Eloxx Acquires Zikani to Focus on Ribosomal RNA-Targeted Gene Therapy

Rare Daily Staff

Rare disease focused biotech Eloxx Pharmaceuticals said it has acquired Zikani Therapeutics in an all-stock transaction, which the companies say has the potential to create a leader in ribosomal RNA-targeted therapies for the treatment of rare diseases and cancer.

The deal adds Zikani’s preclinical stage pipeline in rare diseases and oncology targeting RNA and ribosomal mutations.

Under the terms of the merger agreement, stockholders of Zikani received approximately 7.6 million Eloxx common shares, valued at approximately $25 million, and own approximately 16 percent of the combined company.

Sumit Aggarwal, previously the president and CEO of Zikani, has been named president and CEO of Eloxx, and Vijay Modur, who was Zikani’s chief scientific and medical officer, has been named Eloxx’s head of research and development.

“With the strength of our ELX-02 program for cystic fibrosis, this acquisition provides us with the opportunity to amplify the potential of our innovative science by developing a new class of therapies to treat diseases with limited to no treatment options under the stewardship of leaders with a proven ability to translate technology into treatments for patients,” said Tomer Kariv, Eloxx chairman.

Eloxx is developing novel RNA-modulating drug candidates (designed to be eukaryotic ribosomal selective glycosides) that are formulated to treat rare and ultra-rare premature stop codon diseases. Premature stop codons are point mutations that disrupt protein synthesis from messenger RNA, and have been identified in more than 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.

The company’s lead compound ELX-02 is currently in phase 2 clinical trials in cystic fibrosis (CF) patients affected by nonsense mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. The investigational therapy has shown strong activity across a full range of mutations in CF preclinical models. In phase 1 testing, ELX-02 was generally well- tolerated and demonstrated high bioavailability with consistent pharmacokinetics across both single and multiple-dose studies.

In addition to CF, the combined company plans to file an IND in 2022 for what could potentially become the first oral therapy for protein restoration for patients with nonsense mutations in recessive dystrophic epidermolysis bullosa (RDEB) and junctional epidermolysis bullosa (JEB). RDEB is an incurable, extremely painful, and often fatal skin blistering condition caused by a lack of collagen type VII that is estimated to affect more than 3,000 people worldwide. JEB is the most severe form of EB, with most patients dying in infancy.

By extending the application of ribosomal RNA modulation to the read through of nonsense mutations in tumor suppressor genes, the combined company is also rapidly advancing Zikani’s preclinical research for familial adenomatous polyposis (FAP), an inherited pre-cancerous colorectal disease frequently caused by nonsense mutations in the adenomatous polyposis coli (APC) gene.

Nonsense mutations cause approximately 10 to 12 percent of rare inherited diseases. ELX-02 along with Zikani’s TURBO-ZM library of compounds are anticipated to significantly expand to include the treatment of many other rare diseases and certain cancers. Zikani’s TURBO-ZM platform allows rapid synthesis of novel compounds that can be optimized to modulate the ribosome in a disease specific manner.

“The combined capabilities of Eloxx and Zikani in chemistry, biology, regulatory and drug development, including Zikani’s TURBO-ZM synthetic chemistry platform for designing macrolide-based ribosome modulating agents (RMAs), along with a committed leadership team and talented employees, will further accelerate our ability to impact the lives of those who have rare diseases with the type of urgency and novel thinking that they deserve,” said Aggarwal.

In connection with the acquisition, Silvia Noiman and Martijn Kleijwegt have stepped down from the Eloxx Board. Alan Walts and Raj Parekh who have both served as Zikani directors, were appointed to fulfill the vacancies and serve out the remaining terms of office.

Photo: Sumit Aggarwal, president and CEO of Eloxx