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Celgene Corporation and bluebird bio Complete Enrollment of Pivotal KarMMa Study of anti-BCMA Car T Cell Therapy bb2121 in Patients with Relapsed and Refractory Multiple Myeloma

On November 27, 2018 Celgene Corporation (Nasdaq: CELG) and bluebird bio, Inc. (Nasdaq: BLUE) reported the completion of enrollment for the KarMMa pivotal study of bb2121, the companies’ lead investigational anti-BCMA CAR T cell therapy candidate for patients with relapsed and refractory multiple myeloma (Press release, bluebird bio, NOV 27, 2018, View Source [SID1234531638]). bb2121 is being developed as part of a Co-Development, Co-Promote and Profit Share Agreement between Celgene and bluebird bio.

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"We continue to be excited about bb2121 as a potential first-in-class BCMA-targeted therapy for patients with multiple myeloma," said Alise Reicin, M.D., President, Global Clinical Development for Celgene. "We would like to thank everyone who enabled this achievement, especially the patients and caregivers, and we congratulate the physicians and others involved in the KarMMa study, including our dedicated partners at bluebird bio. We look forward to seeing the data from this study and are progressing our broader bb2121 development program as we advance closer toward delivering this important new option to appropriate patients in need."

"We are committed to developing new treatment options to improve the care of patients with multiple myeloma, and completing enrollment of the KarMMa study moves us closer to this goal," said David Davidson, M.D., chief medical officer, bluebird bio. "As we advance our clinical studies of bb2121 in earlier lines of therapy in collaboration with our partners at Celgene, we remain very grateful to the patients, families and healthcare providers who have made this program possible."

KarMMa is a pivotal, open-label, single-arm, multi-center phase 2 study evaluating the efficacy and safety of bb2121 in patients with relapsed and refractory multiple myeloma. In November 2017, bb2121 was granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration and PRIority Medicines (PRIME) eligibility by the European Medicines Agency. The BTD designation and PRIME eligibility were based on preliminary clinical data from the phase 1 CRB-401 study.

The FDA action date for the bb2121 NDA is anticipated in 2020. bb2121 is currently an investigational therapy; safety and efficacy have not yet been established. bb2121 has not been approved for use by any health authority.

Asterias Biotherapeutics Announces Fourth Patient Dosed in First-in-Human Clinical Study of Immunotherapy VAC2 in Non-Small Cell Lung Cancer

On November 27, 2018 Asterias Biotherapeutics, Inc. (NYSE American: AST), a biotechnology company dedicated to developing cellular immunotherapies to treat cancer and cell-based therapeutics to treat neurological conditions associated with demyelination, reported enrollment and dosing of the fourth subject in the first-in-human Phase 1 clinical trial of VAC2 in the United Kingdom (Press release, Asterias Biotherapeutics, NOV 27, 2018, View Source [SID1234531636]). This is the first patient enrolled and dosed at Queen Elizabeth Hospital, Birmingham, UK. This initial clinical trial, which is being sponsored, managed and funded by Cancer Research UK, will examine the safety and tolerability of VAC2 in non-small cell lung cancer (NSCLC) as the study’s primary endpoints. Secondary and tertiary endpoints of the study include evaluations of the immunogenicity of VAC2 in NSCLC.

"We are excited to be participating in this clinical trial using Asterias’ groundbreaking dendritic cell technology, VAC2 for NSCLC," commented Gary Middleton, Professor of Medical Oncology at The University of Birmingham and Principal Investigator at Queen Elizabeth Hospital, the trial center. "The patient received their sixth and final injection of VAC2 this week and there have been no reported complications."

"We remain excited about the potential of VAC2 for NSCLC and we are thankful for Cancer Research UK’s sponsorship of the clinical trial and look forward to more patients being enrolled at Queen Elizabeth Hospital, as well as at the third trial site that CRUK is planning on opening," commented Michael Mulroy, President and Chief Executive Officer of Asterias. "The enrollment timeline for this trial remains on track and we are continuing to evaluate further development of VAC2 as a monotherapy or in combination with other therapies in various cancer indications that may benefit from this therapy."

About VAC2

VAC2 is an innovative immunotherapy product that contains mature dendritic cells derived from pluripotent stem cells. These non-patient specific (allogeneic) VAC2 cells are engineered to express a modified form of telomerase, a protein widely expressed in tumor cells, but rarely found in normal cells. The modified form of telomerase invokes enhanced stimulation of immune responses to the protein. Similar to an earlier, Asterias-sponsored, hematological cancer program using an autologous approach, the VAC2 dendritic cells instruct the immune system to generate responses against telomerase and, through this mechanism, target tumor cells. VAC2’s mode of action is complementary to and potentially synergistic with other immune therapies such as checkpoint inhibitors or other immune pathway inhibitors.

About Non-Small Cell Lung Cancer and the VAC2 Trial

Lung cancer (both small cell and non-small cell) is the leading cause of cancer-related death, accounting for about one-quarter of all cancer deaths and more than colorectal, breast, and prostate cancers combined. Non-small cell lung cancer (NSCLC) accounts for about 80% to 85% of lung cancers, according to the American Cancer Society. The three main types of NSCLC are adenocarcinoma, squamous cell carcinoma, and large cell carcinoma. The American Cancer Society’s estimates for lung cancer in the United States for 2017 are: about 222,500 new cases of lung cancer, and about 155,870 deaths from lung cancer. Despite the large number of people afflicted by non-small cell lung cancer, patients remain vastly underserved due to a scarcity of effective treatments. According to statistics published by Cancer Research UK, the five year survival rate for lung cancer patients in England and Wales is less than 10%.

As currently designed, the first VAC2 clinical trial will enroll up to 24 subjects into one of two cohorts, depending on the stage of their non-small cell lung cancer. The first cohort will evaluate VAC2 in up to 12 subjects with advanced non-small cell lung cancer. Subjects in this cohort, who carry the major histocompatibility gene, HLA-A2, will receive six weekly injections of VAC2 and will be followed for safety, immune responses to telomerase and overall clinical survival. These survival results will be compared directly to a control group who meet all of the other inclusion/exclusion criteria but do not possess the HLA-A2 gene. Assuming safety is demonstrated in the first cohort, enrolment will advance to a second cohort. In the second cohort, early stage subjects who have had successful resection of their tumor with no evidence of metastasis will be enrolled. Up to 12 subjects in this second cohort who carry the major histocompatibility allele HLA-A2 will receive six, weekly injections of VAC2 and will be followed for safety, immune responses to telomerase, overall clinical survival and time to relapse. These survival results will again be compared directly to a control group who meet all of the inclusion/exclusion criteria of cohort 2 but are not HLA-A2+. Subjects will be followed for one year for immune response to telomerase and for 2 years for the survival endpoints. The supply of VAC2 to be used in this trial is being manufactured by Cancer Research UK’s Biotherapeutics Development Unit. Asterias and Cancer Research UK are exploring the combination of VAC2 with an immune pathway inhibitor.

Sandoz receives eighth European Commission approval for a biosimilar with Ziextenzo® (pegfilgrastim)

On November 27, 2018 Sandoz, a Novartis division and the pioneer and global leader in biosimilars, reported that the European Commission (EC) granted marketing authorization for biosimilar Ziextenzo (pegfilgrastim) (Press release, Novartis, NOV 27, 2018, View Source [SID1234531635]).

Ziextenzo is indicated to reduce the duration of neutropenia and incidence of febrile neutropenia in adult patients treated with cytotoxic (anti-cancer) chemotherapy for malignancy with the exception of chronic myeloid leukemia and myelodysplastic syndromes.[1] These indications match those of the reference medicine.**[1],[3]

"Despite advancements in cancer treatment, febrile neutropenia remains one of the most significant complications of chemotherapy and is a major cause of morbidity,"[4] said Stefan Hendriks, Global Head of Biopharmaceuticals, Sandoz. "With the approval of Ziextenzo, a long-acting version of oncology supportive medicine filgrastim, we look forward to providing a treatment option that delivers the possibility of further reducing both the personal and financial burden of cancer."

This approval was based on comprehensive analytical, preclinical and clinical data. In these studies, Ziextenzo matched the reference medicine in terms of safety, efficacy and quality.[5]-[8] Pegfilgrastim, the active substance in Ziextenzo, is a long-acting form of filgrastim, which stimulates the production of white blood cells.[1]

"Pegfilgrastim biosimilars, such as Ziextenzo, mark a true advancement for people with cancer. These medicines help deliver optimized long-acting dosing and patient convenience while creating savings for our hard-pressed health systems," said Dr. Paul Cornes, oncologist and member of the Continuing Medical Education program of the European Association of Hospital Pharmacists and Core Lecturer for the European School of Oncology, United Kingdom.

Sandoz remains committed to making access happen for patients and leads in biosimilars with eight approved biosimilars worldwide, including five in the last 18 months. We look forward to making available a robust portfolio of biosimilar medicines that enables early and expanded access for patients as well as generates healthcare savings worldwide.

About Ziextenzo (pegfilgrastim)
Pegfilgrastim is a long-acting form of filgrastim. Filgrastim is very similar to a natural protein (granulocyte-colony stimulating factor) – also known as G-CSF – produced by a person’s own body. Filgrastim may be used to reduce the duration of neutropenia (low white blood cell count) and the occurrence of febrile neutropenia (low white blood cell count with a fever). Febrile neutropenia is caused by cytotoxic chemotherapy (medicines that destroy rapidly growing cells); white blood cells are important as they help your body fight infection.[1]

The Center for Cell and Gene Therapy at Baylor College of Medicine to Present at the 60th American Society of Hematology Annual Meeting

On November 27, 2018 Marker Therapeutics, Inc. (NASDAQ:MRKR), a clinical-stage immuno-oncology company, today reported that data from three abstracts—including an oral presentation—were accepted for presentation at the 60th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting (ASH 2018) (Press release, TapImmune, NOV 27, 2018, View Source [SID1234531632]). The studies describe results achieved using multi-tumor antigen specific T cells that were developed at the Baylor College of Medicine in the laboratories of Dr. Ann Leen and Dr. Juan Vera, and exclusively licensed to Marker.

Among the highlights, results from an ongoing Phase 1 study in patients with multiple myeloma will be reviewed in an oral presentation by Premal Lulla, M.B.B.S., Assistant Professor of Medicine at the Center for Cell and Gene Therapy, Hematology-Oncology, at the Baylor College of Medicine.

ASH 2018 will take place in San Diego, CA from December 1-4.

Oral Presentation Details:

Title: Safety and Efficacy of Multiantigen-Targeted T Cells for Multiple Myeloma (Abstract # 1014)

Date: Monday, December 3, 2018

Time: 7:30 p.m. PST (San Diego Convention Center, Ballroom 20D)

Poster Presentation Details:

Title: Targeting Lymphomas Using Non-Engineered, Multi-Antigen Specific T Cells (Abstract #1685)

Date: Saturday, December 1, 2018

Presentation Time: 6:15 p.m. – 8:15 p.m. PST (San Diego Convention Center, Hall GH)

Title: Adoptive T-Cell Therapy for Acute Lymphoblastic Leukemia Targeting Multiple Tumor Associated Antigens (Abstract #2693)

Date: Sunday, December 2, 2018

Presentation Time: 6:00 p.m. – 8:00 p.m. PST (San Diego Convention Center, Hall GH)

DNAtrix’s Oncolytic Virus Expressing OX40 Ligand Treats First Patient in Recurrent Glioblastoma Clinical Trial

On November 26, 2018 DNAtrix, a leader in oncolytic virus immunotherapies for cancer, reported the treatment of the first patient with DNX-2440, an oncolytic virus expressing OX40 ligand (OX40L) (Press release, DNAtrix, NOV 26, 2018, View Source [SID1234534217]). The first-in-human Phase 1 study is evaluating the safety and efficacy of DNX-2440, administered by Alcyone’s Microtip Cannula at the time of biopsy, to patients with recurrent glioblastoma for whom surgery is not possible or planned. DNX-2440 is the second DNAtrix oncolytic adenovirus to advance into the clinic.

DNX-2440 is engineered from DNAtrix’s highly potent oncolytic adenovirus, DNX-2401 (tasadenoturev), which has already demonstrated the ability to selectively kill tumor cells and trigger a powerful immune response directed against the tumor in several clinical studies. In addition to these mechanisms, DNX-2440 expresses OX40L, a critical costimulatory molecule known to enhance antitumor immune responses.

Extensive preclinical data have shown that DNX-2440 elicits tumor-specific immune memory and an abscopal effect (shrinking of an untreated tumor distant from the treated tumor), leading to prolonged survival in difficult-to-treat animal models of cancer, including gliomas, melanomas, and breast and lung cancers.

"Based on our observations from past studies with DNX-2401, we expect DNX-2440 to be safely administered and well tolerated, and to enhance clinical responses and prolong survival in patients with this devastating disease," said Ricardo Diez Valle, MD, Principal Investigator of the study at Clínica Universidad de Navarra, in Spain.

"DNX-2440 should trigger an immune response directed to tumors that are otherwise refractory to single agent immunotherapies," said Frank Tufaro, PhD, CEO of DNAtrix. "We now have two unique products in development and are planning to evaluate DNX-2440 for other malignancies, reinforcing our commitment to developing oncolytic immunotherapies for difficult to treat tumors."