On June 20, 2018 Taiho Oncology, Inc. reported a Phase I study to examine the efficacy of TAS-120, an investigational irreversible pan-fibroblast growth factor receptor (FGFR) inhibitor as a potential treatment for patients with advanced solid tumors, including cholangiocarcinoma (CCA), who were previously treated with chemotherapy or other therapies including other FGFR inhibitors (Press release, Taiho, JUN 20, 2018, View Source [SID1234527409]). This is a presentation of updated clinical data for TAS-120 in cholangiocarcinoma, a rare cancer with limited treatment options. These data were presented as oral and poster presentations on Wednesday, June 20 at 2:50 PM CEST at the ESMO (Free ESMO Whitepaper) 20th World Congress on Gastrointestinal Cancer 2018 (ESMO-GI) in Barcelona, Spain, June 20 to 23.

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"We’re very pleased to see that TAS-120 has shown great promise among CCA patients in this study," said Robert Winkler, MD, senior vice president and head of Clinical Development, Taiho Oncology, Inc. "With such a broad range of tumor types with high unmet medical need, Taiho Oncology has remained committed to pioneering innovative research in specific areas that we hope will ultimately lead to an expanded range of potential therapies."

In this study of 45 patients with CCA harboring FGF/FGFR aberrations (e.g., FGFR2 gene fusions, FGF/FGFR mutations, amplifications and re-arrangements), TAS-120 demonstrated clinical activity in patients with CCA with FGFR2 gene fusions, and showed efficacy in patients who progressed on prior FGFR inhibitors. In 28 patients with FGFR2 gene fusions, 71 percent experienced tumor shrinkage and seven achieved confirmed partial response (cPR). The objective response rate in these patients was 25 percent, and 15 patients (54%) experienced stable disease as their best response, with seven still on treatment. The overall disease control rate was 79 percent. In 17 patients with other FGF/FGFR aberrations who received TAS-120, 18 percent achieved cPR. In 13 patients who had received prior FGFR inhibitors, 31 percent achieved cPR.

"These results are encouraging for this rare and difficult-to-treat cancer," said Milind Javle, MD, professor, Gastrointestinal Medical Oncology, The University of Texas MD Anderson Cancer Center, Houston, and investigator on the Phase I TAS-120 study. "Given the limited number of patients diagnosed each year with cholangiocarcinoma in the U.S., we recognize the sense of urgency for and importance of ongoing research to advance potential new therapies."

The most common treatment-related adverse events (AEs) of all grades in all patients were hyperphosphatemia (78%), increased aspartate aminotransferase (29%), dry skin (29%), diarrhea (27%) and dry mouth (27%). Grade ≥3 treatment-related AEs were reported in 51 percent of patients (51%); the most common was hyperphosphatemia in 22 percent of patients.

A Phase II study of TAS-120 in CCA patients has been initiated. The abstract for this presentation is available on the ESMO (Free ESMO Whitepaper)-GI website at View Source

In May 2018, the U.S. Food and Drug Administration Office of Orphan Drug Development granted Taiho’s investigational TAS-120 orphan drug status for the treatment of cholangiocarcinoma.

About FDA Orphan Drug Designation
The mission of the FDA Office of Orphan Products Development (OOPD) is to advance the valuation and development of products (drugs, biologics, devices, or medical foods) that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions. The Orphan Drug Designation program provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or preventive of rare diseases/disorders that affect more than 200,000 persons but are not expected to recover the costs of developing and marketing a treatment drug. The program has successfully enabled the development and marketing of more than 350 drugs and biologic products for rare diseases since 1983. In contrast, the decade prior to 1983 saw fewer than 10 such products come to market.

About Cholangiocarcinoma
Cholangiocarcinoma (CCA), also known as bile duct cancer, is not common. About 8,000 people in the United States are diagnosed with CCA each year. This includes both intrahepatic (inside the liver) and extrahepatic (outside the liver) cancers. CCA can occur at younger ages, but it is seen mainly in older people. The average age of people in the United States diagnosed with cancer of the intrahepatic bile ducts is 70, and for cancer of the extrahepatic bile ducts it is 72. The chances of survival for patients with CCA depend to a large extent on its location and how advanced it is when it is found.1

The main treatment for CCA is surgery. Radiation therapy and chemotherapy may be used if the cancer cannot be entirely removed with surgery and in cases where the edges of the tissues removed at the operation show cancer cells (also called a positive margin). Both stage III and stage IV cancers cannot be completely removed surgically. Currently, standard treatment options are limited to radiation, palliative therapy, liver transplantation, surgery, chemotherapy and interventional radiology.2

On June 20, 2018 Aminex Therapeutics, Inc., a clinical-stage drug development company focused on advancing a novel cancer immunotherapy, reported it received clearance from the Food and Drug Administration to initiate the first Phase 1 clinical trial of its immuno-oncology drug candidate AMXT 1501, a small molecule polyamine uptake inhibitor, and the approved drug DiFluoroMethylOrnithine (DFMO, eflornithine), a polyamine synthesis inhibitor (Press release, Aminex Therapeutics, JUN 20, 2018, View Source [SID1234527410]). This immunotherapy treatment will be evaluated in patients with a broad range of advanced solid tumor cancers.

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The open label, multicenter, dose-escalation trial will evaluate the safety and tolerability of AMXT 1501 alone, and in combination with DFMO, in more than 50 patients with advanced solid tumors. The trial will include a dose-ranging stage followed by an expansion phase. Evaluation of pharmacokinetics, pharmacodynamics and clinical response rates will be assessed. The trial is listed on the NIH clinical trial registry www.clinicaltrials.gov (Identifier: NCT03536728). NEXT Oncology of San Antonio, Texas is the first of four clinical sites planned for this trial. The first cancer patient received an initial dose on June 12, 2018.

"Initiating this first trial of our unique immunotherapy approach represents a significant milestone for Aminex Therapeutics. We are extremely excited to begin clinical evaluation of this investigational therapy that has demonstrated significant promise in many preclinical studies involving multiple animal solid tumor cancer models," said Jim Skaggs, Chief Executive Officer and Chairman of the Board of Aminex Therapeutics. "The primary goal of this trial is to determine the safety and appropriate dosage of AMXT 1501 and DFMO in combination. Efficacy indications will also be assessed. We are also pleased to announce the closure of a $10 million series B financing to support our clinical development efforts."

Aminex Therapeutics’ immunotherapy approach combines the company’s oral, small molecule polyamine uptake inhibitor, AMXT 1501, with the off-patent polyamine synthesis inhibitor, DFMO, approved for African sleeping sickness. AMXT 1501 is designed to work in conjunction with DFMO to constrain the production and uptake of polyamines – molecules found in high concentrations in cancer cells and linked to immune system suppression. Reducing polyamines and, in turn, decreasing myeloid-derived suppressor cells in the tumor microenvironment, allows for activation of the immune system to attack solid tumors. In preclinical studies involving multiple cancer models, AMXT 1501 + DFMO has been shown to be especially effective against cancers driven by MYC and RAS, two major oncogenes known to promote the development of many types of solid tumor cancers.

Aminex Therapeutics, which is led by an experienced management team, board of directors and scientific advisory board, announced the strengthening of its leadership team with the earlier appointments of Roger Ulrich, Ph.D. to its Board of Directors and Stephen B. Baylin, M.D., as member of the Scientific Advisory Board and clinical advisor. Dr. Ulrich is a founder of Calistoga Pharmaceuticals and formerly with Acerta Pharma, Merck, Abbott Laboratories and The Upjohn Company. He currently serves as director for Acerta Pharma, Remedy Pharmaceuticals and is a Senior Advisor to Frazier Healthcare Partners. Dr. Baylin is Virginia and D.K. Ludwig Professor of Oncology and Medicine, Co-Director of the Cancer Biology Division and Associate Director for Research Programs of the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins.

On June 20, 2018 SillaJen, Inc., (KOSDAQ:215600), a clinical-stage, biotherapeutics company focused on the development of oncolytic immunotherapy products for cancer, reported the first patient has been enrolled in REN026, the Phase 1b clinical trial of Pexa-Vec (pexastimogene devacirepvec) in combination with cemiplimab (REGN2810) for the treatment of renal cell cancer (RCC) (Press release, SillaJen, JUN 20, 2018, View Source [SID1234527430]). The first patient was enrolled in the United States, with expansion to sites in South Korea and Australia anticipated over the coming weeks.

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SillaJen is collaborating with Regeneron to evaluate Pexa-Vec, SillaJen’s lead clinical candidate, in combination with Regeneron’s cemiplimab, an anti-PD1 monoclonal antibody. The aim of the trial is to assess the safety and efficacy of the combination in patients with unresectable or metastatic renal cell carcinoma. The study will also investigate the immune modulating potential of Pexa-Vec given concurrently with checkpoint inhibitor therapy by evaluating multiple blood and tissue biomarkers.

"Given the initial activity seen with Pexa-Vec monotherapy and the potential of oncolytic viruses to enhance anti-tumor immunity, combining Pexa-Vec with cemiplimab is quite rational and has the promise to build upon the activity of checkpoint inhibitor therapy alone in RCC. This trial will not only assess the clinical activity of the two agents but allow us to assess in more depth the changes elicited in anti-tumor immunity following treatment by examining peripheral blood and tumor samples. This will give us proof of concept data that will help us expand this approach to other tumor types," stated James Burke, M.D., chief medical officer at SillaJen.

About Pexa-Vec and the SOLVE Platform
Pexa-Vec is the most advanced product candidate from SillaJen’s proprietary SOLVE (Selective Oncolytic Vaccinia Engineering) platform. The vaccinia strain backbone of Pexa-Vec has been used safely in millions of people as part of a worldwide vaccination program, and over 300 cancer patients have been treated with Pexa-Vec to date. Pexa-Vec was engineered to target common genetic defects in cancer cells by deleting their thymidine kinase (TK) gene, thus making Pexa-Vec dependent on the cellular TK expressed at persistently high levels in cancer cells. Pexa-Vec is also engineered to express granulocyte-macrophage colony stimulating factor (GM-CSF) protein. GM-CSF complements the cancer cell lysis of the product candidate, leading to a cascade of events resulting in tumor necrosis, tumor vasculature shutdown and sustained anti-tumoral immune attack. Pexa-Vec has been shown to be effective when delivered both intratumorally and systemically by intravenous administration. Pexastimogene devacirepvec (Pexa-Vec) is currently being evaluated in a worldwide Phase 3 clinical trial for advanced primary liver cancer, and more information can be found at: View Source

On June 19, 2018 Celyad (Euronext Brussels and Paris, and NASDAQ: CYAD), a clinical-stage biopharmaceutical company focused on the development of CAR-T cell therapies, reported the first Acute Myeloid Leukemia (AML) patient was dosed with the first injections at the third and final dose level of CYAD-01 in the hematological arm of the Phase 1 THINK trial (Press release, Celyad, JUN 19, 2018, View Source [SID1234527393]).

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"The observed tolerability profile of CYAD-01 to date as a stand-alone administration, along with the signs of clinical activity observed in relapsed refractory AML, highlight its potential for further development for both hematological cancers and solid tumors," said Dr. Christian Homsy, CEO of Celyad. "We have started administering the third dose level of CYAD-01 in a relapsed refractory AML patient in the THINK trial with no toxicity observed after the first injections. We look forward to completing the dose-escalation segment of the study, and, potentially an expansion phase, and reporting on the interim results of the THINK trial later this year at scientific congresses. In addition, we are happy to report that the first patient to receive a second cycle of CYAD-01 has been injected earlier this month."

The open label Phase 1 THINK trial is being conducted in the US and in Europe. The dose-escalation segment, conducted in parallel in solid cancers and in hematologic cancer groups, includes three dose levels: 3×108, 1×109 and 3×109 CYAD-01 cells per dose. At each dose level, the patients receive three successive administrations, two weeks apart, of CYAD-01 at the specified dose.

We expect that a total of three AML patients will receive the third and final, highest dose level. To date in the THINK trial, CYAD-01 has already shown signs of clinical activity at lower doses in AML patients who have received one cycle of CYAD-01 treatment per protocol ranging from complete response to stable disease. Celyad previously reported the world’s first complete response by a CAR-T cell therapy, without pre-conditioning, in a patient with refractory and relapsed AML.

Based on the promising signs of activity observed to date, Celyad has started to evaluate if a second cycle of administration of CYAD-01 could improve or prolong the clinical responses. A first patient at the second dose level has successfully started his second cycle of treatment without any toxicity observed to date.

On June 19, 2018 Johnson & Johnson (NYSE: JNJ) reported it will host a conference call for investors at 8:30 a.m. (Eastern Time) on Tuesday, July 17, to review second-quarter results (Press release, Johnson & Johnson, JUN 19, 2018, View Source [SID1234527394]). Alex Gorsky, Chairman and Chief Executive Officer and Joseph J. Wolk, Executive Vice President, Chief Financial Officer will host the call.

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Investors and other interested parties can access the webcast/conference call in the following ways:

The webcast and presentation material are accessible at Johnson & Johnson’s website www.investor.jnj.com. A replay of the webcast will be available approximately three hours after the conference call concludes.
By telephone: for both "listen-only" participants and those financial analysts who wish to take part in the question-and-answer portion of the call, the telephone dial-in number in the U.S. is 877-869-3847. For participants outside the U.S., the dial-in number is 201-689-8261.
A replay of the conference call will be available until approximately 12:00 a.m. on July 25, 2018. The replay dial-in number for U.S. participants is 877-660-6853. For participants outside the U.S., the replay dial-in number is 201-612-7415. The replay conference ID number for all callers is 13680884.
The press release will be available at approximately 6:45 a.m. (Eastern Time) the morning of the conference call.