OneMedResearch recently initiated coverage on Opexa Therapeutics, a biotechnology company developing patient-specific cellular therapies for the treatment of autoimmune diseases. Below, company President and CEO, Neil Warma, discusses Opexa’s lead candidate, Tcelna, a personalized T-cell immunotherapy treatment of multiple sclerosis.
Click below to hear full audio interview and see transcript that follows.
Matthew Margolis: Greetings from OneMedRadio. I’m Matt Margolis. Today, we’re speaking with Neil Warma, president and CEO of Opexa Therapeutics, a biotechnology company developing patient-specific cellular therapies for the treatment of autoimmune diseases. Opexa’s lead candidate, Tcelna, is a personalized T-cell immunotherapy treatment of multiple sclerosis. Opexa trades on the NASDAQ under the symbol OPXA. Thank you for joining us, Neil.
Neil Warma: Well thanks very much, Matt, for the opportunity.
MM: So let’s start with an overview of the company, and secondary progressive multiple sclerosis. What is this condition, what are some global trends?
NW: Sure, Matt. Well as you mentioned at the outset, Opexa is developing a novel personalized therapy that really has the potential in numerous autoimmune diseases. We are currently preparing to conduct a phase II clinical trial in secondary progressive multiple sclerosis and if we’re successful, this could be the first safe and effective treatment for these patients who currently have limited treatment options.
You specifically were asking about secondary progressive and we’ve taken the choice over the past six months to focus on this specific indication of MS. Secondary progressive MS is the more advanced form of multiple sclerosis. Most patients are initially diagnosed with the relapsing/remitting form of multiple sclerosis. This is a chronic neurological disease so over the course of several years, almost all of these relapsing/remitting patients do evolve or do progress to the more advanced form known as secondary progressive MS. SPMS or secondary progressive MS is characterized really by a steady progression of clinical neurological damage, which may be accompanied by relapses but doesn’t have to be and really at any one time approximately half of all MS patients will meet the diagnosis criteria of secondary progressive.
Looking at treatment options for these patients, currently there’s only a single product with the distinct labeling to treat the large subgroup of MS subjects who have progressed to secondary progressive. The drug that is indicated for secondary progressive is rarely utilized in the population because of the side effects. They are significant and this puts an extra burden of responsibility on to the prescribing physician as well. So really treatment options for patients with secondary progressive MS are very limited. So therefore treatment alternative for SPMS subjects has great potential, which is why we have high hopes for Tcelna as that could be the treatment of choice for patients with secondary progressive MS.
MM: So what is T cell based therapy and why is this particular strategy opportunistic to target MS and other autoimmune diseases?
NW: Well T cells are the main cells that compromise our immune system and they play obviously a central role in cell-mediated immunity. So a T cell therapy, T cell based therapy therefore is one that will impact the immune system or immune cells and autoimmune diseases are those disease in which an individual’s immune system is faulty or dysfunctional.
So a T cell therapy such as the one that we’re working on here at Opexa, Tcelna, it’s a therapy that can be used to treat a multitude of autoimmune diseases. So a T cell platform if you will could theoretically be applied to a number of autoimmune diseases. Our first focus here at Opexa as we’ve mentioned is multiple sclerosis and this specific disease is well suited to our antigen specific disease approach.
Multiple sclerosis is caused by a subpopulation of immune cells that become activated and begin to attack nerve fibers in the brain. For MS patients, their immune system is unable to control these destructive cells. They have an autoimmune disease, therefore, their dysfunctional immune system doesn’t allow them to protect themselves against these individual T cells. So Tcelna, Opexa’s therapy, will selectively target only these pathogenic or destructive T cells and Tcelna does not impact the entire T cell population like other MS drugs. Because we’re selective in only targeting these pathogenic T cells, we believe our side effects are that much less than other drugs.
To look at it from a higher level, Tcelna or T cell therapy aims to restore the function of the body’s immune system and because of this impact on the overall immune system, we do believe it has applications in several other autoimmune diseases.
MM: Can you discuss some intriguing data surrounding the anticipated phase IIb trial?
NW: Well it’s interesting. I mean we’re planning to conduct a IIb study in secondary progressive patients. So looking at the data that we’ve generated in secondary progressive and we’ve treated really a range of patients from relapsing/remitting to secondary progressive and we’ve seen benefits kind of across the range of MS patients. So in theory, we think it could be really a therapy of choice for all MS patients. The side effect profile for Tcelna seems to be really superior we believe to all other products and the efficacy seems to be at least as good as the best. I think our mechanism of action for Tcelna does set us apart from other drugs.
When we look at the data that we’ve generated from secondary progressive patients, it is quite encouraging. We treated a number of patients. There were a number of clinical studies done by us and also by Baylor College of Medicine and when we look at the patients across the board. 80% of all the patients treated with Tcelna showed no disease progression after two years, which is highly encouraging. Also, in these secondary progressive patients, the majority of them were having relapses at study entry and what we saw following Tcelna treatment was that the relapse rate for these patients dropped almost to zero. So in 21 years of cumulative patient follow-up on this group of patients we were investigating in our earlier studies, only one patient had one relapse. Additionally in the two years following treatment, the secondary progressive patients showed no worsening of physical or psychological conditions and these are two quality of life indicators.
So really across those three endpoints if you will, we’re very encouraged by the data that we’ve generated in secondary progressive. If we can continue to develop Tcelna and see data such as this developing both a safe and effective therapy, again it could be one of the dominant therapies in this indication. Currently, the MS market generates approximately $11B of annual revenue so again we’re strongly believing in the potential for Tcelna.
MM: Now in November, the FDA granted fast track designation to Tcelna, can you discuss receiving this FDA fast track approval?
NW: Certainly. It was a significant milestone for the company. We have developed kind of very strong relationships with the FDA here in the US and also with Health Canada, the regulatory authority in Canada and we submitted our dossier to the FDA a number of months ago requesting fast track. FDA grants fast track only in diseases of significant unmet need and to therapies that demonstrate the potential to have a significant benefit in these diseases. So by granting us fast track, the FDA recognizes that certainly the secondary progressive is a disease of high unmet medical need and that Tcelna has the potential to benefit these patients. So again, we were very pleased to get this endorsement from the FDA really as it recognizes the potential of the therapy and it should also speed our time to commercialization we hope.
MM: So in that light, maybe you can talk a little bit about a development and commercialization timeline. Your most recent preliminary meeting with prospective clinical trial investors is particularly interesting.
NW: Certainly. As we’ve stated, we are planning to initiate a phase IIb clinical trial in secondary progressive patients and then if this is successful, we would envision in moving immediately in a phase III pivotal trial and this would be subject to FDA approval. Each of these trials will likely be a two-year trial and if the data from both of these trials are encouraging, we would then look to seek approval by FDA following such a clinical program.
The recent meeting you referred to at the annual neurology conference, the AAN was very successful. We had some of the biggest names in MS, some of the key opinion leaders in MS in attendance. Really, we spent a fair amount of time to speak about the specific trial design, the phase II trial design. Also issues around patient enrolment, side questions, and answers so it was a very motivating discussion for us and again many of the MS leaders reflected on the potential of Tcelna in secondary progressive MS so again highly encouraging.
Operationally, we believe we remain on track to initiate the trial in the coming months. We’re all working diligently here to finalize preparations and as we’ve also stated in order to initiate the trial, we need to secure additional financing either through a potential partnership or additional capital raise. This continues to be an important focus for us as well.
MM: Now your proprietary T-cell technology platform is said to generate multiple drug opportunities. So where else are you focusing? What is your progress with those candidates?
NW: Very good question. We’ve had a lot of internal discussion about the potential of the platform kind of across numerous autoimmune diseases and we believe as we’ve stated earlier in this interview that there is potential in many other autoimmune diseases and we’ve discussed such relevant diseases, such as type 1 diabetes, rheumatoid arthritis, all of these are antigen driven diseases. There’s also some orphan and niche indications that could be very attractive to us as well. We have a strong patent position that should allow us to expand nicely into other disease areas as well. So we’ve had a lot of internal discussion. We’re certainly, you know, focused on the MS trial and getting that up and moving forward, but again just to reiterate, the potential to expand the pipeline beyond multiple sclerosis we believe is quite high.
MM: And what challenges and opportunities do you see going forward?
NW: Well, there’s always challenges and opportunities for any company, certainly in the biotech industry as well. I think challenges for us, you know, certainly being a public company, we are influenced by kind of the capital markets both positively and negatively, the volatile capital markets. I think for us as well building and maintaining evaluation for Opexa that is more reflective of our true potential is also a challenge. We do believe given the state of development and the novelty of the platform, we are undervalued so working to build value in the company is very important.
As far as opportunities, I think there are several key ones. I think firstly, you know, we are pleased. We do have a novel therapy that has demonstrated promising clinical efficacy and a superb safety profile really in an area of significant medical need and with substantial market opportunity as well. So we are really in the position of advancing one of the more promising therapies for MS through clinical development towards commercialization so a huge opportunity for us. I think if we’re successful, the return on investment for our shareholders will be significant as well.
We also have the opportunity coming at it very motivated by our discussions and interactions with patients and we really have the opportunity to bring to market really for the first time a safe and effective treatment for secondary progressive MS patients. Again, our interactions from the patient community make this a highly motivating element and opportunity for us tremendously.
MM: What are your key strategies for building shareholder value?
NW: Neil Warma: I believe key to building shareholder value is executing on the upcoming MS clinical trial first and foremost. We will certainly continue the evaluation of additional autoimmune diseases that we can potentially treat with our T cell platform, but really the focus is on the upcoming MS trial. Should this trial be successful as we believe it will be, the value generation for Opexa would be significant and we will have delivered a substantial return on investment for our shareholders.
MM: That was a company snapshot of Opexa Therapeutics with Neil Warma, president and CEO. Opexa trades on the NASDAQ under the symbol O-P-X-A. This is Matt Margolis with OneMedRadio signing off.