Deflazacort

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 42nd Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Monday, January 8, 2024
Nikolaus Friedreich, Regulation, Conference, Eladocagene exuparvovec, Economics, PTC, EMA, FDA, MAA, Friedreich's ataxia, Duchenne muscular dystrophy, FA, NDA, Deflazacort, Prescription, HD, Civil service commission, Therapy, Bank statement, PTC Therapeutics, Cardinal, DMD, PKU, European, Duchenne, AADC, Patient, ALS, GAAP, CHMP, BLA, Pharmaceutical industry

SOUTH PLAINFIELD, N.J., Jan. 8, 2024 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will present an update on its commercial progress and R&D pipeline at the 42nd Annual J.P. Morgan Healthcare Conference today, Monday, January 8, at 10:30am EST/7:30am PST. Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2023 accomplishments and highlight potential 2024 value-creating milestones. Preliminary 2023 unaudited financial results and 2024 financial guidance will also be provided. The presentation is being webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com.

Key Points: 
  • Matthew B. Klein, M.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2023 accomplishments and highlight potential 2024 value-creating milestones.
  • The presentation is being webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com .
  • PTC announced strategic portfolio prioritizations, which resulted in reductions in both operating expenses and headcount of approximately 25% and 30%, respectively.
  • PTC maintains all economics associated with up to $250 million in the remaining commercial sales milestones associated with Evrysdi global net sales.

PTC Therapeutics Provides an Update on Commercial Progress and R&D Pipeline at 40th Annual J.P. Morgan Healthcare Conference

Retrieved on: 
Monday, January 10, 2022
PKU, Amyotrophic lateral sclerosis, Cardinal, Brazilian Health Regulatory Agency, Growth, Duchenne muscular dystrophy, Standard of care, Committee, Volanesorsen, DMD, Phenylketonuria, Investor, Biologics license application, CHMP, Patient, Duchenne, PTC Therapeutics, Spinal muscular atrophy, SG, Conference, NASDAQ, GAAP, ALS, ANVISA, Classification, PTC, Therapy, European Medicines Agency, Committee for Medicinal Products for Human Use, Union, Seizure, AADC, Mitochondrial disease, Huntington's disease, Prescription, European Directive on Traditional Herbal Medicinal Products, Mission statement, Key, FDA, EMA, BLA, SMA, Roche, Medical device, Animal, Video game, Pharmaceutical industry, Medicine, Vaccine, Ataluren, Risdiplam, Deflazacort

SOUTH PLAINFIELD, N.J., Jan. 10, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will present an update on its commercial progress and R&D pipeline at the 40th Annual J.P. Morgan Healthcare Conference today, Monday Jan. 10th at 7:30am EST.

Key Points: 
  • SOUTH PLAINFIELD, N.J., Jan. 10, 2022 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) will present an update on its commercial progress and R&D pipeline at the 40th Annual J.P. Morgan Healthcare Conference today, Monday Jan. 10th at 7:30am EST.
  • Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2021 accomplishments and highlight upcoming potential value-creating milestones.
  • The presentation will be webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com .
  • PTC successfully advanced its clinical pipeline in 2021:
    APHENITY, a Phase 3 registration-directed trial of PTC923 in phenylketonuria (PKU) was initiated.

Polymyalgia Rheumatica Treatment Market to Record a Robust Growth at a CAGR of 13.60% for the Study Period (2018-2030), states DelveInsight

Retrieved on: 
Monday, March 22, 2021
Steroids, Glucocorticoids, Organ systems, Medical specialties, Corticosteroids, Prodrugs, Polymyalgia rheumatica, Hepatotoxins, Rheumatology, Prednisone, Deflazacort

The Polymyalgia Rheumatica market report also proffers an analysis of recent Polymyalgia Rheumatica treatment algorithm/practice, market drivers, market barriers, and unmet medical needs.

Key Points: 
  • The Polymyalgia Rheumatica market report also proffers an analysis of recent Polymyalgia Rheumatica treatment algorithm/practice, market drivers, market barriers, and unmet medical needs.
  • Clinicians often use the rapid resolution of symptoms to low prednisone doses (20 mg) to confirm Polymyalgia Rheumatica diagnosis.
  • Other alternatives to Glucocorticoids have been evaluated for Polymyalgia Rheumatica treatment, but results have been disappointing to date.
  • The corticosteroid drug deflazacort is being studied for the treatment of Polymyalgia Rheumatica.

Polymyalgia Rheumatica Treatment Market to Record a Robust Growth at a CAGR of 13.60% for the Study Period (2018-2030), states DelveInsight

Retrieved on: 
Monday, March 22, 2021
Steroids, Glucocorticoids, Organ systems, Medical specialties, Corticosteroids, Prodrugs, Polymyalgia rheumatica, Hepatotoxins, Rheumatology, Prednisone, Deflazacort

The Polymyalgia Rheumatica market report also proffers an analysis of recent Polymyalgia Rheumatica treatment algorithm/practice, market drivers, market barriers, and unmet medical needs.

Key Points: 
  • The Polymyalgia Rheumatica market report also proffers an analysis of recent Polymyalgia Rheumatica treatment algorithm/practice, market drivers, market barriers, and unmet medical needs.
  • Clinicians often use the rapid resolution of symptoms to low prednisone doses (20 mg) to confirm Polymyalgia Rheumatica diagnosis.
  • Other alternatives to Glucocorticoids have been evaluated for Polymyalgia Rheumatica treatment, but results have been disappointing to date.
  • The corticosteroid drug deflazacort is being studied for the treatment of Polymyalgia Rheumatica.

Amber Specialty Pharmacy Selected by PTC Therapeutics, Inc. to Dispense EMFLAZA® for Duchenne Muscular Dystrophy Patients

Retrieved on: 
Thursday, October 8, 2020
Retail, Health, Supermarket, Managed care, Specialty, Pharmaceutical, Biotechnology, Muscular dystrophy, Articles, Biotechnology, Steroids, Life sciences, PTC Therapeutics, South Plainfield, New Jersey, Pharmacy, Dystrophy, Deflazacort, Duchenne, Specialty pharmacy

EMFLAZA is indicated for the treatment of Duchenne muscular dystrophy in patients ages 2 and older.

Key Points: 
  • EMFLAZA is indicated for the treatment of Duchenne muscular dystrophy in patients ages 2 and older.
  • Amber Specialty Pharmacy is one of three specialty pharmacies selected by PTC Therapeutics to dispense EMFLAZA as part of a limited distribution network.
  • Access to EMFLAZA and our work with Duchenne muscular dystrophy patients nationwide further confirms our commitment and growth in the rare and orphan disease space as a specialty pharmacy."
  • Amber Specialty Pharmacy has built an outstanding reputation by providing personalized support and quality clinical care to patients and families.

Santhera Announces Publication on Molecular Distinctions of Vamorolone Compared to Corticosteroids

Retrieved on: 
Monday, September 14, 2020
Branches of biology, Steroids, Drugs, Glucocorticoids, Prodrugs, Mineralocorticoids, RTT, Corticosteroids, Glucocorticoid receptor, Deflazacort, Prednisolone, Prednisone

Vamorolone and the active metabolites of prednisone and deflazacort were compared regarding their molecular interactions with the target receptor (glucocorticoid receptor, GR), and required accessory proteins (co-activators, co-repressors).

Key Points: 
  • Vamorolone and the active metabolites of prednisone and deflazacort were compared regarding their molecular interactions with the target receptor (glucocorticoid receptor, GR), and required accessory proteins (co-activators, co-repressors).
  • Vamorolone has one less single contact point with the GR compared to prednisone and deflazacort, and this changes conformation of the accessory protein binding sites.
  • The data further show that vamorolone uniformly weakens co-activator associations, which leads to loss of gene transcriptional activities associated with safety concerns of corticosteroids.
  • In contrast, vamorolone retains co-repressor binding necessary for anti-inflammatory activities associated with efficacy of corticosteroids.

PTC Therapeutics Reports Second Quarter 2020 Financial Results and Provides a Corporate Update

Retrieved on: 
Wednesday, August 5, 2020
Chemical compounds, Organic compounds, Steroids, Biotechnology, PTC Therapeutics, South Plainfield, New Jersey, Ataluren, Duchenne, Dystrophy, Deflazacort

SOUTH PLAINFIELD, N.J., Aug. 5, 2020 /PRNewswire/ --PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported financial results for the second quarter ending June 30, 2020.

Key Points: 
  • SOUTH PLAINFIELD, N.J., Aug. 5, 2020 /PRNewswire/ --PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported financial results for the second quarter ending June 30, 2020.
  • Key Second Quarter and Other Corporate Updates:
    Our Duchenne muscular dystrophy franchise had strong performance in the second quarter.
  • Translarna net product revenues were $38.6 million for the second quarter of 2020, compared to $57.8 million for the second quarter of 2019.
  • Emflaza net product revenues were $36.2 million for the second quarter of 2020, compared to $27.6 million for the second quarter of 2019.

PTC Therapeutics Receives FDA Approval for the Expansion of the EMFLAZA® (deflazacort) Labeling to Include Patients 2-5 Years of Age

Retrieved on: 
Friday, June 7, 2019
Muscular dystrophy, Organic compounds, Biotechnology, Deflazacort, Glucocorticoids, Pregnanes, Prodrugs, Medicine, PTC Therapeutics, Drugs, Duchenne, Dystrophy

Emflaza was first approved by the FDA in February 2017 for the treatment of Duchenne in patients 5-years and older.

Key Points: 
  • Emflaza was first approved by the FDA in February 2017 for the treatment of Duchenne in patients 5-years and older.
  • "We are excited to be able to bring Emflaza to younger boys living with Duchenne muscular dystrophy," said Stuart Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics.
  • PTC has developed a support program, called PTC Cares which is dedicated to helping patients, caregivers, and prescribers understand the prescription process, and financial assistance programs for PTC products.
  • EMFLAZA is indicated for the treatment of Duchenne muscular dystrophy in patients two years of age and older.

PTC Therapeutics Reports Third Quarter 2018 Financial Results and Provides a Corporate Update

Retrieved on: 
Monday, November 5, 2018
Biotechnology, PTC Therapeutics, South Plainfield, New Jersey, Deflazacort, PTC, Chemistry, Organic chemistry

SOUTH PLAINFIELD, N.J., Nov. 5, 2018 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported financial results for the third quarter ending September 30, 2018.

Key Points: 
  • SOUTH PLAINFIELD, N.J., Nov. 5, 2018 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and reported financial results for the third quarter ending September 30, 2018.
  • Translarna net product revenues were $30.4 million for the third quarter of 2018, compared to $32.0 million reported in the third quarter of 2017.
  • Emflaza net product revenues were $22.6 million for the third quarter of 2018, compared to $9.8 million reported in the third quarter of 2017.
  • GAAP R&D expenses were $54.4 million for the third quarter of 2018, compared to $30.0 million for the same period in 2017.

PTC Therapeutics Announces Winners of Global Duchenne Muscular Dystrophy Patient Group STRIVE Awards in Recognition of World Duchenne Awareness Day

Retrieved on: 
Friday, September 7, 2018
Biotechnology, PTC Therapeutics, South Plainfield, New Jersey, Deflazacort

The announcement is made as the international Duchenne community marks World Duchenne Awareness Day.

Key Points: 
  • The announcement is made as the international Duchenne community marks World Duchenne Awareness Day.
  • It's a true privilege for PTC Therapeutics to be able to provide the support necessary to make these excellent projects possible."
  • Founded 20 years ago, PTC Therapeutics has successfully launched two rare disorder products and has a global commercial footprint.
  • In addition, PTC has launched its global disease awareness website, DuchenneAndYou.com to engage the broader Duchenne community and provide valuable information regarding the disease state.