Medgenics scores Orphan Drug designation for Infradure Hepatitis D indication...

Press release posted 6/21/12 on Reuters.com. A victory for Medgenics Infradure and their proprietary proprietary tissue-based Biopump platform technology which uses a biopsy of the patients own skin to deliver therapeutic proteins currently delivered via injection. The FDA has designated Infradure an orphan drug for the treatment of Hepatitis D here in the States, while clinical trials are ongoing in Israel for other indications, most notably, for Hepatitis C. This orphan drug designation doesn't ensure the FDA will grant Infradure an expedited review, but clearly that is a probability which could speed Infradure to market for a Hep D indication while trials for the remaining targeted indications carry on. 


CEO Sees FDA's Designation as Significant Validation of Medgenics' Novel Technology for Treatment of hepatitis D

Thu Jun 21, 2012 11:05am EDT

In near record time, Andrew L. Pearlman, Ph.D., President and Chief Executive Officer of Medgenics (AMEX:MDGN) has helped position his relatively new publicly traded company at a much higher level-- one that most other biotech firms usually take far longer to reach.

Earlier this week on Tuesday, his firm raised $9.5 million via a Private Placement and twenty-four hours later, he announced that Medgenics' INFRADURE(TM) for the treatment of hepatitis D had received Orphan Drug Designation from U.S. regulators at the FDA.

The significant news effectively made every protein therapy and hepatitis drug developer (not to mention investors) looks up and take notice.

"We see this as a really significant validation of our technology by the world's toughest regulatory agency," explains Pearlman. "This coming literally just a month after the FDA approved our first clinical trials- which went straight to Phase IIB- in the United States."

Asked to explain the significance of having achieved this designation for Medgenics' proprietary tissue-based Biopump(TM) platform, Pearlman doesn't miss a beat.

"First of all, Orphan Status is a very really significant milestone for any company in pharma or biotechnology," Pearlman says. "Because Orphan Drug Status means that the FDA recognizes that the treatment that you're developing is promising and looks like it can address the need for a specific patient population base."

Medgenics has been diligently developing the same proprietary tissue-based Biopump(TM) platform technology for the sustained production and delivery of therapeutic proteins using the patient's own skin biopsy for the treatment of a range of chronic diseases including anemia, hepatitis C and hemophilia among others. Pearlman believes this approach has multiple benefits compared with current treatments, which include regular and costly injections of therapeutic proteins and if he's right, his firm may have just taken one giant step toward convincing the rest of the world he was right.

"The reason why this is so important for our Company is that this is the first Orphan designation that we've attained for the use of our Biopump(TM) system and the beauty of this situation for us is that with Orphan designation, often what happens is that firms are granted an accelerated approval path by the FDA and although that's not guaranteed, we feel we have a good chance and are certainly hoping for that. Also, after a designation like this, the actual size of the clinical trials one needs to conduct is usually considerably smaller than you have to do for any of the major indications," says Pearlman.

"In Israel, we are hoping to be approved to commence the studies of our Biopump(TM) technology in Hepatitis C. Now Hepatitis C is an enormous indication. One hundred and eighty million people in the world have it and the INFRADURE Biopump(TM) has a very appealing and advantageous value proposition for the treatment of hepatitis C. It has an even more, I would say, compelling case for treating hepatitis B and there are some 350 million patients in the world with hepatitis B. All of these indications would use the same INFRADURE Biopump(TM) approach. The Biopump(TM) stimulates your body's immune system to fight the viral infection- whether its hepatitis B, C or D. The advantage of the combined approach that we're doing in going after hepatitis D as an orphan drug in the United States while commencing the clinical testing in hepatitis C in Israel is that we believe that we will demonstrate safety and efficacy in both of these indications, but that in the case of hepatitis D, there is a very improved likelihood for a relatively rapid route to a product approval compared to hepatitis B or hepatitis C-in which there would likely be much larger clinical trials and a longer path toward approval."

Following a late morning confirmation about the FDA news, shares of the company soared 25.36% to $8.65 +1.75 during regular market hours and headed higher in early trading on Thursday. If trading has been any indication thus far, the low-float stock may continue to appreciate in value as the market continues to digest the significance of the news. Particularly since some of Big Pharma's recent buyouts have targeted Drug makers developing hepatitis treatments.

Even some which have shown, arguably, less promise and innovation than Medgenics' own novel platform-- which aims to reduce some of the terrible side effects many patients endure with other treatments-have been acquired at huge premiums

Pearlman is focused on making sure that his firm will continue to build its pipeline and add value beyond its current $83.73 million market cap by executing and continuing to share positive developments like this one with investors.

"This is now the second approval that we've gotten for our technology, for our company and we think it will give a lot more confidence to would-be partners and institutional investors who may have been looking for a clear sign from a regulatory agency that we have a straight shot for an approvable product within a reasonable time."

Medgenics gets OK to start Phase IIa trial of their EPODURE biopump...

Article posted 3/23/12 on Proactive Investors.com. Israel-based Medgenics gets Israeli Ministry of Health OK to being a Phase IIa trial of it's EPODURE biopump to treat HCV, anemia & haemophilia. The technology will allow patients to continuously produce and maintain their own proteins without dangerous variations in PK.   


Medgenics gets approval to begin Phase IIa clinical study on EPODURE biopump

Fri 11:17 am by Giles Gwinnett

Medgenics has received the green light to begin a Phase IIa clinical study on its EPODURE which is used to treat anaemia.

The company has received clearance from the Israeli Ministry of Health to begin the study, it said.

The firm's biopump system sees a tiny sliver of the patient's tissue taken and modified to carry the gene to enable it to continuously produce the required protein to treat the condition.

It is is developing three applications of the technology - to treat anaemia, hepatitis-C and haemophilia.

The study, announced today, will involve 20 patients and will asess EPODURE's ability to replace months of routine injections for anaemic patients who have end-stage renal failure - or kidney disease.

Each patient will receive an individually targeted initial dose of EPODURE biopumps designed to produce levels of the protein that would replace the routine injections over a period of 4-12 months.

President and CEO of the firm Andrew Pearlman said he expected the phase IIa study to build on the positive clinical results from the earlier Phase I/II study of EPODURE to treat pre-dialysis patients with chronic kidney disease.

"This is the first clinical study that will permit adjustment of the EPODURE dose based on patients' needs as is currently done in standard EPO (erythropoietin) treatment, and will be standard in future EPODURE use.

"This represents a significant change from the fixed-dose study we previously conducted in pre-dialysis patients," he said.

Dr Pearlman added that the company's EPODURE Biopump technology offered potential advantages over current therapy because it enabled patients to continuously produce and deliver their own proteins.

"Sustained delivery of EPO is expected to help keep hemoglobin within the target range to reduce the risks of hemoglobin variability, while avoiding the possible risks posed by EPO concentrations many times the normal physiological range, as observed with EPO administrations.

"EPODURE could potentially be a safer anemia management tool. Additionally, the cost benefits for the management of anemia could be highly significant," he said.

Seeking Alpha.com: Will Medgenics Become The Next Hepatitis C Take Over Candidate?

Posted on 3/12/12 on Seeking Alpha.com. The following comments by the Viral Matters editor: Seeking Alpha contributor Ray Dirks speculates that Medgenics - with it's proprietary EPODURE (for anemia) and INFRADURE (for interferon) biopump devices - would be an attractive candidate for take over. EPODURE might be a go, but the market for INFRADUR could be severely limited to disease states outside of HCV if 'interferon free' therapy becomes a reality.  As it is only in preclinical trials, INFRADUR has plenty of time to see where the 2nd and 3rd generation of anti-HCV Direct Acting Antivirals take us

Will Medgenics Become The Next Hepatitis-C Take Over Candidate?

By Ray Dirks - Seeking Alpha

Interest in the hepatitis-C space has been hot since the euphoria of Big Pharma's acquisition of Inhibitex (INHX) and others began to spread further in early January. Smaller biotechs are being snapped up by larger pharmaceutical companies driven by the need to pump up their product pipelines.

Last November, Gilead Sciences (GILD) bought Princeton, NJ-based Pharmasset for $11 billion for the next generation of hepatitis-C treatments. Vertex Pharmaceuticals, working with Merck, is offering a pill for hepatitis-C, but it has to be taken three times a day though stands the chance to make $2 billion in a couple of years, when approved.

Then there was the Bristol-Myers Squibb (BMY) acquisition of Inhibitex for $2.5 billion and Roche's (RHHBY.PK) purchase of Anadys Pharmaceuticals for $230 million. Let's not forget that Novartis (NVS) and Enanta Pharmaceuticals are in collaboration to develop and sell a brand-new hepatitis-C inhibitor for nearly $500 million.

When one looks at Medgenics (MDGN), at a market cap of only $46 million, we have have to wonder how a company with an innovative approach to treating hepatitis-C would not be of interest to a bigger pharmaceutical partner or buyer. The firm has a very unique way to treat the disease which may not have been recognized by investors yet, but we're betting that will soon change.

Last month when shares were trading at around the $3.50 level, we made a case for the disruptive technology being developed by Medgenics, focusing on the value it has for the $90 billion protein therapy market that is projected to grow to over $130 million over the next several years. Shares nearly doubled after that report and have since seen a healthy pull back.

We've looked specifically at Medgenic's EPODURE product, a proprietary biopump that produces erythropoietin within the patient's own body, now in Phase I/II clinical trials to treat anemia in chronic kidney disease. The anemia market is dominated by Amgen's Epogen that carries with it a high price tag and serious side effects.

Now we'd like to draw attention to another of Medgenic's products in development, using its biopump technology - INFRADURE, that produces interferon-alpha and is currently in the preclinical stage for the treatment of hepatitis-C. In the U.S., hepatitis-C ranks second only to alcoholism as a cause of liver disease and is the leading reason for liver transplants. The Center for Disease Control and Prevention estimates that 3.2 million people in the U.S. have chronic hepatitis-C (far out-passing HIV) and costs due to treating the disease now exceed $600 million each year, with a projection of growing to $11 billion in 2019.

Interferon, or the more popular form, peginterferon-alpha, is usually used in combination with ribavirin, another antiviral medication. Treatment is expensive, around $20,000 per year for a 48-week injected dose, which puts it out of reach in developing countries where it is badly needed. Further, only 50% of patients benefit. There is clearly a medical call for a cheaper and more effective drug.

Medgenic's INFRADURE provides an answer. Like EPODURE and other products under development, INFRADURE uses Medgenic's biopump platform technology where the patient's own tissue, taken during a biopsy, is processed then inserted into the biopump to continuously produce and deliver interferon-alpha, a natural protein produced by the body. Also like EPODURE, this approach would eliminate weekly and expensive injections, particularly since Hepatitis-C progresses very slowly.

Two drugs are on the market to treat hepatitis-C: Pegasys, made by Genentech and PEG-Intron, made by Schering-Plough. Both are meant for weekly injections. Both are very similar and meant to be used in combination with ribavirin. Side effects for this combination are startling, the most reported ones being mental illness, thoughts of hurting or killing yourself or others, unusual thoughts or behaviors, the compulsion to use street drugs if they had been used in the past, alcohol abuse, and aggressiveness. Heart disease and autoimmune disorders are also common. Pegasys and PEG-Intron used with ribavirin causes birth defects or death in an unborn baby.

For example,the FDA's review of Schering Plough's application for PEG-Intron included a number of patients that either committed or attempted suicide, murdered others, relapsed to drug or alcohol addiction, and underwent severe depression. As we mentioned, heart attacks and heart valve disease also appeared, as well as lupus, extremely low red blood cell count, kidney failure, partial blindness, and hearing loss.

INFRADURE will go into human clinical trials in the first half of this year. Based on laboratory and animal data that reported up to six months of consistent delivery of interferon-alpha, Phase I clinical trial results should show efficacy based on results seen with EPODURE.

We draw your attention to the value of Medgenic's platform technology. Besides anemia and hepatitis-C, Medgenic's product is being designed for use in hemophilia, multiple sclerosis, arthritis, obesity and diabetes.

We advise our readers to watch this company going forward as we continue to expect even more bullish developments and positive news flow going forward.

Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

Medgenics developing new therapeutic therapy for anemia...

Article by Ray Dirks from Seeking Alpha.com. Although not directly related to Hepatitis C drug development, patients on the current treatments for Hepatitis C carry the risk of developing anemia. Sometimes this requires the administration of a medicines that increases red blood cell production, but is expensive and carries it's own risks, such as red cell aplasia or cardiac events. The Seeking Alpha investiment website covers a new treatment for anemia in development from a company called Medgenics. They have developed a mini-bio pump delivery system that results in sustained levels of erythropoietin naturally with very little side effects and at a fraction of the cost. They are entering phase 1 trials for Hepatitis C treatment-related animeia later this year.

Medgenics' Disruptive Biotechnology Is One To Watch
By Ray Dirks
Medgenics (AMEX:MDGN) has, what I believe to be, the most disruptive biotechnologies of the last 20 years. This company is poised to own the 90 billion plus protein therapy market. We are talking about an enormous indication. Nomura (the big investment bank) calls this one of the top 10 biotech companies to watch over the next 5 years.
Currently the only form of treatment for Anemia is Epogen. Epogen is a synthetic version of the Erythropoietin that increases your red blood cell production. It is a $12B a year product monopolized by Amgen (AMGN) and affects nearly 1-2 billion worldwide.
When you are diagnosed with anemia, you go to your doctor. Your doctor is going to tell you that you have an abnormally low hemoglobin count and he will prescribe an epogen shot to get your hemoglobin into check and into a 10-12 count range which is considered healthy.

These shots cost about $25K a patient per year and are very painfully administered. What is going to occur is a spike of your Epo level from very low up to a 100x, which can lead to high blood pressure, a percentage increase of fatal cardiac events (as per the FDA) and all sorts of side effects. Over the next 48 hours, your Epo will crash back to a low level after which you will be treated with another shot of epogen, and this happens over and over again for the rest of your existence.

It's a terrible way to live, costs the system billions and is one of the worst FDA procedures on the market. In fact, Amgen has already received a black box warning because of the side effects and increased risks and is under major scrutiny for reimbursements issues as well.

The holy grail of Anemia treatment is to treat the patient without the infections and without that "spike" in Epo which is deadly in nature and provides a terrible quality of life.

Medgenics has developed a therapeutic system that can provide a dosage of the sustained levels (no spikes) of Erythropoietin naturally with literally no side effects and only two primary clinic visits for a fraction of the money. Let me explain exactly what they do and how they do it:

Medgenics takes a small piece of your skin about the size of a one inch toothpick from your abdomen.

This piece of tissue is then processed in a lab by inserting a small piece of DNA that teaches it to continuously produce the missing protein; which in this case is Erythropoietin.

Once this engineered piece of tissue is making the protein you are missing and at the levels needed, it is injected back under your skin and the result is a mini biobump that produced natural Epogen without the spikes, without the shots, without the side effects or dropouts. And for a fraction of the cost of Amgen's Epogen shots.

The Phase I/II study was done with 20 patients, all people diagnosed with anemia obviously. In all 20 patients you have biopumps producing Erythropoietin safely for as long as 36 months without any follow up, within that 10-12 count and without the injections and their dangerous spikes.

The most compelling thing about this story is that this isn't just a treatment for only Anemia; it is a platform that can be used on ALL protein therapies. In fact the company is entering Phase 1 for Hep C (Interferon) in Q2 of this year. Hepatitis C/Interferon is a 3.5 billion dollar Market this year. Gilead (GILD) paid 11 billion for a HEP C company… Inhibitex (INHX) was taken over for 2.5 billion (another Hep C company).

Medgenics already has an active program for Hemophilia developed with Baxter (BAX), and they will also be starting programs for:

* Growth Failure/growth hormone
* Multiple Sclerosis/Interferon Beta
* Diabetes/Insulin
* many others

In summary you have a company that has:

* $50 million invested
* the most disruptive technology in biotech today
* a management team that is arguably from top to bottom the best I have ever seen. Former head of Stanford Medicine, and an entrepreneur who sold his last company for $600 million
* Baxter collaboration and the potential for many more collaborations on multiple indications
* already finished Phase I/II and entering Phase IIB, this is NOT pre Clinical; this is tested and proven in human
* a ridiculously low valuation relative to its competitors.
* the potential of two probably partnerships in the near term
* a science which is not a one trick pony, it is a platform that covers every protein therapy which is more than a 91 billion dollar market
* largest shareholders are Joel Kanter and Isaac Blech. Joel has just sold Clarisonic for $600 million. Isaac has founded over 35 billion in companies

Enough of the science. Let me walk you through the value proposition of the investment. There are two public companies and one collaboration that I will use as comparisons.

Protalix (PLX) has a platform technology and is at about the same stage of clinical as Medgenics, but is only for an indication of 25,000 patients and trades at a 520 million dollar market cap.

Prolor (PBTH), which has a treatment which only increases the half lives of protein therapies, trades at a 300mm market cap.

And of course Zymogenetics which only has an interferon lasting slightly longer than the usual 5-6 hours for Hep C, was bought by Bristol-Myers Squibb (BMY) for $545 million.

Medgenics' market value is currently approximately $32 million and is clearly poised to be the leader in a multi billion dollar space, and I believe will far exceed the market value of the the companies mentioned in this article.

Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.