Therapeutic Areas

Developing new hope for patients and families

Pulmonary Arterial Hypertension (PAH) is a degenerative and life-threatening disease that makes it difficult for the heart to pump blood to the lungs to be oxygenated and may ultimately lead to heart failure. Current PAH treatment options only address the symptoms, slowing but not preventing disease progression. New therapies that address the underlying cause of disease are urgently needed.

In January 2017, we acquired exclusive, worldwide rights to develop and commercialize tacrolimus and ascomycin for the treatment of PAH and related vascular diseases.

Pulmonary arterial hypertension is a degenerative disease with current treatment options that only address the symptoms to slow the progression of the disease. We are excited about the potential of tacrolimus and ascomycin to significantly improve the quality of life and life expectancy of PAH patients.

Advancing innovative therapies for PAH

Therapies that address the underlying cause of PAH are urgently needed. The U.S. Food and Drug Administration (FDA) approved tacrolimus in 1994 for use in lowering the risk of organ rejection in patients undergoing kidney transplant, and it is currently indicated for use in additional organ transplant settings and to treat atopic dermatitis.

Researchers completed a randomized, double-blind Phase 2a clinical trial with 23 class 1 and 2 PAH patients titrated to target blood levels. All target blood levels were well tolerated with no drug related serious adverse events, nephrotoxicity or incident diabetes.

In addition, tacrolimus was provided for compassionate use in three class 3 or 4 PAH patients. The compassionate use demonstrated dramatically reduced rates of hospitalizations and functional class improvements were observed. We are currently focused on a proprietary formulation of tacrolimus for clinical development program and for commercial use.

Links and Information

Helpful Links and Resources