On May 15, 2017 OncoGenex Pharmaceuticals, Inc. (NASDAQ: OGXI) reported financial results for the first quarter ended March 31, 2017 (Filing, Q1, OncoGenex Pharmaceuticals, 2017, MAY 15, 2017, View Source [SID1234519122]).

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Recent Events

• In January 2017, Achieve Life Science, Inc. (Achieve) and OncoGenex announced they entered into a definitive merger agreement.

• In February 2017, OncoGenex announced that apatorsen results from two randomized Phase 2 clinical trials were presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2017 Genitourinary Cancers Symposium, held February 16th- 18th in Orlando. Clinical data from trials in bladder and prostate cancers demonstrated apatorsen was well-tolerated and improved patient outcomes when administered in combination with standard-of-care treatments.

• In March 2017, Achieve announced a strategic collaboration with the National Center for Complementary and Integrative Health (NCCIH) at the National Institutes of Health (NIH) to conduct non-clinical studies in support of an overall clinical development plan for cytisine as a smoking cessation treatment. As part of the collaboration, Achieve is providing cytisine to the NIH to conduct a series of non-clinical studies required by the U.S. Food and Drug Administration (FDA) to support the submission of an Investigational New Drug (IND) application. The collaboration commenced in March 2015 and results of the studies are expected in the second-quarter of 2017.

• In March 2017, the Society for Research in Nicotine and Tobacco (SRNT) held a symposium on cytisine research at its annual conference held in Florence, Italy. The symposium was chaired by Professor Nancy Rigotti, MD, Massachusetts General Hospital/Harvard Medical School, with presentations from Associate Professor Natalie Walker, PhD, National Institute for Health Innovation, University of Auckland, on "Cytisine versus Varenicline for Smoking Cessation: Two Clinical Trials from the Australasian Cytisine Trialist Group," and "The Challenge to Getting Cytisine Licensed For Use Worldwide: Policy Considerations." Dr. Walker was the principal investigator of the 1,310 patient phase 3 CASCAID trial published in the New England Journal of Medicine in December, 2014 titled "Cytisine versus Nicotine for Smoking Cessation".
Financial Results
As of March 31, 2017, the company’s cash, cash equivalents, and short-term investments were $16.5 million compared with $25.5 million as of December 31, 2016. Based on current expectations, OncoGenex believes that its cash, cash equivalents, and short-term investments will be sufficient to fund its currently planned operations for at least the next 12 months.
Revenue for the three months ended March 31, 2017 decreased to zero from $2.9 million for three months ended March 31, 2016. Total operating expenses for the three months ended March 31, 2017 were $3.3 million compared to $7.4 million for the same period in 2016. Net loss for the three months ended March 31, 2017 was $3.3 million compared to $3.7 million for the same period in 2016.
As of May 15, 2017 OncoGenex had 30,087,485 shares outstanding.

On May 15, 2017 GTx, Inc. (Nasdaq: GTXI) reported financial results for the first quarter of 2017, and highlighted recent accomplishments and upcoming milestones (Press release, GTx, MAY 15, 2017, View Source [SID1234519120]). The Company has two ongoing clinical trials of enobosarm (GTx-024) as a potential treatment 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 is also completing preclinical studies in its Selective Androgen Receptor Degrader (SARD) program that are required prior to initiating a clinical trial in men with castration-resistant prostate cancer (CRPC), which is planned for the first half of 2018.

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"We look forward to several important data milestones during this year. This includes top-line results which we expect to announce in the third quarter of this year from an ongoing Phase 2 trial of enobosarm in women with advanced, ER+, AR+ breast cancer and from our clinical trial to treat postmenopausal women with SUI. We also continue to generate interest in the SARD program, which is focused on developing a novel treatment for advanced prostate cancer." said Robert J. Wills, Ph.D., Executive Chairman of GTx.

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 two dose cohorts in the trial are being treated independently for the purpose of assessing efficacy. The study is fully enrolled and 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 an orally-administered 18 mg dose of enobosarm in up to 55 women with advanced, AR+ TNBC. 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 later this quarter 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: Earlier this year, the Company announced it has added additional clinical sites to its ongoing Phase 2 proof-of-concept clinical trial of 3 mg of enobosarm in postmenopausal women with SUI. These sites are now enrolling patients, and the Company expects to announce top-line results from this trial in the third quarter of 2017.

An abstract on preliminary data from the ongoing Phase 2 clinical trial has been accepted for podium presentation at the annual meeting of the International Continence Society in Florence, Italy on September 13, 2017. The principal investigator, Dr. Kenneth Peters, will present these encouraging data from the first cohort of patients enrolled in the study.
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.

Results from preclinical studies to support the potential efficacy of the SARM GTx-026, GTx-027 and enobosarm for DMD treatment were published in the journal Human Molecular Genetics; GTx SARMs increased body weight, lean mass and physical function in preclinical models 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 compounds from its extensive patented SARD portfolio and has now selected lead compounds that are undergoing further preclinical studies including toxicology studies required for a first in human clinical trial which is planned during the first half of 2018. Preclinical SARD data recently were presented at the following medical meetings:

The discovery and early mechanistic evaluation studies were presented at the Endocrine Society’s annual meeting, ENDO 2017.
Data on the effect of SARDs on enzalutamide-resistant prostate cancer were presented at the annual meeting of the European Association of Urology.
Data confirming N-terminal domain binding and efficacy, in preclinical models of AR-SV-expressing castration-resistant prostate cancer, was presented this week at the American Urological Association Meeting, 2017, in Boston. N-terminal binding facilitates inhibition of androgen-mediated cancer proliferation in both native and mutant receptors.
First Quarter 2017 Financial Results

As of March 31, 2017, cash and short-term investments were $16.5 million compared to $21.9 million at December 31, 2016.
Research and development expenses for the quarter ended March 31, 2017 were $4.2 million compared to $4.0 million for the same period of 2016.
General and administrative expenses were $2.1 million for both the quarter ended March 31, 2017 and March 31, 2016.
The Company recognized a non-cash gain of $8.2 million for the quarter ended March 31, 2016 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 subsequent to the first quarter of 2016.
Net loss for the quarter ended March 31, 2017 was $6.3 million compared to net income of $2.1 million for the same period in 2016. Net income for the quarter ended March 31, 2016 included the non-cash gain of $8.2 million related to the revaluation of the Company’s warrant liability.
GTx had approximately 16.0 million shares of common stock outstanding as of March 31, 2017. 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.

On May 15, 2017 Ignyta, Inc. (Nasdaq: RXDX), a biotechnology company focused on precision medicine in oncology, reported that the U.S. Food and Drug Administration (FDA) has granted a Breakthrough Therapy Designation (BTD) to entrectinib "for the treatment of NTRK fusion-positive, locally advanced or metastatic solid tumors in adult and pediatric patients who have either progressed following prior therapies or who have no acceptable standard therapies (Press release, Ignyta, MAY 15, 2017, View Source [SID1234519116])." Entrectinib is the company’s investigational, orally available, CNS-active tyrosine kinase inhibitor targeting tumors that harbor NTRK1/2/3, ROS1, or ALK gene fusions.

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The FDA’s Breakthrough Therapy Designation is intended to expedite development and review timelines of potential new medicines for use in the treatment of a serious or life-threatening condition when preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints [1].

"The granting of Breakthrough Therapy Designation enables us to continue high quality engagement with the FDA during the development of entrectinib, and we greatly appreciate the commitment by FDA to move this investigational drug forward," said Jonathan Lim, M.D., Chairman and CEO of Ignyta. "We believe this designation validates the broad potential of entrectinib as a novel treatment for patients, regardless of age, with TRK-positive tumors, a group of cancers for which there currently is no approved treatment and which represents a clear unmet medical need."

About Entrectinib

Entrectinib is a novel, orally available, CNS-active tyrosine kinase inhibitor targeting tumors that harbor activating alterations to NTRK1/2/3 (encoding TRKA/TRKB/TRKC), ROS1 or ALK. Entrectinib is the only TRK inhibitor with clinically demonstrated activity against primary and metastatic CNS disease, and does not have undesirable off-target activity. This product candidate is in a Phase 2 clinical trial called STARTRK-2, which is the second of the "Studies of Tumor Alterations Responsive to Targeting Receptor Kinases." The trial is a global, multicenter, open label, potentially registration-enabling Phase 2 clinical trial of entrectinib that utilizes a basket design with screening of patient tumor samples for the relevant targets. Such a basket design takes full advantage of entrectinib’s demonstrated preliminary clinical activity across a range of different tumor types and molecular targets.

On May 15, 2017 Corvus Pharmaceuticals, Inc. (Nasdaq:CRVS), a clinical-stage biopharmaceutical company focused on the development and commercialization of novel immuno-oncology therapies, reported that it will present an oral abstract presentation at the 2017 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting taking place June 2-6 in Chicago, Illinois. The abstract for the presentation will be available on the ASCO (Free ASCO Whitepaper) website on May 17 at 5:00 p.m. ET (Press release, Corvus Pharmaceuticals, MAY 15, 2017, View Source [SID1234519115]). Following are details for the oral presentation.

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ORAL PRESENTATION

ABSTRACT #: 3004
TITLE: Safety and clinical activity of adenosine A2a receptor (A2aR) antagonist, CPI-444, in anti-PD1/PDL1 treatment-refractory renal cell (RCC) and non-small cell lung cancer (NSCLC) patients
PRESENTER: Lawrence Fong, M.D., Leader, Cancer Immunotherapy Program, Co-Director, Parker Institute of Cancer Immunotherapy at University of California, San Francisco
PRESENTATION DATE AND TIME: Monday, June 5, 2:27 p.m. CT
LOCATION: Hall D1

On May 15, 2017 Cellular Biomedicine Group Inc. (NASDAQ: CBMG) ("CBMG" or the "Company"), a clinical-stage biopharmaceutical firm engaged in the development of effective immunotherapies for cancer and stem cell therapies for degenerative diseases, reported the addition of a new independent Phase I clinical trial of the Company’s ongoing CARD-1 study in patients with chemorefractory and aggressive DLBCL (Press release, Cellular Biomedicine Group, MAY 15, 2017, View Source [SID1234519114]). The Company and Shanghai Tongji Hospital (Tongji) are conducting a single arm, non-randomized study to evaluate the safety and efficacy of C-CAR011 (Anti-CD19 single-chain variable fragment (scFv) (41BB-CD3ζ)) therapy in relapsed or refractory B cell Non-Hodgkin Lymphoma (NHL). The trial will enroll 15 patients comprised of DLBCL, Primary Mediastinal Large B-Cell Lymphoma (PMBCL) and Follicular Lymphoma (FL).

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"Driven by Shanghai’s regional demand, Tongji’s CAR-T expertise, the requirement to confirm site to site consistency and our need to prepare for the next phase of a confirmatory clinical trial, the new trial will benefit patients in Shanghai and provide CBMG with incremental data in safety and tolerability of C-CAR011 in more chemorefractory and aggressive DLBCL patients comprised of unique histogenesis and those with the most common indolent form of non-Hodgkin lymphoma (NHL). " said Mr. Tony Liu, Chief Executive Officer for CBMG.

Mr. Tony Liu added, "Due to our robust clinical pipeline, we believe the Company’s stock is currently undervalued. The management and our scientific team are committed to delivering long-term clinical benefits to patients that have the potential to address very large cancer and knee osteoarthritis markets and create long-term value for shareholders. We believe that CBMG has one of the very few leading integrated chemistry, manufacturing, and controls (CMC) facilities in the world for a cell therapy company, which when fully built out in China, will have the manufacturing capacity to support the treatment of 10,000 cancer and 10,000 knee osteoarthritis patients per year. With a healthy balance sheet and an efficient deployment of capital that will enable CBMG to execute on its clinical developments over the next twelve months, we are well equipped to further our clinical trials including the addition of new cancer indications by adding more top cancer centers in China for DLBCL and ALL trials using our C-CAR011 product. As a reminder, each year China has approximately five million new cancer patients, which far surpasses the U.S. We are pleased with our CAR-T patient screening and trial enrollment progress thus far and are on track to share our topline clinical data in the fourth quarter of this year as it becomes available. We look forward to evaluating new interests in expanding our clinical development and CAR-T partnerships with leading hospitals in major cities in China."

2017 Business & Technology Highlights

In 2016, commenced patient enrollment in China for its CARD-1 ("CAR-T Against DLBCL") Phase I clinical trial utilizing CBMG’s optimized proprietary C-CAR011 construct of CD19 chimeric antigen receptor T-cell (CAR-T) therapy for the treatment of patients with refractory Diffuse Large B-cell Lymphoma (DLBCL);
Announced addition of second clinical trial site for its Chimeric Antigen Receptor T-cell (CAR-T) Phase I Clinical Trial for its CARD-1 Trial in patients with refractory Diffuse Large B-cell Lymphoma (DLBCL) in Shanghai with Tongji Hospital;
Commenced CALL-1 ("CAR-T against Acute Lymphoblastic Leukemia") Phase I clinical trial in China utilizing its optimized proprietary C-CAR011 construct of CD19 chimeric antigen receptor T-cell (CAR-T) therapy for the treatment of patients with relapsed or refractory (r/r) CD19+ B-cell Acute Lymphoblastic Leukemia (ALL);
Received the first disbursement of $1.2 million in the $2.29 million grant by California Institute for Regenerative Medicine (CIRM), California’s stem cell agency, to support pre-clinical studies of AlloJoinTM, CBMG’s "Off-the-Shelf" Allogeneic Human Adipose-derived Mesenchymal Stem Cells for the treatment of Knee Osteoarthritis in the United States;
Completed expansion of its 30,000 square foot facility in Huishan High Tech Park in Wuxi, China, with 20,000 square feet of the Wuxi GMP facility dedicated to advanced stem cell culturing, centralized plasmid and viral vector production, cell banking and development of reagents;
Began construction of a new GMP facility in "Pharma Valley" in Shanghai Zhangjiang High-Tech Park, which will consist of 40,000 square feet dedicated to advanced cell manufacturing;
Established a strategic research collaboration with GE Healthcare Life Sciences China to co-develop certain high-quality industrial control processes in Chimeric Antigen Receptor T-cell (CAR-T) and stem cell manufacturing, and form a joint laboratory within CBMG’s new Shanghai Zhangjiang GMP-facility dedicated to the joint research and development of a functionally integrated and automated immunotherapy cell preparation system.

About PMBCL & FL
Primary mediastinal B-cell lymphoma (PMBCL) belongs to the group of aggressive diffuse large B-cell lymphomas. Its molecular signature and clinical features resemble classical Hodgkin lymphoma. It constitutes approximately 2 % to 4 % of all non-Hodgkin lymphomas (around 6 % of diffuse large B-cell lymphomas (DLBCL)).
Follicular lymphoma (FL) is the most common indolent (slow- growing) form of NHL, accounting for approximately 12 percent of all B-cell NHLs.