– In 2020, continued to advance genetic medication pipeline with execution of IND-enabling research for AK-OTOF and normal alignment with FDA on the trail to a 2022 IND submission for AK-antiVEGF
– Raised roughly $349 million in gross proceeds, which is anticipated to fund operations for no less than two years
– Attributable to latest third-party manufacturing delays, IND submission for AK-OTOF program now anticipated within the first half of 2022; all different IND-enabling actions stay on monitor
– Establishing inner cGMP manufacturing infrastructure and capabilities in 2021
BOSTON, March 29, 2021 (GLOBE NEWSWIRE) — Akouos, Inc. (Nasdaq: AKUS), a precision genetic medication firm devoted to creating potential gene therapies for people dwelling with disabling listening to loss worldwide, at this time reported monetary outcomes for the fourth quarter and full yr ended December 31, 2020 and offered enterprise highlights.
“2020 was a yr of super progress for Akouos, marked by continued development of our pipeline, growth of our staff, and strengthening of our capital place to additional our management within the growth of precision genetic medicines for interior ear situations,” mentioned Manny Simons, Ph.D., founder, president, and CEO of Akouos. “We proceed to be excited by the nonclinical knowledge reported up to now, which show the sturdy restoration of operate of AK-OTOF. Attributable to third-party manufacturing delays, together with impacts from the continuing COVID-19 pandemic, we now count on to submit the IND for AK-OTOF within the first half of 2022. All different IND-enabling actions stay on monitor. We proceed to work with a number of third-party producers to advance cGMP campaigns, for each the AK-OTOF and AK-antiVEGF IND submissions deliberate for 2022, and we proceed to construct our inner cGMP manufacturing infrastructure and capabilities.”
Enterprise and Pipeline Highlights for 2020, Latest Developments, and Anticipated Milestones
- Continued to construct pipeline with potential for broad applicability for interior ear situations – In 2020, Akouos made progress in direction of Investigational New Drug (IND) submissions to the U.S. Meals and Drug Administration (FDA) for AK-OTOF, a gene remedy supposed for the therapy of otoferlin gene (OTOF)-mediated listening to loss, and AK-antiVEGF, a gene remedy supposed for the therapy of vestibular schwannoma. Earlier this yr, Akouos accomplished inner manufacturing of AK-antiVEGF for an IND-enabling good laboratory practices (GLP) toxicology examine. Moreover, in 2020, the Firm chosen a product candidate for the AK-CLRN1 program. In 2021, the Firm plans to announce a product candidate for its GJB2 program, and targets for its hair cell regeneration and autosomal dominant listening to loss packages.
- Up to date timeline for AK-OTOF IND submission – The submission of an IND to FDA for AK-OTOF is now deliberate for the primary half of 2022 because of third-party manufacturing delays, together with delays associated to the COVID-19 pandemic. The manufacturing course of for Akouos’s product candidates, together with AK-OTOF, has resulted in seven (of seven) profitable at-scale batches of AAVAnc80 vectors. These third-party and inner manufacturing actions have been carried out utilizing the identical clinical-scale, and deliberate commercial-scale, manufacturing course of supposed for cGMP manufacturing of Akouos’s product candidates. Other than the third-party cGMP manufacturing delays, all different IND-enabling actions stay on monitor.
- Enhancing entry to genetic testing for eligible people with auditory neuropathy – In January 2021, Akouos introduced the Resonate™ program with Blueprint Genetics. This system supplies entry to the Blueprint Genetics Complete Listening to Loss and Deafness Panel that features greater than 230 genes related to genetic types of listening to loss, serving to people and their healthcare suppliers higher perceive a person’s situation and foster connections inside the deaf and laborious of listening to neighborhood.
- Knowledge presentation at ARO supporting Akouos’s novel supply strategy – In February 2021, Akouos offered knowledge on the Affiliation for Analysis in Otolaryngology (ARO) that demonstrated that intracochlear administration of AAVAnc80 is effectively tolerated by non-human primates at doses that obtain environment friendly transduction of goal cells. Akouos’s novel supply strategy makes use of a minimally invasive surgical strategy, a supply machine that’s designed for supply of a product candidate in a set quantity and at a managed circulation fee, and direct intracochlear administration that permits for distribution of product candidates alongside the total size of the cochlea.
- Expanded Akouos staff and board of administrators with a number of appointments – By the top of 2020, Akouos had expanded to a complete of 67 staff members with experience throughout a variety of essential capabilities, together with analysis, scientific growth, regulatory, high quality, technical operations, finance, and authorized. The Firm added Heather Preston, Saira Ramasastry, and Vicki Sato to the board of administrators, and named Arthur Tzianabos as chairman.
- Ended 2020 with a powerful money place of $308.0 million to proceed to drive progress throughout the Firm and inside the pipeline – In February 2020, Akouos executed a Sequence B financing elevating $105.0 million in gross proceeds. In June 2020, Akouos accomplished an upsized preliminary public providing elevating $244.4 million in gross proceeds.
Fourth Quarter and Full 12 months 2020 Monetary Outcomes
- Money Place – Money, money equivalents, and marketable securities have been $308.0 million as of December 31, 2020, as in comparison with $25.1 million as of December 31, 2019. Akouos expects its money stability to fund operations for no less than the subsequent two years.
- Analysis and Growth (R&D) Bills – R&D bills have been $8.0 million for the fourth quarter of 2020 and $34.3 million for the total yr ended December 31, 2020, in comparison with $8.5 million for the fourth quarter of 2019 and $20.5 million for the total yr ended December 31, 2019. The rise was primarily because of the elevated efforts in IND-enabling research for AK-OTOF and the expansion within the variety of R&D staff and their associated actions, in addition to the expense allotted to R&D associated to Akouos’s leased services.
- Normal and Administrative (G&A) Bills – G&A bills have been $4.6 million for the fourth quarter of 2020 and $14.6 million for the total yr ended December 31, 2020, in comparison with $1.1 million for the fourth quarter of 2019 and $3.4 million for the total yr ended December 31, 2019. The rise was primarily because of the progress within the variety of G&A staff and different administrative bills associated to working as a public firm, in addition to the expense allotted to G&A associated to Akouos’s leased services.
- Web Loss – Web loss was $12.5 million, or $0.37 loss per share, for the fourth quarter of 2020 and $48.6 million, or $2.77 loss per share, for the total yr ended December 31, 2020, in comparison with $9.5 million, or $14.31 per share, for the fourth quarter of 2019 and $25.7 million, or $42.49 loss per share, for the total yr ended December 31, 2019.
Akouos is a precision genetic medication firm devoted to creating gene therapies with the potential to revive, enhance, and protect high-acuity physiologic listening to for people dwelling with disabling listening to loss worldwide. Leveraging its precision genetic medication platform that comes with a proprietary adeno-associated viral (AAV) vector library and a novel supply strategy, Akouos is targeted on creating precision therapies for types of sensorineural listening to loss. Headquartered in Boston, Akouos was based in 2016 by leaders within the fields of neurotology, genetics, interior ear drug supply, and AAV gene remedy.
Cautionary Word Relating to Ahead-Trying Statements
Statements on this press launch about future expectations, plans and prospects, in addition to some other statements concerning issues that aren’t historic information, could represent “forward-looking statements” inside the which means of The Non-public Securities Litigation Reform Act of 1995. These statements embrace, however are usually not restricted to, statements regarding the initiation, plans, and timing of our future scientific trials and our analysis and growth packages, the timing of our IND submissions for AK-OTOF and AK-antiVEGF, our expectations concerning our manufacturing capabilities and timelines, and the interval over which we consider that our current money, money equivalents and marketable securities can be adequate to fund our working bills. The phrases “anticipate,” “consider,” “proceed,” “might,” “estimate,” “count on,” “intend,” “could,” “plan,” “potential,” “predict,” “undertaking,” “ought to,” “goal,” “will,” “would,” and related expressions are supposed to establish forward-looking statements, though not all forward-looking statements include these figuring out phrases. Precise outcomes could differ materially from these indicated by such forward-looking statements on account of numerous vital components, together with: our restricted working historical past; uncertainties inherent within the growth of product candidates, together with the initiation and completion of nonclinical research and scientific trials; whether or not outcomes from nonclinical research can be predictive of outcomes or success of scientific trials; the timing of and our skill to submit functions for, and procure and keep regulatory approvals for, our product candidates; our expectations concerning our regulatory technique; our skill to fund our working bills and capital expenditure necessities with our money, money equivalents, and marketable securities; the potential benefits of our product candidates; the speed and diploma of market acceptance and scientific utility of our product candidates; our estimates concerning the potential addressable affected person inhabitants for our product candidates; our commercialization, advertising, and manufacturing capabilities and technique; our skill to acquire and keep mental property safety for our product candidates; our skill to establish extra merchandise, product candidates, or applied sciences with vital business potential which can be in line with our business targets; the impression of presidency legal guidelines and laws; dangers associated to aggressive packages; the potential that our inner manufacturing capabilities and/or exterior manufacturing provide could expertise delays; the impression of the COVID-19 pandemic on our enterprise, outcomes of operations, and monetary situation; our skill to keep up and set up collaborations or receive extra funding; and different components mentioned within the “Threat Elements” included within the Firm’s Quarterly Report on Type 10-Q for the three months ended September 30, 2020 filed with the Securities and Alternate Fee, and in different filings that the Firm makes with the Securities and Alternate Fee sooner or later. Any forward-looking statements contained on this press launch communicate solely as of the date hereof, and the Firm expressly disclaims any obligation to replace any forward-looking assertion, whether or not on account of new data, future occasions or in any other case.
Condensed Consolidated Stability Sheet Knowledge
|December 31, 2020||December 31, 2019|
|Money, money equivalents and marketable securities||$||308,010||$||25,078|
|Convertible most well-liked inventory||—||58,690|
|Complete stockholders’ fairness (deficit)||310,614||(32,801||)|
Condensed Consolidated Statements of Operations and Complete Loss
(in hundreds, besides share and per share knowledge)
|Three months ended December 31,||12 months ended December 31,|
|Analysis and growth||$||7,977||$||8,475||$||34,297||$||20,473|
|Normal and administrative||4,646||1,134||14,583||3,410|
|Complete working bills||12,623||9,609||48,880||23,883|
|Loss from operations||(12,623||)||(9,609||)||(48,880||)||(23,883||)|
|Different revenue (expense):|
|Change in honest worth of most well-liked inventory tranche legal responsibility||—||—||—||(2,260||)|
|Different revenue (expense), web||(291||)||(2||)||(287||)||(11||)|
|Complete different revenue (expense), web||75||113||280||(1,858||)|
|Weighted‑common widespread shares excellent, fundamental and diluted||34,217,475||663,659||17,550,847||605,824|
|Web loss per share attributable to widespread stockholders, fundamental and diluted||$||(0.37||)||$||(14.31||)||$||(2.77||)||$||(42.49||)|
Katie Engleman, 1AB
Courtney Turiano, Stern Investor Relations