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Edited Transcript of RIGL earnings conference call or presentation 7-Nov-17 10:00pm GMT

Q3 2017 Rigel Pharmaceuticals Inc Earnings Call

South San Francisco Nov 10, 2017 (Thomson StreetEvents) -- Edited Transcript of Rigel Pharmaceuticals Inc earnings conference call or presentation Tuesday, November 7, 2017 at 10:00:00pm GMT

TEXT version of Transcript

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Corporate Participants

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* Anne-Marie S. Duliege

Rigel Pharmaceuticals, Inc. - Chief Medical Officer and EVP

* Dolly A. Vance

Rigel Pharmaceuticals, Inc. - Executive VP of Corporate Affairs, General Counsel & Corporate Secretary

* Eldon C. Mayer

Rigel Pharmaceuticals, Inc. - Chief Commercial Officer and EVP

* Raul R. Rodriguez

Rigel Pharmaceuticals, Inc. - CEO, President & Director

* Ryan D. Maynard

Rigel Pharmaceuticals, Inc. - CFO and EVP

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Conference Call Participants

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* Alexander Xenakis

BMO Capital Markets Equity Research - Associate

* Joseph Pantginis

H.C. Wainwright & Co, LLC, Research Division - MD & Senior Healthcare Analyst

* Kyung Yang

Jefferies LLC, Research Division - MD and Senior Equity Research Analyst

* Tessa Thomas Romero

JP Morgan Chase & Co, Research Division - Analyst

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Presentation

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Operator [1]

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Good afternoon, and welcome to Rigel Pharmaceuticals Financial Conference Call for the Third Quarter of 2017. (Operator Instructions) I would like to remind you that this call is being recorded for replay purposes at Rigel's website. (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.

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Dolly A. Vance, Rigel Pharmaceuticals, Inc. - Executive VP of Corporate Affairs, General Counsel & Corporate Secretary [2]

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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 Investors Relations page on 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.

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Raul R. Rodriguez, Rigel Pharmaceuticals, Inc. - CEO, President & Director [3]

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Thank you, Dolly, and welcome, everyone. Thank you for your time this afternoon. This past quarter has been an exciting one as we work to potentially bring a 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 to expect from Rigel in the next 6 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 in ITP to establish the medical need, had discussions with the FDA on an approval path and then planned our commercial efforts.

We are now delivering on that strategy. We conducted a Phase III program in ITP and announced results late last year. We filed the U.S. 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're 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 is a foundational expertise at Rigel. We use our understanding of the immune system and apply it to various therapeutic areas.

And finally, we moved various programs forward via partnerships, an example, fostamatinib in North American territories where we would put partnerships in place.

On Slide 9, here's why we're so excited about the fostamatinib opportunity. The indications we're pursuing are attractive orphan indications with sufficient patient populations to make an impact, but still addressable by a focused company. Each of these indications have significant unmet medical needs and are treated by products with limitations. In the case of IgA nephropathy or autoimmune hemolytic anemia, nothing has been approved.

In ITP, only the TPO agents are approved, and they have limitations according to KOLs and to patients with ITP. So why might fostamatinib contribute to these areas? First, it has a unique mechanism of action that may address the underlying autoimmune basis of these autoimmune diseases. This is different than any of the currently available agents. Second, we have shown an important patient benefit in ITP. In our Phase III studies, our trial showed a timely, a robust and an enduring benefit in platelet responses.

Today, we will introduce you to an additional measure of platelet -- of clinical benefit, that is a clinically relevant platelet response.

This platelet -- clinically relevant platelet response was 43% in our studies. Anne-Marie will discuss this in a few minutes.

In autoimmune hemolytic anemia, we have shown a 35% response rate in hemoglobin improvement in our Phase II study. In IgA nephropathy, we have shown a trend towards improvement in proteinuria and an acceptable safety profile in the lower-dose cohort of this trial. We hope to show even better results with the higher dose of fostamatinib in the second cohort, which fully enrolled last quarter.

Finally, we're excited about fostamatinib's large safety database. We have a 5,000 patient year safety database in several autoimmune diseases, with the observed adverse events being similar across these. This has allowed us to propose a paradigm for addressing each of the most frequent adverse events while still allowing patients to continue to benefit from fostamatinib. The safety experience also helps tremendously in our regulatory discussions.

On Slide 10, a quick review of our 4 key accomplishments for this quarter. In September, we had a teleconference with the FDA for our mid-cycle communications meeting, which is a standard meeting built into the FDA's review process. At that time, the FDA confirmed that they were not planning to schedule an Oncology Drug Advisory Committee, or ODAC, meeting to publicly review our NDA submission. They also confirmed that they expect to complete their review by April 17, 2018, which is the date supplied by the FDA under the Prescription Drug User Fee Act, also known as PDUFA. Anne-Marie will give us further details on these interactions as well as our regulatory plans in Europe. In October, we announced top line results from our Stage 1 of our Phase II study of fostamatinib for the treatment of patients with warm antibody autoimmune hemolytic anemia, or AIHA. Stage 1 of the study, which included 17 patients, showed a response rate of 35% and met the prespecified primary efficacy endpoint. In light of these results, we'll begin enrolling Stage 2 of the study, which will have an identical study design and include 20 additional patients. We will also pursue discussions with the regulatory -- on the regulatory approval of path for the product in autoimmune hemolytic anemia. We also completed a very successful fundraiser in October, which sets us up very well for executing on our plans. Ryan will give us further information on this.

And finally, our launch preparations. We have made substantial progress in understanding the ITP opportunity as well as hiring the commercial organization to commercialize fostamatinib. Eldon will provide some insights into this in his presentation.

So with that, let's dive right in. Anne-Marie?

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Anne-Marie S. Duliege, Rigel Pharmaceuticals, Inc. - Chief Medical Officer and EVP [4]

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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 respond to 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 new drug to patient population with unmet treatment need. As we've noted before, the NDA included data from our FIT Phase III clinical program, 2 pivotal studies, 047 and 048, as well as a continuing long-term open-label extension study 049. Overall, patients who responded to fostamatinib had a timely, robust and sustained response to treatment. The FIT Phase III clinical program confirmed that fostamatinib has a consistent and predictable safety profile aligned with a large safety database.

As shown on Slide 12, Rigel focus on fostamatinib for chronic ITP because there is significant unmet medical need. There are approximately 65,000 patients with chronic ITP in the U.S. and is 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 path of physiology 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 increased risk of bleeding, often with minor form of bleedings. However, at times, episodes such as GI or brain hemorrhage can be more severe and possibly lethal. Treatment options exist, but they are limited, often associated with side effects and the response can wane over time. There is no treatment that specifically prevent platelet destruction. SYK kinase is a key player in the immune system destruction of platelets in ITP that's why fostamatinib as a SYK inhibitor with a unique mechanism of action is of particular interest.

Moving to Slide 13, you can see that there were 2 similar Phase III trials followed by open-label extension. Dose increase was allowed at week 4, and patients with no response has the option to switch to the long-term extension study as of week 12.

Slide 14 provides the main baseline characteristics of patients in the Phase III program. This is a patient population typical of ITP, middle-age female patients, but these were patients with severe form of ITP. As shown by median duration of ITP prior to study entry of 8.5 years, but some patients had decades of ITP disease prior to entry, multiple prior ITP treatments and finally, very low platelet counts as baseline with a median of 16,000.

I now want to review with you how we're evaluating efficacy in this disease as shown on Slide 15. First, the stable response rate is defined in the protocol, which is at least 4 out of 6 biweekly platelet counts greater than 50,000 per microliter, in absence of rescue medication, during weeks 14 to 24. This was agreed upon with the FDA and corresponds to a high bar into the platelet response.

In further analysis, we looked at the ASH guidelines, which highlight the importance of maintaining platelet counts greater than 30,000. Hence, in collaboration with our advisers, key opinion leaders, we investigated a more overall clinically-relevant response. We looked at patients with platelet count during the first 12 weeks of treatment of greater than 50,000 per microliter in the absence of rescue medication. And the duration of response is defined by not dropping below 30,000 at 2 consecutive visits. We call this clinically-relevant platelet response. The response was consistent between the 2 randomized control studies and the open-label extension study.

On Slide 16, on the left panel is the primary efficacy endpoint from studies 047 and 048. The treatment effect is a stable response of 18%. On the right panel, we now see that the response rates of patients who crossed over from placebo to fostamatinib in study 049 is 23%, similar to that observed in studies 047 and 048.

Slide 17 reminds us that responses on fostamatinib are durable. 82% of the stable responders maintained response for at least 1 year, and response durations of more than 2 years have now been observed. The key point is that the platelet response is long-lasting. In fact, the median duration of response has now been reached and as of now exceeds 28 months.

Looking at the serious adverse events of bleeding, on Slide 18, in the 2 pivotal trials, we've seen how the response that this response is clinically relevant. Indeed, there is no such serious adverse events of bleeding among the stable responders during the controlled portion of the study as compared to the nonresponders and the placebo patients.

Now we move on to Slide 19. The most recent post-hoc analysis that we have completed for the ITP trial. I mentioned earlier, this analysis looks at the overall clinical benefit and the potential clinical utility of fostamatinib in ITP, which may have direct bearing on the commercial potential.

I will remind you that using our defined clinically-relevant platelet response, as stated on Slide 15 and, again, here on Slide 19, we found that 43% of the fostamatinib treated subjects and 14% of placebo-treated subjects met this criteria. The median duration of response for fostamatinib patients was greater than 28 months using the April 2017 data cutoff submitted to the FDA for the 120-day safety update.

Slide 20 shows the median platelet count over time for patients who had fostamatinib clinically-relevant response in green versus the fostamatinib nonresponders in blue and the placebo patients in orange. The median platelet count for the 43 subjects with the platelet response shown in green was timely, robust and enduring compared to the nonresponders and the placebo patients.

On Slide 21, you can see that 60% of these 43 responders do so within 2 weeks and 80% within 8 weeks. This is important because doctors treated with fostamatinib patients will know quite rapidly whether the patients find benefits from fostamatinib or not and certainly by week 12.

As a reminder, on the next slide, I just would like to do a quick review of the adverse events for the pivotal trials. You see that most of the adverse events were mild or moderate. And there were more frequent serious adverse events in the placebo group compared to the fostamatinib treatment. This is because the bleeding events were less frequent in the fostamatinib arm. The most frequent adverse events observed were diarrhea, hypertension and hepatic enzyme elevations.

So in conclusion of our summary of the fostamatinib program in ITP, the totality of the clinical data across ITP and the larger rheumatoid arthritis database support a favorable benefit-risk profile for patients exposed to fostamatinib. In responder ITP patients, treatment with fostamatinib results in higher platelet counts, pure bleeding events and manageable safety implications.

For non-responding patients, any safety events are easily manageable in routine clinical practice and their risk is limited to duration of up to 12 weeks. If approved by the FDA, fostamatinib may be an important new treatment option by blocking platelets destruction and generating a clinically relevant response in patients with chronic or persistent ITP.

Slide 24 shows a quick overview of the U.S. regulatory interactions to date. The orphan designation was granted in August 2015. We submitted an NDA earlier this year, and this NDA was accepted for review by the FDA. We have also provided the 120-day safety update. The mid-cycle communication -- during this mid-cycle communication, the FDA confirmed that no advisory committee meeting was planned. And overall, there was no showstopper in our interaction and in our responses to questions from the FDA.

And finally, we expect the PDUFA date by -- of April 2018, which remains unchanged. In addition, we had BIMO inspection at Rigel and at 2 clinical sites, which went well.

In regards to filings with European authorities, on Slide 25, we believe that we have data that also merits submission of Marketing Authorization Application, or MAA, to the EMEA. We have requested National Scientific Advice meeting in Spain, the U.K. and the Netherlands, and a meeting date has been set up with the U.K. This concludes my ITP review.

And I will now move on to discuss our autoimmune hemolytic anemia program. Autoimmune hemolytic anemia, or AIHA, is due to the presence of warm antibodies that react with protein antigens on the surface of red blood cells at body temperature. The destruction of red blood cells leads to oxygen to reduce oxygen delivery to the tissues, resulting in fatigue and dyspnea when exercising.

In some patients, the symptoms can be more severe with dyspnea at rest and varying degrees of fatigue. In rare cases of even more severe anemia, the clinical syndrome may become life-threatening. It is an ultra-rare disease with approximately 40,000 adult patients in the U.S. Similarities of treatment used with ITP exist with steroid, rituximab, other immunosuppressive agents and splenectomy being available. But in fact, there are no medical treatments that are specifically approved for autoimmune hemolytic anemia.

So there are several reasons to study fostamatinib in autoimmune hemolytic anemia. One, the unique mechanism of action, which may addresses the cause of autoimmune hemolytic anemia. The clear treatment effect in ITP is an indicator of the value of SYK inhibition to treat autoimmune cytolytic disease, such as hemolytic anemia. There is also an objective endpoint, the change in hemoglobin. And finally, we benefit again from this large safety database of more than 5,000-patient here, primarily in RA and in ITP.

Slide 21 -- 27 summarizes the design and the results so far. The design of the study is an -- of this Phase II study is an open-label, Simon 2-stage study. The primary endpoint is the response rate in the first 12 weeks without the need for rescue therapy. And the response is defined as a hemoglobin of at least 10 grams per deciliter and at least 2 grams per deciliter increase from baseline. On a top line, preliminary basis, the Phase II study achieved the prespecified primary efficacy endpoint for Stage 1.

The patient population on Slide 28 is also characteristics of the general patient population with autoimmune hemolytic anemia, mostly middle-aged women, and of course, they have a low hemoglobin as a sign of the anemia.

On Slide 29, we'll review the hemoglobin response in Stage 1 of this trial. 17 patients had at least one post-baseline hemoglobin measurement. We observed a response rate of 35%, 6 out of 17, on fostamatinib. Four patients responded during the 12-week evaluation period. And on a preliminary basis, an additional 2 patients met the response criteria in the extension study after 12 weeks of dosing. Two of the 17 patients withdrew early from the study due to non-safety-related reasons and will be replaced per study protocol. Further verification and a compressive analysis of the Phase II data will continue and will be presented in the near future.

In terms of safety profile, on Slide 30, there was no new adverse events in the program. Three patients with serious adverse events, all assessed as non-treatment related by the investigators. One patient recovered and continued on treatment. Two patients had serious adverse events resulting in fatalities. In both cases, these patients were immunosuppressed due to steroids and in one of them also due to CLL. One patient had skin necrosis with infection and another patient, an elderly gentleman, had pneumonia.

So in summary, on Slide 31, six out of 17 patients, approximately 35% of patients, had a response. Two subjects reached response during the extension period of the study. There was no new safety signal and no drug-related serious adverse events. Two patients dropped out and will be replaced per protocol. And we have achieved the primary endpoint successfully.

In terms of next step, we are now planning to enroll in Stage 2 of the trial for an additional 20 patients total. The orphan drug designation application is in progress. And we're working on our regulatory strategy to define an optimal path to approval.

Now I would like to pass you to Eldon for a look at commercial activity. Eldon?

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Eldon C. Mayer, Rigel Pharmaceuticals, Inc. - Chief Commercial Officer and EVP [5]

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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 our first product to market. Among 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, ensure that our approach to commercialization is correct. And 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 are approximately 65,000 adult patients with chronic ITP in the U.S., 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 that -- a year. The remaining 27% of those patients, or 17,400, are steroid refractory and receive any of the currently available treatments, such as rituximab, TPO agents, splenectomy, et cetera. And this represents the addressable market for fostamatinib.

As part of our market analysis, we analyze the claims records of approximately 6,000 adult chronic ITP patients over a 5-year period. And we're able to see how most ITP patients typically progress through the various lines of therapy, this is shown on Slide 35.

As a general comment, it's important to note that because the chronic ITP population is a heterogeneous disease, it's difficult for a clinician to predict which patient might respond to a given treatment, if any, and therefore, many different therapies are used and the sequencing of treatment is highly variable. So given all of that, it's actually not surprising how fragmented this market actually is and a no single treatment dominates any line of therapy. It's also important to understand that patients frequently cycle on and off the various available treatments and in addition, steroids are frequently used concomitantly with all of these treatments, although this kind of steroid usage is not shown on the slide.

Moving on to the next slide, another important area we learned about as a result of our analysis and market research thus far is the unmet treatment need that exists for this market. First, we were able to confirm that among most physicians treating ITP, the primary pathogenesis of adult chronic ITP is thought to be immune system platelet destruction, as Anne-Marie mentioned. And yet, this disease pathway is not specifically addressed by any currently available treatment. Additionally, there are number of other areas that physicians have expressed as unmet needs, including a high rate of additional treatments utilized after splenectomy and rituximab, a high rate of fluctuation in platelet levels that exist over the course of currently available treatment, and some safety concerns that exist with these treatments.

Over the course of our preparation for the launch -- excuse me, of course, our preparation for launch goes well beyond market analysis. Our commercial team has been very busy with launch preparation. And on this slide, you can see just a few examples of our key initiatives. I'm pleased to say that we're well on track to being ready for launch in Q2 of next year.

And lastly, on Slide 38, you will see an overview of the structure of the commercial team we've been building. I'm pleased to report that we've been successful with identifying and hiring a team with extensive industry and launch experience in both rare diseases and in large markets and in small as well as large companies. We plan to build a seasoned sales team with a strong background in hematology, oncology and oral oncolytics, and we've already hired all of our key leadership, including a national sales director. We're very excited about the opportunity to bring fostamatinib to market, which we believe can make a difference in the lives of patients with ITP if it needs other treatment options over what is currently available, and I'm confident that with Rigel management and this team, we'll be able to do that successfully.

Now I'll turn the call over to Ryan for financial update.

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Ryan D. Maynard, Rigel Pharmaceuticals, Inc. - CFO and EVP [6]

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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 runway comfortably into 2019, including the potential launch cost for fostamatinib. Any upfront amounts received from successfully partnering out rights to fostamatinib in Europe or Asia would add to this runway.

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 potential commercial launch in the second quarter of 2018.

Now on to some of our financial results from Q3. We reported revenues from collaborations 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 total 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 costs and expenses was a result of the reduction in workforce undertaken in September of '16 and the completion of the pivotal Phase III clinical trials in 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 systems 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.

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Raul R. Rodriguez, Rigel Pharmaceuticals, Inc. - CEO, President & Director [7]

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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 of 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 an 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.

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Questions and Answers

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Operator [1]

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(Operator Instructions) Our first question comes from Eun Yang from Jefferies.

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Kyung Yang, Jefferies LLC, Research Division - MD and Senior Equity Research Analyst [2]

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Question on first in autoimmune hemolytic anemia. So you're moving into Stage 2 [expression] cohort. Are you expecting to start that Stage 2 trial with additional 20 patients by end of this year?

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Raul R. Rodriguez, Rigel Pharmaceuticals, Inc. - CEO, President & Director [3]

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Thank you, Eun

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Anne-Marie S. Duliege, Rigel Pharmaceuticals, Inc. - Chief Medical Officer and EVP [4]

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Eun, there may be 1 or 2 patients that may come up before the end of the year depending what's happening at the existing sites that are continuing to enroll in the trial. But more importantly, in order to enroll the majority of the patients in cohort 2, we will open new sites and that will start next year.

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Kyung Yang, Jefferies LLC, Research Division - MD and Senior Equity Research Analyst [5]

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I see. Do you think you need this additional 20 patients from Stage 2 in order to meet with the FDA to talk about -- to discuss about regulatory path?

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Anne-Marie S. Duliege, Rigel Pharmaceuticals, Inc. - Chief Medical Officer and EVP [6]

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No, we intend to go and talk to the FDA once we have completed Stage 1 and looked at all the data in detail .

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Kyung Yang, Jefferies LLC, Research Division - MD and Senior Equity Research Analyst [7]

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Okay. That would be first quarter next year?

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Anne-Marie S. Duliege, Rigel Pharmaceuticals, Inc. - Chief Medical Officer and EVP [8]

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First half of next year. But possibly, first part [ability] first half of next year.

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Kyung Yang, Jefferies LLC, Research Division - MD and Senior Equity Research Analyst [9]

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Okay. And then, also, you submitted late-breaker abstract for ASH on this data. So if it's not accepted, then would you provide us with the detailed data by year-end?

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Raul R. Rodriguez, Rigel Pharmaceuticals, Inc. - CEO, President & Director [10]

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I think, Eun, this is Raul. 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 for that.

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Kyung Yang, Jefferies LLC, Research Division - MD and Senior Equity Research Analyst [11]

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Okay. But you will find out if it's accepted by November 22, correct?

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Raul R. Rodriguez, Rigel Pharmaceuticals, Inc. - CEO, President & Director [12]

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That's correct. And we do have an oral presentation on the ITP data at ASH, [Dr. Forcell]. So we'll inform you and the rest of our community in terms of if we do have a late-breaker or not.

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Kyung Yang, Jefferies LLC, Research Division - MD and Senior Equity Research Analyst [13]

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Okay. The last question is on the partnership of first ITP outside the U.S. I think on the last call, you mentioned that products progress on the NDA in the U.S., maybe important for securing a deal for a substantial amount of money. So should we expect that any kind of a partnership ex-U.S. to be expected after the PDUFA date?

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Raul R. Rodriguez, Rigel Pharmaceuticals, Inc. - CEO, President & Director [14]

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

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Operator [15]

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Your next question is from Anupam Rama from JP Morgan.

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Tessa Thomas Romero, JP Morgan Chase & Co, Research Division - Analyst [16]

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This is Tessa filling in for Anupam this evening. Given that fostamatinib represents a novel mechanism of action for the treatment of ITP in a sense addressing the disease from the opposite side to TPOs to boost platelets, how should we be thinking about the potential for combination use? Do you have any plan to study fosta in combination use?

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Anne-Marie S. Duliege, Rigel Pharmaceuticals, Inc. - Chief Medical Officer and EVP [17]

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

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Operator [18]

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Our next question comes from Do Kim from BMO Capital.

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Alexander Xenakis, BMO Capital Markets Equity Research - Associate [19]

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This is Alex on for Do. I wanted to know a little bit more color around label negotiations with the FDA and what you think the chances are for broad label for fostamatinib?

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Anne-Marie S. Duliege, Rigel Pharmaceuticals, Inc. - Chief Medical Officer and EVP [20]

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Yes, so we're going to initiate our discussion with the FDA shortly on label negotiation. We are asking for broad label, which is justified by the protocol, with protocol where defined -- where patients had failed one treatment without specifying which treatment or the number of previously failed treatment before. And this is what we have requested. So we'll see. At this point, the FDA has not given us an indication otherwise. But a little bit too early to say that.

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Alexander Xenakis, BMO Capital Markets Equity Research - Associate [21]

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Okay. And with the new clinically relevant platelet response amongst the physicians that you've talked to, how do they view this endpoint with respect to other endpoints? Have they seen a stable response and intermittent responses?

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Anne-Marie S. Duliege, Rigel Pharmaceuticals, Inc. - Chief Medical Officer and EVP [22]

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They certainly evaluate as more clinically relevant endpoint. As I indicated during the presentation, the stable response rate is the one defined by the FDA as a high bar and similar to what has been used to approve TPO agents before. So based on the guidelines and based most importantly on the clinical experience of these physicians, we do not need to see consistently platelet count of 50,000 rather an increasing platelet counts of approximately -- up to 30,000 and above. And most importantly, a reduction in bleeding episodes is what matters for physicians, and of course, to the patients as well.

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Alexander Xenakis, BMO Capital Markets Equity Research - Associate [23]

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And just one more question. How variable is the time to response from patient to patient that you see in ITP? And also, is there a similar time to response that you see in AIHA patients?

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Anne-Marie S. Duliege, Rigel Pharmaceuticals, Inc. - Chief Medical Officer and EVP [24]

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Yes, as indicated, the response is pretty rapid. 60% of the responders will respond within 2 weeks. Approximately 80% of the responders within 8 weeks, and the totality of the responders by week 12. And we seem to see approximately the same rate in autoimmune hemolytic anemia, although 2 additional responders responded up to 12 weeks.

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Operator [25]

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Our next question is from Joe Pantginis from H.C. Wainwright.

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Joseph Pantginis, H.C. Wainwright & Co, LLC, Research Division - MD & Senior Healthcare Analyst [26]

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

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Raul R. Rodriguez, Rigel Pharmaceuticals, Inc. - CEO, President & Director [27]

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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 the next upcoming step. That is obviously scheduled prior to our PDUFA date.

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Joseph Pantginis, H.C. Wainwright & Co, LLC, Research Division - MD & Senior Healthcare Analyst [28]

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And any additional things that might be required like facilities?

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Raul R. Rodriguez, Rigel Pharmaceuticals, Inc. - CEO, President & Director [29]

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Well, it's a very good question. We have not had any facility inspections as yet. That might be required or sometimes, I'm told, they are not required. So it's a bit variable in that.

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Joseph Pantginis, H.C. Wainwright & Co, LLC, Research Division - MD & Senior Healthcare Analyst [30]

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Sure. That's good. And then just wanted to go back to AIHA for a second. Do you have any more color with regard to the time lines of enrolling the second stage?

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Anne-Marie S. Duliege, Rigel Pharmaceuticals, Inc. - Chief Medical Officer and EVP [31]

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

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Operator [32]

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

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Raul R. Rodriguez, Rigel Pharmaceuticals, Inc. - CEO, President & Director [33]

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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 our regulatory and commercial milestones and on our pipeline. And as always, thank you for your support. We greatly appreciate it. Good afternoon.

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Operator [34]

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