On December 7, 2021 LiquidLung, Inc., a biotechnology company dedicated to radically improving the detection, diagnosis and treatment of pulmonary disease, reported that the U.S. Patent and Trademark Office (USPTO) has issued a Notice of Allowance for its patent application No. 17/003,775 for claims related to the non-invasive diagnosis of lung cancer (Press release, LiquidLung, DEC 7, 2021, View Source [SID1234596578]).

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The technology, which is centered around a comprehensive portfolio of novel RNA biomarkers, developed in concert with Liquid Biosciences, a leading bio-analytics provider and dedicated partner to LiquidLung. The technology utilizes various mathematical gene expression signatures that uniquely span early detection and confirmatory diagnosis of lung cancer, both pre- and post-imaging, as well as the diagnostic classification of several prevalent histological subtypes of the disease.

In an independent validation study the lung cancer technology was applied to a blood-based dataset comprised of fully adjudicated in vivo patient samples consisting of patients with confirmed lung cancer and those without lung cancer, the technology achieved 97% sensitivity and 85% specificity for lung cancer detection.

Further, the technology correctly detected 100% of patients with stage I, 89.9% with stage II, 100% with stage III and 100% with stage IV. In addition to detecting lung cancer generally across all stages of disease, the technology also achieved strong sensitivity and specificity for distinguishing between small cell lung cancer (SCLC), non-small cell lung cancer (NSCLC), adenocarcinoma, squamous cell carcinoma and large cell carcinoma. Notably, all cited performance was derived from the biomarkers alone, with absolutely no mathematical or scientific value being derived from patient demographics, nodule information, smoking history or any other clinical risk factors, which introduces a defined opportunity to improve upon the already impressive early data with little investment in R&D.

"Imagine a non-invasive testing platform that can rule more high-risk patients with lung cancer into LDCT imaging for further evaluation earlier, reduce negative biopsy procedures for patients with suspicious pulmonary nodules discovered incidentally or through routine screening, and also accelerate the time to life-saving interventions and personalized treatments," stated Founder and Chairman, Marty Keiser. "The comprehensive nature of our to-be patented technology enables us to accurately and efficiently unlock value for patients across the entire care continuum for lung cancer, all while fitting nicely into existing workflows and standards of care."

LiquidLung is one of three companies created out of IV BioHoldings (IVBH), a bio innovation studio known for its progressive approach to R&D, company creation, productization and commercialization. IVBH made headlines in October when it aligned the public debut of its first-in-category pipeline of RNA technologies with the announcement of a strategic lab partnership with P4 Diagnostix, intended to accelerate clinical development and validation of the IVBH assets within lung cancer, non-alcoholic fatty liver disease (NAFLD) and breast cancer.

"The recent worldwide recognition and appreciation for the clinical utility of RNA, from prevention to detection to treatment, has created a timely tailwind for IVBH as we begin to publicly unveil the vast intellectual property portfolio that we have been quietly and strategically amassing since 2018," stated IVBH Chief Commercial Officer, Elizabeth Cormier-May, who also leads the studio’s women’s health company, Mammogen. "The USPTO decision is a significant milestone for LiquidLung and further establishes IVBH’s position as a leader in the rapidly accelerating RNA revolution," Cormier-May continued.

IVBH is currently scaling operationally to support the lung cancer, NAFLD and breast cancer programs through near-term clinical milestones with multiple phases of clinical data generation expected throughout 2022 across all programs.

On December 7, 2021 Immunicom, Inc., a clinical-stage biotech pioneering subtractive therapies for advanced cancers, reported that promising preliminary data from a clinical trial investigating Immunopheresis therapy for resistant melanoma and other solid cancers at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 2021’s 36th annual meeting (Press release, Immunicom, DEC 7, 2021, View Source [SID1234596577]). The clinical trial is being conducted at Sheba Medical Center and uses Immunicom’s proprietary, subtractive LW-02 column to selectively remove immunosuppressive soluble tumor necrosis factor receptors (sTNF-Rs) via apheresis. By depleting TNF receptors from plasma, this therapy is designed to free the cytokine TNF-alpha, thereby unleashing its potent anti-cancer effects.

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The basket oncology clinical trial (NCT04142931; n =30 patients), which is being conducted under the direction of Dr. Ronnie Shapira, MD, and Prof. Gal Markel, MD PhD, is evaluating the safety and the clinical effectiveness of Immunopheresis as a monotherapy, and in combination with Bristol Myers Squibb’s anti-PD-1 immune checkpoint inhibitor, OPDIVO (nivolumab), in heavily pretreated patients. Preliminary data were presented from Part A (Immunopheresis therapy alone) from six patients—three with melanoma, and three with triple negative breast cancer (TNBC)—who have failed multiple prior lines of standard chemo and immunotherapies. Of these six heavily pretreated patients, three completed the 12-week study regimen, and three were withdrawn due to clinical progression. Two patients are still alive after a year (median overall survival 26.6 weeks). Immunopheresis therapy was well-tolerated in these late-stage patients, and no treatment-related serious adverse events were reported. The therapy was demonstrated to significantly reduce the levels of sTNF-Rs in plasma. Immune profiling of blood and tumor specimens showed evidence of upregulation of protein biomarkers and cell populations that are associated with improved activity of the immune system. Specifically, the tumor specimens showed two developments: 1) an increased infiltration of CD8+ T cells, which are known to have anti-tumor cytotoxic effects, and 2) the expression of immune checkpoint proteins PD1 and PD-L1 that show evidence of turning "cold" tumors into "hot" tumors. Part B of the study—evaluating Immunopheresis therapy in combination with nivolumab—is currently ongoing. The SITC (Free SITC Whitepaper) 2021 Congress abstract is available on Immunicom’s Publications page.

"Our preliminary data, which show renewed immune modulation and intra-tumoral antitumor activity, support our hypothesis that Immunopheresis therapy has the potential to overcome immunotherapy resistance, which could be a significant breakthrough in refractory cancer patient care," said Professor Gal Markel, former Director of the Ella Lemelbaum Institute for Immuno-Oncology, and now Director of Davidoff Center & Deputy Director General, Rabin Medical Center at Clalit Health Services. "Immunicom’s innovative subtractive treatment approach to neutralize cancer’s ability to block the patient’s natural immune-defense mechanisms is especially attractive in these heavily pretreated patients, and it offers the potential for achieving much better clinical outcomes with fewer treatment side effects," added Dr. Ronnie Shapira, a melanoma expert, and Head of the Onco-Gynecological Cancer Unit at the Ella Lemelbaum Institute for Immuno-Oncology.

Speaking to the announcement, Amir Jafri, Immunicom’s Founder and CEO, added, "The clinical trial at Sheba Medical Center is one of three ongoing, groundbreaking clinical trials to assess Immunicom’s subtractive therapies. Right now, our other clinical trials are evaluating the LW-02 column for treating additional multiple cancers, including metastatic TNBC, non-small cell lung cancer, melanoma, and renal cell carcinoma."

Immunicom’s various clinical trials are being conducted in collaboration with world-renowned research organizations and thought leaders, including:

Poland – at Jagiellonian University of Krakow Hospital, under the direction of Principal Investigator, Professor Piotr Wysocki, MD, PhD

Turkey – at Acıbadem Altunizade Hospital (Istanbul), a member of the Acıbadem/IHH Healthcare Group, under the direction of Principal Investigator, Prof. Dr. Gokhan Demir, MD, PhD

Subtractive Therapy – Immunopheresis and the LW-02 Column

Immunicom’s innovative Immunopheresis approach uses the LW-02 column to extract specific immune-suppressive cytokine receptors produced by cancer tumors. Selective removal of these targeted cytokine receptors is intended to neutralize cancer’s ability to block a patient’s natural immune defense mechanisms—which are significantly compromised in late-stage, metastatic disease—thereby re-energizing the immune system to aggressively fight the cancer. Immunopheresis is a "subtractive therapy" in contrast to drugs that are "additive." Subtractive therapy is designed to avoid the side effects, toxicity, and negative impact on a patient’s quality of life that are typical of other cancer treatments.

Based on Immunicom’s clinical program, the LW-02 column could be used either in combination with other therapies, or as a stand-alone treatment. The LW-02 Immunopheresis column has already received Breakthrough Device Designation for stage IV metastatic cancers from the U.S. Food and Drug Administration (FDA) and European regulatory clearance (CE Mark certification) for use in adults with advanced, refractory TNBC. Immunicom has obtained ISO 13485 certification for its manufacturing and related quality systems.

For an overview of Immunopheresis and how this breakthrough technology works, watch Immunicom’s How it Works video.

Immunopheresis and the LW-02 column is considered an investigational therapy by the U.S. FDA and other regulatory authorities. The clinical efficacy of the LW-02 column has not yet been demonstrated. Clinical investigations evaluating the clinical efficacy of the LW-02 column for TNBC are ongoing.

On December 7, 2021 Tempus, a leader in artificial intelligence and precision medicine, reported six abstracts accepted for poster sessions at the 2021 San Antonio Breast Cancer Symposium (SABCS) taking place December 7 – 10 (Press release, Tempus, DEC 7, 2021, View Source [SID1234596576]). The presented findings highlight the unique insights that Tempus’ data and smart diagnostics generate to advance breast cancer research.

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"The research that Tempus is presenting at this year’s symposium underscores the impact that our comprehensive genomic profiling data has on enabling clinicians to make more informed treatment decisions while also powering research for future therapeutics," said Dr. Kimberly Blackwell, Chief Medical Officer at Tempus.

Tempus will present six posters that highlight research to support the use of more personalized treatments for breast cancer patients, conducted with researchers from institutions including Rush University Medical Center, Columbia University Irving Medical Center, and Washington University School of Medicine. Three abstracts selected for poster presentation at SABCS 2021 are highlighted below:

PD14-01: Comprehensive molecular characterization of patients with metastatic invasive lobular carcinoma (ILC): Using real-world data to describe this unique clinical entity
Overview: This study characterizes the genomic and transcriptomic landscape of advanced or metastatic ILC and co-mutational landscape of CDH1-mutant disease. Researchers retrospectively analyzed 201 de-identified patients with ILC or mixed lobular/ductal histology who were tested via Tempus xT, with abstracted pathology reports. CDH1 mutations were present in 65.3% of all metastatic ILC cases. PIK3CA mutations were more common in patients with CDH1-mutant disease than in patients with CDH1-wildtype disease, suggesting that therapies targeting PIK3CA may be further investigated for their actionability in CDH1-mutant metastatic ILC.
Session: Spotlight Poster Discussion 14
Date: Friday, December 10, 2021
Time: 7:00 am – 8:30 am CT
P3-08-04: The genomic and transcriptomic landscape of PIK3R1-mutated breast cancers
Overview: This study investigates the significance of PIK3R1 mutations in metastatic breast cancer. Researchers retrospectively analyzed a de-identified cohort of 4,296 patients with breast cancer who underwent Tempus xT testing. Patients with PIK3R1 mutations have more frequent mutations associated with poor outcomes and endocrine therapy resistance (PTEN and NF1) compared with patients who are PIK3R1 wild type.
Session: Poster Session 3
Date: Thursday, December 9, 2021
Time: 7:00 am – 8:30 am CT
P3-09-04: Genomic landscape of HER2-negative advanced or metastatic breast cancer with PIK3CA gain-of-function mutations
Overview: The study provides insight into possible mechanisms of therapeutic resistance to alpelisib/fulvestrant and identifies potential targeted pathways. Researchers retrospectively analyzed a cohort of 2,918 patients with HER2-negative advanced or metastatic breast cancer with PIK3CA gain-of-function mutations sequenced with Tempus xT, xO or xE. From this cohort, researchers found that there is substantial genomic heterogeneity among PIK3CA-mutated HER2- advanced/metastatic breast cancers, however future studies are needed to assess the prognostic and predictive role of the identified co-mutations and other candidate gene alterations.
Session: Poster Session 3
Date: Thursday, December 9, 2021
Time: 7:00 am – 8:30 am CT
Additional Tempus-affiliated abstracts accepted for poster presentation at SABCS 2021 include:

PD2-01: A platform of CDK4/6 inhibitor-resistant patient-derived breast cancer organoids illuminates mechanisms of resistance and therapeutic vulnerabilities
Session: Spotlight Poster Discussion 2
Date: Wednesday, December 8, 2021
Time: 7:00 am – 8:30 am CT
GS3-09: Loss of ASXL1 tumor suppressor promotes resistance to CDK4/6 inhibitors in ER+ breast cancer
Session: General Session 3
Date: Thursday, December 9, 2021
Time: 10:45 am CT
P5-08-05: Preclinical activity of KB-0742, an oral, highlight selective, CDK9 inhibitor, in cell lines and in MYC-high expressing, patient-derived models of multiple breast cancer subtypes
Session: Poster Session 5
Date: Friday, December 10, 2021
Time: 7:00 am – 8:30 am CT
The complete list of Tempus authored and affiliated abstracts can be found on the Tempus publications page.

On December 7, 2021 Daiichi Sankyo reported that New data from the TROPION-PanTumor01 phase 1 trial of datopotamab deruxtecan (Dato-DXd), a TROP2 directed DXd antibody drug conjugate (ADC) being developed by Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) and AstraZeneca (LSE/STO/Nasdaq: AZN), continue to show encouraging durable tumor response and disease control in patients with metastatic triple negative breast cancer (TNBC) with disease progression following standard treatment (Press release, Daiichi Sankyo, DEC 7, 2021, View Source [SID1234596575]). These data were featured during an oral presentation (GS1-05) at the 2021 San Antonio Breast Cancer Symposium (#SABCS21).

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TNBC accounts for approximately 10 to 15% of breast cancer cases and is associated with higher disease recurrence and worse prognosis compared to other breast cancer subtypes.1,2,3 It is estimated that only 12.2% of patients with metastatic TNBC survive five years and median overall survival is generally less than two years.2,3

An objective response rate (ORR) of 34% was observed in 15 of 44 patients treated with datopotamab deruxtecan (6 mg/kg [n=42] and 8 mg/kg [n=2]) as assessed by blinded independent central review. Fourteen confirmed complete/partial responses (CR/PRs) with one additional CR/PR awaiting confirmation and stable disease in 17 additional patients were reported after a median follow-up of 7.6 months (range, 4 – 13 months). Median duration of response (DOR) was not reached (range, 2.7 – 7.4+ months) with the majority ongoing at the data cut-off of July 30, 2021. A disease control rate (DCR) of 77% was observed.

In a subgroup of 27 patients with measurable disease and previously untreated with a topoisomerase I inhibitor-based ADC, an ORR of 52% was reported with datopotamab deruxtecan. Thirteen confirmed CR/PRs with one additional CR/PR awaiting confirmation and stable disease were reported in nine additional patients after a median follow-up of 8.8 months (range, 4 – 13 months). A DCR of 81% was observed in this subgroup of patients.

"Despite recent advances in the treatment of triple negative breast cancer, a significant need remains to improve patient outcomes, underscoring the importance of developing new and effective therapies," said Ian E. Krop, MD, PhD, Associate Chief, Division of Breast Oncology, Susan F. Smith Center for Women’s Cancers, Dana Farber Cancer Institute. "These preliminary results with datopotamab deruxtecan in pre-treated patients with metastatic triple negative breast cancer are very encouraging and further evaluation of this TROP2 directed ADC is warranted."

The overall safety profile of datopotamab deruxtecan in TNBC in TROPION-PanTumor01 was consistent with what has been previously reported with no new safety signals. Treatment emergent adverse events (TEAEs) occurring in ≥15% of patients included nausea, stomatitis, vomiting, fatigue, alopecia, mucosal inflammation, constipation, headache, decreased lymphocyte count, decreased neutrophil count, pyrexia, anemia, pruritis, hypokalemia, diarrhea and cough. Grade 3 or greater treatment-related TEAEs occurred in 23% of patients. No cases of interstitial lung disease (ILD) were observed.

"These updated results continue to show the promise of datopotamab deruxtecan as a durable treatment strategy for patients with previously treated triple negative breast cancer, a historically difficult-to-treat form of breast cancer," said Gilles Gallant, BPharm, PhD, FOPQ, Senior Vice President, Global Head, Oncology Development, Oncology R&D, Daiichi Sankyo. "We are committed to further developing datopotamab deruxtecan and establishing where this specifically engineered TROP2 may be most effective in treating triple negative breast cancer."

"Patients with metastatic triple negative breast cancer face rapid disease progression with currently available treatment options and unmet need remains high for these patients," said Cristian Massacesi, MD, Chief Medical Officer and Oncology Chief Development Officer, AstraZeneca. "Based on these encouraging results of datopotamab deruxtecan in the triple negative breast cancer cohort of TROPION-PanTumor01, plans are underway to initiate a registrational phase 3 study."

Patients were treated with a median of three prior therapies in the metastatic setting (range, 1-10), including taxanes (91%), platinum-based chemotherapy (52%), immunotherapy (43%), topoisomerase I inhibitor-based ADCs (30%; sacituzumab govitecan, n=10; trastuzumab deruxtecan, n=2; patritumab deruxtecan, n=1) and PARP inhibitors (16%). As of data cut-off on July 30, 2021, 30% of patients remained on treatment with datopotamab deruxtecan.

Summary of TROPION-PanTumor01 TNBC Results

Efficacy Measure

All TNBC Patients
(n=44)i,

Patients without Prior Topoisomerase I-Based ADC and with Measurable Disease
(n=27)ii

ORR, %

34% (n=15)

52% (n=14)

(CR/PR) (confirmed)

n=14

n=13

(CR/PR) (pending confirmation)

n=1iii

n=1iii

SD, %

39% (n=17)

33% (n=9)

PD, %

18% (n=8)

15% (n=4)

DCR, %

77% (n=34)

81% (n=22)

CR; complete response; DCR, disease control rate; ORR, objective response rate; PD, progressive disease; PR, partial response; SD, stable disease

i Includes response evaluable patients who had ≥1 postbaseline tumor assessment or discontinued treatment. Postbaseline tumor assessments were not yet available for 2 patients at the data cut-off. Three patients were not confirmed to have a target lesion per BICR and therefore had a best overall response of non-CR/non-PD.

ii Includes response evaluable patients who had ≥1 postbaseline tumor assessment or discontinued treatment. Postbaseline tumor assessments were not

yet available for 1 patient at the data cut-off.

iii Includes patients with an unconfirmed response but are ongoing treatment.

About TROPION-PanTumor01
TROPION-PanTumor01 is a first-in-human, open-label, two-part, multicenter phase 1 trial evaluating the safety, tolerability and preliminary efficacy of datopotamab deruxtecan in patients with advanced solid tumors refractory to or relapsed from standard treatment or for whom no standard treatment is available, including non-small cell lung cancer (NSCLC), TNBC, hormone receptor (HR) positive/HER2 negative breast cancer, small cell lung cancer, urothelial, gastric and esophageal cancer.

The dose escalation part of the study, which was limited to patients with NSCLC, assessed the safety and tolerability of increasing doses of datopotamab deruxtecan to determine the maximum tolerated dose and/or recommended dose for expansion. The dose expansion part of the study further assessed the safety and tolerability of datopotamab deruxtecan in patients with additional solid tumors. Based on the preliminary efficacy and safety profile, the 6 mg/kg dose has been chosen for further development.4,5

Safety endpoints include dose limiting toxicities and serious adverse events. Efficacy endpoints include ORR, DCR, DOR, time to response, progression-free survival and overall survival. Pharmacokinetic, biomarker and immunogenicity endpoints also are being evaluated.

About Triple Negative Breast Cancer
Approximately 10 to 15% of breast cancers are considered triple negative, a breast cancer subtype that is defined by tumors that test negative for estrogen and progesterone hormone receptors (HRs) as well as human epidermal growth factor 2 receptor (HER2).1,2,6 An estimated 260,000 new cases of TNBC were reported globally in 2018 with it being more common in younger women and those who are Black.1,2,7 Compared to patients with other breast cancer subtypes, prognosis for patients with metastatic TNBC is generally worse and the disease is more likely to recur following treatment with initial chemotherapy.1,3 Five-year survival of metastatic TNBC is estimated at 12.2% and median overall survival is generally less than two years.2,3

About TROP2
TROP2 (trophoblast cell-surface antigen 2) is a transmembrane glycoprotein overexpressed in several types of solid tumors, including breast cancer.8 TROP2 overexpression has been detected in multiple breast cancer subtypes, including approximately 80% of patients with TNBC.9,10,11 High TROP2 expression is an unfavorable prognostic factor for overall survival in all types of breast cancer.12

About Datopotamab Deruxtecan (Dato-DXd)
Datopotamab deruxtecan (Dato-DXd) is an investigational TROP2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, datopotamab deruxtecan is one of three lead ADCs in the oncology pipeline of Daiichi Sankyo, and one of the most advanced programs in AstraZeneca’s ADC scientific platform. Datopotamab deruxtecan is comprised of a humanized anti-TROP2 IgG13 monoclonal antibody, developed in collaboration with Sapporo Medical University, attached to a topoisomerase I inhibitor payload, an exatecan derivative, via a tetrapeptide-based cleavable linker.13

A comprehensive development program called TROPION is underway globally with trials evaluating the efficacy and safety of datopotamab deruxtecan across multiple solid tumors, including NSCLC, TNBC, HR positive/HER2 negative breast cancer, small cell lung cancer, urothelial, gastric and esophageal cancer. Trials in combination with other anticancer treatments, such as immunotherapy, are also underway.

About the Daiichi Sankyo and AstraZeneca Collaboration
Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialize datopotamab deruxtecan in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights. Daiichi Sankyo is responsible for the manufacturing and supply of datopotamab deruxtecan.

About Daiichi Sankyo in Oncology
The oncology portfolio of Daiichi Sankyo is powered by our team of world-class scientists that push beyond traditional thinking to create transformative medicines for people with cancer. Anchored by our DXd antibody drug conjugate (ADC) technology, our research engines include biologics, medicinal chemistry, modality and other research laboratories in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in the U.S. We also work alongside leading academic and business collaborators to further advance the understanding of cancer as Daiichi Sankyo builds towards our ambitious goal of becoming a global leader in oncology by 2025.

On December 7, 2021 Legend Biotech Corporation (NASDAQ: LEGN), a global, clinical-stage biotechnology company developing and manufacturing novel therapies, reported the submission of a New Drug Application (NDA) to the Japanese Ministry of Health, Labour and Welfare (MHLW) for ciltacabtagene autoleucel (cilta-cel) by its collaboration partner, Janssen Pharmaceutical K.K. (Janssen) (Press release, Legend Biotech, DEC 7, 2021, View Source [SID1234596574]). Cilta-cel is an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR)-T cell therapy for the treatment of adults with relapsed or refractory multiple myeloma who have received at least three prior therapies, including a proteasome inhibitor (PI), an immunomodulatory agent (IMiD), and an anti-CD38 antibody.

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The submission is based on results from the Phase 1b/2 CARTITUDE-1 study conducted in the US and Japan, which evaluated the efficacy and safety of cilta-cel for patients with relapsed or refractory multiple myeloma.1 Cilta-cel is currently under regulatory review by several health authorities around the world, including the United States and Europe.

"Today’s submission is an encouraging step in our mission to provide a potentially transformative cell therapy option to patients with multiple myeloma," said Ying Huang, PhD, CEO and CFO of Legend Biotech. "We look forward to closely collaborating with our partner Janssen and the MHLW in order to make cilta-cel available to patients living with relapsed or refractory multiple myeloma, who have exhausted several standard-of-care treatments and are facing poor prognoses."

About Ciltacabtagene Autoleucel

Cilta-cel is an investigational chimeric antigen receptor T cell (CAR-T) therapy, formerly identified as JNJ-4528 in the U.S. and Europe and LCAR-B38M CAR-T cells in China, that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and in earlier lines of treatment. The design consists of a structurally differentiated CAR-T with two BCMA-targeting single domain antibodies. In December 2017, Legend Biotech, Inc. entered into an exclusive worldwide license and collaboration agreement with Janssen Biotech, Inc. (Janssen) to develop and commercialize cilta-cel. In addition to a Breakthrough Therapy Designation (BTD) granted in the U.S. in December 2019, cilta-cel received a Priority Medicines (PRiME) designation from the European Commission in April 2019, and a BTD in China in August 2020. In addition, Orphan Drug Designation was granted for cilta-cel by the U.S. FDA in February 2019, and by the European Commission in February 2020. A Biologics License Application seeking approval of cilta-cel was submitted to the U.S. FDA and a Marketing Authorization Application was submitted to the European Medicines Agency.

About the CARTITUDE-1 Study

CARTITUDE-1 (NCT03548207) is a Phase 1b/2, open-label, multicenter study evaluating the safety and efficacy of cilta-cel in adults with relapsed or refractory with multiple myeloma who have received at least 3 prior lines of therapy or are double refractory to a proteasome inhibitor (PI) and immunomodulatory drug (IMiD), received a PI, an IMiD, and anti-CD38 antibody and documented disease progression within 12 months of starting the most recent therapy.1

About Multiple Myeloma

Multiple myeloma, an incurable blood cancer that starts in the bone marrow and is characterized by an excessive proliferation of plasma cells.2 In Japan, there were more than 7,000 new cases of multiple myeloma and nearly 5,000 deaths.3 Although treatment may result in remission, most patients experience relapse.4 Relapsed myeloma is when the disease has returned after a period of initial, partial or complete remission and does not meet the definition of being refractory.5 Refractory multiple myeloma is when a patient’s disease is non-responsive or progresses within 60 days of their last therapy.6,7 While some patients with multiple myeloma have no symptoms at all, most patients are diagnosed by symptoms that can include bone problems, low blood counts, calcium elevation, kidney problems or infections.8 Patients who relapse after treatment with standard therapies, including protease inhibitors and immunomodulatory agents, have poor prognoses and few treatment options available.9