– In 2020, continued to advance genetic drugs pipeline with execution of IND-enabling research for AK-OTOF and common alignment with FDA on the trail to a 2022 IND submission for AK-antiVEGF
– Raised roughly $349 million in gross proceeds, which is predicted to fund operations for no less than two years
– As a consequence of 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 observe
– Establishing inside cGMP manufacturing infrastructure and capabilities in 2021
BOSTON, March 29, 2021 (GLOBE NEWSWIRE) — Akouos, Inc. (Nasdaq: AKUS), a precision genetic drugs firm devoted to creating potential gene therapies for people residing with disabling listening to loss worldwide, in the present day reported monetary outcomes for the fourth quarter and full yr ended December 31, 2020 and supplied enterprise highlights.
“2020 was a yr of super progress for Akouos, marked by continued development of our pipeline, growth of our group, and strengthening of our capital place to additional our management within the improvement 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 information reported so far, which display the sturdy restoration of perform of AK-OTOF. As a consequence of 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 observe. 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 inside cGMP manufacturing infrastructure and capabilities.”
Enterprise and Pipeline Highlights for 2020, Current Developments, and Anticipated Milestones
- Continued to construct pipeline with potential for broad applicability for interior ear situations – In 2020, Akouos made progress in the direction of Investigational New Drug (IND) submissions to the U.S. Meals and Drug Administration (FDA) for AK-OTOF, a gene remedy meant for the remedy of otoferlin gene (OTOF)-mediated listening to loss, and AK-antiVEGF, a gene remedy meant for the remedy of vestibular schwannoma. Earlier this yr, Akouos accomplished inside 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 as a consequence 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 inside manufacturing actions had been carried out utilizing the identical clinical-scale, and deliberate commercial-scale, manufacturing course of meant for cGMP manufacturing of Akouos’s product candidates. Aside from the third-party cGMP manufacturing delays, all different IND-enabling actions stay on observe.
- Bettering entry to genetic testing for eligible people with auditory neuropathy – In January 2021, Akouos introduced the Resonate™ program with Blueprint Genetics. This system gives 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 throughout the deaf and onerous of listening to group.
- Knowledge presentation at ARO supporting Akouos’s novel supply strategy– In February 2021, Akouos offered information on the Affiliation for Analysis in Otolaryngology (ARO) that demonstrated that intracochlear administration of AAVAnc80 is nicely 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 gadget that’s designed for supply of a product candidate in a hard and fast quantity and at a managed move fee, and direct intracochlear administration that permits for distribution of product candidates alongside the complete size of the cochlea.
- Expanded Akouos group and board of administrators with a number of appointments – By the tip of 2020, Akouos had expanded to a complete of 67 group members with experience throughout a spread of important features, together with analysis, medical improvement, 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 development throughout the Firm and throughout 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 Yr 2020 Monetary Outcomes
- Money Place – Money, money equivalents, and marketable securities had 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 following two years.
- Analysis and Improvement (R&D) Bills – R&D bills had been $8.0 million for the fourth quarter of 2020 and $34.3 million for the complete yr ended December 31, 2020, in comparison with $8.5 million for the fourth quarter of 2019 and $20.5 million for the complete 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 workers and their associated actions, in addition to the expense allotted to R&D associated to Akouos’s leased amenities.
- Common and Administrative (G&A) Bills – G&A bills had been $4.6 million for the fourth quarter of 2020 and $14.6 million for the complete yr ended December 31, 2020, in comparison with $1.1 million for the fourth quarter of 2019 and $3.4 million for the complete yr ended December 31, 2019. The rise was primarily because of the development within the variety of G&A workers 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 amenities.
- Internet Loss – Internet 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 complete 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 complete yr ended December 31, 2019.
Akouos is a precision genetic drugs firm devoted to creating gene therapies with the potential to revive, enhance, and protect high-acuity physiologic listening to for people residing with disabling listening to loss worldwide. Leveraging its precision genetic drugs platform that comes with a proprietary adeno-associated viral (AAV) vector library and a novel supply strategy, Akouos is concentrated 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 Concerning Ahead-Wanting Statements
Statements on this press launch about future expectations, plans and prospects, in addition to every other statements relating to issues that aren’t historic information, might represent “forward-looking statements” throughout the which means of The Non-public Securities Litigation Reform Act of 1995. These statements embrace, however aren’t restricted to, statements referring to the initiation, plans, and timing of our future medical trials and our analysis and improvement packages, the timing of our IND submissions for AK-OTOF and AK-antiVEGF, our expectations relating to our manufacturing capabilities and timelines, and the interval over which we imagine that our present money, money equivalents and marketable securities will probably be adequate to fund our working bills. The phrases “anticipate,” “imagine,” “proceed,” “may,” “estimate,” “count on,” “intend,” “might,” “plan,” “potential,” “predict,” “challenge,” “ought to,” “goal,” “will,” “would,” and comparable expressions are meant to determine forward-looking statements, though not all forward-looking statements comprise these figuring out phrases. Precise outcomes might differ materially from these indicated by such forward-looking statements because of varied vital elements, together with: our restricted working historical past; uncertainties inherent within the improvement of product candidates, together with the initiation and completion of nonclinical research and medical trials; whether or not outcomes from nonclinical research will probably be predictive of outcomes or success of medical trials; the timing of and our potential to submit purposes for, and procure and keep regulatory approvals for, our product candidates; our expectations relating to our regulatory technique; our potential 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 medical utility of our product candidates; our estimates relating to the potential addressable affected person inhabitants for our product candidates; our commercialization, advertising, and manufacturing capabilities and technique; our potential to acquire and keep mental property safety for our product candidates; our potential to determine extra merchandise, product candidates, or applied sciences with vital industrial potential which are per our industrial aims; the affect of presidency legal guidelines and rules; dangers associated to aggressive packages; the potential that our inside manufacturing capabilities and/or exterior manufacturing provide might expertise delays; the affect of the COVID-19 pandemic on our enterprise, outcomes of operations, and monetary situation; our potential to keep up and set up collaborations or acquire extra funding; and different elements 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 because of new info, future occasions or in any other case.
Condensed Consolidated Steadiness Sheet Knowledge
|December 31, 2020||December 31, 2019|
|Money, money equivalents and marketable securities||$||308,010||$||25,078|
|Convertible most well-liked inventory||—||58,690|
|Complete stockholders’ fairness (deficit)||310,614||(32,801||)|
Condensed Consolidated Statements of Operations and Complete Loss
(in 1000’s, besides share and per share information)
|Three months ended December 31,||Yr ended December 31,|
|Analysis and improvement||$||7,977||$||8,475||$||34,297||$||20,473|
|Common and administrative||4,646||1,134||14,583||3,410|
|Complete working bills||12,623||9,609||48,880||23,883|
|Loss from operations||(12,623||)||(9,609||)||(48,880||)||(23,883||)|
|Different earnings (expense):|
|Change in truthful worth of most well-liked inventory tranche legal responsibility||—||—||—||(2,260||)|
|Different earnings (expense), web||(291||)||(2||)||(287||)||(11||)|
|Complete different earnings (expense), web||75||113||280||(1,858||)|
|Weighted‑common frequent shares excellent, fundamental and diluted||34,217,475||663,659||17,550,847||605,824|
|Internet loss per share attributable to frequent stockholders, fundamental and diluted||$||(0.37||)||$||(14.31||)||$||(2.77||)||$||(42.49||)|
Katie Engleman, 1AB
Courtney Turiano, Stern Investor Relations