On November 14, 2017 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ:APTO) (TSX:APS), a clinical-stage company developing highly differentiated therapeutics that target the underlying mechanisms of cancer, reported financial results for the three months ended September 30, 2017 and reported on corporate developments. Unless specified otherwise, all amounts are in Canadian dollars (Press release, Aptose Biosciences, NOV 14, 2017, View Source;p=RssLanding&cat=news&id=2316911 [SID1234522060]).

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The net loss for the quarter ended September 30, 2017 was $3.3 million ($0.14 per share) compared with $4.0 million ($0.31 per share) in the quarter ended September 30, 2016. Total cash and cash equivalents and investments as of September 30, 2017 were $13.6 million (or $10.9 million US dollars) which, based on current operations, provide the Company with sufficient resources to fund research and development and operations into Q4 2018.

"During the third quarter, we made important progress with both of our novel small molecule compounds for the treatment of certain hematologic malignancies," said William G. Rice, Ph.D., Chairman, President and Chief Executive Officer. "We’ve completed manufacture of high quality drug substance and explored multiple formulations of CG’806, our oral first-in-class pan-FLT3/pan-BTK inhibitor. We triggered PK and dose range finding studies with CG’806 in order to prepare the agent for advancement into the clinic for patients with acute myeloid leukemia and certain B-cell malignancies. We also generated renewed excitement about the potential for APTO-253, a clinical-stage compound that effectively inhibits expression of the c-Myc oncogene, and we believe we have effectively addressed the formulation and manufacturing setbacks that led to a clinical hold for APTO-253. Our goal is to return APTO-253 to the clinic for the treatment of patients with AML or MDS."

Corporate Highlights
Successful completion of APTO-253 formal root cause studies – Aptose successfully completed Formal Root Cause Studies for the manufacturing setback related to the clinical batch supply that failed stability testing and established a Corrective and Prevention Action (CAPA) plan.

Manufacture of APTO-253 clinical supply has begun – The Company has initiated the process to manufacture a cGMP clinical supply that will be required for a potential return of APTO-253 to the clinic. Upon manufacture of the new clinical supply, Aptose plans to perform stability, sterility, mock infusion, animal bridging and blood compatibility studies. Following completion of those studies, we would plan to submit findings to the FDA to seek release of the clinical hold and allow return of APTO-253 to the open Phase 1b trial in patients with acute myeloid leukemia (AML) or myelodysplastic syndrome (MDS).

CG’806 manufacturing and preclinical testing progress – Aptose produced a highly purified batch of drug substance (API) to support pharmacokinetic (PK), formulation evaluation and dose range finding studies of CG’806. The PK and formulation evaluation studies have been completed, and the dose range finding preclinical studies are expected to begin before or shortly after year-end. Separately, the Company has initiated the process to manufacture multi-kilogram batches of GLP-grade API for use in the formal GLP/IND-enabling animal toxicity studies.

Intellectual property protection for CG’806 – During the third quarter, Aptose continued to strengthen its patent portfolio. In September, Aptose and partner CrystalGenomics announced that the United States Patent and Trademark Office issued U.S. Patent No. 9,758,508 which claims numerous compounds, including the CG’806 compound, pharmaceutical compositions comprising the CG’806 compound, and methods of treating various diseases. The patent is expected to provide protection until the end of 2033. In August, Aptose received notice allowing U.S. Patent Application No. 14/655,954. The allowed ‘954 application claims numerous compounds, including the CG’806 compound, pharmaceutical compositions comprising the CG’806 compound, and methods of treating various diseases caused by abnormal or uncontrolled activation of protein kinase in a mammal by administering a compound, including the CG’806 compound.

Strengthened financial position – The Company recently announced that it entered into a Common Shares Purchase Agreement with Aspire Capital Fund, LLC ("Aspire Capital") to sell up to US$15.5 million of common shares to Aspire Capital. Under the terms of the agreement, Aspire Capital made an initial investment of US$500,000 to purchase APTO common shares at US $1.40 per share on October 31, 2017. In addition, Aspire Capital has committed to purchase up to an additional US$15.0 million of APTO common shares, at Aptose’s request from time to time during a 30 month period beginning on the effective date of a registration statement related to the transaction, and at prices based on the market price at the time of each sale. Under terms of the agreement, the Company also issued 321,429 common shares to Aspire Capital as consideration for Aspire Capital entering into the Purchase Agreement.

On November 14, 2017 AmpliPhi Biosciences Corporation (NYSE American: APHB), a clinical-stage biotechnology company focused on the development of therapies for antibiotic-resistant infections using bacteriophage technology, reported financial results for the third quarter ended September 30, 2017 (Press release, AmpliPhi Biosciences, NOV 14, 2017, View Source [SID1234522059]).

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"I’m excited to report on the progress AmpliPhi made in the third quarter of 2017," said Paul C. Grint, M.D., CEO of AmpliPhi Biosciences. "We have dosed six patients suffering from life-threatening infections under expanded access programs at two major teaching hospitals and remain on track to meet our previously announced goal of treating ten patients by year end. We have also continued our engagement with the scientific community to increase awareness of phage-based therapies as a promising technology to combat increasing antibiotic resistance. Looking forward, we expect to be well-positioned to treat up to an additional twenty patients by the end of first half of 2018, as we collect the data to prioritize lead indications for potential Phase 2 and/or registrational studies, while working with the FDA on a potential pathway to registration."

Third Quarter 2017 and Recent Business Highlights

Administered first-in-human intravenous treatment of AB-SA01 for a patient suffering from a life-threatening Staphylococcus aureus (S. aureus) infection of the heart (endocarditis). AB-SA01 was administered intravenously to the patient over two weeks and was well tolerated.
Announced the publication of preclinical data demonstrating the activity of AB-PA01 in reducing biofilms. The paper, titled "Activity of Bacteriophages in Removing Biofilms of Pseudomonas aeruginosa Isolates from Chronic Rhinosinusitis Patients," was published in the journal Frontiers in Cellular and Infection Microbiology in September 2017.
Announced publication of a case study detailing the successful treatment of a critically ill patient with a multidrug-resistant (MDR) Acinetobacter baumannii (A. baumannii) infection in the journal Antimicrobial Agents and Chemotherapy in August 2017.
Received a Research and Development (R&D) Tax Incentive cash rebate of USD $2.0 million from the Australian Tax Office based on the company’s R&D spending in Australia during 2016.
Third Quarter 2017 Financial Results

Cash and cash equivalents as of September 30, 2017 totaled $7.7 million. AmpliPhi anticipates that its current financial resources will provide sufficient cash to fund operations until mid-2018.
R&D expenses for the quarter ended September 30, 2017 totaled a net benefit of $0.8 million compared to an expense of $1.7 million for the same period of 2016. The decrease reflects receipt during the quarter of approximately $2.0 million from the Australian tax authority. R&D expenses, excluding any benefit from tax incentive payments, for the three months ended September 30, 2017 and 2016 were $1.2 million and $1.7 million, respectively. The decrease of $0.5 million was primarily attributable to a decrease in professional and consulting fees as well as decreased clinical expenses.
R&D expenses for the nine months ended September 30, 2017 were $1.8 million, net of approximately $2.0 million of tax incentive payments received from the Australian tax authority. There were no similar tax incentive payments recognized during the same period of the prior year. R&D expenses, excluding any benefit from tax incentive payments, for the nine months ended September 30, 2017 and 2016 were $3.8 million and $4.9 million, respectively. The decrease of $1.1 million was primarily related to decreases in consulting fees, professional recruitment fees and clinical expenses.
General and administrative (G&A) expenses for the quarter ended September 30, 2017 totaled $1.6 million compared to $1.8 million for the same period of 2016. G&A expenses for the nine months ended September 30, 2017 were $6.3 million compared to $6.9 million for the same period of 2016.
Net cash used in operating activities for the nine months ended September 30, 2017 was $6.8 million, as compared to $9.1 million for the nine months ended September 30, 2016.
There were 9.5 million shares of common stock outstanding as of November 8, 2017.
Conference Call and Webcast

AmpliPhi will hold a conference call today beginning at 4:30 p.m. Eastern time (1:30 p.m. Pacific time). The conference call dial-in number is (866) 652-5200 for domestic callers and (412) 317-6060 for international callers, and the passcode is 10114360. A live webcast of the call will be available on the Investor Relations section of www.ampliphibio.com.

A recording of the call will be available for 48 hours beginning approximately two hours after the completion of the call by dialing (877) 344-7529 for domestic callers and (412) 317-0088 for international callers. Please use passcode 10114360 to access the recording. A webcast replay will be available on the Investor Relations section of www.ampliphibio.com for 30 days, beginning approximately two hours after the completion of the call.

On November 14, 2017 Cellular Biomedicine Group, Inc. presents the Company’s presentation on November 14, 2017 at the Biotech and Money Investival Showcase held in London (Presentation, Cellular Biomedicine Group, NOV 14, 2017, View Source [SID1234522050]).

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On November 14, 2017 NewLink Genetics Corporation (NASDAQ:NLNK) reported the appointment of Eugene P. Kennedy, M.D., FACS, to the role of Chief Medical Officer. He will be responsible for leading all clinical development, medical affairs and related functions. Dr. Kennedy’s previous role at NewLink Genetics was Vice President of Clinical and Medical Affairs (Press release, NewLink Genetics, NOV 14, 2017, View Source [SID1234522048]).

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"Gene’s considerable experience in clinical trial design and implementation has made him an invaluable team member at NewLink," said Charles J. Link, Jr., MD, Chairman, Chief Executive Officer and Chief Scientific Officer. "We are glad he has taken on this new responsibility and know he will continue to contribute the critical expertise we need as we commence with enrollment of our pivotal trial for patients with advanced melanoma."

Dr. Kennedy joined NewLink in 2014 from Thomas Jefferson University in Philadelphia, PA where he served as Associate Professor of Surgery and held leadership positions as Chief of the Section of Pancreaticobiliary Surgery and Co-Director of the Jefferson Pancreas, Biliary, and Related Cancers Center. Previously, Dr. Kennedy has held faculty positions at the Johns Hopkins Hospital in Baltimore, MD and the Louisiana State University School of Medicine in New Orleans, LA. Dr. Kennedy obtained his undergraduate education at the University of Virginia and received his medical degree from the Medical College of Virginia. He completed a residency and fellowship in Surgery and Surgical Oncology as well as a research fellowship in tumor immunology at the Johns Hopkins Hospital.

On November 14, 2017 Exicure, Inc., a Delaware corporation (the "Company"), the pioneer in gene regulatory and immunotherapeutic drugs utilizing three-dimensional Spherical Nucleic Acid (SNA) constructs, reported its financial results for the third quarter ended September 30, 2017 (Press release, Celsion, NOV 14, 2017, View Source [SID1234522047]).

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"The third quarter was a transformational period for the Company. We continued to advance two SNA drug candidates toward their Phase 1 clinical trials and began our life as a public company," said David Giljohann, Chief Executive Officer of the Company. "We also strengthened our financial position with an initial private placement of approximately $20 million in gross proceeds, followed in the fourth quarter with two subsequent closings, raising an additional approximately $11 million in gross proceeds. This financing will support the clinical development of our lead programs where we have continued our strong progress. By early 2018 we look forward to having three clinical stage drugs."

The Merger and Private Placement

The Merger—On September 27, 2017, we announced the completion of a reverse merger transaction with Max-1 Acquisition Corporation. Immediately after the completion of the merger, Max-1 Acquisition Corporation changed its name to Exicure, Inc. and continued the historical business of Exicure Operating Company, our wholly owned subsidiary. In connection with the merger, we expect to commence trading on the OTC Markets in early 2018.

In accordance with "reverse merger" or "reverse acquisition" accounting treatment, our historical financial statements as of period ends, and for periods ended, prior to the reverse merger will be replaced with the historical financial statements of Exicure Operating Company prior to the reverse merger, in all future filings with the U.S. Securities and Exchange Commission, or SEC. The consolidated financial statements after completion of the reverse merger will include the assets, liabilities and results of operations of the combined company from and after the closing date of the reverse merger.

The Private Placement— On September 26, 2017, we sold 6,767,360 shares of our common stock at a purchase price of $3.00 per share, raising thereby approximately $20.3 million in gross proceeds pursuant to an initial closing of a private placement offering.

Subsequently, on October 27, 2017, we sold 1,878,335 of our common shares at a purchase price of $3.00 per share for gross proceeds of approximately $5.6 million in the first subsequent closing of the private placement. On November 2, 2017, we sold 1,858,501 of our common shares at a purchase price of $3.00 per share for gross proceeds also of approximately $5.6 million in the second subsequent closing of the private placement. In aggregate we have raised approximately $31.5 million of gross proceeds through the private placement.

Third Quarter Business Update

AST-008—In the third quarter of 2017, we received authorization from the Medicines and Healthcare products Regulatory Agency (MHRA) in the United Kingdom to conduct a Phase 1 clinical trial with AST-008 in the United Kingdom. AST-008, an SNA consisting of a TLR9 agonist, is being developed for immuno-oncology applications. While we ultimately plan to clinically advance AST-008 in combination with checkpoint inhibitors, the Phase 1 clinical trial will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single and multiple doses of AST-008 by subcutaneous administration in healthy volunteers. We expect to start the Phase 1 clinical trial during the fourth quarter.

XCUR17—During the third quarter of 2017, we submitted a clinical trial application to conduct a Phase 1 clinical trial for XCUR17 to the Bundesinstitut für Arzneimittel und Medizinprodukte (BfArM), the medical regulatory body in Germany. 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 is a microplaque study in patients with mild to moderate psoriasis. We are currently working with BfArM to finalize the trial protocol.

Purdue Pharma L.P. Collaboration—During the third quarter of 2017, the ongoing Phase 1b clinical trial for AST-005 was completed. AST-005 is an SNA containing TNF antisense oligonucleotides and is intended to be applied in a gel to psoriatic lesions. The Phase 1b clinical trial was conducted in Germany and was intended to evaluate the safety and tolerability of AST-005. We expect topline results from this clinical trial to be available in late 2017.

Third Quarter 2017 Financial Results and Financial Guidance

Cash Position—As of September 30, 2017 Exicure had cash and cash equivalents of $22.9 million compared to $19.6 million as of December 31, 2016. Gross proceeds from the issuance of common stock during the third quarter were $20.3 million with net proceeds of $17.1 million.

Research and development (R&D) expenses—R&D expenses for the third quarter were $3.5 million compared to $2.4 million for the same period in 2016. This increase was due primarily to the costs associated with preparing both AST-008 and XCUR17 for clinical trials.

General and administrative (G&A) expenses—General and administrative expense was $1.3 million for the third quarter compared to $0.8 million for the same period in 2016. This increase in expenses was driven primarily by an increase in non-cash stock compensation costs and certain costs incurred in connection with our merger and private placement.

Net Loss—Net loss for the third quarter was $2.3 million compared to a net loss of $3.5 million for the same period in 2016. The decrease in net loss was driven principally by earning $2.5 million in collaboration revenue attributable to our collaboration with Purdue compared to no revenue for the same period in 2016. The increase in revenue was offset by the increase in R&D expenses described above.

Guidance—We believe that our cash and cash equivalents, as of the date of this press release, are sufficient to fund our current operating plans into 2019.