Eloxx presented findings from the Phase 1 single ascending dose (SAD) and multiple ascending dose (MAD) studies and additional preclinical data for ELX-02
Eloxx will provide an update on the Phase 2 programs for ELX-02 in cystic fibrosis and cystinosis and provide a business update on
Eloxx to host webcast and conference call on
“We are extremely pleased with the emerging profile of ELX-02 and believe that the consistency of our data with those from previous studies with already approved drugs for cystic fibrosis demonstrating that functional increases in CFTR protein across a growing list of CFTR genotypes were correlated with improvements in FEV1, and lung function derisk our Phase 2 studies,” said Dr.
“With the successful completion of our Phase 1 programs, we are pleased to have advanced ELX-02 into Phase 2 clinical trials, and we look forward to providing an update on the programs on
The data presented for ELX-02 at the
In a Poster presentation titled: “Pharmacokinetics, Safety, and Tolerability of Multiple Ascending Doses of ELX-02 in
- ELX-02 shows linear and dose proportional PK following subcutaneous administration twice weekly.
- There were consistent and dose proportional increases in Cmax, AUCt, and AUCinf across the dose range on Day 1 and Day 29, with no accumulation.
- Elimination is primarily renal as parent compound and is essentially complete within 24 hours post-dose.
- To date, ELX-02 has been generally well tolerated in clinical studies, with 105 volunteers exposed, no reported SAEs or renal findings.
- Collectively, these data support the future evaluation of ELX-02 in Phase 2 trials with nonsense mediated CF.
- Phase 2 trials will use the 50 mg/mL concentration with daily administration which achieves comparable exposure.
In a Poster presented by Dr.
- Single subcutaneously injected doses of ELX-02 between 0.3 mg/kg and 7.5 mg/kg showed an acceptable safety profile without severe or serious drug-related adverse events, including a lack of renal and ototoxicity events.
- ELX-02 shows rapid absorption, high bioavailability (98%) and linear and dose proportional PK following subcutaneous administration.
- Elimination is primarily renal as parent compound.
- To date, ELX-02 has been generally well tolerated in clinical studies, with 105 volunteers exposed, no reported SAEs or renal findings.
- The tolerability of the 7.5 mg/kg single dose supports the safety of the planned exposures and dose range being used in Phase 2.
- Collectively, these data support the future evaluation of ELX-02 in Phase 2 trials with nonsense mediated diseases.
In a Poster presentation titled: “Investigational Drug ELX-02 Mediates CFTR Nonsense Mutation Read-through to Increase CFTR MRNA, CFTR Protein Translation and CFTR Function”, which was also included in an
- Pronounced ELX-02 mediated CFTR read-through is demonstrated in FRT, transgenic mice, and patient-derived HBE cells and organoids. Significant and meaningful activity is observed against the top 5 most prevalent nonsense alleles, representing >75% of the CF nonsense population. We continue to identify new responsive genotypes.
- ELX-02 results in a pronounced increase in both CFTR protein expression and mRNA stability further supporting proposed mechanism of action.
- ELX-02 preclinical efficacy-associated exposures translate to the selected Phase 2 clinical trial ascending dose ranges and exposures.
ELX-02 is an investigational agent not approved by any regulatory agency for therapeutic use and is currently in Phase 2 clinical trials for cystic fibrosis and cystinosis.
Conference Call and Webcast Information:
Date:
Time:
International Dial-in Number: (210) 874-7715
Conference ID: 8339658
Live Webcast: accessible from the Company's website at www.eloxxpharma.com under Events and Presentations or with this link: https://edge.media-server.com/mmc/p/ni34pajh
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Forward-Looking Statements
This press release contains forward-looking statements, which are generally statements that are not historical facts. Forward-looking statements can be identified by the words "expects," "anticipates," "believes," "intends," "estimates," "plans," "will," "outlook" and similar expressions. Forward-looking statements are based on management's current plans, estimates, assumptions and projections, and speak only as of the date they are made. We undertake no obligation to update any forward-looking statement in light of new information or future events, except as otherwise required by law. Forward-looking statements involve inherent risks and uncertainties, most of which are difficult to predict and are generally beyond our control. Actual results or outcomes may differ materially from those implied by the forward-looking statements as a result of the impact of a number of factors, including: the development of the Company’s read-through technology; the approval of the Company’s patent applications; the Company’s ability to successfully defend its intellectual property or obtain necessary licenses at a cost acceptable to the Company, if at all; the successful implementation of the Company’s research and development programs and collaborations; the Company’s ability to obtain applicable regulatory approvals for its current and future product candidates; the acceptance by the market of the Company’s products should they receive regulatory approval; the timing and success of the Company’s preliminary studies, preclinical research, clinical trials, and related regulatory filings; the ability of the Company to consummate additional financings as needed; as well as those discussed in more detail in our Annual Report on Form 10-K and our other reports filed with the Securities and Exchange Commission.
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Contact:
barbarar@eloxxpharma.com
(203) 274-2825
SOURCE:
Source: Eloxx Pharmaceuticals