Respivant Sciences Supports International Rare Disease Day Through Awareness Activities

Respivant Sciences, a clinical-stage biopharmaceutical company dedicated to developing new therapies for patients with rare respiratory diseases, today announced its support of Rare Disease Day on February 29, 2020 with a series of events meant to observe and recognize advancements in the field of rare diseases and to support and strengthen patient communities. Respivant joins forces with more than 25 million Americans living with a rare disease and health care advocates around the world to elevate public understanding of rare diseases and call attention to the special challenges these people face.

Respivant also announced it has achieved an important milestone in the SCENIC trial, a Phase 2b study of RVT-1601 for the treatment of Idiopathic Pulmonary Fibrosis (IPF). Respivant has enrolled 50% of the total 180 patients targeted for SCENIC. RVT-1601 is an inhaled mast cell stabilizer with pleiotropic immune modulating activity being developed by Respivant for IPF that has received orphan drug designation by the FDA.

"Respivant is committed to making a difference for rare disease communities by supporting innovative drug development that improves the lives of people living with rare diseases, including Idiopathic Pulmonary Fibrosis,” said Bill Gerhart, CEO of Respivant Sciences. “Participation in activities such as Rare Disease Day, by engaging with the communities and the families we aim to serve, helps us deliver on our mission of developing life-changing medicines for people with rare respiratory conditions where few options, if any, exist.”

In support of International Rare Disease Day, Respivant is participating in the following activities:

• Charlene Marshall, who is living with IPF in Toronto, will visit with Respivant employees at their headquarters in San Diego. She will share her personal challenges with IPF and inspirational words relating to patients’ quest for a cure.
• Employees will observe the day by wearing Rare Disease Day pins from Global Genes as well as rubber bracelets from the Breathe Support Network.
• Respivant participated in the NORD Show Your Stripes Challenge, sharing in the creative energy of millions of people around the world with rare diseases — and the millions who care about them — to raise awareness and generate action.
• Respivant sponsored a webinar produced by the Pulmonary Fibrosis Foundation to educate and increase awareness of the often-debilitating impact of cough on IPF patients.
• Respivant attended the Pulmonary Fibrosis Foundation’s 10th annual fund raiser “Belts for Broadway” in New York this week.
• Respivant launched a social media campaign on Twitter, on Facebook, and on LinkedIn to promote awareness of Rare Disease Day generally, as well as IPF specifically.
• For additional activities taking place in the US and abroad, please visit the Rare Disease Day 2020 event pages on the EURORDIS and NORD websites.

About IPF

Idiopathic pulmonary fibrosis (IPF) is a progressive lung disease characterized by scarring (fibrosis) of the lung for unknown (idiopathic) reasons. Chronic inflammation associated with pulmonary fibrosis contributes to disease progression and also hypersensitizes cough receptors. Approximately 75% 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 disease. There are two approved drugs that have the demonstrated potential to slow progression of IPF; however, neither drug reduces cough nor otherwise improves quality of life. For more information, please visit www.ipfcough.com.

About RVT-1601 and the SCENIC Trial

RVT-1601 is an inhaled mast cell stabilizer with pleiotropic immune modulating activity delivered via a hand-held, silent, electronic nebulizer (eFlow®). Delivery of RVT-1601 directly to the lungs of pulmonary fibrosis patients is believed to be the most effective way to treat pulmonary fibrosis. Preclinical animal studies demonstrate the significant role chronic inflammation plays in the progression of fibrosis and the potential for RVT-1601 to be anti-fibrotic. Also, a Phase 2a clinical trial in IPF patients demonstrated the potential for RVT-1601 (formerly PA-101) to significantly reduce cough due to IPF and improve quality of life. Importantly, RVT-1601 was well-tolerated in this study, with a safety profile comparable to placebo.

SCENIC is a large, global Phase 2b clinical trial designed to:

• Confirm the results of the Phase 2a trial
• Select the appropriate dose for a Phase 3 clinical trial
• Evaluate the potential of RVT-1601 to slow progression of the underlying IPF disease

For more information about the SCENIC Trial, please visit www.SCENICtrial.com.

About Respivant

Respivant Sciences, a wholly owned subsidiary of Roivant Sciences, is a clinical-stage biopharmaceutical company dedicated to developing new therapies for patients with rare 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. Respivant is currently conducting a Phase 2b study of RVT-1601 to assess its potential to treat IPF. For more information, please visit www.respivant.com.

About Roivant

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.

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