Industry Update: Regulations, approvals, acquisitions
Latest business developments compiled from April 01 — May 31, 2016.
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 from March 01 — 31 2016, scheduled to be published in Volume 11 Issue 6 of Regenerative Medicine.
Guidelines
ISSCR
The International Society for Stem Cell Research (ISSCR; www.isscr.org), the world’s largest professional organization of stem cell scientists, released newly updated guidelines for stem cell research and the development of new clinical therapies. Guidelines for Stem Cell Research and Clinical Translation [6]. At their core, the new guidelines preserve the imperative for a specialized oversight process for research involving human embryos, in recognition of the unique sensitivities surrounding such research. Responding to advances in science, the guidelines encompass a broader and more expansive scope of research and clinical endeavor than before, imposing rigor on all stages of the research, addressing the cost of regenerative medicine products, and highlighting the need for accurate and effective public communication.
Green light
Cytori
Cytori Therapeutics (CA, USA; www.cytori.com) announced that the European Commission has granted orphan drug status to a broad range of Cytori Cell Therapy® formulations when used for the treatment of hand dysfunction and Raynaud’s Phenomenon in patients with scleroderma. Key benefits of this broad orphan drug designation include: (1) the ability to pursue multiple therapeutic formulations for systemic scleroderma that are not restricted to the processing of the adipose-derived cellular therapeutics at the bedside with the Celution® System or other current technology, (2) allows adipose-derived cellular therapeutics that are cryopreserved for later administration and (3) provides a pathway for Cytori to expeditiously seek conditional market approval in the EU.
SanBio
San Bio (CA, USA; www.san-bio.com) is now able to conduct the world’s first global Phase 2 clinical trial for chronic traumatic brain injury with allogeneic stem cells in Japan. The 30-day review period of Clinical Trial Notification of the regenerative cell therapy SB623 for traumatic brain injury in Japan, which was submitted to the Pharmaceuticals and Medical Devices Agency (PMDA; www.pmda.go.jp/english/# ) in March 2016, has passed without further concern. The STEMTRA “Stem cell therapy for traumatic brain injury” trial will study the safety and efficacy of SB623 cell therapy in treating patients with chronic motor impairments following a traumatic brain injury. Enrollment began in the United States in October 2015 and, going forward, will include clinical trial sites and patients in Japan. The trial will enroll 52 subjects, and the inclusion of Japanese patients is expected to accelerate overall enrollment. Additional information about this clinical trial is available at http://clinicaltrials.gov (ID: NCT 02416492).
Stemedica
Stemedica Cell Technologies (CA, USA: www.stemedica.com) received an FDA investigational new drug (IND) approval for a US-based, Phase IIa clinical study using its ischemic tolerant, adult allogeneic itMSC to treat subjects with traumatic brain injury. The study is sponsored by Stemedica with ProMedica Health Care Systems of Toledo, Ohio serving as both the trial site and a co-financial supporter of the trial. The clinical trial is titled “A Phase IIa, Open-Label Study to Assess the Safety, Tolerability, and Preliminary Efficacy of a Single Intravenous Dose of Allogeneic Human Mesenchymal Stem Cells to Subjects with Moderate Traumatic Brain Injury.”
TRACT
TRACT Therapeutics (IL, USA: www.tracttherapeutics.com) has received US FDA approval to initiate a Phase 2 clinical trial to investigate the efficacy and safety of its novel therapy for kidney transplant recipients. The therapeutic approach, utilizing a patient’s own cells, expanded in the laboratory and infused back to the patient after an organ transplant, shows promise in restoring immune balance so the body will accept the foreign organ instead of rejecting it. The Phase 2 study of 120 subjects will evaluate the efficacy and safety of a single Treg infusion in kidney transplant recipients and is projected to initiate enrollment in a few months.
Acquisitions
Promethera and Cytonet
Promethera Biosciences (Belgium; www.promethera.com) has acquired key assets of cell therapy developer Cytonet for an undisclosed amount of the buyer’s shares. The deal would create a broader and more diversified pipeline to address a wide range of liver diseases–including fibrosis, nonalcoholic steatohepatitis, acute chronic liver failure, and hemophilia. The combined company would offer Cytonet’s precommercialization-stage Heparesc® cellular therapy involving primary human hepatocytes, as well as Promethera’s HepaStem® and H2Stem® human liver-derived stem cell therapies for inborn liver metabolic diseases and acquired liver diseases with high unmet medical needs.
Name change
SiDanSai
As part of its strategy for market expansion, SiDanSai Biotechnology (China; www.sidansai.com) has officially changed its English name to Innovative Cellular Therapeutics (ICT) (www.ictbio.com), reflecting the global vision of ICT in making continuous breakthroughs in cell therapy and benefiting human wellness. The Chinese name of SiDanSai remains unchanged.
SiDanSai has recently released its first batch of CAR-T clinical data after completing a CAR-T trial with 23 patients suffering from relapsed and refractory leukemia, of which 20 patients have achieved complete remission, proving an 87% complete remission rate.
References
[6 ]http://www.isscr.org/docs/default-source/guidelines/isscr-guidelines-for-stem-cell-research-and-clinical-translation.pdf?sfvrsn=2 (Accessed June 29, 2016).