On August 7, 2018 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported that it has appointed Samuel Agresta, M.D., M.P.H., as Chief Medical Officer where he will oversee global clinical development and regulatory affairs for the company (Press release, Infinity Pharmaceuticals, AUG 7, 2018, View Source [SID1234528758]). Dr. Agresta brings more than 20 years of experience in the practice of academic medicine and oncology drug development, including the recent approvals of two targeted therapies for the treatment of acute myeloid leukemia (AML).

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"We are very pleased to welcome Dr. Agresta to the Infinity team as he brings tremendously valuable global development and regulatory experience having led the development and approval of enasidineb (IDHIFA) and ivosidenib (TIBSOVO), two novel clinical oncology medicines, while at Agios," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "Under Dr. Agresta’s leadership, both medicines received US regulatory approval during the last year, with full approval of ivosidenib granted two weeks ago for patients with relapsed and/or refractory AML based on the results of a single arm, Phase 1 study. Sam’s knowledge and expertise in innovative oncology and precision medicine drug development will be invaluable in advancing IPI-549 for patients."

"This is an exciting time for me to be joining the Infinity team. IPI-549’s mechanism of action represents a novel and promising approach to improve upon current immuno-oncology treatments," said Dr. Agresta. "I am very encouraged by the clinical and translational data presented to date and look forward to building on this foundation in the ongoing and future IPI-549 trials, including with our clinical collaborators Bristol-Myers Squibb and Arcus, to demonstrate the potential impact of IPI-549 in immuno-oncology therapy. We will also work in collaboration with our investigators and regulators to advance IPI-549 with the goal of approval to benefit as many cancer patients as possible."

Dr. Agresta joins Infinity from Agios Pharmaceuticals, Inc., where he played a pivotal role in the development of the company’s oncology programs, including IDHIFA and TIBSOVO, as Vice President and Head of Clinical Development. IDHIFA and TIBSOVO are both first-in-class, orally available, targeted medicines for the treatment of patients with AML that harbor IDH mutations. Prior to Agios, Dr. Agresta worked on the development of oncology therapeutics at Merrimack Pharmaceuticals and Genentech after spending ten years practicing academic medicine. He began his career at the Moffitt Cancer Center, where he served as Associate Medical Director in the Sarcoma Oncology Unit. Dr. Agresta earned an M.D. from Tulane University School of Medicine as well as an M.P.H. from Tulane University School of Public Health and Tropical Medicine.

On August 7, 2018 Infinity Pharmaceuticals, Inc. (NASDAQ: INFI) reported its second quarter 2018 financial results and provided an update on the company, including its progress with IPI-549, a first-in-class oral immuno-oncology product candidate that selectively inhibits phosphoinositide-3-kinase-gamma (PI3K-gamma) and targets immune-suppressive tumor macrophages/myeloid-derived suppressor cells (MDSCs) (Press release, Infinity Pharmaceuticals, AUG 7, 2018, View Source [SID1234528757]).

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"We are particularly pleased to welcome Sam Agresta to the Infinity team as Chief Medical Officer. Sam brings extensive experience in novel oncology drug development, having led the recent approvals of Agios’s two targeted therapies based on compelling Phase 1 data," said Adelene Perkins, Chief Executive Officer and Chair of Infinity Pharmaceuticals. "We continue to be encouraged by our progress in all aspects of our business. Building on the datasets we presented at the recent American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, including showing that IPI-549 was well-tolerated, clinically active and on-mechanism as a monotherapy and in combination with nivolumab, we look forward to presenting more mature data from the combination expansion cohorts in the second half of this year. We are also enthusiastic about partnering with Arcus and testing IPI-549 in combination with its very promising novel agents, in a new collaboration we established in June."

Infinity is evaluating IPI-549 as a monotherapy and in combination with Opdivo (nivolumab), a PD-1 immune checkpoint inhibitor, in collaboration with Bristol-Myers Squibb, in the MARIO-1 Phase 1/1b study in approximately 200 patients with advanced solid tumors. In addition, Arcus Biosciences will initiate two triple combinations investigating IPI-549 with their dual adenosine receptor antagonist, AB928, anti-PD-1 antibody, AB122, and chemotherapy in triple negative breast cancer and ovarian cancer. One triple combination therapy will evaluate IPI-549 in combination with AB928 and AB122 and the second will evaluate IPI-549 in combination with AB928 and chemotherapy, with topline data expected in 2019.

Recent developments include the following:

IPI-549

Continued Progress with the MARIO-1 Phase 1/1b Study of IPI-549: The monotherapy and combination dose-escalation portions of this study have been completed. Enrollment is complete in the mesothelioma combination expansion cohort and is ongoing for the other five disease-specific combination expansion cohorts currently underway, as well as for the seventh combination expansion cohort of patients pre-selected for having high baseline blood levels of myeloid derived suppressor cells (MDSCs), which began enrolling patients in May 2018.
Second Quarter 2018 Financial Results

At June 30, 2018, Infinity had total cash, cash equivalents and available-for-sale securities of $49.2 million, compared to $47.8 million at March 31, 2018.
R&D expense for the second quarter of 2018 was $3.7 million, compared to $3.9 million for the same period in 2017. The decrease in R&D expense was primarily due to a reduction in bonus and stock compensation offset by an increase in clinical development expense for IPI-549.
General and administrative expense was $3.4 million for the second quarter of 2018, compared to $6.2 million for the same period in 2017. The decrease in G&A expense was primarily due to a reduction in bonus and stock compensation.
Net loss for the second quarter of 2018 was $7.0 million, or a basic and diluted loss per common share of $0.12, compared to a net loss of $17.0 million, or a basic and diluted loss per common share of $0.34 for the same period in 2017.
Financial Outlook
Infinity’s 2018 financial guidance is:

Net Loss: Infinity expects net loss for 2018 to range from $35 million to $45 million.
Cash and Investments: Infinity expects to end 2018 with a year-end cash, cash equivalents and available-for-sale securities balance ranging from $15 million to $25 million.
Cash Runway: Based on its current operational plans, Infinity expects that its existing cash, cash equivalents and available-for-sale securities will be adequate to satisfy the company’s capital needs through the third quarter of 2019. Infinity’s financial guidance excludes additional funding or business development activities and does not include the potential $22 million payment from Verastem upon the first regulatory approval of duvelisib, or a potential $2 million payment from PellePharm, a private company, upon initiation of a Phase 3 study for the hedgehog inhibitor program, which Infinity licensed to PellePharm in 2013. Verastem announced that its New Drug Application for duvelisib was accepted by the U.S. Food and Drug Administration (FDA) and that it was given priority review with an FDA action date of October 5, 2018. With the potential Verastem payment, Infinity expects that its cash runway would extend into 2020.
Conference Call Information
Infinity will host a conference call today, August 7, 2018, at 4:30 p.m. ET to discuss these financial results and company updates. A live webcast of the conference call can be accessed in the "Investors/Media" section of Infinity’s website at www.infi.com. To participate in the conference call, please dial 1-877-316-5293 (domestic) or 1-631-291-4526 (international) five minutes prior to start time. The conference ID number is 8037589. An archived version of the webcast will be available on Infinity’s website for 30 days.

About IPI-549 and the Ongoing Phase 1/1b Study
IPI-549 is an investigational first-in-class, oral, immuno-oncology product candidate targeting tumor-associated myeloid cells through selective phosphoinositide-3-kinase-gamma (PI3K-gamma) inhibition, thereby reducing pro-tumor macrophage function and increasing anti-tumor macrophage function. In preclinical studies, IPI-549 demonstrated the ability to reprogram macrophages from a pro-tumor (M2), immune suppressive function, to an anti-tumor (M1) immune activating function and enhance the activity of, and overcome resistance to, checkpoint inhibitors.1,2 As such, IPI-549 may have the potential to treat a broad range of solid tumors and represents a potentially additive or synergistic approach to restoring anti-tumor immunity in combination with other immunotherapies such as checkpoint inhibitors.

The ongoing Phase 1/1b study being conducted by Infinity is designed to evaluate the safety, tolerability, activity, pharmacokinetics and pharmacodynamics of IPI-549 as a monotherapy and in combination with Opdivo in approximately 200 patients with advanced solid tumors.3 The study includes monotherapy and combination dose-escalation components, in addition to monotherapy expansion and combination expansion components. The monotherapy dose-escalation and expansion components are complete. The combination dose-escalation component is also complete, and combination expansion cohorts are enrolling.

The combination expansion component of the study includes multiple cohorts designed to evaluate IPI-549 in patients with specific types of cancer, including patients with non-small cell lung cancer (NSCLC), melanoma and head and neck cancer whose tumors show initial resistance or initially respond to but subsequently develop resistance to immune checkpoint blockade therapy. The combination expansion component also includes a cohort of patients with triple negative breast cancer (TNBC) who have not been previously treated with immune checkpoint blockade therapy, a cohort of patients with mesothelioma, a cohort of patients with adrenocortical carcinoma and a cohort of patients with high baseline blood levels of MDSCs.

IPI-549 is an investigational compound and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

On August 7, 2018 Dynavax Technologies Corporation (NASDAQ: DVAX) reported financial results for the second quarter ended June 30, 2018 (Press release, Dynavax Technologies, AUG 7, 2018, View Source [SID1234528754]). The net loss for the quarter was $39.4 million, or $0.63 per share, compared to $20.3 million, or $0.41 per share, for the quarter ended June 30, 2017. Cash, cash equivalents and marketable securities totaled $216.0 million at June 30, 2018.

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Recent Highlights

HEPLISAV-B [Hepatitis B Vaccine (Recombinant), Adjuvanted]

100% of Medicare-insured lives, 94% of commercially-insured lives, and 73% of lives under state Medicaid plans are covered

219 of our largest targeted customers have received P&T committee approval, of whom 91 have progressed to purchase and 24 have implemented the use of HEPLISAV-B throughout their system

Another 198 target customers have sub-committee or P&T committee review scheduled

Q2 sales of $1.3 million compared to $0.2 million in Q1

Immuno-oncology

Encouraging SD-101 Phase 1b/2 advanced melanoma data in combination with KEYTRUDA in patients naïve to anti PD-1 therapy for 2 mg dose selected for Phase 3:

Overall response rate (ORR) of 70%

80% ORR in patients with low PD-L1

6-month progression free survival (PFS) rate of 76%

AEs related to SD-101 treatment were transient, mild to moderate flu-like symptoms

End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) scheduled

Three abstracts accepted for presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Annual Meeting, October 19-23, 2018

"The launch is progressing as planned and I continue to expect HEPLISAV-B will become the standard of care for vaccination of adults against hepatitis B. During my field visits, I have personally witnessed the strength of the product profile and label in motivating customers to switch vaccines," said Eddie Gray,

chief executive officer of Dynavax. "Our efforts to date are beginning to pay off with increasing sales, which we expect will accelerate during Q4 and into 2019, when we expect HEPLISAV-B to be cash generative before year end. In addition, we continue to advance our SD-101 clinical program which has shown encouraging results so far in both melanoma and head and neck carcinoma patients. We look forward to updating these data later this year."

Financial Results

Cash, cash equivalents and marketable securities of $216.0 million at end of the second quarter, with $75 million available from the February 2018 term loan agreement

Net product revenue was $1.3 million for the quarter ended June 30, 2018, which consists of sales of HEPLISAV-B in the U.S. Product revenue from sales is recorded at the net sales price which includes estimates of product returns, chargebacks, discounts and other fees.

Cost of sales, product was $5.2 million for the quarter ended June 30, 2018 and consists of certain fill, finish and fixed overhead costs for HEPLISAV-B incurred after FDA approval and costs relating to excess capacity at our Dusseldorf manufacturing facility associated with resuming operations after receiving FDA approval of HEPLISAV-B and pre-filled syringes.

Cost of sales, amortization of intangible assets was $2.3 million for the quarter ended June 30, 2018 and consists of amortization of the intangible asset recorded as a result of milestone and sublicense payments relating to HEPLISAV-B.

Research and development expenses for the quarter ended June 30, 2018 and 2017, were $16.3 million and $14.8 million, respectively. The increase in 2018 reflects increased compensation and related personnel costs related to the ongoing development of SD-101, DV281 and earlier stage oncology programs. Additionally, in the current quarter, manufacturing related costs incurred by our Dusseldorf facility that were previously included in research and development expense are now accounted for as excess capacity in our cost of sales, product.

Selling, general and administrative expenses for the quarter ended June 30, 2018 and 2017, were $15.7 million and $5.6 million, respectively. The increase is primarily due to an overall increase in HEPLISAV-B sales, marketing and commercial activities, including full-deployment of a contract sales force, post-marketing studies and consultants for commercial development services.

Conference Call and Webcast Information

Dynavax will hold a conference call today at 4:30pm ET/1:30pm PT. To access the call, participants must dial (800) 239-9838 in the U.S. or (323) 794-2551 internationally, and use the conference ID 2303066. The live call will be webcast and can be accessed in the "Investors and Media" section of the company’s website at www.dynavax.com. A replay of the webcast will be available for 30 days following the live event.

A replay of the conference call will be available for two weeks and can be accessed by dialing (844) 512-2921 in the U.S. or (412) 317-6671 internationally. The conference ID for the replay will be 2303066.

About Hepatitis B

Hepatitis B is a viral disease of the liver that can become chronic and lead to cirrhosis, liver cancer and death. The hepatitis B virus is 50 to 100 times more infectious than HIV,i and transmission is on the rise.

In 2015, new cases of acute hepatitis B increased by more than 20 percent nationally.ii There is no cure for hepatitis B, but effective vaccination can prevent the disease.

In adults, hepatitis B is spread through contact with infected blood and through unprotected sex with an infected person. The CDC recommends vaccination for those at high risk for infection due to their jobs, lifestyle, living situations and travel to certain areas.iii Because people with diabetes are particularly vulnerable to infection, the CDC recommends vaccination for adults age 19 to 59 with diabetes as soon as possible after their diagnosis, and for people age 60 and older with diabetes at their physician’s discretion.iv Approximately 20 million U.S. adults have diabetes, and 1.5 million new cases of diabetes are diagnosed each year.v

About HEPLISAV-B

HEPLISAV-B is an adult hepatitis B vaccine that combines hepatitis B surface antigen with Dynavax’s proprietary Toll-like Receptor (TLR) 9 agonist to enhance the immune response. Dynavax has worldwide commercial rights to HEPLISAV-B.

For more information about HEPLISAV-B, visit View Source

About SD-101

SD-101, the Company’s lead clinical candidate, is a proprietary, second-generation, Toll-like receptor 9 (TLR9) agonist CpG-C class oligodeoxynucleotide. Dynavax is evaluating this intratumoral TLR9 agonist in several clinical studies to assess its safety and activity, including a Phase 2 study in combination with KEYTRUDA (pembrolizumab), an anti-PD-1 therapy, in patients with advanced melanoma and in patients with head and neck squamous cell cancer, in a clinical collaboration with Merck. Dynavax maintains all commercial rights to SD-101.

On August 7, 2018 BioLineRx Ltd. (NASDAQ: BLRX) (TASE: BLRX), a clinical-stage biopharmaceutical company focused on oncology and immunology, reported the positive results from the lead-in period of the GENESIS trial, a double-blind, placebo-controlled Phase 3 trial comparing BL-8040 in combination with granulocyte colony-stimulating factor (G-CSF), to G-CSF alone, for the mobilization of hematopoietic stem cells (HSCs) used for autologous transplantation in multiple myeloma patients (Press release, BioLineRx, AUG 7, 2018, View Source;p=RssLanding&cat=news&id=2362414 [SID1234528751]).

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The open-label, single-arm, lead-in period of the study was designed to include up to 30 patients, with Data Monitoring Committee (DMC) review after completion of approximately 10, 20 and 30 patients, in order to assess safety and efficacy following treatment with BL-8040 plus G-CSF. Results of the first 11 patients show that BL-8040 in combination with standard G-CSF treatment is safe and tolerable. In addition, the data show that 9/11 patients (82%) reached the primary endpoint threshold of ≥ 6×106 CD34 cells/kg with only one dose of BL-8040 and in up to 2 apheresis sessions. Furthermore, 7/11 patients (64%) reached the threshold of ≥ 6×106 CD34 cells/kg in a single apheresis session only. These data demonstrate the potential of BL-8040 treatment to reduce the number of administrations and apheresis sessions, as well as hospitalization costs, related to the preparation of multiple myeloma patients for autologous HSC transplantation.

"Autologous HSC transplantation following high-dose chemotherapy has significantly improved outcomes for multiple myeloma patients," said Dr. John F. DiPersio, Chief, Division of Oncology at the Washington University School of Medicine, and lead investigator of the study. "Current practice involves mobilizing HSCs from the bone marrow to the peripheral blood, after which the cells are collected by apheresis. Results so far show that mobilizing HSCs with a single BL-8040 dose combined with G-CSF is not only safe and tolerable, but also demonstrates robust efficacy regarding the number of collected cells, and may reduce the number of required apheresis sessions to a single session in the majority of patients. This is a very encouraging result that, if corroborated in the placebo-controlled part of the trial, will be of great value to patients as well as to the medical community. I am looking forward to participating in the trial and to potentially improving the quality of treatment available to multiple myeloma patients."

"We are extremely encouraged by these results. Based on the robust data received from the first 11 patients, the DMC issued a positive recommendation to stop the lead-in part of the study and move immediately to the randomized placebo-controlled part of the study," stated Philip Serlin, Chief Executive Officer of BioLineRx. "This is the first Phase 3 trial for our lead BL-8040 program, and as such, it is an important milestone in BL-8040’s comprehensive development plan. We look forward to the top-line results from the randomized, double-blind, placebo-controlled part of the study, which are expected in 2020."

About the GENESIS Study

The GENESIS study is a Phase 3, randomized, double-blind, placebo-controlled, multicenter study, evaluating the safety, tolerability and efficacy of BL-8040 in combination with G-CSF, compared to placebo and G-CSF, for the mobilization of CD34 HSCs for autologous transplantation in multiple myeloma patients. The placebo-controlled part is designed to include 177 patients in more than 25 centers. Treatment will include 5 days of G-CSF, with a single dose of BL-8040 or placebo on day 4 with the option to expand treatment to up to 8 days of G-CSF and up to 2 days of BL-8040. Apheresis for collection of CD34 cells will be performed on day 5. An additional 3 apheresis sessions may be conducted if needed in order to reach the goal of ≥ 6×106 mobilized CD34 cells/kg.

The primary objective of the study is to demonstrate the superiority of a single dose of BL-8040 in combination with G-CSF, over placebo and G-CSF, in the mobilization of ≥ 6×106 CD34 cells/kg in up to 2 apheresis sessions, in preparation for autologous stem cell transplantation in multiple myeloma patients. Secondary objectives include time to engraftment of neutrophils and platelets, durability of the engraftment, as well as safety and other efficacy parameters.

About BL-8040

BL-8040 is a short synthetic peptide for stem cell mobilization and for treatment of hematological malignancies and solid tumors. It functions as a high-affinity best-in-class antagonist for CXCR4, a chemokine receptor that is directly involved in the retention of stems cells in the bone marrow, as well as tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in more than 70% of human cancers and its expression often correlates with disease severity.

HSCs express CXCR4 and are retained in the protective bone marrow niche via binding to CXCL12 (also known as SDF-1). Blocking of the CXCR4-SDF1 interaction by BL-8040 leads to the mobilization of HSCs into the peripheral blood. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of HSCs.

Furthermore, BL-8040 induce mobilization of leukemic cells and immune-cells from the bone marrow, thereby sensitizing leukemic cells to chemo- and bio-based anti-cancer therapy, as well as a direct anti-cancer effect by inducing cell death (apoptosis). BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

On August 7, 2018 Stony Brook University, iCell Gene Therapeutics, and the University of Louisville, have received Food and Drug Administration (FDA) clearance for an Investigational New Drug (IND) for the treatment of relapsed and refractory T-cell leukemia and lymphoma (Press release, Stony Brook University, AUG 7, 2018, View Source [SID1234528670]). The approach is the first to use chimeric antigen receptor engineered T-cells directed against the target protein CD4 (CD4CAR). Together, Stony Brook University, the University of Louisville, and iCell Gene Therapeutics expect the first in-human Phase I clinical trial to begin accruing patients before the end of 2018.

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"We are excited to partner with the University of Louisville and iCell Gene Therapeutics to offer this innovative first-in-human CAR T cell immunotherapy clinical trial for patients who are suffering from these extremely difficult to treat T cell lymphomas and leukemias," said Huda Salman, MD, Principal Investigator for the IND and an oncologist at Stony Brook University Cancer Center. "CD4CAR T cells may prove to be a promising and novel therapy in this setting."

"The development of this trial using CD4 as a target is the first of what we expect to be many CAR T-based clinical trials available to our patients over time," said Yusuf Hannun, MD, Director of the Stony Brook University Cancer Center. "The pending trial is an example of the type of bench-to-bedside research that is building up at Stony Brook due to the growing expertise and collaborative research environment we are creating and new opportunities that will emerge upon the opening of our Medical and Research Translation (MART) Building."

William Tse, MD, FACP, Chief of the Blood and Marrow Transplantation at the University of Louisville School of Medicine, is the Co-PI of the CD4CAR clinical trial at University of Louisville site.

About CD4CAR

CD4CAR is a novel development for the treatment of CD4+ T-cell malignancies. The CD4-redirected chimeric antigen receptor (CAR) T-cells are engineered to express an anti-CD4scFV antibody domain. CD4CAR has received Orphan Drug Designation by the FDA for Peripheral T cell Leukemia (PTCL) in 2016. Over the past few years, CAR T-cell therapy has proven its efficacy in clinical trials for various types of leukemia, lymphoma, and myeloma. CAR T-cell therapy is a type of adoptive immunotherapy. A CAR-engineered T-cell is genetically modified to express a protein on its surface with the capability to bind to a target protein on another cell. The CD4CAR is manufactured from the patient’s own cells to target CD4 expressed on tumor cells. Once these cells are infused back into a patient’s body through an IV, they multiply and attack tumor cells efficiently throughout the body.

About T-cell leukemias and lymphomas

Although there are clinical development programs ongoing with CAR T-cells for B-cell hematological malignancies, CD4 positive T-cell malignancies (T-cell lymphomas (TCLs) and T–cell acute lymphoblastic leukemia (T-ALLs)), have not been targeted by a CAR therapy in a human trial. TCLs account for 15–20 % of all non-Hodgkin’s lymphomas (NHLs), while T-ALLs affect about 25% of ALLs in adults. These malignant entities are significantly more difficult to treat in comparison to B-cell malignancies. Furthermore, T-cell malignancies almost exclusively have poorer outcomes with few exceptions, lower response rates, shorter times of disease control and survival. As a result, the standard of care for T-cell malignancies is not well-established and the only potential curative approach is allogeneic blood and marrow transplant (BMT) for which patients need to achieve complete disease control and to have suitable marrow donors. This leaves many patients with no curative options.