Investigational Spinocerebellar Ataxia Drug Get Green Light From FDA


The phase 2 clinical trial of CAD-1883 will assess the efficacy and safety of the drug compared to placebo.

Jodie Morrison

Jodie Morrison

Cadent Therapeutics has announced that is has received an investigational new drug approval (IND) from the FDA for CAD-1883, which is being assessed for the treatment of spinocerebellar ataxia (SCA), which affects between 6000 and 11,000 individuals in the US.

“The FDA acceptance of our second IND application for CAD-1883 signifies another important milestone for Cadent. With our clinical proof of concept data in hand for the SK channel, we now look forward to driving to phase 2 efficacy data in our lead indication of SCA, where no current approved therapies exist for this undeserved population,” stated Jodie Morrison, chief executive officer of Cadent Therapeutics, in a statement.

In June of 2019, CAD-1998 was granted orphan drug designation by the FDA for the treatment of SCA. The novel, first-in-class, positive allosteric modulator of SK channels increases calcium sensitivity, causing a flow at lower calcium concentrations and restoring neuronal firing regularity while improving motor function.

Phase 1 results revealed that the drug was well-tolerated, with subjects experiencing only transient adverse events (AEs). The multicenter, placebo-controlled, randomized, phase 2 trial will assess safety and efficacy profiles. Outcomes will be recorded on changes from baseline in the Scale for the Assessment and Rating of Ataxia across a 12-week treatment period. Following the oral administration of CAD-1883, investigators will also observe multiple objective biomarkers.

The company has not yet announced when the trial will begin.

The investigational agent has also been examined for the treatment of essential tremor (ET). A phase 2 trial (NCT03688685) initiated in late 2018 that included 25 participants demonstrated an observed signal, with responder analysis ongoing. According to Cadent’s website, results of the proof of concept study in ET supported the pivot to a lead indication for CAD-1883 for SCA. The company currently has no further studies in ET planned.2


1. Cadent Therapeutics announces FDA acceptance of IND application for CAD-1883 for Spinocerebellar Ataxia (SCA)[news release]. Cambridge, MA: Cadent Therapeutics. January 23, 2020. Accessed January 24, 2020.

2. Our pipeline. Cadent Therapeutics website. Accessed January 24, 2020.

Related Videos
Michael Levy, MD, PhD
Michael Kaplitt, MD, PhD
Michael Kaplitt, MD, PhD
video 4 - "Amyloid Cascade Hypothesis of Alzheimer’s Disease"
Video 3 - "Amyloid Precursor Protein and Amyloid Beta Species in Alzheimer’s Disease"
Svetlana Blitshteyn, MD, FAAN, director and founder of Dysautonomia Clinic
© 2024 MJH Life Sciences

All rights reserved.