On June 18, 2018 Ziopharm Oncology, Inc. (Nasdaq:ZIOP) reported the U.S. Food and Drug Administration (FDA) placed on clinical hold a Phase 1 trial to evaluate CD19-specific CAR-T therapies manufactured under point-of-care and requested additional information in support of the investigational new drug (IND) application for the trial (Press release, Ziopharm, JUN 18, 2018, View Source [SID1234527386]).

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Ziopharm, Precigen, Inc., a wholly-owned subsidiary of Intrexon Corporation (NYSE: XON) and The University of Texas MD Anderson Cancer Center, the IND sponsor, are seeking to conduct a clinical trial to evaluate CAR+ T cells manufactured with Sleeping Beauty technology as an investigational treatment for patients with relapsed or refractory, CD19+ leukemias and lymphomas. CAR+ T cells very-rapidly manufactured with the Sleeping Beauty platform for this third-generation trial are designed to co-express CD19-specific chimeric antigen receptor, or CAR, membrane-bound interleukin 15 and a safety switch. The FDA has requested additional information relative to Chemistry, Manufacturing and Controls. Ziopharm and its partners will address the FDA’s requests, and the initiation of this trial may be delayed.

"We know what is needed to address the hold issues and are looking forward to responding to the agency in a timely manner," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. "We are undertaking cutting-edge science and are on the verge of a paradigm shift based on our approach to very-rapidly manufacture CD19-specific T cells within two days using our non-viral approach to CAR-T therapy based on the Sleeping Beauty platform."

Ziopharm believes this feedback from the FDA does not affect timelines for the Company’s planned trial at the National Cancer Institute using the Sleeping Beauty platform to target solid tumors infusing TCR-modified T cells. The second-generation clinical trial evaluating Sleeping Beauty-manufactured CD19-specific CAR-T cells continues enrolling and infusing patients at MD Anderson.

On June 18, 2018 Gamida Cell, a leading cellular and immune therapeutics company, reported that preliminary data from an ongoing phase I study from the nicotinamide-based natural killer cell expansion, or NAM-NK, program will be presented during the Inaugural AACR (Free AACR Whitepaper) International Meeting on Advances in Malignant Lymphoma, held June 22 – 26 in Boston, Massachusetts (Press release, Gamida Cell, 18 18, 2018, View Source [SID1234527379]).

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Veronika Bachanova, M.D., Ph.D., hematologist/oncologist at the Blood and Marrow Transplantation and Cellular Therapy Program of the University of Minnesota, and lead investigator of the investigator-sponsored phase I NAM-NK clinical study at the Masonic Cancer Center, will present a poster of early patient outcomes from the study in patients with refractory non-Hodgkin lymphoma and multiple myeloma.

Title: Phase I study of nicotinamide-expanded related donor Natural Killer (NK) cells for the treatment of relapsed/refractory CD20+ non-Hodgkin lymphoma (Abstract: B05)
Session: Poster Session B: Therapeutics and Clinical Trials in Lymphoma 2
Location: Salon F, Boston Marriott Copley Place
Date: Sunday June 24, 2018 11:45 AM – 1:45 PM

About NAM-NK
Gamida Cell applied the capabilities of its NAM-based cell expansion technology to highly functional NK cells to develop NAM-NK, an innate immunotherapy for the treatment of hematologic and solid tumors in combination with SoC antibody therapies. NAM-NK addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs of NK cells expanded in culture. NAM-NK is in phase I development (NCT03019666) through an investigator-sponsored trial in patients with refractory non-Hodgkin lymphoma and multiple myeloma.

On June 18, 2018 TG Therapeutics, Inc. (NASDAQ: TGTX), reported an oral presentation of clinical data from its ongoing Phase 2 study evaluating umbralisib (TGR-1202), the Company’s PI3K delta inhibitor, in patients with relapsed or refractory Chronic Lymphocytic Leukemia (CLL) who are intolerant to prior BTK or PI3K delta inhibitor therapy (Press release, TG Therapeutics, JUN 18, 2018, View Source [SID1234527378]). Data from this trial were presented over the weekend during an oral session at the 23rd Congress of the European Hematology Association (EHA) (Free EHA Whitepaper).

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Michael S. Weiss, the Company’s Executive Chairman and Chief Executive Officer, stated, "We are pleased to present data evaluating umbralisib in patients intolerant to currently approved BTK or PI3K therapies during the EHA (Free EHA Whitepaper) annual congress. While there have been great advancements in recent years in the treatment of CLL, this study confirms that there are many patients still in need of an alternative treatment option and that umbralisib can be used safely and effectively in those patients who were not able to tolerate a prior BTK or PI3K therapy. The rate of patients withdrawing from kinase treatment for CLL in real world settings has been estimated to reach upwards of 40%, representing a significant unmet medical need." Mr. Weiss continued, "We are extremely pleased with the data presented at ASCO (Free ASCO Whitepaper) and EHA (Free EHA Whitepaper) this month and we look forward to presenting the topline response rate data from the UNITY- CLL Phase 3 trial by the end of summer 2018."

Highlights from the oral presentation include the following:

Oral Presentation: A Phase 2 Study to Assess the Safety and Efficacy of Umbralisib (TGR-1202) In Patients with Chronic Lymphocytic Leukemia (CLL) Who Are Intolerant to Prior BTK or PI3K-delta Inhibitor Therapy (Abstract Number S808)

This presentation includes data from patients with CLL who are intolerant to prior BTK or PI3K delta inhibitor therapy who were then treated with single agent umbralisib (TGR-1202). To be eligible for the study patients had to have received prior treatment with a BTK inhibitor (ibrutinib, acalabrutinib) or a PI3K delta inhibitor (idelalisib, duvelisib) and discontinued therapy due to intolerance within 12 months of starting treatment on this study. Forty-seven patients were evaluable for safety of which 46 were evaluable for Progression Free Survival (PFS), (1 patient had a confirmed Richter’s Transformation (RT) at enrollment which did not meet eligibility criteria).

Highlights from this presentation include:

●Umbralisib demonstrated a favorable safety profile in patients intolerant to prior BTK or PI3K therapy

●Only 13% discontinued due to an adverse event, of which only one patient discontinued due to a recurrent adverse event (AE) also experienced with prior kinase inhibitor therapy

●Median progression free survival (PFS) and overall survival has not been reached with a median follow-up of 9.5 months

●In this relapsed/refractory CLL population, of which 77% required treatment within 6 months of prior KI discontinuation, 64% had a high-risk molecular / genetic marker and 6% had an ibrutinib resistance mutation, significant clinical activity has been observed

PRESENTATION DETAILS
The above referenced presentation is now available on the Publications page, located within the Pipeline section, of the Company’s website at www.tgtherapeutics.com/publications.cfm.

On June 18, 2018 Crinetics Pharmaceuticals, Inc., a clinical stage pharmaceutical company focused on the discovery, development and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, reported the appointment of Alan S. Krasner, M.D. as Chief Medical Officer (Press release, Crinetics Pharmaceuticals, JUN 18, 2018, View Source [SID1234527377]).

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"We are delighted to welcome Alan to the Crinetics management team," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "Alan has extensive experience in clinical development of endocrine drugs at both big pharma and smaller biotech companies. We are fortunate to have his leadership to oversee our development programs as we advance our product candidates into and through the clinic."

Dr. Krasner joins Crinetics from Shire Pharmaceuticals where he was a Senior Medical Director and served as Global Development Lead for Natpara, the first recombinant human intact parathyroid hormone treatment for hypoparathyroidism. Prior to Shire, he worked at Biodel and Pfizer conducting clinical research at various stages of development in diabetes and obesity. He obtained his undergraduate and M.D. degrees from Northwestern University and received his clinical training in Internal Medicine and Endocrinology at Johns Hopkins University.

On June 18, 2018 ImmunoGen, Inc. (Nasdaq: IMGN), a leader in the expanding field of antibody-drug conjugates (ADCs) for the treatment of cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for its lead program, mirvetuximab soravtansine (Press release, ImmunoGen, JUN 18, 2018, View Source [SID1234527375]). The designation is for the treatment of patients with medium to high folate receptor alpha (FRα)-positive platinum-resistant ovarian cancer who received at least one, but no more than three prior systemic treatment regimens, and for whom single-agent chemotherapy is appropriate as the next line of therapy.

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"We are pleased the FDA has granted Fast Track designation for mirvetuximab soravtansine," said Mark Enyedy, ImmunoGen’s President and Chief Executive Officer. "Patients with platinum-resistant ovarian cancer have a poor prognosis and we are encouraged by the FDA’s recognition of the significant need for new therapeutic options that may be addressed by mirvetuximab as monotherapy. This important designation is based on the promising safety and activity findings observed to-date and we look forward to working closely with the FDA as we advance the development of mirvetuximab."

The FDA’s Fast Track Designation is intended to facilitate development and expedite review of drugs to treat serious and life-threatening conditions so that an approved product can reach the market expeditiously.

Mirvetuximab soravtansine is being evaluated in the FORWARD I Phase 3 trial. The trial is designed to randomize 333 patients 2:1 to receive either mirvetuximab soravtansine or the physician’s choice of single-agent chemotherapy. Eligibility criteria include patients with platinum-resistant ovarian cancer that express medium or high levels of FRα who have been treated with up to three prior regimens. The primary endpoint of this study is Progression Free Survival, which is being assessed in the entire study population and in the subset of patients with high FRα expression. Enrollment of FORWARD I was completed ahead of schedule in April 2018 and ImmunoGen expects to report top-line results from the FORWARD I trial in the first half of 2019.

ImmunoGen is partnering with the Gynecologic Oncology Group Foundation Inc., a leader in clinical research in gynecologic malignancies, on FORWARD I, which is being conducted in North America and Europe. This trial is intended to support full marketing approval of mirvetuximab for patients with platinum-resistant ovarian cancer.

Mirvetuximab soravtansine is also being assessed in multiple combinations in the FORWARD II trial. FORWARD II is a Phase 1b/2 study of mirvetuximab in combination with Avastin (bevacizumab), or Keytruda (pembrolizumab) in patients with FRα-positive platinum-resistant ovarian cancer, primary peritoneal, or fallopian tube tumors, as well as a triplet combination of mirvetuximab plus carboplatin and Avastin in patients with platinum-sensitive ovarian cancer.

About Mirvetuximab Soravtansine

Mirvetuximab soravtansine (IMGN853) is the first folate receptor alpha (FRα)-targeting ADC. It uses a humanized 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.

About Ovarian Cancer and FRα

It is estimated that 22,000 women are diagnosed annually with ovarian cancer in the US. With more than 14,000 deaths each year, ovarian cancer accounts for more deaths than any other cancer of the female reproductive system.1

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

There is a significant need for more effective, better-tolerated therapies for recurrent ovarian cancer. It is estimated that approximately 19,000 women in the US and approximately 24,000 women in the EU have platinum-resistant ovarian cancer requiring second-line or later treatment.2 ImmunoGen estimates that 60% of ovarian cancer cases have medium or high FRα expression.