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Audentes Therapeutics Reports Third Quarter 2017 Financial Results and Provides Corporate Update

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.

NewLink Genetics Appoints Eugene Kennedy, M.D. as Chief Medical Officer

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 [SID1234522068]).

"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.

Molecular Templates, Inc. Reports Third Quarter 2017 Financial Results

On November 14, 2017 Molecular Templates, Inc. (Nasdaq:MTEM), a clinical-stage oncology company focused on the discovery and development of the company’s proprietary engineered toxin bodies (ETBs), which are differentiated, targeted, biologic therapeutics for cancer, reported financial results for the third quarter of 2017 (Press release, Molecular Templates, NOV 14, 2017, View Source [SID1234522067]). As of September 30, 2017, cash and cash equivalents totaled $68.2 million.

"The completion of our merger with Threshold Pharmaceuticals as well as the $60M in financings in the third quarter mark a significant milestone for Molecular Templates," said Molecular Templates’ Chief Executive Officer and Chief Scientific Officer, Eric Poma, Ph.D. "The merger and financings put us in a strong position to continue to execute on the development of our lead program, MT-3724, in aggressive lymphomas as well as make strides toward the clinic in 2018 with our next generation of ETBs currently in preclinical development. Additionally, we are excited about our ongoing research collaboration with Takeda Pharmaceuticals as we look to expand the application of our ETB technology."

Company Highlights and Upcoming Milestones

Corporate
On August 1, 2017, we successfully completed the reverse merger with Threshold Pharmaceuticals, Inc. (previously Nasdaq ticker: THLD) and concurrently changed the Company’s name to Molecular Templates, Inc. (new Nasdaq ticker: MTEM).

On August 1, 2017, we closed an equity financing for $40 million; as well as a private placement of $20 million to Millennium Pharmaceuticals, Inc., a wholly owned subsidiary of Takeda Pharmaceutical Company Ltd.

MT-3724
On October 24, 2017, we announced the identification of the maximum tolerated dose (MTD) for MT-3724 and the initiation of and first patient dosed in an expansion cohort, as part of the second portion of the ongoing Phase I study focused on relapsed/refractory diffuse large B-cell lymphoma (DLBCL) patients. The expansion cohort is expected to better define the overall response rate to MT-3724 in heavily pre-treated DLBCL patients. Initial results of this expansion cohort are expected to be announced in 1H18.

Molecular anticipates initiating a Phase II monotherapy study in relapsed and refractory DLBCL patients in 2018.
Takeda Collaboration

Expanded Takeda collaboration in June 2017 with Multi-Target Research and Licensing Collaboration Agreement to develop next-generation oncology therapies, including stock purchase.

MT-4019
Continued development of MT-4019, an ETB candidate that is designed to target CD38-expressing myeloma cancer cells.

Plan to submit an IND application to the FDA in mid-2018 to initiate a Phase I clinical trial in the United States.
Research

Several other ETB candidates in pre-clinical development targeting both solid and hematological cancers where the differentiated mechanism of action innate to ETBs, ribosome inactivation, could play a significant role in treating cancer.

Financial Results
The net loss attributable to common shareholders for the third quarter was $(11.1) million, or $(0.62) per basic and diluted share. This is compared to a net loss attributable to common shareholders for the same period in 2016, of $(3.6) million, or $(11.89) per basic and diluted share. As of September 30, 2017, cash and cash equivalents totaled $68.2 million, which includes $11.2 million received from the merger with Threshold Pharmaceuticals in August 2017 and the receipt of $60 million for two financings that closed on the same day.
Revenues for the third quarter of 2017 were $0.6 million, compared to no revenues during the same period in 2016. Revenues for the third quarter of 2017 related to research and development revenues from our collaboration with Takeda.

Total research and development (R&D) expenses for the third quarter of 2017 were $2.5 million, compared with $2.3 million for the same period in 2016. The $0.2 million increase in R&D expenses in the third quarter of 2017, compared with the same period in 2016, was primarily due to severance benefits related to the merger with Threshold.

Total general and administrative (G&A) expenses for the third quarter of 2017 were $4.0 million, compared with $0.8 million for the same period in 2016. The $3.2 million increase in G&A expenses in the third quarter of 2017, compared with the same period in 2016, was primarily due to costs associated with the Merger and being a publicly traded company.

Revenues for the nine months ended September 30, 2017 were $2.6 million, compared to $1.5 million for the same period in 2016. Revenues for the nine months ended September 30, 2017 were primarily comprised of research and development revenues from our collaboration with Takeda. Revenues for the same period in 2016 comprised of grant revenue from the Cancer Prevention & Research Institute of Texas ("CPRIT").

Total R&D expenses for the nine months ended September 30, 2017 were $4.8 million, compared to $7.2 million for the same period in 2016. The $2.4 million decrease in R&D expenses for the nine months ended September 30, 2017, compared the same period in 2016, was primarily due to decreased spending for outsourced preclinical costs.
Total G&A expenses for the nine months ended September 30, 2017 were $8.2 million, compared to $2.6 million for the same period in 2016. The $5.6 million increase in G&A spending for the nine months ended September 30, 2017, compared to the same period in 2016, was primarily due to costs associated with the Merger and being a publicly traded company.

The net loss attributable to common shareholders for the nine months ended September 30, 2017 was $(17.2) million, or $(2.75) per basic and diluted share, compared to a net loss attributable to common shareholders of $(9.6) million or $(32.01) per basic and diluted share, for the same period in 2016.

Ligand Presents Analyst Day Presentation

On November 14, 2017 Ligand Presents Analyst Day Presentation (Presentation, Ligand, NOV 14, 2017, View Source [SID1234522066]).

GTx Provides Corporate Update and Reports Third Quarter 2017 Financial Results

On November 14, 2017 GTx, Inc. (Nasdaq:GTXI) reported financial results for the third quarter of 2017 and highlighted recent accomplishments and upcoming milestones (Press release, GTx, NOV 14, 2017, View Source;p=RssLanding&cat=news&id=2316925 [SID1234522065]).

"During the quarter, we achieved a key milestone for the company when we reported positive results from our first clinical trial in stress urinary incontinence," said Robert J. Wills, Ph.D., Executive Chairman of GTx. "Remarkably, 18 out of 18 women who received enobosarm for 12 weeks responded. These responses also appear to be durable, lasting months after dosing. These exciting results provided the basis for our recently initiated, placebo-controlled clinical trial of enobosarm for the treatment of SUI."

"Recent data suggests 50 percent of women report SUI, for which there are no FDA-approved pharmaceutical therapies. An orally-available therapy would offer numerous advantages over existing treatments including surgery, and would provide a significant new option that many women would choose in order to address this medical condition," said Kenneth M. Peters, M.D., Chairman of Urology, Oakland University William Beaumont School of Medicine and the principal investigator in the trial.

Third Quarter 2017 Clinical Highlights and Anticipated Milestones

Stress Urinary Incontinence (SUI):

Enobosarm, a Selective Androgen Receptor Modulator (SARM), is being evaluated in Phase 2 clinical development for SUI, the Company’s lead indication. Recent important milestones are summarized as follows:

Reported positive results from the Phase 2 proof-of-concept (POC) clinical trial of enobosarm 3 mg administered orally in post-menopausal women with SUI. With the inclusion of the final patient completing treatment in the POC clinical trial, data from the 18 evaluable patients completing the required 12 weeks of daily treatment showed a clinically meaningful reduction (50 percent or greater) in stress leaks per day, compared to baseline. The mean decrease in stress leaks per day was 81 percent overall (5.17 mean leaks/day at baseline to 1.0 mean leaks/day at 12 weeks).
Patients are being followed for an additional 28 weeks post-treatment to assess the durability of treatment effect. Durability of response for patients who completed the 28-week observation phase has resulted in a 41 to 100 percent reduction in stress leaks/day from baseline (N=6). For those patients who have not completed the 28-week observation phase, the durability of response, measured beginning 4 weeks post dosing, continues to be sustained.
Highlighted the Phase 2 POC results at the International Continence Society (ICS) annual meeting in a poster entitled, "Kegels in a Bottle: Preliminary Results of a Selective Androgen Receptor Modulator (GTx-024) for the Treatment of Stress Urinary Incontinence in Post-Menopausal Women", which subsequently was voted best poster for the conference.

Initiated a second clinical trial, Assessing Enobosarm for Stress Urinary Incontinence Disorder (ASTRID): a randomized, double-blinded, placebo-controlled, Phase 2 trial to assess the efficacy and safety of two doses of enobosarm (1 mg and 3 mg) administered orally in post-menopausal woman with SUI compared to placebo. The primary endpoint of the trial is the percentage of patients with at least a 50 percent reduction in mean leaks/day, compared to baseline. This trial is expected to enroll approximately 400 patients across 70 clinical sites in the U.S. Top-line results are expected to be available by the end of 2018.
Breast Cancer:

Enobosarm is also being evaluated as a hormonal therapy for women with estrogen receptor positive (ER+) and androgen receptor positive (AR+) breast cancer in a Phase 2 clinical trial for this advanced breast cancer population. As reported earlier for the 9 mg cohort, the Phase 2 trial pre-specified threshold for success, clinical benefit response (CBR), was attained and therefore met the primary efficacy endpoint. In addition, the 18 mg cohort has also met the primary efficacy endpoint. The trial has now completed enrollment of the predefined number of evaluable patients in both dosage arms with at least 44 patients in each of two cohorts receiving 9 mg or 18 mg daily doses of enobosarm.

In the 9 mg cohort, following 24 weeks of treatment, a total of 14 patients achieved a CBR out of 49 evaluable patients confirmed as AR positive (28.6%), with two patients achieving a partial response and 12 reporting stable disease. Currently, four patients in this cohort remain on study. In the 18 mg cohort, with 48 evaluable patients, 12 patients achieved a CBR (25%) at 24 weeks with one patient demonstrating a partial response and 11 patients reporting stable disease. Three patients remain on study in the 18 mg cohort. Both doses of enobosarm appear to be safe and generally well tolerated. A complete summary of the study results will be submitted for presentation or publication in 2018.

Although both the 9 mg and 18 mg cohorts met the primary efficacy endpoint in the Phase 2 clinical trial, after evaluating the drug development environment for breast cancer, where treatment paradigms are shifting to immunotherapies and/or combination therapies, the Company has decided that the time and cost of conducting the necessary clinical trials for approval in this indication do not warrant further development of enobosarm in this indication at this time.

Duchenne Muscular Dystrophy (DMD):

SARMs have also been evaluated in preclinical models of DMD, in which GTx SARMs have increased lean muscle mass and physical function. The Company is pursuing a potential strategic collaboration with biopharma companies experienced in orphan drug development to continue the development of a SARM for the treatment of DMD.

Prostate Cancer:

The Company has a Selective Androgen Receptor Degrader (SARD) preclinical program to evaluate its novel SARD technology in castration-resistant prostate cancer (CRPC). The Company has ongoing mechanistic preclinical studies designed to select the most appropriate compound to advance into a first-in-human clinical trial.

Third Quarter 2017 Corporate Highlights and Financial Results

During the quarter, GTx raised net proceeds of $45.6 million in a private placement of its common stock and warrants to purchase its common stock. GTx sold 5,483,320 immediately separable units, comprised of an aggregate of 5,483,320 newly-issued shares of common stock and warrants to purchase up to 3,289,988 additional shares of common stock. Both the common stock and warrants have been registered for resale with the Securities and Exchange Commission.

As of September 30, 2017, cash and short-term investments were $53.6 million compared to $21.9 million at December 31, 2016.

Research and development expenses for the quarter ended September 30, 2017 were $5.9 million compared to $4.6 million for the same period of 2016.

General and administrative expenses for the quarter ended September 30, 2017 were $2.6 million compared to $2.3 million for the same period of 2016.

Net loss for the three months ended September 30, 2017 was $8.5 million compared to a net loss of $6.9 million for the same period in 2016.

Net loss for the nine months ended September 30, 2017 was $21.2 million compared to a net loss of $10.9 million for the same period in 2016. The nine months ended September 30, 2016 included a non-cash gain of $8.2 million due to the change in fair value of the Company’s warrant liability. During the first quarter of 2016, the Company modified its outstanding warrants with no further adjustment to the fair value of these warrants being required.
GTx had approximately 21.5 million shares of common stock outstanding as of September 30, 2017. Additionally, there are warrants outstanding to purchase approximately 6.4 million shares of GTx common stock at an exercise price of $8.50 per share and approximately 3.3 million shares of GTx common stock at an exercise price of $9.02.