On November 14, 2017 Audentes Therapeutics, Inc. (Nasdaq: BOLD), a biotechnology company focused on developing and commercializing gene therapy products for patients living with serious, life-threatening rare diseases, reported its financial results for the quarter ended September 30, 2017, and provided an update on the company’s recent achievements and anticipated upcoming milestones (Press release, Audentes Therapeutics, NOV 14, 2017, View Source;p=RssLanding&cat=news&id=2316918 [SID1234522069]).

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"I am pleased with the excellent progress we have made to advance our pipeline toward key value inflection milestones, including the recent completion of dosing for the first cohort in ASPIRO, the Phase 1 / 2 clinical study of AT132 for the treatment of XLMTM," stated Matthew R. Patterson, President and Chief Executive Officer. "While we are only weeks into the study, we are encouraged by the progress to date and look forward to sharing preliminary safety and efficacy data from the first cohort of patients in early January 2018. This will be the first of many catalysts over the coming months as we execute on our ambitious goal of creating the world’s leading gene therapy company focused on developing a multi-product pipeline to treat serious, life-threatening rare diseases with high unmet medical need."

Recent Achievements & Upcoming Key Events

AT132 for X-Linked Myotubular Myopathy:
Completed dosing of the first cohort of patients in ASPIRO, the Phase 1 / 2 clinical study of AT132. To date, AT132 has been well-tolerated by all patients with no significant treatment-related safety signals.
Announced interim results from INCEPTUS, a prospective natural history run-in study in patients with XLMTM. The primary objectives of INCEPTUS are to characterize the clinical condition of children with XLMTM, identify subjects for potential enrollment in ASPIRO, and serve as a longitudinal baseline and with-in patient control for ASPIRO. Preliminary results from INCEPTUS confirm the significant neuromuscular and respiratory deficits experienced by XLMTM patients, and provide insight into the relevance and sensitivity of assessments used in the ASPIRO study.
Received approval for the Clinical Trial Authorisation (CTA) application for AT132 from the United Kingdom’s (U.K.) Medicines and Healthcare Products Regulatory Agency (MHRA), and anticipate initiation of U.K. enrollment in the first quarter of 2018 for ASPIRO.
Plan to report preliminary clinical data from ASPIRO in early January 2018
AT342 for Crigler-Najjar:
Plan to discuss initial results of LUSTRO, the prospective natural history run-in study being conducted in patients with Crigler-Najjar Syndrome, on the third quarter 2017 earnings call
Plan to dose first patient in VALENS, the Phase 1 / 2 clinical study of AT342, in the first quarter of 2018, and to report preliminary data in the second quarter of 2018
Received approval for the CTA application for AT342 from the MHRA, and anticipate initiation of U.K. enrollment in the first quarter of 2018 for VALENS
AT982 for Pompe Disease:
Conducting a comprehensive in vivo construct selection and dose-ranging study evaluating a range of AAV8 and AAV9 vector constructs designed to express GAA selectively within tissues relevant to Pompe disease, including skeletal muscle, motor neurons and liver. Candidate vectors are being evaluated across a broad range of neuromuscular function and biochemical endpoints at multiple dose levels.
Plan to select optimal construct and file IND in the first half of 2018
AT307 for CASQ2-CPVT:
Completed successful pre-IND/CTA meetings with both the U.S. Food and Drug Administration (FDA) and MHRA, and have incorporated input from both agencies into IND filing plans
Expect to file IND in the first quarter of 2018
Appointed Fulvio Mavilio, Ph.D. as Vice President Scientific Affairs, Europe. Dr. Mavilio is an internationally recognized scientific leader who has made important contributions to the field of molecular genetics and gene therapy for rare diseases.
Third Quarter 2017 Financial Results

Cash Position: As of September 30, 2017, Audentes had cash, cash equivalents and short-term investments of approximately $156.0 million.
R&D Expenses: Research and development expenses were $20.9 million for the third quarter of 2017 compared to $12.5 million for the same period in 2016, an increase of $8.4 million. The increase was primarily due to increased research and development expenses for our AT132 and AT307 programs, increases in our research and development headcount and higher facility costs as we made additional investments in our manufacturing and research facilities. For the nine months ended September 30, 2017, research and development expenses were $54.2 million compared to $32.2 million for the same period in 2016.
General and Administrative: General and administrative expenses were $4.3 million for the third quarter of 2017 compared to $2.9 million for the same period in 2016, an increase of $1.4 million. The increase was primarily due to increased G&A headcount and increases in other G&A expenses related to regulatory compliance and operations as a public company. For the nine months ended September 30, 2017, general and administrative expenses were $12.1 million compared to $8.0 million for the same period in 2016.
Net Loss: Net loss was $25.0 million for the third quarter of 2017, compared to a net loss of $15.4 million for the same period in 2016. For the nine months ended September 30, 2017, our net loss was $65.9 million as compared to $40.0 million for the same period in 2016.
Conference Call
At 4:30 p.m. Eastern Time today, Audentes management will host a conference call and a simultaneous webcast to discuss its third quarter 2017 financial results and provide a corporate update. To access a live webcast of the conference call, please visit the Investor and Media page of the Audentes website at www.audentestx.com. Alternatively, please call 1-833-659-8620 (U.S.) or 1-409-767-9247 (international) and dial the conference ID 2599735 to access the call.

A replay of the webcast will be available on the Audentes website for approximately 30 days.

Molecular Templates has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Molecular Templates, 2017, NOV 14, 2017, View Source [SID1234522056]).

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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 Seres Therapeutics, Inc. (NASDAQ:MCRB), The University of Texas MD Anderson Cancer Center (MD Anderson), and the Parker Institute for Cancer Immunotherapy (Parker Institute) reported a collaboration to evaluate the potential of Seres’ microbiome therapies to improve the outcomes of cancer patients treated with currently-available immunotherapy (Press release, Seres Therapeutics, NOV 14, 2017, View Source [SID1234530894]).

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The collaborators plan to initiate a randomized, placebo-controlled clinical study at MD Anderson, sponsored by the Parker Institute, in patients with advanced metastatic melanoma. The clinical trial will evaluate the impact of an anti-PD-1 checkpoint inhibitor with adjunctive microbiome therapy on patient outcomes. Seres is developing SER-401, a preclinical stage oral microbiome therapy comprising a rationally-designed consortium of live bacteria, to improve the efficacy and safety of immunotherapy.

Published studies provide preclinical and clinical evidence demonstrating that the composition of bacteria in the gastrointestinal microbiome may impact response to checkpoint inhibitor therapy.1 On Nov. 2, 2017, Science published research by Jennifer Wargo, M.D. and colleagues from MD Anderson indicating that the composition of the gut microbiome may influence checkpoint inhibitor response in melanoma patients.2 This research also demonstrated that the favorable microbiome properties found in checkpoint inhibitor responder patients are able to be transferred to mice. The results provide support for the clinical study of microbiome therapeutics to augment the clinical benefit of cancer immunotherapy.

Seres also received an exclusive option, with pre-defined financial terms, to license intellectual property rights from MD Anderson related to the use of bacteria in combination with checkpoint inhibitors.

"MD Anderson, and in particular Dr. Wargo’s laboratory, is leading the charge to better understand the microbiome and the response to immune checkpoint inhibitors," said Roger J. Pomerantz, M.D., President, CEO and Chairman of Seres. "We look forward to combining our insights and capabilities with both MD Anderson and the Parker Institute to advance microbiome therapies to augment Immunotherapy in cancer patients toward the clinic, with the ultimate goal of improving outcomes for patients facing life-threatening tumors with significant unmet medical need."

"Immunotherapy has represented an important advance for melanoma and other cancers. However, in the majority of patients, the response is not adequate to durably control disease," said Jennifer Wargo, M.D., Associate Professor of Genomic Medicine and Surgical Oncology at MD Anderson. "Modulation of the microbiome is a promising approach that may improve the therapeutic benefit of checkpoint therapy."

"This collaboration between the Parker Institute, Seres and MD Anderson exemplifies the mission of the Parker Institute for Cancer Immunotherapy to unlock the promise of immunotherapy by rapidly progressing next generation treatments into clinical trials," said Fred Ramsdell, Ph.D., Vice President of Research at the Parker Institute of Cancer Immunotherapy. "If this novel approach is successful at altering the microbiome and more importantly, also leads to better cancer patient responses to immunotherapy, this would mark an important milestone for the entire field."

References

1. Chen C. and Mellman I., Elements of cancer immunotherapy and the cancer-immune set point, Nature, 2017
2. Wargo J. et al., Gut Microbiome Impacts Response to Anti-PD-1 Immunotherapy in Melanoma Patients, Science, 2017