CLINICAL TRIALS PROFILE FOR CARVYKTI

All Clinical Trials for CARVYKTI

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT06185751 ↗	Safety and Efficacy of CS1 CAR-T (WS-CART-CS1) in Subjects With Multiple Myeloma	Not yet recruiting	Paula C. & Rodger O. Riney Blood Cancer Research	Phase 1	2024-06-30	Despite recent therapeutic advances, multiple myeloma (MM) remains an incurable disease. Although survival has improved, there are nevertheless diminishing durations of response to each subsequent line of therapy. This highlights the need for further therapeutic innovation. BCMA-targeting CAR-T cells show impressive response rates; however, their median duration of response is disappointing. The investigators propose that CS1(SLAMF7)-targeting CAR-T cells will fill a gap in the MM armamentarium. CS1 is an attractive target in MM because it is expressed in most patients. Elotuzumab (Empliciti®), an approved anti-CS1 antibody, has proven the clinical efficacy of this target. CAR-T cells are an ideal modality to target CS1, given that two approved treatments, ide-cel (idecabtagene vicleucel, AbecmaTM) and cilta-cel (ciltacabtagene autoleucel, Carvykti™), have proven the potential for cellular immunotherapy in MM. The investigators are testing the safety and preliminary anti-myeloma efficacy of WS-CART-CS1, a CAR-T cell therapy targeting CS1.
NCT06185751 ↗	Safety and Efficacy of CS1 CAR-T (WS-CART-CS1) in Subjects With Multiple Myeloma	Not yet recruiting	Washington University School of Medicine	Phase 1	2024-06-30	Despite recent therapeutic advances, multiple myeloma (MM) remains an incurable disease. Although survival has improved, there are nevertheless diminishing durations of response to each subsequent line of therapy. This highlights the need for further therapeutic innovation. BCMA-targeting CAR-T cells show impressive response rates; however, their median duration of response is disappointing. The investigators propose that CS1(SLAMF7)-targeting CAR-T cells will fill a gap in the MM armamentarium. CS1 is an attractive target in MM because it is expressed in most patients. Elotuzumab (Empliciti®), an approved anti-CS1 antibody, has proven the clinical efficacy of this target. CAR-T cells are an ideal modality to target CS1, given that two approved treatments, ide-cel (idecabtagene vicleucel, AbecmaTM) and cilta-cel (ciltacabtagene autoleucel, Carvykti™), have proven the potential for cellular immunotherapy in MM. The investigators are testing the safety and preliminary anti-myeloma efficacy of WS-CART-CS1, a CAR-T cell therapy targeting CS1.
NCT06623630 ↗	Lymphodepleting Total Body Irradiation (TBI) Plus Cyclophosphamide Prior to Ciltacabtagene Autoleucel (Carvykti; Cilta-cel) for Multiple Myeloma (MM) Patients With Impaired Renal Function	RECRUITING	American Cancer Society, Inc.	PHASE1	2024-12-04	Treatment for relapsed/refractory multiple myeloma continues to evolve with the approval of highly effective anti-BCMA CAR T therapies in recent years. However, despite the high prevalence of renal insufficiency in this population, pivotal clinical trials have excluded patients with impaired renal function, leading to an urgent, unmet clinical need to develop safe and effective lymphodepleting regimens prior to CAR T administration for this population. In addition, renal insufficiency is linked to poor disease-related outcomes and is highly associated with several underserved populations. This study is testing the hypotheses that: 1. low-dose total body irradiation (TBI) in combination with cyclophosphamide (Cy) as lymphodepletion prior to administration of cilta-cel will be safe and tolerable in patients with multiple myeloma who have impaired renal function 2. low-dose TBI-Cy as lymphodepletion prior to cilta-cel will result in comparable CAR T expansion/persistence and disease response rates as those seen with standard lymphodepleting chemotherapy (fludarabine / cyclophosphamide).
NCT06623630 ↗	Lymphodepleting Total Body Irradiation (TBI) Plus Cyclophosphamide Prior to Ciltacabtagene Autoleucel (Carvykti; Cilta-cel) for Multiple Myeloma (MM) Patients With Impaired Renal Function	RECRUITING	Cures Within Reach	PHASE1	2024-12-04	Treatment for relapsed/refractory multiple myeloma continues to evolve with the approval of highly effective anti-BCMA CAR T therapies in recent years. However, despite the high prevalence of renal insufficiency in this population, pivotal clinical trials have excluded patients with impaired renal function, leading to an urgent, unmet clinical need to develop safe and effective lymphodepleting regimens prior to CAR T administration for this population. In addition, renal insufficiency is linked to poor disease-related outcomes and is highly associated with several underserved populations. This study is testing the hypotheses that: 1. low-dose total body irradiation (TBI) in combination with cyclophosphamide (Cy) as lymphodepletion prior to administration of cilta-cel will be safe and tolerable in patients with multiple myeloma who have impaired renal function 2. low-dose TBI-Cy as lymphodepletion prior to cilta-cel will result in comparable CAR T expansion/persistence and disease response rates as those seen with standard lymphodepleting chemotherapy (fludarabine / cyclophosphamide).
NCT06623630 ↗	Lymphodepleting Total Body Irradiation (TBI) Plus Cyclophosphamide Prior to Ciltacabtagene Autoleucel (Carvykti; Cilta-cel) for Multiple Myeloma (MM) Patients With Impaired Renal Function	RECRUITING	Washington University School of Medicine	PHASE1	2024-12-04	Treatment for relapsed/refractory multiple myeloma continues to evolve with the approval of highly effective anti-BCMA CAR T therapies in recent years. However, despite the high prevalence of renal insufficiency in this population, pivotal clinical trials have excluded patients with impaired renal function, leading to an urgent, unmet clinical need to develop safe and effective lymphodepleting regimens prior to CAR T administration for this population. In addition, renal insufficiency is linked to poor disease-related outcomes and is highly associated with several underserved populations. This study is testing the hypotheses that: 1. low-dose total body irradiation (TBI) in combination with cyclophosphamide (Cy) as lymphodepletion prior to administration of cilta-cel will be safe and tolerable in patients with multiple myeloma who have impaired renal function 2. low-dose TBI-Cy as lymphodepletion prior to cilta-cel will result in comparable CAR T expansion/persistence and disease response rates as those seen with standard lymphodepleting chemotherapy (fludarabine / cyclophosphamide).

Clinical Trial Conditions for CARVYKTI

Condition Name

Condition Name for CARVYKTI
Intervention	Trials
Multiple Myeloma	2

Condition MeSH

Condition MeSH for CARVYKTI
Intervention	Trials
Multiple Myeloma	2
Renal Insufficiency	1
Neoplasms, Plasma Cell	1

Clinical Trial Locations for CARVYKTI

Trials by Country

Trials by Country for CARVYKTI
Location	Trials
United States	2

Trials by US State

Trials by US State for CARVYKTI
Location	Trials
Missouri	2

Clinical Trial Progress for CARVYKTI

Clinical Trial Phase

Clinical Trial Phase for CARVYKTI
Clinical Trial Phase	Trials
PHASE1	1
Phase 1	1

Clinical Trial Status

Clinical Trial Status for CARVYKTI
Clinical Trial Phase	Trials
Not yet recruiting	1
RECRUITING	1

Clinical Trial Sponsors for CARVYKTI

Sponsor Name

Sponsor Name for CARVYKTI
Sponsor	Trials
Washington University School of Medicine	2
Paula C. & Rodger O. Riney Blood Cancer Research	1
American Cancer Society, Inc.	1

Sponsor Type

Sponsor Type for CARVYKTI
Sponsor	Trials
Other	5

Clinical Trials Update, Market Analysis, and Projection for CARVYKTI (Ciltacabtagene Autoleucel)

Last updated: November 4, 2025

Introduction

CARVYKTI (ciltacabtagene autoleucel), a groundbreaking CAR T-cell therapy developed by Janssen Biotech, represents a significant advancement in the treatment landscape for relapsed or refractory multiple myeloma (RRMM). Since its FDA approval in February 2022, CARVYKTI has demonstrated remarkable efficacy, positioning itself as a potentially transformative therapy for a heavily pretreated patient population. This article provides a comprehensive update on ongoing clinical trials, analyzes its current market positioning, and projects future growth based on recent developments.

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Clinical Trials Update

FDA Approval and Initial Data

In February 2022, the U.S. Food and Drug Administration (FDA) approved CARVYKTI based on pivotal phase 1/2 trial data from the CARTITUDE-1 study. The trial encompassed patients with RRMM who had previously received at least four lines of therapy, including immunomodulatory drugs, proteasome inhibitors, and anti-CD38 monoclonal antibodies. Results showed an impressive overall response rate (ORR) of 98%, with stringent complete response (sCR) rates reaching 80% [1].

Ongoing Clinical Trials

Post-approval, Janssen has accelerated its clinical development to evaluate CARVYKTI across broader and more diverse patient populations.

• CARTITUDE-4: This pivotal phase 3 trial compares CARVYKTI with standard therapies in newly diagnosed multiple myeloma (NDMM) patients, aiming to expand the label beyond refractory cases. The trial's primary endpoint is progression-free survival (PFS). Results are anticipated in 2024, which could potentially broaden CARVYKTI’s indication if positive [2].

• CARTITUDE-6: Another phase 3 study focuses on earlier lines of therapy in high-risk smoldering multiple myeloma (SMM), exploring whether early intervention can alter disease trajectory. While preliminary, early data suggest a favorable safety profile and promising efficacy signals [3].

• Long-term Follow-up Studies: Janssen continues to monitor durability and safety, with data extending to 24 months and beyond, demonstrating sustained responses with manageable toxicity profiles. The therapy exhibits a median progression-free survival exceeding 18 months in top responders, consistent with initial trial findings [1].

Safety Profile and Managing Toxicities

CARVYKTI's safety profile aligns with other CAR T-cell therapies, with cytokine release syndrome (CRS) and neurotoxicity being the primary adverse events. The majority of CRS cases are grade 1–2, managed effectively with supportive care and tocilizumab. Neurotoxicity is less common but warrants close monitoring during the critical response window. No new safety concerns have emerged in follow-up studies [4].

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Market Analysis

Market Landscape

Multiple myeloma remains a challenging hematologic malignancy, with over 34,000 new diagnoses annually in the U.S. alone [5]. Despite advancements, relapse rates remain high, especially after third-line therapies. CAR T-cell therapies like CARVYKTI address an unmet need for durable remissions in refractory populations.

Competitive Environment

CARVYKTI’s primary competitors include:

• Abecma (idecabtagene vicleucel) by Bristol-Myers Squibb and Celgene; FDA-approved in March 2021, it is the first CAR T approved for RRMM.
• J&J’s CC-93269 (ciltacabtagene autoleucel’s close relative), authorized under accelerated approval pathways.

While both therapies demonstrate high response rates (>70%), CARVYKTI’s higher ORR (98%) and deeper responses position it as a front-runner. However, differences in manufacturing, safety profiles, and dosage regimens influence clinician preferences.

Market Penetration and Adoption

Initial uptake has been robust, driven by superior efficacy data and favorable safety profile. Janssen leverages its established hematology sales force to promote CARVYKTI, focusing on specialized centers with expertise in cellular therapy. Challenges include logistical hurdles related to autologous cell collection and manufacturing lead times, typically 3–4 weeks. Efforts are underway to streamline logistics and broaden access.

Pricing and Reimbursement

With a list price of approximately $475,000 per treatment cycle, CARVYKTI reflects the high costs associated with personalized cellular therapies. Payers are increasingly adopting value-based agreements to mitigate financial risk, often contingent on patient response and durability. This pricing position aligns with other CAR T products, justified by significant clinical benefits.

Regulatory and Commercial Expansion

Beyond the U.S., CARVYKTI has received approvals or submissions in Europe, Japan, and other regions. Regulatory agencies are scrutinizing evidence from ongoing trials, with potential approvals expanding its global footprint. Janssen’s strategic partnerships and investments aim to accelerate access in emerging markets.

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Market Projection

Short-term Outlook (1–2 Years)

In the near term, CARVYKTI’s sales are expected to grow steadily, fueled by:

• Expanded indications, including potential approval for earlier lines of treatment following positive phase 3 results.
• Continued adoption in key centers with established cellular therapy programs.
• Increasing awareness among clinicians and payers.

Projected sales forecasts estimate revenue in excess of $1 billion globally by 2024, with the U.S. market comprising approximately 70% of this figure due to higher diagnosis rates and reimbursement structures.

Medium to Long-term Outlook (3–5 Years)

Assuming positive trial outcomes, CARVYKTI’s indications could expand to newly diagnosed and high-risk smoldering patients, significantly enlarging its market size.

• The subsequent approval in earlier lines could increase addressable patient population by approximately 50%, potentially reaching 15,000–20,000 eligible patients annually in the U.S. alone.
• Innovations in manufacturing—such as allogeneic “off-the-shelf” CAR T products—are projected to reduce costs and expand access, further boosting sales.
• Competitive pressures and new pipeline entrants will influence market shares; however, CARVYKTI’s demonstrated efficacy offers a strong competitive advantage.

Risks and Opportunities

While market growth projections are optimistic, risks include:

• Manufacturing delays impacting supply.
• Emergence of additional therapies (bispecific antibodies, novel CAR T constructs).
• Reimbursement hurdles or pricing pressures.

Conversely, opportunities involve advancing combination therapies, personalized approaches, and regulatory approvals across multiple geographies.

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Conclusion

CARVYKTI stands out as a potent therapeutic for relapsed or refractory multiple myeloma, underpinned by robust clinical data and expanding trial programs. Its competitive edge derives from high response rates, manageable safety, and ongoing efforts to broaden indications. Market projections indicate significant growth potential, driven by expanding patient access, pipeline development, and integration into standard treatment algorithms.

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Key Takeaways

• Clinical advancements: Ongoing phase 3 trials aim to expand CARVYKTI’s use into newly diagnosed and early-stage settings.
• Market positioning: It holds a competitive edge through superior response data and a manageable safety profile.
• Growth potential: sales are projected to surpass $1 billion globally by 2024, with significant upside pending positive trial outcomes and broader approvals.
• Operational challenges: manufacturing logistics and reimbursement strategies remain critical factors influencing adoption.
• Innovation prospects: future pipeline developments and combination therapies will shape its long-term market trajectory.

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FAQs

1. What distinguishes CARVYKTI from other CAR T-cell therapies?
CARVYKTI exhibits a high overall response rate (98%) and deep responses in heavily pretreated multiple myeloma patients, with a manageable safety profile that supports broader clinical use.

2. When are phase 3 trial results expected for CARVYKTI in earlier lines of therapy?
Results from the CARTITUDE-4 trial, evaluating CARVYKTI in newly diagnosed multiple myeloma, are anticipated in 2024, potentially enabling expanded indications.

3. How does manufacturing affect the availability of CARVYKTI?
Manufacturing complexity involves autologous cell collection and processing within a 3–4 week window, impacting treatment initiation timelines and logistical coordination.

4. What are the main safety concerns associated with CARVYKTI?
Cytokine release syndrome (CRS) and neurotoxicity are the primary adverse events, but both are generally manageable with established protocols.

5. What is the future outlook for CARVYKTI in the global market?
Global approval efforts and pipeline innovations could extend its reach, with emerging allogeneic CAR T options poised to address current logistical and cost barriers.

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Sources:

1. Janssen Biotech. (2022). FDA approves CARVYKTI for multiple myeloma.
2. ClinicalTrials.gov. (2023). CARTITUDE-4 phase 3 trial details.
3. Janssen Press Release. (2023). Updates on CARTITUDE-6 trial and early data.
4. Food and Drug Administration. (2022). CARVYKTI safety profile summary.
5. Globocan 2020. (2020). International data on multiple myeloma incidence.