As the drug development pipeline is one fraught with obstacles, with the process from bench to bedside taking as long as 15 years and at a cost of approximately $USD2.6 billion, conducting high quality clinical trials is of paramount importance. With factors to consider such as recruitment and cohort diversity to infrastructure and skilled support staff, while being mindful of cost, trying to find all the pieces to perfectly fit a complex jigsaw puzzle is difficult.
As a niche, neuroscience-focused CRO, Neuroscience Trials Australia is proud of its track record in working with innovative companies in developing novel drugs and devices in the treatment of neurodegenerative diseases and neurological disorders. One such exciting collaboration is with Cognition Therapeutics, a drug discovery and development company based in Pittsburgh, Pennsylvania (US). With the company’s focus on investigating small molecule compounds that target the toxic proteins that cause the cognitive decline associated with diseases such as Alzheimer’s disease, the formation of this partnership made sense.
The team at Neuroscience Trials Australia worked with Cognition Therapeutics for three years in facilitating a Phase I and a small Phase II clinical trials in Australia with the drug Elayta (CT1812). The drug is a proprietary first-in-class, orally available small molecule to treat Alzheimer’s disease and is unique in its disease-modifying nature, high bioavailability and significant penetration across the blood brain barrier. Elayta has the potential to stop memory loss and facilitate the regeneration of synapses, as it targets sigma-2 receptor complex on neuronal synapses, thereby displacing bound and inhibiting further binding of beta amyloid oligomers.
With such promise shown in pre-clinical studies, it was important to ensure the highest quality first-in-human clinical trials to demonstrate the safety and efficacy of Elayta. With the research led by Susan Catalano at Cognition Therapeutics, Phase I clinical trials began in December 2016 in the US. A short time thereafter, Neuroscience Trials Australia helped Cognition Therapeutics conduct Phase I and II clinical trials in Australia, in parallel.
Throughout 2017, these trials demonstrated Elayta was safe and well tolerated in patients with mild-to-moderate Alzheimer’s Disease at three different doses for 28 days. It was found that there were no clinically significant drug-drug interactions, which is important as a way to manage symptoms, patients are often on treatment regimens involving multiple drugs. Notably, treatment with Elayta led to changes to the cerebrospinal spinal concentrations of several key synaptic proteins that are dysregulated in Alzheimer’s disease, including neurogranin & synaptotagmin-1.
Not only that, but we were able to drive this research further, beyond merely assisting in conducting clinical trials. We were able to help Cognition Therapeutics navigate convoluted regulatory frameworks and obtain Fast Track status for Elayta with the Food and Drugs Administration (FDA) in the US. In addition to this, we assisted the company in starting a subsidiary in Australia, which was enabled by the generous R&D tax incentives. We were also able to support Cognition Therapeutics in opening up this Elayta study to a global cohort.
Today, Elayta has progressed into the next stage of clinical testing. Recently, Cognition Therapeutics received two multi-year grants, totalling $USD6.6 million, to fund further clinical studies investigating the drug’s unique synaptoprotective mechanisms of action. This successful collaboration between Neuroscience Trials Australia and Cognition Therapeutics exemplifies the importance of complementary and synergistic partnerships that are driven by a shared dedication to quality and excellence, and a commitment to improve the lives of those affected by neurological disorders.