PTC Therapeutics, Inc. - PTCT STOCK NEWS

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.

On March 15, 2021, PTC Therapeutics announced results from a real-world study involving 92 patients with Duchenne and Becker muscular dystrophy who switched from prednisone to EMFLAZA (deflazacort) following FDA approval. The study indicated that most patients experienced improvement in disease progression after switching, with 95% of DMD and 90% of BMD patients reporting effective results. The study emphasized the need for better disease management and the potential of EMFLAZA in altering the natural history of DMD, reflecting PTC's commitment to offering differentiated treatments.

The SMA Foundation and PTC Therapeutics (NASDAQ: PTCT) have expanded their collaboration to advance research in regenerative medicine for Spinal Muscular Atrophy (SMA) and related neuromuscular disorders. The partnership aims to fund academic institutions to develop new treatments, filling a therapeutic gap in SMA care. This initiative builds on their successful history, including the recent approval of Evrysdi for SMA. SMA is a serious genetic disorder affecting 1 in 11,000 babies, leading to muscle atrophy and loss of function.