Exicure has a broad pipeline of SNA-based therapeutics to treat a range of diseases with great unmet medical need. The unique properties of the SNA constructs allow the use of oligonucleotide-based therapies to treat diseases in a range of tissue types.

Exicure Pipeline Chart

(1) AST-005 is the subject of the Purdue Collaboration, which is described in more detail in our registration statement filed with the SEC on November 28, 2017.


Psoriasis is an autoimmune disease known to cause red, scaly patches to appear on the skin, particularly around the elbows, knees or scalp, although it can occur anywhere on the body. Unlike eczema, a psoriasis plaque is well defined, and therefore it is easy to see how each plaque is outlined.

There are a variety of potential treatments available for psoriasis, but given that it is an autoimmune disorder, some of these treatments only manage symptoms, while others are more systemic in nature, and attempt to address the immune response. Available treatments include biologics, which are generally administered via injection or intravenously, orally administered systemic drugs, as well as a variety of non-drug solutions, including phototherapy, topical treatments and alternative medicine solutions.

The biologics market alone represents a $4B market worldwide, and yet the most commonly prescribed biologic has been shown to increase the risk of infection. In addition, many patients develop antibodies against the treatment, which ultimately renders it ineffective.1

Exicure has taken advantage of the unique ability of SNA constructs to penetrate skin and deliver oligonucleotides to the epidermal layer to develop a compound to address the problem of psoriasis that combines the benefits of biologics with the ease of application of topical steroids. Exicure is developing a topical treatment designed to target TNF mRNA, which has been validated as a target to alleviate and improve the symptoms of psoriasis.

This unique approach of an SNA containing anti-TNFα antisense oligonucleotides developed as a topically applied treatment has the potential to significantly reduce the presence of TNFα protein, and therefore reduce symptoms. Exicure has demonstrated that application of SNA constructs to human psoriatic skin reduces the levels of TNFα mRNA. Based on these results, Exicure is currently planning clinical studies to determine the efficacy of the SNA technology in patients with psoriasis.


Cancer is characterized by abnormal cell growth with potential for spreading to other sites of the body. Normal cells may become cancerous due to mutation in or overexpression of certain proteins. The immune system can typically recognize these abnormalities and clear the cancer through a process known as immune surveillance. However, a hallmark of many cancers is the ability to suppress immune surveillance mechanisms. Therefore, a key unmet medical need is to bolster the body's natural defenses across a broader range of cancers to restore the cancer-clearing immune surveillance capabilities of the body.

In the last 10 years, there have been numerous advances in the approach to treating cancer using immunotherapy, which reactivates the immune system to fight cancer. This approach can potentially offer initial relief from cancer, longer-term protection against recurrences and present fewer side effects than chemotherapeutics.

Exicure is utilizing its SNA technology to mobilize the body's natural defense against cancer. Exicure's lead immunotherapy compound, AST-008 (initially being investigated in selected solid and hematological tumors) is a toll-like receptor 9 agonist designed to utilize the SNA's beneficial properties to drive a potent anti-cancer immune response. The treatment could accompany other systemic therapies while driving a stronger immune response than other technologies.