- Cancer therapy developer CytRx Corp. claims it has ironed out an approval pathway with the Food and Drug Administration for its lead candidate.
- The clinical-stage company said Wednesday the FDA has agreed to accept a rolling submission of the New Drug Application (NDA) for aldoxorubicin, a soft tissue sarcoma (STS) treatment. CytRx is pursuing a fast track designation for the filing, and plans to turn in the first pieces of the application in the fourth quarter.
- The American Cancer Society estimates roughly 12,400 cases of STS will be diagnosed and nearly 5,000 people will die from the disease in 2017.
Correction: In a previous version of this article, we incorrectly stated aldoxorubicin was compared to placebo in the Phase 3 trial. The drug was compared to an investigators' choice of treatment. BioPharma Dive regrets the error.
For drugmakers, and particularly tiny biotechs without any marketed products, finding a route that gets their applications in front of FDA reviewers is like seeing the light at the end of the tunnel. CytRx, however, may have longer to go.
The data that aldoxorubicin — and CytRx, to an extent — rests on is shaky at best. Results of a randomized, Phase 3 study released last June showed the drug failed to significantly outperform investigator's choice of treatment, with relapsed and refractory STS patients in the treatment arm demonstrating median progression free survival of 4.17 months versus 4.04 months for the other drugs.
CytRx blamed an earlier clinical hold that led to insufficient follow-up analysis for two-thirds of the 433 patients enrolled in the trial, and six months later touted updated results that found a subgroup of 246 patients did in fact show statically significant responses when taking the company's drug as opposed to chemotherapy.
Investors, however, have remained skeptical. The companies shares barely budged from around 39 cents apiece following the positive subgroup analysis announced Dec. 29. They have remained under 50 cents per share since then, though stock did rise more than 9% to 48 cents per share in Wednesday morning trading.
Adding to the skepticism were recent charges filed by the Securities and Exchange Commission against 27 individuals and entities that engaged in stock promotion schemes, namely through paying writers to publish bullish articles on financial news websites.
The commission called out a situation involving CytRx in an April 10 cease and desist order, charging that in December 2013, a business CytRx commissioned for social media and marketing services paid a writer $300 for a Seeking Alpha story on the drugmaker's Phase 2 results for aldoxorubicin. The writer never disclosed this payment, however, according to the commission.
CytRx anticipates U.S. commercial launch for its product in 2018 should the drug secure approval. It is also looking at foreign markets, and intends to discuss with the European Medicines Agency filing an application for European approval.
"We see the biggest market as being in the U.S. and Canada," CytRx CEO Steven Kriegsman said during an April 19 investor call. "There could be other significant markets. We haven't touched on Asia yet; that would be a separate filing. But that would also constitute orphan drug approval. So, the market potential obviously with the broad indications in the U.S. is quite substantial."
Kriegsman added his company has enough capital to push aldoxorubicin to market. The company reported $57 million in cash and cash equivalents as of Dec. 31. If that's not enough, however, CytRx is in confidential talks with "a number" of potential strategic partners.
Aldoxorubicin's filing falls under section 505(b)(2) of the Food, Drug and Cosmetics Act, meaning the agency can make its approval decision based on data not generated by a medication's primary developer. CytRx pointed to Celgene's Abraxane (paclitaxel), Johnson & Johnson's Doxil (doxorubicin liposomal) and Ipsen's Onivyde (irinotecan liposome injection) as examples of other anti-cancer therapies that have successfully made it to market via that approval route.