Industry Update: Regulations, approvals, acquisitions

Written by Dusko ILIC

Latest regulations, approvals, acquisitions compiled from August 1— September 30 2015

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 August 1— September 30 2015, scheduled to be published in Volume 11 Issue 1 of Regenerative Medicine.

Green light

Cell Cure Neurosciences

Cell Cure Neurosciences (Israel; has received from the US FDA Fast Track designation for OpRegen®, a cell-based therapeutic product consisting of RPE cells designed to block the progression of the severe dry-form of AMD.


Kiadis Pharma (the Netherlands; has obtained regulatory approvals from the national authorities both in Canada and in Belgium to start a further Phase II clinical trial with its lead product ATIR101â„¢. ATIR101 is a cell-based product designed to enable stem cell transplantations from partially matched (haploidentical) family donors for blood cancer patients who do not have a matching stem cell donor available. Previously announced results obtained in prior and ongoing clinical studies confirm the safety and efficacy of a single dose of ATIR101 at 2 x 106 cells/kg, with no grade III-IV (life-threatening) acute Graft versus Host Disease (GvHD) occurring, despite the fact that no prophylactic immune suppressants were used. Furthermore, only limited severe infections and relapses were reported in those studies. The aim of the new Phase II trial is to test whether the administration of a second dose of ATIR101 to a stem cell transplantation at a later point during treatment could further improve the transplantation outcome. The study will commence in Canada and Belgium and the Company plans to expand the study to the USA and other European countries pending regulatory approvals. A total of 15 leukaemia patients with acute myeloid leukaemia (AML), acute lymphoblastic leukaemia (ALL) or myelodysplastic syndrome (MDS) will be enrolled. Kiadis expects to enroll the first patient into this study in Q4 2015. Further information about this clinical trial are available at (ID: NCT02500550).


Mesoblast (Australia; announced that its licensee JCR Pharmaceuticals (Japan; has received full approval from the Japanese Ministry of Health, Labour and Welfare for TEMCELL® HS Inj (JR-031), an allogeneic MSC product. TEMCELL is a treatment for children and adults with acute Graft Versus Host Disease, a severe complication arising from hematopoietic cell transplants. TEMCELL has been developed in Japan by JCR utilizing technology under a license from Mesoblast. Under its agreement with JCR, Mesoblast is entitled to receive a milestone payment on this product regulatory approval, as well as royalties and other payments at pre-defined thresholds of cumulative net sales.


Pluristem Therapeutics (Israel; announced that Japan’s Pharmaceuticals and Medical Devices Agency (PMDA; has cleared the Company’s PLX-PAD cells for use in clinical trials in Japan. This clearance is required in order to apply for approval to conduct a Phase II study of PLX-PAD in critical limb ischemia through Japan’s accelerated regulatory pathway for regenerative medicine. This regulatory pathway generally allows for conditional, time-limited marketing approval after a single successful Phase II trial.



Allergan (CA, USA; has resubmitted its Supplemental Biologics License Application (sBLA) for Botox, providing the US FDA with additional study data on the effects of the injectable drug on lower limb spasticity. The resubmission includes further data from a double-blind, placebo-controlled study involving 468 patients. The study showed that as early as week two, a single injection reduced post-stroke spasticity in the lower limbs. Allergan says the improvement in muscle tone was clinically meaningful, the drug was well-tolerated and no new safety signals were observed. The US FDA has assigned a six-month review period for the sBLA for treatment of ankle and toe muscles, putting the Prescription Drug User Fee Act (PDUFA) date sometime in the first quarter of 2016, according to Allergan. In April, the FDA approved Botox (onabotulinumtoxin A) to treat increased stiffness in two thumb muscles, expanding a previous approval in 2010 for the elbow, wrist and fingers in patients 18 and older with upper limb spasticity.