Another new deal values ACCP's drug delivery technology - Analyst Blog

Grant Zeng, CFA

Another new deal values ACCP's drug delivery technology

Early this morning (April 13, 2011, Access Pharmaceuticals Inc. (ACCP) announced that it has entered into an agreement with a major global pharmaceutical company to exploit its CobaCyte and CobOral technology for the targeted delivery of RNAi therapeutics.  

Access will provide the pharmaceutical company with CobOral and CobaCyte siRNA formulations for evaluation of gene knockdown following oral and intravenous administration.  Though the terms of the agreement have not been disclosed, Access indicated that any successful formulation developed will be jointly owned by the Parties and subject to a subsequent full licensing agreement.  

As a reminder, Access is an emerging biopharmaceutical company focused on developing a range of pharmaceutical products for the treatment of cancer and cancer supportive care primarily based upon the company's three key drug delivery technologies: Synthetic Polymer Targeted Drug Delivery System; Cobalamin Mediated Oral Drug Delivery System (CobOral) and Cobalamin Mediated Targeted Drug Delivery System (CobaCyte).

RNAi is typically initiated by the introduction of small fragments of RNA, typically siRNA or miRNA, into cells at disease sites. Due to their large size and high negative charge, these RNA fragments are not able to cross cell membranes. Therefore, to develop effective RNAi therapeutics, a delivery system must be developed that can transport the siRNA into cells, and release undamaged siRNA into target cell cytoplasm.  Access' CobOral and CobaCyte delivery technologies, which are based on vitamin B12, are particularly well-suited for this purpose.  Most human cells have a requirement for vitamin B12 which is served by cell surface receptors which facilitate absorption of this vitamin.  In many diseases, the demand for vitamin B12 is increased, with a corresponding upregulation of the receptor. Using the 'Trojan Horse' principle, the CobaCyte nanoparticle technology can utilize the vitamin B12 uptake mechanism to transport siRNA into cells whereupon native siRNA can be released for incorporation in messenger RNA (mRNA) to initiate the beneficial therapeutic effect. In this way, CobaCyte offers the potential for targeted delivery of siRNA following intravenous administration. The fact that Access' vitamin B12 technology also facilitates oral drug delivery (the CobOral technology) indicates that it may also be possible for this technology to provide effective siRNA treatments by oral drug delivery.

The Company has made great progress in its CobOral and CobaCyte siRNA delivery programs over the past year, demonstrating the efficiency and safe delivery needed for a viable RNAi therapeutic. In July and October, 2010, Access entered into pre-licensing feasibility agreement with two leading biotechnology companies to develop an oral formulation of its currently-marketed, proprietary injectable drugs (diabetes) and prostate cancer compound respectively. Access will utilize its proprietary CobOral Drug Delivery Technology to develop oral formulations of the drugs for pre-clinical testing. Although the name and the terms of the agreements have not been disclosed due to competitive reasons, management indicated that any successful formulation developed will be subject to a subsequent full-licensing agreement.

Big pharma companies have expressed strong interest in both Cobalamin-based platforms for various indications. The signing of this agreement serves as further validation of ACCP's CobaCyte technology which has a unique ability to deliver inactivated siRNA particles to disease target sites. 

We are impressed with the progress Access has made so far about its CobOral/CobaCyte drug delivery technology. We believe the agreement further validates Access's drug delivery technology. It will also strengthen the company's balance sheet through upfront and milestone payments if any full license agreement is signed. This is highly likely in our view.

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