Rigel Pharmaceuticals, Inc. (RIGL) Q2 2017 Earnings Report, Transcript and Summary

Good afternoon, and welcome to Rigel Pharmaceutical's Financial Conference Call for the Second Quarter of 2017. 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 would like to turn this conference over to our first speaker, Dolly Vance, who is Rigel's Executive Vice President, Corporate Affairs and General Counsel. Ma’am you may begin.

Hello and welcome to our financial results and business update conference call. The financial press release for the first quarter of 2017 and a slide presentation for today’s call can be viewed in the Investor Relations section of our website, at www.rigel.com. Joining me on the call today from Rigel are Raul Rodriguez, our CEO; Ryan Maynard, our CFO; Anne-Marie Duliege, our Chief Medical Officer and Esteban Masuda, Ph.D., Senior Vice President of Research. 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 risks 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.

Thanks, Dolly, and welcome everyone. Thank you for your time this afternoon. As you know this quarter was one where we achieved a major milestone in our transition into a commercial stage biotech company. Now more than ever, we are closer to bring you a new therapeutic option to patients with chronic immune thrombocytopenia or ITP. During this call, we will recap our most recent achievements and share why we are very optimistic about the near future. In June we announced that the FDA had accepted the NDA we submitted for TAVALISSE or fostamatinib disodium in patients with chronic or persistent ITP. We expect the FDA to complete its review by April 17 of 2018 under the Prescription Drug User Fee Act also known as PDUFA. As you might imagine, it was quite rewarding to accomplish a significant milestone. It is a credit to our employees and their dedication and hard work. The NDA included data from our fifth Phase 3 clinical program including the two pivotal studies 047 and 048 as well as the continuing long term open-label extension study 049. Overall, patients who responded to fostamatinib had a timely robust and an enduring response to treatment. This is important because the rapid response provides an early feedback as to whether fostamatinib may be a viable treat for treating a patient with ITP. The robust response allows patients just hold steady platelet counts of about 100,000 per microliter of blood which will allow for proper clotting. Fostamatinib may also provide an enduring benefit with responding patients maintaining their platelet counts for a median duration of 16 months of treatment of fostamatinib, and this number of month is increasing. This data provides confidence that the disease can potentially be controlled in the long term. As we mentioned before, the fifth Phase 3 clinical program confirmed that fostamatinib has a consistent and predictable safety profile. This profile aligned with the safety database of over 5,000 patient years. Now, I'd like to ask my colleagues to give you an update on other programs we are working on at Rigel. Besides the ITP program and the NDA submission, I've asked Anne-Marie Duliege to give us an update on our Phase 2 clinical studies for fostamatinib in IgA Nephropathy as well as our Phase 2 study for fostamatinib for the treatment of autoimmune hemolytic anemia. This quarter we also selected a molecule from our Iraq program for the preclinical development. I have asked Esteban Masuda to join us to give us his perspective on the Iraq as a target and moving our candidate into the clinic. Now, I will turn the call over Anne-Marie and Esteban. Following their presentations, Ryan will report on second quarter financials. Anne-Marie?

Thank you, Raul. First, I want to echo Raul’s sentiment in thanking all my colleagues for their ongoing hard work and dedication. It was very rewarding to have the FDA accept our NDA submission and we look forward to working with the agency and hope to bring this drug, this new drug to a patient population with unmet medical need. As shown on Slides 9 and 10, Rigel focused on fostamatinib through chronic ITP because there was a significant unmet medical need. There are approximately 65,000 adult patients with chronic ITP in the US and it qualifies as an orphan disease. The chronic ITP patient population is very genius, meaning that it is challenging to predict which patient will respond to which of the current available treatment if any. [6.39]: On Slide 11, we have reminded you of the design of the Phase 3 program to similar placebo-controlled trials where patients are randomized in a 2 to 1 ratio to fostamatinib at the starting dose of 100 milligrams twice a day or placebo. During the trials, patients who did not meet the criteria of 50,000 platelets at week three were allowed to dose increase and those who did not meet that criteria by week 12 were allowed to roll over to the extension studies. On Slide 12, we have summarized the key baseline characteristics. [8.00]: Slide 13 provides the overall platelet response rate in the combined 047 and 048 study here we have outlined not just the stable patients which were probably 18% but also the intermediate patients approximately 11%. The definition of an intermediate platelet response was patient with at least 2 consecutive platelet counts greater than 50,000 of course in the absence of medication at any time during the control trial. This is important because when accessing which patient benefits from fostamatinib it is not just the stable responders, but also there's intermediate responders and I will show you why on the next slide. A total of 29% or approximately 30% of patients had some response to fostamatinib compared to 2% in the placebo. On Slide 14, we provide the million platelets counts over time for each of the sub groups. The stable responders in red have a timely response and robust platelet response that is maintained over time. Indeed with an increase in average above 50% at week two and if you please continue too immediate platelet counts of approximately 100,000 maintained until week 24. The patient with an intermediate response in Green have also quick benefit their increase has seen a two weeks on average and its benefit is maintained at approximately 50,000 platelets over time. So from 16,000 to 50,000 platelets, that’s a very good outcome for these patients. This is also clinical benefit for these patients. On the next Slide, Slide 15, we show here the captain mail – of time to first response, which means time to platelet counts above 50,000 through those subgroups. It shows that the stable responder and the intermediate responder have a response abruptly and in two weeks for the majority of them and that they get to a response by about 8 to 12 weeks by contrast the placebo in the non responders have a very low platelet count and never get to a median of response. On the next Slide, we show here the critical clinical benefit that this patient have from being treated with fostamatinib in both among the stable responders and the intermediate responders, none of this group had any – bleeding during the placebo control portion of the study. By contrast, patients who did not have response to fostamatinib or patients in the placebo group had at least the portion of them had experienced either a severe adverse event of bleeding. Similarly and that on Slide 17 patients who met the definition of stable response of intermediate response had a limited need for rescue medication and in fact that was essentially during the first week of treatment where they had their platelets increased over time. By contrast patients who didn't have response over placebo patient required more often and more frequently medication. On the next Slide, 18 we provide a summary and we want to remind you of the adverse events profile of fostamatinib, well defined based on our total experience in ITP but also having been informed previously by large database in the patient population. Adverse reactions are defined as occurring exactly 5% of the fostamatinib patients across both trials and at least twice as often as in the placebo group. The most frequent reactions are GI symptoms from diarrhea and nausea as well as hypertension and increases in transmitters. In summary, the response is consistent across the 2 Phase 3 trials with the stable response rate of 18% and the medium platelet counts around 8,500 to 100,000 over time. Additionally 11% of the patients have an intermediate response with 1 million platelet counts of approximately 50,000 over time for an overall response rate of nearly 30000 and this is nearly 30% of the patient. The response is similar across subgroups gender age product treatment or baseline things that are less or above 50,000. The million platelets response is rapid in those responded patients, approximately two weeks. There is a lower need for rescue therapy and no bleeding episodes of serious adverse events and longer responders. In fact the adverse events are mostly none to moderate in severity and finally the 049 study is ongoing and continues to providers with further evaluation of the long term safety and efficacy of fostamatinib in ITP. With the NDA now accepted by the FDA, Slide 20. Slide 20 shows our completed and expected regulatory milestones. To-date our communications with the FDA since the MDA was accepted, have been straightforward and would answer the questions as needed. We're also working with our clinical and manufacturing partners to make sure that we're ready for the FDA’s expected -- and pre approval inspections respectively. We're also preparing for an oncology drug advisory committee or ODAC meeting in the event we have one. Now I want to spend a bit of time providing an overview of our additional clinical studies of fostamatinib in other indication. Moving to Slide 21, Raul mentioned our study of fostamatinib in our --. It’s the most common – worldwide. This causes information and scarring of the kidney, people with the condition are at risk of serious complications from kidney dysfunction including high blood pressure and renal failure. We reported results from the first cohort in the Phase 2 study in January 2017. The primary efficacy endpoint was the main change in Herzliya from baseline at week 24 shown on Slide 22. The study shows that at 24 weeks fostamatinib was well tolerated with no new safety signal detected. The initial data suggests a possible trend towards a greater reduction in proteinuria in fostamatinib patients relative to placebo. On Slide 23, the second cohort shows in the Phase 2 study is regarding the efficacy 50 and fostamatinib as measured by changing renal function and --. The second cohort evaluate a higher dose of fostamatinib 150 mg twice a day compared to the first cohort which evaluated in the dose of 100 mg twice a day. I am happy to announce that the study has completed enrolment this week and we will report the results in early 2018. Finally, Raul also mentioned that our source study continues to enroll. As you will see on Slide 24, there is a large unmet treatment need among patients needing with warm autoimmune hemolytic anemia or AIHA. AIHA is indeed a real serious disorder where the immune system produces antibodies that result in the destruction of the bodies’ own. The sold study is Phase 2 study as explained on Slide 25, stage 1 of the Phase 2 study will evaluate the safety and efficacy of fostamatinib at 100 mg twice a day for 12 weeks in approximately 17 patients with hemolytic anemia was previously received at least one treatment for the disease but benefitted and are still anemic. Stage 1 is continuing enrolment and we expect results from this by the end of the year. Stage 2 will have an identical study design and begin enrolment after the stage 1 results are evaluated and reported. Continuing to our research update, I will now turn the call over to Esteban to discuss our IRAK programs. Esteban?

Thank you, Anne-Marie. Today, I’d like to introduce our IRAK inhibitor program and our clinical candidate R509. Slide 27, why IRAK? Well IRAK is a regulator of toll-like receptors and toll-like receptors are sensors of tissue damage are clinical in chronic inflammation. IRAK also regulates the receptors for the IL-1 family of cytokines which are themselves key mediators of inflammation. Thus IRAK signaling plays a key role in the detection, the induction and the mediation of inflammation. But our preclinical results validated IRAK1-4 therapeutic targets for inflammatory and autoimmune diseases including gout, psoriasis and lupus. In search for inhibitors of toll receptors signaling in Slide 28, we used cell based screens using primary human cells. In doing so, Rigel has discovered and developed, IRAK 1/4 inhibitors that completely operates toll receptors and IL-1 receptors signaling. Importantly, this complete inhibition has shown in the left and in plan, it’s associated with potent inhibition of both IRAK4 and IRAK1 kinases. In contrast, inhibitors of not only IRAK4 and not IRAK1 are seen in the blue panel exhibit partial inhibition of cytokine release. We believe this to be a key differentiation factor between Rigel IRAK1/4 inhibitors and other programs that inhibit IRAK4 collectively. [20.30]: Finally, we have selected our R509 as a prodrug both R835 improving insolubility, where we dose R509 orally to the deliver R835 into the systemic circulation. R509 in Slide 30 by virtue of inhibiting both cell receptors and IL1 receptors has several potential clinical uses. They can be mediated by cell receptors such as lupus and multiple sclerosis or mediated by IL1 receptor such as in gouty arthritis and pustular psoriasis. And we'll show you a couple of examples using animal model next. In Slide 31, we show R509 inhibiting a cell receptor [indiscernible] dependent model of joint inflammation in rates. In the panel below, we're seeing the blue line, those a dose dependent integration of the onset of disease when dosing of R509 starts with a zero. Remarkably, R509 also inhibits ongoing disease when those in a treatment mode are shown with a green line, where R509 dosing begins at Day 12 when disease is already established. Altogether, these results are consistent with IRAK1/4 playing a role in both initiating and sustaining chronic inflammation. In Slide32, we show R509 inhibiting an IL1 dependent model of gout, which is induced here by intra-articular administration of monosodium urate crystals in rat joints. In the left panel below, we can see R509, dose-dependent configuration of inflammation in the green bar relative to the vehicle in grey. The other color bars are positive controls. In the right panel, R509 also in the green bar dose-dependently decreases pain sensations relative to the vehicle in grey, as it is expected from block in IL1. In conclusion, in Slide 33 we are very excited to introduce R509 as a potent IRAK1/4 inhibitor with proven efficacy in multiple models of chronic inflations. Currently work is ongoing to enable the imitation of clinical studies with R509 in the first half of 2015. Thank you. Now, I will turn over the call to Ryan for a financial update.

Thank you, Esteban. For the second quarter of 2017, we reported a net loss of $19.1 million or $0.16 per basic and diluted share, compared to a net loss of $13.5 million or $0.15 per basic and diluted share in the same period of 2016. There were no contract revenues from collaborations in the second quarter of 2017. Contract revenues from collaborations of $8.6 million in the second quarter of 2016 were comprised of $4.8 million from the amortization of the $30 million upfront payment and $95,000 in FTE fees from our license agreement with Bristol-Myers Squibb, as well as payments of $3.7 million that we received from BerGenBio. We reported total costs and expenses of $19.3 million in the second quarter of 2017, compared to $22.2 million for the same period in 2016. The decrease in costs and expenses was primarily due to the decreases in personnel costs and research-related costs as a result of the reduction in force in September of last year, partially offset by the increase in costs related to the preparation for the potential commercial launch of fostamatinib in ITP. For the six months ended June 30, 2017, we reported a net loss of $34.5 million, or $0.29 per basic and diluted share, compared to a net loss of $31 million, or $0.34 per basic and diluted share, for the same period of 2016. As of June 30, 2017, we cash, cash equivalents and short-term investments of $82.3 million, compared to $74.8 million as of December 31, 2016. We expects that these amounts as of June 30 will be sufficient to support its current and projected funding requirements, including the preparation for the potential U.S. launch of fostamatinib through at least the next 12 months. We also continue to evaluate ex-U.S. partnerships for fostamatinib and other partnering opportunities across our pipeline. Back to you Raul.

Thanks very much Ryan. And before, I turn the call over to your questions, I'd like to provide to you a few other updates. With the goal of supporting our upcoming regulatory and commercial launch, first, we have made numerous hires and I'd like to just highlight a number of these. First, Dana Pizzuti, who was Vice President of Regulatory Affairs at Gilead Sciences since 2007 and facilitated the approval of 14 new medicines during her tenure there, joins Rigel as Senior Vice President of Regulatory Affairs and Clinical Quality Assurance. Dana brings tremendous experience across the broad range of regulatory clinical, NDA and commercial stage functions. At Gilead, she managed an organization of 500 worldwide and was responsible for regulatory strategies for all of Gilead's approved and investigation of new drugs. Also joining us is Giovanna Matthews. Most recently, she was CEO of LifeWatchCare/ ValidCare. She joins Rigel as Executive Director Market Access. She has great experience across all areas of market access and reimbursement including managed care, specialty pharmacy, distribution, contracting and patient support services. And soon, Sandra Tong will join as Vice President, Clinical Science and Drug Safety. Most recently she was Vice President of Clinical Research at Plexxikon. She has over 20 years of clinical development and medical experience in small molecule immunology oncology and anemia across all stages development. Finally, Scott Henley has joined Rigel as Vice President Clinical operations. Previously Scott was Vice President, Clinical Operations at Titan Pharmaceuticals and has over 17 years of experience in this area. These additions are highly qualified and enthusiastic people and we are excited to have them join Rigel at this time. We look forward to their contributions. They obviously are very talented, had many choices in terms of places they could go and they chose Rigel. I think it speaks for the opportunity here. With our NDA submission for fostamatinib accepted and our other studies under way with fostamatinib in other rare diseases and progress made with our IRAK program molecule, I think we've had a very productive quarter. So, with that in mind, I'd like to outline for you what will happen at Rigel over the next months. Most importantly, we will work collaboratively with the FDA to review our NDA submission. Secondly, we will continue to build our capabilities to allow us to achieve commercial readiness here in the U.S. We will continue to be with patients, patient advocates and physicians who will help us understand the burden of ITP and the need for new treatment options. As an aside, this past week and a week – a team of us from Rigel attended the Platelet Disorder Support Association annual meeting in Phoenix. This is a very well organized patient advocacy organization advocating for patient suffering from ITP. We have a very good relationship with them and this conference allowed us to hear the patient side of ITP and the frustration, the unpredictability and the substantial burden of this disease and how it affects the quality of life for these patients. It was very clear to us that there is a tremendous medical need in this area. It's a really inspiring conference where you hear that side of the story. So, we came back very energized to finish our work with the NDA and move this product into the marketplace. And finally, we continue to work with other partners to continue develop molecules and move them forward in their pipelines and obviously as partnership opportunities. So, at this time, I'd like to open up your calls – the call up for your questions?

[Operator Instructions] Our first question comes from Eun Yang of Jefferies. Your line is open.

This is Carmen [ph] on for Eun. Thanks for taking the question. So, regarding the ex-U.S. partnership for fostamatinib, what would you need to secure a deal and by when would you hope to have a partnership in place?

Thanks for the question. So, we're having discussions with numerous companies across different geographies, some more European focus, some more Asia focused. I think what we need is progress in terms of the NDA in the U.S. I think that might be a pre-requisite simply because that will provide the final validation that some of these parties would need to find a deal. As you can imagine, it probably is difficult for some of these parties to sign a deal ex-U.S. for a substantial amount of money which is what we would like. And still not have the final approval in the U.S. So, probably that's a requirement in terms of that. In Asia, particularly in Europe, in Asia, the opportunity is a little bit different with more of an emphasis of IgA Nephropathy, so I think there in addition they probably would need some results from our IgA Nephropathy Phase II trial which we'll have early next year. So, those are probably the two major gating issues for deals in those respective territories.

Got it. Great. Thanks. And then the AIHA indication, how many patients do you expect have data for in the update around year-end? Could that be presented at ASH [ph]? And finally, do you expect that the number of patients presented at the end of the year would be sufficient to engage the FDA regarding potential requirements for registration trial or if not, when do you expect you would engage the FDA?

Thanks for that question. So we will have 16 or so patients, 17 I think is the target by the end of the year; so we expect to have that number. And hopefully that will give us sufficient data to be able to decide to move forward with autoimmune hemolytic anemia. So it will be at the end of the year, it's not going to be for ASH. I think the ASH abstract does line with this week and so we're not going to present it at ASH but we will hope to have that data then. I think we usually put a press release out first week of January in terms of a pipeline update, so it probably would be part of that update that we give that. And then in terms of FDA engagement, that would follow after that result and our review of the data. So sometimes, probably around mid-year, Anne-Marie?

Yes.

Okay, great. Thanks for taking the questions.

Thank you. Our next question comes from Anupam Rama of JPMorgan. Your line is open.

Hey guys, it's Eric in for Anupam this evening, thanks for taking the questions. I guess coming out of VA channel for the conference that you described earlier, I'm just wondering in terms of some of the pre-commercial activities you're doing, if you could give us the sense of where you think leases is in terms of awareness among hematologists currently, as well as how kind of concentrated the DPH reading physician audience is in the U.S.?

Thanks for that question. We did have a presentation at the European Hematology Association meeting where we reviewed some of the data that you've seen today here now, now there are some new data that Anne-Marie presented to you. The PDSA meeting is more of a bit of a different conference, it's really oriented more patients as well; so it's a bit of a different one than that, it's not just straightforward medical conference like AISH or AIHA. In terms of awareness, I think the awareness is still very low regards [indiscernible], we haven't really begun to market the product and obviously won't until sometime next year. So at this point we're communicated on a medical level at these conferences and we hope to have some presentations at the AISH meeting at the end of the calendar year to be able to share more with you, obviously that isn't -- we've submitted but obviously don't know for certain that those have been accepted as yet. So we're working towards that. The physician audience that we're targeting is heam, hemox [ph] and we're working on helping identify who would the actual prescribers are and that's a step for our commercial team. And they are working hard on that, the folks that I outlined in terms of joining the company have that as one of their missions and other people that I see here are working on identifying those physicians and really working on the positioning of the product, obviously not yet but in the near future on the pricing of the product and we'll be able to communicate that to you. In fact at a future call, financial call such as this I'm going to invite our head of commercial operations to come and give you an update on some of those key activities. So please join us at the subsequent call for that update.

Thanks, that's very helpful. Maybe just another question here on hemolytic anemia; it would be at year-end, I'm just wondering if you could kind of got us throughout this and thinking about the threshold for activity in terms of hemoglobin response rate or duration of response that you kind of need to see here to get counsel [ph] with advanced net program for us. Thanks.

So it's actually part of the protocol. We want to see an improvement in hemoglobin as we are correcting anemia and want to see at least 10 gram at the minimum and 2 gram above baseline. So this is a criteria that M1 would say there is a difference -- maybe a real difference in hemoglobin. Also what we would like to see is clearly a sustained response, at least for some portion of the 12 weeks duration of the study.

Got it. Thanks for taking the question guys.

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

Hi, how is it going? It's Alex on for Do. I had a first question going back to the slides and I was wondering on slide -- sorry, I'm just going back right now; on Slide 14 in the presentation. I was wondering following the intermediate patients why overtime they seem to decline?

Yes, very good question. Easy, if we have put the end less than two visit to the study. You would see that sign from weeks 18, there is a much more number of patients because some of the patients decide to go directly into an open label part of the trial and so this is because the end are about three or four and in section that we see that.

Okay.

It's really a factor that we've put a little note in there.

Got you.

It's not that they are actually dropping.

It's just a few patients that participated in that and some patients have gone to a new enabled extension.

Got you. Thank you. And then also within ITP and in light of this IPO we were wondering sort of bigger picture what's management's impression of Dova [ph] Pharmaceuticals ITP drug was? And if at all likely effect probably the commercial strategy in anyway?

I don't -- you know, I'm not in a position to really comment on their drug but at some point later I can. I don't think it's a serious difference from what's already available. So I don't think that that will have a significant bearing on our commercial potential.

And going over to AIHA, we were wondering with the enrollment has management identified any bottlenecks to recruiting patients, we know that the 17 patient enrollment has lasted maybe just say a little longer than expected and if that's the case, should we expect a more expedited enrollment for the phase -- for the park two portion?

You know, I think it is a rare disease and I think that's one thing we -- given that we've worked in ITP, it was hard to recruit the ITP patients, we succeeded. GA nephropathy [ph] was also -- is also a rare disease and it took us a while today Anne-Marie and that was able to announce we succeeded there as well, and the same thing for hemolytic anemia; it's a rare disease, if the patients are not well identified, many of them have the disease because there is nothing for them, they frankly live in the shadows. And as a result it's taken a while to have the trial enrolled but I think we've made very good progress and would be able to give you an update on those results at the end of the year. So it's just the nature of rare diseases, all of these actually were a struggle and we've worked hard, we overcame those struggles and succeeded in completing enrollment.

Alright. Thank you very much for taking the questions.

Thank you. I would now like to turn the conference back over to Raul for any closing remarks.

Thank you and thank you for your questions. The second quarter has been a very good quarter for Rigel, we've made very good progress with our NDA, that was fantastic, that is the most important objective for the company as to drive that NDA towards an approval early next year. And I think this year was -- this quarter was a very good quarter and we continue to work very hard and the many activities related to that. We also are preparing for commercial launch in the U.S., so great deal of activity related to that market research, recruiting people in key areas, and we'll give you an update on those activities at the next call. And then finally, with our IRAK program, I'm delighted to have that molecule selected and the work ongoing to make that molecule -- take that molecule into the clinic next. It's an opportunity that's tremendous and Esteban laid out it's potential in a wide number of potential areas and that's very exciting to have something with that level of potential and significantly differentiated versus some of the competitors in the IRAK space; I think that's wonderful. So I think it was a very good quarter for the company. I thank you for your continued support and look forward to interacting with you in the future.

Thank you. Ladies and gentlemen, this concludes today's conference. Thank you for your participation and have a wonderful day. You may all disconnect.