PTC Therapeutics (PTCT) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Commission (EC) once again adopted a negative opinion on the annual renewal of the conditional marketing authorization of Translarna (ataluren) to treat nonsense mutation Duchenne muscular dystrophy (nmDMD). PTC Therapeutics plans to further request a re-examination of the negative opinion regarding Translarna in the EU. Meanwhile, the marketing authorization for Translarna will remain in effect ...

- PTC to submit a request for re-examination - WARREN, N.J., June 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the CHMP has issued a negative opinion on the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nmDMD. This opinion follows the return of the previously issued negative opinion by the European Commission (EC) for re-review. PTC plans to request re-examination of the opinion. The marketing authorization for Transl ...

This rise in share price is attributable to the company's progress with its pipeline development. Last week, management reported encouraging interim data from the mid-stage PIVOT-HD study evaluating PTC518, its investigational treatment for Huntington's disease (HD). Following 12 months of treatment with PTC518, patients achieved dose-dependent lowering of mutant huntingtin (mHTT) protein in the blood and cerebrospinal fluid (CSF). The FDA also removed a partial clinical hold on this study, which was previo ...

WARREN, N.J., June 21, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that on June 17, 2024, the company approved non-statutory stock options to purchase an aggregate of 1,380 shares of its common stock and 6,260 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to nine new employees. The awards were made pursuant to the Nasdaq inducement grant exception as a component of the new hires' employment compensation. ABO ...

- Conference call and webcast to be held June 20th at 8:00 am EDT - PTC is a global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to innovate to identify new therapies and to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines. PTC's mission is to provide access to best-in-clas ...

- Review of European marketing application for PKU now initiated - WARREN, N.J., May 28, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the sepiapterin MAA for PKU has been validated and accepted for review by the EMA. "The validation of the MAA is an important step towards making sepiapterin available to children and adults affected by PKU in Europe," said Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics. "The European submission is the first of several g ...

PTC Therapeutics, Inc. (PTCT) announced that the European Commission (EC) has decided against adopting the Committee for Medicinal Products for Human Use's (CHMP) negative opinion of January 2024 on the annual renewal of the conditional marketing authorization of Translarna (ataluren). Translarna is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. Following the decision, the EC has returned the opinion t ...

- European Commission decides against adoption of negative opinion - - Translarna authorization remains active in Europe - - 2024 revenue guidance paused - WARREN, N.J., May 20, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the European Commission (EC) has decided not to adopt the CHMP's negative opinion of January 24, 2024 on the annual renewal of the conditional marketing authorization of Translarna™ (ataluren) and has returned the opinion to the CHMP for re-evaluation. A ...

- PDUFA target action date of November 13, 2024 - WARREN, N.J., May 14, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) announced today that the FDA has accepted for filing the Biologics License Application (BLA) for Upstaza™ (eladocagene exuparvovec), a gene therapy for the treatment of AADC deficiency. The application has been granted Priority Review with a target regulatory action date of November 13, 2024. "We are excited to be one step closer to bringing an approved therapy to patients with ...

Financial Data and Key Metrics Changes - Total revenue for the first quarter was $210 million, with DMD franchise revenue of $161 million, reflecting strong commercial performance [5][20][42] - Non-GAAP R&D expenses decreased to $107 million from $180 million year-over-year, indicating strategic portfolio prioritization [21] - Cash, cash equivalents, and marketable securities totaled $885 million as of March 31, 2024, up from $877 million at the end of 2023, providing a strong balance sheet for future initiatives [43] Business Line Data and Key Metrics Changes - Translarna generated $104 million in revenue during the quarter, while Emflaza net product revenue was $57 million, demonstrating effective brand protection strategies [37][42] - The global customer-facing team delivered $178 million in revenue from five marketed products, with a focus on growth and diversification [15] Market Data and Key Metrics Changes - The company is expanding its geographic presence in Latin America and the Middle East, with ongoing regulatory filings and approvals anticipated for products like Tegsedi and Waylivra [19] - The European market continues to be a significant contributor to Translarna revenue, accounting for approximately 45% of total revenue, consistent with the previous year [80] Company Strategy and Development Direction - The company plans to submit the NDA for sepiapterin in mid-2024 and for vatiquinone in late 2024, aiming to address significant unmet needs in the PKU and DMD markets [13][33] - The strategy includes maximizing Translarna revenue in Europe while preparing for the U.S. launch of Upstaza, the first approved gene therapy for AADC patients [39] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in achieving all planned regulatory and clinical milestones for 2024, highlighting a strong start to the year [12][35] - There is significant anticipation for the potential availability of Translarna in the U.S. market, with positive feedback from physicians and patients regarding its efficacy [54][80] Other Important Information - The company is actively working on regulatory submissions in Brazil and Japan for sepiapterin, with a focus on meeting the needs of PKU patients globally [38] - The CARDINAL study for utreloxastat in ALS patients is on schedule to report top-line results in the fourth quarter of 2024, with expectations for positive outcomes [14] Q&A Session Summary Question: What work has been done around Europe, Japan, and Brazil regarding upcoming filings? - The company has established commercial infrastructure in these regions and is actively engaging with treatment centers and patient communities to prepare for launches [24] Question: How much did the EU contribute to Translarna revenue this quarter? - The contribution from the EU is consistent with the first quarter of last year, representing around 45% of total revenue [80] Question: Can you provide insights on reimbursement progress in Europe for AADC gene therapy? - The company is seeing favorable pricing and reimbursement negotiations in Europe, which are expected to carry over into the U.S. market upon launch [84] Question: What are the expectations for the efficacy of the Huntington study? - The company aims for a statistically significant benefit on the ALSFRS scale, with a clinically meaningful effect defined as a 2.5 point difference between treatment and placebo groups [104] Question: What is the status of Translarna in Europe? - The European Commission has not yet adopted the CHMP opinion, and the company is actively ensuring patient access to Translarna while awaiting further updates [88]