SAN DIEGO and BASEL, Switzerland, May 20, 2019 /PRNewswire/ -- Respivant Sciences, a clinical-stage biopharmaceutical company dedicated to developing new therapies for patients with respiratory diseases, today announced it has dosed the first patient in the SCENIC trial, a Phase 2b study of RVT-1601 for the treatment of persistent cough in patients with idiopathic pulmonary fibrosis (IPF). RVT-1601 is a proprietary inhalation formulation of a mast cell stabilizer with pleiotropic immune modulating activity delivered directly to the lungs via a proprietary electronic nebulizer device.
"The majority of people with IPF suffer from a persistent, hard-to-treat cough that can be physically and psychologically debilitating," said Bill Gerhart, CEO of Respivant Sciences. "The need for new medicines that can improve the quality of life of IPF patients is as important to patients as the effort to slow or stop progression of the underlying disease. We're hopeful the results from our SCENIC trial will confirm and build upon the positive results from our previously conducted Phase 2a clinical trial."
IPF is a progressive lung disease with no known cure and a median time to death or transplant of 3 to 5 years. Approximately 130,000 adults in the United States and as many as five million adults worldwide are believed to have IPF1. An estimated 75 percent of people with IPF experience a persistent, hard-to-treat, dry chronic cough, which for many can be the single most debilitating aspect of this disease2. A 2015 FDA report entitled "The Voice of the Patient, Idiopathic Pulmonary Fibrosis" identifies severe coughing as one of the most prevalent and burdensome symptoms of the disease, with patients describing coughing fits that can be prolonged, uncontrollable, and violent, leading to pain, exhaustion and the inability to breathe.
"Cough is a frequent and often devastating symptom for patients suffering from IPF. There are no therapies approved specifically for IPF cough, and therapies otherwise available off-label typically do not provide relief of cough for these patients," said Fernando Martinez, Chief of the Division of Pulmonary and Critical Care Medicine at Weill Cornell Medicine and New York-Presbyterian Weill Cornell Medical Center. "As such, promising new treatments targeting this difficult problem are urgently required. The assessment of a new therapy for this condition will be enthusiastically welcomed by both patients and clinicians."
In a previous Phase 2a clinical trial in 24 patients with IPF cough, RVT-1601 was well tolerated and produced a statistically significant and clinically meaningful reduction in cough frequency3. SCENIC is a 180-patient Phase 2b trial of RVT-1601 being conducted at clinical sites in the U.S., Canada, and Europe that is now enrolling qualifying patients. Three different doses of RVT-1601 will be compared to placebo after 12 weeks of dosing. The objective primary endpoint is the change in 24-hour cough frequency vs. placebo as measured by a cough monitor. Secondary endpoints include patient reported outcomes such as change in cough-related quality of life and cough severity. Following the double-blind treatment period, all enrolled patients will participate in a 12-week open-label treatment period on RVT-1601. For more information, please visit www.clinicaltrials.gov.
Respivant also announced the launch of a new website – IPFCough.com – designed to be a resource for IPF patients and their caregivers, as well as to increase awareness of the impact of cough on IPF patients. IPF patients naturally experience stress and anxiety about their terminal diagnosis, but many patients are as distressed about the impact their unrelenting cough has on their lives. IPFCough.com will be a valuable resource for IPF patients seeking information, support, and help with their cough. For more information, please visit www.IPFCough.com.
About IPF Cough
Approximately 75 percent of people with idiopathic pulmonary fibrosis experience a persistent, hard-to-treat, dry chronic cough, which for many can be the single most debilitating aspect of this disease. For those with IPF, their unrelenting persistent cough can lead to embarrassment, depression, social isolation and an overall decline in their quality of life. IPF cough can also lead to shortness of breath, exhaustion, chest pain, migraines, and other debilitating physical conditions. While there are two approved drugs that have the demonstrated potential to slow progression of IPF, neither drug reduces cough nor otherwise improves quality of life.
About RVT-1601 and PARI eFlow
RVT-1601 is a proprietary inhaled formulation of a mast cell stabilizer with pleiotropic immune modulating activity delivered directly to the lungs via a proprietary electronic nebulizer device, the investigational PARI eFlow®. Delivery of RVT-1601 directly to the lungs of pulmonary fibrosis patients may be effective for treating IPF cough as well as potentially the underlying disease. RVT-1601 works by inhibiting inflammatory mediators that contribute to cough and to the progression of fibrosis4.
The PARI eFlow® is a portable, handheld device that produces a soft mist that patients can easily inhale. This delivery system achieves high lung deposition and distribution of RVT-1601, thus offering the potential to optimally reduce chronic cough and the morbidities associated with it. RVT-1601/eFlow is a proprietary closed-system design regulated as a drug/device combination that prevents intentional or unintentional misuse of the drug or device.
SCENIC is a randomized, double-blind, placebo-controlled, dose-ranging Phase 2b trial evaluating the safety and efficacy of inhaled RVT-1601 for the reduction of cough in patients with a confirmed IPF diagnosis. In a Phase 2a clinical trial, RVT-1601 (formerly PA101) was well-tolerated by IPF patients and produced a statistically significant and clinically meaningful reduction in cough frequency. SCENIC is designed to confirm RVT-1601 reduces cough and improves quality of life for IPF patients with this condition, and to select the optimal dose of RVT-1601 for a Phase 3 program.
The objective primary endpoint is change in 24-hour average cough frequency from baseline to end of week 12 of RVT-1601 vs. placebo. Secondary outcome measures include changes in cough severity, assessed using visual analog scale (VAS), and cough-specific quality of life, assessed using the Leicester Cough Questionnaire (LCQ), a 19-item questionnaire designed to measure the impact of cough in three domains: physical, psychological and social.
The potential of RVT-1601 to slow progression of IPF will also be evaluated in SCENIC. Cough is an independent predictor of IPF disease progression5, therefore reducing cough may slow progression of the disease. In addition, positive results in multiple animal models of pulmonary fibrosis demonstrate the potential of the active pharmaceutical ingredient in RVT-1601 to have direct anti-fibrotic activity6.
Recruitment for SCENIC is planned in the U.S., Canada, and Europe. Topline data are expected by the end of 2020. For more information, please visit www.clinicaltrials.gov.
Respivant Sciences, a wholly owned subsidiary of Roivant Sciences, is a clinical-stage biopharmaceutical company dedicated to developing new therapies for patients with respiratory diseases. Respivant leverages the Roivant platform to develop therapies that address high unmet medical needs while driving greater efficiency in research, clinical development, and commercialization. The company's lead candidate, RVT-1601, is an inhaled mast cell stabilizer with pleiotropic immune modulating activity delivered directly to the lungs through a proprietary electronic nebulizer device (eFlow) for the treatment of respiratory diseases. Respivant is currently conducting a Phase 2b study of RVT-1601 to assess its potential to reduce IPF cough and also to slow progression of the underlying IPF disease. For more information, please visit www.respivant.com.
Roivant Sciences aims to improve health by rapidly delivering innovative medicines and technologies to patients. It does this by building Vants – nimble, entrepreneurial biotech and healthcare technology companies with a unique approach to sourcing talent, aligning incentives, and deploying technology to drive greater efficiency in R&D and commercialization. For more information, please visit www.roivant.com.
1Lederer and Martinez, New Eng Jrnl Med 2018
3Birring S. et al, Lancet Resp Med 2017
4Wygrecka M et al. Am J Pathology 2013; Overed-Sayer, et al 2014; Kawanami, 1979; Rosa & Fantozzi, 2013; Gatti et al, 2006; Jonhson, 2016; Okayama, 1992; Yazid S et al. Plos One 2013; Shimbori C, et al, Thorax 2019
5Ryerson, et. al, Respirology 2011
6Shimbori C, et al, Thorax 2019; Hemmati et al., 2002; Harchegani et al., 2017; Respivant unpublished data
SOURCE Respivant Sciences