Industry Update: Regulations, approvals, acquisitions…
Latest regulatory, approval and acquisition developments compiled from 1—30 June 2015
Latest developments in the field of stem cell research and regenerative
medicine compiled from publicly available information and press releases
from non-academic institutions 1—30 June 2015, scheduled
to be published in Volume 10 Issue 7 of Regenerative Medicine.
Green light
Juno
US FDA has accepted Investigational New Drug (IND) application from Juno Therapeutics (WA, USA; https://junotherapeutics.com) for its drug JCAR017, which is an immunotherapy for non-Hodgkin lymphoma. The drug works by taking the patient’s own cells out, essentially training them to fight the cancer cells, then infusing them back into the patient’s body. Early tests showed a 91% remission rate in patients with leukemia and lymphoma.
Q Therapeutics
Q Therapeutics (UT, USA; www.qthera.com) has received clearance from the US FDA for IND) application for the initiation of Phase 1/2a clinical trials of its Q-Cells® product in patients with amyotrophic lateral sclerosis. Q-Cells, the Company’s first patented cellular therapeutic product candidate, are glial-restricted progenitor cells — early descendants of neural stem cells that produce only glia, which make up to 50% percent of cells in the brain.
Stemedica
Stemedica Cell Technologies (CA, USA; www.stemedica.com) has received the US FDA’s IND approval for a US-based, Phase IIa clinical study using its allogeneic stem cell therapy to treat subjects with mild to moderate dementia due to Alzheimer’s disease. Stemedica’s bone marrow-derived, allogeneic ischemia-tolerant (it) MSCs are grown under hypoxic conditions that more closely resemble the environment in which they live in the body. Compared to other MSCs, itMSCs secrete higher levels of growth factors usually associated with angiogenesis and healing.
TxCell
US FDA has accepted IND application from TxCell (France; www.txcell.com) for their lead product Ovasave®, currently in a phase 2b clinical trial for the treatment of patients with refractory Crohn’s disease. The activation of the IND authorizes TxCell to extend the CATS29 study to the U.S. The CATS29 study is currently on-going in Europe following its start in December 2014. There are currently 30 study sites operating the study in 6 countries in the EU. It has been designed to include 160 severe refractory Crohn’s disease patients. Additional information on the CATS29 study can be found at www.clinicaltrials.gov (ID: NCT02327221).
Pending
TxCell
TxCell (France; www.txcell.com) has temporarily put its pilot manufacturing facility activities at Besançon, France on partial hold. The decision was made in agreement with the French regulator, Agence Nationale de Sécurité du Médicament, (ANSM; http://ansm.sante.fr). Manufacturing of new products by TxCell at Besançon will pause until compliance is ensured. This is expected before the end of the year. Treatment of patients in the CATS29 study (an ongoing phase 2b clinical trial with Ovasave to treat refractory Crohn patients) for whom at least one injection had been given, will continue. This is subject to the implementation of a risk analysis pre-approved by ANSM. Additional information on the CATS29 study can be found at www.clinicaltrials.gov (ID: NCT02327221).
Name Change
Cardio3 Biosciences into Celyad
Having expanded beyond its initial focus on cardiovascular diseases through the acquisition of CAR-T assets, Cardio3 Biosciences (Belgium) changed name into Celyad (www.celyad.com).
Acquisitions
AMAG and Cord Blood Registry
Cord Blood Registry (CA, USA; www.cordblood.com) has signed a definitive agreement to be acquired by AMAG Pharmaceuticals (MA, USA; www.amagpharma.com), a specialty pharmaceutical company that focuses on maternal health and hematology, for US$ 700 million. The transaction is expected to close in the third quarter upon the receipt of regulatory approvals and satisfaction of other customary conditions.
NantCell and VivaBioCell
NantCell, a member of the NantWorks group (CA, USA; www.nantworks.com) has completed the acquisition of privately held VivaBioCell (Italy; www.vivabiocell.it). VivaBioCell’s automated “GMP-in-a-Box” medical devices enable safe, efficacious, standardized and affordable medical treatments by creating a next generation tissue engineering manufacturing capability. Current applications include developing stem cell for the treatment of osteoarthritis as well as bone regeneration for dental/maxillofacial reconstruction. This automated “GMP-in-a-Box” system will be utilized to develop next generation manufacturing for immune cells utilized in the treatment of cancer such as natural killer cells. As part of the transaction, NantCell also acquired VivaBioCell’s diagnostic product, capable of identifying patients with systemic sclerosis and systemic lupus erythematosus.