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 (NASDAQ: PTCT) will present a company overview at the Cantor Virtual Global Healthcare Conference on September 17, 2020, at 9:20 a.m. ET. The presentation will be available via live webcast on the PTC Therapeutics website, with an archive accessible for 30 days post-event. PTC focuses on developing innovative treatments for rare disorders, driving investment through a robust pipeline aimed at addressing unmet medical needs.
PTC Therapeutics announced a significant $20 million milestone payment from Roche triggered by the first commercial sale of Evrysdi™ (risdiplam) in the U.S. The FDA approved Evrysdi™ on August 7, 2020, for treating spinal muscular atrophy (SMA) in individuals 2 months and older. This oral treatment can be administered at home, enhancing patient accessibility during the COVID-19 pandemic. Evrysdi™ aims to improve survival motor neuron protein levels, addressing a critical unmet need in SMA care.
On August 18, 2020, PTC Therapeutics (NASDAQ: PTCT) approved non-statutory stock options for 49,165 shares and 14,175 restricted stock units (RSUs) to five new employees as part of their compensation. The stock options have an exercise price of $49.13 and vest over four years, rewarding 25% after the first year, with additional vesting every three months. The RSUs will vest similarly over four years. These inducement grants comply with NASDAQ Listing Rule 5635(c)(4) and are aimed at enhancing the company's talent acquisition strategy.
PTC Therapeutics (NASDAQ: PTCT) announced the European Medicines Agency's acceptance of its Marketing Authorization Application for Evrysdi™ (risdiplam) to treat spinal muscular atrophy (SMA). This follows the PRIME designation granted by the EMA, facilitating accelerated review. The milestone triggers a $15 million payment from Roche to PTC. Evrysdi™ has shown significant clinical efficacy and safety in trials, including improvements in motor functions. The FDA previously approved the drug for patients aged 2 months and older. This progress aims to broaden access for SMA patients globally.
PTC Therapeutics, Inc. announced FDA approval for Evrysdi™ (risdiplam), the first at-home treatment for spinal muscular atrophy (SMA) in patients aged 2 months and older. Evrysdi demonstrated significant improvements in motor function and survival rates across two pivotal trials, involving over 450 patients. Evrysdi will be launched in the U.S. within two weeks, with Genentech marketing the product. The approval builds on a strong clinical program showcasing its efficacy and safety, making it a notable advancement in SMA treatment.
PTC Therapeutics, based in South Plainfield, N.J., announced that Mary Frances Harmon, its Senior Vice President of Corporate and Patient Relations, has been named one of the most inspiring leaders in life sciences by PharmaVOICE magazine. Harmon is recognized for her dedication to improving patient lives and her work in establishing relationships with patients and advocacy groups. With over 30 years in the pharmaceutical industry, she has initiated programs like the STRIVE awards to support Duchenne muscular dystrophy patients. PTC continues to emphasize its commitment to patient-focused initiatives.
PTC Therapeutics (PTCT) reported Q2 2020 revenues of $75.2 million, down from $85.5 million YoY, driven by a decrease in Translarna sales.
However, Emflaza experienced a growth of over 30% YoY. R&D expenses surged to $176.5 million, including one-time charges of $53.6 million from Censa Pharmaceuticals acquisition.
Despite a net loss of $181.4 million, PTC holds over $1 billion in cash, positioning it well for future developments.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a conference call on August 5, 2020, at 4:30 p.m. ET to report its Q2 2020 financial results and business outlook. Investors can join the call by dialing (877) 303-9216 domestically or (973) 935-8152 internationally and using passcode 5897698. A live webcast will also be available on the company's website. PTC focuses on developing and commercializing innovative medicines for rare disorders and aims to provide access to transformative treatments for patients with unmet medical needs.
PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that the European Medicines Agency's CHMP recommended removing the statement regarding the lack of efficacy in non-ambulatory patients from the SmPC for Translarna™ (ataluren). This change allows healthcare providers to make treatment decisions based on clinical judgment for non-ambulatory patients. The recommendation may facilitate reimbursement for these patients. Translarna, which treats Duchenne muscular dystrophy caused by nonsense mutations, has demonstrated potential benefits in slowing disease progression and preserving lung function.
PTC Therapeutics, Inc. (NASDAQ: PTCT) has received FDA authorization to initiate a Phase 2/3 clinical trial for PTC299, a dihydroorotate dehydrogenase (DHODH) inhibitor, aimed at treating COVID-19. The trial, starting imminently in the U.S. and expanding globally, will evaluate the drug's efficacy in reducing SARS-CoV-2 replication and managing inflammation associated with COVID-19. The study will involve around 340 patients, measuring clinical efficacy against a placebo through respiratory improvement. PTC299 is also under investigation for oncology applications, having shown promising results in prior trials.