Welcome to your Update from the Clinic for the month of December. Athersys and NeoStem both confirm completion of enrollment for phase 2 studies while bluebird bio announces the first patient transplanted in its phase 1/2 study of HGB-205 in patients with beta-thalassemia and sickle cell anemia. Read on to find out more.
Cleveland-based Athersys (ATHX), which recently landed a spot on Deloitte’s Technology Fast 500 list, announced completion of enrollment for its phase 2 study of MultiStem® for patients with ulcerative colitis. The study is being performed in conjunction with Pfizer (PFE), after the company formed a collaboration agreement with the pharmaceutical giant back in 2009 to push the technology through the clinic. Treatment centres are found throughout the U.S., Canada and Europe.
Like Mesoblast’s (MSB) mesenchymal stem cell-based Prochymal cell therapy product, which it recently acquired from Osiris Therapeutics (OSIR), MultiStem® is an “off-the-shelf” allogeneic product that can be administered to any patient without the need for immune suppression and can be stored for lengthy periods of time in cryopreservation. Athersys recently received an orphan designation for MultiStem® in Europe for the prevention of graft-versus-host disease (GVHD).
NeoStem (NBS) announced completion of enrollment for its PreSERVE phase 2 study in patients with a form of acute myocardial infarction known as ST segment elevation myocardial infarction (STEMI). Patients will be treated with NeoStem’s AMR-001 autologous cell therapy, composed of a patient’s own CD34+/CXCR4+ cell population derived from the bone marrow, and receive a single intra-coronary injection of 10 million cells. The therapy acts to reverse the effects of heart attack through the development of microvasculature, which increases blood flow to damaged tissue. NeoStem’s management believes the therapy will reduce downstream cardiac events post-heart attack, presenting a pharmacoeconomic benefit to the health-care industry at a time when STEMI is on the rise due to the aging “baby boomer” population.
bluebird bio (BLUE), a cell therapy company tackling rare genetic and orphan diseases in the clinic using novel gene therapy platforms, announced the treatment of its first patient with HGB-205 in a phase 1/2 study of the therapy in patients with beta-thalassemia and sickle cell anemia. HBG-205 is branded as LentiGlobin and is an autologous HSC transplant therapy.
That brings an end to clinical programs in 2013. Keep your eye on Neuralstem (CUR) in 2014. The company is expected to release initial top-line data from its phase 2 ALS trial sometime in the first quarter of the year. It is also expected to enroll its first patient in a phase 1 spinal cord injury trial. Another, more significant, catalyst for the company will be the potential for the FDA to award Neuralstem a Breakthrough Therapy designation (BTD) for its NSI-566 neural stem cells sometime in the second half of 2014. NSI-566 is currently being investigated for treating a number of indications, including amyotropic lateral sclerosis (ALS), chronic spinal cord injury, and ischemic stroke. A BTD offers a company fast tracking through the clinic, and intensive regulatory guidance, for products with preclinical data providing evidence of substantial improvement on at least one clinically significant endpoint over available therapies.
Disclaimer: “Update from the Clinic” is a blog post generated by news flow from public regenerative medicine (RM) companies around the globe. As CCRM has public RM companies in its industry consortium, and the number of such companies is relatively limited on a global scale, Mark Curtis will sometimes include CCRM consortium members in his review. This blog post is provided for general information only and nothing contained in the material constitutes a recommendation for the purchase or sale of any security. The author is not a shareholder of any public RM company. To see a list of CCRM’s industry consortium members, please visit http://ccrm.ca/industry-consortium