Developing targeted therapeutics for rare diseases
Our novel and proprietary platform technology targets therapeutics exclusively to activated macrophages
Our lead therapeutic 101-PGC-005 ('005) has been granted a Rare Pediatric Disease Designation (RPDD) for treatment of Systemic Juvenile Idiopathic Arthritis (sJIA) flares
'005 is currently being tested in a Phase 3 clinical trial in India (CTRI/2024/01/061531) for COVID-19/ARDS to demonstrate its powerful and safe anti-inflammatory activity
Additional molecules in development enable targeted delivery of: aldosterone, rapamycin, and alpha-linolenic acid
The Right Targets
Activated systemic and tissue resident macrophages are the primary drivers of inflammation in sJIA
Our platform can even safely target areas previously inaccessible to GCs such as the CNS
GC Sparing
Eliminates the risks of traditional GCs such as immunosuppression, adrenal suppression, osteoporosis, stunted growth, and hyperglycemia
Improved Efficacy
By exclusively targeting macrophages, therapeutic effects are increased in the relevant tissue.
Further, with zero off-target effects, combination treatments can be used for best outcomes
Improved Safety
'005 has no effect on the HPA axis, allowing the innate immune system to function properly without cortisol suppression
Development Overview
Phase 1
FIH dose escalation study in 15 subjects (Israel)​
Complete
2021
Clinical Trials
Phase 1
PK study in 6 subjects (India)
Complete
2023
Phase 1
CSF PK study in 7 volunteers (India)
In Progress
2024
Phase 2
Safety and efficacy in 62 hospitalized ARDS/COVID-19 patients; 1:1 randomization vs. dexamethasone (India)
Complete
2023
Phase 3
Safety and efficacy in 270 hospitalized ARDS/COVID-19 patients; 1:1 randomization vs dexamethasone (India)
In Progress
2024
Phase 1
PK study as a direct comparitor to dexamethasone suppression test
Pending
2025
Phase 2/3
Safety and efficacy in treating sJIA flares
Pending
2025
Regulatory Milestones
October, 2024
Rare Pediatric Disease Designation (RPDD) granted for treatment of sJIA flares
Novemebr, 2024
Pre-IND meeting with FDA to discuss US-based clinical study plans and Breakthrough Therapy Designation