A NEW GENERATION OF THERAPIES
Peter Nordkild, CEO Asarina Pharma
“For us, GAMSAs represent a paradigm shift in the treatment of stress- and compulsion-related conditions. Selective, specific and safe, GAMSAs can modulate the dramatic effects of GABA, one of the body’s most powerful neurotransmitters— with no broader CNS or GABA impact, meaning no severe side effects. “Sepranolone will be our first launched GAMSA, but we believe it will not be our last. We have a portfolio of GAMSA compounds patented which we believe will be of significant therapeutic value.”
PETER NORDKILD, CEO ASARINA PHARMA
WHAT ARE GAMSAs?
Neurosteroids like ALLO, THDOC, Androstanediol and Pregnanolone that act on the GABA-A receptor have been of great pharmaceutic interest for many years. Many have already been launched as standalone therapies. But key challenges remain.
Such is the potency of these substances that development and safety have often been a concern. GABA-active neurosteroids are amongst the body’s most powerful compounds, quite capable of inducing seizures, anesthesia or worse.
Built on the work of Asarina Pharma’s CSO and Founder Prof Torbjörn Bäckström, a new subgroup of neurosteroids – GAMSAs – has now been developed that specifically modulate the effect of these main neurosteroids, but has no CNS effect or impact on the GABA receptor itself. The result could be a new treatment modality for stress- and compulsion-related compounds—with no major side effects.
GAMSAs: SELECTIVITY AND SAFETY
GABA-AModulating Steroid Antagonists directly target the effects of neurotransmitters at different GABA-A receptor subtypes with high specificity. Their effect is confined to a very specific subtype of the GABA-A receptor, meaning minimal effect on any other CNS mechanism.
As a treatment this ensures:
- Fine-tuned receptor activity without overstimulation
- High selectivity
- Minimal off-target effect
“I believe we are on the crest of a new wave of understanding of just how broad the impact of Allopregnanolone really is. Compulsivity impacts on so many different conditions, from ADHD and OCD/B through to eating disorders and addiction.”
Prof Marco Bortolato, University of Utah
GAMSAs: A NEW FAMILY OF THERAPIES FOR STRESS- AND COMPULSION-RELATED CONDITIONS
GAMSAs represent an entirely new treatment modality for many conditions. Selective, specific and safe, they offer the prospect of a new family of compounds for stress- and compulsion-related conditions that are neither anti-psychotic, nor psychotherapeutic.
Until now, top-line Tourette syndrome treatments, for example, have been either anti-psychotic, associated with severe side effects, or CBIT, known for frequent cases of recidivism. Menstrual Migraine treatment has not involved any neurosteroid or neuroendocrinological mechanism—even though it has been widely acknowledged that analgesic and CGRP antibody treatments remain ineffective.
Professor Torbjörn Bäckström’s work has been instrumental in transforming understanding of how powerfully GABA-A modulating steroids impact our mood, behavior and neurological symptoms. Professor Bäckström has developed, synthesized and patented over 30 GAMSAs, with potential GAMSA compounds available for development to treat:
- Stress-related fatigure
- Cognitive impairments
- Balance treatments