Prosensa Holding (NASDAQ:RNA)
Q2 2013 Earnings Conference Call
August 28, 2013 08:00 ET
Celia Economides - Director, IR
Hans Schikan - CEO
Berndt Modig - CFO
James Gordon - JPMorgan
Joseph Schwartz - Leerink Swann
Chris Marai - Wedbush
Jan De Kerpel - KBC Securities
John Savin - Edison Investment Research
Good day and welcome to the Prosensa quarter results presentation the conference call. (Operator Instructions). At this time I would like to turn the conference over to Ms. Celia Economides, Director of Investor Relations. Please go ahead.
Thank you operator and thank you all for joining us. On behalf of Prosensa I would like to welcome everyone to our first quarterly earnings call. With me today are Hans Schikan, Chief Executive Officer, Berndt Modig, Chief Financial Officer and joining us for the Q&A are Giles Campion, Chief Medical Officer and Luc Dochez, Chief Business Officer.
Earlier today, we issued a press release containing results for the second quarter of 2013 which is available on our website at Prosensa.com and our 6K was filed with the SEC earlier this morning.
We will our full second-quarter interim financial statements and management discussion and analysis later on today. During today's call we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, using these statements about our future expectations, clinical development and regulatory timelines. The potential success of our product candidates and financial projections. Actual results may differ materially from those indicated by these statements.
Forward-looking statements during the call include statements around our exon-skipping drug pipeline and financial position. Such forward-looking information involves risks and uncertainties that could significantly affect expected results. These risks and uncertainties are discussed in the company’s SEC filings, including but not limited to, the company’s Form 6-K containing Q2 press release and certain sections of the company’s registration statement on Form F-1. In addition, any forward-looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. While we may elect to update these forward-looking statements at some point in the future, we specifically disclaim any obligation to do so even if our views change.
With that let me pass the call over to Hans Schikan.
Thank you Celia and good morning and good afternoon everyone. As Celia stated this is our first quarterly earnings as a newly public company. I should can imagine it's an incredibly exciting time for us. I like to begin by giving you a brief overview of Prosensa. Many of you are aware of our story thus we want to remind everyone that even though we now are a public company our mission has not changed. It's still about innovation, RNA modulation and meeting the next medical needs. But most importantly we’re here for patients particularly those affected by rare genetic disorder Duchenne Muscular Dystrophy or DMD, our clinical portfolio of RNA-based product candidates is focused on the treatment of DMD.
RNA modulating therapies provide a powerful tool for targets modulation of gene expression. Our unique proprietary technology platform employee’s single-stranded RNA-based antisense oligonucleotides to correct mutated messenger RNA goals and life-threatening diseases.
DMD is one of the most prevalent rare genetic diseases globally affecting up to one in 3500 boys and invariably fatal. There is currently no proven disease modifying therapy for DMD. We have transformative second-quarter culminating with our successful IPO at the end of June. We issued 6.9 million shares at $13 per share and raised gross proceeds of almost $90 million.
This provides a substantial base to support the developments of our rapidly advancing DMD portfolio and other business objectives going forward. We are incredibly proud of our investigation of DMD portfolio which now includes six identify compounds, all of which have received orphan drug status in the United States and the European Union. In addition Drisapersen as the lead investigational product candidate, which has been exclusively licensed to GlaxoSmithKline also received orphan drug designation in Japan, reinforcing the potential global reach of the program.
The compounds used innovative technique called exon-skipping to provide personalized medicine approach to treat different populations of DMD patients, depending on their genetic mutation. Our exon-skipping technology is based on the interference some exon-inclusion signals, (inaudible) or pre-messenger RNA in order to induce a skipping of targeted exon from the mature messenger RNA, and to restore the level of functional transcripts. The most advantaged product candidate of our DMD portfolio drives a person can address a variety of mutations in the dystrophin gene such as the release of exon-50 or exons 48 to 50 that affects an estimated 13% of patients with the disease. Drisapersen is in Phase III developments with our licensee GlaxoSmithKline.
GSK funds all developments, manufacturing and commercialization for Drisapersen and we have a very collaborative relationship with the company. Since GSK has ultimate responsibility for Drisapersen some questions related to this investigational medicine can be addressed during our call however, there may be questions that will need to be referred to GSK for a response.
We believe that Drisapersen which was recently granted breakthrough therapy designation by the U.S. Food and Drug Administration could offer a broad number of amenable DMD patients and an important much-needed treatment option and we’re dedicated to advancing the rest of our portfolio to help the smaller sub-populations of boys with this devastating neuromuscular disease.
Breakthrough therapy designation which was created by the FDA Safety and Innovation Act last year is reserved for a drug that it's intend is to treat serious or life-threatening condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvements on the clinically significant end point over available therapies if any.
According to it (inaudible) made available by the FDA last Tuesday in the period between October 1, 2012 and the 23rd of August, 2013. The agency has received in total 82 requests for breakthrough therapy designation, 25 of which were granted and 32 were denied with an order of 25 pending. Breakthrough Therapy Designation has all the features of a fast track designation including actions to expedite developments and review. Breakthrough Therapy Designation also enables intensive guidance from the agency in drug developments and provides access to organizational committee in evolving senior managers.
GSK in collaboration with Prosensa is working closely together with regulators and all the stakeholders in an effort to make this product candidate available to patients as quickly as possible. We look forward to an incredibly busy and (inaudible) remainder of the year. s news flow continues (inaudible) program. Today more than 300 patients have participated in clinical studies of Drisapersen and more than 50 trial sites in 25 countries.
In April 2013 Dr. John Kraus of GSK present his results for the so-called 117 or the amount (ph) two clinical trial. This is a Phase II double-blind exploratory parallel group, placebo-controlled clinical study to assess two dosing regimens of Drisapersen for efficacy, safety, vulnerability, and pharmacokinetics in ambulant subjects would be in these.
We were very pleased that the primary endpoints in this study for the dosing regimen of 6 milligrams per kilogram body weight once per week was met. Statistically and clinically meaningful difference 35 meters in the 6 minute walk test between the continuous dosing regimen of 6 milligrams per kilogram body weight per week and placebo was achieved at 24 weeks. Several orphan drugs have obtained regulatory approval based on a change in the 6 minute walk test of approximately 30 meters.
Earlier this week new evidence was published by our two peer reviewed articles in the medical journal Muscle and Nerve on the 6 minute walk test by Dr. Craig McDonalds and co-authors. Their data further validates the use of a 6 minute walk test as a primary end point to measure disease progression and walking ability in ambulatory DMD trials. Moreover the finding supports the clinically meaningful range to be in the range of 20 to 30 meters which can serve as the targeted treatment effects in 12 months trials in ambulatory DMD.
Preliminary results which have been posted on GSK’s clinical study register suggest that treatment for Drisapersen was in general associated with increased levels of the dystrophin expression when compared with pretreatment levels.
Off the 18 patients in the continues treatment arm on 6 milligrams per kilogram per week, 13 showed an increase in baseline dystrophin by at least one testing methods, namely, immuno fluorescence assay or the (inaudible) or exon-skipping progress out of 18 placebo patients, 17 did not show an increase from baseline.
Regarding safety there were no significant safety concerns related to Drisapersen identified in this 177 study. Injection side reactions and renal adverse events, including subclinical proteinuria were observed in all treatment arms including in the placebo arm but occurred more frequently in the continuous and intermittent treatment arms. Drisapersen was generally well tolerated and no subject discontinued treatment.
The next update of Drisapersen is approaching at the end of September, where new data from the open label study on Drisapersen the longest running extension study in DMD patients worldwide will be available at the DIA FDA Oligonucleotide-Based Therapeutics Conference, in Washington, DC from 25th until the 27th of September.
Also at this scientific meeting there will be presentations addressing (inaudible) and renal effects around Drisapersen and antisense oligonucleotide type therapies. Moreover, there will be an update on the overall portfolio of Prosensa DMD product candidates. This data with appropriate scientific context for the 117 Phase 11 clinical trial for Drisapersen are expected to be made available by GSK at the scientific meeting in the fourth quarter.
DEMAND V or DMD114876 is a multicenter Phase II placebo-controlled dose comparison study which is running in 13 sites in the United States. The purpose of this exploratory study is to determine if Drisapersen is effective in the treatment of ambulant boys with DMD.
Two doses of Drisapersen and placebo are used in this study mainly 6 milligram per kilogram per week and 3 milligrams per kilogram per week. Following the treatment periods of 24 weeks the study has a 24 weeks post treatment phase. The results after the treatment period of 24 weeks are expected to be made available and presented by GSK at a scientific meeting in the fourth quarter. Furthermore the study results of the larger Phase III study in 186 patients the so called DMD 114044 or DEMAND III study are expected to be made available by GSK at an appropriate scientific meeting in the fourth quarter of 2013.
This study is a multi-center randomized double-blind placebo control study investigating the once weekly subcutaneous administration of Drisapersen at 6 milligram per kilogram dosing in 186 boys. The primary objective of the study is to demonstrate a mean improvement of 30 meters in the 6 minute walk distance at 48 weeks compared to the placebo. As previously stated the 6 minute walk test is evaluated clinical end-point.
The study runs in 33 sites and the last patients was recently dosed. Outside of Drisapersen we’re very excited about our developments in the study of the natural history of DMD and for other compounds in our DMD portfolio. Together with GSK we have embarked on a global study to evaluate the natural history of DMD.
This study is aiming to enroll 260 patients across the U.S., Europe and Latin America and can be very helpful for comparative purposes for the product candidates which we’re developing in less prevenient patient populations with DMD. During the 10th bi-annual meeting of the European Pediatric Neurology Society, the EPNS in Brussels from September 25 until 28, 2013 we will present more details on this natural history study including the methodology, the study design, the status, the phenotype and the types of the lesions for currently enrolled subjects.
On August 1, 2013 we announced that we’re part of a Pan European Consortium which has be awarded a Framework Program 7, an FP7 research grant of 6 million euro from the European Commission to support the ongoing clinical study of our third novel DMD development candidates grew 45.
The project titles consortium for products across Europe in (inaudible) Scope DMD if expected to run for three years, PRO44our next most advanced product candidate addresses a separate sub-population of DMD patients by using exon-44 skipping and is intended for approximately 6% of all DMD patients. PRO44 is in Stage 1-2 clinical developments in Europe to assess the safety and efficacy of the navigation for DMD patients with a mutation around exon-44 in gene dystrophin protein. Data from this study will be available at the upcoming World Muscle Society Congress from the 1st until the 5th of October in Asilomar, California and expansion study is anticipated to begin during the fourth quarter of this year.
We have four additional compounds that address other distinct sub-populations of DMD patients. Off these PRO45 which may held around 8% of all DMD patients enter clinical trials in the first quarter of 2013. We expect the first patient to be dosed this quarter for PRO53 which is also targeting existing sub-population of 8% of all DMD patients.
PRO52 and PRO55 intended for respectively 4% and 2% of all DMD patients are in advanced pre-clinical development. By year end we expect having four compounds in the clinic for the treatment of DMD. With regard to our agreement with GSK PRO45 is paired with PRO53 and PRO52 is paired with PRO55 because each product candidate in respective pairings addresses a similar size patient sub-population and is at a comparable stage of development. GSK has opt-in rights to one of the two compounds PRO45 or 53 and Prosensa will retain rights to the compound not licensed by GSK. Moreover Prosensa will have opt-in rights to commercialize the compounds licensed to GSK in specified countries within the EU territory. The same principle applies to PRO52 and PRO55.
Last but not least we filed a research program prospect reaching the new and innovative application of our exon-skipping technology platform to specifically target rare mutations in the dystrophin. Most DMD patients (ph) in a hotspot region between exon-42 and 55. Outside this region the subsets of patients who are amenable to exon-skipping become smaller. The prospect program which is still in the discovery phase looks promising aims to help these patients with more rare deletions in their dystrophin gene in an efficient manner.
In August, 2008 European Patent 1619249 or abbreviated 249 was granted to the originator of our exon-skipping technology the Leiden University Medical Center. This patent relates to exon-skipping using oligonucleotide with a certain length directed to the interior of a broad range of exons including exons 43 to 46 and 62 through 53 as well as a number of method claims comprising the use of oligonucleotide. In 2009 AVI Biopharma now Sarepta filed an opposition against European Patent 249 with the European Patent Office and requested revocation of the patents as granted, enlarging amongst orders lack of novelty, innovative step and sufficiency of disclosure.
We on behalf Leiden University Medical Center in turn requested maintenance of the patents on the basis as granted. The opposition division of the European Patent Office residing in all proceedings on the 16th of November, 2011 in Munich in Germany maintained European Patent 249 in amended form. The allowed patents as maintained protects and more (ph) on other things the skipping of exon-51 in the dystrophin gene using the 14 to 40 nerve antisense oligonucleotides as a potential therapy to treat DMD.
We believe that the patents has maintained in the amended (ph) form body opposition decision of the European Patent Office provides protection for our elite product candidate Drisapersen. We and Sarepta both have the right to appeal the written decision of the EPO. We have the (inaudible) the grants of appeals to the EPO and will await the further follow-up from the Board of Appeal at the European Patent Office. Timings are difficult to predict, that is anticipated that this procedure can take some time before it will be finalized.
Finally we also announced changes to our supervisory board, David Mott already member of our supervisory board was appointed Chairman of the Supervisory Board upon completion of our IPO. David who is currently General Partner at New Enterprise Associates brings over 25 years of experience in the biotech industry to Prosensa. I would now like to turn the call over to our Chief Financial Officer, Berndt Modig who will discuss financial highlights from the quarter. Berndt?
Thank you Hans. The consolidated financial statements or Prosensa have been prepared in accordance with IFRS as issued by the International Accounting Standards Board or ISB. The consolidated financial statement are presented in euros which is the company’s functional and presentation currency, therefore all financial numbers that I will present here in this call less otherwise noted will be in euros. After the closing of our IPO, our total outstanding ordinary shares amount to 35.9 million shares and on July 3, 2013 to-date we receive our IPO proceeds Prosensa’s cash and cash equivalent amounted to 94.3 million or a $122.2 million.
Prosensa cash and cash equivalent as of June 30, 2013 were 30.8 million compared to 40.7 million as of December 31, 2012. Our cash consumption excluding cash flows from financing in the first half of 2013 was 10.5 million. Revenue was 2 million for the second quarter 2013 and 4.4 million for the six months ended June 30, 2013 compared to 1.2 million and 3.2 million for the comparable periods in 2012 due to increased license income of 0.5 million and collaboration revenue of 0.3 million for the second quarter 2013 and increased license income of 0.5 million and collaboration revenue of 0.7 million for the six months ended June 30, 2013.
All of our license revenue and collaboration revenue were generated under the GSK Agreement.
Research and development expense was 4.5 million in the second quarter 2013 and 8.6 million for the six months ended June 30, 2013, compared to 2.7 million and 4.5 million for the comparable periods in 2012. Research and development expense mainly increased for the second quarter of 2013 as well as for the six months ended June 30, 2013 due to the commencement of the phase I/II study of PRO45, the preparations for the initiation of the phase I/II study of PRO53 as well as higher intellectual property expenses.
General and administrative expense was 2.1 million in the second quarter of 2013 and 3.9 million for the six months ended June 30, 2013, compared to 0.9 million and 2.0 million for the comparable periods in 2012. The increase is primarily due to expenses related to the IPO for an amount of 0.8 million recorded in the second quarter 2013 and 1.5 million for the six months ended June 30, 2013.
Net loss for the second quarter 2013 was 4.7 million and 8.2 million for the six months ended June 30, 2013, compared to 2.4 million and 5.1 million for the comparable periods in 2012. I will now turn the call back over to Hans for some closing remarks. Hans?
Thank you Berndt and thanks to everyone for joining the call today. We hope that you’re as excited as we are by the position of the company. We have many key milestones in store for the remainder of 2013 and look forward to updating you as we know more. I would now like to turn the call back to the operator who will open the line for questions.
We will now take our first question from (inaudible) of Citigroup. Please go ahead.
This is Christian for (inaudible). Recently data from the 117 study was put on GSKs website with more data about just an dystrophin expression and shows that about 72% of patients had some improvement, can you quantify in terms maybe how robust the actual benefit was?
Indeed GSK has posted on their clinical registry sites the results of the 117 study as available at that moment in time including the results of the dystrophin restoration in the various treatment arms and these results show that based on three different tests there has been a lot exon-skipping to RT-PCR and immuno fluorescence assay and in 13 out of 18 patients treated with a continuous treatment arm there was an dystrophin expression when compared to baseline. So post-treatments compared to pre-treatments as in 17 out of 18 patients that was not the case in the placebo arm, so that illustrates that the general finding that we have in dystrophin expression with this methodology that we can make that statement but in terms of quantifying that’s also with a look at for example functional outcome and that is for more challenging and on all the case.
I had another follow-up, so it seems like you’re now guiding the 876 Phase II study to read out in Q4 and previously it was in Q1 will that get a percent at the same time as the 044 study and any chance to the data will at WMS in October. Thanks.
Okay regarding first and foremost at which scientific conference GSK will be presenting data that has not been announced and disclosed yet. So far the guidance will be that data will be made available at forth coming scientific meeting and WMS is one of those scientific meetings but so far it is not clear whether the presentations can take place there. So guidance on which scientific conference yet. With regard to the 876 study previously it was mentioned that the full study results will be available in the first quarter of 2014, if you look at the fact that last patient will come out of that study somewhere in November of this year then that means that data could be mostly likely available in the first quarter of 2014, that’s more recent guidance is now that the 24, a week’s result after the initial treatment periods of 24 weeks can be made available already at a scientific conference in the fourth quarter on this year. So that a need whereby the 24 week results are available earlier than the results of the full study which will then be most likely be available still in the first quarter of 2014.
We will now take our next question from James Gordon of JPMorgan. Please go ahead.
James Gordon - JPMorgan
One question was on to do with the timing of data disclosure and I saw your comments just now DEMAND III and DEMAND V both could be presented in next quarter, presuming that means you really have the results of those in-house. If you did have the option would the A&Bs (ph) have the same conference or a different conference and should we expect a fresh release announcing the result ahead of conference presentation or only at the same time at the conference and then just one another question I had which was assuming you do get positive DEMAND III data what are your latest thoughts on when you be filing in the U.S. and with the Breakthrough Designation what’s the earliest time you could actually see U.S. approval?
First for most let’s go to your first question about the scientific conferences. GSK has not given any guidance yet as to at what conference which data will be presented, that’s it will be at a forthcoming scientific meeting and in the fourth quarter of this year. So it could very well be the data will come out at the same conference, it could also be different conferences but no guidance has been given yet and just to bear in mind for WMS for example there is the possibility to submit a late breaking abstract which is still possible until later this week and then of course it's still is up to the organization of the WMS to accept such late breaking abstract. So no guidance is given whatsoever on which data will be presented on which conference until that is very clear, normally it's not GSKs policy to preannounce presentations on scientific conferences prior to them being disseminated but of course in case that data are very relevant and very material for Prosensa ones that data estimates are known then we will discuss what can be brought into the public domain as to when data are available as well there.
Your second question about filing in the U.S. that’s also GSK guidance, GSK has informed the public in the past during an MDA meeting that filing will be possible as from the first quarter of 2014 onwards, that statement was made by Dr. John Kraus of GSK at an MDA meeting but that was prior to the notification by the FDA that breakthrough therapy designation was achieved for Drisapersen, after the breakthrough designation was achieved no further guidance was given as to when filing could occur. So the latest set of information we have as from the first quarter of 2014.
We will now take our next question from Joseph Schwartz of Leerink. Please go ahead.
Joseph Schwartz - Leerink Swann
I was wondering if you could address the recent clinical data that GSK released in terms of the secondary endpoints like the timed function test and maybe draw relevance to the ability of the technology to improve strength versus function and what the relevance of each of those to patient outcomes and then also I noticed that there were some it looked like CK (ph) improvements and maybe you could put that as a context for us.
The results of this 117 study were presented for very first time at the Cold Spring Harbor Meeting on the 11th of April then GSK posted some of these results and more extensive data as well on their clinical trial register in late July. Now the plan will be as for many of these studies by GSK that they will also will go for a manuscript that at a certain moment in a peer review journal and in that context more data and more discussion will be provided around those data. So for the time being I would not like to comment on the second end points in that study apart from the fact that what was presented already earlier by GSK and many of these secondary endpoints showed the trends in the same direction as the primary end point and that’s where I would like to leave it because the most important element of the study of course was that the primary end point on the six minute walk test was met in this study and that’s to our knowledge is the first time data has been achieved by a company in the DMD space with an exon-skipping compound. So we’re very pleased with that result but more data, more context around the data will be provide most likely in peer reviews publication in a scientific journal.
Joseph Schwartz - Leerink Swann
Okay and can I just also maybe get your thoughts on how your strategy might vary depending on whether the FDA grants saw approval to Drisapersen or not and how much impact that could have on Drisapersen uptick in either direction?
I fully understand the question, at the same time it's not our goal to comment on the registration strategies for other companies. We’re doing our utmost and GSK is doing it's utmost together with us to ensure that Drisapersen can come to patients as swiftly and as quickly as possible. As I mentioned the latest statements by GSK would be to go for a filing as from the first quarter of 2014 onwards, thereafter GSK obtained Breakthrough Therapy Designation which means that the FDA is committed to really work with GSK in order to assess the data as swiftly and as efficiently as possible in order to put every single effort (ph) in that process so that’s all very promising but of course to what extent that will be impacted by the possibility of another company in getting accelerated approval or not that is something which is not in our let’s say position to comment upon that but we’re doing our utmost to Drisapersen as quickly as possible to patients.
We will now take our next question comes from Chris Marai of Wedbush. Please go ahead.
Chris Marai - Wedbush
First couple of questions on your Phase III trial, number one I saw that you started enrolling the open label study in Q3. I’m just wondering if you can comment on the enrollment in that, how many patients have rolled over from the Phase III into the open label study.
That data has not been disclosed yet by GSK but let me just give some more information about that open label extension possibility, so both for patients coming from the 117 trial as well as from the 004 trial so the original Phase II trial and the Phase III trial, the patient have the option to go into an extension trial and what I know is that the majority of patients is actually going to extension trials with more specific data we would have to refer to GSK because that number is also changing as we speak.
Chris Marai - Wedbush
And then if I may ask another, just curious about your PRO0044 follow on candidate. You know I was wondering specifically you know you’re looking at studying both IV and subcutaneous what was the reason for that and then also with regards to these follow on compounds how do you anticipate you know the sequence to impact the tissue distribution of these, is that more of a function of the backbone chemistry or the actual sequence? Thanks.
First and foremost on the issue of a subcutaneous and IV the current formulation of our antisense oligonucleotides is in the range of around 200 milligrams per ML which means that if you don’t want to inject too large volumes you’re bound by certain limitations there, that’s also the reason why to be at six milligrams per kilogram and the drug can be administered in a subcutaneous manner. In the PRO044 trials we wanted to compare pharmacokinetics both on subcutaneous and IV use and to also to allow to go higher in dose at a certain if that will be required, the data of the PRO044 Phase I/II study are currently being analyzed and are currently being worked up and will be presented at a forth coming scientific meeting so then more context around PRO044 will be given there. That’s the background as to why we’re looking there at both subcutaneous and IV possibilities. Having said that at the same time we do think that having a subcutaneous administration form absolutely offers potential advantages in terms of the potential opportunity for future home treatments so that patients will not have to go every week to the hospital for an infusion so that may offer advantages in that form as well.
But Phase I/II data will be presented and later on in time. And your other question related to tissue distribution of course in patients we do not know exactly how the (inaudible) are distributed in various tissues. We do have preclinical models where we do see a different uptick and also different dystrophin restoration in various muscles but in (inaudible) we have only so far that TB (ph) frontier at the side of our biopsies. So in that respect we don’t have a good insight into different muscle types how that will be related to different uptick or different distribution or different dystrophin restoration. So we only can look at preclinical models there or a data which we know from patients in various muscle types there.
Chris Marai - Wedbush
And if I could just slide in one more real quick, you know the therapy is focused on 117, you know I thought it was interesting that the results presented by GSK really highlighted limited proteinuria compared to previous studies and I was wondering was there a change in how that was measured or defined? I mean it seems pretty straight forward but the results were quite different. You know or was it perhaps a result of patient selection, could you may be elaborate a little bit on why we think there is such a limited incidence of proteinuria compared to earlier studies. Thanks.
At the Cold Spring Harbor Meeting on the 11th of April, GSK presents it's also the safety profile of dystrophin in 117 study and there it was shown that in 39% of all patients on the placebo arm renal adverse events were noticeable and in comparison to slightly over 70% in the continuous treatment arm and the intermittent treatment arm. So in other words the 39% of the placebo arm was lower in presenting the treatment arms. The more detail which were posted on the clinical trial registry if the few dilutes (ph) of various probably side effects were noticed and in some cases you will see both proteinuria and other cases you will see urine containing protein. So this all will be coming out in that scientific peer reviewed journal which is most likely going to be written around the results of the 117 trial so more context not only around the efficacy, around the dystrophin, around safety will then come out in such a peer review publication. I hope that’s an answer to your question?
We will now take our next question from (inaudible) of Emerging Growth Equities. Please go ahead.
I will just focus three questions on the Phase III trial, just wondering now I’m thinking out loud here what fraction of the 186 boys being enrolled in the Phase III are likely to be under seven years? I mean was there a pre-determined cut off of 10% or 20% something like that?
So far no data have been released on baseline values in the Phase III study, the Phase III study which was originally designed to include 180 patients assumed the 10% dropout rates, in the meantime 186 patients that have been recruited and what we understand is that the dropout rates from the study is less than 10% so it means that more patients can be evaluated than the original 162 on which the design of this study was based. But no data are available yet on baseline values here or ages of boys or other criteria when they start with this study, because all that will be coming out on forthcoming scientific conferences when GSK will be presenting this data.
As and when the data is analyzed is there or was there a predefined subgroup specifically stratifying by age and baseline six minute walk distance?
There is no stratification in the Phase III but of course like with every study there is a statistical analytical plan where co-variance can be taken into accounts to look at potential impacts of a number of parameters like and I’m just giving these are the examples like each base line geography where patients come from and so on. So far no guidance or no information has been given by GSK on either that’s is compliant or on the data related to the baseline. I expect that will all come out at the moment that data will be presented at a scientific conference or later on in peer review journals.
And one last thing, since most of these guys are going to be on corticosteroids, so do you think that the 55 meter standard deviation would be sufficient to factor in the variability of the younger boys and thank you so much?
The 55 meter comes also from the result of the study, the Phase III study was designed with a 90% power to look at a 30 meter difference between the placebo controlled patients and patients on 6 milligrams per kilogram per week in 221 randomization and we believe that the power of this study is such that it can take into account those potential variables as well especially if you take into account that this co-variance impact will be looked at as well in this for analytical plan. So we’re confident that the study was designed to be able to see potential difference between placebo and the active dosing arm.
We will now take our next question from Jan De Kerpel of KBC Securities. Please go ahead.
Jan De Kerpel - KBC Securities
Two questions, first on PRO044 results to be disclosed later this year, I was wondering will that data package be sufficient for GSK to make decision on through listed options or not and or can you describe what kind of timelines we should anticipate with respect to GSK making a decision for the next step and then second sorry to come back on this it has been addressed before but on the 117 data clinical registry is closed by GSK if you look in the adverse event table regarding put in proteinuria, you also say protein urine present and I was wondering can you clarify for us what does exactly is the difference between protein urine presence as a measurement, is it less stringent than proteinuria and following from that have the thresholds that have been used previously to classify a patient having proteinuria or not, were this the same thresholds that were used? Thank you.
Maybe I will start with the second question first, the definitions of protein present in urine or proteinuria have not been disclosed yet as such they are not available right now but it can have relationship whether it's a spot urine test or 24 hour urine collection that could be the background there and so far the data have been presented in the context of the aggregates renal effects of Drisapersen on two different dosing regime and the placebo arm and that aggregate context including the subclinical proteinuria showed that it's indeed 39% of patients on the placebo arm experienced a renal adverse event including the subclinical proteinuria where it was 70% in the continuous treatment arm. So when you look at the table in the clinical trial registry you see many, many more different statements but I would refer to the presentation by GSK in the Cold Spring Harbor Meeting where they tried to aggregate those various adverse events into a number of summary headlines. But once that the publication will come out on this study I’m confident that more color will be given on that difference as well.
Then about your first question regarding PRO044 as to what extent will there be likelihood when or how GSK will exercise it's optional this compounds, and as you know in these PRO044 GSK has global exclusive option of 44 and according to the agreements they can exercise that option whenever they wanted to or want to. In the case of PRO044 our Phase I/II clinical study very much followed the same design as our PRO51 initial work which means that we have been treating patients for five consecutive weeks by means of a subcutaneous and in the case of PRO044 in the number of patients also with an IV formulation then had a follow-up of those patients in order to look at dystrophin expression.
With PRO51, 51 which was also published in the New England Journal of Medicine, we did observe a dose response after the increasing doses and in-depth study. For PRO044 that data will be presented later but what I can say now already is that patients who are amenable for 44 skip, the peer to have higher background dystrophin than patients with 51 and amenable deletion (ph) or 51 amenable skip I should say which means that it is more difficult to see differences between pre and post treatment, biopsies with PRO044. So that means that most likely more work will need to be done and in terms of clinical outcome on PRO044 but that data will be presented with more context at one of the oncoming scientific conferences but GSK can exercise the optional PRO044 whenever they want to.
They don’t have to do that’s at a certain moments when data are available, they can do it at any moment.
We will now take our next question comes from John Savin of Edison. Please go ahead.
John Savin - Edison Investment Research
I had a question about the market, I have been trying and work some, look at some figures. Would you agree that the possible prevalent in the United States is about 75,000 patients there is 24 between 5 and 24, and you will be targeting about 13% of those. So maybe around a thousand patients affected by Duchenne (ph). Would you agree those figures are roughly right? It's bit difficult to say because the data hasn’t been fully recorded.
Yeah, well based on our analysis and that is based on prevalence of upto 1 in 3500 effective by this and based on a life span of approximately 25 years which is still very conservative and based on inputs from various patient organizations we have come to a global number of around 75,000 patients that does includes apart from the U.S., Japan and Europe also other countries where patients who are affected by a rare disease like Duchenne first and foremost diagnosed but also potentially treated and we’re the company who is making those products available can be reimbursed for those treatments. So 75,000 is our global number of which about 15,000 in the U.S. so 13% of 15,000 will then be a little less than the 2000 patients for the U.S that’s what we have based on model zone and similar figures of course are applicable for European (ph) those jurisdictions. The 75,000 does include also countries like Brazil from a rare disease perspective is a country where many rare disease patients in fact can be treated but is not very often on the radar screen of DMD patient so far but 15,000 for the U.S. will be our number.
John Savin - Edison Investment Research
Okay do you have any finding or indicative figures for investors about pricing as yet?
As a matter of fact the short answer is no because the first thing which we have to do of course for GSK to go this we have to come to a final data set and then show efficacy and safety in the logical and customer data and then look at the impacts of these treatments also in terms of pricing them. So far no data there yet but coming from general results, having worked in the rare disease phase in this area of course of rare disorders it's not uncommon to have price ranges between $2000 and $4000 per patient per year. Don’t see this as guidance for Drisapersen, it is GSK who will decide on this pricing, this is purely based on my personal experience coming from (inaudible) and all these finds of course all impact which direct will have and not yet fully known of course.
John Savin - Edison Investment Research
Just out of interest would you put - you’re obviously including wheelchair (ph) patients in this figure as well. Do you feel those won't be treated?
Yes the 75,000 patients in relevant countries is indeed a number which includes patients who are already wheel chair bound and of course the hope will be that the impact of the data will be such that all patients can treated even non-ambulant patients but it's too early to make that judgment now already. So within 75,000 for the model yes we have included those patients. I think it's almost approaching one hour so operator I don’t know whether there are any still urgent questions. Operator?
We have no further questions at this time.
That is great. In that case we will close the call here and I really would like to thank you all for being in this first earnings call by Prosensa. As I mentioned we’re looking forward to an incredibly exciting remainder of the year and we will keep you informed as good as we can of upcoming data and results of our portfolio. Thank you very much.
That will conclude today’s conference call. Thank you for your participation. Ladies and gentlemen you may now disconnect.
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