Two weeks ago, Micromet (MITI) hosted its annual R&D day, where it discussed plans for 2009 and beyond. The meeting provided plenty of information regarding the company’s technology and drug candidates, but more importantly, it served as an appetizer for next month’s EHA meeting. As a reminder, Micromet is expected to present data from 2 trials evaluating its lead agent, blinatumumab (MT103), in two forms of blood cancer: Non-Hodgkin Lymphoma (NHL) and Acute Lymphoblastic Leukemia (ALL).
During the R&D day, the company (intentionally and unintentionally) shared some previously undisclosed results from the trials. The new information, which includes impressive efficacy signals from both studies, further solidifies blinatumumab’s position as one of the most promising investigational agents in oncology. Based on its spectacular performance, blinatumumab has a high chance of getting approved as soon as 2012.
Fast route to market
Although the NHL study represents a much larger commercial opportunity, the ALL trial will garner most of the attention. As I explained in my previous article, the ALL study evaluates blinatumumab’s effect on patients who have undergone chemotherapy but still have disease remnants in their bone marrow (minimal residual disease or MRD). These patients usually have very poor prognosis since they are destined to relapse within less than a year. The only available treatment option for some of these patients is stem cell transplant, a highly aggressive and toxic procedure which is associated with a 20-30% mortality rate. Micromet decided to enroll only patients who cannot tolerate or are unwilling to undergo stem cell transplant, as these patients have no approved treatment options.
The high medical need combined with the low prevalence of ALL make this clinical program a fast route to market, with a possible approval already in 2012. More importantly, the study could serve as the ultimate proof of concept for blinatumumab’s ability to deal with one of the central challenges in cancer, post-treatment residual disease that eventually leads to disease relapse.
Micromet’s strategy is similar to that of other biotech companies, such as Exelixis (EXEL) and Arqule (ARQL). All three have a promising compound with a very large potential market, but they decided to pursue approval for niche indications that represent the fastest and safest route to approval. Micromet’s blinatumumab targets CD19, a protein highly expressed on the majority of blood cancers, but decided to initially go after a distinct subgroup of ALL.
