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PTC Therapeutics, Inc. (NASDAQ: PTCT) is a pioneering global biopharmaceutical company dedicated to discovering, developing, and commercializing orally-administered small-molecule therapeutics. The company is focused on post-transcriptional control processes in RNA biology, which are crucial for regulating protein synthesis and ensuring proper cellular function. PTC's innovative approach targets a range of therapeutic areas, including neuromuscular disorders, oncology, and infectious diseases, with a particular emphasis on treatments for orphan and ultra-orphan disorders.
One of PTC's notable achievements is the development of Translarna™ (ataluren), a treatment for nonsense mutation Duchenne muscular dystrophy (nmDMD). Despite a recent setback with the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) issuing a negative opinion, the European Commission has asked for a re-evaluation, keeping the medication available in Europe. This reflects the strong backing from the patient community and expert physicians who believe in its efficacy and safety.
The company’s robust product pipeline also includes Upstaza™ (eladocagene exuparvovec), a gene therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. This therapy has recently been submitted for review by the U.S. FDA and granted Priority Review status. Upstaza has shown transformative clinical benefits in clinical trials and compassionate use programs, demonstrating significant neurological improvements in patients.
Additionally, PTC is advancing sepiapterin for the treatment of phenylketonuria (PKU). The company's Marketing Authorization Application (MAA) for sepiapterin has been accepted by the EMA, with submissions planned in the U.S., Brazil, and Japan. Sepiapterin has demonstrated significant reductions in blood phenylalanine levels, offering a potential new treatment option for PKU patients who currently have limited therapeutic choices.
Financially, PTC has shown strong revenue performance and is well-positioned for future success. Recent financial results indicate a thriving operational status, supported by a diversified portfolio and strategic partnerships. The company's commitment to providing best-in-class treatments for rare disorders, combined with its global commercial infrastructure, underscores its strategy to maximize value for all stakeholders.
PTC's mission is to leverage its scientific and clinical expertise to bring innovative therapies to patients in need, ensuring access to treatments that can significantly improve quality of life. For more information, please visit their official website.
PTC Therapeutics announced FDA acceptance of its New Drug Application (NDA) resubmission for Translarna™ to treat nonsense mutation Duchenne muscular dystrophy (nmDMD). The resubmission is supported by Study 041 results showing significant benefits in multiple endpoints after 72 weeks of treatment, including improved six-minute walk distance and other mobility measures. The STRIDE registry data demonstrated a 3.5-year delay in loss of ambulation and 1.8-year delay in reaching critical lung function threshold. As this is a resubmission following a previous complete response letter, no FDA action date has been provided.
PTC Therapeutics announced the approval of inducement grants for nineteen new employees on Oct. 23, 2024. The grants include 13,690 non-statutory stock options and 20,250 restricted stock units (RSUs). The stock options have an exercise price of $42.48 per share, vesting over four years with 25% vesting after the first year and 6.25% quarterly thereafter. The RSUs also vest over four years, with 25% vesting annually. These awards were granted under Nasdaq Listing Rule 5635(c)(4) as part of the new employees' compensation packages.
PTC Therapeutics (NASDAQ: PTCT) has announced it will host a webcast conference call on Thursday, November 7, at 4:30 p.m. EST to discuss its third quarter 2024 financial results and provide a business update. Participants are advised to register in advance and dial in 15 minutes before the call to avoid delays. The webcast will be accessible through PTC's Investor Relations website, with a replay available for 30 days after the call.
PTC Therapeutics announced that the CHMP of the European Medicines Agency has maintained its negative opinion on renewing the conditional marketing authorization for Translarna™ (ataluren) for treating nonsense mutation Duchenne Muscular Dystrophy (nmDMD) after re-examination. The opinion will be reviewed by the European Commission, with a decision expected in about 67 days.
PTC's CEO, Matthew B. Klein, M.D., expressed disappointment, stating that the CHMP based its decision on the primary analysis subpopulation of Study 041 instead of considering the totality of evidence, including data from three placebo-controlled trials and the STRIDE registry. He emphasized that the decision goes against the wishes of physicians, patients, and families in Europe.
Translarna will remain authorized in Europe pending the EC review. PTC will continue to make Translarna available to patients and ensure the EC has all possible evidence to support continued authorization.
PTC Therapeutics announced that the FDA has set a target regulatory action date of July 29, 2025, for the review of the New Drug Application (NDA) for sepiapterin. The NDA was submitted for the treatment of phenylketonuria (PKU) in pediatric and adult patients, covering all age groups and disease subtypes. Additionally, the results of the Phase 3 APHENITY trial were published in The Lancet.
The NDA includes data from the APHENITY trial and the ongoing open-label extension study, which shows the durability of sepiapterin's effect and patients' ability to liberalize their diet while maintaining phenylalanine (Phe) control. Recent results indicate that approximately 60% of subjects achieve protein intake above the age-adjusted recommended daily allowance for an unaffected individual while maintaining Phe levels < 360 μmol/L.
PTC Therapeutics (NASDAQ: PTCT) announced positive updates on its vatiquinone Friedreich ataxia (FA) program. Two long-term extension studies met their pre-specified endpoints, showing statistically significant evidence of durable treatment benefit on disease progression. Key findings include:
1. MOVE-FA study: 144 weeks of vatiquinone treatment resulted in a 3.7-point benefit (p<0.0001, N=70) on the modified Friedreich Ataxia Rating Scale (mFARS).
2. Earlier study: 24-months of vatiquinone treatment showed a 4.8-point benefit on mFARS (p<0.0001, N=41).
PTC plans to submit a New Drug Application (NDA) for vatiquinone in December 2024, following alignment with FDA on key aspects. The company aims to provide a potentially meaningful therapy for all FA patients, particularly children and adolescents for whom no approved therapies exist.
PTC Therapeutics (NASDAQ: PTCT) announced that the FDA has accepted for filing the New Drug Application (NDA) of sepiapterin for treating pediatric and adult patients with phenylketonuria (PKU). The NDA is based on the phase 3 APHENITY trial results, which showed a mean reduction in phenylalanine (Phe) levels of 63% in the overall treated population and 69% in classical PKU subjects. 84% of subjects achieved Phe control (<360 µmol/L) as per treatment guidelines, and 22% showed Phe level normalization.
The NDA also includes data from the APHENITY open-label extension study and the Phe tolerance sub-study. The latter demonstrated that about 60% of subjects achieved protein intake above the age-adjusted recommended daily allowance while maintaining Phe control, suggesting sepiapterin could enable diet liberalization for PKU patients.
PTC Therapeutics (NASDAQ: PTCT) announced that the FDA has granted Fast Track designation to its PTC518 program for the treatment of Huntington's disease. This designation is awarded to promising therapies for diseases with high unmet needs. PTC518 was discovered using PTC's innovative splicing platform, which also led to the development of Evrysdi® for spinal muscular atrophy.
In June 2024, PTC reported positive 12-month interim data from the PIVOT-HD study, showing durable dose-dependent lowering of mutant Huntingtin protein in blood cells (up to 43% at 10mg) and CSF. The results also indicated dose-dependent favorable clinical effects on key disease measurements, including total motor score and cUHDRS scale. Importantly, PTC518 demonstrated a safe and well-tolerated profile with no treatment-related NfL spikes.
PTC Therapeutics (NASDAQ: PTCT) has announced the approval of inducement grants for nine new employees on Sept. 8, 2024. The grants include 16,180 non-statutory stock options and 23,725 restricted stock units (RSUs). These awards are part of the new hires' compensation packages and comply with Nasdaq Listing Rule 5635(c)(4).
The stock options have an exercise price of $31.54 per share, based on the closing price on Sept. 6, 2024. They have a 10-year term and vest over four years, with 25% vesting on the first anniversary and 6.25% every three months thereafter. The RSUs also vest over four years, with 25% vesting annually. Both are subject to continued employment with PTC Therapeutics.
PTC Therapeutics, Inc. (NASDAQ: PTCT) has announced its participation in three upcoming investor conferences in September 2024. The company will present overviews at:
- Morgan Stanley 22nd Annual Global Healthcare Conference on Sept. 5 at 9:15 am ET
- 2024 Wells Fargo Healthcare Conference on Sept. 6 at 12:00 pm ET
- Cantor 2024 Global Healthcare Conference on Sept. 17 at 2:30 pm ET
All presentations will be webcast live on PTC Therapeutics' website under the Investor section's Events and Presentations page. The webcasts will remain archived for 30 days after each presentation. Investors are advised to connect to the website several minutes before the start of each webcast to ensure timely access.
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