1stOncology™: Cancer Intelligence Service – Page 13171 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Gamida Cell and the CIBMTR Announce Collaboration to Advance Research for Life-Saving Cellular Therapy

On September 11, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, and the CIBMTR (Center for International Blood and Marrow Transplant Research) reported the entry into a research agreement to collect and analyze health outcomes data in patients with hematologic malignancies who receive an allogeneic hematopoietic stem cell transplant (HSCT, or bone marrow transplant) from various donor sources (Press release, Gamida Cell, SEP 11, 2019, View Source [SID1234539444]). The CIBMTR is an organization that collaborates with the global scientific community to advance hematopoietic cell transplantation (HCT) and cellular therapy worldwide.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

The newly launched observational study by the CIBMTR and Gamida Cell will include both retrospective and prospective data contemporaneous to the international, randomized, Phase 3 study of omidubicel, Gamida Cell’s investigational advanced cell therapy designed to enhance the life-saving benefits of bone marrow transplant. Topline data from the ongoing Phase 3 study is anticipated in the first half of 2020, and initial data from the multi-year observational study is anticipated next year. Omidubicel has not yet been approved for marketing in the United States or any other jurisdiction.

"We are pleased to announce this collaboration with Gamida Cell, a company aiming to make bone marrow transplant an option for more patients facing life-threatening blood diseases," said Mary M. Horowitz, M.D., M.S., chief scientific director of the CIBMTR. "This collaboration will leverage the CIBMTR’s deep experience collecting and analyzing data on both the short- and long‐term outcomes of patients undergoing a bone marrow transplant. We look forward to contributing to efforts to better understand real-world clinical outcomes."

"This agreement marks the beginning of Gamida Cell’s health outcomes research initiatives, and we are excited to partner with the CIBMTR, an organization with deep expertise in bone marrow transplantation and cellular therapy," stated Julian Adams, Ph.D., chief executive officer of Gamida Cell. "We are committed to improving outcomes for patients who are in need of a bone marrow transplant and look forward to better understanding the variables that influence health outcomes, as well as elucidating how omidubicel may fit into the treatment landscape."

Randomized clinical trials comparing different donor types suggest that clinical outcomes may vary significantly depending on the donor type. The goal of this real-world, observational study is to better understand the variables that influence the health outcomes of patients receiving a transplant from a source other than a fully matched family donor. As part of the research agreement, the CIBMTR will use its registry, which consists of clinical outcomes data on more than 500,000 stem cell transplants, to analyze long‐term safety and efficacy data for patients with hematologic malignancies who underwent a bone marrow transplant with an alternative donor source following myeloablative conditioning. The criteria for inclusion of patients and the endpoints evaluated in the analysis will be consistent with the design of the Phase 3 study of omidubicel.

About Omidubicel
Omidubicel (formerly known as NiCord), the company’s lead clinical program, is an advanced cell therapy under development as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant solution for patients with hematologic malignancies (blood cancers). Omidubicel is the first bone marrow transplant product to receive Breakthrough Therapy Designation from the U.S. Food and Drug Administration and has also received Orphan Drug Designation in the U.S. and EU. In a Phase 1/2 clinical study, omidubicel demonstrated rapid and durable time to engraftment and was generally well-tolerated.1 A Phase 3 study evaluating omidubicel in patients with leukemia and lymphoma is ongoing in the U.S., Europe and Asia.2 Omidubicel is also being evaluated in a Phase 1/2 clinical study in patients with severe aplastic anemia.3 The aplastic anemia investigational new drug application is currently filed with the FDA under the brand name CordIn, which is the same investigational development candidate as omidubicel. For more information on clinical trials of omidubicel, please visit www.clinicaltrials.gov.

Omidubicel is an investigational therapy, and its safety and efficacy has not been evaluated by the U.S. Food and Drug Administration or any other health authority.

FORMA Therapeutics Announces Data for IDH1m Inhibitor Olutasidenib in Glioma to Be Presented at the 2019 Society for NeuroOncology Annual Meeting

On September 11, 2019 FORMA Therapeutics, Inc. reported that abstracts for the company’s next generation IDH1m inhibitor, olutasidenib (FT-2102), have been accepted for two presentations at the 2019 Society for NeuroOncology Annual Meeting, taking place November 20-24, 2019 in Phoenix, Arizona (Press release, Forma Therapeutics, SEP 11, 2019, View Source [SID1234539443]). Olutasidenib was designed to have a differentiated efficacy, durability and safety profile, as well as blood brain barrier penetrance, and is intended to treat all cancers with IDH1 mutations, including acute myeloid leukemia (AML), myelodysplastic syndrome (MDS), gliomas and other solid tumors.

"I am proud of the progress FORMA has made in the first eight months of 2019 and am truly excited for this next phase of growth for our company centered on our wholly-owned therapeutic pipeline focused on rare hematologic diseases and cancers," said Frank Lee, CEO of FORMA Therapeutics. "Gliomas are the most common, aggressive and difficult-to-treat primary brain tumors, with current treatment options limited to surgery, chemotherapy and radiation. Furthermore, gliomas typically affect relatively young patients. We look forward to presenting data regarding the brain penetrant activity of olutasidenib as well as initial results from our ongoing Phase 1b/2 study in patients with relapsed/refractory IDH1 mutant gliomas this November at SNO."

Olutasidenib Status in Gliomas/Solid Tumors:

An ongoing Phase 1b/2 study of olutasidenib is enrolling patients with gliomas or other advanced solid tumors with an IDH1 mutation.
Data from olutasidenib clinical trials have been submitted for presentation at medical meetings in the fourth quarter of 2019. Abstracts accepted for presentation at SNO include:
Oral Presentation: FT-2102 – A Potent and Selective Brain Penetrant Inhibitor of Mutant Isocitrate Dehydrogenase
Date and Time: Wednesday, November 20, 2019, 9:45-9:55 AM, MST
Oral Abstract Session: BBB Physiology at the SNO-SCIDOT Joint Conference on Therapeutic Delivery to the CNS
Presenter: Maria Ribadeneira, PhD, FORMA Therapeutics
E-Talk Presentation: Phase 1 study of FT-2102, an inhibitor of mutant IDH1, in patients with relapsed/refractory IDH1 mutant gliomas: preliminary safety and clinical activity
Date and Time: Saturday, November 23, 2019, 5:20 PM – 5:24 PM, MST
E-Talks Session: Group 1: Adult Therapeutics/Immunology Rare Tumors
Presenter: Macarena de la Fuente, MD, Sylvester Cancer Center, University of Miami
About Olutasidenib (FT-2102)

FORMA’s most advanced clinical asset, olutasidenib was designed as a potent and selective next generation inhibitor of mutated isocitrate dehydrogenase 1 (IDH1m) intended to treat patients with relapsed/refractory acute myeloid leukemia (RR/AML) or myelodysplastic syndrome (MDS), and patients with glioma and other solid tumors with an IDH1 mutation. IDH1 is a natural enzyme that is part of the normal metabolism of all cells, but when mutated its activity can promote blood malignancies and solid tumors. IDH1 mutations are present in up to 16% of patients with AML and over 80% of patients with low-grade gliomas. In AML, hypermethylation driven by IDH mutations inhibits normal differentiation of progenitor cells leading to accumulation of immature blasts. Quality of life declines with each successive line of treatment for AML and well-tolerated treatments in relapsed disease remain an unmet need. In gliomas, IDH1 mutations occur early in the tumor pathogenesis and persist throughout progression from a neural stem or progenitor cell. Gliomas are the most common, aggressive and difficult-to-treat primary brain tumors and high-grade gliomas are associated with poor long-term prognosis, with a median survival ranging from 5 to 7 years. Treatment options for relapsed glioma are limited. The rationale for targeting IDH1m is to reverse the oncogenic hypermethylated state to reduce tumor burden through induction of differentiation of immature blasts in AML and by slowing or stopping cancer cell growth in glioma.

In addition to the ongoing Phase 1b/2 study in patients with gliomas and other advanced solid tumors with an IDH1 mutation, a multi-cohort study with olutasidenib as a single agent and in combination with azacitidine is currently enrolling patients with AML and MDS, including a pivotal arm with olutasidenib as a single agent in R/R AML. Patients with treatment-naïve AML and R/R MDS with IDH1m are also being evaluated. Top-line data in patients with R/R AML are expected in the first half of 2020. If there is a positive outcome in R/R AML patients, a new drug application (NDA) is expected to be filed in the second half of 2020.

Independent Researchers Find Genprex’s TUSC2 Prevents Tumor Growth in Triple-Negative Breast Cancer

On September 11, 2019 Genprex, Inc. (NASDAQ: GNPX), a clinical-stage gene therapy company, reported that independent researchers reported in a recent study that TUSC2, a tumor suppressor gene and the active agent in Genprex’s Oncoprex immunogene therapy, prevented tumor growth in triple-negative breast cancer (TNBC), which is currently considered an incurable cancer with limited therapeutic options (Press release, Genprex, SEP 11, 2019, View Source [SID1234539442]). Genprex has no affiliation with these researchers.

The study, published in Nature, first found MicroRNA-138 as a diagnostic biomarker for TNBC, which currently lacks targeted therapies due to its inability to express the estrogen and progesterone hormone receptors and the human epidermal growth factor receptor 2 (HER2), thus the name for triple-negative breast cancer. Depletion of miR-138 was found to lead to apoptotic cell death in vitro and prevented tumorigenesis in vivo. TUSC2 was found to be a direct target of miR-138, and TUSC2 mimics the effects of miR-138 knockdown, preventing tumor growth. The researchers deduced that TUSC2 is a downstream tumor suppressor that is directly repressed by miR-138.

The study reports that triple-negative breast cancer is an extremely aggressive subtype of breast cancer which is associated with poor prognosis and high mortality rates. The lack of targeted treatment for triple-negative breast cancer makes it an increasingly feared diagnosis.

Genprex is conducting clinical and pre-clinical research to evaluate the effectiveness of TUSC2 when combined with targeted therapies and immunotherapies for non-small cell lung cancer. Existing pre-clinical data also suggest that TUSC2 may be effective against glioblastoma, head and neck cancer, kidney cancer, and soft tissue sarcomas. Now, this new independent study raises the possibility that TUSC2 expression, through miR-138 targeting, may also be used to treat the most aggressive subset of breast cancer.

"The results of the study evaluating TUSC2 for the treatment of triple-negative breast cancer are encouraging," said Rodney Varner, Genprex’s Chairman and Chief Executive Officer. "We believe that the data reported in this Nature article by independent researchers supports our belief that TUSC2 may be effective to treat a variety of cancers, including some of the most deadly types of cancer."

OncoCell MDx, Inc. Raises $22.2 Million in Series B Financing

On September 11, 2019 OncoCell MDx, Inc., a pan-disease immunogenomics platform company developing novel noninvasive blood-based tests to optimize patient care, reported it has raised $22.2 million in a Preferred Series B financing (Press release, OncoCell MDx, SEP 11, 2019, View Source [SID1234539441]). The financing was led by Savitr Capital, LLC (Savitr) with participation from existing investors.

OncoCell MDx has raised more than $30 million to date in support of its vision to simultaneously detect and stage cancer and other diseases using a single blood sample. This funding bolsters the company’s balance sheet and supports ongoing development and commercialization of its pan-disease diagnostic testing platform. The company plans to launch a blood test for detection and grading of aggressive prostate cancer next year.

"OncoCell is harnessing the power of the immune system and leveraging their proprietary immunogenomics technology to diagnose and stage disease early when there is the greatest opportunity for cure," said Andrew Midler, Managing Member at Savitr.

"We are developing and commercializing novel noninvasive tests that provide accurate diagnostic information quickly to physicians and their patients to help optimize patient treatment and improve patient outcomes," said Mark McDonough, President and CEO of OncoCell MDx.

"In a relatively short time, we have built a database of over 2,000 patient subjects analyzed with RNA-Seq and identified unique immunogenomic signatures that can discriminate healthy patients from those with indolent versus aggressive disease, he said. In prostate cancer, we’ve developed a blood test to enable improved staging and determine if a patient can safely forego invasive surgery or radiation therapy. Then, by virtue of our noninvasive approach, the patient can be serially tested to rule out disease progression, reducing the cost of care and improving quality of life."

DelMar Pharmaceuticals to Present at the Fall Investor Summit on September 16, 2019

On September 11, 2019 DelMar Pharmaceuticals, Inc. (Nasdaq: DMPI) ("DelMar" or the "Company"), a biopharmaceutical company focused on the development of novel cancer therapies, reported that CEO Saiid Zarrabian will deliver a presentation on the Company and meet with investors at the Fall Investor Summit on September 16, 2019 at 1:30 PM ET in Track 1 at Essex House, 160 Central Park South in New York City (Press release, DelMar Pharmaceuticals, SEP 11, 2019, View Source [SID1234539440]). The conference is being held September 16-17, 2019.

Interested investors may register for the conference at https://fall-investor-summit.events.issuerdirect.com/signup, or may request a meeting time by contacting John Marco at Core IR, either by calling 516-222-2560 or via [email protected].