While Medgenics charges ahead with regulatory approval for its stand-alone, patient friendly device, competitors are scrambling to fill the need for PEG-interferon and ribavirin therapy, the current standard known all too painfully to patients of the hepatitis virus. Alternatives are becoming a motley bag of therapies, some good, some not so good. The race is on for an effective treatment that can improve patient compliance and reduce side effects.
Earlier this year it was reported that hepatitis C virus (HCV)-related deaths in the US occur more frequently that those due to HIV. The World Health Organization finds that HCV, heretofore largely neglected in the European Union countries, now infects 9 million people there and shows a very high co-morbidity with HIV. Highly aggressive hepatitis D virus, for which Medgenics has been given Orphan Drug status, increases the risk of liver-related deaths and overall mortality in HIV patients in Europe, where it is more widespread than here. HCV has gained notoriety as the world's most common blood-borne illness infecting between 1% and 2% of Americans. Frightening statistics show that up to 20% of sufferers will develop cirrhosis; there has been a 300% increase in those requiring liver transplant in the last 10 years; and 47% with HCV go on to develop liver cancer with a very high fatality rate. Other types of hepatitis are just as, if not more, potent and dangerous.
Medgenics' object is to replace noxious, long-term injections of PEG-interferon known for its extremely unpleasant side effects that in many cases lead to patient non-compliance, reversing interferon's benefits toward sero-conversion. With an implanted INFRADURE(TM) delivering the interferon protein that is produced by the patient's tissue, assuming results are comparable to PEG-interferon, compliance problems would likely fade.
The technology involves a simple medical procedure: needle biopsy extracts a sample of skin roughly half the size of a toothpick; there is a 7-10 day processing time to transfer the proper protein-producing gene through a viral vector and initiate manufacturing. The newly-created biopump is grafted back into the abdomen to heal in place. More or fewer Biopumps can be implanted as needed, adjusted to the patient's serum protein levels until optimal. So far, a single pump in one early clinical trial patient has produced reliable, safe results for 36 months. Medgenics' highly-scalable processing plants exist in the US and in Israel where the cost of making a biopump is expected to be low.
At this year's assembly of hepatologists at the American Association for the Study of Liver Disease (AASLD), a scientific gathering devoted specifically to problems of the liver, a number of Medgenics' competitors presented potential treatments for HCV. Medgenics reported that renowned experts at the conference affirmed the potential of INFRADURE in the treatment of hepatitis B and D.
Patients' dislike for interferon, the only available treatment for HCV, has led to many attempts at alternatives, the most desirable being a tablet made out of the NS5B protein inhibitor, albeit with varied results. Gilead Sciences (NASDAQ:GILD) appears to be the frontrunner with a sofosbuvir/ribavirin combination in Phase III. Results presented at the Liver Meeting showed 93% and 61% of clinical trial subjects who would or could not use interferon had a prolonged positive response in HCV genotype 2 and 3, respectively. Gilead is expected to approach the FDA with permission to test their new drug sometime in 2013. Sofosbuvir is also being tested in a Phase II trial with Bristol-Myers Squibb Co. (BMY) who is rebounding after withdrawing from trials of an NS5B inhibitor last summer after a patient death.
It should be noted that non-type 1 genotypes, which make up the largest worldwide population of hepatitis virus patients, would benefit most from treatment with INFRADURE(TM) as it is most effective in this sub-population. Also, oral hepatitis drugs do not respond in genotype 1, where PEG-interferon and ribavirin remains the currently approved standard-of-care. Thus, oral therapy for HCV does not pose an extreme competitive threat.
Some hopefuls in the NS5B race, however, have met with an undesirable fate. Last month, BioCryst Pharmaceuticals (NASDAQ:BCRX) withdrew its new drug application for an oral treatment based on an HCV NS5B protein inhibitor from the FDA after worries about toxicity in preclinicals. In other words, the drug killed rats, not a good sign for human testing. Following this, its $101 million, all stock merger with Presidio Pharmaceuticals was stopped, leaving the company with only one mid-phase pipeline compound, for gout, and no choice but to downsize.
Vertex Pharmaceuticals (NASDAQ:VRTX) has had success with INCIVEK, a new combination therapy co-developed with Merck & Co. (MRK) that adds an antiviral, telaprevir to standard interferon/ribavirin therapy but because the body rapidly builds resistance to it, the combo treatment time is lengthened significantly and costs run ridiculously high - up to $85,000 versus $36,000 for interferon and ribavirin alone. Vertex has two non-exclusive deals to test its new HCV regimens for genotype 1 with Johnson & Johnson (JNJ) and GlaxoSmithKline plc (GSK).
Also during the Liver Meeting, Achillion Pharmaceuticals (NASDAQ:ACHN) surprised investors by withholding efficacy data from a drug interaction study of its lead NS5A all-oral combination, placing it well behind Gilead and causing a 12% drop in the shares.
Roche Holding AG (RHHB) had acquired Anadys Pharmaceuticals in 2011 for several hundred million dollars to get setrobuvir, a NS5B inhibitor in human testing, in addition to danoprevir, bought from InterMune (ITMN), a similar class of compound in trials. Roche also licensed mericitabine, another NS5B, from Pharmasset (now part of Gilead).
As you can see, each pharma company has a potentially valuable piece of the entire HCV puzzle. Medgenics' INFRADURE(TM), intended only to replace PEG-interferon injections, stands a good chance of approach from pharmaceutical companies that are testing combinations that still require the standard therapy of PEG-interferon and ribavirin.
In other company news, full patent protection for the EPODURE Biopump has been granted in the US, joining countries Australia, Japan, China, and Korea and bringing the patent portfolio to 36 issued, 81 pending.
Also, Medgenics' third quarter (ending September 30) results included a very modest rise in research and development costs, to $1.8 million, considering the progress made in human clinical trials - earlier this year, two HCV trials were approved to start in Israel and Orphan Drug status was bestowed by the FDA for rare but deadly hepatitis D. Operating expenses were trimmed by $400,000 as consultant fees dropped and ten cents was shaved off net loss from ($0.35) to ($0.25), for a quarterly burn of roughly $3 million. Cash stood at $8.9 million. There is no long-term debt. In sum, Medgenics' clinical and regulatory progress is highly encouraging and, with their participation at the Liver Meeting, they are now more broadly recognized by the medical community at large. About The Analyst: Sharon Di Stefano Sharon di Stefano was one of the first analysts on Wall Street to cover the career of Sol Barer as Chairman and CEO of Celgene Corp. She has spent 20 years as a healthcare analyst, beginning her career at Smith Barney, Harris Upham & Co. specializing in medical devices, pharmaceuticals, healthcare information technology, and biopharmacology. Ms. di Stefano had also served as Senior Venture Officer for the Edison Innovation Fund, implemented through the New Jersey Economic Development Authority that provided funding for early-stage life sciences companies. Ms. di Stefano received a Masters of Science degree, in Business, from Johns Hopkins University in 1986, and a Bachelor of Arts from the University of Delaware in 1984 with a minor in biology.
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