Taksta (fusidic acid) / Aceragen - LARVOL DELTA

Oral ARV-1801 Given in Combination With Intravenous Ceftazidime or Meropenem for Treatment of Melioidosis in Hospitalized Patients (clinicaltrials.gov) - P2 | N=125 | Completed | Sponsor: Arrevus Inc. | Recruiting ➔ Completed

Combination therapy • Trial completion

Oral ARV-1801 Given in Combination With Intravenous Ceftazidime or Meropenem for Treatment of Melioidosis in Hospitalized Patients (clinicaltrials.gov) - P2 | N=100 | Recruiting | Sponsor: Arrevus Inc. | Trial completion date: Jan 2023 ➔ Nov 2023 | Trial primary completion date: Jan 2023 ➔ Sep 2023

Combination therapy • Trial completion date • Trial primary completion date

Study to Determine the Efficacy&Safety of ARV-1801(ACG-701) for the Treatment of Cystic Fibrosis Pulmonary Exacerbations (clinicaltrials.gov) - P2 | N=0 | Withdrawn | Sponsor: Aceragen | N=80 ➔ 0 | Recruiting ➔ Withdrawn

Enrollment change • Trial withdrawal • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Study to Determine the Efficacy&Safety of ARV-1801(ACG-701) for the Treatment of Cystic Fibrosis Pulmonary Exacerbations (clinicaltrials.gov) - P2 | N=80 | Recruiting | Sponsor: Aceragen | Not yet recruiting ➔ Recruiting

Combination therapy • Enrollment open • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Aceragen Announces Recommendation of Data Monitoring Committee in Terra Study (GlobeNewswire) - "Aceragen...today announced the recommendation of its independent Data Monitoring Committee (DMC) in regard to the on-going TERRA Phase 2 study of ACG-701 in patients with melioidosis. The DMC is responsible for providing oversight of safety and efficacy for this study and has recommended the TERRA study continue without modification....We are very pleased that the DMC recommended that the trial continue. We look forward to delivering the data from the study later this year,'...continues to actively enroll patients with a data read-out expected in the fourth quarter of 2023."

DSMB • P2 data • Infectious Disease

Idera Pharmaceuticals Announces Name Change to Aceragen, Inc. and Provides Near-Term Strategic Outlook (GlobeNewswire) - "A Phase 2 trial of ACG-701 in CF PEx (the REPRIEVE study), a randomized double-blinded, placebo-controlled study, was initiated in December 2022 at clinical sites in the United States in collaboration with the CF Foundation’s Therapeutic Development Network (TDN)....Data from the REPRIEVE study is expected in 2H 2023.."

P2 data • Cystic Fibrosis

Study to Determine the Efficacy&Safety of ARV-1801/ACG-701 for the Treatment of Cystic Fibrosis Pulmonary Exacerbations (clinicaltrials.gov) - P2 | N=80 | Not yet recruiting | Sponsor: Aceragen

Combination therapy • New P2 trial • Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Pulmonary Disease • Respiratory Diseases

Idera Pharmaceuticals Reports Third Quarter 2022 Financial Results and Provides Corporate Update (GlobeNewswire) - "REPRIEVE Study in Cystic Fibrosis: Randomized, double-blind, placebo-controlled Phase 2 study evaluating treatment with ACG-701, a proprietary oral formulation of sodium fusidate, in newly diagnosed pulmonary exacerbations in CF patient. The REPRIEVE study is on track for initiation by the end of 2022, with data anticipated in Q3 2023."

New P2 trial • P2 data • Cystic Fibrosis

Oral ARV-1801 Given in Combination With Intravenous Ceftazidime or Meropenem for Treatment of Melioidosis in Hospitalized Patients (clinicaltrials.gov) - P2 | N=100 | Recruiting | Sponsor: Arrevus Inc. | Not yet recruiting ➔ Recruiting

Combination therapy • Enrollment open

pH-Dependent Spectrum Expansion of Sodium Fusidate Against Bacterial Biothreats (ASM Microbe 2022) - "To these ends, ACG-701 is a proprietary oral formulation of SF developed by Aceragen for cystic fibrosis and melioidosis...The enhancement of activity at lower pH is of particular interest for combating biothreat agents as the environment of the pulmonary tract (as the primary site of infection during inhalation) and the intracellular lifecycle of some agents necessitates the pathogen may reside in more acidic environments. These results support further development of SF as a useful agent in the biodefense space."

Cystic Fibrosis • Fibrosis • Genetic Disorders • Immunology • Infectious Disease • Pulmonary Disease • Respiratory Diseases

Aceragen Announces $3.5 Million Development Award from Cystic Fibrosis Foundation to Progress Novel Therapy for CF-Related Exacerbations (GlobeNewswire) - "Aceragen...today announced that its wholly-owned subsidiary, Arrevus, Inc., has received an award from the Cystic Fibrosis Foundation for up to $3.5 million to support the clinical development of ACG-701, a proprietary formulation of sodium fusidate, as a treatment for cystic fibrosis-related pulmonary exacerbations. There are currently no FDA-approved therapies for this indication....'We appreciate the generous support of the Cystic Fibrosis Foundation and are looking forward to 2022 as we work closely with the Foundation to initiate clinical development for ACG-701 in CF and multiple other indications,'..."

Financing • New trial • Cystic Fibrosis

Oral ARV-1801 Given in Combination With Intravenous Ceftazidime or Meropenem for Treatment of Melioidosis in Hospitalized Patients (clinicaltrials.gov) - P2; N=100; Not yet recruiting; Sponsor: Arrevus Inc.

Clinical • Combination therapy • New P2 trial

Aceragen Announces Acquisition of Arrevus, Expands Late-Stage Rare Disease Pipeline (GlobeNewswire) - "Aceragen...today announced the acquisition of Arrevus...The transaction adds ARV-1801 (now ACG-721), an oral, small molecule treatment in late-stage development for indications including cystic fibrosis....ACG-721 is intended for the treatment of cystic fibrosis pulmonary exacerbations and other orphan infectious diseases. Phase 2/3 trial for cystic fibrosis pulmonary exacerbations expected to begin in the second half of 2022."

Licensing / partnership • New P2/3 trial • Cystic Fibrosis

Biotech startup Arrevus receives FDA Orphan Drug Designation for cystic fibrosis treatment (WRAL TechWire) - "Arrevus...announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to ARV-1801 for the treatment of cystic fibrosis (CF)....Orphan Drug Designation provides for close guidance by the FDA, potentially accelerating time to marketing approval and also includes the potential for orphan drug grants, tax credits, and seven-year market exclusivity upon marketing approval....Arrevus is planning to initiate a phase 2 study to examine ARV-1801 in the treatment of pulmonary exacerbations in patients with cystic fibrosis."

New P2 trial • Orphan drug

Oral Sodium Fusidate (CEM-102) for the Treatment of Staphylococcal Bone or Joint Infections (clinicaltrials.gov) - P2/3; N=30; Completed; Sponsor: Arrevus Inc.; Active, not recruiting ➔ Completed

Clinical • Trial completion

Combined Sleep Restriction and Circadian Disruption Negatively Alter Bone Turnover Markers in Women (ENDO 2019) - "...We hypothesized that cumulative sleep restriction and concurrent circadian disruption would decrease P1NP levels in women and that the magnitude of decline would be greater in younger compared to older women, as previously reported in men (JCEM 102(10) 2017)...Abstracts presented at a news conference are embargoed until the date and time of the news conference. The Endocrine Society reserves the right to lift the embargo on specific abstracts that are selected for promotion prior to or during ENDO."

Clinical • Late-breaking abstract

Combined Sleep Restriction and Circadian Disruption Negatively Alter Bone Turnover Markers in Women (ENDO 2019) - "...We hypothesized that cumulative sleep restriction and concurrent circadian disruption would decrease P1NP levels in women and that the magnitude of decline would be greater in younger compared to older women, as previously reported in men (JCEM 102(10) 2017)...Abstracts presented at a news conference are embargoed until the date and time of the news conference. The Endocrine Society reserves the right to lift the embargo on specific abstracts that are selected for promotion prior to or during ENDO."

Clinical