Today’s update from the 2025 ASH Annual Meeting highlights new and existing therapies that could improve care for patients at all stages of multiple myeloma. Read the key takeaways below, and be sure to catch up on the first day.

For a deeper dive into the most important findings from ASH, join us for our Conference Highlights Webinar on December 17. Register here.

For day 2, ASH presentations focused on existing therapies and asked two very important questions: 1) how well do they work, and 2) what can be done to improve them?

Answering this first question, new data covering the effectiveness of Isa-VRd (Sarclisa, Velcade, Revlimid, and dexamethasone) in different patient populations was presented, as well as updated data on Carvytki (cilta-cel), a CAR T-cell therapy that shows strong potential for providing long-lasting disease control for patients whose myeloma has relapsed.

To answer the second question, researchers presented on CELMoDs, a new class of oral therapies designed to enhance the immune response in myeloma. Studies showed encouraging results when CELMoDs were paired with different treatments across various stages of the disease.

New data support the use of Isa-VRd as a standard of care in newly diagnosed patients, with potential impact on quality of life

New data from a Phase 3 study suggest that Isa-VRd is more effective than Isa-Rd in treating older, high-risk NDMM patients who cannot undergo a stem cell transplant. As a reminder, Sarclisa is a monospecific antibody, similar to Darzalex.®

The study included over 200 patients aged 65–79 with high-risk disease features, such as certain genetic changes or high levels of a blood protein called β2-macroglobulin. Results showed:

• Thirty-one percent of Isa-VRd patients became MRD-negative compared to 13 percent of Isa-Rd patients.
• Isa-VRd patients were more than twice as likely to achieve MRD negativity.
• No new or unexpected side effects were reported.

Another study highlighted the impact of Isa-VRd treatment on quality of life. In over 600 newly diagnosed patients who were eligible for a stem cell transplant, Isa-VRd was linked to pain reduction, improved fatigue, and overall favorable quality of life compared to VRd alone.

IsaVRd is currently FDA-approved in the U.S. for NDMM patients not eligible for a stem cell transplant. However, some patients may access IsaVRd off-label, meaning it is used in ways not specifically approved by the FDA, based on a doctor’s judgment. Talk to your care team to see if IsaVRd may be an appropriate option for you.

Carvykti^® keeps multiple myeloma under control for longer than standard 3-drug therapy

A large, Phase 3 study concluded that a single infusion with the CAR T-cell therapy Carvykti helped keep multiple myeloma under control longer than a standard 3 drug therapy, offering promise as an earlier treatment option for patients with relapsed/refractory myeloma. In this study, Carvykti was compared to two standard regimens: PVd (Pomalyst®, Velcade®, and dexamethasone) or DPd (Darzalex®, Pomalyst®, dexamethasone). Even after 2.5 years, three out of four patients treated with Carvykti still had their disease under control, compared to less than half of those treated with standard therapies. No new side effects were reported.

These data suggest Carvykti can provide safe and lasting disease control when used earlier in patients who have relapsed.

Can CELMoDS enhance immunotherapy in early disease and late relapse?

CELMoDs, which include the drugs iberdomide and mezigdomide, are oral treatments designed to help the immune system target and attack myeloma cells. CELMoDs work in a similar way as immunomodulatory agents, such as Revlimid® or Pomalyst®, but are designed to be more potent and effective. Oral treatments offer additional benefits in that they are easy to take and are available to anyone regardless of where they live.

One essential quality of CELMoDs is their ability to “rev up” the immune response. At ASH, many studies were focused on how well CELMoDs can enhance existing therapies. Whether combined with early-line therapies like stem cell therapy (ASCT) or later-line therapies like Elrexfio (elranatamab, a bispecific antibody), researchers are exploring whether CELMoDs can be effective across different stages of the disease—helping to deepen responses, achieve no detectable disease, and provide options for long-term treatment.

While CELMoDs are not yet available for routine clinical use, researchers are actively working to better understand their safety and effectiveness for potential future approval.

• For newly diagnosed multiple myeloma patients: A Phase 2 clinical trial in 120 patients tested different doses of iberdomide as maintenance therapy after a stem cell transplant. Iberdomide helped deepen responses in many patients, with about half achieving MRD-negativity, meaning they had no detectable cancer, and over 80% remaining cancer-free after two years. The most common side effect was low white blood cell counts, which sometimes led to infections; serious side effects were uncommon. Now that researchers have identified an effective and tolerable dose, Iberdomide can now be studied in larger trials.
• For patients who have relapsed following one to three prior lines of therapies: In a Phase 2 clinical trial, 30 patients with relapsed or refractory multiple myeloma were treated with a four-drug combination of iberdomide, Kyprolis®, Darzalex® and dexamethasone. All patients had previously received 1-3 prior lines of therapies, including Revlimid®. Ninety-two percent of patients responded to 4-drug combination treatment. Importantly, these patients were able to stay on iberomide alone for long-term treatment, with regular and ongoing MRD monitoring to make sure the disease stayed under control. While some patients had low blood counts, which could increase the risk of infection, it was manageable with medication or dose adjustments.
• For patients who have relapsed following two to four prior treatments: A Phase 1b study tested a combination of elranatamab, a BCMA-targeted bispecific antibody, and oral iberdomide in 22 patients with relapsed/refractory multiple myeloma who had received two to four prior treatments. After about six months, 91% of patients responded to treatment, and 46% had a complete remission or better. Most patients had side effects, usually mild or moderate, including fatigue, low blood counts, and diarrhea. Serious side effects were less common.

Stay tuned for tomorrow’s final dispatch from ASH 2025, where we’ll bring you even more exciting updates on groundbreaking research that is shaping the future of multiple myeloma care.

The 67th ASH Annual Meeting is underway in Orlando, where more than 30,000 experts have gathered to share the newest research in multiple myeloma and other blood cancers. The MMRF team is here on the ground, connecting with leaders in the field and bringing you updates as they happen.

Over the next three days, we’ll highlight promising new research and key insights shaping the future of myeloma treatment, many of which align with MMRF’s strategic research priorities and address critical unmet patient needs.

On Day 1 of ASH, studies focused on addressing the unmet needs of high-risk patients as well as finding ways to improve existing standards of care for all patients along their journey.

To dive deeper into these findings and additional updates from ASH, read more below and join us for our Conference Highlights Webinar on December 17. Register here to hear from leading experts and stay informed on the latest advancements.

As a reminder, many of the studies being presented, particularly oral abstracts, are early in nature. While the results shared at ASH can be very strong and encouraging, much larger trials will need to be completed to confirm these initial findings, especially in patient populations that are representative of the real-world patient population in the U.S.

New CAR T Treatment Tested in High-Risk, Newly Diagnosed Patients

High-risk multiple myeloma is a more aggressive form of the disease, often marked by certain genetic changes, high levels of specific blood markers, or myeloma growing outside the bone marrow. A real-world study presented today reinforced that these high-risk features can make myeloma harder to treat and more likely to return sooner, highlighting the need for closer follow-up or additional therapy for these patients.

A small, phase 1 study presented early data on a potential new treatment for high-risk newly diagnosed patients who are not eligible for a stem cell transplant. Sixteen patients received an infusion of the CAR T-cell therapy eque-cel. All had a deep response within the first month, including complete responses and minimal residual disease (MRD) negativity, meaning no detectable myeloma using very sensitive tests. Most patients stayed MRD-negative for two years, keeping disease under control during this follow-up period.

Common side effects included CRS (cytokine release syndrome), low blood counts, and infections, but no cases of ICANS (Immune Effector Cell-Associated Neurotoxicity Syndrome) or other neurological toxicity were reported. While these results are promising, more patients and longer follow-up time are needed to confirm the benefits of eque-cel as an early treatment option for this high-risk group.

Improving Treatment Strategies for Newly Diagnosed Patients

New research shows real progress in treating people who are newly diagnosed with multiple myeloma. Three studies presented today look at different treatment approaches, including updated drug combinations, a CAR T-cell therapy that can be made in one day instead of weeks, and an immune-based treatment typically used for treating patients with relapsed/refractory disease.

• KRd leads to more complete and deeper responses than VRd in NDMM patients
  For newly diagnosed patients who may not be able to tolerate four drug treatments, what is the best way to ensure three drug combinations are still effective? One Phase 3 study of 250 patients compared two common triplet combinations for newly diagnosed multiple myeloma patients to see what works better: KRd (Kyprolis®, Revlimid®, dexamethasone) vs VRd (Velcade®, Revlimid®, dexamethasone). Both combinations worked well, but KRd led to more complete and deeper responses than VRd (see table below). This means that M protein levels were absent from the blood and plasma cells were not detectable in the bone marrow.

Side effects were similar between the two treatments: serious side effects occurred in about 7 in 10 patients on KRd and 6 in 10 on VRd, the most common of which were low white blood cell counts and infections. Patients treated with KRd had fewer nerve-related problems than VRd, but slightly more heart-related effects, while patients treated with VRd had more neuropathy than patients treated with KRd.

• “Next-day” CAR T-cell therapy shows strong results and may open door for shorter wait times
  A Phase 1 clinical trial tested a new CAR T-cell therapy for newly diagnosed patients that targets two proteins on the surface of myeloma cells—BCMA and CD19—instead of just one. All patients responded, and 97% had a complete response. While CRS was common side effect, it was manageable with treatment, and no patients had any serious nerve-related side effects. Importantly, this CAR T-cell therapy can be made overnight, rather than the typical timeframe of weeks seen with other CAR T therapies, potentially making the treatment available to those who need it sooner.
• Bispecific treatments brought in earlier along the patient journey
  With the hope of making therapeutic responses last longer, bispecific antibody treatments used for treating relapsed/refractory patients are now being tested as an alternative to traditional chemotherapy and stem cell transplant for newly diagnosed patients:
  • One such treatment is Lynozyfic.™ In an early Phase 2 study, all 14 people treated had a deep response, and everyone tested was MRD-negative at six months. Side effects of Lynozyfic included infections, rash, bone pain, or low blood counts, which were mild for most patients.
  • Another is a combination of Tecvayli^® and Darzalex.^® Both Tecvayli and Darzalex are FDA-approved for patients with relapsed/refractory multiple myeloma; Darzalex is also approved for newly diagnosed patients when used in certain combination treatments. In a Phase 2 study of 37 newly diagnosed multiple myeloma patients ages 65 and older patients received this “all-antibody” combination as their initial therapy. Seventy-eight percent had a very good partial response or better after four treatment cycles, and eventually all patients responded. More than half (51%) had no detectable myeloma six months after starting treatment, and no patients experienced disease worsening. Common side effects included low blood counts and infections, which were mostly mild to moderate.

New CAR T-cell therapy shows promise in treating relapsed/refractory patients with limited options

A Phase 2 study tested anito-cel, a CAR T-cell therapy designed to more easily attach to myeloma cells using a small “hook”—a feature that may help reduce certain side effects seen with other CAR T-cell therapies. Key findings from 117 patients included:

• 97 percent responded, and 68 percent reached a complete remission or better, usually within about one month.
• Most patients remained cancer-free at the one-year mark.
• The most common side effects were low blood counts and mild to moderate cytokine release syndrome, which typically resolved quickly. Serious infections and neurological problems were uncommon.

These data suggest that anito-cel may offer strong, long-lasting control of myeloma, with manageable side effects, even for people whose disease has stopped responding to many other treatments.

We’re hopeful about the progress shared today at ASH—and we’ll be back tomorrow with more updates for the myeloma community.

People living with multiple myeloma often relapse after several lines of therapy. When this happens, it can feel like options are running out. The recent approval of Lynozyfic (linvoseltamab-gcpt) is an important step forward in expanding treatment option for patients.

Lynozyfic was approved in July 2025 for adult patients with relapsed or refractory multiple myeloma who have received at least four prior lines of therapy.^[1] Here are some important details about Lynozyfic:

• Lynozyfic is a bispecific antibody—a type of treatment that helps cells in your immune system find and attack myeloma cells.
• Lynozyfic works in a way that’s similar to other bispecific treatments like Tecvayli and Elrexfio—they all target a protein called BCMA, which is found on myeloma cells. This makes it easier for the immune system to recognize and attack those cancer cells.
• Bispecific antibodies like Lynozyfic are generally more accessible, because they don’t require the individualized manufacturing process or lengthy preparation time associated with CAR T-cell therapy.

A clinical trial with myeloma patients who had received at least four prior treatments showed that 70% of them responded to Lynozyfic treatment. Of these individuals:

• Half of the patients responded within 1 month of treatment
• For 70%, the response has continued for a year (see figure below).

^[1] Including a proteasome inhibitor (such as Velcade, Kyprolis, or Ninlaro), an immunomodulatory agent (such as Revlimid or Pomalyst), and an anti-CD38 monoclonal antibody (such as Darzalex or Sarclisa).

Lynozyfic is given through an intravenous infusion:

• Treatment starts with two step-up doses, which are small doses that gradually increase
  • After each step-up dose, you’ll have to stay in the hospital for 24 hours to be monitored for side effects
• After you’ve received the step-up doses, you’ll receive the standard dose once a week for a total of 12 doses without any hospital stay
• After the first 12 full doses, you’ll receive one full dose every 2 weeks for 5 weeks
• If you’re responding, your doctor may decide to drop your treatment to once every 4 weeks; if not, your treatment will continue on the every-2-weeks schedule

Like many myeloma treatments, Lynozyfic can cause side effects. The most common is cytokine release syndrome, which can cause fever, chills, or trouble breathing. If these signs occur, it’s important to report them to your care team right away.

Several options are available for avoiding or reducing side effects. Neurologic problems—in particular, immune effector cell-associated neurotoxicity syndrome (ICANS)—can also occur, but they are not as common as CRS.

Because of the side effect risks, Lynozyfic is only available through a special safety program. If you receive Lynozyfic, you’ll be given a wallet card to remind you and your care providers of what to watch for.

If you’re a caregiver for a myeloma patient, make sure the patient carries their wallet card and watch the patient for symptoms. If any develop, report them to the care team right away.

As with any myeloma treatment, it’s important for you and your caregiver to have open conversations with your care team about whether Lynozyfic is a good fit. If you’ve already been through several treatments, Lynozyfic offers a new chance at controlling your myeloma.

Interested in learning more about Lynozyfic? Talk to an MMRF patient navigator at the Patient Navigation Center at 1.888.841.MMRF (Monday through Friday, 9:00 AM to 7:00 PM ET) or email patientnavigator@themmrf.org.

Additional Resources

• Regeneron. Lynozyfic (linvoseltamab-gcpt) Patient Support.
• MMRF. Fast Facts in Myeloma. Bispecific Antibodies for Multiple Myeloma: What You Need to Know.
• MMRF. MMRF Patient Toolkit. Immunotherapy.
• MMRF. MMRF Patient Toolkit. Multiple Myeloma Treatment Overview.
• MMRF. MMRF High-Impact Topic Video. Bispecific Antibodies.

Less than a year after enrolling its first patient, the Horizon One clinical trial already has important wins to celebrate: Not only is it pioneering important research for patients with relapsed and refractory multiple myeloma, but it’s enrolling a patient population that’s far more representative of the disease than other trials to date.

The Horizon Clinical Trials program, which is run by the MMRF’s Multiple Myeloma Research Consortium^® (MMRC^®), is innovative by design, starting with the fact that it’s aiming to optimize treatments for patients with significant unmet need. Horizon One is enrolling patients with relapsed and refractory multiple myeloma, while the forthcoming Horizon Two trial will enroll high-risk, newly diagnosed patients.

Unlike a traditional clinical trial, Horizon is what’s known as an adaptive platform clinical trial. This unique design allows investigators to test several treatments at the same time and has built-in flexibility to add or remove therapies in real time—all with the goal of generating data and insights at an accelerated pace.

Speed is especially important for the patients in Horizon One. While there are currently more than 15 FDA-approved drugs for the disease, the devastating reality is that most patients will relapse when the disease becomes resistant to a therapy. Others may eventually become refractory (unresponsive) to many kinds of treatment.

“This patient population needs more options, and they need them fast,” said the MMRF’s Chief Medical Officer Hearn Jay Cho, MD, PhD. “These patients and their providers still face complex questions about what treatments will be most effective and in what order. Horizon is designed to answer those critical questions that other types of trials can’t.”

Horizon is innovating in another way: by enrolling a study population that is representative of multiple myeloma patients across the United States. In doing so, the Horizon trial can generate new insights and answer questions that impact all patients.

Historically, clinical trials in multiple myeloma have not been as inclusive.

About 20 percent of multiple myeloma patients in the U.S. are Black, but those patients have been significantly underrepresented in clinical trials. In fact, one analysis of multiple myeloma trials conducted over a 13-year period found that only four percent of enrolled patients were Black.

The average age of a multiple myeloma diagnosis is 69, and two-thirds of people diagnosed with the disease are 65 years or older. But studies have found that these patients are also left out of research. One analysis found that patients over the age of 70 are half as likely to enroll in multiple myeloma clinical trials, and another found that nearly two-thirds of trial participants over a 16-year period were under the age of 65, not at all representative of the disease’s true age demographic.

But the MMRC’s Horizon trial is working to reach the broadest myeloma population—and succeeding.

As of now, more than 25 percent of patients enrolled in Horizon One identify as Black. Seventy-one percent of patients are 71 or older, with more than 20 percent between 81 and 90 years old, percentages that are rarely, if ever, seen in other myeloma trials.

Horizon One is also working to enroll across a mix of geographical locations, including in rural areas and at 25 community hospitals and clinics. In fact, the MMRC Horizon One trial recently opened the first-ever myeloma clinical trial in Flint, Michigan.

Meeting these impressive goals has required considerable collaboration that the MMRC—with its vast network of 13 major medical centers and researchers and a leadership team with decades of expertise and industry connections—is uniquely positioned to facilitate.

Working closely with the MMRC’s Health Equity Advisor Dr. Craig Cole of Karmanos Cancer Institute, the Horizon team has surveyed MMRC sites, collecting data and insights about why patients enroll and, importantly, why they don’t. Dr. Cole and the MMRC team have also brought all 13 MMRC sites together to share learnings and wins.

“Clinical trials have been exclusionary to older people, people of color, and people who live far from academic medical centers. We can enroll those populations by knowing why patients don’t consent to the clinical trial,” Dr. Cole said. “We’re making cultural change. The MMRC sites are sharing stories and ideas and thinking outside the box about how we can improve what we’re doing.”

Many of the trial’s wins, like the launch in Flint, came out of simply having intimate, one-on-one conversations out in the community.

“I did a church basement talk in Flint at an African American church, and I gave my awareness talk about what myeloma is,” Dr. Cole said. “And this little old lady said, ‘You need to bring clinical trials to us. I don’t want to hear about myeloma and all your fancy drugs that you give in Detroit.’ I took it back to the MMRC and said, ‘A little old lady told me to bring a clinical trial to Flint, and I think we really need to do that.’ And the MMRC leadership made that happen. Now, that little old lady can go down the street, get her bispecific, go home, and hang out with her grandchildren.”

Understanding that cures don’t come out of silos, the MMRF and MMRC have a long history of making their learnings and data available to the wider multiple myeloma field. Looking forward, the Horizon trial investigators plan to share their experiences and findings for other researchers, pharma, academic medicine, and others to learn from.

“We’re so proud to conduct trials like Horizon that will help us better understand the disease across a representative population and regardless of where someone lives,” said MMRF’s Senior Vice President of Clinical Research and Regulatory Affairs Katie Wozniak. “That’s how we’ll improve care and find cures for everyone diagnosed with multiple myeloma.”

The MMRF is excited to share that the FDA has approved Blenrep (belantamab mafodotin) combinations for multiple myeloma patients who have relapsed and have received at least two prior lines of treatment. The announcement was eagerly anticipated, especially after the FDA took some time to weigh the data presented at July’s Oncology Drug Advisory Committee (ODAC).

This is encouraging news for our community. As an IV infusion like Sarclisa or Kyprolis, Blenrep provides an additional off‑the‑shelf treatment option that can help more people access care from their doctor’s office or cancer center. Also, unlike CAR T or bispecific therapy, patients can begin treatment without hospitalization needed to manage certain side effects like CRS or ICANS.

What to Expect with Blenrep

Clinical trial data showed how safe and effective Blenrep (in combination with Velcade and dexamethasone) was for patients compared to Darzalex, Velcade, and dexamethasone. Here were the highlights:

• Improved responses: Adding Blenrep to standard treatment helped patients live almost three times longer without their cancer getting worse.
• Manageable side-effects: The most common serious side effect with Blenrep was eye-related problems (such as blurred vision or dry eyes), which were usually manageable with dose changes, eye drops, or treatment breaks, but often required close monitoring. Other side effects seen were typical for myeloma therapies (like low blood counts and tiredness), and doctors monitored patients regularly to help manage these issues.

Next Steps for Patients

Now that Blenrep is available, it is important to have a conversation with your care team if this treatment could be a viable option for you. Here are some questions to ask that help start the discussion:

• How does Blenrep compare with the other treatments I’m considering?
• What side effects should I watch for, especially related to my eyes, and how will we monitor and manage them throughout treatment?
• How often will I need to come in for infusions and eye exams, and what will my schedule look like day-to-day?
• Will this treatment affect my daily activities, like driving, working, or traveling, and what support resources are available?

In addition to your care team, the MMRF Patient Navigation Center can offer support in answering these questions and give one‑on‑one guidance tailored for  you. The MMRF Education Hub also offers additional materials to help better understand treatment options and navigate you along your myeloma journey.

In late 2024, the MMRF launched the Horizon Clinical Trials Program through the Multiple Myeloma Research Consortium^® (MMRC), a collaborative research network of leading myeloma treatment centers. Horizon uses an innovative trial design called an adaptive platform, which makes it possible to test multiple therapies at the same time to determine the best combination, sequence, and duration of treatment to get the best outcomes for patients. The adaptive design also allows researchers to work together and move faster than they can in a traditional clinical trial model.

For the first trial within the Horizon program (Horizon One), we are enrolling patients with relapsed and refractory myeloma across 13 sites to study dosing approaches with Tecvayli^® (teclistamab), a bispecific antibody treatment. The second platform trial of the program, Horizon Two, is scheduled to launch soon and enroll high-risk, newly diagnosed patients.

Horizon Two will reimagine how we treat high-risk newly diagnosed multiple myeloma. We spoke with Dr. Sham Mailankody of Memorial Sloan Kettering (MSK) Cancer Center about what patients can expect — and why this moment marks a turning point in myeloma research.

Dr. Sham Mailankody, Memorial Sloan Kettering (MSK) Cancer Center

Why do people with high-risk myeloma need different treatment options?

Over the past 25 years, treatments for multiple myeloma have improved a lot, especially for patients with what we call “standard-risk” disease. But people with high-risk myeloma haven’t seen those same benefits. About 1 in 4 newly diagnosed patients fall into this high-risk category, and unfortunately, these patients either do not respond to their initial therapy, or relapse within two years — whereas most patients are in remission for at least four or five years after their first therapy.

What is the Horizon Two trial, and how is it different from other studies?

Horizon Two is a national clinical trial created specifically for people who are newly diagnosed with high-risk myeloma. In the past, this group was often left out of research. The trial is designed to be flexible and patient-friendly. It allows multiple treatment options to be tested at once, and new ones can be added over time. Even patients who have already started their first cycle of treatment may still qualify. This approach helps us learn faster and include more people in the process.

What kinds of treatments are being tested in Horizon Two?

We’re looking at advanced treatments called immunotherapies — including CAR T-cell therapy and bispecific antibodies. These therapies have already worked well for patients with advanced or relapsed myeloma. Now we’re testing whether using them earlier, right after diagnosis, might lead to stronger and longer-lasting responses.

Will the trial consider how treatment affects patients’ quality of life?

Absolutely. Some treatment arms in the trial will explore time-limited therapy, meaning patients could stop treatment after a certain amount of time if they’re doing well. We’re also collecting direct feedback from patients and advocates to guide future parts of the study. Better treatment doesn’t just mean controlling the disease — it means improving how patients feel and live.

How is Horizon Two making it easier for more people to take part in research?

Horizon Two is available at many locations across the country — not just at large academic hospitals, but also at regional and community centers that work closely with myeloma experts. For example, patients don’t have to travel to Manhattan to take part in the trial at MSK; there may be a site closer to home. This setup helps reduce travel time and costs, making it easier for people, including those in rural or underserved areas, to access cutting-edge research and care.

How important is collaboration in making a study like this happen?

It’s absolutely necessary. Horizon Two is the result of years of collaboration through the MMRF and MMRC. It brings together top cancer centers, patient advocates, drug companies, and government regulators. Everyone plays a role: from designing the trial to enrolling patients. This kind of teamwork is what makes real progress possible. 

What does success look like for Horizon Two?

Our biggest goal is to close the gap in outcomes between high-risk and standard-risk patients. If we can do that, we may one day no longer need the term “high-risk.” That would be a huge step forward for more effective treatments and for a more equitable future for everyone living with myeloma. 

Between declining government funding for research and a difficult drug development arena marked by higher costs, greater risk, and more competition for less capital, multiple myeloma is at a crossroads. Cures are within reach, but to realize them, the community needs to confront these challenges head-on—something the Multiple Myeloma Research Foundation (MMRF) is uniquely poised to do. 

One of the most important ways we’ll push the field forward now is through the Myeloma Investment Fund (MIF), our venture philanthropy arm. Over the last six years, the MIF has helped advance six treatments to clinical trials, invested $23 million in 20 companies, and generated a two-to-fourfold return on its initial investment in three of those companies.  

Now, the MIF is scaling up when myeloma needs it most and will soon become a self-sustaining, evergreen fund— reinvesting all the returns it generates into new companies and ideas.  

The MIF is the first and only mission-driven philanthropic venture fund focused on accelerating a cure for multiple myeloma. The MIF provides biotechnology companies with financial and strategic support, access to data, and connections to investors, researchers, and pharma representatives.  

Not only that, but the MIF leverages the MMRF’s vast expertise and its more than 25 years at the forefront of myeloma research.  

“We join the boards of many of these companies and play an important advisory role,” said MIF Managing Director Stephanie Oestreich, PhD, MPA. 

The companies the MIF invests in are developing some of the most transformative potential treatments in myeloma—with a special focus on relapsed patients who have run out of options. 

“An estimated 70 percent of new therapeutic targets are being developed by venture-backed companies, and only 30 percent are with pharma,” Oestreich said. “Venture capital like the kind the MIF provides is critical to move tomorrow’s innovations forward.”  

MIF investments provide funding at a pivotal point in the drug development process, when early breakthroughs are at risk of stalling because they lack funding or direction. By stepping in at this phase, the MIF helps bridge the gap between research and real-world application.  

“At a time when early-stage funding is harder to secure than ever, the MIF’s early support is essential—helping companies attract more investors and ensure that the best new ideas in myeloma don’t get left behind,” the MMRF’s President and Chief Executive Officer Michael Andreini said.  

MIF-backed companies are working on some of the most exciting new technologies and developments today: next-generation CAR T-cell designs and treatments (such as in vivo CAR T, which engineer T-cells in a patient’s body instead of a lab), natural killer cell–directed therapies, antibody-drug conjugates (which have revolutionized treatment of other cancers), and more. Our portfolio also includes companies investigating novel ways to improve patients’ immune response to better recognize, attack, and kill myeloma cells.  

The companies the MIF invests in are also highly regarded. In fact, in September, two such companies—Umoja and Stylus Medicine—made Endpoints’ annual list of the 11 most exciting biotech startups of 2025. Endpoints is one of the most prestigious news outlets covering biopharma, and its annual Endpoints 11 list is highly anticipated in the field—representing “the best and the brightest in biotech.”   

Andrew Scharenberg, MD, the co-founder and CEO of Umoja Biopharma, which the MIF invested in at the start of 2025, said that having the backing of the MIF and MMRF is crucial in more ways than one.  

“Because the MIF and MMRF are trusted by doctors and scientists, their support helps open doors,” he said. “When others see that the MIF believes in what we’re doing, it gives them confidence that our work is promising and based on solid science. That support helps us move faster and bring new treatments to patients sooner.” 

The final day of IMS 2025 brought a powerful close to the meeting, underscoring how far the field has come in advancing personalized care for myeloma patients. From promising new drugs to smarter ways of tailoring treatment using biomarkers, today’s presentations made it clear: The future of myeloma therapy is not just about more options—it’s about better-matched options.

Researchers are now focusing on how to deliver the right treatment to the right patient at the right time, using tools like genetic profiling and immune markers to guide decisions.

Read on for what stood out to us.

New Treatments on the Horizon for Patients at All Stages of Disease

Day four showcased a pipeline of innovative treatments poised to transform myeloma care, from precision-targeted therapies that could redefine treatment standards to enhanced delivery methods that are more convenient for patients.

• On-body-injector for Sarclisa (isatuximab): For myeloma patients, having options that reduce discomfort and fit more easily into daily life is critical. In a phase 2 and phase 3 trial, patients with relapsed or refractory disease tried Sarclisa given through a small wearable injector instead of the traditional IV drip. Surveys looking at quality of life were given to these patients, who reported less pain, less discomfort, fewer side effects than expected, and meaningful time savings compared to IV treatment. Overall, more patients were satisfied with the on-body injector and said they would recommend it to others.
• Iberdomide: Iberdomide is part of a new class of drugs called CELMoDs. CELMoDs are related to IMiDs like Revlimid and Pomalyst, helping to boost the immune system’s ability to fight myeloma. Like IMiDs, they are also pills. In small clinical trial of 75 newly diagnosed patients who were ineligible for transplant, a combination of the oral CELMoD iberdomide with Darzalex (daratumumab) and dexamethasone (IberDd) led to responses in about 95% of people treated. Over 2 years, more patients in this study began to experience deeper responses. Side effects of the IbderDd regimen were manageable, and included infections, diarrhea, and rashes.
• Sonrotoclax: Sonrotoclax is a new oral drug designed to target a specific genetic change called t(11;14) that is found in approximately 20% of patients, and is similar to Venclexta (venetoclax), which is sometimes used off-label for patients with this genetic feature. In a phase 1/2 trial of 50 patients with relapsed/refractory myeloma, 81% responded to a combination of sonrotoclax and dexamethasone. Responses were often seen within three weeks and lasted over a year for many patients.
• HDP-101: HDP-101 is a new anti-BCMA antibody-drug conjugate (ADC) that delivers a powerful toxin directly to myeloma cells while sparing other healthy cells. In a phase 1/2 study of 42 relapsed/refractory patients, HDP-101 was generally safe, especially when given in split doses with premedication to reduce side effects like low platelets and liver changes. There was no evidence of eye-related side effects due to the drug; eye-related side effects are a common side effect with belantamab mafodotin, which is also an anti-BCMA ADC. About 50% of patients who were treated at higher doses responded to this therapy. Additional studies are underway to determine the dose that best balances safety and effectiveness. In this study, patients had already gone through about 7 different therapies, with some trying as many as 15. Even after many relapses, having new treatment options available is very encouraging. It means there is still hope and progress for patients who need more choices.

Strengthening Maintenance Therapy

As doctors work to improve myeloma treatments, researchers are studying whether using more than one treatment for maintenance therapy can help lower the chance of relapse. In a phase 3 study, patients who took a combination of Darzalex and Revlimid for two years did much better at keeping myeloma to very low, almost undetectable levels, compared to patients who took Revlimid alone. Just as important, the side effects were mild and very similar to what patients usually experience with Revlimid by itself. As discussed on day 3, these studies are especially important for patients who may have a higher risk of their disease returning quickly.

The Increasingly Important Role of Biomarkers

During the last day of IMS, we also heard updates on the growing role of biomarkers—biological signs in the body that help doctors better understand each patient’s type of myeloma. These discoveries build on earlier research from the MMRF’s CoMMpass^SM study, which is one of the most important studies ever conducted to better understand new targets for drug development and define which patients are more likely to have aggressive disease. Researchers across the field continue to leverage CoMMpass data.

• Biomarkers to predict risk: Earlier this year, experts introduced a new way to define high-risk multiple myeloma, a more aggressive form of the disease that is harder to treat. Two studies shared today showed that this new definition is not only accurate in predicting worse outcomes among high-risk patients, but it more effective than traditional staging systems, providing strong support for the use of this new definition.
• Biomarkers to guide treatment planning: Biomarkers are also helping doctors choose the best treatments for each patient. Several presentations focused on using biomarkers to predict how well a patient might respond to newer treatments like CAR T-cell therapy and bispecific antibodies, including who may be more likely to experience certain side effects. There’s also progress in using blood-based tests for monitoring, which could reduce the need for more invasive procedures like bone marrow biopsies.

These updates reflect a powerful shift in how myeloma is being treated—from relying solely on standard drug regimens to using a deeper understanding of each patient’s unique disease to guide care. As more targeted therapies and biomarker-driven tools move closer to routine use, the path forward looks increasingly personalized, offering new hope for better outcomes and a higher quality of life for patients at every stage of their journey.

Day three of IMS 2025 brought into focus three transformative trends reshaping multiple myeloma care: emerging effective strategies for early relapse, making powerful treatments more accessible to patients, and bringing later-line therapies to earlier stages of treatment.

Here, the MMRF recaps what stood out from today’s sessions.

Experts Highlight Strategies for Managing Early Relapse

The first plenary session of the day brought several myeloma experts together to outline key considerations for managing early relapse.

• Patients and their care teams should keep a close eye on myeloma markers in the blood (like M protein levels), and, where possible, access testing for minimal residual disease in the bone marrow. Don’t wait for other symptoms to reappear. To optimize outcomes, patients should begin treatment when these biochemical markers begin to rise again.
• Quadruplet therapies and combination maintenance therapy are key to helping patients with a high risk of relapsing.
• When deciding what is next after relapse, patients and providers must consider treatment logistics and access. With so many options now available in early relapse—from triplet therapies to immunotherapies like CAR T—shared decision-making is more important than ever whereby patients and their care teams evaluate the potential benefits and risks of available options.
  
• In the next few years, bispecific therapies will continue to make their way into clinical trials as earlier lines of treatment, even in high-risk newly diagnosed patients. The MMRF’s Horizon II platform is helping to lead this initiative.

These discussions signal a future where earlier intervention, innovative therapies, and collaborative decision-making can significantly improve outcomes for patients facing relapse.

Expanding Access to Bispecifics

Bispecific antibody therapies are typically given in hospital settings because they come with the risk of complications like cytokine release syndrome (CRS), a common, flu-like side effect of T cell-engaging treatments that can cause fevers, chills, and low blood pressure. Because of this, access to these powerful treatments is more limited.

We were excited to hear data at IMS showing that bispecific antibody therapies can safely be given in the community setting, without requiring a hospital stay, enabling more patients to greater access.

• Several studies, including a large real-world study of more than 200 patients, found that step-up dosing of Tecvalyi (teclistamab) and Talvey (talquetamab) can be safely given in outpatient or community clinics. Step-up dosing means slowly increasing the dose to help lower the risk of serious side effects like CRS. With careful monitoring and a clear care plan, side effects in outpatient settings were comparable to those in the hospital—showing that CRS can be managed safely by an outpatient team outside the hospital.
• Equally as exciting was another real-world study showing that relapsed/refractory patients who had a strong response to bispecific antibody therapy—targeting BCMA, GPRC5D, or both—were able to stop continuous treatment. The study analyzed 78 patients who had stopped treatment (not because of disease worsening) and were closely monitored. Nearly 70% of these patients stayed in remission for at least two years. Factors that lead to high risk of relapse included presence of extramedullary disease (myeloma outside the bone marrow) and a higher number of previous therapies.

Bringing Later Line Therapies Upfront

With numerous promising treatments now available for multiple myeloma, determining the optimal timing and sequence of therapies has become increasingly important to improve outcomes and patients’ quality of life. This has been a research focus at the MMRF through our innovative Horizon Clinical Trials Program conducted in collaboration with 14 leading cancer centers.

We were excited to hear and share research updates from our colleagues who are similarly examining the potential of giving treatments to newly diagnosed patients or patients with precursor disease that have been shown to be highly effective in relapsed/refractory patients.

• A Phase I clinical trial of 37 newly-diagnosed patients showed that a combination of the bispecific Elrexfio (elranatamab), Revlimid +/-Daralex may be effective for those not eligible for a stem cell transplant. In this study, 97% responded to the treatment, and most had deep responses within just a few months. Side effects like low blood counts and infections were common but manageable, and most cases of CRS were mild.
• A Phase 2 trial of 74 newly-diagnosed patients who are ineligible for a transplant may benefit from Sarclisa and Velcade, Revlimid, and dexamethasone (Isa SC-VRd). Isa SC is unique in that it is delivered beneath the skin through a special a device worn on the belly. The Isa SC treatment regimen that has shown already promise in clinical trials patients with relapsed/refractory myeloma. After eight months, 88% of patients who received Isa SC-VRd had a very good response or better, with 24% achieving complete remission, and 35% showing no detectable disease.
• Lastly, the newly approved bispecific treatment Lynozyfic (Linvoseltamab) was recently tested in high-risk smoldering multiple myeloma patients. While this study was small (only 19 patients who completed at least one treatment cycle), all of them responded and have not progressed to active myeloma. Side effects (such as infections and CRS) were manageable and did not lead to any treatment discontinuations.

Updates on New Treatments for Patients with Relapsed/Refractory Myeloma

We conclude by sharing exciting updates on arlo-cel, a type of CAR T-cell therapy that targets GPRC5D. We shared promising results from a phase 1 study on the first day of IMS. Today, researchers presented data from a second study that showed similarly encouraging results. In this phase 1 study of 31 relapsed/refractory patients who had received one to three prior lines of therapy, 96% responded to arlo-cel. Side effects—including CRS, loss of taste, and nerve-related symptoms—were common but mostly mild and resolved on their own.

Day three’s presentations demonstrate how myeloma care is evolving to become more accessible and strategically timed—bringing us closer to more personalized strategies that maximize both effectiveness and patients’ quality of life.

Saturday marks the final day of the IMS 2025 meeting. Stay tuned for highlights from the concluding sessions.

MMRF continues to drive the critical global research collaborations that lead to scientific breakthroughs and improved patient outcomes

Norwalk, Conn., September 20, 2025 – Today, the Multiple Myeloma Research Foundation® (MMRF) announced that data generated from several MMRF initiatives were presented at the prestigious 22nd International Myeloma Society (IMS) Annual Meeting. The MMRF team presented five posters, while new findings from its Immune Atlas and CoMMpass℠ programs underpinned 18 additional presentations, reflecting the organization’s unique ability to drive key discoveries, collaborative research, and innovative clinical trials in multiple myeloma.

“Year after year, the MMRF leads research that accelerates the pace of discovery and directly translates into better treatments for patients with multiple myeloma,” said MMRF Chief Scientific Officer George Mulligan, PhD. “Insights from MMRF-led programs are powered by our large clinical and genomic datasets as well as our innovative clinical trial platforms. These demonstrate how our collaborative, data-driven approach is fueling discoveries to deepen our understanding of multiple myeloma.”

The novel MMRF-led research presented at IMS 2025 demonstrates the impact of the foundation’s flagship programs and illustrates how the MMRF’s approach to integrating research within trials addresses key questions that other trials cannot. Among the highlights:

• New reports from the Immune Atlas program include a podium presentation describing how the immune system changes during multiple myeloma treatment.
• The MyDRUG℠ study—a precision medicine trial that selected treatment to match patients’ specific tumor mutations—showed how quadruplet therapy, including daratumumab, reshapes T cell populations in the bone marrow. This highlights the importance of immune function for current therapies.

In addition, the MMRF presented two reports highlighting the positive impact of the organization’s patient education and navigation programs. These initiatives empower people diagnosed with multiple myeloma to optimize their care, better communicate with their healthcare teams, and improve their outcomes.

Abstracts Presented by MMRF
Title: Daratumumab-based Quadruplet Therapy in Functional High-risk Relapse/Refractory Multiple Myeloma Patients Induces Changes Associated with CD8 T Cell Activation and Expansion in the Immune Microenvironment
Abstract: PA-204

Title: CoMMpass Explorer: An Interactive Platform to Explore Clinico-Genomic Data from Newly Diagnosed Multiple Myeloma Patients from the CoMMpass Observational Trial
Abstract: PA-205

Title: Exploration of Tumor Immune Gene Expression to Stratify Progression Risk in Newly Diagnosed Multiple Myeloma Patients from CoMMpass With 1q Cytogenetic Abnormality
Abstract: PA-230

Title: Assessing Positive Behavioral Change in Myeloma Care: An MMRF Longitudinal Study
Abstract: PA-111

Title: Navigating Myeloma: Behavioral and Emotional Outcomes from a Patient-Centered Navigation Resource
Abstract: PA-112

Abstracts on MMRF Immune Atlas Program
Title: Longitudinal Multi-Omic Profiling Uncovers Immune Escape and Predictors of Response in Multiple Myeloma
Abstract: OA-73

Title: Integrating Microenvironment with Tumor Multi-Omic using Unsupervised Machine Learning to Model Heterogeneity Refines Multiple Myeloma Subtypes and Reveals Immune-Based Clusters with Prognostic Impact
Abstract: PA-234

Title: Ancestry-Associated Dysregulation of the Bone Marrow T Cell Compartment in Multiple Myeloma Revealed via scRNA-seq and CyTOF Profiling
Abstract: PA-254

Title: Multi-hit Cytogenetics Risk Events Associated with IFN-I Suppression across TME of NDMM Patients and Poor Outcomes
Abstract: PA-255

Title: Elevated Levels of Circulating Tumor Cells (CTCs) in Newly-Diagnosed Multiple Myeloma (NDMM) Patients Reflect a Highly Proliferative and Genomically Complex Profile
Abstract: PA-231

About Multiple Myeloma
Multiple myeloma is a cancer of the plasma cells that develops in bone marrow. This year, an estimated 35,750 new cases of multiple myeloma will be diagnosed—making it the second-most common blood cancer in the U.S.—and 12,590 people will die from the disease. New targeted agents and therapies have resulted in better outcomes, but most multiple myeloma patients eventually relapse.

About the Multiple Myeloma Research Foundation (MMRF)
The Multiple Myeloma Research Foundation (MMRF) is the largest nonprofit in the world solely focused on accelerating a cure for each and every multiple myeloma patient. We drive the development and delivery of next-generation therapies, leverage data to identify optimal and more personalized treatment approaches, and empower myeloma patients and the broader community with information and resources to extend their lives. Central to our mission is our commitment to advancing access so that all myeloma patients can benefit from the scientific and clinical advances we pursue. Since our inception, the MMRF has raised over $600 million for research, opened nearly 100 clinical trials, and helped bring 15+ FDA-approved therapies to market, which have tripled the life expectancy of myeloma patients. To learn more, visit www.themmrf.org.

Media Contact:
Adam Silverstein, Scient PR: adam@scientpr.com