On July 22, 2014 ZIOPHARM Oncology reported the expansion of synthetic immuno-oncology programs in conjunction with Intrexon to include chimeric antigen receptor T-cell (CAR-T) therapy (Press release Intrexon, JUL 22, 2014, View Source;p=RssLanding&cat=news&id=1949640 [SID:1234500637]). Additionally the Company has provided an update on its development efforts with the proprietary RheoSwitch Therapeutic System (RTS) platform, an inducible regulator for expression of therapeutic molecules through administration of an oral activator ligand, as well as its clinical program with Ad-RTS-IL-12, a novel DNA-based therapeutic candidate for the controlled expression of IL-12.

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CAR-T cells represent an emerging, high value immunological therapy that can target and destroy cancer cells displaying "personalized" fingerprints, yet current approaches feature challenges associated with toxicity, off-target effects, and uneconomical manufacturing. Intrexon possesses the integrated technology platforms, molecular engineering, systems biology, and cell engineering capabilities required to overcome these challenges and fully realize the potential of CAR‐T cell therapies. Most significantly, utilization of the RTS platform will facilitate exquisite regulation of one or more bioeffectors in CAR-T cells enabling physicians to control systemic effects of cell therapies with an appropriate dosing regimen of the oral activator ligand (veledimex), and eventually bring about improved safety and efficacy of these and related therapeutic strategies. Further preclinical work is underway in this promising area of study, and ZIOPHARM and Intrexon expect to provide a progress update in the second half of 2014.

Samuel Broder, M.D., EVP of Scientific and Public Affairs at Intrexon, said, "As a leader in the second generation of biotechnology, Intrexon is applying industrial engineering principles to synthetic immunology to potentiate important biotechnology platforms enabling end-to-end solutions for complex biologic challenges. In particular, the utilization of our proprietary RheoSwitch platform may be especially advantageous in CAR-T treatments."

To date, the RheoSwitch platform has been shown to function as a regulatable switch in an array of cell types for multiple proteins, and RTS expansion into CAR-T therapy is further demonstration of the breadth of Intrexon’s single and multi-genic expression and control technologies. According to data from ClinicalTrials.gov, more than 1,000 clinical trials utilizing gene therapy are currently underway, with the majority in either Phase I or Phase II. Intrexon’s proprietary switch system is uniquely positioned as the first clinically-evaluated gene switch with in vivo data showing the ability to control gene expression with a broad dynamic range. The RTS platform provides a mechanism for titrating therapeutic effects on a patient-specific and predictable basis, as well as a safety switch to rapidly turn off gene-expression. The ability to administer or withdraw the veledimex pill to sustain continued treatment cycles is a key benefit exclusive to RTS technology.

"We are excited by the prospects of applying our advanced synthetic immuno-oncology toolkit towards targeted immunotherapies like CAR-T," said Jonathan Lewis, M.D., Ph.D., CEO of ZIOPHARM. "We also look forward to expanding RTS applications in novel therapeutic strategies for cancer where the ability to control gene expression is essential."

With respect to the Ad-RTS-IL-12 clinical programs, ZIOPHARM continues to conduct Phase II studies in melanoma and breast cancer using Ad-RTS-IL-12 as a monotherapy. Additionally, the Company is evaluating future trials with IL-12 in potential combination therapies with other immune-targeting agents in various cancers including melanoma and breast. ZIOPHARM also plans to initiate a Phase I trial to evaluate Ad-RTS-IL-12 as a single agent in the treatment of patients with Glioblastoma Multiforme in the second half of 2014.

"The development of potent yet tightly controlled cancer depleting therapies such as CAR-T and other targeted cellular products through the molecular rewiring of immunologic gene programs adds to the foundation of our multifaceted strategy in synthetic immuno-oncology," remarked Gregory Frost, Ph.D., SVP of Intrexon’s Health Sector.

On July 22, 2014 MonoSol Rx, the developer of PharmFilm drug delivery technology, reported that it has licensed Zuplenz (ondansetron) to Galena Biopharma, a biopharmaceutical company developing and commercializing innovative, targeted oncology treatments that address major unmet medical needs to advance cancer care (Press release, MonoSol Rx, JUL 22, 2014, View Source [SID:1234508915]). Zuplenz is an oral soluble film (OSF) for the prevention of chemotherapy-induced, radiotherapy-induced, and postoperative nausea and vomiting.

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The licensing agreement includes an undisclosed upfront payment from Galena, as well as double digit royalty payments from sales of Zuplenz. Zuplenz is the first oral soluble lingual film product approved by the U.S. Food and Drug Administration (FDA) as a prescription medication, based on MonoSol Rx’s proprietary PharmFilm technology.

Keith J. Kendall, Co-President and Chief Operating Officer of MonoSol Rx, remarked, "Partnering with Galena Biopharma to provide Zuplenz to patients in need further reinforces MonoSol Rx’s position as a leader in the oral soluble film pharmaceutical market. Through this partnership, we hope to empower supportive care providers with a more comfortable, easier-to-use formulation of ondansetron that addresses a significant unmet medical need, particularly among cancer patients. With the increased availability of Zuplenz, clinicians will be able to improve the level of care they provide by utilizing our innovative drug delivery technology to eliminate swallowing issues and discomfort associated with chemotherapy, radiotherapy, and surgery."

"We are pleased to be working with MonoSol Rx to offer Zuplenz to patients in need," said Mark J. Ahn, Ph.D., President and Chief Executive Officer of Galena Biopharma. "MonoSol Rx’s oral film delivery technology represents a substantive advancement in terms of ease of use and compliance for supportive care patients, where difficulty swallowing can be a major issue. Providing patients with an alternative solution for nausea and vomiting has long been a concept that oncologists and the general surgeon and oncology nurse communities have sought, and that we are eager to deploy."

Zuplenz is a unique formulation of ondansetron using MonoSol Rx’s proprietary PharmFilm technology, co-developed with partner APR Applied Pharma Research s.a. of Switzerland. It is based on a novel and proprietary oral drug delivery technology platform and consists of a polymeric OSF containing ondansetron. Once placed in the mouth, it dissolves in a few seconds, allowing the medication to be absorbed and delivered without water.

On July 22, 2014 OBI Pharma reported hat it has completed patient enrollment in its Phase 2/3 clinical trial evaluating the safety and efficacy of its lead compound, OBI-822, an active immunotherapy for the treatment of metastatic breast cancer (Press release OBI Pharma, JUL 22, 2014, View Source;fn=news_content&no=11 [SID:1234501492]).

"Completing enrollment in the Phase 2/3 clinical trial is a major milestone in the development of OBI-822", said Michael N. Chang, Ph.D., Chairman of the Board of OBI. "Our next target is a global phase 3 trial – now that there is a current IND with the US FDA and an IND application already submitted in China. We look forward to the results of this trial being confirmed once the course of treatment is completed in all enrolled patients early next year."

"The cancer active immunotherapy, OBI-822 guides the immune system to attack breast cancer cells, with minimal side effects, [as observed in the OBI’s Phase 1 and Phase 2/3 clinical trials]" said Amy Huang, General Manager of OBI. "OBI-822’s anti-cancer potential goes beyond breast cancer. All tumor cells expressing Globo series glycan (Globo H, SSEA-3 and SSEA-4) are possible treatment targets for OBI-822. According to the latest research findings reported by Taiwan’s Academia Sinica, the Globo H vaccine can potentially treat up to 16 different types of cancer. In fact, OBI Pharma and Mackay Memorial Hospital jointly initiated a Phase 2 ovarian cancer trial in November 2013 which is currently on-going."

Earlier clinical results
Phase 1 studies of the OBI-822/OBI-821 combination conducted at MSKCC induced both IgM and IgG responses demonstrating that Globo H-specific antibodies can effectively induce complement-mediated cytotoxicity as well as antibody-dependent cytotoxicity. The most commonly observed side effects in the Phase I clinical studies of metastatic breast and prostate cancers were mild local skin reactions and minor flu-like symptoms.

Clinical Study Design
The Phase 2/3 randomized controlled trial (RCT) was initiated in December, 2010 with an enrollment target of 342 subjects. The study enrolled female subjects with metastatic breast cancer who achieved stable disease (SD), partial response (PR), or complete response (CR) after at least 1 regimen of anticancer therapy. Subjects were randomized to receive either active treatment (OPT‐822/OPT‐821) plus cyclophosphamide or control (PBS) plus cyclophosphamide in a 2:1 allocation.

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On July 11, 2014 ProStrakan Group plc("ProStrakan") a subsidiary of global specialty pharmaceutical company, Kyowa Hakko Kirin Co.,Ltd. ("KHK"), reported that it has entered into an agreement with Novo A/S, a private limited liability company fully owned by the Novo Nordisk Foundation,to purchase the entire share capital of Archimedes Pharma Limited ("Archimedes") for a consideration of £230 millionin cash (Press release, ProStrakan, JUL 11, 2014, View Source [SID1234572119]). Archimedes is a fast growing European-based specialty pharmaceutical company providing novel and advanced treatments within the therapeutic areas of pain, oncology and critical care. Its largest product is PecFent, a fentanyl nasal spray used in the management of breakthrough cancer pain in patients who are already receiving maintenance opioid therapy for chronic cancer pain. The product was developed using Archimedes’ proprietary drug delivery technology platform,which is protected by several patents. Archimedes also has a diversified product portfolio of both promoted and non-promoted pharmaceutical products marketed to healthcare professionals across Europe. It has a strong European footprint with commercial operations in the UK, France, Germany and Spain. Archimedes is based in Reading, UK, and has a development facility in Nottingham, UK. In 2013, Archimedes recorded revenues of £41million, growing33% from the previousyear.

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ProStrakan’s acquisition of Archimedes furthers KHK’s strategic vision of becoming a global specialty pharmaceutical company. The acquisition of Archimedes also delivers several strategic benefits to ProStrakan. The transaction significantly expands ProStrakan’s critical mass in Europe by growing its businesses in four of the largest European markets: UK, France, Germany and Spain. In each of these countries Archimedes has sales and marketing infrastructure which will complement ProStrakan’s current operations. In 2013, ProStrakan generated revenues of £155million, growing 20% from the previous year. Archimedes brings a strong product portfolio of high growth brands which expands ProStrakan’s offering in the core therapeutic areas of oncology and oncology supportive care. These brands are expected to be a key driver of growth for ProStrakan over the next several years. In addition, the enhancement of the infrastructure and portfolio broadens the reach of ProStrakan,helping it to position itself for the launch of several exciting new products currently being developed by ProStrakan’s parent company, KHK. One of these products, KW-0761, is a novel antibody being developed for haematology indications such as adult T-cell leukaemia/lymphoma. Dr Tom Stratford, Chief Executive Officer of ProStrakan, said :"The acquisition of Archimedes represents a rare opportunity to enhance the scale of our successful European operations by adding a strong portfolio of high value medicines as well as further strengthening our teams in major European markets. "Since joining KHK three years ago, ProStrakan has grown from strength to strength, culminating in the delivery of a positive profit contribution for the KHK group in 2013. The acquisition of Archimedes is a further step in KHK’s strategy of becoming a global specialty pharmaceutical company, as it significantly expands its presence in Europe."

Closing of the transaction is subject to anti-trust approvals in Germany .Lazard is acting as exclusive financial advisor to KHK and ProStrakan, and Wragge Lawrence Graham & Co. is acting as legal counsel to KHK and ProStrakan

On July 10, 2014 Bristol-Myers Squibb reported that it, following discussions with the U.S. Food and Drug Administration (FDA), the company is planning a third quarter submission of a Biologics Licensing Application (BLA) for Opdivo (nivolumab) for previously treated advanced melanoma (Press release Bristol-Myers Squibb, JUL 10, 2014, View Source [SID:1234500639]). This will mark the second tumor type for which Bristol-Myers Squibb has a regulatory submission underway for Opdivo in the U.S.

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"We continue to collaborate closely with the FDA on Opdivo and the planned submission in advanced melanoma represents an important step forward in our company’s commitment to deliver innovative treatment options for patients with cancer," said Michael Giordano, MD, Head of Oncology Development, Bristol-Myers Squibb.

The advanced melanoma BLA is based on data from Checkmate -037, a multinational, multicenter, randomized open-label Phase 3 trial evaluating Opdivo compared to dacarbazine (DTIC) or carboplatin/paclitaxel in patients with unresectable or metastatic melanoma who have been previously treated with Yervoy (ipilimumab) and, if BRAF-mutation positive, a BRAF inhibitor regimen.

Bristol-Myers Squibb has proposed the name Opdivo (pronounced op-dee-voh), which, if approved by health authorities, will serve as the trade name for nivolumab.