Pipeline

RedHill is building a largely de-risked and advanced-stage clinical development pipeline of proprietary, oral, small molecules, with multiple near-term milestones. The pipeline is primarily focused on the development of drugs for gastrointestinal diseases, infectious diseases and oncology, and includes ongoing collaborations with various arms of the U.S. Government as well as other pharmaceutical companies:

Drug Candidate Pre-Clinical Phase 1 Phase 2 Phase 3 NDA
Opaganib

Opaganib, a proprietary investigational host-directed and potentially broad-acting drug, is a first-in-class, orally administered sphingosine kinase-2 (SPHK2) selective inhibitor with anti-inflammatory, antiviral and anticancer activity, targeting multiple potential indications, including gastrointestinal acute radiation syndrome (GI-ARS), viral infections such as COVID-19, Ebola virus disease and additional viruses as part of pandemic preparedness, several cancers and diabetes and obesity-related disorders.

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Gastrointestinal acute radiation syndrome
U.S. Gov. collaborations

Opaganib is being evaluated as a potential new therapeutic approach to mitigate gastrointestinal acute radiation syndrome (GI-ARS) in collaboration with the NIAID RNCP following the FDA Animal Rule regulatory pathway.

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Acute respiratory distress syndrome (ARDS), including COVID-19 and influenza
Phase 2/3 COVID-19 (ARDS)

Opaganib is being evaluated for its potential to treat acute respiratory distress syndrome (ARDS) caused by viruses such as COVID-19 and influenza. Data from the global Phase 2/3 study in hospitalized patients with severe COVID-19 pneumonia offer a compelling rationale for further investigation into opaganib’s potential in treating virus-induced ARDS, potentially supporting a Phase 2/3 study in this indication.

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Ebola virus
U.S. Gov. collaborations

Opaganib is being evaluated as a potential treatment for Ebola virus disease in collaboration with the U.S. Army and BARDA. Supported by initial funding from the BARDA research and development contract, the collaboration seeks to advance opaganib’s development to combat infections and control Ebola virus disease outbreaks.

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Cholangiocarcinoma
Phase 2 study

Opaganib completed a single-arm Phase 2a clinical study in patients with advanced, unresectable cholangiocarcinoma and has been granted FDA Orphan Drug Designation for this indication.

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Prostate cancer
Phase 2 study

Opaganib is undergoing a Phase 2 study in combination with Bayer’s darolutamide in men with metastatic castration-resistant prostate cancer (mCRPC) and has completed a prior investigator-sponsored Phase 2 study evaluating its safety and efficacy in progressive mCRPC treated with androgen-signaling blockers, abiraterone or enzalutamide.

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RHB-204

RHB-204 is an investigational, proprietary, fixed-dose, orally administered, antibiotic combination therapy targeting Crohn’s disease in Mycobacterium avium subspecies paratuberculosis-positive (MAP+) patients. It is a next-generation formulation of RHB-104, which demonstrated positive results in a Phase 3 Crohn’s disease study, meeting both primary and secondary endpoints. RHB-204 combines the same antimicrobial agents as RHB-104 with potent intracellular, anti-mycobacterial, and anti-inflammatory properties, but with an optimized dosing profile designed to support enhanced tolerability, safety and adherence.

RHB-204 also has potential for the treatment of pulmonary nontuberculous mycobacterial (NTM) disease caused by Mycobacterium avium complex (MAC).

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Crohn’s disease
Phase 2 study planned

An innovative Phase 2 study of RHB-204 is planned in patients with moderate to severe Crohn’s disease who test positive for Mycobacterium avium subspecies paratuberculosis (MAP).

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NTM disease
Phase 3-stage

A previously ongoing U.S. Phase 3 study evaluating RHB-204 for the treatment of pulmonary nontuberculous mycobacterial (NTM) disease caused by Mycobacterium avium complex (MAC) was terminated due to a low patient accrual rate.

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RHB-107

RHB-107 (INN: upamostat) is a first-in-class, once-daily, orally administered serine protease inhibitor targeting multiple potential indications, including viral infections such as COVID-19 & Ebola virus disease, oncology and inflammatory gastrointestinal diseases.

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COVID-19
Phase 2 study

RHB-107 is being evaluated in the ongoing U.S. Government-supported, Phase 2 platform trial (PROTECT study) for early COVID-19 outpatient treatment.

RHB-107 has already completed a positive U.S. Phase 2 study in non-hospitalized symptomatic COVID-19 patients, which evaluated the safety and tolerability profile of RHB-107, along with efficacy signals.

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Ebola virus
U.S. Army collaboration

RHB-107 is being evaluated as a potential treatment for Ebola virus disease in collaboration with the U.S. Army.

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RHB-102

RHB-102 is a patent-protected, proprietary, once-daily, bimodal extended-release, oral tablet formulation of the antiemetic drug ondansetron, a 5-HT3 antagonist, targeting potential indications including oncology support, acute gastroenteritis and gastritis, and IBS-D at two dose strengths, 12mg and 24mg.

RHB-102 is being developed by Hyloris Pharmaceuticals outside of North America. Learn more.

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Gastroenteritis and gastritis
Completed positive U.S. Phase 3 study

RHB-102 (24 mg) successfully completed a randomized, double-blind, placebo-controlled U.S. Phase 3 study meeting the primary outcome measure, demonstrating its safety and efficacy in the treatment of acute gastroenteritis and gastritis.

RHB-102 is being developed by Hyloris Pharmaceuticals outside of North America. Learn more.

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IBS-D
Completed positive U.S. Phase 2 study

RHB-102 (12 mg) successfully completed a positive randomized, double-blind, placebo controlled U.S. Phase 2 study meeting the primary outcome measure, demonstrating its safety and efficacy in the treatment of diarrhea-predominant irritable bowel syndrome (IBS-D).

RHB-102 is being developed by Hyloris Pharmaceuticals outside of North America. Learn more.

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Oncology Support
Comparative bioavailability studies

RHB-102 (24 mg) is in development for the management of chemotherapy and radiotherapy induced nausea and vomiting (also referred to as CINV and RINV).

RHB-102 is being developed by Hyloris Pharmaceuticals outside of North America. Learn more.

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* Estimated timeline/indication in the pipeline is subject to changes in development plans and regulatory requirements/ clarifications, including complementary /additional studies.

** The safety and efficacy of the agents for the indications under investigation have not been established.