An inhaled PDGF receptor inhibitor for the treatment of pulmonary arterial hypertension

DESCRIPTION provided by applicant Pulmokine is developing a novel inhaled PDGF receptor PDGFR kinase inhibitor PK as a treatment for Pulmonary Arterial Hypertension PAH The purpose of this Phase IIB Small Market Bridge Award is to progress PK through a phase A clinical trial for safety PAH is an orphan disease with high morbidity and mortality despite currently available treatments Pulmokineandapos s current Phase II SBIR grant was designed to develop the formulation for inhalation of the API determine pharmacokinetics and maximum tolerated dose in the rat These studies have been accomplished and set the stage for IND enabling studies The IND enabling studies will consist of in vivo animal toxicology studies in two species rat and dog as required by the FDA These studies are currently funded separately through a Stage B VITA contract awarded to Pulmokine through the NHLBI The phase IIB small market award will provide the means to perform a phase A first in human exposure clinical trial in healthy volunteers and to perform longer term toxicology studies After completion of the Phase A clinical trial a combined phase B A clinical trial in patients with PAH will be performed with investor funds The primary endpoint of the phase B A trial will be safety with efficacy as a secondary endpoint After completion of the phase B A trial Pulmokine will form a strategic alliance or partnership with a larger pharmaceutical company or undergo acquisition Such an alliance will allow performance of a phase III clinical trial and commercialization of our drug product as a treatment for PAH The part of the project that will be funded by this phase IIB award includes the following Performance of a phase A safety study of dry powder inhaled PK in healthy human volunteers This phase A clinical trial will consist of a blinded single ascending dose SAD placebo controlled protocol followed by a multiple dose ascending MAD placebo controlled protocol All of the key components are in place to perform this first in human clinical trial cGMP manufacturer of of the API IRIX Pharmaceuticals cGMP manufacture of spray dry powder formulation BEND Research Inc the Clinical site for Performance Parexel Phase Unit Performance of a month GLP toxicology study in the rat This study is necessary per ICH M R guidelines before initiation of the longer phase clinical trial ICH guidelines recommend toxicology studies of the same length as the clinical trial for trials of months duration Since the Phase B A trial is planned for six months we will need the month rat GLP tox study PUBLIC HEALTH RELEVANCE Zisman Lawrence Pulmonary arterial hypertension is a devastating disease with a high morbidity and mortality This project is relevant to public health because it will ultimately lead to a new treatment for pulmonary arterial hypertension Public Health Relevance Statement Page