Merkel cell carcinoma drug IMG901 gets special status in US, EU

IMGN901, an investigational drug to treat Merkel cell carcinoma (MCC) developed by ImmunoGen, Inc., has been granted Orphan Drug designation in US.

IMGN901 is 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.

CD56 is expressed on Merkel cell carcinoma, small-cell lung cancer (SCLC), ovarian cancer, carcinoid and other solid tumors of neuroendocrine origin.

IMGN901 is in early clinical testing for the treatment of Merkel cell carcinoma, small-cell lung cancer and ovarian cancer.

CD56 also is expressed on multiple myeloma and certain other hematological malignancies.

“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, in a news release.

ImmunoGen is 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, he added.

Six patients with metastatic MCC had received IMGN901 in November 2009. 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.

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.

Merkel cell carcinoma 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 Merkel cell carcinoma diagnosed in the US each year.

The incidence of Merkel cell carcinoma 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.

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.

The European Union (EU) Committee for Orphan Medicinal Products (COMP) concurrently also granted IMGN901 orphan medicinal product designation for the treatment of Merkel cell carcinoma.

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.

ImmunoGen, Inc. develops targeted anticancer therapeutics with expertise in cancer biology, monoclonal antibodies and the creation and attachment of potent cell-killing agents.

The company’s TAP technology uses antibodies to deliver one of ImmunoGen’s cancer-cell killing agents specifically to tumor targets.

ImmunoGen’s product pipeline, compounds utilizing the TAP technology are in clinical testing through its collaborations with Genentech (a wholly owned member of the Roche Group), sanofi-aventis, Biogen Idec and Biotest.

The most advanced compound, trastuzumab-DM1 (T-DM1), is in Phase III testing being conducted by Genentech and Roche. Other ImmunoGen collaborative partners include Bayer HealthCare and Amgen.