When there was just silence on October 30, the last day that the FDA was scheduled to decide on approval of GTx’s osteoporosis drug, acapodene, rather than it being a case of 'no news is good news' the omens did not look good. Today, the FDA confirmed that there would be no celebrations at the Memphis-based company by issuing it with a complete response letter.
Despite the fact that GTx shares fell by 28% in early morning trading to a historic low, as previously pointed out by EP Vantage the chances for approval had been looking very slim given the drug’s poor efficacy record in treating bone loss in men undergoing prostate cancer treatment, and safety concerns (Event - GTx could be crushed by osteoporosis drug, October 20, 2009).
True to prediction, it was indeed efficacy and safety concerns that caused the regulator to rain on GTx’s parade. The agency has asked the group to conduct a second phase III trial demonstrating the efficacy and safety of drug in reducing fractures in men with prostate cancer receiving androgen deprivation therapies (ADT), it has also demanded a trial showing that acapodene does not have a negative impact on either disease progression or overall survival.
One of the main concerns facing acapodene is the risk of venous thromboembolic events, especially increased clotting. As such what GTx will need to prove is that even if there is an increased risk of this happening it will be outweighed by the prevention of vertebral fractures.
But it will be a tough task, not only because cancer patients already face an increased risk of clotting, but also because as seen with both acapodene and also Amgen’s Prolia, the FDA is setting a higher bar for drugs that play a supportive role in oncology (FDA delays Amgen’s Prolia but launch is on the horizon, October 19, 2009).
With only $69m of cash on the balance sheet as of June 30, what GTx might also find tough is finding the funds to meet the FDA’s demands.
Today’s share price fall has also made raising money by issuing new shares seriously unattractive, but given its limited options that is what the group may have to do. If GTx does go down this road it will be a move seriously unpopular with shareholders who have not chosen to exit the stock at this point.
The group does, however, have one more chance of turning things round for acapodene. The drug is being studied in prevention of prostate cancer for men with prostatic intraepithelial neoplasia, results are due in the first half of next year and if they are positive, GTx might want to consider finding a partner for the drug in this indication, something that would add much needed cash.
But in the meantime, like many companies disappointed and bruised by the regulatory process, GTx is planning a meeting with the FDA to discuss a way forward, but unless the group can find some way of funding the new trials it might be a very short conversation.