Renovion Announces Positive Top-Line Results from Phase 2 CLIMB Study of ARINA-1 (RVN-301) in Patients with Non-Cystic Fibrosis Bronchiectasis
The CLIMB study was a randomized double-blind placebo-controlled study to assess the safety and efficacy of ARINA-1 in NCFBE.
Renovion Inc., a biopharmaceutical company focused on developing therapies to restore lung health in individuals with mucus-related lung diseases, announced topline results of the Phase 2 CLIMB study of ARINA-1 in individuals with non-CF bronchiectasis (NCFBE). ARINA-1 is a novel nebulized therapy in development for reducing mucus symptoms in individuals with NCFBE, chronic bronchitis, lung transplant, and other respiratory diseases with chronic mucus symptoms.
The CLIMB study was a randomized double-blind placebo-controlled study to assess the safety and efficacy of ARINA-1 in NCFBE. The trial was conducted at nine sites across the US. Participants were randomized (7:3, treatment:placebo), and 40 participants completed the study, with 29 completing in the ARINA-1 arm and 11 in the placebo (0.9% saline) arm. Topline results from the intention-to-treat (ITT) population demonstrate that ARINA-1 is safe and improves quality of life (QOL) in individuals with NCFBE who experience mucus symptoms.
"These results highlight the important role that ARINA-1 could play in the treatment of non-CF bronchiectasis and are extremely encouraging for patients," said Charles Daley, MD, Professor and Chief, Division of Mycobacterial & Respiratory Infections at National Jewish Health in Denver, Colorado and Principal Investigator of the CLIMB trial. "Today, many [NCFBE] patients suffer from chronic cough and sputum production which affects their QOL and may eventually lead to exacerbations, with no approved pharmaceutical therapies available. There is an urgent need for new effective therapies that can break the vicious cycle of excess mucus, inflammation, and infection for these patients."
ARINA-1 was well-tolerated in individuals with NCFBE with mucus symptoms. The most common treatment-related AEs (TRAEs) (event rates (95%CI)) in the ARINA-1 arm (n=29) were cough (0.3, 95%CI 0.1-0.5), dyspnea (0.2, 95%CI 0.1-0.4), and hemoptysis (0.1, 95%CI 0.0-0.3), which are all commonly associated with nebulized therapies and an underlying diagnosis of NCFBE.
Topline ITT efficacy results from the trial demonstrated that ARINA-1 improved quality of life in individuals with NCFBE at day 28. The mean change in SGRQ (MCID, -4) in the ARINA-1 arm from baseline to day 28 exceeded the MCID for the SGRQ in the total score (-8.5) and all three domains (symptoms, -9.6; activity, -8.5; impact, -7.9). This improvement persisted in the total score persisted to day 56 (-6.1), 28 days after stopping therapy. This demonstrates that ARINA-1 not only improved quality of life during treatment, but that it also had a durable effect in these patients after stopping ARINA-1. The placebo group did not achieve an MCID in the total score or any domain of the SGRQ at days 28 (total score, -2.6) or 56 (total score, -1.4). Consistent with the SGRQ, the CAAT (MCID, -2), which is a validated assessment specific for mucus and cough symptoms, also demonstrated MCID improvements in mean change from baseline to day 28 (-3.7). The placebo group did not reach MCID for the CAAT questionnaire (day 28: -1.3).
The topline exploratory analysis of sputum data indicated that ARINA-1 demonstrated improved mucus viscosity and sputum neutrophil elastase (NE) compared to placebo. Further analyses on the mechanism of action and biomarker data are ongoing.
"ARINA-1’s novel mechanism improved quality of life and also improved mucus biomarkers in people with bronchiectasis," said Carolyn Durham, PhD, Chief Scientific Officer of Renovion. " The quality of life data confirms that improving mucus biomarkers, such as viscosity and NE, can reduce symptoms and also improve patient outcomes in this underserved patient population. These data provide a strong rationale for the continued development of ARINA-1 for NCFBE patients, as well as other populations that suffer from mucus symptoms. We look forward to further analyzing the data and discussing next steps with regulatory authorities."
"These results show that ARINA-1 can have a profound benefit for patients. There are currently no approved therapies for bronchiectasis, and to see such a clear impact on symptom reduction with our benign safety profile positions ARINA-1 as an excellent first line therapy for patients. Better SGRQ scores are associated with better outcomes, and we believe that a similar benefit will be seen in chronic bronchitis patients via a study that is already underway, as well as in COPD and asthma patients with mucus," stated Dan Copeland, CEO of Renovion. "Millions of patients with these diseases face the cycle of mucus, inflammation and infection, and we can change the course of disease for patients facing the daily challenges of mucus."
Renovion will discuss study results at the American Thoracic Society Meeting (May 17-22) and present findings at the World Bronchiectasis Conference (July 4-6) in Dundee, Scotland.
About CLIMB
CLIMB (RVN-301) was a randomized, double-blind, placebo-controlled, multi-center, US Phase 2 study to assess the safety, efficacy, and tolerability of ARINA-1 administered twice daily for 28 days in patients with non-cystic fibrosis bronchiectasis (NCFBE). CLIMB was conducted at 9 sites with 40 adult patients diagnosed with NCFBE who indicated that they had mucus symptoms at the time of screening. Patients were randomized 7:3 to receive either ARINA-1 or matching placebo. The primary endpoint was safety at day 28. Secondary endpoints included quality of life measurements at days 28 and 56, and sputum viscosity and neutrophil elastase were measured at day 28.
About Non-Cystic Fibrosis Bronchiectasis (NCFBE)
NCFBE is a severe, chronic inflammatory airways disease characterized by a chronic cycle of mucus, inflammation, and infection that causes permanent bronchial dilation. Most individuals with NCFBE experience severe and life-altering mucus symptoms including shortness of breath, productive and chronic cough, and daily activity impairment, as well as frequent exacerbations that require aggressive antibiotic therapy and / or hospitalization. Individuals experience progressive health and clinical decline. To date, there are no approved treatments that directly target mucociliary clearance or mucus symptoms. There are approximately 500,000 individuals living with NCFBE in the US today.
About Renovion, Inc.
Renovion is a clinical-stage pharmaceutical company focused on developing ARINA-1, novel nebulized therapy delivered to the lungs to treat pulmonary diseases. ARINA-1 clears mucus, reduces damaging inflammation, and has antibacterial properties that have been shown to restore lung health. Renovion is also conducting clinical trials for ARINA-1 for the prevention of Bronchiolitis Obliterans Syndrome (BOS) progression in lung transplant patients in an ongoing Phase 3 trial with a primary endpoint of FEV1 improvement compared to standard of care. Renovion is also conducting an investigational study in patients with chronic bronchitis. The Renovion team has in-depth experience in drug development with extensive scientific expertise in chronic pulmonary conditions and inflammation. For more information about Renovion, visit https://www.renovion.com/ or follow Renovion on LinkedIn (Renovion, Inc.) and Twitter (@RenovionInc).
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