From The Editor | March 14, 2018

The Challenge Of Scaling From 20 Sites To 200

Ed Miseta

By Ed Miseta, Chief Editor, Clinical Leader
Follow Me On Twitter @EdClinical

The Challenge of Scaling From 20 Sites To 200

Immuron is an Australian biotech focused on oral immunotherapy. The company’s pipeline currently includes treatments for ASH, Diabetes, C-Diff, Colitis, NASH, and UPEC. The company recently completed a Phase 2 trial on IMM-124E, it’s product that is expected to help patients with NASH.

Dan Peres, SVP and head of medical for Immuron, recently spoke to me about the challenges of performing NASH trials and preparing to scale-up to a Phase 3 trial.

Ed Miseta: How intimidating is the challenge of scaling up from a Phase 2 trial to a Phase 3 trial?

Dan Peres: Scaling always seems to be the biggest challenge. In the case of our NASH trial, the scale-up will be very critical. Just to give you an idea of what we are dealing with, Immuron is a relatively small company, but we prefer to run everything. Our recently completed Phase 2 study worked with more than 20 sites in the U.S., Israel, and Australia.

The upcoming Phase 3 study will likely involve more than 200 sites and also expand into South America and Western Europe. Obviously the scale-up is going to be immense. We are looking forward to that challenge, but also realize we can’t do it alone. We will attempt to tackle that challenge with partners.

Miseta: What will you look for in a potential partner?

Peres: We have not yet landed on that partner but expect to collaborate with bigger CROs that have experience in this space. There are three CROs that have accumulated a large amount of knowledge in this space by running the larger Phase 3 trials that are currently underway. We believe that is the knowledge a company like ours needs to utilize and we are happy to do so.

Miseta: You mentioned CROs. Do you expect to select one CRO or multiple partners?

Peres: That hasn’t been decided yet. I think it will depend a lot on the location of our clinical sites. But, we do know who the leading CROs are in this space and that is where we will place our focus. For example, Covance and PPD are companies that have done a wonderful job in this space. We want someone with that type of experience because we plan to study NASH for at least the next 5 years in large-scale studies.

We know who the players are, but we will still have to decide whether to go with one or mix a few together. That is certainly a possibility. On one hand, a large international CRO would allow us a more centralized approach to managing such a large study. On the other hand, a smaller and more local CRO could have a geographical advantage. Small CROs can also be better at managing relationships. At the end of the day, we may opt to go with a combination of both if we feel that will be the most effective approach. That model could allow a centralized CRO to manage the local CROs. We used a mixed approach in our Phase 2 trial, which was partly because we are a small company and were used to doing things ourselves. For that study it seemed better to let small CROs run the study for us.

Miseta: Will protocol design be a challenge for you?

Peres: I don’t think so. The study protocol should not be much of an issue because it’s already been defined by the FDA. We know what the endpoints are, so the big issues for us will be around the scale-up process. We ran the Phase 2 study as a small company, but moving forward will rely more heavily on relationships with our partners.

Miseta: Patient recruitment will be a challenge for you, and you will be reaching out to sites to help with that effort. Do you get personally involved with that?

Peres: I do, and it’s something that I enjoy. I spend a lot of time visiting sites, and I would guess about 40 percent of my time during the Phase 2 study was spent doing that. I’m a great believer in the fact that good relationships help drive the recruitment effort. It takes a lot of effort to develop those relationships and to pay close attention to every detail that goes into the clinical site.

Moving forward, that will not be feasible with the same frequency I’ve done in our smaller studies, but I will still have to maintain some sort of close, ongoing relationship with all of our best performing sites. Learning how to implement our methodology of finding patients in the larger scale will be a learning experience for us.

Data will be a big concern for us as well. You also can’t forget that site personnel will be collecting the data you submit to regulators. Therefore, we are focused on what we can do to get the highest quality data from those sites. If you get good data you can shorten the study, which will save us money and also allow us to get the treatment to patients faster. There is currently no treatment available for NASH, so speed is very important to us.

Miseta: Being a small company I assume you use a contract manufacturer as well. Will scaling up the manufacturing be a challenge as well?

Peres: That’s a good question. Right now I would say it is a bit of a guessing game here. I’m not sure that we’re going to use the same CMO now that we are moving to a much larger study. I can tell you we currently have a marketed compound based on the same technology. It is on the market in Canada and Australia. The product is called Travelan and is marketed as a dietary supplement. So in one sense, we have already scaled up and there will not be an issue with scaling production. The question is more around what we will need for this specific study. That has yet to be determined.