Between 3 and 5 million people in the U.S. suffer from critical limb ischemia (CLI), characterized by severe obstruction of the arteries by fat deposits. Blood flow is restricted to the extremities including hands, feet and legs. Pain, skin ulcers, and even amputation and death result from his severe peripheral artery disease. CLI costs the U.S. and Europe about $25 billion annually.
Treatment options are limited, resulting in unacceptably high rates of amputation and death. Standard of care today includes a lifetime of medication to treat underlying conditions such as high blood pressure, high cholesterol and diabetes. Endovascular treatments such as inserting catheters and stents are another option. Arterial surgery, which involves removing or bypassing arteries, is done in severe cases. Amputation is the last resort for patients who are not eligible for surgery.
Regenerative medicine offers a new solution that could save limbs and lives. PLX-PAD, a cell therapy from Pluristem Therapeutics (NASDAQ:PSTI), is entering a pivotal Phase III trial in the U.S. and Europe for the treatment of CLI. Treatment would simply include two injections of PLX-PAD cells. No surgery and no ongoing medication are involved.
250 patients will be enrolled in the study, staring in the second half of 2017. The U.S., UK, Germany, and the European Union have cleared the trial to commence. The EU in particular is very supportive of Pluristem’s cell therapy. An $8 million grant has been given to help pay for the trial in Europe. The European Medicines Association (EMA) has also accepted PLX-PAD for the treatment of CLI into its adaptive accelerated approval pathway. This means Pluristem could get conditional approval for commercial sales of PLX-PAD cells based on this one pivotal Phase III study.
A 59% reduction in amputations at 6 months post treatment was evidenced in a prior Phase I trial, which compared PLX-PAD to patients treated with standard of care in published data. Additional supporting data for CLI approval in Europe and a Biologics License Application (BLA) in the U.S. is coming from Pluristem’s Phase II trial in the treatment of Intermittent Claudication (IC), a less severe peripheral artery disease. Top line data from that trial is expected in early 2018.
In addition to Europe and the U.S., Pluristem also has clearance to conduct a single 75 patient study in Japan which may lead to early conditional marketing approval and early reimbursement in the Asian nation.
Governments around the world are looking for more cost efficient and patient friendly ways to treat their again populations. This has resulted in accelerated approval pathways and funding in major world healthcare markets for regenerative medicines. Pluristem’s PLX cells are ideally suited to meet this need.