Eloxx Acquires Zikani with Hopes to Rule the RNA Wave for Rare Disease

Eloxx Pharma has experienced a string of changes over the past year, coming to a head with today’s announcement that the company will be acquiring early-stage biotech Zikani Therapeutics in an all-stock deal.  

With preliminary Phase II results for lead cystic fibrosis drug ELX-02 expected soon, Eloxx is banking on the acquisition positioning the combined company as a leader in the ribosomal RNA-targeted therapies field. Stockholders of Zikani will receive 7.6 million shares of Eloxx, owning around 16% of the combined company. 

In February 2020, Eloxx was already shaking things up with leadership, replacing its CEO by promoting its chief operating officer to the role and tapping the CMO to fill the vacant chairman seat. Now, under the terms of this deal, Zikani’s president and CEO, Sumit Aggarwal, will take the helm at Eloxx. Additionally, Zikani’s CSMO, Vijay Modur, will head up Eloxx’s R&D. 

“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. 

ELX-02's Phase II trials hit a snag last spring when COVID-19 hit, halting hundreds of clinical studies. Resumed in June, the two trials enrolled individuals with CF who have at least one G542X mutation. Stop, or nonsense, mutations cause a midway halt in production of the CFTR protein midway through the process, leaving patients with shortened, non-functional proteins then destroyed by cells. Zikani’s focus on development of RMAs for rare diseases caused by nonsense mutations is a perfect fit. 

According to the Cystic Fibrosis Foundation, nonsense mutations are found in around 13% of CF patients. In January, the foundation announced an award of up to $2 million to Eloxx to support its global Phase II program. The drug’s Phase I showed high bioavailability, while preclinical models showed strong activity across a full range of mutations.  

“The Phase 2 trials are designed to validate the safety of ELX-02 and assess its biological activity. We look forward to completing enrollment in the first four treatment arms by mid-year and reporting data from these treatment arms in the second half of this year,” Modur said. 

While CF is the primary focus, it isn’t the only one. In 2022, the company plans to file an IND to test the drug as a first oral therapy for protein restoration in patients with nonsense mutations in Recessive Dystrophic Epidermolysis Bullosa (RDEB) and Junctional Epidermolysis Bullosa (JEB). RDEB in incurable and can often be fatal, while JEB leaves most patients dying in infancy.  

The applications won’t stop there. ELX-02, in addition to Zikani’s TURBO-ZM portfolio of compounds, has plans to expand into many other rare disease indications and some cancers. It’s estimated that nonsense mutations cause approximately 10-12% of rare inherited diseases. With 85% of proteins considered undruggable, RNA-modulation is seemingly the wave of the future. 

“We are excited about the potential of ELX-02 and combining the companies opens the door to build a leadership position in genetic therapy by rapidly developing treatments that can restore functional proteins in patients with nonsense mutations in their RNA,” said Aggarwal.  

“The combined capabilities of Eloxx and Zikani in chemistry, biology, regulatory and drug development, including Zikani’s TURBO-ZMTM 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."