In the first part of our feature on regenerative medicine last week we described the current state-of-the-industry and how the sector had emerged into biotech’s mainstream. This feature examines some of the recent clinical milestones that have been achieved by companies in the space and how these events are translating into value creation.

Interest in regenerative medicine companies continues to grow and, although just a few weeks into the New Year, the news flow from these firms continues to keep investors engaged.

Take Athersys Inc., of Cleveland, for example, which is developing and commercializing regenerative medicine therapies. It recently completed enrollment in a Phase II study of Multistem cell therapy, an adult-derived “off-the-shelf” stem cell product, for ulcerative colitis under its collaboration with Pfizer Inc., of New York. The double-blind, randomized, placebo-controlled study will evaluate the safety and efficacy of Multistem in subjects with moderate to severe ulcerative colitis. The primary endpoints of the study include incidence and severity of adverse events over 16 weeks of therapy, change in endoscopic score at eight weeks and changes in the Mayo rectal bleeding subscore at four weeks and eight weeks. Pfizer will carry out the study at sites in the U.S., Canada and Europe. Initial results are expected in the second quarter of this year. (See BioWorld Today, Dec. 20, 2013.)

CLOSELY WATCHED

Investors have certainly been following Athersys closely and are impressed with its performance so far. The company’s share price has increased 292 percent since the beginning of 2013, a return that could push even higher if their trial results are positive.

The company also was able to leverage its share value earlier this month selling 5 million shares of common stock and warrants to purchase up to 1.5 million shares of common stock, in an offering that generated gross proceeds of approximately $20.5 million. The warrants have an exercise price of $4.50 per share of common stock and expire on July 15, 2016. Each share of common stock, along with a warrant to purchase up to 0.30 shares of common stock, was offered for a purchase price of $4.10.

Newark, Calif.-based Stemcells Inc. has just reported that a team at the University of Calgary successfully transplanted its first subject in a Phase I/II clinical trial in chronic spinal cord injury, with its HuCNS-SC human neural stem cells. The ninth subject to enroll in the trial, which was initiated in Switzerland, is the first spinal cord injury patient to have undergone transplantation in North America. This expansion from a single-site, single-country study to a multi-site, multi-country program accelerates the current trial, which should complete enrollment of the remaining three patients this quarter, and pave the way for a controlled Phase II efficacy study that StemCells Inc. plans to initiate mid-year.

The company said it plans to enroll 12 subjects with thoracic neurological injuries at the T2-T11 level, classified as complete or incomplete according to the American Spinal Injury Association Impairment Scale.

To date, nine patients have been enrolled and transplanted with HuCNS-SC cells. Each of the first three subjects suffered a complete injury prior to enrolling in the study. Twelve months after transplantation of the HuCNS-SC cells, data showed multi-segment gains in sensory function in two of the first three subjects, one of which converted from a complete injury classification to an incomplete injury. The third subject in this cohort remained stable, 12 months after transplantation.

Cardio3 Biosciences SA, of Mont-Saint-Guibert, Belgium, said it received FDA authorization for for its investigational new drug application for regenerative medicine product C3BS-CQR-1 (C-Cure) as a treatment targeting heart failure. The company’s second Phase III trial, CHART-2, is designed as a prospective, multicenter, randomized, patient-and evaluator-blinded study comparing treatment with C3BS-CQR-1 to a sham treatment. The trial is aimed to recruit a minimum of 240 patients with chronic advanced symptomatic heart failure. The primary endpoint of the trial is the six-minute walk test post-procedure, a commonly used index of cardiovascular performance. (See BioWorld Today, Jan 10, 2014.)

GENE THERAPY IS HOT

Gene therapy also has been a hot topic amongst investors. One of the beneficiaries of this enthusiasm has been Sangamo Biosciences Inc., of Richmond, Calif., with its shares jumping by 222 percent since the start of 2013.

A month after reporting promising preclinical data from its zinc finger nuclease (ZFN) therapy in beta-thalassemia at the American Society of Hematology (ASH) meeting in New Orleans, the company licensed rights to the technology in a collaboration with Biogen Idec Inc. worth up to about $314 million.

The partnership will focus on developing therapies for sickle cell disease (SCD) and beta-thalassemia.

“Building upon emerging science related to fetal hemoglobin regulation, we intend to develop Sangamo’s novel gene-editing technology to create a single approach that has the potential to functionally cure both sickle cell disease and beta-thalassemia,” said Douglas E. Williams, Ph.D., Biogen Idec’s executive vice president of research and development, in a statement.

Sangamo’s proprietary zinc finger nuclease (ZFN) genome-editing technology enables multiple pathways to treat SCD and beta-thalassemia. The technology can be used to precisely target and knock out key regulators of gene expression, or can be used to precisely insert a new corrective gene to replace the defective copy.

Specifically, the ZFN genome-editing technology aims to knock out BCL11A, a key regulatory of the transcriptional switch from fetal to adult beta-globin expression, in hematopoietic stem cells that are isolated and then returned to the patient. (See BioWorld Today, Jan. 10, 2014.)

Cardium Therapeutics Inc. reported data from the first stage of its Phase III trial with its gene therapy Generx (alferminogene tadenovec, Ad5FGF-4) in patients with myocardial ischemia due to arteriosclerosis. The results were favorable enough that the firm said that it is accelerating the study. (See BioWorld Today, Jan. 2, 2014.)

The 100-patient trial launched in March 2012 and known as ASPIRE is testing Generx at cardiology centers in the Russian Federation.