THE VESSEL June 2026 (Final Draft) (003)

THE VESSEL THE VESSEL The Official Newsletter for The Bleeding Disorders Association of Northeastern New York

BDA COMMUNITY EVENTS RE-ENGAGING RE-CONNECTING

In This Issue Staff & Volunteer Needs

Cornhole Registration

Job Skills Training Grant

Event Flyers

Industry and Policy Updates

Scholarship Recipient

Keep an eye on our website for our: Cornhole Tournament at Fort Orange Brewing – July 12th Annual Meeting at Apex Entertainment – Nov. 15th Fall Community Wellness Day formerly UNITE Walk at Thacher Park - Coming this Fall Family Education Weekend at Double H Ranch – Oct. 2nd to 4th

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June 2026

THE VESSEL THE VESSEL The Official Newsletter for The Bleeding Disorders Association of Northeastern New York

The first half of 2026 gave our community a bit of everything, from education about new treatments on the market and advocacy for better health outcomes, to delicious local food. It was a steady flow of opportunities to connect and raise some funds for programs that mean so much to us.

January kicked January kicked us us off off with with aa Takeda Takeda dinner dinner atat Jacob Jacob && Anthony’s Anthony’s inin Saratoga Saratoga Springs, Springs, where where members members learned about learned about VONVENDI, VONVENDI,, aa first-in-class VONVENDI first-in-class recombinant recombinant extended extended half-life half-life therapy therapy for for vWD vWD that that can can often often treat bleeding treat bleeding episodes episodes in in aa single single dose. dose.

In March, In March, members members from from across across New New York York traveled traveled toto Capitol Capitol Hill Hill inin Washington, Washington, D.C. D.C. for for Washington Washington Days and Days and then then back back home home for for Albany Albany Days Days atat the the State State Capitol.

Capitol. Advocacy Advocacy atat both both state state and and federal federal levels levels focused on focused on protecting protecting and and strengthening strengthening access access to to care care and and support support for for individuals individuals and and families families living living with with bleeding disorders. bleeding disorders.

By April, By April, El El Niño Niño had had helped helped melt melt the the last last of of the the snow snow away, away, and and we we gathered gathered atat the the Scarlet Scarlet Knife Knife inin Latham Latham for aa Sanofi for Sanofi educational educational dinner. dinner. Their Their hematology hematology liaison liaison introduced introduced Qfitlia, Qfitlia,, an Qfitlia an antithrombin-lowering antithrombin-lowering siRNA rebalancing siRNA rebalancing therapy therapy for for prophylaxis prophylaxis in in Hemophilia Hemophilia A A and and B, B, with with or or without without inhibitors. inhibitors. The The presentation paired presentation paired effortlessly effortlessly with with aa standout standout spring spring menu menu that that almost almost made made you you forget forget it it was was still still below below freezing outside. freezing outside.

May awakened May awakened our our competitive competitive side side atat The The Bunker Bunker inin Clifton Clifton Park, Park, where where members members of of all all ages ages took took on on indoor golf indoor golf and and aa spirited spirited mini-golf mini-golf showdown showdown fueled fueled by by fantastic fantastic finger finger food. food. Between Between swings, swings, we we learned learned about Novo about Novo Nordisk’s Nordisk’s rebalancing rebalancing therapy, therapy, Alhemo Alhemo,, aa Tissue Alhemo, Tissue Factor Factor Pathway Pathway Inhibitor Inhibitor antagonist antagonist for for Hemophilia A Hemophilia A and and B, B, with with or or without without inhibitors. inhibitors. Later Later that that month, month, our our Pub Pub Crawl Crawl through through Albany’s Albany’s Warehouse District Warehouse District brought brought perfect perfect weather, weather, great great bar bar food food buffet buffet from from Druthers Druthers Brewing, Brewing, and and aa generous generous stream of stream of smiles. smiles.

June wrapped June wrapped up up the the first first half half of of the the year year with with aa visit visit toto aa beloved beloved old-school old-school Italian Italian gem gem inin Albany’s Albany’s Delaware neighborhood.

Delaware neighborhood. Pfizer’s Pfizer’s community community liaison liaison walked walked us us through through HYMPAVZI, HYMPAVZI,, their HYMPAVZI their anti-TFPI anti-TFPI rebalancing therapy rebalancing therapy for for Hemophilia Hemophilia A A and and B, B, with with or or without without inhibitors. inhibitors. It It was was an an evening evening of of delicious delicious homestyle food, homestyle food, warm warm company, company, and and aa bright bright start start to to summer. summer.

We would We would love love for for you you toto join join us us for for what what comes comes next! next!

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June 2026

From the Executive Director As many As many of of you you know, know, II will will be be retiring retiring atat the the end end of of In In other other words, words, we we want want the the external external circumstances circumstances to to the year the year after after serving serving for for six six wonderful wonderful years years as as change change while while we we remain remain the the same. same. We We can can pray pray and and wish wish Executive Executive

Director Director of of the the Bleeding Bleeding Disorders Disorders for for miracles miracles all all day, day, and and even even manifest manifest and and say say all all the the Association of Association of Northeastern Northeastern New New York.

York. Heading Heading into into affirmations affirmations inin the the world, world, but but nothing nothing changes changes ifif we we don’t don’t this new this new chapter chapter of of my my life life (Retirement!

(Retirement! Yikes!)

Yikes!) has has me me take take different different actions. actions. But But change change is is not not enough. enough. You You can can thinking aa lot thinking lot about about change, change, and and about about growth. growth. change your change your location, location, your your hairstyle, hairstyle, even even your your sheets sheets and and towels. You towels.

You don’t don’t grow grow from from that. that. Growing Growing takes takes Growth and Growth and change change are are both both interconnected, interconnected, yet yet deeply deeply determination determination and and work. work. You You broaden broaden your your life life by by learning learning distinct concepts. distinct concepts. Change, Change, the the transition transition from from one one state state from from the the change. change. to another, to another, can can happen happen whether whether you you want want itit or or not, not, while growth while growth isis aa deliberate, deliberate, positive positive evolution. evolution. Change Change Transitions Transitions can can be be daunting, daunting, especially especially when when we’re we’re letting letting may not may not always always guarantee guarantee growth, growth, but but genuine genuine growth growth go go of of the the old old while while facing facing an an uncertain uncertain future. future. I’ve I’ve found found always demands always demands change. change. that growth that growth from from change change involves involves actively actively shifting shifting your your mindset toto view mindset view transitions transitions as as opportunities opportunities for for learning, learning, Growth isis one Growth one of of the the seven seven characteristics characteristics of of aa living living rather rather than than threats. threats. By By leaning leaning into into discomfort, discomfort, breaking breaking thing. As thing.

As adults, adults, we we sometimes sometimes forget forget that that we we grow. grow. down down large large shifts shifts into into manageable manageable steps steps and and practicing practicing Because we Because we are are no no longer longer getting getting taller, taller, we we lose lose sight sight of of some some self-compassion self-compassion you you can can build build resilience resilience and and see see the the how our how our bodies bodies and and our our brains brains are are renewing renewing every every day. day. change change inin your your life life as as an an opportunity opportunity toto learn, learn, pivot pivot and and In fact, In fact, the the concept concept of of “being “being aa grown grown up” up” implies implies that that evolve. evolve. the growing the growing isis all all done. done. Yet Yet even even though though our our bones bones are are not getting not getting any any longer, longer, we we retain retain the the capacity capacity toto grow, grow, Life Life isis aa journey, journey, not not aa destination. destination. Therefore, Therefore, everything everything to embrace to embrace positive positive transformation. transformation. we have we have experienced experienced so so far far isis meant meant toto be be used used toto keep keep us us going, not going, not for for us us to to set set up up camp camp and and lock lock ourselves ourselves up. up.

Without growth, Without growth, stagnation stagnation occurs, occurs, so so to to be be our our very very May May each each chapter chapter of of your your life life be be full full of of the the joy joy of of best selves best selves we we must must strive strive to to grow. grow. But But the the rub rub is is that that to to embracing embracing the the unfolding unfolding of of daily daily miracles, miracles, learning learning from from grow, we grow, we must must change change –– and and change change is is scary! scary! ItIt isis more more the the bumps bumps inin the the road, road, and and growing growing with with every every twist twist and and comfortable, cozy, comfortable, cozy, safe, safe, and and even even enjoyable enjoyable to to remain remain turn. turn. the same. the same. ItIt isis predictable. predictable. ItIt isis the the part part of of life life we we have have ~ Beth ~ Beth Mahr Mahr learned toto navigate learned navigate and, and, dysfunctional dysfunctional or or not, not, there there is is aa feeling of feeling of stability stability that that comes comes with with being being able able toto manage the manage the predictable. predictable. Change Change requires requires you you to to get get outside your outside your comfort comfort zone. zone.

Most of Most of the the time time when when we we say say we we want want to to change, change, it it really means really means we we want want the the problem, problem, circumstance, circumstance, and/or pain and/or pain toto disappear. disappear.

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June 2026

Scholarship Recipient Scholarship Winner!

Scholarship Winner!

The winner The winner of of BDANENY’s BDANENY’s 2026 2026 scholarship scholarship award award isis Jacob Jacob

Pettit, an Pettit, an 18-year-old 18-year-old student student from from Saugerties, Saugerties, NY. NY. Jacob Jacob

currently attends currently attends Saugerties Saugerties Senior Senior High High School School and and plans plans toto attend Alfred attend Alfred State State College.

College. His His long-term long-term goals goals are are toto become become

an electrician an electrician and and aa successful successful business business owner owner after after graduating. graduating.

Jacob isis also Jacob also passionate passionate about about wrestling. wrestling. He He has has been been aa member member of his of his school’s school’s Varsity Varsity wrestling wrestling team team since since the the 10th 10th grade grade and and

strives toto become strives become an an All-American All-American wrestler wrestler inin college. college. But But wrestling isn’t wrestling isn’t his his only only extracurricular extracurricular activity. activity. He He has has also also played JV played JV hockey hockey and and Varsity Varsity lacrosse. lacrosse.

In addition, In addition, he he isis aa member member of of the the National National Honor Honor Society, Society, aa member member of of the the Boy Boy Scouts Scouts and and an an altar altar boy boy at St. at St. Mary’s Mary’s of of the the Snow Snow Church.

Church. Having Having Von Von Willebrand Willebrand disease disease has has taught taught Jacob Jacob aa lot lot about about

perseverance, responsibility perseverance, responsibility and and mental mental fortitude. fortitude. He He writes writes inin his his essay, essay, “[a]s “[a]s aa wrestler, wrestler, hockey hockey player player and lacrosse and lacrosse player, player, II have have actively actively participated participated inin activities activities that that are are very very physical physical and and often often involve involve

contact...There have contact...There have been been times times when when injuries injuries required required me me toto sit sit out out practices practices and and matches matches for for several several weeks until weeks until my my hematologist hematologist cleared cleared me me toto return... return... II have have toto understand understand my my condition condition and and make make smart smart decisions about decisions about my my health health while while still still pushing pushing myself myself as as an an athlete... athlete... Through Through these these experiences, experiences, II have have

developed perseverance, developed perseverance, responsibility, responsibility, and and mental mental strength, strength, qualities qualities that that have have shaped shaped who who II am am and and made me made me truly truly appreciate appreciate every every chance chance II get get toto compete.” compete.”

Jacob’s Scoutmaster Jacob’s Scoutmaster concurs concurs that that Jacob Jacob isis an an exceptional exceptional young young man, man, describing describing him him as as “mature, “mature,

responsible and responsible and compassionate.” compassionate.” Scoutmaster Scoutmaster Cote Cote commends commends Jacob’s Jacob’s sense sense of of adventure adventure and and leadership, leadership,

both among both among his his peers peers as as well well as as with with younger younger Scouts.

Scouts. He He notes notes that that Jacob Jacob has has volunteered volunteered for for four four years years at Wreaths at Wreaths Across Across America, America, aa national national network network of of volunteers volunteers sponsoring sponsoring wreaths wreaths on on the the headstones headstones of of

our nation’s our nation’s service service members. members. Additionally, Additionally, Jacob Jacob isis currently currently working working toward toward achieving achieving the the rank rank of of Eagle Scout, Eagle Scout, the the highest highest achievement achievement inin scouting. scouting.

The BDA The BDA Board Board and and staff staff congratulate congratulate Jacob Jacob on on all all he he has has achieved achieved so so far far and and wish wish him the him the best best of of luck luck in in aa bright bright future! future!

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June 2026

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June 2026

Industry Update Takeda Takeda

In September In September of of last last year, year, the the FDA FDA approved approved the the extended extended use of use of Takeda’s Takeda’s Vonvendi Vonvendi for for routine routine preventive preventive use use in in adults 18 adults 18 years years and and older older for for all all types types of of von von Willebrand Willebrand

disease (VWD), disease (VWD), along along with with treatment treatment of of bleeding bleeding

episodes and episodes and periopera perioperative perioperative tive use use in in children children with with VWD. VWD. In In light of light of this, this, New New York York State’s State’s Medicaid Medicaid program program has has added Vonvendi added Vonvendi to to its its approved approved formulary formulary inin the the newly newly

expanded category, expanded category, however, however, it it will will still still require require prior prior authorization. authorization.

Although Vonvendi Although Vonvendi had had been been approved approved for for years, years, the the

FDA’s expanded FDA’s expanded use use approval approval –– and and Medicaid’s Medicaid’s recent recent

nod –– allow nod allow for for prophylaxis prophylaxis inin adults adults and and aa preventative preventative course of course of action action for for children children with with VWD. VWD. Pfizer Pfizer

Earlier this Earlier this month, month, Pfizer Pfizer received received the the European European Commission’s Commission’s

(EC) (EC) marketing marketing authorization authorization for for expanding the expanding the approved approved Hympavzi Hympavzi (marstacimab) (marstacimab) indication toto treat indication treat hemophilia hemophilia AA or or B B with with inhibitors inhibitors inin patients aged patients aged 12 12 years years and and above. above.

The approval The approval covers covers adults adults and and adolescents adolescents in in the the EuEuropean Union ropean Union (EU) (EU) who who weigh weigh atat least least 35kg 35kg (77 (77 lbs) lbs) and have and have hemophilia hemophilia A A (factor (factor VIII VIII deficiency) deficiency) with with

factor VIII factor VIII inhibitors, inhibitors, or or hemophilia hemophilia B B (factor (factor IX IX defideficiency) with ciency) with factor factor IX IX inhibitors. inhibitors. It It includes includes all all 27 27 EU EU member states, member states, Liechtenstein, Liechtenstein, Norway, Norway, and and Iceland.

Iceland.

Separately, the Separately, the US US Food Food and and Drug Drug Administration Administration has has

granted priority granted priority review review for for Hympavzi’s Hympavzi’s supplemental supplemental biologics license biologics license application application to to extend extend its its indication indication to to patients aged patients aged six six years years and and older older with with hemophilia hemophilia A A or or

B with B with or or without without inhibitors. inhibitors. AA decision decision isis expected expected by by the second the second quarter quarter of of 2026.

2026.

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Currently, in Currently, in the the United United States, States, Hympavzi Hympavzi (marstacimab-hncq) is (marstacimab-hncq) is approved approved as as aa routine routine prophylaxis treatment prophylaxis treatment to to prevent prevent or or reduce reduce the the frequency of frequency of bleeding bleeding episodes episodes for for adults adults and and patients patients 12 years 12 years and and older older with with hemophilia hemophilia A A or or B B without without inhibitors. inhibitors.

Novo Nordisk Novo Nordisk In April, In April, the the New New England England Journal Journal of of Medicine Medicine (NEJM) published (NEJM) published 26-week 26-week results results from from the the phase phase 33 FRONTIER2 trial FRONTIER2 trial evaluating evaluating the the efficacy efficacy and and safety safety of of once-monthly and once-monthly and once-weekly once-weekly denecimig denecimig (Mim8) (Mim8) in in adults and adults and adolescents adolescents 12 12 years years of of age age and and older older with with Hemophilia A, Hemophilia A, with with or or without without FVIII FVIII inhibitors. inhibitors.

Investigational denecimig Investigational denecimig isis aa bispecific bispecific antibody antibody Factor VIIIa Factor VIIIa (FVIIIa) (FVIIIa) mimetic, mimetic, designed designed for for routine routine prophylaxis to prophylaxis to help help the the body body form form blood blood clots. clots.

ItIt isis being being studied studied as as part part of of the the FRONTIER FRONTIER program program across different across different dosing dosing frequencies, frequencies, age age groups, groups, and and severities for severities for people people living living with with hemophilia hemophilia A, A, with with or or without inhibitors. without inhibitors. The The study study compared compared 254 254 adults adults and and adolescents, 12 adolescents, 12 years years and and older, older, receiving receiving onceoncemonthly or monthly or once-weekly once-weekly denecimig denecimig injections injections toto those those who received who received prior prior clotting clotting factor factor prophylaxis prophylaxis treatment during treatment during the the run-in run-in phase phase or or on-demand on-demand treatment. The treatment.

The FRONTIER2 FRONTIER2 study study measured measured how how many bleeding many bleeding episodes episodes participants participants experienced experienced each each year that year that required required treatment. treatment. People People who who received received denecimig once-monthly denecimig once-monthly had had significantly significantly fewer fewer bleeding episodes bleeding episodes compared compared to to their their previous previous treatments. treatments.

Specifically, they Specifically, they experienced experienced nearly nearly 99% 99% fewer fewer bleeds compared bleeds compared to to on-demand on-demand treatment, treatment, and and about about 43% fewer 43% fewer bleeds bleeds than than when when using using their their regular regular preventive clotting preventive clotting factor factor therapy. therapy. Similarly, Similarly, people people who received who received denecimig denecimig once-weekly once-weekly also also had had significantly fewer significantly fewer bleeding bleeding episodes. episodes.

June 2026

Industry Update Cont’d.

They experienced They experienced approximately approximately 96% 96% fewer fewer bleeds bleeds compared to compared to on-demand on-demand treatment, treatment, and and about about 54% 54% fewer fewer bleeds than bleeds than with with their their previous previous preventive preventive therapy. therapy.

Of the Of the 14 14 participants participants enrolled enrolled in in the the long-term long-term followfollow-

Denecimig, which Denecimig, which isis administered administered subcutaneously subcutaneously (under (under the skin), the skin), "mimics" "mimics" the the role role of of Factor Factor VIIIa VIIIa by by bridging bridging Factor IXa Factor IXa and and Factor Factor X. X. This This action action replaces replaces FVIIIa FVIIIa function, which function, which helps helps restore restore the the body's body's thrombin thrombin generation capacity, generation capacity, helping helping blood blood toto clot. clot. The The use use of of denecimig in denecimig in people people living living with with hemophilia hemophilia A, A, with with or or without inhibitors, without inhibitors, is is investigational investigational and and not not approved approved by by any regulatory any regulatory authorities authorities worldwide, worldwide, however, however, in in September 2025, September 2025, Novo Novo Nordisk Nordisk submitted submitted denecimig denecimig for for review to review to the the US US Food Food and and Drug Drug Administration Administration (FDA) (FDA) through aa Biologics through Biologics License License Application Application (BLA), (BLA), aa formal formal request to request to evaluate evaluate aa biologic biologic medicine. medicine.

resulted inin study resulted study discontinuation. discontinuation. Eight Eight participants participants had had

Genentech Genentech Roche Pharmaceuticals Roche Pharmaceuticals (Genentech’s (Genentech’s parent parent company) company) also has also has been been conducting conducting research research on on aa next-generation next-generation mimetic NXT007, mimetic NXT007, currently currently inin Phase Phase IIII trials. trials. ItIt was was developed by developed by optimizing optimizing the the framework framework ofemicizumab ofemicizumab (Hemlibra) to (Hemlibra) to inefficacy inefficacy and and dosing dosing convenience, convenience, increasing the increasing the mimetic mimetic activity activity and and elimination elimination half-life half-life with the with the goal goal of of improving improving potency. potency. NXT007 NXT007 binds binds to to the the same epitopes same epitopes as as emicizumab, emicizumab, but but has has aa much much greater greater binding efficiency. binding efficiency. Findings Findings comparing comparing NXT007 NXT007 to to

emicizumab were emicizumab were reported reported at at the the American American Society Society of of Hematology at Hematology at their their January January meetings. meetings.

Gene Therapy Gene Therapy This month, This month, researchers researchers atat the the American American Society Society of of Hematology (ASH) Hematology (ASH) published published their their findings findings of of aa study study 88

entitled, “Safety, entitled, “Safety, Efficacy Efficacy and and Patient-Reported Patient-Reported OutOutcomes 66 Years comes Years After After Fidanacogene Fidanacogene Elaparvovacin Elaparvovacin Adults Adults with Hemophilia with Hemophilia B.” B.” Patients Patients receiving receiving single-dose single-dose gene gene therapy for therapy for hemophilia hemophilia BB were were asked asked to to enroll enroll inin aa longlongterm follow-up term follow-up for for an an additional additional five five years, years, after after completing the completing the mandated mandated 52-week 52-week check-ins. check-ins.

up, 11 up, 11 completed completed 66 years years of of follow-up. follow-up. During During years years 22 toto 6, 99 serious 6, serious adverse adverse events events were were reported reported inin 44

participants. None participants.

None were were considered considered treatment treatment related related or or

increased alanine increased alanine aminotransferase aminotransferase (liver (liver enzyme) enzyme) levels, however levels, however no no masses, masses, malignancies, malignancies, thrombotic thrombotic

events or events or inhibitors inhibitors were were reported. reported. Factor Factor IX IX activity activity was maintained, was maintained, with with lower lower than than median median annualized annualized

bleeding rates bleeding rates reported reported during during each each year year of of follow-up. follow-up.

Ten participants Ten participants had had no no treated treated bleeding bleeding events. events. None None of of

the 14 the 14 participants participants resumed resumed prophylaxis. prophylaxis. Improvements Improvements in target in target joints joints were were also also observed. observed. Overall, Overall, the the therapy therapy

exhibited aa favorable exhibited favorable safety safety profile, profile, sustained sustained efficacy, efficacy, and improved and improved PRO PRO for for up up toto six six years. years.

A second A second study study was was published published inin March March inin the the NIH NIH

National Library National Library of of Medicine, Medicine, entitled entitled “Phase “Phase II Pilot Pilot

study for study for Hemophilia Hemophilia A: A: AAV8 AAV8 Vector Vector with with Prophylactic Prophylactic Tacolimus-glucocorticoid Achieves Tacolimus-glucocorticoid Achieves Therapeutic Therapeutic FVIII FVIII

Activity.” This Activity.”

This prospective prospective phase phase 11 pilot pilot study study evaluated evaluated

the safety the safety and and efficacy efficacy of of GS001, GS001, an an adeno-associated adeno-associated virus serotype virus serotype 88 (AAV8)-based (AAV8)-based vector. vector. Twelve Twelve adult adult severe hemophilia severe hemophilia A A participants participants were were enrolled. enrolled.

Participants received Participants received GS001 GS001 low low dose dose cohort cohort or or high high

dose cohort dose cohort following following prophylactic prophylactic treatment treatment with with prednisone alone prednisone alone or or prednisone prednisone plus plus tacrolimus tacrolimus starting starting

from one from one week week before before GS001 GS001 infusion. infusion. Both Both cohorts cohorts

showed significant showed significant reductions reductions in in annualized annualized bleeding bleeding rate and rate and factor factor VIII VIII consumption consumption with with grade grade 1-2 1-2 adverse adverse

events. Notably, events.

Notably, transgene transgene was was expressed expressed as as early early as as two two

days after days after vector vector administration administration and and increased increased rapidly rapidly to to aa high high level level within within seven seven days days inin both both dose dose cohorts. cohorts. This This

demonstrates an demonstrates an advantage advantage inin the the gene gene delivery delivery and and expression efficacy expression efficacy of of GS001.

GS001. This This early early onset onset of of

expression isis aa promising expression promising indicator indicator of of the the therapy’s therapy’s potential toto quickly potential quickly achieve achieve hemostatic hemostatic protection. protection.

Overall, GS001 Overall, GS001 infusion infusion was was safe safe and and well-tolerated, well-tolerated, achieving aa significant achieving significant increase increase in in factor factor VIII VIII level level

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Industry Update Cont’d.

Star Therapeutics Therapeutics Additionally, the Additionally, the use use of of relatively relatively low-dose low-dose vectors vectors can can also also Star On April April 21, 21, Star Star Therapeutics Therapeutics aa late late clinical-stage clinical-stage reduce production reduce production costs costs and and enhance enhance drug drug accessibility accessibility and and On biotechnology company biotechnology company focused focused on on the the discovery discovery and and affordability for affordability for HA HA gene gene therapy. therapy.

development of development of life-changing life-changing therapies therapies for for diseases diseases with with significant unmet significant unmet need, need, announced announced that that the the U.S. U.S. Food Food and and Drug Administration Drug Administration (FDA) (FDA) has has granted granted rare rare pediatric pediatric Hemab Therapeutics Hemab Therapeutics disease designation designation (RPDD) (RPDD) and and Breakthrough Breakthrough Therapy Therapy On March On March 5,5, 2026 2026 Hemab Hemab Therapeutics, Therapeutics, aa late late stage stage clinical clinical disease

A Phase A Phase 33 study study is is currently currently underway. underway.

Designation (BTD) (BTD) to to Star’s Star’s lead lead pro-gram, pro-gram, VGA039, VGA039, for for biotechnology company biotechnology company developing developing novel novel prophylactic prophylactic Designation routine prophylaxis prophylaxis to to prevent prevent or or reduce reduce the the frequency frequency of of therapeutics for therapeutics for serious, serious, underserved underserved coagulation coagulation disorders, disorders, routine bleeding episodes episodes inin patients patients with with von von Willebrand Willebrand disease disease announced that announced that the the U.S. U.S. Food Food and and Drug Drug Administration Administration bleeding (VWD). VGA039 VGA039 isis aa differentiated differentiated monoclonal monoclonal antibody antibody (FDA) has (FDA) has granted granted Breakthrough Breakthrough Therapy Therapy Designation Designation (VWD). therapy that that targets targets Protein Protein S, S, with with dual dual actions actions (BTD) for (BTD) for sutacimig sutacimig for for the the prevention prevention of of bleeding bleeding episodes episodes therapy promoting platelet platelet attachment attachment and and enhancing enhancing fibrin fibrin in patients in patients with with Glanzmann Glanzmann thrombasthenia thrombasthenia (GT).

(GT). BTD BTD promoting deposition to to restore restore hemostasis. hemostasis. VGA039 VGA039 could could be be represents the represents the FDA's FDA's formal formal commitment commitment toto facilitating facilitating the the deposition transformative for for patients, patients, as as itit isis designed designed toto prevent prevent or or development and development and review review of of drugs drugs targeting targeting serious serious or or lifelife- transformative reduce bleeding bleeding across across all all types types of of VWD VWD while while reducing reducing threatening conditions threatening conditions where where preliminary preliminary clinical clinical evidence evidence reduce treatment burden burden via via its its once once monthly, monthly, subcutaneous subcutaneous suggests the suggests the potential potential for for substantial substantial improvement improvement over over treatment dosing regimen. regimen. existing standard existing standard of of care. care. BTD BTD was was granted granted based based on on aa dosing

comprehensive data comprehensive data set, set, including including positive positive results results from from the the Upon approval approval of of aa Biologics Biologics License License Application Application (BLA), (BLA), completed Phase completed Phase 22 multiple multiple ascending ascending dose dose portion portion of of Upon this designation designation makes makes Star Star eligible eligible to to receive receive aa Priority Priority Hemab's Phase Hemab's Phase 1/2 1/2 clinical clinical trial trial of of sutacimig sutacimig (HMB-001(HMB-001- this Review Voucher Voucher (PRV), (PRV), which which may may be be redeemed redeemed to to CL101). The CL101).

The data data showed showed consistent consistent and and clinically clinically Review obtain priority priority review review for for aa subsequent subsequent marketing marketing meaningful reductions meaningful reductions inin bleeding, bleeding, including including the the most most obtain application or or transferred transferred or or sold sold to to another another sponsor. sponsor. severe bleeding severe bleeding events events requiring requiring high-intensity high-intensity treatments treatments application VGA039 has has received received Fast Fast Track, Track, orphan orphan drug, drug, rare rare (defined as (defined as those those requiring requiring recombinant recombinant Factor Factor VIIa, VIIa, VGA039 pediatric disease disease and and Breakthrough Breakthrough Therapy Therapy designations designations platelet transfusions, platelet transfusions, plasma, plasma, cryoprecipitate, cryoprecipitate, or or medical medical pediatric from the the U.S. U.S. Food Food and and Drug Drug Administration Administration (FDA).

(FDA). procedures). Sutacimig procedures).

Sutacimig has has also also received received Fast Fast Track Track from

VGA039 has VGA039 has advanced advanced into into aa Phase Phase 33 study study (NCT07115004), VIVID-6, (NCT07115004), VIVID-6, aa global global single single arm arm crosscrossover study study designed designed to to investigate investigate the the safety safety and and efficacy efficacy Sutacimig isis aa subcutaneously Sutacimig subcutaneously administered administered bispecific bispecific over of subcutaneous subcutaneous administration administration of of VGA039 VGA039 as as antibody that antibody that isis designed designed toto bind bind and and stabilize stabilize endogenous endogenous of prophylaxis for for bleeding bleeding in in patients patients with with every every type type of of Factor VIIa Factor VIIa with with one one antibody antibody arm arm and and bind bind to to TLT-1 TLT-1 on on prophylaxis VWD. Interim Interim data data from from the the Phase Phase 1/2 1/2 multidose multidose study study of of activated platelets activated platelets with with the the other other arm. arm. This This mechanism mechanism is is VWD.

Designation and Designation and Orphan Orphan Drug Drug Designation Designation from from the the FDA. FDA.

VGA039 inin adult adult and and adolescent adolescent patients patients with with VWD VWD was was designed to designed to allow allow for for the the accumulation accumulation of of endogenous endogenous VGA039 presented atat the the 67th 67th American American Society Society of of Hematology Hematology Factor VIIa Factor VIIa in in the the body body and and recruitment recruitment of of Factor Factor VIIa VIIa presented (ASH) Annual Annual Meeting Meeting and and Exposition Exposition inin December December directly to directly to the the surface surface of of the the activated activated platelets, platelets, where where it it (ASH) 2025. facilitates hemostatic facilitates hemostatic plug plug formation. formation. Sutacimig Sutacimig is is designed designed 2025. toto be be aa first-in-class first-in-class prophylactic prophylactic treatment treatment for for Glanzmann Glanzmann thrombasthenia (GT) thrombasthenia (GT) with with the the potential potential toto treat treat other other debilitating bleeding debilitating bleeding disorders, disorders, including including Von Von Willebrand’s Willebrand’s disease. disease.

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June 2026

FED UP for Women & Girls Bill For decades, For decades, bleeding bleeding disorders disorders have have been been commonly commonly associated associated with with men men by by both both

medical professionals medical professionals and and the the general general public. public. At At the the same same time, time, women women and and girls girls with with bleeding disorders bleeding disorders ranging ranging from from hemophilia hemophilia toto von von Willebrand Willebrand disease disease (the (the most most common common inherited bleeding inherited bleeding disorder), disorder), have have faced faced aa different different but but significant significant challenge: challenge: their their

symptoms are symptoms are frequently frequently overlooked overlooked or or misattributed. misattributed. Heavy Heavy menstrual menstrual bleeding, bleeding, one one of of

the most the most common common symptoms symptoms of of bleeding bleeding disorders disorders inin women, women, isis often often normalized normalized or or not not

recognized as recognized as aa sign sign of of an an underlying underlying condition, condition, which which can can contribute contribute toto delayed delayed diagnosis diagnosis

and gaps and gaps inin care. care. Recognition Recognition of of bleeding bleeding disorders disorders among among females females was was an an important important aspect of aspect of this this year’s year’s Washington Washington Days.

Days.

On May On May 22, 22, Representatives Representatives Julie Julie Johnson Johnson (D-TX), (D-TX), Joe Joe Wilson Wilson (R-SC), (R-SC), and and Sarah Sarah McBride (D-DE) McBride (D-DE) introduced introduced H.R. H.R. 8794, 8794, the the Fostering Fostering Effective Effective Diagnosis Diagnosis and and Treatment Treatment for Underserved for Underserved Populations Populations (FED (FED UP) UP) with with Bleeding Bleeding Disorders Disorders Act Act of of 2026.

2026. This This bipartisan bill bipartisan bill aims aims toto address address some some of of the the many many barriers barriers faced faced by by women women and and girls girls living living with bleeding with bleeding disorders. disorders.

The FED The FED UP UP with with Bleeding Bleeding Disorders Disorders Act Act calls calls for for an an interagency interagency review review toto identify identify federal programs, federal programs, activities activities and and strategic strategic plans plans related related to to research, research, training training and and education of education of providers, providers, access access to to treatment treatment and and services, services, and and inclusion inclusion inin clinical clinical

trials all trials all as as they they relate relate toto women women and and girls girls with with bleeding bleeding disorders. disorders. The The review review would would

culminate inin aa report culminate report that that would would include include the the findings findings and and identify identify recommendations. recommendations.

Lastly, the Lastly, the bill bill calls calls for for aa national national public public education education and and awareness awareness campaign campaign utilizing utilizing the findings the findings from from the the review review and and report. report.

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Federal Legislative Update FFY 2027 Funding For Bleeding Disorders Programs During Washington Days, advocates asked

The CDC has been without a confirmed leader for all but one month of Mr. Trump’s second term.

President Trump has had some difficulties with his

Congressional offices to support funding for federal bleeding disorders programs for Federal Fiscal Year

previous picks for Surgeon General; He had previously nominated Dr. Casey Means for the post

2027 by signing onto a Dear Colleague letter, originating with Rep. Julie Johnson of Texas. Forty-

but pulled her nomination after questioning in the Senate provoked doubts about her experience and

one bipartisan members signed on and the President’s proposed budget leaves those programs (primarily at CDC and NIH) largely intact. Congress must still approve the budget, but with such a show of support, we are cautiously optimistic that funding for bleeding disorders programs will remain steady.

her stance on vaccines. Although she received a medical degree from Stanford, she never finished her surgical residency program and did not have a medical license. Additionally, she opposed vaccinating newborns against hepatitis B.

CDC Director, Surgeon General Nominees Remain Unconfirmed In April, President Trump nominated Dr. Erica Schwartz, who served as deputy surgeon general in his first administration, to serve as the next director of the Centers for Disease Control and Prevention. Dr. Schwartz retired as a rear admiral in the US Public Health Service Commissioned Corps and is a boardcertified preventative medicine physician. She previously served as the chief medical officer of the US Coast Guard. She has a medical degree from Brown University with a law degree from the University of Maryland and a master’s degree in public health University.

from

the

Uniformed

Means was the second Surgeon General pick whose nomination had to be withdrawn. Mr. Trump withdrew his first nominee, Fox News medical contributor, Janette Nesheiwat after questions were raised about her academic credentials.

Dr. Schwartz and Dr. Saphier’s nominations must be confirmed by the Senate; the Senate Health Committee is headed by Sen. Bill Cassidy, who recently lost his primary election to a Trump-backed candidate, creating friction between Mr. Trump and Mr. Cassidy.

Services

President Trump has also announced a pick to fill the position of Surgeon General, nominating Dr. Nicole Saphier. Dr. Saphier is a radiologist and former Fox News Channel contributor. She is the director of breast imaging at Memorial Sloan Kettering and has a medical degree from Ross University School of Medicine in Barbados.

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Federal Legislative Update cont’d.

BDA Joins Colleagues Urging CMS to Exempt Our Members from Work Requirements The 2025 budget reconciliation bill (H.R.1) signed into law last July introduced new work requirements for certain Medicaid enrollees. The provision makes continued eligibility contingent upon participation in work or other qualified activities. H.R.1 mandates that Medicaid members aged 19-64 who are covered through ACA Medicaid expansion or an 115 demonstration waiver that provides minimum essential coverage must engage in employment, education, a work program or community service to maintain their Medicaid eligibility. These requirements take effect June 1, 2026.

The BDA has signed onto a letter – originating with the Hemophilia Alliance, the National Bleeding Disorders Foundation, the Hemophilia Federation of America and the Coalition for Hemophilia B - to urge CMS to recognize bleeding disorders as serious complex medical conditions ensuring exemption from the new community engagement requirements. The letter urges that CMS and states must define exemptions for “serious or complex medical conditions as per the Social Security Act and that individuals with bleeding disorders should be categorically exempt based on diagnosis or treatment codes.

Furthermore, as a bleeding disorder is a lifelong condition, individuals so diagnosed should only need to prove exemption once. We feel that it is important to safeguard coverage to prevent health deterioration.

Unfortunately, on June 1, 2026, the Centers for Medicare and Medicaid Services (CMS) issued an interim final rule on Medicaid work requirements that adopts a restrictive definition of medical frailty – differing from states’ early expectations. The more restrictive definition of medical frailty adopted by the Trump Administration will be challenging for states to implement and could lead to more people falling through the cracks and losing coverage.

We will continue to advocate for this important exemption for all of our members.

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State Legislative Update Cover Rare Bill (S.965/A.2384) The need for out-of-network care means more out-of-pocket costs to families, and can compound the stress of caring for a child with lasting medical needs. In the U.S., the reality is that the more health care needs you have, especially from specialists, the greater chance you will find your needs won’t be met, even if you have private insurance coverage. In fact, in a study of more than 1800 ACA silver plan networks, 44% of pediatric networks had no specialists who practiced in.

The Cover Rare bill is “[a]n act to amend the insurance law and the public health law, in relation to providing insurance coverage for rare diseases, life-threatening conditions or diseases, degenerative and disabling conditions, or diagnoses involving medically fragile children.” It allows parents of children with rare, fragile, & deteriorating conditions to access out-of-network specialists with crucial expertise for diagnosis & disease-specific care.

Health plans must be notified in advance and are only required to cover these services at their established in-network rates.

During this session, the bill advanced to the third reading on the Assembly floor, however, the original Senate sponsor has left the NYS Senate, therefore we are looking for a new Senate sponsor. There has been a great deal of bipartisan interest in this bill, so we are confident that we will be in a good position for the 2027 session.

Pre-Authorization Reform Pre-authorization is a practice used by health plans and Pharmacy Benefit Managers to manage the utilization of covered treatments and medications.

Under pre-authorization, patients and/or providers must submit additional information to a plan or PBM to obtain selected treatments and medications.

Each health plan and PBM may have their own preauthorization rules and the process may require multiple submissions of information for an initial ruling. Excessive pre-authorization rules are a huge administrative burden for providers and often delay patient care, increasing the cost of treatment without promoting better health outcomes.

This year, Governor Hochul heard the concerns of advocates and included some prior authorization reform measures in her budget bill.

Part HH of Title IV of the Fiscal Year 2026-2027 NYS Budget Bill: Mandates that a prior authorization for a "designated chronic condition" remains valid for at least one year.

Extends for 60 to 90 days the window during which a new enrollee can see an out-of-network provider if they are receiving an ongoing course of treatment.

Requires the Department of Financial Services (DFS) to collect and publish expanded data from health insurers regarding prior authorizations.

Mandates health insurance plans to make their prescription drug formularies publicly available in a standardized, accessible format. * These provisions were all adopted as part of the New York State Budget that was passed on May 28.

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Board of Directors and Staff President: President: Bruce Weiner Bruce Weiner Vice President: Vice President: Chris Huskie Chris Huskie Treasurer: Treasurer: Cindi Keeler Cindi Keeler Secretary: Secretary: Christina Mastrianni Christina Mastrianni

Board Members: Board Members: Mama Yaa Mama Yaa Boakye Boakye Charity Borst Charity Borst Mary Anne Mary Anne Cechetto Cechetto Patricia Marck Patricia Marck Jessica Smith Jessica Smith

Executive Director: Executive Director: Beth Mahr Beth Mahr Operations Manager: Operations Manager: Michael Budka Michael Budka

** Now Now Accepting Accepting Applications Applications for for Seats Seats on on the the BDANENY BDANENY Board Board of of Directors Directors

Qualifications Qualifications

Professional experience Professional experience inin business, business, government, government, philanthropy, philanthropy, or or the the nonprofit nonprofit sector sector and/or aa member and/or member of of the the bleeding bleeding disorders disorders community. community.

A commitment A commitment toto and and understanding understanding of of the the BDANENY BDANENY mission. mission.

An ability An ability toto build build consensus consensus among among diverse diverse individuals. individuals.

Personal qualities Personal qualities of of integrity, integrity, sincerity, sincerity, and and aa desire desire toto improve improve the the lives lives of of individuals individuals and families and families impacted impacted by by an an inherited inherited bleeding bleeding disorder. disorder.

Commitment parameters Commitment parameters

Term –– Three Term Three years, years, renewable renewable

Time –– Board Time Board meetings meetings every every month; month; 10 10 remotely remotely and and 22 in-person; in-person; meetings meetings typically typically last 90 last 90 minutes minutes

Support of Support of BDANENY BDANENY Fundraising Fundraising Events Events To apply To apply toto join join the the Board, Board, please please visit visit www.bdaneny.org/BoardApplication www.bdaneny.org/BoardApplication toto download the download the application. application.

Contact Us .

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333 Broadway, Suite 210 Troy, New York 12180 (518)729-3577 www.bdaneny.org June 2026