On July 22, 2014 ZIOPHARM Oncology reported the expansion of synthetic immuno-oncology programs in conjunction with Intrexon Corporation (NYSE:XON) to include chimeric antigen receptor T-cell (CAR-T) therapy (Press release Ziopharm, JUL 22, 2014, View Source [SID:1234501132]). 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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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 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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

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.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"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.

On July 7, 2014 Novartis reported that the United States Food and Drug Administration (FDA) has granted Breakthrough Therapy status to CTL019, an investigational chimeric antigen receptor (CAR) therapy for the treatment of pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia (r/r ALL) (Press release Novartis, JUL 7, 2014, View Source [SID:1234500616]). The Breakthrough Therapy filing was submitted by the University of Pennsylvania’s Perelman School of Medicine (Penn) which has an exclusive global agreement with Novartis to research, develop and commercialize personalized CAR T cell therapies for the treatment of cancers.

This is the fifth Breakthrough Therapy designation for Novartis, continuing the company’s trajectory as a leader in developing innovative therapies to help treat diseases in which there remains significant unmet medical need. Novartis’ Zykadia(TM) (ceritinib, previously known as LDK378), for the treatment of anaplastic lymphoma kinase positive (ALK+) metastatic non-small cell lung cancer (NSCLC), is one of the first medicines to receive an FDA approval following earlier receipt of Breakthrough Therapy designation by the FDA.

"This Breakthrough Therapy designation underscores the potential of CTL019 as a life-saving therapy for patients with relapsed/refractory ALL, who are in desperate need of new treatment options," said David Epstein, Division Head, Novartis Pharmaceuticals. "Novartis welcomes increased dialogue with the FDA and a potentially expedited review to streamline the development of CTL019 and hopefully bring this promising therapy to patients as quickly as possible."

According to the FDA, Breakthrough Therapy designation is intended to expedite the development and review of new medicines that treat serious or life-threatening conditions if the therapy has demonstrated substantial improvement over an available therapy on at least one clinically significant endpoint. The designation includes all of the fast track program features, as well as more intensive FDA guidance. It is a distinct status from both accelerated approval and priority review, which can also be granted to the same drug if relevant criteria are met.

"This is a major milestone as we are now one step closer in helping address the high unmet needs of this patient population," said Carl H. June, M.D., Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in the Perelman School of Medicine and director of Translational Research in the Abramson Cancer Center of the University of Pennsylvania. "We are excited about the strength of the positive early data seen in pediatric and adult patients with relapsed/refractory acute lymphoblastic leukemia and look forward to building upon these findings as we continue advancing the CTL019 clinical program in Phase II trials."

Novartis recently established the Cell and Gene Therapies Unit under the leadership of Usman Azam, Global Head, to bring an intense focus on advancing innovative cell-based therapies, including the development of CARs. Novartis holds the worldwide rights to CARs developed through the collaboration with Penn for all cancer indications, including the lead program, CTL019.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

On July 7, 2014 APEIRON Biologics reported that a phase I clinical trial with its neuroblastoma immunotherapy APN311 has received all necessary approvals to start recruiting patients. The trial will be locally conducted by Nagoya University Hospital and is part of a long-term collaboration to make this therapy available to patients suffering from this severe type of children’s cancer (Press release, Apeiron Biologics, JUL 7, 2014, View Source [SID:1234502247])r. In Japan, it won a grant by the Japanese government as an Investigator-initiated clinical trial that utilizes collaboration between the Department of Pediatrics and the Center for Advanced Medicine and Clinical Research (CAMCR) at Nagoya University Hospital. "This is a great achievement for us as we have supported this joint effort from the very beginning. Two years ago the ground was prepared when the Austrian Embassy – Commercial Section in Tokyo hosted a scientific meeting for Japanese pediatric oncologists to learn about this innovative therapy of pediatric neuroblastoma developed by Apeiron ", says Dr. Martin Glatz, Commercial Counsellor of the Austrian Embassy. "The meeting two years ago was part of a focus program aiming to bring more Austrian medical research and biotech companies to the Japanese market. The market has seen tremendous changes recently with companies exploring new opportunities and the government addressing regulatory issues," he added. Hans Loibner, PhD, CEO of Apeiron, commented, "We are very happy and proud to be rewarded with this milestone achievement after all the effort that was invested. I would like to particularly thank the physicians from Nagoya University, Dr. Seiji Kojima and Dr. Yoshiyuki Takahashi, as well as Dr. Masaaki Mizuno, Dr. Katsuyoshi Kato and Dr. Shinobu Shimizu at CAMCR, as we owe it to their dedication and tireless work that this trial can now start. We are confident that Japanese patients will benefit from the introduction of APN311 to Japan and look forward to making the next steps towards approval of this promising antibody therapy of neuroblastoma by the Japanese regulatory authorities."

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!