A number of new and innovative medical paradigms are being explored by companies in the life sciences sector, with one of the front-runners being cell therapy—a disruptive technology that has, in the past few years, progressed out of preclinical studies and into clinical development. To learn more about the transformative promise of regenerative medicine, and what investors might expect from companies working in the sector in 2015, The Life Sciences Report asked analysts Jason Kolbert of Maxim Group and Dr. Christopher James of Brinson Patrick to discuss the advances that could generate robust returns on investment in coming years.
Cell Therapy in a Nutshell
Cell therapies have been gaining momentum in the life sciences for more than half a century. The first cell therapies, bone marrow transplants, were performed in the 1960s, and since then the idea of using healthy cells to replace or regenerate diseased cells has expanded into a number of indications, from cancer to cardiac disease to cosmetic therapy. Today, a number of companies are engaged in both clinical and preclinical trials, and there is some consensus that cell therapies will be "mainstream" in the near future.
The new year could prove a bellwether for regenerative medicine. The sheer volume of companies, universities and financial institutions conducting or backing research and development in the field attests to its potential: The Alliance for Regenerative Medicine (ARM), an advocacy group working with legislators, the investment community and regulatory agencies to promote development of cell therapy technologies, has more than 170 members working in the field.
On the horizon: Australian cell-therapy company Mesoblast Ltd. (MSB:ASE; MBLTY:OTCPK) is positioning itself for expedited approval of several of its allogeneic (derived from the same species) cell therapy products in Japan in 2015, following passage of legislation in that country that expedites approval. And though it hit a significant speed bump last month, Dendreon Corp.'s (DNDN:NASDAQ) Provenge (sipuleucel-T), an autologous (derived from and returned to the same patient) cell therapy for metastatic castrate-resistant prostate cancer, was the first FDA-approved cell therapy, and has helped pave a path forward for additional approvals in the U.S.
To get a handle on what investors can expect, and an idea of which companies to keep an eye on, The Life Sciences Report turned to Dr. Christopher James, managing director and senior equity research analyst with Brinson Patrick Securities Corp. and Jason Kolbert, managing director, senior biotechnology analyst and head of healthcare research with Maxim Group. Here's what they had to say about what 2015 might bring in both the sector and for certain companies working in the space.
Great Expectations
Asked which specific indications could be most responsive to cell therapies, and about companies working in those therapy areas, Kolbert provided several names.
The next "major event" in the sector will be results in a Phase 2 trial in ischemic stroke being conducted by Athersys Inc. (ATHX:NASDAQ), with results expected in Q1/2015, the analyst said. A number of other trials are also slated to start in 2015, including NeoStem Inc.'s (NBS:NASDAQ) Phase 3 trial in acute myocardial infarction and Cesca Therapeutics Inc.'s (KOOL:NASDAQ) pivotal trial in critical limb ischemia.
Kolbert is also looking forward to two trials involving Mesoblast's own stem cell product, as well as results from Prochymal (remestemcel-L, human mesenchymal stem cells for intravenous infusion, acquired from Osiris Therapeutics Inc. [OSIR:NASDAQ]). The Mesoblast product is expected to enter a pivotal trial in back pain, and the company is currently enrolling a global trial in congestive heart failure with partner Teva Pharmaceutical Industries Ltd. (TEVA:NASDAQ). The Osiris product is expected to be redeveloped in the U.S. for graft-versus-host disease (GvHD). Prochymal has been conditionally approved in Canada and New Zealand for GvHD in children. In addition, Mesoblast is expected to release data in a Phase 3 Prochymal trial in Crohn's disease.
James believes the area where cell therapy—cell regeneration—could have the biggest impact is also one of the riskiest—in the central nervous system (CNS), treating disorders or injury of the brain or spinal cord. James sees applications in multiple sclerosis (MS) and spinal cord injury, to regenerate neuronal function, and in Parkinson's disease, to address the death of dopaminergic cells. He also likes ocular applications, for treatment of such conditions as age-related macular degeneration (AMD).
James has two cell therapy companies under coverage. Opexa Therapeutics Inc. (OPXA:NASDAQ), with its Tcelna platform technology, is developing therapies in both CNS and the eye. A Phase 2 trial in secondary progressive MS is expected to read out in mid-2016. The company is also developing OPX-212, an autologous T-cell immunotherapy, to treat neuromyelitis optica, an autoimmune condition that leads to blindness.
The second company, StemCells Inc. (STEM:NASDAQ), is in a Phase 2 trial in the treatment of cervical spinal cord injury. On Dec. 18, the company announced that it had transplanted its HuCNS-SC (purified human neural stem cells) into its first patient; James expects final data from that trial in late 2015. Results from the company's Phase 1/2 trial in dry AMD, are expected in Q1/2015—perhaps as early as January.
StemCells' Phase 2 trial in cervical spinal cord injury points out what James sees as one of the regulatory hurdles cell therapies will have to overcome as they progress through the U.S. Food and Drug Administration's (FDA's) approval process. The trial is a randomized, controlled, single-blind study, as opposed to the FDA standard of double-blind pivotal studies.
To treat central nervous system disease or injury, companies must inject cells into a patient, a "surgical" intervention, James explained. But clinicians "can't do a sham surgery on the brain or on the spine. . .that's not ethical." So companies like StemCells must work with the FDA to develop pivotal trial designs that are "less stringent" than the double-blind model.
The Bigger Picture
Kolbert is enthusiastic about the prospects for cell therapies in 2015, and doesn't see much in the way of headwinds. "Cell therapy ultimately lowers the cost of treating disease, and at a time when there is an outcry over high-priced therapies, cell therapy offers promise to lower the cost of treating disease. In terms of politics, the cell therapies we are focusing on are typically not controversial (using adult stem cells, not cells from embryos). As such, we don’t see any controversy. The big change in the landscape now is the acceptance that cell therapy is 'safe.'"
On the plus side of the spectrum is oncology. Using cell therapy in cancer immunotherapy "holds great promise," Kolbert continued. "The ability to rev up the immune system with checkpoint inhibitors, and couple that with therapeutics, like the therapies being developed by ImmunoCellular Therapeutics Ltd. (IMUC:OTCBB) and Agenus Inc. (AGEN:NASDAQ) targeting glioblastoma, and the work that OncoSec Medical Inc. (ONCS:OTCBB), Inovio Pharmaceuticals Inc. (INO:NYSE.MKT) and NeoStem are doing in melanoma, is exciting."
James and Kolbert agree that recent elections, in which Republicans gained control of both houses of Congress, shouldn't affect ongoing work in the cell therapy field. "They have bigger issues to deal with," James commented.
However, James believes cell therapy companies may suffer from an "overhang" following the recent bankruptcy of Dendreon Corp. And Kolbert cautions investors that second- and third-generation technologies always follow. Such is the case with Bavarian Nordic's (BAVA:OMX) Prostvac. Prostvac has the potential to be equivalent to, or even superior to, Provenge, but at a fraction of the cost. The Phase 3 Prostvac trial reports data next year.
Another concern for investors, according to James, is the question of whether cell therapy companies can manufacture their products on a commercial scale. But the analyst believes that concern can be addressed, and cites the StemCells model, in which, according to the company, "[c]ryopreserved lines of donor-derived cells can be reproduced at commercial scale as 'stem cells in a bottle,' then distributed for patient doses as needed, much like an off-the-shelf pharmaceutical product."
