GTx reacquires full rights to Ostarine

GTx, Inc. (Nasdaq: GTXI) today provided a company update and reported financial results for the fourth quarter and full year 2009.

“Following the Complete Response Letter last fall, GTx and our European partner, Ipsen, met with the FDA in December. We believe that there is a path forward for the approval of toremifene 80 mg”

“Following the Complete Response Letter last fall, GTx and our European partner, Ipsen, met with the FDA in December. We believe that there is a path forward for the approval of toremifene 80 mg,” said Mitchell S. Steiner, MD, CEO of GTx.

As for the reacquisition of Ostarine™ and the GTx selective androgen receptor modulator (SARM) program, Dr. Steiner continued: “It was a difficult decision to dissolve our SARM collaboration with Merck. GTx’s near term objective is to generate revenue so that we can transition to a self-sustaining company. Reacquiring Ostarine moves us toward this objective by allowing us to advance our lead SARM into Phase III clinical studies in cancer cachexia which is a large commercial opportunity for our company and a critical unmet medical need for cancer patients.”

• GTx reacquires full rights to Ostarine and rest of SARM program and plans to advance Ostarine into Phase III clinical trials

GTx has reacquired full rights to Ostarine and its entire SARM program following the mutual agreement by GTx and Merck to dissolve their SARM collaboration. GTx is planning to pursue Phase III clinical development of Ostarine for the treatment of cancer cachexia. GTx completed a successful Phase IIb Ostarine clinical trial for cancer cachexia in October 2008 and now plans to have an End of Phase II meeting with the United States Food and Drug Administration (FDA) to discuss the Ostarine Phase III clinical development program.

Ostarine is an oral agent that has demonstrated the ability to increase lean body mass and improve muscle strength and performance in postmenopausal women, elderly men, and men and women with cancer cachexia. Ostarine is GTx’s lead SARM which has been studied in seven Phase I, Phase II, and Phase IIb clinical trials in 582 subjects.

• GTx provides update on End of Review meeting with FDA

The company announced in November 2009 the receipt of a Complete Response Letter from the FDA for the toremifene 80 mg New Drug Application. In December, GTx and its European Partner, Ipsen Biopharm Limited, met with FDA to better understand the two issues cited in the Complete Response Letter. Based on this End of Review FDA meeting, GTx and Ipsen have concluded that there is a path forward to obtain approval for toremifene 80 mg to reduce fractures in men with prostate cancer on ADT. GTx will provide additional details following further discussions with the FDA.

• Toremifene 20 mg Phase III high grade PIN clinical trial results expected this summer

In late February, the last patient completed the Phase III clinical trial evaluating toremifene 20 mg for the prevention of prostate cancer in high risk men with high grade prostatic intraepithelial neoplasia (PIN). GTx has begun the operational steps to conclude the study and expects to announce results this summer. If successful, GTx would move forward with plans to submit a New Drug Application.

• Initiation of the Phase II clinical trial evaluating GTx-758

In late February, GTx initiated a Phase II clinical trial evaluating GTx-758, an oral luteinizing hormone (LH) inhibitor, for the first line treatment of advanced prostate cancer. The GTx-758 Phase II clinical trial is evaluating multiple doses of GTx-758 in 70 males. GTx expects to obtain the results from the clinical trial in the second half of 2010.

Financial highlights for the quarter and year ended December 31, 2009

The net loss for the quarter and year ended December 31, 2009 was $10.9 million and $46.3 million, respectively, compared to $13.9 million and $51.8 million for the same periods in the prior year. Revenue for the quarter and year ended December 31, 2009 was $3.7 million and $14.7 million, respectively, compared to $3.0 million and $13.5 million for the same periods in 2008.

Revenue for the fourth quarter of 2009 included collaboration income of $2.8 million related to our collaborations with Merck and Ipsen, and $862,000 of net sales of FARESTON^® (toremifene citrate) 60 mg, marketed for the treatment of metastatic breast cancer in postmenopausal women. Revenue for the year ended December 31, 2009 included collaboration income of $11.4 million from Merck and Ipsen and $3.3 million of net sales of FARESTON^®.

Research and development expenses for the quarter and year ended December 31, 2009 were $8.2 million and $32.3 million, respectively, compared to $10.6 million and $44.3 million for the same periods in 2008. General and administrative expenses for the quarter and year ended December 31, 2009 were $6.3 million and $27.7 million, respectively, compared to $6.3 million and $23.1 million for the same periods in 2008.

At December 31, 2009, GTx had cash, cash equivalents and short-term investments of $49.0 million.