Navigating the partnering galaxy

June 21, 2023
James Mutamba, PhD, Chief Business Officer of Arrakis

As a small biotech company with new technology at the frontier of the known drug discovery universe, it may feel like you’re charting a course alone in a dark, cold, and empty void with hailing frequencies open, but with no incoming transmissions. But you would be wrong. If you’re intentionally equipped to locate, identify, and steer toward opportunities that fit your drug development aspirations and company goals, a galaxy full of partnering opportunities is out there.

Depending on your focus, there is a wide range of trajectories from which to choose. If you have a lead drug program that will drive most of the value for your company, it may make sense to follow a traditional path that puts you in the orbit of a single major partner, for whom that program is highly strategic.

On the other hand, if you have a platform designed to be an engine for a standalone enterprise, capable of generating dozens of future drugs, then you might plot a course that brings you into the close orbit of multiple complementary partners. This latter course is Arrakis’ strategy. Since our company’s founding in 2015, we have taken the long view and been singularly focused on building an extremely flexible and broadly applicable platform that can develop a host of RNA‑targeted small molecules (rSMs) to deliver precision medicines for dozens of targets that have been out of reach for conventional approaches.

To realize this ambition, we employ a “first-principles” approach to understanding RNA structure. Then, we discover and optimize ligands to these RNA structures to generate small molecule medicines that will either work on their own, or as part of multifunctional drug modalities on targets that drive disease. Having numerous ways to build RNA-based medicines enables our partnering strategy of working with multiple, aligned and highly sophisticated partners in collaborations focused on different drug modalities or disease targets. This strategy has already come to fruition in major partnerships we have established with Roche and Amgen.

At Arrakis, we consider three key elements in finding partners to help us unlock RNA medicine’s full potential: the expansive opportunity space, open collaboration, and broad and deep experience.

Expansive Opportunity Space: A Constellation of Partnerships

Arrakis has historically found itself in the orbit of potential partners, for example larger biopharmas that have hit a roadblock on a targets of interest with their available approaches. We define the opportunity space by identifying key problems we can potentially solve collaboratively, while also advancing key aspects of our platform.

The opportunity space Arrakis traverses is inhabited by a multitude of small molecule drug companies, including some of the largest, most established organizations in our business. Indeed, hundreds of targets that have proven challenging to drug at the protein level can be potentially addressed by going upstream to RNA. Pharma companies interested in probing new ways to reach these once-undruggable targets are potential partners for Arrakis. We all share a common understanding of the benefits and challenges of small molecule therapeutics. Our shared historical framework, based on protein-targeted drug modalities and well-established tools and technologies, provides useful telemetry as we home in on new territory for using small molecules to create functional RNA-targeted medicines.

Our partnership with Roche is primarily centered on the search for rSMs that bind to RNA and directly affect that RNA’s function, for example, its splicing, stability, or translation. This strategic collaboration is focused on exploring rSMs for a broad set of challenging targets selected by Roche across all of its therapeutic areas.

In contrast, our partnership with Amgen integrates the capabilities of both companies’ discovery platforms to engineer RNA degraders. These therapeutics are heterobifunctional molecules that include small molecules that bind to RNA and host cell effector baits that recruit cellular proteins to degrade the RNA, thus selectively destroying RNAs that encode disease-driving proteins.

Arrakis’ unique ability to solve high-resolution RNA crystal structures enables an in-depth approach to finding ligands and understanding how they bind and where they can be derivatized without impacting binding, thereby providing a powerful tool that can be leveraged for a range of drug discovery goals for ourselves and our partners. Another example of an emerging rSM modality, beyond our work with Roche and Amgen, is the rSMs we are designing to be equipped with specific chemistry that generates a covalent bond to RNA. We have built rSMs with covalent warheads that, only upon binding to a given target RNA, selectively and irreversibly modify that RNA, impeding the progress of the translational machinery and thereby preventing expression of the encoded protein. This is a new opportunity space that we look forward to developing with a future partner.

Open Collaboration: Spreading Your Science in the Ecosystem 

Business development and R&D leaders in biotech are always on the hunt for new science that can point the way to discovering novel therapeutics. That is why, as a biotech company seeking potential partnerships, it’s critical to communicate your science at every opportunity, including at scientific meetings, industry forums, and networking events.

At Arrakis, we proactively communicate what we’re building, the challenges we face, and why we believe our broad platform and customized tools for targeting RNA provide the best path to opening up the entire transcriptome to rSM medicines. Sharing how our platform works within the industry ecosystem has allowed potential partners to imagine how to use our tools to solve their own problems and will continue to open up new avenues for exploration. We are interested in complementary partnerships across numerous therapeutic areas, targeting those collaborations in which we interact directly and frequently with partner R&D groups.

Our partnership with Amgen, for example, grew from this willingness to share our science within the industry ecosystem. After scanning several companies in the RNA space, Amgen concluded that we had exceptional expertise to develop RNA-targeted small molecules, and, like us, they envisioned how our RNA binders could help advance a new RNA degradation modality.

As RNA biology comes into ever sharper focus, our platform continues to evolve in tandem, positioning us well to unlock the therapeutic potential of RNA-targeted medicines. We welcome collaboration with experienced partners who recognize the same promise.

Deep Experience and Expertise: Investing in Proven Pioneers 

While it may sound obvious, a critical element for successful collaborations is having deep and complementary experience. In our interactions, pharma companies have expressed that when choosing a partner, they attempt to ensure its leaders have the proven ability to manage the challenges of making a new technology operational and advancing innovative medicines.

The deep experience of our leaders and scientific team at Arrakis has instilled confidence in our partners and allowed us to reference our hard-won lessons. The fact that we speak the same language around small molecule drug development has enhanced our ability to work seamlessly with our partners toward the shared goal of helping patients.

Despite our significant progress to date, we remain self-aware enough to recognize where our expertise lies relative to our partners’. This knowledge has enabled us to identify specific partners with complementary expertise, allowing us to advance our respective missions together.

Arrakis has hailing frequencies open. We send this message to all who would join us on this critical journey—an expedition to unlock the biology of RNA to discover new medicines for millions of people. The expansive opportunities, continually advancing science, and our collective expertise will move us fearlessly forward together.

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