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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

On March 15, 2017 (GLOBE NEWSWIRE) — Advaxis, Inc. (NASDAQ:ADXS), a biotechnology company developing cancer immunotherapies, presented data from the GOG-0265 study at the Society of Gynecologic Oncology’s Annual Meeting on Women’s Cancer in National Harbor, MD (Press release, Advaxis, MAR 15, 2017, View Source [SID1234518121]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

GOG-0265 is a single arm, Phase 2 trial evaluating axalimogene filolisbac for the treatment of persistent or recurrent metastatic (squamous or non-squamous cell) carcinoma of the cervix (PRmCC). The primary endpoints of the study were to assess the safety and efficacy of axalimogene filolisbac in women with PRmCC. The primary efficacy endpoint was overall survival at 12 months from initial treatment with axalimogene filolisbac. The primary safety endpoints were to evaluate the number of patients with dose-limiting toxicities and the frequency and severity of adverse effects.

The final efficacy results of GOG-0265 demonstrated that 38% of patients (n = 19/50) with heavily pretreated PRmCC were alive 12 months following treatment with axalimogene filolisbac. The GOG-0265 study protocol used a logistic model-based calculation to establish the expected 12-month survival rate. The model identified the key prognostic factors of age, race and performance status significantly related to survival from a database of approximately 500 patients with PRmCC who participated in 17 previous phase 2 studies conducted by the Gynecologic Oncology Group (GOG), now part of NRG Oncology. Using this model, the expected 12-month survival rate of patients enrolled in the study was calculated to be 24.5%. As a result, the 38% 12-month survival rate of patients treated with axalimogene filolisbac represents a 52% improvement over the expected survival rate and is the highest 12-month survival rate achieved to date in this setting. The probability of this survival improvement being detected by chance versus a true treatment effect was calculated to be 0.02. A compelling and ongoing complete response of 18.5 months was observed and the longest ongoing survival is 40.6 months.

"The 12-month survival rate of axalimogene filolisbac reached unprecedented levels in this study, which is both impressive and important given the lack of innovation in metastatic cervical cancer," said Warner K. Huh, MD, Division Director of Gynecologic Oncology at the University of Alabama at Birmingham, and Lead Investigator of the study.

The safety profile was consistent with previous clinical experience. The most common Grade 1 or Grade 2 treatment-related adverse events (TRAEs) were hypotension and symptoms related to cytokine release (e.g., nausea, chills, fever). Eighteen out of 50 patients experienced a Grade 3 TRAE and two out of 50 patients experienced a Grade 4 TRAE, which were hypotension and symptoms related to cytokine release.

The abstract was selected by SGO for prominence as an oral late-breaking presentation by Charles A. Leath III, M.D., MSPH, Associate Professor of Obstetrics and Gynecology at the University of Alabama at Birmingham School of Medicine, entitled, "A prospective phase 2 trial of the listeria-based HPV immunotherapy axalimogene filolisbac in second and third-line metastatic cervical cancer: A NRG Oncology Group trial," on March 14 at 2:30 p.m. ET. Slides from the presentation are available at www.advaxis.com/sgo2017.

Highlights from Dr. Leath’s presentation include:

A 38% (n = 19/50) 12-month survival rate in second- and third-line PRmCC treated with axalimogene filolisbac, representing a 52% improvement over the expected 12-month milestone survival rate of 24.5%
Eight patients remain alive as of January 31, 2017 (Range 12.02 – 40.6 months)
Disease control (complete response, partial response, or stable disease) was achieved in 32% of patients based on investigator assessment of best response
A durable complete response in a patient with PRmCC previously treated with chemotherapy and bevacizumab remains ongoing at 18.5 months
Results compare favorably to GOG Study 227C of bevacizumab, which demonstrated a 12-month milestone overall survival (OS) rate of 30% in a similar patient population which subsequently supported regulatory approval in first-line treatment in combination with chemotherapy in 2014
Consistent with its immunotherapy mechanism of action, axalimogene filolisbac demonstrated a promising plateau in the survival curve, indicating potential long-term clinical benefit for a subset of patients with PRmCC
Axalimogene filolisbac was generally well-tolerated, with primarily infusion-associated, low grade, transient TRAEs (≥30%), such as fatigue, chills, anemia, nausea and fever
Only 2 patients experienced grade 4 TRAEs

Advaxis plans to initiate a global, phase 3 randomized registration study in patients with metastatic cervical cancer later this year.

About the Phase 2 GOG-0265 Study

GOG-0265 is an open-label, single arm 2-stage study designed to evaluate the safety, tolerability and efficacy of axalimogene filolisbac to treat PRmCC as conducted by the Gynecologic Oncology Group (GOG), now part of NRG Oncology. Patients who progressed on or after at least 1 prior line of systemic-dose chemotherapy receive one cycle (three doses) of axalimogene filolisbac at 1 x 109 CFU every 28 days. The primary efficacy endpoint was the 12-month survival rate, with secondary efficacy objective to evaluate progression-free survival, overall survival and objective tumor response. The primary safety endpoints were to evaluate the number of patients with dose-limiting toxicities and the frequency and severity of adverse effects.

The expected 12-month overall survival rate (null hypothesis) was established using a prospectively-defined logistic model-based calculation derived from 17 serially conducted GOG/NRG 2-stage studies in PRmCC involving approximately 500 patients, adjusting for prognostic factors (age, performance status, race) significantly related to survival. In accordance with the prior trials, GOG/NRG used a consistent protocol design/data collection methodology for the current 2-stage GOG-0265 study in PRmCC, which contributed to a robust and homogeneous patient dataset for the primary endpoint analysis.

About Cervical Cancer

Cervical cancer is the fourth most common cancer in women worldwide. An estimated 13,000 new cases will be diagnosed in the United States in 2016, and 4,100 people will die of the disease, according to the National Cancer Institute. Persistent HPV infection is the most important factor in the development of cervical cancer, research shows. According to the ICO Information Centre on HPV and Cervical Cancer, about 4.4% of women in the United States are estimated to harbor high-risk cervical HPV infection at a given time, and about 72% of cervical cancers are attributed to high-risk HPV strains. PRmCC is a fatal disease, and the prognosis for women with advanced and recurrent cervical cancer remains poor, with survival of only 4 to 7 months following failure of first-line treatment, research has shown. There is no therapy following failure of first-line treatment. According to the American Cancer Society, the five-year mortality rate for metastatic disease is at just 17%, with the area continuing to be a high unmet medical need.

About the GOG Foundation, Inc.

The GOG Foundation, Inc. (GOG) is a non-profit international organization with the purpose of promoting excellence in the quality and integrity of clinical and basic scientific research in the field of gynecologic malignancies. The GOG is committed to maintaining the highest standards in clinical trials development, execution, analysis and distribution of results. Continuous evaluation of its processes is utilized in order to constantly improve the quality of patient care. The GOG conducts clinical trials for patients with a variety of gynecologic malignancies, including cancers that arise from the ovaries, uterus, cervix, vagina and vulva. General information on many of these trials for medical professionals and the lay public can be obtained from ClinicalTrials.gov.

NRG Oncology is one of four adult US Network groups funded under the newly structured NCI National Clinical Trials Network. NRG Oncology is comprised of three legacy cooperative groups, the National Surgical Adjuvant Breast and Bowel Project (NSABP), the Radiation Therapy Oncology Group (RTOG), and the Gynecologic Oncology Group (GOG).