Russ: Hi, I’m Russ Capper and this is BusinessMakers USA, brought to you by Insperity, inspiring business performance. We’re in Indianapolis, Indiana, and my guest is Dr. Mark Kelley, Founder and Chief Scientific Officer with Apexian Pharmaceuticals. Mark, welcome to the show.

Mark: Thanks for having me.

Russ: You bet. Tell us about Apexian.

Mark: Well, Apexian was formed about 8 years ago based on some discoveries that were made in my laboratory at the IU School of Medicine here in Indianapolis. And they licensed it and got together with some local investors to form Apexian and basically to move what we discovered on the bench to help patients, which is what we’re about, a cancer drug discovery company.

Russ: Let’s get into it but before we do, go back to when you discovered it. I mean, when you saw it, when you saw the results did you light up and go, wow?

Mark: Maybe a slow light up because I’ve been working on this target for over 27 years, this particular target in the cancer cell. But then, yes, we have been studying this for a while and when we had some drugs we took forward to look at how they would affect our target, we realized this has potential to help patients, and that’s what we’re here for.

Russ: Give us a little bit more detail. As we all know, almost all of our lives have been touched by cancer. Everybody is always lighting up when they think, wow, maybe this is it. So, describe for us what you think you have found.

Mark: It’s a unique protein in the cancer cell that drives the cancer cell to grow. That’s what you want to stop to treat cancer. It also talks to a lot of other proteins in the cell, and so what’s unique about, and novel, is we’ve developed a drug, APX3330, that targets this protein and it stops it doing its multiple functions it does in the cell. And it’s very specific, it just hits this protein.

Russ: Ok. And so, in the discovery process you already knew about the protein and came up with the drug. That’s the way you start heading down the path of a solution?

Mark: That’s correct, yes. We had this drug, we started testing it, doing a lot of different models in the lab, and also through Apexian, we are fortunate we had a lot of great chemists on board, actually a lot of local talent and so we were able to make new compounds, too. So, we’re not just a one hit wonder with the one drug, we have a whole pipeline of drugs we’re developing.

Russ: And so, when you put together a staff of scientists and Ph.D.’s, it calls for money and you already mentioned you had some investors here. Are you continuously raising money when you’re doing something like this?

Mark: Yes. It’s privately owned and private investors, mainly angel investors initially, but we’ve been talking to others and we’re still talking to venture capitalists and others. We’re looking for money; we’re in the middle of our Phase I trial in cancer, which is a safety trial for our drugs. We’ve been able to get to that point which is a big step to get able to from the lab into human patients for the trial.

Russ: Sure. A lot of drugs never make it there, right?

Mark: Right, so we’re really happy about that.

Russ: What do you actually have to do to be able to get to that point?

Mark: You have to file what’s called an IND, and that’s a package of material showing that the drug is safe in the animals’ models. You have to show it has some effect of what you’re going after, and then you have to do a lot of safety and toxicity in the animals. You have to make the drug under a procedure where it’s sterile so that you can give it to patients. And all that costs a lot of money, and all of that is really outside the scope of academia, so that’s where Apexian came in.

Russ: Phase I is really in humans to also show that it’s safe?

Mark: Right. You still have to do that.

Russ: Is it possible in Phase I sometimes, I mean, do participants in your Phase I, do some or do all of them maybe have cancer right now?

Mark: In this Phase I they’re all cancer patients who unfortunately have failed standard treatments and so they’re taking our drug. Really, we can’t do this without them. I can’t say enough for the patients who do this. They’re taking our drug and we’re escalating the dose to see what dose we can use for a further trial, one in the future that looks for the effects of it, but this is to make sure it’s safe. The first thing you want to do is a drug that’s safe, you don’t want to do any harm.

Russ: Whether it’s safe or not it’s possible that it may help these people at the same time.

Mark: Yes. We’re still looking—we have biomarkers we look for, we do scans, and we have other things we’re looking for. Again, it’s really to make sure the drug is safe and to get the next dose, but absolutely we’re hoping it has some effect in these patients.

Russ: The best-case scenario, it’s safe and you see some positive impact.

Mark: Absolutely.

Russ: Let’s say we just made it through and it’s safe. The next step then is..?

Mark: In cancer the next step is called a Phase II trial, and that is for efficacy. That’s to show that the drug is now doing what you hope it to do. We’ve been focusing on colon and pancreatic cancer; colon cancer to be metastatic patients who have really failed a lot of the other trials and then they’ll be treated with our drug and other drugs, and same with pancreatic cancer. That would be a Phase II, to look for an effect.

Russ: And Phase II is when you compare it to a placebo?

Mark: Right. The other interesting thing about this, if I can say this, is this drug is very unique and the target, because not only is it affecting the tumor cells, the cancer, but it shows that by blocking this protein and neurons, neurons that control your—at the fingers, the tingle and your sense of—your walking and your feet, that a lot of patients get what’s called peripheral neuropathy and pain. A lot of patients have to come off their treatment when they’re treated with chemotherapy, and even the new, more targeted drugs, even immunotherapy, and our drug appears to prevent that, that neuropathy. So, it’s a win-win. We’re going after the tumor cells and we’re also protecting the neurons so the patients would, one, have a better quality of life, and two, they would be able to stay on their treatments longer to get the drugs that are helping them. So, we’re very excited.

Russ: So, it’s possible that you might have a questionable area in the efficacy of the drug, but just the fact that it cures this neuropathy, you might still take it to the market?

Mark: Absolutely. That’s the neat thing about the target and the drugs, that we really have a couple shots on goal, if you will, that we can look for both the tumor and that. And we have a bunch of different cancer indications we’re looking at.

Russ: Describe the people that work with you in this mission.

Mark: My CEO, Steve Carchedi—most of them have passed through Lilly at one time or another. He worked actually, at one time, with Home Pearce who led Lilly Oncology here. And Homer is head of our scientific board, so Homer is local. Steve has now worked with a number of big pharma and he’s now in Philadelphia but comes here all the time. I have a great CMO, Rich Messmann, as my Chief Medical Officer, is fabulous. He trained at the NCI and is really great for the trials. Roger Miller is the COO who really worked for years doing all of Lilly’s manufacturing, so he had the CMC work for the drug. And then I have Roberta, and Bill, who are our regulatory and toxicology. You look at this group and it’s a senior group of people, it’s not their first rodeo, and that’s really been phenomenal to work with because they know how to do everything. When we filed our anti-neuropathy indication with the FDA, we had approval in a few days, which is very rare. That’s because everything was buttoned up and just perfect. I can’t say enough about having a great team around you, you know it’s all team work.

Russ: I love missions like what you’re on, and particularly difficult missions that last multiple years and what it must be like in that endeavor. I’ve compared it to some of my more normal business offices that, you know, we ring the bell when we get a new customer, and it must be tough at times over there.

Mark: Yeah, it’s tough but I love science. Like I tell my students, you’re discovering things that have never been seen before, when you do stuff, so that’s the great thing. You get to work with really smart people, both my science—my lab at IU, and then also Apexian, I get to use a different part of my brain and I’ve learned so much about the whole thing. We kind of like to say at Apexian a lot, and I’m stealing a quote that we’re not doing things because they’re easy, we’re doing things because they’re hard. It’s no fun, the easy things. And you know, really at the end of the day it’s about the patients. It’s really about the patients, that’s why we’re here.

Russ: So, best case scenario, maybe it’s a not good idea to even project the future and what you do, but if everything turned out great, it was effective, and you got it and went to market and passed the other tests, what’s the soonest time it could possibly be?

Mark: Maybe 3-4 years depending on finances and, you know, some of it is it just takes time to run the trials. We have some other indications that we could even move a little faster, but that’s a good time frame.

Russ: Well Mark, I along with many other people watching sure wish you success in your endeavor, for sure. And thanks a lot for sharing your story with us.

Mark: Thank you very much and thanks for having me.

Russ: You bet.

Mark: And that wraps up my discussion with Dr. Mark Kelley, Founder and Chief Scientific Officer with Apexian Pharmaceuticals. And this is BusinessMakers USA.