Florida-based Cyclo is developing a hydroxypropyl beta cyclodextrin formulation, branded Trappsol Cyclo, in Niemann-Pick disease type C (NPC) and Alzheimer’s disease. NPC is a rare, neurodegenerative disease that causes dementia.
Cyclo recently completed enrollment in a Phase I NPC study in the US and is approaching that point in another early phase trial featuring sites in Europe and Israel. The progress has led Cyclo to start making plans for a pivotal program.
In the coming months, Cyclo will share its NPC pivotal trial design with the US Food and Drug Administration and, after that, the European Medicines Agency. Cyclo will need the support of a contract research organisation (CRO) to execute its development strategy.
Worldwide Clinical Trials has landed the deal to support the pivotal study. The partners will work to get Trappsol Cyclo into a pivotal NPC trial while also gearing up to test the drug in patients with Alzheimer’s.
Cyclo’s interest in Alzheimer’s is underpinned by the results of an expanded access study that gave its drug to one patient with the late-onset form of the disease.
Third parties have supported the work on Trappsol Cyclo overseen to date by Cyclo, which relies on partners to help it in “in managing, monitoring and otherwise carrying out clinical trials and research activities.” These partners are vital to the successful execution of Cyclo’s studies.
In a regulatory filing, Cyclo wrote, “We rely or will rely heavily on these parties for the execution of our clinical studies and control only certain aspects of their activities. Accordingly, we may have less control over the timing and other aspects of these clinical trials than if we conducted them entirely on our own.”
While Cyclo has always relied on partners, the pivotal study will mark a step up in the scale of Cyclo’s clinical development activity and will therefore require the support of a service provider with the experience and resources to run late-phase clinical trials.
Cyclo will need to raise additional money to fund the clinical trials. As of the end of September 2019, Cyclo had $5m (€4.5m) in cash and cash equivalents, a sum its management calculated is enough to see the company through to November 2020.