Arrakis Therapeutics Secures $38m in Funding

Arrakis Therapeutics, a pioneering biopharmaceutical company, has completed a $38 million Series A financing led by Canaan Partners with participation by Advent Life Sciences, Pfizer Inc., Celgene Corporation, Osage University Partners, and biotech industry leader Henri Termeer. The company has also named serial entrepreneur and biotech leader Michael Gilman, Ph.D., as Chairman and Chief Executive Officer.

The proceeds from the Series A financing will be used to advance Arrakis’ proprietary discovery platforms, comprised of a high-throughput, comprehensive system of bioinformatics tools, assays and chemical libraries that can identify new ribonucleic acid (RNA) targets and create new small-molecule drugs. Using its TRYST™ and PEARL-seq™ platforms, Arrakis is developing an internal product pipeline of RNA-targeted small molecules focused on neurology, oncology, and rare genetic disorders. The basis of Arrakis’ unique approach is a proprietary chemical biology technology that interrogates structures of folded RNAs in their native state, within cells, to enable discovery and rational design of potent, selective small-molecule inhibitors of RNA function.

“RNA is the locus of most human biology, yet our current pharmacopeia is largely limited to protein targets,” said Dr. Gilman. “Arrakis intends to re-architect small-molecule drug discovery by redirecting, modifying, and creating tools that enable medicinal chemistry to directly address therapeutically important RNA molecules. Our substantial intellectual property, financial resources, and the extensive experience of our team in discovering and developing novel drugs will enable us to build a pipeline of new small-molecule therapeutics for patients not helped by today’s medicines.”

Targeting RNA with Small Molecules

The transcriptome encompasses all of the RNA transcribed from our genetic blueprint, including coding RNAs, which are translated into proteins, and noncoding RNAs, which regulate the fate and function of both coding RNAs and proteins. There are over 200,000 RNA transcripts in the human transcriptome—a vast pool of potential therapeutic targets. Yet today’s small-molecule medicines address approximately 500 protein targets in a small number of structural classes. Accessing the biology embodied by the rest of the transcriptome has required the development of new therapeutic modalities, such as oligonucleotide drugs and gene therapies, which are at the early stages of their clinical evolution. Arrakis intends to open RNA biology to medicines with the attractive and predictable pharmaceutical properties of small-molecule drugs, including oral bioavailability, broad biodistribution, and efficient cell penetration.

“The importance of targeting RNA in disease is well recognized,” said Russell C. Petter, Ph.D., founder and CSO of Arrakis Therapeutics. “But until now, targeting RNA with small-molecule compounds has been thought to be too challenging. Nevertheless, there are a number of precedents for conventional small molecules that bind and modulate RNA, including several approved drugs. These molecules were all discovered in functional screens, and only later did we learn that they acted on RNA. Our goal is to intentionally discover RNA-modulating small-molecule medicines. We believe that new developments in informatics, structural biology, and biophysical tools now make that possible.”

Colleen Cuffaro, Ph.D., principal at Canaan, who is joining the Arrakis board said, “Arrakis is pursuing a fundamentally different approach to RNA targets. For decades, the pharmaceutical industry has invested billions of dollars to develop tools and techniques to create drugs for protein targets. Arrakis is building on this toolkit to create a platform to discover RNA-targeted drugs. In doing so, I believe Arrakis has the potential to create an entirely new class of medicines for patients with unmet need.”

Industry-leading Experts on Management Team and Board  

Arrakis has assembled an executive team and board of directors comprised of industry veterans with established records of building successful biopharmaceutical companies.

The Arrakis senior management team is comprised of the following:

Michael Gilman, Ph.D., CEO and chairman of Arrakis; former founding CEO of Padlock Therapeutics and Stromedix, as well as executive roles at Biogen and ARIAD Pharmaceuticals

Russell C. Petter, Ph.D., founder and chief scientific officer of Arrakis; former executive roles at Celgene, Avila, Mersana, Biogen, and Sandoz/Novartis

Daniel Koerwer, chief business officer of Arrakis; former executive roles at BIND and Biogen

James Barsoum, Ph.D., senior vice president of biology of Arrakis; former executive roles at RANA, Synta, and Biogen

Raj Parekh, Ph.D., founder of Arrakis and general partner at Advent Life Sciences said, “Our investor syndicate is a collaborative team committed to supporting Arrakis’ pioneering approach, and providing the company the resources needed to bring transformative new drugs to patients.”

The Arrakis board of directors is comprised of the following:

Michael Gilman, Ph.D., CEO and chairman of Arrakis Therapeutics

Colleen Cuffaro, Ph.D., principal at Canaan Partners

Carolyn Green, executive director of strategic investments, External R&D Innovation, Pfizer Worldwide Research & Development

Raj Parekh, Ph.D., founder of Arrakis Therapeutics and general partner at Advent Life Sciences

Alan Walts, Ph.D., founder of Arrakis Therapeutics and venture partner at Advent Life Sciences

This article has been republished from materials provided by Arrakis Therapeutics. Note: material may have been edited for length and content. For further information, please contact the cited source.