Elektor at H.C. Wainwright Conference: Promising Advances in Neurodegenerative Research

On Tuesday, 20 May 2025, Elektor Inc. (NASDAQ:ALEC) presented at the H.C. Wainwright 3rd Annual BioConnect Investor Conference 2025, highlighting its strategic progress in treating neurodegenerative diseases. The conference, led by CFO Mark Ross, underscored Elektor’s promising clinical programs and financial stability, while also addressing past challenges and future prospects.

Key Takeaways

• Elektor’s Phase 3 study for Frontotemporal Dementia (FTD) with a GRN mutation, in partnership with GSK, could be a significant milestone, with a potential $2.2 billion deal.
• The company holds $354 million in cash, ensuring operations into the second half of 2027.
• Elektor’s innovative Elektra Brain Carrier (EBC) technology aims to enhance therapeutic delivery to the brain.
• The FDA’s breakthrough designation for the FTD program highlights regulatory support.
• Data from Elektor’s Alzheimer’s program is expected by the end of 2026.

Financial Results

• Elektor reported $354 million in cash reserves as of the end of Q1 2025.
• The financial runway is projected to extend into the second half of 2027.

Operational Updates

FTD Program:

• The Phase 3 study is fully enrolled with 103 symptomatic and 16 at-risk patients.
• The FDA has granted breakthrough designation, with results anticipated in Q4 2025.

Alzheimer’s Program:

• The Phase 2 study is fully enrolled, with a 76-week treatment period.
• Data is expected towards the end of 2026.

Elektra Brain Carrier (EBC):

• EBC technology is designed to improve the brain penetration of various therapeutics.
• It is customizable for different molecules, including antibodies and enzymes.

Future Outlook

FTD Program:

• Elektor plans to file for full approval based on Phase 3 data.
• A 25% to 40% slowing of disease progression is considered statistically significant.

Alzheimer’s Program:

• Results from the Phase 2 AL-101 study are expected by the end of 2026.

Q&A Highlights

• The FDA supports the use of biomarkers like progranulin in filings.
• There have been no significant changes in the FDA’s review process for Elektor’s programs.

In conclusion, Elektor’s presentation at the H.C. Wainwright Conference showcased its strategic advancements and financial health, positioning it as a key player in the neurodegenerative disease space. For further details, refer to the full transcript below.

Full transcript - H.C. Wainwright 3rd Annual BioConnect Investor Conference 2025:

Andrew Fine, Biotechnology Analyst, HC Wainwright: Hi. Good afternoon, ever 12:00, so good afternoon, everyone. I’m Andrew Fine. I’m one of the biotechnology analysts at HC Wainwright. So it’s my pleasure to introduce the next company, speaking today, which is Elektor.

And here from the company is its chief financial officer, Mark Ross. Thank you.

Mark Ross, Chief Financial Officer, Elektor: Thanks for having us, Andrew. Great to be here.

Andrew Fine, Biotechnology Analyst, HC Wainwright: So maybe the best place to start is just, you know, not sure how familiar everyone in the room might be. Just kind of getting everyone up to speed, and then we’ll, we’ll dive into specific questions from there maybe.

Mark Ross, Chief Financial Officer, Elektor: Yeah. Happy to do it. So, Elektor is a company that’s pioneering disease modifying treatments for unmet needs in neurodegenerative disease. And the company is in an advanced stage from a development perspective. We have a fully enrolled phase three study that’s reading out in q four of this year in a very unmet need in the setting of frontotemporal dementia.

And these are patients that have a GRN mutation, patients that have half the normal levels of an important protein called progranulin. And with our antibody therapy, we’re restoring the levels back to normal. And this is a program that is partnered with GSK in a large partnership that had a $700,000,000 upfront associated with it. There’s a billion and a half in in development and commercial milestones. And we also have a second progranulin elevating antibody that’s part of this partnership that’s in a fully enrolled phase two study in early Alzheimer’s disease.

The progranulin biology is a very important biology in in evolution. And, again, we’re restoring levels back to normal in the FTD GRN population. And in the Alzheimer’s setting, we’re elevating progranular levels in an effort to slow or reduce the the disease. We also have a number of electro brain carrier programs. This is using our proprietary brain penetrant technology, including a program around a beta and a program around the GCase enzyme.

The the a beta program, for those that are following the field, this is an exciting development in Alzheimer’s disease where we’ve seen, for the first time, slowing of disease progression with A beta therapeutics, although the naked antibodies do have some challenges, both from a side effect perspective as well as just from an uptake perspective, from a patient perspective. And we’re seeking to improve on those by having potential best in class plaque removal, but with the brain shuttle also allowing lower dosing, potential subcutaneous dosing, and improving the side effect profile, and potentially significantly reducing ARIA, having a better side effect profile perhaps relative to anemia, and, and, improving the overall, disease modifying, potential. The GCAES program, which we’re pursuing, and this is a subset of Parkinson’s disease that have GBA mutations. And also, we’re interested in in Gaucher’s disease as well as an engineered enzyme that is, again, brain penetrant using our electro brain carrier technology. The current approaches for Gaucher’s, you can administer the enzyme peripherally, but you’re not getting good brain uptake.

And many of these patients have neurologic involvement. And the GBA mutant patients in Parkinson’s disease represent a significant percentage of Parkinson’s. And again, having a brain penetrant enzyme could be a substantial change to the to the care paradigm.

Andrew Fine, Biotechnology Analyst, HC Wainwright: Great. Maybe just going back to the FTD program, maybe you can, you know, kind of set the stage for people in terms of expectations, you know, importance of for granular biology and, you know, how they should be thinking about interpreting the results when they come out?

Mark Ross, Chief Financial Officer, Elektor: Yeah. Abs absolutely. So so maybe first, just, you know, setting the stage in terms of how this antibody works and and some of the phase two data and and then maybe a bit on the the phase three. So the way our antibody works to elevate progranulin is by blocking a degradation pathway and inhibiting something called sartilin, which is a cell surface receptor receptor that is one of the pathways for degrading progranulin and regulating progranulin. So by our antibody binding SORTYLIN, we’re able to show, and we’ve shown this in the phase two in patients, as well as earlier studies, that we can restore the progranule levels back to normal.

And also in the phase two, in addition to showing restoration of progranular levels, we see encouraging changes on other biomarkers. We show reduction of glial fibrillary acidic protein back to net levels seen in presymptomatic patients. We’ve seen improvements in volumetric MRI as measuring brain atrophy or ventricular size. We’ve seen also other biomarkers, including stabilization of Nf L. And, we showed approximately fifty percent slowing of, disease progression as measured by, clinical endpoint, CDR, plus NAC FTLD, some of the boxes, which is basically an adapted CDR, of the boxes in the setting of frontotemporal dementia with two domains, behavior and speech.

And that slowing of approximately fifty percent of disease progression is really a significant finding, in our view, from a clinical standpoint, and is something we’re seeking to show as well in the phase three. The phase three is a much larger study than the phase two. The phase two we had 12 symptomatic patients with FTD GRN. In the phase three we’ve enrolled 103 symptomatic patients with FTD GRN. We also have 16 at risk patients as well that are part of that study.

This is a ninety six week treatment, so it’s twice as long as the phase two. It’s a it’s a study that’s measuring the same endpoint, the CDR plus NAC, FTLD, some of the boxes as the primary. But we also have a number of very important secondary endpoints, clinical functional endpoints, as well as biomarker measurements, including progranulin, GFAB, as mentioned, Nf L, volumetric MRI. And this study, as mentioned, will read out in Q4 of this year. I should also note that the FDA has granted breakthrough designation around this program, and that was based on their review of the phase two data and data we have to date.

So we view this as a very robust study from the standpoint of evaluating this approach therapy in this significant unmet need. And it should be the basis for, if we’re successful with this study, engaging with the FDA around approval.

Andrew Fine, Biotechnology Analyst, HC Wainwright: May maybe in the context of the FDA, maybe you can speak further to the the implications for their their their their feedback to you previously that biomarkers would be accepted and and and considered in the interpretation of the the data and why that’s important and kind of your ongoing dialogue with with the agency ahead of the results.

Mark Ross, Chief Financial Officer, Elektor: Yeah. Yeah. So we we have had an ongoing dialogue, you know, together with with our partner GSK and and and the FDA. It’s been constructive as as mentioned, the breakthrough designation. It’s helped, and, you know, we’ve taken advantage of that in terms of more regular interactions with the FDA.

In mid of last year, we we we had an engagement with the FDA where we were able to confirm, you know, their, you know, support that the biomarkers, particularly, you know, progranulin and other measures, could be supportive evidence in the context of potential filings. We view that as encouraging. You know, our base plan is to file for complete approval with the overall data set. And again, that’s using the the primary endpoint of CDR plus NAC FTLD, some of the boxes within the symptomatic patient population. And we reached agreement with the FDA that primary analysis would be on the symptomatic population.

We’re well powered for that endpoint with 103 symptomatic patients. If we’re able to see a slowing of disease progression of between, you know, twenty five and forty percent, I think we’re well powered for that being statistically significant. If for whatever reason, you know, we don’t see, you know, statistical significance on the on the primary endpoint. We also think there’s a potential strategy with the totality of the data to go back, including, around, some of the biomarkers. There’s there’s no approved therapy, for this disease.

No disease modifying therapies available. It’s a significant unmet need, and, you know, we think, there’s, there’s, potentials, for different avenues to go back.

Andrew Fine, Biotechnology Analyst, HC Wainwright: Okay. That’s helpful. And maybe, you know, it’s it it comes up so much, I guess. Since you’re in the in the throes of dialogue with with the agency on an on an ongoing basis, have have has anything changed? I mean, as as the kind of consistency and level of interaction remain constant, you know, or people I what’s your what’s your take on?

Mark Ross, Chief Financial Officer, Elektor: Yeah. I think just given the current environments, that’s that’s an understandable question. It’s it’s important to note that we’re we’re on the Cedar side, not not the CBR side. There there hasn’t been any significant change that we’ve seen at this point, which is encouraging. Again, given the unmet need, you know, given, you know, the the potential for disease modifying therapy in in this, you know, severe setting, you know, we we think we’re in along with the breakthrough designation, you know, in a in a reasonable place with this robust study.

There there hasn’t been anything that that we’ve seen so far that would suggest that there would be a delay or a change in in the the review process.

Andrew Fine, Biotechnology Analyst, HC Wainwright: That’s very helpful. Maybe we can speak more about the progranulin program now. You know, the company’s done a lot of work, but, you know, speak about, you know, why it’s an important target for for for AD and the the implications.

Mark Ross, Chief Financial Officer, Elektor: Yeah. So as mentioned in the setting of FTD GRN, you have half the normal levels of progranulin if you have one good copy and one bad copy of the gene. And that’s the primary for the phase three ladazinamab program. We also have a second pogranulin elevating antibody, AL-one hundred one. And this is in a phase two study that’s fully enrolled for early Alzheimer’s disease.

And what’s known in genetic data is that if you have partial loss of function of progranulin, you are at higher risk for developing Alzheimer’s Alzheimer’s disease. And in addition, it’s shown that you’re, if you’re able to elevate progranulin in animal models, it can protect against Alzheimer’s disease or, or reduce the severity of Alzheimer’s disease. So that’s the basis of the effort for AL101. And the phase two study is now fully enrolled. It’s a seventy six week study at at different at two different dose levels versus placebo.

And it’s a primary endpoint of CDR, some of the boxes, which is, you know, the standard in this area. And there’s also a a robust set of additional clinical and functional measures and other biomarkers, including amyloid PET and other outcome measures. So from the standpoint of determining if progranular elevation can have a benefit in Alzheimer’s disease, we think this is a robust study. So that’s fully enrolled in April of this year. It’s a seventy six week treatment period.

So, you know, we’re hoping that we can have data, you know, towards the end of twenty twenty six for this program.

Andrew Fine, Biotechnology Analyst, HC Wainwright: And and how is the progranulin program, the study, different from the the TREM two trial zone?

Mark Ross, Chief Financial Officer, Elektor: Yeah. Yeah. So TREM two biology is a very different biology. We, with with progranulin, are, again, increasing the levels of of a protein that can be potentially protective in the context of neurodegenerative disease. TREM2 biology is more the activity of the microglia in the context of pathology such as Alzheimer’s disease.

So very different biology between the two. And, you know, for for we’re granule, and again, we’re we’re basing this on on the strong genetic evidence as well as the the available earlier animal data in this in this context.

Andrew Fine, Biotechnology Analyst, HC Wainwright: Were there lessons, I guess, learned from the the TREM two trial experience in terms of patient selection or or endpoint selection that are now influencing the progranulin program?

Mark Ross, Chief Financial Officer, Elektor: Yeah. I I think from TREMP two, you know, the the there’s a lot still to be understood as it relates to the the biology and how best to impact disease. And we continue to review the data and assess. At the moment, it’s not a priority from a development perspective for us. As it relates to the phase two in early Alzheimer’s disease, in some respects, it’s a similar patient population.

It’s a similar set of endpoints in terms of the phase two that we also pursued for TREMP two. We think that was a very well run study, and it it it answered the question. It it’s not a it’s substantially different patient study from a design perspective or endpoint perspective.

Andrew Fine, Biotechnology Analyst, HC Wainwright: Maybe you can speak about the the ABC platform and, you know, kind of differentiate it in the context of the landscape.

Mark Ross, Chief Financial Officer, Elektor: Yeah. Yeah. We’re very excited about Elektra Brain Carrier, which is our proprietary brain penetrant technology, and we’re applying this to a number of different programs. It’s applicable to antibodies, such as our a beta program. Also applicable to other proteins and enzymes, including our g case engineered program, enzyme replacement program.

And it can also be applied to nucleic acids, just such as siRNAs. And we have a a tau siRNA program. Some of the unique aspects of electro brain carrier are are tunability and versatility in terms of how we’re applying this. One of the key, factors we see is, the, binding affinity, including to the shuttle technology that’s being employed. So a common shuttle is the transferrin receptor, and that’s, you know, used by, you know, the red blood cells to get iron into the brain and, you know, to get iron across the the blood brain barrier and the the endothelial barrier.

So we’re basically using that as others have to get brain penetrance. However, the binding to that transferrin receptor is something that we can really carefully modify in a way to maximize the therapeutic benefit while minimizing safety concerns, such as anemia. So that’s one aspect. Another aspect is how we’re able to customize the epitope as it relates to the efficacy piece, and also how we’re able to customize the effector portion of the construct. So for example, the Fc portion of an antibody and how that’s potentially either increasing immune activity in certain settings where you want to increase, such as in the context of a beta, where you want the immune system to come in and clear the plaque, or in other settings where perhaps you don’t want an immune effect or function at all because you’re trying to minimize any safety concerns.

So versatility around these different pieces is a differentiator for for us.

Andrew Fine, Biotechnology Analyst, HC Wainwright: In the last minute we have, you know, there’s so such a lengthy and deep level of science has gone into the company. Obviously, the, you know, couple setbacks here and there, the macro environment’s been been been challenging, so the valuation doesn’t really reflect any of that. Maybe you can just kind of set the stage in terms of you know, it’s it’s easy sometimes for investors to view things as black and white. But in totality, validation, you know, is is more than just a zero sum game. Right?

So as you as you get closer to data, you know, later this year, you know, you speak about the implications. You know? Speak about the the validation of of the the breadth of work that the company’s done. And there are, you know, there are there are ramifications beyond that.

Mark Ross, Chief Financial Officer, Elektor: Yeah. Absolutely. So, you know, we do believe we’re in a strong position. So we have, you know, 354,000,000 in cash as as of the end of the first quarter, and that’s runway, recently extended into the second half of twenty twenty seven. So that’s a full year beyond

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