Big Hopes, Small Pharma: UB Spin-Off Wins FDA Orphan Designation for Drug made from Tarantula Venom

BUFFALO, N.Y. -- As Rose Pharmaceuticals marks its first anniversary this month, the stockbroker and University at Buffalo researchers who founded the company are celebrating a year of accomplishments.

The start-up's biophysicists have secured state and foundation funding to advance their product -- a peptide found in tarantula venom -- toward clinical trials as a therapy for muscular dystrophy.

The U.S. Food and Drug Administration has designated the firm's peptide, called GsMTx4, as an orphan drug for Duchenne muscular dystrophy. The status qualifies Rose for tax credits and other incentives that could help the firm move the product to the market more quickly.

Click here to see a video interview with UB researcher Frederick Sachs, who is developing the new drug.

Orphan drugs are those that treat rare diseases, and for Rose Pharmaceuticals CFO Jeff Harvey, success can't come quickly enough. Harvey, a stockbroker from Clarence, learned 1½ years ago that his grandson -- now 3 years old, had Duchenne muscular dystrophy -- a fatal affliction that causes patients' muscles to waste away.

The disease affects about one in 3,500 boys. Symptoms including frequent falling typically appear before age 6. Patients often need a wheelchair by age 12. Severe heart and respiratory problems follow. The average life expectancy ranges from the late teens to the early 20s.

Upon learning of his grandson's diagnosis, Harvey took action. He began reading about Duchenne muscular dystrophy and the limited treatments available for its victims. Then, one day last summer, he consulted Google to see if any scientists at UB were studying the disease. His search yielded a promising result: Sachs, a SUNY Distinguished Professor in physiology and biophysics.

Years before, Sachs and Thomas Suchyna, an assistant professor of physiology and biophysics, had discovered that GsMTx4 showed promise as a therapy for Duchenne muscular dystrophy. But Sachs had given up on trying to commercialize the peptide: Several large pharmaceutical companies had expressed interest in his work, inviting him to present his findings, but none had offered to adopt the drug.

The call from Harvey came as a surprise for Sachs.

"I didn't know what to make of it at first," Sachs remembers. "We had lunch together, and I explained the history of my attempts to get big pharma interested. In my discussions with Jeff, it seemed like maybe what we should do is make a biotech company and then try to raise funds to take care of what big pharma wouldn't do."

Within months, the two men launched Rose Pharmaceuticals, which they named after Rosie, Sachs' pet tarantula, and Rose, Sachs' grandmother. Sachs became the new firm's president. Suchyna and Philip Gottlieb, a research associate professor who has worked with Sachs for many years, signed on as vice presidents.

Harvey, the CFO, enrolled in UB's High-Tech Center for Entrepreneurial Leadership, which gives leaders of early-stage life sciences companies the opportunity to network with guest speakers and learn about topics including entrepreneurship, intellectual property, sales, marketing and financing.

Harvey and Sachs poured personal savings into the company, and, so far, the investment looks promising. This summer, the Children's Guild Foundation awarded Sachs and his team $125,000 to improve the therapy. The UB Center for Advanced Biomedical and Bioengineering Technology (UB CAT), funded by the New York State Foundation for Science, Technology and Innovation, has awarded the firm another $80,000. The FDA's orphan drug designation came in September.

Now, the company is seeking financing for clinical trials of the therapy, which improved the motor ability of dystrophic mice and showed no toxicity in the animal tests.

"We want to beat the disease," Harvey says. "My hope is that my grandson will one day have access to this treatment."

"The more time we've spent doing biotech, the more I begin to ask myself, 'Why are we doing this?' It's not to make money," Sachs says. "As I get older, I would feel really good to have been able to help out some kids. To have a kid get out of a wheelchair and walk away would be unbelievably satisfying."

Rose Pharmaceuticals has an option to license the rights to the peptide from UB, and the company is working with the UB Office of Science, Technology Transfer and Economic Outreach to finalize a license agreement.

For Sachs, the progress the team has made on the treatment is a validation of his life's work and the value of basic science: When he identified GsMTx4, he wasn't studying muscular dystrophy.

Instead, he was researching how to open and close mechanosensitive ion channels that his research team had discovered in muscle cells. He stumbled on GsMTx4, which shuts the ion channels, in a blind screen of arachnid venoms.

Only later did he and Suchyna make the connection with muscular dystrophy. Duchenne muscular dystrophy is caused by a mutation in the gene that codes for dystrophin, part of the cytoskeleton that helps muscle cells keep their shape. In patients with the disease, Sachs says, even a small amount of stress causes the ion channels to open, prompting an influx of calcium ions that activates intracellular enzymes that digest proteins, destroying muscle tissue.

"No one in their right mind would have given spider spit to a kid with dystrophy, so it's only through the basic science that you end up here," Sachs says. "If we can carry it all the way to the clinic, that would be satisfying. You learn new things through basic research and through serendipity. If you keep your eyes open, you see things you would never have seen otherwise."

The High-Tech Center for Entrepreneurial Leadership, funded in part by the New York State Department of Labor through the Buffalo and Erie County Workforce Investment Board, is a partnership between the UB School of Management's Center for Entrepreneurial Leadership and UB's New York State Center of Excellence in Bioinformatics and Life Sciences.

The University at Buffalo is a premier research-intensive public university, a flagship institution in the State University of New York system and its largest and most comprehensive campus. UB's more than 28,000 students pursue their academic interests through more than 300 undergraduate, graduate and professional degree programs. Founded in 1846, the University at Buffalo is a member of the Association of American Universities.