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

犬免疫介导的溶血性贫血（IMHA）代表了高度未满足的医疗需求。 IMHA被认为在犬中预后不良，死亡率约为30%-40%。那里 没有FDA批准的药物。对IMHA犬进行的对照良好的前瞻性临床研究 有限，并且使犬IMHA治疗指南定义不清。 犬IMHA的治疗和症状管理具有挑战性。主体 IMHA的免疫抑制药物方案通常包括口服糖皮质激素，但 基于对治疗的反应和疾病的严重程度的个体化治疗计划， needed.临床上需要安全有效的免疫抑制疗法， 治疗IMHA。还需要设计良好的前瞻性临床研究， 用于进一步为治疗指南的制定提供信息。 霉酚酸（MPA）是一种免疫调节剂。对狗的初步研究表明， 已证明MPA作为皮质类固醇节约剂和预防或 IMHA的主要治疗。OKV-1001是一种新型的MPA缓释剂型， 开发以解决可用的人MPA制剂的局限性。OKV-1001 延长释放制剂被设计为通过以下方式改进现有的MPA制剂： 调节药代动力学特征。药代动力学和药效学特性 在用OKV-1001给药的健康狗中观察到的结果与每日一次制剂一致。 OKV-1001治疗IMHA的临床性能将在一项研究中进行研究。 随机、双盲、安慰剂对照、多中心初步研究。这项为期三个月的研究， 60只客户拥有的狗，比较OKV-1001与安慰剂的疗效和安全性， 适当的研究设计，以支持合理预期OKV-1001作为 新诊断的特发性（非关联性）IMHA的连续治疗 至少12个月大的狗。

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.