Ptc Therapeutics Stock Price, News & Analysis

- 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

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.

PTC Therapeutics (NASDAQ: PTCT) has received a positive opinion from the European Medicines Agency's CHMP for Sephience™ (sepiapterin), a treatment for phenylketonuria (PKU) in children and adults. The approval recommendation includes a comprehensive label covering all age groups and disease severities.

The company plans to launch immediately after European Commission ratification, expected in approximately two months. The authorization will apply to all 27 EU member states plus Iceland, Norway, and Liechtenstein, with initial focus on Germany and key European markets offering immediate named patient access.

Additionally, Sephience is under review by the FDA with a PDUFA date of July 29, 2025, and approval applications are being processed in Japan and Brazil.

PTC Therapeutics (NASDAQ: PTCT) has announced its participation in four major healthcare investor conferences in May and June 2025. The schedule includes:

• Bank of America Securities Healthcare Conference on May 13 at 1:00 p.m. ET
• RBC Capital Markets Global Healthcare Conference on May 20 at 2:05 p.m. ET
• William Blair 45th Annual Growth Stock Conference on June 3 at 10:20 a.m. ET
• Goldman Sachs 46th Annual Global Healthcare Conference on June 10 at 9:20 a.m. ET

All presentations will be available via live 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 start time.

PTC Therapeutics (NASDAQ: PTCT) has announced a webcast conference call scheduled for Tuesday, May 6, at 4:30 p.m. ET. The call will present the company's first quarter 2025 financial results along with updates on business operations and future outlook.

Participants are advised to register in advance for dial-in details and join 15 minutes before the start time 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 (NASDAQ: PTCT) has announced the approval of equity compensation packages for 35 new employees on March 24, 2025. The compensation includes 12,000 non-statutory stock options and 28,220 restricted stock units (RSUs).

The stock options were granted at an exercise price of $57.03 per share, matching PTC's closing price on the grant date. These options have a 10-year term and vest over four years, with 25% vesting after the first year and 6.25% quarterly thereafter. The RSUs also follow a four-year vesting schedule, with 25% vesting annually.

These inducement grants, approved by PTC's Compensation Committee, comply with Nasdaq Listing Rule 5635(c)(4) and are part of the new employees' compensation packages.

PTC Therapeutics (NASDAQ: PTCT) announced that the European Commission (EC) has adopted the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) opinion to not renew the authorization of Translarna™ (ataluren) for treating nonsense mutation Duchenne muscular dystrophy.

This decision effectively removes the drug's conditional marketing authorization in the European Economic Area. However, the EC indicated that individual EU countries can use Articles 117(3) and 5(1) of the EU Directive 2001/83 to allow continued Translarna use. PTC Therapeutics plans to work on a country-by-country basis to provide commercial drug where possible.