Breaking The Mold Of Oncology Drug Development

Advancements in research have reached a 30-year pinnacle. Cancer treatment has been, at best, a very slow process, with new technology that is slowly being welcomed by the FDA. One company in particular, ImmunoCellular Therapeutics (IMUC), is working diligently to break the mold of oncology drug development. As a result, I am looking at how the company is progressing the field, along with the past and present trends in oncology.

Looking at all oncology products on the market and how each work, it is striking to note how each targets a single factor in the complex processes that control tumor growth and invasion. Cancer continues to evolve, yet there is a lack of any viable drugs on the market to effectively combat the disease. It is almost like one is attempting to stop traffic in a city by blocking a single avenue: It only slows down traffic but doesn't stop it completely. The clinical outcomes of the last 30 years of oncology research are very similar -- we know how to slow down progression of the disease but can't stop it from spreading and eventually killing the patient.

There are two main culprits as to why we have taken this path of attacking single antigens over the last few decades. The first is that a large number of these drugs were initially developed in academia, which is notorious for emphasizing publication of fundamental understanding of pathways, rather than trying to find a combination of drugs for a cure. The main reason being combination drugs may be harder to study and, more importantly, even harder to publish. The other problem is the regulatory one, whereby the FDA has emphasized studying the effect of each drug entity and doesn't have a clear regulatory pathway for combining 2-3 or more experimental drugs together.

Probably in every other industry, several changes to a product are made simultaneously in next generation products (for example TV or laptop or cell phone) while the drug industry tries to make small incremental changes which take, usually, 10-12 years to prove through a Phase III randomized trial that a certain drug works. The only exception to this case seems to be the vaccine industry where combining various targets (antigens) of a pathogenic agent are considered to be a superior design and have been more successful than single targets.

One company that is trying to break this mold of oncology drug development is Immunocellular Therapeutics, which is developing ways to stimulate the body's own immune system simultaneously against multiple targets, a page stolen from the vaccine world. Each tumor cell has hundreds of antigens that are expressed on the cell surface, however most are also shared with normal cells. A few antigens are different on these cancer cells, and while other companies are pursuing a strategy of targeting only one of these many antigens, such as Dendreon (DNDN), IMUC's approach is to target several antigens simultaneously. Going back to the problem of blocking the traffic, IMUC's approach is to close as many avenues as possible so traffic really comes to a halt, which makes it easier to treat the cancerous cells.

The early stage clinical data from Phase I seems to provide validation to this approach treat the cancerous cells. The results show 40% of patients treated in a small Phase I study had no progression of disease over 3 years in the most aggressive type of brain tumor, glioblastoma. Four years ago, late Senator Kennedy was diagnosed with glioblastoma, declined quickly, and passed away after 15 months, which is normal for those diagnosed with this vicious disease. The 3 year disease free survival for this cancer is only 5%, so to have a 40% survival rate is quite a high achievement even from a small trial.

Over the last few months, IMUC has announced a number of collaborations with leading medical centers such as Johns Hopkins to buy rights to additional cancer antigens and to combine those in its cocktail to create vaccines targeting multiple sites on cancer cells. With Phase II trial results coming up next year along with additional indications, such as ovarian cancer, being initiated, a positive trial result from IMUC would be a big game changer in how we develop the next generation of drugs for cancer.

Since the beginning of this year, IMUC has seen a significant price appreciation on the back of accelerated enrollment and aggressive technology acquisitions. If Amgen's (AMGN) acquisition of Biovax and Micromet (MITI) are any indications of large pharma's willingness to acquire these products, for over a billion dollars based on Phase II data, then there is clearly a very significant upside if the trial results are positive for this seemingly transcendent technology.

The last thing that could add fuel to this story is the pending legislation, Transforming the Regulatory Environment to Accelerate Access to Treatment Act (TREAT), proposed by Senator Hagan (D-NC) that is aimed at giving an accelerated approval for products like ICT-107, the product developed by IMUC, based on early stage trials where they have demonstrated dramatic response. This could bring ICT-107 to patients quickly while the company continues to accumulate additional data, and make a significant impact to the treatment of glioblastoma, a disease where only two new drugs have been approved over the last 30 years. As the treatment of cancer continues to evolve, and the targeting of multiple antigens becomes common, we should finally see the advancements in cancer that we are so desperate to witness, and IMUC is leading the charge.

Disclosure: I am long IMUC.

Additional disclosure: The material in this article is for informational purposes only and should not be used to make any investment decisions.