What was the appeal and opportunity you saw that made you want to join the AzurRx team?

I was semi-retired and working on different projects with several companies and I had a chance to look at AzurRx’s data in cystic fibrosis (CF) for the treatment of exocrine pancreatic insufficiency (EPI) and a couple of things appealed to me. First, the data from the company’s two Phase 2 studies was compelling.  Second, I saw that there is significant potential for a non-porcine synthetic product in this setting. Finally, I saw that the company was extremely undervalued and believed that, with proper execution, the only direction for valuation was up.  I have a history of turning companies around, and really enjoy doing it.  I saw the opportunity to do it again with AzurRx.

MS1819, the company’s lead product candidate, definitely has the potential to be a life changing therapy. 

Can you characterize the size of the market AzurRx is targeting?  

Our lead indication is targeting a condition known as EPI which affects about 35,000 CF patients and about 100,000 chronic pancreatitis patients in the U.S. In patients with EPI, the pancreas is damaged and does not produce the digestive enzymes, particularly lipase, needed to break up food in the GI tract so that it can be absorbed. Patients with EPI struggle with a nutritional deficit – they cannot gain or keep weight, which is critical for CF patients. The current standard of care is to replace these enzymes with porcine derived enzymes – a therapy known as PERT (pancreatic enzyme replacement therapy).  With PERT therapy, CF patients with EPI have to take 25-40 pills daily throughout their entire lives. In addition to the high pill burden, PERT therapy has risks.

Our lead product, MS1819, is a yeast derived, synthetic, regenerative oral medication that has the potential to be a substitute for the porcine-derived product. MS1819 has an encouraging safety profile and does not confer the risk of fibrosing colonopathy.  Also, we believe it can dramatically reduce the daily pill burden -- down to 8-16 pills per day.

MS1819, targets both the CF and chronic pancreatitis (CP) communities. The CF market is basically comprised of branded generics, with the main product being Creon from AbbVie, which has around US$1 billion in sales.

The barrier to entry is high and no one has been able to displace Creon and Zenpep. Given our unique profile, we believe that we will be able to add new patients to the mix and create a marketplace for our product. With at least a US$1.2 billion market for just Creon and Zenpep in the U.S., even if we were to capture just a small percentage of it, we see this as a very attractive market.   

CP offers a huge opportunity, especially in Europe and Asia.

What studies does AzurRx need to perform to gain approval from FDA to market the drug?

We are preparing to initiate a Phase IIb program for MS1819 in CF in the second quarter, with data expected by the first quarter of 2021. We then anticipate meeting with the FDA to discuss a Phase 3 trial design. We are preparing to be Phase 3 ready by mid-2021 and, assuming that we only need to do a single study, we hope to launch commercially in CF in late 2022 or early 2023. For CP, we are planning another study to be run in parallel with our CF study and are planning to have a second indication for MS1819 for the treatment of CP in 2024.  

Can you discuss the company’s financial position and the resources available to execute on your plan?

AzurRx has used two financial instruments. First, we established a US$15 million equity line with Lincoln Park Capital, to remove a financing overhang that was weighing on our share price. Second, we raised US$6.9 million in a private placement that, together with the equity purchase agreement, gives us capital until 2021.  Additionally, we have an active business development program aimed at generating potential partnerships that could provide us access to non-dilutive sources of capital. Our efforts are targeting pharmaceutical development companies that are interested in the gastroenterology space and looking to fill their pipelines with a differentiated agent offering attractive margin potential. Because our development program is relatively efficient and inexpensive, it would not be a big reach for a pharmaceutical company to come in and co-develop or partner with us.

What steps is AzurRx taking to show value to the market?

My goal as CEO is to execute on our clinical, regulatory and business development initiatives as seamlessly and in as timely a fashion as possible.  We have a lean organization that is operating with a very clear mission.  We expect to complete our combination study and provide top line results by year end. Our Phase IIb study in CF will begin enrolling patients next quarter, and we anticipate releasing topline results in early 2021.  We have designed both studies to showcase the safety and efficacy of MS1819 and believe that their results will be significant events to unlock value.  Because the combination study is open label, it is possible that we may provide interim updates as the trial progresses. Assuming successful outcomes, we look forward to moving MS1819 into at least one pivotal trial in 2021. As I indicated, we have an active business development program and are constantly evaluating potential partnering opportunities.  Naturally, should we decide to pull the trigger on a collaboration, we would only do so if we believed that it would provide value to both patients and our stakeholders.