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

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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.