On March 9, 2018 ChemoCentryx, Inc., (Nasdaq: CCXI), reported financial results for the fourth quarter and full year ended December 31, 2017 and provided an overview of the Company’s recent corporate highlights (Press release, ChemoCentryx, MAR 9, 2018, View Source [SID1234524611]).

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"2017 was a truly remarkable year for ChemoCentryx," said Thomas J. Schall, Ph.D., President and Chief Executive Officer of ChemoCentryx. "We made marked progress in the global Phase III ADVOCATE trial of our lead compound avacopan in patients with ANCA vasculitis; we submitted a Conditional Marketing Authorization (CMA) application in Europe for avacopan for ANCA treatment, and we successfully expanded the Company’s clinical development program to include treatment of the devastating kidney diseases C3G and FSGS. 2018 is also off to a very strong start, exemplified by the EMA’s validation of our CMA application, resulting in a milestone payment of $50 million, itself a part of a considerably strengthened financial position of up to $100 million in new capital commitments. Our commercial planning efforts are underway too. We are advancing towards our goal of building a fully-integrated biopharmaceutical company, delivering precision medicines to patients suffering from serious diseases. Make no mistake, clinical momentum is strong; our advance continues. We intend to extend further the reach of avacopan, beginning clinical studies in hidradenitis suppurativa, a debilitating and disfiguring chronic skin disease, later this year."

Recent Highlights

• ChemoCentryx’s Phase III ADVOCATE pivotal trial of avacopan for the treatment of ANCA vasculitis has 200 sites activated and 220 patients enrolled to date. The trial will evaluate the safety and efficacy of avacopan following 52 weeks of treatment and will include approximately 300 patients. In addition to testing the effect of avacopan on improving active vasculitis, the ADVOCATE trial will also test avacopan’s efficacy in preventing the recurrence of vasculitis, one of the major limitations of the current standard of care for patients with ANCA vasculitis.

• In December 2017, ChemoCentryx strengthened its balance sheet with up to $100 million in new capital commitments, including a milestone of $50 million from partner Vifor Pharma upon the European Medicines Agency’s validation of the Company’s Conditional Marketing Authorization (CMA) application for avacopan for the treatment of patients with ANCA vasculitis, along with the growth capital financing agreement of up to $50 million. Such additional capital is expected to provide ChemoCentryx sufficient funding to advance avacopan through data from the Phase III ADVOCATE trial, as well as associated potential registration filings in the U.S. and EU.
• Patient enrollment is ongoing in a clinical trial evaluating avacopan in a second renal indication, C3 Glomerulopathy (C3G), a rare disorder that often affects the young, requiring dialysis and often kidney transplant. In addition, the Company’s CCR2 inhibitor CCX140 is being evaluated in two sub-populations of primary Focal Segmental Glomerulosclerosis (FSGS), an orphan kidney disease for which there is no approved treatment option. One trial involves non-nephrotic primary FSGS patients, whose disease cause is idiopathic; and the other trial is for primary FSGS patients with nephrotic syndrome, where reduction in proteinuria may constitute the registration endpoint.

• ChemoCentryx intends to initiate the clinical development of avacopan in hidradenitis suppurativa (HS), an inflammatory and chronic skin disease characterized by recurrent, painful, boil-like nodules under the skin. The Company plans to initiate clinical studies with avacopan in HS by the end of 2018.

• In anticipation for potential commercialization in the U.S., William (Bill) J. Fairey, Jr. joined ChemoCentryx as Executive Vice President and Chief Operating Officer to lead the Company’s commercial strategy along with other key operational functions of the Company. Mr. Fairey brings extensive experience in commercialization, marketing, and operations from his 25 years in the pharmaceutical industry and most recent position as President of Actelion Pharmaceuticals U.S.

• In January 2018, data from the ongoing Phase Ib clinical trial of CCX872, the Company’s second CCR2 inhibitor, in locally advanced/metastatic pancreatic patients were presented at the 2018 ASCO (Free ASCO Whitepaper)-SITC Clinical Immuno-Oncology Symposium, demonstrating promising overall survival (OS) of all patients randomized of 29% at 18 months with CCX872 and FOLFIRINOX combination therapy. This compares favorably with previously published OS rates of 18.6% at 18 months using FOLFIRINOX alone to treat pancreatic cancer patients with metastatic disease.
Fourth Quarter and Full Year 2017 Financial Results

Pro forma cash, cash equivalents and investments, including remaining upfront commitments and milestone payments, totaled $195.2 million at December 31, 2017.

Revenue was $56.3 million for the fourth quarter of 2017, compared to $4.9 million for the same period in 2016. For the full year ended December 31, 2017, revenue was $82.5 million, compared to $11.9 million for 2016. The increase in revenue from 2016 to 2017 was due to the CMA application validation milestone, amortization of the upfront license fee commitments from Vifor pursuant to the avacopan and CCX140 agreements and collaboration revenue for development services under the CCX140 agreement. These increases were partially offset by a decrease in grant revenue from the FDA to support the clinical development of avacopan for the treatment of patients with ANCA vasculitis.

Research and development expenses were $12.9 million for the fourth quarter of 2017, compared to $9.2 million for the same period in 2016. Full year 2017 research and development expenses were $49.5 million compared to $37.9 million in 2016. The increase from 2016 to 2017 was primarily due to the initiation and patient enrollment of the avacopan Phase III ADVOCATE trial in patients with ANCA vasculitis and start-up expenses related to the Phase II clinical trials in patients with FSGS and C3G. These increases were partially offset by lower Phase II clinical development expenses primarily due to the completion of the avacopan CLEAR and CLASSIC clinical trials for the treatment of ANCA vasculitis in 2016 and lower Phase I development expense due to the completion of enrollment in the clinical trial for CCX872 in patients with advanced pancreatic cancer in 2016.

General and administrative expenses were $4.1 million for the fourth quarter of 2017, compared to $3.6 million for the same period in 2016. Full year 2017 general and administrative expenses were $16.5 million, compared to $14.7 million in 2016. The increase from 2016 to 2017 was primarily due to higher intellectual property related expenses and accounting related fees associated with preparing to meet the requirements pursuant to the Sarbanes-Oxley Act of 2002, partially offset by lower travel expenses.

Net income for the fourth quarter of 2017 was $39.7 million, compared to a net loss of $7.7 million for the same period in 2016. Full year 2017 net income was $17.9 million, which compares favorably to the $40.0 million net loss in 2016.

Total shares outstanding at December 31, 2017 were approximately 48.8 million shares.

The Company expects to utilize cash and investments between $65 million and $75 million in 2018.

Conference Call and Webcast

The Company will host a conference call and webcast today, March 9, 2018 at 8:30 a.m. Eastern Time / 5:30 a.m. Pacific Time. To participate by telephone, please dial 877-303-8028 (Domestic) or 760-536-5167 (International). The conference ID number is 4887708. A live and archived audio webcast can be accessed through the Investors section of the Company’s website at www.ChemoCentryx.com. The archived webcast will remain available on the Company’s website for fourteen (14) days following the conference call.

On March 9, 2018 VBL Therapeutics (Nasdaq:VBLT), a clinical-stage biotechnology company focused on the discovery, development and commercialization of first-in-class treatments for cancer, reported that it will host a conference call and live audio webcast on Thursday, March 15 at 8:30am Eastern Time to report fiscal year ended December 31, 2017 financial results (Press release, VBL Therapeutics, MAR 9, 2018, View Source [SID1234524634]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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On March 9, 2018 Exicure, Inc., the pioneer in gene regulatory and immunotherapeutic drugs utilizing three-dimensional, spherical nucleic acid (SNA) constructs, reported full year financial results for the year ended December 31, 2017 and provided an update on corporate progress (Press release, Exicure, MAR 9, 2018, View Source [SID1234524612]).

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"Exicure is realizing the promise of our SNA technology through ongoing clinical advancements, including the launch of a Phase 1 clinical trial of AST-008, our TLR9 agonist being developed for immuno-oncology applications," said Dr. David Giljohann, Chief Executive Officer of Exicure. "2017 was a foundational and transformative year for Exicure. By raising approximately $31.5 million in gross proceeds through a private placement financing and completion of a reverse merger, the company has the capital resources to drive our therapeutic pipeline into 2019. In 2018, we anticipate Phase 1 results from both AST-008, and XCUR17, our therapeutic candidate for psoriasis."

Corporate Progress

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Launched the Phase 1 clinical trial of AST-008, a TLR9 agonist for immuno-oncology applications. Received authorization from Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom to conduct a Phase 1 clinical trial of AST-008. The company began dosing healthy subjects during the fourth quarter of 2017.
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Filed a clinical trial application for XCUR17. In February of 2018, Exicure received approval from Bundesinstitut für Arzneimittel und Medizinprodukte (BfArM), the medical regulatory body in Germany, to conduct a Phase 1 clinical trial.
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Completed reverse merger transaction and private placement financing. Through a series of steps beginning on September 26, 2017, Exicure completed a reverse merger and raised approximately $31.5 million in gross proceeds through a private placement of its common shares.
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Advanced towards public market trading. Exicure’s Form S-1 was declared effective on February 6, 2018. The company currently awaits FINRA’s approval of Form 211. Subsequent to FINRA approval, the company will finalize steps to be represented on the OTCQB market.
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Strengthened management team. Jocelyn Trokenheim joined the company as Vice President, Head of Business Development. Ms. Trokenheim most recently served as Vice President of Corporate Development at Takeda Pharmaceuticals where she was responsible for global strategic transactions, such as large-scale M&A, divestitures and strategic partnering.

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 expects this trial to be completed in mid-2018. This clinical trial is evaluating the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple doses by subcutaneous administration in healthy subjects. The company ultimately plans to clinically advance AST-008 in combination with checkpoint inhibitors.

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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 proposed Phase 1 trial of XCUR17 is a microplaque study in patients with mild to moderate psoriasis. BfArM, the German regulatory authority, has approved the Phase 1 clinical trial. The company expects the first patient to be dosed in early 2018 and expects the clinical trial to be completed in mid-2018.

AST-005: AST-005 is an antisense SNA that targets the mRNA encoding TNF. AST-005 is the subject of our collaboration with Purdue Pharma L.P. Purdue Pharma L.P. has completed subject dosing in the Phase 1b clinical trial for AST-005. The Phase 1b clinical trial was conducted in Germany and was intended to evaluate, among other things, the safety and tolerability of AST-005.

2017 Financial Results and Financial Guidance

Cash Position: As of December 31, 2017, Exicure had cash and cash equivalents of $25.8 million compared to $19.6 million as of December 31, 2016. In 2017, Exicure raised approximately $31.5 million in gross proceeds from the private placement of common stock.

Research and Development (R&D) Expenses: Research and development expenses were $14.1 million for the year ended December 31, 2017 compared to $13.7 million for the year ended December 31, 2016. The increase in research and development expense of $0.4 million was primarily due to a net increase in costs related to the company’s clinical development programs of $2.2 million and higher employee-related expenses of $0.4 million, mostly offset by lower platform and discovery-related expense of $2.2 million.

General and Administrative (G&A) Expenses: General and administrative expenses were $7.0 million for the year ended December 31, 2017, compared to $3.5 million for the year ended December 31, 2016. The increase in general and administrative expenses of $3.5 million was primarily due to the write-off of costs related to financing, non-capitalized costs related to the reverse merger, higher stock-based (non-cash) compensation expense, and compliance costs and other costs associated with our transition to being a public company.

Net Loss: Net loss was $12.0 million for the year ended December 31, 2017, compared to net loss of $16.9 million for the year ended December 31, 2016.

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, 2017 is sufficient to meet its anticipated cash requirements for the next twelve months.

Upcoming Events

Dr. David Giljohann, Chief Executive Officer of Exicure, will be giving a presentation on March 20th at the American Chemical Society (ACS) national meeting in New Orleans at the Ernest N. Morial Convention Center. Dr. Giljohann will also be presenting at the Future Leaders in Biotech conference on March 23rd at the Millennium Broadway Hotel in New York.

On March 9, 2018 CTI BioPharma Corp. (CTI BioPharma) (NASDAQ: CTIC) reported that results from the Phase 3 PERSIST-2 clinical trial of pacritinib (an investigational JAK2 inhibitor) have been published online in JAMA Oncology (Press release, CTI BioPharma, MAR 9, 2018, View Source;p=RssLanding&cat=news&id=2337281 [SID1234524602]). The randomized, international, multicenter study compared the efficacy and safety of pacritinib at two dose levels, compared with best available therapy (BAT), which included ruxolitinib (a JAK1/JAK2 inhibitor), in patients with myelofibrosis and thrombocytopenia (defined as platelet counts ≤100 x 109/L). The publication can be accessed at: View Source

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In the intent-to-treat patient population of the study, the combined pacritinib arms (400mg once daily and 200mg twice daily dosing, 149 patients total) demonstrated a significant improvement of 35% or more in spleen volume reduction (SVR) at 24 weeks of treatment in 27 patients (18%) compared to 2 patients (3%) out of 72 patients in the BAT arm, which included treatment with ruxolitinib (P=0.001). The combined pacritinib treatment arms also demonstrated a greater than 50% reduction in total symptom score (TSS) in 37 patients (25%), compared to 10 patients (14%) in the BAT arm (P=0.079). Additionally, an exploratory analysis of the 74 patients who received pacritinib 200mg twice daily showed an improvement of 35% or more reduction of SVR in 16 patients (22%; P=0.001 vs BAT) and 50% or greater reduction of TSS in 24 patients (32%; P=0.01 vs BAT).

Pacritinib was generally well tolerated. The most commonly reported (≥15%) nonhematologic adverse events with pacritinib were gastrointestinal events, fatigue, peripheral edema, and dizziness, and those with BAT (including 19 patients with watchful-waiting only) were abdominal pain, fatigue, diarrhea, and peripheral edema. The majority of common nonhematologic adverse events were grade 1 or 2 in severity. Diarrhea was the most frequently observed adverse event with pacritinib (53% grade 1/2; 4% grade 3) most often occurring during weeks 1 to 8. The incidence of diarrhea was lower with pacritinib twice daily dosing compared to once daily dosing (48% vs 67%, respectively). Diarrhea was manageable with standard antidiarrheal agents (e.g., loperamide) and generally resolved within 1 to 2 weeks. The rate of on-study death was lowest with pacritinib twice daily (6%) compared to BAT (9%) and pacritinib once daily (14%).

Cardiac events were reported at similar rates in all arms (32%, pacritinib once daily or twice daily; 28%, BAT) and were most commonly peripheral edema in all arms. Grade 3 or 4 cardiac events were reported in 13 patients (13%) treated with pacritinib once daily, 7 patients (7%) treated with pacritinib twice daily, and 9 patients (9%) treated with BAT.

Bleeding events were reported at similar rates in all arms (36%, 42%, and 41% of patients treated with pacritinib once daily, twice daily, and BAT, respectively) and were most commonly epistaxis in all arms. Grade 3 or 4 bleeding events were reported in 7 patients (7%), 15 patients (14%), and 7 patients (7%) treated with pacritinib once daily, twice daily, and BAT, respectively.

"Pacritinib was shown to reduce both spleen volume and total symptom score, two very important clinical measures, in myelofibrosis patients with thrombocytopenia including those patients who received prior treatment with ruxolitinib," stated John Mascarenhas, M.D., Adult Leukemia Program, Tisch Cancer Institute, Icahn School of Medicine at Mount Sinai. "Clinical improvements in hemoglobin levels and reduction in transfusions were also seen in patients who received pacritinib, and pacritinib had a generally manageable safety profile."

Dr. Mascarenhas continued, "Myelofibrosis is a difficult and progressive disease, and patients with thrombocytopenia that have already received JAK2 inhibitor therapy have an especially poor prognosis. These study results indicate there is important potential clinical benefit of pacritinib for these patients."

The PERSIST-2 trial results were previously presented at the 58th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting, in December 2016.

CTI BioPharma is currently enrolling patients in the PAC203 study, which is evaluating the safety and efficacy of three dosing schedules, including 200mg twice daily (BID), 100mg twice daily (BID), and 100mg once daily (QD).