NSAA

First Clinical Trial Results of Gene Therapy (GNT0004) for Duchenne Muscular Dystrophy Presented at International Myology 2024 Congress

Retrieved on: 
Tuesday, April 23, 2024
Science, Biotechnology, Research, Pharmaceutical, General Health, Health, Genetics, Clinical Trials, Safety, Myology, Dystrophin-associated protein complex, CPK, NSAA, Immunohistochemistry, Duchenne, Duchenne muscular dystrophy, Disease, Patient, Ageing, Pharmaceutical industry

Today, Professor Francesco Muntoni, principal investigator of the international multicenter gene therapy trial for Duchenne Muscular Dystrophy with GNT0004 product, sponsored by Genethon, presented the innovative trial design and its initial results at the Myology 2024 international scientific congress, currently taking place in Paris.

Key Points: 
  • Today, Professor Francesco Muntoni, principal investigator of the international multicenter gene therapy trial for Duchenne Muscular Dystrophy with GNT0004 product, sponsored by Genethon, presented the innovative trial design and its initial results at the Myology 2024 international scientific congress, currently taking place in Paris.
  • The trial combines phases I/II/III with a dose escalation phase, followed by a pivotal phase at the selected dose.
  • The trial has been approved by French and UK health authorities and includes ambulant boys aged 6 to 10 suffering from Duchenne Muscular Dystrophy.
  • Note that the dose chosen is lower than those used in other gene therapy trials for Duchenne muscular dystrophy.

Edgewise Therapeutics Announces Positive Two-Year Topline Results from the ARCH Open Label Trial of Sevasemten (EDG-5506) in Adults with Becker Muscular Dystrophy (Becker)

Retrieved on: 
Monday, April 15, 2024
Biotechnology, Pharmaceutical, Health, Clinical Trials, TNNI2, Neuromuscular disease, University, Safety, Pharmacokinetics, MDA, Muscle weakness, NSAA, Duchenne, Patient, Muscular dystrophy, University of Florida, Multimedia, CK, Duchenne muscular dystrophy, Myopathy, Biomarker, Therapy, ARCH, Pharmaceutical industry

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced positive two-year topline results from the ARCH trial.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced positive two-year topline results from the ARCH trial.
  • ARCH is an open label, single-center study assessing safety, tolerability, impact on muscle damage biomarkers, pharmacokinetics (PK) and functional measures with sevasemten (EDG-5506) in adults with Becker.
  • Sevasemten is an orally administered small molecule designed to prevent contraction-induced muscle damage in dystrophinopathies including Becker and Duchenne muscular dystrophy (Duchenne).
  • ET to discuss the ARCH two-year data, and will be joined by Dr. Byrne, who will share his perspective of sevasemten and Becker.

Italfarmaco Receives FDA Approval for Duvyzat™ (givinostat) in Duchenne Muscular Dystrophy

Retrieved on: 
Friday, March 22, 2024
Research, FDA, Genetics, Clinical Trials, Other Health, Biotechnology, General Health, Pharmaceutical, Health, Science, HDAC, ITF, MAA, The Lancet, Caregiver, Disease, Magnetic resonance imaging, PPMD, Patient, Rehabilitation, Lancet Neurology, University, EMA, Doctor of Philosophy, MD, Duchenne muscular dystrophy, Pediatrics, Therapy, NSAA, DMD, Food, Pharmaceutical industry

“We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval.

Key Points: 
  • “We are grateful for the support of those living with DMD and their dedicated caregivers, which played a central role in helping us reach this landmark FDA approval.
  • The FDA approval highlights the dedication of Italfarmaco’s research and clinical teams to achieve this milestone for the company.”
    The approval is based on the results of the pivotal multicentre, randomised, double-blind, placebo-controlled phase 3 EPIDYS trial ( NCT02851797 ).
  • Italfarmaco has significantly expanded its U.S. presence through the formation of a new fully owned subsidiary, ITF Therapeutics LLC.
  • Italfarmaco has a global presence and is also working with other regulatory agencies.

Results from Italfarmaco Pivotal Phase 3 EPIDYS Study of Givinostat in Duchenne Muscular Dystrophy (DMD) Published in The Lancet Neurology

Retrieved on: 
Tuesday, March 19, 2024
Science, Biotechnology, Research, Pharmaceutical, Health, Hospitals, Genetics, Clinical Trials, VL, Civil service commission, Givinostat, Neurology, Catholic higher education, Diarrhea, MAA, The Lancet, Magnetic resonance imaging, Safety, Abdominal pain, NDA, Manuscript, Rehabilitation, Lancet Neurology, University, OLE, EMA, Degenerative disease, Doctor of Philosophy, MD, European Medicines Agency, Duchenne muscular dystrophy, European Commission, NSAA, FDA, DMD, Pediatrics, PDUFA, Pharmaceutical industry

Italfarmaco S.p.A. announced today that the full results from the Company’s pivotal phase 3 EPIDYS clinical trial with givinostat in ambulant boys 6 years of age and older with Duchenne muscular dystrophy (DMD) have been published in The Lancet Neurology.

Key Points: 
  • Italfarmaco S.p.A. announced today that the full results from the Company’s pivotal phase 3 EPIDYS clinical trial with givinostat in ambulant boys 6 years of age and older with Duchenne muscular dystrophy (DMD) have been published in The Lancet Neurology.
  • The full manuscript, titled, “Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomized, double-blind, placebo-controlled, phase 3 trial,” is published on The Lancet Neurology website .
  • Participants attended study site visits every 12 weeks for 72 weeks and were evaluated for all endpoints within the study protocol.
  • Upon completion of the 18-month double-blind period, EPIDYS study participants were eligible to enroll in an open-label extension study (OLE study) to receive givinostat on an ongoing basis.

FDA Approves Nonsteroidal Treatment for Duchenne Muscular Dystrophy

Retrieved on: 
Thursday, March 21, 2024
Research, HDAC, Neurological disorder, Walking, Abdominal pain, Dietary supplement, Risk, Inflammation, Patient, QT interval, Public, Animal, Duchenne muscular dystrophy, Diarrhea, Food, Human services, Platelet, Facial muscles, Life expectancy, Heated tobacco product, Fever, Atrophy, Safety, Division, NSAA, Muscle weakness, Cosmetics, Vaccine, Multimedia, FDA, DMD, Muscular dystrophy, Triglyceride, Pharmaceutical industry

SILVER SPRING, Md., March 21, 2024 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older.

Key Points: 
  • SILVER SPRING, Md., March 21, 2024 /PRNewswire/ -- Today, the U.S. Food and Drug Administration approved Duvyzat (givinostat) oral medication for the treatment of Duchenne Muscular Dystrophy (DMD) in patients six years of age and older.
  • Duvyzat is the first nonsteroidal drug approved to treat patients with all genetic variants of DMD.
  • "This approval provides another treatment option to help reduce the burden of this progressive, devastating disease for individuals impacted by DMD regardless of genetic mutation."
  • DMD is the most common childhood form of muscular dystrophy and typically affects males.

Catalyst Pharmaceuticals Announces Publication of Santhera Pharmaceutical’s VISION-DMD Vamorolone (AGAMREE®) Study Results in the Peer-Reviewed Journal Neurology

Retrieved on: 
Wednesday, February 21, 2024
Vamorolone, Catalyst Pharmaceuticals, NSAA, Safety, Prednisone, DMD, Growth, Duchenne muscular dystrophy, Neurology, Biomarker, BMI, Patient, LSM, JAMA Neurology, Pharmaceutical industry

Catalyst holds the exclusive rights to commercialize AGAMREE® (vamorolone) in North America.

Key Points: 
  • Catalyst holds the exclusive rights to commercialize AGAMREE® (vamorolone) in North America.
  • Based on the published study, Santhera has reported that the results of the VISION-DMD study support the long-term efficacy and safety profile of vamorolone and conclude that vamorolone was generally well tolerated, consistent with the 24-week study findings, as published previously in JAMA Neurology [2].
  • This study [VISION-DMD] was conducted to determine the efficacy and safety of vamorolone over 48 weeks and to study crossover participants (prednisone to vamorolone; placebo to vamorolone).
  • Improvements of motor outcomes seen with 6 mg/kg/d of vamorolone at 24 weeks of treatment were maintained to 48 weeks of treatment.

Edgewise Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Thursday, February 22, 2024
Health, Other Health, Clinical Trials, Pharmaceutical, Cardiology, Biotechnology, Muscular dystrophy, Research, American Heart Association, Education, R9, MESA, Ageing, Fast Track, NSAA, McArdle, DUNE, Myopathy, Therapy, WMS, LYNX, ODD, Safety, Biomarker, Pharmacokinetics, HCM, Clinical trial, Caregiver, FOX, FDA, Muscle contraction, Engagement, Duchenne muscular dystrophy, ARCH, Administration, Patient, Disease, Pharmaceutical industry

Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the fourth quarter and full year of 2023 and recent business highlights.

Key Points: 
  • Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the fourth quarter and full year of 2023 and recent business highlights.
  • “In 2023, we made tremendous progress advancing our novel muscle-targeted therapeutics in the clinic,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise.
  • Research and development (R&D) expenses were $27.7 million for the fourth quarter of 2023, compared to $23.8 million for the immediately preceding quarter.
  • General and Administrative (G&A) expenses were $6.2 million for the fourth quarter of 2023, compared to $5.7 million for the immediately preceding quarter.

Santhera Announces Publication of Efficacy, Safety and Tolerability Data with Vamorolone (AGAMREE®) in Patients with Duchenne Muscular Dystrophy in Neurology

Retrieved on: 
Wednesday, February 14, 2024
Vamorolone, NSAA, Safety, Prednisone, DMD, Growth, Duchenne muscular dystrophy, Neurology, European, Biomarker, BMI, Patient, Disease, LSM, JAMA Neurology, Pharmaceutical industry

“Vamorolone is a dissociative corticosteroid that selectively binds to the glucocorticoid receptor and has shown similar efficacy and reduced safety concerns in comparison with prednisone in Duchenne muscular dystrophy (DMD) [3].

Key Points: 
  • “Vamorolone is a dissociative corticosteroid that selectively binds to the glucocorticoid receptor and has shown similar efficacy and reduced safety concerns in comparison with prednisone in Duchenne muscular dystrophy (DMD) [3].
  • This study [VISION-DMD] was conducted to determine the efficacy and safety of vamorolone over 48 weeks and to study crossover participants (prednisone to vamorolone; placebo to vamorolone).
  • There was an increase in BMI after 24 weeks of treatment that then stabilized for both vamorolone groups.
  • Improvements of motor outcomes seen with 6 mg/kg/d of vamorolone at 24 weeks of treatment were maintained to 48 weeks of treatment.

Jazz Pharmaceuticals to Present New Data Highlighting Advancements in Solid Tumors and Rare Blood Cancers at Upcoming Oncology Meetings

Retrieved on: 
Thursday, November 30, 2023
Bone marrow, Trae Elston, Research, Safety, ASH, NACLC, Fulvestrant, Acute leukemia, International Association, MD Anderson Cancer Center, LBL, Breast cancer, ESMO, Jazz Pharmaceuticals, Patient, ASCO, MBC, Acute lymphoblastic leukemia, Conference, NSAA, Abstract, HER2, Lung cancer, Jazz, Society, ALL, HR, Pharmaceutical industry

DUBLIN, Nov. 30, 2023 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced that the Company and its partners will present two abstracts at the 2023 San Antonio Breast Cancer Symposium (SABCS) from December 5-9; 11 abstracts at the 65th Annual American Society of Hematology (ASH) Annual Meeting from December 9-12; and two abstracts at the International Association for the Study of Lung Cancer (IASLC) 2023 North America Conference on Lung Cancer (NACLC) from December 1-3. New data include updated findings from a Phase 2a trial of the investigational HER2-targeted bispecific antibody zanidatamab in combination with palbociclib and fulvestrant as a chemotherapy-free option for HER2+/ HR+ metastatic breast cancer (mBC).

Key Points: 
  • New data include updated findings from a Phase 2a trial of the investigational HER2-targeted bispecific antibody zanidatamab in combination with palbociclib and fulvestrant as a chemotherapy-free option for HER2+/ HR+ metastatic breast cancer (mBC).
  • "SABCS will also feature data from a trial of zanidatamab in neoadjuvant breast cancer.
  • An investigator-sponsored (MD Anderson Cancer Center) SABCS poster presentation featuring results of a Phase 1 trial evaluating neoadjuvant zanidatamab in patients with stage 1 node-negative HER2+ breast cancer as a single-agent chemotherapy-free option.
  • Treatment‐related adverse events (TRAEs) ≥ grade 3 occurred in 126/228 (55%) patients with no TRAEs that led to death.1
    The Jazz and partner-supported presentations at SABCS 2023 are:

Edgewise Therapeutics Reports Third Quarter 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Thursday, November 9, 2023
Biotechnology, Health, Pharmaceutical, Physical Therapy, Cardiology, LYNX, Research, FOX, DUNE, Ageing, Education, Biomarker, Muscular dystrophy, Muscular Dystrophy Association, NSAA, G&A, Myopathy, Glycogen storage disease type V, American Heart Association, WMS, R, Muscle contraction, Pharmacokinetics, Dystrophinopathy, Disorder, Glycogen, PK, Clinical trial, Duchenne muscular dystrophy, McArdle, Safety, HCM, Therapy, Patient, MESA, R9, Administration, Pharmaceutical industry, Edgewise, Duchenne, Becker

Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the third quarter of 2023 and recent business highlights.

Key Points: 
  • Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today reported financial results for the third quarter of 2023 and recent business highlights.
  • “I’m thrilled with the progress we’ve made in 2023 across our skeletal and cardiovascular programs,” said Kevin Koch, Ph.D., President and Chief Executive Officer of Edgewise.
  • Research and development (R&D) expenses were $23.8 million for the third quarter of 2023, compared to $19.6 million for the immediately preceding quarter.
  • General and Administrative (G&A) expenses were $5.7 million for the third quarter of 2023, compared to $5.8 million for the immediately preceding quarter.