Ptc Therapeutics Stock Price, News & Analysis

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.

PTC Therapeutics, Inc. (NASDAQ: PTCT) has approved non-statutory stock options to purchase 86,130 shares and 33,125 restricted stock units (RSUs) for 96 new employees, as per the NASDAQ inducement grant exception. All stock options have an exercise price of $42.85, the closing price on April 8, 2022, and vest over four years with specific conditions. The RSUs also vest over four years. This decision was made by PTC's Compensation Committee in alignment with NASDAQ Listing Rule 5635(c)(4).

PTC Therapeutics (NASDAQ: PTCT) has initiated the PIVOT-HD Phase 2 clinical trial to evaluate PTC518 for Huntington's disease, a condition lacking current treatments targeting its underlying cause. This global trial will start in the U.S. and is divided into two phases: a 12-week placebo-controlled phase focusing on pharmacology and a 9-month phase assessing biomarker effects. Previous Phase 1 results indicated a dose-dependent lowering of huntingtin protein, which is critical for disease management.

PTC Therapeutics (NASDAQ: PTCT) announced the presentation of three posters on EMFLAZA™ (deflazacort) for Duchenne muscular dystrophy (DMD) at the MDA Clinical & Scientific Conference from March 13-16, 2022, in Nashville, TN. This marks the first presentation of data regarding steroid treatment outcomes in non-ambulatory DMD patients, supporting deflazacort's advantages over prednisone. Key presentations include associations between both steroid treatments and disease progression markers and milestones. A hybrid industry forum on corticosteroid treatment is set for March 15.