Dystrophin

Wave Life Sciences Reports Fourth Quarter and Full Year 2023 Financial Results and Provides Business Update

Retrieved on: 
Wednesday, March 6, 2024

In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK.

Key Points: 
  • In the fourth quarter of 2023, Wave initiated dosing in RestorAATion-1, which resulted in a $20 million milestone payment from GSK.
  • Revenue was $29.1 million for the fourth quarter of 2023 as compared to $1.2 million in the prior year quarter.
  • Net loss was $16.3 million for the fourth quarter of 2023 as compared to $43.7 million in the prior year quarter.
  • ET to review the fourth quarter and full year 2023 financial results and pipeline updates.

PepGen Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Corporate Developments

Retrieved on: 
Wednesday, March 6, 2024

BOSTON, March 06, 2024 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today reported financial results for the fourth quarter and full year ended December 31, 2023, and highlighted recent corporate developments.

Key Points: 
  • “With important data readouts expected from our two lead programs, 2024 has the potential to be a transformational year for PepGen.
  • People with myotonic dystrophy type 1 (DM1) currently have no approved treatment options that target the root cause of the disease.
  • Financial Results for the Three Months and Twelve Months ended December 31, 2023
    Cash and cash equivalents were $110.4 million as of December 31, 2023.
  • Net loss was $78.6 million for the year ended December 31, 2023, compared to $69.1 million for the same period in 2022.

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

Retrieved on: 
Tuesday, March 5, 2024

WALTHAM, Mass., March 05, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the fourth quarter and full year 2023 and recent business highlights.

Key Points: 
  • (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today reported financial results for the fourth quarter and full year 2023 and recent business highlights.
  • Net loss: Net loss for the quarter ended December 31, 2023 was $66.6 million, or $1.09 per basic and diluted share.
  • This compares with a net loss of $38.8 million, or $0.74 per basic and diluted share, for the quarter ended December 31, 2022.
  • Net loss for the year ended December 31, 2023 was $235.9 million, or $3.95 per basic and diluted share.

Wave Life Sciences Announces Upcoming Presentations at MDA Conference that Highlight Best-in-Class Potential for WVE-N531 in Duchenne Muscular Dystrophy

Retrieved on: 
Tuesday, February 27, 2024

CAMBRIDGE, Mass., Feb. 27, 2024 (GLOBE NEWSWIRE) -- Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on unlocking the broad potential of RNA medicines to transform human health, today announced its upcoming presentations at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference, taking place March 3-6 in Orlando, FL.

Key Points: 
  • Wave’s poster presentations will highlight the best-in-class potential of WVE-N531 in Duchenne muscular dystrophy (DMD), which is currently being evaluated in the Phase 2, potentially registrational FORWARD-53 study.
  • The presentations will also illustrate the impact of Wave’s novel PN chemistry on pharmacology of its exon skipping oligonucleotides.
  • These data suggest that WVE-N531 muscle concentrations in the clinic may be higher in heart and diaphragm than in skeletal muscle.
  • “At Wave, we increasingly continue to regard exon skipping as the preferred mechanism for altering DMD disease progression in those amenable to this approach.

Sarepta Therapeutics Announces Fourth Quarter and Full-Year 2023 Financial Results and Recent Corporate Developments

Retrieved on: 
Wednesday, February 28, 2024

Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2023.

Key Points: 
  • Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today reported financial results for the fourth quarter and full-year 2023.
  • Beginning in the fourth quarter of 2023, amortization of in-licensed rights (formerly included within depreciation and amortization expense) and income tax (benefit) expense are no longer excluded from the non-GAAP results.
  • Non-GAAP financial results for the fourth quarter and full-year 2022 have been updated to reflect this change for comparability.
  • All relevant non-GAAP measures are reconciled from their respective GAAP measures in the attached table “Reconciliation of GAAP Financial Measures to Non-GAAP Financial Measures.”

Avidity Biosciences Receives FDA Rare Pediatric Disease Designation for AOC 1044 for Treatment of Duchenne Muscular Dystrophy in People with Mutations Amenable to Exon 44 Skipping

Retrieved on: 
Tuesday, February 20, 2024

In addition to receiving Rare Pediatric Disease Designation, AOC 1044 has been granted Orphan Designation by the FDA and the European Medicines Agency (EMA), and Fast Track Designation by the FDA.

Key Points: 
  • In addition to receiving Rare Pediatric Disease Designation, AOC 1044 has been granted Orphan Designation by the FDA and the European Medicines Agency (EMA), and Fast Track Designation by the FDA.
  • "We are pleased that the FDA has granted Rare Pediatric Disease designation to AOC 1044, adding to the Orphan Drug and Fast Track designations already granted.
  • Avidity plans to provide a first look at AOC 1044 data in people living with DMD44 in 2H 2024.
  • The EXPLORE44 trial is a randomized, placebo-controlled, double-blind, Phase 1/2 clinical trial to evaluate AOC 1044 in healthy volunteers and participants with DMD mutations amenable to exon 44 skipping (DMD44).

Satellos Announces Promising Preliminary Data in Facioscapulohumeral Muscular Dystrophy

Retrieved on: 
Tuesday, February 13, 2024

Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSXV: MSCL) (OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today positive preliminary data showing SAT-3247 can improve skeletal muscle function in a mouse model of facioscapulohumeral muscular dystrophy (FSHD).

Key Points: 
  • Satellos Bioscience Inc. (“Satellos” or the “Company”) (TSXV: MSCL) (OTCQB: MSCLF), a public biotech company developing new small molecule therapeutic approaches to improve the treatment of muscle diseases and disorders, announced today positive preliminary data showing SAT-3247 can improve skeletal muscle function in a mouse model of facioscapulohumeral muscular dystrophy (FSHD).
  • We look forward to presenting these data at the MDA Clinical and Scientific Conference 2024 and advancing SAT-3247 into clinical trials for Duchenne muscular dystrophy mid-year.”
    Neil Camarta, Co-Founder and CEO of the FSHD Canada Foundation, said “I’m very pleased.
  • I’m looking forward to working with Satellos to accelerate the development of this promising drug candidate, because time is muscle!”
    FSHD is the third most common muscular dystrophy behind Duchenne (& Beckers) and myotonic dystrophy.
  • FSHD is an adult onset muscular dystrophy that results in the progressive destruction of muscle tissue, owed to the erroneous expression of a gene product called DUX4.

EMA confirms recommendation for non-renewal of authorisation of Duchenne muscular dystrophy medicine Translarna

Retrieved on: 
Sunday, February 4, 2024

This medicine is used for treating patients with Duchenne muscular dystrophy whose disease is caused by a type of genetic defect called a ‘nonsense mutation’ in the dystrophin…

Key Points: 


This medicine is used for treating patients with Duchenne muscular dystrophy whose disease is caused by a type of genetic defect called a ‘nonsense mutation’ in the dystrophin…

Sarepta Therapeutics Announces Positive Data from Part B of MOMENTUM, a Phase 2 Study of SRP-5051 in Patients with Duchenne Muscular Dystrophy Amenable to Skipping Exon 51

Retrieved on: 
Monday, January 29, 2024

SRP-5051 is a next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.

Key Points: 
  • SRP-5051 is a next-generation peptide phosphorodiamidate morpholino oligomer (PPMO) treatment for patients with Duchenne muscular dystrophy who are amenable to exon 51 skipping.
  • Consistent dystrophin expression was seen in ambulatory (4.76%, n=11) and non-ambulatory (5.67%, n=9) participants at 28 weeks.
  • Hypomagnesemia has previously been identified in patients taking SRP-5051 and was managed and monitored through prophylactic magnesium supplementation as part of the study protocol.
  • Hypomagnesemia has been seen in earlier clinical studies of SRP-5051 and, throughout MOMENTUM Part B, supplemental magnesium was administered prophylactically.

Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients Demonstrating Promise of the FORCE™ Platform in Developing Therapeutics for Rare Muscle Diseases

Retrieved on: 
Wednesday, January 3, 2024

“We are excited that Dyne’s first clinical data in two programs have demonstrated proof-of-concept and validated the promise of the FORCE™ platform in developing targeted therapeutics for rare muscle diseases.

Key Points: 
  • “We are excited that Dyne’s first clinical data in two programs have demonstrated proof-of-concept and validated the promise of the FORCE™ platform in developing targeted therapeutics for rare muscle diseases.
  • Dyne anticipates providing its next clinical data update from the ACHIEVE trial in the second half of 2024.
  • Key initial findings from DELIVER include:
    Muscle delivery: DYNE-251 showed a mean 657 ng/g PMO muscle drug concentration at 6 months.
  • Dyne anticipates providing its next clinical data update from the DELIVER trial in the second half of 2024.