Infinity announces preliminary results of IPI-926 Phase 1 study in patients with solid tumors

Infinity Pharmaceuticals, Inc. (Nasdaq:INFI) today announced promising preliminary results from a Phase 1 study of IPI-926, its novel, oral small molecule that targets the Hedgehog pathway. In the study, IPI-926 was well tolerated and resulted in clinical activity in patients with basal cell carcinoma (BCC). These data demonstrate the ability of IPI-926 to inhibit the Hedgehog pathway, further supporting the Phase 1b/2 study of IPI-926 in combination with Gemzar® (gemcitabine) in patients with previously untreated, metastatic pancreatic cancer, which is enrolling patients. These data were described in a poster presentation at the European Society for Medical Oncology (ESMO) Congress in Milan, Italy.

"This study provides important information about the tolerability and anti-tumor activity of IPI-926 in patients with solid tumors and confirms our hypothesis that inhibition of the Hedgehog pathway may be an important new approach to treating the broad range of cancers in which this pathway is implicated," said Antonio Jimeno, M.D., Ph.D., Associate Professor, University of Colorado School of Medicine, and a lead investigator in this study. "I look forward to further exploration of the clinical potential of IPI-926 in multiple indications."

"We are encouraged by the clinical data of IPI-926 showing a pharmacokinetic profile that supports once daily dosing as well as activity in patients with basal cell carcinoma. We look forward to following the numerous patients who remain on study and reporting the full set of data in the future," stated Julian Adams, Ph.D., president of research and development at Infinity. "Having demonstrated that IPI-926 has on-target activity against the Hedgehog pathway, these results further support our Phase 1b/2 trial in pancreatic cancer. We are currently evaluating additional indications in which to advance IPI-926."

Trial Design and Results

The Phase 1 study of IPI-926 was designed as an open-label, dose-escalation study in patients with advanced and/or metastatic solid tumor malignancies. Patients received IPI-926 administered orally once-daily on 28 day cycles at doses ranging from 20 mg to 200 mg. In addition to the initial dose escalation phase, several expansion cohorts were enrolled at the 130 mg dose level, including a cohort of patients with locally advanced or metastatic BCC. Trial endpoints include safety and tolerability, pharmacokinetics, pharmacodynamics and anti-tumor activity. Further dose escalation in this study is ongoing to determine the maximum tolerated dose of IPI-926.

At the time of the data presentation, 60 patients have been enrolled, including 24 patients with BCC. IPI-926 has been well tolerated. The most common adverse events observed were Grade 1 and 2 fatigue and nausea. Grade 3 transaminitis was observed in four patients; however, all events of transaminitis were asymptomatic and reversible. No Grade 4 or 5 related AEs were observed. Steady state exposure to IPI-926 was achieved after three weeks on study, confirming the potential for once daily dosing.

In the BCC cohort, 17 patients were enrolled who were naïve to treatment with a Hedgehog pathway inhibitor. To date, four clinical partial responses have been observed in this group of patients. As the majority of patients with BCC have undergone treatment for less than 24 weeks, more time on study will be required to fully assess the clinical activity of IPI-926 in patients with BCC. Only one patient with BCC naïve to treatment with a Hedgehog pathway inhibitor has discontinued from the study due to progression of disease, and this patient was on trial for more than 18 months. Among patients with non-BCC solid tumors enrolled in the study, three patients have shown stable disease for at least six months.

Information regarding clinical trials for IPI-926, including participating clinical trial sites, is available at www.clinicaltrials.gov.