CAD-1883 for treatment of tremor and spinocerebellar ataxia

Essential tremor is a neurological disorder characterized by uncontrollable shaking in different parts of the body, including the head, arms, hands, neck, and chin. It is the most common movement disorder, affecting 10 million people in the U.S. alone.

Spinocerebellar ataxia is a genetic, degenerative neurological condition that affects approximately 6,000 people in the U.S. Patients are readily identified through genetic testing and most often carry genetic abnormalities called “poly-Q expansions,” like to those found in patients with Huntington’s disease. The disease is progressive and over time results in ongoing damage to the cerebellum, a part of the brain that regulates motor control and balance.

CAD-1883 is a first-in-class selective positive allosteric modulator of SK channels (small conductance, calcium-activated potassium ion channels) discovered in a research collaboration between Saniona and Cadent Therapeutics. By increasing the calcium sensitivity of the SK channels, CAD-1883 causes the potassium current to flow at lower calcium concentrations, potentially restoring neuronal firing regularity and improving motor function.

Cadent Therapeutics is developing CAD-1883 for the treatment of essential tremor and spinocerebellar ataxia, two neurological movement disorders. In preclinical disease models, CAD-1883 has demonstrated the ability to improve motor control and reduce tremor. A Phase 2 trial was initiated in the fourth quarter of 2018 for the treatment of essential tremor. Cadent Therapeutics has completed a Phase 2a study of CAD-1883 in essential tremor, demonstrating an improvement in the "Essential Tremor Rating Assessment Scale Performance Score". In May 2019, the FDA granted Orphan Drug Designation to CAD-1883 as an investigational treatment for spinocerebellar ataxia. In January 2020, Cadent Therapeutics received an acceptance from the FDA to initiate a Phase 2a clinical trial in patients with spinocerebellar ataxia.

Saniona has a 3.4% ownership in Cadent Therapeutics as of December 31, 2019. In addition to ownership in Cadent Therapeutics, Saniona is eligible to receive royalties on any potential products developed and commercialized from the SK-program including CAD-1883.

Cadent Therapeutics is a precision neuroscience company developing novel medicines that tune and modulate brain rhythms to restore motor and cognitive function in patients with serious neurological disease. The company leverages its unique precision neuroscience approach combining target specificity, patient selection, drug design and optimization, and novel quantitative endpoints to create first-in-class molecules to treat movement and cognitive disorders. Currently in early clinical development, Cadent Therapeutics is rapidly advancing its pipeline of therapies to treat spinocerebellar ataxia, essential tremor and schizophrenia. Investors include Atlas Venture, Clal Biotechnology Industries, Slater Technology Fund and Novartis.