Pillar Two

Medical Countermeasures

A host directed broad-spectrum antiviral candidate for filovirus and high-consequence pathogen preparedness.

Pipeline

Candidate
Preclinical
Phase I
Phase II
Phase III
FDA Review
Marketed
Status
OKG-0101 IVInfluenza A/BIntravenous
Preclinical
OKG-0801EbolaIntravenous
Preclinical
Orphan designation
OKG-0802MarburgIntravenous
Preclinical
OKG-0803SudanIntravenous
Preclinical
OKG-0804NipahIntravenous
Preclinical
Additional targets under evaluation include Zika and Tacaribe.

Overview

Treatment and post-exposure prophylaxis in one molecule

Ranpirnase is being advanced as a medical countermeasure against filoviruses and other high-consequence pathogens.

Ebola is nearest to use in humans, on the strength of pre-existing data and the immediate need for an intermediate solution while vaccines are developed. The Ebola Bundibugyo strain is the top priority.

By The Numbers

OKG-0801: Antiviral Activity Against Ebola

47.8–70.2

Selectivity indices eliminating EBOV in cultures of Vero E6 cells

70%

Of mice protected from progressive infection by a single intravenous dose of 0.1 mg/kg ranpirnase

100%

Of female mice survived infection after intraperitoneal administration of 0.1 mg/kg ranpirnase for ten days beginning one hour post challenge

Orphan Drug Designation

Granted 14 July 2017 for the treatment of Ebola virus disease

Ranpirnase is protective against EBOV in mice when administered prophylactically or as post-exposure prophylaxis. Mice that survived after treatment regained lost weight and showed exponentially reduced viral load. High viral clearance and survival were achieved at doses considerably below the scaled mouse equivalent of the safe human dose.

Antiviral effect of ranpirnase against Ebola virus. Hodge et al. Antiviral Research 132:210, 2016. Data generated in collaboration with USAMRIID.

Competitive Position

How ranpirnase compares with current Ebola countermeasures

AttributeRanpirnase (investigational)Monoclonal antibody therapiesVaccines
Primary useTreatment and post-exposure prophylaxisTreatment onlyPrevention only
Strain dependencyPotentially strain-agnostic, mechanism-basedZaire ebolavirus onlyZaire ebolavirus only
Timing of usePre- or post-exposurePost-diagnosisPre-exposure / outbreak control
RouteIntravenous, development-dependentIntravenous infusionIntramuscular injection
Cold chainStandard biologic, expectedRefrigeratedUltra-cold or multi-step
ManufacturingSingle proteinHigh complexity, multiple antibodiesViral-vector production
Stockpiling suitabilityHigh, anticipatedModerateLimited by storage constraints
Role in layered defenceTherapeutic backstopFrontline treatmentPopulation-level prevention

Regulatory Strategy

Evaluating a potential path under the FDA Animal Rule

Okogen is evaluating a potential development pathway under the FDA Animal Rule, subject to FDA agreement on the animal models, efficacy endpoints, exposure bridging, human safety database and CMC requirements. This pathway enables approval without human efficacy trials and algins with U.S. biodefense priorities.