What does Nemucore do? ▼
We have partnered with some of the top AML clinics in the world: namely the H. Lee Moffitt Cancer Center and Research Institute in Tampa, FL and the Memorial Sloan Kettering Cancer Center in New York, NY. Together, we designed a Phase 1b/2 clinical trial protocol using NMI-900 and our companion diagnostic. Now, we’re raising funds to get this combination into clinical trials to help patients with AML.
Why do only some patients respond to the cancer drug you’re developing? ▼
At Nemucore, we’re working on identifying which drug works for those 70% of patients that don’t have an identifiable genetic mutation. Right now we have a potentially game-changing solution for patients suffering from Acute Myeloid Leukemia.
What’s the new approach you’re using? ▼
Our focus is finding an effective treatment for the 70% of patients that don’t have identifiable genetic mutations in their DNA. For our lead drug, NMI-900, we’ve identified RNA signatures that indicate if a patient’s cancer is sensitive to the drug. Just by measuring a specific group of RNA molecules in the cancer cell, we can tell whether NMI-900 could help a patient by destroying his or her cancer.
What is Nemucore’s history? ▼
We raised about $7 million through several National Cancer Institute and foundation grants, which helped us run until 2015. Our research was going well, but we couldn’t scale the company fast enough. We had an opportunity to look at a molecule that GlaxoSmithKline had created and sub-licensed to Cancer Research UK (CRUK), which is the largest cancer foundation in the world. We took a look at the molecule and found it had a lot of potential. Within four days, we had a nondisclosure agreement in place and we had access to all of the documentation on the drug.
Since then, we’ve been focused on creating the right clinical development approach to run successful clinical trials.
How has your Acute Myeloid Leukemia drug performed in trials in the past? ▼
Tell me about the trial you are conducting now. ▼
This new Phase 1b clinical trial was designed with prominent leaders in AML, specifically the folks at Moffitt Cancer Center in Tampa, FL, and at Memorial Sloan Kettering in New York, NY. It’s for patients with Acute Myeloid Leukemia (AML), and also high-risk Myelodysplastic Syndrome (MDS), which is a potential precursor to leukemia. In this trial, we will use both the companion diagnostic and NMI-900.
At the beginning, this trial will simply collect data about the companion diagnostic: we will test each patient’s tumor a few times throughout the trial while he or she receives NMI-900 once or twice a week. This data will help us retrospectively validate that the diagnostic does in fact predict who will benefit from NMI-900. Once we have treated about 40 patients with NMI-900 and analyzed the results of the diagnostic, we can use the data and work with the FDA to open an expansion trial, in which we will use the diagnostic first, then assign patients to receive NMI-900 if the diagnostic’s results predict it could help them.
Currently, we have physicians prepared to run the trial both at Moffitt Cancer Center, Massachusetts General Hospital, and Memorial Sloan Kettering Cancer Center.
How will you make money? ▼
How can you determine how well the drug is working? ▼
How well does Nemucore’s AML drug appear to work? ▼
With this particular patient population, the likelihood of five-year survival is about 27%. In these particular patients, because they haven’t responded to regular chemotherapy, they may die in six to eight months. So if the drug is working, they’re going to get better right in front of the doctor’s eyes. That’s the difference with this type of disease — the patients the doctors are treating are really sick when they enroll them in the trial, and they’re going to get better even before they can prove it on a biopsy or a blood sample. Not only will they feel better but they will likely live longer with fewer or no symptoms.
The patients in the CRUK trial did well on the drug by exhibiting a “manageable” safety profile. One of them, in particular, received 11 months worth of treatment. Her cancer stabilized. Any time a patient enrolls in enroll in a Phase 1 trial, he or she has a resistant cancer, or a cancer that has been heavily pretreated that no longer responds to available treatments. Not only do these patients have aggressive cancers, but their body has really suffered the side effects of multiple chemotherapeutics because they’ve often tried everything else. When you see patients do well in that setting, it’s very encouraging
How many people could potentially use this drug? ▼
Where do you see Nemucore going? ▼
What are you currently most worried about? ▼
Tell me more about your partnerships with the blood cancer experts. ▼
At the Moffitt Cancer Center in Tampa, we’re working with Drs. Alan List and David Sallman. Dr. List is the President and CEO of Moffitt. His specialty is hematological oncology. Earlier in his career, he was a pioneer in the development of a drug called Revlimid. Revlimid is a wonderful drug that helps patients with Myelodysplastic Syndrome or Multiple Myeloma. It also generates close to about $8.2 billion a year in revenue, and it’s the major product for the pharmaceutical company Celgene. Dr. Sallman is Hematologic Oncologist whose research interests focus on the development of targeted therapeutics for patients with AML and MDS.
These are not just the physicians you’d find at a community health center. At the American Society Hematological Malignancies conference, these doctors would be the rock stars walking down the red carpet. We think from a clinical and scientific perspective, we’ve got a super strong story
How much financing do you need to accomplish your goals? ▼
Who are your competitors? ▼
Just last year, there were two new drugs approved to treat AML. One of those approvals can only treat 10% of acute myeloid leukemia patients, and the other one is only good for about 20% of acute myeloid leukemia patients. In order to be a candidate to receive those two drugs, you have to have a targetable mutation. Whereas, with our drug, you don’t have to have that targetable mutation. With our companion diagnostic, we can get a pretty good indication, before administering it, whether or not it will work. So we’re fundamentally different than our competitors that were approved this year, which I think is an important differentiator.
How much will your drug cost? ▼
Typically what happens in the U.S. is, you get your FDA approval in a specific patient population, and you work with the Centers for Medicare and Medicaid Services to identify key aspects of the patient population that you’re going into and your expected price point.
In the UK, where there’s a single-payer system, they take into account the economics of the drug and the outcome of the drug trials. One of the great things about having had that first trial run in the UK is that we can have a more in-depth conversation about health economics with their National Institute for Council on Economics Health.
How did a small company like yours develop an innovative drug like this? ▼
As molecular biology got more sophisticated, we could really discern which patient had what type of profile and which patients were sensitive versus resistant. Big Pharma did a great job coming up with these molecules. But to move the needle for Big Pharma, they need that drug to make $500 million six or seven years after they initially discover it, and that wasn’t the case with drugs like ours.
So smaller biotech companies like Nemucore — who don’t have to worry so much about scale at the outset and who could find the six patients for whom this particular drug will work — can create a lot of value: patients can have better quality of life and insurers no longer have to pay for what is essentially end of life care. You can improve patients’ ability to survive disease and that end-stage life care will be less expensive.
We had some underlying scientific principles. We were able to build and develop on those, and take ideas from several different organizations and bring them together. We’ve become the ultimate integrators. That integration has really taken off with our clinical advisors. Could someone come and beat us today? Probably not. Could someone come in five or ten years with differentiated therapy in that similar vein? Yeah, that’s probably going to happen. But for now, I think we’ve been the ultimate keeper of this technology and have succeeded in figuring out how to deploy it and integrate it versus being the best chemist in the lab.
What’s your timeline? When do you expect your first drug to be approved? ▼
What kinds of feedback do you get from your investors? ▼
Who’s on your team? ▼
Our board is made up of people with pretty incredible backgrounds. There’s Dr. Seiden, who right now is the Chief Medical Officer of the McKesson Corporation and the President of U.S. Oncology; Brian Constantino, who took the healthcare advisory practice at PricewaterhouseCoopers and built it from a $100 million business to a $1.1 billion business when he left 15 years later; Jim Farinholt, who’s started a handful of biotech companies at this point after coming from an investment banking background; Doug Bailey, who’s been an entrepreneur since he left MIT in the early 1970s and helped build Endocyte Pharmaceuticals. It’s a pretty esteemed board and they’ve bought into the story, the science, the chemistry, the clinical aspirations of what our amazing little company can do.
On the frontline of our clinical program is our director of clinical affairs, Allison Morse, who I have a tremendous amount of respect for. Allison was an Oncology Nurse Practitioner for 20 years before coming to work for Nemucore a year ago. Her passion is getting the best for patients and the ability to bring new therapies to the patients, especially those who don’t have a lot of options.
Finally, we have about 57 investors in the company who mean the world to me and our team as they have been pioneers in our effort to bring our novel approach to the clinic. When we are successful, there will be a lot of patients grateful for the risk our investors took to bring them our life-saving approach.