Combination of subcutaneous ELX-02 with ivacaftor did not achieve statistical significance for efficacy endpoints in Phase 2 study in Class 1 CF
ELX-02 was well tolerated with no drug-related serious adverse events observed
Evidence of activity for ELX-02 observed; efficacy signal potentially confounded by variability due to low drug exposure
Path forward for ELX-02 for the treatment of Class 1 CF to be determined together with
Company to host conference call and webcast today,
“We are disappointed that ELX-02 failed to achieve statistical significance for its key efficacy endpoints in this Phase 2 trial in combination with ivacaftor for the treatment of Class 1 CF. Despite this setback, we were pleased to observe that ELX-02 was well tolerated and demonstrated additional evidence of activity in this underserved patient population. We will work closely with the
Topline Results of ELX-02 Phase 2 Combination Trial in Class 1 CF Patients
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). Topline results are summarized below:
- 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.
- Eloxx 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 expected to be at least 50-fold greater than with subcutaneous delivery.
- Lung drug exposure with inhaled delivery of ELX-02 expected to be at least 50-fold greater than with subcutaneous delivery.
About Class 1 CF
CF patients with a Class 1 nonsense mutation remain highly underserved with no approved disease modifying therapies. An estimated 10-12% of CF patients are Class 1 patients with one or both alleles harboring nonsense mutations, leading to less than full length CFTR proteins on the cell membrane in these patients.
Conference Call and Webcast
Eloxx’s management will host a conference call and webcast today at
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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, the expected timing of trials 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 quarter ended
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.
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Source:
Source: Eloxx Pharmaceuticals
