Welcome to your Update from the Clinic for the month of February. Northwest Biotherapeutics received approval from Health Canada to proceed with its ongoing Phase 3 clinical study in Glioblastoma multiforme at clinical sites in Quebec. Sangamo and bluebird bio both had news on the regulatory progress of their respective beta-thalassemia programs. Kite Pharma follows the path of Juno, establishing its own manufacturing capability to manufacture CAR-T cells.
Northwest Biotherapeutics (NWBT) received a go-ahead from Health Canada to launch its Phase 3 study investigating DCVax-L in Glioblastoma multiforme (GBM) at sites in Canada. Initially, clinical study sites will include the Montreal Neurological Institute and Hospital, and Sherbrooke Hospital. The former is considered one of the world’s leading neuroscience research centres. NWBT’s multi-centre, placebo controlled Phase 3 study of DCVax-L will ultimately enroll 348 patients with GBM and is under way at 51 sites in the U.S. Patients are also being enrolled in Europe, where the lead site is King’s College Hospital in London. Another 30 sites are anticipated across Europe.
NWBT has been pursuing an aggressive clinical and regulatory strategy to ensure it can complete clinical development as rapidly as possible (GBM has a very low incidence), and that training is provided to a broad number of clinical sites, such that patients have a high degree of access to the therapy. DCVax-L became the first therapy to receive a Promising Innovative Medicine (PIM) designation from the UK authorities under its Early Access to Medicine Scheme (EAMS), and is the first immunotherapy to receive a hospital exemption under German Drug Law (any patient with GBM can receive the therapy before it is approved, not just those enrolled in clinical studies).
It is no surprise bluebird bio (BLUE) received a Breakthrough Therapy designation for LentiGlobin BB305. The company recently announced data from a Phase 1/2 showing that some beta-thalassemia patients are transfusion free after receiving the gene therapy product. LentiGlobin is an ex vivo gene therapy composed of a patient’s own bone marrow stem cells transduced with a gene encoding human beta-globin. The company has competition though: Sangamo BioSciences (SGMO), which is working on a different gene therapy paradigm (in collaboration with Biogen Idec), received acceptance of an investigational new drug (IND) from the FDA to launch clinical development of its zinc-finger protein (ZFP) therapeutic. SGMO’s ZFP genome editing technology acts by disrupting certain transcriptional regulators in a patient’s hematopoietic stem cells (HSCs), reverting the switch from expression of the mutant form of adult beta-globin back to functional fetal gamma-globin.
Pluristem (PSTM) reported on significant findings from a study investigating the utility of its PLX-R18 cells for the treatment of acute radiation syndrome (ARS) that was conducted by the U.S. National Institutes of Health (NIH). PLX-R18 cells, derived from the placenta, were injected into the muscle of animals, and showed a statistically significant difference in their ability to re-establish production of white blood cells, red blood cells and platelets after animals received an intense dose of radiation. Interestingly, intramuscular administration of the cells had a systemic effect on bone marrow healing, suggesting that the cells primarily exert their effect through secreted proteins. Additional animal studies will be required before FDA approval of the therapy; however, clinical studies in humans will not be required, as the therapy will be approved under the Animal Rule (human studies are not ethical or feasible).
The first subject has been treated in a pivotal study investigating BioTime’s (BTX) Renevia™ in individuals with HIV-associated lipoatrophy. Approximately 50% of people using anti-retrovirals today to manage HIV experience a slow degradation of the fat tissue under their skin. BTX’s product combines adipose-derived cells from the patient with an injectable matrix. The preparation is in liquid form and can be injected through a small gauge syringe. Once inside the body, the matrix polymerizes around the cells, forming a supportive scaffold upon which they can populate. The study is being conducted in Spain.
Only a month after Juno Therapeutics (JUNO) announced it leased a space in Washington State to use for GMP manufacture of its array of clinical CAR-T candidates, Kite Pharma (KITE) announced it is expanding its clinical and commercial manufacture capacity. The company leased a second space in El Segundo, California, directly adjacent to the Los Angeles International airport. KITE already has a facility in Santa Monica, California, under construction, which will provide 18,000 sq. ft. of space for clinical manufacture activities. The El Segundo facility will provide an additional 44,500 sq. ft., which can be expanded to over 60,000 sq. ft., if necessary. The move by KITE underscores what could be a trend with developers of cell-based immunotherapies to complete GMP manufacture using their own staff and facilities. KITE will continue to use its contract manufacturer to support clinical development of KTE-C19 until its own facilities are complete.
Disclaimer: “Update from the Clinic” is a blog post generated by news flow from public regenerative medicine (RM) companies around the globe. As CCRM has public RM companies in its industry consortium, and the number of such companies is relatively limited on a global scale, Mark Curtis will sometimes include CCRM consortium members in his review. This blog post is provided for general information only and nothing contained in the material constitutes a recommendation for the purchase or sale of any security. The author is not a shareholder of any public RM company. To see a list of CCRM’s industry consortium members, please visit http://ccrm.ca/industry-consortium