Our Research and Development Programs
Our research and development programs include the development of three SNA therapeutic candidates to address unmet medical needs in the treatment of mild to moderate psoriasis and solid tumors as well as early stage research activities in neurology, ophthalmology, respiratory and gastrointestinal applications, and the development of SNAs that target IL-4RA. These early stage research activities are described in more detail in the sections entitled “—Early development programs” and “—SNA targeting IL-4RA for atopic dermatitis.” Research and development expense for the years ended December 31, 2017 and 2016 was $14.1 million and $13.7 million, respectively.
The table below sets forth the stage of development of our current SNA therapeutic candidates:
TNF = Tumor Necrosis Factor; IL-17RA = Interleukin 17 Receptor Alpha; TLR9 = Toll-like Receptor 9
(1) AST-005 is the subject of the Purdue Collaboration described below in “—Purdue Collaboration.”
Regulatory documents are prepared and submitted to the appropriate health authority to enable clinical trials in any given jurisdiction. In the United States, this document is called an IND application, while in other jurisdictions, this document is often called an IMPD, which is submitted as part of a CTA. The content and scope of an IND and a CTA are similar.
AST-005—topically applied SNAs for psoriasis
AST-005 is a therapeutic candidate for the treatment of mild to moderate psoriasis, which is often defined as psoriasis that affects less than 10% body surface area and is generally not treated with systemic antibody therapy. AST-005 is an SNA containing TNF antisense oligonucleotides and is intended to be applied in a gel to psoriatic lesions. The clinical success of systemically delivered anti-TNF antibodies has validated TNF as a clinically relevant therapeutic target for psoriasis. We believe that the local inhibition of TNF with AST-005 may result in targeted therapeutic activity in patients with mild to moderate psoriasis, where, given their systemic risks, treatment with anti-TNF antibodies falls outside of the current American Academy of Dermatology guidelines.
In our Phase 1 clinical trial, AST-005 resulted in no drug associated adverse events, and AST-005 reduced the production of TNF mRNA in psoriatic skin after topical application. We believe these results demonstrate the potential therapeutic application of AST-005 in the tissue of interest, and that SNAs are potentially capable of regulating gene expression in human skin. On December 2, 2016, we entered into the Purdue Collaboration for further development of AST-005 in mild to moderate psoriasis and in other indications. Pursuant to the Purdue Collaboration, Purdue is conducting a Phase 1b clinical trial in psoriasis patients in Germany to evaluate the effect of higher concentrations of AST-005 gel on TNF mRNA and downstream mRNA expression. Patient dosing is complete and no serious adverse events have been reported. We expect to have the topline results of the clinical trial in early 2018.