Oral Presentation at FSHD IRC highlights preclinical data that demonstrated prevention of muscle weakness by reducing DUX4 expression.
| Oral Presentation at FSHD IRC highlights preclinical data that demonstrated prevention of muscle weakness by reducing DUX4 expression AOC 1020 on track to be in the clinic by end of 2022 for the treatment of FSHD SAN DIEGO, June 17, 2022 /PRNewswire/ -Avidity Biosciences Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), joins in activities to raise awareness for Facioscapulohumeral muscular dystrophy (FSHD) in support of World FSHD Day and highlights preclinical results supporting AOC 1020 for the treatment of FSHD at the 29th Annual FSHD Society International Research Congress (FSHD IRC) in Orlando, Florida. Currently, there are no approved therapies for the treatment of FSHD.
“We are grateful for our partnership with the global FSHD community as we collectively work together toward developing treatments for one of the most common forms of muscular dystrophy. We continue to work closely with the community to advance AOC 1020 for the treatment of FSHD into the clinic by the end of this year,” said Sarah Boyce, president and chief executive officer. FSHD is a rare, hereditary muscle-weakening condition that affects approximately 16,000-38,000 people in the United States. It is marked by life-long, progressive loss of muscle function and causes significant pain, fatigue, and disability. FSHD is an autosomal dominant disease caused by the abnormal expression of DUX4 (double homeobox 4), a gene involved in embryonic development but not typically expressed in muscles. This abnormal expression of DUX4 leads to a series of downstream events that result in skeletal muscle wasting and compromised muscle function with onset often in teenage and early adult years. “Our preclinical data, presented at FSHD IRC, support our approach of directly targeting DUX4 with AOC 1020. The data demonstrate that a murine version of AOC 1020 prevents muscle weakness by blocking expression of DUX4 in a FSHD mouse model of disease. These findings are particularly important for progressive diseases like FSHD where aberrant and sporadic expression of DUX4 leads to cumulative damage across different muscle groups,” said W. Michael Flanagan, Ph.D., Avidity’s chief technical officer. Every June 20th, people around the world join in activities to raise awareness for FSHD through World FSHD Day and to recognize patients and families around the world who are affected by FSHD. To mark FSHD awareness day, Avidity is participating in the FSHD IRC as well as the FSHD Connect Conference, an educational conference specifically for people living with FSHD and their families. Both conferences are being held in Orlando, Florida and organized by the FSHD Society, the world’s largest research-focused patient organization for FSHD. Oral Presentation at FSHD IRC Key highlights from the oral presentation include:
Avidity is also presenting data from a real-world data analysis of patients with FSHD before and after their diagnosis. The results highlight the increased morbidity and medical and financial burden patients face as they progress through their journey living with FSHD. The presentations will be available later today on Avidity’s website at https://www.aviditybiosciences.com on the Publications page. About Facioscapulohumeral muscular dystrophy (FSHD) About Avidity Forward-Looking Statements Avidity cautions readers that statements contained in this press release regarding matters that are not historical facts are forward-looking statements. These statements are based on the company’s current beliefs and expectations. Such forward-looking statements include, but are not limited to, statements regarding: the progression of the AOC 1020 pre-clinical or clinical program and timing of a planned clinical trial; the pharmacological activity or duration of action of AOC 1020 in a clinical trial; and the development of treatments for muscular dystrophy. The inclusion of forward-looking statements should not be regarded as a representation by Avidity that any of these plans will be achieved. Actual results may differ from those set forth in this press release due to the risks and uncertainties inherent in the business, including, without limitation: Avidity is early in its development efforts; Avidity’s approach to the discovery and development of product candidates based on its AOC platform is unproven, and the company does not know whether it will be able to develop any products of commercial value; potential delays in the commencement, enrollment and completion of clinical trials; disruption to its operations from the COVID-19 pandemic or the war in Ukraine; the success of its preclinical studies and clinical trials for the company’s product candidates; the results of preclinical studies and early clinical trials are not necessarily predictive of future results; Avidity’s dependence on third parties in connection with preclinical testing and product manufacturing; unexpected adverse side effects or inadequate efficacy of its product candidates that may limit their development, regulatory approval and/or commercialization, or may result in recalls or product liability claims; regulatory developments in the United States and foreign countries, including acceptance of INDs and similar foreign regulatory filings and the proposed design of future clinical trials; Avidity could use its available capital resources sooner than it currently expects; and other risks described in prior press releases and in filings with the Securities and Exchange Commission (SEC). Avidity cautions readers not to place undue reliance on these forward-looking statements, which speak only as of the date hereof, and the company undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement, which is made under the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Company Contact: Media Contact:
SOURCE Avidity Biosciences, Inc. | ||
Company Codes: NASDAQ-NMS:RNA |
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