On October 29, 2019 Deciphera Pharmaceuticals, Inc. (Nasdaq:DCPH), a clinical-stage biopharmaceutical company addressing key mechanisms of tumor drug resistance, reported the presentation of updated results from its ongoing Phase 1 study of ripretinib, a broad-spectrum KIT and PDGFRα inhibitor, in patients with second-line through fourth-line plus GIST, as well as its Phase 1 study of DCC-3014, an oral inhibitor of CSF1R, in patients with advanced solid tumors (Press release, Deciphera Pharmaceuticals, OCT 29, 2019, View Source [SID1234549967]). The data are being presented today at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in Boston.

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"These updated results continue to underscore the potential of our diverse pipeline of product candidates, all generated using our proprietary kinase switch control inhibitor platform, to improve the lives of cancer patients," said Matthew L. Sherman, M.D., Executive Vice President and Chief Medical Officer of Deciphera. "Of note, we believe ripretinib continues to demonstrate strong clinical benefit in post-imatinib GIST patients, particularly in the second-line setting. These results bolster our confidence in the ongoing INTRIGUE pivotal Phase 3 clinical study, which is designed to support potential regulatory approvals in patients with second-line GIST."

Ripretinib

Updated results from the Company’s ongoing Phase 1 study of ripretinib in patients with second-line through fourth-line plus GIST included data from 142 GIST patients receiving 150 mg of ripretinib once daily (QD) as the starting dose, which is the dose being utilized in the Company’s INVICTUS and INTRIGUE registration-enabling studies, as of an August 10, 2019 data cutoff date. The table below includes local, investigator-assessed objective response rate (ORR) by best response as determined by Response Evaluation Criteria in Solid Tumors (RECIST) version 1.1, median duration of response, median progression free survival (mPFS) and mean treatment duration.

Line of Therapy

2nd Line

(n=31)

3rd Line

(n=28)

≥4th Line

(n=83)

ORR (confirmed responses only)

19%

14%

7%

Median Duration of Response

80 weeks

NE(1)

76 weeks

mPFS

46 weeks

36 weeks

24 weeks

Mean Treatment Duration(2)

56 weeks

58 weeks

45 weeks

(1) NE = not estimable; (2) Includes 64 patients who elected for intra-patient dose escalation from 150 mg QD to 150 mg twice daily (BID).

Data from GIST patients receiving ≥ 100 mg of ripretinib daily (n=178) in the ongoing Phase 1 study, as of an August 10, 2019 cutoff date, including 2nd line (n=37), 3rd line (n=31), and ≥4th line (n=110) patients were (a) ORR (confirmed responses only): 2nd line (22%), 3rd line (13%), ≥4th line (7%); (b) median duration of response: 2nd line (80 weeks), 3rd line (NE), ≥4th line (48 weeks); (c) mPFS: 2nd line (46 weeks), 3rd line (40 weeks), ≥4th line (24 weeks); (d) mean treatment duration (includes 72 patients who elected for intra-patient dose escalation to 150 mg BID): 2nd line (53 weeks), 3rd line (54 weeks), ≥4th line (48 weeks).

Ripretinib was generally well tolerated and the updated adverse events were consistent with previously presented Phase 1 data in patients with GIST. Grade 3 or 4 treatment-emergent adverse events (TEAEs) in >5% of patients were increase in lipase level (n=25; 18%), anemia (n=11; 8%), and abdominal pain (n=11; 8%).

DCC-3014

The Company’s Phase 1 study of DCC-3014 was designed to evaluate the safety, pharmacokinetics and pharmacodynamics of multiple doses of DCC-3014 in patients with advanced solid tumors. The Company expects to present preliminary data from initial tenosynovial giant cell tumor (TGCT) patients at the 2019 Connective Tissue Oncology Society (CTOS) Annual Meeting being held November 13-16 in Tokyo, Japan.

As of the data cut-off date of September 10, 2019, increasing doses of DCC-3014 were assessed in seven dose cohorts across 36 patients with advanced solid tumor tumors. This included one dose cohort that received 10 mg once daily and six dose cohorts that received a three to five day loading dose regimen at doses of up to 50 mg followed by a schedule of daily, once-weekly or twice-weekly maintenance dosing with DCC-3014.
Data demonstrated dose-proportional exposure for DCC-3014 and exposure to DCC-3014 was associated with an increase in plasma CSF1 and IL-34, rapid and sustained reduction of CD16+ monocytes in peripheral blood, and substantial decreases in CD163+ macrophages in tumor.
DCC-3014 was generally well-tolerated, with most treatment-emergent adverse events (TEAEs) Grade 1 or 2. Most common related TEAEs ≥10% were fatigue (n=6;17%), diarrhea (n=4; 11%), and nausea (n=4; 11%). Grade 3 or 4 related TEAEs occurred in 4 patients, which were grade 3 aspartate aminotransferase (AST) increase, grade 4 lipase increase, grade 3 amylase increase, and grade 3 colitis. Serious adverse events were reported in 17 patients; none of which were related to DCC-3014.
The dose escalation evaluation is ongoing to determine a recommended phase 2 dose for advanced solid tumors and diffuse-type TGCT.
A copy of each presentation is available at www.deciphera.com/science/presentation-publications/.

About Ripretinib

Ripretinib is an investigational tyrosine kinase switch control inhibitor that was engineered to broadly inhibit KIT and PDGFRα mutated kinases by using a unique dual mechanism of action that regulates the kinase switch pocket and activation loop. Ripretinib is currently in clinical development for the treatment of KIT and/or PDGFRα-driven cancers, including gastrointestinal stromal tumors, or GIST, systemic mastocytosis, or SM, and other cancers. Ripretinib inhibits initiating and secondary KIT mutations in exons 9, 11, 13, 14, 17, and 18, involved in GIST, as well as the primary D816V exon 17 mutation involved in SM. Ripretinib also inhibits primary PDGFRα mutations in exons 12, 14 and 18, including the exon 18 D842V mutation, involved in a subset of GIST. In June 2019, the U.S. FDA granted Fast Track Designation to ripretinib for the treatment of patients with advanced GIST who have received prior treatment with imatinib, sunitinib and regorafenib. For more information about the Company’s clinical trials with ripretinib, please visit www.clinicaltrials.gov.

Deciphera Pharmaceuticals has an exclusive license agreement with Zai Lab (Shanghai) Co., Ltd. for the development and commercialization of ripretinib in Greater China (MainlandChina, Hong Kong, Macau and Taiwan). Deciphera Pharmaceuticals retains development and commercial rights for ripretinib in the rest of the world.

About DCC-3014

DCC-3014 is an investigational, orally administered, potent and highly selective inhibitor of CSF1R. DCC-3014 was designed using the Company’s proprietary switch control kinase inhibitor platform to selectively bind to the CSF1R switch pocket. DCC-3014 has greater than 100-fold selectivity for CSF1R over other closely related kinases and has an even greater selectivity for CSF1R over approximately 300 other human kinases. CSF1R controls the differentiation and function of macrophages including Tumor Associated Macrophages (TAMs) whose density within certain tumors including cancers of the breast, cervix, pancreas, bladder and brain correlates with poor prognosis. Tumors induce TAMs to suppress a natural immune response mediated by cytotoxic T-cells, a type of lymphocyte that would otherwise eradicate the tumor; a process known as macrophage checkpoints. Through inhibition of CSF1R, DCC-3014 has in preclinical studies demonstrated potent macrophage checkpoint inhibition as both a single agent and in combination with PD1 inhibitors. DCC-3014 is currently being evaluated in a Phase 1 clinical study. For more information about the clinical trial design please visit www.clinicaltrials.gov (NCT03069469).

On October 29, 2019 CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody therapeutic technology platform, reported the initiation of the Phase 2 PROCLAIM (Probody Clinical Assessment In Man) CX-072-002 program evaluating the anti-PD-L1 Probody CX-072, in combination with the anti-CTLA-4 antibody, YERVOY (ipilimumab), in patients with relapsed or refractory melanoma (Press release, CytomX Therapeutics, OCT 29, 2019, View Source [SID1234549966]).

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"The initiation of this first Phase 2 study for CX-072 marks the ongoing advancement of our innovative pipeline of Probody therapeutics and reflects our vision for this novel checkpoint inhibitor to become a differentiated centerpiece of combination therapies in multiple cancer types," said Sean McCarthy, D.Phil., president, chief executive officer and chairman of CytomX Therapeutics.

"Patients whose melanoma has progressed despite prior treatment with checkpoint inhibition remain a significant unmet medical need. This exciting study leverages our unique technology platform to enable a more powerful combination therapy directed against the two best validated pathways in immuno-oncology and could represent a significant advance in outcomes for these patients who have few treatment options," said Amy Peterson, M.D., chief development officer of CytomX Therapeutics.

About the PROCLAIM-CX-072-002 Ipilimumab Combination Study

PROCLAIM-CX-072-002 is an open-label, multi-center Phase 2 clinical study (NCT03993379) that allows for the testing of CX-072 in combination with ipilimumab in patients with unresectable or metastatic melanoma whose disease has progressed or relapsed following treatment with a PD-1/PD-L1 immune checkpoint inhibitor. This study will assess the efficacy and tolerability of a fixed dose of 800 mg of CX-072 every three weeks in combination with ipilimumab at the full labelled combination dose and schedule of 3 mg/kg every three weeks for four cycles. CX-072 therapy will be continued once every two weeks after the completion of the combination phase until disease progression. The primary objective is overall response rate (ORR) as defined by Response Evaluation Criteria in Solid Tumors (RECIST) v1.1 with secondary objectives evaluating the safety and tolerability of CX-072. The cohort utilizes a Simon 2 Stage design with approximately 40 patients being enrolled into Stage 1 with additional patients being enrolled into Stage 2, pending the outcome of Stage 1. CytomX anticipates initial data from Stage 1 in 2020.

Additional information on this trial is available at ClinicalTrials.gov using the identifier NCT03993379.

Unmet Need in Relapsed Refractory Melanoma

Melanoma is a life-threatening form of skin cancer. The incidence of melanoma has been increasing over the last 40 years, with about 150K newly diagnosed patients across major markets in 2018. In the unresectable/metastatic setting, approximately 60% of melanoma patients will receive immune checkpoint blockade (~35% of BRAF+ patients and ~75% of BRAF WT) yet ~85% of those patients will progress. For patients with unresectable/metastatic melanoma who progress after PD-1/L1 therapy, there are limited treatment options available.

Updated Top-Line Results from the Phase 1 PROCLAIM-CX-072-001 Ipilimumab Combination Study

This open-label, dose-finding study evaluated CX-072 in combination with ipilimumab in patients with advanced solid tumors. Preliminary data from this trial was first presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2018 Annual Meeting and enrollment is complete. Updated data, as of an October 2019 snapshot, showed that among 27 evaluable patients who received ipilimumab (3, 6 or 10 mg/kg) combined with CX-072 (0.3, 1, 3 or 10 mg/kg), the disease control rate (stable disease or better) was 37%. Five patients achieved confirmed objective responses by RECIST v1.1 including one complete response, for an ORR of 19% in these heavily pretreated patients. The median duration of response was 14.6 months (1.9 – 21.2 months) with 4 of the 5 responders still on treatment as of the latest data snapshot.

The recommended combination dose for further investigation is 3 mg/kg of ipilimumab and 10 mg/kg of CX-072 (dose equivalent of 800 mg). This combination was generally well tolerated with no new safety signals observed. Of the 27 patients treated across all doses, Grade 3/4 treatment related adverse events (TRAE) were reported in 9 (33%) patients and Grade 3/4 immune related adverse events (irAEs) were reported in 6 (22%) patients. Of the 20 patients treated with ipilimumab at 3 mg/kg at varying doses of CX-072, Grade 3/4 TRAEs were reported in 5 (25%) patients and Grade 3/4 irAEs were reported in 3 (15%) patients.

The Company is preparing data from the Phase 1 PROCLAIM-CX-072-001 study for publication.

On October 29, 2019 Curis, Inc. (NASDAQ: CRIS), a biotechnology company focused on the development of innovative therapeutics for the treatment of cancer, reported that the Company will release its third quarter financial results on Tuesday, November 5, 2019, after the close of US markets (Press release, Curis, OCT 29, 2019, View Source [SID1234549965]). Management will host a conference call on the same day at 4:30 p.m. ET.

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To access the live conference call, please dial (888) 346-6389 from the United States or (412) 317-5252 from other locations, shortly before 4:30 p.m. ET. The conference call can also be accessed on the Curis website at www.curis.com in the ‘Investors’ section. A replay of the financial results conference call will be available on the Curis website shortly after completion of the call.

On October 29, 2019 Corvus Pharmaceuticals, Inc. (Nasdaq: CRVS), a clinical-stage biopharmaceutical company focused on the development and commercialization of precisely targeted oncology therapies, reported financial results for the third quarter ended September 30, 2019 (Press release, Corvus Pharmaceuticals, OCT 29, 2019, View Source [SID1234549964]).

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"There continues to be strong enrollment in all three of our ongoing clinical studies and we are scheduled to present data related to each at medical meetings during the fourth quarter," said Richard A. Miller, M.D., president and chief executive officer of Corvus. "The highlight will be the updated data from the CPI-006 study in an oral presentation at SITC (Free SITC Whitepaper) in November, which will add to the biologic and clinical evidence supporting CPI-006’s unique dual-mechanisms of action, both in monotherapy and in combination with ciforadenant. We will also provide updated data supporting the potential for our Adenosine Gene Signature to serve as a biomarker capable of identifying patients that are most likely to respond to therapies targeting the adenosine pathway. In December, we will present the first clinical data on CPI-818 in patients with T-cell lymphoma."

Recent Achievements
CPI-006: Immunomodulatory Anti-CD73 Antibody

Continued enrollment of up to 350 patients with advanced cancer in a Phase 1/1b adaptive design clinical trial evaluating CPI-006 as a single agent and in combination with ciforadenant or pembrolizumab.
Selected recommended dose of 18 mg/kg and initiated the disease expansion phase in the monotherapy arm of the study.
The trial also continues to enroll patients in the dose escalation phase in the ciforadenant combination arm of the study.
Ciforadenant (CPI-444): A2A Receptor Antagonist of Adenosine

Continued enrollment of patients with renal cell cancer (RCC) in an amended Phase 1b/2 clinical trial evaluating ciforadenant in combination with Genentech’s Tecentriq (atezolizumab), an anti-PD-L1 antibody. The RCC patients in the trial have failed treatments with anti-PD-(L)1 antibodies and tyrosine kinase inhibitors. This trial is also evaluating the use of a novel gene expression biomarker known as the Adenosine Signature, that may have the potential to predict patients most likely to respond to therapy and form the basis for future biomarker driven studies.
Began enrolling patients with prostate cancer in the amended Phase 1b/2 clinical trial to evaluate activity of ciforadenant and Tecentriq in this disease
CPI-818: A small molecule ITK inhibitor

Continued enrolling patients with T-cell lymphomas, including peripheral T-cell lymphoma (PTCL), cutaneous T-cell lymphoma (CTCL) and others, in a Phase 1/1b study with CPI-818, an ITK inhibitor.
Clinical study sites now open in the United States, Australia and South Korea.
Anticipated Upcoming Events

Updated clinical and immunologic data from the Phase 1/1b clinical trial of CPI-006 monotherapy and combination with ciforadenant is scheduled to be presented in an oral presentation at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) annual meeting in November 2019. This will build upon data presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting in June 2019 that support a new immuno-oncology approach with CPI-006 via activation of immune cells and the inhibition of adenosine production.
Updated data on the identification and role of biomarkers in adenosine pathway therapies, and specifically on the correlation of clinical activity with the Adenosine Gene Signature biomarker in renal cell cancer, is expected to be presented in a poster presentation at SITC (Free SITC Whitepaper).
Preclinical and early clinical data regarding CPI-818 is scheduled to be presented in a poster presentation in December 2019 at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. Additional data is expected to be presented in an oral presentation at the T-cell Lymphoma Forum in January 2020.
Updated clinical data from the amended Phase 1b/2 clinical trial of ciforadenant in combination with atezolizumab in prostate cancer is anticipated to be presented at the ASCO (Free ASCO Whitepaper) Genitourinary Cancers Symposium (GU) in February 2020.
Financial Results
At September 30, 2019, Corvus had cash, cash equivalents and marketable securities totaling $86.4 million, as compared to cash, cash equivalents and marketable securities of $114.6 million at December 31, 2018. Corvus expects full-year 2019 net cash used in operating activities to be between $38 and $40 million and estimates ending 2019 with cash, cash equivalents and marketable securities of between $75 and $77 million.

Research and development expenses for the three months ended September 30, 2019 totaled $9.0 million, as compared to $8.4 million for the same period in 2018. The increase of $0.6 million was primarily due to an increase in CPI-006 and CPI-818 program and related costs, partially offset by a reduction in ciforadenant program costs.

The net loss for the three months ended September 30, 2019 was $11.0 million, compared to a net loss of $10.5 million for the same period in 2018. Total stock compensation expense for the three months ended September 30, 2019 and 2018 was $1.8 million.

Conference Call Details
Corvus will host a conference call and webcast today, Tuesday, October 29, 2019, at 4:30 p.m. ET (1:30 p.m. PT), during which time management will provide a business update and discuss the third quarter 2019 financial results. The conference call can be accessed by dialing 1-800-458-4121 (toll-free domestic) or 1-720-543-0206 (international) and using the conference ID 8706779. The live webcast may be accessed via the investor relations section of the Corvus website. A replay of the webcast will be available on Corvus’ website for 90 days following the call.

About Corvus Pharmaceuticals
Corvus Pharmaceuticals is a clinical-stage biopharmaceutical company focused on the development and commercialization of precisely targeted oncology therapies. Corvus’ lead product candidates are ciforadenant (CPI-444), a small molecule inhibitor of the A2A receptor, and CPI-006, a humanized monoclonal antibody directed against CD73 that exhibits immunomodulatory activity and blockade of adenosine production. These candidates are being studied in ongoing Phase 1/1b and 1b/2 clinical trials in patients with a wide range of advanced solid tumors. Ciforadenant is being evaluated in a successive expansion cohort trial examining its activity both as a single agent and in combination with an anti-PD-L1 antibody. CPI-006 is being evaluated in a multicenter Phase 1/1b clinical trial as a single agent, in combination with ciforadenant, and with pembrolizumab. Corvus’ third clinical program, CPI-818, an oral, small molecule drug that has been shown to selectively inhibit ITK, is in a multicenter Phase 1/1b clinical trial in patients with several types of T-cell lymphomas. For more information, visit www.corvuspharma.com.

Tecentriq is a registered trademark of Genentech.

About Ciforadenant
Ciforadenant (CPI-444) is a small molecule, oral, checkpoint inhibitor designed to disable a tumor’s ability to subvert attack by the immune system by blocking the binding of adenosine in the tumor microenvironment to the A2A receptor. Adenosine, a metabolite of ATP (adenosine tri-phosphate), is produced within the tumor microenvironment where it may bind to the adenosine A2A receptor present on immune cells and block their activity. CD39 and CD73 are enzymes on the surface of tumor cells and immune cells. These enzymes work in concert to convert ATP to adenosine. In vitro and preclinical studies have shown that dual blockade of CD73 and the A2A receptor may be synergistic.

About CPI-006
CPI-006 is a potent humanized monoclonal antibody that reacts with the active site of CD73, blocking the conversion of AMP to adenosine. This antibody also possesses immunomodulatory activity resulting in activation of lymphocytes and effects on lymphocyte trafficking, which are independent of adenosine. In vitro studies of CPI-006 have shown it is capable of substantially inhibiting the production of adenosine by blocking the CD73 enzyme.

About CPI-818
CPI-818 is a small molecule drug given orally that has been shown to selectively inhibit ITK (interleukin-2-inducible T-cell kinase). It was developed to possess dual properties: to block malignant T-cell growth and modulate immune responses. ITK, an enzyme, is expressed predominantly in T-cells and plays a role in T-cell and natural killer (NK) cell lymphomas and leukemias, as well as in normal immune function. Interference with ITK signaling can modulate immune responses to various antigens. The inhibition of specific molecular targets in T-cells may be of therapeutic benefit for patients with T-cell lymphomas – similar to the role of Bruton’s tyrosine kinase (BTK) in B-cells. BTK is now an established target for treating various B-cell lymphomas, and two BTK inhibitors, ibrutinib and aclarabrutinib, have been approved by the U.S. Food and Drug Administration for lymphoma indications.

On October 29, 2019 Cellectis (Paris:ALCLS) (NASDAQ:CLLS) (Euronext Growth: ALCLS; Nasdaq: CLLS), a biopharmaceutical company focused on developing immunotherapies based on gene-edited off-the-shelf CAR T-cells (UCART), reported the Company has dosed the first patient in its UCARTCS1 clinical trial, MELANI-01, the first allogeneic off-the-shelf CAR-T product candidate the U.S. Food and Drug Administration (FDA) has cleared to enter into clinical development for relapsed/refractory multiple myeloma (R/R MM) (Press release, Cellectis, OCT 29, 2019, View Source [SID1234549963]). The UCARTCS1 clinical trial is a Phase 1 dose-escalation study to evaluate the safety, expansion, persistence and clinical activity of UCARTCS1 cells in R/R MM patients.

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"This first patient dosing for our MELANI-01 clinical trial is an important advancement, as our team has worked tirelessly to develop and take the CS1 target from the lab to the clinic," said Dr. André Choulika, Chairman and CEO, Cellectis. "In taking this next clinical step, we look forward to deepening our understanding of UCARTCS1 as a potential new treatment option for relapsed/refractory multiple myeloma patients in the future."

The MELANI-01 clinical trial is currently open at MD Anderson Cancer Center in Houston, Texas, under the supervision of Dr. Krina Patel, Principal Investigator, Study Coordinating Investigator, Assistant Professor, Department of Lymphoma/Myeloma, Division of Cancer Medicine at MD Anderson Cancer Center, as well as Hackensack Meridian in New Jersey under the supervision of Dr. David Siegel, Director of the Multiple Myeloma Institute at John Theurer Cancer Center (JTCC) at Hackensack University Medical Center. Another site is planned to open at Weill Cornell Medicine in New York under the leadership of Dr. Adriana Rossi, Associate Clinical Director, Myeloma Center and Assistant Professor of Medicine, Division of Hematology and Medical Oncology.

About Multiple Myeloma (MM)
Multiple myeloma is a cancer that affects a type of white blood cells called plasma cells that are specialized mature B cells, which secrete antibodies to combat infections. Multiple myeloma is characterized by the uncontrolled proliferation of neoplastic plasma cells in the bone marrow, where they overcrowd healthy blood cells. Although MM is a chronic disease and an exact cause has not yet been identified, researchers have made significant progress over the years in managing the disease through better understanding MM’s pathophysiology. The progress in finding a cure needs to be continued as The American Cancer Society estimates that 32,110 new cases of MM will be diagnosed, and 12,960 deaths are expected to occur in 2019 in the U.S. alone.

About UCARTCS1
UCARTCS1 is an allogeneic, off-the-shelf, gene-edited T-cell product candidate designed for the treatment of multiple myeloma. CS1 (SLAMF7) is highly expressed on MM tumor cells and is an attractive target. The limitation so far has been the presence of the CS1 target on the surface of T-cells, which has hindered the access to CAR-Ts. For example, the introduction of a CAR construct in T-cells induces cross T-cell reactivity and leads to destruction of the CS1+ T-cell population during manufacturing. Cellectis solved this issue by using TALEN gene editing to knock out the CS1 gene from T-cells before introducing the CS1 CAR construct.