Avidity Biosciences Reports Fourth Quarter and Year-End 2023 Financial Results and Recent Highlights
- Avidity is well-positioned financially with $595 million at the end of 2023 and a cash runway through 2025.
- The company plans to initiate the global Phase 3 HARBOR™ trial for AOC 1001 for DM1 and share data from clinical programs for FSHD and DMD44.
- Avidity is revolutionizing RNA delivery with their AOC technology and aims to bring potential new treatments to people living with rare diseases.
- The collaboration with Bristol Myers Squibb focuses on cardiovascular targets and includes potential milestone payments of up to $2.2 billion.
- Key appointments like Eric B. Mosbrooker as Chief Strategy Officer highlight Avidity's organizational growth.
- Financially, collaboration revenue totaled $2.2 million for Q4 2023, R&D expenses increased due to advancements in AOC programs, and G&A expenses rose to support expanded operations.
- None.
Insights
The robust financial position of Avidity Biosciences, with $595.4 million in cash, cash equivalents and marketable securities, provides the company with a solid foundation to advance its clinical programs and pipeline. This financial cushion is critical as the company's R&D expenses have increased significantly, from $150.4 million in the full year 2022 to $191.0 million in 2023. The increase in R&D spending is a direct reflection of the company's commitment to advancing its AOC 1001, AOC 1020 and AOC 1044 programs. Investors should note the cash runway through 2025, indicating that the company is funded for its near-term operations without the immediate need for additional capital raising, which could dilute existing shareholders.
Avidity's progress in advancing AOC 1001 into a global Phase 3 trial for DM1 is a significant milestone. The long-term efficacy and safety data from the MARINA-OLE trial, to be presented at the MDA Clinical & Scientific Conference, will be crucial in assessing the drug's potential market impact. The Orphan and Fast Track designations for AOC 1020 and AOC 1044 by the FDA and EMA suggest a regulatory acknowledgment of the unmet medical need and could result in expedited review processes. The positive data from early-stage trials may increase the likelihood of successful Phase 3 outcomes, which is a critical inflection point for biopharmaceutical companies in terms of stock valuation and partnership opportunities.
Avidity's expanded collaboration with Bristol Myers Squibb, with $100 million upfront and potential milestone payments up to $2.2 billion, is indicative of the industry's interest in AOC technology and its potential applications. The cardiovascular targets, in particular, represent a significant market opportunity. Investors should be aware of the competitive landscape and the potential for Avidity to capture market share with its novel RNA therapeutics, especially given the rare disease focus which often commands high drug prices and can lead to substantial revenue streams upon successful product launches.
Avidity is on-track to initiate global Phase 3 HARBOR™ trial of AOC 1001 for DM1 in mid-2024
Company to share first-look at long-term efficacy and safety data from MARINA-OLE™ trial in people living with DM1 at MDA Clinical & Scientific Conference and via webcast on March 4, 2024
Avidity to report data from clinical programs for people living with FSHD and DMD44 later this year
"With proven delivery of RNA to muscle and robust efficacy and long-term safety data from our ongoing clinical development programs, we are revolutionizing the delivery of RNA with our AOC technology," said Sarah Boyce, president and chief executive officer at Avidity. "2024 is poised to be a transformative year for Avidity as we plan to initiate the global Phase 3 HARBOR™ trial for people living with DM1 and report data from all three of our clinical development programs to treat rare muscle diseases – DM1, FSHD and DMD44. We also plan to advance our cardiology and additional skeletal muscle programs toward clinical development. We know how important these potential new treatments are for the patient community and are working diligently to bring these much-needed therapies to people living with rare diseases."
"We are pleased to be in a strong financial position, with
Clinical Development Programs - Achievements & Updates
AOC 1001
- In October 2023, Avidity announced new positive AOC 1001 data demonstrating improvement in multiple additional functional endpoints and favorable long-term safety and tolerability in people living with myotonic dystrophy type 1 (DM1). These data augmented previously presented positive topline data from the Phase 1/2 MARINA® trial in April 2023 showing improvements in myotonia, muscle strength and mobility.
- Avidity will share a first-look at long-term efficacy and safety data from the MARINA-OLE™ trial of AOC 1001 in people living with DM1 via poster presentation during the 2024 MDA Clinical & Scientific Conference March 3-6 in
Orlando, Florida . The company will also host a live webcast event on March 4 at 8:00 a.m. ET as part of its ongoing investor and analyst event series. - Avidity is on-track to initiate the global Phase 3 HARBOR™ trial of AOC 1001 for adults living with DM1 in mid-2024.
AOC 1020
- In February 2023, the FDA and the European Medicines Agency (EMA) granted Orphan Designation for AOC 1020 for facioscapulohumeral muscular dystrophy (FSHD), and in January 2023, the FDA granted AOC 1020 Fast Track Designation for FSHD.
- In the second quarter of 2024, Avidity is planning to share preliminary data in approximately half of the study participants in the Phase 1/2 FORTITUDE™ trial of AOC 1020 in FSHD.
AOC 1044
- In December 2023, Avidity announced positive AOC 1044 data in healthy volunteers showing unprecedented delivery to muscle and up to
1.5% exon skipping from the Phase 1/2 EXPLORE44™ clinical trial for the treatment of Duchenne muscular dystrophy mutations amenable to exon 44 skipping (DMD44). - In August and October 2023, the FDA and EMA, respectively, granted Orphan Designation for AOC 1044 for DMD44. The FDA granted AOC 1044 Fast Track Designation in April 2023 and Rare Pediatric Disease Designation in February 2024 for DMD44.
- In the second half of 2024, Avidity is planning to share 5 mg/kg cohort data from the Phase 1/2 EXPLORE44™ trial of people living with DMD44.
Collaboration Announcements
- In November 2023, Avidity announced the expansion of a global licensing and research collaboration with Bristol Myers Squibb. The expanded collaboration will focus on the discovery, development and commercialization of up to five cardiovascular targets with
$100 million $2.2 billion
Organizational Highlights
- Avidity announced in January 2024 the appointment of Eric B. Mosbrooker as Chief Strategy Officer. Mr. Mosbrooker previously served as a member of Avidity's Board of Directors.
Fourth Quarter and Year-End 2023 Financial Results
- Cash, Cash Equivalents and Marketable Securities: Cash, cash equivalents and marketable securities totaled
$595.4 million $160.5 million $100 million $60.5 million - Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity's partnership with Eli Lilly and Company and totaled
$2.2 million $2.8 million $9.6 million $9.2 million - Research and Development (R&D) Expenses: R&D expenses include external and internal costs associated with research and development activities. These expenses were
$52.8 million $45.6 million $191.0 million $150.4 million - General and Administrative (G&A) Expenses: G&A expenses primarily consist of employee-related expenses, professional fees, insurance costs and patent filing and maintenance fees. These expenses were
$16.1 million $10.4 million $54.2 million $37.7 million
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 is focused on revolutionizing the field of RNA with its proprietary AOCs, which are designed to combine the specificity of monoclonal antibodies with the precision of oligonucleotide therapies to address targets and diseases previously unreachable with existing RNA therapies. Utilizing its proprietary AOC platform, Avidity demonstrated the first-ever successful targeted delivery of RNA into muscle and is leading the field with clinical development programs for three rare muscle diseases: myotonic dystrophy type 1 (DM1), Duchenne muscular dystrophy (DMD) and facioscapulohumeral muscular dystrophy (FSHD). Avidity is broadening the reach of AOCs with its advancing and expanding pipeline including programs in cardiology and immunology through internal discovery efforts and key partnerships. Avidity is headquartered in
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: plans to initiate a global Phase 3 trial for people living with DM1; the anticipated release of data from the MARINA-OLE™, FORTITUDE™ and EXPLORE44™ trials and the timing thereof; the characterization of safety, tolerability and functional data associated with Avidity's clinical development programs; plans for the progression of research and development initiatives, including in cardiology and skeletal muscle; Avidity's collaboration with Bristol Myers Squibb; Avidity's financial position, cash balance and ability to fund its operations; Avidity's growth and related needs; plans for the progression of Avidity's pipeline and clinical programs for AOC 1001, AOC 1044 and AOC 1020, and the timing thereof; the potential of Avidity's product candidates to treat rare diseases and Avidity's efforts to bring them to people suffering from applicable diseases; the potential of AOCs to target a range of different cells and tissues beyond the liver, and to treat cardiac and immunological diseases, and Avidity's position in the RNA field.
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 Avidity's business and beyond its control, including, without limitation: Avidity may not be able to resolve the partial clinical hold related to the serious adverse event which occurred in the Phase 1/2 MARINA® trial, which may result in delays in the clinical development of AOC 1001; additional data related to Avidity's current clinical programs that continues to become available may be inconsistent with the data produced as of the respective data cutoff dates, further analysis of existing data and analysis of new data may lead to conclusions different from those established as of the date hereof, and such data may not meet Avidity's expectations; unexpected adverse side effects to, or inadequate efficacy of, Avidity's product candidates that may delay or limit their development, regulatory approval and/or commercialization; Avidity's approach to the discovery and development of product candidates based on its AOC platform is unproven; potential delays in the commencement, enrollment, data readouts and completion of preclinical studies or clinical trials; the success of its preclinical studies and clinical trials for the company's product candidates; Avidity's dependence on third parties in connection with preclinical and clinical testing and product manufacturing; Avidity may not realize the expected benefits of its collaborations; regulatory developments in
Investor Contact:
Geoffrey Grande, CFA
(619) 837-5014
investors@aviditybio.com
Media Contact:
Navjot Rai
(619) 837-5016
media@aviditybio.com
Avidity Biosciences, Inc.
Selected Condensed Financial Information
(in thousands, except per share data)
(unaudited)
Statements of Operations | Three Months Ended December 31, | Twelve Months Ended December 31, | |||||
2023 | 2022 | 2023 | 2022 | ||||
Collaboration revenue | $ 2,193 | $ 2,769 | $ 9,560 | $ 9,224 | |||
Operating expenses: | |||||||
Research and development | 52,817 | 45,610 | 190,968 | 150,404 | |||
General and administrative | 16,119 | 10,384 | 54,190 | 37,733 | |||
Total operating expenses | 68,936 | 55,994 | 245,158 | 188,137 | |||
Loss from operations | (66,743) | (53,225) | (235,598) | (178,913) | |||
Other income, net | 6,300 | 2,754 | 23,378 | 4,918 | |||
Net loss | $ (60,443) | $ (50,471) | $ (212,220) | $ (173,995) | |||
Net loss per share, basic and diluted | $ (0.79) | $ (0.88) | $ (2.91) | $ (3.34) | |||
Weighted-average shares outstanding, basic and diluted | 76,052 | 57,296 | 73,012 | 52,162 | |||
Balance Sheets | December 31, | December 31, | |
Assets | |||
Current assets: | |||
Cash, cash equivalents and marketable securities | $ 595,351 | $ 610,727 | |
Prepaid and other assets | 15,956 | 12,215 | |
Total current assets | 611,307 | 622,942 | |
Property and equipment, net | 8,381 | 6,254 | |
Restricted cash | 295 | 251 | |
Right-of-use assets | 8,271 | 8,755 | |
Other assets | 301 | 598 | |
Total assets | $ 628,555 | $ 638,800 | |
Liabilities and Stockholders' Equity | |||
Current liabilities: | |||
Accounts payable and other liabilities | $ 52,315 | $ 46,867 | |
Deferred revenue, current portion | 28,365 | 5,041 | |
Total current liabilities | 80,680 | 51,908 | |
Lease liabilities, net of current portion | 6,213 | 7,582 | |
Deferred revenue, net of current portion | 40,898 | 1,235 | |
Total liabilities | 127,791 | 60,725 | |
Stockholders' equity | 500,764 | 578,075 | |
Total liabilities and stockholders' equity | $ 628,555 | $ 638,800 |
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SOURCE Avidity Biosciences, Inc.
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