We are providing the following business update.
Recent Developments
On September 14, 2022, the Company announced topline
results from the Phase 2 clinical trial of ELX-02 in combination with ivacaftor in Class 1 CF patients with at least one nonsense mutation.
The combination trial of ELX-02 with ivacaftor was well tolerated but did not achieve statistical significance for efficacy endpoints,
including changes from baseline in sweat chloride concentration (SCC) and percent forced expiratory volume (FEV1).
The Phase 2 combination clinical trial of ELX-02
was designed to evaluate safety and assess biological activity in G542X nonsense mutation Class 1 CF patients as monotherapy and in combination
with ivacaftor. The trial included a 1-week monotherapy period (1.5 mg/kg daily subcutaneous) followed by a four week combination period
(1.5 mg/kg daily subcutaneous and 150 mg ivacaftor twice daily).
ELX-02 was generally well tolerated in the trial,
with no treatment-related serious adverse events noted. Overall, the study did not achieve statistical significance for efficacy endpoints
in the Phase 2 study in Class 1 CF for efficacy endpoints, including changes from baseline in SCC and FEV1. No incremental improvement
was observed with ivacaftor combination. Evidence of activity for ELX-02 was observed, as patients with higher baseline sweat chloride
levels demonstrated increased responses as indicated by SCC (p=0.00013 at Day 35). Trial results were potentially confounded by high variability
in sweat chloride and lung function measurement. The Company believes this variability could have been caused by very low drug exposures
in the lung. Steady state lung drug levels in patients from this trial were on average 20%, or 2µM, of the lowest levels at which
drug activity has previously been seen in preclinical testing. Lung drug exposure with inhaled delivery of ELX-02 is expected to be at
least 50-fold greater than with subcutaneous delivery.
Forward-looking Statements
This Current Report on Form 8-K 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 current report, including without limitation, statements regarding 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 Quarterly Report on Form 10-Q for the quarterly period ended June 30, 2022, 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.
All forward-looking statements speak only as of
the date of this Current Report on Form 8-K 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.