Industry Update by Dusko Ilic: Regulations, approvals, acquisitions…

Written by Regenerative Medicine

Latest developments compiled from 1 July until 31 August 2014

Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions from 1 July until 31 August 2014, scheduled to be published in Volume 9 Issue 6 of Regenerative Medicine.


Green light

BioTime

BioTime (CA, USA; www.biotimeinc.com) has received notice from the FDA’s Center for Devices and Radiologic Health that Premviaâ„¢ has been cleared for marketing as a Class II medical device. Premviaâ„¢is the first FDA-cleared member of BioTime’s HyStem family of hydrogels, which are designed to mimic the natural structures of the human body’s extracellular matrix. According to the FDA clearance, the product is indicated for the management of wounds including: partial thickness, full-thickness, tunneling wounds, pressure ulcers, venous ulcers, diabetic ulcers, chronic vascular ulcers, donor skin graft sites, post-Moh’s surgery, post-laser surgery, podiatric wounds, wound dehiscence, abrasions, lacerations, second degree burns, skin tears and draining wounds.

BioTime/Asterias

BioTime’ (CA, USA; www.biotimeinc.com) subsidiary Asterias Biotherapeutics (CA, USA; http://asteriasbiotherapeutics.com) has received clearance from the US FDA to initiate a Phase 1/2a clinical trial of its product, AST-OPC1, in patients with complete cervical spinal cord injury. The approved trial follows the successful completion of the Phase 1 clinical study of the product, and is designed to assess safety and activity of escalating doses of AST-OPC1 in patients with complete cervical spinal cord injuries, the first targeted indication for AST-OPC1 and the first of future product registration clinical trials.

AST-OPC1 is a population of cells derived from human embryonic stem cells that contains oligodendrocyte progenitor cells (OPCs). OPCs and oligodendrocytes perform supportive functions for nervecells in the central nervous system. The foundation for this newly cleared Phase 1/2a clinical trial comes from results from the Phase 1 clinical trial of AST-OPC1, which met its primary endpoints of safety and feasibility when administered to five patients with neurologically-complete, thoracic spinal cord injury. These five patients were administered a low dose of two million AST-OPC1 cells and have been followed to date for 2 to 3 years. No serious adverse events were observed associated with the delivery of the cells, the cells themselves, or the short-course immunosuppression regimen used. There was no evidence of expanding masses, expanding cysts, infections, cerebrospinal fluid leaks, increased inflammation, neural tissue deterioration or immune responses targeting AST-OPC1 in these patients. In four of the five subjects, serial MRI scans performed throughout the 2 to 3 year follow-up period indicate that reduced spinal cord cavitation may have occurred and that AST-OPC1 may have had some positive effects in reducing spinal cord tissue deterioration.

The new Phase 1/2a clinical trial will be an open-label, single-arm study testing three escalating doses of AST-OPC1 in 13 patients with subacute, C5-C7, 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 to assess the safety and activity of the product. Selection of the clinical trial sites is well underway and Asterias expects to begin patient enrollment during the first quarter of 2015. The new clinical trial differs from the original clinical study in that doses up to 10 times higher will be tested. In addition, the trial will focus on patients with neurologically complete cervical spinal cord injuries. Because of the anatomy of the spinal cord and the existence of more sensitive outcomes measures to assess movement of the arms and hands, it is currently believed that detection of efficacy is much more likely to occur in patients with cervical injuries. It is this patient population that Asterias anticipates will be the target for the first registration clinical trials of AST-OPC1. Asterias expects that the results of the Phase 1/2a clinical trial will provide support for a Phase 2b expansion study that will be conducted to more thoroughly demonstrate safety and efficacy of the product.

CorMatrix Cardiovascular

CorMatrix Cardiovascular (GA, USA; www.cormatrix.com) has received the US FDA clearance to market the CorMatrix® ECM® for Vascular Repair. The product is intended for use as a patch material for repair and reconstruction of peripheral vasculature including the carotid, renal, iliac, femoral, and tibial blood vessels. The CorMatrix ECM for Vascular Repair may be used for patch closure of vessels, as a pledget, or for suture line buttressing when repairing peripheral vessels. It is constructed from a multi-laminate sheet of decellularized, noncrosslinked, lyophilized extracellular matrix (ECM) derived from porcine small intestinal submucosa.

Hospital Israelita Albert Einstein

Hospital Israelita Albert Einstein (HIAE; Brazil; http://apps.einstein.br/english/) earned cord blood bank accreditation form the Foundation for the Accreditation of Cellular Therapy (FACT; NE, USA; www.factwebsite.org). This is the first FACT accreditation in Brazil and South America for cord blood bank. HIAE also heads South America in previously receiving FACT accreditation of their cell therapy program for adult and pediatric allogeneic and autologous cell transplantation.

TiGenix

TiGenix (Belgium; www.tigenix.com), the European leader in cell therapy, announced today that the Committee for Medicinal Products for Human Use has renewed for an additional five years its marketing authorization for ChondroCelect® in all of the 31 countries of the EU and European Economic Area. EU legislation requires manufacturers of prescription medicines to apply for renewal of the marketing authorization after a five-year term and based on a re-evaluation of mainly safety data, only products that continue to have a positive risk-benefit ratio have their marketing authorization renewed.

ViaCyte

ViaCyte (CA, USA; www.viacyte.com) has received International Standards Organization (ISO) 13485:2003 certification, an internationally recognized quality standard for medical devices, from the British Standards Institution (www.bsigroup.co.uk), one of the world’s leading certification bodies. ISO 13485:2003 specifies requirements for a comprehensive quality management system for the design and manufacture of medical devices. ViaCyte was assessed and deemed to comply with the ISO’s requirements with respect to the design and manufacture of medical devices for encapsulation and delivery of cells with its Encaptra® drug delivery system.

In unrelated news, US FDA gave ViaCyte the go-ahead to start a phase 1/2 clinical trial in patients with type-1 diabetes after reviewing their application for an Investigational New Drug application. The trial will see if their implantable device, the VC-01â„¢, is safe and shows signs of being beneficial to patients. A promising treatment has received almost US$ 40 million in funding from the stem cell agency, the California Institute for Regenerative Medicine (CIRM; www.cirm.ca.gov). ViaCyte has also completed a private equity financing transaction, providing the Company with US$ 5.4 million through the sale of Series C-1 Preferred Stock, together with warrants to purchase common stock. The first closing of the Series C-1 financing occurred in July 2013. This additional investment brings the total invested in the Series C-1 financing to US$ 16.5 million. A nonprofit corporation and several individuals participated in the offering.

Acquisitions

Cellectis and Takara

Cellectis (France; www.cellectis.com) sold its stem cell subsidiary Cellectis AB to the Japanese Company Takara Bio (www.takara-bio.com). The Company is getting out of the stem cell business and it is now concentrating activities in the field of oncology through the development of Chimeric Antigen Receptor T-cell (CAR-T) immunotherapy products generated through its allogeneic CAR-T platform, both on its own as well as in partnership with Servier (France; www.servier.com) and Pfizer (NY, USA; www.pfizer.com).

ReproCELL, Reinnervate and BioServe

To accelerate its iPSC business, ReprocCELL (Japan; www.reprocell.com) acquired Reinnervate (UK; www.reinnervate.com) and BioServe Biotechnologies (MD, USA; www.bioserve.com). Through the combination of Reinnervate’s 3D cell culture technologies and ReproCELL’s induced pluripotent stem cell (iPSC) technologies, the next generation of iPSC products with better functionality and usability for customers will be developed and launched globally. Through the acquisition of BioServe, ReproCELL will now have access to large variety of patient derived cells in disease areas such as Alzheimer’s, Parkinsons and Cardiovascular disorders. This will accelerate the development and commercialization of diverse patient-derived iPSC products where there is a rapidly growing market need.