Ptc Therapeutics Stock Price, News & Analysis

PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call on May 4, 2021, at 4:30 p.m. (ET) to discuss its first quarter 2021 financial results. Investors can access the call by dialing (877) 303-9216 domestically or (973) 935-8152 internationally. The call will be available for replay on the company’s website for 30 days after the event. PTC emphasizes its focus on developing differentiated medicines for rare disorders, reinforcing its commitment to providing best-in-class treatments for patients with unmet medical needs.

PTC Therapeutics (NASDAQ: PTCT) has granted stock options for 75,085 shares and 33,740 restricted stock units (RSUs) to 79 new employees as a part of their compensation package. Approved on April 13, 2021, these inducement grants comply with NASDAQ Listing Rule 5635(c)(4). The options have an exercise price of $47.31, based on the stock's closing price on the grant date. The vesting period spans four years, with a portion vesting annually and quarterly, contingent on continued employment.

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced positive results from Part 2 of the pivotal FIREFISH trial for Evrysdi™ (risdiplam) in infants with type 1 spinal muscular atrophy (SMA). After 24 months, 93% of infants were alive, with 83% surviving without permanent ventilation. Key motor milestones improved significantly, such as sitting without support (from 29% at 1 year to 61% at 2 years). The treatment was associated with fewer hospitalizations, and safety profiles remained consistent. Evrysdi promotes SMN protein production, crucial for healthy motor neuron function.

PTC Therapeutics (NASDAQ: PTCT) has launched the PTC Pinpoint Direct™-CP Spectrum, a no-charge genetic testing program for individuals in the U.S. with symptoms of cerebral palsy (CP) but without evidence of brain injury. This initiative aims to reduce misdiagnosis and connect patients with potential genetic disorders, which may have existing treatments. The program collaborates with Genome Medical for genetic counseling and Invitae for genetic testing, enhancing patient access to care and eliminating unnecessary treatments.

PTC Therapeutics, Inc. (NASDAQ: PTCT) has been awarded the 2021 Don Clifton Strengths-Based Culture Award by Gallup. This recognition honors organizations that excel in developing engaging workplace cultures focused on employee strengths. CEO Stuart Peltz emphasized the company's commitment to a collaborative environment, especially amid challenges posed by the COVID-19 pandemic. PTC is dedicated to discovering and commercializing innovative medicines for rare disorders, leveraging its scientific expertise and robust global commercial infrastructure.

PTC Therapeutics (NASDAQ: PTCT) will host a webinar on April 15 at 9 a.m. E.T. discussing its PTC518 Huntington disease (HD) program. The webinar will cover PTC's splicing platform and showcase the ongoing Phase 1 clinical trial of PTC518. Preliminary results indicate that PTC518 effectively reduces Huntingtin protein levels in both brain and blood. Participants can join via phone or a live webcast on the PTC website, with a replay available post-event. Huntington disease affects about 45,000 people in the U.S., primarily those aged 30-50.

PTC Therapeutics (NASDAQ: PTCT) has triggered a $20 million milestone payment due to the first commercial sale of Evrysdi™ (risdiplam) in the EU. The drug, approved by the European Medicines Agency on March 30, treats spinal muscular atrophy (SMA) in patients aged 2 months and older. Evrysdi is accessible in Germany and will soon be available in France. Currently approved in 38 countries, the drug's commercial prospects are bolstered by ongoing reimbursement negotiations. PTC reports Evrysdi shows efficacy in pivotal clinical trials, enhancing its market position.

PTC Therapeutics has received marketing authorization from the European Commission for Evrysdi™ (risdiplam), a treatment for spinal muscular atrophy (SMA) in patients aged 2 months and older. This approval, based on the FIREFISH and SUNFISH studies, covers SMA Type 1, 2, and 3 patients with one to four SMN2 copies. Evrysdi, which utilizes PTC’s innovative splicing technology, was previously granted PRIME and Orphan Drug Designations in Europe. Over 3,000 patients have been treated with Evrysdi, which is now approved in 38 countries and undergoing submissions in 33 more.

PTC Therapeutics (NASDAQ: PTCT) announced encouraging two-year data from the SUNFISH trial for Evrysdi™ (risdiplam) at the 2021 MDA Conference. Patients with Type 2 or Type 3 spinal muscular atrophy showed sustained or improved motor function after 24 months. Key measures like MFM32, RULM, and HFMSE indicated motor function stability and improvement. Adverse events decreased in the second year, with upper respiratory infections being the most common. Evrysdi aims to enhance SMN protein production, crucial for motor neuron health, and is marketed in the U.S. by Genentech.