Gretchen Schweitzer - IR, MacDougall Biomedical Communications Jörn Aldag - Chief Executive Officer Dr. Charles Richard - SVP, Research and Development, Neuroscience.

Michael Schmidt - Leerink.

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..

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 place undue reliance on these forward-looking statements, and we assume no obligation to update these forward-looking statements even if new information becomes available in the future. I would now like to turn the call over to uniQure’s Chief Executive Officer, Jörn Aldag..

Thank you, Gretchen. Welcome and thank you all on the call and webcast for joining us for review of our third quarter activities and updates on our pipeline products.

Many of you were at ASH or tuned into our R&D Day last month in New York City where we introduced our patients’ first strategy and our three area therapeutic heads who are spearheading that strategy, Charlie Richard in Neuroscience; Deya Corzo in Liver/Metabolism; and Patrick Most in Cardiovascular.

In addition, we heard from Harald Petry, our Chief Scientific Officer about our strategy to maintain our leadership in gene therapy technologies and manufacturing. Each presented detailed descriptions of our programs in their respective areas which you can find online through our website at www.uniqure.com.

In the third quarter and subsequently we received an additional $53 million in our cardiovascular collaboration with Bristol-Myers Squibb.

Included in the total was a 15 million target designation fee triggered by BMS’ selection of three new collaboration targets, in addition to S100A1 for congestive heart failure and the purchase of an additional 1.3 million ordinary shares of uniQure priced at $29.67 per share.

The purchase price represented approximately 26% premium over uniQure’s closing price per ordinary share on August 7, 2015. BMS now owns 9.9% of uniQure’s outstanding ordinary shares. To-date, we have received a total of $140 million from BMS in the context of the agreement that was signed about eight months ago.

On the corporate front, we announced in July the appointment of Charlie Richard, M.D., Ph.D., to the position of Senior Vice President, Research and Development, Neuroscience, to lead the Company’s growing portfolio of gene therapies targeting neurological diseases, including the development programs for gene therapies for the treatment of Sanfilippo B syndrome and Parkinson’s disease as well as preclinical programs in Huntington’s disease and other rare CNS disorders.

Also in July, Deya Corzo, M.D. who joined the Company in spring 2014 as Vice President, Medical Affairs, was promoted to Senior Vice President, Therapeutic Area Head, Liver/Metabolism.

Deya is a clinical geneticist and a pediatrician by training and leads the efforts for the hemophilia B clinical trial program as well as research and development for hemophilia A and other liver diseases, both are available on this call. Last week, Hans Preusting, Chief Business Officer, resigned to pursue new entrepreneurial opportunities.

He will remain the consultant for the Company following his departure in January 2016. Hans has been with the Company for nine years and has played an important in bringing uniQure to where it is now.

He’s been a great guy to work with and we will not forget his significant contributions in building our manufacturing expertise and then in forging important business and scientific alliances. I personally thank him and wish him the best for his next adventure.

And now our hemophilia B Phase 1/2 program, we have completed dosing the first low dose cohort of five patients. So that’s complete. This product candidate consists of our proprietary AAV5 vector carrying a vial size human Factor IX gene cassette that we have exclusively licensed from St. Jude’s Children’s Research Hospital in Memphis, Tennessee.

We promised the first look at the results to run the end of this year and are now planning to provide preliminary top line readout of data early January 2016, just before JP Morgan. We will show 12-week over a follow-up data including six expression levels and safety data from the first two patients of that study.

Any additional data from the remaining patients in the low dose cohort or other measures of the trial will be presented at a future medical meeting which we expect to happen in the first half of 2016, could be ASGCT or ISTH or EHA.

On September 19, Professor Marc Tardieu, representing in consortium of French Institutions including the AFM foundation for Muscular Diseases, Institut Pasteur, INSERM, and uniQure’s collaborators presented the results from a Phase 1 study of our Sanfilippo B program.

The 12 months follow-up of patients treated with our gene therapy product provided the first clinical validation of gene therapy in a lysosomal storage disease.

It proved that our AAV5 vector can effectively deliver a target gene into the CNS and achieve durable expression of NAGLU protein of 14% to 17% at 12 months post treatment in the cerebrospinal fluid.

We’re incredibly excited that the trial also demonstrated that incremental cognitive development was maintained over a full-year in all four patients aged 20 months to 53 months at study onset. From the patient perspective that is the most critical outcome of any such study.

During our R&D Day, we discussed initiating a further analysis of patient samples to measure glycosaminoglycans, GAG levels in the CSF. Last week, Professor Tardieu reported at a conference in Geneva that a decrease of GAG levels in the CSF was not detected.

This result correlates with a previously published study by Ausseil and colleagues using uniQure’s AAV5 NAGLU gene therapy vector in a canine model of Sanfilippo B. In that study, durable NAGLU expression resulted in significant and widespread reductions in brain tissue GAGs but also not in CSF GAGs.

Given the encouraging positive clinical results, we are now moving forward to prepare the pivotal trial and the future pathway with regulatory authorities. We will also closely monitor our patients to the end of the 30-month study near to year end 2016.

Sanfilippo B syndrome represents a high unmet medical need and gene therapy holds significant premise for these patients. We expect to be able to leverage this proof of concept into other diseases including lysosomal storage diseases affecting the brain.

Turning now to Glybera, on October 28, 2015, the Charité University Clinic in Berlin, Germany, announced the treatment of the first patient with Glybera as a commercially-available gene therapy.

This represents a very special event for all of us at uniQure and for me personally as this first commercial sale of a regulated gene therapy product in the western world marks the end of a long validation journey and is a major milestone in what we believe is the beginning of a new era in medicine.

That said, we do need to face the reality that the disease that Glybera treats familial lipoprotein lipase deficiency or LPLD is extremely rate, making eligible patient identification challenging. In addition, reimbursement decisions by health authorities in various countries in the EU have been lengthy and are not yet definitive.

This situation is unique to Glybera. It should not be extrapolated to other gene therapies. As a result of these developments, we have made a number of tough decisions regarding the future of Glybera. We have prioritized investments in other programs with high unmet medical needs in our pipeline and decided not to pursue U.S.

regulatory approval for LPL deficiency. This is mostly due to the additional clinical studies that the FDA indicated will be required.

We will continue to meet all of our obligations to patients, to our partner Chiesi and to EMA including the execution of the post-approval Phase IV clinical trial and the continuation of the patient registry to assess the long-term clinical benefit of Glybera.

We have also recorded a non-cash impairment charge in the third quarter of 11.6 million associated with certain intangible assets related to Glybera on our balance sheet. The impairment was due to a reduction in the estimated number of patients we expect to be treated with Glybera in the EU territories and in the U.S.

The approval of Glybera has pushed the entire gene therapy industry back into the limelight. It is also a great validation of our competence in developing manufacturing and getting gene therapies approved.

Having been successful in developing and gaining regulatory approval for Glybera in the EU against the historical backdrop of concerns about the safety and efficacy of gene therapy, it is an achievement that will remain forever associated with uniQure and one that we are very proud of having achieved.

Nevertheless, we feel that it is important for us at uniQure to focus our efforts on our developing pipeline of gene therapy programs that address patient populations for which there are no disease modifying treatment options like Sanfilippo B, Huntington’s disease and Parkinson’s disease and for those where gene therapy may fundamentally change the treatment paradigm and allow patients to be free of chronic therapy like hemophilia A and B among others.

All of these programs have well defined and measurable endpoints to provide a clear cost benefit assessment to facilitate future approval, reimbursement, and adoption. To conclude, we have made tremendous progress on advancing our pipeline of programs this year and particularly in the last quarter.

As we conveyed on our R&D Day, we are investing our resources into technology, manufacturing, and a pipeline of programs that we believe will bring the promise of gene therapy to patients in multiple indications over the next five years. Thank you for joining us in the call. We will now take questions..

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

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..

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..

Okay, great. Thanks.

And then, are you still on track to begin the second dose cohort in January?.

Yes..

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..

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..

Thank you. [Operator Instructions] There are no further questions from the audience..

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..

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