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Eli Lilly: Gene Therapy Clinical Trial Restores Hearing in First Participant Within 30 Days

INDIANAPOLIS, INDIANA — Akouos, Inc., a wholly owned subsidiary of Eli Lilly and Company (NYSE: LLY), announced positive initial clinical results from the Phase 1/2 AK-OTOF-101 study, which demonstrated restored hearing within 30 days of AK-OTOF administration in the first participant, an individual with over a decade of profound hearing loss.

Results, including initial data from a second participant who received AK-OTOF, will be presented during the Late Breaking Presidential Symposium at the 2024 Association for Research in Otolaryngology (ARO) MidWinter Meeting.

Details of the presentation are:

• Title: Clinical development of AK-OTOF gene therapy for OTOF-mediated hearing loss Session: Late Breaking Presidential Symposium Date/Time: Saturday, February 3, 10:00 a.m. PT Location: Anaheim Marriott, Anaheim, California

The first participant to receive AK-OTOF in the study, an 11-year-old at the time of administration with profound hearing loss from birth, experienced restored hearing within 30 days. Hearing was restored across tested frequencies, achieving thresholds of 65 to 20 dB HL, and within the normal hearing range at some frequencies at the Day 30 visit.

Both the administration procedure and the investigational therapy were well tolerated, with no serious adverse events reported.

“Gene therapy for hearing loss is something physicians and scientists have been working toward for over 20 years,” said Professor John Germiller, M.D., Ph.D., attending surgeon and Director of Clinical Research in the Division of Otolaryngology at Children’s Hospital of Philadelphia, and a principal investigator who administered AK-OTOF. “These initial results show it may restore hearing better than many thought possible.”

Gene Therapy Treatment for Hearing Loss

In the AK-OTOF-101 trial, eligible participants receive a single, unilateral intracochlear administration of AK-OTOF, with hearing restoration assessed by audiometry and auditory brainstem response (ABR). Participants in cohort 1 receive AK-OTOF at 4.1E11 total vector genomes.

“Children with OTOF-mediated hearing loss often have profound loss from birth, yet only a fraction have genetic testing to receive a definitive diagnosis,” said Dr. Oliver Haag, pediatric otolaryngologist and Head of Otolaryngology at Sant Joan de Deu Hospital in Barcelona, and an investigator in Akouos’s AK-OTOF-NHS-002 Natural History Study.

“This trial and study show the power of international collaboration for rare genetic conditions. It is gratifying to see this effort benefit the first participant.”

Hearing loss is the most common sensory condition. With no approved drugs to restore hearing, it represents significant unmet need. Delivering a healthy gene to target cells in the inner ear has potential to enable hearing for some cases, including an estimated 200,000 with OTOF-mediated loss. OTOF-mediated loss is the first monogenic form investigated in a gene therapy trial.

“We are grateful to the participants, families, investigators and collaborators working together on this pioneering trial,” said Emmanuel Simons, Ph.D. , M.B.A., CEO of Akouos and SVP, Gene Therapy at Lilly. “These initial results highlight the potential impact genetic medicines could have and reinforce our mission to make healthy hearing available to all.”

AK-OTOF has been granted Orphan Drug Designation and Rare Pediatric Disease Designation by the FDA and a positive opinion on orphan designation by the European Medicines Agency.

About AK-OTOF and the AK-OTOF-101 Clinical Trial AK-OTOF (AAVAnc80-hOTOF) is a dual adeno-associated viral (AAV) vector-based gene therapy designed to restore auditory function by gene transfer and durable expression of normal, functional otoferlin protein to the inner hair cells of the cochlea. AK-OTOF utilizes AAVAnc80, a capsid with high transduction efficiency for inner hair cells, together with a strong ubiquitous promoter to achieve expression of otoferlin, observed only in the target inner hair cells, at levels that have the potential to restore high acuity physiologic hearing. The Akouos delivery device, being developed in parallel specifically for intracochlear administration, enables a minimally invasive surgical approach to deliver AK-OTOF throughout the cochlea. The AK-OTOF-101 Clinical Trial (NCT05821959) is a Phase 1/2 trial that is assessing the safety, tolerability, and bioactivity of escalating doses of AK-OTOF delivered via the Akouos delivery device. More information about the trial can be found at https://www.clinicaltrials.gov/study/NCT05821959.

About the OTOF-mediated Hearing Loss Natural History Study The AK-OTOF-NHS-002 Natural History Study (NCT05572073) is designed to characterize the natural history of OTOF-mediated hearing loss, including progression of physiologic responses and audiologic outcomes over time, potential genotype-phenotype relationships, and longitudinal assessment of clinical outcomes. More information about the study can be found at https://www.clinicaltrials.gov/ct2/show/NCT05572073.

About Lilly Lilly unites caring with discovery to create medicines that make life better for people around the world. We’ve been pioneering life-changing discoveries for nearly 150 years, and today our medicines help more than 51 million people across the globe. Harnessing the power of biotechnology, chemistry and genetic medicine, our scientists are urgently advancing new discoveries to solve some of the world’s most significant health challenges, redefining diabetes care, treating obesity and curtailing its most devastating long-term effects, advancing the fight against Alzheimer’s disease, providing solutions to some of the most debilitating immune system disorders, and transforming the most difficult-to-treat cancers into manageable diseases. With each step toward a healthier world, we’re motivated by one thing: making life better for millions more people. That includes delivering innovative clinical trials that reflect the diversity of our world and working to ensure our medicines are accessible and affordable.

Source: Eli Lilly

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Gene Therapy

Lilly to acquire hearing loss company akouos, the boston biotech’s lead drug treats a form of congenital hearing loss, by alla katsnelson, special to c&en, october 19, 2022.

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Eli Lilly and Company is set to acquire Boston-based Akouos, which is developing gene therapies to counter hearing loss, for $487 million. That sum could climb to $610 million if Akouos’s lead product, which treats a genetic form of hearing loss called otoferlin deficiency, achieves certain clinical trial milestones.

For Lilly, the deal helps expand a nascent presence in genetic medicine , which includes a research center being built in Boston. Akouos is one of a handful of companies developing treatments to prevent sensorineural hearing loss, which can be caused by genetic mutations, drugs such as chemotherapies and antibiotics, and other factors.

Akouos’s front-runner product , AK-OTOF, is one of at least three gene therapies being developed to treat otoferlin deficiency, a profound, congenital hearing loss currently treated with cochlear implants. The disorder is caused by mutations in the otoferlin gene that stifle the ability of the ear’s sensory cells, called hair cells, to transmit neural signals.

The US Food and Drug Administration cleared Akouos’s Investigational New Drug (IND) application for AK-OTOF on Sept. 13, and the company inked its deal with Lilly on Oct. 18, just a day after another Boston-based biotech, Decibel Therapeutics , received IND clearance for a gene therapy targeting the same condition.

The strategies being pursued by Akouos and Decibel have some key differences. For example, while Akouos plans to test its treatment in children over age 2, Decibel, which is working in partnership with Regeneron Pharmaceuticals, will also test in a younger cohort of infants.

“In the longer term, I think that could be a key differentiator, being that you really want to treat these patients as young as possible,” says Jack Allen, a senior research analyst who covers the cell and gene therapy space for the investment firm Robert W. Baird & Co.

The involvement of big companies like Lilly and Regeneron is good news for the hearing-loss space, Allen says. “General interest from these larger players for gene therapy for the ear is positive in terms of providing early validation for these approaches.”

CORRECTION:

This story was updated on Oct. 31, 2022, to correct the description of Akouos. It is developing treatments to prevent all sensorineural hearing loss, not just sensorineural hearing loss caused by genetic mutations. And those treatments do not include drugs such as chemotherapy, antibiotics, and other factors. Those are things that can cause sensorineural hearing loss.

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Akouos (akus) surges on $610m buyout deal with eli lilly.

Shares of Akouos AKUS skyrocketed 88.16% after the company announced a buyout agreement for approximately $610 million with pharma giant Eli Lilly LLY. The stock has rallied 55.1% in the year-to-date period against the industry’s decline of 24.2%.

Image Source: Zacks Investment Research

Per the terms of the definitive agreement, Lilly will acquire all outstanding shares of Akouos for an equity value of $487 million, or $12.50 per share, in cash, and one contingent value right (CVR) up to $3.00 per share. The offer price of $12.50 represents a premium of 78% of Akouos’s closing price on Oct 17.

The acquisition, approved by the board of directors of the companies, is expected to close in the fourth quarter of 2022 and is subject to other customary closing conditions.

For Lilly, the acquisition is expected to strengthen its gene therapy profile by adding Akouos’s pre-clinical pipeline of gene therapies to treat monogenic inner ear conditions.

Shares of Lilly have rallied 22.6% in the year against the industry’s decline of 0.1%.

Akouos’s portfolio includes its lead product candidate, AK-OTOF, which recently received an FDA clearance for its investigational new drug (IND) application. The company will soon initiate a phase I/II, first-in-human, pediatric study for treating patients with otoferlin gene ( OTOF )-mediated hearing loss. Currently, no approved treatment options exist for patients with OTOF-mediated hearing loss.

The company also has additional product candidates in its portfolio, including AK-CLRN1 for Usher Type 3A, an autosomal recessive disorder characterized by progressive loss of hearing and vision, GJB2 for a common form of monogenic deafness and hearing loss and AK-antiVEGF for treating vestibular schwannoma.

Akouos, Inc. Price

Akouos, Inc. price | Akouos, Inc. Quote

Zacks Rank and Key Picks

Akouos and Lilly both carry a Zacks Rank #3 (Hold).

Some better-ranked stocks worth considering in the same sector are ACADIA Pharmaceuticals ACAD and BioMarin Pharmaceuticals BMRN, each carrying a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 (Strong Buy) Rank stocks here .

Acadia’s loss per share estimates for 2022 have narrowed down from $1.30 to $1.29 in the past 30 days. The loss per share for 2023 has also narrowed down from 67 cents to 60 cents in the same time frame.

Earnings of Acadia beat estimates in two of the trailing four quarters, while missing the same on the remaining two occasions. The average negative earnings surprise for ACAD is 6.83%.

BioMarin’s earnings per share estimates for 2022 are down by a cent at $1.98 in the past 30 days. The same for 2023 has improved by a cent from $3.80 to $3.81 in the same time frame.

Earnings of BioMarin beat estimates in all of the trailing four quarters. The average earnings surprise for BMRN is 98.90%.

Want the latest recommendations from Zacks Investment Research? Today, you can download 7 Best Stocks for the Next 30 Days. Click to get this free report   BioMarin Pharmaceutical Inc. (BMRN) : Free Stock Analysis Report   Eli Lilly and Company (LLY) : Free Stock Analysis Report   ACADIA Pharmaceuticals Inc. (ACAD) : Free Stock Analysis Report   Akouos, Inc. (AKUS) : Free Stock Analysis Report   To read this article on Zacks.com click here.   Zacks Investment Research

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Lilly to acquire gene therapy company Akouos

18-Oct-2022 - Last updated on 18-Oct-2022 at 14:09 GMT

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Founded in 2016, Boston-headquartered Akouos has integrated expertise across otology, inner ear drug delivery, and gene therapy.

Its lead candidate AK-OTOF, a gene therapy for the treatment of hearing loss due to mutations in the otoferlin gene ( OTOF ​), received FDA clearance for its Investigational New Drug (IND) application in September to initiate a Phase 1/2 pediatric trial (In April 2021, the FDA granted both Orphan Drug Designation and Rare Pediatric Disease Designation for the therapy).

Lilly will pay $487m in cash with the potential for the deal’s value to rise to $610m upon certain clinical and regulatory milestones.

Building a gene therapy portfolio

Aside from AK-OTOF, additional pipeline programs span across multiple inner ear conditions, and include AK-CLRN1 for Usher Type 3A, an autosomal recessive disorder characterized by progressive loss of both hearing and vision;  GJB2 ​ (which encodes connexin 26) for a common form of monogenic deafness and hearing loss; and AK-antiVEGF for the treatment of vestibular schwannoma.

The deal has been approved by the boards of directors of both companies.

Lilly established its gene therapy program via its acquisition of New York’s Prevail Therapeutics in 2021 for $880m in cash, which saw the US pharma giant gain AAV9-based gene therapies for patients with neurodegenerative diseases.

"I am proud of the commitment and passion of our team, which has established Akouos as a pioneer in inner ear genetic medicine, as demonstrated by our work to advance the first investigational therapy for a genetic form of hearing loss into clinical development," ​said Emmanuel Simons, Ph.D., M.B.A., co-founder, president, and chief executive officer of Akouos.  "Joining Lilly – a company that shares our purpose to make life better for people around the world – will help us accelerate the development of a broad pipeline of inner ear genetic medicines."   ​

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