Avidity Biosciences Reports Fourth Quarter and Year-End 2023 Financial Results and Recent Highlights
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
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
"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 inApril 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 inOrlando, Florida . The company will also host a live webcast event onMarch 4 at8:00 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 theEuropean Medicines Agency (EMA) granted Orphan Designation for AOC 1020 for facioscapulohumeral muscular dystrophy (FSHD), and inJanuary 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 inApril 2023 and Rare Pediatric Disease Designation inFebruary 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 withBristol Myers Squibb . The expanded collaboration will focus on the discovery, development and commercialization of up to five cardiovascular targets with$100 million upfront and potential cumulative milestone payments to Avidity of up to$2.2 billion .
Organizational Highlights
- Avidity announced in
January 2024 the appointment ofEric 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 as ofDecember 31, 2023 , which reflects$160.5 million raised in 2023, inclusive of approximately$100 million through our collaboration withBristol Myers Squibb and$60.5 million through our "at the market offering" program. -
Collaboration Revenue: Collaboration revenue, including reimbursable expenses, primarily relates to Avidity's partnership with Eli Lilly and Company and totaled
$2.2 million for the fourth quarter of 2023 compared with$2.8 million for the fourth quarter of 2022, and$9.6 million for the full year 2023 compared with$9.2 million for the full year 2022. -
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 for the fourth quarter of 2023 compared with$45.6 million for the fourth quarter of 2022, and$191.0 million for the full year 2023 compared with$150.4 million for the full year 2022. The increases were primarily driven by the advancement of AOC 1001, AOC 1020 and AOC 1044, as well as internal and external costs related to the expansion of the company's overall research capabilities. -
Gener
al 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 for the fourth quarter of 2023 compared with$10.4 million for the fourth quarter of 2022, and$54.2 million for the full year 2023 compared with$37.7 million for the full year 2022. The increases were primarily due to higher personnel costs and professional fees to support the company's expanded operations.
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: 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
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:
(619) 837-5014
investors@aviditybio.com
Media Contact:
(619) 837-5016
media@aviditybio.com
Selected Condensed Financial Information
(in thousands, except per share data)
(unaudited)
Statements of Operations |
Three Months Ended |
|
Twelve Months Ended |
||||
|
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 |
|
|
|
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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