On March 31, 2017 Mateon Therapeutics, Inc. (OTCQX:MATN), a biopharmaceutical company developing vascular disrupting agents (VDAs) for the treatment of orphan oncology indications, reported that updates on several of its preclinical programs are being presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in Washington, D.C (Press release, Mateon Therapeutics, MAR 31, 2017, View Source [SID1234518365]).

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"These abstract presentations show the breadth of some of our early preclinical development programs," stated William D. Schwieterman, M.D., Mateon’s President and Chief Executive Officer. "As this work matures, we look forward to it complementing our current core clinical programs in platinum-resistant ovarian cancer and acute myeloid leukemia."

Poster presentations by Mateon and/or its collaborators at the AACR (Free AACR Whitepaper) annual meeting are as follows:

Abstract #2952 – The novel Cathepsin L/K inhibitors KGP94 and KGP207 prevent M0 to M2 macrophage differentiation and macrophage mediated pro-tumor functions.
Section: Tumor Microenvironment 3
Date and Time: Monday, April 3, 2017, 1:00 p.m. – 5:00 p.m. Eastern Time

Abstract #3203 – Targeting tumor hypoxia with prodrug conjugates of potent small molecule inhibitors of tubulin polymerization
Section: Novel Molecular Targets 2
Date and Time: Tuesday, April 4, 2017, 8:00 a.m. – 12:00 p.m. Eastern Time

Abstract #4899 – The small molecule Cathepsin L and K inhibitor KGP-94 impairs the metastatic phenotype of osteosarcoma cells
Section: Therapeutic Intervention of Cancer and Metastases
Date and Time: Tuesday, April 4, 2017, 1:00 p.m. – 5:00 p.m. Eastern Time

The above abstracts have been published and can be viewed on the AACR (Free AACR Whitepaper) Annual Meeting website.

On March 31, 2017 MacroGenics, Inc. (NASDAQ: MGNX), a clinical-stage biopharmaceutical company focused on discovering and developing innovative monoclonal antibody-based therapeutics for the treatment of cancer, as well as autoimmune disorders and infectious diseases, reported the presentation of eight nonclinical posters based on its molecules and antibody-based technologies at the 2017 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Washington, D.C (Press release, MacroGenics, MAR 31, 2017, View Source [SID1234518363]). Three of these posters are being presented by the Company’s collaboration partners, Janssen Research & Development, LLC and ImmunoGen, Inc.

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"MacroGenics’ record of antibody-based therapeutic innovation continues," said Scott Koenig, M.D., Ph.D., President and CEO of MacroGenics. "Beyond our broad, growing pipeline of clinical assets across immuno-oncology and autoimmune disorders today, we have maintained our investment in preclinical research to yield our future product candidates for delivering therapeutics to patients in need. The posters featured at AACR (Free AACR Whitepaper) reflect advances in our DART and TRIDENT multi-specific platform technologies, including the ability to target multiple checkpoints with a single recombinant molecule and T cell-mediated cytotoxic mechanisms as well as the deployment of multiple linker-drug conjugate technologies with novel cancer targets. I’m very excited about the potential for each of these programs."

MacroGenics AACR (Free AACR Whitepaper) 2017 Poster Presentations

Each of the posters featured at AACR (Free AACR Whitepaper) relating to a MacroGenics-developed molecule are described below and after their presentation at AACR (Free AACR Whitepaper), may be accessed under "Events & Presentations" in the Investors section of the Company’s website at View Source

B7-H3-targeted Antibody-drug Conjugate: "Preclinical Development of a Duocarmycin-Based Antibody-Drug Conjugate (ADC) Targeting B7-H3 for Solid Cancer" (presentation date is Sunday, April 2 from 1:00 — 5:00pm ET; poster section 2, board #23).
ADAM9 ADC: "ADAM9: Target Validation, Antibody Discovery and Preclinical Data Supporting ADAM9 as an Antibody-Drug Conjugate Therapeutic Target for Solid Tumors" (presentation date is Sunday, April 2 from 1:00 — 5:00pm ET; poster section 2, board #19).
ADAM9 ADC: "Novel Antibody-Drug Conjugates Targeting ADAM9-expressing Solid Tumors Demonstrate Potent Preclinical Activity" (presentation date is Sunday, April 2 from 1:00 — 5:00pm ET; poster section 2, board #18). This poster is being presented by ImmunoGen, Inc.
CD137 DART: "Tumor-Antigen Expression-Dependent Activation of the CD137 Costimulatory Pathway by Bispecific DART Proteins" (presentation date is Tuesday, April 4 from 8:00am — 12:00pm ET; poster section 26, board #15).
PD-1 x CTLA-4 DART: "Co-targeting PD-1 and CTLA-4 Inhibitory Pathways with Bispecific DART and TRIDENT Molecules" (presentation date is Tuesday, April 4 from 8:00am — 12:00pm ET; poster section 26, board #10).
Duvortuxizumab (MGD011): "Potent Antitumor Activity of Duvortuxizumab, a CD19 x CD3 DART Molecule, in Lymphoma Models" (presentation date is Tuesday, April 4 from 8:00am — 12:00pm ET; poster section 26, board #9). This poster is being presented by Janssen.
Duvortuxizumab (MGD011): "Quantitative Prediction of Human Pharmacokinetics for Duvortuxizumab from Cynomolgus Monkey Data: a Translational Pharmacokinetic Modeling Approach" (presentation date is Tuesday, April 4 from 1:00 — 5:00pm ET; poster section 3, board #29). This poster is being presented by Janssen.
5T4 x CD3 DART: "A 5T4 x CD3 Bispecific DART Molecule with Extended Half-life for T-cell Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)s" (presentation date is Tuesday, April 4 from 1:00 — 5:00pm ET; poster section 26, board #23).

On March 31, 2017 Loxo Oncology, Inc. (Nasdaq:LOXO), a biopharmaceutical company innovating the development of highly selective medicines for patients with genetically defined cancers, reported that larotrectinib (LOXO-101) data will be presented at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2017 Annual Meeting taking place April 1 – 5, 2017, in Washington, DC (Press release, Loxo Oncology, MAR 31, 2017, View Source [SID1234518362]).

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The abstract and poster describe initial clinical data across the larotrectinib program for all patients with TRK fusion primary CNS cancers. The cases include three patients with glioblastoma: one patient treated under an expanded access protocol and two patients treated in the ongoing Phase 2 NAVIGATE trial. In the cases described, larotrectinib showed preliminary evidence of anti-tumor activity. The expanded access patient, described in detail in the abstract, had progressed through prior radiation, temozolomide and bevacizumab and demonstrated a brief mixed radiographic response on larotrectinib in the context of a molecularly complex tumor (select regions of the tumor harbored a TRK fusion, while others did not). The two patients treated in NAVIGATE, described in the poster, were enrolled following progression on chemoradiation and temozolomide (both cases) and bevacizumab (one case). The NAVIGATE patients remain on therapy at four months with radiographic evidence of treatment effect, as of a March 13, 2017 data cut-off date. Glioblastomas are highly aggressive CNS tumors, particularly in the post-bevacizumab setting where median overall survival is typically three to six months. Global regulatory discussions have established that primary CNS tumors, including glioblastoma, will not be included in the primary efficacy analysis dataset intended to support initial drug approval, though they are being enrolled in a dedicated treatment arm of NAVIGATE.

"Glioblastomas have historically defied rational targeted therapy approaches, so we are encouraged that larotrectinib may have a role in treating TRK fusion presentations of this devastating disease. We hope these early data lead to increased molecular profiling and referrals to appropriate clinical trials," said Josh Bilenker, M.D., chief executive officer of Loxo Oncology. "We knew it would be necessary to evaluate these patients separately from our primary efficacy dataset, which relies on a RECIST overall response rate primary endpoint. Given the limitations around response assessment in neuro-oncology, randomized survival trials remain the gold standard for evaluating new drugs in primary CNS cancers. However, patients with systemic tumors that have metastasized to the brain are included in our primary efficacy dataset, though they have been exceedingly uncommon. As illustrated in our adult Phase 1 dataset and this AACR (Free AACR Whitepaper) poster, we are pleased with larotrectinib’s ability to enter the CNS in a tolerated fashion and address TRK fusion tumors."

Abstracts from the AACR (Free AACR Whitepaper) Annual Meeting 2017 are available online on the conference website.

The details of the poster presentation are as follows:

Date: April 5, 2017, 8:00am – 12:00pm ET
Title: Potential role of larotrectinib (LOXO-101), a selective pan-TRK inhibitor, in NTRK fusion-positive recurrent glioblastoma
Session: Late-Breaking Research: Experimental and Molecular Therapeutics II
Abstract Code: LB-302
Poster Board Number: 10
Session Location: Poster Section 34

About Larotrectinib (LOXO-101)
Larotrectinib (LOXO-101) is a potent, oral and selective investigational new drug in clinical development for the treatment of patients with cancers that harbor abnormalities involving the tropomyosin receptor kinases (TRKs). Growing research suggests that the NTRK genes, which encode for TRKs, can become abnormally fused to other genes, resulting in growth signals that can lead to cancer in many sites of the body. In an ongoing Phase 1 clinical trial, larotrectinib has demonstrated encouraging preliminary efficacy. Larotrectinib is also being evaluated in the NAVIGATE global Phase 2 multi-center basket trial in patients with solid tumors that harbor TRK gene fusions, and the SCOUT Phase 1/2 trial in pediatric patients, including patients with advanced cancer, TRK gene fusions and infantile fibrosarcoma. Larotrectinib has been granted Breakthrough Therapy Designation and Rare Pediatric Disease Designation by the U.S. FDA. For additional information about the larotrectinib clinical trials, please refer to www.clinicaltrials.gov. Interested patients and physicians can contact the Loxo Oncology Physician and Patient Clinical Trial Hotline at 1-855-NTRK-123 or visit www.loxooncologytrials.com.

On March 31, 2017 Kite Pharma, Inc. (Nasdaq:KITE) reported that it has completed the rolling submission with the U.S. Food and Drug Administration (FDA) of the Biologics License Application (BLA) for axicabtagene ciloleucel (previously known as KTE-C19) as a treatment for patients with relapsed or refractory aggressive non-Hodgkin lymphoma (NHL) who are ineligible for autologous stem cell transplant (ASCT) (Press release, Kite Pharma, MAR 31, 2017, View Source [SID1234518361]).

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"Last month, we announced positive results from our ZUMA-1 pivotal trial with axicabtagene ciloleucel," said Arie Belldegrun, M.D., FACS, Chairman, President, and Chief Executive Officer of Kite. "We look forward to working closely with the FDA during the review of axicabtagene ciloleucel and the possibility of bringing this therapy to patients with aggressive NHL whose outlook is dismal with current therapy."

In December 2015 axicabtagene ciloleucel received Breakthrough Therapy Designation (BTD) by the FDA for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL). If approved, Kite plans to commercially launch axicabtagene ciloleucel in 2017. Kite is also planning a regulatory submission to the European Medicines Agency (EMA) for axicabtagene ciloleucel in 2017.

"The Leukemia & Lymphoma Society (LLS) applauds Kite for achieving this significant milestone and bringing this promising therapy closer to patients with lymphoma who desperately need new options," said Louis J. DeGennaro, Ph.D., LLS President and Chief Executive Officer. "LLS has supported companies that are working to dramatically change cancer treatment through the development of immunotherapy for the past two decades, and we immediately recognized the great opportunity to support Kite’s CAR-T program in 2015 through our Therapy Acceleration Program (TAP). Partnerships created through TAP, now in its tenth year, have the potential to bring several breakthrough therapies, such as axicabtagene ciloleucel, to patients in the coming year."

The ZUMA-1 pivotal trial for axicabtagene ciloleucel for the treatment of patients with aggressive NHL was supported in part by funding from LLS’ TAP.

About axicabtagene ciloleucel

Kite’s lead product candidate, axicabtagene ciloleucel, is an investigational therapy in which a patient’s T cells are engineered to express a chimeric antigen receptor (CAR) to target the antigen CD19, a protein expressed on the cell surface of B-cell lymphomas and leukemias, and redirect the T cells to kill cancer cells. Axicabtagene ciloleucel has been granted Breakthrough Therapy Designation status for diffuse large B-cell lymphoma (DLBCL), transformed follicular lymphoma (TFL), and primary mediastinal B-cell lymphoma (PMBCL) by the U.S. Food and Drug Administration (FDA) and Priority Medicines (PRIME) regulatory support for DLBCL in the EU.

On March 31, 2017 Diffusion Pharmaceuticals Inc. (NASDAQ:DFFN), a clinical stage biotechnology company focused on the development of novel small molecule therapeutics for cancer and other hypoxia-related diseases, reported financial results for the full year of 2016 and provided an overview of recent operational highlights (Press release, Diffusion Pharmaceuticals, MAR 31, 2017, View Source [SID1234518358]).

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David Kalergis, Chairman and Chief Executive Officer, stated: "The recently received proceeds from our private placement provide Diffusion with resources to move forward with our Phase 3 clinical program testing of trans sodium crocetinate (TSC) in newly diagnosed GBM patients, as well as advance its use in other indications. Diffusion staff, expert consultants and key opinion leaders continue to work together to craft the most cost-effective programs with optimal chances for success."

Corporate Highlights

In November 2016, our common stock was approved for listing, and commenced trading on the NASDAQ Capital Market;

In February 2017, data from our Phase 1/2 clinical trial evaluating the safety and efficacy of TSC in newly diagnosed glioblastoma multiforme was published in the print edition of the peer-reviewed Journal of Neurosurgery;

In March 2017, we completed an initial closing of our Series A Convertible Preferred Stock offering to accredited investors in private placement;

In March 2017, U.S. Patent 9,604,899 entitled "Bipolar Trans Carotenoid Salts and Their Uses" was granted by the United States Patent and Trademark Office. This patent expands the coverage of the therapeutic use of TSC and other related compounds to five hypoxia-related conditions including congestive heart failure, chronic renal failure, acute lung injury (ALI), chronic obstructive pulmonary disease (COPD) and respiratory distress syndrome (RDS).
Year End 2016 Results

Research and development expenses were $7.3 million during the year ended December 31, 2016, compared to $3.9 million during the year ended December 31, 2015. This increase was primarily a result of an additional $1.2 million in expenses related to animal toxicology studies, an increase of $1.0 million in active product ingredient manufacturing costs and an additional $0.5 million in costs related to the TSC pancreatic cancer program. Additionally, a $1.0 million noncash impairment charge was recognized for the abandonment of future development efforts related to the RES-440 IPR&D asset. Salaries and wages expense and stock compensation expense increased by $0.3 million and $0.4 million, respectively, due to an increase in headcount. The overall increase in research and development expense was offset by a $0.9 million decrease in spend related to GBM trials.

General and administrative expenses were $11.1 million for the year ended December 31, 2016, compared to $2.5 million for the year ended December 31, 2015. The increase was primarily attributable to $4.1 million in professional fees incurred in connection with preparing to operate as a public company, merger and transaction related fees and fees related to investment bank advisory services. There was also a $2.5 million noncash charge recognized upon settlement of a litigation matter. In addition, insurance expense increased by $0.8 million due to an increase in directors and officer’s insurance and salaries and wages and stock compensation expense increased by $0.4 million and $0.4 million, respectively, due to an increase in headcount.

Net loss was $18.0 million for the year ended December 31, 2016, compared to a net loss of $6.7 million for the year ended December 31, 2015. The increase in the net loss was primarily due to higher expenses associated with research and development and general and administrative costs.

Cash and cash equivalents were $1.6 million as of December 31, 2016, compared to $2.0 million as of December 31, 2015.