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Eloxx Pharmaceuticals Reports Fourth Quarter 2021 Financial and Operating Results and Provides Business Update

Key Takeaway: Eloxx Pharmaceuticals Reports Fourth Quarter 2021 Financial and Operating Results and Provides Business Update Expect topline data from cystic fibrosis (CF) Phase 2 expansion treatment arms evaluating combination with ivacaftor by the end of the first half of 2022 Cystic Fibr

Full Press Release Details

Eloxx Pharmaceuticals Reports Fourth
Quarter 2021 Financial and Operating Results and Provides Business Update
Expect topline data from cystic
fibrosis (CF) Phase 2 expansion treatment arms evaluating combination with ivacaftor by the end of the first half of 2022
Cystic Fibrosis Foundation (CF
Foundation) to provide an award of up to $15.9 million for the ongoing ELX-02 clinical program
New development program for ELX-02
for the treatment of Alport syndrome, a rare kidney genetic disorder; initiation of proof-of-concept clinical trial expected in second
On track to start First in Human
Phase 1 study in 2022 with ZKN-013 that is being developed for the treatment recessive dystrophic epidermolysis bullosa (RDEB) patients
with nonsense mutations cells
Treatment study of ZKN-013 in
APCMin mouse model of familial adenomatous polyposis (FAP) demonstrates significant reductions of
40% in polyp count and 50% in polyp burden leading to improved survival
WATERTOWN, Mass., March 31, 2022 (GLOBE NEWSWIRE) -- Eloxx
Pharmaceuticals, Inc. (NASDAQ: ELOX), a leader in ribosomal RNA-targeted genetic therapies for rare diseases, today reported its financial
results for the three months and year ended December 31, 2021 and provided a business update.
"The recently announced
funding from the CF Foundation, together with positive topline clinical results from the monotherapy arm of Phase 2 of the ELX-02 clinical
trial announced in November 2021, has increased our confidence in the potential of ELOX-02 to transform the lives of Class 1 CF patients
with nonsense mutations," said Sumit Aggarwal, President and Chief Executive Officer of Eloxx. "In addition to the ELOX-02
CF program, we believe we continue to make significant progress across our portfolio of product candidates. With the addition of a new
program in Alport syndrome, a rare kidney disease, we believe we are beginning to fully capture the potential of ELX-02 as a novel readthrough
agent while at the same time, our oral RMAs continue to advance forward with encouraging preclinical data in RDEB and FAP that demonstrate
the potential of ribosome modulation."
Fourth Quarter 2021 and Subsequent Highlights
Class 1 Cystic Fibrosis
Recessive Dystrophic Epidermolysis Bullosa and Junctional
Epidermolysis Bullosa (JEB)
Familial Adenomatous Polyposis and Targeted Oncology
Fourth Quarter 2021 Financial Results
For the three months ended December 31, 2021, we incurred a
net loss of $12.1 million, or $0.14 per share, which includes $1.6 million in stock-based compensation. For the same period in the prior
year, we incurred a net loss of $6.1 million, or $0.15 per share. Since the closing date of Eloxx's acquisition of Zikani Therapeutics,
Inc. in April 2021, the results of Zikani's operations have been included in the Eloxx' condensed consolidated financial statements.
Our research and development expenses (R&D) were
$7.9 million for the three months ended December 31, 2021, which includes $0.6 million in stock-based compensation. For the same period
in the prior year, R&D expenses were $2.6 million. The increase was primarily related to expenses related to the continued development
of ELX-02 due to the impact of the COVID-19 pandemic in the prior year period, an increase in salaries and other personnel related costs,
and an increase in operational facilities.
Our general and administrative (G&A) expenses were
$3.7 million for the three months ended December 31, 2021, which includes $1.0 million in stock-based compensation. For the same period
in the prior year, G&A expenses were $3.1 million. The increase was primarily related to an increase in stock-based compensation expense,
an increase in salaries and other personnel related costs, as well as an increase in legal, accounting and other professional fees.
As of December 31, 2021, we had cash and cash equivalents
of $42.3 million, which combined with $7.0 million from the CF Foundation, we expect will be sufficient to fund our operations into the
second quarter of 2023.
About Nonsense Mutations
Nonsense mutations cause a premature stop codon in the mRNA
resulting in less than full length or loss of function proteins. These remain highly underserved with no approved disease modifying therapies.
An estimated 10-12% patients across over 8,000 inherited genetic rare diseases harbor nonsense mutations in one or both alleles harboring
About Eloxx Pharmaceuticals
Eloxx Pharmaceuticals, Inc. is engaged in the science
of ribosome modulation, leveraging its innovative TURBO-ZMTM 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 clinical development, focusing on cystic fibrosis (US Trial NCT04135495, EU/IL Trial NCT04126473). Eloxx also has
preclinical programs focused on select rare diseases, including inherited diseases, cancer caused by nonsense mutations, kidney diseases,
including autosomal dominant polycystic kidney disease, as well as rare ocular genetic disorders.
For more information, please visit www.eloxxpharma.com.
Forward-looking Statements
This press release contains forward-looking
statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements other than statements of present
and historical facts contained in this press release, including without limitation, statements regarding our future financial results,
the expected timing of trials and results from clinical studies of our product candidates and the potential of our product candidate to
treat nonsense mutations 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 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 preclinical studies
and clinical trials and other research and development activities; the competition for patient enrollment from drug candidates in development;
the impact of the global COVID-19 pandemic on our clinical trials, operations, vendors, suppliers, and employees; 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 financial in the future through product licensing, public or private equity or debt financing
or otherwise; 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 Annual Report on Form 10-K for the fiscal year ended December, 2021, 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 &
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.
Investors John Woolford
Laureen Cassidy laureen@outcomescg.com
ELOXX PHARMACEUTICALS, INC.
AND SUBSIDIARIES UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
(Amounts in thousands, except
share and per share data)
December 31, 2021 December 31, 2020
ASSETS
Current assets:
Cash and cash equivalents $ 42,268 $ 24,668
Restricted cash 299 56
Prepaid expenses and other current assets 913 1,169
Total current assets 43,480 25,893
Property and equipment, net 216 133
Operating lease right-of-use assets 1,443 421
Other long-term assets - 30
Total assets $ 45,139 $ 26,477
LIABILITIES AND STOCKHOLDERS' EQUITY Current liabilities: Accounts payable $ 1,379 $ 481
Accrued expenses 4,196 2,924
Current portion of long-term debt - 5,239
Advances from collaboration partners 3,723 805
Current portion of operating lease liabilities 657 389
Total current liabilities 9,955 9,838
Long-term debt 11,996 6,376
Operating lease liabilities 804 33
Total liabilities 22,755 16,247
Total stockholders' equity 22,384 10,230
Total liabilities and stockholders' equity $ 45,139 $ 26,477
ELOXX PHARMACEUTICALS, INC.
AND SUBSIDIARIES UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS
(Amounts in thousands, except
Last updated: Mar 31, 2022