A “significant number of patients” could see a cure for multiple myeloma within the next two decades, one expert told BioSpace. Here are five therapies that could change the treatment paradigm.
The third approval under the FDA’s Commissioner’s National Priority Voucher program went to Johnson & Johnson’s multiple myeloma combo treatment Tec-Dara last week, continuing recent momentum seen in this space.
It also comes on the heels of a relatively rare occurrence in the pharma industry:
the comeback of a once-disgraced drug. GSK’s Blenrep—withdrawn from the market in November 2022—secured a new FDA approval last October as a combo regimen in the third-line setting for multiple myeloma.
With Blenrep back on the market, GSK rejoins its fellow Big Pharms in a multiple myeloma market that also includes J&J and Takeda, Pfizer and Bristol Myers Squibb.
Despite a powerhouse roster chipping away at the cancer, however, multiple myeloma remains a disease that is treated and managed, not cured, Saad Usmani, scientific advisory board member at the International Myeloma Foundation, told BioSpace in an email.
“There is growing interest now in defining how long we treat patients,” Usmani said, adding that the field is now at a place where it can consider the possibility of a cure. “This is the next phase of our research in multiple myeloma.
“I am very confident that we will be able to provide 20–25+ year disease control and potentially cure a significant number of patients in the next decade,” Usmani continued, adding that he expects to have better predictive models for prognosis and tools to monitor disease response. “We will have defined duration of therapy for patients who will be able to enjoy good quality of life.”
Last month, the FDA released new guidance aimed at getting novel drugs with early efficacy signals to patients more quickly, signaling the evolving nature of the space.
Here, BioSpace looks at some of the most promising emerging therapies—medicines that, if they prove their mettle in the clinic, could bring the industry closer to the future that Usmani envisions.
Regeneron Refines Dosing, Safety with Lynozyfic
Kicking off this list is Regeneron’s bispecific antibody Lynozyfic, which in July last year notched the FDA’s accelerated approval for the fifth line treatment of relapsed or refractory multiple myeloma.
The approval comes after an initial rejection in August 2024, though the regulator at the time found no problems with Lynozyfic’s safety or efficacy, instead citing issues with a third-party manufacturer.
Lynozyfic targets both the BCMA and CD3 proteins, in turn bridging T cells with their target: malignant B cells. Data from the Phase 1/2 LINKER-MM1 study, which formed the foundation of Lynozyfic’s regulatory package, showed a 70% objective response rate in treated patients, including a 45% complete response rate or better.
With Lynozyfic, Regeneron is a late-comer to the scene, Rajesh Kumar, functional head of Forecasting at DelveInsight, told BioSpace in an email. Still, the company has nevertheless managed to set itself apart. “Regeneron leveraged its late-mover advantage to refine dosing and safety,” according to Kumar.
Kumar also pointed to a standout element of the LINKER-MM1 study: allowing patients who hit a certain efficacy bar to switch to a monthly dosing schedule. This dosing profile, Kumar said, offers “a convenience edge over competitors’ biweekly schedules,” which in turn could drive physician and patient preference.
Lynozyfic’s accelerated approval could mean a potential $600 million revenue opportunity for Regeneron, analysts at BMO Capital Markets estimated in a July 3 note to investors. The company continues to develop Lynozyfic for multiple myeloma, looking at its potential to enter earlier—even potentially pre-malignant—treatment settings.
AbbVie, Ichnos Glenmark Go From Two Targets to Three
In July 2025, AbbVie and Ichnos Glenmark Innovation linked up to take the bispecific attack on multiple myeloma one step further, adding another cancer marker to the mix. At the heart of the collaboration is ABBV-2001, a trispecific antibody that works by helping the body recognize the malignant cells and boosting the immune system’s anti-cancer response.
For this technology, AbbVie paid $700 million upfront and promised up to $1.225 billion in development, regulatory and commercial milestones, plus royalties.
“The dual tumor-antigen targeting strategy is designed to enhance avidity even at low antigen expression while improving safety versus first-generation bispecific antibodies,” Aparna Thakur, assistant project manager of Forecasting and Analytics at DelveInsight, told BioSpace in an email, explaining that ABBV-2001 binds to BCMA and CD38 on myeloma cells, and to CD3 on T cells.
This trispecific approach is potentially the first in its class, Thakur said. “The hope is that the trispecific binding nature of this drug candidate will result in an improved clinical outcome and potentially outperform bispecific competitors.”
AbbVie and Ichnos are running the Phase 1 TRIgnite-1 study in patients with relapsed or refractory disease, which is set to complete in July 2027. Early data presented last June demonstrated a 74% overall response rate (ORR), including a 30% high-complete or stringent-complete response rate. All patients in this trial had been heavily pretreated.
The companies will release more data from TRIgnite-1 this year.
Caribou Targets ‘Paradigm Shift’ With Off-The-Shelf CAR T Therapy
For California-based Caribou Biosciences, the best answer to multiple myeloma is one that’s already been proven: CAR T therapies. Indeed, many of the biggest pharma companies have shown the efficacy of this approach, with the FDA approving products such as Bristol Myers Squibb’s Abecma and Johnson & Johnson’s Carvykti.
The question for Caribou isn’t whether CAR T is effective, CEO Rachel Haurwitz told BioSpace in an email, but about access. “Access is a major constraint” for current CAR Ts, Haurwitz continued, estimating that roughly 1 in 10 patients who need the therapy actually get it.
There are several reasons for this, she explained, but one big factor is that current CAR Ts are autologous—meaning they use cells that come from the patients themselves. Autologous cell therapies “are mostly administered in larger academic institutions,” Haurwitz said.
“Many [multiple myeloma] patients cannot wait the weeks to months necessary to receive a dose,” she added.
Autologous CAR T therapies are also more challenging to manufacture and deliver to patients due to stringent FDA requirements, according to an industry report from DelveInsight.
Caribou’s solution is CB-011, an allogeneic CAR T—meaning it can be available off-the-shelf. Instead of using cells from a patient, CB-011 takes samples from healthy donors, which then undergo genome editing using the biotech’s proprietary platform, Haurwitz explained. The result is a construct that can seek out malignant myeloma cells while also minimizing the risk of immune rejection.
Phase 1 data released last November support this mode of action. In the fourth-line-or-later setting, CB-011 resulted in a 92% ORR, including a 75% complete response rate. Safety was “manageable,” Caribou said at the time, with no cases of graft-versus-host disease—a common risk with allogeneic transplants. The biotech is enrolling patients in the dose expansion portion of this study and expects to share more data later this year, Haurwitz said.
“An allogeneic CAR-T cell therapy . . . would potentially be paradigm shifting for MM treatment,” she told BioSpace. “It would allow patients to be treated with a single dose closer to where they live, without the need to wait for manufacturing.”
C4’s Cemsidomide Picks Up MOMENTUM in 2026
2026 is shaping up to be a big year for C4 Therapeutics and its lead asset, the multiple myeloma drug cemsidomide.
Designed to be taken orally, cemsidomide is a small-molecule degrader of IKZF1/3, zinc finger transcription factors that are highly expressed in multiple myeloma and, in turn, drive the expression of genes that help the cancer cells survive and grow. Two of the most common cancer drugs today—Bristol Myers Squibb’s Revlimid and Pomalyst—similarly target and destroy these two transcription factors.
In September last year, C4 released Phase 1 clinical data for cemsidomide, touting a 50% ORR at a 100-ug dose level when used alongside dexamethasone. A lower, 75-ug dose resulted in a 40% ORR. This early-stage study focused on heavily pretreated patients with relapsed or refractory disease.
The readout also showed that degradation of IKZF1 reached over 50% after cemsidomide treatment, while IKZF3 breakdown exceeded 80%—which C4 said at the time supported the drug’s mechanism of action. The company added that cemsidomide was “generally well tolerated” across all doses tested, though there was one case of febrile neutropenia that was classified as grade 4—a severe life-threatening adverse event that needed urgent medical attention. No deaths were reported.
The Massachusetts biotech will accelerate the advancement of cemsidomide this year, with its Phase 2 MOMENTUM trial combining the drug with dexamethasone set to start this quarter. By midyear, C4 also plans to release additional Phase 1 data for cemsidomide, building up to initial MOMENTUM findings in the back half of 2027.
Exicure Wants To Make Stem Cell Transplants Easier
Exicure is taking a unique approach to multiple myeloma with cell mobilizer burixafor, aiming to facilitate smoother stem cell transplantations.
Specifically, burixafor is an oral drug that blocks CXCR4, a receptor involved in how cells move from one part of the body to another, according to the biotech’s website. By disrupting the CXCR4 signaling cascade, burixafor allows the release of stem cells from the bone marrow into the bloodstream, eventually taking them to the peripheral circulation where they can be more readily harvested.
CXCR4 has been implicated in cancer, with heightened expression linked to worse outcomes, according to Exicure.
While burixafor itself doesn’t directly address multiple myeloma, it facilitates stem cell transplantation, a relatively common treatment option for patients with this malignancy. The procedure involves bombarding the patient and their bone marrow with an aggressive regimen of chemotherapy to reduce and hopefully deplete the cancer cells there.
Then, stem cells, which were removed ahead of time—with the help of agents like burixafor—can be reintroduced into the body to rebuild the bone marrow.
Topline Phase 2 data released in December last year showed that nearly 90% of patients treated with burixafor were able to have at least 2 × 10⁶ CD34+ cells/kg harvested within two sessions of leukapheresis sessions—a procedure where white blood cells are separated from a blood sample—indicating that the drug effectively mobilized stem cells.
According to ClinicalTrials.gov, the mid-stage study should have been completed last September, though Exicure has neither released complete data for it nor outlined its development roadmap for burixafor.
