I invited Neuralstem CEO, Richard Garr, to do a Q&A interview and he kindly accepted. The interview provides some novel insights into this major biotech in the stem cell sector.
1. How is Neuralstem doing today? What programs are underway that you find particularly exciting?
Garr: Neuralstem is moving on all cylinders these days on both our cell therapy and small molecule clinical programs. We have just completed all the transplantations of our phase two ALS patients, with the data lock coming early in January, and hopefully the phase2/3 starting early next year; and in September we expect to transplant our first SCI patient at UCSD (chronic thoracic ASI-a patients). We continue to dose phase 1 patients in our stroke trial in China. On the small molecule side, the MGH group presented our 1b data in MDD patients at ASCP this past june and it was frankly, stunning; showing clinically meaningful improvement in all the patients who took the medicine as well as reaching statistical significance in treatment of depression measures, as well as cognitive improvement. It is a first in class neurogenic drug, and we we will be going into a phase two we believe sometime near the end of the first quarter next year.
2. How are the financials?
Garr: The financials are stronger than they have ever been, and the last Q showed roughly $30 million in cash and equivalents. Our burn rate continues to be a fraction of the industry norm thanks to our near virtual model, and generous funding of our ALS trial by the NIH and ALSA.
3. Where do you see the company in 5 years? 10 years?
Garr: We expect to commercialize the cell therapeutics ourselves, certainly in the U.S. With partners world wide in selective markets. We expect to take the small molecule drug through, at least phase two, but ultimately to partner it out. We have a wholly owned subsidiary in China, and a partner with an option on markets in Korea, Vietnam, Malaysia, Indonesia and Singapore, so we see a great deal of our growth also coming in Asia in the long term in cell therapy.
4. What makes Neuralstem unique as a stem cell biotech company?
Garr: I don’t know that we are unique, but we are built differently than most. We were built to move our technology forward, in every therapeutic area to which it might apply; we don’t look for other technologies for a particular disease, and we don’t look to expand beyond what we own. So we, from that point of view, we are perhaps more focused than other companies on a particular way of treating diseases. On the cell therapy side, we only have looked at incurable disease from the start; we believe that that is what our technology is capable of, and that has been our goal from the beginning. That is now and has always been the ethos of our company.
5. How important is patient involvement in the stem cell biotech arena? Building on that, what is your view of Right To Try laws?
Garr: We have always been a much more “patient centric” company than most. And we have always had an intimate relationship with various patient advocacy groups. It is part and parcel of our make up. And yes, that is why I got involved with the RTT movement. There are roughly 30,000 ALS patients, 5,000 newly diagnosed each year; and there is literally nothing our there for them to significantly improve their quality of life or their life span. We believe our ALS therapy is doing both; our trials have not yet been powered to demonstrate that to the level that it meets various existing FDA early access programs, and I think that with the right safeguards RTT can provide a structure to build such programs around. My view of RTT is that it is dependent on, and must work with the FDA. It is built so that it relies on FDA diligence on safety; and I can tell you from our own experience that when you develop treatments for terminal diseases (and the law only applies to terminal diseases) you have spent (at least) tens of millions of dollars before you ever get into a phase one human trial, and you have a great deal of safety data before and after such trials. The law also requires that you be actively engaged in at least a second FDA trial, so these eligible treatments are under the constant villigance of the FDA in order to be eligible. I understand that there are many issues to be worked out in terms of liability and payments to mention just a few; but these are the types of issues this industry deals with every day, this is not peace in the middle east we are trying to solve here. And so when critics of RTT bring up issues with the implementation, to me that is a separate issue from whether or not RTT is the right thing to do; and while those are serious issues, they are “second level” issues. Also, it is a transitory program; that is, it’s only meant to provide access until the drugs are either approved by the FDA and available, or shown not to work. As Ted says, it’s a right to try, not a right to cure. Everyone involved understands that.
6. What is the IP portfolio like for Neuralstem? I understand there has been a conflict with StemCells, Inc. and Greg Schiffman commented on that in my interview with StemCells, Inc. leadership. Can you please let us know the Neuralstem perspective on this?
Garr: I won’t comment on the ongoing litigation other than to say this; the end will speak clearly for itself. With respect to our IP position, it should be understood that NONE of our patents are being challenged in this litigation, only STEM’s patents are being challenged in this litigation. We have patents issued worldwide which cover both our cell therapy and small molecule platforms.
7. How important is social media in this day and age for biotechs? As both a scientist and blogger myself, I note your blog as being unique and valuable to patients, investors, and the larger community. What prompted you to do a blog? What has your experience been like with it so far?
Garr: I think that social media is an essential part of communication today, with all the stakeholders in a company. I started the blog because of the intense interest in what we are doing from the patient and caregiver communities, and as it has evolved, that has become the main focus. It is complicated by the fact that we are a public company which brings in an entirely new and different set of rules and regulations that inform what you can and can’t say (about ongoing trial data for instance) but those are simply rules that one lives with. Social media is changing the way patients communicate with each other. That’s a genie you can’t put back in the bottle. We see patients in trials blogging about their progress (or lack thereof). They are not like juries where you can quarantine them until they reach a decision. It’s all about community, and patients and their support groups are always looking for more community in these stressful situations, it is a human instinct, and I believe a good one. We reach out to comfort each other, that is what is behind the exponential growth here. The industry is going to have to figure out a way to accommodate the new reality of social media and clinical trials, just like every other industry has had to learn how to adapt to the internet age.