Operator
Operator
Ionis Pharmaceuticals, Inc. (IONS)
Q3 2011 Earnings Call· Mon, Nov 7, 2011
$73.45
+2.45%
Same-Day
-9.06%
1 Week
-16.43%
1 Month
-16.67%
vs S&P
-14.84%
Operator
Operator
Stanley Crooke
Management
Good afternoon, and thanks everyone for joining us on today’s conference call to discuss our third quarter financial results. Lynne will discuss our financials, and after that I’ll give you a brief update on our activities for the rest of the year. Joining us on the call today are Lynne Parshall, Chief Operating Officer and CFO; and Kristina Lemonidis, Director of Corporate Communications. Kris, will you read our forward-looking language statement please?
Kristina Lemonidis
Management
Thanks, Stan. Good afternoon everyone. A reminder to everyone this web cast includes forward-looking statements regarding Isis’ financial positions and outlook, Isis business, the planned commercialization of mipomersen and the therapeutic and commercial potential of Isis technology and products in development. Any statement describing Isis’ goals, expectations, financials or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercializing drugs that are safe and effective for use in human therapeutics, and in the endeavor of building a business around such drugs. Isis’ forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Isis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Isis, and as a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Isis programs are described in additional detail in Isis’ annual report on Form 10-K for the year ended December 31, 2010 and its most recently quarterly report on Form 10-Q , which are on file with the SEC. Copies of these and other documents are available from the company. With that, I’ll turn the call over to Lynne.
Lynne Parshall
Chief Operating Officer
Thanks, Kris. As usual, I’ll assume that you’ve read the details of the third quarter financial results in our press release, and I’ll of course be happy to take questions at the end of today’s call. We continue to make excellent progress this year. Our most notable success has been the submission of the mipomersen or rather Kynamro marketing application to the EMA. With this submission we are one step closer to commercializing Kynamro, and our more recent activities continue the momentum following the NDA submission. The three upcoming Kynamro activities that you should focus on are first Genzyme remains on track to file an NDA this month. Second Genzyme is actively preparing to launch Kynamro next year in both the US and Europe, including expanding its already leading commercial launch team. Third Genzyme has reached an agreement with the FDA for a special (inaudible) assessment on the design of the FOCUS FH study. This 12 month study will start this year, and is designed to support the following goals. Expansion of the FH population in the US can include severe heterozygous FH offering an alternative dosing regimen of 3 times a week dosing, so that we can offer our patients different dosing options. And potentially broadening the FH indication beyond severe in Europe. We are enthusiastic about the opportunity to invest to expand the Kynamro profile in its potential markets. In addition to Kynamro our progress this year is also evident in the maturing of our pipeline. We initiated clinical development on five drugs and two new drugs and continue to report the encouraging data for many of the drugs in our pipeline. We’re just beginning a busy fourth quarter in which we will be reporting clinical data on a number of our drugs. And Stan will provide more…
Stanley Crooke
Management
Thanks Lynne. Our primary focus in 2011 has been and remains moving mipomersen towards the market. We have completed the submission for marketing approval in the EU and as Lynne mentioned Genzyme and we are finalizing the US submission, and the US submission will be filed this month. The interactions we have had with both the US and EU regulatory authorities have been productive and give us confidence that our filings are appropriate and approvable for the indications we are seeking. We believe we have a comprehensive of registrational regulatory dossier. Mipomersen is focused on patients within obvious, severe unmet medical needs, FH is a stable cardiovascular disease. We have completed 4 randomized Phase III trails, each study showed compelling efficacy and an acceptable safety profile, each study demonstrated that mipomersen has unique lipid lowering profile. Kynamro was the only drug that lowers all atherogenic lipids. Our long-term open-label extension study has demonstrated continuing efficacy, and consistent safety. Our preclinical dossier is robust, and other parts of the filing such as CNC are very strong. Recently a circulation published in an article written by Doctor Frederick [ph] and colleagues. In this article they have reported that the effects of lipids lowering drugs in the life of homozygous FH patients, the study showed that a mean reduction of 26% in LDL cholesterol was associated with delayed cardiovascular events and prolonged survival in homozygous FH patients. This answers a question that we have been asked quite a number of times, and that is can the linear relationship of LDL cholesterol that cardiovascular risk be extrapolated to patients with extremely high levels of the LDL-C and in particular extrapolated the homozygous FH patients. The answer is an unequivocal yes. In this group, a 26% reduction in LDL cholesterol has extended the average patient’s…
Operator
Operator
(Operator instructions) Our first question comes from the line of Salveen Richter of Collins Stewart. You may proceed.
Yodi Morango - Collins Stewart
Analyst · Collins Stewart. You may proceed
This is Yodi Morango [ph] sitting in for Salveen. I wanted to ask about the reason for the change in dosing frequency to 3 times a week in FOCUS FH and how do you see that affecting compliance, and also the risk of ISR, and I have a follow up question.
Stanley Crooke
Management
We are not changing dosing. This is a continuous step – the continuation of the plan that we laid out years ago. We have weekly dosing. We have already shown that daily and three times a week, and our objective is to both – in this study we will be doing weekly dosing, as well as three times a week dosing. And the objective with the three times a week dosing is to provide patients the option of using the drug weekly or more frequently and giving the patient as many options as possible with our drug, to find the most convenient way to administer for each patient being different is what we think is the right way to provide the maximum in compliance. So there is no change at all in the plan that we have laid out. We are just pursuing the plan as we described over the last several years.
Yodi Morango - Collins Stewart
Analyst · Collins Stewart. You may proceed
Thank you and how many arms will this study have?
Stanley Crooke
Management
It will have a once a week, three times a week, placebo arms.
Yodi Morango - Collins Stewart
Analyst · Collins Stewart. You may proceed
Thank you and one quick follow-up question, you mentioned on the last call and also today that Genzyme’s marketing strategy is focused on educating physicians and patients, what has been done to date on the physician education front, but also on the payer front? Thank you.
Stanley Crooke
Management
I will answer briefly and then Lynne can provide more detail. I think the most important thing that has happened over the last couple of years has happened independently of Genzyme, and also has been facilitated by Genzyme and that is the identification of FH as a significant cardiovascular disease that is tremendously under diagnosed. If you look at the guidance that has been published by the National Lipid Association you will see that it encourages early diagnosis, cascade screening, and the much more aggressive treatment as well as referral of patients to lipidologists. In addition, Genzyme is engaged in quite a number of focus groups on other activities that are contributing and Lynne you probably want to add some more to that.
Lynne Parshall
Chief Operating Officer
Sure. Genzyme has actually now for the last year and a half or two years had a significant presence at all of the major medical meetings focused on cardiovascular health and on lipids. They have sponsored CME events and again they are focused in working both at medical meetings as well as with patient advocacy groups is on early diagnosis and aggressive treatment of these patients and identifying them and getting the patients into care of lipidologists, who are best suited to treat them. In addition of course, both in the US and in Europe, Genzyme is working with the various payer agencies. They have a very strong infrastructure to do that augmented of course now by the Sanofi infrastructure, and that has been an ongoing effort for quite some time.
Stanley Crooke
Management
Next question please.
Operator
Operator
Our next question comes from the line of Eric Smith of Cowen and Company. You may proceed.
Eric Smith - Cowen and Company
Analyst · Eric Smith of Cowen and Company. You may proceed
Good afternoon, and thanks for taking my questions. Lynne in terms of the potential for the Kynamro acceptance to slip into Q1, if it does we will just assume that you will fall a little bit short, $25 million short of the operating loss guidance, and cash year-end guidance that you have previously given. Is that how we should think about that?
Lynne Parshall
Chief Operating Officer
Yes, I think that is a fair way to think about it.
Eric Smith - Cowen and Company
Analyst · Eric Smith of Cowen and Company. You may proceed
Okay, and then Stan on the SPA, what does it say exactly you need to achieve in this 12 month trial in order to get the broader, less severe label for heterozygous FH in the United States. You just need to lower cholesterol or achieve a certain reduction or have some kind of safety?
Stanley Crooke
Management
Yes, Eric, all we are doing is the same thing that mipomersen has done in 4 previous randomized trials, and in the long term open level study. It boils down to lowering cholesterol in exactly the way we did before, and getting additional safety experience.
Eric Smith - Cowen and Company
Analyst · Eric Smith of Cowen and Company. You may proceed
Is there anything about the profile to date safety wise that makes this a risk in terms of the upcoming trial?
Stanley Crooke
Management
I am as confident as I have ever been in the clinical trial with this disease, and 100% successful.
Eric Smith - Cowen and Company
Analyst · Eric Smith of Cowen and Company. You may proceed
So, just simply showing the same safety profile you have seen in the previous four pivotals that would enable you to get approval in the broader population?
Stanley Crooke
Management
Yes, and that is exactly what I have been saying for some years that is the message we got from the European authorities, and now we have agreed with the FDA on specific study design and numbers of patients, and the requirements for meeting the FDA’s needs as well.
Eric Smith - Cowen and Company
Analyst · Eric Smith of Cowen and Company. You may proceed
And if this trial is…
Stanley Crooke
Management
All good news. It is all really good news.
Eric Smith - Cowen and Company
Analyst · Eric Smith of Cowen and Company. You may proceed
Clearly. If this trial then provides the expected result, how much broader could the European label become in terms of less severe heterozygous FH?
Stanley Crooke
Management
Of course, nothing is certain until you actually sit down and negotiate the label, but I think I mentioned that we would hope that we would achieve any patient who had greater than 160 LDL cardiovascular events on maximum lipid lowering therapy.
Eric Smith - Cowen and Company
Analyst · Eric Smith of Cowen and Company. You may proceed
Okay, and the last question, I’m just not sure I’ve heard the date of the R&D day in January, was that the 5th or 25th of the month?
Stanley Crooke
Management
January 5, right after the first of the year. The reason for that Eric is that we just have so much important data coming out right in the fourth quarter with APOCIII, factor 11, TTR, SGLT 2, all the other things we just think it is going to be a lot of information for people to digest, and we’re going to have to help you understand it.
Eric Smith - Cowen and Company
Analyst · Eric Smith of Cowen and Company. You may proceed
Okay, we will be top line press releasing some of that, or will we wait until January to see?
Stanley Crooke
Management
No, we will be putting out press releases on most of it, probably, yes, you will see a spate of press releases coming.
Eric Smith - Cowen and Company
Analyst · Eric Smith of Cowen and Company. You may proceed
Okay, thanks a lot.
Stanley Crooke
Management
Lynne do you want to add anything to or subtract anything from what I said to Eric?
Lynne Parshall
Chief Operating Officer
The FDA wanted us to treat more patients with the drug, reflecting the very much larger market that is represented by the severe FH patients. But the study looks very, very much like all the other studies we have completed.
Eric Smith - Cowen and Company
Analyst · Eric Smith of Cowen and Company. You may proceed
I’m probably going to push my luck with this last question, but is there any kind of expected time line in enrolment and gathering data from the trial or is it too early to say?
Lynne Parshall
Chief Operating Officer
I think it is too early to say. Once we start enrolling patients, I think when we get a few months into the enrolment, we will have a much better handle on it, but I hate to project right now.
Eric Smith - Cowen and Company
Analyst · Eric Smith of Cowen and Company. You may proceed
Thank you.
Stanley Crooke
Management
I will say that there is a lot of enthusiasm for mipomersen and the trial, and so getting underway. And you know, you don’t need to listen to me, all you have to do is look at all the reviews that have come out about mipomersen over the last couple of years, and you can get a sense of how interested the cardiovascular community is in the drug.
Eric Smith - Cowen and Company
Analyst · Eric Smith of Cowen and Company. You may proceed
Thanks and good luck.
Stanley Crooke
Management
Yes. Great news that is the bottom line. Great news.
Operator
Operator
Our next question comes from the line of Eileen Flowers [ph] of Jefferies & Company. You may proceed.
Eileen Flowers
Analyst
Hi, it is Eileen dialing in for Eun Yang this evening, can you talk about whether you anticipate a 10 month interim review for mipomersen at the FDA? Thanks. Jefferies & Company: Hi, it is Eileen dialing in for Eun Yang this evening, can you talk about whether you anticipate a 10 month interim review for mipomersen at the FDA? Thanks.
Stanley Crooke
Management
Did you understand the question Lynne?
Lynne Parshall
Chief Operating Officer
Yes, the question is what is the length of the review, I think until we file and have a chance to talk to the agency about the review time, it is probably premature to contemplate that.
Eileen Flowers
Analyst
Okay, thank you. Jefferies & Company: Okay, thank you.
Operator
Operator
Our next question comes from the line of Ted Tenthoff of Piper. You may proceed. Ted Tenthoff - Piper Jaffray & Co: Great. Thank you very much on the good news update looking forward to a busy fourth quarter. I'm actually out in San Francisco at the liver meeting, and one of your competitors, Santaris is going to be reporting some data on miR-121 in HCV. And maybe you could give us some update on what the litigation there is and also on the good news that has been coming out of Regulus of late?
Stanley Crooke
Management
There is no update on the litigation. It is our belief that the miR-122 we own, we and Regulus and that is based on the patents that we have. So we are quite excited to see the positive data that Santaris is generating, and we will pursue the appropriate steps to assure that are patents are in force as we always have.
Stanley Crooke
Management
Thanks, thanks. And we saw that nice paper out of Regulus recently. Any other updates from those other things [ph]?
Stanley Crooke
Management
Making good progress. Understanding how the drugs work, beginning to understand the microRNA pathway and how to exploit it to get a therapeutic index that is appropriate. So, we are very pleased with where Regulus stands, and we are very pleased with the patent position that we hold. Ted Tenthoff - Piper Jaffray & Co: Great. Thanks for the update. It has been exciting progress.
Stanley Crooke
Management
And as Lynne mentioned, I think we’re very excited to see the NDA going in this month for Kynamro. That is another big event for us, and so it makes for an exciting fourth quarter and a great year for us. Lynne, do you want to add anything to the questions about litigation?
Lynne Parshall
Chief Operating Officer
No.
Operator
Operator
(Operator instructions) And our last question comes from Charles Polsky of William Harris Investors. You may proceed.
Charles Polsky - William Harris Investors
Analyst · William Harris Investors. You may proceed
Hi, Lynne and Stan. Thanks for taking the question, and it is a small question, so as it relates to dosing mipomersen three times a week, is there any accumulated data on patients in other trials maybe who have been dosed three times a week and does the smaller dosing tend to result in less injection site reactions that are accumulated patient treatment you have so far?
Stanley Crooke
Management
Yes, Charles. We did a study where we compared daily, three times a week and weekly dosing for both B-100 reduction and LDL reduction, and other lipid reduction, as well as tolerability and injection site reactions. And as you would expect, as you go to lower doses you have less injection site reactions. We are not very concerned about the injection site reactions, weekly or whatever. So yes, as you go to lower doses administered more frequently, you have less injection site reactions, but of course you are giving more injections. And so it is a balance. We think some people will like giving themselves smaller injections three times a week, and we think some will like giving themselves a larger injection once a week and will just depend on the patient. And the whole goal here is to provide improving presentations of the drug and additional options for patients, so that each patient can find his best way of getting the drug. We are not doing daily in this study because we want to continue to look at the proper presentation of daily administration, the right injector and all that, and so that is taking some additional time. But eventually I would expect that we will also offer daily injections as well. But the data are very clear. As you would expect, the pharmacokinetics of the drug support weekly administration at one dose, thrice weekly at another dose, and daily injection at a one seventh of the weekly dose just as the pharmacokinetics will tell you.
Charles Polsky - William Harris Investors
Analyst · William Harris Investors. You may proceed
Thanks Stan.
Operator
Operator
And we have no further questions at this time. I would now like to turn the call back over to Dr. Crooke for any closing remarks.
Stanley Crooke
Management
Well, thank you very much. We think that we are in a very exciting moment for Isis. And in this call, we have told you that Genzyme and we will be filing our NDA this month for Kynamro. We have told you that the FDA has approved our special protocol assessment for the study that we believe will allow Kynamro to advance to much larger markets. We told you that we will be reporting data, important clinical data on multiple drugs over the next quarter, and we look forward to providing all of that information to you as this next quarter unwinds, and to summarize it and contextualize it for you in January 5 in New York. Thank you very much.
Operator
Operator
Ladies and gentlemen, that concludes today’s conference. Thank you so much for your participation. You may now disconnect. Have a great day.