1. BOSTON, Sept. 07, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos, will present at two upcoming virtual investor conferences in September:

    • H.C. Wainwright 23rd Annual Global Investment Conference. Pre-recorded fireside chat available for on-demand viewing starting on Monday, September 13 at 7:00 a.m. EDT
    • Morgan Stanley Virtual Global Healthcare Conference. Fireside chat on Monday, September 13 at 5:00 p.m. EDT

    An on-demand webcast of the H.C. Wainwright fireside chat and…

    BOSTON, Sept. 07, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos, will present at two upcoming virtual investor conferences in September:

    • H.C. Wainwright 23rd Annual Global Investment Conference. Pre-recorded fireside chat available for on-demand viewing starting on Monday, September 13 at 7:00 a.m. EDT
    • Morgan Stanley Virtual Global Healthcare Conference. Fireside chat on Monday, September 13 at 5:00 p.m. EDT

    An on-demand webcast of the H.C. Wainwright fireside chat and a live webcast of the Morgan Stanley fireside chat will be accessible through the investors section of www.akouos.com. To access the webcasts, please go to the Akouos website approximately 15 minutes prior to the start time to ensure adequate time for any software downloads that may be required. An archived replay will be available on Akouos's website for 90 days following the conference.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com 

    Investors:

    Courtney Turiano, Stern Investor Relations 

    Courtney.Turiano@sternir.com 



    Primary Logo

    View Full Article Hide Full Article
  2. - Announced European Commission designation of AK-OTOF as an orphan drug for the treatment of otoferlin gene-mediated hearing loss

    - Advanced both AK-OTOF and AK-antiVEGF towards planned IND submissions in the first half of 2022 and in 2022, respectively

    - Continued to broaden application of genetic medicines platform to treat additional inner ear conditions

    BOSTON, Aug. 12, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today reports financial results for the second quarter ended June 30, 2021 and provides business highlights.

    "During the past quarter, we have continued to advance the…

    - Announced European Commission designation of AK-OTOF as an orphan drug for the treatment of otoferlin gene-mediated hearing loss

    - Advanced both AK-OTOF and AK-antiVEGF towards planned IND submissions in the first half of 2022 and in 2022, respectively

    - Continued to broaden application of genetic medicines platform to treat additional inner ear conditions

    BOSTON, Aug. 12, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today reports financial results for the second quarter ended June 30, 2021 and provides business highlights.

    "During the past quarter, we have continued to advance the development of our two lead programs," said Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos. "All IND-enabling activities for both AK-OTOF and AK-antiVEGF remain on track to support our planned submissions in 2022, including the completion of dosing in the GLP toxicology study for AK-antiVEGF. We also continue to strengthen our precision genetic medicine platform and progress our earlier stage programs towards the announcement of a product candidate for the treatment of GJB2-mediated hearing loss and targets for hair cell regeneration and autosomal dominant hearing loss, planned for later this year. Our team's exceptional expertise in neurotology, genetics, inner ear drug delivery, and AAV gene therapy has helped establish our leadership in the field of inner ear genetic medicine, and enables us to drive forward the development of gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide."

    Business Highlights

    • AK-OTOF is on track for planned IND submission in the first half of 2022 – All IND-enabling activities for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene (OTOF)-mediated hearing loss, continue to advance as planned.



      • Along with the Orphan Drug Designation and Rare Pediatric Disease Designation previously granted by FDA, the orphan drug designation for AK-OTOF in the European Union could accelerate our development and provide other potential benefits
      • Following the Hearing Loss Association of America-sponsored Patient-Focused Drug Development meeting for people and families living with sensorineural hearing loss, the Company convened a community advisory board of families affected by OTOF-mediated hearing loss to learn about their experiences living with the condition and gain their input on the clinical development plan for AK-OTOF
    • Advanced AK-antiVEGF toward planned IND submission in 2022 – IND-enabling activities for AK-antiVEGF, a gene therapy intended for the treatment of vestibular schwannoma, continue to make progress, including completion of dosing for the good laboratory practice (GLP) toxicology study.
    • Applying genetic medicines platform beyond lead programs to address broader range of inner ear conditions – In 2021, the Company plans to announce a product candidate for its GJB2 program, and targets for its hair cell regeneration and autosomal dominant hearing loss programs.

    Second Quarter 2021 Financial Results

    • Cash Position – Cash, cash equivalents, and marketable securities were $271.8 million as of June 30, 2021, as compared to $308.0 million as of December 31, 2020. Akouos expects its cash balance to fund operations for at least the next two years.
    • Research and Development (R&D) Expenses – R&D expenses were $17.1 million for the second quarter ended June 30, 2021, compared to $9.9 million for the same period in 2020. The increase was primarily due to the increased efforts in IND-enabling studies and increased manufacturing costs for AK-OTOF and AK-antiVEGF and the growth in the number of R&D employees and their related activities, as well as the expense allocated to R&D related to Akouos's leased facilities.
    • General and Administrative (G&A) Expenses – G&A expenses were $5.7 million for the second quarter ended June 30, 2021, compared to $2.7 million for the same period in 2020. The increase was primarily due to the growth in the number of G&A employees and other administrative expenses related to operating as a public company, as well as the expense allocated to G&A related to Akouos's leased facilities.
    • Net Loss – Net loss was $22.7 million, or $0.66 per share, for the second quarter ended June 30, 2021, compared to $12.5 million, or $11.14 per share, for the same period in 2020.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, and timing of our future clinical trials and our research and development programs, the timing of our IND submissions for AK-OTOF and AK-antiVEGF, the potential receipt of exclusivity and other benefits from Orphan Drug Designation in the European Union and Orphan Drug Designation and Rare Pediatric Disease Designation in the United States, and the period over which we believe that our existing cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our limited operating history; uncertainties inherent in the development of product candidates, including the initiation and completion of nonclinical studies and clinical trials; whether results from nonclinical studies will be predictive of results or success of clinical trials; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents, and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing, and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations and any changes in such laws and regulations; risks related to competitive programs; the potential that our internal manufacturing capabilities and/or external manufacturing supply may experience delays; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the "Risk Factors" included in the Company's Quarterly Report on Form 10-Q for the three months ended March 31, 2021 filed with the Securities and Exchange Commission, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.



    Condensed Consolidated Balance Sheet Data

    (Unaudited)

    (in thousands)

           
      June 30, 2021 December 31, 2020
          
    Cash, cash equivalents and marketable securities $271,751 $308,010
    Total assets  316,583  333,350
    Total liabilities  40,055  22,736
    Total stockholders' equity  276,528  310,614



    Condensed Consolidated Statements of Operations and Comprehensive Loss

    (Unaudited)

    (in thousands, except share and per share data)

                
     Three Months Ended  Six Months Ended
     June 30,  June 30, 
     2021  2020  2021  2020 
            
    Operating expenses:           
    Research and development$17,119  $9,937  $28,377  $17,971 
    General and administrative 5,665   2,664   10,555   5,168 
    Total operating expenses 22,784   12,601   38,932   23,139 
    Loss from operations (22,784)  (12,601)  (38,932)  (23,139)
    Other income (expense):           
    Interest income 554   80   1,063   180 
    Other expense, net (510)  (2)  (957)  (4)
    Total other income, net 44   78   106   176 
    Net loss$(22,740) $(12,523) $(38,826) $(22,963)
    Net loss per share attributable to common stockholders

    basic and diluted
    $(0.66) $(11.14) $(1.13) $(25.05)
    Weighted‑average common shares outstanding, basic

    and diluted
     34,372,262   1,124,251   34,324,477   916,521 
    Other comprehensive income (loss):           
    Unrealized gain (loss) on marketable securities (23)     5    
    Total other comprehensive income (loss) (23)     5    
    Total comprehensive loss$(22,763) $(12,523) $(38,821) $(22,963)
                

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com



    Primary Logo

    View Full Article Hide Full Article
  3. BOSTON, Aug. 11, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on the company's application for orphan drug designation for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss. The positive opinion was subsequently adopted by the European Commission. AK-OTOF was previously granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) by the U.S. Food and Drug Administration for this same indication…

    BOSTON, Aug. 11, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products (COMP) issued a positive opinion on the company's application for orphan drug designation for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss. The positive opinion was subsequently adopted by the European Commission. AK-OTOF was previously granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) by the U.S. Food and Drug Administration for this same indication.

    Otoferlin gene (OTOF)-mediated hearing loss is a form of sensorineural hearing loss caused by mutations in the OTOF gene. The OTOF gene encodes otoferlin, a protein that enables the inner hair cells of the cochlea to release neurotransmitter vesicles in response to stimulation by sound to activate auditory neurons. Most individuals with OTOF-mediated hearing loss have Severe to Profound sensorineural hearing loss from birth and approximately 20,000 individuals are affected in the United States and Europe. AK-OTOF is designed to treat the underlying cause of OTOF-mediated hearing loss through delivery of a transgene using a dual vector technology that results in expression of normal, functional otoferlin protein in the affected cells, namely inner hair cells, in the cochlea.

    "Severe to Profound sensorineural hearing loss from birth caused by mutations in the OTOF gene is a high unmet need, especially given that there are currently no approved pharmacologic treatment options available," said Katie Wachtel, M.S. and vice president of regulatory affairs of Akouos. "The orphan drug designation granted by the European Commission is an important step towards advancing the global development of AK-OTOF, a gene therapy with potential to restore physiologic hearing and provide long-lasting benefit to individuals with OTOF-mediated hearing loss. Along with the ODD and RPDD designations previously granted by FDA, the orphan drug designation for AK-OTOF in the European Union could accelerate our development of AK-OTOF and our progress towards our mission of healthy hearing available to all."

    Orphan drug designation in the European Union is granted by the European Commission based on an opinion issued by the EMA COMP. An orphan drug designation provides a number of benefits, including fee reductions, regulatory assistance, and the possibility to apply for a centralized European Union marketing authorization. Marketing authorization for an orphan drug can lead to a ten-year period of market exclusivity.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, and timing of our future clinical trials and our research and development programs, and the potential receipt of exclusivity and other benefits from Orphan Drug Designation in the European Union and Orphan Drug Designation and Rare Pediatric Disease Designation in the United States. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our limited operating history; uncertainties inherent in the development of product candidates, including the initiation and completion of nonclinical studies and clinical trials; whether results from nonclinical studies will be predictive of results or success of clinical trials; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents, and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing, and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations and any changes in such laws and regulations; risks related to competitive programs; the potential that our internal manufacturing capabilities and/or external manufacturing supply may experience delays; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the "Risk Factors" included in the Company's Quarterly Report on Form 10-Q for the three months ended March 31, 2021 filed with the Securities and Exchange Commission, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com



    Primary Logo

    View Full Article Hide Full Article
  4. - Leadership team expanded with appointment of Kathy Reape, M.D. as chief development officer

    - AK-OTOF granted both Orphan Drug Designation and Rare Pediatric Disease Designation by FDA and is on track for planned IND submission in first half of 2022

    - Nonclinical data supporting future clinical development of AK-OTOF and AK-antiVEGF presented at the American Society of Gene and Cell Therapy (ASGCT) 24th Annual Meeting

    - Construction for an internal cGMP manufacturing facility completed, and expansion of laboratory footprint underway, contributing to continued infrastructure build to support future development activities

    BOSTON, May 13, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company…

    - Leadership team expanded with appointment of Kathy Reape, M.D. as chief development officer

    - AK-OTOF granted both Orphan Drug Designation and Rare Pediatric Disease Designation by FDA and is on track for planned IND submission in first half of 2022

    - Nonclinical data supporting future clinical development of AK-OTOF and AK-antiVEGF presented at the American Society of Gene and Cell Therapy (ASGCT) 24th Annual Meeting

    - Construction for an internal cGMP manufacturing facility completed, and expansion of laboratory footprint underway, contributing to continued infrastructure build to support future development activities

    BOSTON, May 13, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today reports financial results for the first quarter ended March 31, 2021 and provides business updates.

    "We continue to demonstrate our leadership in the field of inner ear genetic medicines. Recently, FDA granted AK-OTOF, our lead product candidate, what we believe are the first Orphan Drug and Rare Pediatric Disease designations for a genetic form of hearing loss. At the ASGCT Annual Meeting, our team presented nonclinical data demonstrating the unique applicability of our genetic medicine platform to potentially restore, improve, and preserve physiologic hearing for a monogenic condition and a condition of complex etiology," said Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos. "These accomplishments have been made possible by our team of leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy, including the recent addition of Dr. Kathy Reape as chief development officer. We continue to build our team's capabilities, infrastructure, and facilities to support our planned IND submissions for AK-OTOF and AK-antiVEGF in 2022, and are progressing our earlier stage programs towards candidate selection or target announcement."

    Business Highlights

    • Appointment of Kathy Reape, M.D. as chief development officer Dr. Kathy Reape joined Akouos as chief development officer and brings over 20 years of experience in clinical drug development and medical affairs to Akouos, most notably from her recent role as chief medical officer of Spark Therapeutics.
    • Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) granted by FDA for AK-OTOF – AK-OTOF is a gene therapy intended for the treatment of otoferlin gene (OTOF)-mediated hearing loss, a condition that often results in Severe to Profound sensorineural hearing loss from birth and does not currently have any pharmacologic treatment options available. The receipt of these designations could support acceleration of development of AK-OTOF.
    • Presented nonclinical data at the ASGCT 24th Annual Meeting reflecting broad applicability of genetic medicine platform to treat inner ear conditions In May 2021, Akouos presented nonclinical data that support the future clinical development for both AK-OTOF and AK-antiVEGF, a gene therapy intended for the treatment of vestibular schwannoma, at the ASGCT 24th Annual Meeting. The digital presentations are located at https://akouos.com/gene-therapy-resources/.
    • Continued advancement of both AK-OTOF and AK-antiVEGF towards expected IND submissions in 2022 – Akouos continues to make progress towards an investigational new drug application (IND) submission for AK-OTOF planned in the first half of 2022. The Company expects to submit an IND for AK-antiVEGF in 2022. Both programs utilize key components of Akouos's genetic medicine platform, which incorporates a proprietary AAVAnc capsid library, including AAVAnc80, and a novel delivery approach that leverages minimally-invasive surgical techniques familiar to otologic surgeons.
    • Building infrastructure to support future development – Akouos expanded its facility at 645 Summer Street in Boston, MA to support additional research, development, and manufacturing activities. In 2021, the Company plans to complete build of infrastructure and capabilities to enable internal cGMP manufacturing expected to support clinical development of product candidates, at the anticipated commercial scale.

    First Quarter 2021 Financial Results

    • Cash Position – Cash, cash equivalents, and marketable securities were $286.6 million as of March 31, 2021, as compared to $308.0 million as of December 31, 2020. Akouos expects its cash balance to fund operations for at least the next two years.
    • Research and Development (R&D) Expenses – R&D expenses were $11.3 million for the first quarter ended March 31, 2021, compared to $8.0 million for the same period in 2020. The increase was primarily due to the increased efforts in IND-enabling studies for AK-OTOF and AK-antiVEGF and the growth in the number of R&D employees and their related activities, as well as the expense allocated to R&D related to Akouos's leased facilities.
    • General and Administrative (G&A) Expenses – G&A expenses were $4.9 million for the first quarter ended March 31, 2021, as compared to $2.5 million for the same period in 2020. The increase was primarily due to the growth in the number of G&A employees and other administrative expenses related to operating as a public company, as well as the expense allocated to G&A related to Akouos's leased facilities.
    • Net Loss – Net loss was $16.1 million, or $0.47 loss per share, for the first quarter ended March 31, 2021, compared to $10.4 million, or $14.74 loss per share, for the same period in 2020.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, and timing of our future clinical trials and our research and development programs, the timing of our IND submissions for AK-OTOF and AK-antiVEGF, our expectations regarding our manufacturing capabilities and timelines, and the period over which we believe that our existing cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our limited operating history; uncertainties inherent in the development of product candidates, including the initiation and completion of nonclinical studies and clinical trials; whether results from nonclinical studies will be predictive of results or success of clinical trials; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents, and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing, and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations; risks related to competitive programs; the potential that our internal manufacturing capabilities and/or external manufacturing supply may experience delays; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the "Risk Factors" included in the Company's Annual Report on Form 10-K for the year ended December 31, 2020 filed with the Securities and Exchange Commission, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. 





    Condensed Consolidated Balance Sheet Data

    (Unaudited)

    (in thousands)

      March 31, 2021 December 31, 2020
           
    Cash, cash equivalents and marketable securities $286,619  $308,010 
    Total assets  321,859   333,350 
    Total liabilities  25,057   22,736 
    Total stockholders' equity  296,802   310,614 





    Condensed Consolidated Statements of Operations and Comprehensive Loss

    (Unaudited)

    (in thousands, except share and per share data)

      Three months ended March 31,

      2021  2020 
           
    Operating expenses:      
    Research and development $11,258  $8,034 
    General and administrative  4,890   2,504 
    Total operating expenses  16,148   10,538 
    Loss from operations  (16,148)  (10,538)
    Other income (expense):      
    Interest income  509   100 
    Other expense, net  (447)  (2)
    Total other income, net  62   98 
    Net loss $(16,086) $(10,440)
    Weighted‑average common shares outstanding, basic and diluted  34,284,419   708,204 
    Net loss per share attributable to common stockholders, basic and diluted $(0.47) $(14.74)
           

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com



    Primary Logo

    View Full Article Hide Full Article
  5. - Intracochlear delivery of a dual AAVAnc80 vector encoding human otoferlin results in full-length protein expression in inner hair cells of non-human primates and in durable protein expression sufficient for sustained restoration of auditory function in Otof knockout mice

    - Multiple analyses demonstrate in vitro transduction with dual AK-OTOF vector results in full-length otoferlin expression, with no detection of truncated proteins

    - Long-term, local expression of anti-VEGF protein is robust and well tolerated following intracochlear administration of AK-antiVEGF in non-human primates

    - Akouos continues to progress towards planned IND submissions for AK-OTOF in the first half of 2022 and for AK-antiVEGF in 2022

    BOSTON, May 11, 2021 (GLOBE…

    - Intracochlear delivery of a dual AAVAnc80 vector encoding human otoferlin results in full-length protein expression in inner hair cells of non-human primates and in durable protein expression sufficient for sustained restoration of auditory function in Otof knockout mice

    - Multiple analyses demonstrate in vitro transduction with dual AK-OTOF vector results in full-length otoferlin expression, with no detection of truncated proteins

    - Long-term, local expression of anti-VEGF protein is robust and well tolerated following intracochlear administration of AK-antiVEGF in non-human primates

    - Akouos continues to progress towards planned IND submissions for AK-OTOF in the first half of 2022 and for AK-antiVEGF in 2022

    BOSTON, May 11, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today presented nonclinical data supporting the future clinical development of both AK-OTOF, a gene therapy intended for the treatment of otoferlin gene (OTOF)-mediated hearing loss, and AK-antiVEGF, a gene therapy intended for the treatment of vestibular schwannoma, in three digital presentation sessions at the virtual American Society of Gene and Cell Therapy (ASGCT) 24th Annual Meeting.

    "We are excited to share new data that highlight the potential of genetic medicines for inner ear conditions with the broader gene therapy community," said Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos. "Inner ear conditions represent one of the largest areas of unmet need in medicine today, and one of the challenges in this area is the ability to efficiently address the broad range of conditions that collectively affect hundreds of millions of individuals worldwide. The nonclinical data presented today for the AK-OTOF and AK-antiVEGF programs demonstrate how we are leveraging our genetic medicines platform and multiple AAV-mediated modalities, including gene transfer and therapeutic protein expression, to begin to address that challenge."

    "Nonclinical data presented at ASGCT for AK-OTOF continue to support the potential to restore physiologic hearing and provide long-lasting benefit to individuals with OTOF-mediated hearing loss. In Otof knockout mice, AK-OTOF administration results in durable expression of human otoferlin protein sufficient for sustained restoration of auditory function. In addition, data presented indicate that expression of exogenous secreted protein at or above reported biologically active levels, driven by a ubiquitous promoter, is well tolerated in non-human primates following administration of AK-antiVEGF. These IND-enabling nonclinical studies are promising and support future clinical development. Our team continues to work towards submission of INDs for AK-OTOF and AK-antiVEGF expected in 2022," said Greg Robinson, Ph.D., chief scientific officer of Akouos.

    In Vitro and In Vivo Analyses of Dual Vector Otoferlin Expression to Support the Clinical Development of AK-OTOF (AAVAnc80-hOTOF Vector)

    Presenting Author: Eva Andres-Mateos

    Abstract Number: 355

    Otoferlin plays a critical role in exocytosis of synaptic vesicles at the inner hair cell synapse, and mutations in OTOF, the gene encoding otoferlin, are associated with autosomal recessive sensorineural hearing loss. AK-OTOF is designed to deliver normal OTOF by utilizing a dual vector approach, which encodes the 5' and the 3' components of OTOF. Multiple analyses demonstrate in vitro transduction with dual AK-OTOF vector results in full-length human otoferlin (RNA and protein), with no detection of truncated proteins from either AK-OTOF or its component vectors (5'hOTOF and 3'hOTOF). A one-to-one ratio of the AK-OTOF component vectors appears to be optimal for efficient reconstitution of full-length human otoferlin. In cynomolgus macaques, full-length human otoferlin protein expression is detected in inner hair cells of non-human primate (NHP) cochleae by both immunohistochemistry and immunodetection one month following intracochlear administration of AAVAnc80-FLAG.hOTOF.

    The digital presentation is located at https://akouos.com/gene-therapy-resources/.

    Durable Recovery of Auditory Function Following Intracochlear Delivery of AK-OTOF (AAVAnc80-hOTOF Vector) in a Translationally Relevant Mouse Model of Otoferlin Gene (OTOF)-Mediated Hearing Loss

    Presenting Author: Ann Hickox

    Abstract Number: 569

    Otoferlin gene (OTOF)-mediated hearing loss is caused by mutations in the OTOF gene and is typically characterized by a congenital, Severe to Profound sensorineural hearing loss. The physiologic deficiency resulting from OTOF mutations is localized; specifically, synaptic transmission between the inner hair cell and the auditory nerve is affected, as measured by an absent or abnormal auditory brain stem response (ABR). Gene therapy for OTOF-mediated hearing loss is expected to confer the greatest benefit when cochlear integrity is preserved, as represented by present otoacoustic emissions (OAEs). Individuals with OTOF-mediated hearing loss typically experience a decline in cochlear integrity within the first decade of life, indicated by initially present, then absent, OAEs.​ In an Otof knockout mouse model that recapitulates the human phenotype, administration of AK-OTOF, an adeno-associated viral gene therapy vector encoding human otoferlin under the control of a ubiquitous promoter, results in durable restoration of auditory function, as measured by ABRs, and may preserve OAEs.

    The digital presentation is located at https://akouos.com/gene-therapy-resources/.

    Demonstration of Tolerability of a Novel Delivery Approach and Secreted Protein Expression Following Intracochlear Delivery of AK-antiVEGF (AAVAnc80-antiVEGF Vector) in Non-Human Primates

    Presenting Author: John Connelly

    Abstract Number: 358

    Data published from previous clinical trials show that systemic VEGF inhibitor therapy can reduce vestibular schwannoma (VS) tumor volume and improve hearing in some participants with mutations in the NF2 gene. However, toxicity limits the potential of this systemic delivery approach from being a viable treatment option for vestibular schwannoma. The exposure and tolerability of local expression of anti-VEGF protein following bilateral, intracochlear administration of AK-antiVEGF was evaluated through analyses of protein levels, as well as physiologic and histologic evaluations, in NHPs. Long-term, local expression of anti-VEGF protein, driven by a ubiquitous promoter, is robust and well tolerated in NHPs following intracochlear administration of AK-antiVEGF. Computational modelling supports the potential for diffusion of anti-VEGF protein at or above reported biologically active levels to the site of the VS tumor.

    The digital presentation is located at https://akouos.com/gene-therapy-resources/.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, and timing of our future clinical trials and our research and development programs, the timing of our IND submissions for AK-OTOF and AK-antiVEGF, our expectations regarding our manufacturing capabilities and timelines, and the period over which we believe that our existing cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our limited operating history; uncertainties inherent in the development of product candidates, including the initiation and completion of nonclinical studies and clinical trials; whether results from nonclinical studies will be predictive of results or success of clinical trials; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents, and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing, and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations; risks related to competitive programs; the potential that our internal manufacturing capabilities and/or external manufacturing supply may experience delays; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the "Risk Factors" included in the Company's Annual Report on Form 10-K for the year ended December 31, 2020 filed with the Securities and Exchange Commission, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com



    Primary Logo

    View Full Article Hide Full Article
  6. BOSTON, May 07, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced the appointment of Dr. Kathy Reape as chief development officer. Dr. Reape brings over 20 years of experience in the pharmaceutical industry, including significant gene therapy translational and development expertise, to Akouos.

    "We welcome Dr. Reape to the Akouos team at this exciting time in the company's growth, as we continue to advance the AK-OTOF and AK-antiVEGF programs towards our planned IND submissions in 2022," said Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer. "As…

    BOSTON, May 07, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced the appointment of Dr. Kathy Reape as chief development officer. Dr. Reape brings over 20 years of experience in the pharmaceutical industry, including significant gene therapy translational and development expertise, to Akouos.

    "We welcome Dr. Reape to the Akouos team at this exciting time in the company's growth, as we continue to advance the AK-OTOF and AK-antiVEGF programs towards our planned IND submissions in 2022," said Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer. "As we continue to forge a path for development of genetic medicines for individuals living with disabling hearing loss, Dr. Reape's broad clinical development expertise and notable recent experience in preparing the marketing application and securing approval for the first FDA-approved in vivo gene therapy for the treatment of a rare inherited retinal disorder will prove invaluable."

    Dr. Reape was most recently Chief Medical Officer at Spark Therapeutics where she oversaw clinical development, pharmacovigilance, and medical affairs activities and was a key member of the team responsible for the development and commercialization of the first FDA-approved in vivo gene therapy, LUXTURNA®, for an inherited retinal disease caused by mutations in both copies of the RPE65 gene. She also oversaw the development of Spark's pipeline of gene therapies addressing CNS disease, hemophilia, metabolic disorders, and inherited retinal dystrophies. Prior to Spark, Dr. Reape was senior vice president of clinical development focusing on global brands research and development at Allergan and Actavis. She holds both her undergraduate and M.D. degrees from the University of Pennsylvania and completed her internship and residency at the University of Florida and the University of Medicine and Dentistry of New Jersey.

    "I am excited to work alongside a team of experts in neurotology, genetics, inner ear drug delivery, and AAV gene therapy who have the unique combination of expertise to progress our shared mission, healthy hearing available to all," said Dr. Reape. "Akouos is positioned to be a leader in genetic medicines for inner ear conditions, an area for which there are no pharmacologic treatment options and is generally underserved. I look forward to working with the Akouos team and the deaf and hard-of-hearing community to evaluate whether gene therapies, such as AK-OTOF and AK-OTOF, could address the needs of individuals and families affected by disabling hearing loss."

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.



    Contacts




    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com



    Primary Logo

    View Full Article Hide Full Article
  7. BOSTON, May 07, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos, will participate in a fireside chat at the Bank of America 2021 Virtual Healthcare Conference on Wednesday, May 12 at 2:00 p.m. EDT.

    A live webcast of the fireside chat will be available on the investors section of the company's website at www.akouos.com. To access the webcast, please go to the Akouos website approximately 15 minutes prior to the start time to ensure adequate time for any software downloads…

    BOSTON, May 07, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos, will participate in a fireside chat at the Bank of America 2021 Virtual Healthcare Conference on Wednesday, May 12 at 2:00 p.m. EDT.

    A live webcast of the fireside chat will be available on the investors section of the company's website at www.akouos.com. To access the webcast, please go to the Akouos website approximately 15 minutes prior to the start time to ensure adequate time for any software downloads that may be required. A replay of the webcast will be available on Akouos's website for 30 days following the conference.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com



    Primary Logo

    View Full Article Hide Full Article
  8. BOSTON, April 27, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that new nonclinical data will be presented at the American Society of Gene and Cell Therapy 24th Annual Meeting, which is being held virtually from May 11 to 14, 2021.

    Three digital presentations will highlight nonclinical data that support future clinical development of AK-OTOF, a gene therapy intended for the treatment of otoferlin gene (OTOF)-mediated hearing loss, and AK-antiVEGF, a gene therapy intended for the treatment of vestibular schwannoma. These digital presentations will occur on Tuesday, May 11…

    BOSTON, April 27, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that new nonclinical data will be presented at the American Society of Gene and Cell Therapy 24th Annual Meeting, which is being held virtually from May 11 to 14, 2021.

    Three digital presentations will highlight nonclinical data that support future clinical development of AK-OTOF, a gene therapy intended for the treatment of otoferlin gene (OTOF)-mediated hearing loss, and AK-antiVEGF, a gene therapy intended for the treatment of vestibular schwannoma. These digital presentations will occur on Tuesday, May 11, 2021 from 8:00 to 10:00 a.m. EDT.

    Details are as follows:

    Digital Presentation Title: In Vitro and In Vivo Analyses of Dual Vector Otoferlin Expression to Support the Clinical Development of AK-OTOF (AAVAnc80-hOTOF Vector)

    Presenting Author: Eva Andres-Mateos

    Session Title: AAV Vectors - Preclinical and Proof-of-Concept Studies

    Abstract Number: 355

    Digital Presentation Title: Durable Recovery of Auditory Function Following Intracochlear Delivery of AK-OTOF (AAVAnc80-hOTOF Vector) in a Translationally Relevant Mouse Model of Otoferlin Gene (OTOF)-Mediated Hearing Loss

    Presenting Author: Ann Hickox

    Session Title: Neurologic Diseases

    Abstract Number: 569

    Digital Presentation Title: Demonstration of Tolerability of a Novel Delivery Approach and Secreted Protein Expression Following Intracochlear Delivery of AK-antiVEGF (AAVAnc80-antiVEGF Vector) in Non-Human Primates

    Presenting Author: John Connelly

    Session Title: AAV Vectors - Preclinical and Proof-of-Concept Studies

    Abstract Number: 358

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Contacts



    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com



    Primary Logo

    View Full Article Hide Full Article
  9. BOSTON, April 13, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss. Akouos anticipates that it will submit an investigational new drug application (IND) for AK-OTOF in the first half of 2022.

    Otoferlin gene (OTOF)-mediated hearing loss is a form of sensorineural hearing loss caused by mutations in the OTOF gene. The OTOF gene encodes otoferlin…

    BOSTON, April 13, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene-mediated hearing loss. Akouos anticipates that it will submit an investigational new drug application (IND) for AK-OTOF in the first half of 2022.

    Otoferlin gene (OTOF)-mediated hearing loss is a form of sensorineural hearing loss caused by mutations in the OTOF gene. The OTOF gene encodes otoferlin, a protein that enables the inner hair cells of the cochlea to release neurotransmitter vesicles in response to stimulation by sound to activate auditory neurons. Most individuals with OTOF-mediated hearing loss have Severe-to-Profound sensorineural hearing loss from birth and approximately 20,000 individuals are affected in the United States and Europe. AK-OTOF is designed to treat the underlying cause of OTOF-mediated hearing loss through delivery of a transgene using a dual vector technology that results in expression of normal, functional otoferlin protein in the affected cells, namely inner hair cells, in the cochlea.

    "There are currently no pharmacologic treatment options for individuals with OTOF-mediated hearing loss, or for any other form of sensorineural hearing loss. The nonclinical data reported to date for AK-OTOF demonstrate durable recovery of auditory function and support future clinical development." said Jen Wellman, chief operating officer of Akouos. "We believe these are the first Orphan Drug and Rare Pediatric Disease designations granted by FDA for a genetic form of hearing loss, and represent an important milestone for the field of inner ear genetic medicines. The receipt of both designations could help us accelerate development of AK-OTOF, a therapy that we believe has the potential to restore physiologic hearing and provide long-lasting benefits to these individuals and their families."

    The Office of Orphan Products Development of FDA grants ODD to drugs and biologics intended for the treatment, diagnosis, or prevention of rare diseases, or conditions affecting fewer than 200,000 people in the United States. The designation affords Akouos the potential for certain benefits, including up to seven years of post-approval market exclusivity, assistance in the drug development process, tax credits for clinical development, and exemptions from certain FDA fees.

    Rare pediatric disease designation is granted by FDA to encourage development of treatments for serious or life-threatening rare diseases in which the disease manifestations primarily affect individuals aged from birth to 18 years. Under the Priority Review Voucher program, and subject to FDA approval of AK-OTOF for the treatment of OTOF-mediated hearing loss, Akouos may be eligible to receive one priority review voucher, which could then be redeemed to receive priority review for a subsequent marketing application for a different product or sold or transferred to another sponsor.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the timing of our IND submission for AK-OTOF and the potential receipt of a priority review voucher and other benefits from the ODD and RPDD. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our limited operating history; uncertainties inherent in the development of product candidates, including the initiation and completion of nonclinical studies and clinical trials; whether results from nonclinical studies will be predictive of results or success of clinical trials; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our ability to realize the expected benefits of ODD and RPDD; the potential for changes in regulatory requirements; our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents, and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing, and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations; risks related to competitive programs; the potential that our internal manufacturing capabilities and/or external manufacturing supply may experience delays; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the "Risk Factors" section included in the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2020 filed with the Securities and Exchange Commission, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com



    Primary Logo

    View Full Article Hide Full Article
  10. BOSTON, April 08, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., founder, president, and CEO of Akouos, will participate in a fireside chat at the 20th Annual Needham Virtual Healthcare Conference on Wednesday, April 14 at 4:30 p.m. ET.

    A live webcast of the fireside chat will be available on the investors section of the company's website at www.akouos.com. To access the webcast, please go on to the Akouos website approximately 15 minutes prior to the start time to ensure adequate time for any software downloads that may be required. A…

    BOSTON, April 08, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., founder, president, and CEO of Akouos, will participate in a fireside chat at the 20th Annual Needham Virtual Healthcare Conference on Wednesday, April 14 at 4:30 p.m. ET.

    A live webcast of the fireside chat will be available on the investors section of the company's website at www.akouos.com. To access the webcast, please go on to the Akouos website approximately 15 minutes prior to the start time to ensure adequate time for any software downloads that may be required. A replay of the webcast will be available on Akouos's website for 14 days following the conference.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com



    Primary Logo

    View Full Article Hide Full Article
  11. - In 2020, continued to advance genetic medicine pipeline with execution of IND-enabling studies for AK-OTOF and general alignment with FDA on the path to a 2022 IND submission for AK-antiVEGF

    - Raised approximately $349 million in gross proceeds, which is expected to fund operations for at least two years

    - Due to recent third-party manufacturing delays, IND submission for AK-OTOF program now expected in the first half of 2022; all other IND-enabling activities remain on track

    - Establishing internal cGMP manufacturing infrastructure and capabilities in 2021

    BOSTON, March 29, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals…

    - In 2020, continued to advance genetic medicine pipeline with execution of IND-enabling studies for AK-OTOF and general alignment with FDA on the path to a 2022 IND submission for AK-antiVEGF

    - Raised approximately $349 million in gross proceeds, which is expected to fund operations for at least two years

    - Due to recent third-party manufacturing delays, IND submission for AK-OTOF program now expected in the first half of 2022; all other IND-enabling activities remain on track

    - Establishing internal cGMP manufacturing infrastructure and capabilities in 2021

    BOSTON, March 29, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today reported financial results for the fourth quarter and full year ended December 31, 2020 and provided business highlights.

    "2020 was a year of tremendous progress for Akouos, marked by continued advancement of our pipeline, expansion of our team, and strengthening of our capital position to further our leadership in the development of precision genetic medicines for inner ear conditions," said Manny Simons, Ph.D., founder, president, and CEO of Akouos. "We continue to be excited by the nonclinical data reported to date, which demonstrate the durable recovery of function of AK-OTOF. Due to third-party manufacturing delays, including impacts from the ongoing COVID-19 pandemic, we now expect to submit the IND for AK-OTOF in the first half of 2022. All other IND-enabling activities remain on track. We continue to work with multiple third-party manufacturers to advance cGMP campaigns, for both the AK-OTOF and AK-antiVEGF IND submissions planned for 2022, and we continue to build our internal cGMP manufacturing infrastructure and capabilities."

    Business and Pipeline Highlights for 2020, Recent Developments, and Anticipated Milestones

    • Continued to build pipeline with potential for broad applicability for inner ear conditions – In 2020, Akouos made progress towards Investigational New Drug (IND) submissions to the U.S. Food and Drug Administration (FDA) for AK-OTOF, a gene therapy intended for the treatment of otoferlin gene (OTOF)-mediated hearing loss, and AK-antiVEGF, a gene therapy intended for the treatment of vestibular schwannoma. Earlier this year, Akouos completed internal manufacturing of AK-antiVEGF for an IND-enabling good laboratory practices (GLP) toxicology study. Additionally, in 2020, the Company selected a product candidate for the AK-CLRN1 program. In 2021, the Company plans to announce a product candidate for its GJB2 program, and targets for its hair cell regeneration and autosomal dominant hearing loss programs.
    • Updated timeline for AK-OTOF IND submission – The submission of an IND to FDA for AK-OTOF is now planned for the first half of 2022 due to third-party manufacturing delays, including delays related to the COVID-19 pandemic. The manufacturing process for Akouos's product candidates, including AK-OTOF, has resulted in seven (of seven) successful at-scale batches of AAVAnc80 vectors. These third-party and internal manufacturing activities were performed using the same clinical-scale, and planned commercial-scale, manufacturing process intended for cGMP manufacturing of Akouos's product candidates. Apart from the third-party cGMP manufacturing delays, all other IND-enabling activities remain on track.
    • Improving access to genetic testing for eligible individuals with auditory neuropathy – In January 2021, Akouos announced the Resonate™ program with Blueprint Genetics. The program provides access to the Blueprint Genetics Comprehensive Hearing Loss and Deafness Panel that includes more than 230 genes associated with genetic forms of hearing loss, helping individuals and their healthcare providers better understand an individual's condition and foster connections within the deaf and hard of hearing community.
    • Data presentation at ARO supporting Akouos's novel delivery approach In February 2021, Akouos presented data at the Association for Research in Otolaryngology (ARO) that demonstrated that intracochlear administration of AAVAnc80 is well tolerated by non-human primates at doses that achieve efficient transduction of target cells. Akouos's novel delivery approach utilizes a minimally invasive surgical approach, a delivery device that is designed for delivery of a product candidate in a fixed volume and at a controlled flow rate, and direct intracochlear administration that allows for distribution of product candidates along the full length of the cochlea.
    • Expanded Akouos team and board of directors with multiple appointments – By the end of 2020, Akouos had expanded to a total of 67 team members with expertise across a range of critical functions, including research, clinical development, regulatory, quality, technical operations, finance, and legal. The Company added Heather Preston, Saira Ramasastry, and Vicki Sato to the board of directors, and named Arthur Tzianabos as chairman.
    • Ended 2020 with a strong cash position of $308.0 million to continue to drive growth across the Company and within the pipeline – In February 2020, Akouos executed a Series B financing raising $105.0 million in gross proceeds. In June 2020, Akouos completed an upsized initial public offering raising $244.4 million in gross proceeds.

    Fourth Quarter and Full Year 2020 Financial Results

    • Cash Position – Cash, cash equivalents, and marketable securities were $308.0 million as of December 31, 2020, as compared to $25.1 million as of December 31, 2019. Akouos expects its cash balance to fund operations for at least the next two years.
    • Research and Development (R&D) Expenses – R&D expenses were $8.0 million for the fourth quarter of 2020 and $34.3 million for the full year ended December 31, 2020, compared to $8.5 million for the fourth quarter of 2019 and $20.5 million for the full year ended December 31, 2019. The increase was primarily due to the increased efforts in IND-enabling studies for AK-OTOF and the growth in the number of R&D employees and their related activities, as well as the expense allocated to R&D related to Akouos's leased facilities.
    • General and Administrative (G&A) Expenses – G&A expenses were $4.6 million for the fourth quarter of 2020 and $14.6 million for the full year ended December 31, 2020, compared to $1.1 million for the fourth quarter of 2019 and $3.4 million for the full year ended December 31, 2019. The increase was primarily due to the growth in the number of G&A employees and other administrative expenses related to operating as a public company, as well as the expense allocated to G&A related to Akouos's leased facilities.
    • Net Loss – Net 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 full year ended December 31, 2020, compared to $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 full year ended December 31, 2019.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, and timing of our future clinical trials and our research and development programs, the timing of our IND submissions for AK-OTOF and AK-antiVEGF, our expectations regarding our manufacturing capabilities and timelines, and the period over which we believe that our existing cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our limited operating history; uncertainties inherent in the development of product candidates, including the initiation and completion of nonclinical studies and clinical trials; whether results from nonclinical studies will be predictive of results or success of clinical trials; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents, and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing, and manufacturing capabilities and strategy; our ability to obtain and maintain intellectual property protection for our product candidates; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations; risks related to competitive programs; the potential that our internal manufacturing capabilities and/or external manufacturing supply may experience delays; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the "Risk Factors" included in the Company's Quarterly Report on Form 10-Q for the three months ended September 30, 2020 filed with the Securities and Exchange Commission, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. 

    Condensed Consolidated Balance Sheet Data

    (Unaudited)

    (in thousands)

           
      December 31, 2020 December 31, 2019
          
    Cash, cash equivalents and marketable securities $308,010 $25,078 
    Total assets  333,350  45,162 
    Total liabilities  22,736  19,273 
    Convertible preferred stock    58,690 
    Total stockholders' equity (deficit)  310,614  (32,801)

    Condensed Consolidated Statements of Operations and Comprehensive Loss

    (Unaudited)

    (in thousands, except share and per share data)

                 
      Three months ended December 31, Year ended December 31,
     2020  2019  2020  2019  
                 
    Operating expenses:                         
    Research and development$7,977  $8,475  $34,297  $20,473  
    General and administrative 4,646   1,134   14,583   3,410  
    Total operating expenses 12,623   9,609   48,880   23,883  
    Loss from operations (12,623)  (9,609)  (48,880)  (23,883) 
    Other income (expense):                
    Change in fair value of preferred stock tranche liability          (2,260) 
    Interest income 366   115   567   413  
    Other income (expense), net (291)  (2)  (287)  (11) 
    Total other income (expense), net 75   113   280   (1,858) 
    Net loss$(12,548) $(9,496) $(48,600) $(25,741) 
    Weighted‑average common shares outstanding, basic and diluted 34,217,475   663,659   17,550,847   605,824  
    Net loss per share attributable to common stockholders, basic and diluted$(0.37) $(14.31) $(2.77) $(42.49) 
                 

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com

     



    Primary Logo

    View Full Article Hide Full Article
    • The Resonate™ program provides access to a potential genetic diagnosis by offering genetic testing at no cost to eligible individuals, their insurance, or their healthcare providers
    • A genetic diagnosis may help individuals and their healthcare providers better understand an individual's condition and foster connections within the deaf and hard of hearing community
    • In the future, as novel genetic medicines for disabling hearing loss enter clinical trials, a genetic diagnosis could also help individuals with auditory neuropathy and their healthcare providers determine their potential eligibility​

    BOSTON, Jan. 11, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential…

    • The Resonate™ program provides access to a potential genetic diagnosis by offering genetic testing at no cost to eligible individuals, their insurance, or their healthcare providers
    • A genetic diagnosis may help individuals and their healthcare providers better understand an individual's condition and foster connections within the deaf and hard of hearing community
    • In the future, as novel genetic medicines for disabling hearing loss enter clinical trials, a genetic diagnosis could also help individuals with auditory neuropathy and their healthcare providers determine their potential eligibility​

    BOSTON, Jan. 11, 2021 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, and Blueprint Genetics, a Quest Diagnostics company, today announced the Resonate program. The program offers genetic testing to eligible individuals with auditory neuropathy at no cost to participants, their insurance, or their healthcare providers.

    Every year, thousands of children are born with disabling hearing loss and the majority of cases of congenital hearing loss are due to an underlying genetic cause. However, few individuals receive a genetic diagnosis and a key barrier is the availability and accessibility of genetic testing. The Resonate program seeks to overcome the barriers to genetic testing for individuals with auditory neuropathy, empower them to make informed choices, and provide potentially valuable insight to inform medical management.

    "Akouos is committed to deepening the community's understanding of the genetic forms of auditory neuropathy, a type of sensorineural hearing loss. Today, there are no pharmacologic therapies approved for the treatment of hearing loss. The development of new therapeutic options for underserved patient populations has become increasingly reliant on identification of the underlying genetic cause of a genetic disease, disorder, or condition," said Manny Simons, Ph.D., founder, president, and CEO of Akouos. "We hope the Resonate program, along with our efforts to better understand the genetic causes and natural history of multiple forms of sensorineural hearing loss, will enable the research and development of novel genetic medicines​ for individuals with disabling hearing loss."

    "There are many different genetic changes that cause hearing loss. These changes can be complex and difficult for routine genetic testing to detect. Understanding the genetic cause of hearing loss requires a high-quality genetic testing strategy that includes the mitochondrial genome, difficult-to-sequence genes, and deep intronic variants in addition to copy number variants in order to increase the diagnostic potential for the program participant. We are excited to work with Akouos to offer our comprehensive genetic testing approach to individuals with auditory neuropathy," says Tero-Pekka Alastalo, M.D., Ph.D., executive medical director of Blueprint Genetics.

    The Resonate program is available in the United States and plans to expand to additional geographic regions throughout 2021. To be eligible for the program, individuals can be any age, and must have a current or prior clinical diagnosis of auditory neuropathy, or a medical history consistent with auditory neuropathy. Auditory neuropathy is a hearing disorder in which the inner ear successfully detects sound, but has a problem with sending sound from the ear to the brain. In many cases, genetic mutations cause auditory neuropathy. The Resonate program provides access to the Blueprint Genetics Comprehensive Hearing Loss and Deafness Panel that includes more than 230 genes associated with genetic forms of hearing loss. To help understand their results, participants in the United States also have access to genetic counseling provided by the program at no cost to participants, their insurance, or their healthcare providers.

    For more information on the Resonate program, individuals and families please visit www.akouosresonate.com and healthcare providers please visit www.blueprintgenetics.com/resonate-program.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy. www.akouos.com

    About Blueprint Genetics

    Blueprint Genetics, a Quest Diagnostics company, is a leading specialty genetics and bioinformatics company focused on providing genetic testing for inherited diseases. The company is based in Helsinki and Seattle, with a customer base spanning over 70 countries. www.blueprintgenetics.com

    Cautionary Note Regarding Akouos Forward-Looking Statements

    Statements in this press release about Akouos's future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the expectations for the Resonate program and the roll-out of the Resonate program. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: Akouos's plans to develop and, if approved, subsequently commercialize Akouos's product candidates; the timing of and Akouos's ability to submit applications for, and obtain and maintain regulatory approvals for, Akouos's product candidates; Akouos's expectations regarding Akouos's regulatory strategy; Akouos's expectations regarding Akouos's ability to fund Akouos's operating expenses and capital expenditure requirements with Akouos's cash, cash equivalents and marketable securities; the potential advantages of Akouos's product candidates; the rate and degree of market acceptance and clinical utility of Akouos's product candidates; Akouos's estimates regarding the potential addressable patient population for Akouos's product candidates; Akouos's commercialization, marketing and manufacturing capabilities and strategy; Akouos's expectations regarding Akouos's ability to obtain and maintain intellectual property protection for Akouos's product candidates; Akouos's intellectual property position; Akouos's ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with Akouos's commercial objectives; the impact of government laws and regulations; Akouos's competitive position and expectations regarding developments and projections relating to Akouos's competitors and any competing therapies that are or become available; developments and expectations regarding developments and projections relating to Akouos's competitors and Akouos's industry; the impact of the COVID-19 pandemic on Akouos's business, results of operations, and financial condition; Akouos's ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the "Risk Factors" included in Akouos's Quarterly Report on Form 10-Q for the three months ended September 30, 2020 filed with the Securities and Exchange Commission, and in other filings that Akouos makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and Akouos expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

    Akouos

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com

    Blueprint Genetics

    Tero-Pekka Alastalo, executive medical director

    tpa@blueprintgenetics.com

    Saara Salonoja, communication specialist

    saara.salonoja@blueprintgenetics.com



    Primary Logo

    View Full Article Hide Full Article
  12. BOSTON, Nov. 23, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., M.B.A., CEO, President and co-founder, participated in a pre-recorded fireside chat that will be published today as part of the Piper Sandler 32nd Annual Virtual Healthcare Conference taking place December 1 to 3, 2020.

    The fireside chat will be available for on-demand viewing through the investor relations section of Akouos's website at www.akouos.com, beginning today, Monday, November 23, 2020, and will be available for 14 days following the conference.

    About Akouos

    Akouos…

    BOSTON, Nov. 23, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., M.B.A., CEO, President and co-founder, participated in a pre-recorded fireside chat that will be published today as part of the Piper Sandler 32nd Annual Virtual Healthcare Conference taking place December 1 to 3, 2020.

    The fireside chat will be available for on-demand viewing through the investor relations section of Akouos's website at www.akouos.com, beginning today, Monday, November 23, 2020, and will be available for 14 days following the conference.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com



    Primary Logo

    View Full Article Hide Full Article
  13. - Expanded leadership team with appointment of Sachiyo Minegishi as CFO and promotion of Jennifer Wellman to COO -

    - Continued progress towards 2021 IND submission for AK-OTOF, a gene therapy intended for the treatment of hearing loss due to mutations in the OTOF gene -

    - Execution on build of internal manufacturing capabilities and infrastructure to support future research and clinical trials is on track -

    BOSTON, Nov. 12, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today reported financial results for the third quarter ended September 30, 2020 and provided updated business highlights…

    - Expanded leadership team with appointment of Sachiyo Minegishi as CFO and promotion of Jennifer Wellman to COO -

    - Continued progress towards 2021 IND submission for AK-OTOF, a gene therapy intended for the treatment of hearing loss due to mutations in the OTOF gene -

    - Execution on build of internal manufacturing capabilities and infrastructure to support future research and clinical trials is on track -

    BOSTON, Nov. 12, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today reported financial results for the third quarter ended September 30, 2020 and provided updated business highlights.

    "We continue to advance AK-OTOF, a gene therapy intended for the treatment of hearing loss due to mutations in the OTOF (otoferlin) gene, towards a 2021 IND filing. Today, individuals with OTOF-mediated hearing loss have no therapeutic options. Our novel gene therapy candidate has the potential to restore hearing for these individuals, who typically have no functional hearing at birth," said Manny Simons, Ph.D., founder, president and CEO of Akouos. "Also, despite the challenging environment around us, the team is executing on our plan to establish internal manufacturing capabilities and infrastructure to support IND-enabling studies and clinical trials. Our progress towards our long-term mission, making healthy hearing available to all, is a testament to our deeply committed, experienced team and our strategic partners."

    Business and Pipeline Highlights

    • Appointed Sachiyo Minegishi as chief financial officer – In October 2020, Sachiyo Minegishi joined Akouos as chief financial officer. Ms. Minegishi has 20 years of experience in the biotechnology and pharmaceutical industry, serving in various roles at global companies, as well as in investment banking, most recently leading the gene therapy program for sickle cell disease at bluebird bio.



    • Promoted Jennifer Wellman to chief operating officer – In October 2020, Akouos announced the promotion of Jennifer Wellman to chief operating officer, from her prior role as senior vice president of regulatory and quality. Ms. Wellman brings to Akouos over 20 years of experience in adeno-associated viral (AAV) vector gene therapy research and development, and was previously co-founder and head of product development strategy at Spark Therapeutics, Inc.



    • Continued progress towards IND filing in 2021 to enable a Phase 1/2 clinical trial for AK-OTOF, a gene therapy intended for the treatment of hearing loss due to mutations in the OTOF gene Through a targeted delivery of a proprietary ancestral AAV, known as AAVAnc80, containing the OTOF gene, Akouos aims to restore otoferlin expression, potentially restoring physiologic hearing and providing long-lasting benefits to individuals with OTOF-mediated hearing loss. Akouos plans to submit an IND application to the FDA in 2021 to conduct a Phase 1/2 clinical trial. The planned Phase 1/2 clinical trial consists of two parts. The first part is a dose escalation phase designed to assess the safety, tolerability and bioactivity of AK-OTOF, administered to trial participants through a single unilateral intracochlear injection. The second part is a cohort expansion phase designed to assess safety and efficacy.



    • Execution on build of internal manufacturing capabilities to support future research and clinical activities is on track Akouos is leveraging its expertise in gene therapy to develop internal, scalable manufacturing capabilities to support research, including IND-enabling studies, and current Good Manufacturing Practice activities for clinical trials. The plans to internalize manufacturing will enable more influence on the manufacturing process, associated analytics, and supporting quality systems, as well as increase the ability to control timelines, costs, and intellectual property.

    Third Quarter 2020 Financial Results

    • Cash Position Cash, cash equivalents and marketable securities were $320.1 million as of September 30, 2020, as compared to $25.1 million as of December 31, 2019. Akouos expects the cash balance to fund operations for at least the next two years.

    • Research and Development (R&D) Expenses R&D expenses were $8.6 million for the third quarter ended September 30, 2020, compared to $4.4 million for the same period in 2019. The increase was primarily due to the increased efforts in preclinical IND-enabling studies for AK-OTOF and the growth in the number of R&D employees and their related activities, as well as the expense allocated to R&D related to Akouos's leased facilities.

    • General and Administrative (G&A) Expenses G&A expenses were $4.5 million for the third quarter ended September 30, 2020, compared to $0.9 million for the same period in 2019. The increase was primarily due to the growth in the number of G&A employees and other administrative expenses, as well as the expense allocated to G&A related to Akouos's leased facilities.

    • Net Loss Net loss was $13.1 million, or $0.85 loss per share, for the third quarter ended September 30, 2020, compared to $8.2 million, or $13.21 loss per share, for the same period in 2019.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, plans, and timing of our future clinical trials and our research and development programs, our expectations regarding our manufacturing capabilities, and the period over which we believe that our existing cash, cash equivalents and marketable securities will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our plans to develop and, if approved, subsequently commercialize our product candidates; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our expectations regarding our ability to fund our operating expenses and capital expenditure requirements with our cash, cash equivalents and marketable securities; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing and manufacturing capabilities and strategy; our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates; our intellectual property position; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations; our competitive position and expectations regarding developments and projections relating to our competitors and any competing therapies that are or become available; developments and expectations regarding developments and projections relating to our competitors and our industry; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the "Risk Factors" included in the Company's Quarterly Report on Form 10-Q for the three months ended June 30, 2020 filed with the Securities and Exchange Commission, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.



    Condensed Consolidated Balance Sheet Data

     (Unaudited)

    (in thousands)

            
      September 30, 2020 December 31, 2019
          
    Cash, cash equivalents and marketable securities $320,074  $25,078 
    Total assets  342,247   45,162 
    Total liabilities  20,939   19,273 
    Convertible preferred stock     58,690 
    Total stockholders' equity (deficit)  321,308   (32,801)
             



    Condensed Consolidated Statements of Operations and Comprehensive Loss

    (Unaudited)

    (in thousands, except share and per share data)

                 
      Three Months Ended  Nine Months Ended
      September 30,  September 30, 
      2020  2019  2020  2019 
             
    Operating expenses:            
    Research and development $8,641  $4,377  $26,612  $11,886 
    General and administrative  4,478   887   9,646   2,267 
    Total operating expenses  13,119   5,264   36,258   14,153 
    Loss from operations  (13,119)  (5,264)  (36,258)  (14,153)
    Other income (expense):            
    Change in fair value of preferred stock tranche liability     (3,013)     (2,260)
    Interest income  21   70   201   298 
    Other income (expense), net  9   (3)  5   (8)
    Total other income (expense), net  30   (2,946)  206   (1,970)
    Net loss $(13,089) $(8,210) $(36,052) $(16,123)
    Net loss per share attributable to common stockholders, basic and diluted $(0.85) $(13.21) $(3.01) $(27.48)
    Weighted‑average common shares outstanding, basic and diluted  15,334,241   621,581   11,991,870   586,728 
    Other comprehensive income:            
    Unrealized gain on marketable securities  8      8    
    Total other comprehensive income  8      8    
    Total comprehensive loss $(13,081) $(8,210) $(36,044) $(16,123)
                 



    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com

    Primary Logo

    View Full Article Hide Full Article
  14. BOSTON, Oct. 01, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, announced the appointment of Sachiyo Minegishi as chief financial officer, effective today. In addition, the company announced that Jennifer Wellman has been promoted to chief operating officer, from her prior role as senior vice president of regulatory and quality.

    "Sachiyo is a strategic leader with deep experience in business development and commercialization strategy, specifically in developing gene therapies for rare diseases. Her leadership will prove invaluable as we continue to build Akouos as a fully-integrated genetic…

    BOSTON, Oct. 01, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, announced the appointment of Sachiyo Minegishi as chief financial officer, effective today. In addition, the company announced that Jennifer Wellman has been promoted to chief operating officer, from her prior role as senior vice president of regulatory and quality.

    "Sachiyo is a strategic leader with deep experience in business development and commercialization strategy, specifically in developing gene therapies for rare diseases. Her leadership will prove invaluable as we continue to build Akouos as a fully-integrated genetic medicine company, and continue to advance our program pipeline of AAV-based genetic medicines with the potential to treat a broad range of inner ear disorders," said Manny Simons, Ph.D., M.B.A., CEO, president and co-founder. "Jennifer's promotion to chief operating officer is reflective of her significant contributions to Akouos and the integral role she has played in our growth over the last several years. We look forward to her continued leadership in this expanded role."

    Ms. Minegishi has 20 years of experience in the biotechnology and pharmaceutical industry, serving in various roles at global companies, as well as investment banking. Most recently, Ms. Minegishi led the gene therapy program for Sickle Cell Disease at bluebird bio to initiation of Phase 3 studies and regulatory alignment on clinical path to registration. Earlier, Ms. Minegishi held leadership roles at Aegerion Pharmaceuticals, Human Genome Sciences, Genzyme, and Amgen. She began her career in investment banking at Merrill Lynch, with a focus on execution of equity financing and mergers and acquisitions in the biotechnology industry. Ms. Minegishi earned a B.S. in Chemical Engineering and Economics from the Massachusetts Institute of Technology and an M.B.A. from the Wharton School of the University of Pennsylvania.

    "As Akouos enters this critical stage of advancing towards the clinic, I am excited to work alongside the entire team at Akouos and hearing loss community to develop precision genetic medicines for patients with disabling hearing loss," said Ms. Minegishi. "Akouos is uniquely positioned to rapidly advance towards the goal of restoring, improving, and preserving hearing and I am honored to have the opportunity to contribute my expertise."

    Ms. Wellman has over 20 years of experience in adeno-associated viral (AAV) vector gene therapy research and development. Prior to joining Akouos in early 2018, Ms. Wellman was co-founder and head of product development strategy at Spark Therapeutics, Inc. While at Spark, and at Children's Hospital of Philadelphia, she led the regulatory and clinical development for several AAV gene therapies, including Luxturna®, the first FDA-approved gene therapy for a genetic disease. Earlier in her career, she was at Avigen, Inc. in various R&D positions with increasing responsibilities. Ms. Wellman holds an Honours B.S. in Microbiology and Immunology from Queen's University (Canada) and an M.S. from University of New Haven.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com

    Primary Logo

    View Full Article Hide Full Article
  15. BOSTON, Sept. 24, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., M.B.A., CEO, President and co-founder, will present at two upcoming virtual investor conferences in October:

    • Jefferies Gene Therapy/Editing Summit. Fireside chat on Thursday, October 1 at 8:00 a.m. ET
    • Chardan Annual Genetic Medicines Conference. Fireside chat on Monday, October 5 at 8:30 a.m. ET

    A live webcast of each fireside chat will be accessible through the investors section of the company's website at www.akouos.com. To access the webcasts, please go on to the Akouos website approximately…

    BOSTON, Sept. 24, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., M.B.A., CEO, President and co-founder, will present at two upcoming virtual investor conferences in October:

    • Jefferies Gene Therapy/Editing Summit. Fireside chat on Thursday, October 1 at 8:00 a.m. ET
    • Chardan Annual Genetic Medicines Conference. Fireside chat on Monday, October 5 at 8:30 a.m. ET

    A live webcast of each fireside chat will be accessible through the investors section of the company's website at www.akouos.com. To access the webcasts, please go on to the Akouos website approximately 15 minutes prior to the start time to ensure adequate time for any software downloads that may be required. Replays of each webcast will be available on Akouos's website for 14 days following the conferences.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com

    Primary Logo

    View Full Article Hide Full Article
  16. BOSTON, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., M.B.A., CEO, President and co-founder, will participate in a fireside chat at the Cantor Global Virtual Healthcare Conference on Tuesday, September 15 at 8:00 a.m. ET.

    A live webcast of the fireside chat will be accessible through the investors section of the company's website at www.akouos.com. To access the webcast, please go on to the Akouos website approximately 15 minutes prior to the start time to ensure adequate time for any software downloads that may be required…

    BOSTON, Sept. 08, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced that Manny Simons, Ph.D., M.B.A., CEO, President and co-founder, will participate in a fireside chat at the Cantor Global Virtual Healthcare Conference on Tuesday, September 15 at 8:00 a.m. ET.

    A live webcast of the fireside chat will be accessible through the investors section of the company's website at www.akouos.com. To access the webcast, please go on to the Akouos website approximately 15 minutes prior to the start time to ensure adequate time for any software downloads that may be required. A replay of the webcast will be available on Akouos's website for 14 days following the conference.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com

    Primary Logo

    View Full Article Hide Full Article
  17. - Upsized IPO in June 2020 raised $244.4 million in gross proceeds -

    - Company plans to advance lead product candidate, AK-OTOF, for the treatment of hearing loss due to mutations in the OTOF gene, to IND submission in 2021 and advance development programs spanning multiple inner ear disorders -

    BOSTON, Aug. 13, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today reported financial results for the second quarter ended June 30, 2020 and provided an update on recent business highlights.

    "Our upsized initial public offering, which we completed in June, leaves us in a strong…

    - Upsized IPO in June 2020 raised $244.4 million in gross proceeds -

    - Company plans to advance lead product candidate, AK-OTOF, for the treatment of hearing loss due to mutations in the OTOF gene, to IND submission in 2021 and advance development programs spanning multiple inner ear disorders -

    BOSTON, Aug. 13, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today reported financial results for the second quarter ended June 30, 2020 and provided an update on recent business highlights.

    "Our upsized initial public offering, which we completed in June, leaves us in a strong position to continue to advance our lead product candidate, AK-OTOF, for the treatment of hearing loss due to mutations in the OTOF (otoferlin) gene, into the clinic and further develop our pipeline of programs that leverage various AAV-mediated modalities to treat multiple inner ear disorders," said Manny Simons, Ph.D., founder, president and CEO of Akouos. "We have built a team with deep experience and expertise, which will be a tremendous asset as we continue to execute on our mission to make healthy hearing available to all."

    Recent Business Highlights

    • Appointed Alan Smith, Ph.D., as chief technology officer – In June 2020, Alan Smith, Ph.D., joined Akouos as chief technology officer. Dr. Smith brings over 30 years of experience in cellular therapeutics and previously served as executive vice president, technical operations at Bellicum Pharmaceuticals.



    • Strengthened board of directors with appointment of Saira Ramasastry – In June 2020, Saira Ramasastry joined the board of directors of Akouos. Ms. Ramasastry currently serves as managing partner of Life Sciences Advisory, a firm that she founded to provide strategic advice and business development solutions for life science companies.



    • Expanded the role of Arthur Tzianabos, Ph.D. to chairman of board of directors – In August 2020, Akouos announced the appointment of its board member Arthur Tzianabos to chairman of the board of directors.



    • Completed upsized Initial Public Offering – In June 2020, Akouos completed its initial public offering of 14,375,000 shares of common stock at a public offering price of $17.00 per share, including the full exercise of the underwriter's option to purchase additional shares. Gross proceeds from the IPO were $244.4 million and net proceeds from the offering, after deducting underwriting discounts, commissions and offering expenses, were approximately $223.8 million.



    • Presented two posters at the American Society of Gene & Cell Therapy 23rd Annual Meeting – In May 2020, Akouos presented two posters highlighting Akouos's use of AAVAnc80 vector technology and its potential to address many forms of hearing loss.

    Second Quarter 2020 Financial Results

    • Cash Position Cash and cash equivalents were $333.0 million as of June 30, 2020, as compared to $120.2 million as of March 31, 2020. Total cash, cash equivalents and marketable securities at June 30, 2020 includes total net proceeds of approximately $223.8 million from the company's IPO in June 2020. Akouos expects the cash balance to fund operations for at least the next two years.
    • Research and Development (R&D) Expenses R&D expenses were $9.9 million for the second quarter ended June 30, 2020, compared to $4.4 million for the same period in 2019. The increase was primarily due to contract manufacturing costs, the initiation of preclinical IND-enabling studies for AK-OTOF, and the growth in the number of R&D employees and their related activities, as well as the expense allocated to R&D related to Akouos's leased facilities.
    • General and Administrative (G&A) Expenses G&A expenses were $2.7 million for the second quarter ended June 30, 2020, compared to $0.7 million for the same period in 2019. The increase was primarily due to the growth in the number of G&A employees and other administrative expenses, as well as the expense allocated to G&A related to Akouos's leased facilities.
    • Net Loss Net loss was $12.5 million, or $11.14 loss per share, for the second quarter ended June 30, 2020, compared to $2.0 million, or $3.42 loss per share, for the same period in 2019.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Cautionary Note Regarding Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, and timing, of our future clinical trials and our research and development programs, and the period over which we believe that our existing cash and cash equivalents will be sufficient to fund our operating expenses. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: our plans to develop and, if approved, subsequently commercialize our product candidates; the timing of and our ability to submit applications for, and obtain and maintain regulatory approvals for, our product candidates; our expectations regarding our regulatory strategy; our expectations regarding our ability to fund our operating expenses and capital expenditure requirements with our cash and cash equivalents; the potential advantages of our product candidates; the rate and degree of market acceptance and clinical utility of our product candidates; our estimates regarding the potential addressable patient population for our product candidates; our commercialization, marketing and manufacturing capabilities and strategy; our expectations regarding our ability to obtain and maintain intellectual property protection for our product candidates; our intellectual property position; our ability to identify additional products, product candidates, or technologies with significant commercial potential that are consistent with our commercial objectives; the impact of government laws and regulations; our competitive position and expectations regarding developments and projections relating to our competitors and any competing therapies that are or become available; developments and expectations regarding developments and projections relating to our competitors and our industry; the impact of the COVID-19 pandemic on our business, results of operations, and financial condition; our ability to maintain and establish collaborations or obtain additional funding; and other factors discussed in the "Risk Factors" included in the Company's final prospectus related to its initial public offering filed with the Securities and Exchange Commission, and in other filings that the Company makes with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.



    Condensed Consolidated Balance Sheet Data

     (Unaudited)

    (in thousands)

           
      June 30, 2020 December 31, 2019
          
    Cash and cash equivalents $ 333,004 $ 25,078 
    Total assets   354,154   45,162 
    Total liabilities   21,898   19,273 
    Convertible preferred stock   —   58,690 
    Total stockholders' equity (deficit)   332,256   (32,801)







    Condensed Consolidated Statements of Operations and Comprehensive Loss


    (Unaudited)

    (in thousands, except share and per share data)

                 
      Three Months Ended  Six Months Ended
      June 30,  June 30, 
      2020  2019  2020  2019 
             
    Operating expenses:            
    Research and development $ 9,937  $ 4,366  $ 17,971  $ 7,509 
    General and administrative   2,664    749    5,168    1,380 
    Total operating expenses   12,601    5,115    23,139    8,889 
    Loss from operations   (12,601)   (5,115)   (23,139)   (8,889)
    Other income (expense):            
    Change in fair value of preferred stock tranche liability   —    3,013    —    753 
    Interest income   80    100    180    228 
    Other income (expense), net   (2)   (2)   (4)   (5)
    Total other income (expense), net   78    3,111    176    976 
    Net loss and comprehensive loss $ (12,523) $ (2,004) $ (22,963) $ (7,913)
    Net loss per share attributable to common stockholders, basic and diluted $ (11.14) $ (3.42) $ (25.05) $ (13.91)
    Weighted‑average common shares outstanding, basic and diluted   1,124,251    586,616    916,521    568,811 

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com

     

    Primary Logo

    View Full Article Hide Full Article
  18. Alan Smith, Ph.D., Joins as Chief Technology Officer

    Saira Ramasastry Joins Board of Directors; Arthur Tzianabos, Ph.D., Role Expanded to Chairman

    BOSTON, Aug. 05, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced the appointment of Alan Smith, Ph.D., as chief technology officer. In addition, the company announced that Saira Ramasastry has been appointed to its board of directors as audit committee chair, and board member Arthur Tzianabos, Ph.D., has been appointed chairman of the board.

    "Alan brings terrific experience in the development and manufacturing of complex…

    Alan Smith, Ph.D., Joins as Chief Technology Officer

    Saira Ramasastry Joins Board of Directors; Arthur Tzianabos, Ph.D., Role Expanded to Chairman

    BOSTON, Aug. 05, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for individuals living with disabling hearing loss worldwide, today announced the appointment of Alan Smith, Ph.D., as chief technology officer. In addition, the company announced that Saira Ramasastry has been appointed to its board of directors as audit committee chair, and board member Arthur Tzianabos, Ph.D., has been appointed chairman of the board.

    "Alan brings terrific experience in the development and manufacturing of complex biologics, and a proven track record of building teams and establishing infrastructure to support in-house GMP manufacturing capabilities," said Manny Simons, Ph.D., founder, president, and chief executive officer of Akouos. "Alan joins Akouos at a pivotal time as we prepare to advance our lead program, AK-OTOF, to IND submission next year. We are also excited to welcome Saira, an esteemed life science leader, to our board of directors, and delighted to expand Arthur's role to chairman of our board. Together, these appointments will be instrumental as Akouos continues to grow into a fully integrated genetic medicine company developing innovative potential therapies for a variety of inner ear disorders."

    Dr. Smith joins Akouos with more than 30 years of experience in research and development, manufacturing, and quality in the areas of cell and gene therapies. He has contributed to more than 25 FDA regulatory submissions for cell therapy products and devices. Prior to Akouos, Dr. Smith was executive vice president, technical operations at Bellicum Pharmaceuticals, where he led cell product manufacturing, viral vector manufacturing, process development, assay development, GMP supply chain and logistics, worldwide facilities functions, and the design, construction, and startup of multiple GMP manufacturing facilities. Previously, Dr. Smith was vice president of research and development and cellular therapeutics for LifeNet Health and its subsidiary, The Institute of Regenerative Medicine. Earlier in his career, Dr. Smith served as president and chief executive officer for Cognate BioServices Inc. and chief operating officer and senior vice president of research and development for Osiris Therapeutics, Inc. Dr. Smith is also a former adjunct professor at Eastern Virginia Medical School, California State University, Long Beach and Utah State University. He holds a B.S. in chemistry from Southern Utah University and a Ph.D. in biochemistry from Utah State University.

    Ms. Ramasastry is managing partner of Life Sciences Advisory, a firm that she founded to provide strategic advice and business development solutions for life science companies. Ms. Ramasastry is also a health innovator fellow of the Aspen Institute and a member of the Aspen Global Leadership Network. Prior to founding Life Sciences Advisory, Ms. Ramasastry was an investment banker with Merrill Lynch & Company, where she helped establish the biotechnology practice and was responsible for origination of mergers and acquisitions, and strategic and capital markets transactions. Prior to joining Merrill Lynch, she served as a financial analyst in mergers and acquisitions group at Wasserstein Perella & Co., an investment banking firm. Ms. Ramasastry currently serves on the board of directors for Vir Biotechnology Inc., Glenmark Pharmaceuticals Ltd., and Sangamo Therapeutics, Inc. She holds a B.A. in economics with honors and distinction and an M.S. in management science and engineering from Stanford University, as well as an M. Phil. in management studies from the University of Cambridge, where she is a guest lecturer for the Bioscience Enterprise Programme and previously served on the Cambridge Judge Business School Advisory Council.

    Initially appointed as an independent director to Akouos's board of directors in July 2018, Dr. Tzianabos has now been appointed to serve as chairman. Dr. Tzianabos is currently the chief executive officer and president of Homology Medicines, Inc., leading the efforts to develop genetic medicines for patients with rare genetic diseases.  Previously, Dr. Tzianabos spent nine years at Shire Plc, where he worked on the development and launches of multiple treatments for patients with rare genetic disorders. Prior to joining Shire, Dr. Tzianabos was an Associate Professor of Medicine at Harvard Medical School and maintained laboratories at the Channing Laboratory, Brigham and Women's Hospital and the Department of Microbiology and Molecular Genetics at Harvard Medical School. He serves on the board of directors for Stoke Therapeutics, Inc., the Alliance for Regenerative Medicine, and the development board for the University of New Hampshire's College of Life Sciences and Agriculture. Dr. Tzianabos holds a B.S. in biology from Boston College and a Ph.D. in Microbiology from the University of New Hampshire.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for individuals living with disabling hearing loss worldwide. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Contact

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com

    Primary Logo

    View Full Article Hide Full Article
  19. BOSTON, Aug. 03, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for people worldwide who live with disabling hearing loss, today announced that Manny Simons, Ph.D., M.B.A., CEO, President and co-founder, will participate in a fireside chat at the BTIG Virtual Biotechnology Conference on Monday, August 10 at 10:30 a.m. ET.

    A live webcast of the fireside chat will be accessible through the investors section of the company's website at www.akouos.com. To access the webcast, please go on to the Akouos website approximately 15 minutes prior to the start time to ensure adequate time for any software downloads that may be required. A replay of…

    BOSTON, Aug. 03, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing potential gene therapies for people worldwide who live with disabling hearing loss, today announced that Manny Simons, Ph.D., M.B.A., CEO, President and co-founder, will participate in a fireside chat at the BTIG Virtual Biotechnology Conference on Monday, August 10 at 10:30 a.m. ET.

    A live webcast of the fireside chat will be accessible through the investors section of the company's website at www.akouos.com. To access the webcast, please go on to the Akouos website approximately 15 minutes prior to the start time to ensure adequate time for any software downloads that may be required. A replay of the webcast will be available on Akouos' website for 14 days following the conference.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Contacts

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com

    Primary Logo

    View Full Article Hide Full Article
  20. BOSTON, June 30, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss, today announced the closing of its initial public offering of 14,375,000 shares of common stock at a public offering price of $17.00 per share, including 1,875,000 additional shares of common stock issued upon the exercise in full by the underwriters of their option to purchase additional shares, for gross proceeds of $244.4 million, before underwriting discounts and commissions and offering expenses payable by Akouos. All shares were offered by…

    BOSTON, June 30, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss, today announced the closing of its initial public offering of 14,375,000 shares of common stock at a public offering price of $17.00 per share, including 1,875,000 additional shares of common stock issued upon the exercise in full by the underwriters of their option to purchase additional shares, for gross proceeds of $244.4 million, before underwriting discounts and commissions and offering expenses payable by Akouos. All shares were offered by Akouos.

    Akouos's common stock began trading on the Nasdaq Global Select Market under the ticker symbol "AKUS" on June 26, 2020. 

    BofA Securities, Cowen, and Piper Sandler acted as joint book-running managers for the offering. BTIG acted as lead manager for the offering.

    A registration statement relating to the offering of these securities was declared effective by the Securities and Exchange Commission (the "SEC") on June 25, 2020. Copies of the registration statement can be accessed by visiting the SEC website at www.sec.gov. This offering was made only by means of a prospectus. A copy of the final prospectus relating to the offering may be obtained from BofA Securities, Inc., NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, or by email at dg.prospectus_requests@baml.com; Cowen and Company, LLC, c/o Broadridge Financial Solutions, Attn: Prospectus Department, 1155 Long Island Avenue, Edgewood, NY 11717, by email at PostSaleManualRequests@broadridge.com or by telephone at (833) 297-2926; or Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, via telephone at (800) 747-3924 or via email at prospectus@psc.com.

    This announcement does not constitute an offer to sell, or the solicitation of an offer to buy, securities, and shall not constitute an offer, solicitation or sale in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that jurisdiction. Any offers, solicitations or offers to buy, or any sales of securities will be made in accordance with the registration requirements of the Securities Act of 1933, as amended.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery, and AAV gene therapy.

    Contact

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com

    Primary Logo

    View Full Article Hide Full Article
  21. BOSTON, June 25, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss, today announced the pricing of its initial public offering of 12,500,000 shares of common stock at a public offering price of $17.00 per share, for gross proceeds of $212.5 million, before underwriting discounts and commissions and offering expenses payable by Akouos. The offering is expected to close on or about June 30, 2020, subject to the satisfaction of customary closing conditions. In addition, Akouos has granted the underwriters an option…

    BOSTON, June 25, 2020 (GLOBE NEWSWIRE) -- Akouos, Inc. ("Akouos") (NASDAQ:AKUS), a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss, today announced the pricing of its initial public offering of 12,500,000 shares of common stock at a public offering price of $17.00 per share, for gross proceeds of $212.5 million, before underwriting discounts and commissions and offering expenses payable by Akouos. The offering is expected to close on or about June 30, 2020, subject to the satisfaction of customary closing conditions. In addition, Akouos has granted the underwriters an option for a period of 30 days to purchase up to 1,875,000 additional shares of common stock at the initial public offering price, less underwriting discounts and commissions. All shares are being offered and sold by Akouos. 

    Akouos' common stock is expected to begin trading on the Nasdaq Global Select Market under the ticker symbol "AKUS" on June 26, 2020.

    BofA Securities, Cowen and Piper Sandler are acting as joint book-running managers for the offering.  BTIG is acting as lead manager for the offering.

    A registration statement relating to the offering of these securities was declared effective by the Securities and Exchange Commission (the "SEC") on June 25, 2020. Copies of the registration statement can be accessed by visiting the SEC website at www.sec.gov. The securities referred to in this release are to be offered only by means of a prospectus. A preliminary prospectus describing the terms of the offering has been filed with the SEC and forms a part of the effective registration statement. When available, a copy of the final prospectus relating to the offering may be obtained from BofA Securities, Inc., NC1-004-03-43, 200 North College Street, 3rd floor, Charlotte, NC 28255-0001, Attn: Prospectus Department, or by email at dg.prospectus_requests@baml.com; Cowen and Company, LLC, c/o Broadridge Financial Solutions, Attn: Prospectus Department, 1155 Long Island Avenue, Edgewood, NY 11717, by email at PostSaleManualRequests@broadridge.com or by telephone at (833) 297-2926; or Piper Sandler & Co., Attention: Prospectus Department, 800 Nicollet Mall, J12S03, Minneapolis, MN 55402, via telephone at (800) 747-3924 or via email at prospectus@psc.com.

    This announcement does not constitute an offer to sell, or the solicitation of an offer to buy, securities, and shall not constitute an offer, solicitation or sale in any jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of that jurisdiction. Any offers, solicitations or offers to buy, or any sales of securities will be made in accordance with the registration requirements of the Securities Act of 1933, as amended.

    About Akouos

    Akouos is a precision genetic medicine company dedicated to developing gene therapies with the potential to restore, improve, and preserve high-acuity physiologic hearing for people worldwide who live with disabling hearing loss. Leveraging its precision genetic medicine platform that incorporates a proprietary adeno-associated viral (AAV) vector library and a novel delivery approach, Akouos is focused on developing precision therapies for forms of sensorineural hearing loss. Headquartered in Boston, Akouos was founded in 2016 by leaders in the fields of neurotology, genetics, inner ear drug delivery and AAV gene therapy.

    Forward-Looking Statements

    Statements in this press release about future expectations, plans and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements". These statements include, but are not limited to, statements relating to the expected trading commencement and the closing date. The words, without limitation, "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these or similar identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: the uncertainties related to market conditions and the completion of the public offering on the anticipated terms of the offering or at all, and other factors discussed in the "Risk Factors" section of the preliminary prospectus that forms a part of the effective registration statement filed with the SEC. Any forward-looking statements contained in this press release are based on the current expectations of Akouos' management team and speak only as of the date hereof, and Akouos specifically disclaims any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise.

    Contact

    Media:

    Katie Engleman, 1AB

    katie@1abmedia.com 

    Investors:

    Courtney Turiano, Stern Investor Relations

    Courtney.Turiano@sternir.com  

    Primary Logo

    View Full Article Hide Full Article