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PRIMA BIOMED COMPLETES RECRUITMENT FOR SECOND PATIENT COHORT IN MELANOMA TRIAL AND WILL PRESENT AT ICI CONFERENCE IN BOSTON

On March 15, 2017 Prima BioMed Ltd (ASX: PRR; NASDAQ: PBMD) ("Prima" or the "Company") reported that the second cohort comprising six patients has now been fully recruited for its TACTI-mel (Two ACTive Immunotherapeutics in melanoma) clinical trial being conducted in Australia (Press release, Prima Biomed, MAR 15, 2017, View Source [SID1234518142]). Patients with unresectable or metastatic melanoma that have had a suboptimal response to KEYTRUDA were dosed with the higher 6 mg dose of IMP321 in combination with KEYTRUDA.

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Prima’s Chief Medical & Scientific Officer, Dr Frédéric Triebel, will be presenting the TACTI-mel clinical trial at the Immune Checkpoint Inhibitors conference at the Sheraton Hotel Boston, Massachusetts, held on March 15-16, 2017.
The presentation will be delivered at 1:30pm on Thursday 16 March, 2017 EDT.
A copy of these presentation slides will be made available on the Prima BioMed website.
Further information on the conference can be found at http://immune-checkpoint.com/about/about

About IMP321
IMP321, a first-in-class Antigen Presenting Cell (APC) activator based on the immune checkpoint LAG-3, represents one of the first proposed active immunotherapy drugs in which the patient’s own immune system is harnessed to respond to tumour antigenic debris created by chemotherapy. As an APC activator IMP321 boosts the network of dendritic cells in the body that can respond to tumour antigens for a better anti-tumour CD8 T cell response.

GTx Provides Corporate Update and Reports Fourth Quarter and Year-End 2016 Financial Results

On March 15, 2017 GTx, Inc. (Nasdaq: GTXI) reported financial results for the fourth quarter and year ended December 31, 2016, and highlighted recent accomplishments and upcoming milestones (Press release, GTx, MAR 15, 2017, View Source [SID1234518139]). The Company has two ongoing clinical trials of enobosarm (GTx-024) in women with advanced breast cancer and one ongoing trial with enobosarm as a potential treatment for stress urinary incontinence (SUI) in postmenopausal women. The Company has identified lead compounds from its selective androgen receptor degrader (SARD) portfolio and has preclinical studies underway that are required prior to initiating a clinical trial in men with castration-resistant prostate cancer (CRPC) during the second half of 2017.

"In 2016, we broadened our clinical development efforts for enobosarm beyond breast cancer with the initiation of our clinical trial in SUI in postmenopausal women. Early results in this study are very promising, and we have added additional clinical sites and expect to receive top-line results from this trial in the third quarter. Later this year, we also expect to initiate a first in human clinical trial of a SARD in men with advanced prostate cancer," said Robert J. Wills, Ph.D., Executive Chairman of GTx. "There is a great deal of interest in our SARD program based on data we have generated that demonstrate our SARD compounds degrade and inhibit multiple forms of the androgen receptor, including AR splice variants, and may therefore potentially treat CRPC in men who are non-responsive to current androgen therapy."

Corporate Highlights and Anticipated Milestones

Enobosarm in Breast Cancer: The Company’s lead product candidate, enobosarm, a selective androgen receptor modulator (SARM), is being developed as a targeted treatment for two advanced breast cancer indications: (i) estrogen receptor positive (ER+) and androgen receptor positive (AR+) breast cancer, and (ii) AR+ triple negative breast cancer (TNBC). For both clinical trials, the primary efficacy endpoint is a determination of clinical benefit (CB), which is defined as a complete response, partial response or stable disease.

ER+/AR+ Breast Cancer: The Company has an ongoing open-label, multi-center Phase 2 clinical trial of enobosarm in women with advanced, ER+, AR+ breast cancer. Patients receive orally-administered enobosarm (9 mg or 18 mg) daily for up to 24 months. The first stage of evaluation was assessed among the first 18 evaluable patients for each cohort. At least 3 of 18 patients per cohort achieved CB at week 24, and the trial has proceeded to the second stage of enrollment. In Stage 2, if at least 9 of 44 evaluable patients achieve CB at week 24, the trial will have successfully demonstrated its primary endpoint, and those patients achieving CB at 24 weeks will be able to continue treatment for a total of up to 24 months. The two dose cohorts in the trial are being treated independently for the purpose of assessing efficacy. To date, in this ongoing clinical trial:

CB has been demonstrated in both the 9 mg and 18 mg dose cohorts in Stage 1 of the trial, allowing both cohorts to advance to Stage 2 of the trial.
A sufficient number of evaluable patients have already demonstrated CB at week 24 in the 9 mg dose group for the study to achieve the pre-specified primary efficacy endpoint in this ongoing clinical trial. Of the 40 patients in the 9 mg dose cohort whose AR status has been confirmed AR+, 10 patients have demonstrated CB at week 24, 23 patients have discontinued either at or prior to week 24, and 7 patients remain on study and have not yet reached week 24. There are another 5 patients who have been enrolled to the 9 mg cohort whose AR status has not yet been confirmed. Of the 10 evaluable patients achieving CB, 2 had a partial response and 8 had stable disease. The majority of adverse events are grade 1 and 2, and the most common adverse events reported (occurring in ≥10% of patients) include nausea (31%), fatigue (18%), and arthralgias (13%). Elevations in transaminases (ALT and AST) during enobosarm treatment were mild with the majority being grade 1 or 2.
An abstract submitted by Dr. Beth Overmoyer, the lead principal investigator for the clinical trial and a medical oncologist with the Dana-Farber Cancer Center Institute, detailing data from Stage 1 of the 9 mg cohort, has been accepted for the "poster walk" at the European Society for Medical Oncology IMPAKT Breast Cancer Conference, which is being held May 4-6, 2017, in Brussels, Belgium.

Enobosarm appears to be safe and generally well tolerated. The independent Safety Monitoring Committee met in December 2016, and recommended that the clinical trial continue as planned. The trial will continue with a daily dose of either enobosarm 9 mg or 18 mg until 44 evaluable patients in each cohort have completed treatment in order to better characterize the CB response. The Company expects to report top-line results from this study in the third quarter of 2017.

AR+ TNBC: The Company also has an ongoing open-label, multi-center Phase 2 clinical trial to evaluate the efficacy and safety of orally-administered enobosarm in up to 55 women with advanced, AR+ TNBC. Patients receive 18 mg of enobosarm once daily for up to 12 months. Stage 1 of the trial is being assessed among the first 21 evaluable patients, and if at least 2 of 21 patients achieve CB at week 16, then the trial can proceed to Stage 2 of enrollment of up to a total of 41 evaluable patients. The primary efficacy objective of the trial is CB response following 16 weeks of treatment in 41 evaluable patients.

The Company expects to have sufficient data from Stage 1 of the trial by the second quarter of 2017 to determine if patient enrollment should continue into Stage 2 of the trial.
SARMs in Non-Oncologic Indications: The Company also is developing SARMs as potential treatments for both stress urinary incontinence (SUI) in postmenopausal women and Duchenne muscular dystrophy (DMD), a rare disease characterized by progressive muscle degeneration and weakness.

Stress Urinary Incontinence: Enrollment continues in the Company’s ongoing Phase 2 proof-of-concept clinical trial of 3 mg of enobosarm in postmenopausal women with SUI. The Company expects to have top-line results from the trial in the third quarter of 2017.

Duchenne Muscular Dystrophy: Utilizing data developed from its preclinical development efforts, 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.

SARDs in Prostate Cancer: the Company’s selective androgen receptor degrader (SARD) technology is being evaluated as a potentially novel treatment for men with castration-resistant prostate cancer (CRPC), including those who do not respond or are resistant to currently approved therapies. The Company believes that its SARD compounds will degrade multiple forms of the androgen receptor, including AR splice variants, such as AR-V7, along with mutant versions of the receptor.

Castration-Resistant Prostate Cancer: The Company has screened dozens of compounds from its extensive patented SARD portfolio and has now selected lead compounds that are undergoing further preclinical studies required for a first in human clinical trial, including toxicology studies. It is anticipated that a first in human clinical trial of a SARD will be initiated during the second half of 2017.

The preclinical studies supporting the SARD program have been accepted for presentation and received recognition of merit at several upcoming international meetings, including the Endocrine Society’s annual meeting, ENDO 2017, from April 1-4, 2017, and the annual meeting of the European Association of Urology being held from March 24-28, 2017.

Fourth Quarter and Year-End 2016 Financial Results

As of December 31, 2016, cash and short-term investments were $21.9 million compared to $29.3 million at December 31, 2015.
Research and development expenses for the quarter ended December 31, 2016 were $4.6 million compared to $3.9 million for the same period of 2015. Research and development expenses for the year ended December 31, 2016 were $17.2 million compared to $13.6 million for the year ended December 31, 2015.
General and administrative expenses for the quarter ended December 31, 2016 were $2.3 million compared to $2.1 million for the same period of 2015. General and administrative expenses for the year ended December 31, 2016 were $8.7 million compared to $8.2 million for the year ended December 31, 2015.
The net loss for the quarter ended December 31, 2016 was $6.9 million compared to a net loss of $3.2 million for the same period in 2015. The net loss for the quarter ended December 31, 2015 included a non-cash gain of $2.7 million related to the change in the 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 subsequent to the first quarter of 2016.
The net loss for the year ended December 31, 2016 was $17.7 million compared to a net loss of $18.7 million for the year ended December 31, 2015. The net loss for the years ended December 31, 2016 and December 31, 2015 included a non-cash gain of $8.2 million and $3.1 million, respectively, related to the change in the fair value of the Company’s warrant liability.
GTx had approximately 15.9 million shares of common stock outstanding as of December 31, 2016. Additionally, there remain warrants outstanding to purchase approximately 6.4 million shares of GTx common stock at an exercise price of $8.50 per share.

CytRx Reports 2016 Financial Results

On March 15, 2017 CytRx Corporation (NASDAQ: CYTR), a biopharmaceutical research and development company specializing in oncology, reported financial results for the year ended December 31, 2016, and provided an overview of recent accomplishments and upcoming milestones for its research and development programs (Press release, CytRx, MAR 15, 2017, View Source [SID1234518133]).

"2016 was a pivotal year for CytRx. We achieved several key milestones, including reporting results from our pivotal Phase 3 trial evaluating aldoxorubicin as a treatment for patients with relapsed or refractory soft tissue sarcomas (STS)," said Steven A. Kriegsman, CytRx’s Chairman and CEO. "We look forward to sharing the results of this trial with the U.S. Food and Drug Administration (FDA) this quarter and discussing the regulatory path for a NDA submission for alddoxorubicin. We also hope to present the Phase 3 trial results to the medical community in a peer-reviewed forum in the coming months."

Mr. Kriegsman continued, "Beyond STS, we are also evaluating aldoxorubicin in a Phase 2b trial versus topotecan in patients with second-line small cell lung cancer (SCLC), and we remain on track to report top-line results from that trial by the end of the second quarter of the year."

Upcoming Milestones

Hold Type B pre-NDA meeting with the FDA in March 2017 to obtain input on the Company’s planned NDA filing strategy for aldoxorubicin as a new treatment for patients with STS. Subject to the outcome of this meeting, CytRx intends to file a NDA with the FDA or consider other alternatives which could include strategic alliances or partnerships.
Provide an update of the meeting following official receipt of regulatory guidance from the FDA.
Report top-line results from the global Phase 2b clinical trial evaluating aldoxorubicin versus topotecan in patients with second-line SCLC by the end of the second quarter of 2017.
Present results from the Company’s pivotal Phase 3 clinical trial evaluating aldoxorubicin compared to investigator’s choice in patients with relapsed or refractory STS at a major medical meeting during 2017.
Report overall survival results, a secondary endpoint, from the pivotal Phase 3 clinical trial evaluating aldoxorubicin compared to investigator’s choice in patients with relapsed or refractory STS during 2017.
Present updated data from the ongoing Phase 1b/2 clinical trial of aldoxorubicin in combination with ifosfamide/mesna in patients with advanced sarcomas during 2017.
Select the first internally developed ultra-high potency drug conjugate candidate for future development during 2017.
Fourth Quarter 2016 and Recent Highlights

Granted Type B Pre-NDA Meeting with FDA for Aldoxorubicin. In January 2017, CytRx announced that, in response to a request from the Company, the FDA has agreed to a Type B pre-NDA meeting at which the Company will seek input on its plans for a NDA for aldoxorubicin as a treatment for patients with STS.

Strengthened the Balance Sheet with $8.1 Million in Financing. In December 2016, CytRx announced its entry into securities purchase agreements with existing institutional investors to purchase shares of its common and preferred stock at a purchase price of $0.42 per share in a registered direct offering with gross proceeds of $8.1 million.

Chief Medical Officer Dr. Dan Levitt was Appointed Chief Operating Officer and Dr. Earl W. Brian Joined the Board of Directors. In December 2016, CytRx appointed Chief Medical Officer Daniel Levitt, MD, PhD, to the additional role of Chief Operating Officer. The Company also appointed Earl Warren Brien, MD, a noted sarcoma surgeon, industry consultant, and private healthcare investor, to its Board of Directors.

Reported Updated Results from Pivotal Phase 3 Trial of Aldoxorubicin in Second-Line STS In November 2016. CytRx announced positive updated results from its pivotal Phase 3 clinical trial evaluating aldoxorubicin compared to investigator’s choice in patients with relapsed or refractory STS. The study, which enrolled 433 patients, demonstrated a statistically significant improvement in progression-free survival (PFS) between aldoxorubicin and investigator’s choice therapy in 246 patients with leiomyosarcoma and liposarcoma, (p=0.007). Aldoxorubicin also demonstrated a statistically significant improvement in PFS over investigator’s choice in 312 patients treated in North America (p=0.028). Aldoxorubicin performed better than investigator’s choice for the entire study population and narrowly missed statistical significance (p=0.12). Adverse events were as expected and comparable to prior trials. Patients continue to be followed for overall survival (OS), a secondary endpoint, and CytRx expects the OS data to be available in 2017. CytRx plans to submit the results of this clinical trial for presentation at an upcoming major scientific meeting in 2017.

Presented Positive Aldoxorubicin Combination Clinical Trial Results at ESMO (Free ESMO Whitepaper) 2016 and CTOS 2016. In October 2016, CytRx presented a poster highlighting interim results from its ongoing Phase 1b/2 clinical trial of aldoxorubicin in combination with ifosfamide/mesna in patients with advanced sarcomas at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2016 Congress. In November 2016, the Company presented a poster highlighting updated interim results from the same Phase 1b/2 trial. Of 36 evaluable patients, 35 (95%) achieved clinical benefit (13 (36%) with a partial response and 22 (61%) with stable disease). While the combination did have certain toxicities, none were treatment-limiting.

Full Year 2016 Results

CytRx reported cash, cash equivalents and short-term investments of $57.0 million as of December 31, 2016.

Net loss for the year ended December 31, 2016, was $50.8 million, or $(0.63) per share, compared with a net loss of $58.6 million, or $(0.97) per share, for the year ended December 31, 2015. In 2016, the Company recognized a non-cash gain of $3.8 million on the fair value adjustment of warrant derivative liability primarily related to warrants issued in July 2016, compared to a non-cash gain of $4.4 million in 2015 related to warrants in issued in August 2011, which expired in August 2016. The Company reported licensing revenue of $200,000 in 2016, compared to $100,000 in 2015.

Research and development (R&D) expenses were $35.9 million for 2016, and included development expenses of $27.0 million for aldoxorubicin, approximately $2.3 million for pre-clinical development of new albumin-binding cancer drugs (German lab), and approximately $4.8 million for general operation of our clinical programs. R&D expenses were $43.4 million for 2015.

General and administrative (G&A) expenses were $16.0 million for 2016, compared with $19.7 million for 2015. G&A expenses decreased by approximately 19% primarily due to a significant decrease in legal fees of $4.2 million offset by pre-commercialization costs incurred in the first half of 2016. They also included non-cash stock-compensation expense of $4.9 million and $5.8 million for 2016 and 2015, respectively.

Mateon Therapeutics Announces Initial Data from Third Cohort of Phase 1b Study of OXi4503 in Relapsed/Refractory AML

On March 15, 2017 Mateon Therapeutics, Inc. (OTCQX:MATN), a biopharmaceutical company developing vascular disrupting agents (VDAs) for the treatment of orphan oncology indications, reported preliminary data from the third dose cohort of the ongoing Phase 1b study OX1222 in patients with relapsed/refractory Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS) (Press release, Mateon Therapeutics, MAR 15, 2017, View Source [SID1234518125]). OX1222 is a dose-ranging Phase 1b study of OXi4503 combined with cytarabine. The third dose cohort enrolled four patients who received a dose of 6.25 mg/m2 of OXi4503 in combination with an intermediate dose (1g/m2/day x 5 days) of cytarabine.

One patient (25%) in the third dose cohort – who has a high risk TP53 gene mutation – had a complete remission. Two other patients demonstrated evidence of AML blast reduction after one cycle, one of which is receiving additional cycles of OXi4503 plus cytarabine therapy, the other of which has since progressed. The fourth patient did not show a response and experienced progressive disease. There were no dose-limiting toxicities observed in this cohort, and the safety review committee has recommended that we proceed to the fourth cohort, which is now enrolling patients at an OXi4503 dose of 7.81 mg/m2.

"We are excited to continue to see responses in this severe and intractable patient population that currently has few treatment options," stated William D. Schwieterman, M.D., President and Chief Executive Officer. "We hope the data from this study will continue to improve with higher doses of OXi4503 in subsequent cohorts."

Results from the first two cohorts of OX1222 were initially presented at the 58th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) on December 6, 2016, indicating two out of 10 patients (20%) achieved complete remission. With an additional three months of follow-up data now available on these lower-dose patients in remission (3.75 and 4.68 mg/m2), Mateon is providing updated results – one patient remains in remission nine months following treatment and one patient remained in remission for approximately six months following treatment before the disease recurred.

Similar to the first two cohorts, OXi4503 was generally well tolerated in the third cohort. Adverse events were similar to the first two cohorts, with no significant adverse events in the third cohort.

Mateon reminds investors that a webcast of today’s presentation at the 29th Annual ROTH Conference at 7:30 am pacific time will be available on the company’s website at www.mateon.com in "Events & Presentations" under the "Investors & News" tab. Today’s presentation includes an overview of the mechanism of action of OXi4503 in hematological cancers such as AML and preclinical data from the OXi4503 AML program.

BIO-PATH HOLDINGS REPORTS FULL YEAR 2016 FINANCIAL RESULTS

On March 15, 2017 Bio-Path Holdings, Inc. (NASDAQ: BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid cancer drugs, reported its financial results for the full year ended December 31, 2016 and also provided an update on recent corporate developments (Filing, Q4/Annual, Bio-Path Holdings, 2016, MAR 15, 2017, View Source [SID1234518122]).

"We continue to make progress in our Phase 2 clinical trial of prexigebersen for the treatment of acute myeloid leukemia (AML) and are pleased to have six important clinical sites actively evaluating and enrolling patients," said Peter Nielsen, President and CEO of Bio-Path Holdings. "Additionally, we were honored to welcome Dr. Craig Hooper to our Scientific Advisory Board. He is a recognized world leader in glioblastoma research and will be a valuable asset to Bio-Path as we advance our DNAbilize immunotherapy program into the clinic."

Recent Corporate Highlights

· Presented Clinical Data Evaluating BP1001 as a Treatment for Chronic Myelogenous Leukemia at the 58th Annual American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. In December, Ana Tari Ashizawa, Ph.D., Director of Research at Bio-Path, delivered a poster presentation of prexigebersen (BP1001) data as a treatment for chronic myelogenous leukemia (CML). The results demonstrated that BP1001 decreased the proliferation of Gleevec (imatinib)-resistant CML cells in a dose-dependent manner. In addition, BP1001 pretreatment enhanced the inhibitory effects of Sprycel (dasatinib) in CML cells, leading to cell death. Five CML blast phase patients were enrolled in the first cohort (5mg/m2 BP1001) of the Phase 1 BP1001 clinical study. Two CML patients, who had T315I mutation, showed significant reductions in circulating blasts during treatment. One patient’s blasts were reduced from 89% to 12%, while another patient’s blasts were reduced from 24% to 7%.

· Appointed D. Craig Hooper, Ph.D., to Scientific Advisory Board. In February, the Company announced the appointment of D. Craig Hooper, Ph.D., to its Scientific Advisory Board (SAB). Dr. Hooper is a Professor of Cancer Biology and Neurological Surgery at Thomas Jefferson University. He has published over 140 papers in peer-reviewed journals and serves on the editorial boards of the Journal of Immunology Research, Scientific Reports and the Journal of Immunology. In 2016, he was inducted into the National Academy of Inventors (NAI).

Financial Results for the Full Year Ended December 31, 2016

The Company reported a net loss of $6.8 million, or $0.07 per share, for the year ended December 31, 2016, compared to a net loss of $5.5 million, or $0.06 per share, for the year ended December 31, 2015. The increase was primarily due to the release of drug material for the Company’s Phase II clinical trial for prexigebersen in AML and associated clinical trial costs.

Research and development expenses for the year ended December 31, 2016 increased to $5.5 million, compared to $3.0 million for the year ended December 31, 2015. General and administrative expenses for the year ended December 31, 2016 increased to $3.0 million, compared to $2.5 million for the year ended December 31, 2015.
Change in fair value of the Company’s warrant liability for the year ended December 31, 2016 resulted in non-cash income of $1.7 million. The Company did not have a warrant liability in the comparable period for 2015.

As of December 31, 2016, the Company had cash of $9.4 million, compared to $8.9 million at December 31, 2015. Net cash used in operating activities for the year ended December 31, 2016 was $8.1 million compared to $5.0 million for the comparable period in 2015. Net cash used in investing activities was $0.3 million for the year ended December 31, 2016. The Company did not use any cash in investing activities for the comparable period in 2015. Net cash provided by financing activities for the year ended December 31, 2016 was $9.0 million.