US Hereditary Angioedema Association


IN THIS ISSUE
hae day :-) Women With HAE Virtual Capitol Hill Campaign Voter Voice HAEA Round Table Physician Spotlight	HAE Speaks Podcast #BeyondHAE Youth Podcast Clinical Trial Updates Education Webinar Virtual Meet & Greets

Dear HAEA Friends,

On May 16th, 2012 and every year since, people with HAE, caregivers, families, healthcare practitioners, scientists, and the pharmaceutical industry commemorate hae day :-). This special day of action includes HAE awareness and educational activities. Also, hae day :-) serves as a rallying point for sustaining the dedication of a truly unique group that has achieved something unprecedented for an ultra rare condition: 8 FDA approved medicines with 13 in various stages of development.

The HAEA community always recognized the need for federal legislative action to promote national awareness aimed at improving the lives of people with HAE. In the fall of 2011, the HAEA community participated in an outreach campaign seeking an elected representative who would champion our cause. National in its scope, this effort proved successful when the Senior senator from Hawaii (and then President Pro Tempore of the Senate) Daniel K. Inouye agreed to sponsor legislation designating a national day of HAE awareness. Known as an able negotiator and alliance builder, Senator Inouye used his skills to gain support from his colleagues before sending Resolution 286 to the floor of the United States Senate on Tuesday, January 31st, 2012. The Resolution received bi-partisan support with unanimous approval for establishing May 16th as hae day :-).

Every year on May 16th, the HAEA community raises awareness by hosting fundraisers, distributing educational materials such as the HAEA ER Toolkit, and/or sharing personal stories on social media platforms. In addition, the success in engaging Congress led to our annual Capitol Hill Day, a popular and well attended event that provides opportunities for us to engage and inform our elected representatives.

We look forward to you joining the community and participating in celebrating hae day :-) 2022.

Sincerely,

Tony Castaldo

Anthony Castaldo
US HAEA President & CEO

Join the HAE community this hae day :-) by sharing your personal story to elevate and amplify our voices, and we will send you a storytelling tool kit that includes a tripod, access to our storyvine recording system, and a thorough training on how to tell your story! Whether you prefer to share your hae story in written or video formats, we can't wait to hear from you.

Time is running out! Please submit your story TODAY to be featured on the HAEA website and social media channels.

Share Your Rare Story Here!

Women with HAE Face Unique Challenges When Going Through Puberty, Pregnancy, And Menopause

Connect with Dr. Raffi Tachdjian and Lisa Facciolla (HAE Advocate) on Tuesday, June 28 at 7:30 PM ET, for the first of three Women with HAE special series webinars – Pediatrics through Puberty. This special series webinar event will answer frequently asked questions about how young girls experience Hereditary Angioedema (HAE), and provide tips on the management of the condition. It is a great resource for parents and children who are interested in learning more about how HAE impacts the health of young girls.

We invite you to submit questions in advance to Lisa Facciolla at lisa@haea.org, and Dr. Raffi Tachdjian will answer them live during the webinar.

Individuals who register for the event will be mailed a FREE printed copy of the recently released Women with Hereditary Angioedema booklet that has been developed to help women and young girls with HAE navigate three specific life stages:

1) pediatrics through puberty;
2) family planning and pregnancy; and,
3) menopause and aging

This FREE resource is now available! If you are interested in ordering the Women and HAE booklet for yourself or other women in your life affected by HAE, you can access the order form here.

For more information or questions on the Women and HAE booklet or the Women with HAE – Pediatrics through Puberty webinar, please contact Lisa Facciolla at lisa@haea.org.

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. Capitol Hill visits provide an opportunity for people in our community to use their advocacy skills by sharing their stories with elected officials, and highlighting issues that are important to the HAE community. Our experience shows that our elected officials respond when we take the time to explain the needs of the HAEA community.

The HAEA invites you and your family to participate in a Capitol Hill “Visit” Campaign from June 7 through June 10, 2022!

The Campaign will kick-off on Tuesday, June 7 at 1:00 PM ET/10:00 AM PT, with an informative virtual event that will focus on the key issues that affect the HAE community and training on how to be an effective advocate.

We need HAEA friends to either write a letter or tell your story in a virtual meeting with one of your elected officials.

Register early to ensure you have the opportunity to connect with your local legislators through the HAEA’s Capitol Hill Campaign!

The HAEA will work with you to coordinate writing a letter or to schedule a virtual Capitol Hill visit. The visits will take place from June 8-10 and will focus on:

1) gaining sponsorship and support for legislation crafted by the HAEA, and

2) advocating for additional NIH research funding which is crucial for the continued advancement of HAE therapies.

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

The HAEA Will Be Launching A New Automated System For Advocacy: Voter Voice!

Advocating for HAE just got easier! With Voter Voice – an automated system to help you advocate – the HAEA will be able to send you emails with issues that are pressing for our community while providing you with the tools to make your voice heard in just a few steps. If you choose to sign on, you can use one of our pre-written letters to send to your government representatives. When you write in your address, Voter Voice will find your elected officials and send the letter directly to them.

If you are interested in participating in advocacy outreach through Voter Voice with the HAEA, please contact Jess Myers at Jessica@haea.org. or fill out the advocacy form on the HAEA website here.

HAEA Round Table

Join us at the HAEA Round Table, where members of our community are able to talk about how they have addressed the unique challenges of 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, June 14th at 6:30 PM ET. This event will feature children ages 16-20 and will showcase their answers to questions related to their personal experiences living with HAE.

HAEA Round Table Calendar:

• June 14th
For Youth 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 Baby Boomers 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 and April HAEA Round Tables, you can access the recordings here!

Throughout 2022, the US HAEA is spotlighting Hereditary Angioedema (HAE) treating physicians who are making a positive impact in the HAE community.

This month, we are recognizing HAE treating physician, Mark Davis-Lorton, MD!

"The biggest piece of advice I can give to patients with rare diseases is to understand as much as they possibly can about their own disease and teach every person they come in contact with about their disease (every nurse, every healthcare worker). If you are seeing a doctor who does not understand what you are going through, explain it to them! Understand your disease enough such that you can explain it to new providers. You may be the only person that the healthcare provider has ever come in contact with that has the disease, and your interaction may help in the diagnosis of others"

Thank you Dr. Davis-Lorton for your dedicated service to the HAE community!

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 25: Lisa and Mike discuss hae day :-) a special day dedicated to Hereditary Angioedema. Listen in as Lisa and Mike talk about the importance of hae day :-) and the HAEA 2022 hae day:-) campaign.

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 - HAEA Round Table: How Does It Feel to Be a Kid with HAE?

Youth Podcast series

This month's episode of the #BeyondHAE podcast features four young people with HAE between the ages of 7 and 11 who talk about what it's like to have HAE. These young individuals discussed relevant topics such as what it's like for them to grow up with HAE, the fear of needles, discussing their HAE diagnosis with teachers and friends, and more!

#BeyondHAE is the only podcast made exclusively by young people with HAE for 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 for the duration of the trial, before it is available to everyone. Participation in a clinical trial also provides regular touchpoints with an HAE physician throughout the trial period, and allows you to have a more active role in your health care. Your participation in a trial is your decision, and you have the ability to withdraw if the trial is not working for you without judgment or jeopardizing your health care.

Following is a list of clinical trials currently underway. If you are interested in receiving more information on clinical trials, or are interested in participating in any of the trials listed below, please reach out to the HAEA Research Team at research@haea.org.

CSL312 Study

The US HAEA is currently assisting CSL Behring in recruiting people with HAE for the CSL312_3002 Garadacimab study, a worldwide clinical research study to investigate the safety and effectiveness of an investigational subcutaneous medication given once a month to prevent Hereditary Angioedema (HAE) attacks. In this study, doctors will be evaluating the long term use of garadacimab.

The trial is currently fully recruited for adult participants, however, a few more adolescent participants (ages 12 - 17) are needed to meet the required numbers.

This is 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.

RAPIDe-1 & CHAPTER-1 Studies

The US HAEA is currently assisting Pharvaris in recruiting people with HAE for two clinical research studies to investigate the safety and effectiveness of an investigational oral drug for both acute and prophylactic use of Hereditary Angioedema (HAE) attacks.

In the RAPIDe-1 study, doctors will be evaluating how effective three different doses of the study medication (PHVS416) are in relieving symptoms associated with HAE attacks. The study drug is in the form of soft capsules taken by mouth. In the CHAPTER-1 study, doctors will be evaluating the effectiveness of PHVS416 as a potential oral treatment to prevent HAE attacks.

To qualify for the RAPIDe-1 study, participants must be diagnosed with HAE Type I or II and be between the ages of 18 and 75 years old, with at least 3 attacks in the last four months or 2 attacks in the last two months.

To qualify for the CHAPTER-1 study, participants must be diagnosed with HAE Type I or II and be between the ages of 18 and 75 years old, with at least 3 attacks in the last 3 months or 2 qualifying attacks during the screening period. The use of an approved on-demand medication to treat acute attacks is permitted.

The OASIS Study

The US HAEA is currently assisting IONIS in recruiting people with HAE for The OASIS study, which aims to find out whether the study drug (called donidalorsen) is safe and effective at reducing or preventing HAE attacks.

This is a Phase 3 worldwide placebo-controlled trial where doctors are studying donidalorsen injections to determine safety and effectiveness at reducing or preventing HAE attacks. Participants may be eligible to participate in an open-label extension study at the end of The OASIS study, where all participants receive donidalorsen.

To qualify for The OASIS study, participants must be 12 years of age or older with a documented diagnosis of HAE. Participants must experience at least two attacks during the screening period, and have access to and the ability to use at least one or more acute medications to treat HAE attacks.

KOMPLETE & KONFIDENT Studies

The US HAEA is currently assisting Kalvista in recruiting people with HAE for the KOMPLETE and KONFIDENT studies to understand the effects of investigational oral medications called KVD824 and KVD900.

The KOMPLETE study is a placebo-controlled trial where doctors are studying three different doses of KVD 824-201 to see which one may be best as a safe prophylaxis treatment for HAE attacks. To qualify for the KOMPLETE study, participants must be 18 years of age or older and diagnosed with HAE Type I or II. You will also need to have access to an on-demand medication to use for HAE attacks, and be willing to use a study-approved method of birth control during the study.

The KONFIDENT study is a placebo-controlled trial where doctors are studying two different doses of KVD 900 to see which one may be best as a safe 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. Participants will need to have two attacks in the last three months, have access to an on-demand medication to use for HAE attacks, and be willing to use a study-approved method of birth control during the study.

HAERMONY1 Study

The US HAEA is currently assisting BioMarin in recruiting people with HAE for the HAERMONY1 study, which is an investigational gene therapy called BMN 331 to improve HAE symptoms in adults 18 years of age and older diagnosed with HAE due to C1-INH deficiency.

To qualify for the HAERMONY1 study, participants must be 18 years of age or older and diagnosed with HAE due to C1-INH deficiency. You will also need to use preventative medication to prevent attack or an on-demand medication during HAE attacks. Participants must also have at least one HAE attack per month on average.
*Additional study requirements apply.

For those who qualify, participation lasts about 5 years after receiving the study drug. There are weekly and monthly visits during the first year. After the first year, the visits occur every three or every six months.

If you have HAE Type I, Type II, or HAE with Normal C1-Inhibitor and are interested in learning about new clinical studies to prevent attacks, please contact an HAE advocate at 866-798-5598 to see if there will be an active clinical trial site in your area.

HAEA Treatment Education Series Webinar

Oral Attack Prevention That Is Big For HAE Yet So Small For Your Day

Date: Thursday, May 26th, 2022
Time: 7:00 PM ET / 4 PM PT
Speakers: Daniel Soteres, MD, Steve (BioCryst Patient Ambassador) & Rochelle (BioCryst Care Coordinator)
Sponsored by: BioCryst
Webinar Link: https://haea.zoom.us/j/81407149178
Phone Number: +1 646 558 8656
(Webinar ID: 814 0714 9178)

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 present our programs, resources, and relevant HAE topics while hosting an opportunity for the HAE community members to get to know each other better. 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?

"This is a great opportunity to learn more about HAEA and connect with other patients. Community is so important in this journey with a rare disease and the HAEA Meet & Greet is a great place to begin to build those relationships."
~ Nancy G.

Upcoming HAEA Virtual Meet & Greet Events

Tuesday, May 24th, 6:30 PM ET

Tuesday, June 21st, 6:30 PM CT


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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.