On February 22, 2017 Bellicum Pharmaceuticals, Inc. (Nasdaq:BLCM), a leader in developing novel, controllable cellular immunotherapies for cancers and orphan inherited blood disorders, reported the presentation of updated clinical results from its multicenter BP-004 clinical trial of BPX-501 and rimiducid at the 2017 BMT Tandem Meetings (Press release, Bellicum Pharmaceuticals, FEB 22, 2017, View Source [SID1234517790]). Results on 73 patients with more than six months of follow-up demonstrated that the use of BPX-501 following an alpha/beta-depleted, haploidentical hematopoietic stem cell transplant (haplo-HSCT) resulted in rapid immune recovery in patients with inherited blood disorders and hematological cancers.

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"The results from our BPX-501 clinical program continue to demonstrate positive and consistent results across multiple centers and diseases, underscoring its potential to address the needs of a wide range of pediatric patients who lack access to a suitable matched donor," commented Rick Fair, President and CEO of Bellicum Pharmaceuticals. "The data reported today, including incidences of GvHD- and disease-free outcomes in children with blood cancers and genetic diseases, compare favorably to historical outcomes in patients receiving a matched unrelated donor (MUD) transplant, the comparative population for our ongoing registration trial in Europe."

Updated Results of BP-004 Study
To date, 122 pediatric patients have undergone treatment with BPX-501. Investigators reported on 91 patients with 100 days of follow-up, 73 patients with six months, and 45 patients with more than one year of follow-up. Genetic blood diseases (n=54) include SCID (Severe Combined Immune Deficiency) (n=11), thalassemia major (n=8), Wiskott-Aldrich syndrome (n=6), sickle cell disease (n=3), and several others. Hematological cancers (n=37) include ALL (acute lymphoblastic leukemia) (n=22), AML (acute myeloid leukemia) (n=13), and others.

Patients receiving BPX-501 following a haplo-HSCT demonstrated rapid immune reconstitution by month six, including full recovery and normalization of T-cells, B-cells and immunoglobulins. Cumulative incidence of treatment-related mortality remains very low in the BP-004 study, with six-month and one-year survival rates of 98.4 percent and 97.2 percent, respectively, and no mortality associated with use of BPX-501 or rimiducid. Of 73 patients with more than six months of follow-up, 22 percent had acute Grade 1-2 GvHD, 2.7 percent had Grade 3, and there were no cases of Grade 4 GvHD. In the BP-004 study, rimiducid was used on six patients experiencing GvHD that was not controlled by the use of standard treatments. In all five cases of uncontrolled acute GvHD, the administration of rimiducid rapidly resolved the symptoms. As previously reported, there was one case of severe chronic GvHD attributed to cells from the donor graft, and unrelated to BPX-501, in a malignant patient that did not resolve with administration of rimiducid.

The poster presentation can be accessed in the Events and Presentations section of the Bellicum website.

About BPX-501
BPX-501 is an adjunct T-cell therapy administered after allogeneic HSCT, comprising genetically modified donor T cells incorporating Bellicum’s CaspaCIDe safety switch. It is designed to provide a safety net to eliminate alloreactive BPX-501 T cells (via administration of activator agent rimiducid) should uncontrollable GvHD occur. This enables physicians to more safely perform stem cell transplants by adding back BPX-501 engineered T cells to speed immune reconstitution and provide control over viral infections, without unacceptable GvHD risk. The ongoing BP-004 clinical study of BPX-501 is being conducted at transplant centers in the U.S. and Europe.

About the BMT Tandem Meetings
The BMT Tandem Meetings are the combined annual meetings of the Center for International Blood & Marrow Transplant Research (CIBMTR) and the American Society for Blood and Marrow Transplantation (ASBMT). Attended by investigators, clinicians, laboratory technicians, clinical research professionals, nurses, pharmacists, administrators, and allied health professional attendees, the scientific program addresses the most timely issues in hematopoietic cell transplantation.

On February 22, 2017 Immune Design (Nasdaq:IMDZ), a clinical-stage immunotherapy company focused on oncology, reported that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for its investigational intratumoral therapy, G100, for the treatment of follicular non-Hodgkin’s lymphoma (Press release, Immune Design, FEB 22, 2017, View Source [SID1234517787]).

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Orphan Drug Designation is granted by the FDA Office of Orphan Drug Products to products that treat rare diseases. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation provides the sponsor certain benefits and incentives, including a period of marketing exclusivity for the first marketing application, if regulatory approval is received for the designated indication, potential tax credits for certain activities and waiver of certain administrative fees.

G100 is a product candidate from Immune Design’s GLAAS discovery platform. It contains a potent synthetic small molecule toll-like receptor-4 (TLR-4) agonist, Glucopyranosyl Lipid A (GLA), and is the lead product candidate in Immune Design’s Antigen Agnostic approach. It leverages the activation of both innate and adaptive immunity, including Dendritic Cells, in the tumor microenvironment to create an immune response against the tumor’s preexisting diverse set of antigens. A growing set of clinical and preclinical data have demonstrated the ability of G100 to activate tumor-infiltrating lymphocytes, macrophages and dendritic cells, and promote antigen-presentation and the recruitment of T cells to the tumor. The ensuing induction of local and systemic immune responses has been shown to result in local and abscopal (shrinking of tumors outside the scope of the localized treatment) tumor control in preclinical studies. G100 was evaluated in a Phase 1 study in Merkel cell carcinoma patients and produced a 50% overall response rate per protocol and a favorable safety profile. Currently, G100 is being evaluated as both a monotherapy (with local radiation) and in combination with Merck’s anti-PD-1 agent, pembrolizumab, pursuant to a clinical collaboration with Merck, in a randomized Phase 1/2 trial in patients with follicular non-Hodgkin’s lymphoma.

CMB305, Immune Design’s prime-boost cancer immunotherapy product candidate, has previously been designated orphan drug status for soft tissue sarcoma by the FDA, and each of the two agents comprising CMB305 also have both U.S. and European Orphan drug designation for soft tissue sarcoma.