PTC Therapeutics, Inc. - PTCT STOCK NEWS

PTC Therapeutics reported unaudited 2024 total revenue of approximately $814 million, exceeding guidance. The company's DMD franchise generated strong performance with Translarna™ revenue of ~$340 million and Emflaza® revenue of ~$207 million. The company ended 2024 with a cash balance of ~$1.1 billion.

Key developments include four FDA approval applications submitted in 2024, including Kebilidi™ (approved November 2024) and sepiapterin for PKU (decision expected July 2025). PTC closed a significant collaboration with Novartis for PTC518, receiving $1 billion upfront with potential for $1.9 billion in additional milestones.

For 2025, PTC anticipates total revenues between $600-800 million and projects GAAP R&D and SG&A expenses of $805-835 million. Important upcoming milestones include PIVOT-HD data readout and sepiapterin regulatory decisions in Q2 2025.

PTC Therapeutics (NASDAQ: PTCT) has announced that its CEO, Matthew B. Klein, M.D., will deliver a presentation at the 43rd Annual J.P. Morgan Healthcare Conference. The presentation is scheduled for Monday, January 13, 2025, at 11:15 a.m. PST / 2:15 p.m. EST.

The event will be accessible via webcast on PTC Therapeutics' website under the Investors section's Events and Presentations page. Viewers can access the webcast at https://ir.ptcbio.com/events-presentations, and the recording will remain available for 30 days after the presentation. Attendees are advised to connect several minutes before the start time to ensure proper connection.

PTC Therapeutics (NASDAQ: PTCT) has submitted a New Drug Application (NDA) to the FDA for vatiquinone, aimed at treating children and adults with Friedreich ataxia (FA). If approved, vatiquinone would become the first authorized therapy for children with FA. The application is supported by data from the placebo-controlled MOVE-FA study and two long-term studies, demonstrating significant disease progression slowdown and safety across all age groups.

This marks PTC's fourth FDA approval application in 2024, following their successful AADC gene therapy approval in November 2024, and pending NDAs for sepiapterin (PKU treatment) and Translarna™ (Duchenne muscular dystrophy treatment).

PTC Therapeutics (NASDAQ: PTCT) has announced the approval of inducement grants for eight new employees on December 13, 2024. The grants include non-statutory stock options to purchase 1,725 shares of common stock and 5,060 restricted stock units (RSUs). The stock options were priced at $46.37 per share, matching the closing price on the grant date, and have a 10-year term. Both options and RSUs vest over four years, with options vesting 25% after one year and 6.25% quarterly thereafter, while RSUs vest 25% annually. These awards were approved by PTC's Compensation Committee as part of new hire compensation packages under Nasdaq Listing Rule 5635(c)(4).

PTC Therapeutics (NASDAQ: PTCT) has entered into a global license and collaboration agreement with Novartis for its PTC518 Huntington's disease program. The deal includes a $1.0 billion upfront payment and potential earnings of up to $1.9 billion in development, regulatory, and sales milestones. PTC will share profits in the U.S. on a 40/60 basis and receive tiered double-digit royalties on ex-U.S. sales.

Novartis will take over global development, manufacturing, and commercialization responsibilities after completing the ongoing PIVOT-HD study's placebo-controlled portion, expected in H1 2025. Interim results from June 2024 showed PTC518 achieved durable, dose-dependent reduction in HTT protein levels and demonstrated favorable safety profiles.

PTC Therapeutics (NASDAQ: PTCT) has announced an agreement to sell its Rare Pediatric Disease Priority Review Voucher (PRV) for $150 million. The company received the PRV on November 13, 2024, alongside FDA approval of KEBILIDI™ for treating AADC deficiency in children and adults. The transaction's completion is subject to standard closing conditions, including the expiration of the HSR Antitrust Improvements Act waiting period.

PTC Therapeutics announced that its global Phase 2 placebo-controlled CardinALS study for utreloxastat in ALS patients failed to meet its primary and secondary efficacy endpoints. While the study showed a modest numerical benefit on the primary endpoint and correlation with lowering plasma neurofilament light chain (NfL), statistical significance was not achieved (p= 0.52). The drug was safe and well-tolerated, but due to lack of efficacy and biomarker signal, further development has been discontinued.

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.