PTC Therapeutics' (PTCT) CEO Stuart Peltz on Q2 2014 Results - Earnings Call Transcript

Aug.10.14 | About: PTC Therapeutics (PTCT)

PTC Therapeutics (NASDAQ:PTCT)

Q2 2014 Results Earnings Conference Call

August 07, 2014, 08:30 AM ET

Executives

Jane Baj - Director, Corporate Communications

Stuart W. Peltz - CEO

Shane Kovacs - CFO

Mark Rothera - Chief Commercial Officer

Analysts

Jeremiah Shepard - Credit Suisse

Tazeen Ahmad - Bank of America Merrill Lynch

Christopher Marai - Oppenheimer & Co.

Debjit Chattopadhyay - ROTH Capital Partners

Operator

Thank you for holding for PTC's Second Quarter 2014 Conference Call. At this time all participants are in a listen-only mode. Following the formal remarks, PTC management will open up the lines for question-and-answer period. Please be advised that call is being taped at the company’s request and will be archived on the company’s website until August 21st, which is two weeks from today. 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 Jane Baj of PTC.

Jane Baj

Thank you, operator, and good afternoon, everyone. Thank you all for joining us for PTC's second 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 of PTC. Stuart will provide opening remarks and share some highlights of the quarter and recent events. Mark will provide an update on our reimbursed expanded access program and our preliminary launch plans for Translarna. 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 second 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 and regulatory timelines, the potential success of our 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 which is 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 W. Peltz

Thanks Jane, and good morning, everyone. Welcome to our second quarter 2014 investor conference call. We are pleased to report on the significant progress we have made in the second quarter across each of our research clinical regulatory and commercial fronts. I am extremely proud to report that we recently received approval from the European Commission granting conditional marketing authorization for ataluren in the EU for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients age five years and older. The brand name for ataluren is Translarna.

This is an extremely important milestone for Duchenne community and for our company. Translarna is the first treatment to be approved for Duchenne. The conditional marketing authorization allows PTC to market Translarna in 28 countries that are member states of the European Union as well as the European economic area members. We will be working with the regulators, payers, physicians and patient organizations to make Translarna available as quickly as possible in Europe and also in countries that recognize the European approval.

PTC's confirmatory ACT DMD trial is also on track to complete enrollment in the near-term and we expect to have top-line data from the trial in the second half of 2015. We expect the positive data from this trial will support full approval in the EU, U.S. and rest of the world. ACT DMD is one of the largest studies ever to be conducted in this disorder.

Given the recent outcome from our regulatory dialogue in Europe combined with feedback the FDA has provided to some of our peer companies working at Duchenne. PTC is engaging in further dialogue with the FDA to discuss potential pathways to accelerate bringing the Translarna to United States patients.

I will now turn the call over to Mark Rothera, our Chief Commercial Officer. Mark is responsible for establishing our global commercialization strategy for Translarna and brings a wealth of experience to PTC. Mark has spent 25 years in the biopharmaceutical sector of which the last 15 has been spent launching orphan drugs internationally for rare diseases. Mark has built international operations from the very early stages at PathoGenesis Chiron Biopharmaceuticals, Shire Human Genetic Therapies and Aegerion Pharmaceuticals. Mark will outline our current commercialization efforts. Mark?

Mark Rothera

Thanks, Stu. It is a pleasure to share with you our preliminary launch plans of Translarna and to update you on activities that are already underway. I am truly honored to be part of the team that will bring the first ever treatment for the underlying cause of the disease to Duchenne muscular dystrophy patients.

PTC has the worldwide rights to Translarna and ultimately aims to develop a global footprint for its commercialization. Since I joined PTC approximately 18 months ago we began developing the organizational structure that would ultimately serve as the basis for Translarna's commercialization in Europe. This included bringing on a leading team of professionals with both rare disease and biotech build experience in key countries including Germany, UK, France, Italy, Spain and the Nordic region. This team has been instrumental in our local country effort including enrollment in our ACT DMD study, dialogue with key opinion leaders and patient advocacy group as well as patient identification activities.

As we grow our commercial organization, this group is being supported by strong global and regional capabilities including market access, medical affairs, marketing, quality and supply chain. Our team is highly focused on the country specific processes which are the next step in the journey of bringing commercial access to Translarna for nmDMD patients in Europe. We have begun our commercialization efforts and plan to launch Translarna in selected countries beginning in the first half of 2015. The market access process and timelines vary from country to country and can take over 18 months in some cases.

We have very aware though that for patients and their families living with DMD, a rapidly progressing muscle into these every day counts. In an effort to accelerate access to Translarna prior to commercial availability we recently announced the initiation of a reimbursed expanded access program or EAP in Europe and other countries that will recognize the EMA approval.

Where mechanisms exist and in accordance with local regulations in these countries, PTC will make Translarna available for nonsense mutation Duchenne muscular dystrophy patients to reimburse EAP program. Funded name patient programs have already been authorized in Turkey and Spain and recently the French Medicine Agency has granted an ATU cohort. The ATU cohort allows all nmDMD patients matching certain inclusion criteria to receive the therapy now rather than wait the outcome of a 12 to 18 months pricing and reimbursement process. I am happy to say that PTC has already begun shipments of Translarna to patients enrolled in the EAP program.

I look forward to sharing more of our commercialization plans and milestones achieved on future calls. With that I'll turn the call back over to Stu.

Stuart W. Peltz

Thank you Mark. In addition to our efforts in nonsense mutation DMD we’re also highly focused on the development of Translarna as a treatment for nonsense mutation cystic fibrosis. Nonsense mutations account approximately 10% of all CF patients and a nonsense mutation CF is one of the most severe forms of the disease because of the complete lack of reduction of the CFTR protein. Available treatments today do not address the underlying cause of the disease for these patients.

We recently initiated ACT CF, our confirmatory Phase 3 clinical trial of Translarna in nmCF patients. This is a global randomized double blind placebo controlled study that will enroll approximately 200 patients, six years of age or older with nonsense mutation CF who are not receiving chronic inhaled aminoglycosides. To remind you in our previous Phase 3 trial, a subgroup analysis of patients not receiving chronic inhaled tobramycin show that patients receiving Translarna had a mean relative benefit in FEV1 of 5.1% versus placebo and also 41% decrease in pulmonary exacerbations.

ACT CF has been designed to exclude patients who are using chronic inhaled aminoglycosides such as tobramycin. Aminoglycosides are ribosome-binding drugs that were shown to confound Translarna's activity. The primary endpoint in ACT CF trial is lung function as measured by relative change of percent predicted FEV1 and pulmonary exacerbations as a key secondary endpoint. Patients are randomly assigned to Translarna 40 mg per kg per day or placebo. We anticipate that we will complete enrollment in the second half of 2015 with data expected approximately a year later.

We are currently evaluating our strategy and timing for potential EU submissions seeking conditional approval nonsense mutation CF in Europe. Based on our knowledge and experience we gained from our submission in DMD we plan to file for conditional approval when the ACT CF trial was well underway.

In addition to DMD and CF, we also plan to develop Translarna for other nonsense mutation based disorders. As previously mentioned Translarna is both a product and a pipeline. It's estimated that on average 11% of patients with any monogenetic disorder have a nonsense mutation as the cause of the disease. In the past several years, there have been over 20 publications by independent investigators demonstrating Translarna activity in multiple nonsense mutation preclinical cell and animal based disease models in multiple different organ systems.

We have spent a considerable amount of time evaluating potential clinical development of Translarna in additional indication and based on this evaluation and discussions with outside experts, we have selected mucopolysaccharidosis type 1 also referred to as MPS I as the next indication. MPS I is an inherited genetic disorder caused by a deficiency in an essential enzyme that is responsible for the breakdown of byproducts of chemical reactions in the body cells.

Globally, MPS I occurs in about one in every 100,000 births. It is estimated that 60% to 80% of cases of MPS I are caused by a nonsense mutation. Enzyme replacement therapies around the market, there are many significant unmet medical needs for the development of new treatments that can target the underlying cause of this disorder.

In preclinical models of Hurler's disease Translarna has demonstrated that it crosses the blood brain barrier and penetrates skeletal and cardiac tissues. We therefore, believe that Translarna has the potential to address the underlying cause of MPS I caused by nonsense mutation in these tissues. It is our goal to initiate a Phase 2, proof-of-concept study by the end of this year.

Let's now turn to our alternative splicing platform where the lead compound is focused on another genetic disorder, Spinal Muscular Atrophy or SMA. To remind you, this program is a collaboration with both Roche and the SMA Foundation. SMA is a rare disorder that is the leading genetic cause of death in infants and toddlers. The SMA Foundation estimates that Spinal Muscular Atrophy affects one in every 10,000 children born. It is caused by a defective SMN1 gene leaving the SMA protein production solely as a result of the expression of the SMN2 gene. This leads to reduce levels of the SMN protein causing significant muscle atrophy and eventually death by the age of two in the most severe form of the disease.

In January of this year, a Phase 1A, single ascending dose, placebo controlled study and healthy volunteers was initiated. The primary objectives of this study were to explore safety and pharmacokinetics of the drug candidate, RG7800. This study has now completed and a multiple dose clinical study in SMA patients is currently in preparation. Preliminary findings in the Phase 1a study indicates that RG7800 was well-tolerated at all dose levels studied. There were no series of adverse events or withdrawals due to the adverse events and no dose related trends were identified. Importantly, RG7800 had a dose dependent effect on SMN2 splicing, as shown by a change in the ratio of full length SMN2 mRNA to Delta 7 SMA2 mRNA which may be interpreted as proof of mechanism in terms of expected pharmacodynamic effect.

We are looking forward to the continued development of this exciting potential therapy for this devastating disease in collaboration with our partners. Furthermore, we believe that the results we have achieved to-date in this program provide strong validation for alternative splicing platforms which were separately focused on the discovery of compounds in marginally splicing to include or exclude exons in other RNA transcripts to help treat other rare and neglected disorders.

Primarily, I would also like to highlight a few of the earlier stage programs in our pipeline. As you may appreciate there is a lack of novel treatments being developed for tackling the emerging problem of resistance. Our bacterial program is moving forward and is on track to declare a development candidate later this year. This program is based on a novel chemical scaffold and could potentially address the significant need for new treatment options to combat drug-resistant gonorrhea.

We also plan to initiate a Phase 1 study for PTC596, our development candidate for our BMI1 program. PTC596 is a first-in-class oral potent and selective inhibitor of BMI1 protein expression. Elevated levels of BMI1 are associated with more aggressive drug-resistant tumors and a poor prognosis in a wide variety of cancers, including Glioblastoma. Reducing levels of BMI1 therefore, represents a promising new therapeutic strategy to treat drug-resistant cancers. We would like to acknowledge the support from the Welcome Trust for their Anti-Bacterial BMI1 programs.

I'd also like to discuss a change on our executive team. After many years of dedicated service, our President, Claudio Hirawat has informed of our plans to step down from that role. I would like to take a moment to acknowledge her contributions to PTC. I first met Claudia over 15 years ago, when I was founding PTC. In the early years, Claudia brought organizational discipline to the business as we set up the company and began our initial stages of growth. Over the years her contributions grew immensely as she played a leading role across business development and other key corporate functions. Our corporate collaborations were a direct result of Claudia's efforts for providing both critical source of funding for the business and an opportunity to expand our research capabilities and platform.

She also played a critical role in our successful efforts to obtain a positive opinion from the CHMT for Translarna in Duchenne muscular dystrophy. Claudia is not leaving us. We expect to form a new relationship with Claudia going forward that will allow us to continue to benefit from her expertise. On behalf of the entire organization, I would like to thank Claudia for all she has done for us.

Before we open up the floor to Q&A, let me ask Shane to review our financial results. Shane?

Shane Kovacs

Thanks, Stu. I am happy to report that PTC continues to be in a very strong financial position to execute upon our strategy and build a leading commercial company focused on rare and neglected disorders. We finished the quarter ending June 30th with nearly $227 million of cash and marketable securities on our balance sheet, which should be sufficient capital to fund our operations into mid-2016. Here are the financial highlights from our quarter and our updated financial guidance.

The company reported a net loss of approximately $25.1 million for the second quarter of 2014 compared to approximately $14.6 million for the second quarter of 2013. Total revenues for the second quarter of 2014 were approximately $1.7 million compared to total revenues in second quarter of 2013 of approximately $6.9 million.

Research and development expenses were $18.3 million for the second quarter including $2.2 million in non-cash stock-based compensation expense which compares to $14.7 million for the same period in 2013, including $1.1 million in non-cash stock-based compensation expense. This increase primarily results from additional costs associated with our clinical trials, including the manufacturing of drug product for our studies and regulatory costs associated with the efforts to obtain conditional approval for Translarna in Europe.

General and administrative expenses were $8.7 million for the second quarter of 2014 including $2.1 million in non-cash stock-based compensation expense compared to $6.6 million for the same period in 2013, including $0.8 million in non-cash stock based compensation expense. The increase primarily results from additional costs associated with efforts to successfully obtain conditional approval for Translarna in Europe pre-commercial activities and public company costs.

Based on the approval we recently received from the European Commission we have begun executing against our plans related to pre-launch activities for Translarna in Europe and selected other territories. As we transform from an R&D company into an international commercial organization we are actively putting in place the resources and infrastructure that support the launch. As Mark mentioned earlier, this includes activities and systems related to commercialization such as CMC, quality, compliance, regulatory and medical affairs as well as recruitment. We have also been busy establishing legal entities and setting up our international banking operations.

As a result of this incremental investment we are updating our guidance for 2014. Total cash operating expenses for 2014 are now expected to be between $103 million and $113 million excluding expected non-cash stock-based compensation expense of approximately $17 million for total operating expenses of approximately $120 million to $130 million. We expect to end 2014 with approximately $160 million to $170 million in cash, cash equivalents and marketable securities. We currently have 30.1 million shares issued and outstanding which includes 0.7 million shares in restricted stock grants.

With that, let's open the call for questions. Operator?

Question-and-Answer-Session

Operator

(Operator Instructions). Your first question comes from Jason Kantor with Credit Suisse. Your line is open

Jeremiah Shepard - Credit Suisse

Good morning. This is Jeremiah filling in for Jason.

Stuart W. Peltz

Hi, Jeremiah.

Jeremiah Shepard - Credit Suisse

Congratulations on all the success you have had this quarter. First question regarding Translarna in Europe, how are the patients treated in Europe? Are they primarily treated in specialized centers? And also do you feel like you have IDed most of the possible patient population there?

Stuart W. Peltz

Thanks for that, Jeremiah, let me ask Mark to sort of fill you in on this.

Mark Rothera

Sure. As you know Europe is whole collection of different countries, so it does vary between countries but predominantly the treatment is in reference or specialist centers for muscular disease. In respect to identification again it varies substantially so you do have some countries where it's very sophisticated in terms of patient diagnosis and others where it’s pretty basic still. So one of our efforts is to help sort of the whole stream of patient identification work to be enhanced in Europe. And that's of course substantially easier in the presence of the new therapy.

Stuart W. Peltz

Any other questions?

Jeremiah Shepard - Credit Suisse

Yes, another follow-up is, in terms of prepping the market for the product, how much more do you need to do than what you have already done now? And then in terms of the reimbursement discussions, does it go faster in cases of a rare disease? You mentioned a 12- to 18-month timeline; could it be more toward the 12-month time frame?

Mark Rothera

I think having an orphan drug with a very high unmet medical need certainly helps the case substantially. And there are countries where it will be faster than others. So, some of the Northern European countries like the Nordic countries, Germany, UK are likely to be at the faster end of that spectrum. And then there will be others where there isn't a specific dispensation for orphan drugs but we're expecting to launch in the first half of 2015.

Stuart W. Peltz

And then we're obviously also in those areas where we can bring drug to patients through expanded access program, we're doing that as well, both in Europe as well as outside.

Jeremiah Shepard - Credit Suisse

And in regards to the SMA program, you provided some detail today, but is there a potential meeting or some sort of timeline that we could see even more data maybe at a scientific meeting or medical meeting?

Stuart W. Peltz

Yeah we'll be working with Roche and the SMA Foundation at the appropriate time to be able to -- that will be appropriate to disclose. This is -- it's a partnership between all three of us so this is what we're able to disclose today.

Jeremiah Shepard - Credit Suisse

And just last question, in regards to what you have seen, I know it's still early, but is it -- given the fact that you're looking at splicing and you are seeing a signal, is there any threshold that you can theorize from your preclinical work that could be meaningful in terms of providing therapeutic benefit in organs or in the central nervous system?

Stuart W. Peltz

No, so I think again it is early on a single dose. I think what the important point here thought and it think that's just unique to having healthy volunteers having both in SMN1 and SMN2. So one could at least look to see if what's going on and that is to see the splicing of the SMN2 to go more to SMN1 such that you get, you have an opportunity to see in early read of proof of mechanism. So I think that's a good thing and that, the preclinical data we have and that's what actually one of the advantages having an orally bio-available molecule that you can look in other tissues so looking at the [periphery] and in blood, that gives us a nice opportunity we were able to do that in animals to be able to look in tissues both in blood as well as in the brain. We're not obviously able to that in humans. So we'll be able to -- it will be determined early -- it's to be determined when we'll be able to disclose that.

Jeremiah Shepard - Credit Suisse

Thank you for taking our questions.

Stuart W. Peltz

Thank you.

Operator

Our next question comes from Geoff Meacham with JPMorgan. Your line is open.

Unidentified Analyst

Hi, it's actually Mike in for Geoff. Congrats on all the progress and thanks for taking our questions. With respect to the launch in Europe you mentioned sort of targeting first half of '15 you may be provide a little bit color on what's specific countries you are targeting for the first half?

Stuart W. Peltz

Yeah sure, Mark, why you don't just tackle that?

Mark Rothera

Sure as I mentioned in the last question I think the natural inclination from a launch point of view, earlier launches is to launch in those Northern European countries where the market access processes faster. So Nordic countries such as Denmark and Germany where there is an ability to launch before you've completed the negotiations on pricing and reimbursement, and the UK typically these are faster countries. But as I say we're also very happy that we've been able to initiate the provision of reimbursed drug through early access or expanded access programs in a number of countries where effectively we are kind of quasi beginning to launch in those countries.

Unidentified Analyst

With respect to those expanded access programs could you may be give us sense of pace of enrollment in the ones that are up and running and sort of how that compares to your expectations just trying to get a sense of the enthusiasm?

Stuart W. Peltz

Sure we just started this, so it’s hard to give you precise numbers but we're already working in Spain, France and Turkey and others are being set up. So I think from a point of view of higher unmet medical and demand it isn’t probably what you expect for a disorder like BMD with there is such higher unmet medical need.

Unidentified Analyst

Got it.

Mark Rothera

The other thing I would say is we have through the cohort program if you like a broader access platform whereas in some countries it’s on a named patient basis individual patient by individual patient. But certainly by getting requests from other parts of the world, in Latin America Canada, et cetera. So we are aware of the needs of the patients here and we'll do what we can to provide early access.

Unidentified Analyst

Great, thanks.

Stuart W. Peltz

Thank you.

Operator

Our next question comes from Tazeen Ahmad with Bank of America. Your line is open.

Tazeen Ahmad - Bank of America Merrill Lynch

Hi. Good morning. Thanks for taking my questions. I guess a couple for Shane. Right now we are assuming a U.S. tax rate on your sales, and I was just wondering if there is any kind of opportunity to get a lower rate for ataluren for any of the indications and for any of the products in your earlier stage pipeline?

Shane Kovacs

Yeah, thanks Tazeen, for that question. We are working on our tax planning today I am not sure in a position to give you any guidance on what our tax rate would be at this point in time.

Tazeen Ahmad - Bank of America Merrill Lynch

But there is an opportunity to potentially get a lower rate?

Shane Kovacs

Yeah, we are working on tax planning, to how that works with our international commercial operations.

Tazeen Ahmad - Bank of America Merrill Lynch

Okay, I think you filed a mixed shelf today. Have you disclosed the amount of that and what you would potentially plan to use the funds for?

Shane Kovacs

Yeah, I mean it's really a housekeeping item as you know most companies once they become eligible to file a shelf which is after you've been public for at least one year as more of the housekeeping item just go ahead and file an S3. And we are above that threshold that you don't actually need to file against a specific number. Today we have no plans for additional capital raising and so it's really just to a matter of process.

Tazeen Ahmad - Bank of America Merrill Lynch

Okay. For SMA, can you speak to the involvement that your team has in designing ongoing and planned trials? And can you speak a little bit to what the next phase of development for the product would include?

Stuart W. Peltz

Sure I mean that's really right now in the planning stages. The next phase is to go into patients and we're hopeful that will be aggressive in that. But we're not at the spot yet where we can actually define for you the precise plan.

Tazeen Ahmad - Bank of America Merrill Lynch

So, would that be a Phase 1b trial or would it be a Phase 2 potentially?

Stuart W. Peltz

So this is -- we haven’t really disclosed much except to say that it's been planned to be in SMA patients.

Tazeen Ahmad - Bank of America Merrill Lynch

Okay, and the last question I have is, now that you have the official approval from EU for ataluren, can you comment on how many people you currently have on the ground right now in the EU, and when you expect your commercial team to be fully onboarded?

Stuart W. Peltz

Yeah we're working hard to build the team, may be let me have Mark talk a little bit about that.

Mark Rothera

The important thing is that you don't need a large team to cover countries in Europe for very large or for very rare disease of this nature. I think what counts most is having the right sorts of people with the right experience that know not only the particularities of dealing with a rare disease where you have a lot of stakeholders but also know about building a company. And I think what we focus on is really getting the right people in on the ground. We're very pleased by the progress we're making on that respect.

Tazeen Ahmad - Bank of America Merrill Lynch

Okay. Thanks.

Stuart W. Peltz

Thank you.

Operator

(Operator Instructions) Our next question comes from Christopher Marai with Oppenheimer. Your line is open.

Christopher Marai - Oppenheimer & Co.

Hi, good morning guys. Thanks for taking my question. Just with respect to the expanded access program and potential for reimbursement there, what are we looking at in terms of price of ataluren in those selected markets? Are you providing any guidance there or what do you anticipate will be the price there and how will that differ potentially from the price in markets that are, I guess, launched under the conditional approval pathway? Thanks.

Stuart W. Peltz

Hey Chris thanks for the question. We're not disclosing that right now it may or may not be near the planned out commercial price and so we're not disclosing the pricing of the early access program.

Mark Rothera

However as you can imagine we do expect the ultimate commercial price to be in line with other ultra-orphan drugs when looking at first-in-class therapies you're treating an area of very high unmet medical need and a very small patient population. But we've not really engaged in that negotiation of the country level yet.

Christopher Marai - Oppenheimer & Co.

Okay, then. Then with respect to the program in MPS I, I was just wondering, it's actually a proof-of-concept study. Do you think that, that study will look at endpoints similar to those for Aldurazyme? How should we look at really that market opportunity given current drug out there, and could this potential proof of concept study also be in some way registrationally worthy? Thanks.

Stuart W. Peltz

So thanks for that, it's MPS I this is probably approximately 60% to 80% of patients have a nonsense mutation we're doing a proof of concept study where we're going to be looking at urine and blood gag levels and change those with treatment. You may recall where we work with group to show that we could see reductions of that in a Hurler mouse model. And again one of the things we think is an advantage is the fact that ataluren is able to distribute widely both in terms of the CNS heart as well bone that this would certainly give us an advantage. But first step is really a rapid proof of concept.

Christopher Marai - Oppenheimer & Co.

Right. And then would you have any clinical endpoints, like SCC or six-minute walk tests?

Stuart W. Peltz

We're putting the final touches on the protocol on that but I don't think that probably will be -- will be in the proof-of-concept studies.

Christopher Marai - Oppenheimer & Co.

Okay. And then, finally, when do you think we'll see readout from that proof-of-concept study? Will that be before or after the DMD and CF readout? Thanks.

Stuart W. Peltz

That's always a little I should exactly we're hoping to have results by the end of next year.

Christopher Marai - Oppenheimer & Co.

Great. Thanks for taking the questions. Congrats on the quarter.

Stuart W. Peltz

Thanks a lot. I appreciate it.

Mark Rothera

Thanks Chris.

Operator

Our next question comes from Debjit Chattopadhyay with ROTH Capital Partners. Your line is open.

Debjit Chattopadhyay - ROTH Capital Partners

Hey congratulations guys on the progress so far. Most of my questions have been answered. I'm just wondering if you can just provide some update on the NVR gonorrhea program and the BMI 1 program going forward in terms of clinical timelines for that?

Stuart W. Peltz

Yes thanks for the questions Debjit. We're working towards filing the IND for the BMI program by the end of this year to get that rolling and in terms of the -- so that would be just probably an early Phase 1 in refractory cancer patients. And then the Gonorrhea program we expect to choose a development candidate later this year.

As I said in the talk that this is based on a novel chemical scaffold, really a brand new scaffold, anti-bacterial scaffold which we think is really quite important. And so it's going into safety toxic -- we would plan to have that in safety toxicology in 2015.

Debjit Chattopadhyay - ROTH Capital Partners

Thank you so much. And just one more, on the choice of the MPS versus, say, Hurler's or any of the other indication, what drove that choice for choosing MPS I versus, say, Hurler's?

Stuart W. Peltz

Yes thank you. So MPS I or Hurler's were chosen really as because we think there is a nice pharmacodynamic biomarker that we could readily determine in a relatively short period of time. We still think that there is high unmet medical need, the fact that it can pass the blood brain barriers it uses the same formulation that we have. So and then we think it has a good biomarker and I think the combination of all of that, there will be others that we're obviously looking at but we think that this would be a very good first choice.

Debjit Chattopadhyay - ROTH Capital Partners

Thank you so much and congratulations.

Stuart W. Peltz

Thank you.

Shane Kovacs

Thanks Deb.

Operator

And I am showing no further questions I will now turn the call back over to management for closing remarks.

Stuart W. Peltz

Thank you operator. As we enter the second half of the year we're excited about moving towards the commercial launch of Translarna for DMD in Europe. We look forward to completing enrollment in our ACT DMD study and advancing Translarna in both CF and in MPSI. In addition to our efforts on Translarna we are advancing our early stage programs also towards clinical development.

And clearly what we'll do is communicate with you as we achieve these and other milestones. And again we thank you for your interest in PTC and for joining the call today. Thank you.

Operator

Thank you ladies and gentlemen. That does conclude today's conference. You may all disconnect and everyone have a great day.

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PTC Therapeutics (NASDAQ:PTCT): Q2 EPS of -$0.86 misses by $0.11. Revenue of $1.7M (-75.4% Y/Y) in-line.