THEVESSEL THE VESSEL
THEVESSEL THE VESSEL
Thefirsthalfof2026gaveourcommunityabitofeverything,fromeducationabout newtreatmentsonthemarketandadvocacyforbetterhealthoutcomes,todelicious localfood.Itwasasteadyflowofopportunitiestoconnectandraisesomefundsfor programsthatmeansomuchtous.
January kicked us off with a Takeda dinner at Jacob &Anthony’s in Saratoga Springs, where members January us a at & Anthony’s in learnedabout learned about VONVENDI,afirst-in-classrecombinantextendedhalf-lifetherapyforvWDthatcanoften , a first-in-class recombinant extended half-life therapy for vWD that can often treatbleedingepisodesinasingledose. treat bleeding episodes in a single dose.
InMarch,membersfromacrossNew In March, members from across New YorktraveledtoCapitolHillinWashington,D.C.for York traveled to Capitol Hill in Washington, D.C. for Washington Washington Days Days andthenbackhomefor and then back home for AlbanyDays Albany Days attheStateCapitol.Advocacyatbothstateandfederallevels at the State Capitol. Advocacy at both state and federal levels focusedonprotectingandstrengtheningaccesstocareandsupportforindividualsandfamilieslivingwith focused on protecting and strengthening access to care and support for individuals and families living with bleedingdisorders bleeding disorders.
ByApril,ElNiñohadhelpedmeltthelastofthesnowaway,andwegatheredattheScarletKnifeinLatham
By April, El Niño had helped melt the last of the snow away, and we gathered at the Scarlet Knife in Latham for a Sanofi educational dinner. Their hematology liaison introduced a Qfitlia Qfitlia, an antithrombin-lowering , an siRNA rebalancing therapy for prophylaxis in Hemophilia A and B, with or without inhibitors. The rebalancing therapy prophylaxis in A B, or presentationpairedeffortlesslywithastandoutspringmenuthatalmostmadeyouforgetitwasstillbelow presentation paired effortlessly with a standout spring menu that almost made you forget it was still below freezingoutside. freezing outside.
May awakened our competitive side atThe Bunker in Clifton Park, where members of all ages took on
May awakened our competitive side at The Bunker Clifton where members of all ages took on indoorgolfandaspiritedmini-golfshowdownfueledbyfantasticfingerfood.Betweenswings,welearned indoor golf and a spirited mini-golf showdown fueled by fantastic finger food. Between swings, we learned about Novo Nordisk’s rebalancing therapy, about Novo Nordisk’s rebalancing therapy, Alhemo Alhemo, a Tissue Factor Pathway Inhibitor antagonist for , a Tissue Factor Pathway Inhibitor antagonist for Hemophilia A and B, with or without inhibitors Later that month, our Pub Crawl through Albany’s Hemophilia A and B, with or without inhibitors. Later that month, our Pub Crawl through Albany’s WarehouseDistrictbroughtperfectweather,greatbarfoodbuffetfromDruthersBrewing,andagenerous Warehouse District brought perfect weather, great bar food buffet from Druthers Brewing, and a generous streamofsmiles. stream of smiles.
June wrapped up the first half of the year with a visit to a beloved old-school Italian gem inAlbany’s June wrapped up first year a to a gem in Albany’s Delaware neighborhood. Pfizer’s community liaison walked us through neighborhood. community through HYMPAVZI HYMPAVZI, their anti-TFPI , rebalancing therapy for HemophiliaAand B, with or without inhibitors. It was an evening of delicious rebalancing therapy for Hemophilia A and B, with or without inhibitors. It was evening of delicious homestylefood,warmcompany,andabrightstarttosummer. homestyle food, warm company, and a bright start to summer.
The Official Newsletter for The Bleeding Disorders Association of Northeastern New York
From the Executive Director
As many of you know, I will be retiring at the As many of you I will be retiring at the end of end of the year after serving for six wonderful years as year years as
Executive Director of the Bleeding Disorders
Executive Director of the Bleeding Disorders Association of Northeastern New Association of Northeastern New York. Heading into York. Heading into thisnewchapter ofmylife (Retirement!Yikes!)hasme this chapter of life (Retirement! Yikes!) has thinkingalotaboutchange,andaboutgrowth. thinking a lot about change, and about growth.
Growth and change are both interconnected, yet deeply
Growth and change both interconnected, deeply distinct concepts. Change, the transition from one state distinct concepts. Change, the transition from one state to another, can happen whether you want it or not, another, can you or not, whilegrowthisadeliberate, positive evolution.Change while growth is a deliberate, positive evolution. Change may not always guarantee growth, but genuine growth may not always guarantee growth, but genuine growth alwaysdemandschange. always demands change.
In other words, we want the external circumstances to In other we want the external circumstances to change while we remain the same. We can pray and wish we same. can pray for miracles all day, and even manifest and say all the for miracles all day, and even manifest and all the affirmations in the world, but nothing changes if we don’t affirmations the world, but nothing changes we don’t take different actions. But change is not enough. You can take different actions. change is enough. change your location, your hairstyle, even your sheets and change your your hairstyle, even your sheets and towels. You don’t grow from that. Growing takes towels. You don’t grow from that. Growing takes determination and work.You broaden your life by learning determination and work. broaden life by learning fromthechange. from the change.
Transitions can be daunting, especially when we’re letting
Growth is one of the seven characteristics of a living
Growth is one of the seven characteristics of a living thing. As adults, we sometimes forget that we grow. thing. adults, sometimes forget that Becausewearenolongergettingtaller,welosesightof Because we are no longer getting taller, we lose sight of how our bodies and our brains are renewing every day. our our are every In fact, the concept of “being a grown up” implies that In fact, the concept of “being a grown up” implies that the growing is all done. Yet even though our bones are the growing is all done. Yet even though our bones are not getting any longer, we retain the capacity to grow, getting longer, retain the capacity toembracepositivetransformation. to embrace positive transformation.
Without growth, stagnation occurs, so to be our very Without growth, stagnation occurs, be bestselveswemuststrivetogrow.Buttherubisthatto best selves we must strive to grow. But the rub is that to grow, we must change – and change is scary! It is more change – and change is is comfortable, cozy, safe, and even enjoyable to remain comfortable, cozy, safe, and even enjoyable to remain the same. It is predictable. It is the part of life we have the same. It is predictable. It is the part of life we have learned to navigate and, dysfunctional or not, there is a learned navigate and, dysfunctional not, there is feeling of stability that comes with being able to feeling of stability that comes with being able to manage the predictable. Change requires you to get manage Change you to get outsideyourcomfortzone. outside your comfort zone.
Most of the time when we say we want to change, it of the time when change, it really means we want the problem, circumstance, really means we want the problem, and/orpaintodisappear. and/or pain to disappear.
Transitions can be daunting, especially when we’re letting go of the old while facing an uncertain future. I’ve found go of the old while facing an uncertain future. I’ve found that growth from change involves actively shifting your that growth from change involves actively shifting mindset to view transitions as opportunities for learning, mindset to view transitions as opportunities for learning, rather than threats. By leaning into discomfort, breaking rather threats. By leaning into discomfort, breaking down large shifts into manageable steps and practicing down large shifts into manageable and practicing some self-compassion you can build resilience and see the some self-compassion you can build resilience and see the change in your life as an opportunity to learn, pivot and your life as an learn, evolve. evolve.
Life is a journey, not a destination. Therefore, everything
Life is journey, destination. Therefore, everything we have experienced so far is meant to be used to keep us we have experienced so far is meant to be used to keep us going, not for us to set up camp and lock ourselves up. going, not us to set up camp up. May each chapter of your life be full of the joy of each chapter of life be full of the joy of embracing the unfolding of daily miracles, learning from embracing the unfolding of daily miracles, learning from the bumps in the road, and growing with every twist and the bumps in the road, and growing with twist and turn. turn.
~BethMahr ~ Beth Mahr
Scholarship Recipient
ScholarshipWinner! ScholarshipWinner!
ThewinnerofBDANENY’s2026scholarshipawardisJacob The winner of BDANENY’s 2026 scholarship award is Jacob Pettit, an 18-year-old student from Saugerties, NY. Jacob Pettit, 18-year-old student from Saugerties, NY. Jacob currentlyattendsSaugertiesSeniorHighSchoolandplansto currently attends Saugerties Senior High School and plans to attendAlfredStateCollege.Hislong-termgoalsaretobecome attend Alfred State College. His long-term goals are to become anelectricianandasuccessfulbusinessowneraftergraduating. an electrician and a successful business owner after graduating. Jacobisalsopassionateaboutwrestling Hehasbeenamember Jacob is also passionate about wrestling. He has been a member ofhisschool’sVarsitywrestlingteamsincethe10thgradeand of his school’s Varsity wrestling team since the 10th grade and strives to become anAll-American wrestler in college But strives to become an All-American wrestler in college. But wrestling isn’t his only extracurricular activity. He has also wrestling only activity. He playedJVhockeyandVarsitylacrosse. played JV hockey and Varsity lacrosse.
Inaddition,heisamemberoftheNationalHonorSociety,amemberoftheBoyScoutsandanaltarboy In addition, he is a member of the National Honor Society, a member of the Boy Scouts and an altar boy at St. Mary’s of the Snow Church. Having Von Willebrand disease has taught Jacob a lot about at St. Mary’s Snow Having Von taught perseverance,responsibilityandmentalfortitude.Hewritesinhisessay,“[a]sawrestler,hockeyplayer perseverance, responsibility and mental fortitude. He writes in his essay, “[a]s a wrestler, hockey player andlacrosseplayer,Ihaveactivelyparticipatedinactivitiesthatareveryphysicalandofteninvolve and lacrosse player, I have actively participated in activities that are very physical and often involve contact...Therehavebeentimeswheninjuriesrequiredmetositoutpracticesandmatchesforseveral contact...There have been times when injuries required me to sit out practices and matches for several weeksuntilmyhematologistclearedmetoreturn Ihavetounderstandmyconditionandmakesmart weeks until my hematologist cleared me to return... I have to understand my condition and make smart decisionsaboutmyhealthwhilestillpushingmyselfasanathlete...Throughtheseexperiences,Ihave decisions about my health while still pushing myself as an athlete... Through these experiences, I have developedperseverance,responsibility,andmentalstrength,qualitiesthathaveshapedwhoIamand developed perseverance, responsibility, and mental strength, qualities that have shaped who I am and mademetrulyappreciateeverychanceIgettocompete.”
Jacob’s Scoutmaster concurs that Jacob is an exceptional young man, describing him as “mature, Jacob’s Scoutmaster concurs that Jacob is an young man, him as “mature, responsibleandcompassionate.”ScoutmasterCotecommendsJacob’ssenseofadventureandleadership, responsibleandcompassionate.”ScoutmasterCotecommendsJacob’ssenseofadventureandleadership, bothamonghispeersaswellaswithyoungerScouts HenotesthatJacobhasvolunteeredforfouryears both among his peers as well as with younger Scouts. He notes that Jacob has volunteered for four years atWreathsAcrossAmerica,anationalnetworkofvolunteerssponsoringwreathsontheheadstonesof at Wreaths Across America, a national network of volunteers sponsoring wreaths on the headstones of ournation’sservicemembers.Additionally,Jacobiscurrentlyworkingtowardachievingtherankof our nation’s service members. Additionally, Jacob is currently working toward achieving the rank of EagleScout,thehighestachievementinscouting EagleScout,thehighestachievementinscouting.
TheBDABoardandstaffcongratulateJacobonallhehasachievedsofarandwish The BDABoard and staff congratulate Jacob on all he has achieved so far and wish himthebestofluckinabrightfuture! him the best of luck in a bright future!
Industry Update
Takeda
In In September of last year, the FDAapproved the extended September of last year, the FDA approved the extended use of Takeda’s Vonvendi for routine preventive use in use use adults 18 years and older for all types of von Willebrand adults and older for all of Willebrand disease (VWD), along with treatment of bleeding disease (VWD), along with treatment of bleeding episodes and periopera episodes and perioperative use in children with VWD. In use VWD. In light of this, New York State’s Medicaid program has light of this, York Medicaid has added Vonvendi to its approved formulary in the newly added Vonvendi to its approved formulary in the newly expanded category, however, it will still require prior expanded category, however, will still require prior authorization. authorization.
AlthoughVonvendihadbeenapprovedforyears,the Although Vonvendi had been approved for years, the FDA’s expanded use approval – and Medicaid’s recent expanded approval – and Medicaid’s nod – allow for prophylaxis in adults and a preventative nod – allow for prophylaxis in adults and a preventative courseofactionforchildrenwithVWD. course of action for children with VWD.
Pfizer Pfizer
Earlier this month, Pfizer received the European Earlier this month, Pfizer received the European Commission’s (EC) marketing authorization for Commission’s (EC) marketing authorization for expanding the approved Hympavzi (marstacimab) expanding the approved Hympavzi (marstacimab) indication to treat hemophilia A or B with inhibitors in indication to treat hemophilia or B with inhibitors patientsaged12yearsandabove. patients aged 12 years and above.
TheapprovalcoversadultsandadolescentsintheEu-
The approval covers adults and adolescents in the EuropeanUnion(EU)whoweighatleast35kg(77lbs) ropean Union (EU) who weigh at least 35kg (77 lbs) andhavehemophiliaA(factorVIIIdeficiency)with and have hemophilia A (factor VIII deficiency) with factor VIII inhibitors, or hemophilia B (factor IX defi- factor VIII inhibitors, or hemophilia B (factor IX deficiency)withfactorIXinhibitors.Itincludesall27EU ciency) with factor IX inhibitors. It includes all 27 EU memberstates,Liechtenstein,Norway,andIceland. member states, Liechtenstein, Norway, and Iceland.
Separately, the US Food and Drug Administration has Separately, the Food and Administration has granted priority review for Hympavzi’s supplemental granted priority review for Hympavzi’s supplemental biologicslicenseapplicationtoextenditsindicationto biologics license application to extend its indication to patientsagedsixyearsandolderwithhemophiliaAor six years and older with or Bwithorwithoutinhibitors.Adecisionisexpectedby B with or without inhibitors. A decision is expected by thesecondquarterof2026. the second quarter of 2026.
Currently, in the United States, Hympavzi Currently, in the United States, Hympavzi (marstacimab-hncq) is approved as a routine (marstacimab-hncq) is approved as a routine prophylaxis treatment to prevent or reduce the prophylaxis treatment to prevent or reduce the frequency of bleeding episodes for adults and patients frequency of bleeding episodes for adults and patients 12 years and older with hemophilia A or B without years or inhibitors. inhibitors.
NovoNordisk Novo Nordisk
In April, the New England Journal of Medicine In April, New England Journal (NEJM) published 26-week results from the phase 3 (NEJM) published 26-week results from the phase FRONTIER2 trial evaluating the efficacy and safety of FRONTIER2 trial evaluating the efficacy and safety of once-monthly and once-weekly denecimig (Mim8) in once-monthly and once-weekly denecimig (Mim8) in adults and adolescents 12 years of age and older with adults and adolescents years of age and older with Hemophilia A, with or without FVIII inhibitors. A, or Investigational denecimig is a bispecific antibody Investigational denecimig is bispecific antibody FactorVIIIa(FVIIIa)mimetic,designedforroutine Factor VIIIa (FVIIIa) mimetic, designed for routine prophylaxistohelpthebodyformbloodclots. prophylaxis to help the body form blood clots.
It is being studied as part of the FRONTIER program is being studied of the across different dosing frequencies, age groups, and across different dosing frequencies, and severities for people living with hemophiliaA, with or severities for people living with hemophilia A, with or without inhibitors. The study compared 254 adults and without inhibitors. The study compared 254 adults and adolescents, 12 years and older, receiving once- adolescents, years older, oncemonthly or once-weekly denecimig injections to those monthly once-weekly denecimig injections those who received prior clotting factor prophylaxis who received prior clotting factor prophylaxis treatment during the run-in phase or on-demand treatment during the run-in phase or on-demand treatment. The FRONTIER2 study measured how treatment. The FRONTIER2 study measured how many bleeding episodes participants experienced each bleeding episodes participants experienced each year that required treatment. People who received that required People who received denecimig once-monthly had significantly fewer denecimig once-monthly had significantly fewer bleeding episodes compared to their previous bleeding episodes compared to their previous treatments. treatments.
Specifically, they experienced nearly 99% fewer bleeds compared to on-demand treatment, and about bleeds compared to on-demand treatment, and about 43% fewer bleeds than when using their regular 43% fewer bleeds when using their regular preventive clotting factor therapy. Similarly, people preventive clotting factor therapy. Similarly, people who received denecimig once-weekly also had who received denecimig once-weekly also had significantlyfewerbleedingepisodes. significantly fewer bleeding episodes.
Specifically, they experienced nearly 99% fewer
Industry Update Cont’d.
They experienced approximately 96% fewer bleeds They experienced approximately 96% fewer bleeds compared to on-demand treatment, and about 54% fewer compared to on-demand treatment, and about 54% fewer bleedsthanwiththeirpreviouspreventivetherapy. bleeds than with their previous preventive therapy.
Denecimig, which is administered subcutaneously (under Denecimig, which is administered subcutaneously (under the skin), "mimics" the role of Factor VIIIa by bridging the skin), "mimics" the role of Factor VIIIa by bridging Factor IXa and Factor X. This action replaces FVIIIa Factor IXa Factor X. FVIIIa function, which helps restore the body's thrombin function, which helps the body's thrombin generation capacity, helping blood to clot. The use of generation capacity, helping blood to clot. The use of denecimig in people living with hemophilia A, with or denecimig people living with hemophilia A, with or without inhibitors, is investigational and not approved by inhibitors, any regulatory authorities worldwide, however, in regulatory authorities worldwide, however, in September 2025, Novo Nordisk submitted denecimig for September 2025, Novo Nordisk submitted denecimig for review to the US Food and Drug Administration (FDA) review to the US Food and Drug Administration (FDA) through a Biologics LicenseApplication (BLA), a formal through Biologics License Application (BLA), formal requesttoevaluateabiologicmedicine. request to evaluate a biologic medicine.
Genentech
Roche Pharmaceuticals (Genentech’s parent company)
Roche Pharmaceuticals (Genentech’s company) also has been conducting research on a next-generation also has been conducting research on a next-generation mimetic NXT007, currently in Phase II trials. It was mimetic NXT007, currently Phase II trials. It was developed by optimizing the framework ofemicizumab developed (Hemlibra) to inefficacy and dosing convenience, (Hemlibra) inefficacy and dosing convenience, increasing the mimetic activity and elimination half-life increasing the mimetic activity and elimination half-life withthegoalofimprovingpotency.NXT007bindstothe with the goal of improving potency. NXT007 binds to the same epitopes as emicizumab, but has a much greater epitopes emicizumab, but has a much binding efficiency Findings comparing NXT007 to binding efficiency. Findings comparing NXT007 to emicizumab were reported at the American Society of emicizumab were reported at the American Society of HematologyattheirJanuarymeetings. Hematology at their January meetings.
GeneTherapy
Gene Therapy
This month, researchers at the American Society of This month, researchers at the American Society of Hematology (ASH) published their findings of a study 8 (ASH) a entitled, “Safety, Efficacy and Patient-Reported Out- entitled, “Safety, Efficacy and Patient-Reported Outcomes 6 YearsAfter Fidanacogene ElaparvovacinAdults comes 6 Years After Fidanacogene Elaparvovacin Adults with Hemophilia B.” Patients receiving single-dose gene with Hemophilia B.” Patients receiving single-dose gene therapy for hemophilia B were asked to enroll in a long- therapy for hemophilia asked enroll in longterm follow-up for an additional five years, after term follow-up for an additional five years, after completingthemandated52-weekcheck-ins. completing the mandated 52-week check-ins.
Of the 14 participants enrolled in the long-term follow-
Of the participants enrolled in the long-term followup,11completed6yearsoffollow-up.Duringyears2to up, 11 completed 6 years of follow-up. During years 2 to 6, 9 serious adverse events were reported in 4 6, 9 serious adverse events were reported participants. None were considered participants. considered treatment related or related resulted in study discontinuation. resulted in study discontinuation. Eight participants had Eight participants had increased alanine aminotransferase (liver enzyme) increased alanine aminotransferase (liver enzyme) levels, however no masses, malignancies, thrombotic levels, however masses, malignancies, thrombotic events or inhibitors were reported. Factor IX activity events or inhibitors were reported. Factor IX activity was maintained, with lower than median annualized was maintained, with lower than median annualized bleeding rates reported during each year of follow-up. bleeding reported during each follow-up. Ten participants had no treated bleeding events. None of Ten participants had no treated bleeding events. None of the 14 participants resumed prophylaxis. Improvements the 14 participants resumed prophylaxis. Improvements in target joints were also observed. Overall, the therapy were Overall, therapy exhibited a favorable safety profile, sustained efficacy, exhibited a favorable safety profile, sustained efficacy, andimprovedPROforuptosixyears. and improved PRO for up to six years.
A second study was published in March in the NIH A second study was published in March in the NIH National Library of Medicine, entitled “Phase I Pilot National Library of Medicine, entitled “Phase I Pilot studyforHemophiliaA:AAV8Vector with Prophylactic Hemophilia Prophylactic
Tacolimus-glucocorticoid Achieves Therapeutic FVIII Tacolimus-glucocorticoid Achieves Therapeutic Activity.”This prospective phase 1 pilot study evaluated Activity.” This prospective phase 1 pilot study evaluated the safety and efficacy of GS001, an adeno-associated safety efficacy GS001, an adeno-associated virus serotype 8 (AAV8)-based vector. Twelve adult virus (AAV8)-based Twelve adult severe hemophilia A participants were enrolled. severe hemophilia A participants were enrolled. Participants received GS001 low dose cohort or high or high dose cohort following prophylactic treatment with dose cohort following prophylactic with prednisone alone or prednisone plus tacrolimus starting prednisone alone or prednisone plus tacrolimus starting from one week before GS001 infusion. Both cohorts one GS001 cohorts showed significant reductions in annualized bleeding showed significant reductions in annualized bleeding rate and factorVIII consumption with grade 1-2 adverse rate and factor VIII consumption with grade 1-2 adverse events.Notably,transgenewasexpressedasearlyastwo events. Notably, transgene was expressed as early as two days after vector administration and increased rapidly to days after administration and increased rapidly ahighlevelwithinsevendaysinbothdosecohorts.This a high level within seven days in both dose cohorts. This demonstrates an advantage in the gene delivery and demonstrates an advantage the gene delivery and expression efficacy of GS001. This early onset of expression efficacy of This early of expression is a promising indicator of the therapy’s expression is a promising indicator of the therapy’s potential to quickly achieve hemostatic protection. potential to quickly achieve hemostatic protection. Overall, GS001 infusion was safe and well-tolerated, Overall, infusion safe and well-tolerated, achievingasignificantincreaseinfactorVIIIlevel achieving a significant increase in factor VIII level
Industry Update Cont’d.
Additionally, the use of relatively low-dose vectors can also Additionally, the of relatively low-dose also reduce production costs and enhance drug accessibility and reduce production costs and enhance drug accessibility and affordabilityforHAgenetherapy. affordability for HA gene therapy. A Phase 3 study is currently underway. Phase 3 study is currently underway.
HemabTherapeutics Hemab Therapeutics
OnMarch5,2026HemabTherapeutics, alate stageclinical 5, a stage biotechnology company developing novel prophylactic biotechnology developing novel prophylactic therapeutics for serious, underserved coagulation disorders, therapeutics for underserved coagulation announced that the U.S. Food and Drug Administration announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (FDA) (BTD)forsutacimigforthepreventionofbleedingepisodes (BTD) for sutacimig for the prevention of bleeding episodes in patients with Glanzmann thrombasthenia (GT). BTD in patients with Glanzmann thrombasthenia (GT). BTD represents the FDA's formal commitment to facilitating the represents the FDA's formal commitment to facilitating the development and review of drugs targeting serious or life- development and review of drugs targeting serious lifethreatening conditions where preliminary clinical evidence threatening conditions where preliminary clinical evidence suggests the potential for substantial improvement over suggests the potential for substantial improvement over existing standard of care. BTD was granted based on a existing care. BTD was granted on a comprehensive data set, including positive results from the comprehensive data set, including positive results from the completed Phase 2 multiple ascending dose portion of completed Phase 2 multiple ascending dose portion of Hemab's Phase 1/2 clinical trial of sutacimig (HMB-001- Hemab's Phase 1/2 clinical trial of sutacimig (HMB-001CL101). The data showed consistent and clinically CL101). meaningful reductions in bleeding, including the most meaningful reductions in bleeding, including the severe bleeding events requiring high-intensity treatments severe bleeding events requiring high-intensity treatments (defined as those requiring recombinant Factor VIIa, (defined as those requiring recombinant Factor VIIa, platelet transfusions, plasma, cryoprecipitate, or medical platelet transfusions, plasma, cryoprecipitate, medical procedures). Sutacimig has also received Fast Track procedures). Sutacimig has also received Fast Track DesignationandOrphanDrugDesignationfromtheFDA. Designation and Orphan Drug Designation from the FDA.
Sutacimig is a subcutaneously administered bispecific Sutacimig is subcutaneously administered bispecific antibody that is designed to bind and stabilize endogenous antibody that is designed to bind and stabilize endogenous Factor VIIa with one antibody arm and bind to TLT-1 on Factor VIIa with one antibody arm and bind to on activated platelets with the other arm. This mechanism is arm. designed to allow for the accumulation of endogenous designed allow for the accumulation of endogenous Factor VIIa in the body and recruitment of Factor VIIa Factor VIIa in the body and recruitment of Factor VIIa directly to the surface of the activated platelets, where it directly to the surface of the activated platelets, where facilitates hemostatic plug formation. Sutacimig is designed facilitates hemostatic plug formation. Sutacimig is designed to be a first-in-class prophylactic treatment for Glanzmann to be a first-in-class prophylactic treatment for Glanzmann thrombasthenia (GT) with the potential to treat other thrombasthenia (GT) with the potential to treat other debilitating bleeding disorders, including Von Willebrand’s debilitating bleeding disorders, including Von disease. disease.
StarTherapeutics Star Therapeutics
On April 21, Star Therapeutics a late clinical-stage On April 21, Star Therapeutics a late clinical-stage biotechnology company focused on the discovery and biotechnology focused the discovery and development of life-changing therapies for diseases with development of life-changing therapies for diseases with significant unmet need, announced that the U.S.Foodand significant unmet announced that the U.S. Food and Drug Administration (FDA) has granted rare pediatric Drug Administration (FDA) has granted rare pediatric disease designation (RPDD) and Breakthrough Therapy disease designation (RPDD) and Breakthrough Therapy
Designation (BTD) to Star’s lead pro-gram, VGA039, for Designation (BTD) to Star’s lead pro-gram, VGA039, for routine prophylaxis to prevent or reduce the frequency of routine prophylaxis to prevent or reduce the frequency of bleeding episodes in patients with vonWillebrand disease bleeding patients von (VWD).VGA039 is a differentiated monoclonal antibody (VWD). is a differentiated monoclonal antibody therapy that targets Protein S, with dual actions therapy that targets Protein S, with dual actions promoting platelet attachment and enhancing fibrin promoting platelet attachment and enhancing fibrin deposition to restore hemostasis. VGA039 could be deposition hemostasis. could be transformative for patients, as it is designed to prevent or transformative for patients, as it is designed to or reduce bleeding across all types of VWD while reducing reduce bleeding across all types of VWD while reducing treatment burden via its once monthly, subcutaneous treatment burden via its once monthly, subcutaneous dosingregimen. dosing regimen.
UponapprovalofaBiologicsLicenseApplication(BLA), Upon approval of a Biologics License Application (BLA), this designation makes Star eligible to receive a Priority to a Review Voucher (PRV), which may be redeemed to Review Voucher (PRV), which be redeemed obtain priority review for a subsequent marketing obtain priority review for a subsequent marketing application or transferred or sold to another sponsor. application or transferred or sold to another sponsor.
VGA039 has received Fast Track, orphan drug, rare has received Track, orphan drug, pediatric disease and Breakthrough Therapy designations pediatric disease and Breakthrough Therapy designations from the U.S. Food and Drug Administration (FDA). from the U.S. Food and Drug Administration (FDA). VGA039 has advanced into a Phase 3 study VGA039 has advanced into a Phase study (NCT07115004), VIVID-6, a global single arm cross- (NCT07115004), VIVID-6, global single over study designed to investigate over study designed to investigate the safety and efficacy the safety and efficacy of subcutaneous administration of VGA039 as of subcutaneous administration of VGA039 as prophylaxis for bleeding in patients with every type of with every type VWD.InterimdatafromthePhase1/2multidosestudyof Interim data from the Phase 1/2 multidose study of VGA039 in adult and adolescent patients with VWD was VGA039 in adult and adolescent patients with VWD was presented at the 67th American Society of Hematology presented at the 67th American Society of Hematology (ASH) Annual Meeting and Exposition in December (ASH) Annual Meeting and Exposition in December 2025. 2025.
FED UP for Women & Girls Bill
For decades, bleeding disorders have been commonly associated with men by decades, bleeding disorders have been commonly associated with men by medical professionals and medical professionals and the general public. At the same time, women and girls with the general public. At the same time, women and girls with bleeding disorders ranging from hemophilia to vonWillebrand disease (the most common bleeding disorders ranging from hemophilia to von Willebrand disease (the most common inherited bleeding disorder), have faced a different but significant challenge: their inherited bleeding disorder), have faced a different but significant challenge: their symptoms are frequently overlooked or misattributed. Heavy menstrual bleeding, one of symptoms are frequently overlooked or misattributed. Heavy menstrual bleeding, one of the most common symptoms of bleeding disorders in women, is often normalized or not the most common symptoms of bleeding disorders in women, is often normalized or not recognizedasasignofanunderlyingcondition,whichcancontributetodelayeddiagnosis recognized as a sign of an underlying condition, which can contribute to delayed diagnosis and gaps in care. Recognition of bleeding disorders among females was an important and in care. of disorders females was an important aspectofthisyear’sWashingtonDays. aspect of this year’s Washington Days.
On May 22, Representatives Julie Johnson (D-TX), Joe Wilson (R-SC), and Sarah
On May 22, Representatives Julie Johnson (D-TX), Joe Wilson (R-SC), and Sarah McBride (D-DE) introduced H.R. 8794, the Fostering Effective Diagnosis and Treatment McBride (D-DE) introduced 8794, the Fostering Effective Diagnosis and Treatment for Underserved Populations (FED UP) with Bleeding Disorders Act of 2026. This for Underserved Populations (FED UP) with Bleeding Disorders Act of 2026. This bipartisan bill aims to address some of the many barriers faced by women and girls living bipartisan bill aims to address some of the barriers faced by women and girls living withbleedingdisorders. with bleeding disorders.
The FED UPwith Bleeding DisordersAct calls for an interagency review to identify
The FED UP with Bleeding Disorders calls for interagency review to identify federal programs, activities and strategic plans related to research, training and federal programs, activities and strategic plans related to research, training and education of providers, access to treatment and services, and inclusion in clinical education of providers, to treatment and services, and inclusion in clinical trialsallastheyrelatetowomenandgirlswithbleedingdisorders.Thereviewwould trials all as they relate to women and girls with bleeding disorders. The review would culminate in a report that would include the findings and identify recommendations. culminate in a report that would include the findings and identify recommendations. Lastly,thebill calls foranational public education andawareness campaign utilizing L tly, the bill calls for a national public education and awareness campaign utilizing thefindingsfromthereviewandreport. ndings from the review and report.
Federal Legislative Update
FFY 2027 Funding For Bleeding Disorders Programs
During Washington Days, advocates asked Congressional offices to support funding for federal bleeding disorders programs for Federal Fiscal Year 2027 by signing onto a Dear Colleague letter, originating with Rep. Julie Johnson of Texas. Fortyone 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 disordersprogramswillremainsteady.
CDCDirector,SurgeonGeneralNomineesRemain Unconfirmed
In April, President Trump nominated Dr Erica Schwartz, who served as deputy surgeon general in hisfirstadministration,toserveasthenextdirectorof 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 from the Uniformed Services University.
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 breastimagingatMemorialSloanKetteringandhasa medical degree from Ross University School of MedicineinBarbados.
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 previous picks for Surgeon General; He had previously nominated Dr. Casey Means for the post but pulled her nomination after questioning in the Senate provoked doubts about her experience and 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 vaccinatingnewbornsagainsthepatitisB.
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 raisedaboutheracademiccredentials.
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 aTrump-backed candidate, creating friction between Mr. Trump and Mr.Cassidy
Federal Legislative Update cont’d.
BDAJoinsColleaguesUrgingCMStoExemptOurMembersfrom WorkRequirements
The2025budgetreconciliationbill(H.R.1)signedintolawlastJulyintroducednewwork requirementsforcertainMedicaidenrollees.Theprovisionmakescontinuedeligibility contingentuponparticipationinworkorotherqualifiedactivities.H.R.1mandatesthat Medicaidmembersaged19-64whoarecoveredthroughACAMedicaidexpansionoran 115 demonstration waiver that provides minimum essential coverage must engage in employment,education,aworkprogramorcommunityservicetomaintaintheirMedicaid eligibility.TheserequirementstakeeffectJune1,2026.
TheBDAhassignedontoaletter–originatingwiththeHemophiliaAlliance,theNational Bleeding DisordersFoundation,theHemophiliaFederationofAmericaandtheCoalition forHemophiliaB-tourgeCMStorecognizebleedingdisordersasseriouscomplex medical conditions ensuring exemption from the new community engagement requirements.TheletterurgesthatCMSandstatesmustdefineexemptionsfor“seriousor complexmedicalconditionsaspertheSocialSecurityActandthatindividualswith bleedingdisordersshouldbecategoricallyexemptbasedondiagnosisortreatmentcodes.
Furthermore, as a bleeding disorder is a lifelong condition, individuals so diagnosed shouldonlyneedtoproveexemptiononce.Wefeelthatitisimportanttosafeguard coveragetopreventhealthdeterioration.
Unfortunately,onJune1,2026,theCentersforMedicareandMedicaidServices(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 theTrumpAdministration will be challengingforstatestoimplementandcouldleadtomorepeoplefallingthroughthe cracksandlosingcoverage.
Wewillcontinuetoadvocateforthisimportantexemptionforall ofourmembers.
State Legislative Update
CoverRareBill(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 specialistswhopracticedin.
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 theirestablishedin-networkrates.
Pre-AuthorizationReform
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 promotingbetterhealthoutcomes.
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 the2027session.
This year, Governor Hochul heard the concerns of advocates and included some prior authorization reformmeasuresinherbudgetbill.
Part HH of Title IV of the Fiscal Year 2026-2027
NYSBudgetBill:
Mandates that a prior authorization for a "designatedchroniccondition"remainsvalidfor atleastoneyear 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 oftreatment.
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 inastandardized,accessibleformat.
* These provisions were all adopted as part of the New York State Budget that was passed on May 28.
Board of Directors and Staff
President: President:
BruceWeiner Bruce Weiner
VicePresident: Vice President:
ChrisHuskie Chris Huskie
Treasurer: Treasurer:
CindiKeeler Cindi Keeler
Secretary: Secretary:
ChristinaMastrianni Christina Mastrianni
BoardMembers: Board Members:
MamaYaaBoakye Mama Yaa Boakye
CharityBorst Charity Borst
MaryAnneCechetto Mary Anne Cechetto
PatriciaMarck Patricia Marck
JessicaSmith Jessica Smith
ExecutiveDirector: Executive Director: BethMahr Beth Mahr
OperationsManager: Operations Manager: MichaelBudka Michael Budka
* Now Accepting Applications for Seats on the BDANENY Board of Directors
* Now Accepting Applications for Seats on the BDANENY Board of Directors
Qualifications Qualifications
Professional experience in business, government, philanthropy, or the nonprofit sector
Professional experience in business, government, philanthropy, or the nonprofit sector and/oramemberofthebleedingdisorderscommunity. and/or a member of the bleeding disorders community.
AcommitmenttoandunderstandingoftheBDANENYmission. A commitment to and understanding of the BDANENY mission.
Anabilitytobuildconsensusamongdiverseindividuals. An ability to build consensus among diverse individuals.
Personal qualities of integrity, sincerity, and a desire to improve the lives of individuals
Personal qualities of integrity, sincerity, and a desire to improve the lives of individuals andfamiliesimpactedbyaninheritedbleedingdisorder and families impacted by an inherited bleeding disorder.
Commitmentparameters
Commitment parameters
Term–Threeyears,renewable Term – Three years, renewable
Time – Board meetings every month; 10 remotely and 2 in-person; meetings typically – meetings every month; 10 remotely 2 in-person; meetings typically last90minutes last 90 minutes
SupportofBDANENYFundraisingEvents Support of BDANENY Fundraising Events
ly to join the Board, please visit www.bdaneny.org/BoardApplication to ly to join the Board, please visit www.bdaneny.org/BoardApplication to oad the application. oad the
www.bdaneny.org . .