Signals Blog

Welcome to your update from the clinic for the month of May. Long-standing industry pioneer StemCells, Inc. decided to wind down its operations after a failed Phase 2 study in spinal cord injury. Aduro Biotech also saw a failure with its GVAX product in pancreatic cancer. Kite continues to deliver on its regulatory strategy, announcing a further five orphan indications for products in development.

There were some disappointments in May on the clinical front. But perhaps the most unfortunate news is that we’re seeing an industry pioneer, StemCells, Inc. (STEM), close its doors after almost three decades of research into adult stem cells. The decision came shortly after the company announced a significant failure in its Phase 2 study of Hu-CNS-SC in patients with spinal cord injury. While the treatment group in the study showed a trend in improving motor strength and function, the company determined that it was unlikely the primary endpoint of the study would be achieved. After a review of its cash position and strategic options, management determined its only recourse would be to wind down operations in an attempt to monetize the Hu-CNS-SC intellectual property.

The prospects for GVAX technology (irradiated tumour cells engineered to express GM-CSF) in the clinic are looking bleak after Aduro Biotech (ADRO) reported negative results from a phase 2b study in heavily pre-treated metastatic pancreatic cancer. This is a notoriously challenging indication to pursue in the clinic so risk of failure for any therapy is high, though GVAX pancreas in combination with Aduro’s CRS-207 (live, attenuated Listeria) delivered lower efficacy than either of standard-of-care (chemotherapy) or CRS-207 administered alone in single arms. Aduro acquired GVAX from BioSante in early 2013 for only $1M upfront – a bargain price reflecting the uncertainty around the technology’s prospects in the clinic. Fortunately for Aduro the company has two additional platforms to commercialize: CRS-207 and a class of small molecule compounds secreted by Listeria that stimulate immune response via the STING pathway.

Kite Pharma (KITE) now has a total of six orphan designations in the U.S. for its most advanced CAR product, KTE-C19, after receiving an additional five this past month, for primary mediastinal B cell lymphoma (PMBCL), mantle cell lymphoma (MCL), follicular lymphoma (FL), acute lymphoblastic leukemia (ALL), and chronic lymphocytic leukemia (CLL). The company was previously granted an orphan designation for KTE-C19 in the treatment of diffuse large B cell lymphoma (DLBCL). In addition, Kite has designations for five indications in the EU (all but DLBCL). Orphan designations are an important part of regulatory strategy as they provide a manufacturer seven years of exclusivity following registration, wherein no other company may market the same biologic for the same indications.

Although StemCells Inc. will close up shop, both Asterias Biotherapeutics and Neuralstem continue to develop therapies in the clinic for spinal cord injury. Asterias acquired Geron’s IP and is developing PSC-derived oligodendrocyte progenitors, whereas Neuralstem is developing human spinal cord progenitors. There is hope yet.

Disclaimer: “Update from the Clinic” is a blog post generated by news flow from public regenerative medicine (RM) companies around the globe. As CCRM has public RM companies in its industry consortium, and the number of such companies is relatively limited on a global scale, Mark Curtis will sometimes include CCRM consortium members in his review. This blog post is provided for general information only and nothing contained in the material constitutes a recommendation for the purchase or sale of any security. The author is not a shareholder of any public RM company. To see a list of CCRM’s industry consortium members, please visit

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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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