Industry Update: Clinical trials
Latest developments compiled from January 01 — February 28, 2017.
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 from January 01 — February 28, 2017, scheduled to be published in Volume 12 Issue 4 of Regenerative Medicine.
Asterias
Asterias Biotherapeutics (CA, USA; http://asteriasbiotherapeutics.com) has announced positive efficacy results from the company’s ongoing SCiStar Phase 1/2a clinical trial that showed additional motor function improvement at 6-months and 9-months following administration of 10 million AST-OPC1 cells in AIS-A patients with complete cervical spinal cord injuries (SCI; www.clinicaltrials.gov ID: NCT02302157 and study website www.scistar-study.com) . The SCiStar trial is an open-label, single-arm trial testing three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in as many as 35 patients with sub-acute, C-5 to C-7, motor complete (AIS-A or AIS-B) cervical SCI.
BioCardia
BioCardia (CA, USA; www.biocardia.com) has announced that the first patient had been treated at Johns Hopkins Hospital in the pivotal Phase III CardiAMP® clinical trial of a cell-based therapy for the treatment of ischemic heart failure that develops after a heart attack (www.clinicaltrials.gov ID: NCT02438306). The investigational CardiAMP therapy is designed to deliver a high dose of a patient’s own bone marrow cells directly to the point of cardiac dysfunction, potentially stimulating the body’s natural healing mechanisms after a heart attack
Gamida Cell
Gamida Cell (Jerusalem, Israel; www.gamida-cell.com) has announced that the first patient had been transplanted in the Company’s Phase III Study of NiCord®, in development as a cure for patients with blood cancer who do not have a fully matched donor required for bone marrow transplantation (www.clinicaltrials.gov ID: NCT02730299). NiCord® is a stand-alone graft derived from a single umbilical cord blood unit which has been expanded in culture and enriched with stem and progenitor cells using Gamida Cell’s proprietary NAM technology. NiCord has an FDA Breakthrough Therapy Designation as well as FDA and EMA orphan drug designations.
Kiadis
Following regulatory approval from the national authority in Canada (Health Canada), Kiadis (the Netherlands; www.kiadis.com) has initiated randomized, controlled, transatlantic Phase III trial with ATIR101â„¢ (CR-AIR-009). Approximately 195 patients with acute leukemia will be enrolled in total in the Phase III trial and randomized 1:1 to receive a haploidentical allogeneic HSCT using either the Kiadis Pharma approach with a single dose of ATIR101â„¢ or the post-transplant cyclophosphamide approach (also known as the Baltimore Protocol). The trial protocol has also been submitted for approval and is being evaluated by the US FDA as well as several European regulatory authorities. Following approval, the trial will be rolled out to additional study centers in the US and Europe.
MiMedx
MiMedx (GA, USA; www.mimedx.com) has released results from a recent scientific study confirming the ability of MiMedx Purion Processed dehydrated human amnion/chorion membrane (dHACM) allografts to promote cardiac repair following myocardial infarction. MiMedx purion processed dHACM allografts contain a unique array of regenerative cytokines, growth factors and extracellular matrix involved in the regulation of tissue healing and modulation of inflammation, suggesting role for treating infarcted cardiac tissue following heart attack. Allografts were shown to be able to prevent cardiac damage and promote healing in an in vivo mouse model of acute left ventricular myocardial infarction.
SpinalCyte
SpinalCyte (TX, USA; www.spinalcyte.com) has announced the enrollment of its first patient in a study for the development of CybroCell, the first dermal fibroblast cell product for treatment of degenerative disc disease.
Takeda and TiGenix
Takeda Pharmaceutical Company Limited (Japan; www.takeda.com) and TiGenix (Belgium; www.tigenix.com) have announced new data from the Phase 3 ADMIRE-CD clinical trial, which indicated that investigational compound Cx601, a suspension of allogeneic expanded adipose-derived stem cells, maintained long-term remission of treatment refractory complex perianal fistulas in patients with Crohn’s disease over 52 weeks (www.clinicaltrials.gov ID: NCT01372969).