DM1 | Jotup

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

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금요일, 3월 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

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월요일, 3월 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®

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목요일, 2월 29, 2024

Rare disease, Therapy, SAN, Avidity, Caregiver, DMD, Duchenne muscular dystrophy, Facioscapulohumeral muscular dystrophy, Hope, Learning, DM1, RNA, Rare Disease Day, FSHD, Patient, Disease, AOC, Myopathy, Research

"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

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목요일, 2월 29, 2024

Janus, Therapy, Prospectus, Wellington Management Company, Investment, Sale, Exercise, DM1, Safety, SVB Securities, RNA, Webcast, BofA Securities, AOC, Volume Eight, Research, Security (finance)

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)

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수요일, 2월 28, 2024

Research, MicroRNA, Messenger, FDA, Quality of life, DMPK, Food, Virginia Commonwealth University, DM1, Safety, Biomarker, MBNL, Muscle weakness, Myotonic dystrophy, Patient, Disease, Phase, Pharmaceutical industry

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

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수요일, 2월 21, 2024

Traffic barrier, MLH1, PMS2, Huntington's disease, GWAS, FXS, Conference, DM1, MSH3, PMS1, RNA, Therapy, FAN1, Gene, FRDA, CSO, Fragile X syndrome, Friedreich's ataxia, DNA, Vaccine

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

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목요일, 2월 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.

Dyne Therapeutics Announces Upcoming Presentations on Initial Clinical Data From its ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients at the 2024 Muscular Dystrophy Association Clinical & Scientific Conference

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수요일, 2월 14, 2024

Therapy, Nationwide, Principal, DYN, DMD, Duchenne muscular dystrophy, Conference, DM1, Gene therapy, Safety, Muscular Dystrophy Association, Patient, MPH, Asian literature, PMO, Myopathy, MDA, Episcopal conference, Pharmaceutical industry

WALTHAM, Mass., Feb. 14, 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 announced two oral presentations at the Muscular Dystrophy Association (MDA) Clinical & Scientific Conference being held March 3-6, 2024, in Orlando, Florida and virtually.

Key Points:

Both presentations will highlight the initial clinical data from the ACHIEVE and DELIVER trials reported by Dyne in January 2024.
The initial clinical data from the ACHIEVE trial of DYNE-101 in patients with myotonic dystrophy type 1 (DM1) includes 6-month data from the 1.8 mg/kg (approximate ASO dose) cohort (n=16) and 3-month data from the 3.4 mg/kg Q4W cohort (n=16).
Initial 6-month clinical data from the 5 mg/kg (approximate PMO dose) cohort (n=6) of the DELIVER trial of DYNE-251 in patients with Duchenne muscular dystrophy (DMD) who are amenable to exon 51 skipping will also be presented.
Additionally, both presentations will include safety data from multiple cohorts in ACHIEVE and DELIVER trials which was shared in January.

Juvena Therapeutics Receives FDA Orphan Drug Designation for JUV-161 for the Treatment of Myotonic Dystrophy Type 1

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화요일, 1월 23, 2024

Juvena Therapeutics , a biotechnology company unlocking the potential of stem cell-secreted proteins to treat muscle and metabolic diseases including muscular dystrophies and obesity, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation to the company’s flagship candidate, JUV-161.

Key Points:

Juvena Therapeutics , a biotechnology company unlocking the potential of stem cell-secreted proteins to treat muscle and metabolic diseases including muscular dystrophies and obesity, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug designation to the company’s flagship candidate, JUV-161.
JUV-161 is an investigational therapeutic for the treatment of Myotonic Dystrophy Type 1 (DM1), a rare, multi-systemic, autosomal dominant inherited disease and the most common form of adult muscular dystrophy.
“People living with DM1 deserve safe, effective, and rejuvenating treatments that can repair and restore tissue health to improve muscle function and metabolism,” said Dr. Hanadie Yousef, Co-Founder and CEO of Juvena Therapeutics.
Additionally, Juvena has identified multiple secreted protein hits that induce disease-modifying effects across several metabolic organs and therapeutic areas.

Avidity Biosciences Announces 2024 Corporate Priorities and Catalysts for Next Stage of Growth

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금요일, 1월 5, 2024

RNA, Marketing, Cardiology, Facioscapulohumeral muscular dystrophy, Research, DM1, Duchenne muscular dystrophy, SAN, Facial muscles, Therapy, FSHD, AOC, University, DSM-IV codes, Myotonia, Growth, Safety, Senior, Nursing

SAN DIEGO, Jan. 5, 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 its 2024 corporate priorities and catalysts for the next stage of growth. In mid-2024, Avidity plans to initiate the global Phase 3 HARBOR™ trial of AOC 1001 for adults living with myotonic dystrophy type 1 (DM1). The robust data package of AOC 1001 from the Phase 1/2 MARINA® trial and open-label extension study, MARINA-OLE™, has demonstrated consistent improvements across multiple functional endpoints including myotonia, muscle strength and mobility, and long-term favorable safety and tolerability results in people living with DM1. Avidity also plans to report data in 2024 from all three of its ongoing clinical programs targeting three distinct rare muscle diseases: DM1, Duchenne muscular dystrophy with mutations amenable to exon 44 skipping (DMD44) and facioscapulohumeral muscular dystrophy (FSHD) while advancing its cardiology programs toward clinical development. In addition, Avidity announced the appointment of Eric B. Mosbrooker as Chief Strategy Officer.

Key Points:

In mid-2024, Avidity plans to initiate the global Phase 3 HARBOR™ trial of AOC 1001 for adults living with myotonic dystrophy type 1 (DM1).
In addition, Avidity announced the appointment of Eric B. Mosbrooker as Chief Strategy Officer.
Prior to joining Avidity, Mr. Mosbrooker served as the Chief Operations Officer for Cognoa, where he spearheaded commercial initiatives, program management, product development, and business operations.
Mr. Mosbrooker holds a Bachelor of Science in Industrial Engineering from the University of Wisconsin – Madison.