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."