Curis at H.C. Wainwright: Emiflucertib’s Therapeutic Promise

On Wednesday, 10 September 2025, Curis Inc. (NASDAQ:CRIS) presented at the H.C. Wainwright 27th Annual Global Investment Conference. The company highlighted its strategic focus on emiflucertib, an innovative IRAK4 inhibitor, showing promise in treating lymphoma, leukemia, and solid tumors. While the company boasts strong proof-of-concept data, challenges remain in financing and competition.

Key Takeaways

• Curis is advancing emiflucertib, targeting lymphoma (NHL), leukemia (AML), and solid tumors.
• Proof-of-concept data shows significant improvement over standard care in PCNSL and AML.
• The company ended Q2 with approximately $17 million in cash.
• Curis holds a strong intellectual property position extending to 2035.
• Emiflucertib’s combination with BTK inhibitors and FLT3 inhibition shows potential for improved efficacy.

Financial Results

• Curis concluded the second quarter with roughly $17 million, including funds raised in July.
• The company maintains a robust intellectual property portfolio, with patent protection until 2035.

Operational Updates

• Emiflucertib is being developed for CLL and NHL, with a focus on achieving accelerated filing.
• The clinical program is centered on PCNSL, aiming for a quick path to approval due to its orphan drug status.
• In AML, Curis plans a head-to-head study against the current standard, gilteritinib, to demonstrate emiflucertib’s superior efficacy.
• The company is also exploring emiflucertib in solid tumors, with preliminary data expected later this year.

Future Outlook

• Curis aims to expand emiflucertib’s use across NHL, enhancing efficacy and achieving complete remission or MRD negativity.
• In AML, a registrational study is planned to establish emiflucertib as a best-in-class treatment.
• Regulatory bodies like the FDA and EMA are increasingly open to MRD as a clinical endpoint.
• Curis emphasizes the commercial potential of emiflucertib as a first-in-class and best-in-class therapy.

Conclusion

Readers are encouraged to refer to the full transcript for a comprehensive understanding of Curis’s strategic initiatives.

Full transcript - H.C. Wainwright 27th Annual Global Investment Conference:

Jim Dentzer, CEO, Curis: I’m Jim Dentzer, the CEO of Curis. Thank you for taking time today to learn more about us. Over the next twenty five minutes, I’ll walk through a summary of our progress to date. Before I begin, I’d like to remind everyone that during the presentation, we’ll be making forward looking statements, which are based on our current expectations and beliefs. These statements are subject to certain risks and uncertainties, and actual results may differ materially.

For additional details, please see our SEC filings. While Curis has a broad pipeline of novel cancer treatments, we’re focusing our efforts on emiflucertib, the first in class IRAK4 inhibitor, with broad application in lymphoma, leukemia, and solid tumors. We now have proof of concept data in thirty four patients with PCNSL, and a second proof of concept data set with twenty one patients in AML, with both studies demonstrating a significant improvement over standard of care. More to the point, we believe that these two studies represent the tip of the iceberg. The proof of concept results in PCNS-one of the toughest NHL subtypes to treat- give us confidence that it will also work in other NHL subtypes, including CLL.

Similarly, the proof of concept results in relapsed refractory AML give us confidence that it will also work in frontline AML. Cura is one of the most experienced leadership teams in biotech, with each of us having over twenty years of experience developing novel drugs at a company of Dicerna, Biogen, BMS, and Merck. The newest member of our team, doctor Ahmed Handy, brings of developing both and the number two BTK inhibitor as the CMO of Pharmaclex and also at Acerta. Ahmed is spearheading our push into CLL, and we’re very glad he chose to join the team earlier this year. This slide highlights emavutumab’s novel mechanism of actin and how it drives multiple unmet medical needs in lymphoma and leukemia.

On the right side of the graphic, we see how amyloseptide blocks both the TLR and pathways, the two pathways driving disease in AML. On the left side of the graphic, we see the BCR and TLR pathways, the two pathways driving CLL and NHL. In this setting, amivucertib is used in combination with BTK inhibitor to to enable a dual blade. BTK inhibitors block the BC pathway, and amivasertib blocks the TLR pathway. With that, let’s walk through the data that demonstrate how important this novel mechanism is.

We’ll start with the opportunity in CLL and NHL. IRAK four is a compelling target in CLL and NHL because of its ability to downregulate NF kappa b in a way that’s independent from and synergistic with standard of care. In the graphic on the left, we see that NF kappa b overactivity is what’s driving disease in NHL, And two pathways are driving that overactivity. One is the BCR pathway, which is addressed by BTK inhibitors. The other is the TLR pathway, that’s addressed by emivosertib.

We all know that BTK inhibitors work, and they can provide high response rates. But we also know that they struggle to get complete responses. We believe this is because they block one of the two pathways driving disease in CLL and NHL. In the graphic on the right, we see the preclinical data supporting this thesis. Both drugs induce tumor reduction in monotherapy.

But it is only when we combine them that we see the strongest activity and the deepest responses. When we look at the unmet need in CLL and NHL, we have to start with the understanding that everyone loves BTK inhibitors for a reason. They changed the game in NHL and have become an $11,000,000,000 industry that grew 20% last year. But BTK inhibitors have two key drawbacks. First, their inability to provide complete responses means chronic lifelong therapy for patients with the heightened risk of acquiring treatment resistant mutations.

And second, BTK inhibitors come with side effects like bruising, bleeding, fatigue, and cardiac issues that are particularly difficult for older patients. More recent therapies, like the BCL two anti CD twenty combination, which was approved in 2000 have suggested the promise of a one and done or a fixed duration treatment, which is very compelling. Unfortunately, those treatments also come with a very difficult side effect profile. And over the last six years, they’ve been unable to displace BTK inhibitors as standard of care. With emobusertib, we have a therapy that has the potential to provide the good outcome promise of BCL two and CAR T, but with an oral oral therapy and a much more attractive safety profile.

The clinical program for emifacertib in CLL and NHL was designed in anticipation of the potential for accelerated filing. In part a, we explored dose escalation across multiple NHL subtypes. In parts b and c, we’re focusing specifically on PCNS cell. Part b supports an accelerated approval path with a single arm study adding emivasertib to a patient’s BTKi regimen directly after they’ve progressed on BTKi monotherapy. And part c supports the confirmatory path with a randomized study in BTKi naive patients.

In part a, the dose escalation part of the study, we saw a clear pattern of activity across multiple NHL subtypes. Monotherapy and lower doses are active, and there’s a steady increase in activity as we go to higher doses and to combination. Among the NHL subtypes, we selected pCNSL as the subtype to pursue for our fastest path to first label because the regulatory opportunity is so clear. PCNSL is an orphan indication. It’s the hardest NHL subtype to address, and there are currently no drugs approved for it.

So in these early data, we saw an emerging pattern of activity across multiple NHL subtypes. What what then happened is we expanded into pCNSL. We see this in parts b and part c. What happened is that the emivosertib continued its discouraging pattern of anticancer activity. In the BTKi naive group, the emvocerta ibrutinib combination outperforms the data published for ibrutinib sixty three percent versus thirty nine percent.

In the BTKi experienced group, these are patients who have progressed on BTKi monotherapy, so we wouldn’t expect any tumor reduction if they stayed on it. We saw clear reduction in eleven of seventeen patients, and objective responses in seven of them. The duration of responses we can see on the right looks encouraging as well. Three of four CRs have lasted longer than six months, and one patient’s been in remission for over four hundred days, and another patient for over eight hundred. Across all programs in all of our studies.

The safety table on this slide includes specifically with NHL. Treatment was well tolerated. There’s no dose limiting myelosuppression and no DDI. As we push forward into pCNSL, the obvious next step is to expand into the other NHL subtypes wherever BTKi is used. We know BTK inhibitors work, and that they can provide high response rates, but they struggle to get complete responses.

When we add emifusertib to a BTK inhibitor, we can get complete responses. Given the clarity of emafucirteb’s mechanism and the clear pattern of activity we’re seeing even in the hardest to address NHL subtype, we look forward to expanding across NHL wherever BTK inhibitors are used. We expect to enroll our first patient in CLL by year end. As we said earlier, we look to provide the outcomes promised by b c l two and anti c d twenty, but with a better safety profile. The fifty three percent MRD potential with BCL two and anti CD twenty is compelling, but its difficult safety profile has prevented it from displacing BTKi since its 2019 approval.

Our goal is to bridge that gap, maintain the safety profile of the established $11,000,000,000 standard of care, but deepen its efficacy and enable potential enable patients to achieve complete remission or the potential for MRD negativity. From a regulatory perspective, we see the world’s changing. The FDA and EMA are increasingly receptive to MRD as a clinical endpoint. To be fair, this is a relatively recent development. But emofocertib’s potential to deepen responses, eliminate minimal residual disease, and enable time limited treatment seems well timed with where regulatory authorities want to go.

In summary, BTK inhibitors are great. They they change the game in NHL, but they come with limitations, including the need for chronic lifelong therapy and difficult side effects. With emuvaciertib, we can make the already clear market leader an even more effective therapy by combining it with emuvaciertib. And that’s the HR story. Let’s move on to AML.

As I’ve been to AML, we find that emuvocere unique molecular leak opportunity. In AML, both IRAK four and FLT three are in targets of interest, with both of them driving disease in AML. Why is this important? Let’s walk through them. In the graphic on the right, see how the pathway pathway and the FLT3 pathway drive disease in AML, and that emifusorative blocks both of them.

Let’s walk through these two pathways individually to better understand how important this novel mechanism is. First, we’ll start with IRAK four. It’s important to note that there are two isoforms of IRAK four. IRAK four short is the wild type, and IRAK four long is oncogenic. This distinction is demonstrated in the four gray boxes on the left side of the slide.

The box in the upper left is the control. The box in the upper right shows that knocking out IRAK4 stops leukemic activity. In the lower right, we see that adding back the wild type, IRAK4 short, has no effect. And in the box on the lower left, we see that adding back the oncogenic isoform, IRAK4 long, restarts leukemic activity. The red and blue graphic on the right side of the slide shows why the identification of IRAK4 long as an oncogene was so important.

These data are from Cancer Gene Atlas, and they show that IRAK four lung is expressed in nearly all patients with AML. Now we’re not suggesting IRAK four is the only driver of disease in AML. Of course not. There are a lot of drivers in AML. What we’re suggesting is that IRAK four is an important driver.

It’s expressed in nearly every patient, and the current standard of care doesn’t address it. Now let’s look at FLT3, which is mutated in a third of patients with AML, and more specifically, why combining FLT3 inhibition with IRAK4 inhibition is so effective. The graphics on this slide are from the 2019 Milgar paper, which we think does a great job of articulating why FLT3 inhibitors as a class aren’t more effective in treating AML patients who harbor FLT3 mutation. In the graphic on the left, we see that IRAK4 activity increases after treatment with a FLT3 inhibitor. In the middle graphic, we see that blocking either IRAK4 or FLT3 in monotherapy can induce tumor reduction, but the effect is temporary.

You need to combine IRAK four and FLT three to get the longer, deeper response. In the graphic on the right, we see the logical extension that blocking both IRAK four and FLT three results in extended survival. So given the strong synergy between IRAK four and FLT three, the third of the patient population with FLT three AML represents a clear opportunity for amifusertib. In the frontline setting, composite CR rates are typically around sixty percent, with fit patients receiving chemo plusminus store nor quasartanib and unfit patients receiving Venaza. The fit and unfit populations converge in the relapsedrefractory setting, where patients will typically receive gilteritinib and the expected composite CR rate drops to twenty one percent.

In our study, we targeted patients who had progressed after they had failed a FLT3 inhibitor. In the salvage line setting, we might expect that the composite CR rate would drop again, perhaps to ten percent or lower. As we now know, our data were quite a bit better than that. In the waterfall chart left, we see the single conduct swimmer plot in the middle, we see that these responses came quickly and were durable. And in the chart on the right, we see how these data compare to the current standard of care.

Even though the majority of patients treated with gilteritinib were naive to FLT3 inhibition, and the majority of patients on emivosertib had already failed on a FLT3 inhibitor. Emivosertib clearly outperformed gilteritinib, the current standard of care for patients with FLT3 AML. Just as we saw in NHL, the safety data in it all are equally encouraging. To date, we’ve dosed over two twenty five patients across all of our studies. The table on this slide includes the one hundred and two patients with AML that were treated with emivacertib.

Treatment was well tolerated, and no dose limiting myosuppression was observed. We’re currently working with key opinion leaders to design a registrational head to head study randomizing against gilteritinib. It’s a big study, so we’ll want to ensure that we have sufficient financing before we kick it off. But it’s designed to demonstrate that emifasertib is the next generation best in class treatment for all patients with FLT three AML. And that’s it for leukemia.

In short, emofasertib is a novel drug with a compelling monotherapy opportunity in a genetically defined population, with the potential to establish a new standard of care for AML patients with FLT3 mutation. We want to repeat our FLT3 AML study, this time in a head to head against gilteritinib. We think we can. As I mentioned at the end of the presentation, the primary focus of our work has been in NHL and AML, but we’re also working with several partners in academia and the NCI on the potential for emivosertib in solid tumors. In preclinical studies, emivasertib has been shown to potentiate both chemotherapy and immunotherapy in solid tumor malignancies.

Emivasertib is being evaluated in five investigator sponsored trials in solid tumors. We expect preliminary data from these studies beginning later this year, and as these data mature, we hope to gain a better sense of emifasertib’s potential in the competitive landscape and the scope of the potential opportunity in solid tumors. Now that we’ve walked through potential in NHL, AML, and solid tumors, let’s review some of the high level corporate information. Inclusive of the financing in July, we finished q two with roughly $17,000,000, and we have a strong IP position with comp of matter to 2035 before any extensions. In closing, novel targets in biotech are rare, especially ones with such wide commercial potential.

All of us at Curis are grateful for the opportunity to develop this first in class, and we believe best in class, novel therapy for patients with cancer. Thank you for your time today. We appreciate the opportunity to provide this update on our progress.

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