ARIKAYCE targets the MAC infection directly in your lungs

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.