Cyclo Therapeutics (CYTH) investor relations material

Company overview and strategy

• Cyclo Therapeutics leverages over 30 years of cyclodextrin expertise, focusing on rare and neurodegenerative diseases with unmet needs, notably Niemann-Pick Disease Type C (NPC) and Alzheimer's disease.

• Trappsol® Cyclo™ is a proprietary hydroxypropyl beta cyclodextrin formulation designed to facilitate cholesterol transport out of lysosomes, addressing cholesterol accumulation in NPC and potentially Alzheimer's.

• The company has a strategic partnership with Rafael Holdings, providing $24M in committed capital and board representation.

• A definitive merger agreement with Rafael Holdings was signed in August 2024 to focus on Trappsol® Cyclo™ for NPC1.

• Leadership team includes experienced executives from major pharma and biotech companies.

Clinical pipeline and progress

• Trappsol® Cyclo™ is in a pivotal Phase 3 trial (Transport NPC) for NPC; enrollment of 104 patients completed in May 2024, with 48-week interim topline data expected in H1 2025.

• The Phase 3 trial spans 30+ sites across 14 countries, with primary endpoint NPC Composite Severity Score and secondary endpoints including SCAFI and Vineland-2.

• Phase 1 and 2 studies demonstrated safety, tolerability, and potential efficacy in stabilizing or improving systemic and neurological symptoms of NPC.

• Ongoing long-term extension study shows durability of effect, with patients exhibiting slowed disease progression and consistent safety.

• Trappsol® Cyclo™ has Orphan Drug and Fast Track designations in the US and EU, with potential for a Priority Review Voucher.

Clinical data and safety

• Preclinical and clinical data show Trappsol® Cyclo™ effectively mobilizes cholesterol from lysosomes, normalizes cholesterol metabolism, and is detectable in CSF after IV infusion.

• Phase 1/2 studies showed 100% of patients were stable or improved, with 89% showing improvement over expected disease progression on the 5D-NPC-SS scale.

• Safety profile is favorable, with most adverse events mild to moderate and manageable; hearing loss and infusion reactions are monitored, with most hearing events resolving.

• Biomarker data indicate reduced cholesterol synthesis and increased catabolism, supporting the mechanism of action.

• Higher doses (2000–2500 mg/kg) suggest greater benefit in SARA domains such as gait, stance, and speech.