Category: Uncategorized

GTx Reports Third Quarter 2018 Financial Results and Provides Corporate Update

On October 22, 2018 GTx, Inc. (Nasdaq: GTXI) reported financial results for the third quarter ended September 30, 2018, and provided a corporate update (Press release, GTx, OCT 22, 2018, View Source [SID1234530034]).

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!

"During the quarter, we turned our focus to the ongoing selective androgen receptor degrader program and the potential of our novel selective androgen receptor degrader to treat castration-resistant prostate cancer. We expect to select the most appropriate development compounds by year-end, which we plan to take into IND-enabling studies next year," said Robert J. Wills, Ph.D., Executive Chairman of GTx. "Additionally, we are exploring other strategic options for the company with the goal of optimizing the full potential of our development pipeline."

Corporate Development Update

Selective Androgen Receptor Degrader (SARD): Prostate Cancer

The Company has an ongoing preclinical program to evaluate its novel selective androgen receptor degrader (SARD) technology in castration-resistant prostate cancer (CRPC). In some men with CRPC, current prostate cancer therapy is not effective or subject to emerging resistance. The Company believes that its SARDs may be first-in-class dual-interacting androgen receptor (AR) antagonists and degraders, and may therefore potentially treat CRPC in men who are non-responsive to current androgen targeted therapies. Going forward, the Company plans to:

Complete ongoing mechanistic preclinical studies by year-end or early in the first quarter of 2019;
Select the most appropriate SARD compounds to move forward with IND-enabling studies in 2019; and
Potentially advance one of its SARD compounds into a first-in-human clinical trial in 2020
Selective Androgen Receptor Modulator (SARM): Stress Urinary Incontinence (SUI), Breast Cancer

SUI: Enobosarm, a SARM, was evaluated in post-menopausal women with SUI compared to placebo. During the quarter, the Company announced that the ASTRID Trial, a Phase 2 double-blind, placebo-controlled clinical trial of orally-administered enobosarm (3 mg or 1 mg) in post-menopausal women with SUI, did not achieve statistical significance on the primary endpoint for the trial. Enobosarm was generally safe and well tolerated, and reported adverse events were minimal and similar across all treatment groups. The Company is conducting a comprehensive review of all the ASTRID data and is consulting with key experts to fully understand the study outcomes.

Advanced Breast Cancer: Enobosarm was also 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. The trial met the primary efficacy endpoint in the trial; there are three women in the study who continue to respond to treatment after almost two years on enobosarm (two have stable disease, one now has a partial response). Approximately one year ago, the Company determined that treatment paradigms had shifted to immunotherapies and/or combination therapies, and that it was no longer feasible for GTx to conduct further development of enobosarm in breast cancer.

Enobosarm has been evaluated in more than two dozen clinical trials enrolling over 2,200 subjects, in which approximately 1,500 subjects were treated with enobosarm at doses ranging from 0.1 mg to 100 mg. At all evaluated dose levels, enobosarm was observed to be generally safe and well tolerated.

Third Quarter 2018 Financial Results

As of September 30, 2018, cash and short-term investments were $38.1 million compared to $43.9 million at December 31, 2017.
Research and development expenses for the quarter ended September 30, 2018 were $7.5 million compared to $5.9 million for the same period of 2017.
General and administrative expenses for the quarter ended September 30, 2018 were $2.2 million compared to $2.6 million for the same period of 2017.
The net loss for the quarter ended September 30, 2018 was $9.4 million compared to a net loss of $8.5 million for the same period in 2017.
Net loss for the nine months ended September 30, 2018 was $33.0 million compared to a net loss of $21.2 million for the same period in 2017.
GTx had approximately 24.1 million shares of common stock outstanding as of September 30, 2018. Additionally, there are warrants outstanding to purchase approximately 5.3 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.

Anaeropharma Science Signs Collaborative Research Agreement with Chugai on Creation of Novel Oncology Drugs Using Bifidobacterium

On October 22, 2018 Anaeropharma Science Inc. headquartered in Tokyo (hereinafter "Anaeropharma") reported that Anaeropharma Science and Chugai Pharmaceutical Co., Ltd. headquartered in Tokyo (hereinafter "Chugai") have concluded a collaborative research agreement concerning the creation of novel oncology drugs utilizing characteristic features of Bifidobacterium longum through Anaeropharma’s proprietary platform technology, "in situ Delivery and Production System" (hereinafter "i-DPS") (Press release, Anaeropharma Science, OCT 22, 2018, View Source [SID1234530116]). An overview of the contract follows.

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!

A collaborative research agreement concerning the creation of novel oncology drugs utilizing i-DPS technology

Under the agreement, Anaeropharma and Chugai conduct joint research regarding specific oncology substances by use of Anaeropharma’s i-DPS technology and Chugai’s technology. The scope of the agreement is limited to the specific substances predetermined by both companies, and the i-DPS technology will be applied only to those substances.

About i-DPS and its development programs

Bifidobacterium is obligatory anaerobe which exists as enteroflora in the human body, and known as nonpathogenic bacteria. Solid cancers have immature vascular constructs and their interstitial tumors are in the state of hypoxia. The company aims to leverage the recombinant Bifidobacterium technology to create a new class of anti-cancer drugs. The technology offers broad potential of being more effective to solid tumors and generates oncology drugs with less risks of adverse events than conventional anti-cancer drugs.

The leading product developed using i-DPS technology, APS001F, a recombinant Bifidobacterium to express Cytosine Deaminase which converts a prodrug, 5-FC, to an anti-cancer drug, 5-FU, is under a phase 1 clinical trial in the U.S.

Actinium Pharmaceuticals Announces Webinar Showcasing Actimab-A Post Phase 2 Trial Plans and Actimab-MDS Regulatory Update

On October 22, 2018 Actinium Pharmaceuticals, Inc. (NYSE American: ATNM) reported that it will host a conference call and webinar to provide key updates on the advancement of its CD33 program on Friday, October 26, 2018 at 11:00 AM ET (Press release, Actinium Pharmaceuticals, OCT 22, 2018, View Source [SID1234530262]). Actinium’s CD33 program utilizes the Antibody Radio-Conjugate (ARC), lintuzumab-Ac-225 for hematologic indications including Acute Myeloid Leukemia (AML), Myelodysplastic Syndrome (MDS) and Multiple Myeloma (MM). Actinium recently completed its Phase 2 Actimab-A trial in patients newly diagnosed with AML who are over the age of 60 and unfit for intensive chemotherapy. Dr. Gary Schiller Professor Medicine, Hematology-Oncology at UCLA Medical Center and Dr. Tapan Kadia, Assistant Professor of Medicine, Department of Leukemia at the MD Anderson Cancer Center will highlight Actinium’s post Phase 2 trial development plans for Actimab-A in AML.

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!

Actinium is also developing Actimab-MDS, which is intended to be a single dose, chemotherapy-sparing targeted conditioning agent for patients with high-risk MDS. Currently, this patient population either cannot undergo a bone marrow transplant or have poor outcomes. Management will provide an update on the regulatory pathway for Actimab-MDS following positive interactions with the U.S. Food and Drug Administration (FDA).

Dr. Mark Berger, Actinium’s Chief Medical Officer said, "Our CD33 program has progressed significantly in 2018 resulting in a highly valuable body of data that we are using to inform our ongoing development strategy. Our Antibody Radio-Conjugate approach, given its differentiated mechanism of action, has allowed us to expand this program beyond a traditional AML directed approach which is where other CD33 programs in the industry are focused. The unique ability of our ARC’s enable cell killing to occur not just via internalization of the antigen but also from the cell surface and by crossfire. In addition, our ARCs labeled with radioactive actinium are characterized by the high linear energy transfer of alpha radiation which is able to cause double stranded DNA breaks via a single alpha particle hit. This potent cell killing power of alpha radiation when used in an efficiently targeted manner as in our Actimab program enables us to expand into other radio-sensitive CD33 expressing malignancies such as multiple myeloma and now for targeted conditioning for MDS, both of which are indications where Actinium is developing the only CD33 targeting agent. In addition, we have moved into a novel combination trial for patients with significant unmet need with our Actimab-A CLAG-M study. We are pleased to have made this progress, but we believe the next evolution of our CD33 program will be even more exciting. As such, we look forward to highlighting our post Phase 2 trial development plans for Actimab-A with Dr. Schiller and Dr. Kadia."

Sandesh Seth, Actinium’s Chairman and Chief Executive Officer said, "We are excited to introduce these latest initiatives as they clearly establish Actinium’s Antibody Radio-Conjugate based CD33 program as the industry leader. Further, we are about to enter a period that will showcase data from several of the CD33 program initiatives that this team has advanced. Given the inherent nature of our technology, the expertise of our team and strong relationships with thought leaders, we have been able to craft a development strategy that leverages the strengths of our drug candidates and Antibody Warhead Enabling technology platform into indications with high unmet medical needs. The webinar will showcase the attractiveness of using our Antibody Radiation-Conjugate approach in meeting these needs. In addition, it will establish the strategic importance of Actimab-MDS in enabling our company to develop a multi-asset pipeline of targeted conditioning agents which have the potential to improve access and outcomes for patients undergoing bone marrow transplant and cellular therapy in a chemotherapy-free or chemotherapy-sparing manner."

Conference call and webcast Participation Information
Date: Friday, October 26, 2018
Time: 11:00 AM ET
Webcast Registration: View Source
U.S. Participant Dial-in: (718) 865-8336
U.S./Canada Toll Free Dial-in: (855) 427-0225
Conference ID: 4831

Published Late Breaking Abstracts at ESMO 2018: Sunday 21st October


More than 60 Late Breaking Abstracts (LBA’s) are scheduled to be published at this year’s European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress (E.S.M.O 2018). Below you will find the 27 published at the sessions on Sunday 21st October, the third day of the conference and the busiest for Late Breaking Abstracts.

For full analysis identifying new technologies, drugs, targets, start-ups etc. we recommend Commercial Interest at E.S.M.O Annual Meeting 2018: Analytical Tool.

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!

Roche’s investigational personalised medicine entrectinib shrank tumours in people with NTRK fusion-positive solid tumours

On October 21, 2018 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported results from an integrated analysis of the pivotal phase II STARTRK-2, phase I STARTRK-1 and phase I ALKA-372-001 trials that showed the investigational personalised medicine entrectinib shrank tumours (objective response rate; ORR) in more than half (57.4%) of people with neurotrophic tropomyosin receptor kinase (NTRK) fusion-positive solid tumours (Press release, Hoffmann-La Roche, OCT 21, 2018, View Source [SID1234530312]). Objective responses to entrectinib were seen across ten different solid tumour types (median duration of response [DOR]=10.4 months), including in people with and without central nervous system (CNS) metastases at baseline.[1]
Importantly, entrectinib shrank tumours that had spread to the brain in over half of people (intracranial response; IC ORR=54.5%), with more than a quarter of these people having a complete response.[1] The safety profile of entrectinib was consistent with that seen in previous analyses.[1]

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!

"These data demonstrate the potential of entrectinib to treat a range of difficult-to-treat and rare cancers regardless of their site of origin," said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. "Entrectinib has the potential to redefine personalised medicine, which can utilize tests such as next-generation sequencing, to find the right treatment for each individual patient. People with NTRK fusion-positive solid tumours need more options, and we look forward to working with health authorities to bring this potential treatment to patients as soon as possible."

Roche is leveraging its expertise in developing personalised medicines and advanced diagnostics, in conjunction with Foundation Medicine, to develop a novel diagnostics approach using next-generation sequencing that will help identify people with NTRK gene fusions likely to benefit from entrectinib.[2;3]

Entrectinib has been granted Breakthrough Therapy Designation (BTD) by the US Food and Drug Administration (FDA); Priority Medicines (PRIME) designation by the European Medicines Agency (EMA); and Sakigake designation by the Japanese health authorities for the treatment of NTRK fusion-positive, locally advanced or metastatic solid tumours in adult and paediatric patients who have either progressed following prior therapies or have no acceptable standard therapies.[4] These designations are reserved for medicines that demonstrate substantial improvements over existing therapies or where there are unmet medical needs.
These NTRK fusion-positive results will be presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2018 Congress in Munich, Germany on 21 October 2018, 11:24-11:36 am CEST (LBA17). Follow Roche on Twitter via @Roche and keep up to date with ESMO (Free ESMO Whitepaper) 2018 congress news and updates by using the hashtag #ESMO2018.

Roche also recently presented positive results at the IASLC 19th World Conference on Lung Cancer (WCLC) that showed entrectinib shrank tumours (ORR) in 77.4% of people with locally advanced or metastatic ROS1-positive non-small cell lung cancer (NSCLC).[5] In addition, entrectinib demonstrated a durable response of more than two years (DOR=24.6 months).[5] Importantly, entrectinib was shown to shrink tumours in more than half of people with cancer in the CNS (IC ORR=55.0%).[5] The safety profile of entrectinib was consistent with that seen in previous analyses.[5]

Roche plans to submit results from these integrated analyses to global health authorities for the treatment of NTRK fusion-positive solid tumours and ROS1-positive NSCLC.

About the integrated analysis
The integrated analysis included data from 54 people with locally advanced or metastatic NTRK fusion-positive solid tumours (10 tumour types, >19 histopathologies) from the phase II STARTRK-2, phase I STARTRK-1 and phase I ALKA-372-001 trials.[1] The studies enrolled people across 15 countries and more than 150 clinical trial sites. Tumour types evaluated in the studies to date included breast, cholangiocarcinoma, colorectal, gynaecological, neuroendocrine, non-small cell lung, salivary gland, pancreatic, sarcoma and thyroid cancers.

STARTRK-2 is a phase II, global, multicentre, open-label basket study in people with solid tumours that harbour an NTRK1/2/3, ROS1 or ALK-positive gene fusion.[6] The primary endpoint is ORR and DOR is a secondary endpoint.[6] Other secondary outcome measures include time to response, clinical benefit rate, intracranial tumour response, progression-free survival (PFS), CNS PFS and overall survival (OS).[6]
STARTRK-1 is a phase I, multicentre, open-label dose escalation study of a daily continuous dosing schedule in people with solid tumours with NTRK1/2/3, ROS1 or ALK gene fusions in the US and South Korea.[7] The trial assessed the safety and tolerability of entrectinib via a standard dose escalation scheme and determined the recommended phase II dose.[7]
ALKA-372-001 is a phase I, multicentre, open-label dose escalation study of an intermittent and continuous entrectinib dosing schedule in people with advanced or metastatic solid tumours with TrkA/B/C, ROS1 or ALK gene fusions in Italy.[8]
Overall, entrectinib was well tolerated and the majority of adverse events were Grade 1-2, reversible, and managed with treatment interruption or dose reduction.[1] Treatment-related adverse events leading to discontinuation occurred in 3.9% of patients.[1] The most common treatment-related adverse events were altered sense of taste (dysgeusia), fatigue, and dizziness.[1]

About entrectinib
Entrectinib (RXDX-101) is an investigational, oral medicine in development for the treatment of locally advanced or metastatic solid tumours that harbour NTRK1/2/3 or ROS1 gene fusions. It is a selective, CNS-active tyrosine kinase inhibitor designed to inhibit the kinase activity of the TRK A/B/C and ROS1 proteins, whose activating fusions drive proliferation in certain types of cancer.[2;3] Entrectinib can block ROS1 and NTRK kinase activity and may result in the death of cancer cells with ROS1 or NTRK gene fusions.[2;3] Entrectinib is being investigated across a range of solid tumour types, including breast, cholangiocarcinoma, colorectal, gynaecological, neuroendocrine, non-small cell lung, salivary gland, pancreatic, sarcoma and thyroid cancers.[6-8]

Entrectinib has been granted Breakthrough Therapy Designation (BTD) by the US Food and Drug Administration (FDA); Priority Medicines (PRIME) designation by the European Medicines Agency (EMA); and Sakigake designation by the Japanese health authorities for the treatment of NTRK fusion-positive, locally advanced or metastatic solid tumours in adult and paediatric patients who have either progressed following prior therapies or have no acceptable standard therapies.[4]

About NTRK gene fusions
Neurotrophic tyrosine receptor kinase (NTRK) fusion-positive cancer occurs when the NTRK1/2/3 genes fuse with other genes, resulting in altered TRK proteins (TrKA/TrKB/TrKC) that can activate signalling pathways involved in proliferation of certain types of cancer. NTRK gene fusions are tumour-agnostic, meaning they are present in tumours irrespective of site of origin, and have been identified a broad range of solid tumour types, including breast, cholangiocarcinoma, colorectal, gynaecological, neuroendocrine, non-small cell lung, salivary gland, pancreatic, sarcoma and thyroid cancers.[1] There is a high unmet medical need for treatments for people with life-threatening NTRK fusion-positive tumours.