US Hereditary Angioedema Association


IN THIS ISSUE
HAE IN-MOTION^® Advocacy Action Alert HAEA Round Table Women With HAE Health Team Update Physician Spotlight	Community Blog HAE Speaks Podcast #BeyondHAE Youth Podcast Clinical Trial Updates Education Webinar Virtual Meet & Greets

Time is Running Out! Register for the 2022 HAE IN-MOTION^® Step it Up for HAE event TODAY!

Don’t miss your chance to win amazing prizes in the 2022 HAE IN-MOTION^® Step it Up for HAE event! The nine week-long community virtual walk, run, or ride allows you to log your steps and activity directly in our OneCause platform, all while raising HAE awareness! By participating, you can help us reach our target of 50,000 miles by July 31, 2022, and raise funds to help support the HAEA Research Fund.

Register TODAY to receive your 2022 HAE IN-MOTION^® - Step it Up for HAE event swag bag with AMAZING items, including a t-shirt, water bottle, 2022 HAE IN-MOTION® medal, and more!

Grand prizes will be awarded to the participants with:

Top overall fundraising total
Most miles completed
Top gallery points
Top team member recruitment

First, second, and third place winners in each category will receive $100, $50 and $25 gift certificates to the HAEA Shop!

For more information or questions contact Mike Mallory at mikemallory@haea.org.

Have fun with your HAEA family, raise awareness and funds, and compete for a chance to win great prizes! Register for the 2022 HAE IN-MOTION^® - Step it Up for HAE event TODAY!

Support the Safe Step Act to Ensure People with HAE Get the Medications They Need!

Many health insurance plans will not initially fill a physician's prescription, and instead require that a patient fail on an alternative, less expensive treatment. This dangerous practice - commonly referred to as fail first or step therapy - takes treatment decisions out of the physicians' hands and places it with the insurance provider. Given the seriousness of HAE, any interference with continued access to the physician-prescribed medication could prove to be fatal.

The Safe Step Act would protect members of the HAE community who obtain coverage in self-funded or federally regulated insurance plans by (1) establishing exemptions to fail first, and (2) require plans to respond to appeals in a specific time period.

You can take action TODAY by sending our pre-written letter to ask your Senators and Representatives in the House to support this bill. Click HERE to access our letter template!

HAEA Round Table

Join us at the HAEA Round Table, where members of our community discuss how they have dealt with 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, August 9th at 6:30 PM ET. This event will feature young adults ages 16-20 and will showcase their answers to questions related to their personal experiences in living with HAE.

HAEA Round Table Calendar:

• 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 previous HAEA Round Table, you can access the recordings here!

Women with HAE - Join HAE Expert Physician Sandra Christiansen for a Special Webinar On Family Planning and Pregnancy

Connect with Dr. Christiansen and HAE Advocate, Lisa Facciolla, on Tuesday, July 26 at 7:30 PM ET / 4:30 PM PT, for the second of the Women with HAE special series webinars: Family Planning & Pregnancy. The event will be a great resource for anyone who is interested in learning more about what to consider when planning for a family, and what women with HAE should expect and plan for during pregnancy.

We invite you to submit questions in advance to Lisa Facciolla at lisa@haea.org, and Dr. Christiansen will answer these live during the webinar. Individuals who register for this event will be mailed a FREE printed copy of the recently released Guide for Women with HAE 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
3) menopause and aging

If you missed the first webinar you can click here to watch the recording.

Order your copy of the Women and HAE guidebook here.

For more information or questions on the Women and HAE guidebook or the Pregnancy and Family Planning webinar, please contact Lisa Facciolla at lisa@haea.org.

Health Team Update

Did You Know That The HAEA Offers Virtual Support Groups?

The HAEA offers virtual support groups for people affected by HAE and their caregivers. These support groups provide an opportunity to talk about your concerns, discover useful tips on managing stress, and find comfort by sharing with other people with HAE and their caregivers in a safe environment.

HAEA support groups are offered twice monthly. You can choose to attend in the morning or evening depending on what works best with your schedule. Everyone is welcome to join and participate at their comfort level.

If you are interested in joining a virtual support group, please reach out to Troyce Venturella at troyce@haea.org for more information!

The HAEA also offers assistance with medication denials and appeals. If you need help accessing your HAE medications, reach out to our health team today at (877) 839-4232.

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.) HAE-related questions.

Throughout 2022, the US HAEA is spotlighting HAE treating physicians who have made an impact within the HAE community.

This month, we will be recognizing HAE treating physician, John Anderson, MD!

"Over these last 10 years, my nurses and I have had the joy of helping individuals with Hereditary Angioedema transition their treatment to more effective forms of rescue therapy and prophylaxis. Watching their lives change has been amazing! By far this is one of the most rewarding experiences in my life."

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

"My journey to diagnosis was not easy, and there is still so much that I am learning about my diagnosis. However, I am living a full, active, and joyful life because of the decisions my doctor and I have made. I am living beyond my HAE!"

You can read the rest of Kayla’s HAE journey and how advocating for herself led to a successful HAE diagnosis that saved her life in this month’s featured HAEA Community Blog article!

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 27: In this episode of the HAE Speaks podcast, we listen to Abby share her experience and excitement about being a part of the HAEA Brady club.

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: For Kids Ages 12-15

Youth Podcast series

This month's episode of the #BeyondHAE podcast features two young people with HAE between the ages of 12 and 15 who talk about anxiety, stress, and growing up with HAE. This Round Table features topics such as how to talk about your HAE with others, how it feels to have the HAE diagnosis, how to tell when an attack is coming, and how to manage fear of needles. Our two participants, Lexi and Layna, also share advice they would give to other kids who have been recently diagnosed with HAE. Listen now to hear insight from kids with HAE!

#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 an open label extension, which means free access to HAE medicines being studied until they are approved and commercially available. 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.

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, a worldwide clinical research study to investigate the safety and effectiveness of Garadacimab, 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.

CSL312_3002 is an open label study with no placebo phase required, which means that all participants will receive garadacimab for at least one year.

CHAPTER-1 Study

The US HAEA is currently assisting Pharvaris in recruiting people with HAE for a clinical research study to investigate the safety and effectiveness of an investigational oral drug for prophylactic use of Hereditary Angioedema (HAE) attacks. 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 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-HAE study, a Phase 3 worldwide placebo-controlled trial, which aims to determine whether its study drug (called donidalorsen) is safe and effective at reducing or preventing HAE attacks.

Participants may be eligible to participate in an open-label extension study at the end of the OASIS-HAE study, where all participants receive donidalorsen.

To qualify for the OASIS-HAE 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; diagnosed with HAE Type I or II; 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 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; diagnosed with HAE Type I or II; experienced 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, 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; diagnosed with HAE due to C1-INH deficiency; have 6 months of a continuous prophylaxis or on-demand treatment regimen (prior to study enrollment); and a documented attack frequency of at least 1 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

TAKHZYRO (lanadelumab) + You:
Re-Imagine Your Life with HAE

Date: Thursday, July 21st, 2022
Time: 7:00 PM ET / 4 PM PT
Speakers: Douglas R. Lotz, MD & Jack (Person living with HAE)
Sponsored by: Takeda
Webinar Link: https://haea.zoom.us/j/87937005387
Phone Number: +1 646 558 8656
(Webinar ID: 879 3700 5387)
Important Safety Information

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 like to network with other HAEA members? The HAEA Virtual Meet & Greet Events are the perfect venue 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 get to know 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 is a great space to learn that you are not alone."
~ Danita S.

Upcoming HAEA Virtual Meet & Greet Events

Tuesday, July 19th, 6:30 ET

Tuesday, August 8th, 6:30 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.