2020-04-09| Licensing

Arrakis Signs Lucrative Deal with Roche to Develop RNA Targeting Small Molecules

by Ruchi Jhonsa
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By Ruchi Jhonsa, Ph.D.

Arrakis Therapeutics, a startup aimed at targeting RNA announced a strategic collaboration and license deal with Roche to explore new drug candidates for Roche’s existing research programs. The final details of the agreement were laid out last month via videoconference meeting made necessary by the spread of COVID-19. In this licensing deal, the duo will focus on discovering RNA-targeted small molecule drugs against a range of targets across all of Roche’s research and development pipeline. These targets will cover areas of cancer, immunology, infectious diseases, neuroscience, ophthalmology, and rare diseases. Arrakis will receive $190 million in cash upfront along with royalties from sales of any of these products and milestone payments on the progress of each of these compounds exceeding several billion dollars.

Michael Gilman, CEO at Arrakis said this collaboration will be different than many other partnerships in which small biotech does most of the research work and then gives it away to the bigger company. Here, Arrakis will lead the discovery and research work for each target after Roche will do initiate some of the early research. Thereafter, Roche will handle the preclinical and clinical development. As per the agreement, the disease targets will be exclusive for the duo but Arrakis will remain free to work on other targets and collaborate with other companies. This very first deal for Arrakis gives it a chance to validate its products before it takes a big leap.


The Beginning of a New Collaboration

RNA, the molecule that relays genetic information from DNA to protein has long been a target of chemical-based medication. But the challenge in targeting RNA using small molecules has been significant due to its ever-changing unstable shape, high fluidity, and highly mobile nature. In fact, scientists thought it was “simply impossible” or “futile” to target RNA using small molecules till a few years back. However, the outlook of scientists changed when a group of researchers from the Scripps Research Institute devised a method that could make RNA targeting easier. Essentially, the group locked the RNA molecules in one place making them immobile and halting their function.

This idea led to the genesis of many startups including Arrakis Therapeutics in 2015. The company was founded by a group of former Biogen employees with the aim to design small molecules that target specific strands of messenger RNA and stop the production of defective proteins. Arrakis integrates multiple approaches including RNA bioinformatics and structural tools, curated chemical libraries and RNA guided medicinal chemistry to identify RNA targets and small molecules specific for that. The company raised its initial series A funding of $38 million two years after its foundation. At the time they were still figuring out “how to find molecules that can drug RNA”. But last year they hit a jackpot by “getting hits on every mRNA they screened.” “Finally, we cracked the code,” said the company’s CEO Michael Gilman. The company’s internal pipeline includes three oncology targets and one lipid disorder target.

This not only attracted funding from the investors but also attention from several big pharma. Along with $75 million series B funding, the company got attention from Roche. According to Gilman, “Roche seemed to have the highest and most persistent level of interest.” The Swiss firm was the most suitable choice, Gilman says, as it underscored RNA as a valid target considering they were developing in-house small molecule treatment targeting RNA splicing for spinal muscular atrophy. The opportunity was immediately seized by Arrakis and talks between the two parties started about forming a collaboration.


Competitors to Watch Out

Arrakis is expected to face big competition from companies that are chasing RNA as a target. Ribometrix a drug discovery therapeutics company based out of Durham, NC is collaborating with Vertex Pharmaceuticals to discover small molecule drugs targeting functional 3D RNA structures to treat human diseases. The company bagged $30 million in funding in 2018 for the development of these small molecules. Another, startup in the field, Skyhawk Therapeutics who is looking for drugs that could alter RNA splicing inked a $74 million partnership with Biogen, a $60 million deal with Celgene and a deal of undisclosed amount with Genentech, Merck, and Takeda. Since half of Arrakis’s efforts are focused on targeting RNA splicing, Skyhawk can be a big competition for the company.

Related Article: The Rejuvenated Promise of RNA Therapeutics



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