Ptc Therapeutics Stock Price, News & Analysis

PTC Therapeutics (NASDAQ: PTCT) has received a Complete Response Letter (CRL) from the FDA regarding its New Drug Application (NDA) for vatiquinone, intended to treat Friedreich's ataxia in children and adults.

The FDA determined that substantial evidence of efficacy was not demonstrated for vatiquinone and stated that an additional adequate and well-controlled study would be required to support NDA resubmission. CEO Matthew B. Klein expressed disappointment with the FDA's decision and indicated the company plans to meet with the FDA to address the issues raised in the CRL.

PTC Therapeutics (NASDAQ:PTCT) reported Q2 2025 financial results and corporate updates. The quarter was marked by the European and FDA approval of Sephience™ for PKU treatment with broad labeling. Total revenue reached $178.9 million, including $96 million from the DMD franchise. The company reported a net loss of $64.8 million, improved from $99.2 million in Q2 2024.

Key highlights include cash position of $1.99 billion, positive Phase 2 PIVOT-HD study results for votoplam in Huntington's Disease, and ongoing NDA reviews for vatiquinone and Translarna. PTC updated its full-year 2025 revenue guidance to $650-800 million.

The company entered an agreement to purchase Sephience's annual sales obligation for $225 million upfront plus future milestone payments.

PTC Therapeutics (NASDAQ: PTCT) has received FDA approval for SEPHIENCE™ (sepiapterin), a treatment for phenylketonuria (PKU) in patients aged 1 month and older. The approval covers broad labeling for hyperphenylalaninemia (HPA) in sepiapterin-responsive PKU patients.

The approval is supported by significant efficacy and safety data from the Phase 3 APHENITY trial and its long-term extension study. SEPHIENCE has already received European Commission marketing authorization, with approval applications under review in Japan and Brazil.

The company aims to position SEPHIENCE as the future standard of care for PKU patients, with their experienced teams ready to launch the therapy in the United States.

PTC Therapeutics (NASDAQ: PTCT) has scheduled its second quarter 2025 financial results conference call for Thursday, August 7, at 4:30 p.m. ET. The presentation will include financial results and 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 presentation.

PTC Therapeutics (NASDAQ: PTCT) has received European Commission marketing authorization for Sephience (sepiapterin), a treatment for phenylketonuria (PKU) in both children and adults. The approval includes a comprehensive label covering all age groups and disease severities, positioning Sephience to potentially become the new standard of care. The authorization is supported by significant results from the Phase 3 APHENITY trial and its extension study, which demonstrated durable treatment effects and dietary improvements. The approval covers all 27 EU member states plus Iceland, Norway, and Liechtenstein, with the initial launch planned in Germany in early July. Additionally, an FDA decision is expected by July 29, 2025, while applications are under review in Japan and Brazil.

PTC Therapeutics announced the approval of equity compensation awards for 34 new employees on June 16, 2025. The inducement grants include 12,725 non-statutory stock options with an exercise price of $51.87 per share and 27,790 restricted stock units (RSUs). The stock options have a 10-year term and vest over 4 years, with 25% vesting after the first year and 6.25% quarterly thereafter. The RSUs also vest over 4 years with 25% vesting annually. These awards were granted under Nasdaq Listing Rule 5635(c)(4) as part of the new employees' compensation packages and were approved by PTC's Compensation Committee.

PTC Therapeutics (NASDAQ: PTCT) reported Q1 2025 financial results with total revenue of $190 million. The company achieved a net income of $866.6 million, compared to a net loss of $91.6 million in Q1 2024, largely due to a $986.2 million collaboration agreement with Novartis. Key highlights include:

- DMD franchise revenue of $134 million (Translarna: $86M, Emflaza: $48M)

- Strong cash position of $2.0 billion as of March 31, 2025

- Positive CHMP opinion for Sephience™ in April 2025, with FDA decision expected July 29, 2025

- Updated 2025 revenue guidance to $650-800 million

The company received positive regulatory progress for multiple products, including Sephience for PKU patients and Vatiquinone for Friedreich's ataxia. PTC518's Phase 2 PIVOT-HD study met its primary endpoint with promising results.

PTC Therapeutics (NASDAQ: PTCT) announced positive results from their Phase 2 PIVOT-HD study of PTC518 (votoplam) in Huntington's disease patients. The study successfully met its primary endpoint, demonstrating significant dose-dependent reduction in blood Huntingtin protein levels at Week 12 (p<0.0001).

Key findings include:

• Blood HTT reduction of 23% at 5mg dose and 36-39% at 10mg dose across Stage 2 and 3 patients
• Dose-dependent clinical benefits in Stage 2 patients at 12 months
• 24-month data showed favorable trends in clinical scales compared to natural history
• Dose-dependent plasma NfL reduction of 8.9% (5mg) and 14% (10mg) at 24 months
• Favorable safety profile with no treatment-related serious adverse events

PTC Therapeutics (NASDAQ: PTCT) has approved equity compensation packages for 12 new employees as part of their employment agreements. The inducement grants, approved on April 25, 2025, include:

- 7,000 non-statutory stock options with an exercise price of $49.53 per share (closing price on grant date)

- 10,780 restricted stock units (RSUs)

The stock options have a 10-year term and vest over 4 years, with 25% vesting after the first year and 6.25% quarterly thereafter. RSUs vest annually over 4 years at 25% per year. Both are subject to continued employment. These grants were made under Nasdaq Listing Rule 5635(c)(4) as inducement material to employment.