Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal
RNA-targeted genetic therapies for rare diseases, today provided
program updates for ELX-02 and ZKN-013, including Orphan Drug
Designation (ODD) for ELX-02.
Eloxx has significantly advanced the development of ELX-02 for
the treatment of Alport syndrome with Nonsense Mutations (NMAS).
Additionally, with the global licensing partnership with Almirall
announced in March, the company has begun the development of
ZKN-013 for the treatment of Recessive Dystrophic Epidermolysis
Bullosa (RDDEB) and Familial Adenomatous Polyposis (FAP) patients
with nonsense mutations. The publication of a recent paper on
Autosomal Dominant Polycystic Kidney Disease (ADPKD), confirms the
potential of ELX-02 for the treatment of all rare genetic kidney
diseases with nonsense mutations in the disease causing genes.
“The recent ELX-02 program updates, including Orphan Drug
Designation for ELX-02 for the treatment of Alport Syndrome
highlights the significant unmet medical needs of Alport Syndrome
patients with Nonsense Mutations,” said Sumit Aggarwal, President
and Chief Executive Officer of Eloxx. “The designation further
strengthens our belief that ELX-02 has the potential to be
transformational in supporting the ultra-rare subset of Alport
Syndrome patient population with nonsense mutations. We look
forward to engaging the FDA on initiating the larger clinical trial
to support the potential for regulatory marketing approval of
ELX-02 in this patient population.”
ELX-02: Regulatory and Clinical Updates for Alport
Syndrome Nonsense Mutation Program
- ELX-02 Granted ODD from U.S.
FDA: In April, the FDA Office of Orphan Products
Development (OOPD) granted ODD for ELX-02 for the treatment of
Alport Syndrome.
- The designation was based on a
review of the prevalence of NMAS and the data from the
Proof-of-concept Phase 2 Study that Eloxx announced top-line
results for in 2023.
- FDA ODD is granted to
investigational therapies addressing rare medical diseases or
conditions that affect fewer than 200,000 people in the United
States with a reasonable clinical or preclinical hypothesis for
efficacy in the target population.
- Requested a
Pre-Investigational New Drug (IND) meeting with U.S. FDA:
The request for a pre-IND meeting formally initiates communications
with the FDA regarding development of ELX-02 for the treatment of
NMAS.
- The purpose of the pre-IND meeting
will be to request feedback on the Eloxx’s planned clinical trial
in nonsense mutation Alport syndrome patients.
- Presented Clinical Data
Updates at Alport Workshop Meeting in March: Prof. Daniel
Gale presented updated results from the Proof-of Concept Study in 3
NMAS patients treated with ELX-02
- Meaningful reduction in Podocyte
Foot Process Effacement after treatment measured as an average 60%
increase in Filtration Slit Density in post treatment kidney
biopsies in all 3 patients.
- Results confirm the expression of
functional collagen 4 protein in-line with the mechanism of protein
induction of ELX-02.
- Changes in UPCR noted to be
consistent with biopsy results with reduction or stabilization of
proteinuria during or up to 2 months post completion of
dosing.
ZKN-013: Update on Global Licensing Partnership with
Almirall
In March 2024, Eloxx announced an exclusive license agreement
with Almirall to develop and commercialize ZKN-013 in orphan
indications including RDEB and FAP with nonsense mutations.
- Received $3 million in
upfront payment
- Additional payments of
ELX-02 of up to $470 million and tiered royalties on global
sales: Eloxx shall be eligible for additional payments
throughout the potential development phases, including regulatory
and sales milestones of up to $470 million as well as tiered
royalties on any potential global sales
- Initiated Phase 1 Single
Ascending Dose (SAD) study: Initiated Phase 1 SAD study in
Australia with initial results expected in 2nd half of 2024
Recently Published Paper on Autosomal Dominant
Polycystic Kidney Disease (ADPKD) may Have Other Rare Kidney
Disease Applications
A paper was published in April 2024, on ADPKD titled Genetics of
cystogenesis in base-edited human organoids reveal therapeutic
strategies for polycystic kidney disease, authored by Courtney E.
Vishy, Chardai Thomas, Thomas Vincent, Daniel K. Crawford, Matthew
M. Goddeeris, and Benjamin S. Freedman. Freedman and colleagues
engineered base-edited human iPS cells to resolve the functional
genetics of human PKD. They discovered that cyst formation in
diseased organoids could be prevented with a single healthy gene
copy. This insight enables the demonstration of both genetic and
pharmacological therapeutic approaches for clinically relevant
nonsense mutations and may have the potential to support other rare
kidney diseases.
About Nonsense Mutation Alport
Syndrome
Nonsense Mutation Alport syndrome (NMAS) is an ultra-rare Type
IV Collagenopathy characterized by nonsense mutations in the genes
(COL4A3, COL4A4, and COL4A5) that result in a less than full length
(truncated) Type 4 Collagen. This disorder mostly affects children
with a median age at diagnosis of 9 to 20 years. It is
characterized by rapid and progressive damage to the kidneys, ear,
and eyes, starting with worsening of kidney morphology to
proteinuria and finally kidney failure, hearing loss and eye
abnormalities. 90% NMAS patients progress to kidney failure and
hearing loss before the age of 30. There are no approved therapies.
It is estimated that there are approximately 11,000 NMAS patients
in the US and >20,000 patients in US, Europe, Japan and
China.
About Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is engaged in the
science of ribosome modulation, leveraging its innovative TURBO-ZM™
chemistry technology platform in an effort to develop novel
Ribosome Modulating Agents (RMAs) and its library of Eukaryotic
Ribosome Selective Glycosides (ERSGs). Eloxx’s lead investigational
product candidate, ELX-02, is a small molecule drug candidate
designed to restore production of full-length functional proteins.
ELX-02 is in Phase 2 clinical development for the treatment of
Alport syndrome in patients with nonsense mutations. For more
information, please visit www.eloxxpharma.com.
Forward-looking StatementsThis
press release contains forward-looking statements within the
meaning of the Private Securities Litigation Reform Act of 1995.
All statements other than statements of historical facts contained
in this press release, including without limitation, statements
regarding the potential future payments and future benefits under
the license agreement, achievement of key milestones under the
license agreement and the expected timeline for clinical
development and efficacy of ZKN-013 are forward-looking statements.
Forward-looking statements can be identified by the words “aim,”
“may,” “will,” “would,” “should,” “expect,” “explore,” “plan,”
“anticipate,” “could,” “intend,” “target,” “project,”
“contemplate,” “believe,” “estimate,” “predict,” “potential,”
“seeks,” or “continue” or the negative of these terms similar
expressions, although not all forward-looking statements contain
these words. Forward-looking statements are based on management's
current plans, estimates, assumptions and projections based on
information currently available to us. Forward-looking statements
are subject to known and unknown risks, uncertainties and
assumptions, and actual results or outcomes may differ materially
from those expressed or implied in the forward-looking statements
due to various important factors, including, but not limited to:
our ability, or our licensees’ ability, to progress any product
candidates in preclinical or clinical trials; the uncertainty of
clinical trial results and the fact that positive results from
preclinical studies are not always indicative of positive clinical
results; the scope, rate and progress of our, and our licensees’
preclinical studies and clinical trials and other research and
development activities; the competition for patient enrollment from
drug candidates in development;; our ability to obtain the capital
necessary to fund our operations; the cost of filing, prosecuting,
defending and enforcing any patent claims and other intellectual
property rights; our ability to obtain financing in the future
through product licensing, public or private equity or debt
financing or otherwise; our ability to meet the continued listing
requirements of the Nasdaq Capital Market; general business
conditions, regulatory environment, competition and market for our
products; and business ability and judgment of personnel, and the
availability of qualified personnel and other important factors
discussed under the caption “Risk Factors” in our Quarterly Report
on Form 10-Q for the quarterly period ended September 30, 2023, as
any such factors may be updated from time to time in our other
filings with the SEC, accessible on the SEC’s website at
www.sec.gov and the “Financials & Filings” page of our website
at https://investors.eloxxpharma.com/financials-filings.
All forward-looking statements speak only as of the date of this
press release and, except as required by applicable law, we have no
obligation to update or revise any forward-looking statements
contained herein, whether as a result of any new information,
future events, changed circumstances or otherwise.
Contact
InvestorsJohn Woolfordjohn.woolford@westwicke.com
443.213.0506
MediaLaureen Cassidylaureen@outcomescg.com
Source: Eloxx Pharmaceuticals
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