Ptc Therapeutics Stock Price, News & Analysis

PTC Therapeutics announced that Tegsedi^® (inotersen) has received Category 1 classification from Brazil's CMED, allowing for pricing aligned with international standards. Approved by ANVISA for treating hATTR amyloidosis, Tegsedi is the first antisense medicine available in Brazil and is expected to improve the quality of life for approximately 5,000 patients in the country. The drug has shown significant efficacy in clinical studies, enhancing patient outcomes while enabling self-administration.

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced the approval of non-statutory stock options for 51,400 shares and 18,790 restricted stock units (RSUs) on Sept. 22, 2021, for 47 new employees. The stock options have an exercise price of $39.29, equal to the closing stock price on Sept. 24, 2021. The options vest over four years, with 25% vesting on the first anniversary. The RSUs also vest over four years with annual vesting. This is part of the company's strategy to attract talent in alignment with NASDAQ Listing Rule 5635(c)(4).

PTC Therapeutics, Inc. (NASDAQ: PTCT) presented five-year results on its gene therapy, PTC-AADC, showing significant improvements in children with AADC deficiency, a severe neurological disorder. Treated children displayed enhancements in motor and cognitive skills, with many achieving milestones previously unattainable. A notable reduction in respiratory infections from 2.4 episodes annually to 0.3 was reported. The therapy is under review by the European Medicines Agency and a Biologics License Application is expected for submission to the U.S. FDA by year-end 2021.

PTC Therapeutics (NASDAQ: PTCT) will host a conference call on September 23, 2021, at 8:00 a.m. ET to discuss positive results from the PTC518 Phase 1 study in healthy volunteers. Key findings include significant lowering of HTT mRNA and protein levels, confirming PTC518's capacity to penetrate the blood-brain barrier. The company also plans to initiate the Phase 2 study in Huntington's disease patients by year-end. Currently, there are no approved therapies addressing this debilitating condition.

PTC Therapeutics (NASDAQ: PTCT) presented real-world findings from the STRIDE registry, revealing that Translarna (ataluren) delays loss of ambulation by over five years in boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) compared to standard care. The analysis demonstrated a median age of 17.9 years for loss of ambulation in treated boys, versus 12.5 years in those receiving standard care. Additionally, lung function decline was delayed by 1.8 years. The safety profile remains consistent with previous data, emphasizing Translarna's significant impact on enhancing patients' quality of life.

PTC Therapeutics (NASDAQ: PTCT) announced the recipients of its 2021 STRIVE Awards, supporting initiatives for the Duchenne muscular dystrophy community. This year marks the highest number of applications, with 58 submissions. Grants will fund projects aimed at enhancing independent living for those with Duchenne, including a residential model in Brazil, a free physiotherapy clinic, and resources for transitioning to adulthood in the UK. The STRIVE Awards program, initiated in 2015, has supported 35 projects to date, focusing on awareness, education, and patient advocacy.

PTC Therapeutics (NASDAQ: PTCT) announced that Brazil's ANVISA has approved Waylivra™ (volanesorsen) as the first treatment for familial chylomicronemia syndrome (FCS). This approval is based on results from the Phase 3 APPROACH study, which demonstrated a 94% reduction in triglycerides compared to placebo. Waylivra is administered via a subcutaneous injection and targets apolipoprotein C-III production to reduce triglyceride levels, potentially decreasing the risk of pancreatitis. This represents a significant advancement for FCS patients in Brazil, who have limited treatment options.

PTC Therapeutics (NASDAQ: PTCT) has published a manuscript detailing a groundbreaking gene therapy approach for treating AADC deficiency. The study, featured in the European Molecular Biology Organization Journal, highlights the successful infusion of PTC-AADC into the putamen, showing significant improvements in motor skills and quality of life for affected children. This innovative therapy aims to address the unmet need in treating AADC deficiency, which has no current disease-modifying treatments. The therapy is under review by the European Medicines Agency, with a decision expected in Q4 2021.