US Hereditary Angioedema Association


IN THIS ISSUE
hae day :-) HAEA Virtual Capitol Hill Campaign HAEA Round Table Health Team Update Music Therapy Program Scholarships Physician Spotlight	Community Blog HAE Speaks Podcast #BeyondHAE Youth Podcast Clinical Trial Updates Education Webinar Virtual Meet & Greets

Let's Amplify Rare Stories On hae day :-) 2022

Let's make hae day :-) 2022 more meaningful than ever by flooding social media with stories about your individual HAE challenges and triumphs. We invite you to share your personal story with your social media networks on May 16th. You can participate by making a recording using Storyvine, an easy-to-use app that offers a step-by-step guide to creating video content.

Get started now by pledging to participate in this year’s hae day :-) campaign. We will then send you a training video along with a storytelling toolkit that includes: a tripod, a ring light, and access to our storyvine recording system.

We also have a way for you to participate if you prefer not to record a video. By answering a series of questions found here, your HAE journey can be captured and turned into a written story that you can post to your social media platform(s) on hae day :-).

If you have any questions about how to participate on this exciting hae day :-) campaign, please send an email to Jessica@haea.org.

Share Your Rare Story Here!

Join Us for the 2022 HAEA Virtual Capitol Hill Campaign

Every year, the HAEA organizes a large group of people with HAE and caregivers who visit over 50 strategically selected House and Senate offices. During these visits, HAEA friends showcase their advocacy skills by presenting the HAEA community's key public policy goals to legislators. Our experience shows that legislators respond when we take the time to explain the needs of the HAEA community.

We cordially invite you to participate in this year’s Virtual Capitol Hill Campaign on Tuesday, June 7th at 1:00 PM ET, during which we will discuss how you can participate to ensure our voices are heard in Washington and anywhere else where there are challenges that could hinder access to and reimbursement for HAE therapies.

We will engage our elected officials through virtual visits that highlight:

• Issues that will impact the HAE community in 2022
• Access to and reimbursement for HAE therapies
• Protecting financial assistance programs that help with the cost of HAE medicines

If you have any questions about participating in the Capitol Hill campaign, please send an email to Jessica@haea.org.

HAEA Round Table

Join us at the HAEA Round Table, where members of our community - share their experiences about living with HAE. The HAEA Round Table events are broadcast on Facebook Live, so make sure to tune in to the US HAEA official Facebook page to learn more about how others manage their HAE! The next event in the HAEA Round Table series will be on Tuesday, April 12th at 6:30 PM ET. This event will feature kids ages 12-15 and will showcase their answers to questions related to their personal experiences in living with HAE.

HAEA Round Table Calendar:

• April 12th
For Kids with HAE Ages 12-15 - Anxiety, Stress, and Growth with HAE
• June 14th
For Kids with HAE Ages 16-20 - How Do You Talk to Others About Your HAE?
• August 9th
For Young Adults with HAE - Growing Into Adulthood with HAE
• October 11th
For BabyBoomers with HAE - How Does HAE Affect Aging?
• November 1st
For Caregivers and Parents - What is the Best Way to Support Someone with HAE?

If you missed the February HAEA Round Table for Kids ages 5-11, you can access the recording here!

Health Team Update

April is National Stress-Awareness Month

Reading to Relieve Stress

Everyone feels stressed from time to time and that is NORMAL. Whether we are dealing with HAE or typical everyday situations, we need to have ways to relieve our stress, otherwise it can have physical and emotional side effects that make it difficult to live our best life. Here are some positive ways you can address the stress in your life and keep it in check.

• Make up your mind to stay/get healthy - Start by moving 10 minutes a day.
• Make a change in your routine - Get up a few minutes earlier each day to have some time just for YOU.
• Focus on NOW - Dwelling in the past or worrying about the future can cause unnecessary anxiety. If you don't have any control over it, let it go!
• Give yourself a pep talk - Try daily affirmations.
• Laugh a little - watch a funny video or read something to make you smile. Laughter really is the best medicine.
• Meditate - A still mind is a stress free mind. Begin with 10 min a day of stillness.
• Journal - focus on the things that made you happy today.
• Put a positive spin on it - Even if something went wrong in your day, try and list the things that went RIGHT.
• Help someone else - We feel an inner joy when we are able to help put a smile on someone else's face. Smiling is contagious.

When we take care of ourselves, we are better equipped to deal with our HAE! Small changes every day can make a BIG impact on our overall health.

The HAEA Health Team is always here for you! Please reach out to us at 866-798-5598 for assistance with 1.) insurance issues, 2.) finding an HAE expert physician, 3.) updating your member information, 4.) joining a virtual support group, or 5.) an HAE-related question.

The HAEA is offering Scholarships for Youth and Teens with HAE to enroll in a Music Therapy Program

Applications are open during the month of April - APPLY TODAY!

The US HAEA is pleased to offer scholarships for children and teens with a confirmed HAE diagnosis to participate in a virtual music therapy program hosted by the Children's Music Fund of Los Angeles (CMF).

The program will offer 12 virtual sessions with a certified music therapist to explore music through the lens of a creator, producer, and a student.

"I would definitely recommend this program to others because this has opened my creative ability to express my emotions through music." - 2021 CMF Scholarship Recipient

The HAEA and CMF Music Therapy scholarship is available to US citizens with a confirmed HAE diagnosis who can commit to completing 12 one-hour long virtual sessions with a licensed music therapist. The scholarship is available to kids and teens between the ages of 5 and 18.

Apply Today!

Throughout 2022, the US HAEA will spotlight Hereditary Angioedema (HAE) treating physicians who have made an impact within the HAE community.

This month, we will be recognizing HAE treating physician, Jonathan Bernstein, MD.

"I was very interested in the clinical conditions seen by an allergist and clinical immunologist during my residency and very fascinated by the immune system. Through my experience with rare diseases like HAE, it has been very gratifying to see how developing an understanding of the mechanism of action for this genetic disorder has led to a progression of treatments that today allow HAE patients to lead a normal life."

Thank you Dr. Bernstein for your dedicated service to the HAE community!

"HAEA youth members are tough, passionate, engaged, and truly remarkable advocates ready to stir the world with change. I gained a great deal from my experience during Capitol Hill Day and I hope to see more advocates out supporting individuals with HAE, their caregivers, family members, and other community members affected by HAE." - Courtney, Individual with HAE.

You can read more of Courtney’s experience with advocacy at the HAEA’s Capitol Hill Day now on the newly created HAEA Community Blog!

This blog highlights stories written by HAE community members, for HAE community members. A space to amplify our voices through our experiences, reflections, perspectives, and more!

Access the HAEA Community Blog today here.

Do you have a unique story or opinion to share about your HAE? The HAEA is excited to share your experiences in your own words with the HAEA community! You can share your story here.

Contact HAE Advocate, Hannah Carroll at hannah@haea.org for more information!

Podcasts

HAE Speaks Podcast

Download and listen to HAE Speaks, a podcast where people with HAE get the chance to broadcast their stories to the world! HAE Speaks gives you the opportunity to learn more about your own HAE through the shared experiences presented by our monthly hosts. You can download HAE Speaks on Apple Podcasts, Spotify, or wherever you listen to your podcasts!

Episode 24: Aysha shares her HAE journey. Aysha spent many years struggling for answers before an HAE diagnosis. Listen to hear how her years of questions were explained.

LISTEN NOW

Do you have a topic, question, or idea that you would like us to cover? Send your suggestion to Mike Mallory at mikemallory@haea.org.

#BeyondHAE Youth Podcast - Learning To Feel Comfortable With My Condition

Youth Podcast series

This month's episode of the #BeyondHAE Youth Podcast features HAEA Youth Advocate Sydney, who interviews HAEA Engagement Specialist, Lisa Facciolla. In the interview, Lisa talks about her life with HAE, challenges that she overcame, and how she found independence through self-administration.

#BeyondHAE is the only podcast around made exclusively by young people with HAE. Listen to #BeyondHAE to hear different perspectives of how you can live a normal life with HAE. #BeyondHAE is available to download on Apple Podcasts, Spotify, or wherever you get your latest podcasts!

LISTEN NOW

Clinical Trial Updates

What is a Clinical Trial?

Clinical trials are research studies conducted with people affected by HAE to evaluate whether a drug is safe and effective in humans. They are one of the final stages of the medical research and development process and follow strict standards to protect the health and safety of participants. Clinical trials are a critical part of drug development and are required before a treatment can be approved by the Federal Drug Administration (FDA) and become available by prescription.

What are the benefits of participating in a Clinical Trial?

Clinical trials can offer access to HAE medicines at no cost to the participant until the medicine is approved and available to be prescribed. Participation in a clinical trial also provides regular touchpoints with an HAE physician throughout the trial period. Enrolling in a trial is your decision, and you may withdraw at any time.

The US HAEA is helping to recruit for the clinical trials listed below.

If you are interested in participating or receiving more information on clinical trials, please reach out to the kind, compassionate, and knowledgeable HAEA Research Team at research@haea.org or via phone at 866-798-5598.

CSL312 (CSL Behring)

CSL312 is a worldwide clinical trial to investigate the safety and effectiveness of garadacimab, a subcutaneous preventive therapy given once a month to prevent HAE attacks. The medicine is a monoclonal antibody that is targeted to block Factor XIIa, which can be described as the “fuel” in human plasma that ignites an HAE attack.

The trial has reached its goal for adult participants, however, it is still recruiting for adolescents ages 12 - 17.

CSL312 is now an open label study with no placebo phase required, which means that all participants will receive garadacimab for at least one year, and the use of an on-demand medication to help with acute attacks is permitted.

KOMPLETE & KONFIDENT Studies (Kalvista Pharmaceuticals)

Kalvista is currently running two clinical trials to evaluate the safety and effectiveness of oral medicines that work by blocking kallikrein, a key component in the HAE attack biology.

KOMPLETE is a placebo-controlled trial examining three different oral dose levels of KVD824, a medicine designed to prevent HAE attacks taken twice a day. To qualify for the KOMPLETE study, participants must be 18 years of age or older and diagnosed with HAE Type I or II. Also, people in the study must have had two confirmed attacks in a 4 week period or three attacks over an 8 week period.

The KONFIDENT study is a placebo-controlled trial evaluating two different oral dose levels of KVD900, an on-demand treatment for HAE attacks. To qualify for the KONFIDENT study, participants must be 12 years of age or older and diagnosed with HAE Type I or II. In addition, prospective enrollees must have experienced two attacks in the last three months.

RAPIDe-1 & CHAPTER-1 (Pharvaris)

Pharvaris has initiated two clinical trials to investigate the safety and effectiveness of an oral medicine called PHVS416 that has the same biological action as Icatibant, a therapy given by subcutaneous injection that was approved in 2011. Both studies are recruiting participants between the ages of 18 and 75 who have been diagnosed with HAE Type I or II.

The RAPIDe-1 study is evaluating the safety and effectiveness of three different PHVS416 oral dose levels for treating HAE attacks once they have begun (also known as on-demand therapy). To qualify, participants must have experienced at least 3 attacks in the last four months or 2 attacks in the last two months.

PHVS416 is also being tested as a twice a day oral attack prevention therapy in Pharvaris's CHAPTER-1 clinical trial, which requires participants to have experienced at least 3 attacks in the last three months or 2 qualifying attacks during the screening period.

OASIS (IONIS Pharmaceuticals)

The OASIS trial, sponsored by IONIS Pharmaceuticals, is a Phase 3 worldwide placebo-controlled study that is evaluating the safety and effectiveness of a subcutaneous preventive medicine called donidalorsen, which is given either once every 4 or 8 weeks. This medicine uses a technology called antisense, which targets the kallikrein generating system using messenger RNA to suppress the protein that produces kallikrein.

To qualify for the OASIS trial, participants must be 12 years of age or older with a documented diagnosis of HAE Type I or II. Participants must experience at least two attacks during the screening period.

HAERMONY1 Study

BioMarin is testing BMN 331, a gene therapy that uses what is known as an Adenovirus Assisted Vector, called AAV for short, to produce a level of C1-Inhibitor protein sufficient to prevent HAE attacks. In general, the process for developing an AAV involves transforming a naturally occurring adenovirus into a delivery mechanism for gene therapy. This is accomplished by replacing the adenovirus DNA with the DNA of a healthy gene. The resulting AAV is then used to deliver normal copies of the healthy gene. Adenoviruses are used as the delivery mechanism for this technology because, in and of themselves, adenoviruses are not believed to cause any disease or illness.

The study, called HAERMONY1, is open to people who are 18 years of age or older, have been diagnosed with HAE due to C1-Inhibitor deficiency, and average at least one HAE attack per month. Study requirements include frequent visits and a long-term safety follow up (up to 5 years).

HAEA Treatment Education Series Webinar

The Emotional Toll of Living With HAE and Tips To Manage It

Date: Thursday, April 14th, 2022
Time: 7:00 PM E.T. / 4 PM PT
Speaker: John Anderson, MD
Sponsored by: CSL Behring
Webinar Link: https://haea.zoom.us/j/86149655804
Phone Number: +1 646 558 8656
(Webinar ID: 861 4965 5804)

HAEA Virtual Meet & Greet

Are You Interested In Meeting Others In The HAE Community?

Do you have questions for others living with HAE? Or would you just like to network with other HAEA members? The HAEA Virtual Meet & Greet Events are a perfect way to reach out and get to know others affected by HAE. Each month HAEA staff will be presenting on programs, resources, and relevant HAE topics as well as providing an opportunity for the HAE community to meet each other. If you are interested in participating in an HAEA Virtual Meet & Greet, please check out the HAEA Events Calendar, or reach out to Mike Mallory at mikemallory@haea.org.

What would you tell someone who is considering attending an HAEA Meet & Greet?

"It's as if you're hanging out at your favorite coffee house and telling a story with your friends! "
~ Dano K.

Upcoming HAEA Virtual Meet & Greet Events

Tuesday, April 19th, 6:30 PM PT

Tuesday, May 24th, 6:30 PM ET


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The HAEA is an advocacy and research organization committed to actively engaging our community in a wide variety of grassroots activities that promote HAE education and awareness. We provide personalized services to address the unique needs of people with HAE and their families, which includes helping them secure access to and reimbursement for modern HAE medicines. Our great success in supporting clinical research has resulted in a variety of FDA-approved therapeutic options. We work closely with expert physicians to continuously upgrade our community's quality of life through improving diagnosis and knowledge of the condition, and encourage an individualized approach to selecting an optimal treatment. The HAEA is product and company neutral, and continues to enthusiastically support drug discovery research aimed at the next generation of HAE therapies. Copyright © (2022) US Hereditary Angioedema Association All rights reserved. No part of this publication may be reproduced, stored in a retrieval system, or transmitted in any form by any means, mechanical, electronic, photocopying, recording, or otherwise without the prior written permission of the US Hereditary Angioedema Association. For information, send an email to info@haea.org.