AzurRx BioPharma Completes Initial Cohort Enrollment into Part 1 of its RESERVOIR Phase 2 Clinical Trial of Niclosamide for the Treatment of COVID-19 Gastrointestinal Infections

Rhea-AI Summary

AzurRx BioPharma (NASDAQ: AZRX) announced the completion of enrollment for Part 1 of its Phase 2 RESERVOIR clinical trial assessing FW-1022, an oral formulation of niclosamide for treating COVID-19-related gastrointestinal infections. With approximately 20% of COVID-19 patients experiencing GI symptoms, the trial aims to evaluate FW-1022's safety and efficacy in clearing the SARS-CoV-2 virus from the GI tract. Results from the first cohort will be reviewed by the Data Monitoring Committee before proceeding to Part 2, which focuses on efficacy. Topline results are expected in Q1 2022.

Positive

• Completion of enrollment for Part 1 of the Phase 2 RESERVOIR clinical trial.
• Potential therapeutic solution for COVID-19-related GI infections impacting ~20% of patients.
• Expectation of topline results in Q1 2022, indicating progress in clinical development.

Negative

• Dependence on Data Monitoring Committee's favorable assessment to proceed to Part 2.
• Ongoing COVID-19 infection rates may complicate future developments.

BOCA RATON, Fla., Aug. 12, 2021 (GLOBE NEWSWIRE) -- AzurRx BioPharma, Inc. (“AzurRx” or the “Company”) (NASDAQ: AZRX), a company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases, today announced that it has completed enrollment of the initial cohort into Part 1 of its ongoing RESERVOIR Phase 2 clinical trial evaluating FW-1022 as a treatment for COVID-19-related gastrointestinal (GI) infections. FW-1022 is a proprietary oral tablet formulation of micronized niclosamide for the treatment of COVID-19-related GI infections.

Once all patients in this cohort complete their full two weeks of treatment, the trial’s Data Monitoring Committee (DMC) will review the safety data. Successful DMC review will then trigger enrollment into Part 2 of the trial, focused on demonstrating efficacy and extending safety observations.

James Sapirstein, President and CEO of AzurRx, stated, “Completion of enrollment in the first part of our Phase 2 RESERVOIR clinical trial is an important step in our plan to develop niclosamide as a potential treatment for COVID-19 related GI infection. Despite the availability of vaccines, COVID-19 continues to infect people at an alarming rate due to the emergence of more contagious variants. Ultimately, it is our belief that therapeutic solutions will be needed to truly put an end to this pandemic. We believe that niclosamide could play an important role on this front and we look forward to reporting topline results from the RESERVOIR trial in the first quarter of 2022.”

The RESERVOIR clinical trial is designed as a two-part, two-arm, placebo-controlled Phase 2 study. The trial’s primary objectives are to confirm the safety of FW-1022 in the treatment of patients with COVID-19-related GI infections and to evaluate its efficacy in clearing SARS-CoV-2 (SARS2), the virus that causes COVID-19, from the GI tract. The primary efficacy measure of the RESERVOIR trial is the rate of fecal SARS2 clearance (stool sample) assessed by PCR, comparing the niclosamide arm to the placebo arm for up to six weeks. These long-term observation data could indicate that niclosamide treatment has the potential to improve “long haul” COVID-19 symptoms.

James Pennington, M.D., Chief Medical Officer of AzurRx, commented, “Physicians continue to report that the GI infections caused by COVID-19 remain an underappreciated aspect of the disease, even though these symptoms impact almost 20 percent of COVID-19 patients. We believe our micronized oral niclosamide therapy has the potential to target the SARS2 virus directly in the gastrointestinal tract, where research suggests the virus often hides, replicates and causes illness long after the initial infection.”

About Phase 2 RESERVOIR Clinical Trial
The Phase 2 RESERVOIR clinical trial is a two-part, two-arm, placebo-controlled study examining the safety and efficacy of niclosamide in patients with COVID-19 Gastrointestinal Infection.

The two primary objectives of this trial are to confirm the safety of niclosamide in treatment of patients with COVID-19 GI infection, and to demonstrate efficacy in clearing the SARS-CoV-2 (SARS2) virus from the GI tract. Part 1 of the trial will study 9 to 18 patients with COVID-19 and GI positive stools for SARS2. Patients will be treated for 14 days and observed closely for any signs of safety issues. A Data Monitoring Committee will then review the safety profile and if niclosamide is well-tolerated, the trial will move on to Part 2.

Part 2 will be conducted in outpatients with COVID-19 and PCR positive stools for SARS2. Patients will be randomized to either niclosamide 400 mg tablets, three times a day, or to placebo tablets, three times a day. After 14 days of treatment, patients will be taken off study drugs and remain on study observation for up to 6 weeks. The efficacy endpoint is analyzed by time to clearance from stools of SARS2, comparing the niclosamide arm to the placebo arm. Long term observation will also be important to indicate whether niclosamide treatment might improve ‘long haul’ COVID-19 symptoms.

Additional information about the RESERVOIR trial can be found at www.covidgi.com and on ClinicalTrials.gov.

About COVID-19 Gastrointestinal Infections
Gastrointestinal infection symptoms (severe diarrhea, vomiting and abdominal pain) have been reported in approximately 18% of COVID-19 cases.¹ Of the 33 million individuals who are reported to have contracted COVID-19 in the U.S.,² this would translate into 6 million patients having GI infection. Of the 165 million cases globally, it would translate into almost 30 million patients. Furthermore, approximately 10% of patients who were infected with COVID have persistent symptoms months after their initial diagnosis.³ Approximately 86% of these COVID “long haulers” are reported to have GI infection symptoms, with 60% continuing to have diarrhea months after their initial infection.⁴

There is some evidence to support the view that the GI tract is a possible reservoir for recurrence and fecal spread of the SARS-CoV-2 virus as ACE-2, the entry receptor for COVID-19, is highly expressed on GI cells. There currently is no targeted treatment for COVID-19 GI infections.

About Niclosamide
Niclosamide is a prescription small molecule drug listed as an essential medicine by the World Health Organization (WHO). Niclosamide has been safely used on millions of patients for other clinical indications. In the U.S., niclosamide was approved by the U.S. Food and Drug Administration (FDA) in 1982 for the treatment of intestinal tapeworm infections. In addition to its antihelminthic activity, niclosamide has demonstrated anti-inflammatory and anti-viral properties.

There remains an urgent need to develop new medicines that can be manufactured at large scale quickly to treat COVID-19. Niclosamide was recently identified by the Institut Pasteur Korea as a potent inhibitor of SARS-CoV-2, the virus causing COVID-19, with potency 40X greater than remdesivir.⁵ Additionally, emerging evidence confirms the severe GI-related complications of COVID-19 and potential fecal spread of the virus. The Company’s clinical trials may establish that patients treated with an oral and non-systemic niclosamide formulation that delivers high local GI concentrations have decreased viral load and GI-associated symptoms of COVID-19. Importantly, the manufacturing process for niclosamide can be scaled up to supply large populations quickly.

About AzurRx BioPharma, Inc.
AzurRx BioPharma, Inc. (NASDAQ: AZRX) is a clinical stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases. The Company has a pipeline of two gut-restricted GI assets in three clinical indications. The lead therapeutic candidate is MS1819, a recombinant lipase for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis and chronic pancreatitis. AzurRx is also advancing two clinical programs using proprietary formulations of niclosamide, a small molecule with anti-viral and anti-inflammatory properties: FW-1022, for COVID-19 gastrointestinal infections and FW-420, for Grade 1 and Grade 2 Immune Checkpoint Inhibitor-associated colitis and diarrhea in advanced oncology patients. The Company is headquartered in Boca Raton, Florida with clinical operations in Hayward, California. For more information visit www.azurrx.com.

Forward-Looking Statement
This press release may contain certain statements relating to future results which are forward-looking statements. It is possible that the Company’s actual results and financial condition may differ, possibly materially, from the anticipated results and financial condition indicated in these forward-looking statements, depending on factors including whether results obtained in preclinical and nonclinical studies and clinical trials will be indicative of results obtained in future clinical trials; whether preliminary or interim results from a clinical trial will be indicative of the final results of the trial; the size of the potential markets for the Company’s drug candidates and its ability to service those markets; and the Company’s current and future capital requirements and its ability to raise additional funds to satisfy its capital needs. Additional information concerning the Company and its business, including a discussion of factors that could materially affect the Company’s financial results are contained in the Company’s Annual Report on Form 10-K for the year ended December 31, 2020 under the heading “Risk Factors,” as well as the Company’s subsequent filings with the Securities and Exchange Commission. All forward-looking statements included in this press release are made only as of the date of this press release, and we do not undertake any obligation to publicly update or correct any forward-looking statements to reflect events or circumstances that subsequently occur or of which we hereafter become aware.

For more information:
AzurRx BioPharma, Inc.
777 Yamato Road, Suite 502
Boca Raton, FL 33431
Phone: (561) 589-7020
info@azurrx.com

Media contact:
Tiberend Strategic Advisors, Inc.
Johanna Bennett / David Schemelia
(212) 375-2665 / (609) 468-9325
jbennett@tiberend.com / dschemelia@tiberend.com

References:
¹Gut Journal: Vol 69, Issue 6: 2020; JAMA Network: Vol 3, Issue 6: 2020; Lancet Gastroenterol Hepatol: Vol 5, Issue 5: 2020; Cheung Gastroenterology: Vol. 159, Issue 1: 2020
² New York Times. (7/1/21) https://www.nytimes.com/interactive/2020/world/coronavirus-maps.html
³ Rubin, R. “As their numbers grow, COVID-19 “Long Haulers” Stump Experts”. https://jamanetwork.com/journals/jama/fullarticle/2771111 September 23, 2020.
⁴ Davis, et al. “Characterizing Long Covid in an International Cohort: 7 Months of Symptoms and their Impact”. https://www.medrxiv.org/content/10.1101/2020.12.24.20248802v2.full.pdf
⁵ Jeon S, Ko M, Lee J, Choi I, Byun SY, Park S, Shum D, Kim S. 2020. Identification of antiviral drug candidates against SARS-CoV-2 from FDA-approved drugs. Antimicrob Agents Chemother 64:e00819-20. https://doi.org/10.1128/AAC.00819-20.

FAQ

What is the purpose of the RESERVOIR Phase 2 clinical trial for AZRX?

The RESERVOIR trial aims to evaluate the safety and efficacy of FW-1022 in treating COVID-19-related gastrointestinal infections.

What results are expected from the Phase 2 RESERVOIR trial?

Topline results regarding safety and efficacy are expected in the first quarter of 2022.

How many patients are involved in the RESERVOIR clinical trial's initial cohort?

The initial cohort of Part 1 includes approximately 9 to 18 patients.

What is FW-1022 and its role in the trial?

FW-1022 is an oral formulation of niclosamide aimed at treating gastrointestinal infections linked to COVID-19.

What dosage is being tested in the RESERVOIR trial's Part 2?

Part 2 will test niclosamide at a dosage of 400 mg, taken three times a day.