QMT

Dyne Therapeutics Announces New Clinical Data from ACHIEVE Trial of DYNE-101 in DM1 and DELIVER Trial of DYNE-251 in DMD Demonstrating Compelling Impact on Key Disease Biomarkers and Improvement in Multiple Functional Endpoints

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星期一, 五月 20, 2024
CDER, PMO, FSHD, FDA, Dyne, Therapy, Eteplirsen, Facial muscles, Duchenne muscular dystrophy, Dystrophin, MPH, Food, DYN, Clinical trial, Disease, Safety, Event, Patient, Facioscapulohumeral muscular dystrophy, DM1, Male, Update, DMD, PMOS, Biomarker, QMT, Fatigue, Central nervous system, Pharmaceutical industry

New data from both trials demonstrated compelling impact on key disease biomarkers as well as improvement in multiple functional endpoints and favorable safety profiles.

Key Points: 
  • New data from both trials demonstrated compelling impact on key disease biomarkers as well as improvement in multiple functional endpoints and favorable safety profiles.
  • “We are excited to report new clinical data from both our ACHIEVE and DELIVER trials demonstrating meaningful impact on key biomarkers and functional improvement in multiple clinical endpoints that matter to patients.
  • Our robust preclinical work is translating into clinical benefit along with favorable safety profiles for both DYNE-101 and DYNE-251.
  • In the ACHIEVE trial, DYNE-101 demonstrated robust muscle delivery and dose-dependent, consistent splicing correction while also showing improvement in multiple functional endpoints and patient reported outcomes.

Avidity Biosciences Receives FDA Breakthrough Therapy Designation for Delpacibart Etedesiran (AOC 1001) for Treatment of Myotonic Dystrophy Type 1

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星期三, 五月 8, 2024
FDA, Food, RNA, QMT, Initiation, Facial muscles, Natural history, AOC, Therapy, Myopathy, European Medicines Agency, EMA, DM1, Avidity, Breakthrough therapy, SAN, Pharmaceutical industry

SAN DIEGO, May 8, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to delpacibart etedesiran (AOC 1001), the company's lead clinical development program, for the treatment of myotonic dystrophy type 1 (DM1). Delpacibart etedesiran, abbreviated as del-desiran, is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.

Key Points: 
  • Delpacibart etedesiran, abbreviated as del-desiran, is an investigational treatment designed to address the root cause of DM1, an underrecognized, progressive and often fatal neuromuscular disease with no approved therapies.
  • "We are pleased that the FDA has granted Breakthrough Therapy designation to del-desiran for myotonic dystrophy type 1, underscoring the potential of del-desiran to be an effective treatment and the urgency of bringing this treatment to people living with DM1," said Sarah Boyce, president and chief executive officer at Avidity.
  • Avidity is initiating the global pivotal HARBOR™ study of del-desiran this quarter.
  • In addition to receiving FDA Breakthrough Therapy designation, del-desiran has previously been granted Orphan Drug and Fast Track designations by the FDA and Orphan designation by the European Medicines Agency (EMA) for the treatment of DM1.

Avidity Biosciences Announces Positive AOC 1001 Long-term Data Showing Reversal of Disease Progression in People Living with Myotonic Dystrophy Type 1 Across Multiple Endpoints; Same Key Endpoints Agreed for Phase 3 HARBOR™ Trial

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星期一, 三月 4, 2024
Therapy, Division, PD, PRO, Natural history, Doctor of Philosophy, Myotonic dystrophy, MD, Friends, Neurology, Hope, Neuromuscular medicine, Conference, DM1, Facial muscles, Safety, Muscular Dystrophy Association, Biomarker, RNA, Webcast, Patient, Elbow, Family, Disease, QMT, Pediatrics, AOC, Publication, MDA, Volume Eight, Episcopal conference, Partnership, Pharmaceutical industry

SAN DIEGO, March 4, 2024 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced new positive long-term AOC 1001 data from the MARINA open-label extension (MARINA-OLE™) trial showing reversal of disease progression in people living with myotonic dystrophy type 1 (DM1) across multiple endpoints including vHOT, muscle strength and activities of daily living when compared to END-DM1 natural history data. These endpoints are the same key endpoints that will be used in the global Phase 3 HARBOR™ trial for people living with DM1. The primary endpoint in the Phase 3 HARBOR trial is video hand opening time (vHOT), and key secondary endpoints include muscle strength as measured by hand grip strength and quantitative muscle testing (QMT) total score, and activities of daily living as measured by DM1-Activ. Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024.

Key Points: 
  • These endpoints are the same key endpoints that will be used in the global Phase 3 HARBOR™ trial for people living with DM1.
  • Avidity is accelerating the global Phase 3 HARBOR trial initiation to the second quarter of 2024.
  • Avidity also announced delpacibart etedesiran as the approved international nonproprietary name of AOC 1001, abbreviated as del-desiran.
  • ET to discuss new positive long-term del-desiran (AOC 1001) data from the MARINA-OLE™ trial in people living with DM1.