Ptc Therapeutics Stock Price, News & Analysis

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.

PTC Therapeutics (NASDAQ: PTCT) announced the launch of a Phase 2/3 clinical trial for vatiquinone (PTC743) aimed at treating mitochondrial epilepsy, a severe condition affecting children with inherited mitochondrial diseases. With approximately 11,000-13,000 potential patients across the US, EU, Japan, and Latin America, the trial is critical as it addresses a significant unmet medical need. Vatiquinone has shown promise in earlier studies, impacting seizure frequency and related morbidity in over 500 patients. The trial's primary endpoint focuses on reducing seizure frequency in a controlled setting.

PTC Therapeutics, along with the AADC Family Network, has announced October 23 as the first AADC Deficiency Awareness Day in Massachusetts. This designation aims to raise awareness about Aromatic L-amino Acid Decarboxylase (AADC) deficiency, a rare genetic disorder that significantly impacts affected children. To commemorate the day, PTC will host a Facebook Live event featuring industry experts, including Kelly Heger and Matthew Klein. The initiative highlights the ongoing efforts towards finding treatment options for this condition, which currently has no approved therapies.

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced that its drug, Evrysdi™ (risdiplam), received approval from Brazil's ANVISA for spinal muscular atrophy (SMA), marking its second country approval. Additionally, PTC filed a New Drug Application in Japan, which triggers a $7.5 million milestone payment from Roche. Evrysdi™ has been submitted for approval in 15 other markets and is undergoing reviews in four health authorities. The drug offers a new treatment option for SMA, which affects 1 in 10,000 babies. Its oral formulation is significant for patient accessibility during the COVID-19 pandemic.