Advocating for Advanced Therapies

My sense is that we’ve come a long way and that 2013 was actually a very good year — perhaps maybe even the best year ever for regenerative medicines and advanced therapies. Clearly the financial markets have allowed us to do more in terms of raising capital to fund projects in this space, and we are seeing a growing interest in the sector in the investor community.

We’ve seen a number of major financing events over the course of the past year, and we’ve seen the IPO market become an opportunity for companies to access new groups of investors to an extent that it never has been before. We just came off a panel [at the Phacilitate conference, see box below] where the bluebird bio IPO (www.bluebirdbio.com) was a major point of discussion. That financing came together in ways that simply couldn’t have happened just a few years ago. You could argue that the market conditions played a large role in this, but the company did an excellent job putting together the pieces that allowed it to go out and raise money, benefiting from solid management and the closure of timely and significant deals. Establishing a global strategic collaboration with Celgene Corporation in March 2013 was especially important in positioning bluebird bio for a successful IPO. The company is working toward novel, disease-altering gene therapies in oncology by using a patient’s own genetically modified T cells — chimeric antigen receptor (CAR) T cells — to selectively target and destroy cancer cells.

Momentum around gene therapy is growing because companies such as bluebird bio have learned to articulate the value proposition and communicate opportunities in a way that is compelling to investors. Another example is the partnership between Novartis and the University of Pennsylvania’s Perelman School of Medicine (Penn). Their investigational chimeric antigen receptor (CAR) therapy (CTL019) is clearly a major event, and it is capturing the attention of the oncology community with extremely compelling data. This ex vivo gene-modified, autologous cell therapy could provide transformative care. We are just beginning to see the data. Many issues still need to be addressed around developing a model for commercial scale up. Pricing and reimbursement will be big issues, but this is the right discussion: not just whether the technology works, but how it can be translated from really good science and clinical success into something with commercial potential.

Supporting Patient Access to Regenerative Medicine

At the recent Phacilitate Gene and Cell Therapy Conference (27–29 January 2014 in Washington, DC), BPI’s editor in chief Anne Montgomery and publisher Brian Caine spoke with Morrie Ruffin, managing director of the Alliance for Regenerative Medicine (ARM). Ruffin is also managing partner/owner, of Adjuvant, a strategic consulting practice specializing in support for advanced therapies and regenerative medicine companies and organizations.

We asked him to comment about the status of regenerative medicines and advanced therapies today, comparing the current outlook with that of only a few years ago — going back to the beginning of ARM in 2009. Here he offers his insights into the progress made thus far, the role of ARM in advocating for patient access to advanced therapies, and his future outlook for this rapidly advancing industry segment.

Morrie Ruffin ()

And that is the discussion across the board. Other gene therapy companies have raised big sums over the past year, and we are also seeing that happening with cell therapies, allogeneic therapies, and mesenchymal stem cell (MSC) products.

Regenerative Medicine in 2014

2014 will be an important year. The market is anticipating major clinical events in this space that will hopefully drive continued investment. Over the course of this coming year, we anticipate 14 or 15 major clinical events, with phase 2 and phase 3 readouts of clinical data in a number of key indications. It is this type of news that we hope will sustain momentum in this sector.

As an organization, ARM represents what we call next-generation and advanced therapies. Regenerative medicine is the term used to describe them, and most people are comfortable using that term to encompass all these next-generation technologies. In addition to the numerous technologies in clinical development, we already are seeing the benefits of tissue engineering and first-generation regenerative products such as Apligraf and Dermagraft (www.organogenesis.com). We also promote work in the orthopedic space where cartilage regeneration products are gaining a lot of attention. Another area of significant progress is the use of advanced scaffolds and technologies to allow us to build and repair organs.

Such regenerative medical technologies are not expected to follow the linear progression that you might expect to see in other technologies, certainly in traditional drug development. These are new technologies. They involve various different types of partnering models to help build the infrastructure to support them. So it is going to take time. I think that the investors and most of the key participants are beginning to see how these pieces are going to fit together, so that is very exciting.

Over the past year more hospitals announced their work in regenerative medicine. Especially when looking at development of autologous therapies and related apheresis processes, hospitals recognize that this represents a paradigm shift in administering care. Many medical institutions are therefore making significant investments in this space. What we are all trying to understand is how hospitals are going to participate and what role they are going to have in the ultimate commercializing of certain products. What part of the reimbursement are they are going to capture, for example? But they certainly recognize the opportunity. It is very exciting to see the engagement of groups such as the Mayo Clinic, Penn, Sloan Kettering, and MD Anderson as well as all the California hospitals and research institutions such as the Scripps Research Institute, the University of California, San Diego, Stanford, and the Gladstone Institute.

Tool- and service-providers are playing an increasingly important role. Companies such as GE Healthcare, with its Healthy Imagination Fund (www.healthymagination.com), are becoming increasingly public about their commitment to this space. GE wants to become a major provider of the industrial infrastructure to support this next major wave of medical innovation, so it is investing across the board in technologies that will enable that. Life Technologies (recently acquired by Fischer Scientific) is another company that will continue to play a very large role in this space.

This sector involves a number of different key stakeholder groups. A very important part of ARM is participation of patient groups. With the Juvenile Diabetes Research Foundation as one of our charter members, we recognized early on that our ultimate concern is the impact that advanced therapies w
ill have on patients, on their healthcare and clinical outcomes. We expect to work very hard with patient groups to help them address their needs and define the role that they would like to play in providing an important voice for new, transformative therapies. These groups are providing some important early funding in a number of areas — and certainly in a number of technologies in which ARM is involved. But they will be able to do even more as we move forward because their voice and perspective will be essential, including the education of key decision makers in Washington, DC. That is something ARM takes very seriously and it is why we involve patient groups in much of our advocacy work.

ARM also takes increasing access to capital very seriously. Although we are a nonprofit, we believe that an important role for the organization is to raise the profile of this sector in the investor community. To that end, we are planning to increase our various investor and partnering events in this coming year.

The most important benefit of ARM membership is that it enables interactions with all stakeholders. First and foremost, as an advocacy organization we lead efforts to advocate for the sector through direct interactions with key decision makers, whether in the FDA, CMS (Centers for Medicare and Medicaid Services), or other payer communities. Companies know how critical it is to be able to articulate the pharmacoeconomic rationale for a new product and a pricing strategy that will make sense. Getting feedback from experts and key opinion leaders who can communicate effectively to investors and prospective pharmaceutical partners will contribute to the ultimate success of regenerative medicine.

About the Author

Author Details
Morrie Ruffin is managing director of the Alliance for Regenerative Medicine (ARM) and managing partner/owner of Adjuvant; [email protected]; 202-345-3675.