On August 6, 2018 Exicure, Inc. (OTCQB:XCUR), the pioneer in gene regulatory and immunotherapeutic drugs utilizing three-dimensional, spherical nucleic acid (SNA) constructs, reported financial results for the second quarter ended June 30, 2018, and provided an update on corporate progress (Press release, Exicure, AUG 6, 2018, View Source [SID1234528463]).

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"With the addition of a new COO and the topical dosing of XCUR17 in patients with mild to moderate psoriasis, Exicure continues to make progress on our mission," said Dr. David Giljohann, Chief Executive Officer of Exicure. "In June of 2018, we released data at the Cure SMA Annual Conference demonstrating the use of Exicure’s SNA technology in Spinal Muscular Atrophy mouse models. Based on the results of this preclinical study, we intend to further pursue our early stage research activities in neurological applications," added Dr. Giljohann.

Corporate Progress

Began trading on OTCQB market under the ticker symbol XCUR.
Announced the addition of Matthias Schroff as Chief Operating Officer. Dr. Schroff previously served as CEO of Vaximm and Mologen.
Commenced topical dosing in our Phase 1 clinical trial for XCUR17 in patients with mild to moderate psoriasis.
Presented pre-clinical data demonstrating the performance of Exicure’s SNA compound designed for use in spinal muscular atrophy at the Cure SMA Annual Conference with collaborators at The Ohio State University Wexner Medical Center.
Completed third cohort of volunteers in our AST-008 trial and announced the planned enrollment of a Phase 1b/2 trial expected to commence in late 2018 in combination with checkpoint inhibitors.
Pre-Clinical Updates

In June, Exicure and The Ohio State University Wexner Medical Center presented a poster at the Cure SMA Annual Conference titled: "Nusinersen in spherical nucleic acid (SNA) format improves efficacy both in vitro in SMA patient fibroblasts and in Δ7 SMA mice and reduces toxicity in mice." It was observed in the preclinical study that nusinersen in SNA format prolonged survival by four-fold (maximal survival of 115 days compared to 28 days for nusinersen-treated mice) as well as doubled the levels of healthy full-length SMN2 mRNA and protein in SMA patient fibroblasts when compared to nusinersen.

Pipeline Updates

AST-008: AST-008 is an SNA consisting of toll-like receptor 9 (TLR9) agonists designed for immuno-oncology applications. The Phase 1 clinical trial of AST-008 evaluates the safety, tolerability, pharmacokinetics, and pharmacodynamics of AST-008 by subcutaneous administration in healthy volunteers. This trial is expected to be completed in the third quarter of 2018. We currently anticipate preparing and commencing a Phase 1b/2 clinical trial for AST-008 in combination with checkpoint inhibitors in late 2018.

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. Our Phase 1 trial of XCUR17 is a microplaque study in patients with mild to moderate psoriasis. We have dosed 19 of the prospective 25 patients. Full enrollment and trial completion is expected during the fourth quarter.

AST-005: AST-005 is an SNA containing TNF antisense oligonucleotides and is intended to be applied in a gel to psoriatic lesions. AST-005 is the subject of our collaboration with Purdue Pharma L.P. Purdue Pharma notified Exicure that it has declined to exercise its option to develop AST-005 at this time, but that it also intends to retain rights relating to the TNF target. Purdue reserves its right to continue joint development, with Exicure, of new anti-TNF drug candidates and to retain its exclusivity and other rights to AST-005.

Second Quarter 2018 Financial Results and Financial Guidance

Cash Position: As of June 30, 2018, Exicure had cash and cash equivalents of $16.4 million compared to $25.8 million as of December 31, 2017.

Research and Development (R&D) Expenses: Research and development expenses were $3.8 million for the three months ended June 30, 2018, compared to $3.3 million for the three months ended June 30, 2017. The increase in research and development expense of $0.5 million was primarily due to higher employee related expenses resulting from the hire of our Chief Operating Officer and higher platform discovery expenses attributable to costs necessary to maintain our intellectual property portfolio. Clinical development expenses were nearly flat quarter over quarter.

General and Administrative (G&A) Expenses: General and administrative expenses were $2.0 million for the quarter ended June 30, 2018, compared to $2.1 million for the quarter ended June 30, 2018. The decrease in general and administrative expenses of $0.1 million reflects the net change attributable to a decrease in legal and accounting expenses due to the absence of certain financing expenses incurred during 2017 offset by an increase in costs attributable to being a public company.

Net Loss: Net loss was $6.8 million for the quarter ended June 30, 2018, compared to net loss of $3.0 million for the quarter ended June 30, 2017. The $3.8 million increase in net loss is due principally to a decrease in revenue of $2.7 million that mostly reflects the absence of revenue recognized in the prior period related to the amortization of the upfront payment, and certain reimbursable research and development activities, under the Purdue Collaboration. In addition, we had an increase in Other Loss of $0.8 million principally due to the fair value adjustment of our common stock warrant liability.

Cash Runway Guidance: Exicure believes that, based on its current operating plans and estimates of expenses, as of the date of this press release, its existing cash and cash equivalents as of June 30, 2018, will be sufficient to meet its anticipated cash requirements through March 31, 2019.

On August 6, 2018 Alpine Immune Sciences, Inc. (NASDAQ:ALPN), a company focused on discovering and developing innovative immunotherapies to treat cancer, autoimmune/inflammatory, and other diseases, reported the company will present a business overview and update at the 2018 Wedbush PacGrow Healthcare Conference on Tuesday, August 14, 2018 at 3:40 pm Eastern Time in New York, NY (Press release, Alpine Immune Sciences, AUG 6, 2018, View Source [SID1234528462]).

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A live webcast of the presentation will be available online in the investor relations section of the company’s website at View Source A replay of the presentation will be available on the company website for 90 days following the webcast.

On August 6, 2018 Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage biopharmaceutical company focused on creating innovative cancer immunotherapies, reported financial results for the second quarter ended June 30, 2018 and recent corporate updates (Press release, Arcus Biosciences, AUG 6, 2018, View Source [SID1234528461]).

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"We are pleased with the progress we have made in advancing our molecules into studies in patients with three product candidates now in clinical development," said Terry Rosen, Ph.D., Chief Executive Officer at Arcus. "We continue to believe that AB928, our dual adenosine receptor antagonist, has the potential to significantly enhance the activity of anti-PD-1 antibodies as well as certain chemotherapies and have designed our clinical program for AB928 to demonstrate the potential of these combinations in multiple tumor types where the adenosine pathway is believed to play a critical role. The program will include extensive biomarker analysis to identify biomarkers that may be predictive of response and to demonstrate that changes in immune markers correlate with responses and are consistent with AB928’s mechanism. We look forward to reporting initial safety, biomarker and clinical data for our AB928 combinations in patients in the first half of 2019."

Pipeline Updates

AB928 (dual A 2a R/A 2b R antagonist)

Reported the unblinded safety results from the Phase 1 double-blinded, randomized, placebo-controlled trial for AB928 in healthy volunteers. AB928 was found to be safe and well tolerated at all doses evaluated, including the highest dose tested of 200 mg once daily (QD) (with food). At the AACR (Free AACR Whitepaper) Annual Meeting in April 2018, the Company presented pharmacodynamic data demonstrating that an AB928 dose between 75 mg and 150 mg QD should be sufficient to achieve greater than 90% inhibition of the adenosine 2a receptor (A2aR) pathway.
Initiated dosing of patients in dose-escalation trial to evaluate AB928 in combination with AB122. This trial is designed to identify the recommended dose of AB928 that can be combined with a fixed dose of AB122 for the AB928 + AB122 expansion cohorts in the AB928 Phase 1/1b program. Dosing began with 75 mg QD of AB928 and 240 mg of AB122 every two weeks (Q2W). Subsequent dose-escalation cohorts will evaluate doses of 150 mg and 200 mg QD of AB928, or intermediate doses, in combination with 240 mg Q2W of AB122.
Preparing to initiate Phase 1/1b trials to evaluate AB928 in combination with three different chemotherapy regimens. Each trial will evaluate AB928 in combination with a chemotherapy regimen that is considered a standard of care for each tumor type:
–AB928 in combination with Doxil in triple negative breast cancer (TNBC) and ovarian cancer
–AB928 in combination with mFOLFOX in colorectal and gastroesophageal cancers
–AB928 in combination with carboplatin/pemetrexed and pembrolizumab in non-small cell lung cancer (NSCLC)

Each of these chemotherapy regimens induces immunogenic cell death (a hallmark of which is the generation of significant amounts of adenosine), and therefore their anti-cancer activity is believed to be enhanced by A2aR antagonism. The trials will begin with a dose-escalation portion to identify the recommended dose of AB928 for each chemotherapy regimen, which will be followed by expansion cohorts. The dose-escalation portion will enroll patients with the same tumor types as the expansion cohorts. The Company also plans to evaluate other AB928 combinations, e.g. AB928 + AB122, in these trials.

Initiated development of an immunohistochemistry assay with a leading cancer diagnostic company to be used in Arcus’s clinical trials for AB928 and future clinical trials of AB680. This assay will test for expression of multiple proteins, including PD-L1 and CD73, as well as CD8+ T cells, in the Company’s Phase 1/1b program for AB928.
AB122 (anti-PD-1 antibody)

Continued dosing in the ongoing Phase 1 dose-escalation trial in cancer patients in Australia. The Company has identified 240 mg as the recommended dose for AB122 when administered every two weeks and is currently enrolling patients in additional cohorts to explore other dosing schedules.
Preparing to initiate an expansion cohort to evaluate AB122 as a monotherapy in NSCLC. The objective of this cohort is to confirm that the clinical activity of AB122 is similar to that of approved anti-PD-1 antibodies in NSCLC patients.
AB154 (anti-TIGIT antibody)

Received regulatory approval to initiate a Phase 1 trial to evaluate the safety, pharmacokinetics, pharmacodynamics and clinical activity of AB154 as monotherapy and in combination with AB122 in Australia. The dose-escalation portion will first evaluate increasing doses of AB154 as a monotherapy and subsequently in combination with AB122. Once the recommended doses for AB154 as a monotherapy and in combination with AB122 have been identified, the Company plans to initiate expansion cohorts to evaluate AB154 as a monotherapy and in combination with AB122 in selected tumor types. In the future, the Company plans to explore AB154 in combination with some of its other product candidates. The Company also plans to file an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) in the fourth quarter of 2018 to initiate clinical testing of AB154 in the U.S.
AB680 (small molecule CD73 inhibitor)

Initiated pre-clinical development of an oral formulation of AB680. IND-enabling studies of an oral formulation of AB680 are ongoing.
Corporate Updates

In July, Arcus announced that Taiho Pharmaceutical Co., Ltd. exercised its option under the Option and License Agreement entered into in September 2017 to obtain an exclusive development and commercialization license to the Company’s adenosine receptor antagonist program, which includes AB928 and back-up compounds, in Japan and certain other territories in Asia (excluding China).
In June, Arcus and Infinity Pharmaceuticals announced a clinical collaboration to evaluate two triple combination therapies in TNBC and ovarian cancer. These cohorts will be incorporated into Arcus’s Phase 1/1b trial for AB928 in TNBC and ovarian cancer.
In June, Arcus announced the promotion of Jennifer Jarrett to Chief Operating Officer. Ms. Jarrett also continues to serve as the Company’s Chief Financial Officer.
Upcoming Milestones

In the second half of 2018, the Company expects to

Present the final data from the Phase 1 trial of AB928 in healthy volunteers at a medical conference in the fall.
Initiate a Phase 1 trial to evaluate the safety and pharmacokinetic profile of AB680 in healthy volunteers.
Present safety, pharmacokinetic, receptor occupancy and clinical activity data from the ongoing Phase 1 trial of AB122.
In the first half of 2019, the Company expects to

Present initial data from the dose-escalation trials of AB928 + AB122 and AB928 + chemotherapy, which will include data on safety, biomarker analysis and clinical activity for each of the combinations.
Initiate the expansion cohorts for the AB928 + AB122 and AB928 + chemotherapy combinations. Initial data from the expansion cohorts are expected in late 2019.
Report safety and pharmacokinetic data from the Phase 1 trial of AB680 in healthy volunteers.
Initiate clinical testing of AB680 in cancer patients.
Second Quarter and Year-to-Date 2018 Financial Results

Cash Position: At June 30, 2018, cash and investments (which include cash equivalents and both short- and long-term investments) were $277.5 million, compared to $175.7 million at December 31, 2017. The increase was primarily attributable to $124.7 million in net proceeds from the Company’s initial public offering in March.
Revenues: Collaboration and license revenues for the second quarter ended June 30, 2018 were $1.3 million, compared to no revenue for the same period in 2017. Collaboration and license revenues for the six months ended June 30, 2018 were $2.5 million, compared to no revenue for the same period in 2017. The increase in revenues for both periods was attributable to revenues recognized from the Option and License Agreement the Company entered into with Taiho in September 2017.
R&D Expenses: Research and development expenses for the second quarter ended June 30, 2018 were $13.7 million, compared to $7.8 million for the same period in 2017. The increase was primarily driven by the Company’s ongoing clinical studies of AB928 and AB122, pre-clinical and manufacturing costs to prepare two additional programs, AB154 and AB680, for clinical trials, an increase in R&D headcount to support the Company’s other programs and a milestone payable due to the regulatory filing for AB154. Research and development expenses for the six months ended June 30, 2018 were $25.4 million, compared to $13.6 million for the same period in 2017.
G&A Expenses: General and administrative expenses for the second quarter ended June 30, 2018 were $3.5 million, compared to $1.8 million for the same period in 2017. The increase was primarily due to higher legal and accounting fees and additional staff in key areas required to support a public company infrastructure, as well as increased facilities and office expenses related to our expanded facility in Hayward. General and administrative expenses for the six months ended June 30, 2018 were $6.4 million, compared to $3.3 million for the same period of 2017.
Net Loss: Net loss for the second quarter ended June 30, 2018 was $13.5 million, compared to $9.6 million for the same period in 2017. Net loss for the six months ended June 30, 2018 was $26.5 million, compared to $16.8 million for the same period in 2017. The increase in net loss was primarily attributable to the increase in operating expenses noted above.
Based on its current operating plan, the Company expects that its cash and investments as of June 30, 2018 will enable the Company to fund its anticipated operating expenses and capital expenditure requirements into at least the fourth quarter of 2020.

On August 6, 2018 Foundation Medicine, reported the publication of the results of a large study demonstrating that its novel, investigational assay to measure blood tumor mutational burden (bTMB) can help predict response to the anti-PD-L1 immunotherapy, atezolizumab, (TECENTRIQ) in patients with previously treated non-small cell lung cancer (NSCLC) (Press release, Foundation Medicine, AUG 6, 2018, View Source [SID1234528460]). The study, published in the journal Nature Medicine, was the result of a collaboration between Foundation Medicine and Genentech, a member of the Roche Group, and demonstrates the potential of bTMB to expand precision oncology approaches for patients with advanced cancers, including metastatic lung cancer. In addition, these results show that bTMB may be an independent predictor of clinical benefit, regardless of PD-L1 expression as assessed by immunohistochemistry.

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"A significant proportion of patients with advanced lung cancer do not have adequate tissue for traditional biomarker testing. These study results represent an important advance for the liquid biopsy field and suggest that measuring TMB in the blood with our novel assay can help identify patients more likely to benefit from anti-PD-L1 immunotherapy," said Vincent Miller, M.D., chief medical officer at Foundation Medicine. "We look forward to further developing this assay through ongoing clinical trials and ultimately as a companion diagnostic to help oncologists make the most informed decisions possible, even when a tissue sample is not feasible."

In the study, clinical data for the novel bTMB assay was reported from a retrospective analysis of more than 1,000 samples from patients with previously treated, advanced NSCLC who participated in Genentech’s Phase II POPLAR and Phase III OAK clinical trials. The study used samples from the POPLAR trial to identify a range of bTMB thresholds that correlated with clinically meaningful outcomes, which were then confirmed using samples from the OAK study. Within the OAK study, patients with bTMB ≥ 16 total mutations (14 mut/Mb) showed significantly improved progression-free survival when treated with atezolizumab as compared to those patients with bTMB ≥ 16 total mutations (14 mut/Mb) treated with docetaxel chemotherapy (Hazard Ratio=0.65 [95% CI: 0.47, 0.92]; p=0.013). According to the study’s first author, David Gandara, M.D. of the UC Davis Comprehensive Cancer Center, "These are exciting times in lung cancer immunotherapy. Having a blood test that can identify those patients most likely to benefit would be a huge advantage for both physicians and patients. This publication is the first step toward what I anticipate will be full clinical application of this assay."

This bTMB assay is being prospectively evaluated in two Genentech studies: in the Phase III Blood First Assay Screening Trial (BFAST) as a companion diagnostic assay to validate bTMB as a non-invasive biomarker of response to first-line atezolizumab in advanced NSCLC patients, and in the single arm Phase II Blood First-Line Ready Screening Trial (B-F1RST) evaluating atezolizumab monotherapy in first-line NSCLC.

In April 2018, the U.S. Food and Drug Administration (FDA) granted a Breakthrough Device designation for Foundation Medicine’s new liquid biopsy assay, which will expand upon its current liquid biopsy assay to include genomic biomarkers for microsatellite instability (MSI) and bTMB. If approved, this test could be the first FDA-approved liquid biopsy assay to incorporate multiple companion diagnostics (CDx) and multiple biomarkers to inform the use of targeted oncology therapies, including immunotherapies.

On August 6, 2018 Immune Biosolutions Inc. (IBio), a JLABS @ Toronto resident company, reported a research collaboration and license agreement with Janssen Biotech, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson (Press release, Immune Biosolutions, AUG 6, 2018, View Source [SID1234528459]). The agreement, facilitated by Johnson & Johnson Innovation LLC, will focus initially on the discovery and development of therapeutic antibodies in oncology.

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The collaboration will utilize IBio’s proprietary Nebula antibody discovery platform to advance oncology targets that historically have been a challenge for therapeutic development. "IBio’s Nebula platform provides a new angle to antibody discovery with four families of technologies integrated within a single platform to deliver innovative antibody therapies in key disease areas. We are excited to be working with Janssen in this promising new area which we believe has the potential to transform and improve human health," said Frédéric Leduc, chief executive officer of Immune Biosolutions.

Financial terms of the agreement are not being disclosed.