The ARIKAYCE Resource Library
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Information from the NTM Treatment Guidelines
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In the Know Program: Journey to ARIKAYCE
Watch MAC lung disease expert Dr. Gwenn Huitt and Judy, a real ARIKAYCE patient, discuss topics that can help you learn more about MAC and its treatment options, including adding ARIKAYCE.
Hi everyone, and welcome to today's program. Thank you for joining us for In The Know, let's talk about ARIKAYCE. Today's program will focus on the journey to ARIKAYCE, amikacin liposome inhalation suspension, and you'll hear from a leading physician expert and a patient who is taking ARIKAYCE. Before we begin, I'd like to share some important information you should know about ARIKAYCE. ARIKAYCE is a prescription medicine used to treat adults with refractory, difficult to treat mycobacterium avium complex MAC lung disease, as part of a combination antibacterial drug treatment plan or regimen. This product was approved by FDA using the limited population pathway. This means FDA has approved this drug for a limited and specific patient population and studies on the drug may have only answered focused questions about its safety and effectiveness. ARIKAYCE is associated with risk of increased respiratory adverse reactions, including allergic inflammation of lungs, coughing up blood, severe breathing problems, and worsening of COPD.
Now I'd like to introduce Dr. Gwen Huitt, who will lead today's discussion. Dr. Huitt is a physician with 29 years of experience specializing in Infectious Disease at National Jewish Medical and Research Center, and NTM Center of Excellence. She's also a professor in the Department of Medicine, Director of The Adult Infectious Disease Unit, Chair of the Medical Executive Committee and Chair of the Mycobacterial Consult Service at the National Jewish Health in Denver, Colorado. Her research mainly focuses on infectious disease, pneumonia and germ theory of disease. Dr. Huitt is a distinguished physician, having published many articles on NTM, she is also the recipient of many awards, such as America's Top doctor for Castle Connolly Medical Limited. We are delighted and honored to have her here with us as our expert for this program. Over to you, Dr. Huitt.
Well, thank you so much, Nicole. That's a very generous introduction, and I'm really excited to be able to participate in the program, and I will not be the only person speaking during this program, I've got a great colleague joining me, and Judy will really do a very important service for everyone in presenting a patient's perspective of the use of ARIKAYCE in her particular situation. So Judy, if you could introduce yourself and tell us a little bit about yourself.
Sure, thank you, Dr. Huitt. I really appreciate that. Hi everyone. I'm Judy, my husband Mike and I live here in Wisconsin most of the time at our central home. Part of the time we escape the frozen tundra down to Florida. I have worked in the printing industry, starting off actually as a trades operator, as a press operator, dark room, eventually going into sales and sales support. We have 10 wonderful grandchildren, of course, and we're having a great time watching them grow up. I'm looking forward to sharing my story today. Thank you. Back to you, Dr. Huitt.
Great, thanks so much. And I think we're just gonna have a wonderful program and I'm really excited to go forward today. So we do have a packed agenda, and you're all tuning into this because we really wanna focus on the journey to using ARIKAYCE. And during this program, we are going to cover several topics including understanding MAC lung disease, getting started on a multi-drug regimen and how that goes, as well as an overview of when and how we would add ARIKAYCE's treatment and where it might fit in with a particular treatment regimen in a patient that has pulmonary MAC infection. And last but not least, certainly, we're going to give you a few tips and kind of hints about how you might talk to your doctor, how you might approach your own underlying lung disease and infection with pulmonary MAC and just move forward and empower yourself in treatment regarding pulmonary MAC. So let's go ahead and get started. Well, what is MAC lung disease? And who might be at risk for having MAC lung disease?
Well, I think the first thing I always like to tell patients is, there are varying degrees of MAC lung disease, and so every patient is not created equal, and every disease process associated with MAC lung disease may be different, but we need to take it very seriously, because if we don't take it seriously and if we don't address it regarding treatment in particular, and management in the proper timeframe, it can really progress and cause further lung damage over time, if we leave it untreated. NTM lung disease and MAC lung disease in particular is caused by a bacteria that really we find everywhere in the environment, it's particularly a water and dirt-born organism, so we all encounter it all the time, but interestingly, the MAC germs are the most common form of non-tuberculous mycobacteria organisms, and they comprise about 80% of cases of MAC lung disease.
Well, if we're all exposed to it all the time, how come some people MAC, a lung disease and others don't? Well, the honest answer is, we don't know that yet, we're constantly exposed to it. In some of the areas that we're exposed to MAC in the environment would be tap water sources such as showers or perhaps drinking water, shower heads, mist from the misters in the grocery store or out in restaurants when you're sitting under those misters, yeah, you're inhaling a lot of mycobacteria and a lot of that might be MAC. And then if you're a big gardener and you're breathing in dust and dirt outside, we know that MAC is in these sources as well, so really we're constantly based in MAC from environmental exposure. But if we're all exposed all the time, the question I commonly get from patients is, "Well, if we're all exposed, why am I the lucky one who got MAC lung disease?"
Well, we know that there are certain conditions that really predispose a patient to coming down with a more serious form of MAC infection that will lead to MAC disease, and a few of those include bronchiectasis, and we know that about 50% of people with bronchiectasis may actually have active lung disease, and in those patients, MAC lung disease is the most common form of bacteria, or MAC is the most common group of bacteria that affect their lungs. An additional group of patients that are quite vulnerable to a MAC lung infection would be those patients with underlying COPD or Chronic Obstructive Lung Disease, and we know that COPD patients may be up to 16 times more likely to get NTM lung disease because they have underlying structural lung problems just like patients with bronchiectasis, that once these germs repeatedly gain access to the lungs, they can set up shop and cause problems. Additional patient populations that might be vulnerable to MAC lung disease would be those with underlying asthma and recurrent pneumonias.
Additionally, we've known for decades that certain groups of people may be more vulnerable to acquiring MAC and then progressing to lung disease associated with MAC, and in the old days, and I'm old, we used to term this the Lady Windermere syndrome, and the common two groups of patients that we would see would be a smoker, usually a male who was a smoker, that was one group, and then the much more prevalent population was women who were tall and were thin and usually were in their 60s, 70s of age group, they tended to live in coastal areas, particularly in the United States, but we now know it's certainly not limited to those patients in coastal areas. Anyone in the middle part of the United States can certainly acquire MAC as well, but we do see it more commonly along the coast, and some people do have weakened immune systems as well.
Well, what are some of the signs and symptoms if you think you might have MAC lung disease? Well, it's interesting because the MAC lung disease symptoms may really mimic many other diseases and disease processes, so you may have a chronic cough, well, people with COPD often have a chronic cough, rarely DOEs, and so it differs from asthma in that regard. Generally, you're not experiencing wheezing like asthmatics, but asthmatics cough a lot as well. It mimics other disease processes, and because of that, your practitioner, your general practitioner, and turnus family practitioner may be looking in other directions, so it may be a little difficult to diagnose initially. So the common theme I hear from a lot of patients is, "Gosh, it took me years to finally get to the right place and to the right doctor to get diagnosed."
So it's important and empowering, and part of our job today is to empower you as a patient to say, "Well, what are the common symptoms that somebody with MAC lung disease might be experiencing?" And generally speaking, chronic cough, fatigue and shortness of breath are really the common themes. Now layered on top of that might be sputum production and commonly is sputum production. But the rare person just coughs and has shortness of breath and really doesn't produce any sputum, and when these just are relentless and continue to go on despite your doctor prescribing umpteen courses of antibiotics or trying on an antihistamine for allergies or giving you a breathing treatment. If nothing's working, then it's time to look under other rocks and start looking to think that it might actually be MAC Lung disease. Well, I'd like Judy's impression right now what her journey was when she started on this process. If you don't mind, Judy?
No, I don't mind at all. As a matter of fact, I think you described my situation almost to a T. I had been diagnosed 12 plus years ago, but it was a long time getting there, I had... And it seemed like analyst rounds of pneumonia every time I went in vacation, I get sick about the third day and I'd be running a temperature. It's extreme fatigue. I'd lose my appetite, which is not my nature. And my physician kept looking at it and the history of all the different rounds of pneumonia and being sick on a regular basis like that, and she thought maybe we ought to dig a little deeper on the situation, it actually came to a head one night, I was coughing so much and I could not stop coughing and I cracked a rib and I was in so much pain, I didn't realize where that pain was coming from.
My husband took me to the emergency room, and I think that that coming to a head at that point just made my internist look at it harder and get me over to a specialist. It started with a pulmonary physician who ran me through a whole series of tests, of course, including a bronchoscopy, which finally led to the diagnosis of MAC lung disease, and they referred me to an infectious disease specialist at that point. Back to you, Dr Huitt.
Great. Thanks, Judy. And I would just reiterate that this seems like a story that I've literally heard thousands of times. Unfortunately, including the cracked rib portion of the story. I wouldn't say I've had thousands of patients with cracked ribs, but I've certainly had hundreds unfortunately, and because women are women and have thinner bones, may have a little bit of frail bones like osteopenia or osteoporosis, so fragile bones, and they start having a racking cough this can really lead to some problems. I've even had patients with cracked ribs collapse their lungs, so this really can be complicated, and thank you so much for sharing that because I think it's so important for all of the patients that are out there listening to this to recognize that they're not alone, and that if they're experiencing some of these same symptoms, gosh, it seems like this is something that needs to be assessed by their docs, see if they do have NTM lung disease.
Well, Judy just described a little synopsis of how her doctors proceeded with diagnosis, and ultimately we'll talk about treatment of NTM lung disease, but I think it's important... Again, the theme is empowering you as the patient to kinda push us physicians to help you and help you sooner than later, not have to go 10 years, not even have to go five years to make a diagnosis, so if you think you have these symptoms, you've failed multiple courses of antibiotics, you still have this chronic cough, then think that you may have MAC lung disease and talk to your physician about getting tested. Now, in most cases, I would say you're talking to your primary care doc first and foremost. This is going on, they've been trying the best they can, obviously, everyone at some point in time says uncle and I'm gonna have to pass you on to a specialist, which I would say in most cases, before the diagnosis is actually made, patients get referred to a pulmonologist.
And it's important to make this referral because the longer this disease process is not addressed and treated, you are likely to have more permanent lung damage that is gonna lead to worsening bronchiectasis and structural problems, which will make it much more difficult to treat. So some of the things that you can say are illustrated in the slide, just say, "Doc, we've been trying to deal with this for months or years, can you please refer me to a pulmonologist or infectious disease physician?" So empower yourself to have those services at hand. And so what a pulmonologist is generally gonna do, obviously they're gonna take a history in physical, they're going to get an x-ray and hopefully they're going to get... In addition to a chest X-ray, they're gonna get a CAT scan of your lungs, because the chest x-ray just does not diagnose bronchiectasis very well.
So we really need to go the extra step and see, "Gosh, has there been damage that's already been done," and we can see it visually on the CAT scan picture. Then they need to collect some sputum or something from your lungs, some secretions, so that we can send that to the laboratory and see if they are actually able to grow MAC in the lab, and unfortunately, that takes about, I don't know, six to 10 weeks depending on the lab, and I think that's the hardest thing for patients and physicians, is the length of time that it takes to culture this germ in the lab. But in the meantime, your doctor can do several things additionally, like help you learn how to keep your lungs cleaned out using devices that vibrate your lung and you can at least if you produce mucus and stuff, you can clear your lungs out while you're waiting to start treatment.
And after the six to eight to 10 weeks and your lab calls your doc back and says, "Listen, yeah, we're definitely growing MAC," then the time comes where you and your doc have to talk about, "Okay, we appear to have diagnosed me with MAC, what do we do?" Well, the standard regimen that is used is to use a multi-drug antibiotic treatment approach, and that's usually called multi-drug therapy, and usually it consists of three different antibiotics at the same time. And the reason we use three medicines at least is three is better than two, but theoretically, two would be okay, but three is standard of care, is that we want to attack this bacteria from multiple directions so that the germ does not get smart to it and doesn't develop resistance to any one of the three antibiotics.
So we're sort of confusing the germ, so that it can't develop resistance. And that's very, very important because I would say from a physician standpoint, one of the problems that we see a lot is that doctors may just give you one antibiotic at a time, and yes, it helps you initially, but unfortunately, when you test the germ again two months later after you've been on only one medicine, that germ is now resistant to that one antibiotic, so we can't use it anymore. So it just lessens the choices that we have in a treatment regimen. Well, luckily, physicians have a guideline, they have a document that was revised in 2020, and what this document does is it helps doctors know how they should approach not only the diagnosis of NTM lung disease, not specifically MAC, it talks about many of the non-tuberculous mycobacteria, but how do you test and then what are the recommendations for treatments and for monitoring, if you do decide to go on treatment and you and your doctors feel that that's the right way to go.
This document was developed and published by... Gosh, about 20 physicians and scientists who are in the field of NTM lung disease, and we're very lucky to have had an updated document in 2020 to help physicians. And patients have access to this document too. You can just look it up on the internet. So it's available to anyone. Well, the guidelines recommend starting multi-drug therapy rather than watchful waiting. For the most part, especially if you have evidence of severe infection. And what is severe? Gosh, you have a lot of symptoms, you're coughing, you're fatigued, you might be losing some weight. Let's say you have a compromised immune system, but you're plagued by these symptoms and radio-graphically what we would look at is, gosh, you've got evidence of bronchiectasis now, or in the worst case scenario, you've got something called a cavity, meaning a hole in your lung.
Well, this all indicates more aggressive MAC disease and you better start on medicine immediately. Antibiotics meaning at least three drugs, and not just try simple things. It's time to get started on treatment. Well, the guidelines also recommend that you go to see your physician very frequently, and once they start you on a treatment regimen with the multi-drug antibiotics, they need to check your sputum cultures usually every month or at the most, every two months, because we wanna see if the antibiotics are actually working. I mean, you're going to be able to tell your doctor, "Gosh, yes, I'm noticing a decrease in my cough since I've started antibiotics and I'm having less fatigue." So that's one thing we certainly wanna watch as well, but we wanna see what your sputum cultures are doing, and we'll get to it later, but we're also going to be checking some blood tests on you periodically to make sure that you're not experiencing any toxic side effects from any of the medicines that we have you on.
Well, a lot of patients say, "Well, if I'm gonna embark on treatment, how long is that going to be?" Well, the guidelines say, we want to for sure be watching your cultures and have 12 months of negative cultures. So the clock starts ticking the first time you get a sputum that is culture negative, and we try to go for 12 months after the first culture negative 'cause we wanna be sure that we have killed the bacteria. And the multi-drug regimen actually works for most people, but there is a proportion of people that it doesn't work for, and we have to advance the drug regimen and increase the number of antibiotics that a patient is taking and perhaps consider additional modalities rather than just pills.
So what the new NTM guidelines, the 2020 NTM guidelines strongly recommend, is that adding this new nebulized inhaled antibiotic ARIKAYCE to the underlying multi-drug regimen that you've already been taking, if you continue to test positive after taking the three pills for six months and you still haven't gotten one culture negative sputum. So that's really important. And once you reach this point in your treatment course, there's a lot of discussion that needs to be undertaken with you and your physician, and particularly, we wanna often talk about potential side effects and what to expect when you're taking that. So, Judy, can you share with us what you and your physician spoke about when you got to this point?
Sure. Once I had the diagnosis for MAC... Thank you, Dr. Huitt, by the way. I was immediately put on the multi-drug regimen because I was getting sicker, I almost felt like I was fading away, which is not my nature, I'm a active person. And I really wanted to take an aggressive approach to it, I wanted to knock it out of my system. I felt like if I had gotten this diagnosis earlier in the program, I wouldn't have had the lung damage or possible permanent lung damage that I might have experienced at that time. And going on the multi-drug regimen, I thought that was the answer. I did some research and I also saw that that was really the protocol for the MAC lung disease. And two years ago, three years ago, when I realized that there were multiple re-occurrences and the multi-drug regimen just wasn't going to cut it any longer, it was not a good thing. I was disappointed, it was disheartening, and frankly, it was a little scary to find out that the drug regimen that I'd been using for that amount of time was not really working for me.
So we started to look at alternatives, I had talked to my doctor about ARIKAYCE, where I found a product sheet on a community website, I found some support there. I was looking, kind of searching and researching for myself to see what else might be out there. And I found ARIKAYCE, and I found the product sheet and I took it into my infectious disease specialist. She took a look at it, we talked about it, and she thought like I did, it might be another tool in the toolbox, something he could add into my regimen that might provide a hopeful answer for me. And when it came to the point to make that decision, she helped me find the research tools to be comfortable with the process and to think of the ARIKAYCE as another tool in my toolkit that I could apply to my lungs. So it was a journey, but a successful journey here, getting to the point where we were introduced to ARIKAYCE. Thank you, Dr. Huitt, back to you.
Sure. Well, I think that that is very... Fortunately, that's very typical in what I hear patients that come and see me is they've had the opportunity to look on the internet, there's so much information that's out there. Compared to 20 years ago, it's amazing. So if you have access to internet or a library, if you don't have a computer at home, by all means, try to go and educate yourself. Because honestly, there are a few docs that still need educated regarding this product and how to approach NTM lung disease, 'cause not everyone is as well-versed as they should be. So once again, I think my job here is to empower you, the patient, to, as Judy said, have more tools in your toolbox to be an advocate for yourself and get the best treatment possible. And certainly, what the latest guidelines are showing patients should do.
So let's launch into the antibiotic itself, what actually is this antibiotic ARIKAYCE that we talk about? Well, I will tell you, I've been doing this for 30 years and we have never had an FDA approved, and you've heard a lot about FDA approval lately with the current COVID-19 pandemic. So FDA is our government body that diligently looks at any new medication that's coming to market in the United States. So ARIKAYCE is the first and only FDA-approved treatment option that's been designed specifically for adults who do not respond to a multi-drug regimen alone for six months and have continued positive cultures for MAC lung disease. And again, the important thing to always remember about this drug is that this antibiotic really needs to be added onto an existing multi-drug regimen therapy, so you would never take ARIKAYCE by itself, it's an add-on antibiotic. But an important add-on antibiotic, most certainly.
Well, it's also important when we start any medication, we wanna know about the potential downsides to this that we as the patient may need to watch for, and certainly we as the prescribing physician needs to know exists so that you can have this dialogue with your patient. So, ARIKAYCE can cause serious side effects that include: An allergic inflammation of the lungs, it can also cause coughing up of blood, and we refer to this as hemolysis. It can also cause severe breathing problems, can also cause worsening of COPD or Chronic Obstructive Pulmonary Disease, and has also been known to cause serious allergic reaction. While using ARIKAYCE, these side effects may become serious enough that treatment in a hospital is needed. Call your healthcare provider or get medical help right away if you have any of these serious side effects while taking ARIKAYCE, and that's so important. And also, last but not least, do not use ARIKAYCE if you know that you have an allergy to any aminoglycoside or any of the ingredients that are contained in ARIKAYCE.
So, how do you take ARIKAYCE? Well, it's rather novel actually, it's a liquid and it's an inhaled antibiotic that you take using a nebulizer that's pictured on this slide. It's different from other inhaled antibiotics because it's active ingredient, known as amikacin, is contained inside tiny particles called liposomes. And when you inhale the ARIKAYCE medicine, these liposomes contain the medicine, and they then travel into the lungs to get inside the airway to where this MAC germ is hanging out. And once they're inside of the lungs, they release the medicine to fight the MAC bacteria at the site of infection. And you'll see there's a code on this slide that you can use your smartphone on to see how ARIKAYCE works, it's a video that you can watch at the end of our program if you'd like. But there's an illustration on this slide showing the little amikacin, which is the active ingredient, the antibiotic molecules inside the yellow core of the liposome.
And a clinical study was performed, as with any new drug or new antibiotic, certainly. And this looked at 224 people, and it was ARIKAYCE plus the multi-drug regimen, and it was in people with MAC lung disease who either stopped responding or never responded to their previous treatment. And the key goal was, were we able to culture convert these patients, meaning did the sputum ever go to culture negative by month six? Additional measures that were looked at in this study included: Improvement in the distance walked after six minutes, and improvement in overall health, daily life and perceived well-being in patients with lung disease. And that parameter was a questionnaire. Well, what was important in this study is adding ARIKAYCE clearly helped patients test MAC negative for 12 months and beyond, and as illustrated here, the primary results showed that ARIKAYCE helped three times as many patients test MAC negative at six months or of the 224 patients, 65 tested negative versus those patients who were not taking ARIKAYCE, only 10 of 112 tested MAC negative.
Then when we got to the 12-month time period, which is the end of treatment, ARIKAYCE helped seven times as many patients test MAC negative at 12 months. Or in the initial 224, there were 41 patients who continued to test negative in the ARIKAYCE group and only three of 112 in the non-ARIKAYCE were testing negative. And then most importantly, I would say, they tested for three additional months after all antibiotics were off and looking to see whether sputum cultures converted back to positive and only in the ARIKAYCE patients who had tested negative, and then we continued to watch them, all of them, three months after all drugs were stopped, only the patients that had been exposed to ARIKAYCE were MAC negative (16%). So 36 out of 224 and absolutely zero of the patients who were on the multi-drug regimen alone, so that's a very significant finding that was ascertained from this study. And for those who successfully tested MAC negative, ARIKAYCE also helped more patients stay that way.
And as you can see, at the end of treatment of using ARIKAYCE in the regimen, ARIKAYCE helped twice as many patients test MAC negative at 12 months as opposed to those patients in the gray boxes who had not received ARIKAYCE. And again, only ARIKAYCE patients, the patients that we're exposed to ARIKAYCE, once all antibiotics were stopped, were able to be culture negative after three months. So none of the patients who never received ARIKAYCE and 36 out of 65 receiving the ARIKAYCE were able to stay culture negative three months after stopping all medicine. So once again, this is a very important point that ARIKAYCE really did provide a significant benefit if it was used in your treatment regimen. In this clinical study, they also, as I said, looked at whether or not you improved with a six-minute walk test, and there was none on the six-minute walk test and the St. George's Respiratory Questionnaire measurements at the end of six months also showed no improvement in the ARIKAYCE versus the non-ARIKAYCE group. Judy, you certainly had experience with this. And when your doctor and you talked about adding ARIKAYCE, what was your experience once you added ARIKAYCE?
Thank you, Dr. Huitt. We made the decision to add in ARIKAYCE into the toolbox, and I worked with a very collaborative team. I had an infectious disease specialist at the center of my team, I worked with a pulmonary specialist, pulmonary technicians to measure my process and my progress as we went along at each stage of the game, and I felt that there was an opportunity with ARIKAYCE to make that type of progress that I really wanted. I spoke with my infectious disease doctor at length about different clinical trials, about the supporting evidence that would help me make good decisions about how I worked with ARIKAYCE and what it could really do for me, so we discussed that at length.
And it felt like it was a hopeful tool for me, it was something that might provide an answer. I just thought, why not give it a shot? I certainly needed something different after all the wrongs that I had been through previously. I've been very happy with that decision to move forward, I've had two negative sputums, I'm pretty happy to report that. And I have a third culturing currently, my infectious disease specialist and I did a little happy dance. I was one of her first patients when she came on board at the particular medical society, a group that I work with, and she knew how diligent I was about working towards this goal. So it was pretty cool, we thought... We kind of think we might have had our last appointment, let's see what happens. So thank you, Dr. Huitt.
Well, that's a wonderful story. And I will reiterate that I've heard similar stories from my own patients on many occasions, and I have done a happy dance with some of my patients as well, so I think that's a common theme here. Well, the next phase I think that we wanna talk about is how do you talk to your doctor, and how does your doctor talk to you about ARIKAYCE? And I again, want to have the theme that we are trying to empower you to educate you as the patient, so that sometimes you can educate your physician, you can be their teacher. But know the important questions to ask once you're at this point, when you're thinking about ARIKAYCE and adding it to your already failing multi-drug regimen. So typical things that you would ask is, "Gosh, how do I know whether ARIKAYCE is working for me? What kind of monitoring are you gonna be doing on me? Well, do you think ARIKAYCE would be a good option for me? How long am I gonna take the treatment? And how are you gonna monitor me? Not only with sputums, are gonna get CAT scans? Do I have a log book that I... "
Just all these kind of questions that you and your doc sorta get your plan sorted out so that no one is confused about what you're gonna be doing going forward, and quite honestly, this discussion should happen even if you weren't taking ARIKAYCE. Any medicine that we're taking, we should ask our doctor, "What are you trying to do with this medicine, and how are you gonna monitor me?" So the same thing is applicable with ARIKAYCE. And last but not least, I always wanna talk about potential side effects. So you wanna know the good, but we need to know the potential bad, 'cause everything's got a potential dark side. So your doc and you need to know that ARIKAYCE may cause some serious side effects that include, but are not limited to hearing loss or ringing in the ears, and we commonly refer to that as ototoxicity.
Some people may say... Obviously, you know what hearing loss is. But tinnitus, ear buzzing, whooshing of the ears, ear fullness, like you need to pop your ears. So you have to watch for that. We have to watch for worsening kidney problems, or that's referred to as nephrotoxicity, and that's tested by a blood test called a creatine, generally speaking. So you'll be having routine blood monitoring, and you should have been doing this anyway with your baseline medication. And then in some cases, a worsening muscle weakness may occur, and this is particularly in people with underlying neuromuscular diseases. The common one that people think about is myasthenia gravis, so those are serious side effects that we really have to watch you for.
Now, the important thing is, in the clinical study, we noticed that a relatively high percentage of patients that were just on the baseline regimen, 16%, in fact, 16.1% of patients taking just a multi-drug regimen had some serious side effects, and that increased to 19.7% in patients who had ARIKAYCE added into the regimens. And then other side effects, these are the common side effects, and I'm certainly not gonna go through this whole slide, but I would say bar none, cough, when you're dosing yourself, when you're nebulizing or after you complete your nebulization with ARIKAYCE, is by far the most common theme and hoarseness, I would say would be the second thing that patients complain about. And particularly, this seems to be really common in the first month, but seems to subside, sometimes with a little modification and we'll go through that, but I would say those are the most common things that I've experienced in hearing from my patients that they have experienced it.
And in a clinical study that was done, most patients who experience side effects did not require stopping treatment, they didn't want to stop treatment because they weren't serious enough side effects that they felt like they needed to stop treatment, and 81% of patients taking ARIKAYCE along with their multi-drug therapy regimen did not stop treatment even though they had at least one side effect, and I would say my practice definitely would mirror this. But these are not the only possible side effects that can occur with ARIKAYCE, and so it's really important that you call your doctor or pharmacist for medical advice about side effects if they occur, and you may also report these side effects to the FDA, and the number is 1-800-FDA-1088.
So that's important to remember that you have that outlet to also report to the FDA. But certainly, the thing that you can do as a patient as well, is be sure and tell your healthcare provider about all the medicines that you take, not only the prescription medications that a doctor is prescribing, but any over-the-counter medications or herbal supplements or vitamins, and I'm always shocked, often, at how many of these patients don't report... How many patients don't report that to us, and it's really important 'cause there can be significant drug-drug interactions in something that you would not necessarily think would have a drug-drug interaction. So, two academic centers did a study, via phone, with 26 patients that had been prescribed ARIKAYCE during a two-month period, and this was in the United States, and this...
Insmed was not involved with the conceptualization, development, conduct or analysis of this study, and it's not included in the ARIKAYCE full prescribing information, but they provide writing assistance when the authors were publishing this, through a grant through Insmed incorporated, and what they did is they tabulated the side effects that patients were reporting and what strategies did patients and physicians use when these side effects occurred, and so, when they looked at increased coughing... As you can see, there's some bullet points going down, so oftentimes, they'll give you an albuterol treatment. Sometimes just changing the ARIKAYCE nebulize treatment to the evening. Sometimes just maybe giving it only three days a week instead of seven days a week until your vocal cords and lungs settle down a little bit, sometimes you give cough syrup or have you suck on some lozenges.
Warm salt water, commonly a gargle was very helpful to my patients, I would say, then the next group is the hoarseness or dysphonia, as we call it, and I've only had one patient totally lose their voice and all others of the myriad of patients that I've started ARIKAYCE on, I would say to a person, they develop some hoarseness and it was more significant at the beginning of the treatment. As treatment went on, the hoarseness improved over time, but again, we kinda use the same things for hoarseness with the exception of we don't give an inhaler medicine to help with that. Now, some people do develop shortness of breath or what we say dyspnea, I wanna stop here and say, if you experience anything, you should let your physician know what is going on because they're your partner in this and they have solutions that will help you, but you need to let... We can't help you, or do something about it, if we don't know about it. So back to dyspnea or shortness of breath, they might use an inhaler or Bronco dilator. We might just stop the ARIKAYCE for a few days or a couple of weeks and see if things quiet down and then restart it, maybe slow.
Maybe introducing it, not on a daily basis, but just maybe three days a week for a week, and then increase it to four days and gradually get up to full dosing again. If you're short of breath, we want you to limit your physical activity until you're not short of breath, and in people that are already on oxygen, we may sometimes say, gosh, turn up your oxygen flow from two liters to three liters and see if that will help, and then almost to a person too, I would say when they started the nebulizer treatments, they noticed more sputum would come up during the day because any nebulizer antibiotic is slightly irritating to the airways, and so that does tend to increase your volume, but it's not unmanageable in any circumstance. So, I'd like to toss it back to Judy and see if any of these things occurred in her, and did she have good options for mitigating these symptoms?
Thank you, Dr. Huitt. Yes, I did have a little bit of ear ringing, a little bit of nasal drip, I kind of lost my voice, was very hoarse for about a week, which my husband was a little inappropriately happy about... So, some site effects have benefits, but I really knew what to expect, I had a good... As I mentioned earlier, I had a really good collaborative team involved in my diagnosis and in my treatment. You made a great point about talking with your pharmacist. They were a great resource for me also, and I knew what to expect. I was prepared for it. So, nothing was a surprise to me. I kept a lot of notes and I kept referring back to his notes and I just kept... I had really committed to it, I wanted to make sure I kept after it, and I gave myself the opportunity to really let this ARIKAYCE work for me, and the side effects, they were very manageable, so it was all good. Thank you, Dr. Huitt, back to you.
One of the other things I tell my patients or ask them and plead with them to do is keep a log. So keep a notebook with you, and then always bring that notebook into the doctor's visit because I'd like you to... I'd like to know the day that you started any medicine, whether it's ARIKAYCE or something else. I'd like to know the day you started having a particular side effect, what was the side effect? How close did the side effects start appearing after you took your dose? Those little details can be very, very helpful to me, the physician, to help manage these side effects, so keeping good records, which it sounds like you did, is very important.
Self-advocating and tracking.
Absolutely. Well, let's learn a little bit more about ARIKAYCE now and more, I mean, we've got some patient support that's really important, and honestly is not available to everyone for every new medication that they start on, so I think these support programs have been so helpful to my patients and I've gotten their feedback, so I know how important it is. So, there's the ARIKAYCE Knowledge Moves program, and if you're looking to learn more about ARIKAYCE, then this program is great for you 'cause it provides information and some educational resources, patient stories like yours, Judy, and other patients, and tips on how to have conversations with your doctor, if you're thinking about going to... Incorporating ARIKAYCE use into your drug regimen and you can always visit ARIKAYCE.com to enroll in this program.
And I think the feedback from my patients has been that that program has been hugely beneficial to them at the beginning of their process and through the process when ARIKAYCE was being utilized, and the next step then is if you and your doctor decide to go ahead and start on ARIKAYCE, then there's another support program called the Arikares support program, and this program is once you have been prescribed are case by your physician, this program kicks in and provides you additional information and support, and you'll be assigned a care team that's totally dedicated to helping you along the journey with ARIKAYCE in your treatment regimen, and they help in a variety of ways, from navigating your insurance coverage to using your nebulizer, which is a little different than most nebulizer equipment that you might have encountered up to this point in time, and you can call 1-833-ARIKARE.
And someone is there joining on the spot to help you in this enrollment process over the phone. And, again, to a patient, I have personally had feedback from them that they felt that this was a very supportive program for them once they started on... Or were getting ready to start on ARIKAYCE. And so, on this slide, if you wanna use your smartphone and scan this code to hear more stories from patients about their journey once they started ARIKAYCE, you can listen to that and watch that after the program is completed at ARIKAYCE.com. So... Judy, we've talked about a lot of different things in this program, but now that we're sort of coming to the end in talking about these support programs that are available, did you have experience with any, or utilize any of these support programs?
Oh, Dr. Huitt, absolutely. I utilized all the resources I could get. And it was very helpful to me. I enrolled in the Arikares Support Program, with the onboarding of ARIKAYCE into my program, they were helpful with providing guidance and financial support for starting off on the right foot with all of this, the actual training with the Arikares trainer was really easy. She made it almost seamless and easy to do to get started, and I was ready to receive the information. I had done my homework. So they're all great resource. Arikares coordinator is constantly checking and making sure that you have all the information you need, that you have the proper tools to work with. If you have any questions, you can go back to them with everything... It works out very well for me. I found support groups online to sort of add additional information to my progress to and to the Arikares and how it worked, and that was also very helpful. Community blogs and community resources are just an added benefit to the whole program. Back to you, Doctor.
Perfect. And you said it all. I think providing our patients with the resources to empower themselves, to advocate for themselves, to see whether this is the right product for them, and if it's deemed that it is the right product for them, to get even more information about how to navigate... Navigate this whole journey, because sometimes it can be daunting, let's not lie, and I think it's so important to be empowered and have information. Information is power, as we say. So let's summarize what we've spent this last several minutes talking about, and... So we'd started this program out talking about Mac lung disease and that it can be a serious condition, and the reason it can be serious is 'cause it can cause significant inflammation in the lungs and cause changes to the lung tissue that may be permanent, and that's what we want to avoid. So, we need to be sure what is the status of your lung infection, and do we need to start on treatment right away, or if it's serious, we shouldn't just sit and do as we say, watchful waiting. We should get busy and figure out what to do and what treatment regimen to start you on.
And some people are at greater risk for getting Mac lung disease due to their underlying conditions such as bronchiectasis and COPD that we alluded to earlier. Some people have a cough, little cough... Comes and goes, it's not really so severe, but generally people with Mac lung disease have cough, have fatigue and no response or minimal response to things that your doctor may try, and so that's when you wanna think, surely, "Gosh, do we need to look as to whether I have Mac lung disease?" and if we do decide you have Mac lung disease, then fortunately, we have a 2020 document called The NTM treatment guidelines that tells your doctor and you what the treatment recommendations are to start a multi-drug treatment regimen, if we're going to start rather than watchful waiting, and once you've started your three drug regimen, if after six months, your sputum cultures have not converted to negative, then the NTM treatment guidelines recommend adding ARIKAYCE.
And it's important to know that this was so important in those of us that treat NTM Mac lung disease, that ARIKAYCE is the first and only FDA-approved treatment designed specifically for people who don't respond to a multi-drug regimen alone for the first six months, if you haven't converted your student to negative by that time, it's time to think about adding ARIKAYCE. Now, ARIKAYCE is associated with risk of increased respiratory adverse infections or reactions, including allergic inflammation of the lungs, coughing up blood, severe breathing problems and worsening COPD. Be sure that you talk to your doctor if you have any questions about ARIKAYCE, including possible side effects that may occur, and you can also visit ARIKAYCE.com for more information and support. So, we've had quite a journey together, Judy, and after hearing all of this, and thank you so much, first of all, for sharing your experiences, 'cause I think they're so helpful to patients... They're helpful to me actually. Do you have any final thoughts? Words of wisdom?
Well, I don't know how full of wisdom I am, Dr. Huitt, but thank you for thinking so. I am a big proponent of self-advocating and making sure that you understand the parameters that you're entering into, that you understand what you're taking, why you're taking, what is... You had mentioned earlier what the goals are for both you and your doctor that they have an understanding also of your own personal investment, and what are you willing to commit to to your own well-being to have a potential for successful outcome? That was my investment, my doctor understood I was in for the fight, and I haven't been around this long with this NTM. If we can shorten people's journey to get to this point, it certainly would be impactful for many, many people, and I know that from my own personal experience. I just think, again, it's really important to keep hope here even when you're down for the count, and I think it's even more important to have perseverance top of mind, to keep after it, to keep asking questions, to keep advocating, to seek out the answers.
If you're not getting the answers from your doctor, ask them another way. Ask them to follow a different track or to seek out the answers. I had one doctor look at me and ask me if I wanted to be his assistant, and I thought, "That's kind of a compliment. I don't if I'm irritating him or not, but if we can get to the end goal, that's fine with me." And I just think it's really important, as you mentioned, to track it, to keep a log book specific, you might think the small detail doesn't matter, but it really does. And I think having good people around you to help you get to that end goal is very important, too. Arikares and the whole format and the way that it's put together has been very, very helpful to me personally, Dr. Huitt. I really appreciate... I learned something every day. Thank you.
Well, great. And, all my patients always educate me, and it's so important to the physician community to constantly be challenged, honestly, and educated by our patients as well, because honestly, we can always learn right along with you, and we should always be learning, we should strive for new information. So, I think that's the conclusion of the formal presentation, so I'm going to toss back to our moderator, Nicole.
Yes, and thank you to you both for sharing this important information with all of us this evening, as well as your perspectives. I know this is invaluable to all of us. So, once again, thank you to both. So, this concludes our presentation this evening, thank you again so much to our special guests, and for all of you who joined us today. We have additional programs coming up in the future, so we hope to see you again. Thank you.
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In the Know Program: Taking ARIKAYCE
MAC lung disease specialist, Dr. Gwenn Huitt and Elisse, a real ARIKAYCE patient, discuss topics such as talking to your doctor about adding ARIKAYCE to your multidrug treatment regimen.
Hi everyone, and welcome to today's program. Thank you for joining us for In The Know: Let's Talk About ARIKAYCE. Today's program will focus on taking ARIKAYCE amikacin liposome inhalation suspension, and you'll hear from a leading physician expert and a patient who's taking ARIKAYCE. So before we begin, I did wanna take a moment to share some important information that you should know about ARIKAYCE. ARIKAYCE is a prescription medicine used to treat adults with refractory, difficult to treat, mycobacterium avium complex, MAC lung disease, as part of a combination antibacterial drug treatment plan or regimen. This product was approved by the FDA using the limited population pathway. This means that the FDA has approved this drug for a limited and a specific patient population, and studies on the drug may have only answered focused questions about its safety and its effectiveness. ARIKAYCE is associated with risk of increased respiratory adverse reactions, including allergic inflammation of lungs, coughing up blood, severe breathing problems, and worsening of COPD.
Now, I would like to introduce Dr. Huitt. She's a physician with 29 years of experience, specializing in Infectious Disease at National Jewish Medical and Research Center and NTM Center of Excellence. She's also a professor in the Department of Medicine, Director of The Adult Infectious Disease unit, chair of the Medical Executive Committee and chair of the Micro-bacterial Consult Service at National Jewish Health in Denver, Colorado. Her research mainly focuses on infectious disease, pneumonia and germ theory of disease. Dr. Huitt is a distinguished physician, having published many articles on NTM. She's also the recipient of many awards, such as America's Top Doctor from Castle Connolly Medical limited. We are delighted and honored to have her here with us today as our expert for this program. Now over to you, Dr. Huitt.
Well thank you, Nicole, I really appreciate the opportunity to participate in this program this afternoon, I'd like to welcome everyone here for this program. I'm Dr. Gwen Huitt and I really look forward to the next hour because I think we're going to really get into NTM MAC lung disease and treatment options for this, including ARIKAYCE. I would also like to introduce today's special guest, Elise, who is an ARIKAYCE patient. And Elise is going to be sharing her thoughts and experiences with us today throughout the course of today's program. And so right now I'd like to turn it over to Elise and say, Elise, why don't you tell us a little bit about yourself to start out with.
Thank you, Dr. Huitt. My name is Elise, I live in Florida was my husband. We have two children, a son and a daughter, and we also have a roofing business, which keeps us quite busy. I'm a cat person, I admit this. [chuckle] All our cats are rescues, to be quite honest. But I'm looking forward to sharing my story with everyone, and thank you for inviting me. Back to you Dr. Huitt.
Great. Thanks so much. And we're gonna be tossing this back and forth throughout the next hour between us as we go through this program, so I'll be back to you shortly.
So let's look at today's agenda for the program, and this is the program focusing on taking ARIKAYCE. And a few things that we're going to cover are an overview of MAC lung disease and treatment, what is ARIKAYCE and where does it fit in, talking to your doctor about ARIKAYCE and maybe some scripting we can talk about, getting started on ARIKAYCE. And in the end, we are going to focus on a brief summary of what we've talked about. So let's delve right into what is MAC lung disease and who might be at risk for this? Well, MAC lung disease is a serious lung disease that can certainly get worse over time and cause lung damage if left untreated, it's caused by a group of bacteria commonly found in the environment, in water and soil, particularly in large numbers in those areas. And MAC is the most common form of non-tuberculous mycobacteria, or we shortened that long word to say NTM. And over 80% of NTM are MAC, the cases of NTM are attributed to MAC.
Well, when we... As a clinician... I'm a physician, and so when I see a patient who I've diagnosed and deemed that they have MAC NTM lung disease, we need to come up with a treatment regimen. And the initial treatment regimen is called a multi-drug treatment approach, and we sometimes refer to it as multi-drug therapy or multi-drug treatment. But it involves taking more than one antibiotic at the same time, and usually it's three oral medications as the initial treatment regimen. And the important thing is, is we always wanna use more than one. So at a minimum two, and usually it's three, sometimes it's more than three. But the reason we use multiple antibiotics at the same time is that we want to prevent the bacteria from developing resistance to any of the antibiotics, so we sort of come at... Try to attack the germ from a variety of directions with each of these different antibiotics that act in slightly different ways, so we can not only attack the bacteria and hopefully kill it, but also at the same time prevent drug resistance from developing. Well, the 2020 NTM treatment guidelines strongly recommend adding ARIKAYCE and specifically...
Together and said, Okay, if we diagnose somebody with MAC lung disease, how is the clinician to approach this, and the initial treatment is with this multi-drug regimen, usually at least three antibiotics after the patient is diagnosed as having true lung disease due to this group of organisms, in addition, we recommended or the physicians recommended that the patients have sputum tests every one to two months, and the purpose of getting the repeated sputum test is to see, are you positive or are you negative on the sputum cultures, and obviously, the ultimate goal is to make you sputum culture negative, and if after taking the multi-drug regimen of the at least the three different medications that you are starting with, if by month six, we have not been able to... On your sputum cultures find that you have attained culture negativity, then the 2020 NTM treatment guidelines recommend adding ARIKAYCE at that six-month time period if you still are continuing to be positive for the MAC bacteria.
And it's important to remember in addition to this recommendation, and many other recommendations, but specifically the guidelines, continue to recommend that a patient must stay on treatment for a minimum of 12 months after testing culture negative on their sputum culture, and this is really just to ensure that the MAC bacteria is cleared from the lungs. Well, so as a patient is taking this multi-drug regimen treatment course, it works in most people, and yet there is a portion or proportion of folks that have been taking all the medicines they were supposed to at least three medicines up to the six month time period and they have been unable to get a sputum culture that is culture negative, so it doesn't really work for everyone, the multi-drug treatment regimen, so it's then that the guidelines strongly recommend the adding ARIKAYCE to this multi-drug regimen. And it's important to say, "We're gonna continue those drugs, we started you on, but the ARIKAYCE is now recommended to be added as an additional drug to that multi-drug regimen if you are continuing to test positive at that six-month time period."
And at this point, it's very important that you start this dialogue with your physician to say, Gosh, I've been doing everything I'm supposed to do, what's the next step? And what's the best way to move forward so that we can achieve the ultimate goal of getting to that 12 months of culture negativity." Well, Elise you kinda walked this walk, so I think everyone would really like the benefit of telling us what your walk down the MAC road initially with diagnosis and then maybe what your doc might have suggested at that point in time.
Thank you Dr. Huitt. For two years, I was going for my annual check-ups to my doctor, and I complained of a cough and it was not debilitating or anything, but it was a bother-some cough and she just ignored it and said it was probably allergies. Year two, same thing, probably allergies. Then I changed doctors. Something wasn't right. So my other doctor, my new one, listened to my lungs, she decided to send me for a chest X-ray where she found that the bottom part of my lungs were damaged probably from pneumonia, I may have had in years before I didn't know about, then she sent me to the pulmonologist who tested me and diagnosed me with the MAC infection. He started out with three antibiotics right away because of the condition of my lungs it was not a wait and see kind of thing. He told me that they may not work, he said that it was very, very hard to... hard to fight infection, it was very persistent and that the medications might not work, but I was willing to take a chance and get started on treatment as soon as possible, I needed to do something. When I finished the treatment, I was okay for a while. Then I started coughing again I went back to the pulmonologist who diagnosed me again with the MAC infection, I was disappointed but I really wasn't surprised. At that point, he added ARIKAYCE to my treatment plan and I was ready to fight again with something new. Thank you, Doctor.
Great, thank you. And I think this story, I must have heard a thousand times in my career, where people start with a cough that maybe slowly progresses over months to years, honestly, before they finally go through treatment options, saying it's allergies, "Oh, let's give you inhalers, let's try this, let's try that," until you finally get a sputum culture and some radiograph, some X-rays or CAT scans and say, "Gosh, this may indeed be NTM MAC lung infections," and then get started on treatment. Well, we'll come back to you in a bit Elise, thanks so much for that input 'cause that's very illustrative. Well, we've been... And part of the program is to talk about not only the multi-drug treatment approach that we do as standard starting place, but what is ARIKAYCE and where might it fit into a treatment plan for MAC lung disease? Well, ARIKAYCE is the first and only FDA-approved treatment option that is designed specifically for adults who do not respond to the multi-drug treatment regimen alone after six months of treatment. ARIKAYCE is an add-on to an existing multi-drug therapy regimen, and I just want to really hone in on that it is not to be taken by itself, it's always to be added on to a baseline treatment regimen.
So if you start with the three drug regimen, this will the fourth drug added into that regimen. And the other thing about ARIKAYCE is that it is an inhaled antibiotic and you take that particular preparation with a nebulizer device, a specific nebulizer device that we're gonna talk about later. Well, how does ARIKAYCE work? Well, it's quite unique in the way it works. Its active ingredient is amikacin, but that amikacin is contained inside tiny particles called liposomes, and when you inhale the ARIKAYCE medication, the liposomes containing the medicine travel into your lungs, and then once the medicine is inside your lungs, the medicine is released from the liposome to fight the bacteria at the site of the infection, which is inside the lung, and on this slide, there's one of these QR codes that you can point your phone camera at and take a picture of, and when the program is done, you can go back to arikayce.com and see exactly how ARIKAYCE works in the lung, and I think that's always illustrative for folks to look at.
Well, ARIKAYCE can cause some serious side effects including allergic inflammation of the lungs, coughing up blood, and that's called hemoptysis in medical jargon, severe breathing problems, worsening of chronic obstructive pulmonary disease, or we shorten that to say COPD, and it can also cause serious allergic reactions. While using ARIKAYCE, these side effects may become serious enough that treatment in a hospital is needed, call your health care provider or get medical help right away if you have any of these serious side effects while taking ARIKAYCE, your health care provider may ask you to stop using ARIKAYCE for a short period of time or completely stop using ARIKAYCE. Do not use ARIKAYCE if you are allergic to any aminoglycoside or any of the ingredients contained in ARIKAYCE.
Well, when any new medication is proposed for use in humans, it has to be studied, and so the clinical study that was the cornerstone study for ARIKAYCE involved 224 patients, and in the study patients received ARIKAYCE plus the multi-drug regimen in 224 patients with MAC lung disease, who either had stopped responding or never responded to their previous treatment. A key goal was culture conversion, which means testing MAC negative by month six, patients continued ARIKAYCE treatment for 12 months after testing MAC negative. Additional measures evaluated in the ARIKAYCE study included improvement in the distance walked after six minutes, as well as improvement in clinical health, daily life, as well as perceived well-being in patients with lung disease. Well, this is really, really important. So adding ARIKAYCE help patients test MAC negative for 12 months and beyond, and as you can see from this graph, the purple... dark purple bar is the group that had ARIKAYCE plus the multi-drug regimen, and the gray bar is the multi-drug regimen alone without ARIKAYCE, and what you see is a ARIKAYCE helped three times as many patients test MAC negative at six months, than those who were just in the multi-drug regimen alone. ARIKAYCE helped seven times as many patients test MAC negative at 12 months as well.
And additionally, only those patients that were on ARIKAYCE tested MAC negative three months after completing the treatment. Additionally, for those who successfully tested MAC negative ARIKAYCE helped more patients stay culture negative ARIKAYCE helped two times as many patients test MAC negative at 12 months, and that would be in the purple bar as well. Only people taking the ARIKAYCE tested MAC negative at three months after completing treatment, and lastly, only people taking ARIKAYCE tested MAC negative at 12 months after completing treatment, so you see this had a durable effect going forward when the ARIKAYCE was included in the MAC regimen that had the multi-drug regimen plus the ARIKAYCE. Additional study results that were looked at in this ARIKAYCE clinical study show that there was no improvement in the six-minute walk test and the St. George's Respiratory Questionnaire measurements at the end of six months.
Well, now, it's really important for patients to feel empowered so that they have areas that they can get more information about any product, let alone ARIKAYCE. So fortunately, ARIKAYCE does have a program that you can learn more about it, and it's called The ARIKAYCE Knowledge Moves Program, and this program provides information, educational resources, patient stories, and tips on how to have conversations with your physician or health care practitioner should ARIKAYCE be entertained as a treatment option for your MAC lung disease. And you can learn more about the ARIKAYCE Knowledge Moves Program by going to arikayce.com to enroll. Elise, I'd like to hear what your thoughts were and what your experience with ARIKAYCE was once you guys started on it?
Thank you, Doctor. Well, when I went to my pulmonologist the second time, and he told me he had the bad news and the good news, and I knew what the bad news was, so I said, "Just skip to the good news, I know what the bad news is already." He told me that the good news was that there was a medication now called ARIKAYCE that would take the antibiotic straight to my lungs through a nebulizer. I was thrilled, I was hopeful, I was all on board and ready to start the fight with ARIKAYCE now, he said it was made for people just like myself who had already fought MAC infection and unsuccessfully. So when I got the study results, I was very impressed, I was very encouraged, and when I got my first negative sputum test after starting the ARIKAYCE program, I was thrilled I thought, this is great. So far so good. Thank you, doctor.
Well, thanks so much. And each time you're sharing these little snippets of your journey with us, I think this is probably gonna sound very familiar to many people at least, depending what part of the journey that they're on. But hearing it straight from a patient who's been interacting with their physician about this is really important, and hopefully physicians will be able to view this program as well, so they can learn from patients about the best way to interact with the patient when we're talking about ARIKAYCE and how to move forward.
So in that arena, I think it's important to empower our patients as to how do you broach the subject about, "Gosh, doc, I've been taking these medicines and three drugs for six months and I haven't converted my sputum to negative yet. I heard about this drug called ARIKAYCE, and how can we have that discussion to see whether I'm a candidate for this medication, and should it be added to my regimen?" So one of the things that I think is really important that patients need to know is you need to know, "How will I know if the treatment with all the medications is working after six months?" And that is generally several different ways, but for sure, we're going to get ongoing sputum cultures every one to two months, because again, we're watching...
That clock is ticking. Are we getting to culture negativity? And any of you who have MAC lung disease know that these cultures, the sputum cultures, take anywhere from six to eight weeks to get back, so you're always just waiting for these cultures to come back. And I think that must be one of the hardest things is to... It seems like... It's just you're in the waiting game all the time, but unfortunately the germs grow very slowly, so that's what we're left with right now. But if the multi-drug treatment is or isn't working after six months, perhaps we need to think about ARIKAYCE if it isn't working, and is ARIKAYCE right for me? And if I start taking ARIKAYCE, how will I know if I'm getting better? Is my cough gonna go away? Am I gonna produce less sputum? Just what are we expecting to see that might change? Next, how long is the treatment going to be? How many months or weeks am I gonna be taking ARIKAYCE, and along that course, how are we gonna track that? And again, our goal... Remember the 2020 NTM guidelines instruct us that we're really shooting for 12 months of culture negativity on your sputums.
And how do we track our progress? Well, obviously sputum cultures is the first and foremost thing. As the patient, you certainly need to ask us, "Well, gosh, if I'm taking any medicine, ARIKAYCE or anything else, what should I be aware of? What problems have you heard that might occur with this medication? What should I be looking for?" So ARIKAYCE may cause serious side effects, including hearing loss or ringing in the ears, and we call that ototoxicity, worsening kidney problems, and we call that nephrotoxicity, as well as worsening muscle weakness or something called neuromuscular blockade. So certainly you and your physicians need to be aware of these particular side effects. In the clinical study that was done, 19.7% of patients taking ARIKAYCE plus a multi-drug regimen experienced serious side effects compared to 16.1% of patients taking only the multi-drug regimen alone. The most common side effects of ARIKAYCE are listed, but cough during or after a dose of ARIKAYCE was reported in most patients during the first month of treatment. And I would say I certainly have experienced that with my patients and interacting with them, when they have been taking ARIKAYCE.
These are not all of the possible side effects associated with ARIKAYCE. Be sure to call your doctor or pharmacist for medical advice about side effects. You may report side effects to the FDA at 1-800-332-1088. Also tell your healthcare provider about all the medicines you take. This is huge, and I tell all my patients to always have an up-to-date list of the medicines you take, including prescription medicines, as well as over-the-counter medicines and really, for sure, vitamins and herbal supplements, because we don't know some of the interactions between medicine, so we need a comprehensive list of all the medications you're taking. And keep a list of the medicines you take and show it to your physician each time you meet with them and/or the pharmacist when you get a new medication. This is really, really important. Back to that clinical study. Most patients who experienced side effects did not stop treatment, so they were easily manageable. 81% of the patients taking ARIKAYCE plus the multi-drug regimen did not stop treatment, even though they might have had at least one side effect.
Two academic centers did a study via phone with 26 patients that had been prescribed ARIKAYCE during a two-month period, and this was in the United States, and this... Insmed was not involved with the conceptualization, development, conduct, or analysis of this study, and it's not included in the ARIKAYCE full prescribing information. But they did provide writing assistance when the authors were publishing this through a grant through Insmed Incorporated. Well, this slide is a compilation of four of the major side effects that people commonly complain about in management strategies that we might use to help alleviate any of these symptoms. So in the first column it talks about increased cough, the next column is listed as dysphonia, and basically that just means a change in your voice or change in your voice quality.
The next column is dyspnea, and that just as a fancy word for shortness of breath, and then the last one is showing increased sputum production. And so starting in column number one, there are a lot of choices of management strategies that you and your doctor can go over, and they're listed on the left, but it might be using a bronchodilator or the puffer medicine that you may or may not already be on, sometimes just changing the ARIKAYCE administration from morning to evening will help decrease cough 'cause it lets your vocal cords rest a little bit. You might talk with your doctor about the coughing or change in your voice and your doctor may say, "Well, let's hold off on your ARIKAYCE for a few days and see if this improves just by holding the ARIKAYCE."
Additionally, you can take some cough medicines or throat lozenges, or do some warm water, or warm salt water gargles. And those may be quite beneficial, something just as simple as drinking some warm tea or warm water may help either the increased cough or voice changes. When it gets to the column of dyspnea, or shortness of breath, we may need to use some additional things, we still may need to go back to using that bronchodilator medicine or puffer medicine, and additionally, we may need to stop the ARIKAYCE for a little while, but yeah, sometimes it takes limiting your physical activity, so slowing down a little bit for a couple of days, maybe not running, or having a really robust exercise regimen for a couple of days, and lastly, if you are already on supplemental oxygen, you may need to increase your oxygen use, but again, you're going to be discussing all these issues with your physician and going through with them what your symptoms are, so they can work and partner with you about the best approach to handle any of these certain side effects that might be appearing.
Well, if the treatment plan is established, and you and your doc have talked about your goals and how you're gonna track your progress, you need to establish your new treatment plan that includes ARIKAYCE, and as we've just alluded to in the prior slides, we're going to ask the question, "Well, doc how long do you think I'm gonna be taking ARIKAYCE so that I can feel prepared for what's ahead of me?" Understand your goals of treatment, so you know how quickly you may start to see results, and again, "How frequently am I gonna be checking my sputum cultures with you? How often am I gonna be seeing you in clinic?" Things such as that, and then asking your doc how are you gonna track your progress? And if you have questions that are coming up along the way, "Do you have a patient portal access that I can talk to you or do I go through the nurse in the office or just the best way that I can get a hold of you if I'm experiencing any side effects or benefits for that matter, and I need to communicate with you?"
And then for sure, you're gonna wanna keep track of any side effects that you are experiencing, and what I suggest to everyone is make sure that your time and your date, when said side effect or a thing that we're trying to figure out what might be going on. Give me a time and a date and where was it in relationship to when you might have taken any medication. So keep track of what time you take all your meds on, and not only just a new medicine that might be added to your regimen. Well, so back to Elise, can you share with us how you and your doctor strategized regarding some of these issues?
Sure doctor. I discussed with my doctor the issues of the side effects, and it was quite a conversation because there was a lot to speak about. But when I got my ARIKAYCE materials in the mail, that answered really, all my questions, everything was answered. They answered questions I didn't even think I had, because I didn't understand anything about it to begin with, so it was very, very informative. After reading everything, I was willing to take a chance. I went ahead and got started. After a while, I did get the hoarseness for a while, and I did lose my voice at one point for two days, and then at another point for five days. But everybody's different, everybody has different reactions to every different medication. So if something is going on, then you need to really call your doctor and speak to them about that directly. That's what I did, I called my doctor with questions about anything I didn't understand or anything I was experiencing, and they gave me advice. So that's how I handled it. Thank you.
I think what you've just said is so very important, and I think that if I hear a complaint from patients is, "Doc, I can never get a hold of you in a timely fashion," and so I think as important for physicians to know about new medications that we might be using in treatment plans for our patients that we educate our staff, honestly, our nursing staff, our medical assistants to the best of their ability to... Their knowledge base, because they can actually be very helpful to you, and they're hugely important to us, physicians as extensions of helping our patients manage any kind of side effects that they might be having, so I think establishing, how am I gonna get a hold of you in a timely fashion and get some feedback from you or your office staff regarding my complaints or concerns even, is hugely important, and I think that will give you success for sure. And so it sounds like you had a very good interaction with your physician and office staff to help you manage these speed bumps, as I call them.
Well, now let's move to getting started on ARIKAYCE. So as I mentioned earlier, it's an inhaled antibiotic, and it's taken once daily with a nebulizer that you can see illustrated in the slide. As the patient who's taking this medication, you are going to be responsible for cleaning and disinfecting your Lamira handset and aerosol head before using them for the first time, as well as after every time that you use them. You're going to administer this medication once a day in your own home or, quite honestly, anywhere that has a clean, flat surface. The ARIKAYCE administration time is about 14-20 minutes, depending on your ability to take a deep breath, and you're going to inhale once daily dose of ARIKAYCE around the same time each day. And I think this is important for the physician and the staff who is gonna be helping you on this journey, it's important for us to know that you're taking the ARIKAYCE at the same time each day, so that we can try to pinpoint a particular issue, how it may be related to ARIKAYCE, or maybe related to something entirely different. So if you can stay consistent on when you take the ARIKAYCE, that's very helpful for your physician. And keep the ARIKAYCE, of course, as well as all medicines, out of the reach of children.
Well, what should you expect once a decision is made to start you on ARIKAYCE as part of your antibiotic regimen? Well, when it's time for your shipment to arrive, you're gonna be contacted by your specialty pharmacy to schedule your shipment, and you will need to be home to sign for the shipment so that's very, very important. And you also may need some assistance handling these boxes, which may be heavy. At the start of your treatment, you're gonna receive two shipments from the specialty pharmacy, and that's gonna include the first 28-day supply of ARIKAYCE as well as the one-time shipment of the Lamira nebulizer system. And then going forward, you're gonna receive monthly shipments of the 28-day supply of ARIKAYCE, and that's illustrated on this slide.
Well, there is another support program associated with ARIKAYCE, and it's called the Arikares Support Program, and once you're prescribed ARIKAYCE, the Arikares Support Program provides additional information and support, and you will be assigned a care team dedicated to helping you along this journey. And I can just say from personal experience, every one of my patients who have started on ARIKAYCE have nothing but positive things to say about the Arikares Support Program, 'cause they help them navigate the insurance issues as well as... Just every step along the way. They're there to help you. And so the number to contact to get started in this program is 1-833-ARIKARE, A-R-I-K-A-R-E, and there's somebody there to help you start the enrollment process over the phone. And once you're enrolled in the Arikares Support Program, you're then going to be assigned your Arikares coordinator, your point person, to help you with your journey and to answer questions that you have as you proceed with starting to take ARIKAYCE.
So they're going to help schedule an optional device training with your Arikares trainer, and I would say most people really benefit from this as well. They will work with your specialty pharmacy to verify your insurance coverage, as well as coordinate medication shipments with your specialty pharmacy. They also serve as your primary point of contact throughout your treatment journey, and they provide information about potential savings and financial support options, which is hugely important to most patients as well. Once you are enrolled, you can request an Arikares trainer, and your Arikares trainer is a trained nurse or a therapist who will conduct an in-home training session to show you how to take your first dose of medication and make it part of your daily routine. They will also provide voluntary in-home or virtual device training, and this is important in the time of COVID, I must say. They will also provide ongoing clinical support and information throughout your treatment journey.
Well, a lot of folks that I've seen over the years are always searching for ways to connect with other people who have MAC lung disease, and there are three groups and sites here that you can certainly avail yourself of, NTMir, BRONK, and NTM-360 social, as well as Inspire, and all of these three social supports are ways to connect with other people who are going through the same journey, or certainly a similar journey that you are. Arikayce.com also provides a variety of resources that are listed here, such as important questions that you will need to ask your doctor about ARIKAYCE and how to make the most out of your next appointment. So you can write your questions down and just go bang, bang, bang through your questions with your doc. Tools to help locate a doctor who specializes in lung disease, if you don't have a pulmonologist or a pulmonologist who knows much about MAC lung disease.
They also have real patient and physician stories about MAC lung disease as well as ARIKAYCE, and a printable brochure that provides an overview of ARIKAYCE so you can read and reread it. Also they provide you with ways to connect with others who have MAC lung disease through online communities and support groups. And lastly, they provide information about how to take ARIKAYCE, including the important how-to video, and all these resources are available on arikayce.com. Well, Elise, you, once again, went through this journey and did you avail yourself of any of these support groups or information resources, and did you have any issues with receipt of ARIKAYCE or anything of that nature?
I absolutely enrolled in the Arikares Support Program. It was great. They answered questions that I had along the way. I had a delivery problem once. They got that resolved for me with great detail. It was far above and beyond what I expected. They had answered any questions that I had about the equipment or the delivery or the prescription. It was wonderful. I had someone in my corner to help me every step along the way, and I would suggest it to anybody that is undergoing this treatment. The online support also was very good, 'cause you can connect with other patients. No one else knows what you're going through except for someone else that also has it. And it's not in your everyday life, in my case anyway, that I've ever found anybody else that has this. So it's very important to connect with other people, to give them tips on how to navigate things and to give tips on how to navigate things and to just get some support and a backup. So it was great. Thank you.
Well, I think that patient support groups and any information that a patient can avail themselves of is only beneficial. Now, obviously, the information is only as good as it can be, and I think that's one of the negatives perhaps about some support groups, because it's important to know that everybody's MAC lung disease disease may be different, little differences from one person to the next person. People may have a set of side effects. One person may have a set of side effects, but the other person has no problems, or they may have coexisting medical conditions. So it's very important for every patient to remember you are a unique individual, and it's very important that you have this ongoing relationship with your physician as you go on this road. And these extra perks, as I call them, with the information access are just that, and you should definitely participate in those. But understand that each one of us is very unique in the disease and our journey treatments for the disease. So let's summarize what we've just talked about here. So MAC lung disease is a serious condition. It can certainly get worse over time, and this is generally over months to years, not days or weeks. The standard approach to treating MAC is with multiple antibiotics.
So again, we're trying to get at that germ from different directions and try to kill it, coming at it from several different directions, but at the same time, we're giving multiple drugs because we're trying to prevent antibiotic resistance from developing. If a patient still tests positive for MAC after six months of taking the initial multi-drug treatment, the 2020 NTM treatment guidelines strongly recommend adding ARIKAYCE to their regimen. And ARIKAYCE is the first and only FDA-approved treatment option that's designed specifically for adults who do not respond to a multi-drug treatment regimen alone after six months. The inhaled liposomal formulation of ARIKAYCE delivers the medication directly to the lungs at the site where the MAC bacteria infection is. Adding ARIKAYCE is proven to help patients test MAC negative for 12 months and beyond. ARIKAYCE is associated with risk of increasing respiratory adverse reactions, including allergic inflammation of the lungs, coughing up blood, severe breathing problems and worsening of COPD. It's very important to talk to your doctor about establishing an ARIKAYCE treatment plan and planning for any side effects. Once prescribed ARIKAYCE, the Arikares Support Program provides additional information and support. Visit arikayce.com for more information and support.
Well, now that we've come through the summary, Elise, what type of closing thoughts might you have to anyone who's out there and thinking, "Gosh, maybe ARIKAYCE is something I need to discuss with my physician, or I've already made a decision, and I'm getting ready to start, or I've already started?"
Well, in my case, I think that if I was diagnosed earlier, it would have been very helpful. I would have started treatment earlier. Maybe the disease wouldn't have gone to both my lungs or been as prolific as it became. So I would suggest to people to be advocates for themselves. To advocate to your doctor, push them and get the answers to your questions and repeat the questions until you get the answers you need. And when you do start treatment, don't skip. Be persistent. Put it on a schedule. Do it the same time every day. It's much easier that way. That way, you don't skip and you... It's in your routine. That's very important, I believe. For me, I would do it after dinner or before bed, in that time period there. And if you're not getting results after six months of the multi-drug treatment, I would suggest for anyone to ask their doctor about ARIKAYCE. Thank you.
Thank you, Elise. And I tell my folks, the squeaking wheel does get the grease. And so your point of being your own advocate is huge, and that's certainly one of the main reasons forums like this are out there, to allow patients to be the squeaky wheel and get the grease, which is the answers that are certainly out there. Whether it's a patient support group or directly from your physician's office, your physician themselves or the staff in the physician's office, we need to empower you to be able to move forward in your treatment of MAC lung disease. So thanks so much for sharing your journey with us 'cause it's so important. So I guess this closes the formal presentation, and I guess I'm gonna turn it back to our moderator right now.
So this concludes our presentation this evening. Thank you again so much to our special guests, and for all of you who joined us today. We have additional programs coming up in the future so we hope to see you again. Thank you.
Quick Start Guide
Refer to this guide for help getting started with your ARIKAYCE treatment and Lamira® Nebulizer system.
Lamira Nebulizer System Instructions for Use
Comprehensive instructions on how to use and care for your Lamira Nebulizer System, with directions and techniques to deal with situations that may come up during use.
Create Space for Treatment
Create a plan designed to help you decide when, where, and how to fit your treatment into your schedule.
Side Effects Management Survey Findings
Discuss with your doctor how you may be able to manage the most common side effects of ARIKAYCE, including the strategies found here.
My Daily Planner
Keeping track of your journey is helpful for you and your doctor. This resource will guide you in recording your symptoms, progress, and goals.
Doctor Visit Checklist
You may have a lot of questions during your treatment journey, and we want you to be armed with the information you may need. Use this tool to be prepared for your treatment journey.
Reduce MAC Exposure at Home
Uncover how bacteria can go unnoticed in your home. Learn how to prepare your home environment which may help reduce exposure to MAC bacteria.
Checklist for Traveling With Medication
Get tips for how to stay on track with ARIKAYCE treatment while you’re away from home.
The Prescribing Information for ARIKAYCE.
Do you need the Prescribing Information in another language?