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BioWorld: “Myos, Cloud collaborate to 'muscle in' on new therapeutic category”

Originally published in BioWorld™ Today, Volume 25, No. 138
Written by Marie Powers, BioWorld Staff Writer

Myos Corp., which is taking a bifurcated approach in developing products to improve muscle mass, increase mobility and reduce frailty, is going to the "cloud" through a research and development agreement with newco Cloud Pharmaceuticals Inc. Although terms were not disclosed, the partners will focus on identifying and advancing candidates that inhibit targets in the myostatin regulatory pathway and inflammatory mediators associated with sarcopenia and cachexia.

Myos – Greek for "muscle" – will retain rights to the design and structure of any small molecules developed for the muscle health treatment market. Cloud, which is seeking to accelerate early stage drug discovery using cloud computing technology for the synthesis and design of small molecules, will focus its collaborative efforts on furin regulation and Janus kinase 3 (JAK3) inhibition, with rights to move forward with JAK3 work in indications outside muscle health, according to CEO Ed Addison.

Furin is a prototypical proprotein convertase that processes latent precursor proteins into their biologically active forms. Furin convertase plays a central role in the processing of myostatin to its biologically active form, serving as a potentially important target for development of therapeutic candidates. JAK3 functions in signal transduction and is thought to be a factor in complex biochemical pathways central to inflammation.

Candidates created as part of the research relationship will be the first small molecules for Myos as potential therapies to treat age-related muscle loss, or sarcopenia, in addition to cachexia, a wasting syndrome that can occur in patients with cancer, AIDS, congestive heart failure, multiple sclerosis and other chronic illnesses.

The companies hope to report on initial target testing with furin convertase by the end of next year and to identify a candidate to move into an investigational new drug application within 24 months, according to Peter Levy, chief operating officer of Myos, based in Cedar Knolls, N.J. Meanwhile, Myos will continue to assess and identify additional small-molecule candidates for design and evaluation by Cloud, based in Research Triangle Park, N.C.

Myos was attracted by Cloud's use of high-performance computational methodologies to make rational drug design decisions, according to Bob Hariri, executive chairman of Myos.

"We're at a very unique time where the toolsets available to scientists are increasing at a rate beyond Moore's Law," Hariri told BioWorld Today. "If you use a computer as a computational tool to screen, for example, binding domains on a target, you'll find there are 50 or 100 candidate molecules out there already, and there are dramatically more synthetic opportunities available to come up with candidates that would work."

That type of systemized approach, he said, is far superior to the "serendipity" associated with traditional drug discovery.

"At Myos, the team has been looking at some important regulatory pathways, where there's a lot known about specific receptor-mediated events and specific cell-signaling events," he said. "It seemed like a logical place to connect that drug design capability to a company seeking to better understand how to manipulate and control the processes that are behind the growth and retention of muscle mass."

'A VERY POWERFUL REVENUE-GENERATING ENGINE'

The deal actually came together after Kenneth Sorensen, managing director of Array Capital Management and a member of Cloud's board, introduced Addison to Hariri, who previously founded Anthrogenesis Corp., which was acquired by Summit, N.J.-based Celgene Corp. in 2002 for $45 million. Hariri then led the Celgene Cellular Therapeutics unit. (See BioWorld Today, Nov. 15, 2002.)

Addison is a serial entrepreneur in the software space. Cloud, which changed its name last month from Teradiscoveries, focuses on in silico design of new drugs based on structure-based target information.

"We're not limited to kinases," Addison told BioWorld Today. "We can deal with allosteric proteins and flexible proteins."

Both companies are relative newcomers to drug development. Myos, which previously operated as Atlas Therapeutics Corp., was founded to explore the mechanics of the muscle system, which controls vast processes in the human body yet "no major pharmaceutical company had ever created a focused discovery or development effort to identify ways to maximize the performance of the organ and control some of the processes which degrade the system, either as a result of aging or disease," Hariri said.

The company has a commercial product based on the over-the-counter (OTC) supplement fortetropin (MYO-T12), a natural myostatin inhibitor, that is marketed as Myo-X. One of the company's goals is to capitalize on data generated from development of its nutritional supplement – also designed to increase lean muscle mass and stimulate muscle healing – to move into drug design.

Last month, Myos also inked a three-year master service agreement with researchers from Rutgers University to expand the company's basic science platform for further development of fortetropin and other product formulations.

Ultimately, Myos is banking on both arms of the company to yield blockbuster products, with its OTC dietary nutraceuticals helping to support the longer-term drug development operation.

"The two actually buttress and enable one another" for "a very powerful revenue-generating engine," Hariri explained.

Partner Cloud's inverse design platform, developed by Chief Scientific Officer Shahar Keinan while at Duke University, is used to discover novel compounds by exploring "virtual" chemical space. "Our experiments have shown that over 80 percent of the molecules we find are not in the patent literature and have never been synthesized but are very, very good," Addison said.

A strategic relationship with Microsoft Corp. in 2012 to extend the reach of its drug discovery process helped to accelerate its platform and design six drugs in silico.

The company has come on strong in the last six months, Addison said, attracting two biotech and two pharmas to partner with pipeline candidates and garnering several government grants. Cloud is in the process of raising a series B round, expected to close in the third quarter, with a goal of raising $20 million. From there, the company hopes to be self-sustaining by partnering aggressively, with each agreement containing an opportunity to "earn a piece" of the commercialized drugs.

In the deal with Myos, Cloud will design and optimize the therapeutic candidates in muscle inflammation and atrophy, while Myos will conduct much of the ensuing preclinical development for the JAK3 sarcopenia program. The companies will have a similar structure for the furin program.

"With the core competency that we have and with Myos having strength in biochemistry and preclinical development, it seemed to make a good partnership," Addison said.

"We see this as a multiyear, long-term partnership," he added. "It's not going to end just with those two projects."