Hear from real patients about getting MAC-negative

My name is Elisse.

I live in Key Largo, Florida, with my husband. We have two children.

MAC lung disease impacted me before I was getting treatment in a great way.

I was coughing all night. It was miserable because I was exhausted and so was everybody else. It was, it was very disruptive to the…to everyday life.

My journey to diagnosis started a few years before I was diagnosed.

I went to my family doctor for annual checkups. For several years, I complained of a cough or persistent cough.

Every year she would tell me it was allergies...never really did anything about it. So when I changed doctors, my new doctor told me that she listened to my lungs, didn't like what she heard, sent me for a chest x-ray and it came back that my lungs were damaged on the bottom, probably from pneumonia that I may have had.

My pulmonologist diagnosed me and told me I tested positive for MAC. I was bewildered. I had no idea what it was. I had no idea where to turn.

Having a MAC diagnosis sooner I think would have definitely helped me. The disease may not have progressed to both lungs. It may not have been as severe as it was when it was diagnosed, and I would have been able to start treatment earlier.

After the discussion with my doctor about the multidrug regimen, I was ready to fight.

I was on multidrug treatment for MAC disease for 12 months. After I came off multidrug treatment, for a while I was fine and then I started coughing. So I went back to my pulmonologist who again diagnosed me with the MAC infection.

I was disappointed.

The doctor told me that the good news was that there was a treatment available for people such as myself.

And it was an antibiotic that was inhaled and went directly to the lungs.

I would need to take it in addition to the three antibiotics I was already taking.

So he gave me some hope.

I began taking ARIKAYCE...and I was empowered a little bit because I was ready to fight again. I tracked my progress through the sputum tests and the CT scans.

When I had my first negative sputum test, I was thrilled. I said, well, this is great.

I was prepared for the possible side effects for treatment with ARIKAYCE. I had some hoarseness in my voice for a few days. I lost my voice for about five days. Everybody's side effects are different when taking ARIKAYCE. So I would suggest for people to speak to their doctor if they're having any type of side effects.

The support I received from my doctors is when I call them, if I have a side effect or something's going on that I don't understand or that's uncomfortable, I'll call them and they'll give me advice on what to do.

I've gotten support through online support groups, which helped me a lot.

I am enrolled in Arikares Support Program. It's very helpful, and they answer questions and solve problems I may have. My Arikares Coordinator keeps in touch with me concerning my ARIKAYCE treatment. She checks on my refills, if I need anything, if I'm having any problems, and she keeps a line of communication open. It's great to know that I have someone in my corner.

If someone isn't getting answers from their initial doctor, I would persist in asking the questions.

I do have to advocate and ask in-depth questions...detailed questions until I get the answers I want.

For MAC patients that have not seen results on other multidrug treatments, I would suggest they ask their doctor to look into ARIKAYCE for them.

Life with ARIKAYCE has made me more hopeful for the future...and I'm looking forward to great results.

My name is Lynn, I’m 67 years old and I live in Pearl River, Louisiana.

My name is Dr. Nicole Lapinel and I’ve been in pulmonary critical care for the last few years.

Lynn had had a long path—diagnosis, you know, many years before she had actually met me.

When the infectious disease doctor became aware that I had NTM, she told

me about a doctor who was involved in treating NTM, which is Dr. Lapinel.

I was very impressed the first time I met you, I was impressed with the

abundance of knowledge that you had about this illness.

That makes me feel very, very good to hear that. Very comforting for me as a physician.

My first visit was involved. I was a bit intimidated and overwhelmed at this point because it was just so much information for me to absorb.

During that first appointment, I always try to get as much of that

background history as I possibly can. So, all of that is important in terms of setting our pathway, setting our treatment regimen up and everything like that, getting more of that history. And I hope that even though it was overwhelming to kind of hear all that, that you had a sense that we are going to be taking this kind of step by step.

We had put you on a daily regimen and discontinued one of the antibiotics that you were on and then added two more antibiotics to that regimen on that very, very first visit.

I wasn’t excited about taking more medication, but I felt hopeful for the

first time in a long time of being treated properly.

When Dr. Lapinel told me that my sputum samples were still coming back

positive, I was disappointed. I had a lot of hope that I would see more of a result at this point.

Treatment can potentially not be successful for a variety of reasons. That is something that we also talk to our patients about in terms

of managing expectations.

I knew there was a possibility after six months if I was still

producing positive sputum samples, that we were going to add to the medication I was already taking.

So, it was actually six months into your regimen when cultures were

still positive that we actually decided to add a new medication, an inhaled antibiotic, which is called ARIKAYCE.

Knowing that ARIKAYCE was available for people like me, made me

feel hopeful that I could get this disease under control.

I felt confident that this was the appropriate addition to the regimen, because for the first in its time for MAC lung disease, we actually had an evidence-based approved medication that was also FDA approved.

Whenever we’re introducing new medications, we like to go over those side

effects because that’s…that’s part of handling the treatment. I always discuss cough because so many patients are already suffering with cough.

So, I was prepared for just what she said, the cough, the hoarseness, and the shortness of breath. But she also gave me ideas on what I could do to make it better.

I did keep track of my side effects. I took personal notes so that when

I did see you I could relay them to you and not leave anything out.

I enrolled in the Arikares Support Program. They have been very, very

helpful to me. Someone has come out to my house initially to educate

me on how to use the medication. If I had a problem all I had to do was

make a phone call and somebody was always there to help me immediately.

I’ve actually heard truthfully from many patients that they’re very satisfied with the support that they provide through the program.

We started to see that your sputum cultures started coming back negative

within that first six months of treatment on ARIKAYCE. And so that was a very welcoming sign that we were finally getting the effects, you know, that we had hoped for.

When I found out that my sputum sample was negative after taking ARIKAYCE, I was very excited, and I was very grateful.

You finally got to the point of actually seeing that progress from having

consistently positive cultures to turning to negative cultures.

I appreciate you, Dr. Lapinel. You made sure that I got all of the

proper treatments. You didn’t give up when one of them or three

of them didn’t work, you added to it to make sure that we continue to try to fight this illness.

I appreciate you, the commitment, how seriously you’ve taken this. And just overall being just a wonderful, agreeable person to, to kind

of go with on this journey.

I think that all of the work that we’ve done, all of the treatments that she has recommended and put me on, everything through all of this time has been so successful.

My advice for someone just starting ARIKAYCE is to be positive and take one day at a time. As of today, I’m still MAC-negative.

I feel good about the future.

I’m on this quest for knowledge to get answers for both you and me. Join me as I learn more about MAC lung disease and ARIKAYCE, from a researcher, a doctor, and a patient living with this disease. Let's get moving.

Our first stop is at the Insmed Laboratories to learn about the science behind ARIKAYCE.

Now the goal of Insmed, really, is to develop new therapies to treat patients with rare diseases.

Like me.

Yes exactly.

Cool.

And ARIKAYCE represents the first product that came out of these laboratories that we're going to see.

Perfect.

OK great.

And here we go.

Yep so we're entering one of our laboratories.

So cool.

So why don’t you grab a seat.

Alright thank you.

Sure.

So David who's ARIKAYCE for?

People with MAC infections. Before ARIKAYCE once they've exhausted other treatment options if they're still culture positive there's nothing else for them.

Sure.

Now, with ARIKAYCE, it gives them more hope because they can try ARIKAYCE and [it] gives them an opportunity to become MAC-negative.

MAC-negative, that's the goal.

That's right.

I remember when ARIKAYCE was first FDA approved in 2018, that's a big deal, right?

It is, it was a huge moment for us and I was here, I remember that day very well. People were jumping up and down, there was so much excitement and it really is a reflection of the service of the people here, the hard work thinking about the patients, and and it's very rewarding to know that it's finally going to help patients.

What makes ARIKAYCE different?

Well what makes ARIKAYCE different is it's delivered directly to the lung, the site of infection. Our formulation of ARIKAYCE uses liposomes. It's like a tiny ball.

OK…

You can see here, this is kind of like what it looks like.

Right.

But it's very very tiny, and inside the liposome is where we package all of the drug - you can see here.

May I?

Please. So the purple represents the drug molecules, the amikacin.

How does it get delivered?

So this is really very different from what you're used to, taking an oral tablet, for example.

Right, OK.

And how it gets delivered, is using a nebulizer and I'll show you that system.

OK cool.

So we have here…

OK.

The nebulizer, you’ve used it many times, and this was specially designed for ARIKAYCE.

So it's a combo approach, the liposomes and the nebulizer are what helps ARIKAYCE get to the lungs.

Exactly, so you see here, this aerosol mesh…

OK.

You can't see it, but there's thousands of holes inside here. They're very very tiny, and that's what creates the aerosol.

So why mesh?

So what the mesh does, is it pumps the liquid through these holes so you're creating droplets simultaneously and that creates a very high-density aerosol, so this is a very unique design specifically for ARIKAYCE.

May I?

You may, this is called the Lamira nebulizer system.

Cool. Pretty cool actually taking it all apart and studying it and reading up on it. It wasn't as complicated as I might have thought it was initially, but I'm still impressed with it.

You took it apart and put it together very quickly.

I probably put it --

Muscle memory.

Yes. For me, I wanted to like it. I wanted to want to do it, so I bought myself a nice tray so that I had the right tools and I had tools that were attractive to me, I wanted it, I wanted it to look nice. Who wants to be reminded they're sick? I didn't want that, and I know when I have to look at it that way. I wanted it to be attractive and I wanted to want to use it. It just made it easier for me mentally to listen to my inner self and know that I could do it. And I – I’m getting choked up a little bit… to know, that your researchers and your scientists, put so much into this. So thank you. Sorry, I'm sorry. Wanna give me a hug?

The mission of Insmed can be summed up as, simply for the patients.

Walking around here, you can't help but see that mission come to life. It's touching and impressive to learn so many people have dedicated their time to developing ARIKAYCE, for those who had nothing else after their initial treatment.

Let's see what we can learn at our next stop.

Today, we're meeting with the pulmonologist in New York who's been treating MAC lung disease for over 9 years. Although she was never my doctor, she's someone I've had the pleasure of meeting a few times before this.

One of the ways that I think I help my patients the most is by telling them that I really like to take a, a team approach to their management of care. My entire team is available, as well as myself, to be able to assist in any questions that arise, any complications that potentially may arise, so that we can make our patients feel that even though, this might be a long and complicated journey, that we have people that understand, that are informed about what they are going through, and are here to help every step of the way.

I think the whole diagnosis of MAC lung disease and NTM can be a very scary journey especially when you're first embarking on this and I, I sure wish I had known more at the beginning. That would have helped me along the process a little more quickly. I'm not even sure I could pronounce the name of it fluently the first few months. I would imagine, Dr. Lapinel, that it's sometimes challenging to measure how much a patient is really ready to hear. So, in your particular practice, how much how do you gauge that and how do you inform your patients so that they feel cared for and supported in this journey?

That's a wonderful question, right? Because everybody does come to the table having different backgrounds of information, everybody has different journeys, has different awareness of their underlying disease, and you want to inform people, but you also don't want to overwhelm them, um, for concern of, they may take a step back, but overall, um, you know I like to give a basic understanding of what the disease is, um, what the potential course may be, and that while we have different treatment approaches, it's tailored to that individual patient.

I sometimes hear from other people that they don't want to start MAC treatment right away, what do you think?

I really feel that the decision to start treatment is one that needs to be made individually. In many patients as we've learned more about the disease, I caution them that if they are not started on treatment, that it can lead to a worsening of their underlying lung disease over time. And so to that point, I follow the NTM treatment guidelines that recommend treatment over watchful waiting. And the goal of that treatment is to test MAC-negative. The guidelines do in fact discuss the length of treatment and they usually mention that patients should remain on treatment for at least 12 months from the time that they first convert to culture negative. So, somebody that has, had been MAC-positive converts to MAC-negative they must continue treatment for at least 12 months from that point. We want to do everything that we can, to help treat it, and clear it, and prevent it from coming back.

When you say MAC lung disease is difficult to treat, like it was for me, you probably have other patients that it's the same?

And that's why for my patients, that I do decide to start on treatment when we discuss the oral antibiotic regimen that we're putting them on, I actually bring up ARIKAYCE early on in their diagnosis. We are going to follow the guidelines and treat with proven therapies, so the addition of ARIKAYCE is strongly recommended for patients that continue to test positive after six months of treatment. These are points that are really important to know and so I really encourage patients to speak with their physicians when they're embarking on their treatment journey.

So when you found out that ARIKAYCE was approved what was your reaction?  

I was very happy, there was almost a sense of relief. Now we have this drug that's FDA approved, in a limited population, for patients that continue to test positive after six months of a multidrug treatment regimen.

I think a lot of patients want to know what are the chances that ARIKAYCE is going to work for me?

When patients ask me that, I like to refer them to the data from the study on which, for which ARIKAYCE gained you know its FDA approval. So let's head inside to my office so we can review some of the clinical data so I can answer your question a little bit more clearly.

Thank you I'd like that.

It was so interesting to meet Dr. Lapinel and to hear about her range of experience.

Many patients just wanted to know what we could experience on this journey. It was refreshing to hear doctors also find this disease frustrating. After all, we're in this together.

Let's see what we can learn at our next stop.

We're here with Dr. Lapinel, pulmonologist, in New York. Today, she's taking us through the ARIKAYCE study and discussing the potential side effects. The place for that conversation is here in her office.

Dr. Lapinel, I know a lot of patients want to know about ARIKAYCE and how it might affect them specifically. Can you share some of that with us?

Yes, so I think that's a very important question. You know everybody comes to the table and wants to know how is this going to work for me? And I think to best illustrate that point it's important to look at patients who had actually participated in the ARIKAYCE study and see what their experience was. ARIKAYCE was studied in patients who had already been on a multidrug regimen and still had maintained culture positive for MAC after six months of treatment, so they look to see if ARIKAYCE was added to guideline-based therapy, if more people would test negative for MAC, versus people who were just maintained on guideline-based therapy. They also studied how long people maintained MAC-negative cultures after completing treatment. They also included some additional objectives in this study so they looked at improvement in the distance that people walked over 6 minutes, they also looked for improvement in overall health, daily life, and perceived well-being in people with lung disease as measured by a questionnaire. Overall the study showed that adding ARIKAYCE helped more people test MAC-negative and remain MAC-negative even after completing treatment, compared to people who did not add ARIKAYCE to their treatment.

And the charts get into the details of how many people tested MAC-negative at each time point, right?

Yes, that's correct. So if you look at the purple bar at six months, and the gray bar at six months, you'll see that 29% of people that were on ARIKAYCE with guideline-based therapy tested MAC-negative compared to 8.9% that were not on ARIKAYCE.

So 29% compared to less than 9% after six months, is that right?

Yes, so that's three times as many people that tested MAC-negative after adding ARIKAYCE.

Wow, that's pretty significant.

So Judy if you look here, at 12 months, 18.3% versus 2.7% of patients tested MAC-negative after adding ARIKAYCE, that’s seven times as many patients.

When I first heard about ARIKAYCE and I saw this study it was really encouraging.

We didn't have an approved treatment before ARIKAYCE for this patient population. So this data was really great to see and it meant that we had a potentially great tool to combat the disease. So, Judy, one other thing that I like to point out, is that if you look at the end of the study which was three months after completing treatment, 16.1% of patients that were on ARIKAYCE versus 0% of patients that were on [a] multidrug regimen, remained MAC-negative.

Wow, but I'm sure some people test negative without ARIKAYCE?

Yes, it's important to remember for many patients that are started on initial treatment, actually 2/3 of people will successfully complete treatment. ARIKAYCE was studied in the 1/3 of patients who don't get results with that first treatment.

Dr. Lapinel, what are the chances someone will test MAC-negative after six months with just multidrug treatment if they haven't already?

So the chances are slim, and for that reason ARIKAYCE is my go-to treatment.

So I think the question people have, when they look at this data is, how many people in this study stayed MAC-negative?

So, Judy, that's a wonderful point. There's actually an additional analysis that was done to determine how many people that tested MAC-negative remained MAC-negative over time. This analysis found that after completing treatment ARIKAYCE kept working, and you can see that here.

So, if I'm reading this right, the first bar says ARIKAYCE helped twice as many people test MAC-negative at the end of treatment.

Correct, and if you followed the time points you'll see that three months after completing treatment, and 12 months after completing treatment, 55% and 46% of people who tested negative with ARIKAYCE, were still testing negative compared to those who took the multidrug treatment alone just at 0%.

What about those additional study results?

There is no improvement in the six-minute walk test, or the Saint George's respiratory questionnaire after six months of treatment.

When I was going through this, the data is what my doctor and I used to help map my own journey.

And I think it's very helpful.

We've talked a lot about the different conversations you have with patients, what are the things you tell them that they may experience with ARIKAYCE?

Something that I'd like to share with all my patients, no matter what, is the potential side effects that they may experience.

Yes, that was one of the first questions I had when I started treatment.

So I think everyone does, and I always tell my patients about the more serious respiratory side effects that they can experience with ARIKAYCE. So ARIKAYCE can be associated with allergic inflammation of the lungs, coughing up blood, severe breathing problems, worsening of chronic obstructive pulmonary disease or COPD, as well as more severe allergic reactions. So speaking of allergic reaction, ARIKAYCE is a type of antibiotic known as an aminoglycoside. If you're allergic to that type of drug or antibiotic or any of the ingredients, you should not use ARIKAYCE. ARIKAYCE is also associated with hearing loss or ringing in the ears, worsening kidney problems, and worsening muscle weakness.

Do people who add ARIKAYCE experience serious side effects?

We actually see in the study 16.1% on the multidrug treatment alone had these serious side effects compared to 19.7% of people who added ARIKAYCE.

What about the common side effects of ARIKAYCE?

I like to cover those, because you don't want people to be caught off guard with things like changes in their voice or hoarseness, which we see was experienced by 48% of people in the clinical study. Cough is another common one, reported most often during the first month of treatment, it was experienced by 40% of people who took ARIKAYCE. There's also muscle pain and sore throat experienced by 18% of people who took ARIKAYCE. So the one I also mentioned is increased sputum production, which happened in 6%, for people who are already symptomatic from a respiratory standpoint, it's obviously very important to mention that common symptom.

Yeah when I was on ARIKAYCE it wasn't really the cough, but a little bit of post-nasal drip and some ear ringing.

So everybody is different, and patients may have different side effects. And I emphasize it is important to speak with your physician about them. I don't want to hear three months from now about any side effects that one is experiencing because we want to make sure that we can intervene as early as possible.

Do patients ever say, “Alright, now that I've started this new treatment how long until it starts working?”

They do, and in the ARIKAYCE clinical study people who tested MAC-negative were on treatment for one to four months before they received their first negative test.

From experience I had to ask about these things multiple times to really understand them.

And that's OK, many people do. What I want patients to know, is that we're there for them. From a communication and a support standpoint, to help them get what they need.

I really appreciate you sharing your knowledge and information about ARIKAYCE in particular. It's meant a lot to me, thank you.

It's been my pleasure.

The question I needed answered most in my treatment journey: “What's next?” I needed someone to tell me what I could experience and how to manage the side effects along the way.

If there's one bit of advice I can offer to others, it would be to make sure they have the right doctor, who can guide them every step of the way.

Let's see what we can learn at our next stop.

Throughout my MAC treatment journey, I found that it's a rare but wonderful thing to have someone who really gets you, someone who can walk a mile in your shoes, and you in theirs. That someone for me is Elisse.

Hi, this is so great –

Oh my gosh –

(mixed dialogue)

Come in, come in.

Thank you.

So how was your trip?

It was great, thank you again.

Thank you for coming.

Cheers.

I wonder if you could share with me how it felt when you were diagnosed with MAC lung disease.

Oh absolutely. I was shocked. Honestly, it was like a deer in headlights. My primary care doctor took an X-ray of my lungs because she didn't like what she heard. When I saw the X-rays, I could see the damage she pointed out. I already had damage to both my lungs, and there was a great sense of urgency, to get started on treatment right away.

So you were on an initial multidrug treatment for MAC, did that work for you?

It did for a while, I did the treatment and once I was done, the initial multidrug treatment, I was OK for a while.

Could you tell me more about how you heard about ARIKAYCE?

I actually heard about ARIKAYCE from my pulmonologist. I was hopeful that this would help me test MAC-negative, and it did so I was thrilled.

When you were first diagnosed with MAC, what was your experience with your physicians?

Well, my first experience with my infectious disease doctor, uh was not great. I got no information from them, I would ask questions get one-word answers, it wasn’t working, so I called my pulmonologist to ask to find somebody else - which he did, and it was a completely different experience with someone else. I got my questions answered, we had a rapport, I think it's very important to find a doctor that you can work with.

Did anyone give you a bird's eye view of this situation?

I did not have an overview of the whole thing, of where I was going to be going and how long it was going to take to get there. They did tell me that the first time, when I was MAC-positive, my treatment was going to be 12 months. And the second time I was MAC-positive, my treatment was going to be for 18 months, and that was with the ARIKAYCE.

My experience isn't all that dissimilar. It would have been very helpful if someone had just helped me set the expectations. One of the big things that people try to manage is the side effects, how did that work for you?

I actually had hoarseness in my voice a few times and I actually lost my voice twice for a few days each time.

Mine were very similar. I also lost my voice, I kind of expected it. I had gone through the side effects with my doctor, I had a little bit of nasal drip, even like the blogs and the forms I was on, it was helpful in trying to manage all of that. I know that this can be a long journey, what would be your best advice to people with a diagnosis of MAC lung disease?

I would advise them to remember the doctor is there for you and your health, don't be afraid to ask questions.

Meeting up with other people, someone like Elisse, who also has NTM MAC lung disease, and has traveled the same path with me, helps a person not feel alone. You're not alone on this island. Not only with finding ARIKAYCE, but also finding other tools and other support such as good physicians, as a good team that you're working with.

We all know rare diseases can be lonely diseases, but they don't have to be. Here's what I wish someone would have told me: Remember to advocate and ask all your questions, it's a long journey but there’s support to help you persevere. I always say, the best way to get answers is to have honest conversations. On this quest for knowledge I saw the care that went into designing ARIKAYCE. I learned having the right doctor for you helps you on your treatment journey, and a friend reminded me, I'm not dealing with this disease alone.

To the patients watching, stay hopeful. Yours truly, Judy.