CellCept® represents a human therapeutic success story. CellCept (or mycophenolate mofetil, MMF) possesses the unique combination of broad immunomodulating properties, coupled with a well-established safety profile. This impressive clinical profile led CellCept to become one of the first “steroid sparing” agents to reach $1B Blockbuster status. It remains one of the most commonly used therapeutic agents for treating human patients with immune-mediated inflammatory diseases.

Why hasn’t the value of MMF translated over into the veterinary setting? The key to that question, we believe, centers on its suboptimal and clinically limiting “immediate release” pharmacokinetic profile.

People and dogs share the same pathophysiologic inflammatory mechanisms underpinning atopic dermatitis and other inflammatory skin diseases. By extension, the broad immunomodulating properties of MMF have been well established in canines. Based on our experiences studying this medicine in dogs, we believe this therapeutic modality holds significant clinical value for treating dogs with a host of immune-mediated inflammatory diseases.

To unlock the therapeutic potential of the human originator product in canine patients, Okava developed a novel modified-release formulation with clinically meaningful benefits (OKV-1001).

Although the dog is often used as a preclinical species during human drug formulation development, advanced MR drug delivery technologies have not been widely adopted for orally administered companion animal products. OKV-1001 is one of the first modified release formulations ever developed for dogs.

Modified release (MR) oral dosage forms designed to improve the performance of immediate-release formulations (e.g., increase the therapeutic activity compared to the intensity of side effects, reduce the number of drug administrations required during treatment) are commonly developed for human applications. For example, an enteric coated formulation designed to improve the gastrointestinal (GI) tolerability of MPA has been developed.

Immune-mediated hemolytic anemia (IMHA), one of the most common immune-mediated diseases diagnosed in dogs, is a serious and life-threatening condition. It is estimated that up to 50% of dogs diagnosed with IMHA succumb to this disease. In dogs with IMHA, the immune system, which typically fights of invaders and infections, starts to attack its own red blood cells. This results in the red blood cells being destroyed, causing anemia, as well as system inflammation (damage) around your pet’s whole body.

To this day, generic steroid therapy is the most commonly prescribed medicine used to treat IMHA. While steroid therapy may be effective in some patients, the use of steroids are associated with serious adverse events. The lack of safe and effective “steroid sparing” agents represents an area of high unmet medical need. OKV-1001 will address this sizable market segment that is currently limited to chronic steroid use.

Okava was awarded a $500k grant from the FDA to develop OKV-1001 for IMHA. The clinical utility of OKV-1001 will also be investigated in other severe immune-mediated inflammatory diseases such as pemphigus, lupus, and myasthenia gravis.