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Company has three distinct rare disease programs in clinical development - myotonic dystrophy type 1 (DM1), facioscapulohumeral muscular dystrophy (FSHD), and Duchenne muscular dystrophy (DMD)
On track for preliminary assessment of AOC 1001 MARINA™ trial in fourth quarter
SAN DIEGO, Nov. 8, 2022 /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 financial results for the third quarter ended September 30, 2022 and highlighted recent corporate progress.
"Consistent with our guidance for 2022, we have progressed three programs addressing three distinct rare diseases into clinical development, and we remain on track for our preliminary assessment of the MARINA™ trial this quarter," said Sarah Boyce, president and chief executive officer. "Our team continues to execute on our plan as we advance AOC 1020 for FSHD in the FORTITUDE™ trial and AOC 1044 for DMD in the EXPLORE44™ trial. With close to 40 participants enrolled in the MARINA trial, we continue to gather data on AOC 1001 as we work to resolve the recent partial clinical hold on new participant enrollment as swiftly as possible."
"With approximately $425 million, inclusive of additional funds raised subsequent to September 30th, we have cash runway through 2024 allowing us to invest in the expansion of our AOC platform and our three clinical stage programs addressing rare skeletal muscle diseases," said Mike MacLean, chief financial and chief business officer.
Recent Highlights
Third Quarter 2022 Financial Results
About Avidity
Avidity Biosciences, Inc.'s mission is to profoundly improve people's lives by delivering a new class of RNA therapeutics - Antibody Oligonucleotide Conjugates (AOCs™). Avidity's proprietary AOCs are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to target the root cause of diseases previously untreatable with RNA therapeutics. Avidity's advancing and expanding pipeline has three programs in clinical development. AOC 1001 is designed to treat people with myotonic dystrophy type 1 (DM1) and is currently in Phase 1/2 development with the ongoing MARINA™ and MARINA-OLE™ trials. AOC 1020 is designed to treat people living with facioscapulohumeral muscular dystrophy (FSHD) and is currently in Phase 1/2 development with the FORTITUDE™ trial. AOC 1044 is designed for people with Duchenne muscular dystrophy (DMD) mutations amenable to exon 44 skipping and is currently in Phase 1/2 development with the EXPLORE44™ trial. AOC 1044 is the first of multiple AOCs the company is developing for DMD. Avidity is also broadening the reach of AOCs beyond muscle tissues through both internal discovery efforts and key partnerships as the company continues to deliver on the RNA revolution. Avidity is headquartered in San Diego, CA. For more information about our science, pipeline and people, please visit www.aviditybiosciences.com and engage with us on LinkedIn and Twitter.
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: expectations related to Avidity's ability to resolve the partial clinical hold and resume enrollment in and complete the MARINA™ study, the continuation of participants in the MARINA trial and enrollment of participants into the MARINA-OLE™, and the reporting of data from the preliminary assessment of the MARINA study and the timing thereof; the progression of clinical programs for AOC 1001, AOC 1044 and AOC 1020 and the timing thereof; the initiation of the FORTITUDE™ and EXPLORE44™ trials; the potential of Avidity's product candidates to treat rare diseases; Avidity's plans to expand its AOC platform into additional muscle diseases; and the sufficiency of the company's current financial position to fund its development programs, investments in its pipeline and platform, and operations into 2024. 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 may not be able to resolve the partial clinical hold and the analysis related to the underlying cause of the serious adverse event may result in delays in the MARINA study or an inability to compete the study; unexpected adverse side effects or inadequate efficacy of its product candidates that may delay or limit their development, regulatory approval and/or commercialization, or may result in clinical holds, recalls or product liability claims; 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 preclinical studies or clinical trials; 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 and clinical testing and product manufacturing; 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; disruption to its operations from the COVID-19 pandemic or the war in Ukraine; 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.
Investor Contact:
Kathleen Gallagher
(858) 401-7900 x550
investors@aviditybio.com
Media Contact:
Navjot Rai
(858) 401-7900 x550
media@aviditybio.com
Avidity Biosciences, Inc. | |||||||||||||||||||||||||
Selected Condensed Financial Information | |||||||||||||||||||||||||
(in thousands, except per share data) | |||||||||||||||||||||||||
(unaudited) | |||||||||||||||||||||||||
Statements of Operations | Three Months Ended September 30, | Nine Months Ended September 30, | |||||||||||||||||||||||
2022 | 2021 | 2022 | 2021 | ||||||||||||||||||||||
Collaboration revenue | $ | 2,482 | $ | 2,163 | $ | 6,455 | $ | 7,474 | |||||||||||||||||
Operating expenses: | |||||||||||||||||||||||||
Research and development | 37,317 | 24,831 | 104,794 | 68,214 | |||||||||||||||||||||
General and administrative | 10,094 | 6,612 | 27,349 | 18,764 | |||||||||||||||||||||
Total operating expenses | 47,411 | 31,443 | 132,143 | 86,978 | |||||||||||||||||||||
Loss from operations | (44,929) | (29,280) | (125,688) | (79,504) | |||||||||||||||||||||
Other income (expense), net | 1,330 | 6 | 2,164 | 33 | |||||||||||||||||||||
Net loss | $ | (43,599) | $ | (29,274) | $ | (123,524) | $ | (79,471) | |||||||||||||||||
Net loss per share, basic and diluted | $ | (0.82) | $ | (0.68) | $ | (2.45) | $ | (2.01) | |||||||||||||||||
Weighted-average shares outstanding, basic and diluted | 53,069 | 43,265 | 50,432 | 39,477 | |||||||||||||||||||||
Balance Sheets | September 30, | December 31, | |||||||||||||||||||||||
2022 | 2021 | ||||||||||||||||||||||||
Assets | |||||||||||||||||||||||||
Current assets: | |||||||||||||||||||||||||
Cash, cash equivalents and marketable securities | $ | 405,548 | $ | 405,543 | |||||||||||||||||||||
Prepaid and other assets | 8,916 | 5,598 | |||||||||||||||||||||||
Total current assets | 414,464 | 411,141 | |||||||||||||||||||||||
Property and equipment, net | 6,037 | 4,805 | |||||||||||||||||||||||
Restricted cash | 251 | 251 | |||||||||||||||||||||||
Right-of-use asset | 9,264 | 10,784 | |||||||||||||||||||||||
Other assets | 523 | 599 | |||||||||||||||||||||||
Total assets | $ | 430,539 | $ | 427,580 | |||||||||||||||||||||
Liabilities and Stockholders' Equity | |||||||||||||||||||||||||
Current liabilities: | |||||||||||||||||||||||||
Accounts payable and other liabilities | $ | 37,212 | $ | 24,794 | |||||||||||||||||||||
Deferred revenue, current portion | 5,454 | 4,864 | |||||||||||||||||||||||
Total current liabilities | 42,666 | 29,658 | |||||||||||||||||||||||
Lease liabilities, net of current portion | 8,185 | 9,960 | |||||||||||||||||||||||
Deferred revenue, net of current portion | 2,706 | 6,532 | |||||||||||||||||||||||
Total liabilities | 53,557 | 46,150 | |||||||||||||||||||||||
Stockholders' equity | 376,982 | 381,430 | |||||||||||||||||||||||
Total liabilities and stockholders' equity | $ | 430,539 | $ | 427,580 | |||||||||||||||||||||
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SOURCE Avidity Biosciences, Inc.