Ptc Therapeutics Stock Price, News & Analysis

PTC Therapeutics announced that Translarna (ataluren) has received marketing approval in the Russian Federation for treating nonsense mutation Duchenne muscular dystrophy (nmDMD), impacting an estimated 13% of DMD patients. This approval is significant as Translarna is the only medicine targeted at the underlying cause of nmDMD in approved regions. CEO Stuart W. Peltz highlighted the demand from Russian physicians and the identification of nmDMD patients who may benefit. This marks an important milestone in PTC's goal to globally provide access to this treatment.

PTC Therapeutics (NASDAQ: PTCT) has initiated the registration-directed Phase 3 MOVE-FA study for vatiquinone (PTC743) in children and young adults with Friedreich ataxia (FA), a rare neurodegenerative disorder. This pivotal trial comes after vatiquinone showed positive results in a Phase 2 trial, impacting disease severity over 24 months. The trial will enroll approximately 110 participants across multiple regions, with primary endpoints focused on the modified Friedreich ataxia rating scale. The FDA has granted vatiquinone both Orphan Drug and Fast Track Designations.

PTC Therapeutics (NASDAQ: PTCT) will present at the Evercore ISI 3rd Annual HealthCONx Conference on December 1, 2020, at 11:45 a.m. ET. This presentation, featuring a company overview, will be available via live webcast on PTC's website, with an archived version accessible for 30 days. PTC Therapeutics is a global biopharmaceutical company specializing in innovative treatments for rare disorders, emphasizing its commitment to meeting unmet medical needs through a diversified pipeline of transformative medicines.

PTC Therapeutics has received multiple designations from the U.S. FDA for its drug PTC596, intended for treating rare cancers: leiomyosarcoma (LMS) and Diffuse Intrinsic Pontine Glioma (DIPG). These include Orphan Drug and Fast Track designations for LMS and both Orphan Drug and Rare Pediatric Disease designations for DIPG. PTC596, currently in clinical trials, shows potential in targeting and inhibiting cancer cell growth. Each year, around 4,000 LMS and 300 DIPG cases are diagnosed in the U.S., highlighting the critical need for effective treatments in these areas.

PTC Therapeutics (NASDAQ: PTCT) has initiated a Phase 1 clinical trial to evaluate PTC518 for treating Huntington's disease. PTC518, a small molecule designed to selectively reduce Huntingtin protein levels, shows promise with favorable preclinical results, demonstrating effective distribution throughout the central nervous system. The trial aims to establish safety and optimal dosing, with data anticipated in the first half of 2021. This milestone is pivotal in addressing the unmet medical needs of Huntington's disease patients, as no approved therapies currently exist.

PTC Therapeutics, Inc. (NASDAQ: PTCT) has presented data at the Virtual ISPOR Europe 2020 Conference on two severe childhood genetic disorders: Aromatic L-Amino acid Decarboxylase deficiency (AADC-d) and Duchenne muscular dystrophy caused by nonsense mutations (nmDMD). The findings highlight the significant health burdens of these conditions and the urgent need for effective therapies. PTC's gene therapy, PTC-AADC, is under review by the EMA, with a decision expected in the first half of 2021. Additionally, a qualitative study on nmDMD underscores the critical need for therapies that alleviate the comprehensive impacts on patients and caregivers.

PTC Therapeutics (NASDAQ: PTCT) reported a 66% increase in total revenue to $118.4 million for Q3 2020, driven by the launch of Evrysdi for spinal muscular atrophy. The company achieved significant milestones, including $35 million in collaboration revenue from Roche. However, net loss widened to $69.7 million, compared to $60 million last year, with R&D expenses rising to $93 million. Cash and equivalents increased to $1.14 billion. PTC aims to enhance long-term value through innovative therapies.

On October 28, 2020, PTC Therapeutics, Inc. (NASDAQ: PTCT) announced the approval of non-statutory stock options and restricted stock units (RSUs) for 18 new employees. A total of 18,610 stock options and 6,860 RSUs were granted as part of the employees' compensation. The stock options have an exercise price of $51.96 per share and a vesting schedule over four years. The inducement grants align with NASDAQ Listing Rule 5635(c)(4), established to attract new talent within the company.

PTC Therapeutics, trading under NASDAQ: PTCT, will present an overview at two upcoming virtual conferences. The Credit Suisse 29th Annual Virtual Healthcare Conference is scheduled for November 11, 2020, at 9:30 a.m. ET, and the Barclays Virtual Gene Editing & Gene Therapy Summit will occur on November 16, 2020, at 3:30 p.m. ET. These presentations will be streamed live and archived for two weeks on PTC's investor relations webpage. PTC focuses on developing innovative therapies for patients with rare disorders.