Tuberculosis Drug Inches Closer to Approval
Monday, October 22, 2007
A small Rockville biotech company, started by a former government scientist 10 years ago to take on the global problem of tuberculosis, plans to announce today a significant step forward for a drug at the center of its business.
The company, Sequella, is getting orphan drug status from U.S. and European regulators for its potential new antibiotic called SQ109.
The orphan designation is aimed at products that treat rare diseases. While TB is rare in the United States -- there were fewer than 14,000 new cases last year -- it is a scourge in poorer parts of the world, and outbreaks are often made worse when patients don't adhere to long, difficult treatment regimens.
Public health experts fear the disease will widen as TB-plagued countries like China and India become world economic centers.
"National borders go out of the picture when you have an airplane full of people flying around the world," said Mary Ann DeGroote, an expert on TB drug development at Colorado State University. "Many people are coming from high-burden countries."
The orphan drug designation gives Sequella, which has just 16 employees, market exclusivity protections and a speedier, less expensive path to approval of its drug, which the company's executives hope will shorten the treatment time and lessen the number of drugs needed for cure.
Currently, TB patients must take four drugs and be treated for six to nine months or risk facing deadly drug-resistant strains. Failure to adhere to the regimen is particularly problematic in poor areas of the world. An antibiotic that can shorten and ease the treatment process is considered the holy grail. Companies such as Johnson & Johnson and Otsuka Pharmaceuticals in Japan are also working on new antibiotics.
Carol Nacy, Sequella's chief executive, said the orphan drug action by the Food and Drug Administration and the European Medicines Agency "is a big deal for this company. It really makes it a nice working relationship with the FDA, and we always like having good working relations with regulatory agencies."
For Nacy, the pursuit of a new drug for TB is in many ways an act of love. She is a microbiologist by training. "I like bugs a lot," she said recently. "I just liked the whole concept of being able to find a disease, pull out a specimen from a human, isolate bacteria and then cure the human. That is just a great cycle for me."
She is a spunky woman. Depending on what the bacteria look like under a microscope, she might use the word cute as a description.
Nacy was a researcher at Walter Reed Army Institute of Research, which has produced a number of notable local biotech entrepreneurs, including MedImmune founder Wayne Hockmeyer, who was Nacy's boss at the institute. Another alum is serial entrepreneur John Holaday, who founded EntreMed in the early 1990s. Nacy joined EntreMed in 1993 as chief scientific officer, her first foray into the business, after, as she put it, "an 18-year career in basic science curing mice of diseases I gave them."
Nacy remembers asking for a three-year leave of absence from the institute, a rarity in those days. She got it. "They never said no to me, ever," she said. "No didn't mean anything other than that I didn't have the right argument yet."
In 1996, the National Institutes of Health asked her to review a grant program for TB.
"A, I thought it was cute they still thought of me as a scientist; and B, I didn't know a lot about TB and I thought we'd cured it, so how long would it take to review this program?" she said. "So I went and had three days of indoctrination on TB. I walked out of there stunned. I had no idea the statistics were that bad."
She resolved to start a company and tackle the disease. Sequella has raised about $33 million since its founding, with the biggest investment, roughly $6.5 million, coming from a biotech hedge fund investor in New York. The SQ109 drug is in the early stages of human testing, and the company is also developing a skin patch to diagnose the disease.
In the process of getting the company off the ground, Nacy has become one of the few female biotech chief executives in the country, a fact that isn't entirely interesting to her.
"I have never defined myself as a woman first for anything," she said. "I was a scientist who had indoor plumbing, as opposed to a female scientist. I get surprised when people mention the fact that I am a woman CEO. Yeah, and?"
She would rather talk about tuberculosis.