Ptc Therapeutics Stock Price, News & Analysis

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.

PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call on July 29, 2021, at 4:30 p.m. (ET) to report its second quarter 2021 financial results and update on business outlook. Interested participants can access the call by dialing (877) 303-9216 domestically or (973) 935-8152 internationally. The call will also be available via a live webcast on the company's website, with a replay accessible two hours post-call. The event underscores PTC's dedication to providing updates on its pipeline and business strategy focused on rare disorders.

PTC Therapeutics (NASDAQ: PTCT) released new data confirming the clinical benefits of EMFLAZA® (deflazacort) for Duchenne muscular dystrophy (DMD) patients over prednisone. The pooled results showed significant improvement in physical performance metrics, including the six-minute walk test and the North Star Ambulatory Assessment. Experts emphasized the advantages of early EMFLAZA initiation for better patient outcomes. The data also highlighted real-world evidence of EMFLAZA's effectiveness and its role in slowing disease progression in DMD patients.

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced the approval of Evrysdi™ (risdiplam) in Japan for treating spinal muscular atrophy (SMA). This approval, based on pivotal studies in infants and adults, will trigger a $10M milestone payment from Roche. Evrysdi provides an at-home oral treatment option, enhancing accessibility for patients. It has previously gained FDA and EMA approvals for patients aged 2 months and older. SMA, a severe neuromuscular disease, affects approximately 1 in 10,000 babies, making this development significant for patient care.

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced on June 11, 2021, the approval of non-statutory stock options for 118,350 shares and 30,835 restricted stock units (RSUs) to 66 new employees. These grants are part of the employment compensation to attract talent under NASDAQ's inducement grant exception. The stock options have an exercise price of $42.95 and vest over four years, while RSUs vest annually. This initiative aims to enhance PTC's workforce as it focuses on developing innovative treatments for rare disorders, supporting its growth strategy and commitment to patient care.

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced positive interim results from the RAINBOWFISH trial, showing that pre-symptomatic infants with spinal muscular atrophy (SMA) achieved significant motor milestones after treatment with Evrysdi. These milestones include sitting, rolling, crawling, standing, and walking independently. Additionally, interim results from the JEWELFISH safety study show stabilization in motor function and increased SMN protein levels. Evrysdi is FDA-approved for SMA patients aged 2 months and older and is marketed by Genentech.

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced the election of Mary L. Smith to its Board of Directors on June 9, 2021. Smith brings extensive experience from her role as Principal Deputy Director at the Indian Health Service, where she oversaw a $6 billion healthcare division. Currently, she serves as Vice Chair at VENG Group, advising on business strategy and healthcare governance. Smith aims to leverage her background to support PTC's mission in advancing its pipeline of medicines for rare diseases. CEO Stuart W. Peltz emphasized her valuable insights for the company's growth.

PTC Therapeutics announced that the FDA and European Commission have granted Orphan Drug Designation (ODD) for PTC923, aimed at treating hyperphenylalaninemia, primarily caused by Phenylketonuria (PKU). PKU can lead to severe cognitive disabilities and seizures. PTC923, an oral synthetic sepiapterin, is expected to enter a Phase 3 trial, APHENITY, in mid-2021. ODD provides various benefits, including grant funding, tax advantages, and potential market exclusivity in both the U.S. and EU.