Member Spotlight: Q&A with Cyclerion Therapeutics

Sep 03, 2019

Every month, MassBio spotlights a member company and the great work they’re doing to advance the life sciences industry and support the patients we serve. In September, we spoke with Andy Busch, PhD, Chief Innovation Officer at Cyclerion Therapeutics. Andy has extensive R&D and portfolio leadership experience across a broad range of therapeutic categories, including significant expertise in rare and orphan diseases and in the discovery and development of sGC stimulators. Before joining Cyclerion, Andy was chief scientific officer at Shire Plc; head of drug discovery at Bayer; and global head of cardiovascular research at Hoechst and Sanofi-Aventis, where he started his work with sGC modulators and brought the first sGC stimulator to the clinic.

Tell us about your organization, its mission, and current initiatives

Cyclerion is a biotech company focused on discovering, developing, and delivering medicines that treat serious and orphan diseases. Our efforts are centered on a powerful target, called soluble guanylate cyclase (sGC). sGC is located throughout the body and is a critical regulator of a number of biological functions that are essential for human health, such as blood flow, metabolism, inflammation, and fibrosis. We are working to develop therapies called sGC stimulators that enhance these positive effects in specific tissues or organs – for example, the vasculature, the liver, the lungs, and the brain.

We are currently developing potential treatments for diabetic kidney disease (Phase 2), heart failure with preserved ejection fraction (Phase 2), sickle cell disease (Phase 2), neurodegenerative diseases (Phase 1), liver diseases (discovery), and lung diseases (discovery).

How does your organization’s activities help patients now and into the future?

Every day, we’re working toward getting clear answers about the efficacy and safety of the potentially life-changing medications we’re developing. Our goal is to get one or more of these products approved as quickly as possible so they can make a difference for patients living with debilitating, often life-threatening, under-served conditions.

We try to help patients in other ways as well. We make sure that the patient voice is incorporated into everything we do, including the strategic decisions we make as a company and how we design and run our trials. For example, in our clinical studies we frequently use patient-centric endpoints that evaluate patients’ reports of their symptoms and functional or quality of life measures, studying improvements in the symptoms that patients indicate are the most important to them. We also have a patient advisory committee that has significantly informed our understanding of sickle cell disease and the needs of the patient community.

What do you see as the biggest challenge facing the life sciences industry today?

A significant challenge for biopharma is addressing the very different, but equally important, needs of large, heterogenous patient populations and rare disease populations. Forty years ago, pharma was focused almost exclusively on developing blockbuster medications to serve large patient populations. Then, rare diseases came into the spotlight and many companies began developing specialized medications for smaller patient populations, including well-defined sub-populations of large diseases. The pendulum has swung to the point that much of the innovation is now happening in smaller patient populations. With 7,000 rare diseases, many of these conditions are still underserved – but there remain significant unmet medical needs in larger disease areas as well. 

It’s difficult for many companies to serve both small and large populations well. At Cyclerion, our goal is to “follow the science” and pursue the indications that we believe our molecules can best impact. We then employ a “best owner” approach. For orphan and rare indications, we have the expertise and resources to commercialize the medicine ourselves. For larger disease spaces, we intend to partner with leaders in the therapeutic area to get the medicine to patients as efficiently as possible.

What’s next for your organization / what are you focused on in the coming year?

The next 12 months will be very exciting for Cyclerion. We have four key clinical trial read-outs. Our Phase 2 diabetic kidney disease and HFpEF trials, and the Phase 1 study of our molecule for neurodegenerative diseases, will all read out by the end of this year. Our Phase 2 sickle cell disease results will be coming in mid-2020. All of these projects are part of the “dowry” we received when we spun out of Ironwood Pharmaceuticals in April 2019.

At the same time as we’re focused on executing well on our near-term clinical studies, we’re just getting started with our new biotech start-up. We have a fabulous team and an opportunity to build an iconic company by establishing a strategy and culture that supports ongoing innovation.

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