Imagine your
child's Takhzyro
Experience

Takeda Host Renee Seigel: Hi everyone. Thanks so much for joining. As mentioned, I am Renee Siegel. I am a patient marketer with Takeda, and I am happy to be back here as your host for today's webinar.

Takeda has been dedicated to advancing care in the hereditary angioedema space for more than a decade, and we are proud to be able to support the community in a holistic way. Our webinars with the HAEA are a part of that commitment.

In case anyone here is new to the subject, I would like to quickly set the stage. HAE is a rare genetic disease that causes attacks of swelling that can be painful, unpredictable, debilitating, and it affects about one in 50,000 people worldwide.

It involves reoccurring attacks of severe swelling of various parts of the body, including the hands, feet, genitals, abdomen, face, and throat. Swelling in the airway can restrict breathing and that can be fatal.

People with HAE do not have enough C1-INH protein or the C1-INH they do have does not work as well as it should. This causes another protein called kallikrein to become overactive, which results in the excessive release of an attack producing substance known as bradykinin.

The results of this excessive release are fluid leakage and swelling, otherwise known as an HAE attack. An HAE attack can start in one location and then it can spread to another before resolving.

Now getting into the topic of today's webinar, which is Choosing the Path of Prevention, TAKHZYRO and Children - as many of you are aware, there are two types of treatment for HAE.

On demand treatments are used to treat the symptoms of an HAE attack, and preventive treatments are medicines that you can take routinely to help prevent or reduce the frequency and severity of HAE attacks.

This is a really exciting and an important step forward for HAE treatment for younger patients.

TAKHZYRO is now a treatment for a broad range of children and adults with HAE, and it has been prescribed to more than 3250 people since 2018. Since launching TAKHZYRO for pediatrics, we are so excited to see pediatric patients and families beginning to choose TAKHZYRO as a treatment option. We are just so excited to be able to fulfill this need and to show up for the community in this way.

You will hear tonight from both a doctor and a person living with HAE, as previously introduced, who will provide information in their perspectives on having a prevention mindset when it comes to treating HAE.

First, I would like to review the important safety information about TAKHZYRO before we get into this conversation.

What is TAKHZRYO? TAKHZYRO or lanadelumab-flyo, is a prescription medicine used to prevent attacks of hereditary angioedema in people two years of age and older. It is not known if TAKHZYRO is safe and effective in children under two years of age.

TAKHZYRO may cause serious side effects, including allergic reactions. Call your healthcare provider or get emergency help right away if you have any of the following symptoms: wheezing, difficulty breathing, chest tightness, fast heartbeat, faintness, rash, or hives.

The most common side effects seen with TAKHZYRO were injection site reactions such as pain, redness and bruising, upper respiratory infection, headache, rash, dizziness, diarrhea, and muscle aches.

These are not all the possible side effects of TAKHZYRO. For more information, ask your healthcare provider or pharmacist. You may report side effects to the FDA at 1-800-FDA-1088.

TAKHZYRO has not been studied in pregnant or breastfeeding women. Talk to your healthcare provider about the risk of taking TAKHZYRO if you are pregnant, plan to be pregnant, are breastfeeding, or plan to breastfeed. Please see the full prescribing information, including information for patients available at this presentation.

How is HAE impacting your child?

HAE is an unpredictable, debilitating and potentially life-threatening disease. The onset of HAE symptoms can occur at any age, but studies suggest that symptoms may start to appear during childhood. Children with HAE can be impacted in many areas of their life, and the family can be impacted as well.

As mentioned earlier, the HAEA guidelines recommend always having access to on demand treatment and that long-term prevention may be considered for anyone with HAE.

With all of that information, I am now really happy to introduce Dr. Joshua Jacobs, who is an allergy and immunology specialist treating HAE in adults, adolescents and children. He is currently practicing in California.

HCP Speaker Dr. Joshua Jacobs: Thank you, Renee. Glad to be here.

Takeda Host Renee Seigel: So happy to have you. I am also really excited to welcome one of our wonderful long time patient speakers and advocates in the HAE community, Bobbi. Bobbi lives with HAE herself and has two daughters who were diagnosed as children with HAE and are now adults.

Bobbi has years of experience managing her own HAE, being involved in the community, and caring for her daughters when they were children with HAE, and she very kindly agreed to participate in today's webinar.

Patient Ambassador Bobbi P.: Thank you so much, Renee, and hello to all of our attendees. Renee, how about we start with a poll?

Takeda Host Renee Seigel: Yeah, I think that is a great idea, Bobbi. You will have a poll that will pop up on the screen. This is our first question. Who here cares for or helps care for a child with HAE?

If you are a friend of the family, that counts as well. We know everyone here knows that all the support counts and it takes a village. We will give you a second to answer this poll.

From the poll, it actually looks like the majority of the participants here do not help or care for a child with HAE. So, it is really interesting to know that you're here to learn more about this information which is exciting to see.

Patient Ambassador Bobbi P.: My two daughters have HAE. My oldest was diagnosed when she was in fifth grade and my youngest in kindergarten. We have all been through a lot together.

It is great to see that there's a lot of people on here that want to learn more about taking care of children with HAE.

Every adult and child with HAE are different and HAE is so unpredictable so it really can affect any and every area of your life.

Takeda Host Renee Seigel: Yes, that is interesting. Bobbi. Can you tell us a little bit more?

Patient Ambassador Bobbi P.: Sure. Both of my daughters had different experiences with HAE. My oldest was affected a lot more than my youngest with frequent attacks, especially at school.

She went from never missing a day of school to struggling to just have a good day.

It was non-stop educating the school and her teachers about HAE, working with the home health tutor, and just trying to maintain her learning and her schedule.

Ultimately, we had to pull her out of school so she could concentrate on her health.

This was a very difficult and sad decision for us, but we knew that in the long term this was the best decision. She really had her life up ended and struggled with anxiety and depression as well.

HCP Speaker Dr. Joshua Jacobs: That sounds really hard, Bobbi. I have heard similar stories from other families I've worked with.

The predictability of HAE can be difficult for patients and families to deal with. I have children with HAE suffer from attacks in various parts of the body; face, hands, feet, even laryngeal or throat attacks which can be life threatening. These attacks can be different for each individual and can change over time.

Takeda Host Renee Seigel: Yes. Thank you both for that input and let me just jump in for a second to ask another question for our attendees.

I know the majority of you answered that you don't currently care for a child with HAE, but for those that do - Where has HAE most affected the child that you do care for?

We are going to take a moment so that you can answer this question. While everyone answers, I would like to share some examples from a parent that we recently spoke with. Her name is Ada. Ada and her daughter Kenzleigh both have HAE, and they have generously agreed to share some of their stories and experiences with us, which has been helpful and interesting.

When Kenzleigh was about five or six years of age, Ada noticed swells in Kenzleigh's hands and then a swell that started in her eye and eventually moved to her throat.

Kenzleigh had to be intubated, which some of you might imagine was extremely scary for both Ada and Kenzleigh.

When reflecting on the story with Ada, Ada said that she now looks back on this and feels like it is a reminder that attacks are unpredictable, and you can never really know what the next attack will look like.

After Kenzleigh was formally diagnosed with HAE, they both would try to predict when attacks would come. For example, when she bumped herself, fell, or was upset or stressed, but you just never know and could never really predict it.

Patient Ambassador Bobbi P.: Those experiences are powerful and definitely remind me of what it was like when my girls were growing up. I remember back before my daughters and I were on TAKHZYRO, it was really hard for my family, and especially when my oldest was having throat swells on a regular basis, or when all three of us were having an attack at the same time.

Takeda Host Renee Seigel: Yes, definitely.

Well thank you, Bobbi, for sharing that. Why don't we take a second so we can pull up the results in the poll?

So, it looks like for “where has HAE most affected your child?” It actually looks like it's across the board. It is split between at school, at home, socializing, and then actually the majority of you, 70%, said all of the above, so all these areas.

Just kind of reflecting back on what Bobbi was sharing and just in general, the reflections - I agree that these stories are so powerful, but only approved routine preventive treatment options for children 6 to less than 12 years of age required dosing every three to four days. Children with HAE two to less than six years of age, had no approved preventative treatment, making TAKHZYRO the first preventive treatment for this age group.

Dr. Jacobs, would you mind telling everyone a bit about the studies that led to this?

HCP Speaker Dr. Jacobs: I would love to, Renee. It is great news for the HAE community. So really, we are going to start here and we're going to talk about the initial trials for TAKHZYRO HELP trials. The first clinical trial for TAKHZYRO was indeed term to HELP, and this is the clinical trial that led to FDA approval for TAKHZYRO.

It was a 6 1/2-month trial. It included 125 people diagnosed with HAE type one and two, aged 12 years and older. The main goal of the study was to evaluate the ability of TAKHZYRO 300 mg every two weeks to reduce the frequency of HAE attacks. In this study, people taking TAKHZYRO had 87% fewer attacks than those on placebo.

Injection site reactions were the most common side effects of TAKHZYRO in the 6 1/2-month study, with people aged 12 years and older. The next study was completed after FDA approval of TAKHZYRO, and we call this the HELP OLE. OLE stands for open label extension.

Open label means that everyone in this study is getting medicine, and they know what they are receiving. From the standpoint of effect, there may be some influence on results.

This was again a 2 1/2-year study of 212 people diagnosed with HAE aged 12 years and older. The main goal of the study was to evaluate the long-term safety, and that was with TAKHZYRO 300 mg every two weeks. In this study, people taking TAKHZYRO for an average of 2 1/2 years had similar results to those in the 6 1/2-month clinical trial. The most common side effects seen in this long-term open label study were injection site reactions, and those included pain, redness and bruising, upper respiratory infections, and rash.

Now the TAKHZYRO pediatric study is called the Spring Study. It was the largest pediatric trial of any preventive treatment in HAE, and there were twenty-one children in the 52-week open label study, all aged between 2 and 11 years.

The main goals were one, to evaluate the safety of TAKHZYRO 150 mg taken every two weeks or every four weeks, and two, to measure levels of TAKHZYRO in the body for children 2 to 11 years of age. So, in the study, children from two years to six years of age got one dose every four weeks, and children from 6 to 11 got one dose every two weeks. Overall, the safety and tolerability of TAKHZYRO were similar between adults and pediatric patients. 2 to 17 years of age, or 12 to 17 years of age. There were no serious side effects reported. No one discontinued due to the side effects and there were no allergic reactions related to TAKHZYRO.

The most common treatment emergent adverse events in children taking TAKHZYRO 150 mg every two or four weeks were injection site pain, redness and swelling, administration site pain and injection site reactions, again were the most common.

We do need to point out that TAKHZYRO can cause serious side effects in some people, including allergic reactions, and if that happens, people should call their child's healthcare provider or get emergency help. Use of TAKHZYRO in children 2 to 11 years of age was supported by the efficacy data in the 6 1/2-month study.

For people 12 years of age and older, additional data showed that similar levels of TAKHZYRO were reached in the body for adults and children.

A secondary goal of the study was to measure the ability of TAKHZYRO 150 mg taken once every two weeks or four weeks to prevent HAE attacks in children 2 to 11 years of age. Decrease in HAE attack was measured as the number of attacks before the patient started the study, compared to the number of HAE attacks after taking TAKHZYRO.

The study was not designed to understand how well TAKHZYRO works in children. The twenty-one children included knew they were taking TAKHZYRO, and this did not compare TAKHZYRO to another product or a placebo. These details make it difficult to determine how well TAKHZYRO decreased HAE attacks in children. On average, children had 95% fewer HAE attacks compared to before starting the study, and 76% of children were attack free for the entire 52 weeks, or one year of the study.

Patient Ambassador Bobbi P.: Thanks so much for sharing that information, Dr. Jacobs, I am just so excited for our HAE community.

Takeda Host Renee Seigel: Yes, me too.

Patient Ambassador Bobbi P.: I cannot wait to see how it might help the community of children and their caregivers. I was in the clinical trials for TAKHZYRO, as were my daughters, and we started going weeks and then months without an attack. There were tears of joy and it was just overwhelming.

HCP Speaker Dr. Jacobs: That is so great to hear, Bobbi. I noticed the same kind of relief in my pediatric patients who were included in the most recent clinical trial.

Takeda Host Renee Siegel: Yes, and Kenzleigh was also in the pediatric clinical trial, the Spring Study, as Dr. Jacobs mentioned. Ada told us that she wanted to start Kenzleigh on TAKHZYRO as soon as she could because she was having so many face and throat swells. Ada is very proactive and wanted to do as much as she could to help prevent Kenzleigh from having attacks, which for her, TAKHZYRO was a part of that mindset.

Ada says, we have had discussions that TAKHZYRO has really helped manage Kenzleigh's HAE attacks. Before she enrolled in the study, she was having attacks every week.

Patient Ambassador Bobbi P.: So, Dr. Jacobs, I know a lot of people are going to be thinking about injections and how they can make them more comfortable for kids. Can you give us some tips around that?

HCP Speaker Dr. Jacobs: Absolutely can, Bobbi.

Yes, the frequency of the dose depends on the age of the child, right? So, every four weeks for kids two to less than six, and every two weeks for kids six and older. When treating kids, we do think a lot more about how they will handle the treatment itself and we also would expect kids not to love injections. I am 100% on board with creating a reward system for children who are taking TAKHZYRO. This can work for a child, or really a person of any age. For example, after your injection, we will go to the playground, get some ice cream, whatever works best for your particular child. My mantra is comfort and confidence. Do whatever helps to make the child comfortable physically and emotionally and feel confident in the medication.

Help the child create a positive mindset. Talk to the child about the injection, the reason for it, and how TAKHZYRO is going to help them.

Obviously, the way to do that will depend on the child's age and prior knowledge. They may have seen you or another family member administer an injection for example. I would also suggest that you rotate the injection sites. There are three places mentioned in the instructions for use that comes with TAKHZYRO.

Patient Ambassador Bobbi P.: Oh yes, I am very familiar with those, stomach, thigh, and upper arm.

Which for my daughters and I, we usually inject in our stomachs. That is our preference.

HCP Speaker Dr. Jacobs: Exactly. And so, then I would suggest choosing the environment thoughtfully, somewhere the child feels comfortable and safe, and of course, somewhere clean. It can help to make it the same time and place every time if the child seems calm and willing. We want to normalize the experience, make it ordinary and not frightening, and always be encouraging.

Takeda Host Renee Siegel: Definitely. Thank you both for sharing those thoughts and perspectives and from Ada's perspective, she said that she was apprehensive at first, but that they have a good routine going. It takes just a few minutes every two weeks and Kenzleigh is grateful to be taking a medication that helps reduce the frequency of HAE attacks.

Patient Ambassador Bobbi P.: TAKHZYRO was not available when my girls were young, but I think it's such an exciting step for the HAE community.

Takeda Host Renee Siegel: Yes. Based on what we call or reference specialty pharmacy data, TAKHZYRO is currently the number one prescribed preventative treatment in HAE.

We are looking forward to helping even more, and as I mentioned, so excited to see we're beginning to help this population with this approval for this expanded age range.

HCP Speaker Dr. Jacobs: TI would like to also add here that ongoing family conversations are important. Kids grow up, family dynamics change, priorities around activities change, and it is important to be consistent and get the proper dose at the right intervals.

Takeda Host Renee Siegel: Yes, definitely. There is a resource where people can sign up. They can sign up for dosing reminders which could help, especially if something in the family routine as you are referencing Dr. Jacobs changes.

HCP Speaker Dr. Jacobs: Yes, it is crucial to remember that TAKHZYRO is not a cure, and to take it as prescribed at regular intervals. I hate to see patients having breakthrough attacks, but it can happen and is something they need to be prepared for.

Patient Ambassador Bobbi P.: I want to second your suggestion about having conversations not just with your family, but with your healthcare team and anyone else who you might consider part of your circle about everything that is going on. A dear friend of ours who lives close by, was so supportive and valuable to our family during those tough years. I cannot thank her enough for all the time she was there to help with whatever was needed.

I was also so grateful for all the people I have met through our HAE community - they were and still are my rock and sounding board.

HCP Speaker Dr. Jacobs: Indeed, I have open and honest relationships with all my patients.

Takeda Host Renee Siegel: That is great. Speaking of support, Takeda has more than 12 years of experience supporting the HAE community. I want to remind everyone here that the OnePath program is available. You can access a dedicated patient service manager support manager who offers injection training, financial assistance options, and product support. Let us also not forget that caregivers need support too, whether they have HAE or not. Caregiving can be very demanding, as some of you might know, and we have been talking about children with HAE today, and for every child with HAE, there is a caregiver who could use support as well, and OnePath offers support for caregivers too.

Patient Ambassador Bobbi P.: Yes, that is a great thing.

I would also like to tell caregivers that it is OK to give yourself time and not feel guilty about it with younger kids especially. You are their board, your voice, and do not give up. As a parent, you just want them to flourish.

You need to take care of yourself so that you can take care of others. I know it is not always an easy thing to do.

You can also lean on this community. I have spent the last 10+ years participating in numerous activities, and I will continue to do everything I can to support our community. I encourage everyone to get involved.

Takeda Host Renee Sigel: Thank you, Bobbi. You are an inspiration.

We are also really excited to take a few minutes to hear from all of you. But before that, I just wanted to say that there have been more than 3250 people prescribed TAKHZYRO and in addition their caregivers, friends and families are part of our community too.

So, before I hand it back to the HAEA to take some questions, I just want to take a moment to thank Bobbi and Dr. Jacobs, and although they could not be here tonight, I also want to thank Ada and Kenzleigh for allowing us to share their experiences and story.