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Rigel Pharmaceuticals, Inc. (RIGL)

Q3 2017 Earnings Call· Tue, Nov 7, 2017

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Transcript

Operator

Operator

Good afternoon, and welcome to Rigel Pharmaceutical's Financial Conference Call for the Third 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 purposes at Rigel's Web site [Operator Instructions]. And now, I would like to turn this conference call over to our first speaker, Dolly Vance, who is Rigel's Executive Vice President, Corporate Affairs and General Counsel.

Dolly Vance

Analyst

Welcome to our financial results and business update conference call. The financial press release for the third quarter of 2017 was issued a short while ago and can be viewed in the News & Events section of our Investor Relations page of our website, 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 Eldon Mayer, our Chief Commercial Officer. 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.

Raul Rodriguez

Analyst

Thank you, Dolly. And welcome everyone. Thank you for your time this afternoon. This past quarter has been an exciting one as we worked to potentially bring our new therapeutic option to adult patients with chronic immune thrombocytopenia or ITP. During this call, we will recap our most recent achievements including our interactions with the FDA, discuss highlights of clinical data, discuss our commercial efforts to-date as well as give you an update on our financial position. Collectively these activities demonstrate why we are so positive about what you expect from Rigel in the next six to 12 months. On slide 8, a few years ago we made a substantial change to Rigel’s strategy to transition to a commercial stage company. This strategy was based on fostamatinib as an excellent foundation for this pivot. We conducted market research, had discussions with KOLs and ITP to establish the medical need, had discussions with the FDA on approval path and then planned our commercial efforts. We are now delivering on that strategy. We conducted a Phase 3 program in ITP and announced results late last year. We filed the US NDA for fostamatinib in ITP in April and it was accepted in June. We are now managing that NDA towards an approval and Anne-Marie will give us an update on this effort. We are also planning on commercializing fostamatinib in the U.S., Eldon will provide some background and an update on those activities. Our therapeutic areas of focus are immunology, hematology, oncology disorders and rare diseases. Immunology has a foundational expertise at Rigel. We use our understanding of the immune system and apply it to various therapeutic areas. And finally, we move various program via partnerships, an example fostamatinib in North American territories where we will put partnerships in place. On slide…

Anne-Marie Duliege

Analyst

Thank you, Raul. As always I want to thank my colleagues for their ongoing hard work and dedication in support of our NDA submission. It has been very gratifying that we have been able to responsive the FDA’s questions. We will continue to work closely with the agency and remain hopeful that providing FDA approval we'll be able to bring this few drugs to a patient population with unmet treatment needs. As we've noted before, the NDA included data from our fifth preclinical program, two pivotal studies 047 and 048 as well as a continuing long term open label extension study 049. Overall, patients who responded fostamatinib had a timely robust and sustained response to treatments. The fifty preclinical programs confirm that fostamatinib has a consistent and predictable safety profile alignment with a large safety database. As shown on Slide 12, Rigel focused on fostamatinib through chronic ITP because there was a significant unmet medical need. There are approximately 65,000 patients with chronic ITP in the U.S. and it qualified as an orphan disease. ITP is a rare autoimmune condition. ITP arises from various disease mechanisms of platelet destruction and in some cases insufficient platelet production. The pathophysiology of ITP is complex and many factors that contribute to the disease may differ from patient to patient making this a clinically heterogeneous disease. This disease is characterized by an increase risk of bleeding often with -- bleeding. However at times at results such as GI or brain hemorrhage can be more senior and possibly cells. Treatment options exist but they’re limited after with side effects and the response can vein over time. There is no treatment that specifically prevents platelet destruction. Sick time is the key figure in the immune system destruction of ITP. That’s why fostamatinib and stick inhibitor with…

Eldon Mayer

Analyst

Thank you, Anne-Marie. Today I'd like to review several aspects of our launch preparation and in particular some details on how we view the adult chronic ITP market. As Raul mentioned this is part of our company's transition from pure research and development to bringing at first product to market. In our many initiatives thus far our commercial team has spent a significant amount of time making sure we have a good understanding of the ITP market, consider our approach to commercialization is correct I'd like to share a few of those insights with you. In addition I'd like to describe the commercial team we've been building to support the launch as well as some key activities we've been working on to prepare to enter the market. What I'll cover at a future time are some more specific topics such as pricing and market access. First I'd like to review the ITP patient landscape according to our analysis. On this Slide you can see there're approximately 65,000 adult patients with chronic ITP in the US which of course qualifies it as an orphan disease, and in a given year approximately 50% of these patients or 32,500 are estimated to be actively treated but it's important to keep in mind that if these patients are followed for a longer period of time the proportion of them that are treated would increase significantly. For the remaining half of the 65,000 patients we estimate that 23% of those or 15,100 are treated with steroids during the year, the remaining 27% of those patients or 17,400 are steroid refractory and receive any of the current available treatment such as rituximab, [indiscernible] agents, splenectomy etc, and this represents the addressable market for fostamatinib. As part of our market analysis we analyze the claims records of…

Ryan Maynard

Analyst

Thank you, Eldon. The successful financing that we completed last month has put us in a good financial position. The 65.3 million that we actually received from the stock offering basically doubled our cash position and allowed us to extend our run rate comfortably into 2019 including the potential launch cost for fostamatinib. Any upfront amounts received from successfully partnering outrights to fostamatinib in Europe or Asia would add to this run rate. It’s important to note that during this calendar year we have already invested over 8 million in launch preparation cost. This amount is expected to ramp in ‘18 as we look to a potential commercial in the second quarter of 2018. Now, on some of our financial results from Q3. We reported revenues from collaboration of 900,000 in the third quarter of 2017 which related to a payment received from a license agreement with a third-party. Revenues from collaborations of 3.8 million in the third quarter of 2016 represented the remaining amortization of the upfront received from Bristol-Myers Squibb. We reported to costs and expenses of 18.8 million in the third quarter of 2017 compared to 26.5 million for the same period in 2016, this decrease in cost and expenses was the result of the reduction in workforce undertaken at September of '16 and the completion of the pivotal Phase 3 clinical trials on ITP. Now this decrease was partially offset by the increase in costs related to the preparation for the potential launch of fostamatinib in ITP that I just mentioned earlier. As a final note I'd like to point out that the Rigel finance organization has been very focused on building the appropriate system and infrastructure including people to support the potential launch of fostamatinib. The team is very excited to support this phase in our transformation. Back to you Raul.

Raul Rodriguez

Analyst

Thank you very much Ryan. Needless to say this progress is very exciting at Rigel, our team has accomplished significant milestones this year in executing on our strategy and this being the final call, financial call of the year let me recap some of those. We filed our first NDA as a company for fostamatinib in ITP. We managed the regulatory interactions with the FDA successfully. We demonstrated a broader clinical utility to fostamatinib in hemolytic anemia and we made substantial progress in preparing for the product launch next year, and thanks to you we have the capital to accomplish this. In all it's been a excellent year for Rigel. We are confident that we will continue to make substantial progress on our strategy in the coming year and it should be even more monumental. So with that I'll turn it over to, call over to questions.

Operator

Operator

Thank you [Operator Instructions]. Our first question comes from Eun Yang from Jefferies.

Eun Yang

Analyst

Question on fostmatinib in autoimmune hemolytic anemia. So you are moving into say into extension court, are you expecting to start the Phase 2 trial with additional 20 patients by end of this year.

Raul Rodriguez

Analyst

Thank you, Eun. There maybe one or two patients that may come off before the end of the year depending on what's happening at the end of the existing cycles that occurs interim role in the trial, but more importantly in order to involve the majority of patients in cohort two we will open new sites and that will start next year.

Eun Yang

Analyst

I see, do you think you need this additional 20 patients from stage two in order to maybe the FDA to talk about to discuss [indiscernible].

Raul Rodriguez

Analyst

No, we intend to go and talk to the FDA once we have completed stage one and looked at all the data in detail.

Eun Yang

Analyst

Okay, that would be first quarter next year?

Raul Rodriguez

Analyst

First half of next year but possibly [indiscernible] first half of next year.

Eun Yang

Analyst

And then also you submitted a [indiscernible] or abstract for ASH on this data, so if it's not accepted then would you provide us with the data by year end?

Raul Rodriguez

Analyst

I think I’ll give this as well. I think our plan would be to publish that at another presentation perhaps the EHA meeting in Europe or another venue where we would have a broad audience like that.

Eun Yang

Analyst

But you will find that setted by November 22nd, correct?

Raul Rodriguez

Analyst

That’s correct. And we do have an oral presentation on the ITP data at ASH. So we will inform and the rest of our -- the community in terms of if we do have leg breaker or not.

Eun Yang

Analyst

Okay. The last question is on the partnership for first line ITP for the US. I think on the last call you mentioned that perhaps the progress on the NDA in the US may be important particularly a deal for a substantial amount of money. So should we expect any kind of partnership that still to be expected for the PDUFA date?

Raul Rodriguez

Analyst

I think the U.S. approval would be an important milestone just in terms of validation and in de-risking for an ex-US partnership, partnerships, plural. And so I think that’s an important step. We would like and we are engaging with companies already ahead of that so that they are well prepared to go into deeper discussions after that PDUFA date. We are hopefully closing something not that part to the future beyond that. But it’s an important step as you can imagine.

Operator

Operator

Your next question is from Anupam Rama with JP Morgan. Your line is open.

Unidentified Analyst

Analyst

Thanks for taking our question. This is [Casa] filling in for Anupam this evening. Given that fostamatinib represents a novel mechanism of action for the treatment of ITP in a sense addressing a disease from the opposite side to TPOs but how should we be thinking about the potential for a combination use? Do you have any plans to study fosta in combination use?

Raul Rodriguez

Analyst

Our most important objective was to establish the role of fostamatinib in itself as a potential treatment for patient. Down the road we extend that physicians might be interested in looking at this combination even on a case-by-case basis for some of their patients, or potentially as part of an investing responsive trials. We have not defined that yet but we are not excluding that for the future. It will make sense from a mechanistic perspective but it was important to us to understand, establish and clear the role of fostamatinib on its own prior to being potentially as part of the combination.

Operator

Operator

Our next question comes from Do Kim from BMO Capital. Your line is open.

Alex Arfaei

Analyst

Hi, thanks for taking my question. This is Alex on for Do. Wanted to know a little bit more color around label negotiation with the FDA and what you think the chances are around label for fostamatinib?

Raul Rodriguez

Analyst

Yes, so we are going to initiate our discussion with the FDA shortly on label negotiation. We are asking for broad label which is justified by the circle with approval we’re defined where patients with Phase 1 treatment without specifying which treatment or the number of previously treatment before and this is what we have requested. So we will see. At this point, the FDA has not given an indication otherwise, but it will be too early to say that.

Alex Arfaei

Analyst

And with the new clinically relevant platelet response most of the physicians they talk to, how do they view this end point with respect to other endpoints that we've seen, stable response and intermittent response.

Raul Rodriguez

Analyst

They certainly value it as more clinically relevant endpoint, as I indicated during the presentation the stable response rate is the one defined by the FDA as high bar and similar to what has been used to approve [indiscernible]. But based on the guidelines and based most importantly on the clinical experience of the physicians we do not need to see consistently platelet counts of 50,000 rather an increase in platelet count to 30,000 and above and most importantly a reduction in bleeding episodes is what matters for physicians and of course for the patients as well.

Alex Arfaei

Analyst

Thanks and just one more question to how variable is the time to response from patient to patient that you see in ITP and also the similar time to response that you see in AIHA patients.

Raul Rodriguez

Analyst

As indicated the response is pretty rated, 60% of the responders will respond within two weeks. Approximately 80% of the responders within eight weeks, and the totality of the responders by week 12. And we seem to see approximately the same rate in autoimmune hemolytic anemia although two additional responders responded after 12 weeks.

Alex Arfaei

Analyst

Thanks for taking my questions and congrats on the progress.

Operator

Operator

Our next question is from Joe Pantginis from H.C. Wainwright, your line is open.

Joe Pantginis

Analyst

I following up obviously you said you're about to begin your label negotiations besides that can you maybe give a little punch list about what might be outstanding, for example are there any facility inspections that still need to occur.

Raul Rodriguez

Analyst

So just to be clear we don't know when the label, we don't have a clear sense of the label negotiations that is our next upcoming step that is obviously scheduled prior to our [indiscernible].

Joe Pantginis

Analyst

And any additional things that might be required like facilities?

Raul Rodriguez

Analyst

Well, it's a very good question we have not had any facility inspections at yet, that might be required or sometimes I'm told they're not required so it's a bit variable in that sense.

Joe Pantginis

Analyst

And then just wanted to go back to AIHA for a second, do you have any more color with regards to the timelines of enrolling the second stage.

Raul Rodriguez

Analyst

Not really, we're working on this we're waiting to open new sites and actually new countries and we will have a better idea of what to anticipate by early next year.

Joe Pantginis

Analyst

Great, thanks a lot.

Raul Rodriguez

Analyst

Thank you, Joe.

Operator

Operator

I now would like to turn the call back over to Mr. Raul Rodriguez for closing remarks.

Raul Rodriguez

Analyst

Well thank you for your time, we appreciate your questions and your interest, it's been a very exciting year at Rigel. We will continue to give you regular updates on the regulatory and commercial milestones and on our pipeline. And as always thank you for your support we greatly appreciate it. Good afternoon.

Operator

Operator

Ladies and gentlemen thank you for participating in today's conference, this does conclude the program, you may all disconnect.