Emerging Drug Developer: Aileron Therapeutics

John Carroll — Mon, 21 Apr 2008 07:57:19 -0400

Aileron Therapeutics ramps up, aims big

When Joseph A. Yanchik III started Aileron Therapeutics back in the middle of 2005, Apple Tree Partners came in and provided the seed money. He started with two employees and devoted half of his time to its development.

And he largely kept quiet as the biotech took its first steps.

“There are a lot of people who are great about talking about changing medicine,” says Yanchik. “Here in Cambridge, you can’t throw a rock in any direction without hitting a company that’s doing it. We were going to CROs to prove to ourselves that this is real, and then and only then start to ramp it up. But we would do it on the data, not purely on the promise.”

Then Apple Tree and Novartis Venture Funds put up a $7 million round. And last week they put in $10 million more as Aileron revved up the work at its own 10,000-square-foot lab.

These days, Yanchik is fully devoted to Aileron. And he’s become much more voluble about its potential.

“We are working on three programs right now and expect to bring in a couple more shortly,” says Yanchik. Aileron’s CEO expects to get a lead program into the clinic in a matter of months, “driving toward proof-of-concept.”

Outsourcing was the key to start advancing the science in the virtual company mode.

“We started to fill in the gaps,” says Yanchik. “And we used some great CROs; 10 at the time, sending bits and pieces of the development all over the globe.”

That outsourcing model of drug development, says Yanchik, is likely to remain the dominant theme for biotech start-ups. “The days of hitting 100 employees and throwing a problem at them,” he says, “is largely gone.”

Aileron bills itself as a developer of stapled alpha-helical peptides that penetrate cells. The therapeutics are directed at intra-cellular protein-protein interactions that are not influenced by small molecule drugs or biologics.

“This is a completely new way of creating therapeutics,” says Yanchik. “We’re taking bad guy cells most guys are dealing with and turning them back into good guys, using the lock and key that nature created.” Take insulin as an example: “What if you turn cells back on to produce insulin again?”

It’s not a new concept. The research was pioneered by scientists at Harvard and Dana Farber Cancer Research Institute.

“If you know what a peptide is--a component of a protein--we are stripping out of the protein a key,” says Yanchik. “Most disease is controlled by protein-protein interactions within a cell. These are the control points. If you can get at them, you can affect theoretically any disease. It can be locked on or locked off.”

“Peptides are the keys that go into the locks that turn them on or off, the challenge has always been--and this is not a new concept--understanding how to harness them and turn them into drugs. There are peptides on the market like insulin, the problem has been the pharmacokinetics. They’re put in and metabolized and spit out. With insulin, you have to keep giving it.”

To turn peptides into cures, “you need to take peptides and give them small-molecule like properties.”

In animal studies, for example, Aileron has developed a program that spurs apoptosis, eliminating cancer cells. The developer presented data on attacking liquid and solid tumors at the American Association for Cancer Research meeting in San Diego.

To visualize what Aileron is doing, he says, you should think of a protein as a large structure made up of a series of Slinkys – the alpha helix. Snip out one and they unwind. Then the body eats it up and spits it out. But if you pinch a couple of the turns of the Slinky, it can’t unwind, and gets into the cell. “That’s absolutely key.”

If you take all of the four major categories of drugs in development, says Yanchik, you’ll come up with 500 or 600 targets. But stapled peptide technology can have an extraordinarily broad reach.

“With the alpha-helical structure, the Slinky, we can go to 1,000 to 2,000 targets, triple what the entire industry is,” says Yanchik. “The trick is how to get the damn things to work, so they can’t get flushed out in two minutes. Also, almost all drugs treat problems that already exist. What we’re attempting to do is provide a cure. We’re the first to show a gain of function.”

It’s a big task, but Yanchik sees the company staying small. With the new venture funds in hand, Aileron has hired up on the science side-growing the staff to 15--and lit up its own research facility. By the end of the year, the payroll may grow to 18 or 20 people.

Says Yanchik: “We are working on three programs right now and expect to bring in a couple more shortly.”

There are a number of different ways the company could grow. Development collaborations are likely. There could be new venture money, eventually a possible public offering or an acquisition. Given the state of the public markets, says Yanchik, “the likely exit is an acquisition.”

“In the last 90 days we’ve transformed from a virtual to an internal operation,” he adds. “The near term plan is to fire that up and drive this up to the clinic. We’ll continue to talk potential partnerships and when the right ones surface we’ll certainly be ready.”

Aileron, Heritage Pharma each raise $10M in VC

Tue, 15 Apr 2008 06:59:55 -0400

Novartis Venture Funds and Apple Tree Partners have teamed up to lead a $10 million venture round for Cambridge, MA-based Aileron Therapeutics. Aileron says it will use much of the money to expand its research staff and open a 10,000-square-foot research facility. This brings its total venture funds to $20 million.

"With our broad understanding of the design and application of stapled peptides, Aileron is now focused on developing our product pipeline including--driving Aileron's lead programs to the clinic and expanding our ability to pursue new therapeutic indications and targets, such as transcription factors, that have now been shown to be within the reach of our biologic platform," said CEO Joseph A. Yanchik III.

- check out the report from Mass High Tech
- and check out the press release

ALSO: Heritage Pharmaceuticals has completed a $10 million first venture round with McClendon Venture Company. Release

PLUS: The start-up Anaphore in San Diego has pocketed $8 million in a round led by 5AM Ventures and Versant. Release

Joseph Yanchik

Emerging Drug Developer: Aileron Therapeutics

Aileron, Heritage Pharma each raise $10M in VC