• ALS is a progressive and lethal paralysis leading to respiratory failure
• Life expectancy is 2-5 years after diagnosis
• ~250,000 ALS patients worldwide
• 2 available drugs only moderately extend life
• ~10% of cases have a family history of ALS (fALS)
• ~90% of cases are sporadic ALS (sALS)
• In both fALS and sALS motor neuron degeneration is the fundamental process that leads to paralysis, dysfunction etc.
• Raya is focused on addressing processes that contribute to motor neuron degeneration
• Named after father of founder who died of ALS.
• Based in Montreal, Canada and with a presence in Leuven, Belgium.
• Privately held and self-financed. Currently raising a Seed/Series A Round.
• Focused on developing drugs to treat ALS with a focus on all ALS (including sporadic).
• 5-year mission to have 5+ clinical compounds with PoC/pivotal trial data via 5 different mechanisms of action.
• Raya is working closely with several well-known ALS Clinicians & Researchers.
Raya is approaching drug development in ALS and rare neurodegenerative disease in a different way.
• has substantial safety data in humans.
• has a different Mechanism of Actions.
• is an oral, small molecule
• No two ALS patients are identical.
• A synergistic approach is needed
• Combining different MOAs offers the best hope for a treatment.
• “Patient-friendly” design
• Smaller placebo arms
• Master Protocol
• Faster start-up
• Diseases are orphan indications with no adequate treatment options.
• Company is exploring development programs for each.
• Approval could provide the company with a Priority Review Voucher (PRV).
• PRVs have recently sold for $100M USD.
A French version of the website is coming soon!