Cell Therapy Deal Review: August

Author: Mark Curtis, 09/30/15


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
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14 Responses

  1. Klaatu Gort says:

    Your comment regarding Ocata halting a Phase I/II RPE trial due to cells harboring mutations is incorrect. The halted trial was being conducted by RIKKEN, not Ocata. Publishing such obviously false information has severely damaged your credibility. Please post a correction immediately. Thank you.


  2. Klaatu Gort says:

    RIKEN halted their Phase I/II RPE trial due to mutation issues, not Ocata. To date, Ocata’s RPE trials to treat Stargardt’s and dry-AMD have shown stellar results. Please correct this information at your earliest convenience. Thank you.

  3. Ocata says:

    In fact, Ocata has never, ever, stopped a trail and has a 100% safety record. You must have mixed companies up. Please affect changes.

    “In fact, Ocata had to halt a Phase 1/2 study investigating patient-specific iPSC-derived RPE cells in dry AMD after researchers discovered that cells bound for the second patient in the study housed six mutations, including one in oncogene.”

    Again, Ocata has NEVER stopped a trial.

  4. Gizmo says:


    I wonder if you have confused OCATA with RIKEN? I follow OCATA carefully, and am not aware that the company halted any trials due to mutations. In fact, the safety profile of their RPE trials has been exceptionally clean. Perhaps you have access to information that I don’t, but would appreciate some follow-up on this and a correction if necessary.

    Thank You

  5. David says:

    Why are you reporting these egregious errors about Ocata (OCAT? They have 4 years of safety data with not a single instance of any problems, let alone Teratomas. Where are you sourcing this information? Maybe you’re confusing it with the RIKEN or STAP failures? Ocata has already started Phase 2 studies in the US for AMD and will shortly have Pivotal trials for SMD for both Europe and the US.

    Maybe a little due diligence would be in order.

  6. John Redaelli says:

    Hi Mark,

    This is not a fact – “In fact, Ocata had to halt a Phase 1/2 study investigating patient-specific iPSC-derived RPE cells in dry AMD” – You are confusing, Ocata Therapeutics with the Riken study/trials in Japan. See your own reference/link – “concerns remain around their safety” – http://www.signalsblog.ca/the-waiting-game-first-human-ipsc-clinical-trial-on-hold/

    Please, correct/update, when you can…

    Many Thanks!

    John Redaelli
    twenty2John at Twitter – https://twitter.com/twenty2John

  7. investor says:

    “Ocata had to halt a Phase 1/2 study investigating patient-specific iPSC-derived RPE cells in dry AMD”

    The above statement is completely untrue. Ocata’s trials are NOT on hold, and they are using hESC not iPSC.

  8. Joseph Gross says:

    I believe the Ocata AMD trial uses ma-09 hesc cells for both Phase 1 and Phase 2.The IPSC cells may be used for regenerating photoreceptors in the near future.

  9. Terry Jackson says:

    Mr Spencer — You mixed up your researchers, it was Riken whose trial was stopped because of tetromas forming – not Ocata. Check your link.

    Please correct this – it could be very harmful to Ocata. Ocata has a record of four years of successful trials without any tetromas.

  10. Mark Curtis says:

    Dear All,

    My sincerest apologies. I recognize that I have made a mistake and have confused the RIKEN trial with Ocata – the first time that such a thing has happened while writing for Signals. I have contacted the blog administrator to remove the inaccurate information regarding Ocata’s study and will endeavour to ensure this does not happen again.


    • dyo says:

      Thanks Mark

  11. Stacey Johnson says:

    Editor: Incorrect information about Ocata was included in this post and has since been removed. Apologies to Ocata for the error. Thank you to the commenters who alerted us to this mistake.

  12. Gizmo says:


    Thanks very much for the correction. The OCATA community is particularly sensitive to any sort of misinformation because at the moment there are predatory short sellers doing all sorts of unscrupulous things to attack the company and bring the share price down. These same manipulators are assaulting other emerging biotech firms as well. Hopefully the SEC and other authorities are going to get involved and get serious about enforcement of trading rules.

    Much appreciated…!

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