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Welcome to your cell therapy deal review for the month of August. Synthetic biology giant Intrexon closed a large financing; Immune Design announced a collaboration to pair its viral-based immunotherapy platform with a checkpoint inhibitor; and, NantKwest received a discovery grant to investigate NeukoPlastTM in prostate cancer. In other news we saw earnings reported, which provides insight into cash position.

Combinatorial therapeutic approaches that bring together cell therapy, genetic modifications and immunotherapy will be a source of growth in the oncology market for the foreseeable future. CAR and TCR products are designed to elicit cytotoxic CD8+ T cell responses against cancer cells, while checkpoint inhibitors – or “immunological brakes” – are able to inhibit tumour blockade of T cell attack. Taken together, they may prove to exhibit strong synergies in tumour destruction. Immune Design (IMDZ) announced a Phase 2 collaboration to pair its viral-based “prime-boost” immunotherapy (CMB305) targeting NY-ESO-1 with Genentech’s anti-PD-L1 checkpoint inhibitor (atezolizumab). The Phase 2 study will investigate the therapeutics together in patients with relapsed or metastatic synovial sarcoma and myxoid/round-cell liposarcoma. CMB305 is a unique two-pronged approach that elicits expansion of tumour cell-specific CD8+ T cells, via a lentiviral vector that delivers NY-ESO-1 RNA to dendritic cells (LV305), while inducing CD4+ T helper cells, via delivery of recombinant NY-ESO-1 formulated with a proprietary small molecule (G305).

NantKwest (NK), which recently completed the largest biotech IPO in history, on a valuation-basis, was successful in securing $1 million* from the Prostate Cancer Foundation to develop NK cell therapies for prostate cancer. The company will leverage its taNK technology (NK cells engineered to express CARs) to target prostate-specific membrane antigen (PSMA) on prostate cancer cells. The funds will be used to investigate the PSMA-CAR NK cells in preclinical models of androgen sensitive and hormone therapy-resistant prostate cancer. A Phase 1 study will also be initiated.

Juno Therapeutics (JUNO) is sitting on a remarkable pro forma (future) cash position of $1.31 billion, after clearance for its deal with Celgene went through in August. This puts it well ahead of its closest competitor, Kite Pharma, which reported only 25 per cent this amount, or $367 million. bluebird bio (BLUE) is also heavily cashed up with $930 million on the balance sheet (a large piece of which came from the $477 million equity financing that was completed in June of this year). Other cash positions reported from the cell therapy industry included Aduro Biotech ($466 million), Lion Biotechnologies ($112 million), Ziopharm ($118 million), Opexa Therapeutics ($18 million), Fibrocell Sciences ($27 million) and Caladrius Biosciences ($39 million). So, most companies in the industry are looking healthy.

On the financing side of things, Intrexon (XON) successfully raised $230 million. The company is currently partnered with Ziopharm, which is developing an IL-12 immunotherapy, delivered locally to tumours using an adenovirus, and Merck Serono, which joined the CAR T race in March of this year. Ocata Therapeutics (OCAT) closed on a $10 million debt financing from the Silicon Valley Bank, the proceeds of which will be used to progress a Phase 2 study of the company’s pluripotent stem cell (PSC)-derived retinal pigment epithelial (RPE) cells for dry age-related macular degeneration (AMD) and a pivotal study in Stargardt’s macular degeneration (SMD). The company was also successful in raising other non-dilutive funds through a Small Business Innovation Research (SBIR) grant from the National Institutes of Health (NIH) to continue pre-clinical development of its proprietary hemangio-derived mesenchymal cell product, which it will pursue for the treatment of systemic lupus erythematosus (SLE) and lupus nephritis (LN).

*All figures reported are in U.S. dollars.

Erroneous information about Ocata Therapeutics was contained in the first version of this post, which has since been removed. October 29, 2015.

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Mark Curtis

Mark Curtis

Mark is a Business Development Analyst at the Centre for Commercialization of Regenerative Medicine (CCRM), where he collaborates with the team to help evaluate the commercial potential of regenerative medicine and cell therapy technologies. He began his career at Princess Margaret Hospital studying cellular reprogramming of human skin cells. Following this project, he left the laboratory and took on a role with Bloom Burton & Co., a healthcare-focused investment dealer. While there he supported the advisory team in carrying out scientific diligence on early-stage biotechnology companies. Prior to joining CCRM he was a consultant to Stem Cell Therapeutics, a Toronto-based biotechnology company focused on developing therapeutics targeting cancer stem cells. Mark received a Master’s degree from the University of New South Wales in Sydney, where he studied the directed differentiation of embryonic stem cells, and a Bachelor’s degree in Biology, from Queen’s University. Follow Mark on Twitter @markallencurtis
Mark Curtis

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