Earnings Labs

uniQure N.V. (QURE)

Q3 2015 Earnings Call· Mon, Nov 30, 2015

$17.97

+0.39%

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Transcript

Operator

Operator

Welcome to the uniQure Conference Call. Before I hand the call over to uniQure, please be aware that after the discussion from management, there will be a question-and-answer session. [Operator Instructions] I will now turn the call over to Gretchen Schweitzer who will make introduction.

Gretchen Schweitzer

Analyst

Thank you all for joining us today to discuss updates on several of the programs at uniQure. The conference call is being recorded and will be available on the Company’s website after this live call. Please be aware that there are no slides accompanying this discussion. We will begin the call with a statement from uniQure’s CEO, Jörn Aldag after which we will open the call to questions. The Company’s financial results are provided in the press release and Form 6-K filed with the SEC today. We will not be presenting these results on the call, although Matt Kapusta, Chief Financial Officer is on the call to answer any of your financial questions. In addition to Mr. Aldag and Mr. Kapusta, Dr. Christian Meyer our Chief Medical Officer; Dr. Deya Corzo, Senior Vice President, Therapeutic Area Head, Liver/Metabolism; and Dr. Charles Richard, Senior Vice President for Research and Development, Neuroscience will also be available to answer questions during the Q&A. Please note that we are holding this call to provide a forum for discussion and Q&A around the multiple programs on which we are providing an update. We do not plan to hold regular quarterly calls around financial results in the future. I will now read the safe harbor statement. This presentation and discussion contain forward-looking statements. Our actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, risks associated with collaboration arrangement, our and our collaborators’ clinical development activities, regulatory oversight, product commercialization and intellectual property claims, as well as the risks, uncertainties and other factors described under the heading Risk Factors in uniQure’s 2014 Annual Report on Form 20-F filed with the Securities and Exchange Commission on April 7, 2015. Given these risks, uncertainties, and other factors, you should not…

Operator

Operator

[Operator instruction] Thank you. We will now take our first question from Michael Schmidt from Leerink. Please go ahead your line is open.

Michael Schmidt

Analyst

On the hemophilia B Phase 1 study, I was wondering what’s behind the decision to only present data on two patients in January as opposed to all five that have been treated to-date. And then, I have a couple follow-ups. Jörn Aldag: Michael, the key point here is that we are in a study in which we are trying to show safety and initial efficacy, so indications for expression levels. What is important for us is that we have three months data. And looking at the time point at which the initial patients were dosed, the first two patients will be beyond the three months data point, 12 weeks and therefore we feel comfortable to present that data early in January. The additional three patients dosed, obviously already generate results over time but we are not yet certain as to the 12 months threshold and therefore decided that we would present that data only at scientific conferences later in the first half.

Michael Schmidt

Analyst

Okay, great. Thanks. And then, are you still on track to begin the second dose cohort in January? Jörn Aldag: Yes.

Michael Schmidt

Analyst

Great, and then on the Sanfilippo B study, I was wondering, so you mentioned the 30 months follow up update at year-end ‘16. I was wondering if there might be any other updates before that. For example, on all four patients for example, I think you talked about potentially weaning off those patients of the immunosuppressive therapy. I was wondering if there would be any additional updates before year-end ‘16. Thanks.

Dr. Charles Richard

Analyst

This is Charlie from the CNS therapeutic area. I think the next official time point in which we’re doing comprehensive assessments for all the patients are at the 30-month time point. And so the patients are staggered and they’ll be coming in to the major center in Paris for these evaluations, which as you know, include the neurocognitive assessments, the CSF biochemical measurements, and the MRIs for brain atrophy. So, I think the most -- admittedly, there is only four patients in the study. So, the best analysis that we can provide for people would be at the last patient, last visit for all the patients that have completed all the assessments at this last visit. And that’s going to occur about this time next year, so then we’ll need a little bit of time to complete the analysis of the MRIs and the CSF for instance and then be able to report on that. It is true that the patients are followed in phone calls over the year to the principal investigator. As you know, not all of them live close to Paris. But we just don’t think it’s a very rigorous scientific guidance to provide to people if they aren’t undergoing, for instance, some more rigorous neuropsychological testing to provide anecdotal evidence on how they’re doing over the next year. So that said, the end of the year, about this time next year, we’ll have all that data and to be able to report to you.

Operator

Operator

Thank you. [Operator Instructions] There are no further questions from the audience. Jörn Aldag: Well, then, thank you very much. If there are no further questions, we’re concluding the call. Some of you we’ll be seeing in the course of this week in New York. So, we’re looking forward to that and good bye everyone. Thanks a lot.

Operator

Operator

Thank you. This will conclude today’s conference call. Thank you for your participation. Ladies and gentlemen, you may now disconnect.