August 24, 2012
Where is Regenerative Medicine Heading?
BTST: Can you briefly describe what regenerative medicine is exactly?
Buckler: The traditional understanding of regenerative medicine isn’t as focused on modality as it is outcome. And that outcome is to repair, replace or regenerate cells, tissues and organs in the body, or the function of those organs. But the way to get to that end – or the intent to get there – is what differentiates it from predecessor sectors.
Regenerative medicine can involve anything from small molecules to biologics, implantable devices or even genetically modified cells. What a lot of people end up talking about is what’s really new in the sector, and that is the cell-based approach to regenerative medicine.
BTST: Can you discuss the current state of the regenerative medicine industry? Who are the major players, what kind of growth is being seen, and what does the regulatory environment look like these days?
Buckler: Cell Therapy Group tracks the cell therapy industry, rather than the regenerative medicine industry as a whole. We start on the therapeutic side and track products in clinical development that are sponsored by industry.
One of the interesting phenomena is that, because the existing pipeline has turned to be relatively safe, it’s much easier for cell therapies than other types of therapeutic modalities to get into an initial clinical trial designed to produce some kind of efficacy data.
Currently, the pipeline has a large glut of Phase I and Phase I/II or Phase II clinical trials. When looking at the pipeline globally, there are about 180 industry sponsored cell therapy clinical trials in Phase I/II or Phase II, running on a range of different indications.
We’re also tracking 47 late stage clinical trials, Phase III and/or Phase II/III and other pivotal studies sponsored by 37 companies. Indications are narrower in those than in the earlier end of the pipline, with 30% oncology or related, 34% cartilage/bone repair and the rest in other areas.
From a company perspective, less than 20% of the therapeutic companies with a product in clinical development are public and the rest are privately owned.
The industry is still very fragmented comprised of small enterprises. Only a quarter of companies have more than one product in clinical development. On the other end of the spectrum, roughtone one quarter of the sector is still in pre-clinical development. The bulk of the companies in the sector are in clinical development with their lead product in one indication.
BTST: How has the FDA responded to regenerative medicine clinical trials? It seems like they are a bit unconventional for the traditional approval process.
Buckler: The FDA has been unprecedentedly proactive and engaging of the regenerative medicine industry in trying to create a regulatory framework that fits their pre-existing paradigm as well as this new therapeutic modality.
Many people would argue that they’ve erred on the side of caution. The big question is whether regenerative medicine fits into the IND/BLA type of paradigm given that cell therapies have a very high safety profileSome hope that these therapies would be able to go straight to market by complying with the GTPs and other rules.
In my opinion, the FDA has struck a balance in regulating cell therapies that appropriately reflects the risk-based approach they employ in protecting the public
BTST: How has capital flow been to the sector and what are some of your favorite plays?
Buckler: As with many sectors, it has been a tough environment to raise money. We recently recapped the money going into the sector for 1H 2012 in a Cell Therapy Blog post (here). We’ve tracked just over $540 million into the sector year-to-date. A lot of the money has come from venture capital, retail investors, NGOs and others. One of the more active sources of funding has been non-venture funds run by corporations, like pharmaceutical companies, purely on an investment criteria.
We like to watch the late-stage bucket of companies because there’s potential there for some real movement.
In this bucket fall cell therapy companies like Opexa, Newlink, and Aastrom, for example. Opexa Therapeutics (OPXA), which is headed into a Phase IIB trial for MS, recently started spiking on speculation they are shopping around for a partner. NewLink Genetics (NLNK) is in a Phase III trial with a readout projected for a year out that is currently riding the cancer immunotherapy wave. And finally, Aastrom (ASTM) is a late-stage company with a CEO that’s trying to clean up their shareholder base and corporate finances while balancing a pipeline of trials in various indications.
BTST: Thank you for taking the time for this brief interview!
About Lee Buckler
Lee has been an executive in the cell therapy space since 2000. He is a consultant, blogger, analyst, speaker, author and entrepreneur in the cell therapy industry. Lee is the founder and managing director of Cell Therapy Group – a consulting firm focused on the cell therapy and cell-based regenerative medicine industry. He is also co-founder/owner RegenerativeMedicineJobs.com.
After 6+ years with the Stem Cell Technologies group, he spent 2+ years with Progenitor Cell Therapy (one of the leading cell therapy manufacturing companies in the world), and then launched a consulting company (Cell Therapy Group) exclusively focused on the cell therapy, stem cell, and cell-based regenerative medicine sector.
Lee blogs at CellTherapyBlog.com, interacts with an engaged following of 1,600+ on Twitter as @celltherapy, hosts a cell therapy-related stream on YouTube, administers cell therapy industry-related video content on BioBuisiness.TV, and runs the LinkedIn Cell Therapy Industry Group engaged in peer-to-peer exchange of information and opinion among its now 3,500+ members.
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