AstraZeneca reports positive study data for AZD9291 in lung cancer; selumetinib gains FDA orphan drug status

AstraZeneca announced Friday that updated study results show that the experimental drug AZD9291 demonstrated median progression-free survival (PFS) of 13.5 months in patients with advanced EGFR mutation positive non-small-cell lung cancer (NSCLC), who also have the T790M resistance mutation. The results from the AURA study, which build on those released last year, were detailed at the European Lung Cancer Congress.

The ongoing Phase I/II trial is investigating AZD9291 in patients with advanced NSCLC and disease progression following treatment with an EGFR tyrosine kinase inhibitor (TKI). AstraZeneca noted that as of December 2, 2014, 283 patients with EGFR mutation positive advanced NSCLC and acquired resistance to EGFR TKIs were enrolled, including 163 subjects who had T790M tumours confirmed by central testing.

According to the drugmaker, the PFS findings relate to independently reviewed data from 63 patients with T790M tumours treated with AZD9291 at a dose of 80 milligrams per day, and are based on only 38 percent of patients having tumour progression. AstraZeneca added that the updated results also show an overall response rate with AZD9291 of 54 percent and a median duration of response of 12.4 months.

AstraZeneca noted that AZD9291 is a once-daily, selective, irreversible EGFR TKI designed to target both the activating sensitising mutation, EGFRm, and T790M. Antoine Yver, head of oncology, global medicines development at the company, said "we are on track for a regulatory submission of AZD9291 in the US in the second quarter of this year." He added "our...clinical research programme is also investigating the potential of AZD9291 in earlier disease and in combination with other pipeline assets including immuno-oncology molecules." Ongoing studies in lung cancer include AZD9291 in combination with the anti-PDL1 immunotherapy MEDI4736, the MEK inhibitor selumetinib and the MET inhibitor AZD6094.

Separately, the company announced Friday that the FDA granted orphan drug designation to selumetinib for the treatment of uveal melanoma. Yver remarked that "selumetinib could potentially become the first effective treatment for these patients."

AstraZeneca noted that data from a Phase III study evaluating selumetinib in combination with chemotherapy in patients with first-line metastatic uveal melanoma is expected to be available later this year. Meanwhile, the drug, which was originally licensed from Array BioPharma, is also being investigated in Phase III studies in KRAS mutation positive lung cancer and thyroid cancer, and in Phase II in children with neurofibromatosis Type 1.

Earlier this week, AstraZeneca disclosed that the FDA granted orphan drug designation to its anti-CTLA-4 monoclonal antibody tremelimumab for the treatment of malignant mesothelioma. For further information on the orphan drug market, see Orphan Drug Market Access: Payer Insights on the Present and Future.

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