PTC Therapeutics, Inc. - PTCT STOCK NEWS

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.

PTC Therapeutics (NASDAQ: PTCT) announced positive updates on its vatiquinone Friedreich ataxia (FA) program. Two long-term extension studies met their pre-specified endpoints, showing statistically significant evidence of durable treatment benefit on disease progression. Key findings include:

1. MOVE-FA study: 144 weeks of vatiquinone treatment resulted in a 3.7-point benefit (p<0.0001, N=70) on the modified Friedreich Ataxia Rating Scale (mFARS).

2. Earlier study: 24-months of vatiquinone treatment showed a 4.8-point benefit on mFARS (p<0.0001, N=41).

PTC plans to submit a New Drug Application (NDA) for vatiquinone in December 2024, following alignment with FDA on key aspects. The company aims to provide a potentially meaningful therapy for all FA patients, particularly children and adolescents for whom no approved therapies exist.

PTC Therapeutics (NASDAQ: PTCT) announced that the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for treating pediatric and adult patients with phenylketonuria (PKU). The NDA is based on the phase 3 APHENITY trial results, which showed a mean reduction in phenylalanine (Phe) levels of 63% in the overall treated population and 69% in classical PKU subjects. 84% of subjects achieved Phe control (<360 µmol/L) as per treatment guidelines, and 22% showed Phe level normalization.

The NDA also includes data from the APHENITY open-label extension study and the Phe tolerance sub-study. The latter demonstrated that about 60% of subjects achieved protein intake above the age-adjusted recommended daily allowance while maintaining Phe control, suggesting sepiapterin could enable diet liberalization for PKU patients.