First Wave BioPharma Announces Initiation of Phase 2 Trial Investigating Enhanced Adrulipase Formulation in Exocrine Pancreatic Insufficiency in Patients with Cystic Fibrosis

Rhea-AI Summary

First Wave BioPharma (FWBI) announced the initiation of a Phase 2 clinical trial for an enhanced enteric microgranule delivery formulation of adrulipase, targeting exocrine pancreatic insufficiency (EPI) in cystic fibrosis patients. The FDA has reviewed the amended IND with no further comments. This open-label study aims to assess the safety and efficacy of adrulipase in a 12-patient cohort, with primary efficacy assessed through the coefficient of fat absorption (CFA). Patient screening begins in early February 2023, with topline data expected by mid-2023. The new formulation aims to reduce pill burden for patients, which can be as high as 40 capsules a day.

Positive

• Initiation of Phase 2 clinical trial for adrulipase, indicating progress in drug development.
• FDA's lack of further comments on the IND amendment suggests regulatory support.
• New formulation aims to significantly reduce daily pill intake for EPI patients, enhancing treatment adherence.

Negative

• None.

Patient screening to initiate; topline data expected by mid-2023

BOCA RATON, Fla., Jan. 31, 2023 (GLOBE NEWSWIRE) -- First Wave BioPharma, Inc. (NASDAQ:FWBI) (“First Wave BioPharma” or the “Company”), a clinical-stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases, today announced that the Company will initiate its planned Phase 2 clinical trial of an enhanced enteric microgranule delivery formulation for adrulipase for the treatment of exocrine pancreatic insufficiency (EPI) in patients with cystic fibrosis (CF). The U.S. Food and Drug Administration (FDA) has reviewed the Investigational New Drug (IND) amendment and after requesting and receiving a modified protocol, has not provided any further comments in the 60 day period of review.

The Phase 2 multi-center study is designed to investigate the safety, tolerability and efficacy of an enteric microgranule delivery formulation for adrulipase in a titrated dose-escalation study involving an estimated twelve (12) patients. The primary efficacy endpoint is the coefficient of fat absorption (CFA), with secondary endpoints of stool weight, signs and symptoms of malabsorption and coefficient of nitrogen absorption (CNA).

First Wave BioPharma expects to initiate patient screening in early February 2023, with topline data anticipated by mid-2023. Three clinical trial sites in the U.S. will be participating in the trial.

“We are very pleased to announce the initiation of the Phase 2 clinical trial investigating our new formulation of adrulipase as a potential treatment for EPI associated with cystic fibrosis and chronic pancreatitis,” said James Sapirstein, President and CEO of First Wave BioPharma. “Preclinical research has demonstrated the new adrulipase formulation is able to deliver the drug in the intended area of the gastrointestinal tract where it can provide the desired therapeutic effect. This Phase 2 clinical trial is designed to test that capability as a key step in our goal to provide patients with a more effective and convenient therapeutic option for EPI associated with cystic fibrosis and chronic pancreatitis.”

In vitro data suggest the microgranule drug delivery formulation offers improved protection against the acidic pH in the stomach followed by the rapid release of adrulipase in the small intestine where the drug is expected to mix with food and deliver its therapeutic benefit. Additionally, in vitro research indicates the enhanced formulation has the potential to significantly decrease the number of pills a patient would need to take to achieve the desired therapeutic effect. The pill burden for current commercial porcine pancreatic enzyme replacement therapy (PERT) can be as high as 40 capsules per day, creating a substantial challenge for EPI patients.

Phase 2 SPAN Trial Design
The Phase 2 SPAN trial is designed to investigate the safety, tolerability and efficacy of a new enteric microgranule formulation of adrulipase. The SPAN trial is an open-label study that will be conducted at three sites in the U.S. A total of 12 cystic fibrosis patients, 18 years or older are expected to be enrolled. The trial design employs a dose titration strategy. Patients will be screened at baseline to ensure that they have a coefficient of fat absorption (CFA) of at least 80%. Eligible patients will then be switched from their commercial enzyme product to adrulipase. Each patient will be started on a low dose of adrulipase. If the patient is not clinically controlled, the patient will be switched to a medium dose, and if not controlled on this dose, the patient will be advanced to a high dose. The titrations will be carried out over a three week period, after which a CFA will be obtained. End of study CFAs will be compared to the baseline CFAs in a descriptive fashion. A post-treatment safety visit will be conducted one week after completing the treatment period.

About Adrulipase 
Adrulipase is a recombinant lipase enzyme administered as an oral, non-systemic biologic capsule for the treatment of exocrine pancreatic insufficiency (EPI) associated with cystic fibrosis (CF) and chronic pancreatitis (CP). Adrulipase is derived from the Yarrowia lipolytica yeast lipase and is designed to break up fat molecules in the digestive tract of EPI patients so that they can be absorbed as nutrients. EPI is a condition characterized by deficiency of the exocrine pancreatic enzymes, resulting in a patient’s inability to digest food properly, or maldigestion. The deficiency in this enzyme can be responsible for greasy diarrhea, fecal urge and weight loss. There are more than 30,000 patients in the U.S. with EPI caused by cystic fibrosis according to the Cystic Fibrosis Foundation and approximately 90,000 patients in the U.S. with EPI caused by chronic pancreatitis according to the National Pancreas Foundation.

About First Wave BioPharma, Inc.
First Wave BioPharma is a clinical-stage biopharmaceutical company specializing in the development of targeted, non-systemic therapies for gastrointestinal (GI) diseases. The Company is currently advancing a therapeutic development pipeline with multiple clinical stage programs built around its two proprietary technologies – the biologic adrulipase, a recombinant lipase enzyme designed to enable the digestion of fats and other nutrients, and niclosamide, an oral small molecule with anti-inflammatory properties. First Wave is advancing two Phase 2 clinical programs built around adrulipase for the treatment of exocrine pancreatic insufficiency (FW-EPI) in patients with cystic fibrosis (CF) and chronic pancreatitis (CP). In developing adrulipase, First Wave is seeking to provide CF and CP patients with a safe and effective therapy to control EPI that is non-animal derived and offers the potential to dramatically reduce their daily pill burden. The company is also advancing multiple programs involving niclosamide, including FW-UP for ulcerative proctitis and ulcerative proctosigmoiditis, FW-UC for ulcerative colitis, and FW-CD for Crohn’s disease. First Wave BioPharma is headquartered in Boca Raton, Florida. For more information visit www.firstwavebio.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; whether the contemplated offering will be successful and whether the Company will be able to regain and maintain compliance with Nasdaq’s continued listing criteria; the size of the potential markets for the Company’s drug candidates and its ability to service those markets; the effects of the First Wave Bio, Inc. acquisition, the related settlement and their effect on the Company’s business, operating results and financial prospects; 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, 2021 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:
First Wave BioPharma, Inc.
777 Yamato Road, Suite 502
Boca Raton, FL 33431
Phone: (561) 589-7020
info@firstwavebio.com

Media contact:
Tiberend Strategic Advisors, Inc.
David Schemelia
(609) 468-9325
dschemelia@tiberend.com

Bill Borden
(732) 910-1620
bborden@tiberend.com

FAQ

What is the purpose of First Wave BioPharma's Phase 2 trial for adrulipase?

The Phase 2 trial aims to evaluate the safety and efficacy of an enhanced formulation of adrulipase for treating exocrine pancreatic insufficiency (EPI) in cystic fibrosis patients.

When is the topline data expected from the Phase 2 clinical trial?

Topline data from the Phase 2 clinical trial is anticipated by mid-2023.

How many patients will be enrolled in the Phase 2 adrulipase trial?

The Phase 2 trial will enroll an estimated twelve patients.

What are the primary endpoints of the Phase 2 trial for adrulipase?

The primary efficacy endpoint is the coefficient of fat absorption (CFA), with secondary endpoints including stool weight and coefficient of nitrogen absorption (CNA).

What is the significance of the FDA's review of the IND amendment for FWBI?

The FDA's review and absence of comments on the IND amendment indicate regulatory approval and support for the clinical trial process.