# A Reasonable Expectation of Effectiveness  Clinical Trial of OKV-1001 for the Treatment of Primary (Non-Associative) Immune-Mediated Hemolytic Anemia in Dogs

> **NIH FDA R01** · OKAVA PHARMACEUTICALS, INC. · 2020 · $249,657

## Abstract

Immune-mediated hemolytic anemia (IMHA) in dogs represents high unmet medical need.
IMHA is considered to have a poor prognosis in dogs, with mortality rates of ~30%–40%. There
are no FDA approved drugs. Well-controlled, prospective clinical studies for dogs with IMHA are
limited, and leave treatment guidelines for IMHA in dogs poorly defined.
The treatment, and management of symptoms, for canine IMHA is challenging. The mainstay
immunosuppressive drug regimen for IMHA generally includes oral glucocorticoids, but
individualized treatment plans based on response to treatment and severity of the disease are
needed. There is a clinical need for immunosuppressive therapies that are safe and effective in
treating IMHA. There is also a need for well-designed, prospective clinical studies that can be
used to further inform the development of treatment guidelines.
Mycophenolic acid (MPA) is an immune-modulating agent. Preliminary studies in dogs have
demonstrated a potential role for MPA as a corticosteroid-sparing agent and as adjunctive or
primary therapy in IMHA. OKV-1001, a novel extended-release MPA dosage form, is being
developed to address limitations of the available human MPA preparations. The OKV-1001
extended-release formulation is designed to improve upon the existing MPA formulations by
modulating the pharmacokinetic profile. The pharmacokinetic and pharmacodynamic properties
observed in healthy dogs dosed with OKV-1001 are consistent with a once-daily formulation.
The clinical performance of OKV-1001 for the treatment of IMHA will be studied in a
randomized, double-blind, placebo-controlled, multicenter pilot study. This three-month study in
sixty client-owned dogs, comparing the efficacy and safety of OKV-1001 to placebo, is an
appropriate study design to support reasonable expectation of effectiveness of OKV-1001 as an
adjunctive therapy for the treatment of newly-diagnosed idiopathic (non-associative) IMHA in
dogs at least 12 months of age.

## Key facts

- **NIH application ID:** 10086746
- **Project number:** 1R01FD006916-01
- **Recipient organization:** OKAVA PHARMACEUTICALS, INC.
- **Principal Investigator:** ANDERSON WAYNE
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** FDA
- **Fiscal year:** 2020
- **Award amount:** $249,657
- **Award type:** 1
- **Project period:** 2020-06-01 → 2022-05-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/10086746

## Citation

> US National Institutes of Health, RePORTER application 10086746, A Reasonable Expectation of Effectiveness  Clinical Trial of OKV-1001 for the Treatment of Primary (Non-Associative) Immune-Mediated Hemolytic Anemia in Dogs (1R01FD006916-01). Retrieved via AI Analytics 2026-05-24 from https://api.ai-analytics.org/grant/nih/10086746. Licensed CC0.

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