FDA Grants Orphan Drug Designation to IMGN901, Potential Treatment for Merkel Cell Carcinoma

"IMGN901 has shown promising activity among the small number of MCC patients treated to date, suggesting this is a potential registration path for the compound," commented Daniel Junius, President and CEO. "Metastatic MCC is a rare and highly aggressive cancer with no approved treatments today. We are preparing to be ready to initiate pivotal testing of IMGN901 in MCC in 2011 and will make the final decision based on expanded clinical experience and regulatory input. Gaining orphan drug designation for IMGN901 in the US and Europe is an important step in this process."

Six patients with metastatic MCC had received IMGN901 at the time of the AACR-NCI-EORTC meeting in November 2009.1 Among these, two patients had an objective response - including a patient who has had a sustained complete remission - and a third patient had clinically meaningful stable disease.

In the US, the Orphan Drug Act is intended to encourage companies to develop therapies for the treatment of diseases that affect fewer than 200,000 people in this country. Orphan drug designation provides ImmunoGen with seven years of market exclusivity that begins once IMGN901 receives FDA marketing approval for the treatment of MCC. It also provides certain financial incentives that can help support the development of IMGN901 for MCC.

Similarly, in the EU, orphan medicinal product designation is intended to encourage companies to develop therapies for life-threatening or chronically debilitating conditions that affect no more than five in 10,000 people in the EU. Orphan medicinal product designation provides ImmunoGen with ten years of market exclusivity that begins once IMGN901 receives European approval for MCC. It also enables access to certain financial incentives as well as to protocol assistance.

About IMGN901

IMGN901 is an investigational agent designed to kill cancer cells that express CD56, a protein. This TAP compound consists of a CD56-binding antibody with a potent cancer-cell killing agent, DM1, attached to it using an engineered linker. IMGN901 is wholly owned by ImmunoGen.

CD56 is expressed on MCC, small-cell lung cancer (SCLC), ovarian cancer, carcinoid and other solid tumors of neuroendocrine origin, and IMGN901 is in early clinical testing for the treatment of MCC, SCLC and ovarian cancer. CD56 also is expressed on multiple myeloma and certain other hematological malignancies. IMGN901 is in early clinical testing for the treatment of CD56+ multiple myeloma, both when used as a single agent and as part of a combination regimen.

About Merkel Cell Carcinoma (MCC)

MCC is an aggressive neuroendocrine cancer of the skin that typically occurs on the head/neck, most often in individuals of European ancestry. There are approximately 2000 new cases of MCC diagnosed in the US each year.2 The incidence is considered to be increasing. Medicinal therapy is generally used with patients whose cancer has recurred following surgery and with patients who have metastases at the time of diagnosis. Metastatic disease is associated with a poor outcome, with reported median survival timeframes of 5 to 9 months.

Source: ImmunoGen, Inc.