There is an urgent need to expedite the review of drugs to treat severe or life-threatening conditions, including autosomal dominant polycystic kidney disease, cancer, and rare ocular genetic disorders. Eloxx Pharmaceuticals, Inc. (NASDAQ: ELOX) has taken the initiative to develop such drugs. The company has obtained a Fast Track designation for ELX-02. The drug candidate is currently in Phase 2 clinical trials in CF patients affected by nonsense mutations in the CFTR (CF transmembrane conductance regulator) gene.
The trials aim to evaluate the safety of ELX-02 and also assess its biological activity. It may not have obtained approval as an investigational drug from any global regulatory body. However, Sumit Aggarwal, the President and CEO of Eloxx, says that they are confident that it allows working with the urgency needed on behalf of CF patients.
Clinical Trials and Development Plans
A lot is happening behind the Phase 2 cystic fibrosis clinical trial program for ELX-02. The Cystic Fibrosis Foundation is funding a portion of the trial while Professor Eitan Kerem, MD, oversees the global clinical trial as a Senior Medical Consultant to Eloxx.
Records have it that cystic fibrosis patients have a high burden of this disease and very few treatment options. However, the European Cystic Fibrosis Society-Clinical Trial Network (ECFS-CTN) has given ELX-02 Phase 2 program a “high priority” score. Enrollment into the trial is underway in the US, Israel, and Europe after a temporary but long pose of the COVID-19 pandemic.
The Appointment of a Chief Medical Officer
“The Eloxx pipeline has tremendous potential, and I’m thrilled to be joining a team with the skills and commitment to advance meaningful therapies on behalf of patients,” the newly appointed Chief Medical officer, Ali Hariri, outlined.
Hariri will oversee the clinical development efforts of its promising pipeline of therapies to treat rare diseases. In addition, his experience comes in handy in the advancement of several investigational therapies.
