NeoStem gets data safety monitoring board approval to continue phase 2 cardiac therapy trial

NeoStem (NYSE MKT:NBS) (AMEX:NBS) said Wednesday that its Amorcyte subsidiary received approval last week to continue the PreServe phase 2 clinical trial for its AMR-001 cardiac therapy.

The approval followed the first interim data and safety review by the Data Safety Monitoring Board.

The trial is a phase 2, placebo-controlled study that is expected to include 160 patients at more than 40 clinical sites.

The study will test AMR-001, which is designed to preserve heart function following acute myocardial infarction (AMI), more commonly known as a heart attack.

Patient enrollment for the trial began in January and the company anticipates completing enrollment in 2013, with six months initial data readout near the end of 2013.

"We are pleased that, similar to our Phase 1 trial, the first external review of our Phase 2 trial data confirms that there are no safety signals that would preclude the trial from continuing as planned," said chief medical officer of NeoStem, Andrew L. Pecora, M.D.

"The PreSERVE AMI study to date indicates that multiple National Study sites are capable of acquiring the necessary volume of bone marrow to create the AMR-001 product five to seven days after an AMI in a safe and practical manner, and once created the product can be delivered and administered without a safety signal."

The treament consists of a patient's own bone marrow cells, which are processed to create pharmaceutical-grade cells that are then re-injected through coronary arteries into damaged areas of the heart, 6 to 11 days after a patient experiences a heart attack.

Because the treatment is autologous, meaning cells are taken from the same individual that they're transplanted into, it has no risk of rejection and can provide support for an extended period of time.

Peak annual worldwide sales of AMR-001 for this indication could exceed $1 billion, NeoStem said.

The therapy is protected by two issued and multiple pending U.S. patents, with corresponding patent coverage in selected markets globally.

The Amorcyte AMR-001 product development program also extends to congestive heart failure (CHF), with the company preparing to launch a phase 1 trial for this indication in early 2013.

The worldwide CHF patient population is estimated to be four times larger than that of AMI, NeoStem noted.

NeoStem reported yesterday that revenue from continuing operations rose nearly 55 per cent in the second quarter as the cell therapy company continues to complete the divestiture of its Chinese generic pharmaceutical business.

For the three months that ended June 30, revenues from continuing operations, which consist of the company's cellular therapy business in the United States, were $3.4 million, versus $2.2 million a year earlier.
The growth was driven mainly by clinical service revenues in the company's Progenitor Cell Therapy (PCT) manufacturing subsidiary, reflecting increased penetration into the cell therapy marketplace.
Shares of the company were up 2.8 per cent late morning, at 68.4 cents.