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PTC Therapeutics, Inc. (PTCT)

Q3 2014 Earnings Call· Sun, Nov 9, 2014

$69.88

+0.95%

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Transcript

Operator

Operator

Thank you for holding for PTC’s Third Quarter 2014 Conference Call. At this time, all participants are in a listen-only mode. Following the formal remarks, PTC management will open the lines for a question-and-answer session. Please be advised that the call is being taped at the Company’s request and will be archived on the company’s website until December 5th. PTC’s current investor presentation slide deck is available at the same website location. At this time, I’d like to turn the call over to Emily Hill of PTC.

Emily Hill - Director, Investor Relations

Management

Thank you, operator, and good afternoon, everyone. Thank you all for joining us for PTC’s third quarter 2014 financial results conference call. With me here today are Dr. Stuart Peltz, Chief Executive Officer; Mr. Shane Kovacs, Chief Financial Officer and Mr. Mark Rothera, Chief Commercial Officer. Stuart will provide opening remarks and share some highlights of the quarter and recent events. Shane will then provide detailed financial results, and we will open up the call for questions. Earlier this morning, we issued a press release detailing PTC’s third quarter 2014 financial results, which is available on our website at ptcbio.com. During today’s call we may make forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These include statements about our future expectations, regarding clinical developments, regulatory, and commercialization timelines, the potential success of Translarna and other product candidates and financial projections. Actual results may differ materially from those indicated by these statements as a result of a variety of risks and uncertainties including those discussed under the heading special notes regarding forward-looking statements and risk factors in our most recent form 10-Q and our prospective supplement filed on October 10, each of which are on file with the SEC. Any forward-looking statements represent our views as of today only. PTC undertakes no obligation to publicly update any forward-looking statements. With that, let me pass the call over to Stuart.

Stuart Peltz - Chief Executive Officer

Management

Thanks Emily, and good morning, everyone. Welcome to our third quarter 2014 investor conference call. It’s busy and exciting time here at PTC with multiple programs moving forward. A number of important activities are occurring with our lead product Translarna. We are well advanced in our prelaunch activities of Translarna for Duchenne muscular dystrophy in Europe and other parts of the world. We also expect to submit a new drug application to the FDA on a rolling basis for Translarna for DMD in the US by the end of 2014. In addition to our work in DMD, we are currently enrolling the second Phase 3 trial ACT CF with Translarna for patients with nonsense mutation cystic fibrosis. As you’ve heard me say before, Translarna is both the product and a pipeline and we’re actively pursuing additional indication. I’ll tell you a bit more about these later. We are also very committed to utilizing our platform technologies to discover and develop additional therapies for ultra-orphan disorders. For example, our spinal muscular atrophy program also is advancing into a Phase 2 clinical trial called MOONFISH later this month. This program is partnered with Roche and the SMA Foundation. Let me take you through some of these milestones in detail. As you may recall, Translarna is now approved in the EU for the treatment of the Duchenne muscular dystrophy caused by a nonsense mutation in the ambulatory patients age five years and older. Translarna is the first ever treatment to be approved for the underlying cause of DMD. This approval allows us to provide Translarna to patients in 28 countries in the EU as well as in other countries outside the EU that recognize this. Our commercial activities and preparation for the launch of Translarna are well underway. We are highly engaged with…

Shane Kovacs - Chief Financial Officer

Management

Thanks, Stu. I am happy to again report that PTC is in a very strong financial position which will support our important ongoing commercial, regulatory, clinical and research activities. We finished the quarter ending September 30 with nearly $210 million of cash and marketable securities on our balance sheet. In October, we completed a very successful financing through a public offering of common stock where we secured an additional $117.5 million of net cash proceeds. Pro forma for this offering, we had approximately $327 million of cash on our balance sheet at September 30th. We believe that this cash position should be sufficient to fund our operation for the next three years through late 2017. Here are the financial highlights from our quarter and our updated financial guidance. As Stu mentioned earlier, we achieved an important milestone in the third quarter recognizing the first product sales for Translarna in history of PTC via our European reimbursed early access programs. It is important to note that we are booking revenue on a cash basis when payment is received as oppose to when product is shipped. Therefore, in the quarter, we had shipped more products that we are able to recognize as revenue. Total revenues for the third quarter of 2014 were approximately $1.7 million compared to total revenue in the third quarter of 2013 to $16.3 million. This includes revenue of approximately $0.1 million from net product sales of Translarna. Revenue from grants and collaborations was $1.6 million for the third quarter of 2014 compared with $16.3 million for the same period in 2013. This decrease was due to a milestone payment of $10 million that was recognized in the third quarter of 2013 related to the SMA collaboration as well as a decrease in the recognition of non-cash deferred revenue.…

Operator

Operator

(Operator Instructions) And our first question comes from Jason Kantor of Credit Suisse. Please go ahead.

Jason Kantor - Credit Suisse

Analyst

Perfect. Can you hear me?

Stuart Peltz

Analyst

Yes.

Jason Kantor - Credit Suisse

Analyst

Any chance you guys could give us the patient number in terms of how many patients are on these named-patient programs in Europe at the moment?

Stuart Peltz

Analyst

Yes, hi Jason. Good to hear from you. So, these are just continual process, I don’t think we’re putting out numbers right now. Obviously in terms of the number of patients, we’re just not saying, obviously we’re working on those who are genotype and it will be variable from country to country. So, at this time we’re not just putting out that.

Jason Kantor - Credit Suisse

Analyst

And could you give us some expectation maybe quarter-by-quarter, how would you see the actual commercial launch rolling out in 2015? I think that would help.

Stuart Peltz

Analyst

Yes, hi. Let me ask Mark to talk a bit about that.

Mark Rothera

Analyst

Yes, hello. So, we’re really planning for our first commercial launch in Germany in the next few months. And if you look typically countries in the Nordic region are typically some of the fastest commercial reimbursement processes out there. So, coupled with UK, we see sort of Germany, the Nordic region, UK has being in that sort of first wave of launches in the first half 2015.

Jason Kantor - Credit Suisse

Analyst

And do you expect to be launched throughout Europe by the end of 2015, is that -- should we assume that?

Mark Rothera

Analyst

I think we said this before but the pricing reimbursement processes in Europe can take anywhere from around three months to 18 months plus. So, there maybe a few countries in 2016 that will reimburse but I guess the good news is we have made good progress with the early access program in countries like France and recently we are now in Italy, but thanks to the fact that these authorities have reimbursed early access program, it’s essentially like a launch and you are sort of bringing, and there you can’t in these circumstances promote, but you can provide the drug on a reimbursed basis to patients when physicians request it.

Jason Kantor - Credit Suisse

Analyst

Got it. Thank you.

Operator

Operator

Our next question comes from Alethia Young from Deutsche Bank. Please go ahead.

Alethia Young - Deutsche Bank

Analyst

Hi, great. Thanks for taking my question and also let me be the first to congratulate you on your first sales in the Company’s history. I have a couple questions. One, just kind of following up on Jason’s question, can you talk a little bit about perhaps maybe more of it, some flavor for the numbers in some of these early markets or kind of how you think about like the population size there? And then I guess the next question is, kind of how do you think about variance on pricing as far as like is $300 a reasonable average price or how should we think about it country to country and how did this conversation go the kind of established in this whole kind of average price that you are comfortable communicating? Thanks.

Stuart Peltz

Analyst

Hi, Alethia, thanks. So, now that we have the first available treatment for the underlying cause of DMD. There is a real, much greater incentive to genotype of patients, right. So, that’s going on right now. So, let me pass it on maybe to Mark to actually give you the color on what’s you’re…

Mark Rothera

Analyst

If I understood, you’re sort of talking about patient numbers and the pricing. So, I think we said this before but we’re expecting that within the label in Europe. We’re talking around 40% to 50% of nonsense mutation for Duchenne muscular dystrophy patients falling in the label. And as to pricing, I think you’re very well aware that innovative ultra-orphan drugs in this sort of category are usually priced in the range of $200,000 to $400,000. We made it clear that our internal assumption is around $300,000 plus or minus. And because there are so many variables to the price including weight of the patient, the exchange rate and the country specific mechanisms of reimbursement, already directing you to the best estimate of the overall anticipated price for patients for a year from a planning perspective.

Alethia Young - Deutsche Bank

Analyst

Great. Thanks.

Operator

Operator

Our next question comes from Tazeen Ahmad from Bank of America. Please go ahead.

Tazeen Ahmad - Bank of America

Analyst

Hi, thanks for taking my questions. A couple on the SMA trial, so for MOONFISH, you noted that you’re starting with patients that at age two. Can you just kind of tell us sort of the rationalization behind that? Why not start with younger patients? And given the fact that you’re starting the trial imminently, is it possible that we could expect data from that before the end of 2015? And the third question is on the first trial in healthy patients, when do you think we could potentially see additional data from that potentially at a medical meeting?

Stuart Peltz

Analyst

Yes, hi. Thank you for your question. And just to remind you that the 2 to 15 really is really going to be Type 2 and 3 patients initially. So, that’s what we’ll comment. Obviously, we’ll be -- we’re looking at to start a trial also at some point in the Type 1 patients but that will -- that’s not what predominantly this trial will be about. So that was the reason why that would be. In terms of when we anticipate data, obviously this is – we’re starting this month. And so I think if you look in the ClinicalTrials.gov we say we anticipated the completion as of January 2016 that can be faster depending on the enrollment. So, we certainly anticipate within that year and it could even be more rapid than that.

Tazeen Ahmad - Bank of America

Analyst

And then on the healthy patient data, have you discussed with Roche an appropriate venue for that?

Stuart Peltz

Analyst

Yes. So, obviously we did the healthy volunteer data where we showed the proof of mechanism and everyone was pretty excited about that. We want to share this data and we’re looking at what the best scientific venue is to present that in 2015.

Tazeen Ahmad - Bank of America

Analyst

Okay. And I guess one question for Shane, I think in your 2Q filing, you did disclose that you establish a new subsidiary, is that being -- is that being looked after potentially helping you lower your US tax rate, and if so, when do you think you would get some - we would get some clarity on that? Thanks.

Shane Kovacs

Analyst

Yes, well I think we just kind of earlier said in the script about the proximity of our supply chain and our distribution for Ireland being sort of the central hub. And then ultimately I think we said that we anticipate the discorporate structure could result in a blended tax rate in to low teens based on global sales of Translarna.

Tazeen Ahmad - Bank of America

Analyst

Would that be something that you would have a better sense on the closer you get to commercialization?

Shane Kovacs

Analyst

Yes, I mean obviously we still have a lot of tax losses and so when we ultimately be cash flow positive in the coming taxes is in future years, so it is some ways away but I think based on the structure that we have in place and what some of our peers have come to, I think that’s our current expectations.

Tazeen Ahmad - Bank of America

Analyst

Okay. Thanks.

Operator

Operator

Our next question comes from Chris Marai of Oppenheimer. Please go ahead.

Christopher Marai - Oppenheimer

Analyst

Hi. Thanks for taking my questions and congratulations on your first sales. I guess, first, sort of wondering about your extended access demand here. Are these patients had -- are they patients that have previously been enrolled in trials, enrolled orphan ultra drugs or are they’re new patients? And then, could you comment potentially on patients that have been genotypically confirmed already, how many have been confirmed by your estimates in the marketplaces? Thanks.

Stuart Peltz

Analyst

Yes, sure. So, these are patients, so obviously when we were doing the confirmatory I mean the confirmatory actually DMD trial that these patients we wanted those patients in the trial. So, these are all new patients who either don’t fit well within the current trial but within the label. So, that’s this new patient population that are in. In terms of the genotyping, obviously we think that’s an critical part of what we’re going to do because obviously now there is a treatment, approved treatment for patients now with for DMD but that have the particular genotype and that we think as important in order to – obviously to know what the patient population is and we’re making every effort to really make sure that all known patients are genotype and that’s really one of the top things in our list to work on. And Mark, maybe you want to make any comments?

Mark Rothera

Analyst

Yes, I just wanted to echo your words, Stu, that there is variability between countries as a number of patients that are known in genotype. And it is one of our really big areas of focus of putting in place different processes and supporting mechanism and sort of – and that awareness and enable faster and more efficient genotyping of patients. And as you said, I think it’s a very critical to identifying the right patient and Translarna is really a key to the success with Translarna and PTC.

Christopher Marai - Oppenheimer

Analyst

Okay, great. And then just with respect to that question on the patients, have they been previously exposed to Translarna? Could you comment on that, the patients who are now on therapy through your expanded access program?

Stuart Peltz

Analyst

These are all new patients. Patients that have been previously been exposed are on our extension clinical trial program so right now that’s where they’re at. So, these are all brand new patients.

Christopher Marai - Oppenheimer

Analyst

Okay, thanks. That’s helpful. And then just with respect to ex-EU expanded access demand, are you seeing demand from patients outside the EU and have any patient in Brazil started the process to or request the right to the drug? Thanks.

Stuart Peltz

Analyst

So we’re working now in other countries as well outside the Europe including Israel, Colombia as well as in Brazil to try and get expanded access. What we’re trying to do is obviously use the approval process in those countries that will allow you to use expanded access based on the approval in Europe. And so we’re working there as well. Actually we’re also in Canada and other places as well in terms of that for the approval of potential expanded access. So, we’re working on that right now.

Christopher Marai - Oppenheimer

Analyst

Great. And then one last question, just about the rolling NDA in the US, congratulations. I’m just wondering with respect to that, is this going to significantly impact your previously guided timelines with respect to a potential Translarna US launch. Thanks.

Stuart Peltz

Analyst

Thanks. I think that, the way we’re thinking about this is that as I said discussions with the FDA, that will get the various segments of the NDA in and then on completion of the ACT DMD trail the data will come out and we’ll then expeditiously get it in and it’s our hope and I think and our dialog with them given the severe unmet medical need that this would be rapidly reviewed and that, that this was expected in terms of the approval to move it up potentially up to six months. So, I think what we’re thinking about that is that we would think that this can be a launch within the first half of 2016.

Christopher Marai - Oppenheimer

Analyst

Great. Thanks for taking my question. Congratulations on the FL.

Stuart Peltz

Analyst

Thank you.

Mark Rothera

Analyst

Thanks Chris.

Operator

Operator

(Operator Instructions) And our next question comes from Debjit Chattopadhyay from ROTH Capital Partners. Please go ahead.

Debjit Chattopadhyay - ROTH Capital Partners

Analyst

Hey, good morning, guys. And thanks for taking my question. Considering that SMA Type 1 is probably a more – in more a need for disease modifying therapy compared to DMD, what are you thinking about in terms of solid endpoint, given that you have a biomarker to maybe have an isolated approval process for the SMA Type 1 indication as opposed to going for hard endpoint like time to death or post-activation?

Stuart Peltz

Analyst

So, in Type 1, I mean in Type 2 what we’re doing right now is really in patient what we’re looking at is the SMN both RNA and protein levels as well as some function that’s measured by physiology. So, I think those would be the first part which certainly we’re looking at is the potential for as a biomarker as well. And so those are going to be future discussions with the FDA and the European regulators as their thoughts on biomarkers but certainly we’re gathering that sort of information to be able to have those discussions at a future time.

Debjit Chattopadhyay - ROTH Capital Partners

Analyst

And the other pertains to the commercial launch in Europe, are you guys are considering some sort of centralized lab to expedite the genotyping of patients, so that you get as many as possible within the next year or so, Stu, on the drug?

Stuart Peltz

Analyst

Yes, I think what we’re to say that we’re trying whatever avenue is possible to make sure that we get genotype set the rate limiting step is not actually getting the information for genotyping. And so I think really our goal is to identify and do this rapidly as possible.

Mark Rothera

Analyst

And maybe I can add that given the fact that you’re dealing with the multiple set of countries with different languages and different systems, our general preference has been to find a key laboratory in each country, some of them are already exist and funneling patients into that diagnostics has played very well, some of them are not quite so well organized and our job is to enhance awareness. And in fact patients are much more aware now of the need for genotype than they were in the past because there is now a treatment so it increased the demands for genotype.

Debjit Chattopadhyay - ROTH Capital Partners

Analyst

And one last question, since that Translarna is on a conditional approval, have you had any push back from any reimbursement authority regarding your reimbursement rates until your Phase 3 data is out there?

Stuart Peltz

Analyst

Yes, so the conditional approval really just the point that others really an approval with the requirement to do another study analogous to probably like an accelerated approval in that point of view. I think in terms of push back, in terms of this, it’s probably too early to make any comments on the negotiations at this time.

Mark Rothera

Analyst

We’re expecting our negotiations throughout 2015.

Debjit Chattopadhyay - ROTH Capital Partners

Analyst

So, thank you so much and congratulations once again.

Stuart Peltz

Analyst

Thank you.

Operator

Operator

Our next question comes from Ritu Baral from Cowen. Please go ahead.

Ritu Baral - Cowen and Company

Analyst

Hi, guys. I appreciate you taking the question. In regards to the $300,000 price that you mentioned, is that typically a potential pan-European planning price or does that extend to your worldwide internal estimates as well?

Stuart Peltz

Analyst

Yes. So, we’re – we don’t yet have the final commercial price and that’s going to be something that you negotiate country by country. So, I think we’re in the process of submitting all the reimbursement dossiers in the multiple countries and we anticipate price negotiations to occur throughout 2015 and perhaps even beyond. So, it’s hard to actually give you anything more precise than that.

Ritu Baral - Cowen and Company

Analyst

Okay. And then as we look at Canada and Latin America as you mentioned, what sort of general patient numbers exist there? Is the prevalence of nonsense are more to the US and Europe? And also what sort of timeline following the EU reimbursement or approval to those regions generally follow with orphan drugs in particular?

Stuart Peltz

Analyst

Yes, so maybe I’ll just say the normal prevalence that I think that we’ll look at is what we’ve been saying is approximately 13% of the patients. And in the case of Duchenne muscular dystrophy, there is not a particular ponderous effect. In fact about 40% or so are spontaneous type mutation. So, you really see this worldwide and that number is somewhat typical. And so obviously which is a really some high amount of medical need. And so we’re trying to expedite those processes maybe Mark, have you -- give a little bit of the timelines in those countries.

Mark Rothera

Analyst

I would just say that like some of those countries we’ve seen in Europe, countries like Brazil and Canada do have early access mechanisms on a named-patient basis. And I think our initial expectation is that the demand that we’re already feeling from those countries will be better to the named-patient processes.

Ritu Baral - Cowen and Company

Analyst

Do you think that could be a 2015 something that we should consider for our models for 2015 or would that be more of a 2016 thing?

Mark Rothera

Analyst

It could be possibly towards the back-end of 2015 and into 2016, it’s generally a kind of inertia in getting these systems up and running.

Stuart Peltz

Analyst

Perhaps on the – in terms of thinking like the launch in the US when you compare that where we think in the first half of 2016 the US launch is what we’re anticipating. It could be in terms of approvals, approval so it could be similar to that nature, right.

Ritu Baral - Cowen and Company

Analyst

Great, that’s really helpful. And my last question, going back to the genotyping rates, you’ve presented, I think some interesting market data on the overall rate, current level of genotyping in Europe which disagrees with some of the very anecdotal numbers that the European doctors believe their patients are genotyped. What are your current thoughts, if not on the actual numbers patients genotyped, just the current percentage and how that might grow?

Stuart Peltz

Analyst

Yes, I think in terms of the numbers, I don’t think we’ve actually gone through and discussed too many numbers. So, and actually we’re on that point. Maybe Mark, do you want to have?

Mark Rothera

Analyst

Yes, I wonder whether you might be thinking about the fact we reported a 40% to 50% of nonsense mutation in patients from a prevalence point of view being within our label.

Ritu Baral - Cowen and Company

Analyst

Go ahead, sorry.

Mark Rothera

Analyst

Sorry, that’s just an -- I don’t think we’ve given you a specific number of known nonsense mutation patients statistics.

Ritu Baral - Cowen and Company

Analyst

But do you have any sort of internal estimate on the degree that European patients are genotyped right now and how that might grow?

Mark Rothera

Analyst

So, it does vary by country, some are very sophisticated and very centralized, some are little less sophisticated. We don’t have one number to give you at this stage but I think as Stu mentioned earlier, we’re highly focused on helping different medical systems in different countries to be as efficient as possible about the whole diagnostic pathway. We realize that’s really critical even if this is a very target we mentioned.

Ritu Baral - Cowen and Company

Analyst

Great. Appreciate you guys taking the questions.

Operator

Operator

There are no further questions at this time. I would like to turn it back to Stuart Peltz, for closing remarks

Stuart Peltz - Chief Executive Officer

Management

Thank you, operator. It’s been over 15 years of research and development to bring the first commercial therapy to Duchenne patient. We’re very engaged in this launch process and look forward to updating you further. Also, please either join us in New York City or on the webcast for our Science Day on November 24th to hear more about our continuing progress and our developing pipelines. Again, thank you for your interest in PTC and for joining the call today.

Operator

Operator

Ladies and gentlemen that does conclude today’s conference. Thank you for your attendance. You may now disconnect. Everyone, have a great day.