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8-K – Current report

On April 28, 2015 Bio-Path Holdings reported that they have received orphan drug designation from the U.S. Food and Drug Administration (FDA) for its lead compound Liposomal Grb-2 for the treatment of acute myeloid leukemia (AML) (Filing, 8-K, Bio-Path Holdings, APR 28, 2015, View Source [SID:1234503201]). Orphan drug status provides Bio-Path with seven years of exclusivity after receiving formal marketing approval, as well as additional development incentives. The FDA grants this designation to certain drugs that are targeting diseases affecting fewer than 200,000 people in the United States.

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"This designation from the FDA demonstrates the unmet need for an effective therapy for patients suffering from AML," said Peter Nielsen, President and Chief Executive Officer of Bio-Path. "It also marks a key regulatory milestone for Bio-Path and will be valuable as we continue to progress Liposomal Grb-2 through clinical trials and toward potential commercialization."

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Liposomal Grb-2 is currently in a safety segment of a Phase 2 trial in combination with Ara-C, for acute myeloid leukemia; and is being evaluated for chronic myelogenous leukemia.

Pre-clinical development is underway for triple negative and inflammatory breast cancers.

About Acute Myeloid Leukemia

AML is a fast-growing cancer of the blood and bone marrow where the bone marrow makes many cancerous cells called leukemic blasts. Normal blasts develop into white blood cells that fight infection. In AML, the leukemic blasts do not develop properly and cannot fight infections. These leukemic blasts grow quickly and crowd out the bone marrow, preventing it from making the normal red blood cells, white blood cells, and platelets that the body needs.
Nearly 15,000 people in the United States are diagnosed with AML each year.

About Growth Receptor Bound protein-2 (Grb-2)

The adaptor protein Growth Receptor Bound protein-2 (Grb-2) is essential to cancer cell signaling because it is utilized by oncogenic tyrosine kinases to induce cancer progression. Suppressing the function or expression of Grb-2 should interrupt its vital signaling function and have a therapeutic application in cancer. BP-1001 is a neutral-charge, liposome-incorporated antisense drug substance designed to inhibit Grb-2 expression.

PTC Therapeutics’ Cancer Stem Cell Program Targeting BMI1 Enters Phase 1

On April 28, 2015 PTC Therapeutics reported that its oncology program targeting BMI1, a protein linked to drug-resistant cancers, has entered a Phase 1 study in patients with advanced solid tumors (Press release, PTC Therapeutics, APR 28, 2015, View Source [SID:1234503200]). The open-label, first-in-human study will investigate the safety and pharmacokinetics of PTC596, an orally available small molecule. PTC’s BMI1 program is supported by a collaboration with the Wellcome Trust.

BMI1 has been implicated in a wide variety of cancers and has been demonstrated to contribute to therapeutic resistance and treatment failure. BMI1 is thought to play a role in the survival and maintenance of tumor stem cells in many cancers, including central nervous system cancers such as glioblastoma. Elevated levels of BMI1 have been associated with advanced tumor grade and a poor prognosis.

"We’re excited to begin clinical trials for PTC596, an investigational drug that targets an important stem cell regulator, BMI1, which is elevated in a wide array of tumor types," said Robert Spiegel, M.D., Chief Medical Officer, PTC Therapeutics, Inc. "In preclinical models, PTC596 reduced BMI levels leading to depletion of the cancer stem cell population. Importantly, we saw this effect when PTC596 was used alone and in combination with current standards of care."

"Targeting cancer stem cells by BMI1 inhibition is a promising approach to address the challenge of drug-resistant cancers," stated Lillian Siu, M.D., Princess Margaret Cancer Center, Professor of Oncology, University of Toronto. "Cancer is a complex problem and the development of treatments that focus on molecular targets shows promise for the next generation of cancer therapies to make a difference in patients’ lives."

PTC’s collaboration with the Wellcome Trust began in June 2010 when the Wellcome Trust awarded PTC $5.4 million to support the development of drugs that target BMI1.

First Patient Dosed by Alligator Bioscience in a Clinical Phase 1 Multicenter Trial

On April 27, 2015 Alligator Bioscience AB reported that the first patient was dosed early last week in the recently initiated phase 1 clinical trial of the immuno-oncology antibody ADC-1013 (Press release, Alligator Bioscience, APR 27, 2015, View Source [SID1234538694]). ADC-1013 is an agonistic fully human monoclonal antibody targeting CD40, an immune-stimulatory receptor found on antigen-presenting cells such as dendritic cells.

The clinical phase 1 trial is a first-in-human trial for patients with advanced solid tumor disease. Dr. Per Norlén, Chief Medical Officer at Alligator Bioscience, commented that "we are very pleased to confirm that dosing of the first patient was successful. The interest for participation in our first-in-human trial has been massive, allowing dosing of the first patient at Uppsala University Hospital, Sweden, within 3 weeks of starting recruitment. ADC-1013 was well tolerated and not associated with any significant adverse reactions. Currently we have opened 2 clinical sites, and expect all 5 sites to be active shortly."

As mentioned in a previous press release April 7, the study will enroll up to 40 patients during the dose escalation and expansion phases at five centers in the United Kingdom, Denmark and Sweden.

10-Q – Quarterly report [Sections 13 or 15(d)]

Amgen has filed a 10-Q – Quarterly report [Sections 13 or 15(d)] with the U.S. Securities and Exchange Commission (Filing, 10-Q, Amgen, APR 27, 2015, View Source [SID1234503189]).

Data Safety Monitoring Board Recommends Continuation of Phase 3 Study of Zoptarelin Doxorubicin in Advanced Endometrial Cancer

On April 27, 2015 Aeterna Zentaris Inc. (NASDAQ: AEZS) (TSX: AEZ) (the "Company") reported that the independent Data Safety Monitoring Board ("DSMB") for the pivotal Phase 3 ZoptEC (Zoptarelin Doxorubicin in Endometrial Cancer) study with zoptarelin doxorubicin in women with advanced, recurrent or metastatic endometrial cancer, has completed a pre specified first interim futility analysis (Press release, AEterna Zentaris, APR 27, 2015, View Source;q=655 [SID:1234506576]). The DSMB has recommended that the Phase 3 study continue as planned.

David Dodd, Chairman and CEO of Aeterna Zentaris, commented, "We are very pleased with this positive recommendation from the DSMB on our ZoptEC Phase 3 trial in endometrial cancer, and I would like to thank everyone involved in this project for their unwavering dedication. With approximately 90 % of patients enrolled at this time, we are well on our way of completing patient recruitment ahead of the anticipated timeline. We believe that zoptarelin doxorubicin has the potential to become the first FDA approved medical therapy for advanced, recurrent endometrial cancer This could result in its rapid adoption as a novel core therapy for patient treatment & management, and therefore, could represent a significant market opportunity for the Company. Moving forward, we are continuing to develop our commercialization plans regarding zoptarelin doxorubicin in this indication, including establishing additional partnerships in territories that won’t be pursued by Aeterna Zentaris. In addition, contingent on the success of the ZoptEC program, we have additional areas of interest for further therapeutic development, including ovarian, prostate and triple negative breast cancer. Our commitment is to ensure that patients and their physicians have such therapies that can potentially improve and extend the quality of lives."

The ZoptEC Phase 3 trial is an open-label, randomized-controlled study, comparing the efficacy and safety of zoptarelin doxorubicin, a hybrid molecule composed of a synthetic peptide carrier and a well known chemotherapy agent, doxorubicin, to doxorubicin alone. It is being conducted under a Special Protocol Assessment with the U.S. Food and Drug Administration ("FDA"). Patients are centrally randomized in a 1:1 ratio and receive either zoptarelin doxorubicin (267 mg/m2) or doxorubicin (60 mg/m2) intravenously, every 3 weeks and for up to 9 cycles. Response will be evaluated every 3 cycles during treatment, thereafter, every 12 weeks until progression. All patients will be followed for survival as the primary efficacy endpoint ("EP"). Secondary EPs include progression-free survival, objective response-rate, and clinical benefit rate.

At this time, sites initiation has been completed with over 120 sites in operation in North America, Europe and Israel. More than 465 patients out of an expected total of 500 have been recruited. A second interim analysis will be conducted according to protocol at approximately 192 events, with the final analysis planned at an anticipated 384 events.

For more information on this trial, please consult (ClinicalTrials.gov Identifier: NCT01767155; EudraCT No: 2012-005546-38; ZoptEC: Zoptarelin doxorubicin in endometrial cancer).

About Zoptarelin Doxorubicin

Zoptarelin doxorubicin represents a new targeting concept in oncology using a hybrid molecule composed of a synthetic peptide carrier and a well-known chemotherapy agent, doxorubicin. Zoptarelin doxorubicin is the first intravenous drug in advanced clinical development that directs the chemotherapy agent specifically to LHRH-receptor expressing tumors, which could result in a more targeted treatment with less damage to healthy tissue. The Company is currently conducting a ZoptEC (Zoptarelin doxorubicin in Endometrial Cancer) Phase 3 trial in women with advanced, recurrent or metastatic endometrial cancer, while zoptarelin doxorubicin is also in an investigator-initiated Phase 2 trial in prostate cancer. Aeterna Zentaris owns the worldwide rights to this compound except in China (including Hong Kong and Macau) where rights have been out-licensed to Sinopharm A-Think Pharmaceuticals, a subsidiary of Sinopharm, the largest medical and healthcare group in China and on Fortune’s Global 500 list. On April 16, 2015, the Company announced the filing of a patent application intended to strengthen the exclusivity of zoptarelin doxorubicin through a unique, significantly lower cost in the manufacturing process.

About Endometrial Cancer

Endometrial cancer is the most common gynecologic malignancy in developed countries and develops when abnormal cells amass to form a tumor in the lining of the uterus. It largely affects women over the age of 50 with a higher prevalence in Caucasians and a higher mortality rate among African Americans. According to the American Cancer Society, there will be more than 50,000 new cases of endometrial cancer in the U.S. alone in 2015, with about 20% of recurring disease.