Signals Blog


Welcome to your Update from the Clinic for the month of April. Mesenchymal stem cells took a hit as Athersys’ MultiStem came up short, missing its primary endpoint in a Phase 2 study investigating the cells for ischemic stroke. Juno Therapeutics continues to turn heads with its Phase 1/2 data in blood cancers. NeoStem, a little further along in clinical development, enrolled its first patient in a Phase 3 study investigating an immunotherapy for the treatment of melanoma.

The significant, but unfortunately negative, news this month came from Athersys (ATHX), which for the last decade has been developing MultiStem, an allogeneic, off-the-shelf cell therapy product that is currently being deployed across three key disease areas: cardiovascular, inflammatory/autoimmune, and neurological. The company announced that the Phase 2 study investigating MultiStem in patients failed to meet its primary endpoint (Global Stroke Recovery Assessment), as patients in the treatment group showed no improvement at 90 days relative to placebo. The trial also failed two of its secondary endpoints related to stroke rehabilitation.

This is the second failure for MultiStem in the last year. The therapy also failed to meet its primary endpoint in a Phase 2 study in April of last year for ulcerative colitis. Management believes that patient subsets could be the answer, though data within these groups were not significant either. Without a second asset in development, investors are questioning the fate of the company. Japanese drug maker Chugai is also impacted, after making a $200 million deal with Athersys last year to gain rights to MultiStem in Asia.

Patients with “liquid” cancers, like leukemias, are exhibiting exceptional response rates to CAR-T therapeutics. Juno Therapeutics’ (JUNO) recent results are a testament to this. In a Phase 1 study investigating a CD19-directed CAR-T (JCAR017) in patients with relapsed/refractory acute lymphoblast leukemia (ALL), 91 percent of patients achieved complete remission! The technology was initially developed at Seattle Children’s Research Hospital.

In April of 2014, NeoStem entered into an agreement to acquire California Stem Cell, which gave it access to eltrapuldencel-t (formerly melapuldencel-t), an autologous cell-based immunotherapy under development for metastatic melanoma. NeoStem announced that the first patient in the Phase 3 INTUS study has now been enrolled.

Eltrapuldencel-t is a unique therapy amongst the dendritic cell (DC) immunotherapies. Instead of simply pulsing DCs with whole tumour cells to prime them, the protocol calls for a direct linkage between the DC and tumour cell, which is believed to optimize the presentation of the cancer antigens to T cells within the body. The therapy is being developed under a Special Protocol Assessment (SPA), which would allow NeoStem to gain approval for marketing without the need to complete further clinical studies.

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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