|CEO of emerging NanoAntibiotics Says Firm is Poised for Growth in Biotech’s Potent Liver Disease Space|
|Thursday, 07 July 2016 07:10|
Jonathan Adams is CEO of NanoAntibiotics, Inc. (OTCQB:NNAB) [nanoantibiotics.com], which will soon change its name to BioVie. He has been working in pharma/biotech since graduating with his MBA from Dartmouth/Tuck in 1989, and earlier in his career assisted with the launch of Celebrex and many other drug therapies. A key member of his team is Cuong Do, who brings decades of industry experience, including as the former head of strategy for Merck and a senior partner with McKinsey & Company.
Q: Your company has taken on the challenge of treating a dangerous disease called ascites and changing the treatment paradigm. What compelled you to take this on?
Adams: We are pioneering CIPT Technology as a potential breakthrough for the treatment of ascites, a life-threatening complication of advanced liver cirrhosis. A few years ago we recognized a compelling opportunity to develop an improved version of a drug called terlipressin for the treatment of ascites. This drug has been used for decades in about 50 countries for related conditions, but is not available in the US. There’s an extensive collection of clinical studies and publications for a variety of medical applications. Having access to these data has helped us to de-risk our clinical development program. We’re passionate about filling this unmet medical as we pursue a revolutionary approach that could allow both outpatient and inpatient treatment. Our goal is to be the first to deliver an FDA-approved treatment for ascites.
Q: Please explain what ascites is and how many people have it?
Adams: Ascites is the accumulation of large amounts of fluid in the abdomen of patients with chronic liver cirrhosis. The occurrence of ascites signals an important transition in disease severity and carries a 40% mortality rate within two years of diagnosis. Without a liver transplant, these desperately ill patients have a miserable quality of life, and may progress to a condition called hepatorenal syndrome (HRS) which can cause death within weeks.
Liver cirrhosis affects millions of people worldwide and accounts for about 27,000 US deaths every year. An estimated 100,000 Americans have ascites due to liver cirrhosis. Given the relatively small patient population we have applied for FDA Orphan Drug designation, which we had previously obtained for a different compound for ascites.
Q: What sets your technology apart from other treatment options?
Adams: The company has pioneered a patent-pending drug delivery system, called CIPT Technology, targeting a new formulation to enable outpatient administration. A recent European study in a related condition (HRS) showed that this delivery technique reduced side effects by about one-half, while achieving efficacy that was similar to the dosing method commonly used around the world. Two small studies in Italy and Australia have indicated the potential for our therapeutic approach to reduce ascites without causing serious side effects. We are leveraging these studies to design our mid-stage US clinical trial to establish proof-of-concept, which we expect to start next year. Establishing the therapeutic proof-of-concept is a major value driver for biotech companies. For example, Synthetic Biologics (NASDAQ:SYN), began a phase 2b proof-of-concept trial for its new drug candidate SYN-004 last fall. If the results are positive, this should really increase the company’s value.
Our approach could yield the first drug to target ascites at its mechanistic source by interrupting the disease pathway, thereby reducing or eliminating the ascites. If successful it would deliver much-needed relief from this life-destroying disease.
Q: This has been an exciting year for biotech companies like Intercept Pharmaceuticals (Nasdaq: ICPT) and Arrowhead Pharmaceuticals (Nasdaq:ARWR) who are in the liver disease space . Can you provide some insight into your strategy for advancing CIP Terlipressin Technology into the clinic? What other future indications will the company target?
Adams: We are leveraging what is already known about terlipressin in ex-US studies and scientific papers as much as possible as a way to de-risk our clinical program. We held a pre-investigational new drug (“pre-IND”) meeting with the FDA earlier this year, and received guidance for submitting our full IND package. If it is accepted, we plan to begin a mid-stage clinical trial in ambulatory ascites patients next year.
Based on terlipressin approvals in other countries, feedback received from leading medical experts, and investigative studies, there are potential follow-on indications for a variety of related medical conditions. One obvious possibility is to treat bleeding esophageal varices (BEV) for which the drug is used worldwide. Another could be hepatorenal syndrome (HRS) with approvals in several countries. Based on various publications there could be applications in the correction of hyponatremia, pre-liver/kidney transplant, septic shock, and other conditions. Terlipressin is a potent vasoconstrictor with a basic mechanism of action which opens the door to these possibilities.
Q: Does the FDA provide incentives such as Orphan Drug or Breakthrough Designations for these indications?
Adams: We are currently seeking Orphan Drug designation, which if granted would provide 7 years of market exclusivity if we are first to market. Obtaining Orphan Drug status can be a catalyst for attracting investment and growth for small biotech companies. For example Anavex Life Sciences (NASDAQ:AVXL) recently won Orphan Drug for its lead candidate ANAVEX 2-73 for infantile spasms, after having secured it for Rett Syndrome, and this helped to boost the company’s value. Additionally, we will apply for Fast-track and Breakthrough designations, which help to expedite the clinical development and review process.
Beyond Orphan Drug exclusivity we are seeking global patent protection, and recently filed for patent protection in the US, Japan, and an international PCT in Europe (a placeholder for future foreign patent filings).
Q: What is the sales potential for CIP Terlipressin Technology?
Adams: Projected US demand for an effective therapy to manage ascites could reach as high as $500 million if FDA marketing approval is achieved. Future global sales revenues could eventually exceed $1 billion including the follow-on opportunities described above. The company is also pursuing patent protection to bolster market exclusivity. All of these factors point to the company having considerable upside potential.