PTCT PTC Therapeutics Inc.

53.26
+1.31  (+3%)
Previous Close 51.95
Open 52.16
52 Week Low 30.7923
52 Week High 59.89
Market Cap $3,605,873,178
Shares 67,703,214
Float 67,179,439
Enterprise Value $3,360,540,999
Volume 364,286
Av. Daily Volume 447,537
Stock charts supplied by TradingView

Upcoming Catalysts

Drug Stage Catalyst Date
PTC857
Healthy volunteers
Phase 1
Phase 1
Premium membership is required to view catalyst dates, analyst ratings, earnings dates and cash burn data. Click here to unlock and sign up to a 14-day FREE TRIAL.
PTC-AADC
AADC deficiency
BLA Filing
BLA Filing
Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.
PTC299
COVID-19
Phase 2/3
Phase 2/3
Lorem ipsum dolor sit amet, consectetur adipiscing elit. Quisque sapien.

Drug Pipeline

Drug Stage Notes
Vatiquinone
Mitochondrial Epilepsy
Phase 2/3
Phase 2/3
Phase 2/3 trial initiation announced October 26, 2020.
Risdiplam (RG7916)
Spinal Muscular Atrophy (SMA) Type 1
Approved
Approved
FDA Approval announced August 7, 2020.
Translarna
Duchenne muscular dystrophy caused by nonsense mutations (nmDMD)
Phase 3
Phase 3
Received a Refusal to File letter February 23 2016. Appeal rejected October 17, 2016. Decision made to resubmit NDA during 1Q 2017 under protest. Further CRL October 25, 2017. NDA to be refiled.
Emflaza
Duchenne muscular dystrophy (age 2-5)
Approved
Approved
FDA Approval announced for label expansion June 7, 2019.
Translarna
Nonsense mutation cystic fibrosis
Phase 3
Phase 3
Phase 3 data released March 2, 2017 - endpoints not met. Development to be discontinued.

Latest News

  1. SOUTH PLAINFIELD, N.J., Oct. 26, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT), today announced the initiation of a registration-directed Phase 2/3 clinical trial to evaluate vatiquinone (PTC743) in patients with mitochondrial epilepsy, the highly morbid condition of refractory seizures in children with inherited mitochondrial disease. It is estimated that there are approximately 11,000-13,0001 patients with mitochondrial epilepsy in the United States, European Union, Japan and Latin America. Vatiquinone, developed from PTC's Bio-e platform, is an investigational oral small molecule that inhibits 15-Lipoxygenase, a key enzyme that regulates oxidative stress and inflammation response pathways underpinning many neurological disease…

    SOUTH PLAINFIELD, N.J., Oct. 26, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT), today announced the initiation of a registration-directed Phase 2/3 clinical trial to evaluate vatiquinone (PTC743) in patients with mitochondrial epilepsy, the highly morbid condition of refractory seizures in children with inherited mitochondrial disease. It is estimated that there are approximately 11,000-13,0001 patients with mitochondrial epilepsy in the United States, European Union, Japan and Latin America. Vatiquinone, developed from PTC's Bio-e platform, is an investigational oral small molecule that inhibits 15-Lipoxygenase, a key enzyme that regulates oxidative stress and inflammation response pathways underpinning many neurological disease pathologies, including epilepsy. The U.S. Food and Drug Administration has granted PTC743 orphan drug designation and pediatric rare disease designation for mitochondrial epilepsy.  

    "We are excited to initiate this registrational clinical trial to evaluate the first indication for vatiquinone that was identified from our Bio-e platform," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "As a devastating and highly fatal pediatric disorder with no approved treatment options, mitochondrial epilepsy represents an area of significant unmet need. Vatiquinone has been evaluated in over 500 patients to date, and in clinical studies it has demonstrated effects in resolving refractory status epilepticus, decreasing seizure frequency and seizure-related morbidity in certain patients. We look forward to advancing this compound for patients who are clearly in need of treatment."

    The Phase 2/3 trial in mitochondrial epilepsy, named MIT-E, is a randomized, placebo-controlled study evaluating vatiquinone in approximately 60 patients with genetically confirmed mitochondrial disease and associated refractory epilepsy. Enrolled patients will participate in a one-month run-in phase to ensure a minimum number of observable motor seizures. Patients who meet that threshold will then be enrolled in a six-month parallel arm placebo-controlled phase. The study's primary endpoint is the reduction of observed motor seizure frequency in the placebo-controlled phase relative to the run-in phase. Secondary endpoints include the occurrence of status epilepticus, number of hospitalizations, rescue medication use, and caregiver burden.

    About Vatiquinone (PTC743)

    Vatiquinone is an investigational small molecule that inhibits 15-Lipoxygenase, an enzyme that is a key regulator of the oxidative stress and inflammation response pathways that underpin neurological disease pathology. Vatiquinone has been evaluated in a number of clinical studies in which it has been demonstrated to have an impact on mortality risk and a number of neurological and neuromuscular disease symptoms. In more than 500 patients with duration of exposure up to 10 years, vatiquinone has demonstrated a favorable safety profile.

    About the Bio-e platform

    The Bio-e platform is based on a family of enzyme targets, oxidoreductases, that are critical to the generation and regulation of energy key to disease pathology in the CNS and other organ systems. The platform harnesses electron-transfer chemistry to modulate key biological processes beyond the reach of current drug development approaches. The lead compounds from the Bio-e platform, vatiquinone (PTC743) and PTC857, target the enzyme 15-lipoxygenase, a key enzymatic hub that regulates the inflammation and oxidative stress that underpin mitochondrial disease and CNS pathology.

    About PTC Therapeutics, Inc.

    PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.

    For More Information:

    Media:

    Jane Baj

    +1 (908) 912-9167

     

    Investors:

    Lisa Hayes

    +1 (908) 868-8926

     

    Forward-Looking Statements:

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC, including with respect to the expected timing of clinical trials and studies, availability of data, regulatory submissions and responses and other matters; PTC's strategy, future operations, future financial position, future revenues and projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.

    PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the eligible patient base and commercial potential of PTC's products and product candidates; the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.

    As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory or prove to be commercially successful.

    The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

    1Gorman et al. Annals of Neurology.2015; Chevallier et al. Epilepsia. 2014;

    Cision View original content:http://www.prnewswire.com/news-releases/ptc-therapeutics-announces-initiation-of-a-registration-directed-clinical-trial-to-evaluate-vatiquinone-in-mitochondrial-epilepsy-301159255.html

    SOURCE PTC Therapeutics

    View Full Article Hide Full Article
  2. SOUTH PLAINFIELD, N.J., Oct. 22, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) and the AADC Family Network today announced that Oct. 23, 2020, has been officially designated as the first Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Awareness Day in the State of Massachusetts, helping enable parents, caregivers, patient advocacy groups, local legislators, and experts raise awareness, share resources, and work together towards a treatment for this devastating disease.

    To commemorate the day, PTC will be hosting a Facebook Live event on Friday, Oct. 23, at 10 a.m. EDT featuring commentary from advocacy partners, local ambassadors and other industry experts, including:

    • Bob Coughlin, President and CEO of MassBio
    • Kelly Heger

    SOUTH PLAINFIELD, N.J., Oct. 22, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) and the AADC Family Network today announced that Oct. 23, 2020, has been officially designated as the first Aromatic L-amino Acid Decarboxylase (AADC) Deficiency Awareness Day in the State of Massachusetts, helping enable parents, caregivers, patient advocacy groups, local legislators, and experts raise awareness, share resources, and work together towards a treatment for this devastating disease.

    To commemorate the day, PTC will be hosting a Facebook Live event on Friday, Oct. 23, at 10 a.m. EDT featuring commentary from advocacy partners, local ambassadors and other industry experts, including:

    • Bob Coughlin, President and CEO of MassBio
    • Kelly Heger, Founder of AADC Family Network & Foundation
    • Dr. Irina Anselm, Director, Mitochondrial Program & Co-Director, Neurometabolic Program at Boston Children's Hospital
    • Dr. Matthew Klein, Chief Development Officer, PTC Therapeutics

    The discussion will focus on how to raise awareness among parents and caregivers, patient advocacy groups, legislators and experts to work toward a treatment to address the high unmet medical need caused by AADC deficiency. Interested members of the public are invited to join the event via Facebook Live stream and can RSVP for the event on Facebook.

    The initiative was spearheaded by Kelly Heger, AADC deficiency caregiver and founder of the AADC Family Network & Foundation, along with PTC Therapeutics, MassBio and Boston Children's Hospital. Ms. Heger's daughter Jillian was diagnosed with the rare disease at just 5 months old. Since then, Jillian has required 24-hour care from family members, visiting health care professionals, and inpatient hospital teams. Ms. Heger has been a pioneering advocate for AADC awareness and founded the AADC Family Network & Foundation to help the AADC deficiency community access resources and to unite in the campaign for a cure.

    "When Jillian was diagnosed with AADC deficiency, she was one of the first children to receive the diagnosis in the United States," said Kelly Heger, Founder AADC Family Network & Foundation. "As Jillian has progressed on her journey, and as we have learned more about the devastating disease in tandem, raising awareness and finding a cure for the AADC deficiency community has become increasingly important. Declaring Oct. 23 as AADC Deficiency Awareness Day in Massachusetts in partnership with PTC Therapeutics and the State is a significant milestone for the rare disease community, and we are hopeful that it will help propel other advocacy organizations, researchers and policymakers towards our shared goal of finding a cure."

    Representative Angelo D'Emilia introduced Resolution HD 5268 which passed the Massachusetts House of Representatives on Oct.1, declaring Oct. 23 as AADC Deficiency Awareness Day.

    "I am happy to be supporting Oct. 23 as Aromatic L-Amino Acid Decarboxylase (AADC) Deficiency Day here in Massachusetts," said Representative D'Emilia.  "The Heger family is an inspiration and our community is proud to support Kelly as the Founder of the AADC Family Network and all of her dedicated work and advocacy."

    "We are honored to partner with other industry leaders and advocates to declare an important awareness day for the AADC deficiency community," said Mary Frances Harmon, SVP, Corporate Relations of PTC Therapeutics. "It is particularly meaningful that Massachusetts is the first state to declare this proclamation, with its vibrant network of top-tier life science companies, talent, and resources constantly working on the next generation of therapeutics for patients. At PTC, we remain committed to developing a gene therapy for this patient population, in hopes of delivering meaningful clinical benefits to individuals with this devastating disease."

    AADC deficiency is a rare genetic disorder that affects the brain, causes weak muscle tone[1], and results in delays or failure to reach developmental milestones.[2] Patients with severe AADC deficiency have a high risk of death during childhood and there are currently no approved therapies that address the underlying cause.2 AADC deficiency presents with signs and symptoms similar to more common diseases, and is often under and misdiagnosed by physicians, resulting in a long and challenging diagnosis journey for patients and families.

    For additional information about aromatic l-amino acid decarboxylase (AADC) deficiency, please visit:

    About the AADC Family Network & Foundation

    The AADC Family Network & Foundation was originally founded as the ALADD Foundation, Inc. in 1998 and was the first foundation for AADC (Aromatic L Amino Acid Decarboxylase Deficiency). The foundation was established for the charitable and educational purposes of providing research, medical intervention, support and awareness, and to participate in all services and activities designed to carry out and find a CURE for AADC deficiency.

    About PTC Therapeutics, Inc.

    PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.

    For More Information:

    Investors:

    Lisa Hayes

    +1 (732) 354-8687

     

    Media:

    Jane Baj

    +1 (908) 912-9167

     

    Forward-Looking Statements:

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historic fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC; expectations with respect to PTC's gene therapy platform; PTC's strategy, future operations, future financial position, future revenues and projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.

    PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the eligible patient base and commercial potential of PTC's products and product candidates; expectations with respect to PTC's gene therapy platform; the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future; significant business effects, including the effects of industry, market, economic, political or regulatory conditions; changes in tax and other laws, regulations, rates and policies; PTC's scientific approach and general development progress; and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC. You are urged to carefully consider all such factors.

    As with any pharmaceutical under development, there are significant risks in the development, regulatory approval and commercialization of new products. There are no guarantees that any product will receive or maintain regulatory approval in any territory or prove to be commercially successful.

    The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

    1 What is AADC Deficiency? Retrieved from https://aboutaadc.com/what-is-aadc/

    2 PTC Therapeutics. (2020, September 9). PTC Therapeutics Launches PTC PINPOINT - A Genetic Testing Program for Individuals Suspected of Having Neurotransmitter Disorders [Press Release] Retrieved from https://ir.ptcbio.com/news-releases/news-release-details/ptc-therapeutics-launches-ptc-pinpoint-genetic-testing-program 

    Cision View original content:http://www.prnewswire.com/news-releases/ptc-therapeutics-and-aadc-family-network-announce-first-ever-aadc-deficiency-awareness-day-in-massachusetts-301157792.html

    SOURCE PTC Therapeutics, Inc.

    View Full Article Hide Full Article
  3. SOUTH PLAINFIELD, N.J., Oct. 21, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that Evrysdi™ (risdiplam) was approved in Brazil by the National Health Surveillance Agency (ANVISA) for the treatment of spinal muscular atrophy (SMA). The approval was obtained within seven months of Roche's initial submission and marks the second country to approve Evrysdi™. Additionally, PTC announced the filing of a New Drug Application (NDA) for Evrysdi™ for the treatment of SMA with the Japanese Ministry of Health, Labor and Welfare (MHLW). The filing in Japan by Chugai Pharmaceutical Co. Ltd., a member of the Roche group, triggers a $7.5 million milestone payment to PTC from Roche.

    "We are very pleased with both the rapid approval…

    SOUTH PLAINFIELD, N.J., Oct. 21, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that Evrysdi™ (risdiplam) was approved in Brazil by the National Health Surveillance Agency (ANVISA) for the treatment of spinal muscular atrophy (SMA). The approval was obtained within seven months of Roche's initial submission and marks the second country to approve Evrysdi™. Additionally, PTC announced the filing of a New Drug Application (NDA) for Evrysdi™ for the treatment of SMA with the Japanese Ministry of Health, Labor and Welfare (MHLW). The filing in Japan by Chugai Pharmaceutical Co. Ltd., a member of the Roche group, triggers a $7.5 million milestone payment to PTC from Roche.

    "We are very pleased with both the rapid approval in Brazil as well as the filing of Evrysdi™ in Japan," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "These important achievements continue to build momentum for Evrysdi™ globally, and we are working hard alongside our partners in the collaboration to make the medicine available to SMA patients around the world. The strong efficacy, excellent safety profile, and convenient oral formulation of Evrysdi™ are so important to the SMA community. Access to a home administered therapy is particularly important during the COVID-19 pandemic."

    The NDA in Japan is based on data from the FIREFISH study in infants aged 1 to 7 months old with symptomatic Type 1 SMA and the SUNFISH study in children and young adults with Type 2 or 3 SMA. MHLW previously granted orphan drug designation to risdiplam and the NDA application is subject to priority review.

    The U.S. Food and Drug Administration (FDA) approved Evrysdi™ (risdiplam) in August 2020, for the treatment of SMA for adults and children 2 months and older. At this time, applications have been filed for Evrysdi™ in 15 international markets: Australia, Chile, India, Indonesia, Israel, Kuwait, Macedonia, Malaysia, Russia, Singapore, South Korea, Taiwan, Thailand, Ukraine and the United Arab Emirates. In addition, four health authorities worldwide are currently reviewing the application: Canada, China, the European Union and Switzerland.

    Evrysdi™ is based on PTC science and is commercialized in the United States by Genentech, a member of the Roche Group. Roche leads the clinical development of Evrysdi™ as part of a collaboration with the SMA Foundation and PTC Therapeutics.

    About Spinal Muscular Atrophy (SMA)

    Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease that can be fatal. It affects approximately 1 in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual's physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

    About Evrysdi™ (risdiplam)

    Evrysdi™ is a survival motor neuron 2 (SMN2)-directed RNA splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi™ is designed to distribute evenly to all parts of the body, including the central nervous system (CNS). Evrysdi™ is administered daily at home in liquid form by mouth or feeding tube. The U.S. Food and Drug Administration recently approved Evrysdi™ (risdiplam) for the treatment of spinal muscular atrophy for adults and children 2 months and older. Evrysdi™ (risdiplam) is marketed in the United States by Genentech, a member of the Roche Group.

    About the Risdiplam Clinical Studies

    FIREFISH (NCT02913482) - an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of risdiplam in infants and determining the dose for Part 2. Part 2 is a pivotal, single-arm study of risdiplam in 41 infants with Type 1 SMA treated for two years followed by an open-label extension. Enrollment for Part 2 was completed in November 2018. The primary objective of Part 2 was to assess efficacy as measured by the proportion of infants sitting without support after 12 months of treatment, as assessed in the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) (defined as sitting without support for five seconds). The study met its primary endpoint.

    SUNFISH (NCT02908685) - SUNFISH is a two part, double-blind, placebo controlled pivotal study in people aged 2 to 25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using total score of Motor Function Measure 32 (MFM-32) at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. The study met its primary endpoint.

    Clinical Trial Safety Data

    The safety profile of Evrysdi™ was established across FIREFISH and SUNFISH pivotal trials. The most common adverse reactions in later-onset SMA (incidence of at least 10 percent of patients treated with Evrysdi™ and more frequently than control) were fever, diarrhea, and rash. The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, the most common adverse reactions (incidence of at least 10 percent) were upper respiratory tract infection, pneumonia, constipation, and vomiting.

    In addition to FIREFISH and SUNFISH, Evrysdi™ is being evaluated in a broad range of people with SMA, including in:

    JEWELFISH (NCT03032172) - an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi™. The study has completed recruitment (n=174).

    RAINBOWFISH (NCT03779334) - an open-label, single-arm, multi-center study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in babies (~n=25), from birth to 6 weeks of age (at first dose), with genetically diagnosed SMA, who are not yet presenting with symptoms. The study is currently recruiting.

    About PTC Therapeutics, Inc.

    PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.

    For More Information:

    Investors:

    Lisa Hayes

    +1 (732) 354-8687

    Media:

    Jane Baj

    +1 (908) 912-9167

     

    Forward-Looking Statements:

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historical fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC; advancement of PTC's joint collaboration program in SMA, including any potential regulatory submissions, regulatory approvals or commercial prospects; PTC's strategy, future operations, future financial position, future revenues and, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.

    PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future, including Evrysdi™; the enrollment, conduct, and results of studies under the SMA collaboration and events during, or as a result of, the studies that could delay or prevent further development under the program, including any potential regulatory submissions and potential commercialization with regards to Evrysdi™; the eligible patient base and commercial potential of Evrysdi™ or any of PTC's other product candidates; and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC (Securities and Exchange Commission). You are urged to carefully consider all such factors.

    As with any pharmaceutical under development, there are significant risks in the development, regulatory approval, and commercialization of new products. There are no guarantees that any product or product candidate will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Evrysdi™.

    The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

     

    Cision View original content:http://www.prnewswire.com/news-releases/evrysdi-risdiplam-continues-to-make-global-progress-for-the-treatment-of-spinal-muscular-atrophy-301156965.html

    SOURCE PTC Therapeutics, Inc.

    View Full Article Hide Full Article
  4. SOUTH PLAINFIELD, N.J., Oct. 15, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that the Company will host a webcast conference call to report its third quarter 2020 financial results and provide an update on the company's business and outlook on Thursday, October 29, 2020 at 4:30 p.m. (ET) after the closing of the market.

    The call can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 7096445. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion…

    SOUTH PLAINFIELD, N.J., Oct. 15, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that the Company will host a webcast conference call to report its third quarter 2020 financial results and provide an update on the company's business and outlook on Thursday, October 29, 2020 at 4:30 p.m. (ET) after the closing of the market.

    The call can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 7096445. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast replay of the call will be available approximately two hours after completion of the call and will be archived on the company's website for 30 days following the call.

    About PTC Therapeutics, Inc.

    PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.

    FOR MORE INFORMATION PLEASE CONTACT:

    Media:

    Investors:

    Jane Baj

    Lisa Hayes

    +1 (908) 912-9167

    +1 (732) 354-8687

     

     

    Cision View original content:http://www.prnewswire.com/news-releases/ptc-therapeutics-to-host-conference-call-to-discuss-third-quarter-2020-financial-results-301147176.html

    SOURCE PTC Therapeutics, Inc.

    View Full Article Hide Full Article
  5. SOUTH PLAINFIELD, N.J., Sept. 28, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that two-year data from Part 1 of the FIREFISH study demonstrated that infants on Evrysdi™ (risdiplam) continued to improve and achieve motor milestones. Evrysdi™ was approved in August for the treatment of spinal muscular atrophy (SMA) patients two months and older. Data were presented at the 25th International Annual Congress of the World Muscle Society (WMS).

    "The results from the long-term FIREFISH trial demonstrate that SMA patients continue to improve in motor function and gain additional developmental milestones," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "In addition, the results further validate…

    SOUTH PLAINFIELD, N.J., Sept. 28, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that two-year data from Part 1 of the FIREFISH study demonstrated that infants on Evrysdi™ (risdiplam) continued to improve and achieve motor milestones. Evrysdi™ was approved in August for the treatment of spinal muscular atrophy (SMA) patients two months and older. Data were presented at the 25th International Annual Congress of the World Muscle Society (WMS).

    "The results from the long-term FIREFISH trial demonstrate that SMA patients continue to improve in motor function and gain additional developmental milestones," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "In addition, the results further validate the safety and durable efficacy profile of Evrysdi™ and reinforce the continued benefit of the therapy. Since its approval in August, Evrysdi™ has demonstrated a strong commercial launch signifying the need for an oral treatment for SMA patients, especially one that can be taken at home amidst the global pandemic."

    The results from the second year of Part 1 of the FIREFISH study demonstrated that infants treated with the therapeutic dose of risdiplam (17 out of 21 babies) showed that an estimated 88 percent of infants were alive and required no permanent ventilation at two years. In addition, infants reached the following milestones:

    Milestone

    1 year

    2 years

    Sit without support for at least 5 seconds

    (as measured by the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development – Third edition (BSID-III) 

    41% (7/17)

    59% (10/17)

    Upright head control

    53% (9/17)

    65% (11/17)

    Ability to turn over

    12% (2/17)

    29% (5/17)

    Stand supporting weight or with support

    6% (1/17)

    30% (5/17)

    CHOP-INTEND* score of 40 points or more

    59% (10/17)

    71% (12/17)

    Safety for Evrysdi in the FIREFISH study was consistent with its previously reported safety profile and no new safety signals were identified. The most common adverse events (n=21) included fever (pyrexia; 71%), upper respiratory tract infection (52%), cough (33%), vomiting (33%), diarrhea (29%) and respiratory tract infection (29%). The most serious adverse event that occurred in 24% of infants was pneumonia.

    Evrysdi™ has demonstrated a favorable efficacy and safety profile, with the safety profile established across the FIREFISH and SUNFISH trials. To date, there have been no drug-related safety findings leading to withdrawal from any study.

    At the time of the analysis, the youngest infant was 28.4 months and the oldest was 45.1 months old. The median age at enrollment was 6.3 months. Of the infants alive at two years (n=14), 100 percent maintained the ability to swallow and 93 percent (13/14) were able to feed orally. Of the 17 infants treated with the therapeutic dose, two experienced fatal complications of their disease at eight and 13 months of treatment and one infant was withdrawn from the study and sadly died 3.5 months later. None of these were attributed by the investigator as related to risdiplam.

    Evrysdi™ (risdiplam) is being studied in more than 450 people as part of a broad and robust clinical trial program in SMA, with patients ranging from birth to 60 years old, and including pre-symptomatic patients and those previously treated with other SMA-targeting therapies.

    Evrysdi™ is designed to treat SMA by increasing and sustaining the production of the survival of motor neuron (SMN) protein. SMN protein is found throughout the body and is critical for maintaining healthy motor neurons and movement. Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. Evrysdi is marketed in the United States by Genentech, a member of the Roche Group.

    *Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders

    About Evrysdi™ (risdiplam)

    Evrysdi™ is a survival of motor neuron 2 (SMN2) splicing modifier designed to treat SMA caused by mutations in chromosome 5q that lead to SMN protein deficiency. Evrysdi™ is administered daily at home in liquid form by mouth or by feeding tube.

    The FDA approved Evrysdi for the treatment of SMA in adults and children 2 months of age and older. Risdiplam was granted PRIME (PRIority MEdicines) designation by the European Medicines Agency (EMA) in 2018 and Orphan Drug Designation by FDA and EMA in 2017 and 2019, respectively. At this time, Evrysdi™ has been filed in 16 markets: Australia, Brazil, Chile, India, Indonesia, Israel, Kuwait, Macedonia, Malaysia, Russia, Singapore, South Korea, Taiwan, Thailand, Ukraine, and the United Arab Emirates. In addition, four health authorities are currently reviewing the application: Canada, China, EU (European Union) and Switzerland.

    About the Clinical Studies

    Risdiplam is currently being evaluated in four multi-center trials in people with SMA:

    • FIREFISH (NCT02913482) - an open-label, two-part pivotal clinical trial in infants with Type 1 SMA. Part 1 was a dose-escalation study in 21 infants with the primary objective of assessing the safety profile of risdiplam in infants and determining the dose for Part 2. Part 2 is a pivotal, single-arm study of risdiplam in 41 infants with Type 1 SMA treated for two years followed by an open-label extension. Enrollment for Part 2 was completed in November 2018. The primary objective of Part 2 was to assess efficacy as measured by the proportion of infants sitting without support after 12 months of treatment, as assessed in the Gross Motor Scale of the Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) (defined as sitting without support for five seconds). The study met its primary endpoint.
    • SUNFISH (NCT02908685) - SUNFISH is a two part, double-blind, placebo controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA. Part 1 (n=51) determined the dose for the confirmatory Part 2. Part 2 (n=180) evaluated motor function using total score of Motor Function Measure 32 (MFM-32) at 12 months. MFM-32 is a validated scale used to evaluate fine and gross motor function in people with neurological disorders, including SMA. The study met its primary endpoint.
    • JEWELFISH (NCT03032172) - an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics (PK) and pharmacodynamics (PD) in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi™. The study has completed recruitment (n=174).
    • RAINBOWFISH (NCT03779334) - an open-label, single-arm, multi-center study, investigating the efficacy, safety, pharmacokinetics, and pharmacodynamics of risdiplam in babies (~n=25), from birth to 6 weeks of age (at first dose), with genetically diagnosed SMA, who are not yet presenting with symptoms. The study is currently recruiting.

    Clinical Trial Safety Data

    The safety profile of Evrysdi™ was established across FIREFISH and SUNFISH pivotal trials. The most common adverse reactions in later-onset SMA (incidence of at least 10 percent of patients treated with Evrysdi™ and more frequently than control) were fever, diarrhea, and rash. The most common adverse reactions in infantile-onset SMA were similar to those observed in later-onset SMA patients. Additionally, the most common adverse reactions (incidence of at least 10 percent) were upper respiratory tract infection, pneumonia, constipation, and vomiting. 

    About Spinal Muscular Atrophy (SMA)

    Spinal muscular atrophy (SMA) is a severe, progressive neuromuscular disease that can be fatal. It affects approximately one in 10,000 babies and is the leading genetic cause of infant mortality. SMA is caused by a mutation of the survival motor neuron 1 (SMN1) gene, which leads to a deficiency of SMN protein. This protein is found throughout the body and is essential to the function of nerves that control muscles and movement. Without it, nerve cells cannot function correctly, leading to muscle weakness over time. Depending on the type of SMA, an individual's physical strength and their ability to walk, eat or breathe can be significantly diminished or lost.

    About PTC Therapeutics, Inc.

    PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.

    For More Information:

    Investors:

    Lisa Hayes

    +1 (908) 868-8926

     

    Media:

    Jane Baj

    +1 (908) 912-9167

      

    Forward Looking Statements:

    This press release contains forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. All statements contained in this release, other than statements of historical fact, are forward-looking statements, including statements regarding: the future expectations, plans and prospects for PTC; advancement of PTC's joint collaboration program in SMA, including any potential regulatory submissions, regulatory approvals or commercial prospects; PTC's strategy, future operations, future financial position, future revenues and, projected costs; and the objectives of management. Other forward-looking statements may be identified by the words "guidance," "plan," "anticipate," "believe," "estimate," "expect," "intend," "may," "target," "potential," "will," "would," "could," "should," "continue," and similar expressions.

    PTC's actual results, performance or achievements could differ materially from those expressed or implied by forward-looking statements it makes as a result of a variety of risks and uncertainties, including those related to: the outcome of pricing, coverage and reimbursement negotiations with third party payors for PTC's products or product candidates that PTC commercializes or may commercialize in the future, including Evrysdi™; the enrollment, conduct, and results of studies under the SMA collaboration and events during, or as a result of, the studies that could delay or prevent further development under the program, including any potential regulatory submissions and potential commercialization with regards to Evrysdi™; the eligible patient base and commercial potential of Evrysdi™ or any of PTC's other product candidates; and the factors discussed in the "Risk Factors" section of PTC's most recent Quarterly Report on Form 10-Q and Annual Report on Form 10-K, as well as any updates to these risk factors filed from time to time in PTC's other filings with the SEC (Securities and Exchange Commission). You are urged to carefully consider all such factors.

    As with any pharmaceutical under development, there are significant risks in the development, regulatory approval, and commercialization of new products. There are no guarantees that any product or product candidate will receive or maintain regulatory approval in any territory, or prove to be commercially successful, including Evrysdi™.

    The forward-looking statements contained herein represent PTC's views only as of the date of this press release and PTC does not undertake or plan to update or revise any such forward-looking statements to reflect actual results or changes in plans, prospects, assumptions, estimates or projections, or other circumstances occurring after the date of this press release except as required by law.

     

    Cision View original content:http://www.prnewswire.com/news-releases/two-year-data-of-evrysdi-risdiplam-in-infants-with-spinal-muscular-atrophy-demonstrate-continued-improvement-of-developmental-milestones-301138716.html

    SOURCE PTC Therapeutics, Inc.

    View Full Article Hide Full Article
View All PTC Therapeutics Inc. News