Category Archives: Stem Cell Therapy

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The information made available in the Stem Cell Therapy report will definitely facilitate to increase the knowledge and decision-making skills of the business, thus providing an immense opportunity for growth. This will at last increase the return rate and drive the competitive edge within. Being a custom market report, it provides services tailored to the exact challenge. Whether it is survey work, in-depth interviewing, or a combination of multiple methods, marketing report will match the right methodology and personnel to the business need. Proficient team of analysts gather, analyze, and synthesize the data to accomplish challenging tasks while not setting unrealistic expectations.

The report focuses on the major players that are in operation within the market and their competitive landscape present in the market. The report includes an inventory of initiatives taken by the businesses within the past years. The report has mentioned growth parameters in the regional markets along with major players dominating the regional growth such as North America, Europe, China, Japan, Southeast Asia and India.

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Stem Cell Therapy Market Scenario

The report throws light on the competitive landscape, segmentation, geographical expansion, and revenue, production, and consumption growth of the Stem Cell Therapy market. Stem Cell Therapy Market Size, Growth Analysis, Industry Trend, and Forecast, offers details of the factors influencing the global business scope. This report provides future products, joint ventures, marketing strategy, developments, mergers and acquisitions, marketing, promotions, revenue, import, export, CAGR values, the industry as a whole, and the particular competitors faced are also studied in the large-scale market.

Stem Cell Therapy Market competitive landscape provides details by competitor. Details included are company overview, company financials, revenue generated, market potential, investment in research and development, new market initiatives, production sites and facilities, company strengths and weaknesses, product launch, product trials pipelines, product approvals, patents, product width and breath, application dominance, technology lifeline curve. The data points provided are only related to the companys focus related to Stem Cell Therapy market. Leading global Stem Cell Therapy market players and manufacturers are studied to give a brief idea about competitions.

The Following Manufacturers Are Covered In This Report:

Gilead,Novartis,Organogenesis,Vericel

Global Stem Cell Therapy Market Segmented By:

Product Type: Adult Stem Cells, Human Embryonic Stem Cells (hESC), Induced Pluripotent Stem Cells, Very Small Embryonic Like Stem Cells

Application: Regenerative Medicine, Drug Discovery and Development

Latest news and industry developments in terms of market expansions, acquisitions, growth strategies, joint ventures and collaborations, product launches, market expansions etc. are included in the report. The report focuses on the operation and their competitive landscape present within the market. Identification of numerous key players of the market will help the reader perceive the ways and collaborations that players will need to understand the competition within the global Stem Cell Therapy market.

Stem Cell Therapy Market report provides depth analysis of the market recent developments and comprehensive competitive landscape created by the COVID19/CORONA Virus pandemic. Stem Cell Therapy Market report is helpful for strategists, marketers and senior management, And Key Players in Stem Cell Therapy Industry.

Market Dynamics Of Stem Cell Therapy Market

Global Stem Cell Therapy market report has the best research offerings and the required critical information for looking new product trends or competitive analysis of an existing or emerging market. Companies can sharpen their competitive edge again and again with this business report. The report comprises of expert insights on global industries, products, company profiles, and market trends. Users can gain unlimited, company-wide access to a comprehensive catalog of industry-specific market research from this industry analysis report. The market report examines industries at a much higher level than an industry study.

Table of Content: Global Stem Cell Therapy Market Research Report

Chapter 1: Global Stem Cell Therapy Industry Overview

Chapter 2: Global Economic Impact on Stem Cell Therapy Market

Chapter 3: Global Market Size Competition by Industry Producers

Chapter 4: Global Productions, Revenue (Value), according to Regions

Chapter 5: Global Supplies (Production), Consumption, Export, Import, geographically

Chapter 6: Global Productions, Revenue (Value), Price Trend, Product Type

Chapter 7: Global Market Analysis, on the basis of Application

Chapter 8: Stem Cell Therapy Market Industry Value Chain

Chapter 9: Stem Cell Therapy Market Chain, Sourcing Strategy, and Downstream Buyers

Chapter 10: Strategies and key policies by Distributors/Suppliers/Traders

Chapter 11: Key Economic Indicators, by Market Vendors

Chapter 12: Market Effect Factors Analysis

Chapter 13: Global Stem Cell Therapy Market Forecast Period

Chapter 14: Future Of The Market

Chapter 15: Appendix

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Substantial research & development activities carry out by some players that comprises offering training to covering recent information on new technology, materials and techniques to innovative practice solutions, will complement the market growth is also explained. Frequent technological advances, superior portability, and ease of handling for Stem Cell Therapy are boosting adoption in home and alternate care settings as well. Furthermore, non-profit and government initiatives, and awareness programs, and an influx of funding for research studies have positively influenced developments within the industry.

Competitive Landscape and Stem Cell Therapy Market Share Analysis

The major players covered in the Stem Cell Therapy market report are Terumo Medical Corporation, Getinge AB., Medtronic, Edwards Lifesciences Corporation., Smiths Medical, Teleflex Incorporated., Johnson & Johnson Services, Inc., Abbott., Dispocard GmbH, Coloplast Corp., Boston Scientific Corporation or its affiliates., GENERAL ELECTRIC COMPANY, Astler Medicss., Tinomed Healthcare Private Limited., S N Medical Systems, Paras Healthcare, Elesonic Healthcare Private Limited., Ansh Healthcare., Omega Medsurge and Hi Tech Medi Systems. among other domestic and global players.

Global Stem Cell Therapy Market Scope And Market Size

By type, the Stem Cell Therapy market is segmented intoelectron beam computed tomography, nuclear Stem Cell Therapy, echocardiogram (ECG), cardiac catheterization, coronary arteriography, angiocardiography, and others.

By indication, the Stem Cell Therapy market is segmented into congenital heart diseases, coronary artery blockage, defects or injuries to the hearts fourprimary valves, blood clots within the heart, tumours in or on the heart, and others.

By end use, the Stem Cell Therapy market is segmented into hospitals and clinics, diagnostic centres, cardiac care centres, ambulatory centres and home care, academic institutes, and others.

Global Stem Cell Therapy Market: Regional Analysis

The research report includes specific segments by region (country), by company, by Type and by Application. This study provides information about the sales and revenue during the historic and forecasted period of 2021 to 2027. Understanding the segments helps in identifying the importance of different factors that aid the market growth.

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Stem Cell Therapy Market Share, Size Global Forthcoming Developments, Updates, Leading Players,Future Growth, Business Prospects and Future...

Abecma (idecabtagene vicleucel ; ide-cel; formerly bb2121), a chimeric antigen receptor (CAR)-T cell therapy, led to improved survival in patients with multiple myeloma who have been treated with many other lines of therapy, according to updated results from the KarMMa trial presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.1

The favorable benefit risk profile of ide-cel, regardless of the number of prior lines of therapy, supports its role as a treatment option for heavily pretreated relapse refractory multiple myeloma, Dr. Larry D. Anderson, associate professor, UT Southwestern Medical Center, said during a presentation of the poster.

At the December 21, 2020, data cutoff, the average follow-up was 24.8 months (range, 1.7-33.6).

Overall response rate (ORR) the percentage of patients who responded to the treatment was 73% in the overall population, including a 33% complete response rate, where disease could not be detected (CRR; complete response [CR] or stringent complete response [sCR]), 20% with a very good partial response (VGPR), and 20% who had a partial response (PR). ORR rates were 50%, 69%, and 81%, respectively, across the 150, 300, and 450 million CAR T cell-dose arms, including CR/sCR rates of 25%, 29%, and 39%.

Of note, ORR did not vary by the number of prior lines of therapy received. For those who received three prior lines of therapy (a total of 15 patients), the ORR was 73%, including a CRR of 53% and VGPR of 20%, compared with an ORR of 73% in those who received four or more (112 patients) lines of therapy, including a CRR of 30%, VGPR of 23%, and PR of 20%.

Average duration of response (DOR; the time patients disease was stable or in remission after being treated) was 10.9 months, including 9.9 months for the 300 million CAR T cells-dose arm and 11.3 months for the 450 million CAR T cells-dose arm -dose arm. Median DOR was 21.5 months in patients who experienced a CR or sCR. Median DOR by response were 21.5 months among those who experienced a CR; 10.4 months for those with VGPR; and 4.5 months in those with PRs.

Moreover, the rate of event-free 24-month DOR appeared to be similar in patients who received three or four or more lines of therapy. For those who received three lines of prior therapy, median DOR was eight months, compared with 10.9 months in those who received four or more lines of therapy.

Average progression-free survival (PFS) meaning the length of time after treatment when the disease does not get worse was 8.6 months across all target doses, including 5.8 months for the 300 million CAR T cells-dose arm and 12.2 months for the 450 million CAR T cells-dose arm -dose arm. Similarly, average PFS was similar among those who previously received three lines of therapy, compared with four or more prior lines of therapy (8.6 months vs 8.9 months, respectively).

On average, it took patients about one month to respond to therapy and about 2.8 months to experience a CR.

Median overall survival (OS) was 24.8 months, including a median OS of 22.0 months in those who received three lines of prior therapy and 25.2 months in those who received four or more lines of prior therapy. Moreover, OS was 20 months or longer across several key high-risk subgroups, including those aged 65 or older (21.7 months), those with extramedullary disease (20.2 months), and those with triple refractory disease (21.7 months).

Regarding side effects, cytokine release syndrome (CRS; the effect of many inflammatory cytokine immune cells being release into the blood stream) and neurotoxicity (brain and/or nervous system damage) rates were similar, regardless of prior lines of therapy received, and were mostly low grade. In total, 85% and 18% of the overall population experienced at least 1 CRS or neurotoxicity event, respectively.

The safety profile of Abecma was consistent with long-term follow-up, with similar rates of infections and secondary primary malignancies, and no unexpected gene therapy related toxicities were observed. The most common grade 3 to 4 side effects in the overall population were neutropenia (89%), anemia (61%), thrombocytopenia (52%), leukopenia (39%), lymphopenia (27%) and infections (27%).

Long-term results from the KarMMA trial continue to demonstrate frequent, deep, and durable responses in heavily pretreated patients with [relapsed/refractory multiple myeloma], the study authors write in the poster. ORR, CRR, DOR and PFS were consistent with previous reports and patients received similar benefit regardless of the number of prior lines of therapy.

In his presentation, Anderson presented data on long-term efficacy and safety following treatment with Abecma in the pivotal phase 2 KarMMa trial (NCT03361748)-including overall data and by prior line of therapy that patients had received (three compared to four or more), since the FDA label is requiring at least four prior lines, and this study only required three, he added.

In total, 140 patients who had received at least three prior lines of therapy for multiple myeloma including an IMiD, a PI, and an anti-CD38 antibody and were refractory to their last treatment regimen, were enrolled in the study. However, only 128 patients received infusion with Abecma.

Patients were treated with Abecma across the target dose range of 150 (four patients), 300 (70 patients), and 450 (54 patients) million CAR T cells.

ORR served as the primary end point of the study. Secondary end points included CRR, safety, DOR, PFS, OS, pharmacokinetics, minimal residual disease, quality of life and health economics and outcomes research.

At the start of the trial, the average patient age was 61 years (range, 33-78) and patients had a median of six years (range, 1-18) since their diagnosis. A majority of the patients were male (59%), had high tumor burden (51%), B-cell maturation antigen (BCMA) expression 50% or more at screening (85%), ECOG performance status which measures how functional a patient is on a range of 1 (fully functioning) to 5 (dead) of 1 (53%), and Revised International Staging System disease stage of II (70%). Thirty-five percent of patients had high-risk features.2

The median number of prior therapies was six (range, 3-16) and 94% had previously undergone at least one autologous hematopoietic stem cell transplant (94%). Eighty-eight percent of patients required bridging therapy, a kind of pre-treatment before CAR-T cell thearpy. Eighty-nine percent of patients had double-refractory disease, 84% were triple-refractory and 26% were penta-refractory.

Patients who had received three prior lines of therapy had similar baseline characteristics, compared with those who received four or more prior lines, including differences in extramedullary disease, high-risk cytogenetics, prior refractoriness and time since the initial diagnosis to screening.

Patients with relapsed/refractory multiple myeloma previously exposed to immunomodulatory agents, protease inhibitors, and anti-CD38 antibodies have poor outcomes with subsequent therapy using previously approved regimens, with expected response rates in the 26% to 31% range, PFS in the two- to four-month range, and overall survival less than nine months, Anderson explained.

However, the BCMA-directed CAR-T cell therapy previously demonstrated favorable tolerability with deep, durable responses in patients who were heavily pretreated with relapsed/refractory multiple myeloma.As a result, the FDA approved the agent for the treatment of adult patients with relapsed or refractory multiple myeloma after four or more prior therapies, including an immunomodulatory drug, a proteasome inhibitor, and an anti-CD38 antibody, representing the first BCMAdirected CAR T-cell therapy approved.

The study authors noted that is being explored in ongoing clinical trials, including the following:

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Abecma Continues to Improve Survival in Heavily Pretreated Patients With Multiple Myeloma - Curetoday.com

Michael Bishop, MD, a professor of medicine and director of Hematopoietic Stem Cell Transplantation Program at The University of Chicago Medicine, discussesthe different settings where chimeric antigen receptor (CAR) T-cell therapy are used.

Bishop thinks CAR T-cell therapy made a significant impact upon patient care. CAR T cells are indicated for 3 main groups of patients. The first is for non-Hodgkin lymphoma and for pediatric and young adult acute lymphoblastic leukemia (ALL), which has FDA-approved agents. More recent approvals have been for patients with mantle cell lymphoma. Physicians are waiting for what they expect to be the first indication for CAR T-cell therapy in multiple myeloma.

Starting with ALL for the pediatric population, CAR T cells have been a game changer, according to Bishop. These young patients have median overall survivals of less than 6 months, but the high response rates with CAR T are enabling them to potentially go on to an allogeneic stem cell transplant and in some cases, be free of disease.

In the non-Hodgkin lymphoma setting, there are now 3 products indicated for advanced B-cell non-Hodgkin lymphoma, which is significant for patients with totally refractory disease. There is this therapeutic option that is potentially curative. Bishop says investigators are seeing patients out to 5 years without any further therapy after receiving CAR T cells.

Excerpt from:
CAR T Cells Have Reshaped the Hematologic Cancer Landscape - Targeted Oncology

8 of 11 Patients in Dose Escalation Cohorts 2 and 3 Achieved Objective Response

6 of 11 Patients Achieved Complete Response, including 2 Patients Previously Treated with Autologous CD19 CAR T-cell Therapy

Favorable FT516 Safety Profile Was Observed; No FT516-related Serious Adverse Events or FT516-related Grade 3 or Greater Adverse Events

Outpatient Treatment Regimen Was Well-tolerated; No Events of Any Grade of Cytokine Release Syndrome, Immune Effector Cell-Associated Neurotoxicity Syndrome, or Graft-vs-Host Disease

SAN DIEGO, June 04, 2021 (GLOBE NEWSWIRE) -- Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer, today highlighted positive interim Phase 1 data from the Companys FT516 program for patients with relapsed / refractory B-cell lymphoma at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting being held virtually June 4-8, 2021. FT516 is the Companys universal, off-the-shelf natural killer (NK) cell product candidate derived from a clonal master induced pluripotent stem cell (iPSC) line engineered with a novel high-affinity, non-cleavable CD16 (hnCD16) Fc receptor, which is designed to maximize antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. The ongoing Phase 1 dose-escalation study of FT516 is currently enrolling patients in the fourth dose cohort of 900 million cells per dose.

As of the data cutoff date of March 11, 2021, four patients in the second dose cohort of 90 million cells per dose and seven patients in the third dose cohort of 300 million cells per dose were evaluable for assessment of safety and efficacy. Eight of eleven patients achieved an objective response, including six patients who achieved a complete response, as assessed by PET-CT scan per Lugano 2014 criteria (see Table 1). Patients had received a median of three prior lines of therapy and a median of two prior lines containing CD20-targeted therapy. Of the eleven patients, eight patients had aggressive B-cell lymphoma, five patients were refractory to their most recent prior therapy, and four patients were previously treated with autologous CD19 CAR-T cell therapy.

These additional data from our Phase 1 study of FT516 administered off-the-shelf in the outpatient setting continue to reinforce its differentiated safety profile and underscore its potential clinical benefit, said Wayne Chu, M.D., Senior Vice President of Clinical Development of Fate Therapeutics. Based on the favorable therapeutic profile of FT516 that continues to emerge and the potential to treat patients on-demand without delay, we plan to initiate multiple indication-specific, dose-expansion cohorts for patients with B-cell lymphomas to broadly assess FT516 in combination with CD20-targeted monoclonal antibody regimens, including those used as standard-of-care in earlier-line settings.

The ongoing Phase 1 clinical trial in relapsed / refractory B-cell lymphoma is assessing FT516 in an off-the-shelf treatment regimen of up to two cycles, with each cycle consisting of three days of conditioning chemotherapy (500 mg/m2 of cyclophosphamide and 30 mg/m2 of fludarabine), a single-dose of rituximab (375 mg/m2), and three weekly doses of FT516 each with IL-2 cytokine support. The FT516 treatment regimen is designed to be administered in the outpatient setting.

Safety DataNo dose-limiting toxicities, and no FT516-related serious adverse events or FT516-related Grade 3 or greater adverse events, were observed. The FT516 treatment regimen was well tolerated, and no treatment-emergent adverse events (TEAEs) of any grade of cytokine release syndrome, immune effector cell-associated neurotoxicity syndrome, or graft-versus-host disease were reported by investigators (see Table 2). All Grade 3 or greater TEAEs were consistent with lympho-conditioning chemotherapy and underlying disease. Of note, a Grade 3 or greater TEAE of infection was reported in one patient only. There were no discontinuations due to adverse events, and no patients withdrew from the study except in the setting of disease progression. In addition, no evidence of anti-product T- or B-cell mediated host-versus-product alloreactivity was detected, supporting the potential to safely administer up to six doses of FT516 in the outpatient setting without the need for patient matching.

Activity DataAs of the data cutoff date of March 11, 2021, eleven relapsed / refractory patients in the second and third dose cohorts were evaluable for assessment of safety and efficacy. Of the eleven patients, nine patients completed both FT516 treatment cycles and eight patients achieved an objective response, including six patients who achieved a complete response, as assessed by PET-CT scan per Lugano 2014 criteria. Notably, two of four patients previously treated with autologous CD19 CAR-T cell therapy achieved a complete response. Two patients showed progressive disease following the first FT516 treatment cycle and discontinued treatment. The Company previously reported that two patients treated in the first dose cohort (30 million cells per dose) showed progressive disease.

Patient Case StudyThe ASCO presentation featured a case study of a 36-year old male with triple-hit, high-grade B-cell lymphoma with rearrangements of MYC, BCL2, and BCL6 genes. The patient was refractory to all prior lines of therapy with the exception of autologous CD19 CAR T-cell therapy, for which a complete response of two months duration was achieved. The patient was most recently refractory to an investigational CD20-targeted T-cell engager and presented with bulky lymphadenopathy with the largest lesion measuring approximately 10 centimeters. The first FT516 treatment cycle resulted in a complete response with resolution of all metabolically active disease and 85% reduction in the size of target lesions. Subsequent to the data cutoff date of March 11, 2021, the patient completed a second FT516 treatment cycle after which the response assessment continued to show complete response.

As of March 11, 2021 database entry. Data subject to source document verification.CR = Complete Response; PR = Partial Response; PD = Progressive DiseaseCAR = Chimeric antigen receptor; DH/DE = Double-hit / double expressor; DLBCL = Diffuse large B-cell lymphoma; FL = Follicular lymphoma; Gr = Grade; HGBCL = High-grade B-cell lymphoma; iNHL = Indolent non-Hodgkin lymphoma; TH = Triple-hit; Transformed iNHL = Aggressive B-cell lymphoma transformed from indolent non-Hodgkin lymphoma1 Cycle 2 Day 29 protocol-defined response assessment per Lugano 2014 criteria2 Subject did not proceed to Cycle 23 Confirmed DLBCL (transformation from Gr3A FL) subsequent to the data cutoff date of March 11, 20214 Cycle 2 Day 29 protocol-defined response assessment reported subsequent to the data cutoff date of March 11, 2021

CRS = Cytokine Release Syndrome; DL = Dose Level; GvHD = Graft vs. Host Disease; ICANS = Immune Cell-Associated Neurotoxicity Syndrome;M = Million; SAE = Serious Adverse Event; TEAE = Treatment-Emergent Adverse Event1 Includes two subjects in the first dose cohort of 30 million cells per dose

About Fate Therapeutics iPSC Product PlatformThe Companys proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that are designed to be administered with multiple doses to deliver more effective pharmacologic activity, including in combination with other cancer treatments. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Companys first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event and selecting a single engineered iPSC for maintenance as a clonal master iPSC line. Analogous to master cell lines used to manufacture biopharmaceutical drug products such as monoclonal antibodies, clonal master iPSC lines are a renewable source for manufacturing cell therapy products which are well-defined and uniform in composition, can be mass produced at significant scale in a cost-effective manner, and can be delivered off-the-shelf for patient treatment. As a result, the Companys platform is uniquely designed to overcome numerous limitations associated with the production of cell therapies using patient- or donor-sourced cells, which is logistically complex and expensive and is subject to batch-to-batch and cell-to-cell variability that can affect clinical safety and efficacy. Fate Therapeutics iPSC product platform is supported by an intellectual property portfolio of over 350 issued patents and 150 pending patent applications.

About FT516FT516 is an investigational, universal, off-the-shelf natural killer (NK) cell cancer immunotherapy derived from a clonal master induced pluripotent stem cell (iPSC) line engineered to express a novel high-affinity 158V, non-cleavable CD16 (hnCD16) Fc receptor, which has been modified to prevent its down-regulation and to enhance its binding to tumor-targeting antibodies. CD16 mediates antibody-dependent cellular cytotoxicity (ADCC), a potent anti-tumor mechanism by which NK cells recognize, bind and kill antibody-coated cancer cells. ADCC is dependent on NK cells maintaining stable and effective expression of CD16, which has been shown to undergo considerable down-regulation in cancer patients. In addition, CD16 occurs in two variants, 158V or 158F, that elicit high or low binding affinity, respectively, to the Fc domain of IgG1 antibodies. Scientists from the Company have shown in a peer-reviewed publication (Blood. 2020;135(6):399-410) that hnCD16 iPSC-derived NK cells, compared to peripheral blood NK cells, elicit a more durable anti-tumor response and extend survival in combination with anti-CD20 monoclonal antibodies in an in vivo xenograft mouse model of human lymphoma. Numerous clinical studies with FDA-approved tumor-targeting antibodies, including rituximab, trastuzumab and cetuximab, have demonstrated that patients homozygous for the 158V variant, which is present in only about 15% of patients, have improved clinical outcomes. FT516 is being investigated in a multi-dose Phase 1 clinical trial as a monotherapy for the treatment of acute myeloid leukemia and in combination with CD20-targeted monoclonal antibodies for the treatment of advanced B-cell lymphoma (NCT04023071). Additionally, FT516 is being investigated in a multi-dose Phase 1 clinical trial in combination with avelumab for the treatment of advanced solid tumor resistant to anti-PDL1 checkpoint inhibitor therapy (NCT04551885).

About Fate Therapeutics, Inc.Fate Therapeutics is a clinical-stage biopharmaceutical company dedicated to the development of first-in-class cellular immunotherapies for patients with cancer. The Company has established a leadership position in the clinical development and manufacture of universal, off-the-shelf cell products using its proprietary induced pluripotent stem cell (iPSC) product platform. The Companys immuno-oncology pipeline includes off-the-shelf, iPSC-derived natural killer (NK) cell and T-cell product candidates, which are designed to synergize with well-established cancer therapies, including immune checkpoint inhibitors and monoclonal antibodies, and to target tumor-associated antigens using chimeric antigen receptors (CARs). Fate Therapeutics is headquartered in San Diego, CA. For more information, please visit http://www.fatetherapeutics.com.

Forward-Looking StatementsThis release contains "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995 including statements regarding the safety and therapeutic potential of the Companys iPSC-derived NK cell product candidates, including FT516, its ongoing and planned clinical studies, and the expected clinical development plans for FT516. These and any other forward-looking statements in this release are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that results observed in studies of its product candidates, including preclinical studies and clinical trials of any of its product candidates, will not be observed in ongoing or future studies involving these product candidates, the risk that the Company may cease or delay clinical development of any of its product candidates for a variety of reasons (including requirements that may be imposed by regulatory authorities on the initiation or conduct of clinical trials, the amount and type of data to be generated, or otherwise to support regulatory approval, difficulties or delays in subject enrollment and continuation in current and planned clinical trials, difficulties in manufacturing or supplying the Companys product candidates for clinical testing, and any adverse events or other negative results that may be observed during preclinical or clinical development), and the risk that its product candidates may not produce therapeutic benefits or may cause other unanticipated adverse effects. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Companys actual results to differ from those contained in the forward-looking statements, see the risks and uncertainties detailed in the Companys periodic filings with the Securities and Exchange Commission, including but not limited to the Companys most recently filed periodic report, and from time to time in the Companys press releases and other investor communications.Fate Therapeutics is providing the information in this release as of this date and does not undertake any obligation to update any forward-looking statements contained in this release as a result of new information, future events or otherwise.

Contact:Christina TartagliaStern Investor Relations, Inc.212.362.1200christina@sternir.com

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Fate Therapeutics Highlights Positive Interim Data from its Phase 1 Study of FT516 in Combination with - GlobeNewswire

Long-term follow-up data from the KarMMa trial found that treatment with the chimeric antigen receptor (CAR) T-cell therapy, idecabtagene vicleucel (ide-cel; formerly bb2121; Abecma), continues to demonstrate improved survival among heavily pretreated patients with relapsed/refractory multiple myeloma, according to a presentation presented at the 2021 American Society of Clinical Oncology (ASCO) Annual Meeting.1

The favorable benefit risk profile of ide-cel, regardless of the number of prior lines of therapy, supports its role as a treatment option for heavily pretreated relapse refractory multiple myeloma, Larry D. Anderson, MD, PhD, associate professor, UT Southwestern Medical Center, said during a presentation of the poster.

At the December 21, 2020, data cutoff, the median follow-up was 24.8 months (range, 1.7-33.6).

Overall response rate (ORR) was 73% in the overall population, including a 33% complete response rate (CRR; complete response [CR] or stringent complete response [sCR]), 20% with a very good partial response (VGPR), and 20% who had a partial response (PR). ORR rates were 50%, 69%, and 81%, respectively, across the 150, 300, and 450 million CAR T cell-dose arms, including CR/sCR rates of 25%, 29%, and 39%.

Of note, ORR did not vary by the number of prior lines of therapy received. For those who received 3 prior lines of therapy (n = 15), the ORR was 73%, including a CRR of 53% and VGPR of 20%, compared with an ORR of 73% in those who received 4 (n = 112) lines of therapy, including a CRR of 30%, VGPR of 23%, and PR of 20%.

Median duration of response (DOR) was 10.9 months (95% CI, 9.0-11.4), including 9.9 months for the 300 million CAR T cells-dose arm and 11.3 months for the 450 million CAR T cells-dose arm -dose arm. Median DOR was 21.5 months in patients who experienced a CR or sCR. Median DOR by response were 21.5 months (95% CI, 12.5 to not estimable [NE]) among those who experienced a CR, 10.4 months (95% CI, 5.1-12.2) for those with VGPR, and 4.5 months (95% CI, 2.9-6.7) in those with PRs.

Moreover, the rate of event-free 24-month DOR appeared to be similar in patients who received 3 or 4 or more lines of therapy. For those who received 3 lines of prior therapy, median DOR was 8.0 months (95% CI, 3.3-11.4), compared with 10.9 months (95% CI, 9.2-13.5) in those who received 4 or more lines of therapy.

Median progression-free survival (PFS) was 8.6 months (95% CI, 5.6-11.6) across all target doses, including 5.8 months for the 300 million CAR T cells-dose arm and 12.2 months for the 450 million CAR T cells-dose arm -dose arm. Similarly, median PFS was similar among those who previously received 3 lines of therapy, compared with 4 or more prior lines of therapy (8.6 months (95% CI, 2.9-12.1) vs 8.9 months (95% CI, 5.4-11.6)]

The median time to first response was 1 month (range, 0.5-8.8), with a median time to CR of 2.8 months (range, 1.0-15.8).

Median overall survival (OS) was 24.8 months (95% CI, 19.9-31.2), including a median OS of 22.0 months (95% CI, 10.-NE) in those who received 3 lines of prior therapy and 25.2 months (95% CI, 19.9-NE) in those who received 4 or more lines of prior therapy. Moreover, OS was 20 months or longer across several key high-risk subgroups, including those aged 65 or older (21.7 months; 95% CI, 17.1-31.2), those with extramedullary disease (20.2 months; 95% CI, 15.5-28.3), and those with triple refractory disease (21.7 months; 95% CI, 18.2-NE).

In regards to safety, cytokine release syndrome (CRS) and neurotoxicity were similar, regardless of prior lines of therapy received, and were mostly low grade. In total, 85% and 18% of the overall population experienced at least 1 CRS or neurotoxicity event, respectively.

The safety profile of ide-cel was consistent with long-term follow-up, with similar rates of infections and secondary primary malignancies, and no unexpected gene therapy related toxicities were observed. The most common grade 3 to 4 adverse events (AEs) in the overall population were neutropenia (89%), anemia (61%), thrombocytopenia (52%), leukopenia (39%), lymphopenia (27%), and infections (27%).

Long-term results from the KarMMA trial continue to demonstrate frequent, deep, and durable responses in heavily pretreated patients with [relapsed/refractory multiple myeloma], the study authors write in the poster. ORR, CRR, DOR, and PFS were consistent with previous reports and patients received similar benefit regardless of the number of prior lines of therapy.

In his presentation, Anderson presented data on long-term efficacy and safety following treatment with ide-cel in the pivotal phase 2 KarMMa trial (NCT03361748)-including overall data and by prior line of therapy that patients had received (3 vs 4), since the FDA label is requiring at least 4 prior lines, and this study only required 3, he added.

In total, 140 patients who had received at least 3 prior lines of therapy for multiple myeloma including an IMiD, a PI, and an anti-CD38 antibody and were refractory to their last treatment regimen, were enrolled in the study. However, only 128 patients received infusion with ide-cel.

Patients were treated with ide-cel across the target dose range of 150 (n = 4), 300 (n = 70), and 450 (n = 54) million CAR T cells.

ORR served as the primary end point of the study. Secondary end points included CRR, safety, DOR, PFS, OS, pharmacokinetics, minimal residual disease, quality of life, and health economics and outcomes research.

At baseline, the median patient age was 61 years (range, 33-78) and patients had a median of 6 years (range, 1-18) since their diagnosis. A majority of the patients were male (59%), had high tumor burden (51%), B-cell maturation antigen (BCMA) expression 50% at screening (85%), ECOG performance status of 1 (53%), and Revised International Staging System disease stage of II (70%). Thirty-five percent of patients had high-risk features.2

The median number of prior therapies was 6 (range, 3-16) and 94% had previously undergone at least 1 autologous hematopoietic stem cell transplant (94%). Eighty-eight percent of patients required bridging therapy. Eighty-nine percent of patients had double-refractory disease, 84% were triple-refractory, and 26% were penta-refractory.

Patients who had received 3 prior lines of therapy had similar baseline characteristics, compared with those who received 4 prior lines, including differences in extramedullary disease, high-risk cytogenetics, prior refractoriness, and time since the initial diagnosis to screening.

Patients with relapsed/refractory multiple myeloma previously exposed to immunomodulatory agents, protease inhibitors, and anti-CD38 antibodies have poor outcomes with subsequent therapy using previously approved regimens, with expected response rates in the 26% to 31% range, PFS in the 2- to 4-month range, and overall survival less than 9 months, Anderson explained.

However, the BCMA-directed CAR T-cell therapy previously demonstrated favorable tolerability with deep, durable responses in patients who were heavily pretreated with relapsed/refractory multiple myeloma.2 As a result, the FDA approved the agent for the treatment of adult patients with relapsed or refractory multiple myeloma after 4 or more prior therapies, including an immunomodulatory drug, a proteasome inhibitor, and an anti-CD38 antibody, representing the first BCMAdirected CAR T-cell therapy approved.3

The study authors noted that ide-cel is being explored in ongoing clinical trials, including the following:

Read more:
In Some Heavily Pretreated Patients with R/R MM Ide-Cel Continues to Show Deep and Durable Responses - Targeted Oncology

Patients with mantle cell lymphoma (MCL) or chronic lymphocytic leukemia (CLL) who were treated with cirmtuzumab and ibrutinib (Imbruvica) in a phase 1/2 trial showed promising responses with and tolerability of therapy, according to data presented at the American Society of Clinical Oncology (ASCO) Annual Meeting.

The majority of the patients demonstrated a significant reduction in tumor sizes, lead study author Hun Ju Lee, MD, assistant professor of medicine in the Department of Lymphoma & Myeloma at The University of Texas MD Anderson Cancer Center said during the presentation.

ORR for patients with MCL was 83.3% and was 91.1% for patients with CLL. The complete response rates were 38.9% and 14.7% for the MCL and CLL arms, respectively. Ultimately, 94.4% of patients with MCL and 100% of patients with CLL elicited a clinical benefit from the cirtuzumab/ibrutinib regimen.

The median progression-free survival in both cancers was not reached.

Because MCL and CLL are considered incurable, the study aimed to test the efficacy and safety profile of cirmtuzumab, which inhibits tumor promoting activity of onco-embryonic tyrosine kinase receptor ROR1 found in many solid and hematologic cancers, plus ibrutinib in patients with relapsed/refractory MCL or treatment nave or relapsed/refractory CLL.

The study was performed in 3 parts with separate arms. Part 1, for dose escalation; part 2, for dose expansion; and part 3, comparing cirmtuzumab plus ibrutinib with ibrutinib alone in CLL.

Overall, 26 patients with refractory MCL (median age 66.5, 15.4% women) and 34 patients with treatment nave or RR CLL (median age 68, 23.5% women) were enrolled in the study.

For part 1, 12 patients with MCL were enrolled, and 5 into part 2. The median number of prior regimens was 2, including patients relapsing after ibrutinib (n=4), autologous stem-cell transplantation (n=3), autologous stem cell transplantation/allogenic stem cell transplantation (1) and autologous stem cell transplantation /CAR-T (1). For patients with CLL, at least 74% were high risk, as determined by unmutated IGHV, del17p and/or del11q, in parts 1 and 2.

In part 1, cirmtuzumab was given intravenously 5 times every 2 weeks, and then every 4 weeks, at 2 to 16 mg. Three-hundred or 600 mg doses were also examined. Researchers assessed the safety profile of cirmtuzumab during the first 28 days, which was then followed by ibrutinib at approved doses for each indication. A treatment regimen of cirmtuzumab (600 mg) given intravenously 3 times every 2 weeks, and then every 4 weeks, in combination with ibrutinib starting day 0, was chosen as the recommended dosing for parts 2 and 3.

In summary, cirmtuzumab plus ibrutinib is a very well tolerated regimen, Lee noted.

Adverse events with 20% or greater incidence were recorded, including fatigue (n=11), diarrhea (n = 9), contusion (n = 7), dizziness (n = 7) and nausea (n = 7). The efficacy is robust in many of these pre-treated patients, [including] high response, rate durable response [and] encouraging PFS, demonstrating clinical benefit, he concluded.

Of note, the phase 2 study for CLL is completed, and is awaiting a long-term follow-up. Phase 2 for MCL is currently enrolling.

Reference

Lee HJ, Choi M, Siddiqi T, et al. Phase 1/2 study of cirmtuzumab and ibrutinib in mantle cell lymphoma (MCL) or chronic lymphocytic leukemia (CLL).J Clin Oncol. 2021;39(suppl 15; abstr 7556).

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High Rate of Response Noted in MCL and CLL With Cirmtuzumab Plus Ibrutinib - Cancer Network