About our project

The problem we solve: There are currently 466M people worldwide with disabling hearing loss and there are no approved therapies.

About our solution: RHI is developing gene therapy platform to treat persons with hearing loss

Progress to date:

RHI is generating positive proof of concept data on its lead gene therapy product (RHI100). RHI 100 qualifies as a rare disease and will pursue an "orphan status" regulatory path.  RHI a privately held, pre-clinical, "C" Corp and has established a patent estate around its first 3 genetic targets.  CEO's son (15 yrs old) has been diagnosed with permanent hearing loss due to a genetic condition.

About Our Company

How We Help Patients

In 2005, Xavier was born. As with most newborns, he received a newborn hearing screening test which he failed. Xavy's parents were devastated but moved forward as best they could. What followed over the next 4 years were chronic ear infections, multiple medications and numerous trips to hearing specialists. In 2010 at 5 years of age, Xavy was diagnosed with Bi Lateral Sensorineural hearing loss (SNHL) non syndromic. He was fitted for hearing aids which he would wear for the rest of his life, as there are no approved therapies for persons with hearing loss. This fact did not sit well with Xavy’s parents.

Xavy’s father conducted interviews with over 25 Key Opinion Leaders in the hearing loss field, profiled over 21 companies and attended his first scientific conference for hearing loss research called ARO (Association for Research in Otolaryngology). As a result of his research and with the support of the scientific community, a company was created.  The company is called Rescue Hearing Inc. My name is Jim Ayala the CEO and Founder of Rescue Hearing Inc and Xavier is my son.

How We Help Physicians

There are currently no therapies for hearing loss.  Patients diagnosed with hearing loss have only 2 options, hearing aids or cochlear implants.  Rescue Hearing Inc (RHI) is a company developed by scientists and the father of a child born with congenital genetic hearing loss. There are over 100 genes that when mutated can cause hearing loss and RHI is developing a gene therapy platform that will target some of these genes.  RHI's initial gene therapy product (RHI100) targets a genetic mutation that causes progressive hearing loss.  Patients are born with normal hearing then begin losing their hearing in their late teens and early 20s which then progresses to deafness.  These patients perform poorly even when fitted with hearing aids or cochlear implants because the underlying genetic mutation affects both the cochlear hair cells and the undelying nerve bundle (spiral ganglion).  RHI100 will deliver functioning genes via direct injection into the cochlea using a viral vector serotype that has an established safety profile with regulatory agencies.  RHI has filed patents on 3 genetic targets.  

How We Help Hospitals

RHI's gene therapies must be delivered in a hospital setting using a surgical procedure common to otolyrngologists.  Patient pre and post surgical procedures can be done via the Audiology Department.

How We Help Partners

The promise of gene therapy is a one time treatment that will replace a lifetime of expenses and hardship associated with hearing aids and cochlear implants.  The 2018 WHO study on the rise of hearing loss globally states that "unaddressed hearing loss poses an annual global cost of $750 billion (USD)".  Private and public health insurance companies, hospitals, research institutions, healthcare providers and patients all stand to benefit from the development of gene therapies like RHI100.  

Innovation Details

Intellectual Property Summary

RHI PATENT ESTATE BUILT AROUND 3 GENETIC TARGETS:

• TARGET 1&2- PCT WORLDWIDE FILING (MAR 2018), EUROPE, CHINA, JAPAN, HONG KONG, ISRAEL, CANADA, KOREA, BRAZIL
• TARGET 1&2- CIP FILINGS MADE IN DEC 2019 (POC DATA SET)
• TARGET 1&2- COMBO THERAPY PATENT FILED DEC 2019 (DEVICE + GENE THERAPY)
• TARGET 3 – PROVISIONAL FILED MAR 2020

NATURE OF CLAIMS ARE COMPOSITION OF MATTER AND METHODS AND USE.

Clinical Information

Initial gene therapy product (RHI100) will treat adult patients experiencing progressive hearing loss which leads to deafness. These patients seek cochlear implants to address their hearing loss but perform poorly due to their underlying genetic condition.  RHI100 will introduce functioning gene via direct injection to inner ear which will replace malfunctioning gene that is causing hearing loss. Current Development Stage- Proof of Concept- RHI100 positive POC data has demonstrated the following:

• Vector capsid delivers therapy to the correct cells in the inner ear
• RHI100 rescued the hearing of humanized disease model mice (invivo)
• RHI100 rescued humanized, mutant ips cells (invitro)
• The data set will be published in qrt 4 (2020)

Regulatory Status

RHI100 REGULATORY PATH AND TIMELINE- RHI is currently preparing for its Pre-IND meeting with FDA and PEI (Germany).  Timeline:

• Qrt 4 2020- conduct pre-IND meeting w FDA / EMA and apply for orphan / fast track designation
• Qrt 1 2021- Begin Pre-Clinical safety / tox animal studies
• Qrt 1 2022- begin human clinical trial

How we will use the funds raised

Specific use of proceeds in order:

• COMPLETE POC STUDIES AND PUBLISH DATA
• PAY REGULATORY CONSULTANT
  • CONDUCT PRE-IND MEETING W FDA / EMA
  • APPLY FOR ORPHAN / FAST TRACK REGULATORY STATUS
• HIRE CMO TO MANUFACTURE DRUG PRODUCT FOR PRE-CLINICAL AND CLINICAL STUDIES
• HIRE CRO TO CONDUCT PRE-IND ENABLING ANIMAL STUDIES (SAFETY / TOX)
• HIRE CRO TO CONDUCT HUMAN CLINICAL TRIAL
• SG&A OPERATING EXPENSES

Thank You

My name is Jim Ayala, CEO and Founder of Rescue Hearing Inc (RHI).  I have 27 yrs of healthcare industry experience and RHI is my 4 life science stratup.  My son (now 15 yrs old) was born with permanent hearing loss due to a genetic condition.  Because there are no approved therapies for hearing loss, he has only 2 options, hearing aids or cochlear implants.  RHI was founded to provide therapeutic options for persons (like my son) with debilitating hearing loss.  Our scientific team consists of the leading experts in the development of gene therapies for hearing loss that includes Dr. Hinrich Staecker, the inventor and PI of the first and only clinical trial of a gene therapy for hearing loss.  I invite those with passion and courage to join RHI in developing therapies for hearing loss.    

Investor Info

Market Size

According to the 2018 WHO study on hearing loss and deafness, 466 million people worldwide live with debilitating hearing loss (900M in 2050). Our initial gene therapy product RHI100 will target a segment of this market, specifically cochlear implant recepients.  RHI100 will also qualify as an orphan designation.  RHI has completed a study on the estimated incidence and prevalence of the RHI100 genetic target and incorporated this data into RHI100 revenue projections.  Both of the studies are available to qualified investors under CDA

Projected 3 Year Growth

We intend to achieve 2% market penetration in year 3 post launch with estimated gross revenue of $158M in worldwide sales.  A full study on the estimated incidence and prevalence of the RHI100 genetic target with revenue projections is available to qualified investors under CDA.

Revenue Model

Reimbursement:  Product reimbursement will come from patients (private pay and health insurance) and cochlear implant manufacturers (combo therapy=cochlear implant + gene therapy).  Revenue can also be generated via out-licensing of technology platform.  Marketing: intention is to partner during clinical development with pharma/biotech company to provide sales and distribution.  Exit events can occur via acquisition or partnering and could occur within 3 years.   

Competitors

The number of hearing loss companies is growing due to lack of approved therapies.  The “big 3” hearing loss companies all created by institutional investors and VC consortiums (which validates the market) are:

• Decibel
• Frequency therapeutics
• Akouos (recent IPO @ $100M)

Traction

Customers are private pay and insured patients with disabling hearing loss.  Product will be delivered by physicians and hospitals.  Cochlear implant manufacturers (e.g. Medel) have also expressed an interest in our products where which could be delivered as a "combo therapy" (cochlear implant + gene therapy).  Since there are no approved therapies for hearing loss there is an unmet need validated by the number of institutional investors funding or creating hearing loss companies.

Due Diligence Docs

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