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Medivation’s Pidilizumab Shows Potential Clinical Benefit and Safety in Children With Diffuse Intrinsic Pontine Glioma (DIPG)

On June 16, 2016 Medivation, Inc. (NASDAQ: MDVN) reported results from a Phase I/II study of pidilizumab, an investigational antibody with immune-mediated anti-tumor effects, that demonstrated potential clinical benefit in pediatric patients with diffuse intrinsic pontine glioma (DIPG) (Press release, Medivation, JUN 16, 2016, View Source [SID:1234513409]).

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The study, which was exploratory in nature, was designed to assess the safety and tolerability of pidilizumab, as well as key clinical outcomes, such as event-free and overall survival, in this pediatric population. Preliminary data were presented this week in an oral symposium at the International Symposium on Pediatric Neuro-Oncology (ISPNO) by the study’s lead investigator Iris Fried, M.D., Attending Physician, Pediatric Hemato-oncology, Hadassah Medical Center, Jerusalem, Israel.

Diffuse intrinsic pontine glioma (DIPG) is a rare and aggressive pediatric cancer which is responsible for the highest brain tumor mortality in children.1 Approximately 300-400 pediatric brain stem tumors are diagnosed per year in the United States, approximately 75%-80% of which are DIPGs.2 Children with DIPG experience a median overall survival between 9-12 months and a two-year survival rate of less than 10%.1

Data from nine pediatric patients with DIPG who were treated with pidilizumab following completion of standard radiation therapy were presented. The median age of the study population was 6.5 years (range: 3-19 years): eight patients had intermediate risk features and one patient had high risk features. The reported mean event-free and overall survival estimates were 12 and 15.6 months, respectively. Three patients with DIPG remained progression-free at 16.3, 22, and 24 months following diagnosis, with one patient experiencing a partial response. Adverse events of any grade reported in at least one treatment cycle include neutropenia, fatigue, loss of appetite, hypertension, nausea, and lymphopenia; only neutropenia and hypertension were reported as grade 3 adverse events. The study continues to enroll patients.

"Separate studies have revealed the potential of pidilizumab in hematological malignancies, and these results, while in a small cohort in a rare disease, suggest its potential activity in other conditions," said David Hung, M.D., Founder, President and Chief Executive Officer of Medivation. "We are especially encouraged by these results in a devastating disease that typically leads to such rapid and certain mortality, and we remain committed to advancing the clinical development of pidilizumab as we continue to characterize its unique and differentiated mechanism of action."

Despite more than 30 years of clinical research, there have been no improvements in clinical outcomes and there are no approved treatments for DIPG.1,2

10-Q – Quarterly report [Sections 13 or 15(d)]

Spring Bank Pharmaceuticals has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Spring Bank Pharmaceuticals, 2018, JUN 15, 2016, View Source [SID1234527559]).

Apogenix Reports Topline Results from Phase I Trial in Myelodysplastic Syndromes with APG101

On June 15, 2016 Apogenix, a biopharmaceutical company developing next-generation immuno-oncology therapeutics, reported final topline results from a Phase I trial evaluating APG101 in low to intermediate-1 risk transfusion-dependent patients with myelodysplastic syndromes (MDS) (Press release, Apogenix, JUN 15, 2016, View Source [SID1234524579]). The study showed that APG101 was well tolerated. In addition, the trial showed that APG101 efficiently stimulates erythropoiesis in these patients.

The single arm Phase I trial enrolled twenty patients with low to intermediate-1 risk MDS who were transfusion dependent. Patients had to be refractory to erythropoietin-stimulating agents (ESAs). The patients were treated with APG101 over a period of three months and followed for an additional six months. An extension of treatment was not intended. The primary objectives of the study were safety and tolerability. Secondary objectives included changes in transfusion frequency and changes in parameters involved in erythropoiesis.

In the study, treatment with APG101 led to a significant decrease in transfusion frequency for more than six months (end of follow up period) in 44% of the patients. In addition, measurements of parameters involved in erythropoiesis (i.e., number and function of progenitor cells) further supported the activity of APG101 in this patient population. This evidence of in vivo activity of APG101 confirms in vitro data recently published (Oncotarget Vol. 7 No. 12, 2016). More details from the final results of the study are being submitted for presentation at a major medical meeting later this year.

"The topline data from this Phase I trial continue to support the activity of APG101 in MDS patients," said Harald Fricke, M.D., Chief Medical Officer. "We were particularly excited to see that APG101 appeared to decrease the number of transfusions required by this very sick patient population. Our next step will be to initiate a Phase II trial in MDS to evaluate APG101 in various doses in combination with an erythropoietin-stimulating agent, and we are currently soliciting input from key opinion leaders on the design of that trial."

About Myelodysplastic Syndromes (MDS)
MDS is a bone marrow disorder that is characterized by ineffective hematopoiesis and can lead to severe anemia. In most cases, the anemia is treated with blood transfusions that eventually result in an iron overload, which can damage the liver and other organs. At the same time, the number of thrombocytes that are responsible for coagulation and the number of leucocytes that are responsible for immune defense significantly decrease in patients with this disorder. As a result, MDS patients frequently suffer from sudden bleeding and life-threatening infections. In addition, they are at risk of developing acute myeloid leukemia, a type of blood cancer.

About APG101
Apogenix’s lead immuno-oncology candidate APG101 is a fully human fusion protein that consists of the extracellular domain of the CD95 receptor and the Fc domain of an IgG antibody. APG101 is being developed for the treatment of solid tumors and malignant hematological diseases. By blocking the CD95 ligand, which negatively regulates erythrocyte production in MDS patients, APG101 directly addresses the cause of the disorder and could thus potentially provide a cure for MDS.

Exemplar Genetics to Present on Novel Research Models for Oncology at the World Preclinical Congress

On June 15, 2016 Exemplar Genetics reported its President, John Swart, Ph.D., will present today at the 15th Annual World Preclinical Congress during the Preclinical Models in Oncology session (Press release, Exemplar Genetics, JUN 15, 2016, View Source [SID:1234513374]). Dr. Swart’s presentation titled ‘Genetically Engineered Miniswine Models of Cancer’ will provide an overview of Exemplar’s novel ExeGen TP53R167H and ExeGen KRASG12D/+/TP53R167H/+ miniswine research models developed for oncology to help bridge the pre-clinical gap from small animal models to humans.

Exemplar Genetics Logo
Dr. Swart commented, "We believe the development of these new genetically engineered miniature swine models will resolve inadequacies with traditional rodent models of cancer, which while valuable in certain aspects of research, have been inefficient at translating to human clinical trials."

As described in the Journal of Clinical Investigation publication, "Development and translational imaging of a TP53 porcine tumorigenesis model", current cancer research models are informative yet fail to accurately recapitulate human disease, and pre-clinical results utilizing these models often have poor translational value to the clinic. In particular, the lack of a large-animal model that accurately replicates human cancer pathologies has been a significant barrier to the development of effective diagnostics, as well as surgical and therapeutic interventions.

Exemplar’s genetically engineered ExeGen TP53R167H model expresses a mutation in the gene that encodes p53 (TP53), which is orthologous to one commonly found in humans. It is estimated p53 function is compromised in the vast majority of human tumors, through either TP53 gene mutation or alterations targeting the numerous regulators of p53 signaling. Studies with the TP53R167H model demonstrated tumor formation and characteristic chromosomal instability similar to what is seen in humans with mutant p53 alleles. Additionally, Exemplar has developed the ExeGen KRASG12D/+/TP53R167H/+ model which contains a conditional KRAS mutation on the background of TP53-targeted pigs that should allow for the inducement of human-like tumors in a tissue specific manner.

In April 2016, the U.S. Food & Drug Administration (FDA) exercised enforcement discretion in regard to Exemplar’s ExeGen low-density lipoprotein receptor (LDLR) miniswine clearing it for commercial use as a research model. As the first genetically engineered miniswine model reviewed and cleared by the FDA, this powerful investigational platform is available to researchers and drug developers helping forge a more reliable, consistent path from pre-clinical testing through human studies. Exemplar continues to work closely with the FDA to make each of the miniswine research models in its broad, extensive pipeline for use in the evaluation of several human health conditions including rare diseases, cancer, cystic fibrosis, neuromuscular/neurodegenerative disorders, and cardiovascular disease, available to researchers working on solutions for these devastating diseases.

BTG completes the acquisition of Galil Medical

On June 15, 2016 BTG plc (LSE: BTG), the specialist healthcare company, reported that it has completed the acquisition of Galil Medical, a leading provider of cryoablation products for the treatment of kidney and other cancers (Press release, BTG, JUN 15, 2016, View Source [SID:1234513373]).

BTG announced on 6 May 2016 that it had entered into an agreement to acquire Galil.