October 30, 2017
Manufacturing Insights

RXi trying to break through to a Nobel $100B market

Brad Kane
Geert Cauwenbergh, president and CEO, RXi Pharmaceuticals Corp., Marlborough.

Founded: 2006

Employees: 15

Age: 63

Residence: Princeton, N.J.

Education: Master in plant biology and marine biology, Ph.D. in medical sciences, University of Leuven, Belgium

For more than a decade, RXi has been developing drugs using RNAi technology, the discovery of which in 1998 won UMass Medical School researcher Craig Mello and his partner Andrew Fire the Nobel Prize. In January, RXi acquired Cambridge immuno-oncology drug developer MirImmune, Inc. to expand into the potentially lucrative space of preventing cancer cells from forming.

What is RXi trying to do?

I'm trying to replace 50 percent of the antibody market with RNAi's.

How can RNAi's treat disease?

They block or inhibit – highly selectively and highly effectively – bad proteins and multi-proteins. Antibodies can only jump to action when the bad protein is already there, so if we can stop the formation of these bad proteins, then antibodies are not needed.

In a number of diseases, such as ALS – Lou Gehrig's disease – there is a protein that is misformed because of some small issue with messenger RNA. You can block that messenger RNA from producing the bad protein, and then the disease is not going to occur.

How big is the potential RNAi market?

RNAi will be a $100-billion market in the next 15-20 years.

How is your drug development coming?

It takes time. The first four or five years have been fine-tuning the cell-delivery technology. RNAi was discovered in 1998 by Craig Mello and Andrew Fire, and they got a Nobel Prize in 2006.

Way before 2006, companies had started trying to explore their discovery for biological reasons. They were failing one after another because they couldn't get the treatment into the body's cells. People then thought, "Hmm, maybe we should get them in the cells."

Craig Mello (chairman of RXi's scientific board) said, "I want us to show not that this works in the clinic; I want us to focus on the right way of getting it into the cell." That has taken awhile.

How does RXi penetrate the cell?

Cell membranes love cholestrol; they need it for survival. So what RXi did was attach a molecule of cholesterol at the head of our RNAi construct, and as a result, the RNAi strands got sucked into the cell as well. Works like a charm.

What diseases are you trying to treat?

The small problem is we are a tiny little company. We got started with $9 million from a hedge-fund guy, who is long gone and made $67 million. While we have very broad intellectual property, we cannot do everything ourselves. So, we are focusing on treating diseases in dermatology and ophthalmology, and we are looking into immuno-oncology, which is the biggest prize to get.

When will RNAi drugs hit the market?

It won't be ours, but RNAi treatments for the kidneys and the liver from other companies probably will come to market in 2019. That is going to be the breakthrough for RNAi. Once you get approval for a new technology, then the whole space opens up and the money pours in; 2019 should be a great year for RNAi.

When will RXi drugs hit the market?

I would say for dermatology, 3-4 years. Ophthalmology could go faster, depending on the medical need. For immuno-oncology, it is not going to happen immediately, but we might be in the clinic in the second half of 2018.

For immuno-oncology because you are dealing with life and death, especially in the later stages of cancer, the Food & Drug Administration has worked with academia and the industry to allow people to move forward as quickly as possible.

Once RNAi becomes a $100-billion market, what becomes of RXi?

We hope to be part of that success. Because of our cell-delivery technology, we may become the favored one.

Will the company be acquired?

There is a reason there are so few small and medium-sized pharmaceutical companies anymore. In order to get a compound to phase three in drug development, you spend $75-100 million. Then, you have to spend another $100-200 million. If you take all the failures of the compounds that didn't make it, in order to get one drug to market, it costs you $2-3 billion. It is more than building a plane.

One day, I assume somebody will make an offer my board and the shareholders cannot refuse.

This interview was conducted and edited for length and clarity by Brad Kane, WBJ editor.


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