On May 15, 2019 AVEO Oncology (NASDAQ: AVEO) reported two poster presentations at the upcoming American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting being held May 31-June 4, 2019 in Chicago, Illinois (Press release, AVEO, MAY 15, 2019, View Source [SID1234536352]).

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Presentation Details

Title: Efficacy and safety of tivozanib in recurrent, platinum-resistant ovarian, fallopian tube or primary peritoneal cancer
First Author: Wendy M. Swetzig, PhD, Northwestern University Feinberg School of Medicine
Abstract Number: 5538
Poster Session: Gynecologic Cancer
Poster Board: 361
Date and Time: Saturday, June 1, 2019, 1:15-4:15 PM CT
Location: Hall A

Title: TIVO-3: Subgroup analysis of progression-free survival of tivozanib compared to sorafenib in subjects with refractory advanced renal cell carcinoma (RCC)
First Author: Camillo Porta, MD, Associate Professor, Department of Internal Medicine, University of Pavia and Division of Translational Oncology, IRCCS Maugeri, Italy
Abstract Number: 4572
Poster Session: Genitourinary (Nonprostate) Cancer
Poster Board: 398
Date and Time: Monday, June 3, 2019, 1:15-4:15 PM CT
Location: Hall A

About Tivozanib (FOTIVDA)

Tivozanib (FOTIVDA) is an oral, once-daily, vascular endothelial growth factor (VEGF) tyrosine kinase inhibitor (TKI) discovered by Kyowa Hakko Kirin and approved for the treatment of adult patients with advanced renal cell carcinoma (RCC) in the European Union plus Norway and Iceland. It is a potent, selective and long half-life inhibitor of all three VEGF receptors and is designed to optimize VEGF blockade while minimizing off-target toxicities, potentially resulting in improved efficacy and minimal dose modifications.1,2 Tivozanib has been shown to significantly reduce regulatory T-cell production in preclinical models3 and has demonstrated synergy in combination with nivolumab (anti PD-1) in a Phase 1/2 study in RCC. Tivozanib has been investigated in several tumor types, including renal cell, hepatocellular, colorectal and breast cancers. In addition, a new formulation of tivozanib is in pre-clinical development for the treatment of age-related macular degeneration.

On May 15, 2019 Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), reported positive data from the Phase I/II STARTRK-NG study, evaluating the investigational medicine entrectinib in children and adolescents with recurrent or refractory solid tumors with and without neurotrophic tyrosine receptor kinase (NTRK), ROS1 or anaplastic lymphoma kinase (ALK) gene fusions (Press release, Genentech, MAY 15, 2019, View Source [SID1234536351]). The study showed entrectinib shrank tumors (objective response rate; ORR) in all children and adolescents who had NTRK, ROS1 or ALK fusion-positive solid tumors (11 of 11 patients), including two patients achieving a complete response. Of the 11 patients, five patients with primary high-grade tumors in the central nervous system (CNS) had an objective response, including one patient with a complete response. The safety profile of entrectinib was consistent with that seen in previous analyses. Data will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago on Sunday, June 2, 2019, from 8:00 – 8:12 a.m. CDT (Abstract 10009), and was part of today’s official ASCO (Free ASCO Whitepaper) presscast.

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"We are encouraged by the results we have seen with entrectinib in children with pediatric and adolescent cancers, including those with tumors in the brain," said Sandra Horning, M.D., chief medical officer and head of Global Product Development. "The STARTRK-NG study underscores the importance of combining comprehensive genomic profiling with targeted therapies and supports our approach to providing people with personalized medicines developed specifically for their type of cancer."

Additional data for entrectinib across different tumor types and patient populations will also be presented at ASCO (Free ASCO Whitepaper), highlighting the company’s unique approach to personalized healthcare through advances in targeted therapies, diagnostics and data analytics:

Initial results from an integrated analysis of the Phase II STARTRK-2, Phase I STARTRK-1 and Phase I ALKA-372-001 trials evaluating the efficacy of entrectinib in adults with solid tumors and CNS metastases will be presented on Saturday, June 1, 2019, in a poster session from 3:00 – 4:30 p.m. CDT (Abstract 3017).

Results from a real-world data study evaluating time-to-treatment discontinuation and progression-free survival as endpoints for comparative efficacy analysis of clinical trials of entrectinib and crizotinib for the treatment of people with ROS1-positive non-small cell lung cancer (NSCLC) will be presented during a poster session on Sunday, June 2, 2019, from 8:00 – 11:00 a.m. CDT (Abstract 9070).

The FDA recently granted Priority Review for entrectinib for both the treatment of pediatric and adult patients with NTRK fusion-positive, locally advanced or metastatic solid tumors who have either progressed following prior therapies or as an initial therapy when there are no acceptable standard therapies, and for the treatment of people with metastatic ROS1-positive NSCLC. These NDAs are based on results from the integrated analysis of the Phase II STARTRK-2, Phase I STARTRK-1 and Phase I ALKA-372-001 trials, and data from the STARTRK-NG study. The FDA is expected to make a decision on approval by August 18, 2019.

About the STARTRK-NG study

STARTRK-NG is a Phase I/II open-label dose-escalation and expansion study evaluating the safety and efficacy of entrectinib in children and adolescent patients with no curative first-line treatment option, recurrent or refractory extracranial solid tumors or primary CNS tumors, with or without NTRK, ROS1 or ALK fusions. Response, assessed by Investigator, was classified as complete response (CR), partial response (PR), stable disease (SD) or progressive disease (PD) using Response Assessment in Neuro-Oncology (RANO) for CNS tumors, Response Evaluation Criteria in Solid Tumors (RECIST), and Curie score (CS) for neuroblastoma. The study enrolled 29 children and adolescents aged 4.9 months through 20 years (median age of 7 years) who had recurrent or refractory solid tumors, and 28 were evaluated for response. Of the 28 children and adolescents evaluated, 11 children were identified to have tumors with NTRK, ROS1 or ALK fusions and one with ALK F1174L-mutated neuroblastoma. A summary of the results are included below.

Complete responses were observed in two patients with tumors harboring NTRK and ALK fusions: one with an NTRK fusion-positive primary CNS tumor and one with an ALK fusion-positive inflammatory myofibroblastic tumor. Another complete response was observed in one neuroblastoma patient with an ALK F1174L mutation.
Partial responses were observed in nine patients, three unconfirmed at the time of the clinical cut-off date, across NTRK, ROS1 and ALK fusion-positive primary CNS (n=4) and extracranial (n=5) solid tumors.
Median duration of therapy for confirmed fusion-positive responders was 10.51 months (3.8 to 17.7 months), and median time to response was 1.89 months (1 to 1.9 months).
The safety profile of entrectinib was consistent with that seen in previous analyses. Treatment-related adverse events were most frequently National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Grade 1 or 2, leading to discontinuation in 6.9 percent of patients.

About NTRK fusion-positive cancer

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 signaling pathways involved in proliferation of certain types of cancer. NTRK gene fusions are tumor-agnostic, meaning they are present in tumors irrespective of site of origin. These fusions have been identified in a broad range of solid tumor types, including breast, cholangiocarcinoma, colorectal, gynecological, neuroendocrine, non-small cell lung, salivary gland, pancreatic, sarcoma and thyroid cancers.

About entrectinib

Entrectinib (RXDX-101) is an investigational, oral medicine in development for the treatment of locally advanced or metastatic solid tumors that harbor NTRK1/2/3 or ROS1 gene fusions. It is a selective 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. Entrectinib can block ROS1 and NTRK kinase activity and may result in the death of cancer cells with ROS1 or NTRK gene fusions. Entrectinib is being investigated across a range of solid tumor types, including breast, cholangiocarcinoma, colorectal, gynecological, neuroendocrine, non-small cell lung, salivary gland, pancreatic, sarcoma and thyroid cancers.

On May 15, 2019 X4 Pharmaceuticals, Inc. (Nasdaq:XFOR), a clinical-stage biopharmaceutical company focused on the development of novel therapeutics for the treatment of rare diseases, and The Leukemia & Lymphoma Society (LLS) reported a collaboration to accelerate the development of X4’s lead product candidate, mavorixafor (X4P-001) for the treatment of Waldenström’s macroglobulinemia (WM), a rare form of non-Hodgkin lymphoma (Press release, X4 Pharmaceuticals, MAY 15, 2019, View Source [SID1234536349]).

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Mavorixafor was selected for LLS’s Therapy Acceleration Program (TAP), a strategic initiative where LLS builds business alliances and collaborations with biotechnology companies and academic researchers to speed the development of new therapies for blood cancers. Under the collaboration, X4 will conduct a multi-national Phase 1/2 clinical trial to evaluate the safety and assess the preliminary anti-tumor activity of mavorixafor in combination with ibrutinib in WM patients. The trial is planned to commence this year. Lee Greenberger, Ph.D., chief scientific officer of LLS, will also serve as a member of an advisory board to X4, providing important strategy and partnership guidance throughout the trial.

"LLS’s selection of mavorixafor for TAP collaboration and investment reinforces its potential as a novel therapy for Waldenström’s macroglobulinemia. Approximately 30 to 40 percent of WM patients have a CXCR4 mutation, and a number of these patients do not respond well to current therapies," said Paula Ragan, Ph.D., president and chief executive officer of X4 Pharmaceuticals. "We look forward to working closely with Dr. Greenberger and the LLS TAP team to gain valuable data and insights throughout the upcoming clinical trial as we work to bring a new therapeutic option to patients with this rare form of cancer."

Mavorixafor is a first-in-class, oral, small molecule allosteric antagonist of the chemokine receptor CXCR4 and is designed to address certain rare primary immunodeficiency diseases and certain cancers, including lymphomas, in which genetic mutations in CXCR4 create abnormal trafficking of white blood cells and play a role in disease process.

"Through TAP, LLS is committed to advancing the development of promising investigational therapies that we believe have potential to improve standards of care for patients, especially in disease areas with high unmet medical need, such as Waldenström’s macroglobulinemia," said Dr. Greenberger. "Mavorixafor has demonstrated early promise in other disease areas with CXCR4 mutations, including solid tumors, and its potential application among CXCR4-mutant WM patients makes it an excellent fit and an important asset within our program as we work with innovative companies like X4 to uncover and develop cutting-edge therapies for patients with blood cancers."

About Waldenström’s Macroglobulinemia
Waldenström’s macroglobulinemia (WM) is a rare form of non-Hodgkin lymphoma and B-cell lymphoproliferative disorder. According to the American Cancer Society, approximately three per one million people are diagnosed each year, including 1,400 new cases in the United States annually. Recent advancements in whole-genome sequencing have identified genetic mutations in the disease similar to WHIM syndrome, a rare congenital primary immunodeficiency characterized by warts, hypogammaglobulinemia, infection and myelokathexis. Approximately 30 to 40 percent of WM cases express mutations in the CXCR4 gene in the cancer cells. In WM, somatic mutations of CXCR4 are associated with active tumor cells and possible drug resistance, including resistance to anti-CD20 monoclonal antibodies and Burton tyrosine kinase (BTK) inhibitors, such as ibrutinib, the current standard of care. WM patients with this somatic mutation have a dramatically reduced median progression-free survival, or mPFS, of approximately two years, whereas patients without the mutation have a mPFS of well over five years.

About the Therapy Acceleration Program
The Leukemia & Lymphoma Society’s Therapy Acceleration Program (TAP) identifies and funds innovative projects related to therapies, supportive care or diagnostics that have the potential to change the standard of care for patients with blood cancer, especially in areas of high unmet medical need. TAP funding assists both clinical investigators and companies in gaining critical clinical proof of concept data that better enables them to obtain the resources they need or a partner to complete the testing, registration and marketing of new treatments, supportive care and diagnostics for leukemia, lymphoma and myeloma. TAP funding is different from the traditional grant at LLS. The TAP review process is separate from the grant process and LLS’s TAP staff play an active advisory role and closely monitor each approved project. To learn more about how TAP works, please click here.

On May 15, 2019 The SanBio Group (SanBio Co., Ltd. and SanBio, Inc.)(TOKYO:4592), a scientific leader in regenerative medicine for neurological disorders, reported the appointment of Bijan Nejadnik, M.D., as its new Chief Medical Officer in charge of development and regulatory affairs (Press release, Sanbio, MAY 15, 2019, View Source [SID1234536348]).

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Dr. Nejadnik has held many important positions, including key roles at Johnson & Johnson Services, Inc., a major pharmaceutical company, as well as pioneering clinical programs in the pharmaceutical and biotech industry, such as Jazz Pharmaceuticals, Inc., Galena Biopharma, Inc., and Eureka Therapeutics, Inc. At Johnson & Johnson, he was involved in the development of immunological and oncology therapeutics, including the development of infliximab (trade name: Remicade) used for the treatment of rheumatic and other autoimmune disorders. At Jazz Pharmaceuticals, he submitted Biologics License Applications (BLA) for multiple oncology therapeutics and obtained approval from the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA). As Chief Medical Officer at Galena Biopharma, he led the research of immunotherapy for multiple cancers and research in adoptive T-cell therapy in combination with checkpoint inhibitor drugs. As Chief Medical Officer at Eureka Therapeutics, he was involved in the development of genetically modified T-cells and obtained FDA approval for several IND and launched the clinical trials. Dr. Nejadnik thus is a leading figure in clinical development, with diverse experience.

In Japan, using the conditional and term-limited authorization system for regenerative medicine products under the Revised Pharmaceutical Affairs Act, the SanBio Group is currently working toward applying for manufacturing and marketing approval of SB623 for the treatment of chronic motor deficit resulting from traumatic brain injury (TBI) during the fiscal year ending January 31, 2020 (February 1, 2019–January 31, 2020). Further, it plans to initiate a Phase 3 clinical trial of SB623 for the same indication by the end of the fiscal year ending January 31, 2020. Dr. Nejadnik will work on leading these clinical developments for obtaining approval, and in the long term, will work to promote global expansion of the Group.

Damien Bates, M.D., PhD, FRACS, MBA, who until today has served as the Group’s Chief Medical Officer and Head of Research, will continue to contribute to the global development of the regenerative cell medicine SB623 as the Group’s senior advisor.

Accepting the position of Chief Medical Officer, Dr. Nejadnik commented, "I have been involved in the development of many new pharmaceutical drugs with a view to meeting unmet medical needs that lack effective treatment. SanBio is a pioneer in drug development for patients suffering central nervous system disorders. I understand that the development of SB623 has already come close to the stage of product launch in Japan; taking advantage of my past experiences, I will do my best to bring SB623 to patients worldwide as soon as possible."

"I am very pleased to welcome Dr. Bijan Nejadnik as SanBio’s new Chief Medical Officer," said Keita Mori, CEO of SanBio, Co., Ltd. "In Japan, the development program for SB623 as a treatment for chronic motor deficit resulting from TBI has already advanced to a stage that we are preparing for obtaining the approval for launch under the conditional and term-limited authorization system for regenerative medicine products. Going forward, we aim to bring the product to the global market. We expect that Dr. Nejadnik will receive the baton from Dr. Damien Bates and, by drawing on his extensive experience, will make further contributions to the clinical development and submission process towards the approval of SB623."

On May 15, 2019 Mateon Therapeutics, Inc. (OTCQB:MATN), a biopharmaceutical company developing investigational drugs for the treatment of orphan oncology indications, reported financial results for the first quarter of 2019 (Press release, Mateon Therapeutics, MAY 15, 2019, View Source [SID1234536347]).

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For the three months ended March 31, 2019, Mateon reported a net loss of $0.6 million, compared to a net loss of $0.8 million for the three months ended March 31, 2018. As of March 31, 2019, Mateon had cash of $0.2 million. On April 22, 2019, Mateon merged with Oncotelic, Inc., a clinical-stage cancer immunotherapy company focused on TGF-β RNA therapeutics. Because the first quarter of 2019 ended prior to the date of the merger, the financial results reported today do not include any financial results for Oncotelic.

"I am excited about the growth potential for our newly combined company – we have a promising pipeline of next-generation immunotherapies targeting several significant cancer markets where there are significant unmet medical needs," said Vuong Trieu, Ph.D., Chairman and Chief Executive Officer of Mateon and co-founder of Oncotelic. "Earlier this month, we presented two posters on our proprietary self-immunization protocol at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting. These posters showed OT-101’s ability to reactivate immune cells directly around the cancer tissue. Our goal is to advance this candidate in the clinic and have an approvable anti-cancer drug within a few years."

About OT-101
The company’s lead product candidate, OT-101, is being developed as a broad-spectrum anti-cancer drug that can also be used in combination with other standard cancer therapies to establish an effective multi-modality treatment strategy for difficult-to-treat cancers, including high-grade gliomas and pancreatic cancer. The company plans to initiate phase 3 clinical trials for OT-101 in both high-grade glioma and pancreatic cancer. During phase 2 clinical trials in pancreatic cancer, melanoma, and colorectal cancers (Study P001) and high-grade gliomas (Study G004), meaningful clinical benefits were observed and OT-101 exhibited a favorable safety profile. These clinical benefits included long term survival and meaningful tumor reduction. Both partial and complete responses have been observed in the G004 Phase 2 clinical trial of OT-101 as a single agent in patients with aggressive brain tumors and in patients with treatment failure pancreatic cancers.

About the company’s Self-Immunization Protocol (SIP©)
The company’s self-immunization protocol (SIP©) is based on novel and proprietary sequential treatment of cancers with OT-101 (an antisense against TGF-β2) and chemotherapies. This sequential treatment strategy is aimed at achieving effective self-immunization against a patients’ own cancer, resulting in robust therapeutic immune response and consequently better control of the cancer and improved survival. Prolonged states of being cancer-free have been observed in some patients with the most aggressive forms of cancer, raising a renewed hope for a potential cure. The use of OT-101 lifts the suppression of the patient’s immune cells around the cancer tissue, providing the foundation for an effective initial priming, which is critical for a successful immune response. The subsequent chemotherapy results in the release of neoantigens that result in a robust boost of the immune response. The company believes that a rational combination of the Oncotelic SIP platform with immune-modulatory drugs like interleukin 2 (IL-2) and/or immune checkpoint inhibitors has the potential to help achieve sustained and robust immune responses in patients with the most difficult-to-treat forms of cancer.

The mechanism of action of SIP© presentations at the AACR (Free AACR Whitepaper) Annual Meeting are now available for viewing at www.oncotelic.com:

Abstract Number: 3968 / 24: OT-101/Chemotherapy – A novel mechanism of action (MOA) in pancreatic cancer immunization therapy.
Abstract Number: 5029 / 23: OT-101/Chemotherapy – A novel mechanism of action (MOA) in gliomas immunization therapy.