Member Spotlight: Q&A with Eloxx Pharmaceuticals

Jul 02, 2018

Every month, MassBio spotlights a member company and the great work they’re doing to advance the life sciences industry and support the patients we serve. This month we spoke with Robert Ward, Chairman and CEO of Eloxx Pharma, who has over 30 years of leadership experience across nearly every biopharmaceutical function from drug development, commercialization to strategic business development transactions, with commercial success across a variety of different indications.​

Tell us about your organization and your current initiatives

At Eloxx Pharmaceuticals, our team has a shared goal of bringing safe and effective therapies to children and adults suffering from rare genetic diseases as quickly as possible. We are a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel therapeutics to treat cystic fibrosis, cystinosis and other diseases caused by stop codons, or nonsense mutations, which limit production of functional proteins.

Headquartered in Waltham, MA, with R&D operations in Rehovot, Israel, we are a rapidly growing biotech and currently hiring across a wide variety of positions in leadership, research, clinical, regulatory affairs and patient advocacy.

Our lead compound, ELX-02, is a potent read-through inducing drug in several models of genetic disease caused by nonsense mutations and has the potential to be the first disease-modifying therapy for rare diseases that currently have no effective treatments. Based upon favorable results in preclinical trials, we expect to initiate Phase 2 trials in cystic fibrosis and cystinosis by the end of 2018.

How does your organization’s activities help patients now and into the future?

We are currently focused on developing and advancing our lead asset, ELX-02, into Phase 2 clinical trials for cystic fibrosis and cystinosis subject to regulatory review and clearance of our CTA and IND respectively. There is a high unmet medical need in both indications for patients with nonsense mutations.

Eloxx’s technology comes from more than a decade of work pioneered by Dr. Timor Baasov. At the Technion Institute in Israel, he and his team developed designed a library of molecutles from the aminoglycoside scaffold  that preserve the ability to read through stop codons which halt protein production but lack the toxicity properties which limit the long term use of aminoglycosides

Cystic fibrosis is a genetic disease caused by a defective or missing protein. The disease is a progressive and eventually fatal genetic disease that causes a sticky buildup of mucus in internal organs. In the lungs, the mucus clogs the airways and traps bacteria leading to infections, extensive tissue damage and eventually, respiratory failure. Although there are currently FDA approved therapies for cystic fibrosis, for approximately 13% of the CF patient population with nonsense mutations, there are currently no approved therapies. It is estimated that 22% of CF patients has a nonsense mutation on one or both alleles.

Our goal is to develop potential therapies for rare and ultra-rare genetic diseases with high unmet medical need, which have the potential to significantly improve the lives of patients.

What do you see as the biggest challenge facing the life sciences industry today?

One of the biggest challenges facing the life sciences industry today is access and cost. Responsible pricing is paramount to allow patient’s access to medications they need. For rare and ultra rare diseases, the pace of progress is generating a great deal of hope for patients. However, there is a need to balance economic incentives to stimulate the development and marketing of orphan drugs without jeopardizing a patient’s access to therapy.

For example, the importance of Medicaid formulary access cannot be understated. Currently, across the country, many states are implementing formulary restrictions, which limit a patient’s access to treatment.

The rare disease patient community is often left out of these important decisions, and these restrictions may make the difference between life and death for many individuals suffering from rare and ultra rare diseases.

As an organization, we are committed to supporting patients, patient advocacy groups, rare disease organizations. We will continue to advocate for increased visibility and awareness of rare and ultra-rare genetic diseases and access to appropriate therapies. The outlook for pediatric drug development is much improved, and at Eloxx, we continue to do our part to facilitate the momentum.

What’s next for your organization / what are you focused on in the coming year?

Looking ahead in 2018, we will be focused on rapidly growing our organization and advancing ELX-02 in cystic fibrosis and cystinosis. By mid-2018, we expect to submit a clinical trial application (CTA) for cystic fibrosis to the Federal Agency for Medicines and Health Products (FAMHP) in Brussels, Belgium. Around the same time, we expect to submit an investigational new drug (IND) application for cystinosis to the FDA. By the end of the year, Eloxx expects to begin Phase 2 studies in CF and cystinosis, subject to regulatory review and clearance of our CTA and IND.

In 2017, we grew our team by 50% to 18 full-time employees. What’s more impressive is that we currently have 24 openings on our careers page.

If you have a passion for rare diseases and would like to be part of a rapidly growing entrepreneurial culture which promotes diversity, operates through strong teamwork and rewards accountability, and respect, feel free to join us.

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