A1FNC24 Program Book

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POWER POWER I S I N YOU YOU T H E

M A K E A

I F F E R E N C E # A 1 F N C 2 4 A L P H A - 1 F O U N D A T I O N N A T I O N A L C O N F E R E N C E PROGRAM PROGRAM BOOK BOOK M I A M I , F L | J U N E 7 - 9 , 2 0 2 4

T O
D

ALPHA-1 FOUNDATION

NATIONAL CONFERENCE

Patient Confidentiality

Due to the sensitivity of health information, we encourage you to make an informed choice about whether or not to provide your personal information to the organizations exhibiting and participating at this conference.

Please be advised that the Alpha-1 Foundation (A1F) is taking photos and video throughout the conference. All photos and video taken by the official photographer and videographer may be used in A1F publications or reproduced to be used for newsletters, promotional materials, inclusion on websites or any other purpose. You release A1F from any liability connected with the taking of photographs and video. If you do not wish to have your picture used in any publication, please let A1F know in writing.

The Alpha-1 Foundation is pleased to present the 33rd Annual
Photography
Table of Contents 4-5 Things to Know & Conference Highlights 6 2024 Board of Directors 7 Welcome Letter 8 Alpha-1 Foundation Staff 9 Riding For A Reason 10-11 Alpha-1 Awareness Month & A1F Virtual Walk 12-13 Alpha-1 Grant Awards 14 Alpha-1 Research Registry 14 Wave Life Sciences 15 Alpha-1 Advocacy 15 Beam Therapeutics 16 Clinical Trials Education Program 16 The Redwood Study 17 2025 Alpha-1 Foundation National Conference 17 Inhibrx 18 Acknowledgements & Exhibitors 19-21 Conference Agenda 22-32 Speaker Bios 23 Vertex 24 AlphaNet 27 CSL Behring 30 Grifols 33 Takeda 35-39 Note Pages 3 2 2024 Alpha-1 Foundation National Conference � Miami, FL � June 7-9, 2024 The POWER is in YOU to make a difference

THINGS TO KNOW THINGS TO KNOW

Exhibit Hall Hours

Friday at 12:30 pm

Friday, June 7th

12:30 pm – 5:00 pm

Saturday, June 8th

8:00 am – 5:00 pm

Alpha-1 Kids Room

The Alpha-1 Kids Room is open Saturday, June 8th from 8:30 am to 5:00 pm in Tenor room.

The Alpha-1 Kids Room will be closed during lunch from 12:30 pm to 2:00 pm.

Dress Code

Dress is casual for all conference meetings, sessions and dinners.

You may want to bring a sweater or light jacket, as the meeting rooms in the hotel may be cold.

Please avoid wearing perfumes, colognes or other scented items. This can trigger a serious asthma attack for some Alphas.

Cell Phones/Electronics

Please silence cell phones and other electronic communication devices during the general and breakout sessions.

Name Badges

Official conference name badges MUST be worn at all times while in the conference areas in order to enter the exhibit hall, attend sessions and meals. Anyone not wearing an official conference name badge will be asked to leave the conference area.

Company badges may also be worn, but will not be accepted as a replacement of the official conference badges. Supplementing badges with business cards or altering, adding to or defacing the official conference badge is not permitted.

What’s included in your name badge?

Ribbons - If you have a ribbon(s), stick it to the bottom of your nametag. Visit the ribbon wall to add more!

Drink Tickets - There are four (4) drink tickets in your nametag provided by A1F; two (2) for each dinner.

Special Dietary Requests

Participants who have made a special dietary request will have it noted on their name badge in the bottom right hand corner.

The hotel will indicate allergens at buffets, if applicable. If you have questions about allergens, please make sure to let a banquet server know.

CONFERENCE HIGHLIGHTS CONFERENCE HIGHLIGHTS

Friday,

June 7th

Exhibit Hall Grand Opening

Enjoy MIAMI-inspired sounds and bites!

The Power is in You:

Alpha-1 Programs, Services, & Getting Involved

Enjoy an ice cream bar and learn how to make a difference in the Alpha-1 community. Meet Alpha-1 rockstars and volunteers who have been involved in the community and make a difference every day

MIAMI Dinner Reception

A MIAMI inspired evening with A1F! Enjoy Miami inspired food stations, drinks, and a personal appearance by some of Miami’s most famous. Fun giveaways throughout the evening.

Saturday,

June 8th

GLOW PARTY

Dinner Reception

Enjoy Downtown Miami fusion inspired food stations, GLOW entertainment, and drinks. Fun giveaways throughout the evening and the exhibitor passport drawing

Sunday, June 9th

Bring You Back 2025 Beach Bash Brunch

A farewell breakfast celebration you won’t want to miss! Bring your conference tote to brunch to fill your beach bag for 2025 with fun items

Take a video at the 360-photo booth and don't miss your opportunity to win a trip to Disney World!

A1FNC24 T-Shirt

fficial T-shirt will be available for purchase at "Fundraising & Getting Involved" Booth All ions go toward research & related program

Adult T-Shirts: $25 | Kid T-Shirts: $15

Alpha Teddy

Limited Edition Alpha-1 Teddy Bear will be available for purchase at the "Fundraising and Getting Involved" Booth.

1 for $25 | 2 for $40

Available for Purchase

5 5 4 2024 Alpha-1 Foundation National Conference � Miami, FL � June 7-9, 2024

WELCOME LETTER WELCOME LETTER

Welcome to the 2024 Alpha-1 Foundation (A1F) National Conference The theme this year is “The Power is in You to Make a Difference” We are excited to kick off the largest annual gathering of the Alpha-1 community, both in-person and on our virtual platform. This year s theme acknowledges the spirit of the Alpha-1 community and the power we each have to make a difference. Over the next 3 days you will hear from leading Alpha-1 experts. You will meet fellow Alphas You will learn all about the A1F’s programs and resources that can empower you every day and hopefully you will find the power in you to get involved!

Since 1995, A1F has invested over $100 million to support Alpha-1 research at 130 institutions in North America, Europe, the Middle East, and Australia making us the largest private funder of Alpha-1 research in the world We continue to invest boldly and wisely funding only the most promising science to accelerate treatments for the underlying causes of Alpha-1 and to find a cure for everyone diagnosed with this condition

A1F is the leader in providing Alphas and their families with the support, education and resources they need throughout their Alpha-1 journey At A1F the patient is always at the center of everything we do as an organization. The force behind all our success is the Alpha-1 community Its passion fuels our shared mission, and there is no doubt that together, we will achieve our goals.

We are at an exciting and pivotal moment in the history of Alpha-1 The marriage of science, medicine, and technology, along with the leadership of A1F, has us primed to deliver for patients and their families. More advancements are on the horizon today than ever, and our commitment to supporting breakthrough initiatives in Alpha-1 is greater than ever We know we cannot do this alone We will go fast and far together with you: the power of the patient, providing the inspiration that fuels our efforts

This year’s theme, “The Power is in You to Make a Difference ” blends all the programs and activities of A1F with how you can engage and empower yourself as a patient Weaved throughout the conference are opportunities to find and use that “power.” Your involvement is integral in moving our shared mission forward. The Power is in You to:

1

Educate yourself & support others affected by Alpha-1

B O A R D M E M B E R S

Raise awareness & advocate for Alpha-1 2. Make a difference in the Alpha-1 community 3 Participate in research to find a cure for Alpha-1 4

We encourage you to learn more about how to get involved by visiting our official A1F foyer, highlighting our programs and services. The power is in you to connect with all our partners to assist you in your own patient journey Join us at the Exhibit Hall Grand Opening on Friday afternoon at 12 30 pm to learn more

Special thanks to all our presenters for giving their valuable time for this conference and throughout the entire year to make our educational programming top-notch. Thank you to our sponsors for their commitment and dedication to the Alpha-1 Community: Platinum Sponsors: AlphaNet, CSL Behring, Grifols Takeda and Vertex, Gold Sponsor: Accredo by Evernorth, Silver Sponsor: CVS Health Emerging Therapies Sponsors: Beam Therapeutics BioMarin Pharmaceutical, Inhibrx, Korro Bio, Takeda and Wave Life Sciences

Be sure to join us for Sunday s closing event, the “Bring You Back 2025 Beach Bash Brunch ” to get ready for next year’s conference celebrating the Foundation’s 30th anniversary!

To our patients and your families, thank you for your support. This is your conference, find the power in you and make a difference in your life and the lives of others!

Sincerely

Mark Brantly, MD Flor da Jennifer Jopp Minnesota Ann Knebel, PhD RN Maryland Darrell N. Kotton MD Noel G. McElvaney, MD, BCh BAO, FRCPI, FRCPC Ireland Faron Schonfeld Pennsylvania Catherine Vernon Florida Martin Zamora, MD Colorado
Massachusetts
7 6 2024 Alpha-1 Foundation National Conference � Miami, FL � June 7-9, 2024 The POWER is in YOU to make a difference

"Riding for a Reason" Virtual Bike Trek

"Escape

to the Cape" in person at Cape Cod, MA on September 27th - 29th &

VIRTUAL CHALLENGE - RIDING FOR A REASON

We are asking the Alpha-1 community to bike, foot pedal, or e-bike towards their personal goals during the month of September to help raise funds and awareness for Alpha-1. We will stay connected using the hashtag #Alpha1RidingforaReason on social media and weekly Zoom calls!

Get your bikes in gear and ride as many miles as you can to help bring us closer to our mission of finding a cure. Whether you are riding for yourself or in honor of a loved one, this is a great way to make a difference!

IN-PERSON EVENT - ESCAPE TO THE CAPE

Join Team Alpha-1 for a one, two, or three-day Escape to the Cape bike trek in Cape Cod, Massachusetts. We are recruiting riders for the weekend to help us cross the finish line! Riders will enjoy a bike ride along the picturesque coast of the Cape. This is a fun-filled weekend with the Alpha-1 community cheering each other on and raising funds and awareness for the cure.

This event is open to the public and is in partnership with the American Lung Association. Register today: give.alpha1.org/RidingforaReason2024

Keep on track with the “Good Move” activity and fundraising phone app. Fundraising has never been so easy and as much fun!

For more information, please contact Irene Calderon at icalderon@alpha1.org

For Corporate Sponsorship information, please contact Angela McBride at amcbride@alpha1.org

ALP ALPH
Joy Alison
A L PHA-1RIDING
O N S E P T E M B E R 2 0 2 4 #ALPHA1RIDINGFORAREASON
FORAREAS
8 2024 Alpha-1 Foundation National Conference � Miami, FL � June 7-9, 2024

THE P WER TO C NNECT

2024 ALPHA-1 AWARENESS MONTH

November is Alpha-1 Awareness Month, a 30-day initiative that encourages the Alpha-1 community to share stories and resources and raise funds to support the search for a cure.

This year’s theme is “The Power to Connect,” highlighting the importance of connecting with your fellow Alphas through Alpha-1 Support Groups, Alpha-1 specialists in Clinical Resource Centers, legislators on Capitol Hill, and your local community to raise #Alpha1Awareness.

Whether you post on social media, fundraise for the 2024 Alpha-1 Foundation Virtual Walk, or sign up for the Alpha-1 Research Registry, you have the power to connect.

To learn more about events happening during Alpha-1 Awareness Month, visit: alpha1.org/alpha-1-awareness-month/

2024 ALPHA-1 FOUNDATION VIRTUAL WALK

Help us raise funds and awareness for Alpha-1! You have “The Power to Connect” to help find a cure.

Connect with friends and family to boost your fundraising, whether at home, in your neighborhood, or at your local park. Use the “Good Move” app to make it easy and fun!

Start a team or join a team in your area. You have the power to connect to the Alpha-1 community!

Registration is $25 and includes the official 2024 A1F Virtual Walk T-shirt.

For more information, contact Irene Calderon at icalderon@alpha1.org.

REGISTER TODAY! #A1FVW24 give.alpha1.org/VirtualWalk24

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THE GRANTS AWARD PROGRAM THE GRANTS AWARD PROGRAM

The Alpha-1 Foundation hosted a reception on Monday, May 20, 2024, to honor the newly awarded research grant recipients, where over 160 guests joined at The Point, Hilton San Diego, in San Diego, CA, in conjunction with the 2024 American Thoracic Society (ATS) International Conference.

The Foundation announced the in-cycle grant awardees for 2024, 19 grant recipients will receive a total of $3 million for their two-year projects. This year’s grant categories consist of 11 Research Grants, 4 Pilot and Feasibility Grants, and 4 Postdoctoral Research Fellowship Grants.

Since 1995, the Foundation has invested over $100 million to support Alpha-1 Antitrypsin Deficiency (Alpha-1) research and programs at more than 130 institutions in North America, Europe, the Middle East, and Australia. Annual in-cycle grants are a regular part of the Foundation’s overall research program. Of this year’s grant awards fields of study, 63% are lung-focused, 32% are liver-focused, and 5% are focused on Ethical, Legal, and Social Issues relating to Alpha-1 Antitrypsin Deficiency. The research includes a mix of basic research, translational research, and clinical research topics that maintain a well-rounded portfolio.

The Grants Award Program is the Foundation’s mechanism to fund a broad range of research that leads to improvements in the lives of people living with Alpha-1. The specific goals of the program are:

To promote basic science and clinical research related to the alpha-1 antitrypsin protein and Alpha-1;

To attract and train clinical researchers for the study of Alpha-1;

To support and encourage established scientists to work on clinical problems and ethical, legal, and social issues (ELSI) within the field of alpha-1 antitrypsin research (with a preference given to new investigators); and ultimately,

To develop effective therapies for the clinical manifestations of Alpha-1.

For more information on Alpha-1 research, please visit alpha1.org.

ARDEES ARDEES

PhD Ce lular Effects of A1AT Aggregat on with Sing e-Cell and Cross inking Proteomics

& Feasibility nt Awardees

Roche,

Genetic D scrim nation –Tackl ng a Grow ng Issue in Alpha-1 Antitrypsin Deficiency

EHuiliang Wang PhD Genetic Engineered Exosomes for Effic ent DNA Delivery for Alpha-1 Ant trypsin Defic ency

Werder, PhD Investigating the Ce lu ar and Molecular Mechan sms of Respiratory Infect ons n AATD

Research owship Grant Awardees

PhD Regeneration of Lung Epithel a Stem Cell Compartments in a Novel Z-AAT Mouse Model

Mark Murphy, PhD A Detailed Study of Lung Immune Cell Outcomes in AATD

Schiano, PhD Targeting Inflammation as Therapeutic Approach for AATD FGF21 and Cellular Homeostasis in Alpha1-Antitryps n Deficiency

Emily Moser, PhD Alpha-1 Antitrypsin Promotes Vaccine Antibody Responses Suzanne MB, BCh, BAO Rhiannon
ot
Hirofumi Kiyokawa, MD, Valentina
tdoctoral
Ivera Khodzhaeva, PhD Konstantinos Thalassinos,
13 12 2024 Alpha-1 Foundation National Conference � Miami, FL � June 7-9, 2024 The POWER is in YOU to make a difference

Join the Alpha-1 Research Registry

We are recruiting for Alpha-1 related studies and need your participation!

We encourage you and your family members to join the Alpha-1 Research Registry to help advance Alpha-1 research, diagnosis, and treatment.

Anyone diagnosed with Alpha-1 can join the Registry in three steps:

Complete a Registration Form 1.

Review and Sign the Informed Consent 2. Complete the Questionnaire 3

To enroll or for more information, please visit the Participate in Research counter in the A1F Foyer or visit alpha1 org/join-the-alpha-1-research-registry

15
Committed to listening to, learning from, and partnering with the Alpha-1 community wavelifesciences.com Reimagine possible. The John W. Walsh Home Infusion Bill has been reintroduced in the 118th Congress as H.R. 4438 and we need you! Please find link to download the toolkit with all the materials to get continue this campaign: alpha1.org/advocacy-toolkit To get involved and get your state on the map, e-mail Jeanne Kushner, Senior Director of Communications & Policy at jkushner@alpha1.org Become an Alpha-1 Advocate!

The Alpha-1 Foundation Clinical Trials Education Program

Are you interested in better understanding how a study drug comes to fruition? From our Clinical Trials Education Program, you can learn more about different phases and types of clinical trials, how inclusion/exclusion criteria can affect participation in a study, what is informed consent and why it is important, and the next steps to get involved!

To enroll or for more information, please visit the Participate in Research counter in the A1F Foyer or visit alpha1.org/clinical-trials-101

D M E M B E R S

augmentation therapy is given

The ElevAATe Study is actively looking for people, ages 18-80, with a confirmed diagnosis of AATD Emphysema to participate in a clinical trial investigating INBRX-101, a new recombinant form of Augmentation Therapy. Treatment with INBRX-101 may require less frequent dosing but still raise and maintain serum AAT levels. In a completed Phase 1 study, patients treated with 120 mg/kg of INBRX-101 every 3 weeks for up to 3 doses had measurements of AAT levels in the blood that were similar to AAT levels that are seen in people without AATD.1

To learn more about the ElevAATe trial, contact the Alpha-1 Foundation Research Registry at alpha1registry@alpha1.org or call 1-877-228-7321 , extension 252 or 245.

You can also scan the QR code and fill out the pre-screening inquiry form and an Alpha-1 Foundation representative will get back to you with more information on the trial.

Mark Brantly, M Florida

Noel G. McElvaney, MD, BCh, BAO, FRCPI, FRCPC

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©2024 Inhibrx, Inc. All Rights Reserved 3/24
Reference: 1. Kuhn BT, Veale A, Farah H, et al. Recombinant human AAT protein INBRX-101 demonstrates potential to achieve lung penetration and normal functional serum AAT levels in patients with AAT deficiency. Presented at: the ATS International Conference 2023; May 19-24, 2023; Washington, DC. INBRX-101 is an investigational treatment for which we have not yet obtained marketing authorization, and its safety and efficacy have not been definitively established. M I A M I , F L | J U N E 6 - 8 , 2 0 2 5 | # A 1 F N C 2 5 2 0 2 5 A L P H A - 1 F O U N D A T I O N N A T I O N A L C O N F E R E N C E J O I N U S I N C E L E B R A T I N G O U R 3 0 T H A N N I V E R S A R Y A T T H E

Platinum Conference Sponsors: AlphaNet, CSL Behring, Grifols, Takeda, and Vertex

Gold Sponsor: Accredo by Evernorth

Emerging Therapies Sponsors: Beam Therapeutics, BioMarin Pharmaceutical, Inhibrx, Korro Bio, Takeda, and Wave Life Sciences

Fred Walsh & Joe Reidy for organizing the Alpha Angels Memorial Service

The physicians, healthcare providers and others who give so many volunteer hours to the Alpha-1 community as speakers at this conference and throughout the year at our Education Programs.

The 2024 Alpha-1 Foundation National Conference Program Committee who contributed many hours to making this conference a success. Members include: Cathey Horsak Henderson & Alexis Artiles Ojeda, Co-Chairs of the A1F National Conference; Kim Caraballo, Erin Carr, Virginia Clark, MD, Jeanine D’Armiento, MD, PhD, Toya DeLeon, Nitika Gupta, MD, Jon Hagstrom, Peggy Iverson, Darrell Kotton, MD, Jan Ligman, Angela McBride, Laraine Potter, Robert Sandhaus, MD, PhD, FCCP, Scott Santarella, and Andrew Wilson, MD.

EXHIBITORS EXHIBITORS

The Alpha-1 Foundation would like to thank all of the exhibitors participating in this year’s conference. Please take the time to visit their booths in S

B t t r exhibitor passport stamped by all the exhib rday evening!

9:00 –10:00 am

10:0010:15 am

10:15 –11:15 am

Breakfast in Concerto

A1F Welcome & Day 1 Opening Remarks in Symphony 2-4

Scott Santarella, President & CEO, Alpha-1 Foundation

Jon Hagstrom, Chair of the Board of Directors, Alpha-1 Foundation

Alpha Empowerment Hour in Symphony 2-4

Erin Carr | Dan Grimm | Jessica Brown | Melissa Hale

Alpha-1 Research Registry & Patient Presentation in Symphony 2-4

11:15 –11:45 am

11:45 am –12:30 pm

Jeanine D'Armiento, MD, PhD, Medical Liaison, Alpha-1 Foundation

Alison Keaveny, Director of Clinical Research, Alpha-1 Foundation

Nadine Nuchovich, Clinical Research & Global Manager, Alpha-1 Foundation

Douglas Kipperling, Alpha-1 Patient

Platinum Sponsor Presentations in Symphony 2-4

AlphaNet | Charlie Strange, MD, Medical Director

CSL Behring | Tim Kanter, General Manager, CSL Vifor US & Shannen Silvestrini, Director, Marketing-Established Products Group

Grifols | Chris Healey, President at Grifols Shared Services North America

Takeda | Dan Spivak, Head of Alpha-1 Franchise

12:30 –1:30 pm

1:30 –2:30 pm

Exhibit Hall Grand Opening in Symphony 1

Enjoy MIAMI inspired sounds and bites!

Lunch in Concerto

Track 1 in Symphony 4 Track 2 in Symphony 2-3 2:30 pm

Alpha-1 Antitrypsin Deficiency 101: Lung

Robert Sandhaus, MD, PhD, FCCP, Clinical Director, Alpha-1 Foundation

Alpha-1 Antitrypsin Deficiency 101: Liver

ssia Health

o by Evernorth

1 Foundation lphaNet

ded Testing (ACT) Study

3:00 pm

Jeffrey Teckman, MD, Professor of Pediatrics and Biochemistry, Saint Louis University

The ChiLDReN Study Published New Findings

3:30 pm

Jeffrey Teckman, MD 4:00 pm

Q&A Session

Jeanine D’Armiento, MD, PhD, Medical Liaison

Robert Sandhaus, MD, PhD, FCCP

Jeffrey Teckman, MD

5:00 –5:30 pm

6:30 –8:30 pm

Alpha-1 Kids Room Meet & Greet

The Power is in You: Alpha-1 Programs, Services, & Getting Involved

Come enjoy an ice cream bar and learn how you can make a difference in the Alpha-1 community.

Meet Alpha-1 rockstars and volunteers who have been involved in the community and make a difference every day.

We invite Alpha-1 parents to join A1F staff and Dr. Jeffrey Teckman for a meet & greet.

MIAMI Dinner Reception & Entertainment

A MIAMI inspired evening with A1F! Enjoy Miami inspired food stations, drinks, and a personal appearance by some of Miami’s most famous.

Friday, June 7th
CVS He CSL Beh Global Liver Grifo Plasma Protein Association Take 19 18 2024 Alpha-1 Foundation National Conference � Miami, FL � June 7-9, 2024 The POWER is in YOU to make a difference

Saturday, June 8th

7:30 –

9:00 am

8:00 –8:45 am

9:00 –

9:15 am

9:15 –

9:30 am

Breakfast in Concerto

Alpha Angels Memorial Service in Symphony 2-4

Join the Alpha-1 community for the annual Memorial Service to remember all Alpha Angels.

Day 2 Opening Remarks in Symphony 2-4

Scott Santarella, President & CEO, Alpha-1 Foundation

The Alpha-1 Foundation’s Research Agenda in Symphony 2-4

Andrew Wilson, MD, Scientific Director, Alpha-1 Foundation

Alpha-1 Research Panel in Symphony 2-4

Moderated by: Monica Goldklang, MD, Assistant Professor of Medicine (in Anesthesiology), Columbia University, Center for LAM and Rare Lung Diseases

Michael Campos, MD, Professor of Clinical Medicine, University of Miami Miller School of Medicine

Jeanine D’Armiento, MD, PhD

9:30 –

10:30 am

Nitika Arora Gupta MD, DCH, DNB, MRCPCH(UK) FAASLD, Medical Director, Liver transplant and Hepatology Director-Liver Transplant Teen Transition Program, Professor of Pediatrics, Emory University School of Medicine, Children’s Healthcare of Atlanta

Jeffrey Teckman, MD

Mike Wells, MD, Critical Care Medicine, Pulmonology, University of Alabama at Birmingham Andrew Wilson, MD

Emerging Therapies in Alpha-1 in Symphony 2-4

Moderated by: Andrew Wilson, MD and Darrell Kotton, MD

10:30 - 10:35 | Introduction

10:35 - 10:50 | Beam Therapeutics

BEAM-302 Base editing as a potential therapeutic approach for alpha-1 antitrypsin deficiency (Alpha-1) | Nicolas Currier, MD, PhD, Senior Medical Director

11:20 - 11:35 | Korro Bio

KRRO-110, a RNA Editing Modality for the Treatment of Alpha-1 Antitrypsin Deficiency | Kemi Olugemo, MD, FAAN, Chief Medical Officer

11:35 - 11:50 | Takeda

10:30 am–12:30 pm

10:50 - 11:05 | BioMarin Pharmaceutical BioMarin's research program in Alpha-1 Antitrypsin Deficiency (AATD) | Suresh Agarwal, Director, Clinical Pharmacology

11:05 - 11:20 | Inhibrx

INBRX-101: ElevAATing Clinical Development in AATD | Erin Babcock, Director of Clinical Science

The Redwood Study: Explore a New Path for Alpha-1 Liver Disease | Sylvester Uwumarogie, MD, MSc, Associate Medical Director, Global Medical Affairs-GI New Programs

11:50 - 12:05 | Wave Life Sciences

RNA Editing for the Treatment of Alpha-1 Antitrypsin Deficiency | Amy Donner, PhD. Vice President, Head of Medical Communications

12:05 – 12:30 | Q&A Panel

Afternoon Breakout Sessions

Track 1 in Symphony 2

Pre & Post Transplant Process

Juan Salgado, MD

Associate Professor of Medicine, Transplant Pulmonologist, Director for the Lung Transplantation Fellowship, University of Miami Miller School of Medicine

Track 2 in Symphony 3

Track 3 in Symphony 4

Sunday, June 9th

2:40 –3:15 pm

Everyday Living as an Alpha

Monica Goldklang, MD

Assistant Professor of Medicine (in Anesthesiology), Columbia University, Center for LAM and Rare Lung Diseases

MZ Presentation

Mike Wells, MD

Critical Care Medicine, Pulmonology, University of Alabama at Birmingham

Navigating Life with Oxygen

Moderator: Skip Scribner

Patient Panel: Debbi Webb-Howells DC Young

Having a Young Child Diagnosed with Alpha-1

Jean P. Molleston, MD Professor of Clinical Pediatrics, Division Chief, Pediatric Gastroenterology, Hepatology & Nutrition, Indiana University School of Medicine

Having an Adolescent Diagnosed with Alpha-1

Nitika Arora Gupta MD, DCH, DNB, MRCPCH(UK) FAASLD Medical Director, Liver transplant and Hepatology Director-Liver Transplant Teen Transition Program, Professor of Pediatrics, Emory University School of Medicine, Children’s Healthcare of Atlanta 3:15 –

Sleep Apnea

Kori Ascher, MD

Assistant Professor of Clinical Medicine, Division of Pulmonary, Critical Care, and Sleep Medicine, University of Miami Health System

Valve Options

Moderator: D. Kyle Hogarth, MD, FCCP

Patient Panel: Craig Hamilton Lisa Kosak Siobhan Lestina

Transition of Care: Adolescent to Adult Alpha

Nitika Arora Gupta, MD, DCH, DNB, MRCPH(UK), FAASLD 4:15 –4:50 pm

How to Advocate for Yourself as an Alpha-1 Patient

Michael Campos, MD

Professor of Clinical Medicine, University of Miami Miller School of Medicine

Public Policy & Advocacy

Moderator: Jeanne Kushner

Patient Panel: Daniel Grimm

Jennifer Jopp Lisa Kosak

Alpha-1 Kid Success Stories

Moderator: Jeffrey Teckman, MD Patient Panel: Bianca Castillo

Chase Castillo

Catriona Garry

12:30 –2:00 pm Lunch in Concerto & Exhibits in Symphony 1 2:00 –4:50 pm Afternoon Breakout Sessions 7:00 –9:00 pm
& Entertainment in Symphony 2-4
downtown Miami fusion inspired food stations, GLOW inspired entertainment, and drinks. Fun giveaways throughout the evening and the exhibitor passport drawing.
GLOW PARTY Dinner Reception
Enjoy
8:00 –9:30 am Bring You Back 2025 Beach Bash Brunch in Soundgarden
farewell breakfast celebration you won’t want to miss! Bring your conference tote to brunch so you can fill your beach bag for 2025 with fun items. Take a video at the 360-photo booth and don't miss your opportunity to win a trip to Disney!
A
2:00 –2:35 pm
Break
3:35 pm
3:35 –4:10 pm
21 20 2024 Alpha-1 Foundation National Conference � Miami, FL � June 7-9, 2024 The POWER is in YOU to make a difference

SPEAKER BIOS SPEAKER BIOS

Kori Ascher, DO is an Assistant Professor in the Department of Pulmonary, Critical Care, & Sleep Medicine at the University of Miami. She earned her Doctor of Osteopathic Medicine from Nova Southeastern University and holds certifications in Sleep Medicine, Critical Care Medicine, Pulmonary Medicine, and Internal Medicine. Dr. Ascher is actively involved in academic and clinical roles, serving as Assistant Program Director of the Sleep Medicine Fellowship Program. Dr. Ascher is dedicated to improving sleep health and raising awareness of pulmonary and sleep diseases in past endeavors through her organization, Just Breathe Miami, and her contributions to various professional and community initiatives.

Jessica Brown is a dedicated advocate from Tampa, Florida, and the mother to a very active seven-year-old boy, Thomas, who was diagnosed as a ZZ alpha when he was a baby. She holds a Bachelor of Science from the University of Tampa in marketing with an emphasis in public health. Before becoming a mom, Jessica worked in the healthcare field for many years, where she discovered her passion for helping others while working in hospice care. Jessica has been actively involved with the Alpha-1 Foundation and its fundraising efforts for several years. In 2023, she helped raise $10,000 through the “Riding for a Reason” campaign and has contributed to raising over $20,000 since 2020. Her goal is to help increase awareness and find a cure for Alpha-1, ensuring that no other parent must hear that their child is diagnosed with this rare condition. Jessica is passionately working towards making a difference in the lives of those affected by Alpha-1.

Michael Campos, MD received his bachelor’s degree in biology and medical degree from the Universidad Peruana Cayetano Heredia, Lima, Peru in 1994. He completed his Residency (1998) and Chief Residency (1999) in Internal Medicine, followed by fellowships in Critical Care Medicine (1999-2000) and Pulmonary Medicine (2000-2002) at the University of Miami/Jackson Memorial Medical Center in Miami, Florida. In 2003, he joined the faculty at the University of Miami Miller School of Medicine where he currently holds the rank of Professor in clinical medicine. Dr. Campos has worked as Chief of the Pulmonary Section at the Miami Veterans Administration (VA) Medical Center and is an active investigator in many research programs related to Chronic Obstructive Pulmonary Disease (COPD) and Alpha-1 Antitrypsin Deficiency (Alpha-1). He is widely published with over 100 articles in peer-reviewed journals and continues with his interest in patient education.

is a mother and advocate who understands the daily struggles individuals and families impacted by Alpha-1 face. Born out of personal experience and unwavering love for her children Jack and Tess, her journey took a profound turn when Tess was diagnosed with Alpha-1 as an infant. This pivotal moment ignited her passion for advocacy and drove her to become deeply involved in the Alpha-1 community. At the core of Erin's advocacy is a simple yet powerful belief in the importance of compassion, resilience, and determination. She firmly believes that by coming together as a community, we can overcome any obstacle and create a brighter future for those affected by Alpha-1. Whether organizing support group meetings, participating in awareness campaigns, or providing guidance and support to newly diagnosed families, she remains steadfast in her commitment to making a difference.

22 2024 Alpha-1 Foundation National Conference � Miami, FL � June 7-9, 2024

SPEAKER BIOS SPEAKER BIOS

Jeanine D’Armiento, MD, PhD is a Professor of Medicine in Anesthesiology at Columbia University Irving Medical Center. Dr. D’Armiento is the Director of the Center for Molecular Pulmonary Disease in Anesthesiology and Physiology and Cellular Biophysics, and Director of the Center for Lymphangiomyomatosis (LAM) and Rare Lung Disease. Her research focuses on understanding the mechanisms of lung injury and repair. Her laboratory integrates both in vitro and in vivo approaches and is uniquely situated to characterize the molecular changes in the study of lung injury and disease to identify potential therapeutic targets. Dr. D’Armiento’s clinical work focuses on Rare Diseases, and her Center for LAM and Rare Lung Diseases at Columbia University serves one of the largest populations of women with LAM in addition to patients with Alpha-1. Dr. D’Armiento is part of the A1F Clinical Resource Center (CRC) network and provides care to patients with Alpha-1. She serves as Chair of the Executive Committee of the Columbia University Senate and as a Consultant to the Director of the Division of Rare Disease Research Innovations (DRDRI), NCATs.

Monica Goldklang, MD is a pulmonary and critical care physician scientist at Columbia University Irving Medical Center. She grew up in Michigan, completing undergraduate, medical school, and residency training at the University of Michigan. She then moved to Columbia University for a pulmonary and critical care fellowship and postdoctoral research training in the laboratory of Dr. Jeanine D’Armiento. In addition to caring for patients with rare lung diseases including Alpha-1 Antitrypsin Deficiency (Alpha-1) and Lymphangioleiomyomatosis, and working in the ICU, Dr. Goldklang has an active research portfolio. She has several clinical and translational studies in Alpha-1, including an investigation into novel imaging biomarkers of lung disease as well as studies regarding long-term disease trajectory and serum biomarkers following acute exacerbations of Chronic Obstructive Pulmonary Disease (COPD).

lives in Clarence, NY with his wife Debbie and their two children, Noelle and Eli. He was diagnosed as a ZZ Alpha in 2016, and has been on augmentation therapy since. After retiring in 2018 from a 41-year career as a truck driver, Dan became an AlphaNet coordinator. He took the job to learn more about Alpha-1 and soon became involved with the Alpha-1 Foundation. He enrolled in a clinical trial at Columbia University with Drs. D’Armiento and Goldklang, which reinforced his belief in the impact one person can make. Though Dan retired from AlphaNet in February this year, he continues to work closely with the Alpha-1 Foundation in a volunteer capacity.

Nitika Gupta, MD , DCH, DNB, MRCPCH(UK) FAASLD is a board-certified pediatric gastroenterologist and transplant hepatologist in the Department of Pediatrics, Emory University School of Medicine and Children’s Healthcare of Atlanta. After completing pediatric residency in India and transplant training in Birmingham Children’s Hospital, she went on to do pediatric residency, pediatric gastroenterology & hepatology, and transplant hepatology fellowships at Emory University, Atlanta. Over the past 20 years, the focus of her work has been in liver diseases of children and liver transplantation. She has conducted bench research in animal models and now focuses on clinical research and quality of life improvement. She has a special interest in transition from pediatric to adult healthcare and was the founding director of the Teen Transition program for liver transplant recipients. Her research has highlighted racial disparities and increased mortality after transfer from pediatric to adult healthcare which has led to the development of

24
Since 1995, AlphaNet has supported the Alpha-1 Foundation with contributions totaling more than $70 million. AlphaNet is proud to be a sponsor of the 2024 Alpha-1 National Conference. www.alphanet.org 25 The POWER is in YOU to make a difference

SPEAKER BIOS SPEAKER BIOS

new interventions and programs to mitigate these risks resulting in improved outcomes. She lives in Atlanta with her husband and 3 children.

Jon Hagstrom is the Chair of the Board of Directors of the Alpha-1 Foundation. Jon spent over 20 years working in investment banking and management consulting. He was diagnosed as a ZZ Alpha in 2008, after noticing increasing shortness of breath. The course of his disease was quite aggressive, cutting his career short in his early 40s. He received a bilateral lung transplant at Duke University Medical Center in 2016. Jon serves in a variety of roles for A1F, spanning all aspects of the mission. He has a keen interest in research and therapeutic development, serving as a patient representative on the Grant Advisory Committee (GAC), the Therapeutic Development Network (TDN) Steering Committee, the Alpha-1 Research Registry Steering Committee, and the Alpha-1 Biomarker Consortium (A1BC). He is passionate about patient support, serving as the Support Group Leader for NY/NJ, an Alpha-1 Global Advisory Committee member, and a Peer Guide for Alphas facing lung transplants. He is a fierce advocate for fellow Alphas, actively lobbying Congress for better care, championing patient interests to the FDA, and promoting early detection. He has been a member of the Investment and Development Committees since 2019, elected Board Treasurer and Executive Committee member in 2021, and Board Chair in 2022. Jon was recently appointed as the Alpha-1 patient on the ATS Public Advisory Roundtable (PAR). Jon is an avid classical pianist and resides in Dobbs Ferry, New York, with his teenage son.

Melissa Hale Melissa Hale is a ZZ Alpha living just outside of Philadelphia. Melissa was diagnosed with Alpha-1 in 2000 after her pulmonologist could not treat what he thought at the time to be asthma symptoms. Being diagnosed relatively young for an Alpha-1 patient, she was honored and excited to participate in Alpha-1 clinical studies and trials that could ultimately help medical professionals studying Alpha-1, and most of all other Alpha-1 patients.

D. Kyle Hogarth, MD, FCCP received his medical degree from Case Western Reserve University in Cleveland and completed his residency in internal medicine and a fellowship in pulmonary and critical care medicine at the University of Chicago. Dr. Hogarth directs the Alpha-1 Clinical Resource Center at the University of Chicago and is the director of bronchoscopy. Dr. Hogarth’s research focuses on improving quality of life and outcomes for patients with Alpha-1, improving Alpha-1 diagnosis and improving the early detection and management of lung cancer. In 2007, 2011, 2013 and 2014, he received the Department of Medicine’s Outstanding Faculty Clinical Service Award. In addition to his clinical and research work, he is on the editorial board for the journals of CHEST and Proceedings of the American Thoracic Society.

27
26 2024 Alpha-1 Foundation National Conference � Miami, FL � June 7-9, 2024

ZEMAIRA® Alpha1-Proteinase Inhibitor (Human) lyophilized powder for reconstitution for intravenous use

Initial U.S. Approval: 2003

BRIEF SUMMARY OF PRESCRIBING INFORMATION

These highlights do not include all the information needed to use ZEMAIRA safely and effectively. See full prescribing information for ZEMAIRA.

-----------------------------------INDICATIONS AND USAGE----------------------------------

• ZEMAIRA is an alpha1-proteinase inhibitor (A -PI) indicated for chronic augmentation and maintenance therapy in adults with A -PI deficiency and clinical evidence of emphysema (1).

• The effect of augmentation therapy with ZEMAIRA or any A -PI product on pulmonary exacerbations and on the progression of emphysema in A1-PI deficiency has not been demonstrated in randomized, controlled clinical studies (1).

• ZEMAIRA is not indicated as therapy for lung disease patients in whom severe A1-PI deficiency has not been established (1).

-------------------------------DOSAGE AND ADMINISTRATION------------------------------

For intravenous use after reconstitution only (2).

• The recommended weekly dose of ZEMAIRA is 60 mg/kg body weight. Dose ranging studies using efficacy endpoints have not been performed with ZEMAIRA or any A -PI product (2).

• Administer through a suitable 5 micron infusion filter (not supplied) at room temperature within 3 hours after reconstitution (2.2).

• Do not mix with other medicinal products. Administer through a separate dedicated infusion line (2.2).

• Administer at a rate of approximately 0.08 mL/kg/min as determined by the response and comfort of the patient (2.2).

• Monitor closely the infusion rate and the patient’s clinical state, including vital signs, throughout the infusion. Slow or stop the infusion if adverse reactions occur. If symptoms subside promptly, the infusion may be resumed at a lower rate that is comfortable for the patient (2.2).

--------------------------------DOSAGE FORMS AND STRENGTHS---------------------------

ZEMAIRA is supplied in a single-dose vial containing approximately 1000 mg, 4000 mg, or 5000 mg of functionally active A1-PI as a white to off-white lyophilized powder for reconstitution with 20 mL, 76 mL, or 95 mL of Sterile Water for Injection, USP. The amount of functional A1-PI is printed on the vial label and carton (3).

---------------------------------------CONTRAINDICATIONS -----------------------------------

• History of anaphylaxis or severe systemic reactions to ZEMAIRA or A1-PI protein (4).

• Immunoglobulin A (IgA)-deficient patients with antibodies against IgA, due to the risk of severe hypersensitivity (4).

-------------------------------WARNINGS AND PRECAUTIONS-------------------------------

• Observe any signs of hypersensitivity such as tachycardia, hypotension, confusion, syncope, oxygen consumption decrease, and pharyngeal edema when administering ZEMAIRA to patients with known hypersensitivity to an A1-PI product (5.1).

• Patients with selective or severe IgA deficiency can develop antibodies to IgA and, therefore, have a greater risk of developing potentially severe hypersensitivity and anaphylactic reactions. If anaphylactic or severe anaphylactoid reactions occur, discontinue the infusion immediately (5.2).

• Because ZEMAIRA is made from human blood, it may carry a risk of transmitting infectious agents, e.g., viruses, the variant Creutzfeldt-Jakob disease (vCJD) agent and, theoretically, the Creutzfeldt-Jakob disease (CJD) agent (5.3).

-------------------------------------ADVERSE REACTIONS--------------------------------------

• Serious adverse reactions reported following administration of ZEMAIRA in prelicensure clinical trials included one event each in separate subjects of bronchitis and dyspnea, and one event each in a single subject of chest pain, cerebral ischemia and convulsion.

• The most common adverse reactions occurring in at least 5% of subjects receiving ZEMAIRA in all pre-licensure clinical trials were headache, sinusitis, upper respiratory infection, bronchitis, asthenia, cough increased, fever, injection site hemorrhage, rhinitis, sore throat, and vasodilation (6).

To report SUSPECTED ADVERSE REACTIONS, contact CSL Behring Pharmacovigilance at 1-866-915-6958 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

SPEAKER BIOS SPEAKER BIOS

Darrell N. Kotton, MD is the founding Director of the Boston Medical Center/Boston University Center for Regenerative Medicine (CReM) and oversees program development while coordinating the efforts of seven physically-contiguous laboratories in their efforts to understand basic reprogramming mechanisms as well as approaches to the directed differentiation of pluripotent stem cells, following lessons from developmental biology to derive a variety of cell lineages for basic studies and disease models. Dr. Darrell Kotton’s laboratory specializes in stem cell biology with a focus on applying stem cells to model and understand lung development and disease. He has trained graduate students and post-doctoral fellows who have gone on to become faculty members in academic medicine, laboratory PIs, and industry scientists. In his role as an educator, he serves as the PI of the Boston University CTSI’s TL1 training grant in regenerative medicine. Dr. Kotton’s laboratory research has focused primarily on applying mouse and human pluripotent stem cells to investigate the developmental biology of endoderm and the biology of lung injury and repair. His group has developed reprogramming methods, endodermal differentiation protocols for pluripotent stem cells, and gene transfer methods to modulate stem cells or various resident lung cells for durable in vitro and in vivo gene expression. Most recently his laboratory has developed new approaches for generating lung and thyroid lineages from mouse or human pluripotent stem cells in vitro.

Based on September 2022 revision

Jean P. Molleston, MD joined the Pediatric Gastroenterology, Hepatology and Nutrition division at Riley Hospital for Children in 1999 and has served as Division Chief since 2005. She is a pediatric hepatologist and transplant physician; her clinical and research interests include fatty liver (NASH), cholestatic liver disease, metabolic liver disease, CFrelated liver disease and viral hepatitis in addition to general gastroenterology. She has a long interest in medical education and loves to teach. She served in medical school course development and implementation, residency education, and faculty development and mentors’ junior faculty in clinical research.

Juan Salgado, MD is an Associate Professor of Clinical Medicine at the University of Miami Miller School of Medicine where he is also the Program Director of the lung transplantation advanced fellowship tracks. He is a transplant pulmonologist and intensivist at the Miami Transplant Institute, where he leads the ECMO & critical care operations for the Lung Center.

Robert A. Sandhaus, MD, PhD, FCCP is a Professor of Medicine in the Division of Pulmonary, Critical Care, and Sleep Medicine at the University of Colorado School of Medicine and is based at National Jewish Health in Denver, where he founded the Alpha-1 Antitrypsin Deficiency (Alpha-1) Program over 40 years ago. His early research focused on the role of white blood cell proteases in preventing and promoting lung disease. This work led to research and clinical interest in patients with Alpha-1. More recently, he has evaluated the lung dysfunction of patients with Osteogenesis Imperfecta. Dr. Sandhaus has served on the Boards of Directors of the Alpha-1 Association, the Alpha-1 Foundation, AlphaNet, the Alpha-1 Project (TAP), the Association for the Accreditation of Human Research Protection Programs, Global Implementation Solutions, and the Osteogenesis Imperfecta Foundation. For more than 20 years, he has been the Executive Vice President and Senior Medical Director of AlphaNet and the Clinical Director of the Alpha-1 Foundation. More recently, he is also serving as the Medical Director of AlphaNet Canada.

29 The POWER is in YOU to make a difference

1-833-PHONEA1 (1-833-756-6321)

Visit our Booth

• Obtain educational resources

• Learn about PROLASTIN®-C LIQUID and the PROLASTIN DIRECT® program

• Connect and ask questions

I know that PROLASTIN-C LIQUID and the PROLASTIN DIRECT program will be there for me, supporting me every step of the way.

SPEAKER BIOS SPEAKER BIOS

Scott Santarella is the President and Chief Executive Officer of the Alpha-1 Foundation. Scott leads the organization in developing strategies for advancing detection and discovery through clinical and translational research initiatives and investment to achieve the mission. Prior to this position, he served five years as CEO at the Global Lyme Alliance (GLA) where he led the Board from a merged entity of two all-volunteer local charities to a fully operational, professionally staffed, global nonprofit dedicated to Lyme and tick-borne research and education. Scott has over 32 years of experience as a senior executive with a proven track record in strategic planning, creative messaging and marketing, industry partnership development, clinical research innovation, and raising public awareness for neglected, underfunded, and often stigmatized diseases. Prior to joining GLA, he was President and CEO of the Bonnie J. Addario Lung Cancer Foundation (ALCF) in San Francisco (NKA GO2 Foundation for Lung Cancer), where he successfully grew the organization from a regional lung cancer research and patient services organization to a globally recognized leader in the lung cancer community as well as the broader oncology research and patientsurvivor marketplace. Prior to ALCF he was President and CEO of the American Lung Association of New York (ALANY) and spent 10 years as the Executive Director and then Chief Operating Officer of the Multiple Myeloma Research Foundation (MMRF). He received an Executive Leadership Certification from Harvard Business School and holds a Bachelor of Arts in Journalism from the University of Massachusetts, Amherst.

Skip Scribner is an EMT-Paramedic who educates the airline industry on respiratory assist devices, addressing allowed devices, common problems, and misconceptions during travel. He has spoken at the DOT Disability Forum in Cairo (2009) and Miami (2010). Skip is a retired Fire/Paramedic Lieutenant with 29 years of service in the City of Norfolk, VA Fire Rescue. He began his EMS career in a volunteer rescue squad as a neonatal transport paramedic and an original Flight Paramedic on the Nightingale trauma helicopter in Norfolk, VA. A private pilot since 1976, Skip founded Advanced Aeromedical in 1981. Initially focused on long-haul critical care patient transport, his company now helps oxygen-dependent people travel globally, providing logistical support for equipment, oxygen, and travel needs. Whether it's urgently delivering a POC across the country for a morning flight or coordinating oxygen for a cruise passenger, Skip and his team handle it efficiently and effectively.

You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch or call 1-800-FDA-1088.

Jeffrey H. Teckman, MD is the Interim Chairman of the Department of Pediatrics and Director of Pediatric Gastroenterology and Hepatology at the Saint Louis University School of Medicine, where he holds the Drs. James and Patricia Monteleone Endowed Chair. He has been involved in research on liver disease for more than 25 years in both basic science and clinical studies. Although his studies have ranged from viral hepatitis to liver failure in short bowel syndrome, his primary basic science focus has been the liver disease associated with Alpha-1 Antitrypsin Deficiency (Alpha-1). His laboratory was the first to recognize the link between Alpha-1 mutant Z protein in the liver and autophagy, and he published the first unified explanation of the mechanism of liver injury in Alpha-1. Dr. Teckman is the Principal Investigator of the only prospective, multicenter, liver biopsy-based study of adult Alpha-1 liver disease and is the lead author of the largest report of Alpha-1 pediatric liver disease. Dr. Teckman has also served the patient community as an advisor, educator, and advocate.

© 2024 Grifols All rights reserved April 2024 US-PRL-2400066
–Steve
Actual patient since 2007 “ Patient Testimonial
M.,
31 The POWER is in YOU to make a difference

SPEAKER BIOS SPEAKER BIOS

J. Michael Wells, MD is an Associate Professor of Medicine in the Division of Pulmonary, Allergy, and Critical Care Medicine at the University of Alabama at Birmingham (UAB). He joined the faculty in 2012 after completing a pulmonary fellowship at UAB. His research focuses on mechanisms and implications of extracellular matrix remodeling, inflammation, and pulmonary vascular disease in Chronic Obstructive Pulmonary Disease (COPD) and Alpha-1 Antitrypsin Deficiency (Alpha-1). He oversees a basic science laboratory, is the Medical Director for the UAB Alpha-1 Clinical Resource Center (CRC), and is the Assistant Medical Director for the UAB Lung Health Center. He directs animal and cellular experiments, supervises the biospecimen core for an NIH/NCATSfunded clinical trial in Alpha-1, and is an active SPIROMICS and COPDGene investigator. Dr. Wells has received the Young Physician Scientist Award from the ASCI and the Sreedhar Nair Early-Stage Investigator Award in COPD from the American Thoracic Society. Dr. Wells’ research has been continuously supported through NIH and foundation grants, university awards, and industry-sponsored clinical trials.

FIND INFUSION OPTIONS THAT FIT YOUR LIFE

GLASSIA® can be infused in the clinic, at an infusion center, at home by a healthcare provider, or by self-administration— after appropriate training.*

You have infusion options. Talk to your doctor to explore them. Visit glassialiquid.com/connect to sign up for more helpful information.

Andrew Wilson, MD is the Scientific Director of the Alpha-1 Foundation and a Professor of Medicine at the Boston University Chobanian & Avedisian School of Medicine. He is a Pulmonary and Critical Care physician-scientist with a focus on regenerative medicine and stem cell biology. The goal of his research is to advance the understanding of and treatment for genetic causes of Chronic Obstructive Pulmonary Disease (COPD) and the lung and liver diseases associated with Alpha-1 Antitrypsin Deficiency (Alpha-1), largely through the platform of patient-derived stem cells. In association with his research efforts, he has compiled the world’s largest repository of Alpha-1 patient specific induced pluripotent stem cells (iPSCs). He has shared these cells widely with researchers around the world. In addition to laboratory-based science, he also participates in epidemiological studies to define the incidence of and risk factors for both liver and lung disease associated with Alpha-1. He is the founding director of the Alpha-1 Center at Boston University and Boston Medical Center, one of the largest clinical resource centers for patients with Alpha-1 in the Northeast, where he cares for patients with Alpha-1.

If self-administration is deemed appropriate, ensure that you receive detailed instructions and adequate training on how to administer at home or other appropriate setting and have demonstrated the ability to independently administer GLASSIA.

“Having the option to infuse at home on my own* gives me the flexibility to keep doing the things I love, like spending time with my grandchildren.”

—TRUDY D. Actual GLASSIA patient for 2 years

What is GLASSIA?

GLASSIA is a medicine containing human Alpha₁-Proteinase

Inhibitor (Alpha₁-PI) that is used to treat adults with lung disease (emphysema) because of severe Alpha₁-antitrypsin (Alpha₁) deficiency. GLASSIA is not meant to be used as a therapy for lung disease other than severe Alpha₁ deficiency. Effects of GLASSIA on worsening lung function and emphysema progression have not been proven in clinical trials. Long-term effects of Alpha₁ replacement and maintenance therapy have not been studied.

IMPORTANT SAFETY INFORMATION

What is the most important information I need to know about GLASSIA?

• GLASSIA can cause severe allergic reactions including hives, swelling in the mouth or throat, itching, tightness in the chest, trouble breathing, wheezing, faintness or low blood pressure

• If you will be taking GLASSIA outside a healthcare setting, ask your healthcare provider (HCP) about an epinephrine pen and/or other supportive care for certain severe allergic reactions.

Who should not use GLASSIA?

Do not use GLASSIA if you:

• Have immunoglobulin A (IgA) deficiency with antibodies to IgA

• Have a severe allergic reaction to human Alpha₁-PI products.

IMPORTANT SAFETY INFORMATION, CONTINUED

What are the possible or reasonably likely side effects of GLASSIA?

If any of the following problems occur contact your healthcare provider (HCP) or call emergency services right away:

• Worsening or flare-up of your chronic obstructive pulmonary disease (COPD)

• Hives, swelling in the mouth or throat, itching, chest tightness, trouble breathing, wheezing, fainting or dizziness. These could be signs of a serious allergic reaction.

The most common side effects that may occur are headache and upper respiratory tract infections.

Other possible side effects of GLASSIA include:

• Cough

• Sinus infection

• Chest discomfort

• Dizziness

• Increased liver enzymes

• Shortness of breath

• Nausea

• Fatigue

These are not all the possible side effects. Tell your HCP about any side effect that bothers you or that does not go away. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please see the Important Facts About GLASSIA on the next page.

©2021 Takeda Pharmaceuticals U.S.A., Inc. 300 Shire Way, Lexington, MA 02421. 1-877-TAKEDA-7 (1-877-825-3327). All rights reserved. TAKEDA and the TAKEDA logo are trademarks or registered trademarks of Takeda Pharmaceutical Company Limited. GLASSIA is a registered trademark of Kamada Ltd., and used under license. US-GLA-0293v1.0 10/21
32 2024 Alpha-1 Foundation National Conference � Miami, FL � June 7-9, 2024

IMPORTANT FACTS ABOUT GLASSIA (glasssee-ă) [Alpha1-Proteinase Inhibitor (Human)]

Injection Solution – For Intravenous Administration What is GLASSIA?

GLASSIA is a liquid medicine conta ning human Alpha1-Proteinase Inhibitor (A pha1-PI) a so known as alpha1-antitrypsin AAT), wh ch is purified from human blood. The ma n purpose of infusing GLASSIA is to increase the evels of the AAT prote n in your b ood and lungs AAT protein protects the lung tissue by blocking certa n enzyme-caused damage Such damage can ead to severe lung d sease, such as emphysema

Limitations of Use:

• The effects of increasing the AAT protein levels w th GLASSIA or any other A pha1-PI product on worsening pu monary function and progression of emphysema have not been proven n clin cal tria s.

• The ong-term effects of AAT rep acement and maintenance therapy w th GLASSIA have not been stud ed.

• GLASSIA is not ntended as a therapy n individuals w th lung d sease other than severe A pha1-PI def ciency

Who should not use GLASSIA?

Do not use GLASSIA f you:

• Have mmunog obulin A (IgA) defic ency w th antibodies to IgA

• Have had a severe a lerg c react on to human Alpha1-PI products

What is the most important information that I should know about GLASSIA?

Severe allergic react ons can occur w th GLASSIA Your doctor w ll nform you about signs of a lerg c reactions which include hives, swe ling in the mouth or throat, itching, t ghtness in the chest, trouble breathing, wheezing, faintness, low b ood pressure, or ser ous allerg c react on. If you have any of these reactions, discontinue use of the product and contact your phys cian and/or seek immed ate emergency care, depending on the severity of the reaction

If you or your caregiver wi l be administering GLASS A outside a healthcare sett ng, ask your doctor about an ep nephr ne pen and/or other supportive care for certain severe a lergic react ons Ask your doctor to make sure you receive training on how and when to use any prescribed support ve care med cine and keep it close at hand when adm nistering GLASSIA

How should I take GLASSIA?

• GLASSIA is g ven direct y into the bloodstream.

• You can get GLASSIA at your hea thcare professional’s off ce, c inic, hospital, or del vered directly to your home by a healthcare professional from a l m ted network of specia ty pharmacy prov ders

• Your healthcare professional wil dec de f self-infusion in your home is right for you. You should be trained on how to do infus ons by your healthcare profess onal

What should I tell my healthcare professional before I start using GLASSIA?

Before starting GLASSIA, tell your healthcare profess ona if you:

• Have IgA def ciency with ant bodies to IgA

• Have a h story of severe allergic react ons to Alpha1-PI products

What are the possible or reasonably likely side effects of GLASSIA?

• A poss b e side effect to GLASSIA s worsening or flare-up of your chronic obstruct ve pu monary d sease COPD) in which your breathing gets worse than usua

• Ca l your hea thcare professional or go to your emergency department r ght away f you get: Hives, swell ng n the mouth or throat, itching, chest tightness, trouble breathing, wheez ng, fa nt ng or dizz ness These cou d be signs of a serious al erg c react on

• The most common side effects are headache and upper respiratory tract infect ons Other possible side effects of GLASSIA inc ude: cough, s nus infection, chest discomfort, d zz ness, ncreased iver enzymes, shortness of breath, nausea, and fatigue.

These are not a l of the possible side effects for GLASSIA You can ask your healthcare professional for nformat on that is provided to healthcare profess ona s. Talk to your healthcare profess ona about any side effects that bother you or that don’t go away.

How do I store GLASSIA?

Store GLASS A refrigerated or at room temperature

• You can store GLASSIA in the refrigerator (36°F to 46°F [2°C to 8°C]) Do not freeze

• You can store GLASS A at room temperature (up to 77°F [25°C] for up to one month. You must use GLASSIA within one month once you remove it from the refrigerator. Do not re-refrigerate GLASS A once the product has been stored at room temperature

• Keep the GLASSIA vial in the box unt l you are ready to admin ster the product

Check the expirat on date on the carton and via label. Do not use GLASSIA after the expiration date.

How do I get more information about GLASSIA?

The risk information provided here is not comprehensive. To learn more, talk about GLASSIA with your healthcare provider. The FDA-approved Full Prescribing Information ncluding Information for Patients and Instructions for Use can be found at www.glassia.com or call 1-877-TAKEDA-7 (1-877-825-3327).

Manufactured by: Distr buted by Kamada Ltd Baxalta US Inc

Beit Kama Lex ngton, MA MP Negev 8532500 02421

Israe USA

U.S. L cense No. 1826

US-GLA-0282v1 0 Rev 7/21

POWER POWER I S I N YOU YOU T H E T O M A K E A D I F F E R E N C E
35 The POWER is in YOU to make a difference
A L P H A - 1 F O U N D A T I O N N A T I O N A L C O N F E R E N C E M I A M I , F L | J U N E 7 - 9 , 2 0 2 4 POWER POWER I S I N YOU YOU T H E T O M A K E A D I F F E R E N C E 37 36 2024 Alpha-1 Foundation National Conference � Miami, FL � June 7-9, 2024 The POWER is in YOU to make a difference
S I N
T H E T O M A K E A D I F F E R E N C E A L P H A - 1 F O U N D A T I O N N A T I O N A L C O N F E R E N C E M I A M I , F L | J U N E 7 - 9 , 2 0 2 4 39 38 2024 Alpha-1 Foundation National Conference � Miami, FL � June 7-9, 2024 The POWER is in YOU to make a difference
POWER POWER I
YOU YOU

The Alpha-1 Foundation is committed to finding a cure for Alpha-1 Antitrypsin Deficiency (Alpha-1) and to improving the lives of people affected by Alpha-1 worldwide.

The Foundation has invested over $100 million to support Alpha-1 research and programs in 130 institutions in North America, South America, Europe, the Middle East and Australia.

Alpha-1 Foundation

3300 Ponce de Leon Blvd, Coral Gables, FL 33134 ALPHA1.ORG

The Alpha-1 Foundation thanks the generous sponsors who have contributed to support this conference.

PLATINUM SPONSORS

Gold Sponsor

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