On July 3, 2019 Ziopharm Oncology, Inc. ("Ziopharm" or "the Company") (Nasdaq:ZIOP), reported Drew Deniger, Ph.D., will join Ziopharm from the National Cancer Institute (NCI) to lead the company’s program to genetically modify T cells to express neoantigen-specific T-cell receptors (TCRs), effective July 29, 2019 (Press release, Ziopharm, JUL 3, 2019, View Source [SID1234537377]).

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Since 2013, Dr. Deniger has worked at the NCI under Dr. Steven Rosenberg where he has served as Lead Investigator for the group’s efforts in three initiatives: Identifying "hotspot" neoantigens for T-cell therapy; targeting neoantigens in metastatic endometrial and ovarian cancers; and non-viral gene therapy using the Sleeping Beauty platform to generate TCR-modified T cells targeting neoantigens.

"At the foundation of our TCR-T program is the partnership we have developed with Dr. Rosenberg and his team at the NCI. As an integral part of that group, Dr. Deniger has helped harness our Sleeping Beauty technology to express neoantigen-specific T-cell receptors and prepare for the start of the upcoming clinical trial in patients with solid tumors," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. "As a recognized leader in the identification of neoantigens in hotspots, advancing innovative immunotherapy approaches into the clinic, and with years of expertise with the Sleeping Beauty system, we’re delighted to welcome Drew to Ziopharm."

Author of multiple peer-reviewed manuscripts describing detection of neoantigen-reactive T cells for personalized cancer immunotherapy and T-cell responses to hotspot mutations, Dr. Deniger has also written validating publications related to the non-viral Sleeping Beauty transposon-transposase system. Dr. Deniger is the named inventor on patents related to TCRs recognizing mutated p53 and methods of isolating T cells having antigenic specificity for a p53 cancer specific-mutation, and has been the recipient of numerous awards in cancer immunotherapy.

"Evaluating and validating the Sleeping Beauty platform has been one of my research priorities during my time at NCI," said Drew Deniger, Ph.D. "Utilizing Sleeping Beauty to genetically modify T cells is an extremely compelling path to treating solid tumors and I am excited to be able to join Laurence and the Ziopharm team as we advance TCR-T into the clinic. We look forward to building out a broad library of TCRs reactive to neoantigens including within KRAS, p53 and EGFR hotspots for use with the Sleeping Beauty platform in the future."

Prior to his tenure at the NCI, Dr. Deniger earned B.S. degrees in Chemistry and Biochemistry from the University of Texas at Austin, a M.S. in Cancer Biology and a Ph.D. in Immunology from the University of Texas MD Anderson Cancer Center. His post-doctoral instruction was in the laboratory of Dr. Rosenberg, where he was trained in clinical translation of cancer immunotherapy, including tumor infiltrating lymphocytes (TIL), neoantigen-specific T cells and TCR-T therapy.

Ziopharm and the NCI are partnered through January 2022 in a cooperative research and development agreement (CRADA), under the direction of Dr. Rosenberg, Chief of the Surgery Branch of the NCI, supporting clinical work to evaluate a non-viral approach to manufacturing TCR-T with the Sleeping Beauty platform that target solid tumors. With this approach, T cells can be genetically modified to express multiple, neoantigen-specific TCRs, which Ziopharm believes will be foundational technology to successfully targeting and treating metastatic solid tumors.

Last month, Ziopharm announced that the investigational new drug (IND) application submitted by the NCI had received clearance from the U.S. Food and Drug Administration (FDA) for a clinical trial in solid tumors to evaluate TCR-T utilizing Ziopharm’s Sleeping Beauty platform.

On July 3, 2019 The U.S. Food and Drug Administration reported that it has granted accelerated approval to Xpovio (selinexor) tablets in combination with the corticosteroid dexamethasone for the treatment of adult patients with relapsed refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is resistant to several other forms of treatment, including at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody (Press release, US FDA, JUL 3, 2019, View Source [SID1234537379]).

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"While there is no cure for multiple myeloma, there are FDA-approved treatments to target the cancer and slow down the spread of the disease. Sadly, often over time, patients can exhaust all available treatments and still see their disease progress," said Richard Pazdur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research. "Today we approved a treatment under our accelerated approval program that provides a treatment option for patients with multiple myeloma with no available therapy."

Multiple myeloma is cancer that begins in plasma cells (white blood cells that produce antibodies) and may also be referred to as plasma cell myeloma. Abnormal plasma cells build up in the bone marrow, forming tumors in many bones of the body. As more antibodies are made, it can cause blood to thicken and keep the bone marrow from making enough healthy blood cells. The exact causes of multiple myeloma are unknown, but it is more common in older people and African Americans.

Efficacy was evaluated in 83 patients with RRMM who were treated with Xpovio in combination with dexamethasone. At the end of the study, the overall response rate was measured at 25.3%. The median time to first response was four weeks, with a range of one to ten weeks. The median duration of response was 3.8 months. The efficacy evaluation was supported by additional information from an ongoing, randomized trial in patients with multiple myeloma.

Common side effects of patients taking Xpovio in combination with dexamethasone include a low white blood cell count (leukopenia), a low count of neutrophils, a type of white blood cell (neutropenia), low count of platelets (thrombocytopenia) and low amount of red blood cells (anemia). Patients also reported vomiting, nausea, fatigue, diarrhea, fever, decreased appetite and weight, constipation, upper respiratory tract infections and low blood sodium levels (hyponatremia).

Health care professionals are advised to monitor patients for low blood counts, platelets and sodium levels. Patients should avoid taking Xpovio with other medications that may cause dizziness or confusion and avoid situations where dizziness may be a problem. Health care professionals are advised to optimize the patient’s hydration status, blood counts and other medications to avoid dizziness or confusion. The FDA advises health care professionals to tell females of reproductive age and males with a female partner of reproductive potential to use effective contraception during treatment with Xpovio. Women who are pregnant or breastfeeding should not take Xpovio because it may cause harm to a developing fetus or newborn baby. Xpovio must be dispensed with a patient Medication Guide that describes important information about the drug’s uses and risks.

Xpovio in combination with dexamethasone was granted accelerated approval, which enables the FDA to approve drugs for serious conditions to fill an unmet medical need based on an endpoint that is reasonably likely to predict a clinical benefit to patients. Further clinical trials are required to verify and describe Xpovio’s clinical benefit.

The FDA granted this application Fast Track designation. Xpovio also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases. The FDA granted the approval of Xpovio to Karyopharm Therapeutics.

For more information:

National Cancer Institute: Multiple myeloma
FDA: Office of Hematology and Oncology Products
FDA: Approved Drugs: Questions and Answers
FDA: Fast Track, Breakthrough Therapy, Accelerated Approval, and Priority Review
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.

On July 3, 2019 Onco360, the nation’s largest independent Oncology Pharmacy, reported that it was selected to be a specialty pharmacy network partner for Karyopharm’s new product XPOVIO (selinexor) a nuclear export inhibitor indicated in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma (RRMM) who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody (Press release, Onco360, JUL 3, 2019, View Source [SID1234537380]).

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"Onco360 is excited to be selected as a specialty pharmacy provider for XPOVIO patients," said Paul Jardina, President and CEO, Onco360. "The recent approval of XPOVIO unlocks a new treatment option for relapsed or refractory multiple myeloma patients who have failed previous lines of treatment. As a provider of this key treatment, Onco360 can support the highly specialized needs of relapsed or refractory multiple myeloma and their physicians across the states."

Multiple myeloma is an incurable hematological malignancy involving plasma cells. Only 50 percent of patients diagnosed with multiple myeloma survive past five years following initial diagnosis. The National Cancer Institute estimates that in 2018, approximately 30,770 new patients were diagnosed with multiple myeloma in the U.S.1 Unfortunately, most multiple myeloma patients will relapse following the first complete remission and often require continuous treatment to prevent disease progression. Previously, there were no U.S. Food and Drug Administration (FDA)-approved products or regimens for patients with relapsed or refractory multiple myeloma who failed treatment with lenalidomide, pomalidomide, bortezomib, carfilzomib, and/or daratumumab.

XPOVIO is manufactured by Karyopharm Therapeutics, and was approved by the U.S. FDA on July 3, 2019 based on positive clinical trial results from the Phase IIb STORM Trial which demonstrated a 21% overall response rate in heavily-pretreated multiple myelmoma patients.2 XPOVIO is the first and only nuclear export inhibitor that blocks XPO1. For full prescribing information, visit XPOVIO.com.

On July 3, 2019 Seattle Genetics, Inc. (Nasdaq: SGEN) reported that it will report its second quarter 2019 financial results on Tuesday, July 16, 2019 after the close of financial markets (Press release, Seattle Genetics, JUL 3, 2019, View Source [SID1234537368]). Following the announcement, management will host a conference call and webcast discussion of the results and provide a general corporate update. Access to the event can be obtained as follows:

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LIVE access on Tuesday, July 16, 2019

1:30 p.m. Pacific Time / 4:30 p.m. Eastern Time

Telephone 800-458-4121 (domestic) or +1 323-794-2093 (international); conference ID 3271918
Webcast with slides available at www.seattlegenetics.com in the Investors section
REPLAY access

Telephone replay will be available beginning at approximately 4:30 p.m. PT on Tuesday, July 16, 2019 through 5:00 p.m. PT on Friday, July 19, 2019 by calling 888-203-1112 (domestic) or +1 719-457-0820 (international); conference ID 3271918
Webcast replay will be available on the Seattle Genetics website at www.seattlegenetics.com in the Investors section

On July 2, 2019 A2A Pharmaceuticals Inc. (A2A), a biotechnology company committed to the advancement of innovative scientific research and new therapeutic agents, reported the appointment of Dr. Sotirios G. Stergiopoulos as President and Chief Executive Officer (Press release, A2A Pharmaceuticals, JUL 2, 2019, View Source [SID1234537369]). He has held the position as Founder and Chairman of the Board since 2016. Sotirios will maintain a seat on the Board of Directors.

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Dr. Stergiopoulos joins A2A from Ipsen where he served as the Chief Medical Officer, SVP and Head of Global Medical Affairs since January 2017. In this role, he was responsible for the overall medical and clinical governance of the company, as well as the creation of a global medical affairs organization. Dr. Stergiopoulos played a key role in Ipsen’s oncology TA strategy. Sotirios held numerous leadership positions in the pharmaceutical industry with companies including Novartis, Bayer, and Celgene.

Dr. Stergiopoulos is a physician executive with a B.S. from Stony Brook University, an M.D. from Poznan University of Medical Sciences, and an M.B.E.E. from the Johns Hopkins University. He has trained at the National Institutes of Health, the Albert Einstein College of Medicine, and Harvard University.

Edward Painter, who has served as CEO of A2A since 2016, will take the role of Chief Investment Officer and Chairman of the Board. Edward will work with Sotirios to optimize the allocation of capital across A2A’s programs and build the management team.

Mr. Painter remarked, "I am delighted to have succeeded in recruiting Sotirios to the CEO role at A2A. His leadership in the pharmaceutical industry, as well as experience in clinical medicine and Oncology drug development, will be critical to A2A’s advancement of therapeutics for Oncology and other life-threatening diseases into the clinic for the patients that need them."

Dr. Stergiopoulos explained, "A2A’s novel approach to drug development has the potential to bring true best in class therapies to patients suffering with cancer and other difficult to treat diseases. I am encouraged by the platform utilized by A2A to pursue difficult targets and look forward to working alongside Edward and the team at A2A to translate the technology into therapies for patients."