Ptc Therapeutics Stock Price, News & Analysis

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.

PTC Therapeutics announced on August 17, 2021, the approval of inducement grants for 48 new employees, comprising 51,670 stock options and 19,755 restricted stock units (RSUs). The stock options have an exercise price of $39.23 per share, the closing price on the grant date, with a 10-year term and a vesting schedule over four years. RSUs will vest similarly over four years, contingent upon continued service. These grants comply with NASDAQ's inducement grant exception, enhancing PTC's capacity to attract talent critical for its growth.

PTC Therapeutics, Inc. (PTCT) reported a strong second quarter for 2021, achieving total revenues of $116.7 million, a growth of 55% year-over-year. The DMD franchise generated $102 million in net product revenue, reflecting a 36% increase. R&D expenses decreased to $125.5 million, while the net loss narrowed to $118.4 million. With cash reserves at $947.1 million, PTC raised its revenue guidance for the DMD franchise to between $370 and $390 million for the full year. The company has several ongoing clinical trials, including the initiation of a Phase 3 trial for PKU planned for Q3 2021.

PTC Therapeutics (NASDAQ: PTCT) announced the publication of results from Part 2 of the FIREFISH study in the New England Journal of Medicine. The trial demonstrated that infants with symptomatic Type 1 spinal muscular atrophy (SMA) treated with Evrysdi™ showed notable enhancements in survival and motor function compared to historical controls. Key findings include a 90% increase in CHOP-INTEND scores and 85% event-free survival after 12 months. PTC's splicing platform plays a crucial role in developing innovative therapies for SMA.