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 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 OncBioMune Pharmaceuticals, Inc. (OTCQB:OBMP) ("OncBioMune" or the "Company"), a clinical-stage biopharmaceutical company engaged in the development of a proprietary immunotherapy cancer vaccine technology and targeted cancer therapies, reported to inform shareholders that all requisite approvals have been granted to commence a Phase 2 clinical trial of ProscaVax for early-stage prostate cancer to be hosted at Beth Israel Deaconess Medical Center a teaching hospital of Harvard University Medical School in Boston, MA (Press release, Oncbiomune, JUN 20, 2018, View Source [SID1234527408]).

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ProscaVax is OncBioMune’s lead immunotherapy platform candidate consisting of a combination of prostate cancer associated prostate specific antigen (PSA) with the biological adjuvants interleukin-2 (IL-2) and granulocyte-macrophage colony-stimulating factor (GM-CSF).

Per the study protocol, patients to be enrolled will be in what is termed "active surveillance," a disease management option for patients with localized prostate cancer that elect to work with their doctor to monitor the disease for progression before taking more drastic intervention measures, such as surgery or radiotherapy. To the Company’s knowledge, the trial of ProscaVax is the first ever worldwide for a prostate cancer vaccine technology addressing the active surveillance patient population.

"We are making the final preparations to initiate enrollment, including planned site visits," commented Dr. Jonathan Head, Chief Executive Officer at OncBioMune. "Underscored by the compelling data derived from the successfully completed Phase 1a clinical trial and our other research, we are eager to proceed with this study. Our goal is simple: to provide a safe, nontoxic. front-line therapy for prostate cancer patients that are otherwise left with either no therapy or with much more invasive options that typically have very unpleasant side effects, such as impotence or urinary incontinence. I’d like to express my gratitude for all the shareholders that have supported us throughout the process to advance our company to this next stage of ProscaVax development. I believe there is a bright future ahead of us."

Concurrent with the Phase 2 trial of patients in active surveillance, the Company is undergoing the approval process to initiate a Phase 2 trial of ProscaVax in hormone-naïve recurrent prostate cancer patients with increasing PSA at theUrology Clinics of North Texas. More updates on the status of this study are expected soon.

About Prostate Cancer

According to the American Cancer Society (ACS), prostate cancer is the most common type of cancer in men other than skin cancer, with about 1 in 9 men diagnosed during their lifetime. ACS estimates that about 164,690 new cases of prostate cancer will be diagnosed during 2018 and approximately 29,430 men will die from the disease this year. Prostate cancer is the second leading cause of cancer death in men, trailing only lung cancer. Approximately 2.9 million men are living with prostate cancer today. The average age of diagnosis is 66, with the disease considered rare in men under the age of 40.

On June 20, 2018 NewLink Genetics Corporation (NASDAQ:NLNK) reported that updated data for patients with diffuse intrinsic pontine glioma (DIPG) from NLG2105, a Phase 1 study evaluating indoximod in combination with radiation and chemotherapy for the treatment of pediatric patients with progressive brain tumors, will be presented at the International Symposium on Pediatric Neuro-Oncology (ISPNO) in Denver, Colorado (Press release, NewLink Genetics, JUN 20, 2018, View Source [SID1234527407]). The presentation session and time are noted below.

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Poster Session 1: IMMU-25 – Radio-immunotherapy using the IDO pathway inhibitor indoximod for children with newly-diagnosed DIPG, to be presented by Theodore S. Johnson, M.D., Ph.D., Georgia Cancer Center, Augusta University, Sunday, July 1, 5:00PM – 6:30PM MT, Hyatt Regency Denver

About Indoximod
Indoximod is an investigational, orally available small molecule targeting the IDO pathway. The IDO pathway is a key immuno-oncology target involved in regulating the tumor microenvironment and immune escape. Indoximod is being evaluated in combination with treatment regimens including chemotherapy, radiation, checkpoint blockade and cancer vaccines across multiple indications such as AML, DIPG and melanoma.

On June 20, 2018 Moleculin Biotech, Inc., (Nasdaq:MBRX) ("Moleculin" or the "Company"), a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, reported it has received Polish National Office approval to begin its second Phase I/II clinical trial to study Annamycin for the treatment of relapsed or refractory acute myeloid leukemia ("AML") (Press release, Moleculin, JUN 20, 2018, View Source [SID1234527406]).

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"This approval is a significant step forward to be able to treat additional patients with AML and to generate positive clinical data regarding Annamycin," commented Walter Klemp, Chairman and CEO of Moleculin. "Consent from the Polish National Office was the final step required to allow us to begin recruiting patients for this important trial. Enrollment will begin immediately and we expect patients to commence receiving treatment, at the appropriate dose, in this clinical trial during the second half of this year."