Tegoprubart, formerly known as AT-1501, is the first ALS drug invented by a nonprofit to reach human clinical trials. This is one of the ALS Therapy Development Institute’s (ALS TDI) proudest accomplishments. In another milestone for the treatment, it recently successfully completed a Phase 2a clinical trial.

Due to the unique structure of ALS TDI, tegoprubart is making its way through clinical trials under the stewardship of another company – Eledon Pharmaceuticals. As the Drug Discovery Engine for ALS, ALS TDI’s mission is to work to continue to focus on feeding the ALS clinical pipeline. To this end, when a treatment invented in our lab, such as tegoprubart, is ready for clinical trials we will pass it on to a partner like Eledon. This allows us to continue the search for more potential treatments, rather than moving on to the expensive process of advancing a drug through multiple phases of trials.

Recently, Eledon Pharmaceuticals CEO Dr. David-Alexandre Gros and Chief Scientific Officer Dr. Steve Perrin joined ALS TDI for a virtual town hall discussion on tegoprubart. Dr. Perrin, who also formerly served as the CEO of ALS TDI, began with a presentation detailing the preclinical evidence for the efficacy of the drug produced by ALS TDI and followed with the results of the Phase 2a study. Dr. Perrin and Dr. Gros also took time after their presentation to answer questions from the community about tegoprubart, including several about the next steps for the drug. To see a full video of their presentation and Q+A session, you can watch a recording of the Town Hall here.

Below is a selection of edited highlights from the discussion portion of the town hall:


What are the current plans for moving tegoprubart forward?

Dr. Gros: We're focused on designing the next trial and at the same time finding how to fund the trial. We are working on both in parallel. We've been focused on ALS, obviously, since the founding of the company. This is core to our culture. We've been working with a lot of urgency to move the trial forward, as you've seen over the past two years, even with COVID. We'll continue to move with the same urgency toward both of those goals.

Right now, we’re just getting the data. We're looking to share and publish that data. There is a lot of work going on in parallel. But I'll reiterate what I started with. We understand the urgency, it's been the way we've behaved since forming the company, and we will continue to behave in that way.

Have you been able to determine if there is a subset of people with ALS that responds better to tegoprubart than others?

Dr. Perrin: At this time, I think that that's a hard one to answer. We know that about 60% of people have an inflammatory signature that you can measure, including a costimulatory signature. But at this point in time, we don't know what that relates to and if it's actually 100% of people. In autoimmune diseases like lupus nephritis, you often see people have overt flares, and at that point in time, you do see robust increases in inflammation. In ALS that might be the case as well, and we just don't know yet.

We do know, from those studies that have been published, that people with the sporadic form of the disease have inflammation just like people with the genetic forms of the disease. So, we don't feel that we have to stratify based on genetics, but I think we can't answer that question until we get into larger studies. We're not going to segregate people, for instance, that have some level of CD40L as part of enrollment, or at least we're not thinking along those lines.

Are there plans to have an Expanded Access Protocol, or to seek funding from the ACT for ALS, for the upcoming trial of tegoprubart?

Dr. Gros: When it comes to funding, we're looking everywhere we can. In terms of access and EAP, as well as broader access, we'll continue to work with regulatory authorities. That includes the FDA in the U.S., as well as the ALS community, to ensure that we're looking at all aspects of access.

Today, with the trial that we've just run, we do have to think about safety. And while we've shown some supportive data in terms of safety, we have not yet fully elucidated the risk/benefit of tegoprubart. We also have not finalized the regimen of tegoprubart. So, we'll look to do that in this in this upcoming trial. As we progress with the trial, we encourage people who think that they might be eligible to participate in the trial to do so. And by doing so, be able to access tegoprubart.

There are currently two potential ALS treatments currently being considered for approval by regulators, Amylyx’s Albrioza (AMX0035) and Biogen’s Tofersen. If either of these are approved, do you expect people in the next trial of tegoprubart will be able to take these treatments as well?

Dr. Perrin: Obviously, we're watching those drugs very carefully, and how the FDA and other regulatory agencies respond to them. We allowed people on the currently approved drugs, Edaravone and Rilutek to enter our phase 2a study, as long as they were on stable doses. As drugs get approved, we'll have to look and see and evaluate that as we talk to an advisory committee and then the agency about our next trial design. That’s going to be a key component of that discussion.

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