Intellectual Property Of A Biotech: An Analysis

For biotech investors, there is little as frustrating as digging through mounds of medical journals and browsing websites in an attempt to interpret clinical trial results, trial timelines and potential drug market values. Presented clinical data can be confusing, complicated, biased and even contradictory at times. Biotech companies often purposely delay both positive and negative data for their own purposes and may only report a portion of the data from their trials. Potential marketing value for a drug can also be elusive with differing patient targets for specific indications with phrases often seen such as "for breast cancer patients with overexpression of HER2 +2 or +3" or other such jargon that not only makes researching the marketing potential difficult, but it also makes it difficult to perform "apples to apples" comparisons among similar drugs.

The deeper a biotech investor digs for data and marketing potential the more questions than answers can often be found. Some of the most difficult research an investor can undertake is in a biotech's patent portfolio protecting its valuable intellectual property. This property not only increases the market value of the company owning it, but it also protects the drugs' future marketability as it can reduce or prevent competitive products from draining sales from the drug's future and prevent "copycat" drugs from finding loopholes and directly competing with the company. The money and years invested in any drug truly drains a biotech's finances. In order to survive, they must start marketing or licensing their final product at some point in order to survive. When/if "The FDA Approves Drug XYZ" for the biotech is finally announced, the company must be confident in its product's return on investment or the millions of dollars and years of research could be in vain for the company. An analysis of the varied patents a biotech company possesses is necessary for those wishing to predict the company's expected profitability in the event its product is ultimately cleared for marketing.

ImmunoCellular Therapeutics (IMUC) is a development phase biotech with a phase II trial for newly diagnosed glioblastoma multiforme (GBM) expected to complete enrollment sometime in Q2 2012. Interim data analysis is expected in Q4 2012 or Q1 2013, and the company as well as investors are hopeful with the trial garnering much attention as evident from the common stock's price run up from January 10th's $1.05 to the current price hovering around $3.50 per share. The company's press releases have been largely focused on the phase I trial results for ICT-107 for the above-mentioned GBM and the follow up phase II trial. The data is indeed impressive, even for a small patient set. However, investors should also be taking note of the additional press releases as pertaining to patent protections applied for as well as those awarded. The July 10th announcement of the Japanese patent allowance for IMUC's multi-antigen approach to fighting brain cancer is a sure sign that the company is taking its intellectual property protection very seriously. It is this patent protection that will protect the value of the company's pipeline and will also attract Big Pharma suitors for M&A and licensing purposes. To aid current and potential investors in their intellectual property research for ImmunoCellular Therapeutics, an overview of their patent portfolio as granted and applied for is presented below and pertains directly to their current clinical and pre-clinical work. As you read through these patents, remember many are here to protect the company's worldwide license in some of these cases, greatly multiplying the potential financial benefit that will could evident for licensing and marketing purposes.

Granted Patents

US 8097256

US 8,097,256 is titled "Cancer Vaccines and Vaccination Methods" with patent allowance announced on October 10th, 2011. This is a key patent for ImmunoCellular as it is critical in protecting the company's lead product, ICT-107. The invention is for a cancer vaccine and is designed to claim the epitopes (part of the antigens the T-cells, B-cells or antibodies recognize) for any combination of 4, 5 or 6 of the following antigens: tyrosinase-related protein (TRP)-2, Melanoma-associated Antigen-1 (MAGE-1), HER-2, IL-13 receptor α2, gp100, and AIM-2. This patent is specific to claim the invention as a vaccine for gliomas, glioblastoma multiforme (GBM) and astrocytomas. To cover claims for off-the-shelf and patient specific vaccines, the patent coverage is for allogeneic and autologous (the latter like Dendreon's Provenge or Immunotherapy's ICT-107) prepared vaccines. The allogeneic claim will be helpful for future products as vaccines prepared via this approach would be cheaper and much quicker to prepare and would be more readily available for patients needing treatment immediately upon diagnosis or immediately after a front-line treatment. The dendritic cells created in the process are key to immunotherapy (here vaccines) as they are antigen presenters and are what the patient's immunity system is seeking out to have an immune response against. The invention here is not only for the typical epitope but also superagonist epitopes that the dendritic cells present to the immunity system. Superagonist epitopes are modified epitopes that have amino acid substitutions at the function group sites making them generate a more potent immune response than the naturally occurring epitopes. It appears that the keys to this patent are two-fold. First, the vaccine will stimulate an immune response against a wide range of brain cancers with any of the 6 antigens over expressed. A patient with any one of these or multiples of these is a viable candidate. Secondly, the tumors may evolve to present different types of antigens than the one determined in initial testing. A drug targeting only one type of antigen would be ineffective if the tumor evolved to have a different expression. In this case, 6 different antigens are targeted and a mutated version within these 6 antigens would also be targeted.

US 7939090

US 7,939,090 is titled "System and Method for the Treatment of Cancer, Including Cancers of the Central Nervous System". The company announced its allowance on June 21, 2011 noting that the patent covers the combination of a dendritic cell based vaccine combined concurrently with or before chemotherapy is administered at the recurrence of the disease (GBM). Like most cancer vaccines, it is typically not administered as a first line treatment to attack cancer alone but rather is used to prevent recurrence after a front line treatment of resection, chemotherapy or radiotherapy. The patent specifically claims the combination of the vaccine with at least one or a combination of the following chemotherapy agents: temozolomide, procarbazine, vincristine, BCNU, CCNU, thalidomide, irinotecan, isotretinoin, imatinib and/or etoposide. The invention summary states "The dual therapeutic approach of the instant invention may further be implemented to beneficially influence the chemosensitivity of a mammal with cancer, including cancers of the CNS, by vaccinating the mammal with DC prior to and/or concurrently with administration of chemotherapy." This response is well documented with a host of accompanying drawings in the patent application. However, not noted in this patent is the fact that chemotherapy often weakens the immunity system and also somewhat mutes the immune response. Therefore, it appears the best time to administer the vaccine is indeed before or with the chemotherapy agents when the immunity system is still at its strongest. An additional statement in the patent award is also key as it is a very broad and general coverage for the patent stating, "The cancer vaccine used in various embodiments of the instant invention may be selected from any dendritic cell (DC)- based cancer vaccine, and can be administered by routine methods." Essentially, this patent appears to protect any DC-based drug the company develops or licenses in combination with the chemotherapy agents mentioned in the award. This patent award is key and a great addition to ImmunoCellular's intellectual property.

US 8129184

US 8,129,184 is titled "Cancer Stem Cell Antigen Vaccines and Methods". The company announced its allowance on February 1st of this year. It gives protection to the company's pipeline that includes cancer stem cell targeting as part of the treatment protocol. The patent covers various methods of combining dendritic cells with cancer stem cell (NYSE:CSC) antigens, which teaches them to present the cancer stem cell antigens as targets for an immune response. Cancer stem cells are being implicated in the recurrent nature of many cancers. After cancer cells are killed via whatever first line method was used, CSC's often remain as they are more like "normal" human cells and do not reproduce very rapidly and are less susceptible to the agents that typically kill the faster growing and faster reproducing cancer cells. CSC's are capable of mutating and creating another generation of cancer cells often more resistant to the chemotherapy agents or irradiation that was used to destroy the first generation cancer cells. If CSC's can be targeted and destroyed, the recurrent nature of many cancers can be delayed or even prevented thereby helping to create a true cure for cancers. Inherent in the technology is a likely synergistic use of a CSC vaccine for many cancers, which are difficult to destroy because of their propensity to mutate. A potent initial first line treatment may prove to be enough to destroy a cancer in theory if the adaptive nature of the cancer is nullified via an adjuvant CSC targeted immune response. The patent covers not only concepts and administration of the vaccine, but also the methods of preparing the vaccine not only via a patient-specific autologous but also an off the shelf allogeneic approach. Antigens utilized via either approach for directly targeting the CSC's are identified in the patent as any combination of the following: CD133, CD90, CD44, CXCR4, Nestin, Musashi-1 (Msi1), maternal embryonic leucine zipper kinase (MELK), GLI1, PTCH1, Bmi-1, phosphoserine phosphatase (PSP), Snail, OCT4, BCRP1, MGMT, Bcl-2, FLIP, BCL-XL, XIAP, cIAP1, cIAP2, NAIP, or surviving. A mention was even made in the patent of a synthetic peptide as being presented in some embodiments.

Patents Applied For

US 2010/0310643 A1

US 2010/0310643 A1 is titled "CD 133 Epitopes". The application was submitted May 7, 2010 and June 03, 2010 with the former as WO 2010/028066 A3R4 as a provisional. The application claims use of the antigen CD133 and many of its amino acid variants to be utilized as a means of targeting cancer cells via an immunotherapy approach. Specifically, it claims "A method for treating a cancer in a patient, the method comprising administering to the patient a composition comprising antigen-presenting cells, wherein the antigen presenting cells present on their surface a peptide epitope comprising the amino sequence ILSAFSVYV (SEQ ID NO:1) with four or fewer amino acid substitutions". As mentioned in patent 8,129,184 above, CD133 is an antigen targeted for CSC immune response. The company recently presented data at the AACR annual meeting indicating in vitro human cell studies and in vivo studies in mice indicating a safe profile and immunogenicity of two peptides as a potential vaccine to target CD133 CSCs. They indicated plans in using this technology as the basis for ICT-121 for recurrent glioblastoma for its initial indication with clinicals for additional solid tumors to follow. The latter comes as no surprise as CD133 cells are highly enriched in CSC's in colon cancer, hepatocellular carcinoma, prostate cancer, myeloma and melanoma. Like ImmunoCellular's approved patents, this one would cover all aspects of isolation, preparation and administration of the vaccine and its active and modified dendritic (antigen presenting) cells. As ICT-121 trials get underway, this patent's approval will become key if clinicals are favorable not only for its current recurrent GBM indication, but also other indications' possibilities.

US2002/0182219 A1

US2002/0182219 A1 is titled "Cancer Immunotherapy" and was submitted on March 22 and published December of 2002. The patent has yet to be approved, and there have been no recent updates on the application. Although an older application, it is a novel attempt at what could be the future of the war on cancer, a cancer preventative vaccine. Most cancer vaccines in clinicals now are attempts at first line treatment or as an adjuvant for prevention of cancer recurrence. This application is for a vaccine that stimulates an immune response against cells expressing IL-13Rα2 in patients having or at risk for developing a disease having cells expressing IL-13Rα2. Not going into a lengthy discussion on this patent because of its age, the application of the patent could be substantial if/when it is approved. The very nature of using IL-13Rα2 is intriguing as it has potential advantages over other discussed antigens. It is a cell-surface receptor which gives it much exposure in the immune system, especially important in a difficult area such as gliomas in which the blood/brain barrier is difficult to overcome. It is also expressed on a wide range of high-grade gliomas and the testes (which are immune privileged which reduces the chance of a hopeful immune response turning into an autoimmune nightmare by attacking unintended targets/organs). IL-13Rα2 is a great target for anti-cancer immunotherapy because of its size (380 amino acids in IL-13Rα2 and 343 amino acids in the extracellular domain), which gives an immune response multiple epitopes to recognize and target.

These three granted and two requested patents are key to ImmunoCellular's marketability as a suitor, to their future profitability via technology licensing and/or to their future profitability as a pharmaceutical with their own products to push through trials, gain marketing approval for and then produce/market on their own. As clinicals progress and the true value of the company's product line is realized, these patents will become more valuable. The wording in these patents is important as loop holes are closed and the applicability of the technology for several indications will only increase their value and ImmunoCellular Therapeutics' price tag for Big Pharma and individual investors as 2012 progresses.

Disclosure: I have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.