Eloxx Pharmaceuticals, Inc. (OTC: ELOX), a leader in ribosomal
RNA-targeted genetic therapies for rare diseases with nonsense
mutations, today provided significant key corporate updates.
“The demonstration of both collagen alpha 4 and collagen alpha 5
in patients in our proof-of-concept study of ELX-02 in NMAS
patients is an important milestone in the development of this
crucial potential therapy. These new results, presented in a
late-breaking presentation at ASN Kidney Week, strongly justify the
need to conduct a larger trial to confirm these encouraging results
in this underserved patient population,” said Sumit Aggarwal,
President and Chief Executive Officer of Eloxx. “Also, the FDA
allowing for continued dosing of subjects in the Phase 1 trial for
ZKN-013 is a substantial achievement for the program and makes
Eloxx eligible for the next milestone payment in the company’s
agreement with Almirall, once confirmed by our partner,
Almirall.”
Additional Alport Syndrome data presented at the ASN
Kidney Week
In late October, Daniel Gale, Professor of Nephrology at
University College London Department of Renal Medicine, presented
additional results from Eloxx’s proof-of-concept study in three
NMAS patients treated with ELX-02 in a late-breaking presentation
at ASN Kidney Week. The presentation was titled: “Small molecule
premature termination codon readthrough therapy: a Phase 2
pediatric and adult trial in Nonsense Mutation Alport syndrome” at
the ASN Kidney Week conference.
“Although this was a small trial of limited duration, the
appearance of new Type IV Collagen in the glomerular basement
membranes is the first time a missing podocyte protein has been
reconstituted in a human and provides evidence that ELX-02 is
acting as intended in the kidneys of patients. A longer, controlled
study will be needed to quantify the clinical effectiveness of this
therapy,” said Professor Gale.
New protein immunostaining and mass spectrometry analyses that
were presented, confirmed the production of both collagen a4 (IV)
and collagen a5 (IV) proteins in NMAS patients treated with ELX-02.
Prior to treatment, collagen a4 (IV) and a5 (IV) were absent in the
glomerulus in all three patients. Post treatment biopsy of the
patient with the largest improvement in Filtration Slit Density
showed a 34% increase in collagen a4 (IV) immunostaining intensity
and presence of collagen a3/a4/a5 (IV) peptide with a greater than
6-fold increase in collagen a4 and detectable collagen a5
consistent with formation of a functional protein induction. No
collagen alpha 5 was detected pre-treatment in this patient.
These additional data support ELX-02’s mechanism of protein
induction, and the observed morphology change and reduced foot
process effacement in all the patients that was previously
reported. The results also justify the conduct of a larger clinical
trial with longer ELX-02 treatment duration to validate the
observed clinical benefit in NMAS patients.
The demonstrated readthrough of premature stop codons also
suggests ELX-02 has potential in other rare kidney diseases,
including autosomal dominant polycystic kidney disease and
cystinosis.
FDA allows for continued dosing of subjects in Phase 1
trial of ZKN-013
Following successful dosing of the initial subjects, FDA has
allowed continued dosing of healthy volunteers for ZKN-013 for the
treatment of recessive dystrophic epidermolysis bullosa and
junctional epidermolysis bullosa. Eloxx is awaiting confirmation
from Almirall to proceed with the study based on their review of
the results and FDA feedback.
ZKN-013 has been exclusively licensed to Almirall, S.A.
(BME:ALM), who has global rights to develop and commercialize
ZKN-013. Under the terms of the license agreement, Eloxx is
eligible for additional development, regulatory and sales
milestones of up to $470 million as well as tiered royalties on any
potential global sales.
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, the expected timeline for clinical development,
the efficacy of the Company’s product candidates ZKN-013 and
ELX-02, the Company’s expected and planned communications with and
submissions to the FDA and anticipated funding under the bridge
loan 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: the Company’s incurrence of
significant losses; the Company has identified conditions and
events that raise substantial doubt about its ability to continue
as a going concern; its ability, or its 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 the
Company’s, and its licensees’ preclinical studies and clinical
trials and other research and development activities; the
competition for patient enrollment from drug candidates in
development; the Company’s ability to obtain the capital necessary
to fund its operations; the cost of filing, prosecuting, defending
and enforcing any patent claims and other intellectual property
rights; the Company’s ability to obtain financing in the future
through product licensing, public or private equity or debt
financing or otherwise; general business conditions, regulatory
environment, competition and market for the Company’s 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 the Company’s 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 the Company’s other
filings with the SEC, accessible on the SEC’s website at
www.sec.gov and the “Financials & Filings” page of the
Company’s 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, the
Company has 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.
ContactJohn Woolfordjohn.woolford@westwicke.com
443.213.0506
Source: Eloxx Pharmaceuticals
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