Value, Validation Mark Upbeat State of Industry in Regenerative Medicine

In delivering the Alliance for Regenerative Medicine’s (ARM) 2016 state of the industry briefing at the Biotech Showcase event this month in San Francisco, chair of the international advocacy organization representing the regenerative medicine and advanced therapies (RM/AT) community, Edward Lanphier, said 2015 had been a great year for the sector, exemplified by excellent clinical data readouts that have helped attract significant investments and big pharma validation through major partnerships.

The sector continues to evolve and grow and ARM has identified 672-plus companies, including those involved in gene and cell therapies, around the world that are active in the space. That represents a 30 percent increase from the 517 companies identified in 2014. (See BioWorld Insight, Jan. 20, 2015.)

Approximately 52 percent, or 349 companies, are located in U.S., with 185 (27 percent) in Europe and 112 (17 percent) in Asia. Lanphier was particularly enthusiastic about the growing value creation in the sector through the approval of 20 new products and expanding number of ongoing clinical trials. In fact, since the year-earlier report there has been an almost doubling of clinical trial activity, with more than 631 trials identified in 2015; 70 percent of those have progressed to the phase II and phase III stages. More than 40 percent of those are targeting oncology indications.


There is no doubt that oncology trials “have really taken off,” Lanphier noted, “particularly with the explosion in cell-based immuno-oncology.”

CAR T and other cell-based therapies are being advanced by a host of major companies and research institutions. For example, at the American Society of Hematology (ASH) meeting in December, Novartis AG presented the latest findings from an ongoing phase II study of CTL019, an investigational chimeric antigen receptor T cell (CAR T) therapy, which further support its potential in the treatment of children and young adults with relapsed/refractory acute lymphoblastic leukemia (r/r ALL). The study found that 55 of 59 patients (93 percent) experienced complete remissions (CR) with CTL019. Overall survival was 79 percent at 12 months.

Juno Therapeutics Inc. also reported clinical data from separate trials for CAR T cell product candidates, JCAR015 and JCAR014, demonstrating encouraging clinical responses in patients with r/r ALL. The JCAR014 data showed the addition of fludarabine to cyclophosphamide-based lymphodepletion improves CAR T-cell expansion, persistence and disease-free survival in r/r B-cell adult ALL patients treated with a defined cell product.

In the area of gene therapy there are also a number of mature programs in progress. Lanphier referenced Uniqure NV, which reported that four of five patients suffering from severe or moderately severe hemophilia B have been able to discontinue prophylaxis with recombinant factor IX, following treatment with its AMT-060 gene therapy, and Spark Therapeutics Inc.’s phase III top-line data with its compound, SPK-RPE65, for blindness-causing, RPE65-mediated, inherited retinal dystrophies has impressed. That trial demonstrated a highly statistically significant improvement in the intervention group compared to the control group in the primary endpoint, the change in bilateral mobility testing (MT) between baseline and one year. The first two secondary endpoints – full-field light sensitivity threshold testing and MT for the assigned first eye – also showed highly statistically significant improvement. (See BioWorld Today, Jan. 8, 2016, Oct.6, 2015.)

Separately, at the J.P. Morgan Healthcare conference, Spark outlined its comprehensive three-year vision for expanding the company’s portfolio through 2018 and launching its first commercial product in 2017.

In all, the Philadelphia-based company will advance a total of 10 total programs across three therapeutic franchises by 2018, including one commercially approved therapy, two in pivotal-stage trials and at least seven additional programs in clinical proof-of-concept trials.

Those are just a few examples where significant value is being created, and it bodes well for a number of new approvals to come in the not too distant future, Lanphier explained.

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