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.

On November 14, 2017 Aptevo Therapeutics Inc. (Nasdaq:APVO), a biotechnology company focused on developing novel oncology and hematology therapeutics, reported that management will be presenting at the 29th Annual Piper Jaffray Healthcare Conference on Tuesday, November 28, 2017 at 4:00 pm ET at the Lotte New York Palace Hotel (Press release, Aptevo Therapeutics, NOV 14, 2017, View Source;p=RssLanding&cat=news&id=2316919 [SID1234522070]).

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A live audio webcast of the presentation can be accessed at www.aptevotherapeutics.com under the ‘Investors’ section of the website, under ‘Events.’ An audio reply of the webcast will be available approximately 2 hours following the live webcast.

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 Purdue Pharma L.P. reported completion of significant oncology related investments as part of its ongoing efforts to diversify its scientific research into areas of high unmet medical need (Press release, Purdue Pharma, NOV 14, 2017, View Source [SID1234527505]). Through these investments, executed over a multi-year period and capped recently in 2017, Purdue is establishing a portfolio of drug candidates with the potential to deliver new cancer therapies to patients within the next five years.

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"With this formal entry into oncology research and development, Purdue is evolving as a company and renewing its foundational commitment to bring important new medicines to patients and physicians who need them," said Craig Landau, president and CEO, Purdue Pharma. "We are excited by the opportunity and the potential to make meaningful contributions to the field of cancer medicine."

In assembling this portfolio, Purdue now has four drug candidates in development for multiple cancer types:

EDO-S7.1, a novel topoisomerase inhibitor, is designed to work by metabolizing into its active form through enzymes in the gastrointestinal tract that are particularly active in cancer cells. In an interim analysis of a phase 2 trial in patients with therapy refractory advanced biliary tract cancers, the trial met the primary endpoint of rate of disease control (DCR) after first stage. In the trial, commonly observed drug related adverse events were: myelosuppression (including grade 3-4 neutropenia and thrombocytopenia), infection, alopecia, fatigue, nausea, and abdominal pain.1
EDO-S101, also known as tinostamustine, is a novel, first-in-class alkylating deacetylase inhibitor (AK-DACi) compound currently advancing through phase 1 human trials. Preclinical studies suggest that tinostamustine delivers both alkylating activity and pan-histone deacetylase (HDAC) inhibition to simultaneously damage DNA and block damage repair in cancer cells. The development programs for this drug candidate are investigating its potential utility in both solid and hematologic tumors.
EDO-B776 and EDO-B278 are two late pre-clinical stage antibody-drug conjugates. EDO-B776 is being studied for its potential to target the cancer antigen 125 (CA-125) in ovarian cancer. EDO-B278 is an antibody-drug conjugate targeting the human tissue factor and is in development for various solid tumors.
Research on these drug candidates is being directed on behalf of Purdue by Mundipharma EDO GmbH, part of the Mundipharma network of independent associated companies.

"We are pleased to collaborate with Purdue Pharma on the development of these important compounds," said Dr. Thomas Mehrling, chief executive officer, EDO. "Purdue’s strong experience developing novel treatments will translate well to oncology and we are confident that this will be a successful partnership."

Purdue will also continue to selectively seek additional oncology product assets through licensing and acquisition, and the company will maintain a priority interest in candidates with mechanisms complementary to emerging immuno-oncology based treatment paradigms.

On November 14, 2017 Moleculin Biotech, Inc., (NASDAQ: MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of anti-cancer drug candidates, some of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported its financial results for the third quarter ended September 30, 2017 (Press release, Moleculin, NOV 14, 2017, View Source [SID1234522027]). Additionally, the Company announced potential upcoming milestones and recent corporate developments.

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Management Discussion
Walter Klemp, Chairman and CEO of Moleculin, commented, "We continue to make progress in advancing our drug candidates through their various regulatory approval pathways. We accomplished a number of important milestones on Annamycin, one of our lead cancer drug candidates. During the quarter, the Food and Drug Administration ("FDA") allowed our Investigational New Drug application ("IND") to proceed, meaning we could begin clinical trials of Annamycin for the treatment of relapsed or refractory Acute Myeloid Leukemia ("AML"). In preclinical tests, Annamycin has shown little to no cardiotoxicity and an ability to avoid the multidrug resistance mechanisms that often defeat the current first-line therapies for AML. For that reason, we believe Annamycin has the potential to become the first second-line therapy suitable for a majority of relapsed or refractory AML patients. Because first-line therapies fail in a significant majority of AML patients, we believe Annamycin may give new hope to those who have run out of treatment options.

We recently announced that 14 qualified cancer clinics have asked to participate in in the Phase I/II clinical trial that is the subject of our IND, and that will study Annamycin for the treatment of relapsed or refractory AML. Seven of the interested sites are in Poland which we anticipate will give us access to a higher percentage of patients who are ‘treatment naïve’ patients. Study subjects who have not received any experimental therapies before entering our trial are important because we believe such patients may be in a better condition to respond positively to the Annamycin therapy. We have requested permission from the Polish government and we expect to receive authorization before the end of the year, at which point we plan to immediately begin enrolling patients.
We have several other drug candidates that are making progress in their respective development programs. We recently announced that the MD Anderson Cancer Center ("MD Anderson") has submitted an IND request to conduct a physician-sponsored Phase I clinical trial of WP1066 in patients with glioblastoma and melanoma that has metastasized to the brain. Preclinical testing suggests that WP1066 may increase the natural immune response to tumors, suppressing of tumor cell proliferation and tumor cell survival. If that IND is approved, we will then have two "clinical stage" programs under way.

During the quarter, we also announced that we have begun efforts to seek a Clinical Trial Authorization ("CTA") in Poland for the study of WP1220 (part of our WP1066 Portfolio) in the treatment of Cutaneous T-Cell Lymphoma. Additionally, we announced several collaborations, including an agreement with Mayo Clinic to study WP1066 for the treatment of a rare form of childhood brain tumor and two projects with the University of Bergen in Norway, one to conduct research on the ability of WP1122 to inhibit glycolysis and limit tumor growth and the other to dig more deeply into the potential ability of WP1066 to stimulate anti-cancer immune response. In addition, we announced research findings suggesting that one of our WP1122 analogs could be well suited for the treatment of pancreatic cancer."

"We believe one of the key strengths of Moleculin is the breadth and diversity of our drug development portfolio. We now have active development programs with four drug candidates representing three substantially different approaches to treating cancer."

Jonathan Foster, Chief Financial Officer of Moleculin, stated, "I am pleased we have been able to raise $8.7 million during the nine-month period ended September 30, 2017. As a result, we finished the third quarter with $8.7 million in cash and cash equivalents and no debt, compared to $5.0 million at December 31, 2016. During the third quarter we had a cash burn rate of approximately $1.5 million, but anticipate the cash burn rate will increase as we move our cancer drugs into clinical trials. We are highly focused on running our business in an efficient manner, prudently managing our cash burn rate and maintaining a solid cash position."

Third Quarter Highlights and Recent Corporate Developments
Moleculin Appoints Dr. Sandra Silberman as Chief Medical Officer – New Products – November 8, 2017, the Company announced the appointment of Dr. Sandra Silberman as Chief Medical Officer ("CMO") in charge of New Products.
Moleculin Announces MD Anderson has Filed an IND with the FDA on its Drug WP1066 for the Treatment of Brain Tumors – November 1, 2017, the Company announced that responses have been submitted to FDA requests for additional information relating to the physician-sponsored IND application to study WP1066 as a potential treatment for brain tumors.

Moleculin Requests Authorization from the Polish Government to Advance Annamycin – October 24, 2017, the Company announced that it has submitted its request for CTA in Poland which, if allowed, will enable a clinical trial to study Annamycin for the treatment of relapsed or refractory AML in Poland. This will be in addition to the previously announced allowance of Moleculin’s IND filing with the FDA.

Moleculin Announces 14 Qualified Clinical Sites Requesting Participation in Annamycin Trial – October 18, 2017, the Company announced that 14 qualified cancer clinics have requested to participate in its clinical trial to study Annamycin for the treatment of relapsed or refractory AML.

Moleculin Announces Strategic Collaboration to Develop Immune Stimulation Drug – October 11, 2017, the Company announced that it has entered into an agreement to collaborate with the University of Bergen to expand research on WP1066 and early indications of a possible dual ability to increase immune system response to tumors while also suppressing tumor cell proliferation tumor cell and survival.

Moleculin Signs Agreement with First Hospital for Annamycin Trials – October 3, 2017, the Company announced it has entered into an agreement with the first of several hospitals desiring to become treatment sites for its clinical trial to study Annamycin for the treatment of relapsed or refractory AML.

Moleculin Announces FDA Approval of Annamycin IND – September 26, 2017, the Company announced that the FDA has allowed Moleculin’s IND for the study of Annamycin in relapsed or refractory AML to proceed. This allows Moleculin to begin clinical trials of Annamycin in the US.

Moleculin Engages CRO to Begin Clinical Trials of WP1220 for the Treatment of Cutaneous T-Cell Lymphoma – September 13, 2017, the Company announced it has engaged contract research organization ("CRO") Bioscience SA ("Bioscience") to prepare for a proof-of-concept clinical trial in Poland to study Moleculin’s drug candidate WP1220 for the treatment of cutaneous T-cell lymphoma.

Moleculin to Collaborate on Combining its WP1122 Brain Cancer Drug Candidate with Roche’s Drug Avastin – September 6, 2017, the Company announced it has entered into a collaborative agreement with the University of Bergen in Norway to study WP1122 in combination with the drug Avastin (bevacizumab) made by Roche Pharma. Roche Pharma is not a party to the collaborative agreement.

Moleculin Begins Clinical Testing Site Development Efforts in Poland; Selects Bioscience SA as Polish CRO – August 3, 2017, the Company announced it has selected Bioscience SA, a Polish CRO to begin identifying and preparing clinical testing sites in Poland for Annamycin, the Company’s drug candidate for the treatment of relapsed or refractory AML.
Moleculin Strengthens Board of Directors with Appointment of John M. Climaco – July 27, 2017, the Company announced the appointment of John M. Climaco as an independent member of the Company’s Board of Directors, effective July 24, 2017 to fill a board vacancy.

Moleculin Signs Agreement with MD Anderson Cancer Center for Leukemia Drug, Annamycin – July 18, 2017, the Company announced it has signed a new technology license agreement with MD Anderson based on new patent applications Moleculin intends to file relating to its drug Annamycin for the treatment of relapsed or refractory AML.
Moleculin Appoints Lead European Principal Investigator for Planned Annamycin Clinical Trial – July 6, 2017, the Company announced it has appointed Dr. Lidia Gil of Poznan University of Medical Sciences in Poznan, Poland to be the lead European Principal Investigator for its upcoming planned Phase I/II clinical trial of Annamycin for the treatment of relapsed or refractory AML, subject to receipt of Polish approval of the CTA.

Third Quarter Results
Research and Development Expense. Research and development (R&D) expense was $1.1 million and $0.5 million for the three months ended September 30, 2017 and 2016, respectively. The increase of approximately $0.6 million mainly represents an increase of approximately: $0.1 million related to an increase in R&D associated headcount costs, $0.1 million for sponsored research and related expenses; and, approximately $0.4 million associated with developing and testing drug product as we prepare our IND for Annamycin and for the related clinical trials.
General and Administrative Expense. General and administrative expense was $1.3 million and $0.9 million for the three months ended September 30, 2017 and 2016, respectively. The expense increase of approximately $0.4 million was mainly attributable to the increase in headcount and associated payroll costs of $0.2 million, $0.3 million of stock based compensation; and, approximately $0.1 million in legal, accounting, consulting, and other professional expenses. This was offset by a reduction in public listing expenses of $0.2 million.
Net Loss. The net loss for the three months ended September 30, 2017 was $2.9 million which included non-cash income of $0.5 million related to a gain recognized on the expiration of warrants. The net loss also included additional noncash charges for $0.5 million for stock based compensation and other stock based expenses.
Liquidity and Capital Resources

As of September 30, 2017, we had $8.7 million in cash and cash equivalents compared to $5.0 million at December 31, 2016. In February 2017, we completed a public offering of our common stock and warrants, pursuant to which we received approximately $4.5 million in net proceeds, after deducting underwriting discounts and commissions and estimated offering expenses. Additionally, through September 30, 2017, $3.3 million in cash was received from the exercise of warrants issued in our February public offering and $0.4 million from the sale of common stock in our ATM offering. Cash used in operations was $4.9 million for the nine months ended September 30, 2017. This increase over the prior year of $2.3 million was mainly due to to an increase in headcount and general company activity as it prepared its IND for Annamycin and readied for the related, upcoming trials. We believe that our existing cash and cash equivalents as of September 30, 2017 and cash generated already in the third quarter will be sufficient to fund our planned operations into the third quarter of 2018. Such plans are subject to change depending on clinical enrollment progress and use of drug product.