Riquent unsuccessful in getting FDA Approval and taking Product ForwardFintan Walton :
Hello and welcome to PharmaVentures business review here live in San Diego, California. On this show I haveDeirdre Gillespie , who is CEO of La Jolla Pharmaceutical based here in San Diego.
Deirdre Gillespie :
Hello Fintan Walton .
Fintan Walton :
How are you?
Deirdre Gillespie :
Very well, thank you. Pleasure to be here.
Fintan Walton :
Good. Now Deirdre Gillespie , you joined La Jolla Pharmaceutical back in March 2006?
Fintan Walton :
And the company had gone through some, some changes at that time. But things have changed since March 2006 when you joined, could you tell us what, what sort of company you had inherited and the issues that you have had to address at that particular time?
Deirdre Gillespie :
Yes. La Jolla Pharmaceutical has a long history of developing new drugs for autoimmune diseases. And it's lead product is called Riquent, which has been developed for a lupus a fairly rare autoimmune diseases. It have been through a previous Phase III program and have unfortunately just missed " missed it's primary end point in its Phase III drug study. So it was unsuccessful in getting FDA approval. However on review of the data it appeared that perhaps we have been under dosing at that time. So the company was my predecessor actually was very effective in securing a $66 million pipe from top 10 two venture capitalist in December 2005. And that got the company back off to the races.
Fintan Walton :
Right. So you came in with money in the company.
Fintan Walton :
And some clear indication of how to take this product forward?
Deirdre Gillespie :
Of what needed to be put right, what the changes need to be.
Deirdre Gillespie :
So we did that, we got a much bigger Phase III study now, most importantly Fintan Walton , we increased the dose.
Deirdre Gillespie :
In the previous studies that have been 100 milligrams per dose of the drug.
Deirdre Gillespie :
And that have being effective just not enough, not effective enough in and out of the patients.
Fintan Walton :
Right, right.
Deirdre Gillespie :
And because the safety profile was so excellent and with the FDA support we increased the dose twice actually to 300 and 900. So in this study now we have Placebo 300 and 900 and the studies been up in running and it's now in the lateral part of Phase III.
Riquent novel technology and its pipelineFintan Walton :
Okay. Now Riquent, the molecule itself describe it, because it obviously it was important to increase the dosing and why was that, was that because of the nature of the molecule?
Deirdre Gillespie :
lupus is also an autoimmune disease in which you make antibodies to your own cellular components and one of the very big group of antibodies of course is lupus is antibodies to your DNA.
Deirdre Gillespie :
That anti-double-stranded DNA-antibodies. This drug binds to those and sweeps the amount of the blood stream. So Riquent itself is in fact 97% DNA, it's folded little 20-mers ofDNA on a little platform but it's injected IV and then mocks these autoantibodies after the patient's blood stream.
Fintan Walton :
So it's a novel " it's a novel type technology?
Deirdre Gillespie :
Very novel technology, discovered back in the year early 1990's by La Jolla Pharmaceutical, so a long history of discovering the technology, turning into a drug, then to the clinic.
Riquent used to prevent exacerbations and as a much safer drug.Fintan Walton :
So it's very important just to make sure that this new technology, this new concept could be applied in the therapeutic " in the therapeutic way?
Deirdre Gillespie :
It's very important because if the drug is effective and it's looking very promising now, it's also very safe in these patients.
Deirdre Gillespie :
And the current treatments for lupus and there are no approved treatments, the current treatments are just to give the patient high dosages of drugs that suppress the immune system, so steroids or anti- cancer agent.
Deirdre Gillespie :
And these are young women mostly, 90% of them are young woman and high dose steroids and Cyclophosphamide of course have many very bad side effects.
Fintan Walton :
The side effects?
Deirdre Gillespie :
In fact the commonest cause of ill health and death in these patients are side effects from that treatment. most commonly overwhelming infections.
Deirdre Gillespie :
And this drug would not cause that.
Fintan Walton :
Okay. And this drug presumably is a chronic therapy
Fintan Walton :
So it's important that this " and it also works effectively over the long-term here you described?
Deirdre Gillespie :
Yes. And it's unique compared to other drugs coming down the pipe for lupus and that is used to prevent exacerbation of the disease as opposed to treat what is called a flare the patients go on this and this prevents them having exacerbation or increases the time to the next exacerbation.
Deirdre Gillespie :
So it's a unique profile as well.
Raising funds and increase of the dosage of DrugFintan Walton :
Okay. The other think you did when you joined a year later I believe is to raise to further finance, could you describe that. And how much you were able to raise?
Deirdre Gillespie :
We raised $40 million in the first quarter of 2007. In this very large study we have " we have four interim analysis, and the first two was to measure how effectively the higher dosages reduce these particular autoantibodies in the patient, so we did that first analysis in the first quarter of '07 and it was very positive. The high dosages were working much better and then we raised money. And then we did a 12-month interim antibody analysis just recently and that confirmed the antibodies stay down long-term and we've raised money again on the back of that one. Yeah the goal really is to drive " it's a high quality study. We've got " the FDA is very interested in it. We have an approvable letter with fast tracked. We have a Special Protocol Assessment. We have the NDA review previously, so our manufacturing capabilities already being reviewed and approved. We just need to confirm the efficacy, I went through.
Fintan Walton :
Okay. Now in terms of raising the finance in both occasions, was that a difficult task for a company like yours?
Deirdre Gillespie :
It was much harder this year than last that is for sure.
Deirdre Gillespie :
Yes. Last year it was a challenge, but we "we had an oversubscribed book [ph] with it a fully marketed around in 10-days. This year of course the markets are very down indeed and we were lucky that our venture capital investors contributed very significantly to this round. So strong inside participation and then the external investors came in.
Anticipation of the Product Approval and the ongoing studies.Fintan Walton :
Right, now and going back to Riquent when do you expect your product to be approved?
Deirdre Gillespie :
We have three shots of goals, it's in between now and the end of next year.
Deirdre Gillespie :
So we have our first interim efficacy analysis, as this drug reducing the flare in the kidney, in December of this year and second one, in second quarter of next year and then final data at the end of next year.
Fintan Walton :
So that's the end of 2009?
Deirdre Gillespie :
But if we hit it either at the interims we stop and we file. So, exciting times.
Fintan Walton :
So, so that's a very clear objective and for you to achieve what's, what's " what are the products you have coming up behind this Riquent ?
Deirdre Gillespie :
We have -- actually really for practical purpose Riquent is our focus, we do have another very novel small molecule, chemical entity in the research base with a lead compound, some great animal data, but that's a year from IND. So really, I am very focused on a new treatment for lupus one that could really transform the way these patients are treated. Right now the studies looking very good. The dropout rate is low, the compliance is high, the safety is absolutely clean as a vessel and the number of flares the patients are having is down. So really it's " it's which story that's matters.
Fintan Walton :
Right, so you are focused in that, but are you not tempted to try and look for other products as well?
Deirdre Gillespie :
We have been tempted, and we do have been looking, but it is hard for small companies, mostly venture capital backed to get the funding.
Deirdre Gillespie :
And the big, this is a 160 sites in 30 countries. Patients are in these studies for 12 or 15 months, it's going to be 900 patients. So it's actually a very large complex study, just as it's on its own and that's where we need to be acting.
La Jolla interest in Partnering ActivitiesFintan Walton :
Okay. And when you " if at the end of December of 2009 you've hopefully got approval?
Fintan Walton :
What would you like to do with La Jolla Pharmaceutical would you " what's going to happen next?
Deirdre Gillespie :
Since last second antibody dated just recently, and because we are getting towards the end now of the Phase III development, it's being a considerable uptick in partnering interest. So we have a number of ongoing discussions with potential partners. Our plan is to keep some commercial rights in the United States. So may be co-promotion with capital arrangement and then use work with our partner to begin to get the product commercialized, have a little income of our own and then began to build the pipeline.
Fintan Walton :
Okay. So it's " it's you are definitely gonna look for partnering, you're not going to try and keep this all for yourself and settle as a fully integrated pharmaceutical company, with one product?
Deirdre Gillespie :
No, I think that " that's fine. Its critical it lets a focus market is Rheumatologist and Nephrologists, so you don't needed to pick sales force. In my opinion it's hard to market very well first time around if you are a small biotech. I think learning and working with a partner, where the drug is important so it is the right way to go this time.
Fintan Walton :
Right. And it's important for the " for the describer?
Fintan Walton :
To feel confident about the company that selling?
Deirdre Gillespie :
Yes, and to have " yes, we have very good marketing support, all the funds that are needed to do it well as well.
Deirdre Gillespie work experience in US and UKFintan Walton :
One final question, you've worked both as CEO in theUS and also in theUK, which do you find the more interesting, let's put it that way?
Deirdre Gillespie :
It's a sneaky question.
Fintan Walton :
Well, yes. Okay.
Deirdre Gillespie :
It was a little bit difficult to do because I was the CEO of a private company in the UK and now it's a public company here. And then ---
Fintan Walton :
Yes. So it's entirely different really?
Deirdre Gillespie :
It's very different, the small company three or four "I was employee number three in my last company, I'm employee number 100 in this one. And I have none of the popping instructions in the UK. But I am working with venture capitalist, so from that perspective it's very similar. I would say it was harder to get money in the UK than it is in theUS.
Fintan Walton :
Yes, yes.
Deirdre Gillespie :
I think now there is constant problem of course.
Fintan Walton :
Right. So working in this cluster down[ph] in the South California helps a lot?
Deirdre Gillespie :
Yes, this critical matter here lots of biotech's, lots of CEOs, all the VCs are here. It's " I think is almost bigger than San Francisco now in San Diego, Much bigger world I think.
Fintan Walton :
Okay. Well Deirdre Gillespie , thank you very much indeed for coming on the show. Thank you very much indeed.
Deirdre Gillespie :
My pleasure. Thank you Fintan Walton .