Chris Cline - Manager of Investor Relations Steve Aselage - Chief Executive Officer Alvin Shih - Executive Vice President, Research and Development Laura Clague - Senior Vice President and Chief Financial Officer.
Robyn Karnauskas - Deutsche bank Joseph Schwartz - Leerink Partners Do Kim - Nomura Securities.
Good day ladies and gentlemen and welcome to the Retrophin Inc's, First Quarter 2015 Financial Results Incorporate Update Conference Call. At this time all participants are in a listen-only mode. Later we will conduct a question-and-answer session and instructions will follow at that time.
[Operator Instructions] As a reminder, this call is being recorded. I would now like to introduce your host for today’s conference, Chris Cline, Manager of Investor Relations. Sir, you may begin..
Thank you, Kate. Good afternoon everyone and welcome to Retrophin's first quarter 2015 financial results and corporate update call. Here with me today are Steve Aselage, Chief Executive Officer; Laura Clague, Chief Financial Officer; and Dr. Alvin Shih, Executive Vice President and Global Head of R&D.
Before we begin, I would like to caution that comments made during this conference call by management will contain forward-looking statements that involve risks and uncertainties regarding the operations and future results of Retrophin.
I encourage you to review the company’s filings with the Securities and Exchange Commission, which identify specific risk factors that may cause actual results or events to materially differ from those described in the forward-looking statements.
The content of this conference call contains time sensitive information that is accurate only as of today’s date, Mary 11, 2015. The company undertakes no obligation to revise or update any statements to reflect events or circumstances after the date of this conference call. With that, I’ll now turn the call over to Steve.
Steve?.
Thank you, Chris. Good afternoon everyone and thank you for joining us today. We are very happy about the progress we made in the first quarter which was full of achievements that will drive fundamental long term growth to Retrophin and our stakeholders. Operationally, we grew Thiola at a very solid pace.
We executed a highly accretive acquisition of Cholbam which diversifies our commercial portfolio and makes us a leading provider of treatment for bile acid disorders. We significantly de-risk the balance sheet with a successful follow-on equity offering.
From a commercial perspective, I am very pleased with the 23% top line growth over the last quarter in order to capitalize on the momentum we’ve build with Thiola and to effectively launch Cholbam or increasing our sales force to 24 sales representative.
This will expand our commercial reach to cover approximately 2,200 physicians treating cystinuria bile acid synthesis disorder and peroxisomal disorders. Thiola continues to grow meaningfully. As of May 8th, we had more than 725 patients on Thiola. We expect that number to grow significantly throughout 2015 and 2016.
Thiola continues to be considered the standard of care for persistent cystinuria patients and we are working to establish routine monitoring of cystine levels and target urinary cystine concentration levels to up guide treatment of dosing.
We continued to pride ourselves on providing a first class patient experience which appears to be positively impacting compliance. Prior to our re-launch, compliance have been approximately 50% and while it is still very early on, we are now seeing compliance in the 80% to 85% range.
This is a testament to the work being done by our total care hub which provides access and delivery of therapy to patients 24/7 medical support and educational resources. Moving on to Cholbam, which we recently acquired by exercising our option with Asklepion Pharmaceuticals on FDA approval in March.
Cholbam expand our commercial platform and with seven year Orphan Act pursuant in the United States positions us as a leading provider by bile acid treatments.
It is the first FDA approved treatment for pediatric and adolescent patients with bile acid synthesis disorders due to single enzyme defects and patients with peroxisomal disorders who exhibit signs of lever dysfunction.
These patients were generally affected by reduced bile flow, accumulation of toxic bile acid intermedia is the liver and malabsorption of fats and fat soluble vitamins in the diet. If untreated, patients fail to mature physically and may develop life threatening liver injury which can result in liver transplant or death.
The clinical trials of Cholbam treatment have illustrated to potential to prevent progressing of liver disease and facilitate the absorption of fats and fat soluble vitamins which are key to a normal growth rate for patients. From a commercial perspective, we have shipped Cholbam to more than a dozen patients, two of which were new to therapy.
We have roughly ten more patients will receive their first shipment by the end of May. It is important to remember that there were approximately 30 patients receiving therapy in the extension study of Cholbam and our first priority is to continue assisting those patients with the transition to commercial therapy.
We are working now to better define the number of patients that might benefit from therapy with Cholbam. We estimate the insurance for single enzyme defect patients is roughly one to nine per million lives of 20 to 30 new patients per year. They could be potential candidates for Cholbam.
Since chronic Cholbam treatment has a potential to compensate for the panic defect on these patients, we anticipate the majority of successfully treated patients going on therapy will remain on drug for life.
We view the peroxisomal disorder patient population in a very different way based from the literature, we expect insurance will be greater than the single enzyme defect patients that approximately one in 50,000 life.
However, Cholbam in considered not a junk of therapy for the subset of patients that is only able to address the panic component of the multiple organ disorder. Treatment for this mostly pediatric population may not for long life similar to the single enzyme patients.
We are currently doing on in-depth survey of relevant centers of excellence to get a better handle of the number of patients that could benefit from therapy.
While acquisition of Cholbam included worldwide rights and while we assign little value to the international opportunity at the time of the agreement, we do see some potential there if we’re able to restructure some existing distribution agreements. We are working on that now. Ones we are able to gain more clarity on a potential path outside the U.S.
we will show developments. Where pediatric disease priority review voucher was acquired with the Cholbam acquisition after determining there was not a clear strategic benefit to our pipeline candidates. We initiated a process to monetize the voucher. We’ve had considerable inbound interest and discussions are ongoing.
If the process results in a cash return, we would expect to use those proceeds to further support of efforts to develop our pipeline and acquire additional late stage products. Moving on the Chenodal where the patient base remains stable at around 60 patients.
Our discussions with the FDA continued to be constructive for determining an acceptable path rate for addition of CTX to the Chenodal label, the screening study announced last quarter has began and we are aiming to screen up to 500 subject with bilateral cataracts by working with 50 centers in the United States to do high volumes of cataract surgery.
We are hopeful the date from the study will allows us to raise awareness of CTX and lead to a better knowledge about the true problems of this devastating and very difficult to diagnose disease.
Shifting to the pipeline, Alvin and his team continued to make progress which he will update you on shortly, enhance focus this team has been able to operate with after divesting on noncore assets is become a parent.
We continued to be excited about the future potential of Sparsentan for FSGS and RE-024 for PKAN which just recently entered the clinic. Our business development activity has remained aggressive despite starting up the year with remarkable Cholbam transaction, our BD remains on that for strategic acquisitions and partnerships.
We’ve been to create significant value for the company by acquiring underappreciated assets in the past and we plan to continue to do so. With that I would like to now ask Alvin to go through our progress on the pipeline.
Alvin?.
Thank you, Steve. The first months of 2015 were very productive for our research and development. Our focus remains on advancing innovative therapies for rare genetic diseases, as well as optimizing the existing product portfolio through lifecycle management.
On the development portfolio, one of our highest priories is enrolling patients into our Phase 2 DUET study, which is testing our lead assets Sparsentan and focal segmental glomerulosclerosis or FSGS.
Our clinical operations team is working diligently to achieve our target of completing enrollment of 100 FSGS patients in this study by the end of this year. We have more than 30 active sites worldwide will provide further updates as we make progress.
I am happy to share that the Data Monitoring Committee met for the second time recently and has approved the continuation of the study and the opening of the highest dose cohort.
We continue to work hand on hand with NephCure, the leading patient efficacy group in the space as well as NEPTUNE contortion of physicians and academic medical centers who are actively managing FSGS patients.
Shifting attention to the earlier stage pipeline, there has been positive news flow in recent weeks with the progress RE-024 which is out novel therapeutic in development for the treatment of pantothenate kinase-associated neuro degeneration, or PKAN.
In the first quarter, we filed an investigational new drug application with the FDA and last month, the agency gave us clearance to proceed with the Phase 1 study. This study will to aim to assess the safety tolerability and pharmacokinetics of single doses of RE-024 in healthy volunteer subjects.
Our clinical teams preparation allowed us to initiate screening of healthy volunteers for the Phase 1 trial almost immediately after received FDA clearance and we expect to begin dosing in the very near future. Also of note, just last week, the FDA Office of Orphan Products Development granted RE-024 orphan drug designation for the treatment of PKAN.
These two regulatory milestones have allows us to build significant momentum in the development of RE-024 and they further validate the potential for this program to help PKAN patients. With regard to the four patients outside the United States, we’ve been receiving RE-024 as part of physician initiated studies.
I am happy to report that all four patients remain on RE-024 therapy. I want to give credit to the entire R&D team which has worked diligently to move RE-024 forward as quickly as possible, as well as the scientific and patient communities which have been supported by our effort.
This is truly a team effort and we operate with commitment and a shared sense of urgency to help this patient population. Briefly on RE-034, the R&D team continues developmental work which mainly to the initiation of IND enabling studies later this year.
Regarding support of our commercial assets, we’ve remained committed to optimizing the value of the current portfolio of marketed products. For example, our regulatory team is in constructive dialog with the FDA to seek the addition of cerebrotendinous xanthomatosis of CTX to the Chenodal label.
And for able to achieve this label change, we will be able to do more for patients which is difficult to diagnose disease. As Steven alluded to earlier, we’ve also begun work to initiate a prevalent study of CTX.
We know that many CTX patients present with pediatric bilateral cataracts which is one of the key opportunities for early diagnosis before irreversible cognitive decline.
The screening study will have the twofold benefit to helping us understand the true nature of prevalence for this disease, while also raising awareness in the major academic medical centers where diagnosis will be made going forward.
Regarding our newest asset Cholbam, the R&D team is fully engaged in supporting the launch and adding efforts to improve diagnosis and treatment. We’re working to develop a next generation diagnostic which will aid in the identification of bile acids synthesis disorder patients.
We continue to support the commercial and medical teams during the launch of this critical therapy for a small then important group of patients. And last by not least, our discovery scientists are working hard to ensure sustainability of our pipeline via next generation therapeutics for rare diseases.
We are prosecuting novel targets in the rare genetic disease space and we’ll provide updates as we get closer to the clinic. We are also evaluating multiple opportunities to collaborate with patient efficacy groups on early stage of research efforts.
As with everything in the rare disease space, the patients are the center of our research efforts and we look forward to working with patient groups to accelerate the development of therapeutics in multiple rare diseases. With that I will turn it over to Laura.
Laura?.
Thank you, Alvin. From the financial perspective, the first quarter marked significant advancement for Retrophin. Our net products sales continued to grow and our new finance team is focused on efficiencies and cost controls at the end positively impact our ability to reduce expenses.
We’re also able to bolster our balance sheet with net proceeds of 140 million from our follow-on equity offering of approximately 7.9 million shares. This offering was significantly oversubscribed and closed at the end of March. Net product sales consisting of Thiola and Chenodal were 17.4 million in the first quarter.
The 23% increase over the last quarter is largely attributable to the re-launch of Thiola. As Steve pointed out earlier, we continued to expect meaningful growth of Thiola as we make additions to our sales force and target new physicians.
We’re also excited about our recent acquisition of Cholbam which added as third commercial asset and will contribute to net product sales net quarter. We’ve reported GAAP net income of 39.7 million for the first quarter of 2015. Adjusting for extraordinary and onetime expenses, we ended the quarter with a net loss of 1.1 million.
The key elements of the change this quarter are due to the accounting for our Cholbam acquisition including the gain that resulted from valuation of the related where pediatric disease priority review voucher that came with approval. This gain of 48.6 million was reported net of tax.
You will also note on our balance sheet that our intangible assets increased due to the addition of the fair value of the acquired product rights for Cholbam and the acquisition related contingent consideration increased reflecting the future royalties and milestone we will pay for Cholbam.
It’s important to note that we placed a value on a voucher in our Q1 financial statements based on GAAP accounting rules that required at the asset we recorded as fair value at the date of acquisition. And determining that fair value, we have valuated many factors including their recent sales of similar vouchers.
Clearly the final selling price will be determined by the benefit perceived by potential buyers who may see value very differently than what is reflected in the current financials.
Assuming the process results in a cash return, we would expect to use those proceeds to further support our efforts to develop our pipeline and acquire additional late stage products. Research and development expense on a GAAP basis were 10.3 million for the first quarter compared to 6.9 million for the same period last year.
This increase is principally attributed to the extension of the R&D department to support the development of our lead development candidates Sparsentan, RE-023 and RE-034. Notably, we saw a decline in R&D expenses from the four quarter of last year as a result of divestment of noncore asset and lower CMC costs.
Please keep in mind that we expect our R&D cost will not be balanced throughout the year as the timing of expenses for the DUET trial and RE-024 development will vary. On an adjusted basis, R&D expense for the first quarter was 7.8 million. Relevant non-cash expenses for the first quarter included 2.2 million of stock based compensation.
Selling, general and administrative expenses declined slightly on a GAAP basis compared to the same period last year. The reduction in SG&A expense compared to last quarter is due to the absence of extraordinary consulting fees. On ad adjusted basis, SG&A expense for the first quarter was 8 million.
Notable onetime and non-cash adjustments for the first quarter consisted of 5 million related to stock compensation and depreciation and amortization and 2 million of legal expenses.
As of March 31st, 2015 we had approximately 126.3 million in cash and cash equivalents in marketable securities which was boosted this quarter by the earlier mentioned equity offering resulting a net proceeds of a 140 million.
Offsetting this inflow in the first quarter was approximately 32 million of payments to Asklepion for the auction to acquire in the eventual acquisition of Cholbam. To briefly wrap up, the progress made in the first quarter’s financial performance is clear and set this up to successfully execute Retrophin’s strategic plan moving forward.
I will now turn the call back over to Steve for his closing comments.
Steve?.
Thanks Laura. While we are proud of progress we’ve made this start 2015, we have our site set much higher. Retrophin has now been in a better position and create significant value for the stakeholders.
We’re moving into the balance of ‘15 with growing revenue base, we will benefit it from a third growing assets starting in the second quarter, while leveraging stronger cost controls will allow us to continue operating more efficiently.
We now have a stronger balance sheet that will enable flexibility and pursue our business development strategy and pipeline advancement. We look forward to continuing the executing of our strategic plan. With that I will now turn over the call to Chris for questions.
Chris?.
Thanks Steve. Kate, can you please open the line for Q&A..
[Operator Instructions] And our first question comes from the line of Robyn Karnauskas of Deutsche bank. Your line is open. Please go ahead..
Hi guys, thanks for taking my questions. So two questions actually.
Fist regarding Thiola, so you feel like you have reached all the low handing fruits to patients that were out there that they have previously been on therapy and how do we think about those patients you know like this kind of patients that would coming on over the course, how do we think about the year? And then the second question is regarding RE-024, when could we get there and is a stock that these patients staying on drug, do we know that that means efficacy or does that mean that drug is just phase.
And so can we read and see anything as efficacy given, how long these patients have been on drug? Thanks..
Sure. Let me start with the Thiola. I would now say we’ve got all the low hanging fruit, but I think the majority of the patients who are on therapy previously we’re happy with the results we’re getting and really wanted to go back on. Those patients were back on therapy.
What we have focused initial efforts on is getting back to the physicians who were previous prescribers of Thiola and I think we’ve done a pretty good job of covering that prescriber universe. While we haven’t been able to do here is good to add into some of the clinics that don’t have a history of prescribing Thiola.
We feel that there is a significant opportunity there and there is going to be ability to further grow the patient base as we increase our reach.
And as we mentioned previously, we are expanding the sales force with 16 people who did an incredible job and covering most of the major centers, but we feel like by expanding up to 24, we’re going to increase our coverage universe by 500 to 600 additional potential prescribers. We think that’s going to help drive business over the rest of the year..
And Bobyn, let me address your questions on RE-024 or you have some follow-up for Steve..
Just on the follow-up there. So when we think about like the trends when you are going from getting logging for patients, they are going up there and getting new patients.
Should we think about how like the average patients for monthly come in a little bit before those sales people kind of ramp up, so should we expect to see fewer patients for month in the near term that steady stream going into the back half of the year as those patients are brought on therapy or how do we think about that?.
Well I think you may want to think about in terms of we got a big ball of certifications initially and I think the - prior to this call the two data points we gave you showed roughly 50 new patients per month coming in.
The actually accumulation or increase in patients over the last couple of months has been a little bit slower than that, it was between 35 and 40 new patients per month, whether we can keep study or whether we can even ramp it up a little bit with some better coverage remains to be seen.
I can’t give you kind of a crystal ball diagnosis of exactly where the line is going to become stable and see it week-over-week, month-over-month consistency of patient growth. What I can see is we feel comfortable that we are going to see meaningful increases in patients numbers the rest of this year.
But it looks at those point like we’re not going to be able to continue that 50 patients a month trend that you saw late Q4 and early Q1..
Great, thanks..
And let me tackle your questions on RE-024.
The Phase 1 trial is not designed to be a factored study, so we - envision that we’ll get the results on that relatively soon, certainly before the end of the year and the goal would be to have that safety, tolerability and PKAN data and have that date be submitted for publication before the end of the year. So with regard to the - go ahead Robyn..
Sorry, go ahead. Sorry, I was asking the question again..
Yeah, so I would say in terms of the four patients outside the U.S. who are still on therapy, I think it’s an optimistic signal that they in combination what the investigators have chosen to remain on therapy. I don’t want to read more into that date that is wanted before of course these are outside U.S. trials that are really uncontrolled by nature.
I think what it does help us is understand the safety profile which is important and then helps us think about how efficacy will ultimately be measured in trials when we are able to conduct those in the patient setting in the U.S..
Great, thank you..
Thank you. And our next question comes from the line of Joe Schwartz of Leerink Partners. Your line is open. Please go ahead..
Thanks very much and congratulations on all the progress.
First of all if you could give us an update on the process to update the Chenodal label for the CTX indication, what is involved there?.
Yeah. So Joe, the process is that we have to remain in discussion with the FDA and map out a mutually agreeable process by which we get that on label. It’s a very unusual situation, so this is one where it really does require close communication, negotiation with the agency.
So we have met with the agency and have submitted a plan that we believe will be achievable and acceptable. And at this point, we are waiting for feedback from the agency. So when we have something further to report, we will provide an update..
Okay, great.
And then what we can define if anything from the recent DSMB decision to enroll patients in the highest dose cohort of DUET, where there certain rates of DEMA for example that were pre-specified that would have prevented that from happening?.
No, I think all we say at this point is that based on their review of the blinded data they saw fit to open enrolment for the highest dose cohort. So that’s really all I would read into it at this point because the date are blinded..
Okay. And then maybe I can squeeze in a one last one.
How do you expect to develop RE-034, what are the next steps for this and do you think you will wait for Sparsentan do that data before advancing this asset into clinical trial or do you have plans to move ahead regardless?.
Yeah, right now we are trying to maximize our strategic options with this early stage asset. We’re considering multiple development pads and mapping out how we might push this forward into the clinic. As you know we filed a provisional pantothenate application to cover the novel formulation.
And right now that’s forming a basis trying to figure out the appropriate path because this sent to IND enabling studies this year..
Maybe one last, last question, given that Acthar was approved with a pretty non-traditional lease nowadays root at the FDA, how - what was the regulatory pathway be that you envision, would it be something life 505(b)(2) anything more abbreviated in a full-fledged clinical trial program?.
Yeah, right now that’s part of the discussion that we would have to have with the regulators. So I don’t have a definitive answer for you right there..
Okay, well thanks for taking all my questions..
Sure..
Thank you. And our next question comes from the line of Do Kim with Nomura Securities. Your line is open. Please go ahead..
Hi. Thank you for taking my question.
A question for Alvin on RE-024, I was hoping that you could provide more details on the Phase 1 study design like how many doses will be evaluated, what is the dose that being used in the investigator initiated studies fall, maybe the time points for PKAN measurement? And also maybe if you could go over how PKAN is diagnosed and whether there is a system in place to identify the patients or is the population is under diagnosed like some other rare diseases?.
Yeah, so let me try to address those questions and if I forget anything just jump in. So the Phase 1 is intended to dose, as I mentioned it’s a single ascending dose trial and it will cover multiple doses that will bracket the clinical doses that we have already been seeing.
So we intend on starting below and then going higher than the doses that we’ve seeing in the ex-U.S. experience so far. We’ve designed it to be as flexible as possible. Right now the plan is to have five dosing cohorts.
And you asked about PKAN measurements, we will be looking at PKAN over the course of several days and of course most of those measurements would occur early on, so that we make sure we get a good assessment early on in therapy and we’ll got out to a time period of a couple of days..
Great.
Just could you tell us how the population of our patient of PKAN is diagnosed and whether or not that is - the population is under diagnosed like some other rare diseases?.
Sure. Our sense is that just with any rare disease where no current therapy exists, the diagnosis is probably lower than it should be. With PKAN there are a couple of different avenues of diagnosis, the first is radiologic.
So as patients present with this certain consolation symptoms that presumable a movement disorder as well as neurodegeneration, they often get a MRI and then MRI will have a very typical pattern which is called the eye of the tiger sign and so that one way of moving towards the diagnosis.
The other means of diagnosis is by running generic tests to have the PKAN gene sequence to understand if there is a definitive generic defect going on. So those are really the two avenues of diagnosis.
We do think it’s somewhat under diagnosed and part of the development work that we’ll be doing is really trying to map out where these patients are and how many of them there are. Our estimate right now is that they are between one and three per million population, but that’s a number they would have to be verified overtime..
Great, thank you for taking my questions..
Thank you. And I am showing no further question at this time, I’d like to turn the call back over to Chris Cline for any closing remarks..
Thanks Kate. With that I’ll bring the call to an end. Thank you all for joining us today and we look forward to updating on our progress next quarter..
Ladies and gentlemen, thank you for participating in today’s conference. This concludes today’s program. You may all disconnect. Everyone have a great day..