Industry Update: Regulations, approvals & acquisitions

Latest developments compiled from 1–30 September 2016

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Apr 18, 2017
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Asterias Biotherapeutics (CA, USA; announced that its Data Monitoring Committee has reviewed the safety data from the initial cohort of three patients dosed with 2 million cells, and a subsequent five patients in the second cohort dosed with 10 million cells, and has cleared the company to now begin dosing a third cohort of 5–8 complete cervical injury patients (AIS-A patients) with the highest dose of 20 million cells. Concurrently, the study is also proceeding with enrolling the first cohort of 5–8 sensory incomplete cervical SCI patients (AIS-B patients), each of whom will be administered ten million cells. The SCiStar study is an ongoing Phase I/IIa clinical trial funded in part by a US$14.3 million grant from the California Institute for Regenerative Medicine and is designed to evaluate the safety and efficacy of escalating doses of AST-OPC1 in newly injured patients with sensory and motor complete cervical SCI as well as newly injured patients with sensory incomplete SCI. These patients are commonly referred to as AIS-A and AIS-B patients, respectively. Additional information on the Phase I/IIa trial, including trial sites, can be found at (ID: NCT02302157), and at the SCiStar Study website (


Athersys (OH, USA; has received agreement from the FDA under a special protocol assessment for the design and planned analysis of a Phase III Study of MultiStem Treatment for Ischemic Stroke. The Phase III trial titled, MASTERS2, would provide the foundation of the regulatory package to be submitted for marketing approval. Multistem cell therapy is a patented regenerative medicine product based on stem cells isolated from bone marrow or other tissues, that have the ability to promote tissue repair and healing through the production of therapeutic factors produced in response to signals of inflammation and tissue damage. The MASTERS2 clinical trial will be a randomized, double-blind, placebo-controlled clinical trial designed to enroll 300 patients in North America and Europe who have suffered moderate to moderate-severe ischemic stroke. The enrolled subjects will receive either a single intravenous dose of MultiStem cell therapy or placebo, administered within 18–36 h of the occurrence of the stroke, in addition to the standard of care. The primary end point will evaluate disability using modified Rankin Scale scores at 3 months, comparing the distribution between the MultiStem treatment and placebo groups. The modified Rankin Scale shift analysis considers disability across the full spectrum, enabling recognition of large and small improvements in disability and differences in mortality and other serious outcomes, among strokes of different severities. The study will also assess excellent outcome (modified Rankin Scale ≤1, NIHSS ≤1 and Barthel Index ≥95) at 3 months and 1 year as key secondary end points. Additionally, the study will consider other measures of functional recovery, biomarker data and clinical outcomes, including hospitalization, mortality and life-threatening adverse events, and post-stroke complications such as infection.

Furthermore, the company announced the successful completion of Japan's Pharmaceutical and Medical Devices Agency review of the Clinical Trial Notification, allowing the commencement by Healios (Japan, of a confirmatory clinical trial evaluating the safety and efficacy of administration of MultiStem for the treatment of ischemic stroke in Japan (also designated by Healios as HLCM051 in Japan). In accordance with the regulatory system in Japan, a Clinical Trial Notification is equivalent to an Investigational New Drug (IND) application under the FDA. The clinical trial to be conducted in Japan stems from a partnership and license agreement between Healios and Athersys, and consists in a randomized, double-blind, placebo-controlled clinical trial where subjects will receive either a single dose of MultiStem or placebo, administered within 18–36 h of the occurrence of the stroke, in addition to standard of care. The study will evaluate patient recovery through approximately 90 days following initial treatment based on excellent outcome and other neurological, functional and clinical end points. The trial in Japan follows a Phase II study, B01-02 trial, which was completed at 33 clinical sites in the USA and UK, and evaluated safety and effectiveness of the intravenous administration of MultiStem cells within 24–48 h after the occurrence of a moderate-to-severe stroke on 126 subjects.


The EMA has granted access to its Priority Medicines scheme for Bluebird Bio's (MA, USA; proprietary product LentiGlobin in the treatment of patients with TDT. The Priority Medicines initiative provides enhanced support and increased interaction to companies, with the goal of optimizing development plans and speeding regulatory evaluations to bring innovative medicines to patients more quickly. Bluebird Bio is also participating in the EMA's Adaptive Pathways Pilot program, which also aims to expedite patient access to new therapies. LentiGlobin BB305 product aims to treat TDT and severe sickle cell disease (SCD), by inserting ex vivo a functional human β-globin gene into a patient's own hematopoietic stem cells outside the body and then transplanting those modified cells into the patient's blood stream. Promising results from a preclinical proof-of-concept study using gene therapy to treat SCD were published earlier [2]. Bluebird Bio has three ongoing studies evaluating LentiGlobin therapy for the treatment of TDT and severe SCD – the Northstar Study in TDT, the HGB-205 study in TDT or severe SCD and the HGB-206 study in severe SCD.


Cynata Therapeutics (Australia, has received approval from the UK Medicines and Healthcare products Regulatory Agency to proceed with its Phase I clinical trial of CYP-001 in patients with steroid resistant GvHD. CYP-001 is Cynata's lead Cymerus MSC product, developed through the Cymerus technology utilizing iPSC and mesenchymoangioblasts to achieve economic manufacture of MSC at commercial scale. Cynata plans to conduct the Phase I clinical trial, which is titled “An Open-Label Phase I Study to Investigate the Safety and Efficacy of CYP-001 for the Treatment of Adults With Steroid-Resistant Acute Graft Versus Host Disease”, at a number of leading clinical centers in the UK and Australia. Additional centers in other jurisdictions are also being considered. The trial will aim to recruit approximately 16 participants who have undergone a bone marrow transplant or similar procedure, and were subsequently diagnosed with steroid resistant grade II–IV acute GvHD.


The FDA has granted Orphan Drug Designation to Fate Therapeutics (CA, USA; for ProTmune™. The FDA designation is for “prevention of GvHD in patients undergoing allogeneic hematopoietic cell transplantation” and broadly covers diseases, including blood cancers and genetic disorders, for which the procedure is performed. ProTmune™ is an investigational programmed cellular immunotherapy undergoing clinical development for the prevention of acute GvHD and cytomegalovirus infection in patients undergoing allogeneic HCT. The cell therapy is produced by modulating a donor-sourced, mobilized peripheral blood graft ex vivo with two small molecules (FT1050 and FT4145) to enhance the biological properties and therapeutic function of the graft's immune cells. The programmed mobilized peripheral blood graft is administered to a patient as a one-time intravenous infusion.



Regen BioPharma (CA, USA; submitted a revised IND application to the FDA, which will be focusing on using a novel method to unleash the body's innate ability to kill cancer. The package submitted contained preclinical experiments as well as details of the proposed clinical trial, and proposes to combine the company's proprietary dCellVax product with silencing through siRNA of Indoleamine 2,3-dioxygenase, an enzyme that is overexpressed in some cancers and which suppresses immune system response against the cancer. The proposed dCellVax clinical trial involves generation of patient-specific dendritic cells that are modified by gene-silencing to lose expression of the immune checkpoint gene Indoleamine 2,3-dioxygenase. Ten patients with advanced breast cancer will be treated in the proposed clinical trial. The company has also submitted additional data to the FDA supporting its application requesting Orphan Drug status for the use of HemaXellerate in aplastic anemia. HemaXellerate is comprised of cells extracted from the patient's own fat tissue and processed using a proprietary method to induce a biological response in the patient that heals damaged bone marrow and restores ability of the body to generate healthy blood cells. The FDA recently cleared Regen to perform Phase I clinical trials using HemaXellerate in aplastic anemia patients.

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Dusko ILIC

Reader in Stem Cell Science, King's College London

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