADC deficiency. The therapy is approved for both children and adults across all disease severity levels.

KEBILIDI is administered through stereotactic neurosurgery to the putamen of the brain, enabling dopamine synthesis and progressive motor development improvement. The approval is based on ongoing clinical trial PTC-AADC-GT-002, with confirmatory evidence pending from long-term follow-up. PTC Therapeutics has also received a Rare Disease Priority Review Voucher, which they plan to monetize.

PTC Therapeutics reported Q3 2024 total revenue of $196.8 million, leading to increased full-year guidance of $750-800 million. The DMD franchise generated $124 million, with Translarna contributing $72.3 million and Emflaza $51.9 million. The company has three FDA applications under review and plans a fourth submission for vatiquinone in December. Q3 net loss was $106.7 million, improved from $133.0 million in Q3 2023. Cash position strengthened to $1.01 billion as of September 30, 2024, up from $876.7 million at end of 2023.

PTC Therapeutics (NASDAQ: PTCT) has announced its participation in three major healthcare investor conferences in November and December 2024. The company will present overviews at the UBS Global Healthcare Conference on November 13, the Jefferies London Healthcare Conference on November 19, and Citi's 2024 Global Healthcare Conference on December 3.

All presentations will be available via webcast on PTC's investor relations website and will remain accessible for 30 days after each event. Viewers are advised to connect several minutes before the scheduled start times to ensure proper connection.

PTC Therapeutics announced FDA acceptance of its New Drug Application (NDA) resubmission for Translarna™ to treat nonsense mutation Duchenne muscular dystrophy (nmDMD). The resubmission is supported by Study 041 results showing significant benefits in multiple endpoints after 72 weeks of treatment, including improved six-minute walk distance and other mobility measures. The STRIDE registry data demonstrated a 3.5-year delay in loss of ambulation and 1.8-year delay in reaching critical lung function threshold. As this is a resubmission following a previous complete response letter, no FDA action date has been provided.

PTC Therapeutics announced the approval of inducement grants for nineteen new employees on Oct. 23, 2024. The grants include 13,690 non-statutory stock options and 20,250 restricted stock units (RSUs). The stock options have an exercise price of $42.48 per share, vesting over four years with 25% vesting after the first year and 6.25% quarterly thereafter. The RSUs also vest over four years, with 25% vesting annually. These awards were granted under Nasdaq Listing Rule 5635(c)(4) as part of the new employees' compensation packages.

PTC Therapeutics (NASDAQ: PTCT) has announced it will host a webcast conference call on Thursday, November 7, at 4:30 p.m. EST to discuss its third quarter 2024 financial results and provide a business update. Participants are advised to register in advance and dial in 15 minutes before the call to avoid delays. The webcast will be accessible through PTC's Investor Relations website, with a replay available for 30 days after the call.

PTC Therapeutics announced that the CHMP of the European Medicines Agency has maintained its negative opinion on renewing the conditional marketing authorization for Translarna™ (ataluren) for treating nonsense mutation Duchenne Muscular Dystrophy (nmDMD) after re-examination. The opinion will be reviewed by the European Commission, with a decision expected in about 67 days.

PTC's CEO, Matthew B. Klein, M.D., expressed disappointment, stating that the CHMP based its decision on the primary analysis subpopulation of Study 041 instead of considering the totality of evidence, including data from three placebo-controlled trials and the STRIDE registry. He emphasized that the decision goes against the wishes of physicians, patients, and families in Europe.

Translarna will remain authorized in Europe pending the EC review. PTC will continue to make Translarna available to patients and ensure the EC has all possible evidence to support continued authorization.

PTC Therapeutics announced that the FDA has set a target regulatory action date of July 29, 2025, for the review of the New Drug Application (NDA) for sepiapterin. The NDA was submitted for the treatment of phenylketonuria (PKU) in pediatric and adult patients, covering all age groups and disease subtypes. Additionally, the results of the Phase 3 APHENITY trial were published in The Lancet.

The NDA includes data from the APHENITY trial and the ongoing open-label extension study, which shows the durability of sepiapterin's effect and patients' ability to liberalize their diet while maintaining phenylalanine (Phe) control. Recent results indicate that approximately 60% of subjects achieve protein intake above the age-adjusted recommended daily allowance for an unaffected individual while maintaining Phe levels < 360 μmol/L.