PTC Therapeutics develops and commercializes medicines for children and adults with rare disorders, with a life-science business focused on clinically differentiated therapies in areas including neurology and metabolism. Company news commonly covers product revenue, commercial launch activity, financial guidance, investor presentations, and updates from its rare-disease development portfolio.
Recurring developments include Sephience™ (sepiapterin) launch and revenue updates, clinical and regulatory disclosures involving votoplam, vatiquinone and Translarna™ (ataluren), and partnership-related program responsibilities. PTC also reports governance changes and Nasdaq inducement equity grants tied to employee compensation.
PTC Therapeutics (NASDAQ: PTCT) has submitted a New Drug Application (NDA) to the FDA for sepiapterin, targeting the treatment of phenylketonuria (PKU) in both pediatric and adult patients. The submission is based on significant results from the phase 3 APHENITY trial, which showed a 63% mean reduction in Phe levels overall and 69% in classical PKU patients. Notably, 84% of subjects achieved Phe control (<360 µmol/L) and 22% normalized Phe levels. The Phe tolerance sub-study revealed that 60% of subjects could increase protein intake while maintaining Phe control, potentially allowing for diet liberalization. Marketing applications for Europe are under review, with submissions for Japan and Brazil expected later in 2024.
PTC Therapeutics (NASDAQ: PTCT) has announced a webcast conference call to discuss its second quarter 2024 financial results and provide a business update. The call is scheduled for Thursday, August 8, at 4:30 p.m. EDT. Participants can access the call by phone after registering for dial-in details. To avoid delays, it's recommended to dial in 15 minutes before the call starts. The webcast can be accessed on the Investor section of PTC's website. A replay will be available approximately two hours after the call ends and will be archived on the company's website for 30 days.
PTC Therapeutics (NASDAQ: PTCT) announced that the European Medicines Agency's CHMP has issued a negative opinion on renewing the conditional marketing authorization of Translarna™ (ataluren), used for treating nmDMD. This follows a similar negative opinion issued earlier by the European Commission (EC), prompting a request for re-review. Despite the negative opinions, the existing marketing authorization remains valid pending re-examination and EC ratification, allowing Translarna to stay on the market until the end of 2024. CEO Matthew B. Klein expressed disappointment but noted the support of the STRIDE real-world evidence and key opinion leaders in the field. PTC plans to request a re-examination to ensure continued availability of Translarna.
PTC Therapeutics (NASDAQ: PTCT) announced the approval of non-statutory stock options and restricted stock units (RSUs) for nine new employees on June 17, 2024, under the Nasdaq inducement grant exception. The inducement grants include stock options to purchase 1,380 shares at an exercise price of $35.40 per share and 6,260 RSUs. The stock options have a 10-year term and vest over four years, with 25% vesting on the first anniversary of the employee's hire date and 6.25% vesting quarterly thereafter. The RSUs will vest annually over four years, subject to continued employment. These grants aim to attract and retain talent in accordance with Nasdaq Listing Rule 5635(c)(4).
PTC Therapeutics (NASDAQ: PTCT) released interim results from their Phase 2 PIVOT-HD study of PTC518 in Huntington's disease (HD) patients. At Month 12, PTC518 showed dose-dependent reductions of mutant huntingtin (mHTT) protein in blood and cerebrospinal fluid (CSF), with 22% and 43% reductions at 5mg and 10mg doses, respectively. Additionally, clinical assessments indicated a slowing in motor symptom progression, with Total Motor Score (TMS) worsening by 2.0 points (5mg) and 1.3 points (10mg) compared to 4.9 points for placebo. Importantly, PTC518 was found to be safe and well-tolerated. Based on these findings, the FDA lifted the partial clinical hold on the program. The company will discuss these results in a conference call and webcast on June 20, 2024, at 8:00 am EDT.
PTC Therapeutics (NASDAQ: PTCT) announced its participation in William Blair's 44th Annual Growth Stock Conference on June 4, 2024, at 9:00 am ET / 8:00 am CT. The presentation will be webcast live and accessible through the Investor section of PTC Therapeutics' website. The webcast will be archived for 30 days. Attendees should join early to ensure a timely connection.
PTC Therapeutics (NASDAQ: PTCT) announced the validation and acceptance of its marketing application for sepiapterin aimed at treating PKU, by the European Medicines Agency (EMA). This marks an essential phase in making sepiapterin available in Europe. CEO Matthew B. Klein highlighted that this submission is the first of many planned for 2024, with the FDA submission expected by Q3 2024, and additional submissions in Brazil and Japan.
The sepiapterin MAA comprises data from the phase 3 APHENITY trial, which showed a 63% mean reduction in Phe levels, with 84% of participants achieving Phe control <360 µmol/L. Results also indicated dietary liberalization while maintaining Phe control. The open-label extension study confirmed the durability of sepiapterin's effects.
PTC Therapeutics (NASDAQ: PTCT) announced the European Commission's decision not to adopt the CHMP's negative opinion on the annual renewal of the marketing authorization for Translarna™ (ataluren). The authorization remains valid, allowing continued availability for patients in Europe. The EC has requested a re-evaluation, considering more comprehensive data. PTC's CEO emphasized the importance of Translarna for patients with nonsense mutation Duchenne muscular dystrophy. Due to the uncertainty of the continued authorization's impact on 2024 revenue, PTC has paused its revenue guidance, with updates to follow.
PTC Therapeutics announced the FDA's acceptance of the Biologics License Application (BLA) for Upstaza™ (eladocagene exuparvovec), a gene therapy targeting AADC deficiency. The FDA has granted Priority Review status, setting a target action date of November 13, 2024. According to PTC's CEO, Matthew B. Klein, the collected data affirm Upstaza's potential transformative benefits. This acceptance marks a critical milestone in the effort to introduce an approved therapy for AADC deficiency in the U.S.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will be participating in the RBC Capital Markets 2024 Global Healthcare Conference on May 14 and the Bank of America Securities 2024 Health Care Conference on May 16. The presentations will be webcast live on the company's website and archived for 30 days.