Exicure, Inc. Reports Full Year 2018 Financial Results and Corporate Progress

Source:  Exicure, Inc. 3/8/19

Completed Phase 1 clinical trials of two drugs and began public trading

Exicure, Inc. (OTCQB: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) constructs, today reported full year financial results for the year ended December 31, 2018 and provided an update on corporate progress.

“We’re very proud of our achievements on each of our strategic priorities for 2018,” said Dr. David Giljohann, Chief Executive Officer of Exicure. “We drove two programs through Phase 1 clinical trials while laying the foundation for expanding our pipeline in neurology, rare dermatology, ophthalmology and gastroenterology. We also strengthened the management of our company and our access to capital. In the coming year, we expect to execute and advance our ongoing clinical programs, while expanding into new therapeutic areas,” Giljohann concluded.

Corporate Progress
Key achievements for Exicure during 2018 include:

• Completed the Phase 1 clinical trial of AST-008, a TLR9 agonist for immuno-oncology applications showing activation of the immune system and robust cellular response
• Completed a successful Phase 1 clinical trial of XCUR17 showing clinical effect in patients with psoriasis
• Demonstrated, in preclinical animal models, superior bio-distribution and prolonged survival when administering nusinersen in SNA format versus nusinersen in linear format
• Strengthened management team with addition of Dr. Matthias Schroff as Chief Operating Officer
• Commenced public trading on the OTCQB market
• Completed $22.0 million financing in August of 2018 at $4.50/share

Pipeline Updates
 
AST-008: AST-008 is an SNA consisting of toll-like receptor 9, or TLR9, agonists designed for immuno-oncology applications. The company began subject dosing of AST-008 in a Phase 1 clinical trial during the fourth quarter 2017 and the trial completed in September of 2018. AST-008 was shown to be safe and tolerable in all subjects, with no serious adverse events and no dose limiting toxicity. During the fourth quarter of 2018, the FDA opened the IND for AST-008 and informed the Company that our proposed Phase 1b/2 trial may proceed. During early 2019, we opened four clinical sites and began dosing and recruiting patients in that trial.

XCUR17: XCUR17 is an antisense SNA that targets the mRNA encoding IL-17RA, a protein that is considered essential in the initiation and maintenance of psoriasis. In the fourth quarter of 2018, we reported results from the Phase 1 trial of XCUR17. Of the twenty-one treated patients, eleven treated with the highest strength XCUR17 gel were observed to have a reduction in redness and improvement in healing as determined by blinded physician assessments. Further, the highest strength XCUR17 gel showed a statistically significant improvement in psoriasis symptoms versus the vehicle gel. By comparison, seventeen of the twenty-one patients treated with the positive comparator showed a clinical response, while four patients treated with the placebo vehicle had a clinical response.

2018 Financial Results and Financial Guidance
 
Cash Position: As of December 31, 2018, Exicure had cash and cash equivalents of $26.3 million compared to $25.8 million as of December 31, 2017. In 2018, Exicure raised approximately $22.0 million in gross proceeds from the private placement of common stock.

Research and Development Expense: Research and development expense was $14.1 million for the year ended December 31, 2018 compared to $13.1 million for the year ended December 31, 2017. The increase in research and development expense of $1.0 million was primarily due to higher employee-related expense of $1.2 million and higher platform and discovery-related expense of $0.6 million, partially offset by a net decrease of $0.9 million in costs related to our clinical development programs.

General and Administrative Expense: General and administrative expense was $7.8 million for the year ended December 31, 2018 and $7.0 million for the year ended December 31, 2017, an increase of $0.8 million. This increase was due to higher costs of $0.9 million associated with being a public company, including higher expense for investor and public relations and director and officer insurance, as well as higher compensation and related expense of $0.5 million associated with salary increases and the addition of an executive during 2018, partially offset by lower legal and accounting costs of $0.7 million resulting from a change in mix of transaction support in 2018 as compared to 2017.

Net Loss: Net loss was $22.4 million for the year ended December 31, 2018, compared to net loss of $11.0 million for the year ended December 31, 2017. Net loss reflects the changes in expenses discussed above and also reflects that revenue for the year ended December 31, 2018 decreased $9.6 million compared to revenue for the year ended 2017. Revenue in 2017 was primarily related to the Purdue Collaboration and reflects the amortization of the upfront payment on the Collaboration which did not reoccur in 2018.

Cash Runway Guidance: Exicure believes that, based on its current operating plans and as of the date of this press release, its existing cash and cash equivalents as of December 31, 2018 is sufficient to meet its anticipated cash requirements into early 2020.

About Exicure, Inc.
Exicure, Inc. is a clinical-stage biotechnology company developing therapeutics for immuno-oncology, inflammatory diseases and genetic disorders based on our proprietary Spherical Nucleic Acid, or SNA technology. We believe Exicure's proprietary SNA architecture has distinct chemical and biological properties that may provide advantages over other nucleic acid therapeutics and may have therapeutic potential to target diseases not typically addressed with other nucleic acid therapeutics. Exicure's lead program is in a Phase1b/2 trial in patients with advanced solid tumors. Exicure is based outside of Chicago, IL. www.exicuretx.com

Exicure Announces Dosing of First Patient in Phase 1b/2 Immuno-oncology Trial

Source:  Exicure, Inc. 2/25/19

Exicure, Inc. (OTCQB: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) constructs, announced today that it has dosed the first patient in its multicenter, open-label, Phase 1b/2 study of AST-008 combined with pembrolizumab. Enrollment in the trial is open to patients with superficial injectable tumors in advanced or metastatic solid tumor conditions including Merkel cell carcinoma, head and neck squamous cell carcinoma, cutaneous squamous cell carcinoma and melanoma. clinicaltrials.gov NCT03684785
 
“We believe that combining our immune system agonist drug with checkpoint inhibitors is an important strategy for leveraging the patient’s own immune system to fight cancer. We are excited to bring this approach into cancers like Merkel cell carcinoma, where patients have limited success using currently available treatments,” said Exicure CEO Dr. David Giljohann. “It is also an important milestone for Exicure in the development of our platform technology, which allows us to digitally design drug candidates and potentially bring them into clinic faster.”

The primary objective of the Phase 1b dose escalation stage is to assess the safety and tolerability of Exicure’s AST-008 drug alone and in combination with pembrolizumab, and to determine a dose for the Phase 2 stage of the study. Patients in the dose escalation stage may have previously been exposed to antibody checkpoint inhibitors, but not as a requirement for inclusion in the trial. In the Phase 2 portion of the study, Exicure will further evaluate AST-008 in combination with pembrolizumab in patients who have previously received but not responded to anti-PD-1 or anti-PD-L1 antibody therapy.

About Exicure’s AST-008 Drug
AST-008 is a toll-like receptor nine (TLR9) agonist oligonucleotide in a proprietary SNA format with immune-stimulatory properties. SNAs are dense, radial arrangements of nucleic acids (DNA) that have high cellular uptake and an enhanced presentation of the DNA for TLR9 agonism. AST-008 is designed to enter into and activate immune cells to elicit an immune response to treat solid tumors in combination with other agents such as checkpoint inhibitors. We observed that AST-008 showed potent antitumor activity as a monotherapy and synergized with anti-PD-1 antibodies in multiple preclinical tumor models. In a successful Phase 1 trial in healthy volunteers, AST-008 activated key immune cells and cytokines predictive for an anti-tumor effect in patients.

About Exicure, Inc.
Exicure, Inc. is a clinical stage biotechnology company developing a new class of immunomodulatory and gene regulating drugs against validated targets. Exicure's proprietary SNA architecture is designed to unlock the potential of therapeutic oligonucleotides in a wide range of cells and tissues. Exicure's lead programs address inflammatory diseases, genetic disorders and oncology. Exicure is based outside of Chicago, IL. www.exicuretx.com

Exicure Announces License and Development Agreement with Dermelix Biotherapeutics in Rare Genetic Skin Diseases

Source:  Exicure, Inc. 2/19/19

Exicure, Inc. (OTCQB: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) constructs, and Dermelix Biotherapeutics, a biopharmaceutical company focused on the development of innovative treatments for rare genetic skin diseases, today announced a license and development agreement to advance SNA therapeutics in rare genetic skin diseases. Under the terms of agreement, Dermelix licensed worldwide rights to research, develop, and commercialize Exicure’s technology for the treatment of Netherton Syndrome and up to five additional rare skin indications.

“Late last year we demonstrated the safety and clinical efficacy of our platform in a Phase 1 clinical trial in patients with psoriasis. Building on this success, we are now excited to extend the application of our SNA technology into rare dermatological conditions,” said Dr. David Giljohann, Chief Executive Officer of Exicure. “We look forward to combining Exicure’s ability to design and develop nucleic acid therapeutics with Dermelix’s deep knowledge of the clinical development and regulatory pathways for patients with rare dermatological conditions.”

Dr. Nick France, CEO of Dermelix noted, “Our company was founded to address the lack of innovation within the field of rare dermatological conditions. For too long patients have had few, or in many cases no, available treatment options. We believe the SNA platform will facilitate the rapid development of a portfolio of targeted treatment options for these severely debilitating conditions.”

Dermelix will initially develop a targeted therapy for the treatment of Netherton Syndrome (NS). NS is a rare and severe autosomal recessive disorder caused by loss-of-function mutations in the SPINK5 gene, which encodes the serine protease inhibitor LEKTI involved in skin barrier function. NS affects approximately 1 in 200,000 children born each year, and is characterized by severely inflamed, red, scaled, itchy skin, and patients are at increased risk of mortality in the first year of life due to recurrent infections and dehydration as a result of the impaired skin barrier. Currently, there are no approved treatments for NS patients and off-label use of standard of care treatments are of limited utility.

Under the terms of the agreement, Exicure will receive an upfront payment of $1 million at closing of the transaction and will receive an additional $1 million upon the exercise of each of the five options granted to Dermelix. Exicure will be responsible for conducting the early stage development for each indication up to IND enabling toxicology studies. Dermelix will assume subsequent development, commercial activities and financial responsibility. Exicure is eligible to receive potential payments following the achievement of certain clinical, regulatory, and commercial milestones of approximately $166 million per indication in each of six indications. In addition, Exicure will receive low double-digit royalties on annual net sales for SNA therapeutics developed.

About Exicure, Inc.
Exicure is a clinical stage biotechnology company developing a new class of immunomodulatory and gene regulating drugs against validated targets. Exicure's proprietary spherical nucleic acid (SNA™) architecture is designed to unlock the potential of therapeutic oligonucleotides in a wide range of cells and tissues. Exicure's lead programs address inflammatory diseases, genetic disorders and oncology. Exicure is based outside of Chicago, IL. www.exicuretx.com
 
About Dermelix Biotherapeutics, LLC
Dermelix is a privately-held, clinical-stage biopharmaceutical company focused on the development of innovative therapies for rare and debilitating dermatologic conditions with high unmet medical need. For more information, please visit http://www.dermelix.com/.

Exicure Announces US Clinical Sites for Phase 1b/2 Oncology Trial

Source:  Exicure, Inc. 2/5/19

Exicure, Inc. (OTCQB: XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA™) constructs, today announced four of the clinical trial sites for the Company’s Phase 1b/2 trial of AST-008.

“Exicure is thrilled to announce the four initial sites for the Phase 1b/2 clinical trial to evaluate our immune system activating spherical nucleic acid in combination with Keytruda for the treatment of solid tumors,” said Dr. David Giljohann, Chief Executive Officer of Exicure. “We are encouraged by the results of our Phase 1 clinical trial, which demonstrated that our drug is well-tolerated and activates key immune cells and signals. Eligible patients will be able to enroll in trial sites in the US.”

The open-label Phase 1b/2 trial will begin with a dose finding Phase 1b stage in combination with the anti-PD-1 therapy pembrolizumab (KEYTRUDA®), followed by a Phase 2 expansion stage. In the Phase 1b, Exicure will enroll patients with superficial injectable tumors and will prioritize those with Merkel cell carcinoma, cutaneous squamous cell carcinoma, melanoma, and squamous cell carcinoma of the head and neck. Preliminary data from the Phase 1b stage of the trial are expected in late 2019.

The clinical trial sites are as follows:

• Dana Farber Cancer Institute (Boston, Massachusetts)
• Holden Comprehensive Cancer Center at the University of Iowa (Iowa City, Iowa)
• John Wayne Cancer Institute at Providence St. John's Health Center (Santa Monica, California)
• Sylvester Comprehensive Cancer Center at the University of Miami (Miami, Florida)

AST-008 is a toll-like receptor nine (TLR9) activator being developed by Exicure. More information on the Phase 1b/2 trial of AST-008 can be found at clinicaltrials.gov, under identifier number NCT03684785.

KEYTRUDA® (Pembrolizumab; Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.) is an anti-PD-1 therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

About Exicure, Inc.
Exicure, Inc. is a clinical stage biotechnology company developing a new class of immunomodulatory and gene regulating drugs against validated targets. Exicure's proprietary spherical nucleic acid (SNA™) architecture is designed to unlock the potential of therapeutic oligonucleotides in a wide range of cells and tissues. Exicure's lead programs address inflammatory diseases, genetic disorders and oncology. Exicure is based outside of Chicago, IL. www.exicuretx.com