Ptc Therapeutics Stock Price, News & Analysis

PTC Therapeutics announces the election of Alethia Young to its Board of Directors. Young brings over 20 years of experience in healthcare and biotech equity research, currently serving as CFO of Graphite Bio. Previously, she held senior positions at Cantor Fitzgerald, Credit Suisse, and Deutsche Bank. Young expressed enthusiasm about PTC's diverse pipeline aimed at rare diseases, emphasizing her commitment to leveraging her background for the company's growth. CEO Stuart W. Peltz highlighted her industry knowledge as a valuable asset for PTC's future success.

PTC Therapeutics (NASDAQ: PTCT) will present a company overview at the William Blair 42nd Annual Growth Stock Conference on June 9, 2022, at 2:20 p.m. ET. The presentation will be accessible via live webcast on the company’s website, where it will also be archived for 30 days. PTC Therapeutics focuses on developing medicines for rare disorders and aims to provide best-in-class treatments for patients with unmet medical needs. For more details, visit www.ptcbio.com.

PTC Therapeutics announced encouraging preliminary results from its Phase 1B study of unesbulin (PTC596) for advanced leiomyosarcoma (LMS). The study showed an 18.2% objective response rate and a 51.5% disease control rate. Unesbulin was well tolerated, with a recommended Phase 2 dose established. Based on these promising results, PTC has initiated the SUNRISE LMS study to compare unesbulin combined with dacarbazine (DTIC) against placebo. This underscores PTC's commitment to addressing high unmet medical needs in rare cancers.

The FDA has approved a label extension for Evrysdi (risdiplam), enabling its use in infants under 2 months with spinal muscular atrophy (SMA). This groundbreaking treatment, administered at home, has effectively treated over 5,000 patients, demonstrating that pre-symptomatic infants can achieve significant developmental milestones. The approval stems from promising results in the RAINBOWFISH study, showing that infants treated with Evrysdi remained alive without requiring permanent ventilation after 12 months. Evrysdi, targeting SMN protein deficiency, is vital for maintaining healthy motor function.

PTC Therapeutics, Inc. (NASDAQ: PTCT) announced a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) regarding Upstaza™ (eladocagene exuparvovec). This marks the first gene therapy administered directly into the brain, aimed at treating aromatic L-amino acid decarboxylase (AADC) deficiency. If approved by the European Commission, Upstaza would be the first disease-modifying treatment for AADC deficiency, significantly impacting motor and cognitive functions in patients. The expected ratification is within two months, covering all EU states and some additional countries.

PTC Therapeutics reported total revenues of $149 million for Q1 2022, marking a 26% year-over-year growth. The company highlighted strong performance in its Duchenne muscular dystrophy drugs, specifically $128 million in product revenue from Translarna™ and Emflaza®. PTC initiated the PIVOT-HD Phase 2 trial for PTC518 targeting Huntington's disease, with a CHMP opinion on AADC gene therapy anticipated in May. Despite a net loss of $126.7 million, PTC reaffirmed its 2022 revenue guidance of $700-$750 million.

PTC Therapeutics (NASDAQ: PTCT) announced its participation in two investor conferences. The company will give a presentation at the Bank of America Securities 2002 Healthcare Conference on May 10 at 5:20 p.m. PT and at the 2022 RBC Capital Markets Global Healthcare Conference on May 18 at 2:05 p.m. ET. Both presentations will be available via live webcast on the company's website, with an archive accessible for 30 days post-event. PTC focuses on developing medicines for rare disorders, emphasizing its commitment to patients' unmet medical needs.

PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call on May 3, 2022, at 4:30 p.m. ET to discuss its first quarter 2022 financial results and provide a business update. Investors can join the call by dialing (877) 303-9216 in the U.S. or (973) 935-8152 outside the U.S. A live webcast will be available on the company's investor website. PTC is focused on developing medicines for rare disorders and aims to maximize value for patients and stakeholders.

PTC Therapeutics (NASDAQ:PTCT) announced the completion of Scientific Advisory Group and Oral Explanation meetings for its gene therapy treatment, PTC-AADC, aimed at AADC deficiency. The Committee for Advanced Therapies (CAT) will present a draft opinion to the Committee for Medicinal Products for Human Use (CHMP) in May 2022. If approved, PTC-AADC would be the first gene therapy infused directly into the brain, promising substantial benefits for patients suffering from this severe genetic disorder. Current treatment options are limited, making this potential approval significant.