Industry Update: Regulations, approvals, acquisitions
Latest developments in the field of stem cell research and regenerative medicine compiled from publicly available information and press releases from non-academic institutions June 01 — June 30, 2017.
Latest regulations, approvals & acquisitions compiled from 01 — June 30, 2017, scheduled to be published in Volume 12 Issue 7 of Regenerative Medicine.
Green light
ReNeuron
ReNeuron Group (UK; www.reneuron.com) has provided an update on its cell therapy development programmes targeting degenerative diseases of the retina. The FDA has approved the cryopreserved formulation of their human retinal progenitor cell (hRPC) therapeutic candidate and they have now started treating patients with this formulation in our ongoing US Phase I/II study clinical trial in retinitis pigmentosa (RP) patients. The new proprietary formulation enables the hRPCs to be frozen for shipping and storage and easily thawed at the point of clinical use. This freeze-thaw modality provides a greatly enhanced shelf life for the product, lower prospective cost of goods and the capability to ship the cells for clinical and commercial application anywhere in the world. The new hRPC formulation has also facilitated an expansion of ReNeuron’s clinical programmes in ophthalmology. ReNeuron is filing an application with the FDA to expand the Phase II element of the ongoing Phase I/II clinical trial in RP from six to 20 patients. The expanded study is designed to provide the depth and quality of data that, if positive, will allow subsequent progression to a Phase II/III pivotal study in this indication. The company also intend to file an application to start a new US Phase II clinical trial later this year in patients with cone-rod dystrophy, to be conducted alongside the Phase II part of the ongoing RP clinical trial.
Pending
Kadimastem
Kadimastem (Israel; www.kadimastem.com) has received approval from the IRB (Helsinki Committee) of the Hadassah Ein Kerem Hospital for the phase I/IIa clinical trial in ALS patients using the cell therapy product developed by the Company. The commencement of the clinical trial is subject to the approval of Ministry of Health’s Supreme Committee for clinical trials in humans, which is expected to convene in the upcoming months. Kadimastem’s product, AstroRx® is a human embryonic stem cell (hESC)-based treatment for amyotrophic lateral sclerosis (ALS), based on astrocytes produced from stem cells. Kadimastem’s unique technology enables large-scale production of the cells according to Good Manufacturing Practices (GMP) standard. The cells will be injected into the patients’ spinal fluid using a standard injection procedure, performed routinely in hospitals worldwide.
Acquisitions
Cell Medica
Cell Medica (UK; https://cellmedica.com) has announced the acquisition of Catapult Therapy TCR Ltd, a subsidiary of the CGT Catapult (UK; https://ct.catapult.org.uk), and the initiation of a collaboration to establish cell therapy manufacturing for Cell Medica at CGT Catapult’s GMP manufacturing facility in Stevenage, UK. Financial terms were not disclosed. Catapult Therapy TCR Ltd is a special purpose company set up by CGT Catapult, University College London (UCL) Business and Imperial Innovations, and managed by CGT Catapult, for the development of the WT1 T cell receptor (TCR) cell therapy discovered through research at UCL and Imperial College London. The WT1-TCR cell therapy enhances the immune system to fight cancer by genetically engineering the patient’s T cells to target WT1, a tumor-associated antigen which is expressed in both solid tumors and blood cancers. CGT Catapult has been developing the WT1-TCR cell therapy for the treatment of acute myeloid leukemia and myelodysplastic syndrome. Early development work, including initiation of a Phase I trial, was conducted at UCL and Imperial College London with funding from the UK charity Bloodwise. CGT Catapult advanced the product to a larger Phase I/II clinical trial and developed an improved manufacturing process. Having completed the treatment of eight patients with promising results, CGT Catapult will now transfer the WT1-TCR cell therapy rights to Cell Medica for continued development towards regulatory approval. The WT1-TCR cell therapy will be integrated with the Dominant TCR platform technology which Cell Medica licensed from UCL Business in 2016. Applying the Dominant TCR technology to the WT1-TCR cell therapy is expected to result in a more efficacious product with the potential to treat patients with solid tumors such as mesothelioma and ovarian cancer, which have proven very difficult to treat with conventional therapies. Cell Medica is planning to initiate a Phase I/II clinical trial with a Dominant WT1-TCR version in late 2018.
Cell Medica and CGT Catapult have also initiated a collaboration to establish cell therapy manufacturing operations for Cell Medica at the GMP production facility recently built by CGT Catapult in Stevenage. The collaboration will include transferring the current WT1-TCR cell therapy manufacturing process to Stevenage over the next twelve months while Cell Medica and CGT Catapult work to develop a commercial scale production process using advanced manufacturing techniques. Cell Medica will also evaluate the feasibility of manufacturing additional cell therapy products at the site.
Sanpower and Dendreon
Dendreon Pharmaceuticals (CA, USA; www.dendreon.com) has closed a transaction in which Sanpower Group (http://en.sanpowergroup.com), a private Chinese conglomerate, acquired Dendreon from an affiliate of Valeant Pharmaceuticals International (QC, Canada; www.valeant.com) for US$ 819.9 million in cash. By operating as a standalone company, under the ownership of Sanpower Group, Dendreon will strive to further accelerate and expand access to its flagship immunotherapy product PROVENGE® (sipuleucel-T) to prostate cancer patients worldwide. PROVENGE is an autologous cellular immunotherapy indicated for the treatment of asymptomatic or minimally symptomatic metastatic castrate-resistant hormone refractory prostate cancer.