Kira Pharmaceuticals today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to KP104 for the treatment of Paroxysmal Nocturnal Hemoglobinuria.
| CAMBRIDGE, Mass. and SUZHOU, China, July 28, 2022 /PRNewswire/ -- Kira Pharmaceuticals, a global clinical-stage biotechnology company pioneering transformational complement therapies to treat immune-mediated diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to KP104 for the treatment of Paroxysmal Nocturnal Hemoglobinuria (PNH).
PNH is a rare, life-threatening blood disease that arises due to hyperactivity of the complement system. An intricate constellation of protein pathways, the complement system is a key component of innate immunity. Aberrant activity within this system can be a driver of diseases such as autoimmune and inflammatory conditions. KP104 is a bifunctional, first-in-class biologic with a unique dual-approach mechanism of action. Designed to selectively block the alternative and terminal pathways, KP104 provides a powerful and synergistic method of targeting validated drivers of disease in the complement system. KP104 has also been engineered to have an extended half-life and potency, with a formulation that can be used for both IV and subcutaneous administration. “Receiving Orphan Drug Designation is a key milestone for Kira, validating KP104’s differentiated potential to provide a safe and effective treatment for PNH patients,” said Frederick Beddingfield, MD, PhD, CEO at Kira Pharmaceuticals. “As a bifunctional complement therapy targeting both the alternative and terminal pathways, KP104 has the potential to treat complement-mediated diseases where single-target therapies are not adequate. As we enter Phase 2 across multiple indications, we look forward to continued clinical evaluation of KP104 for patients in need.” The FDA’s Orphan Drug Designation program provides orphan status to drugs defined as those intended for the treatment, diagnosis, or prevention of rare diseases that affect fewer than 200,000 people in the United States. KP104 has completed a Phase 1 first-in-human (FIH) study and is entering Phase 2 trials across multiple indications. The Phase 1 clinical trial was a randomized, double blind, placebo-controlled study designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of escalating single and multiple doses of KP104 in healthy volunteers. Kira plans to present this Phase 1 data at a medical conference later this year. About KP104 About Paroxysmal Nocturnal Hemoglobinuria About Kira Pharmaceuticals
SOURCE Kira Pharmaceuticals |
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