On May 31, 2019 Jiangsu Hengrui Medicine reported China regulators approved its PD-1 drug, camrelizumab, as a third-line treatment for recurrent or refractory classical Hodgkin’s lymphoma (Press release, ChinaBio, MAY 31, 2019, View Source [SID1234556331]). Camrelizumab is the third domestic PD-1/PD-L1 drug approved in China; previously, Junshi’s Tuoyi and Innovent’s Tyvyx were granted marketing approvals. Two US-based companies have also entered China’s PD-1/PD-L1 market — Merck with Keytruda (also for melanoma) and Bristol Myers Squibb with Opdivo. Hengrui said it is testing camrelizumab in about 20 China clinical trials.
On May 31, 2019 the cancer vaccine company Ultimovacs ASA reported that it has raised NOK 370 million in an oversubscribed share issue, and will be listed on the Oslo Stock Exchange on Monday, June 3, 2019 (Press release, Ultimovacs, MAY 31, 2019, View Source [SID1234536989]).
Investors showed great interest in Ultimovac’s listing on the Oslo Stock Exchange and the issue was oversubscribed. Large Norwegian and international institutional investors have entered the company as new shareholders. Among the new, large shareholders are also specialized health funds. Ultimovacs experienced a broad interest in subscription of shares and the company will have about 1,500 shareholders on listing.
"It is very gratifying for Norwegian health care and for Ultimovacs that national and international investors show the company this confidence. In today’s uncertain market, it is extra pleasant with such great interest, both from international investors and small savers. I look forward to following the company further, "says Jonas Einarsson, chairman of Ultimovacs and CEO of Radforsk.
The funds Ultimovacs collect at the IPO will finance the company’s development plan for the universal cancer vaccine UV1. The main project is a major clinical study to document the treatment effect of the vaccine. In this study, UV1 will be combined with other immunotherapy in patients with melanoma at about 30 hospitals in Norway, Europe, the United States and possibly Australia.
Ultimovacs has already conducted three minor clinical trials of the vaccine at the Radium Hospital in patients with lung cancer, prostate cancer and mole cancer.
«The cancer vaccine has shown promising clinical signals in the studies we have conducted at Radiumhospitalet. Based on this, we have developed a development plan to document that our vaccine has an effect on cancer patients. I am very pleased that we have now completed a successful stock exchange listing. This means that the practical conditions are now in place to implement our development plan, "says Øyvind Kongstun Arnesen, CEO of Ultimovacs.
On May 31, 2019 Gamida Cell Ltd. (Nasdaq: GMDA), a leading cellular and immune therapeutics company, reported that two presentations on omidubicel1 (formerly known as NiCord), an investigational advanced cell therapy in Phase 3 clinical development designed to enhance the life-saving benefits of hematopoietic stem cell (bone marrow) transplant, took place at the International Society for Cell and Gene Therapy (ISCT) 2019 Annual Meeting being held in Melbourne, Australia (Press release, Gamida Cell, MAY 31, 2019, Gamida Cell Announces Data for Omidubicel Presented at the International Society for Cellular and Gene Therapy 2019 Annual Meeting
[SID1234536776]). The presentations included a summary of clinical and translational data from the completed Phase 1/2 clinical study of omidubicel in patients with high-risk hematologic malignancies, or blood cancers. Results from the Phase 1/2 study showed that patients transplanted with omidubicel had rapid and durable engraftment of neutrophils and platelets, as well as prompt immune reconstitution. An international, randomized Phase 3 study of omidubicel in patients with hematologic malignancies is currently ongoing.2
"In the Phase 1/2 clinical study, patients who received omidubicel had a clinically meaningful reduction in their time to neutrophil and platelet recovery compared to a real-world cohort of patients who received a standard umbilical cord blood transplant. The neutrophil recovery observed with omidubicel also resulted in fewer days spent in the hospital compared to the comparator cohort," said Joanne Kurtzberg, M.D., Director of the Marcus Center for Cellular Cures and the Carolinas Cord Blood Bank at Duke University Medical Center. "These data suggest an important potential step toward making stem cell transplantation safer and more accessible to patients with lethal blood cancers, and I am pleased to be participating in the Phase 3 study currently enrolling patients."
Despite the curative potential of bone marrow transplants, it is estimated that more than 40 percent of eligible patients in the U.S. do not receive one for various reasons, including finding a matched donor.3 Even for patients who do receive a transplant, treatment is not always effective and can lead to serious complications that can dramatically affect quality of life.4 Omidubicel is intended to address the current limitations of bone marrow transplant by providing a therapeutic dose of cells while preserving the cells’ functional therapeutic characteristics.
"At Gamida Cell, our aspiration is to bring the first FDA-approved cell therapy for bone marrow transplantation to patients," stated Ronit Simantov, M.D., chief medical officer at Gamida Cell. "These data demonstrate the potential of omidubicel to give patients with high-risk blood cancers, particularly those who would not otherwise receive a bone marrow transplant from a matched donor, an opportunity for a cure."
Data Presented at ISCT 2019 Annual Meeting
Phase 1/2 Clinical Data
The presentation, "NiCord, an Expanded Cord Blood Product, Accelerates Engraftment After Myeloablative Conditioning," described results from the completed multicenter, Phase 1/2 clinical trial of omidubicel in 36 patients with high-risk hematologic malignancies and no readily available matched sibling or matched unrelated adult donor. The key primary endpoint was the cumulative incidence of neutrophil engraftment at 42 days. Additionally, the omidubicel patient cohort was compared to a retrospective cohort of patients who received standard cord blood transplant using data from the Center for International Blood and Marrow Transplant Research (CIBMTR).
Data from the study demonstrated that patients transplanted with omidubicel had rapid and durable engraftment of neutrophils and platelets. The age-adjusted cumulative incidence of neutrophil engraftment at 42 days following transplantation was 94 percent for omidubicel recipients compared to 85 percent for the CIBMTR cohort. Among patients who engrafted, the median time to neutrophil recovery was 11.5 days (95 percent confidence interval (CI): 9-14 days) for omidubicel recipients compared to 21 days (95 percent CI: 20-23 days) for the CIBMTR cohort (p < 0.001). For patients achieving platelet recovery, the median time to platelet recovery was 34 days (95 percent CI: 32-42 days) and 46 days (95 percent CI: 42-50 days) for the omidubicel and CIBMTR cohorts, respectively (p < 0.001). Omidubicel demonstrated an acceptable safety profile, with hypertension reported as the most common adverse event attributable to omidubicel infusion, and moderate to severe chronic graft vs. host disease reported in 9.8 percent of patients at one year following transplantation. Primary hospital discharge occurred at a median of 20 days following transplantation. Omidubicel recipients spent a median of 73 days alive and out of hospital during the first 100 days following transplantation.
Phase 1/2 Translational Data
The presentation, "Rapid and Robust CD4+ and CD8+ T-, NK-, B- and Monocyte Reconstitution after Nicotinamide-Expanded Cord Blood Transplantation," described in-depth immune reconstitution data from the completed Phase 1/2 clinical study of omidubicel. Immune reconstitution for 27 patients receiving omidubicel was compared to retrospective cohorts of adolescent and young adults with hematologic malignancies receiving unmanipulated cord blood transplantation (unCBT, n = 27) or unrelated bone marrow transplantation (BMT, n =20). The primary endpoint was the probability of achieving CD4+ immune reconstitution (> 50×106/L) within the first 100 days. Secondary endpoints included the recovery of B cells, CD4+ T cells and natural killer (NK) cells during the first year after transplantation. Analyses were performed at the University Medical Centre Utrecht, Laboratory of Translational Immunology.
The analysis showed that 91 percent of patients receiving omidubicel achieved successful immune reconstitution of CD4+ T cells at 100 days after transplantation. Reconstitution of T cells in the omidubicel group (median age 41.5 years) was similar to the unCBT and BMT cohorts (median age 15.4 and 14.3 years, respectively), despite the younger age of the cohorts, who would be expected to reconstitute faster. In addition, reconstitution of a number of cell types, including B cells (p = 0.026) and NK cells (p < 0.001), was significantly faster after transplantation with omidubicel compared to the cohorts, and suggests that omidubicel reconstitutes diverse functions of the immune system.
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).1 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.5 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.6 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.
On May 31, 2019 Vigeo Therapeutics, a clinical-stage biopharmaceutical company developing novel therapeutics to reprogram the tumor immune microenvironment (TIME), reported that preliminary data from its Phase 1 open label trial of VT1021 in patients with advanced solid tumors, will be presented tomorrow, June 1st from 8:00 am to 11:00 am CT at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) annual meeting being held May 31 – June 4, 2019 in Chicago (Press release, Vigeo Therapeutics, MAY 31, 2019, View Source [SID1234536775]).
Vigeo is developing therapies that target the TIME via the induction of thrombospondin-1(Tsp-1) by replicating the biological activity of prosaposin (Psap). Tsp-1 is a naturally occurring potent anti-tumorigenic protein that has been shown to reprogram the TIME and block tumor growth and progression.
"We believe that by reprogramming the TIME, we can significantly improve the outcomes of cancer treatments," said Jing Watnick, Ph.D., M.B.A., chief executive officer of Vigeo. "Emerging clinical data continue to suggest that Vigeo’s VT1021, targeting the tumor microenvironment, is safe and well tolerated and has significant potential in hard-to-treat cancers," she added.
VT1021 in Patients with Advanced Solid Tumors
Dose escalation phase of first in human study with VT1021 is ongoing. Through the first six dose levels tested, VT1021 has been shown to be safe and well tolerated with no serious drug related adverse events. Enrollment for expansion cohorts in specific tumor types is expected to be initiated in the third quarter of 2019.
"The mechanism of action of VT1021 is quite unique," said Gregory Berk MD, chief medical officer of Vigeo. "The molecule has demonstrated an excellent safety profile and PK/PD properties, and is well positioned to be an effective single agent therapy, as well as an ideal partner for standard of care therapies in various oncology indications."
Title: A Phase 1 Study Evaluating the Safety, Pharmacology, and Preliminary Activity of VT1021 in Patients with Advanced Solid Tumors
Date & Time: Saturday June 1, 2019 from 8:00 am – 11:00 am CT
Abstract Session: Developmental Therapeutics and Tumor Biology
Location: Board #144a
Presenter: Michael Cieslewicz, PhD, Vice President for Project Management and Operations, Vigeo
Vigeo’s lead molecule, VT1021, is a small peptide agent derived from Psap, that triggers Tsp-1 production, which reprograms the tumor microenvironment and makes it inhospitable for tumor growth to occur. Pre-clinical results have demonstrated that VT1021, when administered systemically, can cause tumor regression in animal models at both the primary and metastatic sites. VT1021 is currently being evaluated in a Phase 1, open label, multicenter trial that assesses the drug’s safety, tolerability, and preliminary anti-tumor efficacy. The trial’s dose escalation phase was launched in late 2017, and the expansion phase will initiate in 3rd quarter of 2019. An interim readout is expected in the second half of 2020.
On May 31, 2019 ALX Oncology, a clinical-stage immuno-oncology company developing therapies to block the CD47 myeloid checkpoint mechanism, reported that Jaume Pons, Ph.D., President and Chief Executive Officer, will present a company overview at the Jefferies 2019 Healthcare Conference on Wednesday, June 5, 2019 at 4:00 p.m. ET at the Grand Hyatt in New York, NY (Press release, ALX Oncology, MAY 31, 2019, View Source [SID1234536774]).