FDA Approves Novocure’s NovoTTF-100L System in Combination with Chemotherapy for the Treatment of Malignant Pleural Mesothelioma

FDA’s Real-World Evidence Program Framework

NovoCure announced the U.S. FDA has approved the NovoTTF-100L System in combination with pemetrexed plus platinum-based chemotherapy for the first-line treatment of unresectable, locally advanced or metastatic, malignant pleural mesothelioma (MPM). NovoTTF-100L is a non-invasive, antimitotic cancer treatment that delivers Tumor Treating Fields to the region of the tumor. Tumor Treating Fields therapy uses electric fields tuned to specific frequencies to disrupt solid tumor cancer cell division.

The FDA approval of the NovoTTF-100L System was based on the STELLAR trial. The prospective, single-arm trial was designed to study the safety and efficacy of NovoTTF-100L plus chemotherapy first-line in patients with unresectable MPM. The trial included 80 patients with unresectable and previously untreated MPM who were candidates for treatment with pemetrexed and cisplatin or carboplatin. The trial was powered to prospectively determine the overall survival in patients treated with NovoTTF-100L plus chemotherapy. The median overall survival was 18.2 months across all patients treated with NovoTTF-100L plus chemotherapy. The median overall survival was 21.2 months for patients with epithelioid MPM (n=53) and 12.1 months for patients with non-epithelioid MPM (n=21). At one year 62% of patients (n=80) enrolled in the STELLAR trial were still alive. Mild-to-moderate skin irritation was the most common device-related side effect with NovoTTF-100L.

About Malignant pleural mesothelioma

Malignant pleural mesothelioma is a rare, aggressive cancer that develops in the pleura, a thin layer of tissue surrounding the lungs. Inhaling microscopic asbestos fibers is the primary cause of mesothelioma. Once these fibers enter the lungs, they can become lodged in the pleura, accumulating and causing cellular damage that can lead to cancer. Approximately 3,000 people are diagnosed with MPM in the United States annually.

FDA Grants Fast Track Status to Imara Therapeutics’ IMR-687 for Sickle Cell Disease

Imara Therapeutics announced the FDA has granted Fast Track designation to IMR-687, the company’s lead product candidate for the treatment of sickle cell disease. IMR-687 has previously received U.S. Orphan Drug Designation and U.S. Rare Pediatric Designation.

IMR-687 is currently being evaluated in a multi-national Phase 2a clinical trial in adult patients. The randomized, placebo-controlled, multicenter study plans to enroll 54 subjects and is designed to evaluate the safety, pharmacokinetics and preliminary pharmacodynamics of escalating doses of oral IMR-687.

About Sickle Cell Disease

Sickle cell disease is a rare, genetically inherited condition that alters hemoglobin, the protein in red blood cells that transports oxygen throughout the body. The altered hemoglobin distorts red blood cells into a sickle, or crescent, shape. Painful episodes can occur when sickled red blood cells, which are stiff and inflexible, get stuck in small blood vessels. These episodes deprive tissues and organs of oxygen-rich blood and can lead to vaso-occlusive crisis (VOC), acute chest syndrome (ACS), and permanent damage to organs including the liver, spleen, kidney and brain.

About IMR-687

IMR-687 is an orally administered, highly potent and selective phosphodiesterase 9 (PDE9) inhibitor designed to address the underlying pathology of sickle cell disease. IMR-687 has the potential to be a disease-modifying therapeutic for sickle cell disease as well as other hemoglobinopathies. Pre-clinical data demonstrate IMR-687 reduces both the sickling of red blood cells and blood vessel occlusion that cause debilitating pain, organ damage, and early mortality in affected patients.