AzurRx BioPharma Inc. (NASDAQ: AZRX)

AzurRx BioPharma Inc. (NASDAQ: AZRX) is a clinical-stage biopharmaceutical company focused on developing treatments for gastrointestinal diseases using recombinant proteins.

The company's lead drug candidate is MS1819, a recombinant lipase for the treatment of exocrine pancreatic insufficiency (EPI) in patients suffering from cystic fibrosis and chronic pancreatitis.

AzurRx has already completed two Phase 2 clinical trials for MS1819 and is currently pursuing approval through parallel monotherapy and combination therapy pathways.

The company was founded in 2014 and is headquartered in New York City, with scientific operations in Langlade, France, and clinical operations in Hayward, California.

MS1819 Clinical Trials

The two current ongoing clinical trials for MS1819 in cystic fibrosis (CF) are the Phase 2b Option 2 monotherapy trial and the Phase 2 combination therapy trial, using MS1819 together with porcine pancreatic enzyme replacement therapy (PERT), the current standard of care. Pending the Phase 2b trial outcome, the company intends to initiate a Phase 3 trial in cystic fibrosis.

• Phase 2b CF Option 2 Trial – The study was initiated in Q3 2020, using MS1819 doses in enteric capsule form (2240mg and 4480mg). Topline data for the trial is anticipated in Q1 2021.
• Phase 2 CF Combination Trial – The study was initiated in Q4 2019, using daily dose levels of PERT in combination with MS1819 dosages (700mg, 1120mg and 2240mg). Topline data is anticipated in Q2 2021.

These trials are currently addressing the treatment of EPI in patients with cystic fibrosis and chronic pancreatitis – an established global market with an estimated value in excess of $2 billion that has been growing at a CAGR greater than 20% over the past five years.

Results from AzurRx's Phase 2b Option 2 trial of MS1819 in cystic fibrosis patients demonstrate that the non-porcine MS1819 lipase is well-tolerated by patients, with no significant safety signals observed at the 2240mg daily dose level.

"[W]e have evaluated four different enteric capsules and identified the best suitable formulation for MS1819 that provides gastroprotection of enzyme content and delayed release into the duodenum," James Sapirstein, President & CEO of AzurRx, stated in a September 2020 news release (https://ibn.fm/27t4W). "Our clinical program continues to advance, and we are determined to develop MS1819 as a safer alternative to porcine pancreatic enzyme replacement therapy, significantly reducing the pill burden of cystic fibrosis patients."

Financial Highlights

As of July 2020, AzurRx had raised gross cash capital of $22.1 million, including $15.2 million from Series B convertible preferred stock and warrants in July 2020 and $6.9 million from convertible promissory notes and warrants in December 2019 and January 2020. Notably, AzurRx solidified its financial position and created an effectively debt-free balance sheet by exchanging substantially all of its outstanding convertible notes into the Series B convertible preferred stock financing.

The company secured an additional $2.5 million in French Research Tax Credits, received in 2020, for the years 2017-2019 (https://ibn.fm/Qxk7O).

In a letter to shareholder, Sapirstein noted that ensuring the company maintains sufficient capital to support its business operations has been a key focus. He further stated that the company is in "a financially secure position" to complete its two Phase 2 MS1819 clinical trial programs and to begin preparations in 2021 for a pivotal Phase 3 study.

The company has no current plans to access additional financing, as it believes it has enough cash to fund existing operational and clinical objectives through Q3 2021.

Management Team

James Sapirstein is the President and CEO of AzurRx BioPharma. He was previously the CEO and a board member for ContraVir Pharmaceuticals Inc., which is now known as Hepion Pharmaceuticals Inc. (NASDAQ: HEPA). Mr. Sapirstein has almost 36 years of experience in the pharmaceutical industry, with expertise in drug development and commercialization. He currently serves on the Emerging Companies and Health Section boards of the BIO (Biotechnology Innovation Organization) and is Chairman Emeritus of BioNJ. He earned his Bachelor's degree in Pharmacy from Rutgers University and has an MBA in management from Fairleigh Dickinson University.

Daniel Schneiderman is the Chief Financial Officer of AzurRx. He previously served as the CFO of Biophytis SA and its U.S. subsidiary, Biophytis, Inc., clinical-stage biotechnology companies focused on the development of pharmaceutical candidates for age-related diseases. He was appointed to the AzurRx position in January 2020, bringing to the team over 18 years of experience in capital markets and finance operations. Mr. Schneiderman holds a degree in economics from Tulane University.

James Pennington, M.D., is the Chief Medical Officer of AzurRx. Before joining the team, he was the Chief Medical Officer and Senior Clinical Fellow for 11 years at Anthera Pharmaceuticals. Before becoming a part of the biotech industry, Dr. Pennington was on the Medical Faculty of Harvard Medical School for 10 years. He received his medical degree from Oregon Health & Science University.

Martin Krusin is the Senior Vice President for Corporate Development at AzurRx. He has 20 years of experience in business development, strategic marketing, financing and operations in the health care, financial services and consulting sectors. Before joining AzurRx, he was the VP for Business Development at FluoroPharma Medical Inc. Mr. Krusin received his MBA from Columbia Business School in finance and marketing, an MPhil. in political economy from Oxford University and a BA in international relations from Swarthmore College.

Dinesh Srinivasan, Ph.D., is the Vice President for Translational Research at AzurRx. He has over 15 years of experience leading drug discovery and development in the pharmaceutical industry. He began his career as a post-doctorate fellow at Roche Palo Alto. Dr. Srinivasan received his MSc in Biotechnology from the University of Mumbai, India, and a Ph.D. in Pharmacology and Toxicology from the University of Arizona – Tucson.

Ted Stover is the Product Development Director at AzurRx. He joined the company in 2020 to oversee CMC and Project Management. Before joining AzurRx, he spent 20 years focused on manufacturing operations and analytical method development for all stages of pharmaceutical drug development. Mr. Stover earned his MBA from the University of Florida.