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.

On June 24, 2016 Pfizer Inc. (NYSE:PFE) reported that it has completed its acquisition of Anacor Pharmaceuticals, Inc (Press release, Pfizer, JUN 24, 2016, View Source [SID:1234513545]). Under the terms of the transaction, each outstanding share of Anacor common stock has been converted into the right to receive $99.25 net in cash (without interest but subject to required withholding of taxes).

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"Now that Anacor is part of Pfizer, we can accelerate our shared commitment to help patients with inflammatory disease, an area of high unmet medical need," said Albert Bourla, Group President, Pfizer Innovative Health. "We believe that Pfizer is in a position to quickly capitalize on the benefits offered by the combination with Anacor, including the potential for a near-term U.S. product launch and subsequent commercialization of crisaborole, a differentiated asset with compelling clinical data. If approved, crisaborole has the potential to be an important first-line treatment option for patients with mild-to-moderate atopic dermatitis and the physicians who treat them."

The transaction is not expected to impact Pfizer’s current 2016 adjusted financial guidance. Pfizer continues to expect the transaction to be slightly dilutive to Adjusted Diluted Earnings Per Share (EPS)(1) in 2017 with accretion to Adjusted Diluted EPS(1) beginning in 2018 and increasing thereafter.

The Offer

The tender offer for all of the outstanding shares of Anacor common stock expired as scheduled immediately after 11:59 p.m., New York City time, on June 23, 2016. Computershare Trust Company, N.A., the depositary and paying agent for the tender offer, has advised Pfizer that 39,306,909 shares of Anacor common stock were validly tendered into and not validly withdrawn from the tender offer, including 4,300,427 shares tendered by notice of guaranteed delivery for which certificates were not yet delivered, representing approximately 86.1% of the outstanding shares. All of the conditions to the offer have been satisfied and on June 24, 2016, Pfizer and its subsidiary Quattro Merger Sub Inc. accepted for payment and will promptly pay for all shares validly tendered and not validly withdrawn.

Following its acceptance of the tendered shares, Pfizer completed its acquisition of Anacor through the merger of Quattro Merger Sub Inc. with and into Anacor without a vote of Anacor’s stockholders pursuant to Section 251(h) of the Delaware General Corporation Law. As a result of the merger, Anacor became a wholly-owned subsidiary of Pfizer. In connection with the merger, all Anacor shares not validly tendered into the tender offer (other than treasury shares held by Anacor and any shares owned by Pfizer, Quattro Merger Sub Inc. or any person who was entitled to and has properly demanded statutory appraisal of his or her shares) have been cancelled and converted into the right to receive the same $99.25 per share net in cash (without interest but subject to required withholding of taxes) as will be paid for all shares that were validly tendered and not validly withdrawn in the tender offer. Anacor common stock will cease to be traded on the NASDAQ Global Market.

On June 23, 2016 ArQule, Inc. (Nasdaq:ARQL) reported that preliminary data from the ongoing phase 2 portion of a phase 1/2 trial in intrahepatic cholangiocarcinoma (iCCA) with our proprietary fibroblast growth factor receptor (FGFR) inhibitor, ARQ 087, will be presented on June 30, 2016 at the ESMO (Free ESMO Whitepaper) 18th World Congress on Gastrointestinal Cancer in Barcelona, Spain (Press release, ArQule, JUN 23, 2016, View Source [SID:1234513506]). This is a biomarker driven trial designed to enroll at least 20 patients in iCCA with FGFR2 genetic alterations. The company has been granted orphan drug designation by the U.S. Food and Drug Administration and European Medicine’s Agency for ARQ 087 in this indication.

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Presentation Details
Abstract Number: 340 (PD #19)
Poster Title: ARQ 087, an Oral Pan-Fibroblast Growth Factor Receptor (FGFR) Inhibitor, in Patients with Advanced and/or Metastatic Intrahepatic Cholangiocarcinoma (iCCA)
Poster Discussion Time: June 30, 2016 from 11:00 a.m. to 11:30 a.m. CEST
Poster Presentation Time: June 30, 2016 from 5:10 p.m. to 5:40 p.m. CEST
Location: Exhibit Hall

About Intrahepatic Cholangiocarcinoma

Cholangiocarcinoma (CCA) is the most common biliary malignancy and the second most common hepatic malignancy after hepatocellular carcinoma (HCC)1. Depending on the anatomic location, CCA is classified as intrahepatic (iCCA), perihilar (pCCA), and extrahepatic (eCCA). iCCA originates from the intrahepatic biliary ductal system and forms an intrahepatic mass. The average age adjusted incidence rate for iCCA is approximately one in 100,000 per year in the United States and Europe2,3.

About FGFR and ARQ 087

ARQ 087 is a multi-kinase inhibitor designed to preferentially inhibit the fibroblast growth factor receptor ("FGFR") family with demonstrated efficacy in FGFR2 genetic alterations. The FGFR pathway is disrupted in several ways in human cancer, thus providing numerous therapeutic targets for an inhibitor of this pathway. ARQ 087 has demonstrated in vivo inhibition of tumor growth and downstream signaling in tumors whose growth is driven by FGFR targets.

Signals of single agent activity with this drug were observed in phase 1a testing. Phase 1b expansion cohorts with ARQ 087 include patients with cholangiocarcinoma and adrenocortical tumors, as well as those with FGFR translocations, amplifications and mutations. Clinical development of ARQ 087 has advanced into phase 2 for intrahepatic cholangiocarcinoma (iCCA) following the observation of two confirmed responses in this patient population in the phase 1 portion of the program.

On June 23, 2016 Mylan N.V. (NASDAQ, TASE: MYL), one of the world’s leading pharmaceutical companies, reported a €1 million donation to the Princess Máxima Center for Pediatric Oncology, a new national cancer center currently being built in Utrecht, the Netherlands (Press release, Mylan, JUN 23, 2016, View Source [SID:1234513522]).

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The donation from Mylan will support the Princess Máxima Center in its mission of treating and curing children with cancer by enhancing research efforts and supporting patients and their families while at the center for treatment.

Mylan Executive Chairman Robert J. Coury commented, "Mylan’s mission is to set new standards in healthcare and provide access to high quality medicine to patients around the world; however, our mission starts with the communities in which we operate, and we have had the privilege to serve customers and patients in the Netherlands for more than 17 years. The mission of the Princess Máxima Center is closely aligned with Mylan’s and I would like to commend Prof. Pieters for his vision and leadership in raising the standard of care for cancer in the Netherlands and ensuring children with cancer have equal access to the best care available."

Mylan CEO Heather Bresch added, "Every two days a child dies from cancer in the Netherlands and one in four children does not survive their fight with this disease. Through our commitment to providing access to oncology medications to patients around the world, we strive to do our part to help treat and cure cancer, and we believe our partnership with The Princess Máxima Center is just one of the ways we can go beyond medicine to deliver better health for a better world."

Professor Pieters, member of the board of the Princess Máxima Center commented, "We are grateful to Mylan for their generous support of the Princess Máxima Center, which is focused on treating children with cancer using the best possible methods, while also preventing the development of negative side effects in young patients, both now and over the long term. By bringing together expertise in care and research in the Princess Máxima Center, the Netherlands will become a leader in the field of pediatric oncology both in Europe and globally. It is a position that will help us fulfill our ambition: to cure every child with cancer. I am delighted that our collaboration with Mylan will further support the care we provide and our continued research."

Matean Niël, Mylan country manager in the Netherlands added: "I am thrilled to see Mylan supporting a cause in the Netherlands that puts people and patients first. Mylan’s support of the Princess Máxima Center creates a lot of pride among our hundreds of employees based in Bunschoten, Weesp and Zwolle. This initiative reflects our local commitment to institutional care in the Netherlands."