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PTC Therapeutics (PTCT) provides critical updates through this centralized news resource for investors and industry stakeholders. Track official announcements spanning clinical trial progress, regulatory milestones, and strategic partnerships in rare disease therapeutics.
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• Clinical trial results for neuromuscular and oncology programs
• Regulatory filings and orphan drug designations
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• Financial performance and earnings communications
Bookmark this page for direct access to PTCT's evolving developments in post-transcriptional control therapeutics. Monitor breakthrough innovations addressing Duchenne muscular dystrophy, genetic disorders, and underserved patient populations.
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.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call on October 29, 2020, at 4:30 p.m. (ET), to report third quarter 2020 financial results and update its business outlook. Investors can access the call by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) with the passcode 7096445. A live webcast will also be available on the company’s website and a replay will be archived for 30 days. PTC focuses on developing medicines for rare disorders and aims to provide access to innovative treatments.
On September 28, 2020, PTC Therapeutics announced positive two-year data from the FIREFISH study on Evrysdi™ (risdiplam) for treating spinal muscular atrophy (SMA). The study indicated that 88% of infants were alive without permanent ventilation after two years. Significant motor milestones were achieved, including increased independent sitting and head control. The treatment's safety profile remained consistent, with no new safety signals. Evrysdi™ was recently approved and shows a strong commercial launch, highlighting its importance amid the ongoing pandemic.
PTC Therapeutics announced on September 21, 2020, the approval of stock options for 75,670 shares and 12,845 restricted stock units (RSUs) to 22 new employees, as part of their employment compensation. These inducement grants comply with NASDAQ's listing rules and aim to attract new talent. The options have an exercise price of $45.46 per share, with a 10-year term and a four-year vesting schedule. The RSUs will also vest over four years. PTC focuses on developing innovative treatments for rare disorders, driving their investment in a strong pipeline of medicines.
PTC Therapeutics, Inc. (NASDAQ: PTCT) will present a company overview on October 1, 2020, at the Jefferies Virtual Gene Therapy/Editing Summit at 9:00 a.m. ET. The presentation will be live-streamed on their website and available for 30 days afterward. PTC Therapeutics focuses on developing medicines for rare disorders, aiming to provide access to innovative treatments for patients with unmet needs. For optimal viewing, it's recommended to connect a few minutes before the start of the webcast.
PTC Therapeutics (NASDAQ: PTCT) announced the winners of its sixth annual STRIVE Awards, recognizing initiatives aimed at supporting the Duchenne muscular dystrophy community. Awarded projects include an online platform in Argentina, events in Hungary connecting families with healthcare professionals, a support program in Russia, and a student ambassador program in the U.S. These initiatives aim to enhance community connections and awareness during challenging times. Mary Frances Harmon emphasized the importance of patient advocacy amid the ongoing challenges faced by rare disease patients.
PTC Therapeutics (NASDAQ: PTCT) will present a company overview at the Cantor Virtual Global Healthcare Conference on September 17, 2020, at 9:20 a.m. ET. The presentation will be available via live webcast on the PTC Therapeutics website, with an archive accessible for 30 days post-event. PTC focuses on developing innovative treatments for rare disorders, driving investment through a robust pipeline aimed at addressing unmet medical needs.
PTC Therapeutics announced a significant $20 million milestone payment from Roche triggered by the first commercial sale of Evrysdi™ (risdiplam) in the U.S. The FDA approved Evrysdi™ on August 7, 2020, for treating spinal muscular atrophy (SMA) in individuals 2 months and older. This oral treatment can be administered at home, enhancing patient accessibility during the COVID-19 pandemic. Evrysdi™ aims to improve survival motor neuron protein levels, addressing a critical unmet need in SMA care.
On August 18, 2020, PTC Therapeutics (NASDAQ: PTCT) approved non-statutory stock options for 49,165 shares and 14,175 restricted stock units (RSUs) to five new employees as part of their compensation. The stock options have an exercise price of $49.13 and vest over four years, rewarding 25% after the first year, with additional vesting every three months. The RSUs will vest similarly over four years. These inducement grants comply with NASDAQ Listing Rule 5635(c)(4) and are aimed at enhancing the company's talent acquisition strategy.
PTC Therapeutics (NASDAQ: PTCT) announced the European Medicines Agency's acceptance of its Marketing Authorization Application for Evrysdi™ (risdiplam) to treat spinal muscular atrophy (SMA). This follows the PRIME designation granted by the EMA, facilitating accelerated review. The milestone triggers a $15 million payment from Roche to PTC. Evrysdi™ has shown significant clinical efficacy and safety in trials, including improvements in motor functions. The FDA previously approved the drug for patients aged 2 months and older. This progress aims to broaden access for SMA patients globally.