Allos announces favorable survival data from FOLOTYN Phase 2b study

Allos Therapeutics, Inc. (NASDAQ:ALTH) today announced the presentation of favorable survival data from the Company's international, randomized Phase 2b investigational study of FOLOTYN^® (pralatrexate injection) relative to erlotinib in patients with Stage IIIB/IV (advanced) non-small cell lung cancer (NSCLC). In this study, patients receiving FOLOTYN had a 16 percent reduction in the risk of death compared to erlotinib in the overall (intent-to-treat) population>

“Advanced non-small cell lung cancer is an aggressive disease. Unfortunately, nearly all patients will progress after their initial treatment”

"Overall survival remains the most clinically relevant endpoint in assessing the benefit of an investigational therapy to treat cancer," said Karen Kelly, M.D., an investigator of the study, The University of Kansas Medical Center. "I believe the survival data from this randomized Phase 2b study are compelling and warrant exploring Phase 3 options in order to fully understand the potential role of FOLOTYN to treat patients with advanced non-small cell lung cancer."

The objective of this randomized, international, multi-center Phase 2b study (PDX-012) was to estimate the efficacy of FOLOTYN relative to that of erlotinib as assessed by OS, the primary endpoint of the trial. All patients had received one or two prior systemic treatments including at least one prior platinum-based regimen. Secondary endpoints included progression-free survival (PFS)>

Analyses were also performed according to the statistical analysis plan to assess the activity of FOLOTYN and erlotinib in predefined patient cohorts. Most notably, patients with non-squamous cell carcinoma>

The most common Grade 3-4 adverse event observed in patients treated with FOLOTYN was mucositis (23 percent). Other Grade 3-4 adverse events occurring in more than five percent (but less than 10 percent) of patients were fatigue (9 percent), dyspnea (6 percent), neutropenia (6 percent), thrombocytopenia (5 percent) and anemia (5 percent) in patients treated with FOLOTYN, and rash (8 percent), dyspnea (8 percent), anemia (8 percent) and fatigue (5 percent) in patients treated with erlotinib.

"Advanced non-small cell lung cancer is an aggressive disease. Unfortunately, nearly all patients will progress after their initial treatment," said Charles Morris, MB ChB, MRCP, chief medical officer at Allos Therapeutics. "This study is providing Allos and the lung cancer community with important data regarding the efficacy and safety profile for FOLOTYN in advanced NSCLC, where there remains a high unmet medical need. We believe these data demonstrate the clinical activity of FOLOTYN in patients with advanced NSCLC, and we are in the process of exploring potential Phase 3 development options for FOLOTYN in this indication."

These data were presented in an oral presentation at the 35^th European Society of Medical Oncology (ESMO) Congress in Milan, Italy. The company had previously announced the topline results from this trial in July 2010.

Study Design

The objective of this randomized, open-label, international, multi-center Phase 2b study was to estimate the efficacy of FOLOTYN relative to that of erlotinib, marketed as TARCEVA^®, as assessed by overall survival. The trial enrolled 201 current or former smokers with Stage IIIB/IV (advanced) NSCLC who had received one or two previous treatments including at least one prior platinum-based chemotherapy regimen. The primary endpoint of the trial was overall survival. Secondary endpoints included progression-free survival and response rate as compared with erlotinib, and the safety and tolerability of FOLOTYN. The study was not designed to show a statistically significant difference between the two treatment arms. No definition of statistical significance was included in the statistical analysis plan.

The trial - which enrolled patients across 43 locations worldwide (15 U.S. sites and 28 ex-U.S. sites) - was initiated by Allos in January 2008 and completed enrollment in July 2009. The first 35 patients enrolled in the study were randomly assigned one-to-one to receive FOLOTYN (intravenous push on days 1 and 15 of a 28-day cycle; initial dose of 230 mg/m2) or erlotinib (oral, 150 mg daily in a 28-day cycle). Following a protocol amendment, 166 patients were randomly assigned one-to-one to receive either FOLOTYN at 190 mg/m2 or erlotinib at 150 mg/day and then further stratified by light versus heavy smokers. All patients received concurrent vitamin therapy of B12 and folic acid. According to the statistical analysis plan, analyses were conducted to assess the activity of FOLOTYN and erlotinib in predefined patient cohorts, which included light smokers>