Synthetic Biology companies Ziopharm and Intrexon

1. Ziopharm Oncology Inc’s Chief Executive Jonathan Lewis plans to produce less expensive new cancer drugs using synthetic biology.

Lewis says Ziopharm’s most advanced drug in development, palifosfamide for sarcoma and lung cancer, can be a $1 billion a year seller even if reasonably priced. But the linchpin to Ziopharm producing less expensive biotech drugs is an experimental new technology known as synthetic biology developed by its partner Intrexon Corp.

Lewis sees synthetic biology transforming biotech drug manufacture the way Henry Ford’s assembly line changed automobile production.

The current expensive and complicated biotech production model involves protein based medicines manufactured in huge vats in carefully controlled environments. Ziopharm won’t use great vats to create the protein, but use the person to create the protein. Ziopharm expects to file five investigational new drug applications (INDs) seeking approval to begin human trials using synthetic biology next year and eight more in 2013.

Ziopharm drugs

ZIOPHARM Oncology is a biopharmaceutical company engaged in the development and commercialization of a diverse portfolio of cancer therapeutics. The Company is currently focused on several clinical programs.

Palifosfamide (Zymafos™ or ZIO-201) is a novel DNA cross-linker in class with bendamustine, ifosfamide, and cyclophosphamide. ZIOPHARM is currently enrolling patients in a randomized, double-blinded, placebo-controlled Phase III trial with palifosfamide administered intravenously for the treatment of metastatic soft tissue sarcoma in the front-line setting. The Company is also currently conducting a Phase I intravenous study of palifosfamide in combination with standard of care addressing small cell lung cancer and an oral form of the drug for treatment of solid tumors is currently in the advanced preclinical stage of development.

Darinaparsin (Zinapar™ or ZIO-101) is a novel mitochondrial-targeted agent (organic arsenic) being developed intravenously for the treatment of relapsed peripheral T-cell lymphoma likely with a two-stage potentially pivotal study expected to begin in late 2011. An oral form is in a Phase I trial in solid tumors.

Indibulin (Zybulin™ or ZIO-301) is a novel, oral tubulin binding agent that is expected to have several potential benefits including oral dosing, application in multi-drug resistant tumors, no neuropathy and a quite tolerable toxicity profile. It is currently being studied in Phase I/II in metastatic breast cancer.

ZIOPHARM is also pursuing the development of novel DNA-based therapeutics in the field of cancer pursuant to an exclusive channel partnership with Intrexon Corporation. The partnership includes two existing clinical-stage product candidates, the first of which is in a Phase Ib study and the second, entering Phase I study.

ZIOPHARM Oncology, Inc. a drug development company employing small molecule and synthetic biology approaches to cancer therapy, announced today that the U.S. Food and Drug Administration (FDA) has accepted the Company’s investigational new drug (IND) application to begin clinical study of ZIN ATI-001, a novel DNA-based therapeutic candidate also known as Ad-RTS-IL-12 + AL (INXN 2001/1001), in oncology. When initiated, the Phase I study will evaluate safety in addition to immunological and biological effects of the therapeutic candidate in patients with melanoma. ZIN ATI-001 is the second clinical oncology product candidate from the ZIOPHARM-Intrexon Corporation exclusive synthetic biology channel partnership.

ZIN ATI-001 employs an adenoviral vector to deliver, directly into the patient’s own cells, a gene which expresses Interleukin-12 (IL-12), a potent, naturally occurring anticancer cytokine central to the initiation and regulation of cellular immune responses. Production of IL-12 within cells is then tightly regulated by the Intrexon RheoSwitch Therapeutic System™ (RTSTM), a “gene switch” controlled by an orally administered activator ligand (AL). Preclinical studies have shown that the immunological mechanism of action of ZIN ATI-001 is similar to that of ZIN-CTI-001 (DC-RTS-IL-12 + AL), ZIOPHARM’s most advanced DNA-based product candidate, currently in Phase Ib. Positive clinical data of ZIN-CTI-001, the first-ever to demonstrate small molecule-controlled production of an anticancer protein in humans, were recently presented at the 2011 Annual Meeting of the American Society of Clinical Oncology.

2. Intrexon is Ziopharm’s partner

Intrexon Corporation, a next generation synthetic biology company, today announced that it has closed a Series E preferred investment round of $100 million, bringing the company’s total capital raised to more than $259 million. New investors took the majority of the round while existing investors, including Randal J Kirk, Intrexon’s Chairman and CEO, and Third Security, LLC, took the remainder. Perella Weinberg Partners acted as Intrexon’s exclusive financial advisor and placement agent.

The funding will be used to provide working capital for Intrexon’s commercial divisions and the expansion of its UltraVector® platform. UltraVector, which is the underlying architecture and construction methodology for the company’s complex transgenes, is the backbone of Intrexon’s activities in human health care, animal science, agricultural biotechnology and industrial products.

Intrexon technologies:

UltraVector enables the architecture and construction of complex, integrated biofunctional systems featuring multi-program networks and highly variable componentry.

DNA and RNA MOD Engineering enabling rapid development promotors, chromatin modifiers, bioactive RNA species, and RNA motifs to regulate stability or control translation initiation.

Protein Engineering for enhanced or novel biofunctionalities such as novel enzyme inhibitors, localization signals, and fusion proteins with multivariate functionality.

Transcriptional Control Chemistry powering monogenic and multigenic systems inducible through a comprehensive library of activator ligands.

Genome Engineering featuring stable, location-based integration that doesn’t disrupt critical cellular functions.

Cell System Engineering enabling controlled expression of individual traits or entire biosynthetic pathways, with subcellular positioning and enzyme control

Combined with an advanced, rapid design-build-test framework, the outcome is Better DNA™ —DNA that produces the exacting results you desire at a molecular and cellular level. Intrexon’s core technology is a means to rationally engineer biological applications at an unprecedented scale. To our partners, however, it is simply a means to redefine what is possible

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