Arrakis Therapeutics

Press Release.

Amgen and Arrakis Therapeutics Announce Multi-Target Collaboration to Identify Novel RNA Degrader Small Molecule Therapeutics

Collaboration Creates “Targeted RNA Degraders” by Bringing Together Amgen’s Induced Proximity Platform Discovery Expertise and Arrakis’ Leading-edge RNA-targeted Drug Discovery Platform

THOUSAND OAKS, Calif. and WALTHAM, Mass., Jan. 11, 2022 – Amgen (NASDAQ:AMGN) and Arrakis Therapeutics today announced a research collaboration focused on the discovery and development of RNA degrader therapeutics against a range of difficult-to-drug targets in multiple therapeutic areas. This new class of “targeted RNA degraders” consists of small molecule drugs that selectively destroy RNAs encoding disease-causing proteins by inducing their proximity to nucleases.

Under the terms of the agreement, Arrakis will lead research activities for the identification of RNA-targeted small molecule (rSM) binders against a broad set of targets nominated by Amgen. Both parties will collaboratively design and functionalize these molecules to specifically degrade targeted RNAs, and Amgen will lead further preclinical and clinical development activities. Amgen will pay $75 million upfront to Arrakis for five initial programs and will have the option to nominate additional programs. For each program, Arrakis will be eligible for additional payments from Amgen for preclinical, clinical, regulatory and sales milestones, and royalties up to low double digits. Arrakis could potentially receive several billion dollars in future payments if all milestones are met and future program options are exercised.

“Targeted RNA degradation is an exciting area that is pushing the boundaries of drug discovery and design,” said Raymond Deshaies, Ph.D., senior vice president of Global Research at Amgen. “The collaboration with Arrakis combines Amgen’s induced proximity expertise in discovering multispecific molecules to target the biologic mechanisms of disease and Arrakis’ pioneering discovery platform to predict RNA structures and identify small molecules that bind to them, significantly broadening the possibilities of addressing difficult protein targets considered undruggable because they may not have binding sites needed for conventional medicines. Combining this approach with Amgen’s targeted protein degradation induced proximity research already underway has the potential to significantly expand the druggable genome.”

By integrating the capabilities of the two innovative discovery platforms from Amgen and Arrakis, the collaboration creates an opportunity to design and engineer targeted RNA degraders. Amgen has built its Induced Proximity Platform to identify multispecific molecules that harness the power of cell biology by forming novel connections between natural effectors and targets. One end of the molecule binds to the target to be altered (inhibited, activated or destroyed) and the other end binds to a cellular effector that acts on the target, offering the potential to engage a broad range of cellular mechanisms to treat disease. With targeted RNA degraders, the effector, such as a ribonuclease or other RNA modulator, is brought into proximity of the RNA to degrade or otherwise modify the disease-causing RNA of interest. This complements Amgen’s existing efforts to target RNA with siRNA. In this collaboration, Arrakis’ rSM platform will be applied as a drug discovery engine to identify small molecules that bind target RNA. These rSMs will then be functionalized with nuclease recruiters to create heterobifunctional molecules that trigger degradation of disease-relevant RNA targets.

“We are excited to partner with Amgen’s strong research team to pursue a shared goal of creating a new class of medicines that induce degradation of disease-causing RNAs. This collaboration further demonstrates the utility of our proprietary rSM discovery platform for targeting RNA with small molecules and paves the way for creating powerful new treatments for patients,” said Michael Gilman, Ph.D., chief executive officer of Arrakis. “Based on our long-term goal to build a broad and industry-leading platform that adapts state-of-the-art drug discovery tools to target RNA biology, we have enabled a range of different applications and collaborations to leverage our science, a strong capital position and the ability to grow our business and our impact to advance RNA-targeted drug programs for diseases unaddressed by today’s medicines.”

About Amgen

Amgen is committed to unlocking the potential of biology for patients suffering from serious illnesses by discovering, developing, manufacturing and delivering innovative human therapeutics. This approach begins by using tools like advanced human genetics to unravel the complexities of disease and understand the fundamentals of human biology.

Amgen focuses on areas of high unmet medical need and leverages its expertise to strive for solutions that improve health outcomes and dramatically improve people’s lives. A biotechnology pioneer since 1980, Amgen has grown to be one of the world’s leading independent biotechnology companies, has reached millions of patients around the world and is developing a pipeline of medicines with breakaway potential.

Amgen is one of the 30 companies that comprise the Dow Jones Industrial Average and is also part of the Nasdaq-100 index. In 2021, Amgen was named one of the 25 World’s Best Workplaces™ by Fortune and Great Place to Work™ and one of the 100 most sustainable companies in the world by Barron’s.

For more information, visit www.amgen.com and follow us on www.twitter.com/amgen.

About Arrakis Therapeutics

Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. Arrakis is building a proprietary pipeline of RNA-targeted small molecule (rSM) medicines focused on cancer and genetically validated targets in other disease areas. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life science investors. The company is located in Waltham, Massachusetts.

For more information, please visit www.arrakistx.com and engage with us on Twitter @ArrakisTx or on LinkedIn.

Press Release.

Arrakis Therapeutics Enters Strategic Collaboration and License Agreement with Roche for Multi-Target Program Utilizing RNA-Targeted Small Molecule Drug Discovery Platform

Arrakis Receives $190 Million Upfront Cash Payment with Potential for Multi-Billion Dollar Future Payments

Waltham, MA – April 8, 2020 – Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small molecule medicines that directly target RNA, today announced a strategic collaboration and license agreement with Roche (SIX: RO, ROG; OTCQX: RHHBY) for the discovery of RNA-targeted small molecule (rSM) drugs against a broad set of targets across all of Roche’s research and development areas. Under the terms of the agreement, Arrakis will lead discovery and research activities for each target to a defined point, at which time Roche will have the right to exclusively pursue further preclinical and clinical development. Arrakis will receive an upfront payment of $190 million in cash and may also receive preclinical, clinical, commercial and sales milestone payments and royalties for any resulting products. The aggregate potential value of future payments to Arrakis exceeds several billion dollars, subject to regulatory approvals and other conditions being met.

“We are excited to partner with Roche’s strong research and development teams. Together, we share a common vision of accessing new drug targets at the RNA level and thereby discovering novel medicines to treat diseases with high unmet medical need. The collaboration will increase the number of new treatments for patients arising from our proprietary rSM discovery platform,”

said Michael Gilman, Ph.D., Chief Executive Officer of Arrakis.

“In addition to the Roche collaboration, we are further building our capabilities and advancing our wholly-owned rSM programs for diseases unaddressed by today’s medicines.”

“Through our pioneering work in RNA biology, we have built a platform for creating and adapting drug discovery tools that allow us to predict and validate the structure of RNA targets, locate druggable pockets, identify drug-like hits, and conduct medicinal chemistry programs to discover a new class of RNA-targeted medicines optimized for potency, selectivity, and safety. This agreement with Roche underscores the value inherent in our rSM platform and will enable us to continue to make leading discoveries and further scientific contributions in the field,”

said Jennifer C. Petter, Ph.D., Founder and Chief Scientific Officer of Arrakis.

Arrakis is taking a broad approach, targeting multiple mechanisms across the lifecycle of RNA to establish a new paradigm for small-molecule drug discovery. The company’s discovery platform integrates leading-edge RNA bioinformatic and structural tools, curated chemical libraries, RNA-specific assays, and RNA-guided medicinal chemistry. In addition to collaborating with partners, Arrakis is developing an internal pipeline of rSMs to treat a range of serious illnesses including cancers and other diseases where strongly validated targets and drivers of disease have been identified but have proven challenging with other drug approaches and modalities.

About Arrakis Therapeutics

Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. Arrakis is building a proprietary pipeline of RNA-targeted small molecule (rSM) medicines focused on cancer and genetically validated targets in other disease areas. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors. The company is located in Waltham, Massachusetts. For more information, please visit www.arrakistx.com and engage with us on Twitter @ArrakisTx or on LinkedIn.

###

Investor Contact:
Will O’Connor
212-362-1200
will.oconnor@sternir.com

Media Contact:
Kathryn Morris
914-204-6412
kathryn@theyatesnetwork.com

Press Release.

April 18, 2019

Michael Gilman, Ph.D., to expand role to full-time CEO to lead company to clinical stage
Having established first-in-industry RNA-targeted Small Molecule (rSM) platform, Arrakis will focus pipeline in oncology and genetically validated targets in other diseases
Financing led by venBio Partners and Nextech Invest

Waltham, Mass., April 18, 2019 – Arrakis Therapeutics, a biopharmaceutical company pioneering the discovery of a new class of small-molecule medicines that directly target RNA, has completed a $75 million Series B financing co-led by venBio Partners and Nextech Invest, with participation by new investors Omega Funds, HBM Healthcare Investments, GV (formerly Google Ventures), WuXi AppTec Venture Fund, and Alexandria Venture Investments, as well as all existing investors, Canaan Partners, Advent Life Sciences, Pfizer Ventures, Celgene Corporation, Osage University Partners and the estate of Henri Termeer. In addition, the company announced that Michael Gilman, Ph.D., will expand his role to full-time Chief Executive Officer in addition to continuing to serve as Chairman of the Board of Directors.

“With this financing and outstanding syndicate of investors, Arrakis will leap to the next stage of realizing our vision of creating a new class of medicines with RNA-targeted small molecules, or rSMs,”

said Dr. Gilman.

“We have built an end-to-end platform for the discovery of rSMs by creating or adapting tools that allow us to predict and validate the structure of RNA targets, locate druggable pockets, identify drug-like hits, and conduct medicinal chemistry programs to improve potency, selectivity, and safety. We are now operating this platform at scale to create a pipeline of utterly novel rSM medicines. I am excited to commit my full effort, along with the Arrakis team and our investors, to drive our discoveries into powerful new medicines for patients.”

The proceeds from the Series B financing will enable Arrakis to build a pipeline of novel RNA‑targeted small molecules, with the goal of reaching clinical testing with one or more candidates. The company will focus its internal drug development in oncology and genetically validated targets in other disease areas. In addition, the funding will enable Arrakis to continue to refine and expand its first-in-industry rSM discovery platform, including a high-throughput, comprehensive suite of computational tools, biophysical and cellular assays, and chemical libraries that are uniquely designed to create new small-molecule drugs for RNA targets.

“Arrakis Therapeutics is the clear leader in the emerging rSM field. We are pleased to support their differentiated strategy to transform the drug discovery toolkit to focus on RNA and open hundreds of important new targets to therapeutic intervention. Arrakis’ deeply experienced team is uniquely qualified to execute this strategy,”

said Richard Gaster, M.D., Ph.D., Principal at venBio Partners.

“The demand for more effective and better tolerated cancer drugs is high, creating the biggest and fastest growing market in healthcare. RNA is now a validated therapeutic target, and drugging RNA with conventional small-molecule medicines can provide cancer patients with options not achievable by any other means,”

said Jakob Loven, Ph.D., Partner at Nextech Invest.

In conjunction with the Series B financing, Dr. Gaster and Dr. Loven will join the Arrakis Board of Directors.

In its next stage of growth, Arrakis will employ its proprietary rSM drug discovery platform to discover novel RNA-targeted small molecules and advance lead candidates toward clinical testing. Since the company’s inception, Arrakis has systematically reconfigured drug discovery tools for RNA targets and achieved the following:

A systematic approach to identify and validate druggable RNA targets, enabling the company to target multiple aspects of RNA biology; these approaches include:

• in silico tools to identify druggable RNA targets at scale;
• high-throughput molecular biology tools to validate these targets.

Multiple screening methods for identifying tractable targets and chemical matter, including:

• screening of hundreds of targets to date;
• identification of druggable RNA binding pockets;
• deriving the principles of molecular recognition of RNA.

Advancement of rSM drug programs against novel RNA targets and with strong intellectual property, including:

• launch of four programs against RNA targets that encode proteins that are otherwise undruggable;
• chemical biology tools to elucidate the mechanism of action and selectivity of drug candidates;
• an intellectual property estate comprising new methods, compounds and targets.

About Arrakis Therapeutics
Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. The company has developed a proprietary platform to identify new RNA targets and drug candidates to treat diseases unaddressed by today’s medicines. Arrakis is building a proprietary pipeline of RNA-targeted small molecules (rSMs) focused on cancer and genetically validated targets in other disease areas. The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors. The company is located in Waltham, Massachusetts. Please visit www.arrakistx.com.

Press Release.

Veteran management team to re-architect small molecule drug discovery to pursue new RNA targets within vast human transcriptome

Waltham, Mass., February 27, 2017 – 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 of Arrakis.

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 theresources 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

Observers on the Arrakis board of directors are Tim Shannon, M.D., general partner at Canaan Partners; Elaine Jones, Ph.D., VP of Pfizer Ventures; Peter Worland, Ph.D., VP Integrative Research Development at Celgene; and Matthew Cohen, Ph.D., principal at Osage University Partners.

About Arrakis Therapeutics

Arrakis Therapeutics is a biopharmaceutical company pioneering the discovery of a new class of medicines that directly target RNA. The company’s TRYST™ and PEARL-seq™ platforms identify new RNA targets and drug candidates to treat diseases unaddressed by today’s medicines. Arrakis is developing a proprietary pipeline of RNA-targeted small molecules focused on neurologic diseases, cancer, and rare genetic diseases.

Arrakis was founded in 2015 by Russell Petter, Ph.D., Alan Walts, Ph.D., Henri Termeer and Raj Parekh, Ph.D. with a vision to create a proprietary, transformational discovery platform that identifies small-molecule drugs that act directly on disease-causing RNA.  The company was established with seed funding provided by Advent Life Sciences and Henri Termeer.  The company brings together scientific leaders in RNA structure, chemistry and biology, along with a highly experienced management team and the backing of leading life sciences investors. The company is located in Waltham, Mass.  Please visit www.arrakistx.com.

About Advent Life Sciences

Advent Life Sciences founds and invests in early- and mid-stage life sciences companies that have a first- or best-in-class approach to unmet medical needs. The investing team consists of experienced professionals, each with extensive scientific, medical and operational experience, a long-standing record of entrepreneurial and investment success in the US and Europe, and is particularly focused on supporting entrepreneurs and founders to take innovative new medical entities from concept to approval. The firm invests in a range of sectors within life sciences, principally drug discovery, enabling technologies and med tech, always with an emphasis on innovative, paradigm-changing approaches. Advent Life Sciences has a presence in the UK, US and France.  For more information, please visit AdventLS.com.

About Canaan Partners

Canaan Partners is an early stage venture capital firm that invests in entrepreneurs with visionary ideas.  With $4.2 billion under management, a diversified fund and 190 exits to date, Canaan has invested in some of the world’s leading technology and health care companies over the past 30 years. Canaan’s focus areas include fin tech, enterprise/SaaS, marketplace, e-commerce, biopharma, digital health and medtech.  To learn more about our people and our portfolio, please visit canaan.com.

About Pfizer Inc.

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products. Our global portfolio includes medicines and vaccines as well as many of the world’s best-known consumer health care products. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For more than 150 years, Pfizer has worked to make a difference for all who rely on us. For more information, please visit us at www.pfizer.com.

About Celgene Corporation

Celgene Corporation, headquartered in Summit, New Jersey, is an integrated global biopharmaceutical company engaged primarily in the discovery, development and commercialization of innovative therapies for the treatment of cancer and inflammatory diseases through next-generation solutions in protein homeostasis, immuno-oncology, epigenetics, immunology and neuro-inflammation. For more information, please visit www.celgene.com.

About Osage University Partners

Osage University Partners is a venture capital firm focused on investing in startups that are commercializing pioneering university technologies. Osage partners with top research universities to invest in their most innovative startups, and Osage shares its investment profit with its partner institutions. The firm invests in software, hardware, and life science companies at all stages of company development. Osage has partnered with over 80 universities, including 36 of the top 50 U.S. institutions by research expenditures, and has invested in over 60 of their spinouts. Osage University Partners is part of a family of investment funds within Osage Partners, which is based in Philadelphia, PA and manages in excess of $500 million. For more information, visit www.osageuniversitypartners.com.

Contact:
Kathryn Morris
The Yates Network
Tel:  845-635-9828
kathryn@theyatesnetwork.com