CAR T-Cell Therapy for Acute Lymphoblastic Leukemia (ALL) - Market Insight, Epidemiology and Market Forecast - 2032

Key Highlights

• The main treatment options for ALL include chemotherapy, targeted therapy, immunotherapy, surgery, radiation therapy, and stem cell transplant.
• In the last few years, immunotherapy has undergone a new phase of development which is linked to the development of CAR-T cell therapy. It is anticipated that CAR-T cells will bring out the next big leap forward in leukemia immunotherapy. In 2017, the US FDA made a historic decision by approving KYMRIAH (tisangenlecleucel), the first-ever CAR-T cell therapy for the treatment of acute lymphoblastic leukemia. In addition, later in October 2021, the FDA approved another CAR-T cell therapy, TECARTUS (brexucabtagene autoleucel) for adult patients 26 years of age and above with R/R B-cell precursor ALL.
• CAR-T cells provide a long-term benefit with a one-time treatment, avoiding the toxicity of salvage chemotherapy and autologous transplant for patients with high-risk illnesses. The approvals have altered the standard of care for high-risk patients who are either primary refractory or have early recurrence following front-line therapy.
• In 2021, the market size of CAR-T in ALL in the 7MM was approximately USD 75 million, which is further expected to increase by 2032.
• In the 7MM, the US accounted for the largest market size in 2021, with approximately USD 55 million.
• Among the emerging CAR-Ts, obecabtagene autoleucel is anticipated to be the first to enter the market among the upcoming therapies, giving it a competitive edge over other emerging assets.
• Although most clinical studies use autologous CAR-T cells for B-ALL treatment, the administration of allogeneic CAR-T cells has been also reported in a small and limited number of clinical studies. Key players such as Precision Biosciences (PBCAR0191) Cellectis (UCART22), Wugen (WU-CART-007), and Cellectis/Servier/Allogene (UCART19) are currently testing their allogeneic products.
• CAR-T cell therapies need more data supporting their use in earlier lines of therapy. Patients are likely to have better outcomes if they receive CAR-T cell therapy before multiple lines of chemotherapy, like those in the clinical trials that led to FDA approval. Some trials are being designed to establish the efficacy and safety of CAR-T cell therapy in earlier lines. Novartis is expecting the submission schedule of KYMRIAH in first-line high-risk ALL pediatrics and young adult patients in =2026.

The “CAR-T in ALL-Market Insight, Epidemiology and Market Forecast - 2032” report delivers an in-depth understanding of ALL, historical and forecasted epidemiology as well as the CAR-T in ALL market trends in the United States, EU4 (Germany, France, Italy, and Spain), and the United Kingdom, and Japan.

The CAR-T in ALL market report provides current treatment practices, emerging CAR-Ts, market share of individual CAR-Ts, and current and forecasted 7MM CAR-T in ALL market size from 2019 to 2032. The report also covers current treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market’s potential.

Geography Covered

• The United States
• EU4 (Germany, France, Italy, and Spain), and the United Kingdom
• Japan

Study Period: 2019-2032

CAR-T in ALL Understanding and Treatment Algorithm

CAR-T in ALL Overview

Acute lymphocytic leukemia (ALL), also known as acute lymphoblastic leukemia, is a type of cancer that affects the blood and bone marrow. It starts from young white blood cells called lymphocytes in the bone marrow; mainly characterized by an overproduction of immature white blood cells, called lymphoblasts or leukemic blasts. Because the bone marrow is unable to make adequate numbers of red cells, normal white cells, and platelets, people with ALL become more susceptible to anemia, recurrent infections, and bruising and bleeding easily. The blast cells can then spill out of the bone marrow into the bloodstream and accumulate in various organs including the lymph nodes or glands, spleen, liver, and central nervous system (brain and spinal cord). Tests and procedures used to diagnose ALL include blood tests, bone marrow tests, imaging tests, and spinal fluid tests.

The treatment options for ALL include chemotherapy, post-remission therapy (consolidation and maintenance therapy), targeted therapy, immunotherapy, and CAR-T cell therapy. In addition to this, stem cell transplant is also used early in therapy for patients with high-risk subtypes of ALL.

CAR-T in ALL Epidemiology

As the market is derived using a patient-based model, the CAR-T in ALL epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total incident cases of ALL, gender-specific cases of ALL, age-specific cases of ALL, subtype-specific cases of ALL, genetic-mutation specific cases of ALL, and total treated cases of ALL in the 7MM covering the United States, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan from 2019 to 2032.

• Among the 7MM, the US accounted for the highest number of incident cases of ALL.
• The total incident cases of ALL in the US comprised approximately 6,200 cases in 2021 and are projected to increase during the forecast period.
• Amongst EU4 and the UK, Germany accounted for the highest number of incident cases of ALL, while Spain accounted for the lowest number of cases.
• Among the gender-specific cases, males accounted for nearly 3,500 cases, while females accounted for 2,700 in the US in 2021.
• Among the type-specific cases of ALL, the incident cases of B-ALL accounted for nearly 85%, while that of T-ALL accounted for nearly 15% in the US.

CAR-T in ALL Drug Chapters

The drug chapter segment of the CAR-T in ALL report encloses a detailed analysis of marketed and late-stage (Phase III and Phase II) CAR-Ts. It also helps understand the CAR-T in ALL pivotal clinical trial details, recent and expected market approvals, patent details, advantages and disadvantages of each included drug, the latest news, and recent deals and collaborations.

Marketed Drug

TECARTUS (brexucabtagene autoleucel): Gilead Sciences

TECARTUS is a CD19-directed genetically modified autologous T cell immunotherapy, which binds to CD19-expressing cancer cells and normal B cells. Studies demonstrated that following anti-CD19 CAR-T cell engagement with CD19-expressing target cells, the CD28 and CD3-zeta costimulatory domains activate downstream signaling cascades that lead to T cell activation, proliferation, acquisition of effector functions, and secretion of inflammatory cytokines and chemokines. This sequence of events leads to the killing of CD19-expressing cells. In October 2021, the US FDA approved TECARTUS for adult patients with R/R B-ALL. It was approved by the EC in September 2022.

KYMRIAH (tisagenlecleucel): Novartis

KYMRIAH is a CD19-directed genetically modified autologous T cell immunotherapy that involves reprogramming a patient’s T cells with a transgene encoding a chimeric antigen receptor (CAR) to identify and eliminate CD19-expressing malignant and normal cells. The CAR is comprised of a murine single-chain antibody fragment that recognizes CD19 and is fused to intracellular signaling domains. Upon binding to CD19-expressing cells, the CAR transmits a signal to promote T cell expansion, activation, target cell elimination, and persistence of the KYMRIAH cells. In August 2017, the US FDA approved KYMRIAH for the treatment of patients up to age 25 years with R/R B-ALL. It was approved by the EC in August 2018, and by the MHLW in March 2019.

Emerging Drug

Obecabtagene autoleucel (obe-cel): Autolus Therapeutics

Obe-cel is an autologous CD19 CAR-T cell therapy with a unique CD19 CAR. The CAR is designed to have a fast-off kinetic, which mimics physiological T-cell receptor interactions. Autolus Therapeutics recently announced that the FELIX Phase II trial of obe-cel in R/R adult ALL has continued to progress well, and the company is on track to report initial results from the trial in the fourth quarter of 2022. In March 2022, obe-cel was granted Orphan Medical Product Designation by the EMA for the treatment of ALL, having previously received ODD by the US FDA for B-ALL. In April 2022, the US FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to obe-cel for the treatment of adult B-ALL. Obe-cel also received PRIME designation from the EMA and Innovative Licensing and Access Pathway (ILAP) by the Medicines and Healthcare Products Regulatory Agency (MHRA), United Kingdom.

UCART22: Cellectis

UCART22 is an allogeneic-engineered T-cell product candidate designed for the treatment of CD22-expressing hematologic malignancies and is currently being developed for the treatment of B-ALL. Currently, the company is evaluating the drug in Phase I/II BALLI-01 Study in patients with R/R ALL. As of the December 2021 report, the company is enrolling patients in the third dose level of the BALLI-01 Study with a fludarabine cyclophosphamide alemtuzumab lymphodepletion regimen. In December 2021, the company presented preliminary results from this Study at the American Society of Hematology annual meeting.

CAR-T in ALL Market Outlook

Traditional cytotoxic chemotherapy-containing regimens have been the backbone of treatment for adults with ALL for decades. Common complications of traditional chemotherapy can be fatal and include infection, bleeding, thrombosis, neuropathy, osteonecrosis, and the development of secondary cancers, including AML and MDS.

Since the first successful report of CAR-T cell therapy for pediatric ALL more than a decade ago, the field has exploded with new constructs and targets, insights into CAR T-cell persistence, and novel antigen escape mechanisms; first came KYMRIAH (tisagenlecleucel, or tisa-cel, Novartis) for pediatric and young adult patients who had received at least two prior lines of therapy, followed by TECARTUS (brexucabtagene autoleucel, or brexu-cel, Kite) for adults following first relapse. It is anticipated that CAR-T cells will bring out the next big leap forward in leukemia immunotherapy.

Obecabtagene autoleucel (obe-cel; AUTO1), a novel, second-generation CAR product, is currently the most advanced candidate in the CAR-T in the ALL emerging pipeline. CD19 negative relapse is a major cause of treatment failure after CD19 CAR T-cell therapy for pediatric B-ALL. To address this, Autolus has designed AUTO1/22 to target both CD19 and CD22 using the fast-off rate CD19 CAR from obecabtagene autoleucel (obe-cel) combined with a novel CD22 CAR capable of effective signaling in response to low antigen density.

Several companies advancing allogeneic CAR-T cell therapies in ALL include key players such as Precision Biosciences (PBCAR0191), Cellectis (UCART22), Wugen (WU-CART-007), and Cellectis/Servier/Allogene (UCART19), and others.

• The total market size of CAR-T in ALL in the 7MM was approximately USD 75 million in 2021 and is projected to increase during the forecast period (2023-2032).
• Amongst EU4 and the UK, Germany accounted for the largest market size in 2021, while Spain occupied the bottom of the ladder.
• Among the therapies, TECARTUS is expected to generate the highest revenue in the 7MM by 2032.

CAR-T in ALL Uptake

This section focuses on the rate of uptake of the potential CAR-Ts expected to be launched in the market during the study period 2019-2032. The analysis covers market uptake by CAR-Ts; patient uptake by CAR-Ts; and sales of each CAR-T in ALL.

CAR-T in ALL Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III and Phase II. It also analyzes key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for CAR-T in ALL emerging therapies.

KOL Views

To keep up with current market trends, we take KOLs and SMEs’ opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts contacted for insights on CAR-T in ALL evolving treatment landscape, patient reliance on conventional therapies, patient’s therapy switching acceptability, and drug uptake, along with challenges related to accessibility, including Medical/scientific writers; American Cancer Society; Hematologist and Professors; MD, FACS, Chair of the Department of Department of Hematology, University of Texas MD Anderson Cancer Center, and others.

The analysts connected with 30+ KOLs to gather insights; however, interviews were conducted with 10+ KOLs in the 7MM. Their opinion helps understand and validate current and emerging therapy treatment patterns or CAR-T in ALL market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Qualitative Analysis

We perform Qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and Analyst views. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst’s discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

The analyst analyzes multiple emerging therapies based on relevant attributes such as safety, efficacy, target population, and order of entry.

In efficacy, the trial’s primary and secondary outcome measures are evaluated. Further, the therapies’ safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed, and it sets a clear understanding of the side effects posed by the drug in the trials.

Market Access and Reimbursement

Reimbursement is a crucial factor affecting the drug’s market access. Often, the decision to reimburse comes down to the price of the drug relative to the benefit it produces in treated patients. To reduce the healthcare burden of these high-cost therapies, payers and other industry insiders are considering many payment models. Understanding insurance and out-of-pocket costs shouldn’t be overwhelming.

Patients whose healthcare professionals have prescribed TECARTUS therapy can work with Kite Konnect. This integrated technology platform provides information and assistance throughout the therapy process for Kite’s commercialized CAR-T therapies, including courier tracking for shipments and manufacturing status updates. Kite Konnect provides support for eligible patients receiving TECARTUS, and it includes information for the healthcare teams supporting their patients.

The report further provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenario of approved therapies, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report

• The report covers a segment of key events, an executive summary, and a descriptive overview of CAR-T in ALL, explaining its causes, signs, symptoms, pathogenesis, and currently used CAR-T therapies.
• Comprehensive insight into the epidemiology segments and forecasts, disease progression, and treatment guidelines has been provided.
• Additionally, an all-inclusive account of the emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the CAR-T in the ALL market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM therapy outreach.
• The report provides an edge while developing business strategies, by understanding trends, through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM of CAR-T in ALL.

CAR-T in ALL Report Insights

• Patient Population
• Therapeutic Approaches
• CAR-T in ALL Pipeline Analysis
• CAR-T in ALL Market Size and Trends
• Existing and Future Market Opportunity

CAR-T in ALL Report Key Strengths

• Ten Years Forecast
• The 7MM Coverage
• CAR-T in ALL Epidemiology Segmentation
• Key Cross Competition
• Conjoint Analysis
• Drugs Uptake and Key Market Forecast Assumptions

CAR-T in ALL Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs

• What was the CAR-T in ALL Market share (%) distribution in 2019, and what would it look like in 2032?
• At what CAGR is the CAR-T in ALL market expected to grow in the 7MM during the study period (2019-2032)?
• Among the emerging CAR-Ts, which potential CAR-T is expected to disrupt the ALL markets?
• What will be the impact on the sales of TECARTUS and KYMRIAH after the entry of its generics into the market?
• What are the disease risks, burdens, and unmet needs? What will be the growth opportunities across the 7MM concerning the patient population?
• Which emerging CAR-T is going to garner the maximum market share in the 7MM in 2032?
• What are the various recent and upcoming events expected to improve the uptake of CAR-T in ALL?
• How much market share will be captured by CAR-Ts by 2032?
• What are the current guidelines for treating ALL using CAR-Ts in the US, Europe, and Japan?
• What are the recent novel therapies, targets, mechanisms of action, and technologies being developed to overcome the limitations of existing therapies?
• How is the access to CAR-Ts among different countries?

Reasons to buy

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the CAR-T in ALL.
• Insights on patient burden/disease, incidence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Distribution of historical and current patient share based on real-world prescription data along with reported sales of approved products in the US, EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• To understand the future market competition in the CAR-T in ALL market.
• Highlights of access and reimbursement policies of current therapies, barriers to accessibility of expensive off-label therapies, and patient assistance programs.
• To understand Key Opinion Leaders’ perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.

Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.