On December 15, 2017 Ohr Pharmaceutical, Inc. (Nasdaq: OHRP), a clinical-stage pharmaceutical company developing novel therapies for ophthalmic disease, reported financial results for its fiscal year ended September 30, 2017 (Press release, Ohr Pharmaceutical, DEC 15, 2017, View Source [SID1234522662]).

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"Our primary focus at Ohr continues to be the successful completion of the MAKO study, our ongoing clinical trial being conducted to evaluate the efficacy and safety of Squalamine in combination with Lucentis for treatment-naive patients with the wet form of age-related macular degeneration (wet-AMD)," said Dr. Jason Slakter, chief executive officer of Ohr Pharmaceutical. "The MAKO study enrolled a targeted population identified in the prior Phase 2 study which we believe is best suited for Squalamine combination therapy and is consistent with the mechanism of action of Squalamine and the vascular biology of the disease. We remain on track to receive and report top-line efficacy data from the MAKO study in early calendar 2018."

Corporate Highlights for the Fiscal Year Ended September 30, 2017

● Continued progress of the ongoing clinical study in wet-AMD, entitled the MAKO study. The study is a multi-center, randomized, double-masked, placebo controlled clinical trial designed to investigate Ohr’s lead candidate Squalamine in combination with Lucentis in wet-AMD.

○ Topline data from the study are expected in early calendar year 2018.

○ In April 2017, the Company made a strategic decision to amend the MAKO study from the original agreed-upon design to enable efficacy analyses at an earlier date than originally anticipated.

○ More than 200 patients were enrolled in the MAKO study. Patients received monthly Lucentis and either topical Squalamine or placebo eye drops twice daily. The primary endpoint is an assessment of visual acuity gain at nine months.

○ The MAKO study prospectively enrolled the patient population identified from the prior Phase 2 IMPACT study that had the greatest potential to benefit from Squalamine combination therapy.

● Raised approximately $19.5 million in combined net proceeds, after deducting placement agent fees and offering expenses payable, from two public offerings of common stock and warrants that closed on December 13, 2016 and on April 10, 2017.

● In May, the Company appointed the Honorable Mike Ferguson as a Director and Chairman of the Board of Directors.

○ Mr. Ferguson served for nearly a decade in the House of Representatives. As Vice Chairman of the House health subcommittee, he led policy reforms including the creation of the Medicare Part D prescription drug benefit and pharmaceutical and medical device user fee reauthorizations. He is also Senior Advisor and Leader of the Federal Policy Team at Baker Hostetler LLP, one of the nation’s largest law firms.

Financial Results for the Fiscal Year ended September 30, 2017

● For the year ended September 30, 2017, the Company reported a net loss of approximately $23.8 million, or ($0.53) per share, compared to a net loss of approximately $25.8 million, or ($0.82) per share, in the same period of 2016.

● For the year ended September 30, 2017, total operating expenses were approximately $23.8 million, consisting of $5.3 million in general and administrative expenses, $17.4 million of research and development expenses, and $1.2 million in depreciation and amortization. This compares to total operating expenses of approximately $24.6 million, consisting of approximately $7.7 million in general and administrative expenses, $16.5 million in research and development expenses, and $1.2 million in depreciation and amortization in the same period of 2016.

● At September 30, 2017, the Company had cash and cash equivalents of approximately $12.8 million, compared to cash and cash equivalents of approximately $12.5 million at September 30, 2016.

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On December 15, 2017 Cancer Research UK reported the signing of a five-year drug-discovery collaboration between its subsidiary, Cancer Research Technology (CRT), and Celgene Corporation, to discover, develop and commercialise new anti-cancer treatments (Press release, Cancer Research Technology, DEC 15, 2017, View Source [SID1234523153]).

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This arrangement represents an expansion of Cancer Research Technology’s theme-based translational model that now encompasses six industry partnerships, including this new collaboration with Celgene.

The collaboration is centred on mRNA translation, the cellular process of assembling proteins, which is a promising area of research with the potential to produce treatments that can target a fundamental characteristic of cancer cells.

Dr Iain Foulkes, Cancer Research Technology’s CEO, said: "This bold and exciting collaboration between one of industry’s leading innovators, Celgene, and CRT is part of our theme-based drug discovery approach and helps leverage our understanding of cancer biology and the needs of patients to drive the most promising discoveries into the clinic.

"This is our largest drug discovery collaboration to date and represents a major endorsement of the reputation and scale of our capacity and expertise in both drug discovery and clinical development by a leading industry partner."

Cancer Research Technology will lead drug discovery R&D activity and can progress clinical candidates through phase one trials.

Under the terms of the agreement*, Celgene will pay an upfront fee to Cancer Research Technology, and have the option to secure US rights to projects resulting from the collaboration, subject to the payment of additional option fees. Celgene will also have the option to secure Global rights to such projects at the end of phase one clinical trials, subject to the payment of additional option fees.

Cancer Research Technology can receive downstream royalties and development milestones from licensed programs.

On December 15, 2017 H3 Biomedicine Inc., a clinical stage biopharmaceutical company specializing in the discovery and development of precision medicines for oncology and a member of Eisai’s global Oncology Business Group, reported that data on one of its clinical programs has been published in the current issue of Cancer Research (Press release, H3 Biomedicine, DEC 15, 2017, View Source [SID1234522668]). The title of the paper, "H3B-6527 is a Potent and Selective Inhibitor of FGFR4 in FGF19-driven Hepatocellular Carcinoma," was composed by H3 scientists with Anand Selvaraj, PhD, Senior Investigator as lead scientist on the study.

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"We are encouraged by the pre-clinical data of H3B-6527 and the article published in Cancer Research highlights the strength of H3’s unique drug discovery platform that successfully targets specific drivers of hepatocellular carcinoma," said Markus Warmuth, M.D., President and CEO of H3 Biomedicine. "We feel this new research continues to underscore our commitment to FGFR4 inhibition as a novel treatment approach in this therapeutic area and we look forward to advancing this program."

The data were recently presented at the International Liver Cancer Association annual meeting held in Seoul, Republic of Korea.

The publication reports that an oral dosing of H3B-6527 in mice led to dose-dependent pharmacodynamic modulation of FGFR4 signaling and tumor regression in FGF19 altered HCC xenograft models. These data served as proof-of-concept for the approach and led to the clinical introduction of H3B-6527.

The data published in Cancer Research show promising pre-clinical activity of H3B-6527 in hepatocellular carincoma models," said Pete Smith, Ph.D., Chief Scientific Officer, H3 Biomedicine. "H3B-6527 is currently in a Phase I clinical trial and we look forward to discussing the progress of the trial in the coming months."

About H3B-6527
H3B-6527 is a selective, orally bioavailable, and potent inhibitor of fibroblast growth factor receptor 4 (FGFR4) that is being investigated for the treatment of advanced hepatocellular carcinoma (HCC). Aberrant signaling through the FGF19-FGFR4 axis has been shown to drive tumor development and dependency in pre-clinical models of HCC. H3B-6527 has shown sustained tumor regressions in several preclinical models of HCC where FGF19-FGFR4 signaling is aberrantly activated. The safety and preliminary efficacy of H3B-6527 will be explored in patients that are selected using a companion diagnostic that identifies HCC with activated FGF19-FGFR4 pathway activity. H3B-6527 is currently in Phase 1 clinical trials. For more information on the clinical trial, please click here.

On December 15, 2017 Endocyte, Inc. (NASDAQ:ECYT), a biopharmaceutical company developing targeted therapeutics for personalized cancer treatment, reported that it has been selected for addition to the NASDAQ Biotechnology Index (NASDAQ:NBI) (Press release, Endocyte, DEC 15, 2017, View Source [SID1234522667]). Endocyte’s addition to the NBI will become effective prior to market open on Monday, Dec, 18, 2017.

The NASDAQ Biotechnology Index (NBI) contains securities of NASDAQ-listed companies that meet certain eligibility criteria, and are classified according to the Industry Classification Benchmark as either Biotechnology or Pharmaceuticals. These requirements include minimum market capitalization, and average daily trading volume. For more information about the NASDAQ Biotechnology Index visit View Source

On December 15, 2017 Hutchison China MediTech Limited ("Chi-Med") (AIM/Nasdaq: HCM) has reported that it initiated the United States Phase I bridging clinical trial of fruquintinib. Fruquintinib is a highly selective and potent oral inhibitor of vascular endothelial growth factor receptors ("VEGFR") 1, 2 and 3, that has met its primary endpoint in several Phase II and III clinical trials in China for the treatment of colorectal, lung and gastric cancers (Press release, Hutchison China MediTech, DEC 15, 2017, http://www.chi-med.com/chi-med-initiates-fruquintinib-u-s-clinical-trials/ [SID1234522660]). The clinical study in the U.S. is a multi-center, open-label, Phase I clinical study to evaluate the safety, tolerability and pharmacokinetics of fruquintinib in U.S. patients with advanced solid tumors. The first drug dose was administered earlier this month. Additional details about this study may be found at clinicaltrials.gov, using identifier NCT03251378.

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About Fruquintinib Development in China
Colorectal cancer: The China Food and Drug Administration ("CFDA") acknowledged acceptance of the New Drug Application ("NDA") for fruquintinib for the treatment of patients with advanced colorectal cancer ("CRC") in June 2017. Fruquintinib was subsequently awarded priority review status in view of its significant clinical value, according to a CFDA announcement in September 2017. The NDA is supported by data from the successful FRESCO study, a Phase III pivotal registration trial of fruquintinib in 416 patients with CRC in China, which was highlighted in an oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting held on June 5, 2017 (clinicaltrials.gov identifier NCT02314819). The FRESCO study followed an initial Phase I trial in 40 solid tumor patients, a Phase Ib study in 62 CRC patients, and a Phase II clinical trial in 71 CRC patients.

Lung cancer: Fruquintinib is being studied in a Phase III pivotal trial in approximately 520 third-line non-small cell lung cancer ("NSCLC") patients, known as the FALUCA study (clinicaltrials.gov identifier NCT02691299), following a Phase II clinical trial in 91 third-line NSCLC patients. Fruquintinib is concurrently being studied in a Phase II study in combination with Iressa (gefitinib) in first-line setting for patients with advanced or metastatic NSCLC (clinicaltrials.gov identifier NCT02976116).

Gastric cancer: In October 2017, Chi-Med initiated a pivotal Phase III clinical trial of fruquintinib in combination with Taxol (paclitaxel), known as the FRUTIGA study, for the treatment of over 500 patients with advanced gastric or gastroesophageal junction (GEJ) adenocarcinoma (clinicaltrials.gov identifier NCT03223376).

In China, fruquintinib is jointly developed with Eli Lilly and Company.

About Fruquintinib
Fruquintinib (HMPL-013) is a highly selective small molecule drug candidate that has been shown to inhibit VEGFR 24 hours a day via an oral dose, with lower off-target toxicities compared to other targeted therapies. Its tolerability, along with its clean drug-drug interaction profile demonstrated to date, may enable rational combination with other cancer therapies such as chemotherapy and other targeted therapies, which are being studied in our ongoing clinical trials of fruquintinib.

At an advanced stage, tumors secrete large amounts of vascular endothelial growth factor ("VEGF"), a protein ligand, to stimulate formation of excessive vasculature (angiogenesis) around the tumor to provide greater blood flow, oxygen, and nutrients to the tumor. VEGF and VEGFR play pivotal roles in tumor-related angiogenesis, and fruquintinib inhibits the VEGF/VEGFR pathway. This represents an important therapeutic strategy in blocking the development of new blood vessels essential for tumors to grow and invade.