Episode 69 | Kinevant Sciences is researching namilumab – a potential new drug for sarcoidosis

Kinevant Sciences is researching a potential new drug for sarcoidosis - namilumab – which inhibits one of the key proteins believed responsible for granuloma formation and persistence in sarcoidosis.

In Episode 69 of the FSR Sarc Fighter Podcast,  Kinevant CEO Bill Gerhart and Director of Patient Advocacy, Rayne Rodgers discuss the status of namilumab, and how you as a sarcoidosis patient can participate in the Phase 2 clinical trial, RESOLVE-Lung.

Show Notes

• www.kinevant.com
• www.sarcoidosistrial.com
• Click here for information on how to sign up for the clinical trial: https://bit.ly/3DaVsR6
• ClinicalTrials.gov listing for RESOLVE-Lung: https://clinicaltrials.gov/ct2/show/NCT05314517
• ClinicalTrials.gov listing for RESOLVE-Heart: https://clinicaltrials.gov/ct2/show/NCT05351554

Below is a transcript of the interview.  It is auto-generated.  Please excuse typos and misspellings.

John Carlin: Coming up on the Sarkfighter podcast, an exciting new potential treatment for Sarcoidosis.

Bill Gerhart: This particular protein is required for granuloma formation and persistence and that if we inhibit it, we can potentially make a significant difference in Sarkidosis.

John Carlin: The folks at Cotton are ah, researching a drug that is showing promise and shutting off the body's damaging response to the triggers of Sarcoidosis.

Bill Gerhart: So that's what I mean about a precision medicine and why we're so excited about the promise and potential of this particular drug for Sarcodosis.

John Carlin: That interview is coming up.

Bill Gerhart: This is the Sarkfighter Podcast, living with sarchoidosis and other rare diseases. Here's your host, John Carlin.

John Carlin: Hello and welcome to the Sarkfighter Podcast. I'm your host, John Carlin and this episode of the FSR. Sarcfighter podcast is brought to you by kind of antsciences sponsor of the Resolve long pulmonary sarcoidosis clinical trial. For more information, please visit www.sarcodosentrial.com. I do this podcast to offer my fellow shark fighters hope and to help you connect with other shark patients to hear their stories, understand how Sarcidosis affects their lives. Hopefully that helps you understand what you are up against and what you need to do to overcome whether it's the disease or the effects of the medicine or both. Before we jump into our interview today, I want to give you an opportunity to become a leader in the Sarcodosis community by sharing your story. A lot of people have done it here on the podcast, but FSR now is seeking dynamic, dedicated individuals impacted by Sarcodosis. To work alongside the newly launched FSR Global Sarcoidosis Clinic Alliance, volunteers would apply to become community outreach leaders. That's a new title. Who will share their Sarcodosis story with the public to empower others and raise awareness, or apply to be support group leaders, which would be a different category of leadership to facilitate inperson support group meetings at FSR Global Alliance Clinic alliance member locations. Applications are due September 30, which is coming up quickly, about a month from the day that I'm recording here in late August of 2022. Learn more by visiting the FSR website and there will be a link in the show notes, but it's www.stopsarcodosis.org gscaleaders. But another thing that we do here on the Sark Fighter podcast and these actually tend to be the most popular episodes, is talk to researchers and people in the pharmaceutical space about progress in fighting Sarcoidosis. Kind of spread that out, didn't I? Sarcoidosis, well, Sarcoidosis, you know what I mean. Today I have for you a solid dose of hope. The pharmaceutical company kind of Am is working on a drug called Namiliumab and if all goes well, you'll be hearing a lot about it in the coming months and hopefully longer. Now probably if it gets through all the trials and so forth, it'll change and it'll have a, uh, commercial name. And if all goes according to plan and this becomes a drug that's available to Sarcodosis patients and a lot has to happen before that could be the case. Namil You MAB will be known by something else. But in the meantime, let's just get kind of used to hearing that. It's N-A-M-M-I-L-U-M-A-B namel. Umab, okay. And I'll be talking with Bill Gerhardt. He is the CEO of kind of Aunt and also Rainy Rogers, she is the director of Patient Advocacy about how this drug, if it makes it through clinical trials and it's done well so far, can shut down sarcoidosis by switching off the trigger that creates the body's autoimmune response, which, instead of helping, actually damages tissues in our bodies. That's what we call fibrosis. And often that damage is irreparable. And of course, sarcidosis is the snowflake disease. It's different in every single person. Most of the time, like 90%. It happens in the lungs. Sometimes it happens in the heart. So you have cardiac sarcoidosis, and the last thing you want is permanent damage from fibrosis in your heart. But also, uh, in a very small number of cases, it can be in the, uh, neurological system. Like in my case, I have it on my spinal cord. It can be in your eyes. It could be in your spleen, it could be on your skin. We've talked about many, many of those cases here on the sarcfighter podcast. And no matter where it shows up, it's not good. So today we'll be talking primarily about sarcodosis in the lungs, also a little bit about cardiac sarcidosis, but how Amelia MAB looks to be promising in those cases. All right, now, as you know, sarcidosis is essentially granulomas. Noncaseating granulomas is the term that we hear and use that's basically small clumps of immune cells clustering in a part of your body where a signal has indicated that the body needs to defend against an invader of some kind. But there is no invader. There is no insult. But the granulomas come anyway. And instead of helping, they do damage. And if left unchecked, there are all kinds of issues that can happen. And that's basically the story with sarcoidosis. So today on the podcast, we'll be hearing how kind of ant is making steady progress so far with this new drug, Namilium App. All right, now, Bill Gerhardt is so good, the CEO, at breaking down how the drug works in layman's terms, in a way that you can understand. You'll hear him use an analogy where he talks about the tumbling of dominoes. And if you've never been able to figure out what's going on in your body when he starts talking about how the dominoes tumble, you'll start to really understand what's going on with sarcrodosis in your body and then how, uh, this new drug potentially could stop the dominoes from tumbling and by doing that in a very specific, highly targeted kind of way. Now, what I can tell you so far is that Namilia MAB has passed the phase one clinical trial. And the company is now recruiting people, including you, maybe for Phase Two. If that's successful, there will be Phase Three and then FDA approval. And we'll go over the timeline in the interview today. And as I mentioned a moment ago, you may have a role in this. There is a strong need for people to sign on for the trial, and Bill and Rainey will be sharing information about how you can do that. And I can tell you it is not just happening in the United States. So there will be opportunities for people all around the world to potentially participate in this. So maybe you're one of those people who are suffering the effects of prednisone, maybe long term exposure to prednisone, or you're dealing with methotrexate. And a lot of people's bodies don't react well to methotrexate. And, for instance, I know mine didn't, and you're tired of it. So maybe you're wondering where you would go to participate. How would you sign up for this? How would you just get more information about it? And all of that is coming up here on the FSR Stark Fighter Podcast.

Bill Gerhart: Uh.

Speaker C: M um.

John Carlin: Hi.

John Carlin: I hope you're enjoying the Stark fighter podcast.

John Carlin: You may be wondering, what can I do to help?

John Carlin: How can I be a part of the Sarquidosis solution?

John Carlin: It's simple.

John Carlin: Make a donation to Kicks, kick in to stop Sarcidosis.

John Carlin: 100% of the money goes to the.

John Carlin: Foundation for Sarcidosis Research.

John Carlin: Look for a link in the show.

John Carlin: Notes of the Sarfighter Podcast. A reminder from FSR. Over the past year, with generous support from the community, FSR has made incredible progress in efforts to accelerate Sarcodosis research and deliver responsive patient support. Programming spotlighting Sarcodosis to hashtag make it visible. FSR's 2022 update that provides an inside look at the incredible progress made in the last year, as well as upcoming initiatives in the Sarcoidosis world.

John Carlin: So if you want to know more.

John Carlin: About that, visit the FSR website and read the update and learn how your support can help stop Sarcoidosis.

John Carlin: Welcome back to the Sark Fighter Podcast. And joining me now is Bill Gerhardt. He is the CEO of kind of ant. And Randy Rogers. She is the Director of Patient Advocacy as well. Guys, thank you for joining me here on the podcast.

Bill Gerhart: It's great to be here, John.

John Carlin: Uh, yeah, we have a lot to talk about today and some exciting research which could have some fantastic results for people with Sarcoidosis. And Bill, I know you're going to be fielding a lot of these questions in the beginning, and then Rainy will get to you in a little bit when we start talking about the advocacy side of, uh, what you guys are doing at Kind of amp. But Bill, let me just throw it out there. You have a, uh, potential new treatment which is still in the research phase. But, uh, why don't you tell us what Namilia, uh, MAB is, please?

Bill Gerhart: Yeah, sure. Thanks, John. First, um, I'm, um, excited to be here on your podcast. I've been, uh, listening to you for a while now and feels like we're, uh, old friends. So it's a pleasure to be here and we're excited to talk about our drug candidate for, uh, sarcodosis and a clinical trial that we've initiated for pulmonary sarcodosis. Nameliumab is a next generation precision medicine. It's pronounced Nah milieu MAB. I know it's a mouthful, but after we say it 100 times, we get pretty good at it, right. Um, but eventually it will have a brand name. But that's a generic name for now. And to oversimplify, what nameliumab is it's? An antibody that inhibits a key cytokine, a type of protein that we believe contributes to the formation and persistence of granulomas. Antibody drugs are often referred to sometimes as biologics because it mimics M, a type of molecule that is found in our biology, in our body. Antibodies are normally produced by our body to fight diseases and infections. All of us have millions of them circulating within us. For example, most of us all now have antibodies that will attack the Cobid virus, either because we've been infected and or because we've been vaccinated. Antibody drugs are unfortunately expensive to manufacture, but they can be very powerful at precisely inhibiting the activity of a key target, a key protein involved in a disease. You know, uh, when a drug is a biologic, when it is administered either by infusion or injection influxamab or rimicate or Adolylmab or humera are examples of an antibody type drug that are biologic that some of your listeners may be familiar with. Another clue that the drug is an antibody is when the generic name ends in MAB, M-A-B like inflicts a MAB or Adolittle MAB, or in our case, Namilia MAB nameb inhibits a key protein that we think is involved in the formation and persistence of granulomans. Uh, the name of that particular protein is called granulocide macrophage colony stimulating factor, which is a mouthful. It's referred to as GMC SF, in short. But the important thing to know about this particular protein is that it's elevated in the blood, lung fluid and lung tissue of sarcoidosis patients. And the more severe the disease, the higher are, uh, the levels of this particular protein. More importantly, when GMCSF is genetically deleted in animal models, those animals are not able to form granulomas when challenged with a virus or bacterium. And when GMC s theft is overexpressed in animal models, the level of granulomas are significantly higher. So it's the combination of these samples from patients combined with these animal results that are a really good indicator that this particular protein is required for granuloma formation of persistence, and that if we inhibit it, we can potentially make a significant difference in sarcodosis.

John Carlin: Well, that is a lot of information, but, uh, it sounds it was a wonderful explanation, honestly, for what's going on in people's bodies. So you say you try to trigger the um reaction by what would you do? Like give them a ah, virus and see if you couldn't get that to trigger? And that's the evidence that suggests this is working.

Bill Gerhart: Yeah. Not to get too uh, scientific about it, but there are animal models that have been developed that form granulomas when challenged with a bacterium or a virus or a number of any other things that can result in the formation of granulomas. And so in these animal models to test a hypothesis you can actually genetically engineer the animals so that it doesn't express your protein of interest. And in the case where this particular protein that we're targeting Tmcsf, is missing those animals cannot form granulomas in response to that challenge. And more importantly, when we add higher levels of this protein to those animal studies when challenged, you see a significant increase in granuloma. So it's uh, not a guarantee, but it's a great sign that this approach could be effective for treating granuloma at its source. I mean sorry, treating sarcodosis at its source?

John Carlin: Well, it sounds extremely promising just on the face of it, as you've explained it.

Bill Gerhart: Well, we're certainly excited enough to invest uh, a very significant, uh, amount of money and uh, for the team to invest a significant um, percentage of their uh, adult working life to try to develop a new therapy for sarcodosis based on this uh early but uh, promising data that suggests that the drug will work once, uh, it gets into patients. I think maybe to better answer the question why do we think it will work? We need to briefly just traverse the ground that may be well understood by some of your listeners. We all, I think, know that sarcoidosis is an inflammatory disease characterized by the presence of granuloma believed to be formed by an exaggerated immune response to unidentified antigens. Granulomas are clumps of cells that surround an antigen. The purpose of granulomas is to wall off that antigen so that the body is protected from whatever that is. And what do we mean by an antigen? It's a foreign molecule or a molecule that the body thinks is foreign, like a virus or a bacterium, chemicals or maybe dust even or maybe even a protein in the body. Like a fragment of DNA that the body doesn't recognize. And normally the body can clear these granulomas without assistance. But in sarcodosis, those granulomas persist, sometimes leading to inflammation that when unsuccessful at clearing the granulomas, persist. Uh, and it's that chronic inflammation that mostly leads to the symptoms. Patients experience compromised organ function and in some cases scarring or fibrosis of the tissue which can lead to even organ failure. And as you know, and as uh, most of your listeners know, nobody knows for sure why some people get this disease. It's likely due to some genetic predisposition where the immune system doesn't quite work right when a foreign molecule is detected similar to why some people get cancer when exposed to certain environmental exposures. Why is this important to understand for this particular drug? Uh, well, short of understanding what genes are causing the initial dysfunctional immune response and because scientists don't historically know what is actually causing these granulomas to form the best pharmaceutical approaches to treating this disease historically have been broadly acting anti inflammatory or immune suppressive therapies like pregnancy or method. But what we're doing is taking a more precise approach, a, uh, more targeted approach to the specific thing that's happening in sarcodosis based on a better understanding of what's going on. All biological processes have what are called pathways. When one thing happens, a cascade of biological events occur leading to, in this case, an inappropriate immune response to an antigen that fails to resolve on its own. Think of a biological process as a row of dominoes that fall over one after another after the first one is knocked over eventually leading to the last domino falling over. As we better understand what those pathways look like for sarcodosis we can develop new drugs that target a specific step in that pathway, a specific protein in that pathway or, if you like, a specific domino. In my example, that when inhibited, stops the process from occurring stops the formation and persistence of granulomas and or better enables the body's immune system to get rid of the granulomas. If we can prevent or reduce the formation and persistence of granulomas we can prevent or reduce the resulting chronic inflammation that leads to all of the problems that patients are experiencing. And if we can do that with a drug that is safe, well tolerated and conveniently administered then that would be a home run for, uh, patients with this disease. Think of it as a precision medicine. We're not developing a drug with broad anti inflammatory properties like prednisone, which works in the short term. It causes all kinds of problems if patients are unable to taper off after one or two courses. And we're not talking about a drug like methotrexate, which broadly suppresses the immune system, which sometimes works, but it's slow to act and has significant side effects for a lot of people that take it. In my Domino's example, these types of medicines don't knock over just one domino in one pathway. They knock over multiple dominoes and multiple pathways a sledgehammer, if you will, instead of, um, a scalpel leaving not only some, um, efficacy in circuit doses, but sometimes also to a lot of other unintended effects, side effects. So that's what I mean about a precision medicine and why we're so excited about the promise and potential of this particular drug for sarcoidosis.

John Carlin: And why sarcoidosis? We've got about 200,000 sarcoidosis patients in the United States and over a million that we know of in the world. Convenient, uh, is focusing on an orphan disease. Can you kind of give us some idea of why you decided to work on circuitosis.

Bill Gerhart: Yeah, that's a great question. Maybe to answer that question, I can tell you first a little bit about myself and then a little bit about the philosophy and the focus of our company. Um, my background is primarily as the CEO and founder of, uh, bio pharma companies, mostly here in San Diego where I live. So I'm somewhat of a serial entrepreneur, having started a half a dozen companies in my career. By the way, biopharmic can mean different things to different people, but in my case, it means drug development. In other words, designing and executing clinical trials in order to prove that a drug is safe and effective and deserves to be FDA approved and made available to patients. I started a company and helped develop a new medicine for cystic fibrosis, which is a rare disease I helped start, uh, and was the CEO of a company that developed a new medicine for chronic obstructive pulmonary disease, which is the number three cause of mortality in the world. And I was the CEO and founder of a company that was developing a drug for idiopathic pulmonary fibrosis, which is a deadly respiratory disease. But in that case, um, that program failed, which sometimes happens in our industry. Uh, we were in the middle of recruiting for that study when the pandemic hit, and we had to stop recruitment, like a lot of companies. And we tried to finish with the data that we had, and unfortunately, the data wasn't positive enough for us to continue forward. So that's kind of a little bit about my history. And at this point in my career, I'm passionate about working on new treatments for diseases that have few or no good treatment options. Serious diseases with significant unmet needs. And those tend to be rare diseases, which is arbitrarily defined as less than 200,000 in the US. And they frequently have no therapies approved. And I've been looking at Sarcodosis for maybe close to 15 years, but just never found the right approach, um, that I thought would be, um, uh, successful. Um, and the conviction rate has to be pretty high, given what's at stake when you actually go down this path to develop a new drug. So, kind of Ant was formed to specifically focus on inflammation and autoimmune rare diseases. And our first and currently only program is targeting Sarcodosis. Unlike a lot of other developers who start with a drug and look for a disease to treat, our philosophy is to first start with the problem, the disease, and work backwards to identify and eventually develop a drug that we think will most effectively treat that problem. So when you ask the question, Why Sarcoidosis? It's because of all of the different rare diseases that we've been looking at. We think we've got an approach that we have a lot of confidence in, that we think it's going to work. And once you have that conviction, then there's obviously a moral, um, obligation and a business obligation to go try to make that happen. Influenb remicade or adolyma. Pimera are examples of drugs that have been developed for other diseases and they have sort of been repurposed for treating sarcodosis, but they weren't designed or developed to treat specifically sarcoidosis. Not specifically proven effective and approved by the FDA for sarcodosis, but better than no option for sure, other than antiinflammatory or immunosuppressant drugs like Pride Zone or Methotrexate. The advantage of our approach is that we believe it significantly increases our probability of success, the success of the therapy, both in terms of efficacy and in terms of tolerability and safety. A lot about sarcodosis, not well understood. That's just one of the many things that makes it challenging to develop a new therapy for this disease. However, our, uh, growing knowledge and what biological processes cause the disease, and based on our insights on how we can interrupt those processes that lead to this disease with a new therapy, gives us, um, a lot of hope and a lot of excitement about the potential to treat this disease at its source more effectively than what's currently available. So that's our approach generally, and that's our approach specifically for sarcodosis.

John Carlin: Well, um, just speaking on behalf of the sarcodosis community, thank you for choosing sarcodosis to focus on. Um, as you mentioned, there are no on label drugs right now for sarcodosis that are specifically developed for sarcoidosis. So, um, as we look at your research opportunity here, uh, let's hope that it moves forward in a positive direction the way that it seems to be with what you've been able to establish so far. So thank you for that. Um, let me ask you the next question. So you have started now, uh, as we talk about your research, you, um, have started something called the Resolve lung study. Please tell us what that is.

Bill Gerhart: Uh, sure, yeah, no excited to talk about our clinical trial and in fact, we actually have initiated two clinical trials, John. We've initiated a smaller study and cardiac sarcidosis that we refer to as resolve heart, and a larger trial in pulmonary sarcastosis, which we refer to as resolve lung, which we'll mostly discuss here today. And the resolve lung clinical trial is a traditional randomized doubleblind placebocontrolled trial, that is a phase two trial, which is many of our listeners know as a result of listening to other speakers on your podcast, is sort of the middle of the process, if you will, to a drug being approved by the FDA. Phase one is mostly safety. Phase two is efficacy and safety in a smaller number of patients. Phase three is safety and efficacy in a larger number of patients. In our particular case, we've actually designed a pretty significant phase two study. It's a twoarm study. One arm would have, ah, randomized to placebo. The second arm would be randomized to our drug mailing map. It's 100 patients total 50 patients in each arm. Um, and for patients, it would be a one year commitment with monthly clinical visits. And the one year is broken into two periods. The first six months is the randomized, double blind, placebo controlled part of the trial. But at the end of the six months, all patients in the trial will roll over and be in what we call an open label extension. Which means regardless if you were on placebo or active in the first six months of the study, you will be on the therapy in the second six months of the study. So everybody in the clinical trial will eventually get the drug, either in the beginning or in the middle of the trial. The trial, like other pulmonary sarcodosis studies, is designed to measure improvements in lung function and in quality of life, ability to taper off of steroids or other immunosuppressive, therapies changes and improvements in fatigue and other important assessments of effectiveness for patients. Um, patients eligible for the study are broadly patients that are not well controlled on currently available therapies, either because they're on doses of prednisone and methotrexate and they still have symptoms and they're still sick and they still have a poor quality of life, or they've tried to use levels of those drugs that are effective but aren't able to tolerate the side effects. So we hopefully are targeting patients who are of the highest need, who would normally be patients that would be candidates for biologic, um, in order to best demonstrate the potential of the drug. It's a big study, uh, in terms of footprint up to 50 clinical trial sites in seven different countries in the US. As well as the UK and the EU.

John Carlin: So you right now, are in phase two. Are you actively recruiting, you're looking for those patients right now?

Bill Gerhart: We are. We've, uh, just recently opened our first clinical trial sites in the US. And we're starting to screen patients for both studies. And we hope to actually enroll our first patient here in the very near future. Or maybe by the time your listeners, uh, hear this podcast, we will have enrolled our first patient, which would be a huge, exciting moment, uh, for the company, because to get to this point took years, uh, and a significant amount of work by a lot of people at our company in order to get to that point. It's not the end, obviously, because we have to then finish recruitment and, uh, lay the groundwork for the next part of the clinical programs. Everybody will be working forward, but it will definitely be a cause of celebration around our company.

John Carlin: So I want to ask you in just a moment where, um, these, uh, centers are that are offering the trial, because I assume there's some sort of geographic tie for people who may want to, but you can only travel just so far, go to the doctor often enough to make this work. Um, but I just want to get a time marker in here. You're listening to the Sark Fighter podcast, and we are talking with Bill Gerhardt, who is the CEO of kind of Ant, and we'll be hearing from Randy Rogers, the director of Patient Advocacy, here in just a moment. But we are recording, uh, in late August of 2020, uh, two. And just so if you are listening, because a lot of people do go back and listen to episodes in the future, uh, I want to make sure that if you're hearing this and you might be interested, that you kind of have a time marker. So that's what I'm doing. Um, so, Bill, let me get back to ask you, first of all, where, uh, is the research taking place and how long will you be recruiting patients for the study?

Bill Gerhart: Yeah, that's a good question. Um, I'll let Rainy give more information about how patients can find a clinical trial center in the area that they live, um, because we are opening up 20 to 25 sites in the US. It's a little bit of a long list to read off here, but our goal was to have a clinical trial site in every major city so that the majority of patients would have access, uh, to the, uh, drug. And of course, if they need to travel, we will provide, in many cases, assistance so that motivated and interested patients, uh, who potentially qualify the trial can participate.

John Carlin: All right. Uh, because it does disrupt people's lives, uh, to have to go back to the doctor that often, uh, especially if they don't feel that they need to. But on the other hand, uh, the therapy that you are investigating here sounds so promising that I would hope people listening to this, if they qualify, uh, would certainly try to step up. And the other thing that you mentioned, which is different than anything we've talked about on the podcast before, is so you have a placebo where somebody gets essentially a pill that looks like the treatment, and then you have another group that gets the actual treatment. But after six months, I heard you say everybody gets the treatment, which is an interesting take, I know.

Bill Gerhart: Uh, it's included in the study design for two reasons. The first is that it gives us more information about the drug when we can look, for example, at those patients that have been on the therapy for a total of twelve months. And just as importantly, it gives us important information about the drug when we follow patients who have been on placebo for six months, and then they switch over to the, uh, draw for the second six months, and we can see within one patient the difference in outcomes. And that is really valuable information. So that's one reason why we do it. And obviously, the second reason why we do it is because oftentimes patients are reluctant to participate in clinical. Trials because they don't want to take the risk of being in the placebo arm. And unfortunately, and you know this well from other speakers that you've had on the podcast in order to definitively determine if the dropd is safe and effective you have to compare it against a cohort of patients that are identical to the ones that have the drug who are on placebo so that you can make those comparisons. If you can't do that, then scientifically you really can't conclude if the drug is safe and effective. So that's a necessary part of drug development. But by adding this open label extension, we can say to patients if you participate in the study, you take the time and the inconvenience of participating in the study, then this is another reason to participate. And we've really worked hard to try to make it as easy as possible for patients to participate in this clinical trial. First, by opening up as many clinical trial sites as we can to make sure that, uh, the, uh, a center is, uh, nearby a patient who's interested and qualified. Second, we design, uh, the criteria, unlike other studies so that you don't have to be on a high dose of steroids to get in the study. If you are on a high dose of steroid and you want to try to get off, then you can participate in the study. And part of the study design will be to taper you down in, uh, the study. But if you're not on a high dose of steroid, you can still get in the study because we would just keep you at whatever level that you're at now. So you cannot be on a steroid at all and still get into the study. And we think that's a big advantage for a lot of people who traditionally have been excluded from clinical trials. Treatment practices are changing, and people are on high doses of steroids less and less as time goes by. And clinicians and patients are more aggressive about trying to find other options other than staying on high doses because of all of the side effects. So we think that that will be appealing. And then the other thing, uh, that's important to know about this drug is that it's not the first time that it's being evaluated in patients who have a significant disease. This drug has been in more than 300 patients, uh, sometimes up to six months or longer. And so far, we have not seen any safety or even side effects associated with the drug. So we can't be positive in what will happen in sarcosis. But, um, our belief is that the drug will continue to be safe and well tolerated. So oftentimes you hear patients say that they don't want to be a guinea pig and that's the reason for not participating in a clinical trial. But in this case, that shouldn't, uh, be a concern. And patients who, uh, are willing to participate in this trial can participate, uh, with that extra level of confidence and awareness. And then finally, the other thing that makes this drug different than other drugs that have been evaluated for Sarcoidosis, it's a sub queue injection. It's not an infusion. It's not a 1 hour fusion. You don't have to go to a clinic and sit in a chair for an hour. It's a convenient once a month injection that you can get at the clinic or in some centers that are participating in this clinical trial. A mobile nurse come to your home in order to do the assessments and to give you the injection. So we've tried to make it, um, as convenient as possible for patients who are interested in participating in this study to do so.

John Carlin: Very good. Um, I want to just ask you, so you're recruiting now. When do you think and it's a year long process, so when do you think you'll have the results of your phase two and be ready to go into a larger testing in phase three, and assuming that everything progresses as you hope, as we all hope, when, uh, might this be available to patients if everything just tracks according to plan?

Bill Gerhart: Yeah, good question. And I like your caveat at the end, because, um, um, um, often plans don't go as well as you hope, but in this case, our plan is to finish recruitment sometime in the first half of next year, which means that we will get our top line results in the first half of 2024. And in anticipation of positive results from one or both of the clinical trials that we're conducting in Sarcidosis, we are already investing in manufacturing for the phase three part of the program. One of the unique artifacts about developing, uh, particularly a biologic for a disease, is that you have to manufacture your phase three drug product in the same facility on the same conditions as what you would launch and go to market with. Um, if you don't do that, then you take on a lot of unnecessary risk and you potentially delay the drug's approval, uh, and entry into the market. So companies like ours not only are making a significant investment in the clinical trial, but we're making a significant investment in parallel with all of the manufacturing and, um, other requirements to initiate a phase three study so that we can roll into that phase three study in a very short period of time after we get our results from this study.

John Carlin: Excellent. You're listening to the FSR Sark Fighter podcast. And today we are talking with some folks at kind of a who are working on a potential new therapy, let's call it a research opportunity for Sarcoidosis. And I want to bring in Rainy Rogers, who is the director of patient advocacy.

Bill Gerhart: First.

John Carlin: Uh of all rainy welcome. Uh, you've been sitting here graciously, and we haven't heard from you much yet. So, uh, officially, welcome to the podcast.

Rayne Rodgers: Thanks so much, John. Um, the first time I met you, I felt like I was meeting a celebrity. I've listened to so much of, uh, your podcast over the last eight or nine months. So thank you so much for having us. Bill and I are thrilled to be here.

John Carlin: Sure. Uh, I'll tell you what, I'll bring it right up to San Diego as soon as the weather gets cold here in Virginia. How's that sound?

Bill Gerhart: You got a place to stay?

John Carlin: Beautiful. Um, but Randy, I, uh, know that you guys are working with FSR. Can you talk a little bit about that relationship?

Rayne Rodgers: Absolutely. So, um, I'm the Director of Patient Advocacy at Canada, as John mentioned, and really, I wear a number of hats. So my background is in rare disease research, both for pharmaceutical companies as well as for patient foundations like FSR. Um, I actually worked for the Muscular Dystrophy Association and really, uh, here at Kanye, and I'm responsible for a number of different things. So helping to design clinical trials that are easy for people to participate in, making sure that all geographical areas are accommodated for and represented in research, and ensuring ultimately that the patient voice is reflected in everything that we do. So I spend a lot of time listening and a lot of time doing research, um, in partnership, uh, with groups like FSR. So kind of antiserid to be partnering with FSR, as well as a number of, uh, other smaller organizations that are, uh, international as well, that focus in Sarcudosis. We are partnered with FSR on a number of exciting initiatives. So I'll just highlight a few. Uh. So I joke with Bill. I could talk about advocacy all day. But I'll just give some highlights and some color into what we're doing with FSR m ultimately. We feel so lucky as researchers to have FSR as a partner for us to be able to really truly understand what's important to patients and to bounce ideas off of each other as we continue with our phase two research of Namibia Map. So we are part of the Corporate Advisory committee with FSR. We also recently, just last month, many of your listeners may have attended the Global Virtual Patient Summit. We were, um, really excited to represent, uh, our research there. We had a virtual booth. We, uh, had a live chat feature and interacted with people living with Sarcoidosis all over the globe. Had a lot of really great conversations. Uh, there. We had on our booth information about the trial as well. Um, another piece I want to highlight is we're a proud sponsor of the African American Women in Sarcodosis campaign. And that campaign really, uh, is pursuing how to best encourage African American women to participate in research, understanding the barriers to that population, and, uh, making rapid change to enable those populations to have access to these types of opportunities, especially for these ethnic groups that are historically underrepresented in research. Participating in clinical trials is so important because it really helps to ensure that the trial results better reflect both the effectiveness and safety of the drug, but across all patients or all people living with Sarquidosis. So, just a few highlights with FSR, but I'm thrilled to be partnered with them.

John Carlin: Yeah, well, of course. And you just mentioned the African American Women Initiative, uh, which, if people are listening to this podcast for the first time, may not be familiar with that program, but essentially you have a population, uh, when we look at the data that is most, uh, likely to suffer the effects of Sarcodosis and least likely to access effective treatment. And so that's one of the reasons why FSR is trying to, uh, make it easier for that group of people to get the treatment they need and to really kind of focus attention on a group that badly needs it. And so kind of ant is stepping in. And you're one of the sponsors of that program, so congratulations to you on that. I think that's outstanding advocacy. But when we look at, uh, Namib, if people want to get involved with this, and I know that there is a website that is probably available, um, through your website, but that information is also being distributed by FSR, is it not?

Rayne Rodgers: It is, yes. We have developed so we do have our kind of a website, but we've developed a study specific website that is for patients. It's for people that are living with Sarkoidosis who are looking for information about the drug, about the trial, um, you know, where these trial sites are and the ability to ask more questions. So that website, uh, and hopefully we can link it in the show notes. John, if they've seen you do that website is www.starkoidosistrial.com. So super easy to remember. Um, all the information is there. Also, Bill had mentioned study sites, that we have 26 sites in the United States. Um, and we have a total of, uh, approximately 50 sites globally. So we have seven countries, including the United States, that are participating. Um, some of the sites are active. Now, as Bill had mentioned, we're thrilled to have sites actively enrolling, and some of our sites are still in startup.

John Carlin: I know you're actively working with all of these sites right now. Uh, and again, we are speaking in late August of 2022. So if someone is listening to this, when do you think that that will be firmed up completely and that you'll have all your sites lined up and ready to go and to accept patients that might be willing to do this?

Rayne Rodgers: Yeah, absolutely. So it's on a rolling basis. Um, every week we have new sites activating. We're hoping to get all of our sites active here in the next couple of months, uh, specifically in the US. Probably sooner. Um, that link Sarquidosischrial.com, it's going to be updated in real time, so as soon as a new site is active, that site is going to be listed there.

John Carlin: I just want to touch back quickly because Bill, earlier in the podcast is mentioned, there's actually two studies happening here. There's. Resolve heart and resolve lung. As we look at the potential safety and effectiveness of Amelia Map, um, are we talking primarily about lung patients here, or if you heard the word heart and you're a cardiac patient, how would you react to the information we're sharing right now?

Rayne Rodgers: You're right. What we've been talking about with the 50 sites and, uh, international and all that, that is specific to the Resolve Lung Study. The, uh, Resolve Heart Study is in the United States, and we do have information on, uh, our website for that study as well, in terms of how to find more information.

John Carlin: All right, and what level of sarcidosis are you looking for? Are you looking for a patient who's just been diagnosed or for somebody who's been suffering for multiple years or all of the above?

Rayne Rodgers: Sure. Um, so there's a short list of criteria on the website. I'll highlight just a couple of points. Um, so, 18 years or older, diagnosed with sarcoidosis for at least six months, completed a COVID-19 vaccination. I think the fourth one listed there is, um, just around steroid use. And, um, Bill had mentioned this earlier, but willing, uh, to either taper down or stay on a stable dose or no requirement for steroids as well. So, yeah, those criteria. A couple of things there are listed on the website.

John Carlin: All right, outstanding. Um, so, Rainy, is there anything else that you wanted to add in terms of patient advocacy and making kind of ant, uh, making sure that the psychedosis patients are being heard, or anything else you want to add along those lines?

Rayne Rodgers: Absolutely. Uh, just a couple closing statements for me. I mean, ultimately, I just want to really stress how much time our team has spent really considering and trying to understand what's important, not to rare disease patients, but to people living with sarquidosis, um, and what it would take for them to, uh, consider partnering with a company like kind of Ant for research, uh, with Mammalia Map in both pulmonary and cardiac sarcidosis. We've tried to think outside the box to make the trial more accessible. So some of the things Bill had mentioned, we have, um, transportation assistance and, uh, travel reimbursement to study visits. We know that these visits can be, uh, time consuming and potentially financially burdensome. Um, so really trying to, um, cover those costs there ultimately the fact that Namiliamab is an injection and not an infusion, which will reduce, uh, the time needed to participate in a research project such as ours, and the offering of home and televisits. So if a patient is working, um, they need an evening home visit for their objection. This month, they're traveling, they're taking care of a loved one there's so many things that happen on a day to day basis. Really trying to make sure that we're addressing those potential concerns and offering, um, the flexibility and the, uh, resources there so that all patients that are interested have an opportunity to participate. Not just, uh, someone who's living in a close to a big city and potentially retired. And, um, we understand that the Sarquodosis community is very complex and that there's a lot of different, um, things that are important. So really trying to cover all those basis.

John Carlin: Well, the fact that it's a shot is fantastic. I've been through the whole thing at the infusion center with Remicade, and it is very time consuming. You have to build your life around it. There's no other way around it. Um, and now, uh, I've transferred to, um, Humera, and that's a shot that I give myself at home, which is so much easier. And in terms of participating in this study, uh, that's just something that it's that hour of your life that you get back once you get there and go through the waiting room and all the other stuff that happens when you're at the doctor's office. Well, Randy, uh, thank you. And I, uh, want to bring in CEO Bill Your Heart again, uh, at kind of a bill. Do you have some closing thoughts or anything else that you would want to add to our conversation?

Bill Gerhart: Drug development is hard. Most people in the world have a negative impression of pharmaceutical companies, usually because of the high prices charge for drugs, which makes them less accessible to many. I understand that. But the reality is that most drugs are initially discovered and developed by small companies like ours, staffed with people who take huge career risks and investors who risk significant amounts of money to work on just one program or one disease. It takes a hurricane, uh, effort and a lot of courage to nurture a drug candidacy from infancy to improve an entity that's available to patients. Most, as you know, fail along the way. Many of us have nothing to show for the effort after dedicating three to four Five years Longer of Our Livesto program. And a good example was the program that I worked on at IPF, which was five years of our lives and $100 million of invested capital with nothing to show for it. At kind of end, I'm, um, privileged to lead a small team of very dedicated individuals who wake up every morning thinking about nothing but the huge responsibility we have to successfully conduct these clinical trials and develop a new therapy for those with sarcosis, many of whom desperately need a more effective and well tolerated therapy to treat their disease. For us, it's now a mission and a cause that consumes 80% of our waking minutes. We are completely energized by the potential to make a significant difference in the lives of patients that are suffering from Psychnosis. Having spoken to many of them over the last couple of years. We deeply understand the challenges experienced by people with this disease. It's challenging to diagnose, challenging to find the optimal treatment regimen. It has a significant impact on quality of life, and the existing treatment options can be pretty crummy. So here's my call of action here's my call to action from your listeners. Nobody is pleased with the currently available therapies for sarcodosis. But in order to develop new therapies that make a significant difference in this disease, it does take a village. It takes dedicated biopharma or pharmaceutical companies that are willing and able to make the investment in r amp D for an orphan disease for a small number of patients, relatively speaking. But more than this, it takes clinicians who treat this disease to be willing and enthusiastic about participating in clinical research. If they're not willing and enthusiastic about participating in clinical research, we can't evaluate and get new therapies to patients. And patients have to be willing to participate in clinical trials. If you don't have clinicians and patients willing to participate in clinical trials, we can't invest in developing and evaluating new candidates for this disease. I mentioned earlier that biologic drugs are expensive to manufacture. But the biggest drive of cost, the biggest driver of cost, and thus the biggest driver of high prices for drugs for rare disease, is a huge cost to conduct a clinical trial like the one we have initiated for sarcidosis. And statistically, less than 5% of those with the disease are willing to participate in a clinical trial. If it's a large disease, that's not a problem, but if it's a rare disease, that's a big problem. It's been estimated that only 1% of sarcodosis patients who would qualify for a clinical trial would choose to participate in a clinical trial. 1%. It's risky to try to develop a drug for sarcodosis because we don't know what really causes the disease and we don't really know what's required to get the drug approved by the FDA. But on top of those risks, there's that significant cost to recruit and enroll a clinical trial, because it's so hard to find patients that are willing and interested to participate. If the percentage of patients just doubled from 1% to 2%, that would make a huge difference. The more patients who participate in clinical trials, the lower the cost to conduct clinical research for psychedosis, and the more motivated companies would be willing to develop new therapies for this disease. Forget about our company and our disease. I want to just make that pitch for the community of people who have this disease. It's important for people to have that awareness and to know that their participation is important in order for the treatments, for their disease to be developed and for their lives to improve, not just for them, but for successive generations of people that might have this disease. Other pharma companies are watching to see what our experience is and the experience of other companies conducting clinical research in Sarcodosis, can we successfully conduct a large study in sarcidosis? So this is a call to action. If you are clinician who treats Sarcidosis patients, sponsor a clinical trial so that your patients who are interested in qualified can participate. If you're a patient, please ask your clinician about the opportunity to participate in a clinical trial. Yes, it does involve some risk and some, um, inconvenience, but companies like ours need you to partner with us to conduct clinical research in order to develop new therapies that better treat and may eventually cure the disease that affects you and future generations of people with this disease. Without your participation, we can't do it. Help us help you.

John Carlin: Outstanding. That's a fantastic call to action. And, uh, I had never heard these statistics before. The 5%, generally 1% of Sark patients, and you start doing the math, and that doesn't leave you with many patients, potentially, when you look at the United States. Fortunately, the podcast does have a worldwide reach. So hopefully, if somebody's listening in Australia or UK or, um, wherever in Europe, uh, maybe that will help you get this done that much more quickly. So, uh, let's hope that that works out. So, we have been talking with, uh, two folks from kind event CEO bill gerhardt and randy rogers, who is the director of patient advocacy. Guys, thank you so much for what you're doing and for joining me here on the FSR Stark Fighter podcast.

Bill Gerhart: Thanks for the opportunity to share a bit about our company and, uh, the potential for this trial. John, we really appreciate it and thanks for all that you do for the community. It's really inspiring.

Speaker C: Um.

John Carlin: We covered a lot of ground in this interview, but there are some takeaways as you listen to the FSR SARC Fighter podcast. Let me hit the bullet points for you. Kind of Ant is the pharma company. They have a potential new drug to fight Sarcodosis called Namiliumab. It's passed phase one testing. Now entering phase two. It's shown so far that it is safe and has few side effects in 300 patients. And the hope is that further testing will prove that it will suppress the inflammation associated with Sarcoidosis and therefore significantly improve patients quality of life. They are working with various centers right now to get the ball rolling. They need you to participate. And this test is interesting in that eventually, even people who get the placebo initially will eventually receive the treatment from, uh, Namib. I'm working very hard to make that just roll off my tongue. I'm getting better at it. Namilumab. Okay. FSR is partnering with Kind of Ant to help spread the word about the trial. And you can get more information in the Show Notes on Kind of Ant's website and the FSR website.

John Carlin: And, uh, as I said, as well.

John Carlin: As the Show Notes here. Now I can tell you that it costs hundreds of millions of dollars to bring a drug like this through research and testing and then to the market. And let me just say that I, for one, m am glad that those resources, and let's face it, that risk, that financial risk, is being focused on Sarcoidosis, which is classified as a rare disease with fewer than 2000 patients in the United States. There are many, many of these orphan diseases. And it is gratifying that people like Bill Gerhardt and Rainey uh, uh, at kind of ant have decided that Sarcoidosis is where they want to put their focus. And then they're making progress, right? They're making progress. So thank you to Bill and Rainy for joining me here today on the FSR Sark Fighter Podcast. Reminder, uh, the official Sark Fighter song is called Zombie by Mark Styer, and he plays in a band called the White Hot Lizards in Alberta, Canada. Mark is a fellow SARC fighter. The story behind the lyrics is way back in episode twelve, but whenever you hear little bits of that song here on the podcast, you're hearing part of the song Zombie Reduce. Uh, I normally release a podcast every other Monday. As I'm speaking today, my trusty dog Google, my rescue boxer, is curled up on the chair in my office. Google makes my life so much better and I just have to share that with you. Please don't forget to follow me on social media. I'm on Facebook. Just search for Sarkfighter. Also Instagram if you happen to ride Peloton, you hear me talk a lot about Bicycling. I'm on Peloton as Sark Fighter and I do have a cycling blog, Carl and the Cyclist, which has a section called Cycling with Sarcoidosis. Also, if you are new here and you're just trying to figure out what Sarcoidosis is, try listening to episode one. But, uh, actually episode two with Dr. Simon Hart. It is one of the most listened to episodes. My story is episode one. And the backstory to the founding of the foundation for Sarcodosis Research is episode eleven with Andrea and Reading Wilson who started the foundation at their kitchen table. If you would like to tell me something about the podcast, if maybe you'd like to be a guest on the podcast, if you want to, uh, alert me or, uh, let me know about something that's happening, please email me. Carlinagency@gmail.com, uh, that link is in the Show Notes. And if you just want to comment on the podcast, I certainly welcome your comments. So once again, follow me on social media. Please take a link and just help share the podcast, get it out there in the Sarcodosis community using your social media, because I do appreciate your interest. And the more people who listen, the more we can grow this community and the more it can help. So, if nothing else, if you really like the podcast, just tell one person. I certainly appreciate it. Until next time. Keep fighting.