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Novartis' Afinitor Study Met Key Goal Of Tumor-size Reduction In TSC Patients

Swiss healthcare firm Novartis AG (NVS) Friday said a late stage study of its cancer drug Afinitor met its primary endpoint of reducing the size of subependymal giant cell astrocytoma, a non-cancerous brain tumor, in patients with tuberous sclerosis, a rare genetic disorder.

Tuberous sclerosis complex or TSC affects about one to two million people worldwide and is associated with a variety of resulting disorders including seizures, swelling in the brain, developmental delays and skin lesions.

TSC may cause non-cancerous brain tumors known as subependymal giant cell astrocytomas or SEGAs to form in vital organs and can affect many different parts of the body, most commonly the brain and kidney. SEGAs occur in up to 20 percent of patients with TSC.

Afinitor (everolimus) targets mTOR, a protein that acts as an important regulator of tumor cell division, blood vessel growth and cell metabolism. By inhibiting the mTOR activity, everolimus may reduce cell proliferation, blood vessel growth and glucose uptake related to SEGA associated with TSC.

Everolimus is approved by the regulatory authorities of U.S, Canada, Brazil, Guatemala, the Philippines, Columbia and Korea for SEGAs in patients with TSC under the name afinitor. It is known by the name Votubia in Switzerland.

Last month, the Committee for Medicinal Products for Human Use adopted a positive opinion for the approval of everolimus in the European Union.

In countries where everolimus is not approved, brain surgery is the only treatment option for patients with growing SEGAs.

The phase III study, dubbed EXIST-1, evaluated the efficacy and safety of everolimus versus placebo for the treatment of patients with SEGA associated with TSC. In the trial, the patients were randomized to receive either everolimus or placebo at a daily starting dose of 4.5 mg/m². The study met the primary endpoint of SEGA response rate, with 35 percent of the patients receiving everolimus experiencing a 50 percent or greater reduction in SEGA volume.

The study also assessed three key secondary endpoints such as change in seizure frequency, time to SEGA progression and skin lesion response rate. The most clinically notable adverse events were infections and infestations, observed in both patients on everolimus and patients on placebo.

Commenting on the trial results, Dr. Sergiusz Jozwiak, a lead EXIST-1 investigator and Professor, Department of Child Neurology, The Children's Memorial Health Institute, Warsaw, Poland, stated, "This study, which included SEGA patients from infancy to adulthood, provides compelling evidence of the impact of everolimus in reducing SEGA size with a tolerability profile consistent with the previous everolimus trial in this treatment setting."

The phase III study also supports the findings of a phase II study used for the registration of Everolimus in several countries, the company noted.

Novartis will present the phase III study results of everolimus at the International TSC Research Conference in Washington, D.C. on July 9.

NVS closed Thursday's trading at $61.80.

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