Industry Update: Clinical trials

Latest clinical trial developments compiled from 1—31 December, 2015

Latest clinical trial developments in the field of stem cell research and
regenerative medicine compiled from publicly available information and
press releases from non-academic institutions 1—31 December 2015, scheduled to be published in Volume 11 Issue 3 of Regenerative Medicine.

Anika

Anika Therapeutics (MA, USA; www.anikatherapeutics.com) enrolled the first patient in its pivotal HYALOFAST® FastTRACK Phase III clinical study. The HYALOFAST FastTRACK study is a prospective, randomized, active treatment-controlled, evaluator-blinded multicenter study to establish the superiority of a hyaluronan-based scaffold HYALOFAST with autologous bone marrow aspirate concentrate (BMAC) in the treatment of articular knee cartilage defect lesions. The study will enroll approximately 200 patients at up to 30 sites in the U.S. and Europe. HYALOFAST is a biodegradable scaffold that is used to enable cartilage regeneration in patients suffering from cartilage defects. European clinical data demonstrates that patients treated with HYALOFAST plus autologous BMAC in a one-step, minimally invasive arthroscopic procedure were able to successfully regenerate hyaline-like cartilage. HYALOFAST is CE Marked in Europe and is available commercially in 18 countries with more than 6,000 uses to date.

Bellicum

Bellicum Pharmaceuticals (TX, USA; www.bellicum.com) presented interim data from the lead site in an ongoing BP-004 Phase 1/2 clinical trial. Pediatric patients in the study with a variety of genetic diseases achieved disease-free outcomes following a haploidentical, T cell-depleted HSCT followed by an add-back of BPX-501 donor T cells. The study is designed to evaluate whether this regimen is safe and improves immune reconstitution, infection control and overall outcomes. Initial outcomes were reported from the 39 pediatric patients who have received the BPX-501 product (of a total of 49 enrolled) at the European trial site as of November 30. Twenty of these children had non-malignant genetic diseases including Fanconi anemia, beta thalassemia, severe combined immunodeficiency (SCID or “bubble boy” disease), Wiskott-Aldrich Syndrome and others. Nineteen additional patients had blood cancers, with acute lymphoblastic leukemia being the most common. BPX-501 is an adjunct T cell therapy of genetically modified donor T cells incorporating Bellicum’s proprietary CaspaCIDe safety switch. The presented data show that treatment with BPX-501 is safe and well tolerated for patients with non-malignant and malignant diseases, and provides several important immune benefits compared to the clinical site’s historical controls. Additional information about the clinical trial is available at http://clinicaltrials.gov (ID: NCT02065869).

Bone Therapeutics

Bone Therapeutics (Belgium; www.bonetherapeutics.com) has successfully completed recruitment of the second cohort of four patients in its ALLOB® Phase IIA spinal fusion trial without any safety concerns. Spinal fusion is considered the gold standard for the treatment of a broad spectrum of spine disorders and aims to relieve pain and improve function. Although spinal fusion surgery is routine, it is associated with failure rates of up to 35%. Bone Therapeutics’ cell therapy product ALLOB has been designed to accelerate the fusion process and reduce the failure rate of current surgeries. The pilot Phase IIA study will enroll 16 patients with symptomatic degenerative lumbar disc disease who require interbody fusion surgery. An interbody cage is implanted according to the standard-of-care surgical approach, which will be supplemented with ALLOB in combination with bioceramic granules. Safety and efficacy of this treatment will be assessed over 12 months, using clinical and radiological evaluation. The trial is currently running in eight centers across Belgium. Today, the procedure has been performed in eight patients without any complications or safety issues. ALLOB is also being evaluated in the treatment of delayed-union fractures and has already reported strong safety and efficacy results. Recently, the Company initiated a novel Phase IIA study with ALLOB for the treatment of patients with failed spinal fusions. Additional information about the clinical trial is available at http://clinicaltrials.gov (ID: NCT02205138).

Gamida

Gamida Cell (Israel; www.gamida-cell.com) announced positive top line results from the Phase I/II study of NiCord, in development as an experimental treatment for patients with blood cancers. NiCord is an ex vivo cell graft derived from a single cord blood unit, expanded and enriched with highly functional stem cells and immune modulatory cells, utilizing the Company’s proprietary NAM technology. The currently available set of results are documented in a report submitted to the U.S. FDA in preparation for an End-of-Phase II meeting, and the initiation of a Phase III, randomized controlled, registration study of NiCord in hematological malignancies. NiCord aims to provide a superior transplant solution for patients without a suitable matched donor for bone marrow transplantation. Data was reported on the clinical outcomes of 16 patients with high risk hematological malignancies after transplantation of NiCord as the sole graft source. NiCord demonstrated rapid short term engraftment and stable, long term hematopoiesis of multiple cellular lineages. Neutrophil engraftment was achieved for all patients, at a median 10 days post-transplant. Historical reports of un-manipulated cord blood transplants estimate that neutrophil engraftment usually takes between 23-27 days on average. The top line data reported shows that NiCord is comparable in terms of time to engraftment to peripheral blood transplantation, which is considered the gold standard. Results also indicated a good safety profile with potentially reduced morbidity and shorter hospitalization of patients. Additional information about the clinical trial is available at http://clinicaltrials.gov (ID: NCT01221857).

Sanbio and Sunovion

SanBio (CA, USA; www.san-bio.com) has initiated patient recruitment for a Phase IIb clinical trial to further test the safety and efficacy of its proprietary cell therapy. SanBio is working with Sunovion Pharmaceuticals (MA, USA; www.sunovion.com), a wholly owned subsidiary of Sumitomo Dainippon Pharma (Japan; www.ds-pharma.com), on the conduct of the clinical trial. Earlier clinical trial results suggested the therapy’s potential to improve motor function following an ischemic stroke. The ACTIsSIMA “Allogeneic Cell Therapy for Ischemic Stroke to Improve Motor Abilities” trial will examine the safety and efficacy of SB623 cells in patients who have experienced an ischemic stroke in the previous six months to five years and still suffer from motor impairments. SB623 cells are modified allogeneic MSC, derived from bone marrow stromal cells isolated from healthy donors. When administered into neural tissue, SB623 cell therapy is designed to promote recovery from injury by triggering the brain’s natural regenerative ability. The ACTIsSIMA Phase IIb clinical trial follows a Phase I/IIa clinical trial in which the SB623 cell treatment suggested improvements in motor function. The Phase IIb clinical trial will further evaluate the safety and efficacy of SB623 treatment. There are expected to be approximately 60 clinical trial sites throughout the U.S., and total enrollment is expected to reach 156 patients. SanBio and Sumitomo Dainippon Pharma have entered into a joint development and license agreement for exclusive marketing rights in North America for SB623 for chronic stroke. Additional information about the clinical trial is available at http://clinicaltrials.gov (ID: NCT02448641).