Lead molecule targets genetic nonsense mutation in rare diseases
Dose escalating study to enroll 36 subjects
Data expected in the fourth quarter of 2018
SAN DIEGO and REHOVOT, Israel and BOSTON, Nov. 27, 2017 (GLOBE NEWSWIRE) -- Sevion Therapeutics, Inc. (OTCQB:SVON) and Eloxx Pharmaceuticals Ltd., a clinical-stage biopharmaceutical company developing novel translational read-through inducing drugs (TRIDs) for genetic diseases caused by nonsense mutations, announced today dosing of the first subject in its multiple ascending dose (MAD) Phase 1b clinical trial with its lead candidate, ELX-02. The double-blind, placebo-controlled, dose-escalating trial will enroll 36 subjects across 4 dose levels, and is designed to evaluate the safety, tolerability and pharmacokinetics of ELX-02 in healthy volunteers (see clinicaltrials.gov, NCT03309605). The trial is being run in Belgium, and data are expected at the end of the fourth quarter 2018.
“We are very pleased to continue our clinical development program for ELX-02 and are excited about its potential to treat many genetic diseases caused by nonsense mutations,” said Pedro Huertas, M.D., Ph.D., Chief Medical Officer of Eloxx Pharmaceuticals. “We look forward to continue achieving our key clinical milestones.”
ELX-02 is a fifth-generation designer aminoglycoside optimized by medicinal chemistry approaches to act as a translational read-through inducing drug (TRID), enabling the synthesis of full-length proteins. ELX-02 has the potential to treat many rare genetic diseases with high prevalence of nonsense mutations such as cystic fibrosis, cystinosis, mucopolysaccharidosis Type I (MPS I), Rett Syndrome, and Duchenne muscular dystrophy (DMD).
About Eloxx Pharmaceuticals
Eloxx Pharmaceuticals is a clinical-stage biopharmaceutical company developing novel small molecule medicines to treat many rare and ultra-rare genetic diseases caused by nonsense mutations. Nonsense mutations are a class of gene defect that result in premature termination of protein synthesis. As a consequence, patients with a genetic disease caused by nonsense mutations have absent or truncated nonfunctional proteins, accounting for some of the most severe phenotypes in these genetic diseases. Eloxx’s lead product candidate, ELX-02, is an optimized aminoglycoside designed to restore full-length functional proteins. Eloxx Pharmaceuticals was founded in 2013 and maintains offices in Boston, MA, and Rehovot, Israel.
Certain statements included in this press release are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Actual results could differ materially from such statements expressed or implied herein as a result of a variety of factors, including, but not limited to: the Company’s ability to continue as a going concern; the ability of the Company to consummate additional financings; the development of the Company’s antibody technology; the approval of the Company’s patent applications; the Company’s ability to successfully defend its intellectual property or obtain the 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 success of the Company’s license agreements; the acceptance by the market of the Company’s products; the timing and success of the Company’s preliminary studies, preclinical research and clinical trials; competition and the timing of projects and trends in future operating performance; and the quotation of the Company’s common stock on an over-the-counter securities market, as well as other factors expressed from time to time in the Company’s periodic filings with the Securities and Exchange Commission (the “SEC”). As a result, this press release should be read in conjunction with the Company’s periodic filings with the SEC. The forward-looking statements contained herein are made only as of the date of this press release, and the Company undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstances.
Greg Weaver, CFO
Dan Ferry, Managing Director