Biostage is a clinical-stage company specializing in bioengineered organ implants to treat cancer and other conditions involving the esophagus, bronchus and trachea. In August 2017, Biostage transplanted its esophageal implant product into its first patient worldwide, a 75-year old man with cancer. On the heels of this historic accomplishment, I am pleased to share this interview with Jim McGorry, President and CEO at Biostage, Inc. In this interview, we discuss the company’s vision, its research partnerships, clinical trial strategy, and five-year goals.
Please note that this interview was conducted prior to Biostage’s breach and failure to fund by First Pecos.
Read on to learn more about how Biostage is introducing bioengineered organ implants to the regenerative medicine landscape.
Interview with Jim McGorry, President and CEO of Biostage, Inc.
Cade Hildreth: How and when was Biostage founded?
Jim McGorry: The company, called Harvard Bioscience, has been in around the Boston area for almost 100 years. I call it the “garage band” of researchers, because using this name, the founders started out of a garage and made high-end instrumentation for researchers. With the roll-out of different companies and a diverse range of products, from syringe pumps to complex bioreactors, today mostl PhD researchers recognize the name Harvard Bioscience.
Then, about 5 years ago, the company saw a trend in regenerative medicine, with different researchers around the world doing de-cellularization/re- cellularization procedures in lungs, liver, or kidneys, and other tissues and organs. These researchers needed bioreactors, because the field started changing, so the company started supplying these types of tools to scientists around the world.
With a very good knowledge in this area, Harvard Bioscience engineers and physiologists at began serving this market niche across many different countries. The trend toward regenerative medicine was so strong, that they then spun-off a piece of the company to be exclusively focused on it. The original name of this company was Harvard Apparatus Regenerative Technology, but it has since been renamed to be Biostage. That is how the company evolved. We started as a bioreactor company and grew into a biotech company. Right now, we believe that hollow organs – the trachea, the bronchus and the esophagus – are the next opportunities for solving unmet medical needs using regenerative medicine.
Cade Hildreth: How did you become involved with Biostage?
Jim McGorry: Previously, I had worked with two major companies spanning 30 years within the healthcare niche, Baxter Healthcare and then Genzyme. I was on the ground floor of starting Genzyme’s biosurgery business, bringing biotechnology to surgery back in 1996 and 1997. We had the first two commercially available autologous products, Epicel and Carticel, for skin and cartilage. I was involved with that.
I had a very good 12-year career at Genzyme, but after Sanofi purchased them, Genzyme’s former CEO of Henri Termeer, a big name in the industry, said to me, “Regenerative medicine is going to be the next frontier.” I had a background in surgery at Baxter and also oncology since I was on the ground floor in starting Genzyme’s oncology and biosurgery business, so I got involved on the board of what is now Biostage. As of two years ago, I then became the CEO of Biostage.
It’s a very exciting moment to have stepped in as CEO, as our lead product is focused on esophageal cancer. Are you familiar with how esophageal cancer is treated right now?
Cade Hildreth: I know a little bit, but I’m sure that our readers would appreciate more background on it. Could you share it with me?
Jim McGorry: Yes, esophageal cancer currently affects about 19,000 folks in the United States, but almost half a million worldwide. Right now, the approach is a surgical one in which the surgeon does a dissection to take out the tumor. The problem is that this leaves a gap, so there is something missing. The current “Standard of Care” is almost medieval. They go into the peritoneum, take your stomach up through the diaphragm, make this “bagpipe-like organ” into a tube, pull it up into your chest and then attach it. Alternatively, they take your colon up into the peritoneum and the colon doesn’t belong in the chest either. That 12 hour procedure is what is done right now, and the five-year survival for that procedure is about 15%.
Cade Hildreth: What has it been like to move Biostage’s Cellspan Esophageal Implant into clinical trials?
Jim McGorry: There is a dramatic need for Biostage’s technologies. Right now, we are pivoting from large animal studies into human studies, having filed an IND and achieved the implantation of our Cellspan Esophageal Implant into our first patient in August 2017. We have done preclinical studies in 40 large animals so far, where we take a biopsy of the patient’s adipose tissue, isolate and expand the mesenchymal stem cells (MSCs), and then we seed these cells onto a polyethylene scaffold. With 50 years of experience in bioreactor technologies and experience with medical devices, we combine this expertise with a cell-based approach to create a “smart device.” We then deliver it near to where that tumor was removed and allow the patient’s body to heal itself. The paracrine effect, the cell signaling, and the cytokine release combine to create an immunoprivileged implant that can support tissue regenerative.
Right now, I am sitting here with a textbook “Principles of Tissue Engineering”, with three names on it: 1) Robert Lonza, who’s his company was Ocata was bought by Astellas, 2) Bob Langer, a giant name at MIT, and 3) Dr Vacanti, the Chairman of our Scientific Advisory Board.
For those cells on the scaffold, the thought for 25 years was that things would incorporate into the scaffold. However, we see in our technology a totally different paradigm. The body knows what to do. We deliver those cells onto that scaffold using what we call a “Cellspan Implant.” With that, what happens is that after about three weeks, the body doesn’t even need the scaffold anymore. The cells grows right over the top. Then, we do a non-invasive procedure, an endoscopy, and remove the scaffold. So, the only thing left in the body is a biologic response. Next, we hold that open with a stent, so the rest of the inner lumen and the epithelium and the other cell layers continue to grow.
In large animals, we have animals alive a year after implant. This is the area that we are very excited about, the application of our Cellframe technology in hollow organs. I mentioned advanes in skin applications earlier, in terms of Epicell and Carticel. We strongly believe that the next application of a therapeutic breakthrough will combine the use of stem cells with smart devices to precisely deliver therapeutic cells. That is what’s very exciting for us right now as we embrace our identify as a clinical-stage company.
Cade Hildreth: That’s a very exciting advance. What other applications is Biostage exploring?
Jim McGorry: At a broader level, the other application that we are extremely happy to be advancing is a congenital disorder in children referred to as pediatric tracheal atresia. One out of every two or every three thousand children is born with a gap in their esophagus, where the upper and lower parts do not connect. In a partnership with Connecticut Children’s Hospital, we are showing in animal models that the regenerative portion is growing both in length and in circumference.
The promise of our platform technology is quite broad, so we find ourselves with this emerging technology that can solve real unmet medical needs for underserved patient populations.
Cade Hildreth: I know that you have a research partnership with Mayo Clinic. Could you speak to importance of that partnership?
Jim McGorry: Yes, thank you. The Mayo Clinic is one of top regenerative medicine institutions in the world. When you are in Rochester, Minnesota, at the Mayo Clinic, you feel the strength of a major institution. With esphogeal atresia, as that child grows, so can the regenerative portion. Whereas, where you put synthetic material in them, a pediatric patient can grow out of that, and then you have to do additional surgeries, which is what occurs for patients with heart congenital disorders. That is why a regenerative approach to pediatric conditions is exciting. Our partnership with Mayo is with the Chief of Thoracic Surgery, Dr. Dennis Weigel.
Dr. Dennis Weigel, as Mayo’s Chief of Thoracic Surgery, is very interested in the work that we are doing, so we are in the process of coming out with a major manuscript on some of our earlier preclinical animal data. We are also focused on the bronchus and trying to build our technology into a platform. For example, when you think about the lungs, there is the carina, where the trachea splits and goes into your lungs. Unfortunately, there are 150,000 new cases of lung cancer a year, but 40,000 are just in the bronchus. If you have to remove that tumor, there is no way.
Right now, there approach is to connect the lungs. They take the tumor out and now the patient loses 50% of their respiratory capacity. We are applying our technology working with the Mayo Clinic to be able to use our cell frame technology, cells on our bronchial scaffold, to do high-end plumbing. We want to be able to reconnect biological sites that have been removed.
We are also doing other work within the esophagus and the trachea, so Mayo Clinic is a powerful partner. Connecticut Children’s is another important partner, obviously validated by Professor Surgeon Dr. Bechanti, and you’ll be seeing several more collaborations from us on the pediatric side. We will also be working with alongside other top clinicians and researchers from around the country and the world.
Cade Hildreth: What business divisions does Biostage have?
Jim McGorry: Biostage is a small firm into Massachusetts, but a lot of regenerative medicine has started right here. We are tapping to that ecosystem. Currently, we have three parts to our company:
- High-end engineers that know how to build controllers and bioreactors
- A cell biology division, so we can tap into that giant emerging area of cell biology
- Materials science, which is how we make and deliver cells where they need to go
Within regenerative medicine, a key question is, “How do cells know where to go?” We believe that when cells on a scaffold are delivered right to where they need to be, the body knows how to handle things from there.
Cade Hildreth: You mentioned that at one year post-op you have animals that have survived. What is the approximate timeline for recovery and how do you expect that to translate into human patients in terms of hospital time?
Jim McGorry: Right now, if you understand the patient journey that follows after they have the “Standard of Care” procedure in which the surgeon pulls up the colon through the stomach, the quality of life for these patients is very poor. Most of them don’t have an esophageal gastric junction anymore, so unfortunately, they can’t sleep lying down for the rest of their lives. They have to sit up. They also have to eat in very small bites. There can also be gastric leaks and problems involved with the surgeries. It is a very difficult patient journey. As a result of our animal studies, we believe the quality of live will be much better for patients treated using our methods.
We have been closely involved with the FDA, with the pre-IND process, and filed our IND into Q3 2017. Most recently, we transplanted our very first esophageal implant into a patient, a 75-year old cancer patient. That was a major accomplishment, because it was the moment when we pivoted from a pre-clinical into a clinical-stage company.
We are also talking to companies in Japan right now about how our technology can be used over there, and we are talking to other children’s hospitals to advance our pediatric applications. We are excited to continue working with the Mayo Clinic, as well as Connecticut Children’s. We will be advancing those efforts and adding to our advisory and collaborations, because the mission of our company is to address life-threatening areas where patients have limited therapeutic options. We believe our technology has the potential to dramatically change the way these patients are treated.
Cade Hildreth: Congratulations on treating your first patient. What are the next steps for this clinical trial?
Jim McGorry: We hve had very favorable conversations with the FDA. Our initial pilot study will involve six-patients. With our approach, patient progress is easy to measure, because they start with a surgical gap. Within the pilot study, a surgeon is doing an esophagectomy on the patients. After 3 weeks, when the scaffold is removed after, the outcome is very clear, because there has either been cellular growth or a gap remains. We are hopeful that our technology will be a medical breakthrough. If we are successful with the pivotal trial, next we plan to go into a Phase II pivotal registration study and hope to have an efficient path to market. Of course, all of those things will have to be negotiated with the FDA and we will have to see if we can qualify to be fast tracked or receive expedited reviews, but right now we are doing all the work necessary to be able to build out our platform.
Cade Hildreth: You mentioned you’re in touch in companies in Japan. Was this inspired by their accelerated regulatory framework?
Jim McGorry: Yes, but the greater reason is that the Japanese and Asian population has 10-times the incidence of esophageal cancer as compared to the United States. Within the Asian population, there are lower rates of colorectal cancer, but much higher rates of stomach and esophageal cancer. While the reasons are not fully known, esophageal cancer occurs at rates that are 10-times that of the United States.
As you said, the Japanese also have a very favorable regulatory framework for regenerative medicine, so we want to leverage that with the right partner. As a result of my experience with Genzyme, I have a strong background in collaborations, partnerships, and global strategy. You grow a company by working with the right people, which is what we are now pursuing.
Cade Hildreth: What are your five-years goals for Biostage?
Jim McGorry: First and foremost, our goal is validate technology in human patients. That’s the biggest goal, to successfully grow as a clinical-stage company. It was a major accomplishedto transplant our first patient with the Cellspan Esophageal Implant in August.
Our next goal is to partner and collaborate with the right geographies and people. We will continue to pursue pediatrics and congenital abnormalities, so that the treatment modality will adjust to meet the needs of a growing patient. While we are prioritizing the esophagus, we are looking at the bronchial piece next. We plan to work closely with the FDA and international agencies to be able to get expedited and efficient clinical pathways. We hope to change the trajectory of patient lives.
Cade Hildreth: What role has Dr. Saverio LaFrancesca had at Biostage?
Jim McGorry: Dr. Saverio LaFrancesca is absolutely remarkable as surgeon and researcher. He is a heart surgeon. He has done over a thousand heart surgeries and two hundred lung transplants. He trained under the great Denton Cooley at Texas Heart Institute. He has a prominent surgical practice, because he believes in bringing innovation to patients. He is now our President and Chief Medical Officer. We promoted Severio in early 2017, because I wanted to show our total commitment towards clinical development.
Saverio is a remarkable person and is our lead doing the scientific and surgical heavy lifting to bring our products into human patients. His relationships with the Mayo Clinic and Dr. Weigel and and other key surgeons and researchers around the world has been essential to our progress.
Cade Hildreth: How did Dr. LaFrancesca become involved with Biostage?
Jim McGorry: You know, like the hair commercial: He was a customer and now he is the President. All joking aside, he was using the company’s bioreactors, and he knew very well all the things that Harvard Bioscience was doing. He saw that we were looking for the Chief Medical Officer and joined us three years ago. It was through his leadership that we have totally advanced our technology and now find ourselves a clinical stage company. Our predecessor was founded almost a hundred years ago, but the company bought Biostage at the start of 2014 and got listed on NASDAQ at the start of 2014. It’s just over 3 years old on NASDAQ right now.
Cade Hildreth: Final and important question. How can people learn more about Biostage or get in touch with you?
Jim McGorry: Go to Biostage.com. One the site, we include updates about our technology platform, our patients, and the journey they have to go through. We have a video about our Cellframe technology that explains what we are doing and have a manuscript published in collaboration with the Mayo Clinic, as well as news about our company and recent clinical advances.