Rigel Pharmaceuticals, Inc. (RIGL) Q3 2016 Earnings Report, Transcript and Summary

Good afternoon, and welcome to Rigel Pharmaceutical's Earnings Conference Call for the Third Quarter of 2016. All participants are in a listen-only mode. We will be facilitating a question-and-answer session at the end of today's conference. [Operator Instructions]. I would like to remind you that this call is being recorded for replay purpose from Rigel's website. [Operator Instructions]. And now, I will turn this conference over to our first speaker, Dolly Vance, who is Rigel's Executive Vice President, Corporate Affairs and General Counsel.

Hello and welcome to our quarterly earnings conference call. The financial press release for the third quarter of 2016 was issued a short while ago and can be viewed in the news section of our Investor Relations page on our website at www.rigel.com. As a reminder, during today's call, we may make forward-looking statements regarding our financial outlook and our plans and timing for regulatory and product development. These statements are subject to risk and uncertainties that may cause actual results to differ from those forecasted. A description of these risks can be found in our most recent Quarterly Report in Form 10-Q on file with the SEC. Any forward-looking statements are made only as of today's date, and we undertake no obligation to update these forward-looking statements to reflect subsequent events or circumstances. At this time, I would like to turn the call over to our CEO, Raul Rodriguez.

Good afternoon. In addition to Dolly and myself, Ryan Maynard, our CFO; and Anne-Marie Duliege, our Chief Medical Officer are participating in the call. We also have Eldon Mayer, our new Chief Commercial Officer joining us to listen in. On today's call, I would like to review the events of this past quarter. It certainly has been an eventful quarter. Most importantly we completed the Phase 3 clinical studies for fostamatinib and ITP or Immune thrombocytopenia. We would like to use some of this time to review briefly those study results and discuss our next steps and update you on other items. We have continued to look at the data from the FIT Phase 3 clinical program and continue to be very enthusiastic upon fostamatinib. We are encouraged by the consistency of the data across the various trials. Fostamatinib appears to show clear clinical effect and an enduring benefit for those post difficult to treat ITP patients. We are having discussions with the FDA soon about the FIT results in our planned NDA application. And we are hopeful that those discussions will facilitate our submitting the NDA in the first quarter of 2017. Our goal at Rigel is to transform the company into a profitable biotech company with a focus specialized commercial organization aimed at providing new therapeutics to patients with ITP and other indications and their healthcare providers. Towards that goal and in addition to the Phase 3 completion, we undertook two major initiatives this quarter. One, we had a significant restructuring; and two, hiring key senior managers to help drive us towards that goal. In September, we announced the restructuring primarily affecting our research area, reducing our headcount by 46 physicians. While it's never easy to make cuts and affect good employees, the restructuring was an important business decision for Rigel. We have -- we're seeing the most promising and near-term research projects for our own new product development and the conservative resources will allow us to be -- will be directed towards moving fostamatinib towards regulatory and commercial approvals. We have also announced some key management appointments that reflect this new focus. I'm happy to have Eldon Mayer join us here today as Rigel's Chief Commercial Officer. Eldon will be establishing and leading the pre-market and launch planning looking to make our first product of success. In the future, he will provide updates on commercial plan and performance. We also recently hired Joe Lasaga as Vice President of Business Development and Alliance Management. Joe will be working to establish partnerships for fostamatinib outside of the U.S. He will also be working on various other partnering opportunities earlier in the pipeline. And finally we have named Dr. Esteban Masuda as Senior Vice President, Research. He will head our refocused drug discovery efforts. Esteban was the first project leader for fostamatinib and still serves as the go to resource for that product. We look forward to the contributions of these management members and expect to add a few commercially related managers in the coming months. At this time, I would like to turn the call over to Ryan for a report on third quarter financials. Then we will spend some time discussing the fostamatinib results and the next steps. Ryan?

Thank you, Raul. For the third quarter of 2016, we reported a net loss of $22.6 million or $0.24 per share compared to a net loss of $6.7 million or $0.08 per share in the third quarter of 2015. We reported revenues of $3.8 million this quarter that was the final amortization amount related to the upfront payment we received from Bristol-Myers Squibb. As Raul mentioned, we announced a workforce reduction in September of 46 physicians, mostly in the research area. The resulting restructuring charges of approximately $5.8 million were recorded in the third quarter and included $5 million in severance costs, $319,000 of impairment of lab equipments, and $499,000 of non-cash stock-based compensation expense related to a former Head of Research. As of the end of the quarter, we have paid out $3.8 million of the $5 million of severance costs. Total cost and expenses of $26.5 million were reported in this quarter compared to $19.8 million in the second quarter of last year. The primary reason for the increase in cost this quarter was due to restructuring costs I've already mentioned. As of September 30, 2016, we had cash and investments of $85.3 million. During the last quarter, we completed the utilization of the ATM facility through Cantor and have no amount available to draw under that facility. We believe our cash resources on hand are sufficient to take us through the end of 2017. In this forecast we have allocated a substantial amount of cost to prepare Rigel for the potential commercial launch of fostamatinib. This outlook also does not include any milestones from our current partners or any potential upfront payments from new collaborations we will be focused on. If we're successful on those fronts, we will further extend the runway into 2018. At this time, I would like to turn the call over to Anne-Marie Duliege, Rigel's Chief Medical Officer.

Thank you, Ryan. As Raul stated earlier, our team is in the process of evaluating and compiling the data for the full collection of clinical studies for fostamatinib in patients with ITP. For the purpose of today's call, I'd like to review the data that we have previously presented in August and October of this year. And so this is going to be the identical presentation to what we just did. But to begin with let's talk about chronic persistent ITP and this is on Slide 9. As a reminder, ITP is an autoimmune disease in which the body distorts its own platelets. SYK Kinase is a key player in the immune system destructions of platelets in ITP. Fostamatinib is a SYK inhibitor and targets the underlying cause of ITP. The platelet count for normal healthy adult is generally above 150,000 platelets per microliter but ITP patients can have platelet counts for below 30,000, the level at which they are generally considered at risk for spontaneous or trauma induced hemorrhage. People with ITP may not know when or how severely the platelets counts have dropped until they actually experience severe symptoms. So maintaining a stable platelet count has been a significant challenge for patients and their physicians. In addition, patients with ITP are very heterogeneous patient population although there are some treatments available that support the new systems and support platelet growth; there is no one treatment that works for everyone. In addition, it's difficult so I would say it's actually very challenging nearly impossible to predict which treatment will work for which patient. There are approximately 50,000 to 60,000 adult patients with primary ITP in the U.S. making it to be an orphan disease. On the next slide, Slide 10, this is a summary of the structure of the program. The program consisted of two Phase 3 randomized control studies 047 and 048 and one long-term label extension study. So approximately 150 patients were randomized in these two trials each of 75 patients and obviously assigned to one of these two trials and then randomized 2 to 1 to either fostamatinib or placebo. They could drop at some point in time and move on into the extension trial. The primary endpoint in the two placebo randomized control trials was stable platelet counts which is defined as platelet counts above 50,000 on at least four of the six visits that took place in the second part of the studies which is between weeks 14 and week 24. On Slide 11, we have the figure of the primary endpoint in the Phase 1 study. As you may remember nine patients which is 18% in the active group responded compared to no one in the control group and that's and therefore the statistical significance was met, was met at the level of 0.0261. In the Phase 2 study 048, while there was a very same response rate, 18% in the active group, there was one patient in a placebo group that met the criteria for response that was 4% and for this reason the study did not meet significance with a P value of 0.15. And on Slide 13, when we combine these two studies, something we will indicate in our submission of the data to the FDA, then the combined response rate is 18% in the active group on one hand and then 2% one out of 49 in the placebo group and the P value is 0.007. On Slide 14, we have provided the figure of the median platelet counts over time for the various subgroup of patients in the two randomized controls trial. So on the X axis, you have the weeks during 12 and then the median platelet counts on the Y axis. The green line shows the median platelet counts for the 18 responders in the fostamatinib group and what we see here is that the response is pretty rapid. There is a quick increase noticeable early as week two of treatment and the platelets, we continue to increase and reach a level of approximately 100,000 again that's a median count at week 12 and maintain this level consistency thereafter. By contrast, patients who either were fostamatinib non-responders in blue or placebo non-responders in grey had platelet counts that essentially did not change over time and remain largely under 40,000. And then in yellow is the placebo patients responded in the placebo groups, the placebo responder with platelet counts bouncing over doing other studies. Switching now to the summary of the safety finding in this program on Slide 15, remember the two key message from our safety analysis. One, importantly serious adverse events were not more frequent in the active group versus the control group. Second, there where indeed more frequent treatment related adverse events with Fosta compared to placebo patients and they were very well identified inside, this was very consistent with a profile that had been seen in previous programs such as in patients with rheumatoid arthritis. Specifically there is a higher incidence of patients with gastrointestinal complaints mostly nausea and diarrhea, a trend towards a higher frequency of hypertension during the trial and also a trend towards higher frequency of transaminase elevation. These adverse events are largely mild in terms of intensity about 75% of the case or even moderate very rarely are they severe exceptionally. So a well-known safety profile consistent with what we knew before, no new information here and overall these adverse events are all quite manageable. At this point, I would like to switch on to the long-term extension study, which is on 12 -- 16, so here at the time when we did this interim analysis in June, 118 patients had entered into the trial and had monitored data as to June 2016. On Slide 17 we had really two questions in terms of efficacy. Number one naturally was whether we could demonstrate the patients who had responded upon exposure to fostamatinib in the parent trial maintained the response, and the answer was yes, generally patients maintained platelet counts above 50,000 in the long-term extension study and more specifically the median count is 96,000 very consistent with where patients were at the end of the current studies. And as a reminder here, the median exposure to fostamatinib for these group of responders is 13 months which is substantial and that includes six months in the parent study and seven months as a median in the 049 study. The second and separate question is what is the response rate in patients newly exposed to fostamatinib in study 049. So at the time we did this analysis, 43 placebo non-responders had transitioned from the original study to 049 and at that point starting to receive fostamatinib. Of those 43, 36 had sufficient data to evaluate their ability to respond to the drug to fostamatinib and actually six out of 36 patients had a stable platelet response which is 17% at a P value of 0.01 meaning statistically significant. And these are -- this is was a predefined analysis, one could consider it potentially separate from the other 12, independent of the other 12. So this 17% rate is very consistent with the 18% rate of response to fostamatinib that has been observed in studies 047 and 048. And that's really very reissuing. From that we're moving forward with our NDA effort and that's on Slide 18. We have already obtained input from regulatory consultant we will continue to do so, but in the next coming few months, we also intend to obtain input from the FDA and we're putting together our package of top-line results to submit to the FDA. Pending our FDA's feedback, we intend to submit the NDA do stay on time as we had planned in Q1 2017 and providing that the FDA will accept this submission for their own filing to the FDA itself, then we will support the review of this submission by the FDA of course and we also can anticipate that, we are likely to have an advisory committee review, if that indeed happens, we expect that this could happen by the end of next year about Q4 of 2017 and periodically the approval to PDUFA date could be in Q1 2018. Raul, I'll turn to you.

Thank you, Anne-Marie. So we look at the totality of the fostamatinib clinical data and in particularly FIT Phase 3 clinical program, there is some important points that we would like you to remember. One adults with chronic ITP are very much an underserved patient population and their disease is unpredictable and quite serious, it's also an orphan disease. Patients and physicians' team and Heemonks who treat them are in search of new treatment options, our market research has shown that both groups would consider fostamatinib an attractive new medical option for it. Fostamatinib has a unique mechanism of action which targets the underlying immune cascades that causes the platelet destruction in ITP. None of the existing therapies target this autoimmune basis of this disease. The Phase 3 data have shown that fostamatinib works quickly, achieves a stable, and an enduring platelet response in some of these ITP patients, it very much confirms what we saw in the Phase 2 study with fostamatinib and ITP. The rapid response is helpful because it provides an early feedback as to whether fostamatinib will be a viable treatment option for that patient with ITP and the enduring platelet increases keeps patients, gives patient and their doctors confidence that this disease is under control in the long-term, that's very helpful. On the safety side, the Phase 3 program was very much as expected. Rigel previously had accumulated approximately 5,000 patient years of experience with fostamatinib; the safety profile is well defined and quite manageable. Anne-Marie and I had the chance this past summer to attend the Platelet Disorder Support Association Conference in Orlando and we had a chance to meet a number of patients who suffer from ITP and for most of us it's about platelet count increases and it's actually quite critical. We got a chance to learn how serious this disease is for them. They suffer from bruising on a continual basis. They are always fatigued and it really very much limits their lives. Also they are always living in concern that whatever they're on will continue to work or whether they will be able to stay on it in the long-term. So there is a tremendous medical need and I think we came out of that meeting quite satisfied that we're doing something that is going to benefit these patients greatly. As Anne-Marie indicated, our priority is to submit the NDA package, the project is well underway and we hope to have this completed early next year. I want to update you on a couple other clinical efforts as well. We are conducting a Phase 2 clinical study of fostamatinib in patients with IgA nephropathy. IgA nephropathy is a very serious autoimmune disease that severely affects the functioning of kidneys. And many of these patients eventually require dialysis and kidney transplant. We expect to have results from cohort 1, 100 milligram BID cohort by year-end and expect that we will give us a signal that we're on the right track. Further second cohort 150 milligram BID cohort will likely give us a more robust efficacy readout. We're also conducting a Phase 2 study in autoimmune hemolytic anemia with fostamatinib. Autoimmune hemolytic anemia is another rare immune blood disorder but instead of platelets, this disorder produces antibodies resulting in the destruction of red blood cells. There are currently no approved therapies for autoimmune hemolytic anemia. Our Phase 2 study is currently enrolling and we hope to have those results by next year. And finally our research group is selecting a small molecule candidate from our IRAK program. Our plan is to file an IND for that molecule next year. So before closing, I would like to say a few words on our financials. Our restructuring this past quarter has created the flexibility in our financial runway. We have enough cash to take us in to the end of 2017, so we think our financial position is quite good. So I would like to at this point open the call for your questions.

Thank you. [Operator Instructions]. And our first question comes from Daniel Wolle from Piper Jaffray. Your line is open.

Hi guys. Thanks for taking the questions. What do you expect from the meetings with the regulatory agencies regarding fostamatinib? And my second question has to do with AIHI program. Can you provide us with updates on the timing, more specifically than next year and could that update how many patients worth of data you anticipate to see and if there is any particular signal that you're looking for to make a go or no go decision on that? Thank you very much.

Anne-Marie may be you do the first one, I will do the second.

Perfect. So the first one where do we expect from the FDA. We're about sent a small package of data with the top-line results. We really don't want to speak on behalf of the FDA for sure not but it's our sense that on one hand given that this is a orphan disease and we have the orphan status, orphan drug status that these if there is an unmet need and these are serious patients, given the nature of this placebo patients that we described too in the graph, it is quite likely that the FDA will be pragmatic about it and we want to at least to take a deeper look at the data and we do our package. So we will see what they say but we do anticipate that. If they decide indeed to review our package, they will focus as they should on the risk-benefit equation of the drug and we believe here that we have a strong rationale based on what we have described to you on one hand, a very consistent treatment effect albeit in a small patient population; and on the other hand, a well-known list of potential adverse events with a proposal on how to manage them and this is backed up by an incredibly large 50 database that comes not so much from ITP but from the RA program. A very unusual situation for an orphan drug, so that's where we know we believe and we know that the discussion will focus.

On autoimmune hemolytic anemia, this is a rare disease and it's slow to enroll as a result of that, so we have to be a BIT patient. I don't have the confidence now on when exactly we will have that read-up but I think it will be next year and as we get more patients under our belt, we will give you a more granular estimate of that.

And on signal you might be looking for?

Here we're looking very much like we saw with our Phase 2 trial in ITP, we're really looking for are we having a benefit in these patients, are we able to raise hemoglobin levels. And I think we're going to be pragmatic in how we look at that but obviously since there is nothing approved for these patients and there is a substantial medical need, I think we're going to look to see what raised that finger listen, make a decision as to whether that's useful clinically. We may do some market research with that result to verify that's helpful with some doctors who treat autoimmune hemolytic anemia on a regular basis and certainly the investigators in the trial would give us some guidance as to whether that result is something they find attractive. We are yet to do that but we plan on so doing.

Thank you. And our next question comes from Eun Yang from Jefferies. Your line is open.

Thank you. In the past that you said that the autoimmune hemolytic anemia Phase 1 data was expected in early 2017. But today I will take it doesn't sound like it wants to come in only 2017, is that a fair assessment?

Yes it is. I think we changed that guidance may be some time ago or may be a quarter ago or so. It's just been slower to enroll than we had hoped. The patients here with autoimmune hemolytic anemia not very engaged with their disease given that there is no therapy, available therapies for them. So we have had to do much more outreach towards them to get them to effectively come out of the shadows and participate.

Okay. And then this IgA nephropathy, so I think that you mentioned that about 150 milligram dosing may be needed to some effect. So the 100 milligram dose at the core data that we are expecting by end of this year, is it fair that we do not expect much of it because more of the safety?

I think we're hoping for a very good safety out of it but usually we see a higher level of efficacy at the higher dose group which is why we're saying, we think, we will see better efficacy in the second dose cohort. But that's not to say I don't know what the results will be in this first one as yet it's still obviously blinded.

Okay. But very soon you're expecting the data by end of this year, correct?

Yes it is, we should have it by the end of the year and usually we do some announcements the week before JPM and that might be a good time to talk about it.

Okay. And are you still planning to host on events at ASH this year?

We've applied for -- applied for a late breaker session, those are rare and there is many applications, so we're not sure if we're going to get it or not. I hope we do and if so we would like to have a call post that to discuss the presentation.

Okay. When would you find out whether you -- you're [indiscernible] for late breaker presentation?

By the middle of this month roughly.

This November.

Yes ASH is literally in a month from now, so we will know pretty soon.

Okay. So once you get it, you would announce the date, correct?

Yes, there we're presenting, having the presentation and a call of post.

Thank you. And our next question comes from Anupam Rama from J.P. Morgan. Your line is open.

Hey guys this is Eric in for Anupam. Thanks for taking the question. Just for the Phase 3 FIT studies, just looking to better understand what you saw in terms of discontinuation rate, I know the other weekend where you talked about some patients having discontinued for non-response thing going on to the extension study. But wonder if you could provide some detail around any discontinuations related to tolerability or otherwise within the first 12 weeks on study and then I have a follow-up.

You absolutely need and I think the question was [indiscernible]. And the rate of discontinuation due to adverse event is not substantially different, really small and fairly similar between both groups. However clearly and as expected there are -- there is a larger rate of dose reductions in the active group compared to the control group because of adverse events. And this is first of all a reality. But second this is really guided by the protocol, investigators in the protocol or guided to dose reducing issue for diarrhea or hypertension or even transaminases and that's happened. So part of our analysis will be down the road to the FDA to provide a detailed analysis of both dose reductions as well as dose continuation, discontinuation but there is not a substantial difference in terms of discontinuation.

Got it, thanks. That's helpful. And just secondly I'm curious to know among non-responding patients in the first Phase 3 studies, I think went on to the open label extension trial 049, I'm wondering if there are any notable changes in platelet counts with longer time on drug? Thanks.

So are you talking about the non-responders -- the responders sorry?

I think so the responders.

The responders --

Well may be I can just kind of clarify, my understanding from the design is that non -- initial non-responders from those are the Phase 3 studies would have the potential to continue on to the extension study?

Yes that's right, so for these patients nothing has changed at some point, if you're non-responder initially we haven't observed any substantial increase in platelet count after six months. There is no reason for them to be late responders and so at some point, they will probably discontinue the study. The response that we have observed where one in the parent study these 18 patients initially and as we've said the response is pretty rapid as some condition as earlier in two weeks but we've in fourth week we probably see the majority of response happening. And then separately the same thing is happening in 049 this time only for those patients initially randomized to the placebo group that now start receiving fostamatinib in the open label.

Got it, got it. I guess given that as an open label study, is there the chance that you might have an incremental -- incremental update from the still remaining patients that didn't have 24 week follow-up they might take into discussions with FDA?

For the patients that didn't have 12 weeks, yes, so certainly with the product submission to the FDA, we intend to submit, we will have many data as we can on the 049 studies and in past we will make sure that we maximize all the data we can have on this long-term extension because clearly we know that the FDA will be interested by these two questions and also additional safety data that we may generate. So yes we will maximize the data from 049 in our submission.

Thank you. And our next question comes from Do Kim from BMO Capital Markets. Your line is open.

Hi thanks for taking my questions. Just a follow up on the open label long-term extension study. Do you have a sense of why so many non-responders decided to opt into the study given that they hadn't seen any stable platelet response, were there any other benefits they were gauging from fostamatinib positive like body of late?

This is why the uptick to continue to go into 049 is because they were blinded, nobody knew whether they were in active versus control. And so they probably believe that there was agenda they would be in a control route and therefore by going into the open label extension that they would get something new which may lead them to respond. Of course for those patients who actually were not respondents to Fosta that didn't happen and they probably saw that pretty quickly after that.

Okay. And that makes sense. And has the ITP result changed any how you think about or how you approach the Phase 3 study design for the autoimmune hemolytic anemia study?

No, no not really, we didn't adjust that design; it's a design where the primary end point as opposed to being low-pace headcounts is an increase in the value of hemoglobin for these patients.

That's on the current Phase 2 study. We have not begun to think about what the Phase 3 or a pivotal program might look like for autoimmune hemolytic anemia.

Do you think it's possible that you would see a similar response rate given the similarity to these mechanisms?

Really we, yes that's the basis, that's very much the basis of the whole rationale potentially but truly we don't have a sense, nor do we want to make any sort of superstition about what we might see that will be too early. Right now, as Raul indicated, because of the ultra rare nature of this patient population and the sign that these are not acute patients and forever they have been staying with their current medical status and lack of treatment, it simply said a difficult place to enroll and that's not specific to autoimmune hemolytic anemia. In general for all orphan drug situation and we saw that in our original effort in ITP, it takes a while to enroll patients in this trial and we're extremely active in it but that's our focus honestly back.

Okay. Thank you. And may be one commercial question, what are the next steps in pre-launch preparations and what do you think the timing of those would be?

I will take that, yes, hi this is Eldon. I think at this point, I joined the company a few weeks ago, so my next step is to really refine and continue to build-out the business plan and specifically the launch plan of that. So I will be focusing on that just to really determine what is the right approach for this for our company and for the market and for our customers. But specifically one step of that of course will be one step done to determine what the team need to look like and so I'm in a process now of identifying and looking to hire soon some key management that will be instrumental in helping you to build out those plans and to validate some of our addressed strategy that we have and everything else that needs to get done with that including identification of key vendors and so forth. But in the meantime, we have conducted some market research preliminary but we need to go out and do further market research to make sure that we fully understand the market, we fully understand the customers, we will be buying some additional data to validate specifically how the market looks, we will be looking into all the other things of course that go along with that positioning and messaging and branding and all of that but I also will be spending quite a bit of time and looking at market access and what will be needed to make sure that patients have access to it and physicians on a timely basis and all the other preparation it goes along with that, all the behind the scene operations and so forth. So that's a quick overview, feel free to I hope that answers your question. Raul anything you want to add to that?

No, I think that's a very good summary.

Great, thanks for taking my questions.

Sure.

Thank you. This concludes today's Q&A session. I would now like to turn the call back over to Raul Rodriguez for closing remarks.

Thank you for those questions. So on the whole; I think this has been a good quarter and a good year for Rigel. I think we have a product in fostamatinib, we very much believe that, the data from the FIT Phase 3 studies and the long-term extension study as well as the Phase 2 study we conducted earlier show that in total the product provides a consistent, clear, and sustained clinical benefit for some ITP patients and we will have discussions with the FDA soon and we do plan on filing the NDA in the coming months. So thank you very much for your interest in Rigel and we look to keep you informed on any updates.

Ladies and gentlemen, thank you for participating in today's conference. This does conclude the program and you may all disconnect. Everyone have a great day.