Welcome back, everyone. We have an update from Clene, which trades on the Nasdaq under the symbol CLNN. It's a late clinical stage biopharmaceutical company focused on improving mitochondrial health and protecting neuronal function to treat neurodegenerative diseases. Happy to welcome back President and CEO Rob Etherington. Rob, nice to see you again. Please remind our conference viewers what your drug is and what therapeutic areas you're currently targeting.
Thanks, Anna. Pleasure to be here. My drug is CNM-Au8. I'm holding it here in my hands. People with neurodegenerative disease, which include amyotrophic lateral sclerosis, you know, the whole ice bucket folks, formerly called Lou Gehrig's, but we call it ALS, is our key focus. The same asset, same dose, has also been explored in a multiple sclerosis study. We presented late-stage data from that program showing remyelination and neuron protection at the American Academy of Neurology late-break session in San Diego a month ago.
Wonderful. The last time you spoke to our audience, you discussed the regulatory pathway and some upcoming meetings with the FDA in regards to the ALS indications. Give us an update with that status.
All of us who follow the FDA have been watching a number of headlines. The agency, of course, is in the middle of some changes, is an understatement, I should say. Nonetheless, we have a meeting planned that we're expecting later this quarter. That's now late May. You can do the math and figure out we're meeting them in June to discuss our statistical analysis path for our neurofilament data. This requires a little bit of understanding. Neurofilament light is a biomarker that can be measured in the blood, and it's indicative of a problem. In this case, a disintegrating neuron. Let me just give a metaphor, because in the news is President Joe Biden's prostate cancer. We wish him well. What was also disclosed in the headlines in the last 24 hours is that he had not done a recent PSA.
A prostate-specific antigen is an example of a prostate going awry, and it's a blood test. If I have neurodegenerative disease, a blood test that tells me that my neuron is being compromised, attacked, and leaving debris, a skeletal remains of its destruction in the blood is called neurofilament light. The reason why this is relevant is because there is another drug in ALS for a very small subset of ALS patients that was approved on a neurofilament change alone. Clene, presently, has a $45 million funded, bless them, from the National Institutes of Health. They funded a $45 million compassionate use program called an expanded access protocol. That's its proper vernacular. This has enabled 170 participants to come on our drug funded by this NIH work. These are people that are very ill.
They have not been able to get concluded, not been able to get enrolled, rather, into other ALS clinical programs. By consequence, they are looking for every option. We have been able to fund that program with this capital from the National Institutes of Health. What is being assessed there is neurofilament levels. What the FDA said is evaluate that program and let's see if from these many participants, 170, which is about 3x the number we had from the Harvard Healy program, where we already showed them a neurofilament benefit that was statistically significant in a pre-specified way, let's see if this EAP is concordant, if this data syncs together, shows more evidence of that neurofilament benefit. That data comes to Clene in the summer.
This meeting with the FDA is to basically show them our analysis plan and then get that data, verify it's concordant, aligned, supportive, the same as what we already showed them, and then to file the potential for filing a new drug application for the possibility of accelerated approval. By the way, this is all independent from the survival data that we have been showing, and we just did show new data this spring. We have a robust conversation that we'll be having with the FDA coming up. That's the net.
Wonderful. Great news on that front. Assuming you're successful in your clinical trials with the NIH-sponsored EAP, when do you plan on filing an NDA? Will that be under the accelerated approval pathway?
At the moment, we believe that that's the right pathway, accelerated approval. The survival data may change the discussion. Hard to know. We don't know yet. To answer your question on timing, the plan is to file that in the fourth quarter this year. In other words, this could lead, if this data is positive and if the FDA is aligned, to the consideration of an advisory committee for the possibility of approving CNM-Au8 for ALS sometime in 2026.
Wonderful. You also recently presented some interesting multiple sclerosis data at the American Academy of Neurology. Can you share some of those results?
I can. We presented at the late-break session, excuse me, data that showed the CNM-Au8 in our Visionary MS study. And just to give your viewers a little insight to what that is, we enrolled stable, relapsing-remitting multiple sclerosis participants that had chronic optic neuropathy. So lesions, MS lesions in their optical tract had compromised their vision or had compromised their cognition. They had what's called an MS cognitive fog. This is a huge issue. This remains actually one of the leading medical needs in MS patients, is that we've stopped the attack of the MS, but the earlier attacks have caused functional compromise, either cognitively or visually.
What we showed in that late break session is that the long-term experience, numbering over three years of follow-up, showed that the functional changes of vision improvement and cognition improvement that we'd already shown was nicely correlated with what are called periclinical biomarkers, namely MRI and another assessment of the visual field of the optic nerve that is into the visual cortex. What we showed is that we were protecting neurons, and we showed data that was indicative of remyelination. This is a Holy Grail medical need in MS to improve cognition or vision through remyelinating a damaged neuron. Again, this is the same dose, same drug that patients were taking as they are doing so in the ALS program. What we have some opportunity here is a little bit of a pipeline and a product.
Now, most investors are more focused on ALS because ALS is a quicker regulatory path in principle. The MS disease would require us to conclude a phase III program, which still needs to commence. We are some years away before we could commercialize an MS. Both data are very interesting, including two potential strategic partners.
With that said, with your MS program moving forward, are you planning to meet with the FDA in an end-of-phase two meeting? And do you anticipate initiating a phase III?
That meeting will be in the end of the year. We've now got everything we need for that meeting, and we have submitted for the agency to organize that meeting. That meeting, as I say, will be in the second half of the year.
Shay asks, the ALS and MS, were these studies similar in nature? Can you talk a little bit about the size and duration of these studies?
Very different, actually. They both were neurodegenerative disease because ALS and MS is neurodegenerative disease. But the ALS study, one was a six-month study, one was a nine-month study. Shorter because an individual that's been diagnosed with ALS and then is given a sentence of mortality or death, two to five years, ALS patients appropriately don't want to go into a long placebo-controlled study. They want to get as much help as they can, as fast as they can. The MS is different. The MS study was a 12-month study. So six months, nine months for ALS, 12 months for MS, excuse me. And the endpoints were different. In the case of ALS, we're really looking for a global functional scale, as well as whether or not patients are living longer and whether or not they clinically worsen.
In the case of MS, as I said, we're looking for visual change or cognition change.
Amy asks about what are the current approved alternatives. She understands they're very old. What's the largest benefit to your new drug?
Lou Gehrig was diagnosed in the 1930s, and he famously lent his name to the disease. We have corrected that and now call it ALS all the time. It took 60 years for the very first drug to be approved. That drug is really Riluzole. It was approved in 1995 by the US FDA. Subsequently, it was approved in pretty much every other regulatory key market worldwide. This is the tragedy. Today, that is the main drug that I would be prescribed. There is another drug that has been developed in Japan that is available in this country for some individuals and is available in a very small set of countries worldwide called Radicava or Edaravone. It is not often prescribed anymore, unfortunately. That is it. That is it. So really just really Riluzole, a generic drug now for 30 years.
Really, Riluzole, everybody's on it because it gives a few months of extra survival. Other than that, relatively little clinical benefit. What our drug is doing differently is as the neuron is attacked, it loses its energy in order to fight back. Our drug enables the bioenergetic metabolite support that the neuron needs to basically do its function, its daily housekeeping. The way that you and I move and walk and talk and eat and chew and breathe, that requires a lot of energy. It is a great choreography of muscle movement that we do not even think about. In an ALS patient, they are losing this. Our drug is enabling that to improve with the goal being to keep them alive longer is our prayer.
Wonderful. Last question for you. You can close on this. What is your perspective on the shareholder opportunity investing in Clene? Are there any comparables as to what Clene can become?
One good example is there is another ALS company that unfortunately now had to withdraw their market because their confirmatory phase III study was not, in fact, confirmatory in any way. They were kind of trading in the market doldrums at the beginning of the stage as they were a young private company working to get clinical benefit. They got FDA approval, and they quickly were on track to sell $400 million of their drug in year one. That took them to a $2 billion market cap. It is extraordinarily different than our small market cap at the minute as we still wait ourselves for FDA's opinion. You can certainly sense we do believe that we could sell $400 million in year one as well if we were approved. That would be obviously a completely different market cap than our present number.
Rob, fascinating, such important work you're doing. Thank you for this update. We look forward to hearing the positive results the next time you're on our conference.
Thank you, Anna.