Genmab Announces Positive Topline Results in Phase III ANDROMEDA Study of Daratumumab in Light-chain (AL) Amyloidosis

On May 28, 2020 Genmab A/S (Nasdaq: GMAB) reported positive topline results from the Phase III ANDROMEDA (AMY3001) study of subcutaneous (SC) daratumumab in combination with cyclophosphamide, bortezomib and dexamethasone (CyBorD) for patients with newly diagnosed light-chain (AL) amyloidosis (Press release, Genmab, MAY 28, 2020, View Source [SID1234558618]). The study, conducted by Janssen Biotech, Inc. (Janssen), met the primary endpoint of percentage of patients with hematologic complete response. Patients in the study treated with daratumumab in combination with CyBorD had a 53.3% hematologic complete response compared to 18.1% of patients who were treated with CyBorD alone (odds ratio of 5.1 (95% CI 3.2 – 8.2, p<0.0001)).

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Overall, the safety profile of daratumumab SC in combination with CyBorD is consistent with the known safety profile of the CyBorD regimen and the known safety profile of daratumumab.

"We are very pleased with the topline results from the Phase III ANDROMEDA study in AL amyloidosis. We believe the data supports the potential of daratumumab in the treatment of this devastating, progressive disease, for which no approved treatments are available," said Jan van de Winkel, Ph.D., Chief Executive Officer of Genmab.

Janssen, which obtained an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab in 2012, will discuss with health authorities the potential for a regulatory submission for this indication.

About the ANDROMEDA (AMY3001) study

The Phase III study (NCT03201965) included 388 patients newly diagnosed with AL amyloidosis. Patients were randomized to receive treatment with either subcutaneous daratumumab in combination with cyclophosphamide (a chemotherapy), bortezomib (a proteasome inhibitor) and dexamethasone (a corticosteroid) or treatment with cyclophosphamide, bortezomib and dexamethasone alone. The primary endpoint of the study is the percentage of patients who achieve hematologic complete response.

About Light-chain (AL) Amyloidosis

Amyloidosis is a disease that occurs when amyloid proteins, which are abnormal proteins, accumulate in tissues and organs. When the amyloid proteins cluster together, they form deposits that damage the tissues and organs. AL amyloidosis most frequently affects the heart, kidneys, liver, nervous system and digestive tract. There is currently no cure or existing approved therapies for AL amyloidosis though it can be treated with chemotherapy, dexamethasone, stem cell transplants and supportive therapies.1 It is estimated that there are approximately 3,000 to 4,000 new cases of AL amyloidosis diagnosed annually in the U.S.2

About DARZALEX (daratumumab)

DARZALEX (daratumumab) intravenous infusion is indicated for the treatment of adult patients in the United States: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple

LEI Code 529900MTJPDPE4MHJ122

Genmab Announces Positive Topline Results in Phase III ANDROMEDA Study of Daratumumab in Light-chain (AL) Amyloidosis

myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of patients with multiple myeloma who have received at least one prior therapy; in combination with pomalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor (PI); and as a monotherapy for the treatment of patients with multiple myeloma who have received at least three prior lines of therapy, including a PI and an immunomodulatory agent, or who are double-refractory to a PI and an immunomodulatory agent.3 DARZALEX is the first monoclonal antibody (mAb) to receive U.S. Food and Drug Administration (U.S. FDA) approval to treat multiple myeloma. DARZALEX intravenous infusion is indicated for the treatment of adult patients in Europe: in combination with bortezomib, thalidomide and dexamethasone as treatment for patients newly diagnosed with multiple myeloma who are eligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; for use in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone, for the treatment of adult patients with multiple myeloma who have received at least one prior therapy; and as monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, whose prior therapy included a PI and an immunomodulatory agent and who have demonstrated disease progression on the last therapy4. The option to split the first infusion of DARZALEX over two consecutive days has been approved in both Europe and the U.S. In Japan, DARZALEX intravenous infusion is approved for the treatment of adult patients: in combination with lenalidomide and dexamethasone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with bortezomib, melphalan and prednisone for the treatment of patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant; in combination with lenalidomide and dexamethasone, or bortezomib and dexamethasone for the treatment of relapsed or refractory multiple myeloma. DARZALEX is the first human CD38 monoclonal antibody to reach the market in the United States, Europe and Japan. For more information, visit www.DARZALEX.com.

DARZALEX FASPRO (daratumumab and hyaluronidase-fihj), a subcutaneous formulation of daratumumab, is approved in the United States for the treatment of adult patients with multiple myeloma: in combination with bortezomib, melphalan and prednisone in newly diagnosed patients who are ineligible for ASCT; in combination with lenalidomide and dexamethasone in newly diagnosed patients who are ineligible for ASCT and in patients with relapsed or refractory multiple myeloma who have received at least one prior therapy; in combination with bortezomib and dexamethasone in patients who have received at least one prior therapy; and as monotherapy, in patients who have received at least three prior lines of therapy including a PI and an immunomodulatory agent or who are double-refractory to a PI and an immunomodulatory agent.5 DARZALEX FASPRO is the first subcutaneous CD38-directed antibody approved in the U.S. for the treatment of multiple myeloma.

Daratumumab is a human IgG1k monoclonal antibody (mAb) that binds with high affinity to the CD38 molecule, which is highly expressed on the surface of multiple myeloma cells. Daratumumab triggers a person’s own immune system to attack the cancer cells, resulting in rapid tumor cell death through multiple immune-mediated mechanisms of action and through immunomodulatory effects, in addition to direct tumor cell death, via apoptosis (programmed cell death).3,5,6,7,8,9

Daratumumab is being developed by Janssen Biotech, Inc. under an exclusive worldwide license to develop, manufacture and commercialize daratumumab from Genmab. A comprehensive clinical development program for daratumumab is ongoing, including multiple Phase III studies in smoldering, relapsed and refractory and frontline multiple myeloma settings. Additional studies are ongoing or planned to assess the potential of daratumumab in other malignant and pre-malignant diseases in which CD38 is expressed, such as amyloidosis and T-cell acute lymphocytic leukemia (ALL). Daratumumab has received two Breakthrough Therapy Designations from the U.S. FDA for certain indications of multiple myeloma, including as a monotherapy for heavily pretreated multiple myeloma and in combination with certain other therapies for second-line treatment of multiple myeloma.

bluebird bio Announces Live Webcast of EHA Data Review and June Investor Events

On May 28, 2020 bluebird bio, Inc. (NASDAQ: BLUE) reported that the company will host a live webcast to review new data presented at the European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting on Friday, June 12 at 8:00 am ET (Press release, bluebird bio, MAY 28, 2020, View Source [SID1234558634]).

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Investors may listen to the call on June 12, 2020 at 8:00 am ET by dialing (844) 825-4408 from locations in the United States or +1 (315) 625-3227 from outside the United States. Please refer to conference ID number 2796099.

In addition, members of the management team will participate in the following upcoming investor conferences:

Jefferies Virtual Healthcare Conference, Thursday, June 4, at 3:30 pm ET
Goldman Sachs 41st Annual Global Healthcare Conference, Wednesday, June 10, at 9:40 am ET
To access the live webcasts of bluebird bio’s presentations, please visit the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source Replays of the webcasts will be available on the bluebird bio website for 90 days following the events.

Bio-Techne To Present At The Jefferies Virtual Healthcare Conference

On May 27, 2020 Bio-Techne Corporation (NASDAQ:TECH) reported that Chuck Kummeth, President and Chief Executive Officer, will present at the virtual Jefferies Virtual Healthcare Conference on Tuesday, June 2, 2020 at 10:30 a.m. EDT (Press release, Bio-Techne, MAY 27, 2020, View Sourcenews/detail/193/bio-techne-to-present-at-the-jefferies-virtual-healthcare-conference" target="_blank" title="View Sourcenews/detail/193/bio-techne-to-present-at-the-jefferies-virtual-healthcare-conference" rel="nofollow">View Source [SID1234558528]). A live webcast of the presentation can be accessed via Bio-Techne’s Investor Relations website at View Source or through the following link View Source

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Iovance Reports Pivotal Cohort 4 Data for Tumor Infiltrating Lymphocyte (TIL) Therapy Lifileucel from C-144-01 Clinical Study in Advanced Melanoma

On May 27, 2020 Iovance Biotherapeutics, Inc. (NASDAQ: IOVA), a late-stage biotechnology company developing novel T cell-based cancer immunotherapies, reported initial data from pivotal Cohort 4 and updated long-term data from Cohort 2 in the C-144-01 study of lifileucel in advanced melanoma (Press release, Iovance Biotherapeutics, MAY 27, 2020, View Source [SID1234558544]).

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"We are very pleased to announce our pivotal Cohort 4 early data from the C-144-01 clinical study in advanced melanoma today," said Maria Fardis, Ph.D., President and Chief Executive Officer of Iovance Biotherapeutics. "The data from the first 68 patients in Cohort 4, with a 32.4% overall response rate (ORR) at 5.3 months of median study follow up, is highly consistent with what we have observed in Cohort 2 with comparable study follow up. For Cohort 2, median duration of response has not been reached at 18.7 months of study follow up. Available care for metastatic melanoma patients enrolled into our program is chemotherapy, which has been reported to offer a 4-10% response rate with a very short median duration of response. Together, these early data continue to support the potential benefit of the one-time administration of lifileucel TIL therapy in advanced melanoma patients."

Interim Pivotal Cohort 4 Results

Initial results from the pivotal Cohort 4 in the C-144-01 clinical study is available for 68 patients with two radiological assessments, as determined by investigator. Primary endpoint for the C-144-01 Cohort 4 study is ORR by independent review committee and secondary endpoint is ORR by investigator. Lifileucel shows a 32.4% overall response rate (1 complete response and 21 partial responses, 2 of which are yet to be confirmed with follow up visits) and a disease control rate of 72.1% as of the data cut off of 16 Mar 2020. This data is consistent with what was noted in Cohort 2 at 6 months of median study follow up. The ORR was 33% as reported in SITC (Free SITC Whitepaper) 2018 abstract.

The Cohort 4 metastatic melanoma patients have a similar patient characteristic profile to Cohort 2 with high baseline disease burden. They have progressed on multiple prior therapies, including anti-PD-1 and BRAF/MEK inhibitors. The adverse event profile was consistent with Cohort 2 and with the underlying advanced disease, lymphodepletion and IL-2 regimens. Cohort 4 dosed a total of 89 patients, and additional updates on Cohort 4 will be presented at upcoming medical meetings. Iovance remains on track to submit a biologics licensing application (BLA) for lifileucel in late 2020.

Updated Cohort 2 Results

Updated results from Cohort 2 will be presented at the ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program during an oral abstract session titled, "Long-term follow up of lifileucel (LN-144) cryopreserved autologous tumor infiltrating lymphocyte therapy in patients with advanced melanoma progressed on multiple prior therapies." In this Cohort 2 data, lifileucel shows a 36.4% overall response rate (2 complete responses and 22 partial responses) and a disease control rate of 80% (n=66) as assessed by investigators. Median duration of response (DOR) was not reached at 18.7 months of median study follow up (2.2 to 26.9+ months). Durable responses have been observed across a wide age range in metastatic melanoma patients who have received prior anti-CTLA-4 and BRAF targeted treatments, regardless of BRAF mutation status, and equally in patients with PD-L1 high and low status.

The Cohort 2 melanoma patients are heavily pretreated with high baseline disease burden. They have progressed on multiple prior therapies (3.3 mean prior therapies), including anti-PD-1 and BRAF/MEK inhibitors. The adverse event profile was consistent with the underlying advanced disease, lymphodepletion and IL-2 regimens.

The oral abstract session at ASCO (Free ASCO Whitepaper)20 will be available on demand in the ASCO (Free ASCO Whitepaper) Meeting Library at View Source Details of the presentation are as follows:

Title: Long-term follow up of lifileucel (LN-144) cryopreserved autologous tumor infiltrating lymphocyte therapy in patients with advanced melanoma progressed on multiple prior therapies

Authors: Amod Sarnaik, et al.

Session Title: Melanoma/Skin Cancers

Session Type: Oral Abstract Session

Abstract Number: 10006

Location: ASCO (Free ASCO Whitepaper)20 Virtual Scientific Program at View Source

Date/Time: available for on-demand viewing starting at 8:00am ET on May 29, 2020

Furthermore, Iovance will provide results from Cohort 4 as well as Cohort 2 of the C-144-01 study in metastatic melanoma as part of the BLA package. Based on the pooled analysis of Cohort 2 plus 4 (n=134), the overall response rate was 34.3%, including three complete responses, 43 partial responses (two of which are yet to be confirmed with follow up visits) and a disease control rate of 76.1%. Median DOR was not reached at 10.6 months of median study follow up.

Bellicum to Participate in Two Upcoming Virtual Investor Conferences

On May 27, 2020 Bellicum Pharmaceuticals, Inc. (NASDAQ:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers, reported that management will participate in two upcoming virtual investor conferences (Press release, Bellicum Pharmaceuticals, MAY 27, 2020, View Source [SID1234558562]).

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Conference Details:

Jefferies Virtual Healthcare Conference
Date/Time: Wednesday, June 3, 2020 at 4:00 p.m. EDT
Format: Fireside Chat

Investor Summit Virtual Summer Summit
Date/Time: Wednesday, June 10, 2020 at 3:20 p.m. EDT
Format: Presentation

Live webcasts of the fireside chat and presentation may be accessed from the Events & Presentation section of the Bellicum website. An archived version of each webcast will be available for replay immediately following the event.