Industry Update: Clinical trials

Written by Dusko ILIC

Latest clinical trial developments compiled from August 1— September 30 2015

Latest business developments in the field of stem cell research and
regenerative medicine compiled from publicly available information and
press releases from non-academic institutions August 1— September 30
2015, scheduled to be published in Volume 11 Issue 1 of Regenerative
Medicine
.


Asterias

Asterias Biotherapeutics (CA, USA; www.asteriasbiotherapeutics.com), subsidiary of BioTime (CA, USA; www.biotimeinc.com) reported that the third patient was successfully dosed at Chicago-based Rush University Medical Center in a Phase 1/2a clinical trial evaluating activity of escalating doses of AST-OPC1 (oligodendrocyte progenitor cells) in newly injured patients with sensory and motor complete cervical spinal cord injury. This represents the final patient treated at the initial low-dose (2 million cells) safety cohort. The SCi-STAR trial will test three sequential escalating doses of AST-OPC1 administered at up to 20 million AST-OPC1 cells in 13 patients with sub-acute, C-5 to C-7, neurologically complete cervical spinal cord injury. These individuals have essentially lost all sensation and movement below their injury site with severe paralysis of the upper and lower limbs. AST-OPC1 will be administered 14 to 30 days post-injury. Patients will be followed by neurological exams and imaging methods to assess the safety and activity of the product. Additional information on the Phase 1/2a study, including trial sites, can be found at www.clinicaltrials.gov (ID: NCT02302157) and at the SCiStar Study Website www.scistarstudy.com.

Mesoblast

Mesoblast (Australia; www.mesoblast.com) announced additional Phase 2 trial results of its lead product candidate for the treatment of chronic heart failure (CHF). A post-hoc analysis was performed in 30 patients from the Phase 2 trial who had been randomized to receive either placebo or a single administration of 150 million mesenchymal precursor cells (MPC) MPC-150-IM. The results suggest that patients with advanced heart failure may be an optimal target population for treatment with Mesoblast’s MPC therapy.

Ocata

Ocata Therapeutics (MA, USA; www.ocata.com) announced that the first patient has been enrolled in the company’s Phase 2 clinical trial using Ocata’s proprietary retinal pigment epithelial (RPE) cells in patients with atrophic (dry) age-related macular degeneration (AMD). The purpose of the trial is to evaluate safety and explore efficacy as compared to a parallel control group. Interim top-line results from the first cohort is expected in the second quarter of 2016 and the interim read-out on the second two cohorts is expected by the end of 2016. This Phase 2 study will include up to three cohorts of up to 20 subjects each. Each of the cohorts will receive one week of immune suppressive therapy prior to surgery and then different immune suppression regimens following transplantation of the cells, to determine the safety and tolerability of 0, 6 and 12 weeks of post-transplant systemic immune suppression. Untreated patients will receive placebo treatment similar in appearance and administration to those who have received cellular transplants. Further information about this clinical trial are available at http://clinicaltrials.gov (ID: NCT02563782).

Regeneus

Regeneus (Australia; http://regeneus.com.au) has enrolled and treated the first patient under the Phase 1 clinical trial of its Progenza off-the-shelf allogeneic stem cell treatment for knee osteoarthritis. The Safety, Tolerability and Efficacy of Progenza (STEP) trial seeks to evaluate the treatment’s safety and tolerability as well as its effect on knee pain and function. Progenza is produced from MSC derived from the adipose tissue of a healthy donor who has been extensively screened. The company’s proprietary and scalable manufacturing process is capable of producing millions of therapeutic doses from one donor. A review of the sentinel patient’s safety data by the study safety oversight committee identified no safety concerns. Enrolment is now open to the remainder of the first cohort of 10 patients. A second and final cohort of 10 patients will receive a higher dose of cells.

The London Project to Cure Blindness

A pioneering trial of a new treatment derived from stem cells for people with ‘wet’ AMD has commenced at Moorfields Eye Hospital (www.moorfields.nhs.uk) following a successful operation on a patient. This first operation is a major milestone in the London Project to Cure Blindness (www.thelondonproject.org), which was established with the aim of curing vision loss in patients with wet AMD, and is the result of a partnership between the hospital, the University College London Institute of Ophthalmology (www.ucl.ac.uk/ioo), and the National Institute for Health Research (NIHR; www.nihr.ac.uk). Pfizer (NY, USA; www.pfizer.com) joined the partnership in 2009 with the goal of helping to turn the original idea into a potential therapy. The trial is investigating the safety and efficacy of transplanting hESC-derived retinal pigment epithelium derived to treat people with sudden severe visual loss from wet AMD. These cells are used to replace those at the back of the eye that are diseased in AMD. This is done using a specially engineered patch inserted behind the retina in an operation lasting one to two hours. The first surgery was successfully performed on a patient in August 2015 and there have been no complications to date. The trial will recruit 10 patients in total over a period of 18 months. Each patient will be followed for a year to assess the safety and stability of the cells and whether there is an effect in restoring vision.

ViaCyte

ViaCyte (CA, USA; http://viacyte.com), a privately-held regenerative medicine company with the first hESC-derived islet replacement therapy for the treatment of diabetes in clinical trials, opened a second site in its Phase 1/2 trial called STEP ONE, or Safety, Tolerability, and Efficacy of VC-01â„¢ Combination Product in Type One Diabetes. The new site, which is the first in Canada, will be at the University of Alberta Hospitals in Edmonton, AL. Further information about this clinical trial are available at http://clinicaltrials.gov (ID: NCT02239354).