NEW HAVEN, Conn.--(BUSINESS WIRE)--Karos Pharmaceuticals, Inc., a privately held biopharmaceutical company developing therapeutic agents to modulate peripheral serotonin synthesis to treat orphan indications, including pulmonary arterial hypertension (PAH) and carcinoid syndrome, announces several major achievements, including the success of the Phase 1 clinical trials of KAR5585, the closing of the Series B round of financing, US issuance of a composition of matter patent for KAR5585, and the appointment of Ed Mascioli, MD, as Chief Executive Officer.
Karos’ lead agent, KAR5585, is a novel, orally available, best in class, small molecule tryptophan hydroxylase-1 (TPH-1) selective inhibitor being developed to treat pulmonary arterial hypertension (PAH) and carcinoid syndrome. In vivo animal models have demonstrated KAR5585’s ability to reduce vascular remodeling and vascular occlusions in a dose-dependent manner when used for treatment or prevention of the disease pathophysiology and to substantially reduce pulmonary arterial hypertension when used alone or synergistically with standard of care therapies for PAH. KAR5585 met all expectations in the single and multiple ascending dose studies, demonstrating excellent safety, tolerability, robust pharmacokinetics and a dose-dependent reduction in serotonin synthesis. The pharmacokinetic data support BID and possibly QD dosing, and the human safety data confirm the safety profile seen preclinically. The KAR5585 Phase 2 studies in PAH and carcinoid are planned for 2017. KAR5585 has Orphan Designation for PAH.
Dr. Lewis J. Rubin, Professor of Medicine, Emeritus at the University of California San Diego and a scientific/clinical advisor to Karos, commented that “despite several available therapeutic options for PAH there remains an unmet need for additional therapies. The data with KAR5585 suggest this compound, targeting a novel pathogenic mechanism, may have the potential to treat PAH and be synergistic with current therapies” Dr. Rubin explained.
“With strong clinical data, newly issued patent claims, and new funding, Karos is well-positioned to advance our lead therapeutic candidate into Phase II clinical trials in patients” commented Ed Mascioli, the newly appointed CEO. Dr. Mascioli comes to Karos with extensive senior executive drug development and venture capital experience, having been CEO at Affinium Pharmaceuticals (sold to Debiopharm), established the Rare Disease Unit at Pfizer, and investing experiences at SV Life Sciences and MPM Capital. He started his career as an academic physician, on the faculty of Harvard Medical School.
Karos Pharmaceuticals is a privately held, clinical-stage biopharmaceutical company advancing innovative medicines for patients living with rare diseases where the pathophysiology is driven by serotonin, which includes fibrotic, inflammatory, and metabolic diseases. Karos is supported by leading life science investor New Leaf Venture Partners. Please visit www.karospharma.com for more information.
Pulmonary arterial hypertension (PAH), a rare, orphan disease affecting at least 25,000 patients in the US, is growing in incidence. PAH causes significant morbidity and early mortality. Patients are often diagnosed between the ages of 30-50 years due to symptoms of fatigue and difficulty breathing. Current treatments do not control symptoms well and do not alter disease progression sufficiently to prevent mortality. In some cases, lung transplants may be required. Seven-year mortality ranges from 32-65% post diagnosis, according to the National Institutes of Health.