sodium cromoglicate (RVT-1601) / Roivant - LARVOL DELTA

TRANSCATHETER MITRAL VALVE IN VALVE AS A BRIDGE TO MECHANICAL MITRAL VALVE REPLACEMENT IN A PATIENT WITH PREMATURE BIOPROSTHETIC VALVE DEGENERATION AND STENOSIS, SEVERE PULMONARY HYPERTENSION AND SEVERE RIGHT VENTRICULAR DYSFUNCTION (ACC-WCC 2023) - "RHC showed severe pulmonary hypertension (PA 101/42/61 mmHg) from mitral stenosis (mean PCWP 43 mmHg)Decision-making: Options for redo surgical mechanical/bioprosthetic valve replacement vs transcatheter mitral valve in valve were considered by the heart team and was deemed prohibitive risk given the very high risk of hemodynamic collapse and the potential need for ECMO after valve replacement due to severe RV dysfunction... Transcatheter mitral valve in valve intervention can be successfully done to bridge patients to surgical mechanical MVR in patients with severe RV dysfunction and severe pulmonary hypertension."

Clinical • Cardiovascular • Heart Failure • Hypertension • Pulmonary Arterial Hypertension • Pulmonary Disease • Respiratory Diseases

Phase 2b Study of Inhaled RVT-1601 for Chronic Cough in Idiopathic Pulmonary Fibrosis: SCENIC Trial: Multi-Center, Randomized, Placebo-Controlled Study. (PubMed, Am J Respir Crit Care Med) - P2b | "Treatment with inhaled RVT-1601 (10, 40 and 80 mg TID) did not provide benefit over placebo for the treatment of chronic cough in patients with IPF. Clinical trial registration available at www.clinicaltrials.gov, ID: NCT03864328."

Clinical • Journal • P2b data • Chronic Cough • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Infectious Disease • Novel Coronavirus Disease • Pulmonary Disease • Respiratory Diseases

A mutation in DOP1B identified as a probable cause for autosomal recessive Peters anomaly in a consanguineous family. (PubMed, Mol Vis) - "DOP1B that encodes DOP1 leucine zipper like protein B was identified as the putative PA-causing gene in pedigree PA-101...However, this finding does not necessarily implicate a specific role for DOP1B in eye development as the gene is expressed in many tissues. Ultimately, definitive assessment of the contribution of DOP1B to PA pathology awaits identification of mutations in the gene in unrelated patients with PA and functional studies."

Journal • CNS Disorders • Developmental Disorders • Genetic Disorders • Psychiatry

Improved bioavailability of cromolyn sodium using inhaled PA101 delivered via eFlow® nebulizer. (PubMed, Eur Clin Respir J) - "Systemic bioavailability of PA101 was approximately 25% compared to approximately 1% for Nalcrom® and approximately 10% for Intal, respectively. These data warrant further research on the therapeutic potential of PA101 (via eFlow®) in allergic and mast-cell driven diseases."

Journal • Aggressive Systemic Mastocytosis • Asthma • Respiratory Diseases

SCENIC: A Phase 2b Study of Inhaled RVT-1601 for the Treatment of Persistent Cough in IPF (clinicaltrials.gov) - P2b; N=108; Terminated; Sponsor: Respivant Sciences GmbH; N=180 ➔ 108; Trial completion date: Dec 2020 ➔ Jun 2020; Recruiting ➔ Terminated; Trial primary completion date: Oct 2020 ➔ May 2020; COVID-19 Pandemic

Clinical • Enrollment change • Trial completion date • Trial primary completion date • Trial termination • Fibrosis • Idiopathic Pulmonary Fibrosis • Immunology • Respiratory Diseases

Open-label Study of Inhaled RVT-1601 in Preterm Infants (clinicaltrials.gov) - P1; N=8; Completed; Sponsor: Respivant Sciences GmbH; Recruiting ➔ Completed

Clinical • Trial completion

Open-label Study of Inhaled RVT-1601 in Preterm Infants (clinicaltrials.gov) - P1; N=6; Recruiting; Sponsor: Respivant Sciences GmbH; Trial completion date: Mar 2020 ➔ Aug 2020; Trial primary completion date: Feb 2020 ➔ Jun 2020

Clinical • Trial completion date • Trial primary completion date

Respivant Sciences supports international rare disease day through awareness activities (Yahoo Finance) - "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...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."

Enrollment status

Open-label Study of Inhaled RVT-1601 in Preterm Infants (clinicaltrials.gov) - P1; N=6; Recruiting; Sponsor: Respivant Sciences GmbH; Not yet recruiting ➔ Recruiting

Enrollment open

Polymer Microarrays for the Discovery and Optimization of Robust Optical Fibre-based pH Sensors. (PubMed, ACS Comb Sci) - "Polymer microarrays were utilized for the high-throughput screening and discovery of optimal polymeric substrates capable of trapping functional ratiometric fluorescence-based pH sensors. This lead to the identification of poly (methyl methacrylate-co-2-(dimethylamino) ethyl acrylate) (PA101) that allowed, via dip coating, the attachment of fluorescent pH sensors onto the tip of optical fibres that exhibited robust, rapid and reproducible sensing of physiological pH's."

Journal

New funding awards - October 2019 (Duke University School of Medicine) - "Lake Morrison of Pulmonary has received an award from Respivant Sciences, GmbH for a project entitled "Respivant RVT1601-CC-04 SCENIC: Randomized double blind placebo controlled dose ranging efficacy and safety study with inhaled RVT-1601 for the treatment of persistent cough in patients with IPF." Total funding will be $127,541."

Grant

Open-label Study of Inhaled RVT-1601 in Preterm Infants (clinicaltrials.gov) - P1; N=6; Not yet recruiting; Sponsor: Respivant Sciences GmbH

Clinical • New P1 trial

First patient dosed in phase 2b study testing inhaled RVT-1601 for IPF cough (Pulmonary Fibrosis News) - P2b, N=180; SCENIC (NCT03864328); "'The assessment of a new therapy for this condition will be enthusiastically welcomed by both patients and clinicians,' Fernando Martinez, MD, said in a press release."

P2b data

Respivant Sciences announces dosing of first patient in phase 2b trial of RVT-1601 for the treatment of persistent cough due to idiopathic pulmonary fibrosis (PRNewswire) - P2b, N=NA; "'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...'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.'"

P2b data

SCENIC: A Phase 2b Study of Inhaled RVT-1601 for the Treatment of Persistent Cough in IPF (clinicaltrials.gov) - P2b; N=180; Recruiting; Sponsor: Respivant Sciences GmbH; Phase classification: P2 ➔ P2b

Clinical • Phase classification

SCENIC: A Phase 2b Study of Inhaled RVT-1601 for the Treatment of Persistent Cough in IPF (clinicaltrials.gov) - P2; N=180; Recruiting; Sponsor: Respivant Sciences GmbH

Clinical • New P2 trial • New P2b trial