Synthetic Biologics CEO Jeff Riley Discusses ‘Gene Medicine 2.0’

Synthetic Biologics (SYN) is a biotechnology company focused on the development of synthetic DNA-based therapeutics and innovative disease-modifying medicines for serious illnesses. The company is developing, or has partnered the development of, product candidates for the treatment of pulmonary arterial hypertension (PAH), relapsing-remitting multiple sclerosis (MS), cognitive dysfunction in MS, fibromyalgia and amyotrophic lateral sclerosis (ALS). Below, OneMedRadio interviews CEO Jeff Riley.

 

 

Click below to hear audio interview and see transcript that follows.

Matthew Margolis:    Greetings from OneMedRadio, I’m Matt Margolis. Today, we’re joined by Jeff Riley, CEO of Synthetic Biologics, an East Coast-based biotechnology company developing DNA-based therapeutics and disease-modifying medicines for serious illnesses. Synthetic Biologics trades on the AMEX under the symbol SYN. Mr. Riley, thank you for joining us.

Jeff Riley:    Hey Matt, thanks. It’s a pleasure to be here.

MM:     So let’s start with the discussion of your R&D. So what’s your lead candidate? How does it work?

JR:     Matt, I’d love to do so. We basically have three products that are at the clinical stage of development. The one that we’re no longer heavily involved in is for fibromyalgia. It’s called flupirtine [EffirmaTM], and that was partnered in 2010 with Meda, a Swedish drug company.

So we focus on our other two products that are in-house and that we’re moving forward. One is for multiple sclerosis that is called TrimestaTM. We just completed the 164-patient relapsing and remitting trial enrollment of patients. It’s being run out of UCLA by Rhonda Voskuhl, and we expect to have information data from that Phase II trial some time in early 2014. We are also in the process of enrolling another 60 patients for cognitive dysfunction, also in multiple sclerosis in women, and that trial should begin shortly as well.

Our other product, which is very exciting is the zinc acetate formulation and it’s specifically for Lou Gehrig’s disease or ALS. The Phase I trial of that is going to begin in about 2 weeks, and we’ll roll into a Phase II/III trial by third quarter of this year. We’re hoping for data from that Phase II/III trial in first quarter or potentially even second quarter of next year, 2013. Again, there’s really no therapies for ALS, Lou Gehrig’s disease at this point in time, so we feel very, very good that if this product does show efficacy, which has pre-clinically looked like it’s going to do very well, we’ll have something that can help patients substantially. So they’re already in the pipeline.

MM:     And what specifically do you think makes your unique formulation for ALS opportunistic?

JR:  I don’t know if it’s opportunistic. I think there’s a fair amount of data that’s been out in the public demand with respect to zinc and how zinc functions as it relates to copper, especially in central nervous system disorders, Alzheimer’s, Parkinson’s, Lou Gherig’s disease. So the mechanism of action is essentially trying to balance the amount of copper in an individual’s brain when they get an amount of zinc that actually pulls copper. It is complementary to some extent pulling copper from the brain. There’s a bit of a balancing act there, but the theory in the science we had is very solid so we were following on years and years of research to see whether or not we can show that this works in patients.

MM:   So in your opinion, why is there resurgence in gene medicine?

JR:  We call it Gene Medicine 2.0, and in November of last year, we decided to change directions a little bit in the company, and going forward, focus heavier on biologics, which is why we renamed the company Synthetic Biologics. Gene Medicine is, I think, coming into its own similar to monoclonal antibodies and other therapies over the years, in particular, biologics. It really does take 15 to 20 years of hard academic and rigorous research to get a set-up technology or a set of products to a point where you could do the translational work, put those in the humans, and see exactly what’s going to happen.

About 10, 12 years ago, Gene Therapy 1.0 was very famous. Several companies raised billions of dollars. The result was that it was not ready for prime time. It was a little bit premature, and basically, gene therapy dropped off the face of the planet.

Big pharmacies decided to abandon those areas, not all of them, but most of them, and [were] de-prioritized within the discovery functions in these companies.

However, the last 10 years, there’s been a lot of progress made, and I think you’re going to see a resurgence because a lot of the issues from delivery to immunogenicity have been overcome by a lot of great academic research, and we felt that the time is right, and the inflection point was right for attempting it, in particular, small company like us to jump into that world, build a theme around that, and begin to pull these products into the clinic.

MM:     So I wonder if we could circle back to your company re-branding, your recent changes. Maybe you can introduce us to the recent management changes.

JR:     You bet. So I was chairman of the – I became chairman of the company in November, and I took the CEO role in February of this year.

We also hired John Monahan as our chief science officer. John is one of the foremost experts in the gene therapy area. He was also the founder and CEO of the company called Avigen, which was one of the bigger gene therapy companies 10, 12 years ago. It reached the market cap of $2 billion and then crashed back to earth at a later date. It was acquired by somebody else, a fantastic individual, very, very, very knowledgeable about the gene therapy space.

We’ve also hired recently a gentleman named Evan Ballantyne. Evan was the Chief Financial Officer for Clinical Data, where he was for 5 years, and it successfully sold that company last year in 2011 for $1.3 billion. That was an R.J. Kirk company, and we’ll get to that shortly I think when we talk a little bit about our partner in strategies with privately-held company Intrexon.

We’ve also hired Michael Kaleko who used to be at Novartis. He ran Novartis’ gene ocular programs. And we continue to fill out the rest of the team with press releases over the next few weeks. You’ll see filling in with the Chief Clinical Officer as well as, you know, VP of Regulatory goals in addition to what we currently have. So you’ll see a team that’s very focused on the products that we have today from the clinical development perspective, but more importantly, look into the future at biologics and more specifically gene medicine. This team will be one of the best teams out there as far as knowing how to take a product from IND on through.

MM:   And you were eager to discuss your collaboration with Intrexon Corporation. In late March 2012, you announced your Q1 earnings and one major highlight was the execution of this collaboration that occurred in Q4 of 2011. So I was wondering if you could go into a little detail about this company milestone.

JR:   You bet. Alot of folks in the industry, in particular, on the banking side, know the name Randall J. Kirk, also known as R.J. Kirk. This gentleman was very prolific in the biotech and pharma industries with respect to M&A and building companies. His mantra tends to be, you know, he’s going to buy into a company, he’s going to build the company, he doesn’t sell his stock, and he gets the company to a point where there’s a value inflection point, and an M&A transaction occurs. He was involved with General Injectables, Scios Pharma, King Pharmaceuticals, New River was his company. And most recently, on the public side, his company was Clinical Data. I’ve hired his ex-Chief Financial Officer recently for our company.

And during that time period, he’s also started and is now very focused on a company called Intrexon, which is a privately-held company with roughly 300 employees in it. It is focused entirely on DNA therapies, the platforms within DNA therapies as well so based treatments, stem cells, you name it. If it’s biologic, he’s been going out looking at platforms and acquiring those who’re building a platform-based company around those technologies. So we were very pleased back in November, late November of last year when we were able to get a deal done with his company, which was announced. It was specifically around one indication to begin with. We hope to expand that collaboration over the next couple of months, but the initial collaboration is around pulmonary arterial hypertension, and we’re using the RheoSwitch® technology in addition to our expertise.

The two teams are working together to move this further pre-clinical studies and hopefully into the clinic.

MM:      So maybe you can go into some detail about pulmonary arterial hypertension, and specifically, what the market opportunity is there.

JR:    PAH is a nasty disease. Basically, the thickening of the arteries that lead, from the lung to the heart and back. It’s a deadly disease, life span 3 to 5 years after contracting this disease. There are some pretty good therapies out there today, everything from Viagra, which is used to help expand that area in a pinch to some of the prostacyclins, and there’s a variety of companies playing in this area. One of the downsides is that most of the therapies including prostacyclin, there is a dose limiting issue, in that you can’t give a patient too much of that without an adverse reaction.

So with gene therapy, the goal is to try and get the DNA directly into the cells that are at the location of the disease stage itself, so in the pulmonary arteries, where we will then be able to activate those genes to produce the proteins, to produce the prostacyclin, and the theory is that we will be able to get a much higher amount of the product locally at the place where it needs to be with a much lower systemic side effect profile, which will obviously benefit patients greatly. They don’t have as much side effects, but they have the drug that they need to have at that location, so hopefully eliminate the disease state.

MM:  Now I want to circle back to your in-house pipeline. So can you go into some detail about your approval calendar for Trimesta and your ALS formulation?

JR:   We briefly touched on it earlier for the Trimesta for the MS drug. We’re looking at getting Phase II data early next year or early – I’m sorry – early in 2014. Thereafter, we’ll have to look at what the trial date is. It is a pretty big trial, 165 patients, 164 patients. We’ll have to get together with the FDA and see what we need to do for registration trial. After that, the endpoints are such – keep in mind, there are two MS trials we’re running; one for cognition and one for relapsing-remitting MS, and we don’t have a clear picture on if we can accelerate the process after that trial or not. So I can’t give you a clear definitive answer on that per MS specifically.

For Lou Gehrig’s disease and the zinc formulation, that – We know pretty closely what we need to do with that. The Phase I will begin shortly. We’ll go into a Phase II/III later this year, and probably by this time next year, 2013, we will have results from the Phase II/III trial. Again, we’ll have to go to the FDA and talk to them to find out exactly what we needed to do, whether that data is sufficient to be registration file or whether we need to do another Phase III. So again, that’s a much shorter timeline than Trimesta, but again, the market is not as big for Lou Gehrig’s disease as it is for multiple sclerosis.

MM:  And lastly, what opportunities do you see for Synthetic Biologics going forward and what are your key strategies to build shareholder value?

JR:    Going forward, we’re looking at obviously doing the basic blocking and tackling on our existing programs and moving those forward as aggressively as we can. In addition to that, we are very active on the business development front. We are looking at a variety of programs in the gene therapy and cell-base therapy biologics areas, and we will be pulling programs into the company over the next 4 to 6 months. In addition to that, we will probably expand our relationship with Intrexon. Those are in discussions now, and you know, we hope to have a very robust pipeline from discovery stage, pre-clinical stage programs, all the way up to Phase III within the next year or so.

MM:   That was a company snapshot with Jeff Riley of Synthetic Biologics, trading on the AMEX under the symbol S-Y-N. This is Matt Margolis of OneMedRadio signing off.

 

Forward-looking statement:

This interview contains forward-looking statements on Synthetic Biologics’ current expectations and projections about future events. In some cases forward-looking statements can be identified by terminology such as “may,” “should,” “potential,” “continue,” “expects,” “anticipates,” “intends,” “plans,” “believes,” “estimates,” and similar expressions. These statements are based upon current beliefs, expectations and assumptions and are subject to a number of risks and uncertainties, many of which are difficult to predict and include statements regarding the timing and results of our clinical trials and our ability to successfully develop products with superior benefits. The forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those set forth or implied by any forward-looking statements. Important factors that could cause actual results to differ materially from those reflected in Synthetic Biologics’ forward-looking statements include, among others, a failure of our clinical trials to be completed on time or to achieve desired results, or failure by us or our strategic partners to successfully commercialize products and other factors described in greater detail on Synthetic Biologics’ reports on Form 10-K and other filings with the SEC, which are available at www.sec.gov. Such forward-looking statements are current as of the date posted, and Synthetic Biologics expressly disclaims any obligation to update such statements.


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