Industry Update: Clinical trials

Written by Dusko ILIC

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 April 01 — May 31, 2017.

Latest clinical trial developments compiled from April 01 — May 31, 2017, scheduled to be published in Volume 12 Issue 6 of Regenerative Medicine.


Asterias Biotherapeutics (CA, USA; has announced new positive serial magnetic resonance imaging (MRI) data from its ongoing AST-OPC1 SCiStar Phase 1/2a clinical trial in patients with severe spinal cord injury, including no sign of lesion cavities in any patient in both Cohort 1 (treated with 2 million AST-OPC1 cells) and Cohort 2 (treated with 10 million AST-OPC1 cells) at 6 and 12 months follow-up. The MRI results are supportive of the extensive pre-clinical data on AST-OPC1 showing that AST-OPC1 cells durably engraft and help prevent cavitation at the injury site. Cavitation is a destructive process that occurs within the spinal cord following spinal cord injuries, and typically results in permanent loss of motor and sensory function. Additionally, a patient with cavitation can develop a condition known as syringomyelia which results in additional neurological and functional damage to the patient.

Avita Medical

Avita Medical (CA, USA; has announced that they have achieved both co-primary endpoints in its pivotal clinical trial which will soon be submitted for U.S. market approval of its ReCell® device to treat severe burns. These data as well as the results from a previous burns trial will be submitted to the US FDA as part of an application to support Premarket Approval (PMA) for the ReCell® Autologous Cell Harvesting device. The Company is now focused on fulfilling requirements for remaining non-clinical data needed for the PMA submission, which is on track for mid-2017. Based on expected timelines, approval could occur by Q2 2018.


BioTime (CA, USA; has announced new data from the Phase I/IIa clinical trial of OpRegen® in the advanced form of dry age-related macular degeneration (dry-AMD). OpRegen is an investigational therapy in which retinal pigment epithelial (RPE) cells are transplanted into the subretinal space, where they are intended to replace missing RPE cells. Imaging analysis suggests the transplanted OpRegen cells remained in place (engrafted) in an area of the scar that was completely depleted of retinal pigment epithelium (RPE) because of the advanced stages of the disease. Cell engraftment occurred in four of the five patients treated thus far. There was also possible evidence of a biological response with some areas appearing to show structural improvement (a thickening of the thinned area of retina above the scar) without any signs of retinal edema, a fluid build-up that can further compromise vision.

Burst Biologics

Burst Biologics (ID, USA; has obtained approval to begin a multicentre prospective clinical study in Foot and Ankle Surgery patients. Burst Biologics latest product innovation, BioBurst® Fluid, is a cellular tissue product derived from umbilical cord blood. This study will include 10 clinical sites throughout the US with an enrolment of 300 patients. This study will include both ankle fusion patients and various osteotomy procedures. The company designed the study parameters broadly in order to evaluate complex surgeries in challenging patient populations. These populations Include patients with high BMI, Charcot, previous non-unions and smokers.

Capricor Therapeutics

Capricor Therapeutics (CA, USA; has announced positive top-line results from a safety and exploratory efficacy analysis of six-month data from the randomized 12-month Phase I/II HOPE Clinical Trial of CAP-1002 (allogeneic cardiosphere-derived cells), an investigational candidate for the treatment of patients with Duchenne muscular dystrophy (DMD). DMD is a rare, life-threatening genetic disorder for which treatment options are limited. In a 25-patient, randomized, single-dose, three-center clinical trial being conducted in a DMD population with advanced cardiac disease, patients treated with CAP-1002 demonstrated statistically-significant (p<0.05) improvement compared to usual care controls in certain measures of cardiac and upper limb function. CAP-1002 was generally safe and well-tolerated over the initial six-month follow-up period. Capricor expects to report top-line 12-month results from the HOPE-Duchenne Trial in the fourth quarter of 2017.

Capricor Therapeutics

Capricor Therapeutics (CA, USA; has announced that a pre-specified administrative interim analysis performed on six-month follow-up data from the ALLSTAR Trial, an ongoing randomized, double-blind, placebo-controlled, 142-patient Phase II clinical trial of CAP-1002 (allogeneic cardiosphere-derived cells) in adults who have experienced a large heart attack with residual cardiac dysfunction, had demonstrated a low probability of achieving a statistically-significant difference in the 12-month primary efficacy endpoint of percent change from baseline infarct size as a percent of left ventricular mass, measured by cardiac magnetic resonance imaging (MRI). Capricor will continue to perform analyses of the cumulative ALLSTAR data to better understand the basis for this outcome.


Cartiva (GA, USA; has completed enrolment and treatment of all 50 patients in a multi-center study evaluating the safety and effectiveness of Cartiva Synthetic Cartilage Implant (SCI) for first carpometacarpal (CMC) joint osteoarthritis at the base of the thumb. The study was conducted at nine sites in Canada and the U.K. Basal thumb arthritis is estimated to afflict more than two million American adults, and up to one-third of post-menopausal women. Cartiva SCI is a proprietary biocompatible polymer device designed to mimic natural cartilage. It is implanted in the metacarpal base to replace damaged cartilage without destroying or removing healthy tissue. The implant’s design minimizes bone resection while preserving the trapezium. This may provide a quicker, less painful recovery than ligament reconstruction tendon interposition (LRTI) surgery or trapeziectomy.


CollPlant (Israel;, a regenerative medicine company utilizing its proprietary plant-based rhCollagen technology for tissue repair products (recombinant human, “rhCollagen”), has announced positive results from post-marketing surveillance of the Company’s advanced wound care product, Vergenix® FG, for the treatment of patients with chronic, hard to heal wounds in Europe. As part of the surveillance, the Company monitored ten patients following a single treatment with Vergenix®FG in hospitals in Italy and Switzerland. Five weeks post- treatment, patients displayed an average wound closure rate of 80%, with no observed safety or tolerability issues. Vergenix®FG received CE Mark approval in February 2016. The Company has signed distribution agreements covering Italy and Switzerland, and initial product shipments have commenced.


Cynata Therapeutics (Australia; has announced that the first patient has been treated with CYP-001, the company’s first mesenchymal stem cell (MSC) product in its Phase 1 clinical trial in patients with steroid-resistant acute graft versus host disease (GvHD). Commencement of the trial represents a milestone for the Company and is the first time in the world that a patient has been treated with an allogeneic, induced pluripotent stem cell (iPSC)-derived therapeutic MSC product. CYP-001 is manufactured in a scalable process using Cynata’s CymerusTM platform with iPSCs as the starting material. Cynata has sourced its iPSCs from Cellular Dynamics International (WI, USA;, a Fujifilm subsidiary company. Cynata recently announced a strategic alliance with Fujifilm pursuant to which Fujifilm has taken an approximately 9% equity stake in Cynata making it the Company’s largest shareholder. These high quality clinical-grade iPSCs were derived from a single blood donation using a non-viral, non-integrating episomal reprogramming method, without genome modification. The Cymerus process overcomes both the need to source multiple donors and the inherent variability in products derived from multiple donations.


Cytori (CA, USA; has announced that the US FDA has approved an Investigational Device Exemption (IDE) for a pilot clinical trial to evaluate Cytori Cell Therapyâ„¢ in patients with thermal burn injury. This trial, named the RELIEF trial, is designed as a prospective, open-label, parallel group, usual care controlled, multi-center randomized (2:1, active : usual care alone) safety and feasibility study targeting thermal burns. Subjects will have at least one deep partial or full thickness burn wound that is to be autografted with a meshed split thickness skin graft (STSG). Subjects randomized to Cytori Cell Therapy will undergo small volume fat harvest (100 to 150 mL) during scheduled burn surgery followed by intravenous delivery of Cytori Cell Therapy. Subjects randomized to usual care will not undergo a fat harvesting procedure. The RELIEF trial will assess safety and feasibility of intravenous delivery of Cytori Cell Therapyâ„¢ as an adjunct to usual care in patients with thermal burn injuries covering between 20% and 50% of their body surface area. The trial is approved to enrol up to 30 patients in up to 10 U.S. sites. Initiation of RELIEF is dependent upon execution of a contract option by BARDA to provide the necessary funds.


Fibrocell Science (PA, USA; has announced that the Data Safety Monitoring Board (DSMB) has recommended continuation of the Phase 1/2 clinical trial of FCX-007 for the treatment of Recessive Dystrophic Epidermolysis Bullosa (RDEB), following a review of safety data from the first patient treated.  No product-related adverse events were reported. FCX-007 is Fibrocell’s clinical-stage product candidate for the treatment of RDEB, a congenital and progressive orphan skin disease caused by the deficiency of the protein type VII collagen (COL7). FCX-007 is a genetically-modified autologous fibroblast that encodes the gene for COL7 and is being developed in collaboration with Intrexon Corporation (VA, USA; By genetically modifying autologous fibroblasts ex vivo to produce COL7, culturing them and then treating wounds locally via injection, FCX-007 offers the potential to address the underlying cause of the disease by providing high levels of COL7 directly to the affected areas while avoiding systemic distribution. The FDA has granted Orphan Designation to FCX-007 for the treatment of Dystrophic Epidermolysis Bullosa, which includes RDEB. In addition, FCX-007 has been granted Rare Pediatric Disease Designation and Fast Track Designation by the FDA for treatment of RDEB.


jCyte (CA, USA; has launched a phase 2b clinical trial to test the safety and efficacy of its developmental retinitis pigmentosa (RP) treatment, jCell. The company is recruiting 70 patients for the single-masked study, which will began enrollment in April 2017. The multicenter trial is being funded by jCyte, which recently received an US$ 8.3 million matching grant from the California Institute for Regenerative Medicine (CA, USA; RP is a genetic condition that destroys retinal cells and usually strikes people in their teens. Many patients are legally blind by the time they reach 40. Worldwide, nearly 1.5 million people suffer from RP, making it the leading cause of inherited blindness. The trial’s primary goal is to assess changes in visual function following treatment with jCell, focusing on visual acuity, visual fields, contrast sensitivity and the ability to navigate a mobility course. Trial participants will receive a single RPC injection (or a sham injection as a control) in their worst-sighted eye. Progress will be assessed periodically over the following 12 months. Patients in the control group will be allowed to join the test arm after they have completed one year of follow-up.


Longeveron (FL, USA; has announced their Phase I Alzheimer’s disease trial (NCT02600130) will proceed with enrollment at the recommendation of an independent Data Monitoring Committee (DMC) which reviewed data from the trial’s safety run-in phase. The clinical trial is designed to assess the safety, tolerability and efficacy of intravenous infusion of two different doses of Longeveron mesenchymal stem cells (LMSCs) compared to placebo. The DMC reviewed safety data from a five-patient run-in phase where subjects diagnosed with Alzheimer’s disease were given a single infusion of either LMSCs or placebo. Longeveron now has the green light to continue the trial.


Mallinckrodt (UK; has confirmed the enrollment of the first patient in the company’s Phase 2 study assessing the safety, tolerability and efficacy of Stratagraft® regenerative skin tissue as an alternative to autografting full-thickness complex skin defects. StrataGraft regenerative skin tissue, is a viable, full-thickness product being developed for severe burns and other complex skin defects that is not yet approved by the US FDA. It was designed to mimic natural human skin, with both dermal and fully- differentiated epidermal layers. Unlike first generation products, this resorbable tissue can be sutured or stapled and remains intact in the wound bed, providing critical barrier functionality during the wound healing process. StrataGraft is produced using unmodified NIKS® cells grown under standard operating procedures. Because the continuous NIKS skin cell line has been thoroughly characterized, StrataGraft products are virus-free, non-tumorigenic, and offer batch-to-batch genetic consistency. The study population will include patients with up to 49% total body surface area complex skin defects that include a full-thickness component. Primary outcome measures will include the percent area of the StrataGraft treatment site requiring autografting by three months and wound closure of the treatment sites at three months. Targeted enrollment for this study is up to 20 patients with complex skin defects due to acute traumatic skin loss which require surgical excision and autografting. Subjects will be sequentially enrolled in two cohorts of increasing treatment area receiving StrataGraft skin tissue. Study completion is expected by late 2018.


Mesoblast (Australia; has announced that the Phase 3 trial of its allogenic mesenchymal precursor cell (MPC) product candidate MPC-150-IM in patients with moderate to advanced chronic heart failure was successful in the pre-specified interim futility analysis of the efficacy endpoint in the trial’s first 270 patients. This ongoing double-blinded randomized (1:1) trial is currently being conducted across multiple study sites in the U.S. and Canada. It is evaluating MPC-150-IM in adult patients with moderate to advanced New York Heart Association (NYHA) Class II/III chronic heart failure with left ventricular systolic dysfunction. The trial’s primary efficacy endpoint is a comparison of recurrent non-fatal heart failure-related major adverse cardiac events (HF-MACE) in moderate to advanced CHF patients receiving either MPC-150-IM by catheter injection into the damaged left ventricular heart muscle or sham control. After notifying the Company of the interim analysis results, the trial’s Independent Data Monitoring Committee (IDMC) additionally stated that they had no safety concerns relating to MPC-150-IM and formally recommended that the trial should continue as planned. It is expected that the trial will enroll in total approximately 600 patients.


RepliCel (BC, Canada;, a regenerative medicine company focused on developing autologous cell therapies, have reported statically and clinically significant positive data from the interim analysis of its phase I study evaluating RCS-01 (RepliCel’s type 1 collagen-expressing, hair follicle-derived fibroblasts [“NBDS cells”]) for the treatment of aging and sun-damaged skin. The primary objective of this trial was to establish a complete safety profile for intradermal injections of RCS-01 at six months post-injection. Participants in the Germany-based study did not report any serious adverse events at the interim point of the trial. Researchers also gathered compelling positive proof-of-concept data indicating the product’s potential for skin rejuvenation. The study was neither powered for, nor was expected to show statistically significant results of efficacy. However, the nearly two-fold increase in gene expression of collagen-related biomarkers in the skin, after a single injection of RCS-01, was so profound with a single RCS-01 injection, that the results are considered statistically significant.

U.S. Stem Cell

U.S. Stem Cell (FL, USA; have received from the US FDA a reactivation status of the MARVEL phase II/III trial evaluating MyoCell®, an autologous muscle stem cell therapy for the treatment of severe heart damage in heart failure patients. Following the passing of the 21st Century Cures Act, U.S. Stem Cell, Inc. has applied to the FDA for Regenerative Medicine Advanced Therapy (RMAT) Designation for the MyoCell product as part of the MARVEL trial. The trial had previously been placed on “Inactive Status” as patients were not actively being enrolled. The protocol has since been placed on “Reactivation Status” after the FDA reviewed data on the protocol and data collected on patients to date.