Rigel Pharmaceuticals at Cantor Conference: Strategic Growth and Expansion

On Wednesday, 03 September 2025, Rigel Pharmaceuticals (NASDAQ:RIGL) presented at the Cantor Global Healthcare Conference 2025, highlighting its robust growth trajectory and future plans. The company shared both its strong financial performance and strategic ambitions, aiming for significant expansion in the hematology and oncology sectors.

Key Takeaways

• Rigel raised its full-year revenue guidance to $270-280 million, reflecting strong sales growth.
• The company reported a 76% increase in Q2 revenue compared to the previous year.
• Key products TAVALISSE, Reslidia, and GAVRETO have driven substantial revenue growth.
• Rigel is advancing its development programs, including R289 for lower-risk MDS.
• The company maintains financial discipline while seeking additional late-stage product acquisitions.

Financial Results

• First half revenue reached $102 million, with a 76% growth in Q2 over the previous year.
• GAVRETO achieved $11.8 million in net sales in Q2, outperforming previous expectations.
• TAVALISSE and Reslidia reported year-over-year growth of 52% and 36%, respectively.
• Rigel’s cash position increased from $77 million in Q1 to $108 million in Q2.
• The company revised its revenue guidance upwards, anticipating $270-280 million for the year.

Operational Updates

• TAVALISSE is being used in earlier lines of therapy for ITP, improving patient outcomes.
• Reslidia showed a 35% complete response rate in relapsed/refractory AML patients.
• GAVRETO sales surged following its acquisition from Genentech, aided by updated treatment guidelines.
• Rigel is progressing with the R289 study, moving into the dose expansion phase soon.
• Oludecitinib is under study for glioma and other cancers through strategic collaborations.

Future Outlook

• Rigel expects 50% growth this year and an additional 15% in the second half.
• The company aims to continue expanding its commercial business with TAVALISSE, GAVRETO, and Reslidia.
• Strategic plans include advancing development programs and identifying in-licensing opportunities.
• Rigel is committed to maintaining financial discipline while pursuing growth.

Conclusion

For more detailed insights, readers are encouraged to refer to the full conference call transcript below.

Full transcript - Cantor Global Healthcare Conference 2025:

Ayan, Cantor: Everyone. Welcome. I’m Ayan with Cantor. So we have a presentation from Rigel Pharmaceuticals’ CEO and President, Raul Rodriguez, here with us. Raul, take it away.

Raul Rodriguez, CEO and President, Rigel Pharmaceuticals: First, let me thank you for having invited us to present at the conference, well attended. It’s a nice way to kick off the fall season and the second half of the year or third half of the year, I suppose. So important statements here are forward looking. They’re also available on our website. This presentation is available on our website, a more expanded version, actually.

So feel free to look at that and read these important statements. Let me tell you about Rigel. We are a hematology and oncology company and very happy with where we are. But we want to be much, much larger. Much larger, much profit more profitable, bigger products, more products, larger products is the objective.

And I’m going to tell you how we’ve gotten to where we are today, and that will signal to where we’re going into the future. A couple of different parts of the story. One is commercial execution. We have three commercial products: TAVALISSE, indicated for the treatment of ITP, immune thrombocytopenia Reslidia, indicated for IDH mutant relapsed and refractory AML and most recently GAVRETO, indicated for RET fusion positive non small cell lung cancer and thyroid cancer. All three of these products have been growing and contributing to the substantial growth on the top line.

I’ll share a little bit more with you in a minute. On the right side, we are working on developing major opportunities for the company in the hemonc area, led by R289, our dual IRAK1 and four inhibitor that we have in study for lower risk MDS, a large area with substantial medical need. In addition, oludecitinib, Reslidia, we’re studying in various places beyond relapsed and refractory AML, where IDH mutations play an important role. These include glioma and various other hematologic conditions. I’ll touch on that a little bit.

And we continue to look at in licensing and acquiring other products. Reslidia and GAVRETA were acquired in the last couple of years few years, and we look to continue to do that. Late stage, data ready, NDA ready or NDA filed or NDA approved or already on the market. Tie that all with financial discipline, very moderate growth in terms of our overall expenses. What results is in a very profitable company, profitable enough to do a lot of important things in the clinic to take the company to the next level.

And that’s our objective. Here’s how we’ve done in the last few years. Very impressive quarterly and quarterly growth. This first half of this year, 102,000,000 achieved. In Q2 alone, 76% growth over Q2 of last year, a very dramatic growth.

For the year, 102,000,000, kind of comparable to what we did in all of ’twenty three. So you begin to appreciate that the strategy that we have in place to grow our three commercial products, ad commercial products, is really working and working pretty well. I’ll come back to that at the end of the story. Let me take you through each one of these. There are some highlights I’d like to hit.

TAVALISSE and ITP. This is the indication for TAVALISSE, indicated for the treatment of adult chronic ITP for patients who had failed one previously some insufficient response to a previous agent. So here’s what the market looks like. It’s about eighty one thousand patients in The U. S, adults with chronic ITP.

About thirty seven of them are simply watchful waiting. They clearly have the disease, but they don’t require treatment immediately. That is, there’s a mild form of disease, they have low platelet counts, but it’s manageable without therapy, or they’re in some temporary remission that quickly goes away. Beyond that, the first line of therapy is always, almost always, a steroid, twenty thousand patients. We’re indicated for the twenty four thousand patients beyond that, from second, third, fourth and fifth line and beyond.

Now when we launched this product, we were used substantially in the later lines, fourth and fifth line. Doctors used TPO agents, they used Rituxan. Those were generally the first and second line or the second and flip flop the order, but different doctors used those two agents in the earlier lines. As we’ve had the product on the market and have the opportunity to share the data with doctors and have the opportunity to remind them of the product and its benefits, we are now used across all the second, third, fourth and fifth line, not just the more refractory lines. And third line in particular, we’ve been able to penetrate that segment nicely.

And it’s understandable why we would want to penetrate these lines that are earlier. There are much larger numbers in the earlier lines. Obviously, that’s an important part for it. And in addition, the earlier you use the product, the better your results. That is, if you used it in fifth line, well, maybe about half the patients have some benefit.

However, if you use it in second and third line, it’s eighty percent, ninety percent have a benefit. That’s where we want to get at, because that’s where the benefit really accrues to patients and obviously so many more of them. Put those two things together with some time and doctors using this product regularly, it becoming a standard of care, and doctors using it and getting comfortable with it, having some success, it builds on itself. That is, doctors will now use it in earlier lines if they’ve used and succeeded in an earlier line in the past. And any patient you get on this product that succeeds has the potential to succeed for a long time.

So it allows us to carry into each quarter after each quarter a book of business that is pretty solid. And now we work on adding new patients for that quarter and carrying some of those patients on into subsequent quarters. It’s a pretty good business. It’s taken the time to build to this, but we’re really delighted where we are with the product. Let me move on to Reslidia, a mutant IDH1 relapsedrefractory AML.

This is the indication. It’s for patients with refractory AML who are susceptible to an IDH mutation. Now, this is a terrible disease. Every year, about twenty two thousand patients are diagnosed with AML. Every year, about half that number, eleven thousand, die from this indication.

So it’s not well treated as yet significant need here. IDH mutations are present in about six percent to nine percent of AML cases. So it’s a smaller number, but very substantial. We’re delighted to have an IDH1 product here that addresses those. These patients are well identified And especially in the that is, they’ve been genotyped.

And especially in the relapsedrefractory setting, really very limited options available to them. So we were delighted to evaluate this product and in license it a couple of years ago. And here’s what excited us about it. Primarily, this is a phase two study, just I want to outline some of the key data points here. Primarily an elderly population, that’s typical of AML, 71 years old was the median age.

Many of them had high risk factors, two or more prior treatments, and all had none of them have ever had an IDH inhibitor. What we like the most about the product is that the response rate, the CRCRH rate was thirty five percent. Really nice numbers for this indication. And most importantly, a duration of response of nearly twenty six months, a substantial improvement over what was available previously. When you look at the quality of those responses, almost all the cases were CRs rather than CRs and CRHs.

And with those patients, twenty eight months duration, really great duration. If you achieve a response here, you’re likely to keep it for quite some time. And what that tells you is that this product will be used longer. It tells you that these patients are benefiting from this longer. And that’s a really great achievement given how terrible this disease is.

So we’re really pleased with this profile, and that’s why we licensed the product and have done very well in presenting it to doctors, and they’re beginning to use it on a regular basis. Moving on to GAVRETO. GAVRETO is indicated for RET fusion positive non small cell lung cancer and thyroid cancer. I won’t read the whole thing here, but read it online. And here’s what’s excited us about GAVRETO.

GAVRETO had really nice data. RET fusion positive non small cell lung cancer was an area of significant need, And the data here was really compelling. There’s another RET inhibitor by Lilly, Retevmo, that is the predominant product that launched earlier, had more data than GAVRETO. GAVRETO was being marketed by Genentech and Roche across the world, Genentech in The U. S.

And we saw the data. They were looking to return the product to its originator, Blueprint, and Proofprint needed a marketing partner. What excited us about this product is that we thought we could grow in this market, In particular, the areas in gray and light and dark gray, they’re treated with multi kinase inhibitors and chemotherapeutics and immune checkpoint. The data in these patients is inferior with those methods than with a RET fusion product like ours. And we thought we could help those patients that are now receiving those other agents, the older agents, and are not getting good satisfaction with that.

And we can eat into that twenty five percent and give them a better drug for their illness And grow the product from what’s fifteen percent to a higher percentage point. Our predecessors at Genentech had sold the product. And and and what we wanted to do is to change the practice. We were really fortunate that at the same time, the guidelines shifted last year. They shifted to say, if you have a RET fusion positive patient, you should treat them with a RET fusion inhibitor.

In addition, if you have a patient responding with a multi kinase inhibitor or chemotherapeutic, you should switch them to a RET fusion inhibitor. It’s a better approach. So we’re out there saying this. The guidelines shifted in our favor, and that has benefited us tremendously. I’ll show you the impact of this.

So just staying on GAVRETO. We achieved $11,800,000 in net sales last quarter, Q2. Our predecessor, Genentech, had a run rate of seven when they were marketing the product. We’ve only had it for three quarters. So already you see substantial uptick in terms of the sales.

We’re seeing new doctors who use the product and new patients, obviously, who take the product and are benefiting from it. So I think it’s a tremendous achievement for our commercial organization, bringing a new product in. And typically you see a decline in sales in the first six months or so as you lose some patients. We lost very, very few. Excellent job in transferring it over to us.

And in addition to that, a compelling story to tell. And now we’re out there telling this story, the guidelines helping us. We achieved great results with this product. TAVALISSE grew 52% from a year ago, a great achievement and continues to look very promising. And Reslidia also 36% from a growth year, all three products contributing tremendously to the growth of the commercial business of the company.

That’s in The U. S. Outside of The U. S, we deployed partnerships to help us commercialize these products. TAVALISSE has partnered with Grifols in Europe, Kissei in Asia and Medison in Canada and in Israel.

This past year, we achieved launches in South Korea in July, and other countries will follow with TAVALISSE. Reslidia, we’ve also begun partnering. Here, we are partnered with Kisei again, our partner with Reslidia in Japan, South Korea and Taiwan, and entered into an agreement with Doctor. Reddy in Latin America and other countries. And you begin to see that on a quarterly basis, we’re seeing some fairly nice revenue from our partners.

So the royalty revenue is meaningful. In addition, they purchase product from us at a markup, and that becomes meaningful to us as well as that number begins to grow nicely. So the ex U. S. Opportunity is much smaller than The U.

S. Opportunity, but for us, profitable and attractive and incremental to what we’re doing ourselves. Let me move on to the clinical development update. I want to there’s two pieces to that, and I want to start on the in licensing and product acquisition side. So as I said earlier, we’re looking for differentiated products where we feel we can add value that are in the hematology and oncology space.

They have to be late stage. That is, we have to have data that allows us to register the product or it’s already been filed, the NDA has already been filed or the NDA has been approved or the product is already on the market. It could be any one of those stages. Reslidia, the NDA had just been filed, but got rid of the product that had already been on the market. And this allows us to launch the product quickly in the next few years, and it quickly becomes accretive.

That is, it quickly pays for itself. And that’s the objective. We for those two products, we had minimal increases in our infrastructure. And that allows us to have most of the opportunity, the sales of that product drop down to the bottom line fairly quickly. It’s a good strategy.

It’s helped us tremendously with both GAVRETO and Reslidia, and we hope to replicate that at least once, maybe twice in the coming years, and hopefully even products that as attractive as those and maybe even a little bigger. That’s going to be a tremendous portfolio of commercial products in the near term. And it’s going to be cash generating as a business. What do we want to use that cash for? What we want to do is to invest it in large major opportunities that are on the left side of this slide, particularly the R289 IRAK14 inhibitor, which has tremendous opportunity in lower risk MDS, an area of great medical need.

I’ll tell you a little bit about that. So here’s the landscape for lower risk MDS. First line are typically ESAs. Luspatercept is used there. If you have a Del-five deletion, thalidomide is used.

Failing that, you go to either luspatercept or the recently approved of Rytello. Failing one of those, you go to an HMA. Now the response rates are not terribly high across the board here. HMA is about eighteen percent, twenty percent. Luspatercept and Rytello, maybe about thirty eight percent or forty percent, much earlier patients you see there.

’2 eighty nine has potential in a wide gamut of these this landscape, from after ESAs to after an initially approved agent like luspatercept to after an HMA is used. The opportunity is bigger in the middle and on the left side of this slide, smaller after HMAs. Here’s what excites us about this product and its value proposition. There are large numbers of patients, 12,000 more patients that are previously treated. And they are dependent on transfusion on a regular basis, which is a tremendous burden for these patients and the medical system to continuously give them red blood cells on a regular basis to keep them in normal.

A key mechanism underlying MDS is a dysregulated inflammatory signaling, particularly in the bone marrow. That has become to be appreciated much more so in the last five or ten years that there is a key inflammatory cascade, a cytokine storm basically, in the bone marrow that’s driving a lower risk MDS. And targeting IRAK1 and four, we thought, may have a benefit in these patients. What we’ve done is we looked at this because IRAK1 and four blocks toll like receptor and IL-one signaling in places in the body. And we thought that those were two key cascades that if we can help regulate them, we may have a benefit in lower risk MDS.

We tested this in a healthy volunteer study, looking at patient volunteers where we gave LPS protein, cytokine storm ensues, and then we showed that we’re able to ameliorate that with ARQ289 or its other product, eight thirty five. And what we did there is, with healthy volunteers, an acute setting, we hope to replicate that on a chronic setting in a condition like lower risk MDS. The FDA has granted us Fast Track designation, orphan drug designation, which is fantastic, showing that they’re willing to and excited to work with us on this opportunity. And the initial data is very promising. So I’ll show you a tiny bit about that.

Here’s the study we launched. It’s a very standard Phase Ib study. You have a dose escalation phase, where you start at a very low dose and you escalate up. You’re testing for safety, seeing if you see some signals. You also want to see PK and PD results as well.

Having completed that, then you go on to a dose expansion phase. So at the last ASH meeting in 2024, we shared data on dose level one through four of this study. This year we’ve enrolled dose level five and dose level six. And our plan is to share data at the end of this year on all the dose levels. So then you will have data again, they’re small numbers, maybe six patients a dose.

So not that many. It’s hard to be definitive, of course, it’s small numbers. But it does tell you on the safety, it does tell you on the PK, and it does give you some signals on the efficacy side looking at transfusion independence. What we said at our financial call last month is that we met with FDA. We agreed with them on what the dose selection for the dose expansion phase would be, primarily safety, not surprising, and exposure.

And so with that, we’re going forward and we’ll select those two doses. And our plan is to start the dose expansion phase in the next couple months. Possible to even do it at the end of this month in September or early October. So we’ll be able to launch those studies. And now you’ll aggregate 20 more patients a dose.

So after this dose expansion phase, which we’ll start now and go on through next year, we’ll have about 25, 26 patients in each of the dose groups. That’s a pretty robust data set that will tell you if this product has legs and what the dose level is. With that, then we’ll start a registrational study. And we hope to engage with FDA in terms of what that looks like. But drugs have been approved for this indication just recently.

And transfusion independence is likely the primary endpoint that everyone is looking for. So it’s an exciting time for the product. The data from last year, I’ll share a couple of key pieces there. And we’re excited to share these two new dose groups and to start the expansion phase. There’s a tremendous medical need in this area, with agents not providing results for many or even most of the patients that have it.

And a mechanism that is unique, where Rigel is in the lead in this area, with a mechanism that I think is a great fit from mechanism of action to the disease process. Here’s some data from our that we presented at the ASH meeting last year. The safety was good. Incidence of cytopenias are grade three and grade four, and infections were low. At doses above five hundred once a day, plasma concentrations were very good, that is, they reached fifty percent or ninety percent of what we saw with normal healthy volunteers and saw that we were able to get positive results with this product.

Third, we saw three of ten evaluable transfusion dependent patients that achieved transfusion independence. So that’s a really good outcome, especially considering that these patients were highly, highly refractory. Seventy seven percent had failed luspatercept HMAs and seventy three percent had failed luspatercept. High failure rates in those approved agents already, and yet we were able to get three out of ten to two of them to have a positive response. I think that’s a tremendous achievement.

The clinicians who are involved with the study were excited by that result and really encouraged to finish this dose escalation phase and then go on to the dose expansion phase. Also, those patients that achieved a transfusion independent also see all their hemoglobin increase, 2% to 5%, which is quite meaningful. So really nice results for this. Again, all early, and we hope to have more results at the end of this year and really more definitive results probably at the end of next year, around the end of next year. Let me move on to oludecitinib or Reslidia in mutant IDH inhibitors.

Glioma is an area of significant need. This is an area where we’ve, until recently, not made much progress. And a significant problem, twenty thousand patients a year, who have glioma, it’s very deadly. Almost always recurs, and almost always there’s not a good therapy available for these patients. Our predecessor, pharma, where we licensed Elluda, did this very nice study, twenty six patients in highly refractory relapsed patients.

And what they saw is that we had two PRs and about ten stable disease. That is a disease control rate, as they called it, about half the patients. For this patient population, as refractory as kind of worn out and at last stages as we saw, that’s a really good outcome. So we were delighted. It also tells you the product causes the BB, crosses the BBB, and is having an impact there, which is great to see.

So what we’re doing, we did two things acquiring the product. Quickly, put a collaboration in place with the Connect organization. This is a group that’s doing a nationwide umbrella study in high grade glioma. And now they’ve added Eluda as one of the arms in patients who would have IDH mutations. This is called the Targeted D study arm.

This study is now open for enrollment and we hope that they’ll recruit patients and we’re able to see this. This is primarily in a maintenance setting, that as patients come in, they are they have surgery, they have perhaps radiation, and then they go into the study, see if TMZ, standard agent in this area plus ALUTA, has positive results, Do that for twelve months and then switch only to Elluda as a maintenance beyond that. So it’s an exciting program. Excited to collaborate with the Connect organization about it. Ourselves, we’re looking for looking at additional studies where we might conduct in glioma, and we’ll be sharing that with you later on this year.

In addition, we put in place a collaboration with MD Anderson, one of the nation’s premier cancer centers, for the study of Eluda in AML and other cancers. We’ve put a collaboration in place looking at the product in AML, frontline, as well as relapsedrefractory. And the frontline is exciting because we don’t have an indication there. We want to generate data in that setting. Additional high risk MDS in CMML, an area where there’s substantial need, we’re conducting a study with Eyre.

And with them also as monotherapy in CCUS, which is an earlier indication, as well as lower risk MDS, in addition in post transplant in the maintenance setting. So we have four studies and ability to add more to this in terms of what we’re doing with MD Anderson to generate robust data sets across key areas of opportunity for Eluda. So this is what we’re going to accomplish this year. We’re well on our way. Complete dose escalation phase of the Part one Phase Ib study.

We’ve completed that already. Initiate dosing in the expansion phase. We’re going to do that shortly. And interact with FDA on potential registration path. We’ve done initiated those discussions with them and on the dosing of the dose escalation phase, expansion phase, and then now we’re talking to them about the registration path forward, and present the data at the end of the year at a medical meeting.

So look forward to achieving all of those objectives. For Eluda, plan on initiating a Phase II study. We’ll discuss that further with you. And then continue to support our MD Anderson collaboration as well as our KINECT collaboration in glioma. So a lot to accomplish and I think we are checking each of these boxes by the end of the year.

I will pass I will just say a few words on our RIP kinase inhibitor. Our RIPK program was discovered here at Rigel. And there are two pieces to this program. One is an immune program led by R552, now called okadusertib. Lilly is conducting a Phase IIa clinical study in rheumatoid arthritis with this product.

This has potential to be a successor to the JAK programs and other treatments in rheumatoid arthritis that have some issues. And they’re excited about this opportunity. They’ve called this the best RIP kinase inhibitor there is in clinical study right now. And we’ll share data with us and subsequently you at a later date. But we’re excited about the potential of this molecule.

In the CNS side, a second program, also within the same collaboration, is in CNS diseases. Our job was to get Lilly a basket of blood brain barrier penetrating RIPK inhibitors. We’ve done that. And now Lilly will move forward in getting those into the clinic. It’s an exciting program, potential to be very large areas in RA, psoriasis, IBD, on the CNS side, perhaps Alzheimer’s, but multiple sclerosis and other areas.

Major, major areas where a big party with deep, deep pockets like Lilly can really execute well on. So we’re delighted to have them as a partner and delighted to have data producing. We will get milestones, in some cases substantial ones, and we’ll get royalties from this, from both sides of those collaborations. So let me touch on our financial position, because I say this is a chart I’m really happy to share with you. On the bar there, the bar chart, you see there very impressive growth, impressive growth going back quarters.

And so you see continued nice growth and of late, some very substantial increases as all three products have contributed to that growth. TAVALISSE continues to grow nicely GAVRETO continues to grow well And RESLIDIA contributes substantially as well. So all of are doing very well. In addition, collaborations revenue is substantial. The Lilly is a reversal of upfront payment that they gave to us some time ago.

So that’s a noncash item. But it’s nice having it, and it’s nice having all the other revenue there. As you see, a total of about 12 about $2,500,000 in revenue from the other partners in this past quarter. So really good contribution from partnerships as well. And what we did at the last this is a bit of an eye test, apologies for that.

What I wanted to say about this is, one, we’re profitable as a company. We were profitable for the calendar year last year, ’24. So far, we’re even more profitable this year. As you see the first six months, 71,000,000 in profit, 40,000,000 of that is that Lilly reversal non cash. So if you strip that out, still a really attractive profitability, 31,000,000 for the first half of the year.

That’s fantastic. Our cash position went from $77,000,000 at the end of Q1 to $108,000,000 at the end of Q2. So we’re generating cash and we intend to continue to generate cash. We also said that we’re going to raise our guidance from total revenue of $270,000,000 to $280,000,000 previously it was 200,000,000 $210,000,000 And net product sales, we raised net product sales revenue to $210,000,000 to $220,000,000 a significant uptick from our prior guidance at the start of the year. And we said that we’re going to be profitable, net income profitable for the calendar year despite increased investment in our development portfolio, particularly with two eighty nine and Oluda.

This is a chart that is really astounding. Our growth this year is really based on our growth for the last four years. We’ve grown the business at a 32% CAGR every year since ’twenty one, really good growth. And this year, we’re expected to grow at about 50%. Again, a tremendous year for us, one we’re really proud of.

The profitability drops to the bottom line. That allows us to invest in creating major opportunities. Example, $2.89 in low risk MDS that will take the company to yet a very different level in the near future. So really excited about this. And we’re planning on having these four key value drivers for the balance of the year, continue to grow the commercial business, TAVALISSE, GAVRETO and VERSLIDIA.

We have 50% growth above from Q1 to first half of the year to second half, 15% additional growth is what we’re projecting. Advance the development programs, particularly $2.89, into the clinic and share that data and identify additional candidates that we can bring an in license on a semi regular basis and continue to be financially disciplined. That is, continue to show that we’re able to fund what we want to do ourselves and do so effectively. So it’s a great place to be at the company. It’s a growing company.

Sales are growing. Profitability is growing. Cash generation is improving. Things to invest it in that’s going to take the company to the next level in the near future. That’s an exciting time to be here.

So thank you so much for your support and your interest in Rigel.

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