Approaching Parkinson's the Neurologix way




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Video title: Approaching Parkinson's the Neurologix way
Released on: October 23, 2009. © PharmaVentures Ltd
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  • Summary
  • Transcript
  • Participants
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In this interview, filmed at BioPharm in San Francisco, Dr Fintan Walton speaks with John Murdock, CEO at Neurologix, an innovative New Jersey-based biotech company.

They discuss:

• the vision behind Neurologix and its novel therapeutic approach to Parkinson's
• gene therapy as a solution to countering deficiencies
• Dopamine-based therapies and potential alternatives
• Neurologix's frugality and funding
• assistance with the firm's Phase III
• securing the company's financial foundations during the crunch
The vision behind Neurologix.
Fintan Walton:
Hello and welcome to PharmaTelevision News Review, we are here at BioPharm America in San Francisco and on this show I have John Murdock, who is CEO of Neurologix a company based in New Jersey. Welcome to the show.
John Murdock:
Thank you very much Fintan .
Fintan Walton:
Neurologix as I said is a company based in New Jersey here in the US, your company has focus in CNS and you have some very novel ways of approaching the issues that appear in this in the area of CNS particularly Parkinson's, could you just describe to me how your business was formed and how got focus on this particular area?
John Murdock:
Yes, yes. Our business was formed in 1998, the lead investor was Palisade Capital and the vision behind the investment was essentially begin to the commercialize to develop and commercialize some of the innovation of doctors Michael Kaplitt who's currently is at the Cornell Weill University in New York as well as Mathew During, who is a Clinical Professor at Ohio State University and if you go back into the literature you will see that Michael and Matt really laid the foundation for the use of gene therapy especially focusing in CNS.
Fintan Walton:
Right.
John Murdock:
In combination they've written well over a 100 articles. And in fact our Phase I Parkinson's trial was the first gene therapy based trial for any CNS disorder in the world.
Gene therapy as a solution to countering deficiencies.
Fintan Walton:
So the approach the novel approach you are taking is gene therapy?
John Murdock:
Yes. We believe that the gene therapy is an ideal modality to begin we reestablish function in the brain especially where we have focal disease and we can identify a specific protein that's become deficient or impaired. And there is a tremendous need out there, because most of the large molecules and the small molecules create toxic effects as they began to pass through the blood brain barrier.
Fintan Walton:
So your technology is embedded in both the gene therapy vectors that you use as well as the novel approach that you take in producing a therapeutic effect?
John Murdock:
Yes. Well in the first case we were the first- one of the few first companies to begin using the adeno-associated-virus, which has proved to be extremely safe. And essentially that's our fundamental platform using we are using the cell type 2 and what we do in each and every case our lead indication as you've mentioned is for Parkinson's, but we are also very active in looking at Epilepsy and Huntington's as well. And we are currently working with Dr. Paul Greengard at Rockefeller University in New York City on a very interesting therapy for depression. And essential what we'll do is we'll repackage a unique trench gene for each and every one of the indications into the AAV-2 and really establish a new target for that.
Neurologix's novel therapeutic approach to Parkinson's.
Fintan Walton:
Okay. So as you said the first major project that's ongoing at the Neurologix's is Parkinson's?
John Murdock:
Yes, yes.
Fintan Walton:
Can you describe actually how your approach is different?
John Murdock:
Well essentially that indication is just about ready to finish a Phase II in the United States. Our Phase I was kind of interesting to the extent it was written up in both the The Lancet and the PNAS.org and our modality there is extremely novel, historically given the Parkinson's has been very closely identified with the deficiency of dopamine, which in fact is the case, however in late stage Parkinson's patients dopamine is not the only protein that's deficient. There is a protein called gamma aminobutyric acid, which is a major neuromodulator that is produced by dopamine, and quite frankly in these late stages patients what we're seeing as that this lack of dopamine really impacts the control of an area in the basic ganglia called the subthalamic nucleus which controls the signals out through this cerebral cortex, in our case we're directly going to the subthalamic nucleus or the STN and inserting a GAD-gene that synthesis the protein for GABA to reestablish the balance in the STN and provide symptomatic relief to the late stage Parkinson's patients.
Dopamine-based therapies and potential alternatives.
Fintan Walton:
So for doing at this type of trial with gene therapy is it difficult to get clinical recruitment or what are the barriers did you see in other words to trying to perform clinical trials for gene therapy kind of thing?
John Murdock:
Well yeah it's very interest you asked that question, because there has not been a new platform for Parkinson's since Levodopa, there is currently some combination drugs that are out there, but most of the therapies are dopamine based. And for late stage patients the only thing that's available is something called DBS. I presented last week at Rodman & Renshaw Conference and after it was one of the Parkinson's one of a members at the audience came up to talk to me, he was a Parkinson's patient and he was talking about the tremendous pan up demand and the hope that this population has because this really an underserved market. So in our case we assume that we have some difficulty including patients that has not been the case what's so ever. The enthusiasm from patients has been outstanding, neurologists have quickly grasped it, we are in eight leading institutions. And what's clear to them is that there is a very simple method of action and based on our earlier clinical results we are quite confident that we are gonna see a therapeutic effect. So we haven't had the problems that I'll expect you know of course there is the bureaucracy of the IRB's, the IBC's and quite frankly we are going through just about every department in a hospital and in today's environment that's not easy, but with respect to outside of the bureaucracy of the trial it's been very, very easy.
Fintan Walton:
So how does the patient get delivered the gene therapy?
John Murdock:
Well that's good well our given the fact that Dr. Kaplitt is not only a researcher he is a PhD in Molecular Biology, he is also a Stereotactic neurosurgeon. And if Michael were here he would tell you that most of his work is really translational, and as we began to develop the gene therapy it was very clear that one of the things that lot of folks typically forget is the surgical assist where you need to deliver that in the O.R, so we developed the partnership [PharmaDeals ID = 20291] with Medtronic, and Medtronic's developed a very innovative and fusion system that we insert stereo tactically directly into the brain and we began to infuse in the operating room, we move that patient from the operating room and within three hours, this therapy about a hour and a half in the operating room about another hour and a half in the step taking this therapy is completed and the catheter is removed and that patient stays overnight and goes home. So, I mean the direct question is, there is we do, we infuse.
Fintan Walton:
Right. But obviously a lot of technology behind that one?
John Murdock:
Yeah sure, yeah sure.
Fintan Walton:
And in collaboration with Medtronic's which is important part of the equation, I presume?
John Murdock:
Yes.
Securing the company's financial foundations during the crunch.
Fintan Walton:
So taking this approach which obviously is novel and it's got a level of high risk associated like all new therapies but it's particularly high risk. How have you managed to raise capital for a company like Neurologix which has this particular approach and in the background that gene therapies had its ups and down over the last few years?
John Murdock:
Well there is no question. The lead investor was Palisade Capital and this goes back to 1999. And it really takes a special investor, who has the vision and can understand. We've been very, very fortunate at GE Capital pension has come in and been very, very supportive to the company. We've also had a lot of support from the fund out of Dallas Fortworth called(indiscernable). We've been extremely frugal in the use of capital and we're about to rate the finish the Phase II and the company has raised something slightly under $47 million and our company is extremely virtual. I will tell you that there is a lot of questions about gene therapy, but we are beginning to see some optimism, there are number of publications I saw them the other day where we're really moving into the second generation of gene therapy and it's very(indiscernable) because I've been with the company now for about four-years and as I have been joined the company I could see the evolution both in the viral delivery systems, the promoters, the understanding of what the therapy could do, what they couldn't do. And I think we are very, very close to see an advancement. In my view I think gene therapy is going to be as important enabling technology as the micro chip was for computers.
Fintan Walton:
Okay. So the business model in terms of funding is traditional equity rounds of funding or is there a greater reliance on non-dilutive funding through charities and government?
John Murdock:
Well in some of our indications we were very, with our Epilepsy indication we have gotten support for pre-clinical work from the Epilepsy foundation [PharmaDeals ID = 29151]. We are also talking, we are also very optimistic potentially getting some work, have some support for our Huntington's indications from The Huntington's foundation, but most of our investments or most of the money that's coming into the company for developing Parkinson's is all come from equity rounds of financing.
Partnering and assistance with the firm's Phase III trials.
Fintan Walton:
And what is the relationship with pharmaceutical companies? Are you getting an interest from pharmaceutical companies for this particular approach, because it's different for their " it's outside their normal model?
John Murdock:
We are, we are as we get closer to our end point I've been recently very active in talking to number of pharmaceutical companies, the specialty pharmaceutical companies that have some confidence in gene therapy or in small in biologics whether it would be monoclonal seems to be the most interested. If you go to the large pharma companies there is a surgical component to this and they have not really I mean their business model is basically based around that small or large molecules, so this is a little foreign to them. And the science it seems a little foreign as well, but there is a good interest in the specialty pharma's, I am talking to couple of companies right now and I we've been very, very prudent in where we can " because we think this Phase II is a major, major inflection point for the company. So we've sort of pushed off partnering until we get close to that end point which is coming in the first " actually the second quarter of next year. So over the next six months I would expect to be much more active and be in a position to really develop a partnership relationship for the Phase III and ultimate commercialization, because that's basically our business model. We are extremely good at discovery early stage, pre clinical work, early stage product development, manufacturing of generic products, Phase II clinical trials what we get to a point where there are other companies out there that have competences in later stage you know commercial development that we don't and probably would never want to replicate.
Fintan Walton:
Okay. So you just briefly mentioned already some of the other products that you are developing are they reliant on the same gene therapy approach or are there differences in the " in the approach that you're taking with them as well?
John Murdock:
Yeah that's we are using, actually with our infusion system that was developed with Medtronic is the first the infusion system for delivery of gene in the brain, that was actually the first combination drug that was over approved by the US FDA, the vision there is to use that infusion system across the line. What we were essentially doing was changing you know vector viral concentrations, infusion rates, volumes but fundamentally it's the same approach and we are staying with the AAV-2-virus, because we have a good track record with it, it has a nice regulatory profile known to be safe.
Neurologix's frugality and funding.
Fintan Walton:
It's tough times for biotechnology companies particularly in the last year because of the environment, the economic environment, how much has that affected your company?
John Murdock:
Well we became rich. We were always reasonably frugal, but when we saw I guess it was the nuclear winner of 2008 we stepped back and took a look and we wanted to ensure that we could get to our Phase II data without additional funding. So we were thinking, we were actually ready to move one of our preclinical programs into a Phase I and we decided that we would slow down and wait until we actually hit our Phase II end point enter our next round of funding with a little better foundation, financial foundation with company we can be broader in our clinical development, but we've been extremely frugal.
Future highlights of Neurologix.
Fintan Walton:
Okay. And John Murdock, what can we expect now over the next couple of years, what's the what are the highlights we can expect from Neurologix's?
John Murdock:
Well you'll see that we'll you can expect that we will complete our Phase II in June actually we'll announce data in June or July of 2010 shortly thereafter another indication move into the clinical trial there is a crossover component with that trial where, that trial was you know double blind study, so we will bring the controls in. And assuming that our end point make that therapy available we will be transferring into our final manufacturing facility some time in late 2010. And I would expect in early 2011 our Phase III for Parkinson'sto begin with another indication in moving very nicely along in the clinic.
Fintan Walton:
John Murdock, thank you very much indeed for coming on our show. Thank you.
John Murdock:
You are welcome. Thank you.
John Murdock
Chief Executive Officer
Mr. John Murdock was appointed as the President and Chief Executive Officer of the Company on July 17, 2006. Mr. John Murdock has been a director of the Corporation since November 14, 2005. Mr. John Murdock was a Partner of Red Bird Capital, LLC, a private equity firm focusing on early stage medical technology companies, from 2001 until July 2006. From 1996 to 2001, he was President and Chief Executive Officer and a director of Teleflex Instruments &Surgical Services. He holds a B.S. and an MBA from La Salle University and an E.P.S.M. from the Graduate School of Business at Stanford University.
Neurologix
Neurologix is a clinical-stage biotechnology company dedicated to the discovery, development, and commercialization of life-altering gene transfer therapies for serious disorders of the brain and Central Nervous System (CNS). Neurologix's therapeutic approach is built upon the groundbreaking research of its scientific founders and advisors, whose accomplishments have formed the foundation of gene therapy for neurological illnesses. Current company programs address such conditions as Parkinson's disease, Huntington's disease and epilepsy, all of which are large markets not adequately served by current therapeutic options.