GeNO LLC a privately held, advanced development-stage technology company announced that it has submitted an Investigational New Drug (IND) application to the US Food and Drug Administration (FDA) for the GeNO NITROsyl (Inhaled Nitric Oxide) System for the treatment of pulmonary hypertension in patients with pulmonary arterial hypertension (PAH) and in patients with pulmonary hypertension secondary to idiopathic pulmonary fibrosis (PH-IPF). If accepted, the IND will enable GeNO to begin a Phase 2 dose escalation study in which both patient populations will receive up to a maximum of 80 ppm NO delivered for up to 150 minutes with its stand-alone NITROsyl platform.

Pulmonary arterial hypertension (PAH) is a rare progressive disorder that carries a poor prognosis and is associated with significant morbidity and mortality, having an historical survival rate rarely exceeding five years.

In contrast to PAH, no medications are currently approved for treatment of pulmonary hypertension (PH) associated with idiopathic pulmonary fibrosis (IPF). IPF is characterized by progressive fibrosis in the lungs with a median survival time from diagnosis of two to five years and a five-year survival rate of approximately 20%. There is a significant unmet medical need in this patient population for a potent and selective pulmonary vasodilator that can reduce pulmonary arterial pressure (PAP) and pulmonary vascular resistance (PVR) and improve oxygenation.

"The submission of this IND is a major milestone for the company," said GeNO's founder and president, David Fine, Ph.D. "We have worked hard to develop an approach of delivering inhaled Nitric Oxide therapy as an efficient and effective treatment to chronic illness."

Following the dose escalation study, it is planned to study these two populations for 12 weeks in a multicenter, randomized, double-blind, placebo-controlled study using the new GeNO ambulatory NITROsyl platform, which will allow for 24-7 inhalation of nitric oxide by means of a nasal cannula. The ambulatory platform uses no gas bottles and instead uses liquid nitrogen dioxide, which is then vaporized and reduced to NO prior to inhalation.

"Nitric oxide has substantial therapeutic potential in diseases involving the pulmonary vascular system. Its use in patients with PAH is well described in the literature, and has been shown to acutely improve conditions associated with PH in humans, due to its selective pulmonary vasodilatory effects," said Robert F. Roscigno, Vice President. "The results from the dose escalation study will help provide the information required to finalize the design of the first study of the ambulatory GeNO NITROsyl System that will allow continuous administration of nitric oxide in an ambulatory setting. This information will guide the choice of the starting dose, response-guided titration steps, and identify the optimal dose."

"While IPF and PAH are different, we plan to focus on the one common feature, namely the pulmonary hypertension, which is a determinant of survival and at least an element or cause of symptoms. Part of the rationale in fact, is built on this point: there are no treatments for IPF, so we have decided to focus on the main consequence of IPF that is potentially treatable and that contributes to symptoms and survival," said Dr. Lewis J. Rubin, M.D., Emeritus Professor of Medicine at the University of California in San Diego, School of Medicine and its emeritus director of Pulmonary and Critical Care and a medical advisor to GeNO. "The Phase 2 program will allow us to assess whether inhaled NO improves PH in both populations, whether there are safety concerns in either group, and whether the profile of effect is common. Thus, this is the first study to evaluate and compare the effects of a treatment in two distinct forms of PH, although they share a common rationale."

Source: GeNO LLC