Frederick Sachs, PhD.

Frederick Sachs, PhD, president of Tonus Therapeutics, holds the source of a promising new drug targeting muscular dystrophy.

UB Spinoff Opens Headquarters in Center of Excellence

Published January 29, 2013

Story based on news release by Charlotte Hsu

Tonus Therapeutics, a UB spinoff firm developing a promising new therapy for muscular dystrophy, has opened its headquarters in UB’s New York State Center of Excellence in Bioinformatics and Life Sciences.

“The whole project on mechanosensitive ion channels and GsMTx4 and its use in treating muscular dystrophy is 100 percent Buffalo. Everything was discovered here.”
Frederick Sachs, PhD
SUNY Distinguished Professor of Physiology and Biophysics

“Being located in a hub of research activity in Buffalo, close to other entrepreneurs and biotech startups, is important to us,” says Jeff Harvey, the firm's co-founder and chief financial officer.

“The Buffalo Niagara Medical Campus provides ready contact with experienced entrepreneurs and potential clinical and commercial partners, including those within the Center of Excellence.”

Promising Drug Derived from Tarantula Venom

Tonus is developing a drug derived from a peptide that UB scientists discovered in the venom of the Chilean rose tarantula.

The U.S. Food and Drug Administration has designated the peptide, now made by chemical synthesis, an “orphan drug” for muscular dystrophy, a designation recognizing promising methods of treating rare diseases.

Peptide May Suppress Muscular Dystrophy’s Symptoms

The Tonus researchers’ findings stem from their long-term studies of venoms targeting mechanosensitive ion channels—tiny conduits that help control the flow of important substances, such as calcium, into cells.

Due to a defective gene, people with muscular dystrophy lack the protein dystrophin, which helps muscles keep their shape. Consequently, their cell membranes stretch more easily, prompting the ion channels to open, letting calcium flood in.

This chain reaction causes the body to start “digesting muscle from the inside out,” says Tonus President Frederick Sachs, PhD, SUNY Distinguished Professor of Physiology and Biophysics.

The peptide that the UB researchers discovered, GsMTx4, keeps the ion channels shut, offering potential for a drug that could suppress the debilitating symptoms of a host of diseases, including muscular dystrophy.

Drug’s Properties May Help Reduce Cost to Patient

Tonus is also exploring the peptide’s effects on sickle cell anemia, xerocytosis and chronic kidney failure.

Researchers have gathered preliminary data showing that GsMTx4 is nontoxic in mice and does not disturb heart function in mice or ferrets or isolated human heart muscle.

The drug is capable of staying in the body for a long time without breaking down, so it could be possible to deliver low doses infrequently, reducing patients’ costs, Sachs notes.

Child’s Illness Spurred Collaboration

Tonus was founded in 2009 when Harvey, a stockbroker whose grandson has a severe form of muscular dystrophy, joined forces with UB researchers.

In addition to Harvey and Sachs, the other co-founders, both in physiology and biophysics, are vice presidents Thomas Suchyna, research assistant professor, and Philip Gottlieb, research associate professor.

“The whole project on mechanosensitive ion channels and GsMTx4 and its use in treating muscular dystrophy is 100 percent Buffalo,” Sachs says.

“Everything was discovered here.”

UB Partnerships Advance New Firm

Tonus has benefited from several UB resources and partnerships, including: