Industry Update: Business Developments

Latest developments compiled from 1—30 September 2014

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 September 2014, scheduled to be published in Volume 10 Issue 1 of Regenerative Medicine.

Collaborations, partnerships & alliances

Co-development agreement: Fibrocell and Interexon

Fibrocell Science (PA, USA; www.fibrocellscience.com) and Intrexon (MD, USA; www.dna.com), announced plans to file an investigational new drug application with the US FDA in the first half of 2015 for their drug candidate, GM-HDF-COL7 (genetically-modified human dermal fibroblast, collagen VII). The companies are developing this new cell-based therapeutic for the treatment of recessive dystrophic epidermolysis bullosa (RDEB), the most severe form of the rare connective tissue disorder epidermolysis bullosa. Fibrocell and Intrexon commenced preclinical activities of GM-HDF-COL7 earlier this year and have since attained certain regulatory and development achievements including Orphan Drug Designation in June and initiation of clinical material manufacturing in September.

Co-development agreement: Novartis and UPenn

Novartis (Switzerland; www.novatis.com) and the University of Pennsylvania (PA, USA; www.upenn.edu) reached an important milestone in its alliance as unveiling plans for the construction of a first-of-its-kind Center for Advanced Cellular Therapeutics (CACT) on the Penn Medicine campus in Philadelphia. The CACT will become the epicenter for research using Chimeric Antigen Receptor technology (CAR), which enables a patient’s T cells to be reprogrammed outside of the body so when they are re-infused into the patient, the T cells have the ability to “hunt” and destroy the cancer cells. Clinical trials using this approach have made headlines around the world. Plans for the 30,000-Sq. Ft. facility cement the Penn-Novartis alliance, a marquee component of Penn’s efforts in translational sciences that expedite the development of novel therapies for diseases of all kinds. The collaboration was announced in August 2012, when the two organizations entered an exclusive global research and licensing agreement to further study and commercialize novel CAR therapies. The CACT, which will be funded in part through a US$ 20 million investment from Novartis, will be devoted to the discovery, development and manufacturing of these personalized cellular cancer therapies, through a joint research and development program led by scientists and clinicians from Penn and Novartis.

Co-development and licensing agreement: Sumitomo and SanBio

Sumitomo Dainippon Pharma (Japan; www.ds-pharma.com) and SanBio (CA, USA; www.san-bio.com), have entered into a joint development and license agreement for exclusive marketing rights in the US and Canada for SB623, a cell therapy for the treatment of patients with chronic stroke discovered and currently under development by SanBio. SB623 is an allogeneic cell product, derived from bone marrow mesenchymal stromal/stem cells (MSC) isolated from healthy donors. Unlike autologous cell therapy, which requires individualized cell preparation for each patient, SB623 production can be scaled up from a single donor’s cells, enabling delivery of uniform quality products to a large number of stroke patients. In preclinical and clinical studies to date, SB623 has shown beneficial results on stroke disability with no serious adverse events. SB623 is the first cell therapy product for stroke that has been approved by the US FDA for human clinical trials. Phase I/IIa clinical tests have been completed in the US. Further information about that clinical trial are available at http://clinicaltrials.gov (ID: NCT01287936). Currently, preparations are underway to begin a Phase IIb clinical trial in the US. Under the terms of the Agreement, Sumitomo Dainippon Pharma and SanBio will jointly develop SB623 in the US and Canada, and Sumitomo Dainippon Pharma will have the exclusive right to market SB623 in both countries. In consideration of the rights and licenses granted, Sumitomo Dainippon Pharma will make an initial payment of US$ 6 million and milestone payments totaling an additional US$ 74 million during the clinical development of SB623. After market launch, SanBio will supply the finished product to Sumitomo Dainippon Pharma and receive double-digit percentage royalties based on sales. Additionally, SanBio may receive sales milestone payments contingent upon the achievement of annual sales goals, up to a total of US$ 125 million. The development expenses will be shared equally between the two companies.

Licensing agreement: Cynata and University of Wisconsin

Cynata Therapeutics (Australia; www.cynata.com) has entered into a license agreement with Cellular Dynamics International (WI, USA; www.cellulardynamics.com). The agreement provides Cynata with nonexclusive worldwide rights to a clinical grade human induced pluripotent stem cell (iPSC) line manufactured by Cellular Dynamics. Cynata intends to use this iPSC line as high quality starting material to manufacture its Cymerusâ„¢ MSC products for human therapeutic use.

Licensing agreement: ImmunoCellular and Caltech

ImmunoCellular Therapeutics (CA, USA; www.imuc.com) has established a licensing agreement with the California Institute of Technology (www.caltech.edu) for exclusive rights to novel technology for the development of certain antigen specific T-cell immunotherapies for the treatment of cancer. The technology utilizes the patient’s own hematopoietic stem cells to create antigen-specific killer T-cells to treat cancer. ImmunoCellular plans to utilize this technology to expand and complement its dendritic cell-based cancer vaccine platform, with the goal of developing new immunotherapies that kill cancer cells in a highly directed and specific way, and that can function as single agents or in combination approaches. Caltech’s technology addresses the challenge, and limitation, that CAR and T-cell receptor technologies have faced of generating a limited, short-lived immune response. By putting T-cell receptors into stem cells rather than into T-cells, the immune response can be transformed into a long-lived and potent response that could effectively treat previously resilient solid cancers. This observation has been verified in animal models by investigators at Caltech and the National Cancer Institute.

Partnership agreement: CellProThera and BioCardia

CellProThera (France; www.cellprothera.com/?lang=en) and BioCardia (CA, USA; www.biocardia.com) partnered on the EXCELLENT (Expanded Cell Endocardiac Transplantation) trial, a multi-center, controlled Phase I/IIb study to evaluate the safety and efficacy of in vitro expanded peripheral blood CD34+ stem cells output by the CellProThera’s StemXpand® Automated Process and delivered via the BioCardia Helixâ„¢ Transendocardial Delivery System.

Launching new projects, products and services

Bioheart

Bioheart (FL, USA; www.bioheartinc.com) opened of the laboratory and clinic in South Africa. The new facility, named South African Stem Cell Institute (SASCI), immediately began treating patients. Last week a total of 12 patients underwent autologous cell therapies for the treatment of spinal cord injury, diabetes, arthritis, autoimmune disease and more. All of the patients received an in-clinic treatment as well as cell culture expansion and cryopreservation. The patient cells were multiplied and will be preserved in sub-zero temperatures for future treatments as required. Bioheart’s new joint venture has established an important relationship within the South African government. The Ministry of Health will be adopting Bioheart protocols making them the standard of care for patients in South Africa. Bioheart will assume 49% ownership of the new entity.

Health 100

Medical entrepreneur Xia Jie, whose company Health 100 (www.health-100.cn) owns the largest chain of health clinics in China, plans to open facilities in Queenstown, New Zealand to cater for wealthy clients. Health 100 would invest with existing firms Queenstown Regenerative Medicine (www.queenstownrm.co.nz) and the Queenstown Skin Institute (www.skininstitute.co.nz).

Regeneus

Regeneus (Australia; http://regeneus.com.au) announced plans to open the HiQCell Regenerative Medicine Clinic at the Camden Medical Centre in Singapore. The HiQCell treatment involves harvesting a small amount of a patient’s own stem cells from their adipose (fat) tissue and after separating and concentrating the regenerative cells these are re-injected in osteoarthritic-affected joints such as knees, hips and ankles. The HiQCell treatment, which aims to reduce inflammation and repair damaged tissue is carried out under the supervision of the treating medical practitioner. HiQCell has been used to treat more than 500 patients and over 1,000 arthritic joints. The cell therapy procedure is supported by safety data from a randomized controlled clinical trial and long-term safety and efficacy data is tracked through Australia’s first Joint Registry for stem cell therapy.

Royan Institute

Royan Institute’s (Iran, www.royaninstitute.org) spin off stem cell technology firm has received a positive response from the private and the government sector, to launch their activities in Oman. Royan Cord Blood Bank, founded in 2005 under the institute, storing more than 33,000 samples in branches in more than 22 cities nationwide, will be setting up cord blood bank in Oman.