Rigel Pharmaceuticals at Jefferies Conference: Strategic Growth and Innovation

On Wednesday, 04 June 2025, Rigel Pharmaceuticals (NASDAQ:RIGL) presented at the Jefferies Global Healthcare Conference 2025, highlighting its strategic growth and innovation in the hematology and oncology sectors. The company showcased its profitable commercial business and disciplined financial management, while also acknowledging the challenges of maintaining growth momentum in a competitive market.

Key Takeaways

• Rigel Pharmaceuticals projects net product sales of $185 million to $192 million for the year, marking a 32% growth.
• The company’s commercial success is driven by three approved products: TAVALISSE, Reslidia, and GAVRETO.
• Rigel plans to reinvest profits into developing new opportunities, including R289 for lower-risk MDS and Oludasidenib trials for glioma.
• Rigel has formed partnerships with major organizations to expand its product reach and pipeline.

Financial Results

• Net product sales are expected to grow by approximately 32%, reaching $185 million to $192 million.
• Q1 sales for TAVALISSE were $28 million, a 35% increase from the previous year.
• Reslidia sales reached $6 million, growing by 25% year-over-year.
• GAVRETO sales surpassed those of its previous owner, Genentech, within a short timeframe.
• Total revenue is anticipated to be between $200 million and $210 million, with contract revenue contributing $15 million to $18 million.
• Rigel maintains a cash reserve of approximately $77 million and expects to continue achieving positive net income this year.

Operational Updates

• Rigel’s commercial business has experienced a 32% compound annual growth rate since the COVID-19 pandemic.
• TAVALISSE demand continues to rise, supported by a streamlined distribution system.
• The transition of GAVRETO’s acquisition was successful, with no initial sales decline.
• The company is actively promoting Reslidia to increase awareness among patients and healthcare providers.
• Upcoming presentations at ASCO and EHA will feature data on Oludasidenib and GAVRETO.
• R289 is in a Phase 1b study, with enrollment progressing through dose levels.

Future Outlook

• Rigel aims to expand the adoption of TAVALISSE, GAVRETO, and Reslidia, ensuring strong year-over-year growth.
• The company is committed to maintaining financial discipline to support development programs, especially for R289 in lower-risk MDS.
• Rigel plans to explore earlier treatment opportunities and expects dose escalation results for R289 by the end of the year.
• A Phase II study for Oludasidenib in glioma is planned.

Readers are encouraged to refer to the full transcript for a detailed account of Rigel Pharmaceuticals’ presentation at the Jefferies Global Healthcare Conference 2025.

Full transcript - Jefferies Global Healthcare Conference 2025:

Unidentified speaker: This is standard presentation. Welcome, Rahul. Thank you so much, Farshan. It’s nice to see you and my thanks to our colleagues at Jefferies for having invited us again. I appreciate the opportunity.

I’d like to tell you about Rigel and where we are. First, some important forward looking statements here. This is also available on our website, so please feel free to read this. Let me tell you where Rigel is right now. It’s a pretty exciting time for the company.

We have on our as you see on this summary slide here, our goal to grow our hematology and oncology business. I’d like to particularly focus on the commercial aspects of the business where we have three approved products on the market, TAVALISSE for ITP, Reslidia for mutant IDH1 relapsed and refractory AML and Gabretto for RET fusion positive non small cell lung cancer and thyroid cancer. Together this product makes a very potent commercial portfolio and we’ve done very well with it and I’d like to share that with you today. When you couple that strong commercial execution and strict financial discipline at the bottom left of right of the slide, what you have is a company that has reached the point where we are profitable. We are giving guidance to remain profitability.

And what we want to do with those profits from the commercial business is a really important thing because it in turn will transform this company yet again. We’d like to invest them in really exciting development opportunities. R289, our dual IRAK1 and four inhibitor is in a Phase 1b study for lower risk MDS. An exciting opportunity with tremendous medical need where we think this product may have a unique role and may provide an additional alternative to available therapies that I think may really benefit these patients. It’s probably the most exciting thing we’ve ever worked on at Rigel.

In addition, we have oludasidenib, which is approved for mutant IDH1 AML, but our plans is to go beyond that and consider a trial in glioma that we’re hoping to launch later this year. All of that, we continue to look externally for in licensing and acquisition opportunities for products that are late stage, have solid data backing them up, which we think allows us to leverage our current infrastructure and capacity in hematology and oncology areas. You put that together, a strong, growing, profitable commercial business, financial discipline, a development pipeline that is really exciting that will be transformative for the company And we’re able to fund ourselves through our own cash flow and looking externally for additional opportunities. It’s a very exciting place for the company to be, and I’m happy to tell you about it. Let’s start with this summary slide, which I love to share.

So since coming out of COVID, as you see the company’s commercial business has grown quite nicely, 32% CAGR a year. And that’s driven by growth in TAVALISSE shown here in the deep green bars, but then incremental adds by Reslidia starting in 2023 and GAVRETO started in 2024. We acquired GAVRETO halfway through the year in 2024 and already it’s having a substantial impact. And I’ll come back to this, but we expect more of the same this year. Our guidance is for 185,000,000 to $192,000,000 in net product sales this calendar year.

That is continuing approximately 32% growth over this current year and frankly even beyond this. So really exciting about having this as a base business to build on. Here’s a brief summary of Q1 of this year, which I think was an excellent Q1, our best ever. TAVALISSE continues to grow nicely, dollars 28,000,000 in sales, 35% growth over a year prior. Down at the bottom, Braslidia, six million and twenty five percent growth over a year and that’s still in its launch phase, so it’s still growing nicely.

And then Gavretto, our most recent acquisition, we don’t have a comparison, but our Genentech who was selling this product in The U. S. Was selling a 28,000,000 annually. That is about $7,000,000 a quarter. And as you see in a short order, we’ve already exceeded that.

And I can talk to you a little bit more about that. So an exciting business in Q1, we’re looking for continued growth from here for the balance of the year. And here’s why we think this opportunity is so important for TAVALISSE. We continue to see demand growth. We continue to see new patient starts.

And then the carryover that is quarter to quarter, what we see patients is once they’ve stabilized on with TAVALISSE and their ITP is under control, they continue to stay on drug for quite a while. And that’s really beneficial because it allows you to build the business steadily quarter over quarter. And we’ve also taken steps to streamline our distribution system, providing more efficiency and as well as easier access to patients. On Gavretto, so every time you acquire a new product, you generally expect to see sales decline in the initial quarters as that transition happens. Delighted to say that we’d had an excellent transition for this product for Rigel.

And as a result, we saw no decline in sales even in the first quarter where we had the product and we’ve grown it ever since. And that’s a testament to the work that we did, the coordination with our partners Blueprint and Genentech in making that transition work and frankly just a lot of efforts, making sure every patient that was benefiting from GAVRETO continue to do so now that they’re buying it through our distribution system. So demand continues and we expect this to go forward. Reslidia, we continue to make it aware patients and doctors aware of its benefits and as a result we think we continue to grow the product in relapsedrefractory AML. And it’s the education and the sharing of that data that continues and we’re doing an excellent job of it.

I’ll show you a couple pieces of data in a minute about why we continue to be so excited about Reslidia. So let’s start with ITP and TAVALISSE. So TAVALISSE is indicated for the treatment of adult chronic ITP in patients that have had a insufficient response to a prior therapy. That prior therapy is usually a steroid. That tends to be the first line.

And then products like TAVALISSE are considered. So there are about eighty one thousand patients in The U. S. Suffering from adults suffering from ITP. First line is typically a steroid, that’s about twenty thousand.

And then in second line and beyond, about twenty four thousand patients. And that’s where we’re indicated for all of those nodes where a patient is considering a next line of therapy in treating their ITP. At launch we were more used in the refractory lines, fourth, fifth line. And as the product has become an established part of the treatment paradigm, we’re being used across the board second, third, fourth, fifth line. And as a result, we’re growing the business and we’re growing the patient stability with the product.

What I mean by that is this, on the left hand side, the earlier you use this product, second and third line, the better your results are. So as doctors have become familiar and comfortable with this product in the later lines, they moved it up naturally. And as a result, the results are even better. Those patients, once successful on the product, tend to stay on it. And you see that on the right hand side.

The efficacy is a durable type of efficacy. And we’re delighted to have it be used across all those lines and having that durability allows us to really build a nice business here. Let me go on to Reslidia and IDH1 relapsedrefractory AML. So Reslidia is indicated for adult patients with relapsedrefractory AML that have an IDH1 mutation. As you probably know, AML isn’t just a god awful disease.

About twenty two thousand patients are diagnosed every year and you have about eleven thousand deaths a year. So quite a substantial number. IDH mutations are about six to nine percent of overall AML cases And by the time they get to the relapse and refractory setting, they’re generally genotyped. So you know that they have this mutation and you can access a product like Reslidia in setting whether they started as fit or unfit patients. And here’s what got us excited about this product.

It’s a clinical trial, primarily elderly people, that’s who gets AML. A CR CRH rate of thirty five percent. That was slightly better than what was available prior, but what’s really more impressive is the durability of that response. Nearly twenty six months of durability of that response. That’s about three times better than what was available previously.

So that’s an exciting advance in terms of this product and this indication. Now we can say the the the response rates are maybe a little better equal to, but boy, if you have a response, you really hold it for a long time. If you look at the CR, CRH, CR rates, we have about ninety two percent of our responders are CR responders. So pretty high quality. And for those it’s twenty eight months.

So really exciting data if the doctors use the product and stay on it, you can get good responses and hold those responses for a fairly long time. That’s a very sizable advance over what was previously available and it’s something that we’re very proud of and we’re sharing this data obviously with clinicians as we launch this product. GAVRETO in RET fusion positive non small cell lung cancer and thyroid cancer, it’s indicated for those two things, so I’ll skip this chart. So here’s what we’re excited about this product. So GAVRETO is a RET inhibitor, RET fusion for RET fusion positive patients.

And it has actually about a 50% market share as you see on the right. However, about twenty five percent of patients are treated with multi kinase inhibitors and chemotherapeutics and immune checkpoint inhibitors. It happens that those treatment approaches are not very good. They’re not as effective as the RET inhibitors are. So eating into that share, that twenty five percent that you see in the gray bars is our objective.

And growing this product by providing our GAVRETO to those patients and giving them a better treatment than they currently are receiving. And we were fortunate soon after launching acquiring this product that we were able to get and you see here the guidelines had changed to say that the preferred first line treatment for RET positive patients is a RET fusion inhibitor. And that even if a patient is succeeding or being treated with multi kinase inhibitors or chemotherapeutics, they should switch to a RET inhibitor. So strong support from the community in terms of helping us get this type of product out to these patients and hopefully have a real benefit on these patients. So we’re excited about the opportunity here.

The only once daily RET inhibitor available, highly durable responses, shown some results in brain mets, which I think are very supportive in a very well established safety and tolerability profile. So we’re excited about this. It’s still early stages for us. We just acquired it in June of last year. So we’ve only had it for three quarters.

And each of those quarters, we’ve grown the product and look to continue to grow this product nicely. So a very useful addition to the portfolio. Outside The U. S. In The U.

S. Is our sole focus for ourselves. We’ve put partnerships in place for TAVALISSE in Europe with Grifols, Kisei in Asia, Japan and medicine in Canada and Israel. And our partners have gotten the product on the market in various territories and produced fairly nice revenues from us for us this past quarter as you see here from the various partnerships. Included in this is a one time payment for approval in Korea from our partner Kisei, about $3,000,000 And in this is not just royalties, but also supply that we supply our partners for the product at a small markup.

In addition, we’ll put a couple partnerships in place for Reslidia, one with Kissei in Asia, Japan, and another one with Doctor. Reddy and across Latin American territory. So expanding in other countries beyond The U. S, but already a meaningful producer of our revenues. Let me move on to what I think is the most exciting piece of this presentation, the clinical development update.

So the clinical development pipeline has R289 and IRAK1 and four inhibitor, which we’re looking at low risk MDS and there we have both Fast Track as well as orphan designation from FDA. So very good engagement with FDA on this product and this indication. Elutacitinib, we’re looking at beyond relapsedrefractory IDH1 mutant AML. I’ll share a little bit of our trials we’ve launched in other areas as well as considering a glioma study, Phase II study in glioma with this product and I’ll talk to you a little bit about that. We continue to look at in license opportunities.

There’s quite a number of things out there. We’re looking at things that are differentiated, that have data that we think is compelling in hematology or oncology, that have late stage programs. That is, have registrational data in hand, have already perhaps filed an NDA, the NDA is approved and waiting to launch, or the product is on the market and we feel we can add value to the marketing of that product. They have to be synergistic with our current in house capabilities. So this is a strategy of Roger.

One, we’ve executed with two products now quite successfully and have been successful with the launch and execution of those products to now. So we’re excited about continuing that strategy because that plus the development pipeline, especially R289 and low risk MDS has potential to really grow this business very substantially and therefore its profitability and our ability to do even bigger trials and more important trials in larger indications over time. Let’s move on to thank you. One area that I would quickly highlight for you is the various presentations at ASCO and EHA focusing on Oluda and focusing on GAVRETO PRAL in various settings, which I think continue to confirm the benefit of the product, those two products in those respective indications as well as looking at a couple of different facets of patients that we hadn’t studied before. So the ASCO presentations have already occurred and those are available.

And then the EHAZ presentations are up and coming. But I think a robust set of data across those two products at those two conferences. Let me move on to two eighty nine in low risk MDS. Here’s what we’re excited about this. Tremendous value proposition.

So there are about twelve thousand patients with lower risk MDS that have previously been treated. That’s who we’re targeting for for our current trial. And we’re looking at taking those patients who are transfusion dependent and getting them to attain transfusion independence. And that’s a paradigm, the endpoint that the FDA has been looking for and therefore one that we are really excited about what we’re doing with this area because we think we can have a meaningful impact on lower risk MDS patients. The mechanism is novel.

No one else is working in lower risk MDS with an IRAK4 one and four inhibitor. In fact, I could say we’re the only one actively developing an IRAK1 and four molecule overall. And one in four confers us substantial advantages. One in four inhibition is better than IRAK4 alone. We’re able to more profoundly inhibit inflammatory cytokines, including in the bone marrow, which is our target in lower risk MDS.

And that’s an exciting addition to the treatment approach in lower risk MDS. IRAK1 and four inhibition blocks toll like receptor signaling and IL-one family of signaling. Two key inflammatory pathways that if we’re able to do that in the bone marrow, we should have a real benefit. We did a proof of concept study. We took normal healthy volunteers in a hospital setting.

We gave them an LPS protein and as a result, a cytokine storm occurs. And we’re able to demonstrate that with this product, we’re able to greatly diminish and in a dose dependent manner that cytokine storm. Now that’s an acute setting. We caused the insult. We solved the insult.

But if we could do that on a chronic basis, we may have a new approach to treating diseases such as lower risk MDS where the inflammatory cascade that’s going on in the bone marrow is a key driver of the progression of lower risk MDS. Completely new approach and it’s only been in the last really ten years where it’s been acknowledged and now commonly understood that inflammatory process is a key driver of this disease. And we’re excited to have a molecule that is active against two key signaling path pathways because we think we could have a real benefit to these patients. And I’ll show you in a minute why we still believe that there is a significant need for new treatments in this area. And frankly, I think we can provide such a treatment and the opportunity is very profound, transformational for this company.

As I mentioned earlier, we have fast tracked an orphan designation and early but a very encouraging clinical profile. We presented some data at ASH that I’ll quickly review for you today, but it’s showing that the product may have real promise in treating lower risk MDS. And more to come. So here’s the treatment landscape for lower risk MDS. Initial therapies are typical ESAs and transfusions.

Going failing that, then they move into other areas such as luspatercept or imetelstat or Ritalo. And then failing those, they use hypomethylating agents, but not approved this yet. We put little asterisk, little stars, blue stars in areas where we may proceed with this product. We have opportunities after HMAs, much smaller opportunity. The two stars, blue stars on the left are really large opportunities.

And that’s areas that we’d like to explore further. I’ll tell you what we’re doing there. So we’re only we’re beginning to focus on treatment of transfusion dependent lower risk MDS patients where frankly there’s just not enough here to treat these patients. And we think we could be a meaningful contributor to how these patients are treated. Let me tell you about the Phase 1b study that we’re in the middle of, or towards the end of at this point.

So it’s a very typical dose escalation phase study, three plus three design. We started a very low dose, dose level one. We only did three patients there because we were comfortable that really that was just too low a dose. And then the more meaningful doses are dose level two and above. At the ASH meeting in 2024, we presented data on dose level one, two, three, and four.

And we are we since then have recruited dose level five and are now recruiting dose level enrolling dose level six. At the end of this year we should have a good data set on all these dose groups. And we hope to share them at a medical conference at the end of this calendar year. Also this year what we hope to do is to pick two of those doses that look the most compelling and move them into a dose expansion phase. This could be about up to 20 patients a dose and we compare what the two doses look like now with more robust numbers.

So by the end of that expansion phase, we should have roughly 25 patients in each of those two doses where we can make some meaningful differentiation between the two and meaningful comment in terms of this is a real product here. So those are that’s coming shortly and we’ll initiate those expansion phases later this calendar year. Then after that, we will launch a registration study and we’re going to consult with FDA in terms of what that might look like and obviously share with you what that might look like. But importantly this is something that Ritalo, Luspatercept have done already and we know what to do here. We’re not reinventing the wheel here.

We know what we have to show and that is transfusion independence with a safety profile that’s acceptable. And given the mechanism, we’re confident we can show that with this product. So we’re excited about that. You see here in the little blue box in the middle, we also want to try a smaller study to show earlier patients. Are we able to have a benefit in those that have only taken an ESA and are treatment naive to everything else?

And see what the benefit is on that earlier phase as well. I think that could be a very exciting additional opportunity for us. So data’s coming at the end of this year on the dose escalation phase. We will announce that we’ve started a dose expansion phase and what the two doses there are. We will have discussions with FDA in terms of what the plan is going forward to registration and we’ll launch this additional exploratory study in the post DSA patients.

So it’s pretty a lot effort, lot of excitement around this product. We’re incredibly excited about it because we think it has such a meaningful impact on this disease in various settings. And we have importantly the ability to fund this. So you can’t get a more exciting time for the company than this very thing. Let me share with you a few other things.

Safety looks acceptable in most of the cases. AEs were diarrhea, fatigue, other things like that. We had grade three, four AEs as well. There were about two of those of platelet count decreases, pneumonia, ALT increases. You see here data treatment emergent AEs.

The 500 looks pretty clean and even the seven fifty looks pretty clean. I think you see in the footnote there, we did have increases in ALT and AST that might be a dose limiting at the seven fifty dose group. So we’re obviously taking that into account. But in general, low incidence of grade three, four side opinions and infections with the product. So we think a manageable safety profile.

And the data looks exciting. This is data we shared at the ASH of last year. But I want to highlight the two in boxes, the five hundred QD in green and the seven fifty QD in black, what we saw in each of those groups, there were five evaluable transfusion dependent patients in each of those two doses, of which we had two responders. That is about a forty percent response rate in those two patient populations. That’s a pretty good response for this patient.

And considering especially that these were highly treatment refractory populations. That is, going back to this bar here, it was patients who generally had failed luspatercept, they had failed HMA. So really at the extreme right of this. Very difficult to treat patients. But despite that we were able to show good solid responses, forty percent in those two dose groups.

So we’re really delighted by that. And we’re obviously hoping that we could achieve similarly or even better in future doses, and especially if we go into earlier patients where they’re more likely to succeed on this product. So that’s exciting. Here’s a little bit of a summary of the four responders we had. There were four of them.

Three of them were high transfusion burden patients. You see many of them elderly patients. And what’s remarkable here is the number of prior therapies that they had failed. Three of them have failed luspatercept. They failed even experimental agents.

You see fostamatinib there in one of the patients. And yet we were able to get them to good responses. One, even fifty two weeks this is the cutoff of last year we’ll have more updated data later this year. But really impressive results and impressive increases in hemoglobin level as well. Two point three to five percent grams per deciliter versus their baseline.

You want to see that as well as transfusion independence achieved. So really pleased with the results of those earlier dose groups. We’re looking for the same as we go into higher dose groups and selection of one of two of these dose groups to move forward with. Let me move on to Oluda in a couple of areas, but particularly glioma. Glioma continues to be one of the most challenging areas in treatment.

Very difficult to treat these patients. Many of them succumb. Historically, not much has worked. A couple of years ago at the ASCO meeting, vorasidenib showed very solid data, grade two, low grade glioma, that demonstrated that IDH1 inhibition may be really beneficial if you’re able to cross the BBB, the blood brain barrier. And our product is, there’s this trial here that I cite, twenty six patients with Oluda in highly treatment refractory patients.

And what we’re able to show is that we had a couple partial responses, 10 stable disease, that is disease control rate of about half the patients. That’s a really good result given how difficult to treat those patients were. Showing also that the product does obviously in patients cross the BBB and we may have success here. So what we’re doing here is two things. We’ve already launched this Connect target phase two clinical study.

So the Connect organization is conducting an umbrella study across The U. S. With studying high grade glioma. What we’ve agreed to them is that we would add Oluda as a targeted D Phase II study arm. So if those patients have IDH1 mutations they can enroll into the study and receive Oluda as a treatment in a maintenance setting.

So these are newly diagnosed patients and we’re giving the product with TMZ to start and then eventually just Oluda over the course of time. So this study is now open for enrollment. In addition, we’re planning to start a Phase II study in glioma later on ourselves and we’ll share that information with you a little bit later this year. In addition, we have put out collaboration with MD Anderson for ILUTA in various other areas, including AML. Looking at AML in first line as well as relapsedrefractory, higher risk MDS, lower risk MDS and CCUS, as well as transplant in the maintenance setting.

So some pretty exciting opportunities there that are now all open for enrollment. So let me just quickly say some of the milestones we’re looking to do. Complete the dose escalation of R289 in low risk MDS, initiate the expansion phase study, interactions with FDA leading to a registrational program, and present the data at the end of this calendar year. With ALUTA, initiate the Phase II study in Ruglioma and continue to support our collaborations with MD Anderson and CONNECT. I’ll pass on this RIP1 slide.

It’s available on our website for you to look at. Financially, we’re in really good shape. So we here we see have our quarterly results every year. Q1s are always a little bit more challenging and you generally grow out of those strongly and that’s what we expect this calendar year. Cash about $77,000,000 and we at our quarterly call restated our financial outlook.

We anticipate total revenue of 200,000,000 to $210,000,000 comprised of net product sales of 185,000,000 to 192,000,000 as well as contract revenue of 15,000,000 to $18,000,000 So continued strong growth, 30% plus over last year. And importantly, there you see on the bottom right, we continue to project positive net income this year that is being profitable, but funding all the things that I’ve just told you about. So ability to fund our programs ourselves with our own means. And I think that’s going to continue. That is our plan.

So the value drivers for this year, Grob, TAVALISSE, GAVRETO and Reslidia continue to work on adoption of those products and show strong year over year growth. I’ll skip down continued financial discipline, driving substantial cash flow to advance our development programs, particularly February in lower risk MDS, but also in other areas such as that we’re looking at even front earlier line and then Oluda in glioma as well. So and continue to look at external opportunities. And so it’s a very strong position that we’re in this year. I think a growing strong commercial business that’s generating profits and of scale now that we’re able to count on those profits on a regular basis.

Still continued financial discipline, where we’ve really kept a tight lid on our expenses. We expect to spend more on the development side as we develop these programs. And an incredible opportunity to transform the company with two eighty nine and lower risk MDS. That opportunity is tremendous and we’re going to do everything possible to expedite those clinical trials and getting the product on the market for that indication. So again, a fantastic place to be as a company with all the various components, commercial, development, financial, working together to really drive ours into the future.

So with that, I’d like to to thank you for for your interest. Thank you, Rahul. Thank you.

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