On June 28, 2016 FUJIFILM Corporation (President: Kenji Sukeno) reported the progress in a Phase I clinical trial of radioimmunotherapeutic anti-cancer agent FF-21101 in the United States in patients with advanced solid cancers (Press release, Fujifilm, JUN 28, 2016, View Source [SID:1234513609]). In imaging, tumor uptake of antibody FF-21101 was demonstrated in administered patients. Taking these results, it is expected to treat diseases with emission from the radiolabeled antibody. Fujifilm will proceed with the clinical trial and evaluate FF-21101 as an anti-cancer agent.

FF-21101 is an anti-cancer agent consisting of a radioisotope labeled antibody (armed antibody*1), and uses radiation emitted by the radioisotope to directly attack cancer cells. This is why it is expected to have a higher level of efficacy, regardless of the state of patient’s immune system. In addition, its accumulation in cancer tissue can be confirmed by imaging with an administration of antibodies labeled with a radioisotope. To date, tumor uptake of antibody was demonstrated in 3 of 4 patients who have undergone imaging with an administration of radiolabeled FF-21101.
These results were presented on June 27th at the World Innovative Networking in Personalized Cancer Medicine Symposium 2016 (Paris, France) by principal investigator, Vivek Subbiah MD, of The University of Texas MD Anderson Cancer Center*2 (MD Anderson Cancer Center, hereafter), Houston, TX, USA.

Fujifilm has organized the technologies of its Group companies to develop FF-21101. The bio-venture, Perseus Proteomics, has contributed to antibody drug discovery, while the biopharmaceutical contract manufacturer, FUJIFILM Diosynth Biotechnologies, has taken charge of antibody production. The radiopharmaceutical company, FUJIFILM RI Pharma, utilized its technology for developing the diagnostic and therapeutic radiopharmaceuticals.

Fujifilm initiated the Phase I clinical trial of radiolabeled FF-21101 in patients with advanced solid cancers at MD Anderson Cancer Center in the United States, one of the world’s most distinguished facilities for cancer research and treatment, in January 2016. In this trial, FF-21101(111In), antibodies labeled with Indium-111*3, were administered prior to therapy and its biodistribution to normal and cancerous tissues were visualized and quantified by imaging procedures. In addition, for safety estimation in advance, radiation absorbed doses of Yttrium-90*4 in organs were calculated from these data, after which, patients were moved on to the therapeutic dosing of FF-21101(90Y), antibodies labeled with Yttrium-90.

Although the trial is still underway, preliminary observations include:
Imaging revealed tumor uptake in 3 of 4 patients administered FF-21101(111In).
FF-21101 was well tolerated in all patients who went on to receive the therapeutic dose of FF-21101(90Y).

Fujifilm will continue the study to further evaluate tolerability and efficacy in multiple advanced solid tumors at MD Anderson Cancer Center, and will move on to the phase IIa study.

Fujifilm is working on the R&D of innovative pharmaceutical products and creation of their production processes by combining the technologies and know-how accumulated in the photographic film business including analysis technology, nanotechnology, and production technology, with the technological expertise of its core pharmaceutical affiliates such as Toyama Chemical. Defining "oncology", a field with numerous unmet medical needs as its focal area, the company will actively promote R&D to expand business deployment and supply innovative pharmaceutical products so as to contribute to resolving challenges social issues.

*1： Armed antibody: Antibody conjugated with toxin or radioisotope via a chemical linker. It is expected to have a potent anti-tumor effect.
*2：One of the world’s largest cancer centers based in Houston. Texas (USA), specializing in cancer treatment, research, education and prevention. It was established in 1941 with a mission to eliminate cancer. Under the core values of "Caring", "Integrity" and "Discovery", the Center has developed numerous new cancer treatments and is as an international leader in oncology.
*3：A radioisotope which emits gamma rays appropriate for imaging. Its physical half-life is, approximately 67 hours.
*4：A radioisotope which emits beta particle radiation with a range of 5 mm in human body, appropriate for treatment. Its physical half-life is, approximately 64 hours.
　　
About anti-cancer agent FF-21101:
FF-21101 uses antibodies that target P-cadherin*5, which is overexpressed on the surface of solid cancer cells, including lung, pancreatic and colon cancers, and is implicated in tumor growth and cancer metastasis. For the clinical application of FF-21101, tumor uptake will be confirmed in patients administered antibodies radiolabeled with emitter, such as Indium-111 or other radioisotopes. Antibodies labeled with Yttrium-90 namely FF-21101(90Y), will be subsequently administered and directly attacks cancer cells by radiation emitted from the 90Y radioisotope. FF-21101 is expected to be more effective than therapy using the P-cadherin targeted antibody without the 90Y. In animal testing, it has already demonstrated a high efficacy in shrinking human tumors implanted in mice.

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On June 28, 2016 STORM Therapeutics, a spin-out company from the University of Cambridge’s Gurdon Institute focused on the identification and development of small molecule drugs that target RNA-modifying enzymes, reported that it has received £12 million in Series ‘A’ funding from Cambridge Innovation Capital, Merck Ventures, Pfizer Venture Investments and Touchstone Innovations (Press release, STORM Therapeutics, JUN 28, 2016, View Source [SID1234561051]). STORM Therapeutics is based upon the ground-breaking work of its co-founders, Professor Tony Kouzarides and Professor Eric Miska, in the field of RNA epigenetics.

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RNA (ribonucleic acid) is the only direct product of the human genome and acts as the template for the synthesis of all proteins, the molecular machines of the cell. RNA is also known to be a key player in cellular decision-making. There are several large families of RNA-modifying enzymes which catalyse a diverse range of epigenetic modifications of RNA, changing RNA activity and thereby key processes within the cell. There is a growing understanding of the importance of RNA modification in the development of cancer, opening up novel therapeutic targets in cancer treatment.

Professors Kouzarides and Miska and their research groups have identified certain of these RNA-modifying enzymes against which STORM Therapeutics intends to develop potential therapeutics using intellectual property licensed from the University of Cambridge and Cancer Research UK. The company will use the proceeds of the funding to identify small molecule modulators of these novel targets in RNA modification pathways and develop them into new classes of anti-cancer treatments.

Professors Tony Kouzarides and Eric Miska commented: "The work that our research groups are undertaking on non-coding RNA and the enzymes that modify this RNA is giving us incredibly interesting insights into how gene expression can be modified at a cellular level. The funding and support that STORM Therapeutics has received from its investors will allow the development of these insights into a new class of therapeutics ready to be taken into clinical trials."

On June 28, 2016 Bio-Path Holdings, Inc., (NASDAQ: BPTH), a biotechnology company leveraging its proprietary DNAbilize liposomal delivery and antisense technology to develop a portfolio of targeted nucleic acid drugs, reported that it has entered into a sponsored research agreement with Thomas Jefferson University to investigate DNAbilize antisense DNA technology for the development of a brain cancer immunotherapy that works by activating the patient’s own immune system to fight their cancer (Press release, Bio-Path Holdings, JUN 28, 2016, View Source [SID:1234513645]).

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D. Craig Hooper, Ph.D., Department of Cancer Biology, Sidney Kimmel Cancer Center at Thomas Jefferson University and the Principal Investigator of the study, previously evaluated Bio-Path’s DNAbilize liposomal delivery and antisense technology in preclinical studies that demonstrated efficacy, suggesting the potential for a systemic antisense immunotherapy for brain cancers. The objective of the collaboration is to demonstrate that DNAbilize delivered systemically would have an effect in redirecting the immune system to fight a patient’s own cancer.

"Brain cancers, such as glioblastoma, are very aggressive and with median survival of about 15 months, novel treatments are urgently needed," said Peter Nielsen, President and Chief Executive Officer of Bio-Path. "We are excited to continue our work with Dr. Hooper to further demonstrate the potential of DNAbilize to safely and systemically deliver a brain cancer immunotherapy. We are entering the immunotherapy market with a unique approach to triggering the immune system to fight a patient’s cancer. This collaboration offers a significant development opportunity for Bio-Path that has the potential to create a second technology platform of immunotherapy products."

On June 27, 2016 ZIOPHARM Oncology, Inc. (Nasdaq:ZIOP), a biopharmaceutical company focused on new cancer immunotherapies, reported the successful completion of enrollment in the first and second dosing cohorts as well as the initiation of enrollment in a third cohort in the Company’s ongoing multi-center Phase 1 study of Ad-RTS-hIL-12 + orally administered veledimex to treat recurrent or progressive glioblastoma (GBM) or grade III malignant glioma (Press release, Ziopharm, JUN 27, 2016, View Source [SID:1234513578]). Ad-RTS-hIL-12 + veledimex is a novel viral gene therapy candidate for the controlled expression of interleukin 12 (IL-12), a critical protein for stimulating an anti-cancer immune response.

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The primary objective of the study is to determine the safety and tolerability of a single intratumoral Ad-RTS-hIL-12 injection activated upon dosing with oral veledimex. Secondary objectives are to determine the maximum tolerated dose, the immune responses elicited, and assessment of biologic response. The first cohort of seven patients received 20 mg doses of veledimex, the second cohort of six patients received 40 mg doses of veledimex, and the third cohort will receive 30 mg doses of veledimex to refine the effect of activating the immune response within the tumor. The resultant immunologic activity that follows IL-12 expression in the brain suggests that no further dose escalation will be necessary and the optimal dosing may be reached sooner than initially anticipated.

Francois Lebel, M.D., Executive Vice President, Research and Development, Chief Medical Officer at ZIOPHARM, commented: "With the RheoSwitch (RTS) technology, the only switch currently in the clinic that operates on gene transcription, we have demonstrated the ability for veledimex to cross the human blood brain barrier and activate production of IL-12 in GBM tumors in a dose-dependent manner, giving us the potential to precisely tune the balance between activity and tolerability."

Data from 11 patients with recurrent high-grade gliomas were recently presented at the 2016 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting. All of these patients failed at least two prior lines of therapy and underwent partial resection leaving residual tumors, in certain cases with significant tumor burden. Ad-RTS-hIL-12 was administered through direct injection into the brain tumor and veledimex was taken orally to activate the production of IL-12 from the tumor site and stimulate an immune response.

As of May 18th, the date of data collection for the ASCO (Free ASCO Whitepaper) presentation, overall median follow up was 6.2 months, with 10 of 11 recipients alive. IL-12 in the bloodstream was measured and was found to be proportional to the amount of veledimex administered, demonstrating that this orally-delivered activator crossed the blood brain barrier to turn on the RheoSwitch technology in a dose-dependent manner.

To date, toxicities in both dose cohorts were consistent with those previously reported, with a higher incidence of grade 3 or greater adverse events in the 40 mg dose group. All related side effects were reversed upon cessation of veledimex. No subsequent deaths have been reported.

The Company expects to present updated results from the study at a scientific meeting later in the year.

"Overall survival remains the gold standard of therapeutic success in glioblastoma, particularly in high-grade, recurrent disease, where survival is too often measured by just a few months," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of ZIOPHARM. "We remain encouraged by the outcomes of Ad-RTS-hIL-12 as a single agent tuning the immune system in this GBM study. We believe that these early results also have positive implications for our combination approach utilizing this novel gene therapy with immune check point inhibitors. We look forward to additional follow up as we work to fine-tune dosing levels using the RheoSwitch technology."

Ad-RTS-hIL-12 + veledimex has been granted Orphan Drug Designation by the U.S. Food and Drug Administration for the treatment of patients with malignant glioma.

About Glioblastoma

Glioblastoma is an aggressive primary brain tumor affecting approximately 74,000 people worldwide each year.i, ii Recurrent glioblastoma is an aggressive cancer with one of the lowest 3-year survival rates, at 3%, among all cancers.iii For patients who have experienced multiple recurrences the prognosis is particularly poor, with a median overall survival (OS) of 6-7 months, while OS in patients that have failed temozolomide and bevacizumab, or equivalent salvage chemotherapy, is approximately 3-5 months.iv, v

On June 27, 2016 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, as amended ("HSR"), with respect to its proposed acquisition of Celator Pharmaceuticals, Inc. ("Celator"; Nasdaq: CPXX) expired effective June 24, 2016, at 11:59 p.m. (Eastern Daylight Time) (Press release, Jazz Pharmaceuticals, JUN 27, 2016, View Source;p=RssLanding&cat=news&id=2180005 [SID:1234513579]).

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As previously announced on May 31, 2016, Jazz Pharmaceuticals and Celator entered into a definitive merger agreement under which Jazz Pharmaceuticals has commenced a tender offer for all of the outstanding shares of Celator at $30.25 per share in cash, representing total consideration of approximately $1.5 billion. The expiration of the HSR waiting period satisfies one of the conditions required to consummate the tender offer. The closing of the tender offer remains subject to other customary conditions, including the tender of a majority of the outstanding shares of Celator common stock.

The tender offer and withdrawal rights will expire at one minute following 11:59 p.m., New York City Time, on July 11, 2016, unless the tender offer is extended or terminated earlier in accordance with the terms of the definitive merger agreement. The Offer to Purchase dated June 10, 2016, relating to the tender offer has been filed with the United States Securities and Exchange Commission ("SEC") and can be viewed online, along with any amendments thereto, at www.sec.gov.