PTC Therapeutics, Inc. - PTCT STOCK NEWS

PTC Therapeutics (NASDAQ: PTCT) will present an overview at upcoming investor conferences: Wells Fargo's 17th Annual Healthcare Conference on Sept. 8 at 1:55 p.m. EDT and Morgan Stanley's 20th Annual Global Healthcare Conference on Sept. 12 at 4:15 p.m. EDT. These presentations will be available via live webcast on PTC's Investor page and archived for 30 days. PTC focuses on developing treatments for rare disorders, enhancing access to innovative medicines for patients with unmet medical needs.

PTC Therapeutics reported $166 million in total revenue for Q2 2022, marking a 42% year-over-year growth. The DMD franchise showed strong revenue performance with $134 million net product revenue, a 32% increase from the previous year. Notably, Upstaza™ gene therapy received marketing authorization from the European Commission, beginning its commercial rollout. However, the company faced a net loss of $152 million compared to $118 million in Q2 2021. PTC reaffirms its full-year guidance of $700-$750 million in total revenues.

PTC Therapeutics, Inc. (NASDAQ: PTCT) will host a webcast conference call on August 4, 2022, at 4:30 p.m. ET, to discuss its second quarter 2022 financial results and provide a business outlook. Participants can register for the call in advance to receive dial-in details and are encouraged to join 15 minutes early to avoid delays. A replay of the conference call will be available on PTC's website shortly after the event and archived for 30 days. The company is committed to developing treatments for rare disorders.

PTC Therapeutics announced the European Commission's approval of Upstaza™ (eladocagene exuparvovec), the first gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency, applicable in all EU member states and select countries. This therapy is significant for patients aged 18 months and older, offering a disease-modifying treatment option. Clinical trials showed substantial improvements in motor and cognitive skills in treated patients. CEO Stuart W. Peltz emphasized the therapy's potential impact on the quality of life for patients suffering from this severe genetic disorder.

PTC Therapeutics (NASDAQ: PTCT) will hold a conference call on June 21, 2022, at 8:00 a.m. E.T. to discuss topline results from Study 041 of Translarna™ (ataluren) for patients with nonsense mutation Duchenne muscular dystrophy. The call will include a slide presentation accessible via the company’s website. Translarna is a protein restoration therapy aimed at addressing genetic disorders caused by nonsense mutations. More details about Duchenne muscular dystrophy and Translarna can be found on PTC's website.

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.