ISIS Pharmaceuticals' CEO Presents at Review of CHMP Opinion on KYNAMRO Conference (Transcript)

Dec.14.12 | About: Ionis Pharmaceuticals, (IONS)

ISIS Pharmaceuticals, Inc. (ISIS) Review of CHMP Opinion on KYNAMRO Conference Call December 14, 2012 8:30 AM ET

Executives

Stanley T. Crooke – Chairman and Chief Executive Officer

D. Wade Walke – Executive Director, Corporate Communications and Investor Relations

Paula Soteropoulos – Vice President and General Manager-Genzyme’s Cardiovascular Business

Analysts

Jim Birchenough – BMO Capital Markets

Salveen K. Richter – Canaccord Genuity, Inc.

Stephen D. Willey – Stifel, Nicolaus & Co., Inc.

Nicholas A. Bishop – Cowen & Co. LLC

Eun K. Yang – Jefferies & Co., Inc.

Operator

Good day, ladies and gentlemen and welcome to the Isis Pharmaceuticals’ Conference Call to review the KYNAMRO’s CHMP opinion. As a reminder, this call is being recorded for replay purposes.

I would now like to hand the call over to your host for today, Dr. Stanley Crooke, Chairman and CEO. Please proceed.

Stanley T. Crooke

Good morning everyone, and thanks for joining us on the call today. What we will do is, review the opinion made by the European Medicines Agency Committee for Medicinal Products for Human Use known as CHMP. and then we will open up the call for Q&A.

Joining me on today’s call are Paula Soteropoulos, who is Vice President and General Manager, Cardiovascular for Genzyme Corporation and for Sanofi company; Lynne Parshall, Chief Operating Officer and CFO; Richard Geary, Senior Vice President of Development and Wade Walke, Executive Director, Corporate Communications and Investor Relations.

Wade, would you please read our forward-looking language statement?

D. Wade Walke

Thanks, Stan. A reminder to everyone, that this webcast includes forward-looking statements regarding the development activity in therapeutic and commercial potential and safety of KYNAMRO.

Any statements describing Isis’ goals, expectations, financial or other projections, intentions or beliefs including the planned commercialization of KYNAMRO 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 as 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.

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, 2011 and Isis’ most recent Quarterly Report on Form 10-Q, which are on file with the SEC. Copies of these and other documents are available from the Company.

Now I’ll turn the call back over to Stan.

Stanley T. Crooke

Thanks, Wade. Let me begin by saying that we are of course, disappointed by the negative opinion of the CHMP on the marketing authorization of KYNAMRO for the treatment of homozygous FH patients. Genzyme and we remain committed to advancing the development program for this important new therapy for patients who are living with the disease puts them at extreme cardiovascular risk with significantly high LDL cholesterol levels. Given the need of these patients and the limited therapeutic options, we believe KYNAMRO should be available to these patients and we’re working together to reach that goal.

Now I’ll comment on that a bit further. I’ll turn the call over to Paula.

Paula Soteropoulos

Thank you, Stan. We’re also very disappointed by the CHMP’s opinion. nevertheless, we remain very committed to bringing KYNAMRO to patients and families who have a real need for new treatment options. And we plan to request a re-examination of the opinion. During this process, we are committed to working closely with the CHMP to address the committee’s concerns. We expect to have an opinion from the re-examination in the second quarter of 2013.

Looking beyond Europe, one of Genzyme’s strengths, which is bolstered by Sanofi’s resources is our global presence. As such we are making progress in our plans for the global commercialization of KYNAMRO that include obtaining regulatory approval for homozygous FH in markets beyond the U.S. and Europe.

and finally, we continue to prepare for a successful launch of KYNAMRO in United States. following a positive FDA Advisory Committee vote in October, we expect to hear from the FDA by our PDUFA date of January 29 regarding KYNAMRO’s approval in the United States.

And I will turn the call back to Stan.

Stanley T. Crooke

Thanks, Paula. So we believe that KYNAMRO represents an important new therapy for patients who are at significant risk of cardiovascular events. despite being treated with our currently available therapies, and we remain very enthusiastic about the potential benefits that this drug may bring for these patients. Genzyme and we are disappointed with this opinion and the concerns expressed by the CHMP, but we are still optimistic that KYNAMRO will be approved in Europe.

Even though I cannot address the specific concerns expressed by the committee while Genzyme begins the process of re-examination, I can say that each of the issues raised by the CHMP was thoroughly reviewed by the FDA, and the details of their review and of course, the details of our response to questions related to these issues are available in the briefing documents that were prepared by the Advisory Committee.

We also do not believe that today’s opinion from the CHMP; it should have a negative impact elsewhere. and as Paula mentioned, the Genzyme is moving forward with plans to market KYNAMRO in other markets around the world including Japan.

With that, again, I want to thank you for your interest and open the call up for Q&A. Operator, if you can please set us up for questions.

Question-and-Answer Session

Operator

(Operator Instructions) Your first question comes from the line of Jim Birchenough, BMO Capital. Please proceed.

Jim Birchenough – BMO Capital Markets

Yeah, hi, guys. Thanks for hosting the call. A couple of questions, just first on the re-examination process, I’m just wondering if Paula could comment on what the precedent is for initial decisions being reversed on a re-examination, either with their own products or more broadly? And then I have got a couple follow-ups.

Paula Soteropoulos

Sure, Jim. We have had one product that has been reversed, and I can’t speak specifically broadly to our CHMP opinion that have been reversed, but they are definitely other products that had been reversed and the most recent one, of course, is UniQure’s gene therapy treatment.

Stanley T. Crooke

I think…

Jim Birchenough – BMO Capital Markets

Just on the cardiovascular risk concerns, as Stan suggested, that was thoroughly vetted by FDA. Is it the case that there is additional data that CHMP hasn’t considered or are you just trying to get them to take a different perspective on data they’ve already seen?

Paula Soteropoulos

So with regard to the specific concerns in the CHMP, we are not going to discuss further the dialogue that we’re having with the authorities. we’re confident that we’ll be able to address the committee’s concerns and really look forward to continuing the conversation with the CHMP during the re-examination period.

Jim Birchenough – BMO Capital Markets

And just a final question, on the FOCUS FH study, what’s the status of that study in terms of enrollment and expected timelines for that data? Thanks.

Paula Soteropoulos

Stan, do you want me to take that as well?

Stanley T. Crooke

Enrollment has really picked up. so we’re very pleased with where the enrollment process is. and to-date, we’re very pleased with the performance of KYNAMRO with regard to all of the things that we would be able to see right now such as dropouts and that sort of thing. The only other thing I would add Jim, is that as we looked at recent experience and I think there were four re-examinations last year and two were – for two of them, the drug was approved.

Jim Birchenough – BMO Capital Markets

Okay, great. Thanks, very helpful.

Operator

Thank you. Your next question comes from the line of Salveen Richter, Canaccord. Please go ahead.

Salveen K. Richter – Canaccord Genuity, Inc.

Thanks for taking my questions. I am just wondering what you can do to address the concerns here on discontinuation and the liver concerns as well around monitoring?

Stanley T. Crooke

Well, we think we have addressed those concerns with responses we’re going to provide additional clarification and we really can’t say anymore specifically than that. Paula, do you want to add anything or subtract anything from that?

Paula Soteropoulos

No, not specifically.

Salveen K. Richter – Canaccord Genuity, Inc.

Okay. And then what does this mean for the broader indications like severe and heterozygous FH? Should we just assume that there won’t be any filings worldwide in these broader populations going forward?

Stanley T. Crooke

No, we are still committed to moving KYNAMRO forward. And we think is, this benefit justifies that and FOCUS FH is designed to provide support for broadening the indication.

Salveen K. Richter – Canaccord Genuity, Inc.

Great, thank you.

Operator

Thank you. Your next question comes from the line of Stephen Willey, Stifel, Nicolaus. Please go ahead. Your line is now open.

Stephen D. Willey – Stifel, Nicolaus & Co., Inc.

Yeah. hi, I just had a quick question I guess with respect to if indeed the FDA just come back with the favorable decision next month, and you guys are in the position to maybe margin price, how having this, potential uncertainty within Europe would kind of complicate in the initial pricing decision in the United States?

Stanley T Crooke

Paula?

Paula Soteropoulos

Yes. So our pricing margins, we wouldn’t talk about until we’re ready to launch, and our pricing will be responsible pricing for the disease and the treatments and really independent of this opinion, because we are moving forward with this product developments globally and we will continue to look at our pricing with that respect.

Stephen D. Willey – Stifel, Nicolaus & Co., Inc.

So there hasn’t been any kind of expectation then with respect to kind of the total market opportunity or total addressable patient population with respect to KYNAMRO, and therefore there hasn’t really been any kind of formal reevaluation of price at this point, or is that still a work in progress?

Paula Soteropoulos

Well, obviously pricing is the work in progress right up until the time that we launch.

Stephen D. Willey – Stifel, Nicolaus & Co., Inc.

Okay. And then with respect to the monitoring of the liver fat, Stan, I know in the briefing documents, I think they essentially raised the same concerns regarding the lack of correlation between liver fat increases and enzymatic increases. And I think, in the slide deck, you mentioned using ultrasound as a tool for monitoring. I’m just kind of curious as to what your thoughts are around the sensitivity of ultrasound and how adaptable something like that might be?

Stanley T Crooke

I think monitoring of KYNAMRO will prove to be just monitoring ALPs and individual physicians who have the concerns will use the ultrasound or MRIs or what not. And our strengths with ultrasound is that they are helpful of not as precise as we showed serial MRIs are. But certainly I think if you ask 20 experts, you will find that they think as they do provide help.

Stephen D. Willey – Stifel, Nicolaus & Co., Inc.

Okay, thanks.

Operator

Thank you. Your next question comes from the line of Nicholas Bishop, Cowen & Company. Please go ahead.

Nicholas A. Bishop – Cowen & Co. LLC

Hi, good morning. I have a question about the nature of the data that was submitted for the review. One is the CHMP’s Q&A letter says that there were two main studies submitted in support of this application, the homozygous FH and the severe heterozygous FH. Is that accurate, you only submitted those two studies? And then the second part of my question is could you tell me what the two-year retention rate was for homozygous FH patients only and also severe heterozygous FH patients?

Stanley T Crooke

We submitted all four Phase 3 studies to the European authorities, I think what they’re referring to is that those two studies, remember the indication we were filing for was homozygous severe. And so those two studies are the primary studies for those two indications just as homozygous FH study is the primary indication in the U.S. because our indication with homozygous and three other Phase 3 studies were supported.

I don’t recall sitting here what the discontinuation rate in CS in the long-term open-label study for homozygous versus the other populations was. But I would again refer to the briefing document and the answers that we’ve given previously that the study was not designed to evaluate long-term retention and that there were many administrative issues that resolved in patients leaving the study early and in addition to people did actually drop out. We think actually that study is very supportive of the long-term ability of KYNAMRO to be effective. And it’s continuing and we’ll do our best to clarify why we believe, what we believe.

Nicholas A. Bishop – Cowen & Co. LLC

Okay, thanks. And then I have one other on the process so far. Did you have an oral defense at any point during this review? And also are you aware of what the vote was between the rapporteurs, members of the CHMP?

Stanley T Crooke

Paula, do you want to add?

Paula Soteropoulos

Yeah, we don’t discuss the details of our regulatory interactions in general. And so that’s something again, that we keep that dialogue with the European authorities is something that’s between the company and the authorities.

Nicholas A. Bishop – Cowen & Co. LLC

Can you say whether the vote was close in terms of approval versus not or was it skewed pretty far the negative?

Paula Soteropoulos

That detail isn’t normally shared.

Nicholas A. Bishop – Cowen & Co. LLC

It’s not shared with the sponsor or you won’t share it with us?

Paula Soteropoulos

Again, we are not going to be talking about the details, the specific details.

Nicholas A. Bishop – Cowen & Co. LLC

Okay, fair enough. Thanks a lot for answering my questions.

Operator

Thank you. Your next question comes from the line of Eun Yang, Jefferies. Please proceed.

Eun K. Yang – Jefferies & Co., Inc.

Thanks very much. So in the press release, and today’s CHMP opinion is on homozygous patients, did you withdraw severe heterozygous FH indication during the process?

Stanley T Crooke

Yes. What we did was narrow the indication. I think we mentioned that in the conference call. Lynne, do you want to, Paula?

Paula Soteropoulos

Did you want Lynne to start?

Stanley T Crooke

No, go ahead.

Paula Soteropoulos

Again in the process of the discussions with the CHMP, we further and I think that Isis did discuss this in one of their calls, part of their discussion was looking really at the risk benefit of the most severe population homozygous FH and analogous to the U.S. and looking at severe from a longer-term perspective of additional data that we have. So it’s not that we formally withdrew, it’s part of the conversation.

Eun K. Yang – Jefferies & Co., Inc.

And then you mentioned on the call earlier that PDUFA date in the U.S. is a little more than a month away. Based on the discussions that you are having I’m assuming with the FDA, are you confident that you do [basically] approval on the PDUFA date?

Paula Soteropoulos

So we are, I can tell you by our actions, we are aggressively preparing to launch and are nearing launch-ready mode in anticipation of that January 29 PDUFA date.

Eun K. Yang – Jefferies & Co., Inc.

Okay, thanks.

Operator

Thank you. Sir, you have no questions at this time. I would now like to turn the call over to Dr. Stanley Crooke for closing remarks.

Stanley T. Crooke

If there are no more questions, I will thank everyone for their interest. We are, of course, disappointed, but we and Genzyme are committed to see this process through, and we believe these patients with homozygous FH very much deserve KYNAMRO and we are going to work to bring the drug through them. Thanks very much.

Paula Soteropoulos

Thank you.

Operator

Thank you for your participation in today’s conference. This concludes the presentation. You may now disconnect. Good day.

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