Problem to be Solved: Sickle cell disease (SCD) is a global health issue that affects approximately 100,00 Americans and over 13 million people worldwide. As a chronic and multifactorial disease, it is characterized by a persistent milieu of oxidative stress, inflammation, recurrent and painful episodes of sickle crisis, and chronic vasculopathy. The disease places significant financial stress on the United States Healthcare system, with annual costs exceeding $1.1 billion. Each year in the U.S., there are approximately 200,000 emergency room visits by patients seeking treatment for pain crisis and other disease related complications. There remains an unmet need for new interventions that ameliorate chronic vasculopathy and reduce the frequency and severity of pain crises. Product and Long-term Goal: PHD-14 is a small molecule with an excellent safety profile in humans and is being repurposed by Nanometics (d.b.a. PHD Biosciences [PHD]) as a chronic oral therapeutic to improve vascular flow, reduce the frequency of pain crises, and ameliorate organ damage in SCD patients. Technological Innovation: PHD-14 can act by several mechanisms to improve vascular flow and preliminary studies have demonstrated that a single intraperitoneal (i.p.) dose causes statistically significant improvements in the hemodynamic parameters of Berkeley (BERK) mice under ambient conditions and hypoxia / reoxygenation challenged NY1DD mice. Phase I Objectives: This Phase I SBIR project aims to demonstrate the feasibility of oral doses of PHD-14 as a safe and effective therapeutic in BERK. Phase II studies will be guided by feedback from a pre- investigational new drug meeting with the Food and Drug Administration. Commercial Opportunity: Treatment options for SCD are limited and there remains a significant need for new drugs to improve patient outcomes. As infant death rates decrease in developing countries, the target market is expected to rise dramatically over the next 20 years.

Public Health Relevance

CSR To whom it may concern: Application title: A repurposed molecule as an oral therapeutic to improve vascular flow in sickle cell disease. Opportunity Title: PHS 2017-02 Omnibus Solicitation of the NIH, CDC, and FDA for Small Business Innovation Research Grant Applications (Parent SBIR [R43/R44]) Phase I SBIR Research Grant Applications Please assign this application to the following: National Heart Lung and Blood Institute (NHLBI) Small Business: Hematology ZRG1 VH-F (10) Hematology Small Business Activities [SBIR/STTR] Special Emphasis Panels This proposal aims to further the development of PHD-14 as a repurposed oral therapeutic for sickle cell disease (SCD). PHD-14 has an impressive safety record in all human clinical studies and preliminary results have demonstrated that a single intraperitoneal (i.p.) dose improved the hemodynamic properties in two rodent models of SCD. This Phase I SBIR proposal aims to demonstrate the feasibility of PHD-14 as an oral therapeutic for SCD using the Berkeley mouse model. Please make secondary assignment to NCATS. Please make tertiary assignment to NIDDK.

National Institute of Health (NIH)
National Heart, Lung, and Blood Institute (NHLBI)
Small Business Innovation Research Grants (SBIR) - Phase I (R43)
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Special Emphasis Panel (ZRG1)
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Ochocinska, Margaret J
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Nanometics, LLC
New York
United States
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