Pharma Strategy Blog

Hedgehog pathway activity has been demonstrated in malignant glioma. However, its role in tumor growth has not been determined. Here we demonstrate that pharmacological inhibition of the Hedgehog pathway in established orthotopic malignant glioma xenografts confers a survival advantage. Pathway inhibition is measured in transplanted human tumor cells and not in host mouse brain. Correspondingly, survival benefit is observed only in tumors with an operational Hedgehog pathway. These data indicate that Hedgehog signaling regulates the growth of select malignant gliomas. We also demonstrate that Hedgehog pathway component and gene target expression segregate to CD133+ tumor initiating cells. Treated mice eventually succumb to disease, thus, targeting the Hedgehog pathway in CD133+ cells produces significant, but incomplete tumor regression. Therefore, our studies suggest that more complete tumor regression may require the inclusion of other therapeutic targets, including CD133- cells.

Nycomed has submitted a New Drug Application with the US Food and Drug Administration for Daxas (roflumilast) as a once-daily oral treatment for patients with symptomatic chronic obstructive pulmonary disease. The filing is based on results from four Phase III trials, two of which showed positive effects on exacerbation rates and pulmonary function. It is also looking to purchase Solvay.

Benlysta (belimumab) formerly known as LymphoStat-B, met the primary endpoint in BLISS-52, the first of two Phase III trials in patients with systemic lupus erythematosus (SLE). The 865-patient year-long study showed that “a clinically and statistically significant improvement” was shown in patient response rate for belimumab plus standard of care, versus placebo plus standard of care (57.6% for 10 mg/kg Benlysta, 51.7% for 1 mg/kg Benlysta) and 43.6% for placebo).