SKYCLARYS and
Friedreich ataxia
(FA) resources

Recorded Voice: SKYCLARYS^® (omaveloxolone) is a prescription medicine used to treat FA in patients aged 16 years and older. It is not known if SKYCLARYS is safe and effective for use in children younger than 16 years of age. SKYCLARYS may cause serious side effects including an increase in blood liver enzymes in your blood, increase in a blood protein called B-Type Natriuretic Peptide (BNP), which tells how well your heart is working, and changes in cholesterol levels. Your doctor will test these levels before and/or during treatment with SKYCLARYS. The most common side effects of SKYCLARYS include increased liver enzymes, headache, nausea, stomach pain, tiredness, diarrhea, and muscle pain. Before taking SKYCLARYS, tell your healthcare provider about your medical conditions such as liver or heart problems including heart failure or high levels of fat in your blood, high cholesterol, are pregnant or plan to become pregnant, or are breastfeeding or plan to breastfeed. Tell your healthcare provider about all the medicines you take including prescription and over-the-counter medicines, vitamins, and herbal supplements. Taking SKYCLARYS with other medicines can cause serious side effects. SKYCLARYS may affect the way other medicines work, and other medicines may affect how SKYCLARYS works. Do not drink grapefruit juice or eat grapefruit. These may change the amount of SKYCLARYS in your blood. These are not all the possible side effects of SKYCLARYS. For more information, ask your healthcare provider or pharmacist. Visit www.skyclarys.com to obtain the FDA-approved product labeling. You may report side effects to FDA at 1-800-FDA-1088.

Patricia Stark: Hello and thank you for joining me. My name is Patricia Stark. We're here today to talk about SKYCLARYS, the first and only FDA-approved prescription medicine for Friedreich's ataxia or FA. Now, before we dive in, I'd like to introduce my guest, Matthew Lafleur, a columnist for Friedreich's Ataxia News. For the last 5 years, Matt has been writing about his life with FA, and during that time, he has become a fixture in the rare disease community using his platform to reach people all over the world and inform them about some of the issues facing people with FA and those who care for them. Matt, you have written dozens of columns about living with FA, but there is one article in particular that I'd like to talk about because I love the metaphor that you used. It's called "Betting on Dark Horses." Now, in sports or in politics, a dark horse is someone who's maybe a little unknown but who unexpectedly comes from behind to succeed. In your view, how are people with Friedreich's ataxia like dark horses?

Matthew Lafleur: Thank you, Patricia. FA is still a pretty unknown disease for most people. People who have it themselves, they also face random symptoms that vary from person to person, so in FA, we're all kind of familiar with the unknown. I think we can deal with that unknown in 1 of 2 ways. Either we can dread it and live in fear of further disability, or we can remain hopeful and optimistic even when we don't know what's coming, kind of like placing a bet on a dark horse. I think we need to believe in our chances in the face of an unknown future.

Patricia Stark: And you're a bit of a dark horse yourself, Matt, as today you're a successful columnist with Friedreich's Ataxia News, but in your bio, you say that your life doesn't look like you imagined it would when you were a young Cajun boy growing up Opelousas, Louisiana. So, how old were you when you were first diagnosed with FA?

Matthew Lafleur: I was in elementary school so about 9 years old, but honestly, I was just a kid, so I didn't really pay much attention to what it was. I expected there to be a surgery or maybe a medicine that I could take and everything would be better.

Patricia Stark: And sports played a big role in your life at that time, right?

Matthew Lafleur: Yeah. It feels weird to say that now, but back then definitely. I played almost every sport there was. Every season I was playing a different sport. It was really a big part of my identity back then, but after I was diagnosed with Friedreich's ataxia, I was getting weaker and weaker and weaker, and eventually, in junior high, I had to quit playing sports. I didn't understand why, so I asked myself why am I getting so bad? And I didn't really have an answer, so I was really just hard on myself.

Patricia Stark: It must have been difficult to transition away from something you loved at such a young age.

Matthew Lafleur: Well, it really wasn't much of a decision. I was kind of forced into making it because I couldn't keep up with the rest of my teammates. Since most of my friends were in the sports world, it's very difficult and lonely feeling left behind. When I got into high school, I decided to learn more about Friedreich's ataxia. I looked it up, and on the one hand, it was really liberating because I realized that it wasn't my fault that I was getting behind in sports, it was this diagnosis. But I also found out that there was nothing I could do about it. There was no treatment and no cure for it, and eventually, it means that I would end up in a wheelchair, and finding that out that destroyed me.

Patricia Stark: So, then how did you cope with being forced to take such a dramatically different path in your life?

Matthew Lafleur: Even though I can no longer do sports, I kept on doing well at school, so when I first went into college, I was going to be an English teacher. But I realized pretty quickly that I'm not very extroverted, so I didn't really look forward to getting up in front of kids every day and having them judge me and whatnot, so that wasn't for me.

Patricia Stark: So, here was another transition that you were forced to make.

Matthew Lafleur: That's right. I had to reassess what I wanted to do by being a teacher. I loved literature, but what I really wanted was to be there for the kids, to be the cool teacher, you know, the one they could talk to about what's wrong in their life and maybe get some good advice. That’s when I realized there's a whole separate career in that, counseling, so I found one of the best counseling programs in Louisiana and I applied, and thankfully, I got in.

Patricia Stark: So, did you feel like counseling was going to be that right path for you at this point?

Matthew Lafleur: Definitely. I loved everything about counseling. I graduated in the top of my class in grad school, but in the meantime, you know, I still had FA, and FA kept on progressing like it does. It started to affect things like my voice and my arm movements, and I was trying to learn how to drive using hand controls, but I could never quite get the hang of it, so that was big detriment for me. I applied to counseling job after counseling job, and I couldn't ever find one. I went on interview after interview looking for one, but nothing, so that was really devastating for me. I moved two different times trying to find a counseling job, but I just couldn't find one. I ended up moving back home with my parents, and I just realized that I was going to be another victim of FA.

Patricia Stark: But of course, this wouldn't be a real dark horse triumph, though, if it ended there. You eventually found your job with Friedreich's Ataxia News. So, what drew you to that as a possibility?

Matthew Lafleur: Friedreich's Ataxia News was looking to hire a remote columnist, someone to write about their life with FA, and I thought, man, I have education in counseling, and I have a degree in English. I could totally do this. So, I started out as a columnist, and now I do that, and I graduated, and I'm the culture coordinator of the entire company, BioNews, the company that owns Friedreich's Ataxia News.

Patricia Stark: It sounds like really a perfect fit. What does it mean to be a culture coordinator in the rare disease space?

Matthew Lafleur: Mostly I'm trying to build a connection between people all over the world with all different kinds of diagnoses. I'm really just trying to let them know they're not all alone in their disorder. Whether you're the one with the diagnosis yourself, or you're the caregiver of someone with the diagnosis, or you're just working on behalf of those with diagnoses, you really matter. That's what makes it super special about what I do, so I love what I do, and I love where I'm at.

Patricia Stark: It is so great that you've been able to help bring people together and provide information about the issues that people with FA and really any rare disease find important. So, let me ask you, what are some of the sources that you go to for information and support?

Matthew Lafleur: Well, I can't really not mention Friedreich's Ataxia News. That was the hub I would go to get pertinent information about my disorder before I even started writing for them. There are also some patient advocacy groups that give out a lot of good information about my diagnosis, and another cool source that I found for information is support groups online. There are certain social media platforms with groups on them for people living with FA or people who are caregivers of those. People in these groups often bring articles and share new information about FA. The FA community is super special to me. It reminds me of a quote by C.S. Lewis, "A friendship begins when one person says to another, What? You too? I thought I was the only one." In something like FA, something so rare and so random, you often feel like you're all alone in facing it, and that you're the only who has trouble with it, but to find out that other people are out there who know exactly what you're going through and understand what it is is so, so groundbreaking.

Patricia Stark: And what's really remarkable to me, Matt, is that not only have you been able to complete your education, find a meaningful career, and build a life for yourself, but you've been able to do it all while dealing with FA. What are some of the things that you've done to help manage your disease progression over the last couple of years?

Matthew Lafleur: Well, first off, thank you for saying that. I believe that everyone has a burden in their life that they wish wasn't there. That goes for me, that goes for everyone watching, and that even goes for you, Patricia.

Patricia Stark: Sure.

Matthew Lafleur: So, whether the challenge is FA or not, we can either see our burdens as an excuse, or we can see them as an obstacle that we can overcome. So for me, when it comes to my medical care, I see a cardiologist about once a year because heart issues are the most problematic for FA patients. I also do physical therapy around twice a week, and that's a real priority for me. In FA, I know firsthand the old adage is true that if you don't use something you lose it, so physical therapy is a way for me to maintain my abilities. My general practitioner I see is also super helpful. Before he started treating me, he was kind of unaware about FA, but because of our relationship, he really increased his knowledge, and it's really helpful for me. And of course, I also see a neurologist about once a year.

Patricia Stark: So, you have really built a solid team of healthcare professionals to help you stay on top of FA. Well, now, how about in your day-to-day life? Do you have any things that you do to help you get through the day?

Matthew Lafleur: Well, I feel like it's different for everyone, so what works for me might not work for someone else, but there are some things I try to do every day, yeah. For me, it's all about maintaining physical activity, trying to do whatever I can on my own. So, I'm able to still use a manual wheelchair, so I try and do that to keep my body moving. I have a standing frame that I try to go into for a couple hours every day. Getting in the standing position really helps out with things like my circulation. And I also have two trikes that I try to ride a couple times a week. One is powered by my arms, and one is powered by my legs.

Patricia Stark: Well, Matt, it's clear that you have been able to embody that dark horse spirit but actively managing your disease and being resourceful and finding a new and meaningful career, even if it’s not where you thought you would wind up. And there is another sort of a dark horse that you've written about recently, and that's SKYCLARYS, which, as many people know, faced a few bumps along the way.

Matthew Lafleur: Yeah. One of my latest columns is about SKYCLARYS because there's a lot of buzz going on about it in the FA community, so I really wanted to get at it from a patient perspective.

Patricia Stark: So, I'm very curious to hear more about your perspective on SKYCLARYS, but first, I would like to take a minute to talk about some of the specifics of this treatment option. SKYCLARYS is indicated for the treatment of Friedreich's ataxia in adults and adolescents age 16 years and older. It was studied in a 48-week trial called the MOXIe Trial, which included 103 patients with FA. About half were treated with SKYCLARYS, while the other half were given a placebo. Now, to understand the results of the trial, we first have to understand a little bit about how those results were measured. Researchers used a set of movement and coordination tests called the modified Friedreich's Ataxia Rating Scale, or mFARS for short. Results from the mFARS tests are tallied up, and patients are given a score from 0 to 93. A lower score means less physical impairment, while a higher score means more physical impairment. FA is a progressive disease. A large study of untreated patients that did not involve SKYCLARYS showed that FA patients typically see their scores rise by an average of about two points per year, but it's important to know that how fast FA progresses varies from person to person, and mFARS scores will change on an individual basis. Now, at the end of the MOXIe Trial, patients who received SKYCLARYS had lower mFARS scores, meaning less physical impairment compared to untreated patients. This suggests that SKYCLARYS slowed FA disease progression. In the clinical trial, the most common side effects of SKYCLARYS were elevated liver enzymes along with headache, nausea, stomach pain, and tiredness. Some people also experienced diarrhea, muscle aches, and pain in the mouth, throat, or neck. These are not all of the possible side effects of SKYCLARYS. Call your doctor for medical advice about side effects, and please stay tuned at the end of this presentation to see important safety information for SKYCLARYS. Your doctor will continue to monitor your liver enzymes as well as your cholesterol and levels of a certain protein in your blood while taking SKYCLARYS. SKYCLARYS is a once-daily oral treatment. The recommended dose is 150 mg taken as three capsules of 50 milligrams each. Be sure to take SKYCLARYS exactly as your healthcare provider tells you to take it. SKYCLARYS is available through a specialty pharmacy and is not available at retail pharmacies. REACH is an informational resource for patients and caregivers to explore ways to access SKYCLARYS. Please talk with your doctor to see if SKYCLARYS may be an option for you. If you and your doctor decide to start treatment with SKYCLARYS, your doctor can begin the process by submitting a Start Form. Enrollment in REACH support service is not required to receive prescription SKYCLARYS. More information about REACH, including how to get started and how to work with REACH to access support offerings, can be found online. Now, Matt, I'd like to turn it back over to you. You have been following the journey of SKYCLARYS for some time now, so what is your perspective on this treatment?

Matthew Lafleur: This is very exciting news for the community. When it was first approved, I heard from a lot of people in the FA community talking about what a milestone this is and how much the FA community played a big role in getting it approved.

Patricia Stark: And what do you think this means for the FA community as a whole?

Matthew Lafleur: I've heard nothing but excitement for it in the FA community. There's still more work to be done to find a cure. I really hope that the approval of SKYCLARYS; will encourage more people to continue raising funds to research FA, and that people still participate in all sorts of clinical trials.

Patricia Stark: Well, thank you so much, Matt. And to all of you watching, I hope that you found our discussion informative and helpful. Please stay tuned for additional safety information about SKYCLARYS. My name is Patricia Stark, and on behalf of myself, Matt, thank you so much for joining us.

[ISI present in video and available below.]