Eloxx Pharmaceuticals Announces that Two Abstracts for ELX-02 Have Been Accepted for Presentation at the 41st European Cystic Fibrosis Conference

ELX-02 is an investigational, novel translational read-through inducing drug designed to restore synthesis of full-length functional proteins

Late Breaker evaluation of ELX-02 in cystic fibrosis patient organoids with nonsense mutations

Eloxx expects to initiate a Phase 2 clinical trial for ELX-02 in cystic fibrosis by the end of 2018, subject to regulatory review and clearance of our CTA

WALTHAM, Mass., April 18, 2018 -- Eloxx Pharmaceuticals, Inc. (“Eloxx”), (ELX:OTCQ), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis, cystinosis and other diseases caused by nonsense mutations limiting production of functional proteins, today announced that two abstracts, including one Later Breaker, for its lead candidate, ELX-02 will be presented at the 41^st European Cystic Fibrosis Conference taking place on June 6-9, 2018 in Belgrade, Serbia.

“We are pleased to have this opportunity to present new data for ELX-02 in organoids from cystic fibrosis patients with nonsense mutations to the scientific community. These patients have a high burden of disease, and few, if any, treatment options available,” said Dr. Pedro Huertas, Chief Medical Officer of Eloxx Pharmaceuticals. “We plan to initiate a Phase 2 clinical trial in cystic fibrosis patients with nonsense mutations by the end of this year, subject to regulatory clearance of our CTA in Belgium. We are committed to bringing forward therapeutic options which have the potential to transform lives.’”

The details for the two oral ELX-02 presentations are:

Oral Presentation Title: “Translational read-through of CFTR nonsense mutations and inducement of cystic fibrosis transmembrane conductance regulator (CFTR) function by ELX-02”
Presenter: Neal Sharpe, Ph.D., Senior Director, Preclinical Safety, R & D, Eloxx Pharmaceuticals
Time: Thursday, June 7, 2018 from 3:30 pm - 3:45 pm CET, or 9:30 am – 9:45 am ET

Oral Late Breaker Presentation Title: “Evaluation of ELX-02 in Cystic Fibrosis Organoids with Non-Sense Mutations”
Presenter: Dr. Pedro Huertas, Chief Medical Officer, Eloxx Pharmaceuticals
Time: Friday, June 8, 2018 from 5:00 pm - 5:30 pm CET, or 11:00 am - 11:30 pm ET

Listen to a Discussion with the HUB

We hosted a call with Dr. Robert Vries, Managing Director and one of the original scientific founders of Hubrecht Organoids Technology (The HUB).

The HUB is a not-for-profit organization founded by the Hubrecht Institute, KNAW and University Medical Center Utrecht, The Netherlands. 

The HUB is founded on the pioneering work of Prof. Dr. Hans Clevers who discovered methods to grow stem cell-derived human epithelial ‘mini-organs’ (organoids) from tissues of patients with various diseases including cancer and cystic fibrosis. 

In cell culture, organoids stably maintain the genotype/phenotype of the patient’s diseased tissue, thereby representing an in vitro platform for preclinical drug discovery and validation and a tool for precision medicine.

Organoids have become the standard to evaluate therapies for cystic fibrosis patients and the HUB has been testing all of the compounds currently being marketed and in development in cystic fibrosis patient organoids. Published results in the organoids have demonstrated a high correlation with clinical outcomes and specifically FEV1, the primary regulatory endpoint for establishing efficacy in CF.

We invite you to listen to the call to learn more about the organoids:

• How are the organoids being used in CF?
• What is the view from regulators and how they are using the organoid data?
• How are payors using and adopting the organoids?

The dial-in numbers for the replay of the call are: 

U.S. Dial-In Number: (855) 859-2056
International Dial-In Number: (404) 537-3406
Eloxx Pharmaceuticals Conference Call
ID # 9591818

About Eloxx Pharmaceuticals

Eloxx Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing novel RNA-modulating drug candidates that are designed to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA. As a consequence, patients with premature stop codon diseases have reduced or eliminated protein production from the mutation bearing allele accounting for some of the most severe phenotypes in these genetic diseases. These premature stop codons have been identified in over 1,800 rare and ultra-rare diseases. Read-through therapeutic development is focused on extending mRNA half-life and increasing protein synthesis by enabling the cytoplasmic ribosome to read through premature stop codons to produce full-length proteins. Eloxx’s lead product candidate, ELX-02, is a small molecule drug candidate designed to restore production of full-length functional proteins. ELX-02 is in the early stages of clinical development focusing on cystic fibrosis and cystinosis. ELX-02 is an investigational drug that has not been approved by any global regulatory body. Eloxx is headquartered in Waltham, MA, with R&D operations in Rehovot, Israel.

Forward-Looking Statements

Certain statements included in this press release are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and involve a number of risks and uncertainties. These include statements of management’s intentions, beliefs, plans and future expectations and, therefore, you are cautioned not to place undue reliance on them. Such forward-looking statements involve risks and uncertainties and actual results could differ materially from any forward-looking statements expressed or implied herein. The risks and uncertainties that could result in actual results to differ materially from these forward-looking statements expressed or implied herein include, but are 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, clinical trials; and related regulatory filings:; and the continued quotation of the Company’s common stock on the 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 10-K, 10-Qs and other 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 or revise such forward-looking statements to reflect subsequent events or circumstances.

Contact:

Barbara Ryan
[email protected] 
(203) 274-2825