FDA slaps back small-cap's bid to repurpose 50-year-old chemotherapy

04 Aug 2021
endpts.com
When the German drugmaker Medac handed off a decades-old cancer compound to their US subsidiary in February, it thought it would be smooth sailing to market. After all, the drug had recently been approved in Europe and was available in Canada. On Tuesday, though, Medac and that publicly traded subsidiary, Medexus, announced the FDA had rejected the compound, telling the company they would need to get new data and reanalyze their Phase III trial before marketing it in the US. Over-the-counter shares $MEDXF of the small-cap biotech were down 37%, from $6 to $3.79 on the news. The drug Medexus had been pitching is treosulfan, a chemotherapy that researchers have been studying for its cancer-killing potential since at least 1964 and directly in patients since at least 1968. It never entered widespread use but, after periodic trials over the decades, eventually won approval for ovarian cancer. Around 20 years ago, Medac began developing treosulfan as a conditioning agent — a drug meant to clean out a cancer patient’s stem cells and prepare them for a bone marrow transplant. The company argued that it could prove less toxic to patients than the widely-used conditioning agent busulfan. It was an early, low-tech step in a broader, industry-wide effort to make such transplants safer. Last year, the Lancet published the company’s Phase III data showing that, in an open-label, 436-patient study, treosulfan was no worse than busulfan. The trial even showed a small, albeit not statistically significant benefit for treosulfan: 64% of patients who received Medac’s drug went two years without relapse or death, compared to 54% of patients who received busulfan. Investigators attributed the survival advantage to fewer transplant-related deaths in the treosulfan arm. It’s not clear precisely what the FDA’s objections were; the agency does not release rejection letters, leaving that up to companies, who rarely disclose their full contents. Medexus executives said only that the agency requested more data and analyses and that the move came as a shock. “Given the recent Health Canada approval, European Medicines Agency approval in 2019, as well as supporting data from more than 100 publications, we were all surprised by the FDA’s response,” CEO Ken d’Entremont said in a statement. “That being said, Medexus and Medac look forward to continuing to work with the FDA to address their requests in a timely manner, and we remain optimistic for a future, albeit delayed, approval of treosulfan in the United States.”
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