Cancer may soon meet its match. A recent biotech partnership between biologic products leader Mesoblast, synthetic biology leader Intrexon (XON +4.29%), and developmental biotech Ziopharm Oncology (ZIOP +0.00%) aims to develop a new class of cancer therapeutics. If the first molecules show promise in treating lung cancer in preclinical studies, the trio will form a joint venture with the hope of revolutionizing cancer treatment. What, exactly, does each company bring to the table? And where does their idea rank in terms of innovative approaches to treating cancer?
Partnership perfection
This is truly a beautiful industry partnership. Mesoblast will contribute its novel Mesenchymal Lineage Cells, or MLCs, which are stem cells that are found in a number of tissues throughout the body. They're ideal for therapeutic use because they can be produced on an industrial scale with current biomanufacturing technology and are allogenic, meaning they are accepted by all donors. While MLCs can be used for regenerative applications -- such as forming new blood vessels or regenerating heart tissue -- by releasing factors that act on a target tissue, the current partnership will develop MLCs that target specific tumors.
Doing so will require the use of two proprietary Intrexon platforms involved in the current exclusive channel collaboration with Ziopharm. First, Ziopharm will use the company's UltraVector platform to design and optimize MLCs that express therapeutic genes for battling lung cancer. Second, Ziopharm will use Intrexon's RheoSwitch Therapeutic System to control when and where in the body the engineered MLCs become activated -- optimizing the delivery of pharmaceutical payloads.
The timing of the partnership is no coincidence. Ziopharm has been refocusing its clinical efforts on its growing synthetic biology platform after its former lead drug candidate failed a late-stage trial. Of course, Intrexon's 16% stake in the company gives it considerable power in directing operations. And since Intrexon isn't directly involved in the partnership (it's between Mesoblast and Ziopharm, although the latter is utilizing technology from its partnership with Intrexon) you can bet it's doing a lot of behind the scenes work, especially with biotech guru Randal Kirk at the helm.
Meanwhile, Mesoblast is now the world leader in MLC technology after acquiring the competing and complimentary patent portfolio of Osiris Therapeutics (NASDAQ: OSIR) in October. The $50 million deal comes with the potential for an additional $50 million in milestone payments, plus royalties. It will allow Osiris to focus outside of the risky field of regenerative medicine -- similar to a recent move by Geron -- and will allow Mesoblast more flexibility in commercializing its technology without fearing possible patent lawsuits.
Most innovative?
It is still way too early to call this the most innovative oncology platform -- the therapeutics described have yet to even be designed, let alone tested. However, the partnership is part of a larger trend focused on exploiting the weaknesses of cancer tissues and strengths of the body's own immune system through bioengineering and genomic analysis. The concept of anti-cancer MLCs is an very innovative way to combine the industry's understanding of biology in an attempt to treat cancer. What other innovative ideas could revolutionize cancer?
Amgen (AMGN 1.17%) has one of the best just-so-crazy-it-might-actually work ideas for the future of oncology treatments. Talimogene laherparepvec, or TVEC, is an engineered form of the herpes-causing virus HSV-1. Yep, you read that right. The company replaced two genes that allow HSV-1 to defeat the body's immune system with a human gene responsible for producing a growth factor that rallies the body's immune system. In a phase 3 trial evaluating the approach in late-stage melanoma, TVEC demonstrated a durable response rate of 16% compared to injectable forms of granulocyte-macrophage colony-stimulating factor, or GM-CSF (white blood cell growth factors hard-coded into TVEC's genetic code). A similar trial in metastatic melanoma demonstrated a 19% to 1% edge.
Anti-cancer MLCs also have to contend with the novelty of PD-1 drugs being developed by AstraZeneca, Roche, Merck, and Bristol-Myers Squibb. The new class of drugs has hinted at being incredibly effective and well-tolerated in early and mid-stage trials. Better yet, they can likely be developed to treat a range of cancers.
Foolish bottom line
It may be early, but given the exceptional precision in gene expression allowed by synthetic biology and the therapeutic and allogenic nature of MLCs this trifecta is definitely worth keeping an eye on. Ziopharm may be small and lack revenue, but I'm confident that Intrexon can guide its investment to success if the technology proves feasible. Overall this is a great development for the long-term future of Intrexon's business, although it is still years away from providing a meaningful boost to shares. I'll still be sticking to the sidelines.
