On December 1, 2018 BeiGene, Ltd. (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, reported the presentation of clinical data from two ongoing trials of its investigational Bruton’s tyrosine kinase (BTK) inhibitor, zanubrutinib, in patients with mantle cell lymphoma (MCL) (Press release, BeiGene, DEC 1, 2018, View Source;p=irol-newsArticle&ID=2378923 [SID1234531783]). The presentations were made at the 60th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), taking place December 1-4, 2018 in San Diego, CA.

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Results from the pivotal Phase 2 trial of zanubrutinib in Chinese patients with relapsed or refractory (R/R) MCL (ClinicalTrials.gov Identifier: NCT03206970) were featured in an oral presentation, while updated results from the global Phase 1 trial of zanubrutinib in patients with multiple subtypes of B-cell malignancies, including treatment naïve (TN) and R/R MCL (ClinicalTrials.gov Identifier: NCT02343120), were featured in a poster presentation.

"Taken together, we believe that these two studies provide encouraging evidence for the use of zanubrutinib as a potential therapy in patients with MCL," said Jane Huang, M.D., Chief Medical Officer, Hematology, at BeiGene. "The results from 86 patients enrolled in our pivotal Phase 2 study in Chinese patients with R/R MCL presented today at ASH (Free ASH Whitepaper), provide a thorough look into the data included in our first new drug application (NDA) in China for zanubrutinib. Additionally, the results from 48 patients with MCL enrolled in our global Phase 1 study illustrated consistent outcomes for patients studied outside of China. We are excited by the prospect that zanubrutinib may be a differentiated BTK inhibitor with deep, durable responses for patients with MCL and potentially for other B-cell malignancies."

Zanubrutinib was discovered by BeiGene scientists, and is being developed globally as a monotherapy and in combination with other therapies to treat various hematologic malignancies. Zanubrutinib is being studied in several clinical trials as part of a broad development program and was granted Fast Track Designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with Waldenström macroglobulinemia (WM). BeiGene plans to submit an initial NDA to the FDA for zanubrutinib in 2019 or early 2020. The NDAs in China for R/R MCL and R/R chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL) have been accepted by the National Medical Products Administration (NMPA, formerly known as CFDA) and the MCL filing has been granted priority review.

"Zanubrutinib was shown to be highly active in Chinese patients with R/R MCL, as evidenced by a high rate of complete responses characterized by PET-based imaging. It was also generally well-tolerated, and we are hopeful of its potential to become a new treatment option for Chinese patients with MCL and potentially other forms of B-cell lymphomas," said Yuqin Song, M.D., Ph.D., Associate Professor of Medical Oncology, Deputy Director of the Lymphoma Department at Peking University Cancer Hospital in China, and presenter of results from the pivotal Phase 2 trial in Chinese patients.

"The outcomes observed in patients treated outside of China are generally consistent with the experiences observed in Chinese patients with R/R MCL. Importantly, the high response rates that were observed appear to extend to patients with both TN and R/R MCL," commented Constantine Tam, M.D., Disease Group Lead for Low Grade Lymphoma and Chronic Lymphocytic Leukemia at Peter MacCallum Cancer Center and Director of Hematology at St. Vincent’s Hospital, Australia, and lead author of the poster presentation of results from the global Phase 1 trial.

Summary of Clinical Results From the Pivotal Phase 2 Trial in China
Oral Presentation Data Included in BeiGene’s NDA in China for Zanubrutinib in MCL

This single arm, open-label, multi-center, pivotal Phase 2 trial of zanubrutinib as a monotherapy in Chinese patients with R/R MCL enrolled 86 patients who had received a median of two prior lines of therapy (1-4). Patients were treated with zanubrutinib, dosed at 160 mg orally twice-daily (BID). The primary endpoint of the trial was overall response rate (ORR) assessed by independent review committee (IRC) using PET-based imaging according to the Lugano Classification 2014.

As of March 27, 2018, 85 patients with R/R MCL were evaluable for efficacy and 65 patients (75.6%) remained on study treatment. The median follow-up time for patients enrolled in the trial was 35.9 weeks (1.1-55.9). Results included:

The ORR by IRC was 83.5 percent (71/85); the complete response (CR) rate was 58.8 percent (50/85) and the partial response (PR) rate was 24.7 percent (21/85);

The 24-week progression-free survival (PFS) was estimated at 82 percent. The median PFS had not yet been reached;

With 24.1 weeks median follow-up (0.1-41.1), the median duration of response (DOR) had not yet been reached and 90 percent of responders were still in response at 24 weeks;

Zanubrutinib tolerability was generally consistent with previous reports in patients with various B-cell malignancies and the majority of adverse events (AEs) were grade 1 or 2 in severity. The most frequent AEs of any attribution were neutrophil count decreased (31.4%), rash (29.1%), upper respiratory tract infection (29.1%), and platelet count decreased (22.1%);

The most frequently reported (in >5 percent of patients) grade 3 or higher AEs were neutrophil count decreased (11.6%) and lung infection (5.8%);

Four patients (4.7%) had treatment emergent adverse events (TEAEs) leading to death (one case each of traffic accident, cerebral hemorrhage, pneumonia, and unknown cause in the setting of infection); and

Among events of special interest for BTK inhibitors, diarrhea was observed in nine patients (10.5%), all grade 1-2. Major hemorrhage was observed in 1 patient (1.2%) with blastoid variant of MCL who had intra-parenchymal CNS bleeding. No cases of atrial fibrillation/flutter were reported in this trial.
Summary of Updated Clinical Results From the Global Phase 1 Trial

This open-label Phase 1 trial of zanubrutinib as a monotherapy in patients with different subtypes of B-cell malignancies, including MCL, is being conducted in Australia, New Zealand, the United States, Italy, and South Korea. As of July 24, 2018, 48 patients with TN (n=9) or R/R (n=39) MCL have been enrolled in the trial and the median follow-up time was 12.7 months (0.7-38.0). Forty-five patients including six with TN and 39 with R/R MCL, were evaluable for efficacy in this analysis, per the Lugano 2014 classification. At the time of the data cutoff, 26 patients remained on study treatment. Updated results included:

The ORR by investigator was 88.9 percent (40/45); the CR rate was 26.7 percent (12/45) and the PR rate was 62.2 percent (28/45). The majority of patients were assessed via CT-scan; PET scans were optional per trial protocol;

The median DOR was 16.2 months and the median PFS for R/R patients was 18.0 months (0.7-30.7);

Zanubrutinib tolerability was generally consistent with previous reports in patients with various B-cell malignancies and the majority of AEs were grade 1 or 2 in severity. The most frequent AEs of any attribution were petechia/purpura/contusion (33.3%), diarrhea (33.3%), upper respiratory tract infection (29.2%), fatigue (25.0%), and constipation (18.8%);

Grade 3-5 AEs occurred in 56.3 percent of patients. Grade 3-5 AEs of any attribution reported in > three patients included anemia (8.3%), major hemorrhage (6.3%), cellulitis (6.3%), myalgia (6.3%), neutropenia (6.3%), pneumonia (6.3%); and thrombocytopenia (6.3%);

Discontinuation due to AEs occurred in 18.8 percent of patients with all but one event (peripheral edema) determined to be unrelated to study drug; and

There were four deaths due to AEs, which were all determined by the investigators to be unrelated to zanubrutinib treatment.
Investor Webcast

Date and Time: Monday, December 3, 2018 at 20:00 PST (Tuesday, December 4 at 12:00 China Standard Time)
Webcast: A live webcast and replay of the event will be available on BeiGene’s investor website, View Source
About Mantle Cell Lymphoma
Lymphoma is a diverse group of malignancies that originates from B-, T- or NK- cells. Mantle cell lymphoma (MCL) is typically an aggressive form of non-Hodgkin lymphoma (NHL) that arises from B-cells originating in the "mantle zone." In 2013, the incidence of lymphoma was 4.2 per 100,000 and the mortality was 2.2 per 100,000 in mainland Chinai, making it the eleventh most common cancer and the tenth leading cause of cancer death.ii In the United States, about 70,800 new cases of NHL were expected in 2014, with MCL representing about six percent (about 4,200 cases) of all new cases of NHL in the United Statesiii. Mantle cell lymphoma usually has a poor prognosis, with a median survival of three to four years, although occasionally patients may have an indolent course.iv Frequently, mantle cell lymphoma is diagnosed at a later stage of disease.

About Zanubrutinib
Zanubrutinib (BGB-3111) is an investigational small molecule inhibitor of Bruton’s tyrosine kinase (BTK) that is currently being evaluated in a broad pivotal clinical program globally as a monotherapy and in combination with other therapies to treat various B-cell malignancies.

Clinical trials of zanubrutinib include a global Phase 1 trial; a fully-enrolled, global Phase 3 clinical trial in patients with Waldenström macroglobulinemia (WM) comparing zanubrutinib to ibrutinib, the currently approved BTK inhibitor for WM; a global Phase 3 clinical trial in patients with previously untreated chronic lymphocytic leukemia (CLL); a pivotal Phase 2 trial in patients with relapsed/refractory (R/R) follicular lymphoma in combination with GAZYVA (obinutuzumab); and a Phase 3 trial comparing zanubrutinib to ibrutinib in patients with R/R CLL/small lymphocytic lymphoma (SLL). In China, BeiGene has completed enrollment in two other pivotal Phase 2 clinical trials of zanubrutinib in patients with CLL/SLL and WM. New drug applications (NDA) for zanubrutinib in patients with R/R MCL and in patients with R/R CLL/SLL have been accepted by the National Medical Products Administration (NMPA, formerly known as CFDA) and the MCL filing has been granted priority review.

On December 1, 2018 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported late interim results from its BP-004 European registration trial, suggesting that adding rivo-celTM (rivogenlecleucel, formerly BPX-501) to stem cell transplants in patients who lack a matched donor may deliver comparable outcomes to matched unrelated donor (MUD) transplants (Press release, Bellicum Pharmaceuticals, DEC 1, 2018, View Source [SID1234531767]). The data were reviewed in a presentation today at the 60th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) (ASH 2018) by Mattia Algeri, M.D., Cell and Gene Therapy Unit, Ospedale Bambino Gesù, Rome, Lazio, Italy.

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The interim data show a 90.9 percent event-free survival (EFS) rate at 180 days for patients treated with rivo-cel (n=166) after a stem cell transplant from a haploidentical, or partially matched, donor. These results appear comparable to an interim evaluation of patients who received a MUD transplant in the concurrently-run observational study (n=91), demonstrating an 87.7 percent EFS rate at 180 days. The Company expects to report final comparison results from both study populations and file for approval in Europe in 2019. Demonstrating non-inferiority to MUD transplantation is the primary study objective required for rivo-cel product registration in Europe.

"While a stem cell transplant is an established, often curative treatment option for children with cancers and hereditary blood diseases, the majority of patients are unable to find an HLA-matched related donor, which is associated with the best outcomes," commented Dr. Algeri. "While matched unrelated donor transplants are an important option, only half of patients can identify a match. Further, it can take up to several months to do so, a delay that is problematic for many transplant patients. These data suggest that rivo-cel treatment may enable haploidentical stem cell transplant with overall outcomes similar to MUD, making it an attractive alternative for many of these patients."

Study Design and Highlights

The BP-004 trial is a prospective, multicenter, European Phase 1/2 trial in pediatric patients with malignant or nonmalignant disorders. Patients received rivo-cel within a few weeks following a haplo-transplant. No post-transplant Graft versus Host Disease (GvHD) prophylaxis was used. Patients who developed visceral GvHD or were refractory to standard of care treatments were eligible to receive rimiducid. The primary endpoint was EFS at day 180 (events included transplant-related mortality or non-relapse mortality for malignant patients, severe GvHD, and life-threatening infections).

Rivo-cel was well-tolerated, with few patients experiencing non-GvHD adverse events considered to be possibly or probably related to rivo-cel. Overall GvHD rates were low, with few cases of high-grade acute GvHD or chronic GvHD. The majority of patients with visceral GvHD or who were refractory to standard of care treatment for GvHD responded to rimiducid.

The observational comparator trial (C/CP-004) was designed to collect both prospective and retrospective data from pediatric patients with either malignant or nonmalignant disease who underwent a matched unrelated donor transplant during the same time period and at the same treatment centers where the BP-004 rivo-cel study was conducted.

"These interim data increase our confidence that rivo-cel may be an approvable new treatment option for transplant patients with leukemias, lymphomas and genetic blood diseases in Europe upon final data read-out in 2019," commented Rick Fair, Bellicum President & CEO. "If we continue to demonstrate that rivo-cel enables patients without a matched donor to achieve outcomes comparable to a MUD transplant, that would be very clinically meaningful and would fulfill a requirement for European approval. We look forward to reporting final six-month results for all patients in 2019. We also plan to continue to follow these patients to evaluate the impact of rivo-cel in preventing relapse and extending overall survival, particularly in patients with leukemia."

A copy of this ASH (Free ASH Whitepaper) presentation will be made available in the Abstracts & Presentations section of the Company’s website. The Company expects to report topline final results in early 2019 followed by publication of a comprehensive data set.

Analyst and Investor Luncheon Event and Webcast
Bellicum will host a live and webcast analyst and investor luncheon event on December 3, 2018 at 12:00 p.m. – 1:30 p.m. PST in San Diego, CA. Featured speakers include Dr. Alice Bertaina, Associate Professor of Pediatrics (Stem Cell Transplantation), Lucile Packard Children’s Hospital at the Stanford School of Medicine, as well as Bellicum senior management. A webcast replay of the event will be available in the News & Events section of the Bellicum website, and available for at least two weeks following the event.

About Rivo-cel (BPX-501)
Rivo-celTM (rivogenlecleucel) is an allogeneic polyclonal T cell product designed to reduce relapse of leukemia following a stem cell transplant. The cell treatment contains a diverse repertoire of T cells which may contribute to a robust graft vs. leukemia effect. Rivo-cel’s antiviral benefits may also reduce morbidity and mortality in patients susceptible to infection following a transplant. The product’s CaspaCIDe safety switch enables this approach by allowing physicians to reduce the number of alloreactive cells in the event of uncontrolled GvHD. Rivo-cel addresses a major unmet need in adult and pediatric leukemia, lymphoma and genetic blood disease patients following a haploidentical stem cell transplant.

On November 30, 2018 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to the development of programmed cellular immunotherapies for cancer and immune disorders, reported that the U.S. Food and Drug Administration (FDA) has allowed its Investigational New Drug (IND) Application for FT500, the Company’s universal, off-the-shelf natural killer (NK) cell product candidate derived from a clonal master induced pluripotent stem cell (iPSC) line (Press release, Fate Therapeutics, NOV 30, 2018, View Source [SID1234531751]). The clinical trial of FT500 is expected to be the first-ever clinical investigation in the U.S. of an iPSC-derived cell product.

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"The clearance by the FDA of our FT500 IND is a significant milestone and marks the beginning of an exciting new era for the clinical development of cell products," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "Clonal master iPSC lines are a renewable cell source that can uniquely produce cell products which are uniformly engineered and well characterized, can be mass produced in a cost-effective manner, and can be delivered off-the-shelf to treat many patients. This revolutionary paradigm overcomes significant challenges that limit both patient- and donor-derived cell therapy, where heterogeneous populations of primary cells are repeatedly sourced, engineered, expanded and characterized on a batch-by-batch basis resulting in cell therapies with substantial variability in quality, consistency and potency."

The Company plans to initiate first-in-human clinical testing of FT500 in combination with checkpoint inhibitor therapy for the treatment of advanced solid tumors. This study is expected to evaluate the safety and tolerability of multiple doses of FT500, in multiple dosing cycles with nivolumab, pembrolizumab or atezolizumab, in subjects that have progressed or failed on checkpoint inhibitor therapy.

On Monday, December 3, an oral presentation at the 60thAmerican Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition will feature new preclinical data demonstrating that FT500 has enhanced anti-tumor cytotoxicity and promotes T-cell activation and homing. Moreover, in an in vitro three-dimensional tumor spheroid model, FT500 in combination with activated T cells and an anti-PD1 antibody significantly enhance the elimination of target cancer cells, as compared to FT500 or activated T cells alone. Additional iPSC-derived, off-the-shelf cell product candidates from the Company’s iPSC product platform, including its first iPSC-derived chimeric antigen receptor (CAR) T-cell (FT819) and CAR NK cell (FT596) product candidates, will also be highlighted at ASH (Free ASH Whitepaper).

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary iPSC product platform enables mass production of off-the-shelf, engineered, homogeneous cell products that can be administered in repeat doses to mediate more effective pharmacologic activity, including in combination with cycles of 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 Company’s first-of-kind approach involves engineering human iPSCs in a one-time genetic modification event, and selecting a single 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 to treat many patients. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 100 issued patents and 100 pending patent applications.

On November 30, 2018 SELLAS Life Sciences Group, Inc. (Nasdaq: SLS) ("SELLAS" or the "Company"), a clinical-stage biopharmaceutical company focused on the development of novel cancer immunotherapies for a broad range of cancer indications, reported that further correlative data from the Phase 2b trial of nelipepimut-S (NPS, NeuVax) in combination with trastuzumab (Herceptin) for the treatment of women with triple-negative breast cancer (TNBC) will be presented in a poster presentation at the 2018 San Antonio Breast Cancer Symposium (SABCS), taking place December 4-8, 2018 in San Antonio, TX (Press release, Sellas Life Sciences, NOV 30, 2018, View Source;Herceptin-at-the-2018-San-Antonio-Breast-Cancer-Symposium/default.aspx [SID1234531752]). The data will include efficacy results from analysis of key predefined subgroups and the difference in the patterns of disease relapse between the active (NPS plus trastuzumab) versus the control (trastuzumab) arms.

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Details for the presentation are as follows:

Title: Subgroups analysis of a multicenter, prospective, randomized, blinded phase 2b trial of trastuzumab + nelipeptimut-S (NeuVax) vs trastuzumab for prevention of recurrence in breast cancer patients

Poster Session 2: Treatment: Immunotherapy (clinical)

Poster Hall Location: Hall 1

Poster Hall Hours: Thursday, December 6, 2018; 8:30 – 10:00 am ET

Abstract ID: P2-09-01

SELLAS previously announced that the full dataset, as well as correlations between clinical response and HLA type, from the Phase 2b trial of nelipepimut-S in combination with trastuzumab were presented in an oral presentation at the 2018 Annual Meeting of the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) on October 22, 2018 in Munich, Germany, and in a poster presentation at the 2018 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting on November 9 and 10, 2018 in Washington, D.C., respectively

On November 30, 2018 Adaptive Biotechnologies and its collaborators reported it will present 28 studies, including a late-breaker presentation, at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in San Diego, December 1-4, 2018 (Press release, Adaptive Biotechnologies, NOV 30, 2018, View Source [SID1234531753]). The data presented at ASH (Free ASH Whitepaper) builds on the recent FDA clearance of the clonoSEQ Assay to detect and monitor minimal residual disease (MRD) in patients with multiple myeloma or B-cell acute lymphoblastic leukemia (ALL), using DNA from a patient’s bone marrow sample.

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Among the 28 clonoSEQ studies at ASH (Free ASH Whitepaper), new research supports expanded use in myeloma and ALL, efficacy in other blood cancers like chronic lymphocytic leukemia (CLL) and Non-Hodgkin’s Lymphoma (NHL), and ability to detect MRD in blood-based samples. New data generated using clonoSEQ will be presented that demonstrate the value of a highly sensitive, standardized next-generation sequencing MRD test to determine early response to treatment and predict potential relapse in myeloma and ALL patients. Data will also be presented that look at the sensitivity of clonoSEQ and other technologies to assess MRD.

"This year has been a landmark year for minimal residual disease. It’s one of the first new endpoints we’ve seen in hematology clinical trials since progression-free survival," said Chad Robins, chief executive officer and co-founder of Adaptive Biotechnologies. "The volume and the quality of MRD data being presented at ASH (Free ASH Whitepaper) establish that MRD has firmly taken root as a clinical trial endpoint and biomarker that can help predict patient outcomes. With greater reliance on MRD in clinical trials, as well as a growing focus on monitoring MRD to inform patient care, having access to a highly sensitive, standardized test like clonoSEQ is paramount."

clonoSEQ, the first clinical application of Adaptive’s pioneering immune profiling platform, will be featured in a late-breaker presentation, 12 oral presentations and 15 posters. Data on approved, investigational and research uses will be presented across a range of cancers – 14 multiple myeloma, 4 ALL, 4 CLL, 4 mantle cell lymphoma, 1 diffuse large B-cell lymphoma, and 1 Hodgkin’s lymphoma.

Key highlights include:

Abstract Title Date, Time, Location
Multiple myeloma and ALL
Abstract #LBA-2, Late-Breaker Presentation

LBA-2: Phase 3 Randomized Study of Daratumumab Plus Lenalidomide and Dexamethasone (D-Rd) Versus Lenalidomide and Dexamethasone (Rd) in Patients with Newly Diagnosed Multiple Myeloma (NDMM) Ineligible for Transplant (MAIA) Tuesday, December 4, 7:30 AM PT, Hall AB, San Diego Convention Center
Abstract #156, Oral Presentation

One-Year Update of a Phase 3 Randomized Study of Daratumumab Plus Bortezomib, Melphalan, and Prednisone (D-VMP) Versus Bortezomib, Melphalan, and Prednisone (VMP) in Patients (Pts) with Transplant-Ineligible Newly Diagnosed Multiple Myeloma (NDMM): Alcyone Saturday, December 1, 1:15 PM PT, Grand Ballroom 7, Marriott Marquis San Diego Marina
Abstract #123, Oral Presentation

Ixazomib-Lenalidomide-Dexamethasone (IRd) Consolidation Following Autologous Stem Cell Transplantation in Patients with Newly Diagnosed Multiple Myeloma: A Large Multi-Center Phase II Trial Saturday, December 1, 10:00 AM PT, Grand Hall C, Manchester Grand Hyatt San Diego
Abstract #281, Oral Presentation

Multivariable Modeling of Disease and Treatment Characteristics of Adults with B-ALL in MRD-Negative CR after CD19 CAR-T Cells Identifies Factors Impacting Disease-Free Survival Sunday, December 2, 8:30 AM PT, Ballroom 20D, San Diego Convention Center
Abstract #1551, Poster Presentation

Molecular Detection of Minimal Residual Disease Precedes Morphological Relapse and Could be Used to Identify Relapse in Pediatric and Young Adult B-Cell Acute Lymphoblastic Leukemia Patients Treated with Tisagenlecleucel Saturday, December 1,
6:15 PM PT, Hall GH, San Diego Convention Center

Abstract #3272, Poster Presentation

Evaluation of Sustained Minimal Residual Disease (MRD) Negativity in Relapsed/Refractory Multiple Myeloma (RRMM) Patients (Pts) Treated with Daratumumab in Combination with Lenalidomide Plus Dexamethasone (D-Rd) or Bortezomib Plus Dexamethasone (D-Vd): Analysis of Pollux and Castor Sunday, December 2, 6:00 PM PT, Hall GH, San Diego Convention Center
Blood-based MRD Monitoring
Abstract #147, Oral Presentation

Circulating Tumor DNA Dynamics during Therapy Predict Outcomes in Mantle Cell Lymphoma Saturday, December 1, 12:30 PM PT, Pacific Ballroom 20, Marriott Marquis San Diego Marina
Abstract #3137, Poster Presentation

Undetectable-Minimal Residual Disease (U-MRD6) (10-6 sensitivity) Is Associated with Best Progression-Free Survival for Patients Who Achieve Bone Marrow Undetectable MRD4 (10-4 sensitivity) with First-Line FCR Sunday, December 2, 6:00 PM PT, Hall GH, San Diego Convention Center
About Minimal Residual Disease

Minimal residual disease (MRD), also referred to as measurable residual disease, refers to cancer cells that remain in the body after treatment for patients with lymphoid cancers. These cells can be present at levels undetectable by traditional morphologic methods, microscopic examination of blood, or a bone marrow or a lymph node biopsy.

MRD is used by physicians to detect and monitor disease burden in patients and to inform their treatment decisions. Clinical practice guidelines recommend assessing MRD at multiple time points during treatment and maintenance in MM and ALL, and guidelines for both diseases include NGS as a recommended testing method. The prognostic value of MRD assessment has been demonstrated in multiple lymphoid cancers. Controlled trials have shown that even small amounts of disease are profoundly significant for predicting a patient’s long-term clinical outcomes. Therefore, highly sensitive, standardized molecular technologies are needed for reliable detection of MRD.

Measurement of MRD is currently being evaluated as a way to measure efficacy in drug trials, with the potential to expedite the approval of emerging therapies.

About the clonoSEQ Assay

The Adaptive Biotechnologies clonoSEQ Assay has been granted De Novo designation by the FDA as an in vitro diagnostic (IVD) to detect and monitor minimal residual disease (MRD) in patients with multiple myeloma (MM) and B-cell acute lymphoblastic leukemia (ALL) using DNA from bone marrow samples. It identifies and quantifies specific DNA sequences found in malignant cells, allowing clinicians to monitor patients for changes in disease burden during and after treatment. This robust assay provides sensitive and accurate measurement of residual disease that allows physicians to predict patient outcomes, assess response to therapy over time, monitor patients during remission and detect potential relapse. The clonoSEQ Assay is a single-site assay performed at Adaptive Biotechnologies. It is also available as a CLIA-regulated laboratory developed test (LDT) service for use in other lymphoid cancers.

clonoSEQ was reviewed under the FDA’s De Novo premarket review pathway, a regulatory pathway for some low- to moderate-risk novel devices for which there is no legally marketed predicate device.

For important information about the FDA-cleared uses of clonoSEQ, including the full intended use, limitations, and detailed performance characteristics, please visit www.clonoSEQ.com/technical-summary