Welcome to your update from the clinic for the month of February! OncoMed is the leader in news this month with various Phase 1b trials for its cancer stem cell-targeting fusion protein. Immunocellular Therapeutics and StemCells Inc. file orphan drug designations with the European Medicines Agency and FDA for their respective cell therapies. Read on to find out more.
OncoMed (OMED), a specialized therapeutics company developing biologics and monoclonal antibodies with anti-cancer stem cell activity, announced that it has commenced its third Phase 1b for OMP-54F28 in patients with ovarian cancer. OMP-54F28 is a fusion protein consisting of a portion of the Frizzled 8 receptor, which is a critical component of the Wnt pathway and a domain of the human IgG1 antibody. This fusion protein is able to inhibit the Wnt signaling pathway by acting as a decoy receptor capable of binding Wnt ligands.
The treatment is a combination approach together with paclitaxel or carboplatin, two chemotherapeutics agents. The company also announced initiation of a Phase 1b of OMP-54F28, along with the antibody Sorafenib (Nexavar®, co-marketed by Bayer and Onyx Pharmaceuticals), in patients with hepatocellular cancer, the most common type of liver cancer.
Across the pond in Europe, Immunocellular Therapeutics (IMUC) received an Orphan Drug Designation for its autologous dendritic cell therapy ICT-107 in patients with newly diagnosed glioblastoma multiforme, the most common and aggressive malignant brain tumour in humans. Clinicians administer the dendritic cells intradermally after they are mixed with a cocktail of synthetic cancer proteins. The cells then elicit an immune response targeted to cancer cells. IMUC is also developing ICT-121 for recurrent glioblastoma (Phase 1) and ICT-140 for ovarian cancer (preclinical).
Like an Orphan Drug Designation at the FDA, the designation in Europe comes with a number of incentives to therapeutics developers, including 10-years of market exclusivity post-approval, clinical and scientific guidance throughout development, and various fee and tax reductions.
Isreal-based Pluristem Therapeutics (PSTI) has applied for an Orphan Drug Designation with the FDA for its off-the-shelf placenta-derived (PLX-PAD) cells for treating patients with preeclampsia, a condition of pregnant women. The cells, similar in nature to mesenchymal stem cells, release a number of cytokines (molecular messengers) that temper inflammatory response in the body. Preeclampsia is common – it occurs in 6-8% of pregnancies globally – and is characterized by high blood pressure and excessive concentrations of protein in the urine. Without treatment the disease progresses to eclampsia, which may lead to seizures during pregnancy. It is estimated the disease costs the health-care system $3 billion annually.
Finally, StemCells Inc. (STEM) announced that it has completed enrollment in the first of two patient cohorts in a Phase 1/2 study to test its purified human neural stem cells (Hu-CNS-SC) in patients with age-related macular degeneration (AMD). The cohort has eight patients; half will receive 200,000 cells, while the other half will receive 1,000,000 cells. The cells are administered to a small space behind the retina in the eye that is most affected by the disease.
The success of OncoMed in garnering significant partnerships with pharma companies is no surprise given the amount of chatter on cancer stem cell-based therapeutics of late. So far, the company has forged relationships with Bayer, GSK, and Celgene. At the Phase 1b stage, with five candidates in clinical development, OMED already has a staggering $1 billion enterprise value (market capitalization less cash). Its competition is receiving similarly healthy valuations.
VeraStem and Stemline Therapeutics are both at the Phase 1/2 stage with two candidates in clinical development and have enterprise values of $350 and $220 million, respectively. Roche was one of the first of Big Pharma to back the cancer stem cell theory. During 2008 it purchased Arius Research, a company that had generated a monoclonal antibody targeting CD44 on cancer stem cells. Despite Arius being at the preclinical stage, Roche paid an incredible C$191 million in cash to acquire its platform.
It is clear that Big Pharma has begun placing its bets on cancer stem cells. As OncoMed, VeraStem, and Stemline Therapeutics all approach their efficacy trials, now is the time to be watching.
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 http://ccrm.ca/industry-consortium