SCA Therapeutics Development

This page lists some therapeutics (mostly drugs) being developed to modify spinocerebellar ataxia (SCA) and Huntington’s disease (HD) progressions. This page is destined to get horribly out-of-date. We’ll see.

Why include Huntington’s disease?

The SCA that I have (SCA3) and HD both are polyglutamine diseases, and gene-based therapeutics for one can likely be stepping stones for the other. HD prevalence is slightly higher than SCA prevalence and a worse disease, so it’s usually given higher priority.

How different is Friedreich’s ataxia?

Friedreich’s ataxia (FA) and SCA(3) are very different diseases. Will there be leverage between therapeutics, like I assume there will be for polyglutamine diseases such as HD and SCA(3)?

  • Since FA is autosomal recessive, the inheritance pattern is completely different from SCA. Also, FA comes on stronger and is fatal to children.
  • The prevalence of FA is about the same as all the SCAs combined. There are at least a dozen pharmaceutical companies working on therapies for FA but only a handful working on SCA.
  • The genetic defect that causes SCA(3) is in an exon and causes a ubiquitous protein to be toxic primarily to the cerebellum. The genetic defect that causes FA is in an intron and causes a protein to not be created that is needed in various areas.
  • Because of the exon/intron situation, SCA(3) can be modified at the DNA or RNA level, but FA can be modified at the DNA level but not the RNA level.
  • There are no gene therapy approaches that will work for both. Though some believe CRISPR/Cas9 will be able to repair billions of mature neurons, I’m more skeptical.
  • Therapeutic approaches to each disease that don’t involve gene therapy are mostly unrelated.


See here for a general description of trial phases.

The following lists are alphabetical.

Specific to SCA or SCA3 (4 listed)

Bioblast Pharma Ltd.

  • Pipeline [find with Google]
  • Therapeutic for SCA3
    • Code name: Cabaletta.
    • Generic name: trehalose, a disaccharide in the same family as table sugar (sucrose).
    • Administration: intravenous.
    • Described by Bioblast as a mutant protein stabilizer.
    • Described here as an autophagy enhancer.
    • General goal: slow cerebellar degeneration.
    • Stage in 2017-November: phase 2a successful and complete in Israel.
  • Find with Google: Bioblast and ataxia

Bioblast published positive phase 2a trial results on 2017-01-18: SCA3 progression did not worsen in 14 people after 6 months nor in 5 people after 12 months.

Bioblast has spent most of 2017 in financial trouble, seeking partnership or merger opportunities. The company is using JSB Partners to assist in business development. Details as of 2017-11-02.

2018-01-04: Trehalose has been dominating the news with how it might be proliferating some fatal superbugs.

Biohaven Pharmaceutical

  • Pipeline [find with Google]
  • Drug for SCA
    • Code name: BHV-4157 (link goes to my own assessment).
    • Generic name: trigriluzole (link goes to my own assessment).
    • Administration: by mouth.
    • Mechanism of action: reduces glutamate in the brain.
    • General goal: slow cerebellar degeneration by reducing needless excitement of neurons.
    • Stage in 2017-November: Phase 2 is considered to have failed in the U.S.
  • Find with Google: Biohaven and ataxia

Cadent Therapeutics

  • Pipeline [find with Google]
  • Drug for SCA and essential tremor (ET):
    • Name: no public name.
    • I think the and is important. This seems to be for people with SCA and ET, or rather ET caused by SCA. Their website uses 3,000 per 100,000 in the U.S. as having ET and 2 per 100,000 in the U.S. as having SCA. Presumably those with SCA have a higher ET prevalence, but the combination must be quite small—hundreds of people, maybe into the low thousands in the U.S.
    • Good term to know: SK channel.
    • Allosteric activator: improves regulation of brain activity to help restore motor and cognitive function.
    • 2017-10-17: expected transition to clinical phase in 2018.
  • Find with Google: Cadent and ataxia

Shionogi & Co., Inc.

  • Pipeline [find with Google]
  • Drug for SCA
    • Code name: S-0373, or KPS-0373 at partner Kissei Pharmaceuticals
    • Generic name: rovatirelin.
    • Non-peptide mimetic of thyrotropin-releasing hormone (TRH)
      • Mimetic: essentially an imitation. In other words, it’s synthetic TRH.
    • TRH is thought to improve neurologic dysfunction by improving cerebral blood flow.
    • Stage in 2017-November: phase 3 in Japan.
    • Just guessing: possibly a follow-on of some sort to taltirelin, approved in Japan in 2000 as an SCA therapeutic—the first ever, I think.
  • Find with Google

Specific to Huntington’s disease (4 listed)

Ionis Pharmaceuticals, Inc.


Sangamo Therapeutics, Inc.

Vybion, Inc

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