DM1

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.

PepGen Receives U.S. FDA Fast Track Designation for PGN-EDODM1 for the Treatment of Myotonic Dystrophy Type 1

Retrieved on: 
Tuesday, February 20, 2024

“Receiving Fast Track designation from the FDA for PGN-EDODM1 is a significant milestone in our efforts to deliver a potentially transformative therapy to the DM1 community,” said James McArthur, Ph.D., President and CEO of PepGen.

Key Points: 
  • “Receiving Fast Track designation from the FDA for PGN-EDODM1 is a significant milestone in our efforts to deliver a potentially transformative therapy to the DM1 community,” said James McArthur, Ph.D., President and CEO of PepGen.
  • Once a drug candidate receives Fast Track designation, early and frequent communication between the FDA and the drug company is encouraged throughout the entire drug development and regulatory review process.
  • For more information about Fast Track designation, please visit the FDA website ( www.fda.gov ).
  • (ClinicalTrials.gov identifier: NCT06204809)
    The Company previously announced that the FDA granted Orphan Drug Designation to PGN-EDODM1 in September 2023.

Arrowhead Pharmaceuticals Initiates Phase 1/2a Study of ARO-DM1 for Treatment of Type 1 Myotonic Dystrophy

Retrieved on: 
Friday, March 8, 2024

Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities.

Key Points: 
  • Patients with DM1 have muscle weakness and wasting, myotonia, cataracts, and often develop cardiac conduction abnormalities.
  • Pathogenesis of DM1 is driven by an expanded CUG trinucleotide repeat in the 3’-untranslated region of DMPK transcripts.
  • These abnormal transcripts cause mis-regulated splicing, known as spliceopathy, for certain messenger RNAs which are directly linked to the clinical manifestations of DM1.
  • Presentation materials may be accessed on the Events and Presentations page under the Investors section of the Arrowhead website.

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

Retrieved on: 
Monday, March 4, 2024

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.

Avidity Biosciences Honors Rare Disease Day®

Retrieved on: 
Thursday, February 29, 2024

"Today, on Rare Disease Day, we are proud to join the global community in raising awareness for people living with rare diseases," said Sarah Boyce, president and chief executive officer at Avidity.

Key Points: 
  • "Today, on Rare Disease Day, we are proud to join the global community in raising awareness for people living with rare diseases," said Sarah Boyce, president and chief executive officer at Avidity.
  • "Recently, we had the privilege of hosting individuals affected by rare muscle diseases at Avidity.
  • Rare Disease Day takes place on the last day of February each year with the goal to raise awareness of the impact of rare diseases worldwide.
  • EURORDIS established Rare Disease Day in 2008 and coordinates with more than 70 national alliance patient organizations each year to honor those living with rare diseases as well as their families and caregivers.

Avidity Biosciences Announces Oversubscribed $400 Million Private Placement

Retrieved on: 
Thursday, February 29, 2024

SAN DIEGO, Feb. 29, 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 it has agreed to sell 15,224,773 shares of its common stock at a price of $16.50 per share and pre-funded warrants to purchase 9,030,851 shares of its common stock at a price per pre-funded warrant of $16.4990 to a select group of institutional and accredited healthcare specialist investors in an oversubscribed private placement. The pre-funded warrants have an exercise price of $0.001 per share, will be immediately exercisable and will not expire.

Key Points: 
  • Avidity anticipates the gross proceeds from the private placement to be approximately $400 million, before deducting any offering-related fees and expenses.
  • The private placement is expected to close on or about March 4, 2024, subject to customary closing conditions.
  • Leerink Partners, BofA Securities, Cantor and Chardan acted as placement agents for the private placement.
  • Avidity has agreed to file a registration statement with the U.S. Securities and Exchange Commission (the SEC) registering the resale of the shares of common stock issued in the private placement and the shares of common stock issuable upon the exercise of the pre-funded warrants issued in the private placement no later than the 30th day after the closing of the private placement.

ARTHEx Biotech Receives IND Clearance from FDA to Initiate the Phase I-IIa ArthemiR™ Trial of ATX-01 for Myotonic Dystrophy Type 1 (DM1)

Retrieved on: 
Wednesday, February 28, 2024

VALENCIA, Spain, Feb. 28, 2024 /PRNewswire/ -- ARTHEx Biotech S.L., a clinical-stage biotechnology company focused on developing innovative medicines through the modulation of microRNAs, announced that the U.S. Food and Drug Administration (FDA) cleared the Company to initiate the Phase I-IIa ArthemiR™ study of ATX-01 for the treatment of Myotonic Dystrophy Type 1 (DM1). 

Key Points: 
  • The ArthemiR™ trial is a Phase I-IIa double-blind, placebo-controlled, dose escalation study expected to enroll participants with classic Myotonic Dystrophy Type 1 (DM1).
  • ARTHEx will also investigate target engagement at the muscle level through biomarkers, including MBNL levels and splicing index.
  • In addition, the clinical endpoints from the trial will include measures related to muscle function, patient-reported outcomes, and quality of life measures.
  • Patients are eager for new trials, and we are delighted to start enrolment in the coming months."

Rgenta Therapeutics to Present at the 19th Annual Huntington's Disease Therapeutics Conference

Retrieved on: 
Wednesday, February 21, 2024

CAMBRIDGE, Mass., Feb. 21, 2024 /PRNewswire/ -- Rgenta Therapeutics ("Rgenta" or the "Company") today announced it will participate at the 19th Annual Huntington's Disease Therapeutics Conference being held February 26-29, 2024, in Palm Springs, California.

Key Points: 
  • CAMBRIDGE, Mass., Feb. 21, 2024 /PRNewswire/ -- Rgenta Therapeutics ("Rgenta" or the "Company") today announced it will participate at the 19th Annual Huntington's Disease Therapeutics Conference being held February 26-29, 2024, in Palm Springs, California.
  • As part of the annual conference, Travis Wager, Ph.D., President & CSO of Rgenta will participate in the Targeting the DNA repair machinery to modulate somatic instability session on Tuesday, February 27, 2024 at 2:00 PM PT.
  • To date, there has been a lack of successful strategies for directly targeting the PMS1 protein with small molecule inhibitors.
  • For more information about the event, please visit the conference website .

Avidity Biosciences to Present New AOC 1001 Long-term Efficacy and Safety Data from MARINA-OLE™ Trial in People Living with Myotonic Dystrophy Type 1 (DM1) at 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference

Retrieved on: 
Thursday, February 15, 2024

SAN DIEGO, Feb. 15, 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 company will be presenting multiple posters from all three clinical development programs in rare muscle diseases, including new AOC 1001 long-term efficacy and safety data from the MARINA open-label extension (MARINA-OLE™) trial in people living with myotonic dystrophy type 1 (DM1) at the 2024 Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 3-6, 2024, in Orlando, Florida.

Key Points: 
  • ET for a live video webcast event, which will be available on the company's website.
  • The company is hosting Volume 8 of its investor and analyst event series on March 4, 2024, beginning at 8:00 a.m.
  • ET to discuss new AOC 1001 long-term efficacy and safety data from the MARINA-OLE™ trial in people living with DM1.
  • A replay of the webcast will be archived on Avidity's website following the event.