Presentation1

January 7, 2022

By: Randy Pinchuk, Executive Director, Corporate Strategy & Business Development

Strategic collaborations have long played an instrumental role in the development and commercialization of innovative therapeutics. When collaborations are well executed, they can have exponential benefits on the partners, patients, and society. Recent collaborations spurred by the COVID-19 pandemic stretched across industries, borders, and public and private sectors, highlighting the value of such partnerships in the development, advancement and distribution of lifesaving medicines across the world.

Why We Partner

At Marinus, we are constantly seeking to improve the lives of patients in need. In rare and orphan diseases, the urgency to provide new treatment options to patients is particularly critical given the significant unmet medical need and likelihood for patients to become increasingly debilitated over time. As such, our approach to partnering is anchored in making innovative treatments available to patients across the world as quickly and efficiently as possible.

Forging alliances with like-minded innovators in the field can help increase R&D efficiency, expand geographic reach, and strategically manage late-stage commercialization costs for greater patient impact. Importantly, partnering may also provide non-dilutive capital to help support ongoing operational, development, and commercialization requirements while allowing us to build a sustainable framework for innovation so we can continue to make a long-term difference for patients.

Small and large pharmaceutical companies alike can benefit from partnering. As it relates to Marinus’ recent alliance with Orion Corporation, collaborations can help large pharma companies optimize their portfolios and pipelines as they seek new opportunities for growth. For smaller companies like Marinus, which may not have the resources or infrastructure to commercialize in global markets, partnerships can help increase scale quickly and support development and broader adoption of products.

Marinus’ Collaborations

Marinus’ collaboration with Orion Corporation is a great example of two companies partnering to create added value for a broad array of stakeholders, especially patients. As a member of Marinus’ Business Development team, I am proud to have helped structure and execute an alliance that allows both companies to maximize the potential impact we can have on patients as we work to develop and make new treatments available in Europe. Indeed, being able to build an effective, sustainable, and value-creating entity where there wasn’t before is the reason I got involved in partnerships and it is what keeps me motivated to pursue more.

From early in the engagement process with Orion, it was clear that our companies were a good cultural and strategic fit. The Orion team was easy to work with, collaborative in nature, and embodied our values of commitment, innovation and community. In addition, they have extensive expertise in critical strategic areas for Marinus and have the infrastructure to support both our orphan disease and acute care franchises.

Our functional teams are now hard at work supporting launch preparation in Europe as well as advancing a robust clinical development plan to enrich our pipeline. Orion’s clinical and commercial expertise and their presence in the patient advocacy communities across European markets have already proven valuable and will continue to be critical accelerators as we charge forward.

Partnerships are not just limited between corporations; we see them occur outside of industry to help achieve shared strategic goals and, in turn, maximize benefit for the community. For instance, Marinus recently joined forces with the Loulou Foundation, a private foundation dedicated to advancing research on the causes and treatment of CDKL5 deficiency disorder (CDD), and six other biotech and pharmaceutical organizations, to undertake a key observational CDD clinical study to better understand the natural history and the utility of various clinical assessments.

The results of the study will ultimately be shared with the entire scientific and medical community to aid in the design of clinical trials and to inform therapeutic development for CDD and related neurodevelopmental disorders, which will certainly prove to be extremely valuable as we seek to broaden our reach to more patients.

Expansion of Global Partnering Strategy

With European commercial efforts underway, we now turn our global partnership focus to other priority markets where we hope to expand adoption among a broader patient community. Highlighting the value of global partnerships, we will unquestionably have to lean on our future partners to help navigate unique regulatory environments, orphan pathways, and development and commercial requirements in these markets.

In addition, we will continue to build upon our relationships with local and global advocacy organizations to help identify patients which can help support clinical study execution and broaden access and adoption in local markets.

Patient Advocacy Groups Are Key Drivers of Change

Collaborating with patient advocacy groups from the earliest stages of clinical development is integral to successful pharmaceutical R&D. As we think about global expansion to increase patient reach, it wouldn’t be possible without the insights and learnings of patient advocacy groups worldwide. In addition to being key drivers of change, these groups can help to educate and drive awareness across their networks on innovative treatment options.

Patient identification and enrollment can be challenging when running a clinical trial in new markets, especially in rare and orphan diseases with smaller patient populations. To help address these challenges, advocacy organizations can provide a wealth of local knowledge as well as connectivity to patients, physicians, and treatment centers. This type of information can be extremely valuable as we consider development requirements in new markets and can save significant time and resources, thereby supporting the partnering process.

Stronger Together

At Marinus, we believe that the greatest catalyst for driving change through innovation comes from collaborating with like-minded partners who share in our patient commitment. Strong partnerships and open innovation have the power to spark new possibilities that one organization could not otherwise create on its own and we look forward to seeing where our current and future collaborations take us.