Japan’s MHLW Approves Invivoscribe’s LeukoStrat CDx FLT3 Mutation Assay as the CDx for Daiichi Sankyo’s Quizartinib for Treatment of Relapsed/Refractory FLT3-ITD AML. Expands Use to Include Specimens Collected in EDTA

On June 19, 2019 Invivoscribe is reported that on June 5th the Ministry of Health, Labor and Welfare (MHLW) approved our LeukoStrat CDx FLT3 Mutation Assay as the companion diagnostic for Daiichi Sankyo’s Quizartinib for the treatment of FLT3-ITD positive relapse/ refractory acute myeloid leukemia (AML) patients in Japan (Press release, Invivoscribe Technologies, JUN 19, 2019, View Source [SID1234537170]). At the same time the Japanese MHLW added approval in use of EDTA collection tubes to the existing approval of heparin collection tubes used with this assay.

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The QuANTUM-R study demonstrated that quizartinib resulted in a statistically significant improvement in overall survival (OS) compared to salvage chemotherapy when patients were selected with the LeukoStrat CDx FLT3 Mutation Assay. Mutations in the FLT3 gene are among the most important driver mutations in AML.

This milestone further establishes the LeukoStrat CDx FLT3 Mutation Assay as the international gold standard for comprehensive FLT3 assessment for critically ill AML patients.

This PCR-based, in vitro diagnostic test detects internal tandem duplication (ITD) mutations and tyrosine kinase domain (TKD) mutations D835 and I836 in the FLT3 gene in genomic DNA extracted from mononuclear cells obtained from peripheral blood or bone marrow aspirates of patients diagnosed with AML. This test, which is available worldwide, includes software that interprets data, generates standardized mutant/wildtype signal ratios for ITD and TKD mutations, and predicts response to multiple tyrosine kinase inhibitors.

"Once again, our Streamlined CDx program has demonstrated its effectiveness in accelerating submissions and approvals for our partners worldwide. Invivoscribe welcomes opportunities to partner with global pharmaceutical companies interested in developing and commercializing companion diagnostics, whether their therapies are targeting hematologic diseases or solid tumors." said Dr. Jeffrey Miller, Invivoscribe’s CSO and CEO.

The LeukoStrat test is available as a test menu service through Invivoscribe’s wholly-owned subsidiaries, LabPMM LLC (San Diego, CA, U.S.), LabPMM GmbH (Martinsried, Germany) and LabPMM GK (Kawasaki City, Japan). Greater than 95% of patient samples tested using the FDA-approved LeukoStrat CDx FLT3 Mutation Assay and selection of other CLIA-validated PCR-based capillary assays report out results within 48 hours of sample receipt at any of the LabPMM laboratories. LeukoStrat CDx FLT3 Mutation Assay kits are currently distributed in Japan, Europe, and Australia, and are planned for distribution in the United States and China in the future.

Gamida Cell and Lonza Establish Commercial Manufacturing Agreement for Omidubicel

On June 19, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, and Lonza (SWX: LONN), an integrated healthcare solutions provider, reported that the companies have entered into a strategic manufacturing agreement (Press release, Gamida Cell, JUN 19, 2019, View Source [SID1234537169]). The agreement provides for the future commercial production after potential FDA approval of omidubicel, Gamida Cell’s investigational advanced cell therapy currently in clinical development designed to enhance the life-saving benefits of hematopoietic stem cell (bone marrow) transplant. An international, randomized Phase 3 study of omidubicel in patients with hematologic malignancies is currently ongoing, and omidubicel has not yet been approved for marketing in the United States or any other jurisdiction.

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This agreement follows a successful multi-year clinical manufacturing relationship and provides Gamida Cell with a path to commercial supply of omidubicel. Under this multi-year agreement, Lonza will construct and dedicate production suites at its Geleen, NL site, for the anticipated commercial launch. Additionally, the agreement enables Gamida Cell to increase the number of dedicated production suites over time to ensure commercial supply. Gamida Cell also has the option of expanding further into Lonza’s global cell and gene therapy manufacturing network.

"Gamida Cell and Lonza have had a strong relationship for the clinical supply of omidubicel, and we are pleased to extend our relationship as we prepare to potentially bring omidubicel to patients in a commercial setting after potential FDA approval," stated Julian Adams, chief executive officer of Gamida Cell. "The ability to reliably provide an advanced cellular therapy to patients is critical, and this agreement provides Gamida Cell with access to a top-tier manufacturing site for the long-term commercial supply of omidubicel after potential FDA approval. Additionally, this agreement enables the supply of commercial product as we plan for the build out of Gamida Cell’s own commercial-scale cGMP manufacturing facility to augment production."

"This agreement is an example of our long-term manufacturing partnership capabilities and efforts to drive the industrialization of the cell therapy industry. Our cell therapy experience and expertise will enable us to best support Gamida Cell at this important phase in the development of omidubicel," said Alberto Santagostino, SVP, head of cell & gene technologies at Lonza. "We seek to partner with such innovative companies who are pioneering important new treatment options to patients and look forward to enabling Gamida Cell to deliver omidubicel at a commercial scale after potential FDA approval."

BIOGEN TO REPORT SECOND QUARTER 2019 FINANCIAL RESULTS JULY 23, 2019

On June 19, 2019 Biogen Inc. (Nasdaq:BIIB) reported it will report second quarter 2019 financial results Tuesday, July 23, 2019, before the financial markets open (Press release, Biogen, JUN 19, 2019, View Source [SID1234537168]).

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Following the release of the financials, the Company will host a live webcast with Biogen management from 8:00-9:00 am ET. To access the live webcast, please go to the investors section of Biogen’s website at View Source Following the live webcast, an archived version of the call will be available on the website.

BeiGene Announces Updated Results from Two Ongoing Clinical Trials of Zanubrutinib in Patients with Mantle Cell Lymphoma in Presentations at the 15th International Conference on Malignant Lymphoma (ICML)

On June 19, 2019 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 results from two ongoing clinical studies of its investigational BTK inhibitor zanubrutinib in patients with mantle cell lymphoma (MCL) in two presentations at the 15thInternational Conference on Malignant Lymphoma (ICML), taking place June 18-22, 2019 in Lugano, Switzerland (Press release, BeiGene, JUN 19, 2019, View Sourcenews-releases/news-release-details/beigene-announces-updated-results-two-ongoing-clinical-trials" target="_blank" title="View Sourcenews-releases/news-release-details/beigene-announces-updated-results-two-ongoing-clinical-trials" rel="nofollow">View Source [SID1234537167]).

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"With longer follow-up, we continue to be encouraged by the clinical results of zanubrutinib in patients with MCL. The deep, durable responses shown in these two presentations at ICML provide additional support for our new drug application in MCL in China, which is currently under priority review," said Jane Huang, M.D., Chief Medical Officer, Hematology, at BeiGene. "We hope that zanubrutinib will become an impactful treatment for patients with MCL and other B-cell malignancies."

Summary of Updated Clinical Results from the Pivotal Phase 2 Trial Being Conducted in China

This single-arm, open-label, multi-center, pivotal Phase 2 trial of zanubrutinib as a monotherapy in patients with relapsed/refractory (R/R) MCL (clinicaltrials.gov identifier: NCT03206970) is being conducted in China, and enrolled 86 patients who had received a median of two (1-4) prior lines of therapy. 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 the February 15, 2019 data cutoff, 52 patients (60.5%) remained on study treatment. The median follow-up time for patients enrolled in the trial was 18.4 months (0.3-23.5). Results included:

The investigator-assessed (INV) ORR was 83.7% (72/86); the complete response (CR) rate was 77.9% (67/86) and the partial response (PR) rate was 5.8% (5/86). At an earlier data cutoff in March 2018 (8.2 months median follow-up), the ORR, CR and PR were 84.7%, 72.9%, and 11.8% per investigator assessment, and 83.5%, 58.8%, and 24.7% per IRC assessment, respectively;
The 15-month progression-free survival (PFS) by investigator was estimated at 72.1%; median PFS follow-up was 19.1 months (0.0-22.3);
With 16.4 months median follow-up (2.3-19.5), the duration of response (DOR) by investigator at 15 months was 67.4%;
Zanubrutinib tolerability was generally consistent with previous reports of zanubrutinib treatment in patients with various B-cell malignancies. The majority of treatment-emergent adverse events (TEAEs) were grade 1 or 2 in severity, with the most frequently reported being neutrophil count decreased (44.2%), upper respiratory tract infection (34.9%), rash (33.7%), white blood cell count decrease (31.4%), and platelet count decrease (25.6%);
Grade ≧3 TEAEs were reported in 36 patients (41.9%), with the most frequently reported being neutrophil count decrease (18.6%), lung infection (7.0%), white blood cell count decrease (5.8%), and anemia (5.8%);
Five patients (5.8%) had TEAEs leading to death (one case each of pneumonia, cerebral hemorrhage, traffic accident, and two cases of death with unknown cause); and
Among TEAEs of special interest for BTK inhibitors, hypertension was reported in 13 patients (15.1%), petechiae/purpura/contusion in four patients (4.7%), and major hemorrhage in three patients (3.5%); no cases of atrial fibrillation/flutter, secondary primary malignancy, or tumor lysis syndrome were reported in this trial.
"Zanubrutinib demonstrated high activity in patients with R/R MCL, with 84% of patients achieving objective response and now an investigator-assessed complete response rate observed at 78%. The responses achieved have been generally well-tolerated as well," 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 the presenting author of the pivotal Phase 2 trial in Chinese patients.

Summary of Updated Clinical Results from the Global Phase 1/2 Trial

This open-label, multi-center Phase 1/2 trial of zanubrutinib as a monotherapy (clinicaltrials.gov identifier: NCT02343120) in patients with different subtypes of B-cell malignancies, including MCL, is being conducted in the United States, Australia, Italy, South Korea, New Zealand, and the United Kingdom.

As of the December 13, 2018 data cut-off, 53 patients with treatment naïve (TN, n=16) or R/R (n=37) MCL have been enrolled in the trial and the median follow-up time was 15.4 months (0.1-38.2). Forty-eight patients (all 37 R/R and 11 TN) were evaluable for efficacy with median follow-up time of 16.7 months (1.6-38.2) in this analysis, per the Lugano 2014 Classification. At the time of the data cutoff, 27 patients (13 TN and 14 R/R) remained on study treatment. Updated results included:

The investigator-assessed ORR was 85.4% (41/48); the CR rate was 29.2% (14/48) and the PR rate was 56.3% (27/48). The majority of patients were assessed via CT-scan; PET scan was optional per trial protocol;
The median DOR was 16.2 months (0.03-28.2) for all patients. The median PFS for patients with R/R MCL was 17.3 months;
The majority of adverse events (AEs) were grade 1 or 2 in severity. The most frequently reported AEs included contusion (39.6%), diarrhea (34.0%), upper respiratory tract infection (26.4%), constipation (22.6%), fatigue (22.6%), and rash (18.9%);
Grade ≧3 AEs were reported in 54.7% patients, with the most frequent being anemia (9.4%), myalgia (5.7%), cellulitis (5.7%), pleural effusion (5.7%), and pneumonia (5.7%);
Discontinuation due to AEs occurred in 18.9% patients with two determined to be related to study drug (one case each of peripheral edema and subdural hematoma); and
There were five deaths due to AEs, which were all determined by the investigators to be unrelated to zanubrutinib treatment.
"The updated results from this Phase 1/2 global trial suggested that zanubrutinib was generally well-tolerated and highly active in patients with MCL. These data support further evaluation of zanubrutinib in late-stage clinical studies," commented Constantine Tam, M.D., Disease Group Lead for Low Grade Lymphoma and Chronic Lymphocytic Leukemia at the 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 1/2 trial.

Mid-2019 Clinical Data Update Conference Call and Webcast Information:

BeiGene will host a conference call and webcast on Thursday, June 20 at 8:00 a.m. EDT. Investors and analysts are invited to join the conference call using the following dial-in information:
U.S. Toll-Free: +1 (844) 461-9930
U.S. Toll: +1 (478) 219-0535
Hong Kong Toll-Free: +852 800 279 19250
China Toll-Free: +86 800 914 686
Conference ID: 1790069

A live webcast of the conference call can be accessed from the investors section of BeiGene’s website at View Source or View Source An archived replay will be available two hours after the event for 90 days.

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 2015, about 88,200 new cases and 52,100 cancer deaths of lymphoma were expected in Mainland China, making it the 12th most common cancer and the 11th leading cause of cancer death.1 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 States.2 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.3 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) discovered by BeiGene scientists 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 fully-enrolled, global Phase 3 clinical trial in patients with Waldenström macroglobulinemia (WM) comparing zanubrutinib to ibrutinib, currently the only approved BTK inhibitor for WM; a global Phase 3 clinical trial in patients with previously untreated chronic lymphocytic leukemia (CLL)/small lymphocytic lymphoma (SLL); a pivotal Phase 2 trial in patients with relapsed/refractory (R/R) follicular lymphoma in combination with GAZYVA (obinutuzumab); a Phase 3 trial comparing zanubrutinib to ibrutinib in patients with R/R CLL/SLL; and a global Phase 1 trial. In China, BeiGene has completed two pivotal Phase 2 clinical trials of zanubrutinib in patients with R/R MCL and R/R CLL/SLL and the enrollment in the pivotal Phase 2 clinical trials in patients with WM.

Zanubrutinib has been granted by the U.S. Food and Drug Administration (FDA) Fast Track designation for the treatment of patients with WM, and Breakthrough Therapy designation for the treatment of adult patients with MCL who have received at least one prior therapy. The New Drug Applications (NDAs) in China for R/R MCL and R/R CLL/SLL have been accepted by the China National Medical Products Administration (NMPA) and granted priority review. BeiGene plans to submit its first NDA in the U.S. for zanubrutinib in 2019 or early 2020.

Gilead and Nurix Establish Strategic Collaboration to Develop Novel Therapies for Cancer and Other Diseases

On June 19, 2019 Gilead Sciences, Inc. (Nasdaq: GILD) and Nurix Therapeutics, Inc., a company discovering drugs that harness the body’s natural process to control protein levels, reported a global strategic collaboration to discover, develop and commercialize a pipeline of innovative targeted protein degradation drugs for patients with cancer and other challenging diseases (Press release, Gilead Sciences, JUN 19, 2019, View Source [SID1234537166]).

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This press release features multimedia. View the full release here: View Source

Dysregulated and/or mutated proteins play a central role in the development and progression of many human diseases. Nurix’s technology platform is focused on the manipulation of the ubiquitin system and its component E3 ligases, the key enzymes responsible for controlling protein levels in human cells.

Under the multi-year collaboration, Nurix will utilize its proprietary drug discovery platform to identify novel agents that utilize E3 ligases to induce degradation of specified drug targets and Gilead will have an option to license drug candidates directed to up to five targets resulting from the work. Nurix will retain the option to co-develop and co-detail up to two programs in the United States. The collaboration excludes Nurix’s lead degradation program, for which Nurix retains all rights.

"There are many molecular targets involved in disease pathways that have traditionally been challenging to manipulate using conventional approaches," said John McHutchison, A.O., M.D., Chief Scientific Officer and Head of Research and Development, Gilead Sciences. "Nurix’s innovative protein degradation discovery technology provides Gilead with a new strategy to interrogate these drug targets, as we continue to build a pipeline of small molecule therapeutics for patients with cancers and other diseases."

"Gilead is an ideal partner to help us bring potentially transformative treatments to patients," said Arthur T. Sands, M.D., Ph.D., Nurix’s Chief Executive Officer. "This partnership expands our ability to build our pipeline of novel targeted protein degradation drugs based on our established expertise in the field of protein homeostasis, while we continue to independently advance our lead programs into the clinic."

Under the terms of the agreement, Nurix will receive an upfront payment of $45 million and will be eligible to receive up to approximately $2.3 billion in total additional payments based on the successful completion of certain research, pre-clinical, clinical, regulatory and commercialization milestones as well as up to low double-digit tiered royalties on net sales. For those programs that Nurix opts in to co-develop and co-detail, the parties will split development costs as well as profits and losses 50/50 for the U.S., and Nurix will be eligible to receive royalties on ex-U.S. sales and reduced milestone payments.