Allos Therapeutics, Inc announced topline results from the company's randomized phase-2b investigational trial of Folotyn (pralatrexate injection) versus erlotinib in patients with Stage IIIB/IV (advanced) non-small cell lung cancer (NSCLC) who had received one or two prior systemic treatments including at least one prior platinum-based regimen. The objective of the trial was to estimate the efficacy of Folotyn compared to that of erlotinib as assessed by overall survival. The results demonstrated clinical activity of Folotyn in this patient population.

The primary endpoint of the trial was overall survival. Patients receiving Folotyn had a 16 per cent reduction in the risk of death compared to erlotinib in the overall patient population (n=201; hazard ratio (HR)=0.84) and a 13 per cent reduction in the risk of death in the primary efficacy analysis population (n=166; HR=0.87).

Analyses were also performed in predefined patient cohorts, including light vs. heavy smokers, current vs. former smokers, squamous vs. non-squamous histology, and patients who received prior pemetrexed vs. those who had not. The largest reductions in risk of death for Folotyn were observed in patients with non-squamous cell carcinoma (n=107; HR=0.65) and light smokers (n=37; HR=0.63), with the results demonstrating a 35 percent and 37 percent reduction in the risk of death, respectively. Positive trends in overall survival were observed in favour of Folotyn in all other patient cohorts except patients with squamous cell carcinoma and patients who received prior pemetrexed.

The safety profile of Folotyn was consistent with that observed and reported in previous Folotyn solid tumour studies. The most common Grade 3-4 adverse event observed in patients treated with Folotyn was mucositis (23 per cent). Other Grade 3-4 adverse events occurring in more than 5 per cent (but less than 10 per cent) of patients were fatigue, dyspnea, neutropenia, thrombocytopenia and anaemia in patients treated with Folotyn and rash, dyspnea, anemia and fatigue in patients treated with erlotinib.

"We are pleased that the results of this trial demonstrated clinical activity of Folotyn in a randomized study compared to erlotinib, an approved active agent in non-small cell lung cancer," said Charles Morris, MRCP, chief medical officer at Allos Therapeutics. "We will continue to review these important data with our expert advisors to further evaluate the clinical potential of Folotyn in this patient population. We also plan to submit the complete study results for presentation at an upcoming medical meeting."

"This study generated important data regarding the safety and efficacy profile for Folotyn in advanced non-small cell lung cancer, an area of high unmet medical need," said Paul L Berns, chief executive officer at Allos Therapeutics. "We believe these data warrant further analysis to determine the future development strategy based on our assessment of the potential clinical, regulatory and commercial opportunities for Folotyn in this indication."

Allos Therapeutics is a biopharmaceutical company committed to the development and commercialization of innovative anti-cancer therapeutics.