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Eloxx Pharmaceuticals Reports Second Quarter 2021 Financial and Operating Results and Provides Business Update Added Additional Treatment Arm and Provided Enrollment Update for Ongoing ELX-02 Phase 2 Clinical Trials for

Key Takeaway: Pharmaceuticals Reports Second Quarter 2021 Financial and Operating Results and Provides Business Update Added Additional Treatment Arm and Provided Enrollment Update for Ongoing ELX-02 Phase 2 Clinical Trials for Cystic Fibrosis (CF) Nominated ZKN-013 as Lead Candidate for Re

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Pharmaceuticals Reports Second Quarter 2021 Financial and Operating Results and Provides Business Update
Added Additional Treatment Arm and Provided
Enrollment Update for Ongoing ELX-02 Phase 2 Clinical Trials for Cystic Fibrosis (CF)
Nominated ZKN-013 as Lead Candidate for Recessive
Dystrophic Epidermolysis Bullosa (RDEB) and Junctional Epidermolysis Bullosa (JEB) Programs
Advanced Multiple Preclinical Rare Inherited
and Targeted Oncology Programs
Award from Cystic Fibrosis Foundation to Develop Ribosome Modulating Agents (RMAs) with TURBO-ZM Platform
Raised Approximately $52M in Gross Proceeds
Through Public Offering
WATERTOWN, MA - Aug 16, 2021 - 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 ended June 30, 2021, and provided a business update.
"Since the acquisition of Zikani Therapeutics in early April,
we have made tremendous progress across our portfolio of novel therapeutic programs," said Sumit Aggarwal, President and Chief Executive
Officer of Eloxx. "In CF, we remain on track to report data from the ongoing Phase 2 clinical trials for ELX-02 , while we are also
preparing for combination studies of ELX-02 and ivacaftor and commencing efforts to evaluate inhaled delivery of ELX-02. Beyond CF, we
continue to advance our preclinical programs to demonstrate the potential of our oral RMAs. We intend to provide additional insight into
these programs in the coming quarters."
Second Quarter 2021 and Subsequent Highlights
Class 1 Cystic Fibrosis
Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional
Epidermolysis Bullosa (JEB)
Rare Inherited and Targeted Oncology
Second Quarter 2021 Financial Results
For the three months ended June 30, 2021, we incurred a net loss of
$36.1 million or $0.54 per share, which includes $4.0 million in stock-based compensation. For the same period in the prior year, we incurred
a net loss of $7.9 million, or $0.20 per share. Results for the second quarter of 2021 included a $22.7 million acquired in-process research
and development expense related to the acquisition of Zikani.
Our research and development expenses (R&D) were $5.7 million for
the three months ended June 30, 2021, which includes $0.1 million in stock-based compensation. For the same period in the prior year,
R&D expenses were $3.7 million. The increase in R&D expenses was primarily related to an increase in expenses related to the continued
development of ELX-02 as a result of the suspension of our clinical trials due to the impact of the COVID-19 pandemic in the prior year
period and an increase in salaries and other personnel related costs, partially offset by a decrease in stock-based compensation expense.
Our general and administrative (G&A) expenses were $7.4 million
for the three months ended June 30, 2021, which includes $3.9 million in stock-based compensation. For the same period in the prior year,
G&A expenses were $3.8 million. The increase was primarily related to an increase in stock-based compensation expense, an increase
in salaries and other personnel-related costs associated with the merger with Zikani, as well as an increase in expenses attributable
principally to infrastructure related costs including legal, accounting and other professional fees.
As of June 30, 2021, we had cash and cash equivalents of $56.7 million,
which we expect will be sufficient to fund our operations into the first quarter of 2023.
1 J Mol Med (Berl) 2016 Apr;94(4):469-82) and Int J Cancer
2020 Feb 15;146(4):1064-1074)
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 Ribsome 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. 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
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 expected cash burn and 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.
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 Quarterly Report on Form 10-Q for the quarter ended June 30, 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 & Filings" page of our website at https://investors.eloxxpharma.com/financial-information/sec-filings
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
ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES
UNAUDITED CONDENSED CONSOLIDATED BALANCE SHEETS
(Amounts in thousands, except share and per
June 30, 2021 December 31, 2020
ASSETS
Current assets:
Cash and cash equivalents $ 56,734 $ 24,668
Restricted cash 246 56
Prepaid expenses and other current assets 1,579 1,169
Total current assets 58,559 25,893
Property and equipment, net 185 133
Operating lease right-of-use asset 1,866 421
Other long-term assets - 30
Total assets $ 60,610 $ 26,477
LIABILITIES AND STOCKHOLDERS' EQUITY
Current liabilities:
Accounts payable $ 2,024 $ 481
Accrued expenses 3,302 2,886
Current portion of long-term debt 5,686 5,239
Advances from collaboration partners 3,411 805
Current portion of operating lease liability 753 389
Taxes payable 34 38
Total current liabilities 15,210 9,838
Long-term debt 3,637 6,376
Operating lease liability 1,120 33
Total liabilities 19,967 16,247
Total stockholders' equity 40,643 10,230
Total liabilities and stockholders' equity $ 60,610 $ 26,477
ELOXX PHARMACEUTICALS, INC. AND SUBSIDIARIES
UNAUDITED CONSOLIDATED STATEMENTS OF OPERATIONS
(Amounts in thousands, except share and per
Three Months Ended June 30, Six Months Ended June 30,
2021 2020 2021 2020
Operating expenses:
Research and development $ 5,704 $ 3,738 $ 9,777 $ 8,505
General and administrative 7,355 3,848 11,696 8,854
In process research and development 22,670 - 22,670 -
Restructuring charges - - - 3,994
Total operating expenses 35,729 7,586 44,143 21,353
Loss from operations (35,729 ) (7,586 ) (44,143 ) (21,353 )
Other expense, net 329 301 609 480
Net loss $ (36,058 ) $ (7,887 ) $ (44,752 ) $ (21,833 )
Basic and diluted net loss per share $ (0.54 ) $ (0.20 ) $ (0.84 ) $ (0.54 )
Weighted average number of common shares used in computing net loss per share, basic and diluted 66,389,865 40,129,304 53,357,401 40,101,789
Last updated: Aug 16, 2021