National Editor

Privately held GTx Inc. raised $20 million from current shareholders to push its products aimed at men's health - enough cash to "get us to a point where we have data in both our clinical programs," and can decide what to do next, said Mitchell Steiner, GTx's CEO.

Founded in 1997, GTx has Acapodene (toremifine citrate), a selective estrogen receptor modulator, in Phase IIb/III trials to prevent prostate cancer and starting Phase III in October to treat the complications of androgen-deprivation products, such as Lupron and Zoladex, for prostate cancer.

Specifically, Acapodene is intended to treat the premalignant lesion of the prostate known as high-grade prostatic intraepithelial neoplasia, with the goal of heading off invasive prostate cancer. The Phase IIb/III trial is expected to finish in May 2004, with data expected by the third quarter of that year.

In the adjuvant cancer setting, Acapodene may lessen the problems associated with Lupron (leuprolide) and Zoladex (goserelin), both leutinizing hormone-releasing hormone agonists, such as osteoporosis, breast enlargement and hot flashes.

"The biggest complication is osteoporosis and bone fractures," Steiner said. "Their survival is impacted, and they're more likely to get a bone fracture induced by androgen deprivation or the disease itself."

Hot flashes caused by the drugs are "not like the hot flashes in post-menopausal women," he added, but are "pretty severe and it doesn't get better with time. We have one drug to handle three side effects at once."

The Memphis, Tenn.-based company's second clinical program is to develop andarine, a first-in-class selective androgen receptor modulator (SARM) entering Phase II in January for cancer-related cachexia, or muscle wasting. More than one-third of malignancy patients lose upward of 5 percent of their body weight as a result of the disease, and about 20 percent of cancer deaths are caused by the condition.

"SARMs made their debut about 15 or 16 years ago, when AstraZeneca put out tamoxifen, which revolutionized the way we think about the estrogen receptor," Steiner said. London-based AstraZeneca plc sells tamoxifen as Nolvadex.

Andarine, an oral, nonsteroidal drug, builds muscle and bone but is believed to have few of the drawbacks of testosterone therapy, such as stimulating the prostate.

"The old oral agents were all steroids, which means they were giving a lot of liver trouble," Steiner said. What's more, "the big scare that patients have about [testosterone therapy] stimulating subclinical prostate cancer will be addressed" by andarine, he said.

"The beauty of SARMs is that you can design these molecules to do different things," Steiner said. Others in development for different indications include prostarine for benign prostatic hyperplasia and ostarine for male osteoporosis and andropause.

In preclinical stages is another agent, andromustine, for hormone-refractory prostate cancer.

At the start of the year, GTx entered a deal with Madison, N.J.-based Wyeth to further develop the former's vesicular stomatitis virus-based technologies, which GTx has been exploring for use as targeted viral cytolytic agents against HIV-infected cells and cancers. (See BioWorld Today, Jan. 29, 2003.)

Given the latest financing, "assuming we decide we want to do a partnership with the andarine/SARM program, we'll be fine," Steiner told BioWorld Today. What about a partner for Acapodene?

"The door is open, but we're now at a point where we are two years away from having some revenue from this and launching a product," Steiner said. "Our goal today is to see if we can take this one ourselves."

Participants in the financing include J.R. Hyde III, GTx's chairman; Oracle Partners LP, of New York; and Memphis Biomed Ventures I LP, of Memphis.