Industry Update: Regulations, approvals, acquisitions
Latest industry developments compiled from October 01 — November 30 2016
Latest industry developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions from October 01 — November 30 2016, scheduled to be published in Volume 12 Issue 2 of Regenerative Medicine.
Green light
Biological Industries
Biological Industries (Israel; www.bioind.com) have announced that the US FDA has accepted the submission for its Drug Master File (DMF) for the company’s NutriStem® hPSC XF Medium, a chemically defined, xeno-free, serumfree media, which is designed to support the growth of human embryonic stem cells (hESCs) and iPSCs. A DMF is a confidential detailed document submitted to the FDA by a manufacturer that includes the Chemistry, Manufacturing and Controls information about their product. An active DMF enables clinical investigators to cross-reference the DMF in their own sponsored IND-application.
Celyad
Celyad (Belgium; www.celyad.com) has been approved in Belgium to initiate the THerapeutic Immunotherapy with NKR-2) THINK clinical trial. THINK, a multinational open-label Phase Ib study to assess the safety and clinical activityof multiple administrations of autologous natural killer receptor (NKR)-2 T-cells, is the second clinical trial of its NKR-2 product candidate, a CAR-T cell therapy using NK group 2D (NKG2D) ligands as a target, to evaluate safety and efficacy in seven cancer indications including both solid and hematological malignancies. The dose escalation design will include three dose levels adjusted to body weight: up to 3×108, 1×109 and 3×109 NKR-2 T-cells. At each dose, the patients will receive three successive administrations, two weeks apart, of NKR-2 T-cells at the specifffied dose. The dose escalation part of the study will enroll up to 24 patients while the extension phase would enroll 86 additional patients.
Cytori
Cytori Therapeutics (CA, USA; www.cytori.com) announced that the US FDA Office of Orphan Products Development (OOPD) has granted Cytori an orphan drug designation for cryopreserved or centrally processed autologous adipose-derived stem and regenerative cell (ADRC) therapy ECCS-50 for scleroderma. Orphan drug designation is granted by the FDA to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Orphan drug designation provides certain incentives, which may include tax credits towards the cost of clinical trials and waivers from FDA user fees. If a product that has orphan drug designation subsequently receives the first FDA approval for the disease for which it has such designation, the product is entitled to orphan product exclusivity for seven years.
Cytori published two-year clinical follow-up of patients treated with a single administration of ECCS-50 in the SCLERADEC-I pilot trial of Cytori Cell Therapy in patients with hand dysfunction associated with scleroderma [3].
Elanix
Elanix Biotechnologies (Germany; https://elanixbiotechnologies.com) announced that their licensed partner Transwell Biotechnology (Taiwan; www.tw-bio.com), subsidiary of Easywell Biomedicals (Taiwan; www.easywellbio.com/en/index.php) has received investigational new drug (IND) clearance from the US FDA to conduct combined phase I/II clinical trial (https://clinicaltrials.gov; ID: NCT02737748). The study will begin in Japan and Taiwan. The study will compare safety and efficacy of TWB-103 (skin fibroblast progenitors as provided by Elanix Biotechnologies AG) add-on Tegadermâ„¢ to standard of care Tegaderm (skin dressing) alone. Primary objectives are to evaluate the safety and efficacy of TWB-103 in split-thickness skin graft donor site wounds. Enrolment of patients is expected by Q2 2017.
Gamida
Gamida Cell (Israel; www.gamida-cell.com) announced that the US FDA has granted Breakthrough Therapy Designation to the Company’s lead product candidate, NiCord®, in development as a novel graft modality for bone marrow transplantation in patients with high risk hematological malignancies such as leukemia and lymphoma. NiCord is an ex vivo expanded cell graft, utilizing Gamida Cell’s proprietary NAM platform technology (nicotinamide as supplement to the media) to expand cells from umbilical cord blood. NiCord is enriched with highly functional stem and progenitor cells as well as with immune modulatory myeloid derived dendritic cells. An open-label, controlled, multicenter, international, Phase III, randomized study of transplantation of NiCord versus transplantation of one or two non-manipulated, unrelated cord blood units in patients with acute lymphoblastic leukemia or acute myeloid leukemia, myelodysplastic syndrome or chronic myeloid leukemia will begin before the end of the year.
Nature Cell
Nature Cell (Korea; www.naturecell.co.kr/en/) has announced AstroStem’s approval for Phase I and Phase II clinical trials for treatment of Alzheimer’s disease by the U.S. FDA. AstroStem is a therapeutic agent for adult stem cells that collects about 10g of fat from the patient’s own abdominal subcutaneous tissue and separates only pure stem cells into a finished product, which is repeatedly administered intravenously to the patient. Production of the approved clinical trial materials will be done by the RBio Biostar Stem Cell Research Institute (Japan; http://jbiostar.com), which has been developing technology for dementia reduction for more than 10 years.
Red light
ICMR
The Indian Council of Medical Research (ICMR; www.icmr.nic.in/About_Us/About_ICMR.html) has derailed a controversial experiment that would seek to revive brain-dead accident victims. On 11 November, ICMR’s National Institute of Medical Statistics removed the “ReAnima” trial (http://reanima.tech) from India’s clinical trial registry. Anupam Hospital in the north Indian state of Uttarakhand, announced in May 2016 plans to give around 20 brain-dead people a mix of interventions including injections of MSC and peptides, and transcranial laser stimulation and median nerve stimulation. Transcranial laser stimulation involves shining pulses of near-infrared light into the brain; median nerve stimulation is the electrical stimulation of a major nerve that runs from the neck to the arm. Both techniques have been shown to improve cognition in patients with traumatic brain injury. Bioquark (PA, USA; www.bioquark.com) supplies the trial with peptides that are supposedly helping regeneration of brain cells.