Welcome to our dedicated page for Ptc Therapeutics news (Ticker: PTCT), a resource for investors and traders seeking the latest updates and insights on Ptc Therapeutics stock.
PTC Therapeutics, Inc. (NASDAQ: PTCT) is a global biopharmaceutical company focused on clinically differentiated medicines for children and adults living with rare disorders. The PTCT news feed on Stock Titan aggregates company announcements, financial updates and regulatory milestones that PTC discloses through press releases and SEC filings.
Investors following PTCT news can see updates on the commercial performance and global rollout of products such as Sephience (sepiapterin) for phenylketonuria (PKU), as well as revenue contributions from the company’s Duchenne muscular dystrophy franchise, which includes Translarna (ataluren) and Emflaza (deflazacort). News items also cover royalty revenue from Evrysdi (risdiplam) and changes in royalty arrangements, such as the sale of certain Evrysdi royalty rights to Royalty Pharma.
PTC Therapeutics regularly reports quarterly and annual financial results, including total revenue, product revenue, royalty and collaboration revenue, and details on GAAP and non-GAAP R&D and SG&A expenses. Corporate updates often highlight pipeline and regulatory developments, including FDA meetings for programs like votoplam in Huntington’s disease and vatiquinone in Friedreich’s ataxia, as well as ongoing review of the Translarna NDA.
The company also issues news about regulatory approvals and launches in different regions, such as Sephience approvals in the EU, US and Japan, and about R&D-focused events and investor conference presentations. In addition, PTC discloses equity inducement grants under Nasdaq Listing Rule 5635(c)(4), illustrating its use of stock options and restricted stock units as part of employee compensation.
By monitoring the PTCT news page, readers can track how product launches, regulatory decisions, collaborations, royalty transactions and capital markets activities shape the company’s progress in the rare disease biopharmaceutical sector.
PTC Therapeutics reported a 2022 total revenue of approximately $710 million, showing over 30% year-over-year growth. The company projects 2023 total revenue guidance between $940 million and $1 billion. Significant corporate milestones include the approval of Upstaza™ for AADC deficiency and the initiation of several clinical trials. The unaudited net product revenue in 2022 reached $535 million, with guidance for DMD franchise revenue in 2023 between $545 million and $565 million.
PTC Therapeutics, based in South Plainfield, N.J., will present a company overview at the 41st Annual J.P. Morgan Healthcare Conference on January 9, 2023, at 7:30 a.m. PST/10:30 a.m. EST.
The presentation can be accessed via a live webcast on the company’s Investor section and will be archived for 30 days. PTC focuses on developing medicines for rare disorders, aiming to provide access to transformative treatments for patients with unmet medical needs.
PTC Therapeutics (PTCT) announced that the Brazilian Health Regulatory Agency (ANVISA) has approved Waylivra™ (volanesorsen) as the first treatment for familial partial lipodystrophy (FPL) in Brazil. This marks a significant milestone as it's the first global approval for Waylivra for this indication. The approval was based on the Phase 2/3 BROADEN study, which demonstrated an 88% mean reduction in triglyceride levels for patients treated with Waylivra at three months, compared to only 22% for the placebo group. Waylivra is also approved for Familial Chylomicronemia Syndrome (FCS) in Brazil.
PTC Therapeutics has received authorization from the Medicines and Healthcare Products Regulatory Agency (MHRA) in the UK for Upstaza (eladocagene exuparvovec), the first gene therapy aimed at treating AADC deficiency. This rare disorder severely impacts motor development, posing life-threatening challenges. Upstaza has demonstrated clinical efficacy, allowing patients to achieve significant motor and cognitive milestones after treatment. This authorization follows a similar approval in the European Union, furthering PTC's commitment to innovative therapies for rare diseases.
PTC Therapeutics reported a 57% year-over-year revenue growth with total quarterly revenue of $217 million for Q3 2022. Key products: DMD revenue reached $131 million, with Translarna at $77 million and Emflaza at $55 million. The company secured strategic financing from Blackstone for up to $1 billion to support its pipeline. Despite growth, PTC recorded a net loss of $109.3 million and a significant decrease in cash equivalents to $288.4 million since 2021. Full-year revenue guidance increased to $710-$750 million.
PTC Therapeutics has entered a strategic financing collaboration with Blackstone, securing up to $500 million in funding to enhance its product pipeline and business development. This includes $350 million in low-cost capital at the close. The funds will help accelerate PTC's mission to deliver transformative medicines, with a goal of launching one therapy every 2-3 years. Blackstone's investment will be collateralized by PTC's existing products, including Translarna™ and Upstaza®. The collaboration aims to optimize capital structure and support new medicines.
PTC Therapeutics (NASDAQ: PTCT) has confirmed ongoing enrollment for its Phase 2 PIVOT-HD trial of PTC518, aimed at treating Huntington's disease. The 12-month study has received approval in several European countries and Australia, focusing on two dose levels: 5mg and 10mg. Enrollment began in the US but has paused due to FDA requests for additional data. There have been no treatment-related adverse events reported to date. PTC plans to share data from the initial 12-week study phase within the first half of 2023.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call on October 27, 2022, at 4:30 p.m. ET to discuss its third quarter 2022 financial results and business updates. Participants can register for the call online to receive dial-in details and are advised to join 15 minutes early. A replay will be available approximately two hours after the call and can be accessed on the company's website for 30 days. PTC is focused on developing medicines for rare disorders, leveraging its scientific expertise and global commercial capabilities.
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced it will present new data on Translarna™ (ataluren) at the 27th International Annual Congress of the World Muscle Society in Halifax, Nova Scotia, on October 12-14, 2022. Key presentations will highlight updated demographics and safety data, age at loss of ambulation, pulmonary function, and the efficacy of ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD). Translarna is currently licensed in the European Economic Area for nmDMD treatment in ambulatory patients aged 2 and older, while being investigational in the U.S.
PTC Therapeutics (NASDAQ: PTCT) announced the recipients of its 2022 STRIVE Awards, supporting Duchenne Muscular Dystrophy initiatives. The program grants funds to four organizations: ACDM (Brazil) for educational rehabilitation, KASDER (Turkey) for expanding psychological services, MDA Hellas (Greece) for a film on sexuality, and Action Duchenne (U.K.) for skills training. This year, PTC received 40 applications, highlighting diverse needs in the Duchenne community. The STRIVE Awards enhance awareness and support for patients, aiming to provide a broader impact across four countries.