Leaked Audio of Hemlibra Presentation from HFA Symposium



Hemlibra currently has 44 fatalities.


This is the full transcript of the Hemlibra presentation. Click the above video to hear the full audio of this presentation.


Okay. I think we're going to get started.


Good morning, everyone, and welcome. Thank you so much for joining us here today for our symposium. I want to just first of all, introduce myself. My name is Ana Garcia. I'm one of the managers with the Hemophilia Community Clinical Educator Team, and I have been a nurse now for 35 years. So yes, I'm that old. I still can't believe I'm that old. I've been within the hemophilia space now for over 18 years, and I've been here at Genentech in my current position for about a year and a half.


Throughout the years, I've just always enjoyed coming here to HFA's annual meeting. I feel like it just really provides an incredible opportunity to connect, to engage, to learn from the community, to see old friends or make new friends. I was actually texting recently with one of the moms that I've been friends with for a long time. She was asking if I was going to be here. She said, "How ironic we live like seven or eight miles apart." She has other friends within the community, but we always end up seeing each other at HFA. So looking forward to getting to see a lot of my old friends.


Okay. Before we begin, there are a few disclaimers that I want to make sure and disclose with you all, and this will provide some context for you for the information that you're about to receive. So this program is intended to provide general information about Hemlibra and not medical advice for any particular patient. You need to talk with your doctor to discuss any treatment regimen that's appropriate for you. Individual results may vary in taking the Genentech product, and the product may not be appropriate for everyone. This program is presented on behalf of Genentech, and the information presented is consistent with FDA guidelines. This program may be monitored by Genentech for adherence to program requirements. Any adverse events in this presentation today have already been reported to the US Drug Safety, so no action is required by any member of the audience. I am an employee of Genentech and our speaker has been compensated by Genentech today to serve as the speaker for this program. All materials are the property of Genentech and may not be reported or photographed, copied or reproduced. Okay.


Today, I also want to take an opportunity to introduce you all to our great team of people who are the Hemophilia Community Clinical Educators. We like to call them CEs for short. It's a lot easier to say. We're so honored for all of us to be here today. We have a dedicated team of people. They all have nursing degrees and they're committed to being an educational and informational resource about our approved Genentech products, educational programs and event, as well as information about access to Genentech medicines for eligible patients. What they do not do, however, is provide any medical advice.


We will always ask you that if you have any questions about a medical condition to please contact your healthcare provider. When the exhibit four opens, we would love for y'all to come by. We are at booth number 10, and come on in and get to meet some of our CE team who will be there to meet with you guys.


I hope by now that many of y'all are familiar with our company, with Genentech, but if you're not, I'm very honored to be here today to introduce us to you. Our 40 plus year history has been leading us to this very moment that we're at today. We had a role in the genesis of biotech industry itself, and we're most known for our work in cancer. That work has revolved around therapeutic antibodies, which has become a standard of care of several disease areas.


I'm giving you guys a little background regarding Genentech. You know about our CEs. So what I'd like to do is introduce you now to Dr. Joanna Davis, who is going to introduce you to Hemlibra, which is our FDA approved treatment for people with hemophilia A with factor VIII inhibitors. Dr. Davis is the medical director at the University of Miami HTC. She obtained her undergraduate degree from Barnard College of Columbia University. She attended Albert Einstein College of Medicine in New York, and she did her pediatric training at Montefiore Hospital in the Bronx. Her fellowship was for pediatric hematology oncology was at the University of Miami. She has been there since 1982 on staff.


She is very committed to education and to outreach to the medical and patient community. She established a hemophilia lecture series for community health care providers that has reached over 2,500 healthcare providers. She's also created bilingual storybooks for families living with bleeding disorders, and these books have been distributed nationally and are part of NHS educational library. She has served on numerous advisory boards and has many publications in academic journals. She has also engaged in several clinical research projects.

Before I bring Dr. Davis to the stage, I do want to also let you guys know that we will be playing some trivia throughout this presentation. Make sure that you all pay attention because you'll have a clicker there to put your answers in. Before any further ado, I would like to ask Dr. Davis up to the stage.


Good morning. Thank you all for coming out and for wanting to learn about Hemlibra. It's a pleasure to be here at the meeting and it's also a pleasure to be here in Cleveland. It's my first time in Cleveland. So it will be kind of exciting.


Let's just get right to it. What is Hemlibra? Hemlibra is a prescription medication. Your doctor has to prescribe it. It is available for use as routine prophylaxis to prevent or reduce the frequency of bleeding episodes in both adults and children who have hemophilia A and also have developed an inhibitor of factor VII.


What's the most important information? Well, everything is the most important information, but there have been some serious, we'll call, adverse events in the course of the clinical trials, which we'll talk about moving forward to the slide deck. Which we need to discuss upfront. Hemlibra increases the body's ability to perform clot. That's why people with hemophilia are potentially eligible to use it. Before starting any new medication, regardless of what it is, you and your healthcare provider need to have a discussion about the medication.


Of course, Hemlibra in particular, if you are on a bypass agent and you and your physician have determined that this might be an appropriate therapy for you, you need to make sure that you discontinue whatever bypass agent you've been prescribed the day before you start. Hemlibra may cause, as I said, some serious side effects when used in conjunction with one of the bypassing agents, which is an APCC activated prothrombin complex concentrate, which you may know by the brand name of FEIBA. These two potentially serious episodes are called either thrombotic microangiopathy, which is abbreviated TMA because it's too much to say, or just in general blood clots or thrombotic events. If you and your doctor have determined that the use of an APCC to manage a bleeding episode is the appropriate way for you to go while you are using Hemlibra for prophylaxis, then you need to have that discussion and work out a treatment plan so that you're not exceeding more than 100 units per kilogram per day of the APCC.


We're going to cover a lot of ground and intersperse in some fun stuff with Ana. We're going to talk about what Hemlibra is. How does it work? How did we get from the discovery or the laboratory production of this molecule to clinical use today? How do you use it? And again, the serious potential side effects. On your table, you a whole bunch of information, including what I call, they're like really good cookies. But under the cookies, there are a number of brochures. One of them is talking about what do you need to know about Hemlibra. In that is the prescribing information medication guide. The medication guide is specifically written to provide you, your family members, your health care provider with specific information about Hemlibra so that you can avoid serious adverse events. It's written by the FDA. You have it like right there.


As Ana said, this presentation is not intended to give specific medical advice for any one person. This is an informational session so that you can become familiar with a medication, which is relatively new to the market. When in doubt, always call your healthcare provider.

We'll get the serious and common side effects out of the way. As I mentioned before, TMA thrombotic microangiopathy, is a condition in which blood clots can form in small blood vessels. Now, whenever there's a clot, it's sort of blocking a pipe. So what can't get past the blockage to whatever organ or system is meant to go and deliver blood. The signs or symptoms may include, may include confusion if the clots have occurred in the brain, weakness, swelling of arms and legs, yellowing skin and eyes, also jaundice, side or abdominal pain, back pain, nausea, vomiting, not feeling well in general, or passing less urine than you're used to. If you were to be on this medication or any medication and developed these symptoms, your first call should be to your healthcare provider. You should not ignore these symptoms.


The other serious side effects are blood clots, which most often occur in arms and legs, but can certainly occur in the lungs or the brain as well. The symptoms can include swelling, pain in the limbs, shortness of breath, chest pain or tightness, a fast heart rate, coughing up blood, not feeling well, feeling faint, headache, numbness in your face, eye pain or swelling, difficulty seeing. Again, regardless, if you're experiencing any of these symptoms, your first call should be to your healthcare provider.


As I discussed, these potentially serious side effects were observed in a few patients on the clinical trials who were receiving Hemlibra prophylaxis but were using the APCC to manage a breakthrough bleed. Again, as I said before, if you're going to be relying on an APCC to manage any potential break in bleeding while you're on Hemlibra, talk to your health care provider. The recommendation is that you don't receive more than 100 units per kilogram in a 24 hour period.


Let's talk about more common side effects, and I'm sure everyone here has had the experience of treating a [inaudible 00:13:27] bleed. You know that there are minor, but could still be annoying or worrisome, side effects with any kind of an injection. Hemlibra is administered under the skin as a subcutaneous injection. We'll talk more about that later, but that's a big change. But at the actual injection site, a person may experience some local discomfort, maybe some redness, some warmth, itchiness, maybe some tenderness. Some patients reported a headache after receiving the Hemlibra, others some joint pain. These are not all possible side effects.


These are not all possible side effects. You'll have the package insert. It will give you much more details about what anybody may have reported in association with [inaudible 00:14:13], and you can certainly contact the FDA if you are on this medication and you were experiencing a side effect of any sort.


Okay. While I figure out how to reattach this, it's time for some fun stuff without.

Thank you. Okay. So I mentioned earlier, we're going to do a little trivia here. And you guys will look at your table and you'll see that you have these little clippers. And what I'm going to do is I'll spin the wheel, the topic will come up and then there'll be a question. Y'all need to just look at the question and you get the corresponding answer with your clipper, and then we'll see how you all do. So I do want to let you know that this first question is a little, I think probably one of the harder ones. And I'm going to give you a hint and just say to you to think back to what I had said at the beginning, and that may help you figure out the answer. So y'all ready to play? Okay. So let's spin the wheel.


Okay. The topic is, What It Is. And the question is, what is Hemlibra? Is it A, factor eight product replacement? B, hormone to stimulate the production of more clotting factor? C, a newly discovered kind of zebra? D, a therapeutic antibody? Or E, a clot preserver? Okay. Get your answers in.


So some of y'all really were listening to me. Awesome. The answer is D, therapeutic antibody. Hemlibra is a therapeutic antibody.


So what does that actually mean? Well, a therapeutic antibody is a laboratory produced molecule that is built in such a way that it recognizes and attaches to specific proteins to help treat a disease or condition. Definitely talk more about that later.


So how does this work? Here we go, we're talking. Background for the audience, and I'm sure everybody here already knows this. Hemophilia A is a usually inherited bleeding disorder. People with hemophilia A do not have the ability to manufacture a normal amount of clotting factor eight. Clotting factor eight is one of the coagulation factors, which is really important for the formation of a good solid clot. Most of the time, people with hemophilia A are managed with factor eight replacement therapy or factor concentrates, but there are people with hemophilia A who in the course of their lifetime, may develop an inhibitor, which is another antibody that's approaching that the immune system has basically made, and this inhibitor protein attaches to the factor eight that someone doesn't fuse, so basically clears it out of circulation so that it's not helpful. So people with inhibitors to factor eight are, and again, everybody's different, but most people with inhibitors are not able to receive factor eight replacement therapy because this antibody or inhibitor wipes it out.


So how is Hemlibra supposed to work? So here's this nice little graph. Here we have the schema for Hemlibra. This is activated factor nine. And if anybody's bored afterwards, you can talk about clotting cascade, but take my word for it. Here's activated factor nine. In order for the clotting process to continue, factor nine has to get in contact with factor 10, and activate factor 10 so it can go on performing the rest of the body process. You can see here on this little graphic that these two clotting factors have little prongs on it, and Hemlibra is built in such a way that there's receptors for those proteins. Here we go. Hemlibra brings these two together and causes factor 10 to get activated and then it can go off and do its job in terms of completing the rest of the clotting process and [inaudible 00:19:11]. And here we are.


Hi guys. Okay. We're going to see if everyone's been listening here. I'm going to spin the wheel. Okay. So the topic is How It Works. And the question is, how does Hemlibra work? Is it A, replacement factor eight protein? B, replaces a clotting cascade? C, bridges clotting factors? Or D, it turns certain genes on and off? Okay, everybody. Put in your answers.

Okay. That's awesome. I think you guys were really listening. That's great. So the answer is C. Hemlibra is believed to bridge clotting factors to help the clotting cascade continue.

Super. Okay. So again, I believe where we'll bring activated factor nine, together with factor 10, to activate factor 10 and then the rest of the body processes.


So let's talk about clinical studies. This is going to be sort of like peeling back layers of an onion so that you all understand what happened once the Hemlibra had been developed in the lab, went through preliminary studies, and then was brought to what we call a clinical trial, which is a pivotal part of the development of any medication at all.


How does it work in human beings? Hemlibra was studied in two clinical trials named Haven One and Haven Two. Haven One is finished. All the results have been examined, have been statistically analyzed. That study is completed. Haven Two is still ongoing. So the information about what's going on in Haven Two is what we call interim results, because the study is still ongoing.


Both of these studies were involved in determining whether Hemlibra would be effective and safe for use in patients, adults, adolescents, and children who had hemophilia A, but had also [inaudible 00:21:45] an inhibitor. Primary goal of these studies is to determine how well Hemlibra worked to reduce the number of treated bleeds in these patients with factor eight hemophilia A and inhibitors to factor eight. So it was not just, did it reduce the number of bleeds, was the safety of Hemlibra. How did the use of Hemlibra compare to how patients were experiencing bleeds and other complications of hemophilia before they started the Hemlibra, with whatever their treatment regimen had been as prescribed by the treating physician? And the bleeds that were being looked at in terms of reducing the number were basically all bleeds, including joint bleeds, including spontaneous bleeds, including [inaudible 00:08:36].


Haven One was studied in 109 adult and adolescent males with hemophilia Aa who had factor eight inhibitors. The age range for the patients enrolled on this study was 12 to 75 years. And they had to weigh more than 40 kilograms, or about 88 pounds. All of these patients had been prescribed bypassing agents by their healthcare providers. Some of them were managed on demand, meaning they had a bleed, they treated that bleed at that point in time. Others had been prescribed a prophylactic regimen with a bypassing agent, which means they were on a schedule of infusions of the bypassing agent, regardless of whether they were bleeding or not.


The primary goal was to compare how many bleeds patients experienced when they were on Hemlibra, versus what they had had with their previous therapy. Study was both randomized, multicenter and overlaid. Randomized means that the decision for a patient to be assigned to Hemlibra or not was sort of like a flip of the coin. It wasn't that somebody could come in and say, I only want to get this, or I don't want to get that. This was randomly determined. Multicenter means that many hemophilia programs offered the opportunity to their patients to enroll on this study. No one center has so many patients with inhibitors that you could do all this work in one place, because the more patients you have in the role, the more meaningful are the results. And open label meant that both the doctor and the patient knew what the prescribed regimen, or the randomized regimen was for each patient.


So now this gets to be a busy slide, and I'm going to go through it bit by bit because it can be confusing when you're just looking at [inaudible 00:10:35]. So adults and adolescents age 12 to 75, there were 109 all told. 53 of these patients before enrolling had been on on demand therapy with a bypassing agent, as determined by working with their healthcare provider. 53 of these 109 had been treated with on demand bypassing agents. They were randomized, two to one. So two thirds of these 53 were randomly assigned to receive Hemlibra prophylaxis. One third of them were randomized to just continue doing what they had been doing before.


Now, what is Hemlibra prophylaxis? Hemlibra prophylaxis means receiving three milligrams per kilogram of Hemlibra administered as an under the skin injection once a week for four weeks. And then after those four weeks, the dose drops to one and a half milligrams per kilogram administered under the skin once a week for the rest of the time. This period of time here was six months, or 24 weeks.


So these are the patients who were treated on demand before enrolling. There were also 49 patients involved in the study who had all been on a prophylactic regimen with a bypassing agent. All these patients automatically were put onto Hemlibra prophylaxis, or [inaudible 00:26:12] prophylaxis with one medication, he went to prophylaxis with Hemlibra. What was interesting, delving down even more, is 24 of these 49 patients had actually been observed for about six months before they started the Hemlibra. And what did that mean? It meant that they were on a prophylactic regimen as prescribed by their healthcare providers, and they were very meticulous about tracking if they had a bleed, what they were doing when they had the bleed, how did they manage the bleed. So that was their life on prophylaxis.

That type of observation is called a non interventional study, and it actually serves a very, very useful purpose. It allows for looking at how a particular patient did before and after. And it's the same patient. As opposed to, which is obviously a very useful tool to see how patients do in general. But this gave you a little bit more of a direct comparison.


So here were the results. Now let me explain the graph. This is called annualized bleed rate. This study only went for six months. So during those six months, patients experienced bleeds, but in order to standardize it, there's a mathematical formula which says, well, if you had X number of bleeds in this amount of time, we do the math and we would project that if everything stayed the same with you, you would have experienced so many bleeds in the course of a year. Annualized. So for patients in that top part, if you remember, you had the ones that were on demand, the patients who continue with their on-


The patients who continue with their on demand therapy, the ones that were randomized there. Their annualized bleed rate gives you the range. The numbers would work out to be about 23.3 bleeds in the course of a year, annualized bleed rate. If you took the other patients, so two-thirds of that group, for on demands before enrolled who were randomized to receive the Hemlibra prophylaxis, these patients had a 2.9 or only a three times annualized bleed rate. That's a huge reduction. 87% reduction in bleed rate, in treated bleed rate, between receiving on-demand bypassing with [inaudible 00:28:50] Hemlibra prophylaxis. This is considered statistically significant, which means these results could not have happened just by chance. There was an intervention [inaudible 00:29:01] statistically significant.


Another question. What about no bleeds during that period of time? So for the patients who continued to be treated on demand for those six months, only 6% of those patients reported that in that period of time, they had experienced no bleeds at all. Compared to the patients who had gone on to Hemlibra prophylaxis were 63% said that during that timeframe, they had had no bleeds at all once they were on the Hemlibra prophylaxis. Again, this is statistically significant.


Now let's go back to the patients who had been on prophylaxis with a bypassing agent before enrolling. 24 of those have been observed for six months before enrolling in the Hemlibra study. So of those 24 patients, before they enrolled in their observation period on their bypassing agent prophylaxis, their annualized bleeding rate was 15.7. After their six months on Hemlibra prophylaxis, they had only 3.3 annualized bleed rate. Again, 79% reduction in their treated bleed rate once they got Hemlibra prophylaxis. And again, this is also statistically significant.


What about zero bleeds? Well, 30% of these patients in their observation period had zero bleeds during those six months. When they finished the six months on Hemlibra, 71% of them reported that they had had no bleeds. Again, statistically significant.

Good results in a nutshell. Compared to no prophylaxis, on demand treatment only, there was a statically significant reduction in all of bleeds, whether treated or not, treated spontaneously, treated jointly, treated target joint bleeds.


Now, hemophilia, as everyone in the group knows, is not just about having the bleed and treating the bleed. There are consequences, physical consequences, and consequences to your daily life and the life of your family if you have hemophilia and are living with a potential for joint bleeds. So you've got the hemophilia symptoms, if you will, pain, joint, swelling, maybe limitation of motion. But then you have the impact on how somebody goes about his daily life. How are you able to function? Can you walk? How far can you walk? Does it hurt you to move? Can you play with your kids? Can you go and do grocery shopping? That's a whole other issue because maybe you don't want to. But it's an impact. There are quality of life questionnaires. Some of you may have done these at your hemophilia treatment center trying to determine the impact of having hemophilia, perhaps having bleeds, on daily life.

There are some that are set for pediatrics. There are some that are set for adults. The quality of life questionnaire during the HAVEN 1 study was administered to patients over the age of 18. There were 25 patients who completed this, and people who were taking the Hemlibra prophylaxis showed a clear improvement on the quality of life questionnaire when they were on Hemlibra prophylaxis, as opposed to those who continued on with their episodic on-demand treatment, no prophylaxis.


HAVEN 2. This is, again, ongoing. It's single arm, meaning everybody's getting the same treatment. There's no randomization. Open label. For children who are younger than the age of 12 or for adolescents between 12 and 17 who are child sized, weighing less than 40 kilograms. They all received the Hemlibra prophylaxis at the same dosage and an interim analysis was done. These are the results.


The patients were followed for at least 12 weeks. It's of course to HAVEN 1 which was finished and it was six months. At the time of the recording of this data, the range of time for observation was about 38 weeks. Some patients were observed for as few as 12 weeks, some patients were not all the way to 41 weeks. Same dosage. There 23 patients younger than the age of 12 who fit into this interim analysis and their results were eligible. And in a nutshell, it showed that for these 23 patients, there was an 87% reduction. There was an 87% zero bleed performance. It was a significant, significant change. It also looked at specific bleed types. And again, across all three categories, 87% of those children had no bleeds. It was very encouraging as an interim analysis.


There was a 99% reduction in bleed rate in general. This was compared to pediatric patients who either had been on prophylaxis before. And that was [inaudible 00:34:53] or on the one patient who had prior to [inaudible 00:34:56] had been on on demand therapy.


Another term which is important for any study, for any medication that you're taking is pharmacokinetics, abbreviated PK. Many of you may have had PK studies done through your hemophilia treatment center. What it means is it's a study of how a medication, regardless of what it is, moves through a person's system. How high does the level get after you take it? How long does the effect last? I think people are familiar with the idea of maybe being prescribed an antibiotic and your doctor is saying to you, "Look, you need to have a couple of days of it or a couple of doses up to get your level up so that it is effective to treat whatever the infection is."


Well, it's the same thing here. So if you remember, for prophylaxis, those first four weeks involve three milligrams per kilogram once a week. That was to get your level up in the bloodstream to what had been determined should be an effective therapeutic level for prevention of joint bleeds. That was approximately determined to be about 50 micrograms per mL. So it took about four weeks of this higher dose to reach that anticipated therapeutic level. And then you can see moving forward in time, once the dose had dropped, that you're still able to maintain a therapeutic level of Hemlibra in the bloodstream.


Okay. A lot of information. I'm happy to turn it over to Ana for the next round.


Okay. Did y'all get all that? You ready?


100%.


I think you guys are going to do really good on this. I'm feeling good about it. Let's see what we spin to. Oh, good. Clinical trials. Awesome. Here is a question. I want to let y'all know that we're going to have two questions. That was a lot of information. I'm going to have two questions for you guys. This is the first. What word is used in the name of the Hemlibra clinical trials? Is it A, Frank, B, Haven, C, Bridge, D, giraffe, or E, emerge? Okay. Come on, let's get in those answers.


Okay. The answer, you guys were really good on this one, was B. HAVEN 1 and HAVEN 2. Okay. I think that one was a little bit easy, right? So we're going to go for another one here and see how you guys do. Okay. In the HAVEN 1 trials compared with no prophylaxis, Hemlibra showed a statistically significant reduction in A, all bleeds whether treated or not, B, treated spontaneous bleeds, C, treated joint bleeds, D, treated target joint bleeds, or E, all of the above. Okay. Get the answers in.


Oh, wow. You all did awesome. That's right. The answer is E, all of the above. Great job, guys. Okay. We'll give you some more information.


There we go. Okay. So yes, on the HAVEN 1 trial, compared to no prophylaxis, there was a statistically significant reduction in all bleeds whether treated or not, spontaneous bleeds, target joint bleeds. So you guys did awesome. Have another cookie. All right.

What do you tell your healthcare provider before using Hemlibra? Now this is basic general information, which is applicable across the board when you are anticipating a new medication. This is common sense, but needs to be discussed when you're changing the occasions for any reasons. If you are pregnant or plan to become pregnant [inaudible 00:39:41], let your healthcare provider know. It is not known if Hemlibra can cross the placenta and cause damage or issues with the unborn baby. So if you are a female and your situation is such that this might be applicable for you to consider using, it is recommended that you and your partner practice some form of birth control or contraception to avoid pregnancy, just because you don't know what the risks may be to your unborn baby.

If you're breastfeeding or plan to breastfeed, again, it's not known whether the Hemlibra would pass through into the breast milk and affect the baby. If you are taking any medications at all, whether it's over the counter, whether it's prescribed, whether it's a herbal supplement, whether it's vitamins, always let your healthcare provider know because of the potential for interactions, which may either prevent you from using the medication or make a change in your other regimens. In general, what we advise our patients to do down in Miami is just keep a list of what they are taking on a regular basis and keep in your pocket, in your purse, in your phone. So that when you're at the doctor's office, you can say, "This is what I'm doing now."


How do we use the Hemlibra? This is an overview. There are instructions for use, which will come with any dosage you're using. It'll include how to dispose of needles and syringes and how to actually administer the medication. This information is also available at hemlibra.com. You have the information on your table under the cookies.


Okay. This little graphic is a calendar page. There's the weeks, there's the days. Use Hemlibra exactly as discussed with your healthcare provider. It is for routine prophylaxis. It's not for treating a bleed. It's for the prevention and reduction of bleeds. Therefore, you and your healthcare provider need to have worked out a plan.


Therefore, you and your healthcare provider, you two have worked out plan in terms of what you're going to do, what medication you're going to use if you develop a bleed while you are on HEMLIBRA prophylaxis. This medication is given under the skin. This is a sea change here, because it is not given in a vein, it is not given in a port, it is not given in a PICC line and it is not given as an intramuscular injection like an immunization might be. It will be injected once a week by either yourself after you learn, or if you don't want to learn or can't learn or are under the age of seven, it will be administered by a caregiver or a home nurse. It is not recommended the children under seven give themselves this injection. Okay. Thank you.


Okay. Guys listening? I'm going to find out. Spin the wheel. Okay. So the topic is how to use it. And we're going to ask two questions again. Okay. So here we go. So how do you take HEMLIBRA? Is it A, as a pill? B, as an injection under the skin? C, as infusion? D, as an injection into a muscle? Or E, as a cream rubbed into the skin? Okay. Get your answers in.

Okay. Awesome. You guys did great. The answer is B. [inaudible 00:44:05] So HEMLIBRA is given as a subcutaneous injection under your skin. Okay, guys, ready for another one? You ready?


Yeah.


Okay. So next question is, how often do you take HEMLIBRA? Is it A, every time you eat broccoli? B, once a day? C, three times a week? D, once a week? Or E, once a year? Okay. Everybody put in your answers.


Okay. Good job. The answer is D. Excellent. Except for the guy who was talking about broccoli. Once a week under the skin. So your healthcare provider is going to show you how to mix it and how to administer a subcutaneous injection. You probably will be observed the first time in clinic to make sure that you're doing all the steps correctly. And once you've learned, then you're going to be getting it to yourself, or if you're dealing with a child under the age of seven and probably even a child of eight or nine or 10 depending, you as mom or dad or another healthcare provider is going to be giving the injection to that child. So obviously you need to learn how to do it.


Now we have a lot of skin, right? So can you just inject this wherever? Well, actually no. The recommendations are to administer the HEMLIBRA on the thighs, the back of the arms or on the abdomen avoiding about a two inch circle around the umbilicus. Then there are reasons for that, that the absorption of medication may not be normal because of the way the tissues are formulated around the belly button. There's also the potential for perhaps hitting some veins. And this is not an intravenous medication. So in general, if you're going to use the abdomen, you got a lot of space anyway, just to avoid about a two inch circle around the belly button.


How do you use it? This is weight-based, right, three milligrams per kilogram, once a week for four weeks, one and a half milligrams per kilogram once a week after that first four week period. So this is obviously weight-based. If your weight changes while you're on HEMLIBRA, even if you're not scheduled for a visit, call your healthcare provider, say, "Do I need a change in dose?" Don't make that decision on your own.


And again, this is once a week. So go back to our really nifty little calendar graphic, and you can see, for argument's sake, that Mondays are all marked off. Pick a day and stick to it. Pick a day and stick to it. It doesn't have to be Monday, but that's just the graphic. Now life intervenes. Sometimes somebody might actually miss a dose. What do you do? Well, what you don't do is you don't wait until the next week and then take a double dose. You contact your healthcare provider, but you try to make that dose up as close to when you realized you missed it as possible. And then just continue on with your regular weekly dose. Again, talk to your healthcare provider, but do not double up the dose to make up for the one you missed. Okay, y'all ready? Next question. Okay. Missing doses. And the question is, this is going to be a true or false. If you miss a dose, should you double your next dose? A, true. B, false. Put in your answer.


Hey, good job. That's really important to remember. So the answer is B, false. Do not double up the dose and call your healthcare provider. Make the dose up as soon as possible. This is also really important. I think everybody here is very familiar with going into your comp visit or going to see your healthcare provider, having a whole bunch of blood tests done. And some of those tests are done to determine how well your current regimen may be working for you, regardless of whether you have an inhibitor or you don't. If you go to an emergency room, if you are traveling somewhere and you go to see another doctor for whatever reason, those physicians may also order some basic tests of coagulation to try and figure out what's going on and is what you're taking appropriate.


What's important to know, and this is where you get to teach the ER doctor or the community healthcare provider something, is that the use of HEMLIBRA may interfere with some of these very common tests of blood clotting. And the results will not be accurate. And therefore you don't want a healthcare provider to make a decision about what you're supposed to be doing based on inaccurate results. So when you sit with your physician and you're talking about, I want to try this, or maybe this is appropriate, that has to enter into the equation because then you need to have some guidance to be able to inform other healthcare providers that you're on this medication and these tests are not going to be appropriate for monitoring.


This applies not just to the ER, but if you need a surgical procedure, or if you're going to the dentist, some dentists before they're going to do oral surgery on you will want to have the basic coagulation information as well. So again, very important for you to be able to relate that to them understanding. You may want to talk to your healthcare provider about tips for how you get started with this new regimen. Remember, you stop your bypassing agent 24 hours before you begin the HEMLIBRA prophylaxis. You and your spouse or your family need to figure out what might be the best day of the week so that you can be adherent to that schedule.


You need to, again, plan for emergencies. And I think everybody here already has plans in place, but MedicAlert bracelets, medical information on your phone, where some people can program it in so that they just have to hit a button and walk into the emergency room or wherever and all their information is there. What we do a lot in Miami is we provide our patients what we call a travel alert, which is basically, hi, my name is, I have this problem, I see Dr. Davis in Miami. And this is what if I'm in your emergency room is because I may need to have whatever. And here's how we do it. So that may be something to think about. These are not musts, but there's suggestions. And I'm quite sure that many of you have your own system which has worked well for you in terms of having the emergency information available if you need to seek medical attention outside of your immediate hemophilia treatment center.


Okay, y'all, this is the last trivia question of the day, so about if get a hundred percent. Think we can do it? Okay.


Yeah.


Come on. Okay. So let's stand. Planning for emergencies. And the question is, what are things that you should consider to help you plan for an emergency? A, wear a Medical Alert bracelet. B, carry an emergency wallet phone card. C, use the medical ID function to store important information on your phone. D, use a travel alert when traveling. And E, all of the above. Okay. Put in your answers.


Okay, is it going to be 100? That was close. Awesome. 93%. So the correct answer is E, all of the above. And it'll be interesting to find out what other ways that you may devise for making sure that medical information is available. I know that I have a couple of patients who have these things that kids who are in car seats, which fit over the seatbelt or over the harness, which basically has all their medical information there, so in the event of an accident first responders can see all their information up close and personal.


And in general, for more information, talk to your healthcare provider, go to HEMLIBRA.com. You've got the information at your table. And this brings us to the end of this session. I hope it's been useful. This is an exciting time for people with risk of hemophilia and [inaudible 00:54:31] has resources to help you understand your health insurance coverage for HEMLIBRA. There are patient assistance options if you are eligible. Again, you can talk to your CEs. You can go down to the booth. You can find out more from HEMLIBRA.com.


I appreciate the opportunity to spend some time with you this morning. and thank you very much.


End of presentation.

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