
eDSP & A-T - eDSP (Red blood cell encapsulated dexamethasone sodium phosphate) is designed to chronically deliver corticosteroid efficacy without toxicity[5] - The company is conducting a Pivotal Phase 3 NEAT clinical trial in pediatric rare disease Ataxia-Telangiectasia (A-T) with no currently approved treatments and a $1+ billion commercial opportunity[9] - 99 patients with A-T enrolled as of June 25, 2025, including 78 participants in 6 to 9 year-old cohort[9, 46] - The estimated prevalence of A-T is approximately 10,000 patients in the U S, U K, and EU4 countries[28, 53] - The company plans to submit NDA with FDA and MAA with EMA in 2026[49] Financials - The company recently closed up to $22 million private placement of securities – with proceeds of approximately $11.5 million upfront and up to $10.4 million if warrants exercised in full[9] - Financing proceeds plus $31.6 million in cash provides funding through topline results in first quarter of 2026 and into the second quarter of 2026 – or second half of 2026 if warrants exercised[9] - The company has invested $100 million in AIDE technology over 20+ years of research & development[13, 76] Pipeline Expansion - The company sees significant pipeline expansion opportunity for eDSP with Duchenne muscular dystrophy (DMD) as second indication with multiple other rare immunology and autoimmune focused rare disease targets[9, 56, 60] - The company plans to dose first patient in DMD Phase 2 clinical study in 2026[60, 64] AIDE Technology - 20+ years of research & development and $100 million invested in AIDE technology[13, 76]