On May 2, 2017 ZIOPHARM Oncology, Inc. (Nasdaq: ZIOP), a biopharmaceutical company focused on new immunotherapies, reported that an investigator-initiated Investigational New Drug (IND) application to the U.S. Food and Drug Administration (FDA) for a Phase 1 trial infusing the Company’s CD33-specific CAR+ T therapy for relapsed or refractory acute myeloid leukemia (AML) is now active, with the first patient to be enrolled in the study expected to begin treatment in the third quarter of 2017 (Press release, Ziopharm, MAY 2, 2017, View Source [SID1234518785]). The CD33-specific CAR+ T cells incorporate a kill switch designed to eliminate the modified T cells under potential adverse safety conditions.

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"Relapsed AML is an aggressive disease with very poor outcomes," said William G. Wierda, M.D., Ph.D., Professor and Center Medical Director, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center and principal investigator for the CD33 study. "In vivo preclinical animal studies have demonstrated that these CAR-T cells targeting CD33 exhibit specific killing of AML cells, eliminating disease burden, and significantly enhancing survival compared to controls, and I look forward to evaluating the safety and effectiveness of this gene therapy for patients with AML."

Francois Lebel, M.D., Executive Vice President, Research and Development, Chief Medical Officer at ZIOPHARM added, "CAR-T cells expressing CD33 have shown promise in preclinical studies, but to-date, there has been limited experience in humans, representing a significant white space for us in treating AML. We look forward to seeing the positive preclinical results with our CD33-specific CAR-T cells translate to the clinic for relapsed/refractory AML patients who have far too few treatment options. In parallel with this Phase 1 study, we have also begun preclinical studies to evaluate rapid non-viral manufacturing of CAR+ T CD33-specific therapy for treatment of AML under point-of-care."

AML is a rapidly progressing cancer of the blood and bone marrow characterized by uncontrolled proliferation of immature blast cells with multiple associated gene mutations. The American Cancer Society estimates that there were approximately 20,000 new cases of AML and over 10,000 patient deaths from AML in the United States in 2016. A majority of AML patients relapse or present with refractory disease and have overall poor prognosis.
This will be the second CAR target for genetically modified T cells to be studied at The University of Texas MD Anderson Cancer Center under the research and development agreement among ZIOPHARM, Intrexon Corporation (NYSE:XON), and MD Anderson.

On May 1, 2017 Heat Biologics, Inc. ("Heat") (NASDAQ: HTBX), a leader in the development of immunotherapies designed to activate a patient’s immune system against cancer, reported that the company has completed the acquisition of an 80% controlling interest in Pelican Therapeutics, Inc. ("Pelican") (Press release, Heat Biologics, MAY 1, 2017, View Source [SID1234518883]). Headquartered in Austin, Texas, Pelican is a privately held immuno-oncology company focused on developing agonists to TNFRSF25, a differentiated and potentially best-in-class T cell costimulatory receptor. Heat also announced its appointment of industry veteran and Pelican board member Rahul Jasuja, Ph.D., as new Chief Executive Officer of the Pelican subsidiary.

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"We are pleased to complete the acquisition of Pelican, to expand our reach within immuno-oncology," stated Jeff Wolf, Heat’s Founder and CEO. "Pelican’s two product candidates strengthen our portfolio in the emerging T cell activation space. Pelican’s T cell co-stimulator, PTX-25, in combination with Heat’s ImPACT/ComPACT platform, has the potential to dramatically improve the durability of responses by stimulating the production of ‘memory’ CD8+ T cells. The acquisition also brings a $15.2 million grant awarded by the Cancer Prevention and Research Institute of Texas (CPRIT) to advance multiple products through preclinical development and at least one program through a 70 patient Phase 1 clinical trial."

"Furthermore, we are pleased to welcome Dr. Jasuja as new CEO of the Pelican subsidiary. As a board member, he made significant contributions advancing the business strategy and preclinical activities and we believe he is ideally suited to lead the new Pelican subsidiary moving forward."

On May 1, 2017 Aeterna Zentaris Inc. (NASDAQ:AEZS) (TSX:AEZS) (the "Company") reported that the ZoptEC Phase 3 clinical study of Zoptrex (zoptarelin doxorubicin) in women with locally advanced, recurrent or metastatic endometrial cancer did not achieve its primary endpoint of demonstrating a statistically significant increase in the median period of overall survival of patients treated with Zoptrex as compared to patients treated with doxorubicin (Press release, AEterna Zentaris, MAY 1, 2017, View Source [SID1234518774]).

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Dr. Richard Sachse, the Company’s Chief Scientific Officer, stated, "The median overall survival period for patients treated with Zoptrex was 10.9 months compared to 10.8 months for patients treated with doxorubicin. This is not a statistically significant, clinically meaningful increase in overall survival and thus the ZoptEC Phase 3 clinical study did not meet its primary endpoint. In addition, Zoptrex generally performed no better than the comparator drug with respect to the secondary efficacy endpoints. For example, the median period of progression-free survival of the patients in the Zoptrex arm of the study was identical to that for patients in the doxorubicin arm. Finally, there was no meaningful difference between the two arms with respect to safety; the number of patients with cardiac disorders was similar – eight in the Zoptrex arm and nine in the doxorubicin arm. Therefore, the results of the study are not supportive to pursue regulatory approval."

David A. Dodd, the President and Chief Executive Officer of the Company, stated, "We are very disappointed with the outcome of the ZoptEC Phase 3 clinical study. Based on this outcome, we do not anticipate conducting clinical trials of Zoptrex with respect to any other indications. I would like to thank my colleagues within Aeterna Zentaris and our external team of clinical investigators for their dedication to and contributions on this project." Commenting on the Company’s plans, Mr. Dodd continued, "Our focus has now shifted entirely to filing our new drug application for Macrilen and, if the product is approved, to its commercial launch as soon as possible. We will also optimize our resources to be consistent with our focus on Macrilen-related efforts. We continue to believe in the potential that Macrilen provides for us to become a focused specialty pharmaceutical company. Our intention is to submit the Macrilen NDA in the third quarter of 2017 and, if the product receives FDA approval, to commercially launch the product in the first quarter of 2018."

Trieza Therapeutics is a new company developing immunomodulatory oncolytic viruses for the treatment of cancer (Company Web Page, MPM Capital, MAY 1, 2017, View Source [SID1234518761]). Founded as a spin-out from Potenza Therapeutics, Trieza has a portfolio containing multiple pre-clinical candidates.

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On May 1, 2017 ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported that the results from a Phase 1 dose-escalation study evaluating mirvetuximab soravtansine (IMGN853) in patients with folate receptor alpha (FRα)-positive solid tumors were published in the journal Cancer (Press release, ImmunoGen, MAY 1, 2017, View Source [SID1234518773]). The previously disclosed data demonstrated encouraging clinical activity and a manageable safety profile for mirvetuximab soravtansine (IMGN853), and informed the dose that was used in Phase 1 expansion cohorts and the ongoing Phase 3 FORWARD I trial of patients with platinum-resistant ovarian cancer.

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"These Phase 1 results have played an important role in determining the appropriate dose for mirvetuximab soravtansine in the recently initiated Phase 3 FORWARD I trial of patients with platinum-resistant ovarian cancer," said Kathleen Moore, M.D., Associate Professor, Department of Obstetrics and Gynecology at the Stephenson Cancer Center at the University of Oklahoma. "The combination of these data and the recent data published in the Journal of Clinical Oncology further support the dose that has been chosen and patients who have been selected for FORWARD I."

The open-label, Phase 1 dose-escalation study treated a total of 44 patients with recurrent ovarian (52%) or endometrial cancer (25%), along with renal cell carcinoma and non-small cell lung cancer (11% and 9%, respectively). Patients received mirvetuximab soravtansine on day 1 of a 21-day cycle (Q3W dosing) with cycles repeated until dose-limited toxicity or progression, concluding the recommended dose for future trials is 6.0 mg/kg of mirvetuximab (based on adjusted ideal body weight) dosed once every three weeks. On the basis of the study findings, and additional data that demonstrated the importance of FRα expression levels for optimal mirvetuximab sorvatansine activity, the Company designed the Phase 3 FORWARD I trial utilizing this dose in patients with platinum-resistant ovarian cancer, along with a Phase 1b trial evaluating mirvetuximab in combination with standard-of-care chemotherapy and targeted agents.1

Mirvetuximab soravtansine exhibited a manageable safety profile and encouraging preliminary clinical activity. Adverse events (AEs) were generally mild with the majority being grade 1 or grade 2 (least severe grades). The most commonly observed AEs were fatigue, blurred vision and diarrhea.1

The publication, "Phase I dose-escalation study of mirvetuximab soravtansine (IMGN853), a folate receptor alpha-targeting antibody-drug conjugate, in patients with solid tumors," is available on the Cancer website.

About Mirvetuximab Soravtansine

Mirvetuximab soravtansine (IMGN853) is the first FRα-targeting ADC. It uses a FRα-binding antibody to target the ADC specifically to FRα-expressing cancer cells and a potent anti-tumor agent, DM4, to kill the targeted cancer cells.

Mirvetuximab soravtansine is ImmunoGen’s lead program and is in Phase 3 testing as a single agent for the treatment of platinum-resistant ovarian cancer. The candidate is also being assessed in combination regimens for both platinum-resistant and platinum-sensitive disease in Phase 1b/2 FORWARD II trial.

About Ovarian Cancer and FRα

In 2016, approximately 22,300 new cases of ovarian cancer will be diagnosed in the U.S. and more than 14,200 women will die from the disease.2 ImmunoGen estimates that 60% of ovarian cancer cases have medium or high FRα expression.

Standard first-line therapy for ovarian cancer is a platinum-based regimen. Once the cancer becomes platinum-resistant, treatment options include single-agent cytotoxic therapies such as pegylated liposomal doxorubicin, paclitaxel, or topotecan.