"Five in Five":
5 ALS compounds with PoC data in 5 years.
About Us
Mission-driven
For all types of ALS
For all types of ALS
• Named after father of founder who died of ALS.
• Based in Montreal, Canada and with a presence in Leuven, Belgium.
For all types of ALS
For all types of ALS
For all types of ALS
• 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).
Collaborative
For all types of ALS
Collaborative
• 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's unique approach
Raya is approaching drug development in ALS and rare neurodegenerative disease in a different way.
Raya is planning to develop 5 compounds
Each compound:
• has substantial safety data in humans.
• has a different Mechanism of Actions.
• is an oral, small molecule
Due to heterogeneity in ALS, a combinatorial approach is key
• No two ALS patients are identical.
• A synergistic approach is needed
• Combining different MOAs offers the best hope for a treatment.
Raya is planning for a dedicated platform trial (adaptive design)
• “Patient-friendly” design
• Smaller placebo arms
• Master Protocol
• Faster start-up
RT1972 has the potential to treat a few pediatric neurodegenerative diseases
• 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.