The Vessel, September 2021 by BDANENY2

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

SEPTEMBER 2021

End of Summer Un-Wined Cornhole Tourney Raises Funds for BDANENY Despite the unpredictable weather on August 28, a good time was had by all who attended the “End of Summer Un-Wined” event at Galway Rock Winery. 16 teams competed in the cornhole tournament—some of which had clearly been practicing! The venue offered several varieties of their own wines, made right on the premises, as well as several different craft beers provided by local brewery Unified Beer Works in Malta. The winning team was rewarded for their efforts with a customized cornhole set created specifically for the BDA by our very own Chris Huskievery talented woodworker! A huge thank you goes out to the Chrzan family, who masterminded and orchestrated this fun event, from the beer to the brackets. And thank you to all who came out to support this fundraiser—and kick-off to our fall event season! Start practicing for 2022!

In This Issue

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“Bombadier Blood”

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BDANENY Upcoming Events

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Black & Blue Bash

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UNITE Walk

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Industry Update

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Annual Meeting

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Profile: Dr. Kate Halligan

From the Executive Director Stop and smell the roses...you’ve all heard that that’s the way you should live your life; living in the moment and being grateful for the small things. But it’s hard when the day-to-day grind and unexpected crises pop up out of nowhere. It’s especially hard when a medical emergency can happen in the blink of an eye. With the back to school frenzy many of you are now experiencing, it may not seem that there’s any time to take a breath, let alone enjoy it. The season marked by the autumnal equinox ushers in my favorite holiday of the year, Thanksgiving. What could be better than a day off surrounded by food and focused on gratitude? Some may argue that Thanksgiving represents the midpoint in a hectic seasonal rush toward Christmas, but in the spirit of the original Thanksgiving, let’s not take for granted the bounty

that the harvest season bring us. This fall we are fortunate to be able to get together once again—with proper COVID precautions of course! Join with friends old and new for these events:

UNITE Walk on 9/25 in Gavin Park Bombadier Blood on 10/14 in Glenmont Annual Meeting on 10/24 in Schaghticoke Black & Blue Bash on 11/6 in Saratoga Last year at this time, we were facing an uncertain future, still unable to meet together or hug our grandparents. This year, let’s be grateful for what we have and for how far we’ve come. And might I suggest that the best way to live in the moment is at a BDA event, surrounded by a warm and welcoming community.

Meet Dr. Kate Halligan

Albany Medical Center Hires New Pediatric Oncologist/Hematologist On August 30, AMC announced the appointment of Dr. Kate Halligan to the Melodies Center for Childhood Cancers and Blood Disorders at the Bernard & Millie Duker Children’s Hospital at Albany Medical Center. Most recently, Dr. Halligan completed her fellowship in pediatric hematology/oncology at UPMC Children’s Hospital of Pittsburgh. She completed her residency in pediatrics and graduated from Albany Medical College where she also earned a doctorate and master’s degree in cardiovascular science. Her undergraduate degree is from Siena College. She is a member of the Academy of Pediatric Oncology/Hematology, the American Academy of Hematology, the Children’s Oncology Group and the American Academy of Pediatrics. Above: with her husband and three sons at her 2015 graduation from Albany Medical College.

The Hemophilia Federation of America and the National Hemophilia Foundation are pleased to share a new partnership called the Together Project. Through the Together Project, HFA and NHF will work collaboratively on important issues that affect the bleeding disorders community. The first collaboration is focused on mental health and wellness in the bleeding disorders community, raising awareness, providing resources, and sharing coordinated messaging. 3

HFA Reconfigures Board of Directors In 2021, the Hemophilia Federation of America pledged to examine ways in which to reconfigure their Board of Directors to both meaningfully represent the bleeding disorders community, while at the same time streamlining operations. As of August 2020, HFA had 60 Board Members comprised of members representing each chapter, as well as an Executive Committee and several Members at Large. A Board this large makes it difficult for all Board Members to have any meaningful individual participation. Also, with a smaller Board, meetings tend to run more smoothly, and efficiently, because there is less to manage, coordinate and facilitate.

After several months of examination, planning and consulting with key stakeholders, HFA has winnowed down the size of their Board to 15 total members. The new Board structure will be comprised of ten Board Members elected through a defined candidate election process, four elected officer positions (President, Vice President, Secretary and Treasurer – elected by the Board), and one Member Organization Advisory Council member. Advisory and Board Committees remain the same. The Member Organization Advisory council was created to provide HFA with a connection to the community that is vitally important. The Member Organization Advisory Council will provide a direct channel for Member Organizations to hear updates, ask questions about programs and give feedback directly to HFA Board Members and staff. Each current organization gets a seat on the Advisory Council. There are no term limits for Council seats, since the Member Organization can appoint whomever they feel represents them best. The Council’s Leadership Team will consist of four to five Council Members and the Council Representative Board Member. Additionally, the Advisory Council will interview and score all Board of Directors candidates.

Industry Update Sigilon and SIG-001 Sigilon Therapeutics, a biotechnology company that seeks to develop functional cures for chronic diseases through its Shielded Living Therapeutics platform, has been notified by the Food and Drug Administration (FDA) that it’s Phase I/2 study of SIG-001 in patients with severe or moderately severe hemophilia A has been placed on hold. This hold was placed after the company reported a serious adverse event and temporarily halted program enrollment. Of the three patients dosed with SIG-001, one of them, who received the highest dose of the study drug, developed inhibitors to Factor VIII. Sigalon will continue to investigate the adverse event to determine whether or not the reaction is related to the therapy.

cation submission by the second quarter of 2022, followed by an expected six month review by the FDA. In Europe, BioMarin submitted a Marketing Authorization Application to the European Medicines Agency (EMA) on June 28 of this year. In May, the EMA granted the company’s request for accelerated assessment. Accelerated assessment reduces the time to review an application for final approval for human use. The EMA is expected to issue an opinion on human use in the first half of 2022.

FDA Examines Gene Therapy However, on September 6, the FDA suspended testing of BioMarin’s gene therapy for phenylketonuria, an inherited disorder that causes an amino acid to build The Shielded Living Therapeutics program develops up in the body. Researchers in the study found evinon-viral engineered cell-based therapies designed to dence that the virus delivering the corrected gene had fused itself into the genomes of six mice that later deproduce crucial proteins, enzymes or factors needed by patients living with chronic diseases such as hemo- veloped liver cancer. The tumors were found one year after treatment with a much higher dose than Bio Marphilia, diabetes and lysomal diseases. The celltherapies are encapsulated by bio-compatible spheres, in has been giving to humans in early-stage clinical intended to keep the immune system from seeing the testing. cell therapy inside as foreign, which could then trigger Liver cancer has emerged as a concern in various gene an immune response. The investigation into the adtherapy trials. Astellas Pharma’s gene therapy study verse event will explore all factors that could have for myotubular myopathy has seen the death of four lead to the development of inhibitors in the patient, volunteers, each showing signs of abnormal liver including family history and immune stimulation from function. a recent vaccination. Last year, a liver tumor was discovered in a hemophilBioMarin and Valrox ia patient receiving experimental treatment AMT-061 Sigilon is not alone in its quest for a better hemophilia from the biotech company uniQure, but a definitive A therapy. Valrox (valoctocogene roxaparvovec or link between the viruses that deliver gene therapy BMN 270) is a gene therapy being developed by Bio (AAVs) and tumor formation has never been estabMarin. The therapy is currently being tested in two lished in humans, and further investigation into the Phase 3 Clinical Trials. On July 21, BioMarin present- uniQure case has exonerated the treatment. Additioned a five-year update at the International Society on ally, a study following dogs given a hemophilia gene Thrombosis and Haemostasis Virtual Congress. therapy showed that although the AAV did fuse itself to the animals’ genome, it did not lead to cancer. NevIn the United States, BioMarin plans to submit twoyear safety and efficacy data on all study participants, ertheless, in a cautious response to the boom in gene with the goal of targeting a Biologics License Appli- therapy research, the FDA convened a meeting Sep6

Industry Update cont’d. tember 2 and 3 to address gene therapy safety. BioMarin’s announcement of the suspension suggests that the FDA is taking a cautious approach. At the FDA meeting, advisors made several recommendations including more testing in animals, more comprehensive screening of clinical trial participants, particularly for pre-existing liver conditions, and greater follow-up monitoring of treated patients. Advisors also highlighted the inconsistent methods and tools used to assess risk, making it harder to apply recommendations across programs. The FDA’s objective in convening this meeting was to “balance our enthusiasm for this field with caution.” FDA officials have indicated that they expect to be approving between 10 and 20 cell and gene therapies a year by 2025.

pletely restructure several of their manufacturing facilities. Their revamped manufacturing procedures are not expected to bring new product to the market until late in 2023. Facing such a shortage, several organizations including the Hemophilia Alliance, the Hemophilia Federation of America, the National Hemophilia Foundation and the World Federation of Hemophilia came together to explore possible solutions. Their efforts have resulted in an agreement with STAQ Pharma, Inc., an FDA-registered and FDA-inspected 503B outsourcing facility in Denver, CO. STAQ’s facility is able to produce the appropriate concentration necessary to be effectively used as a nasal spray.

CSL Behring and uniQure In May, uniQure closed a deal with CSL Behring to sell their experimental gene therapy for hemophilia B after receiving approval for the deal from regulators in three countries. UniQure received $450 million and could take home as much as $1.6 billion in additional payments contingent on reaching certain commercial and regulatory milestones. CSL Behring already markets several treatments for hemophilia B including Idelvion and Mononine.

Since the facility is based in Colorado, the Boards of Pharmacy in all 50 states must approve STAQ’s application to become a non-resident outsourcing facility with the permission to ship products into the state. Advocates have sent letters to all state boards to request an expedited review of these applications. We are hopeful that this effort will result in a treatment option for patients who currently have no therapeutic equivalent – without waiting until late in 2023. To date, 31 states have approved the biosimilar; 19 states have yet to approve licensing. Not all insurance plans, however, have approved reimbursement.

uniQure remains responsible for completing the ongoing Phase 3 trial, assessing the five-year safety and effectiveness of its AMT-061 therapy in 54 men with moderate to severe hemophilia B. The trial is expected to conclude in March 2025.

Earlier this year, this national coalition successfully petitioned the FDA to add demopressin acetate nasal spray to their 506E National Drug Shortage list. The Hemophilia Alliance’s Board agreed to pay the upfront development costs for STAQ’s product.

CSL Behring and Stimate As we previously reported, in July of 2020, Ferring Pharmaceuticals and its US distributor CSL Behring announced a voluntary recall of STIMATE nasal spray, used to control bleeding in patients with mild hemophilia A and von Willebrand disease. Upon investigation, they determined that they needed to com-

And earlier this month, CSL Behring announced that they will no longer distribute Stimate. Although Ferring has given no indication that they will discontinue the manufacturing, they will have to find another distribution partner before the product can be made available in the US.

Industry Update, cont.d Centessa and SerpinPC In new rebalancing drug development, Centessa Pharmaceuticals has announced that their new hemophilia drug SerpinPC reduced bleeding rates and spontaneous join bleeds in a midstage study. The drug reduced bleeding rates by 88% during the last 12 weeks of treatment at the highest dose, 70% at mid-dose, and 80% at the lowest dose, compared to bleeds the patients experienced prior to receiving the treatment. Spontaneous joint bleeds were reduced by 94% in the highest dose group, 69% in the mid-dose group and 76% at the lowest dose. The drug comes from the serpin family of proteins and is meant to boost production of thrombin by inhibiting activated protein C. Thrombin is an enzyme in blood plasma which causes the clotting of blood by converting fibrinogen to fibrin. SerpinPC has the potential to treat all types of hemophilia regardless of severity or inhibitor status and may be administered subcutaneously. Pfizer and BeneFIX On September 12, the FDA restored it’s approval of BeneFIX as a routine prophylactic treatment for bleeds in children and adolescents under 16 with hemophilia B. The Pfizer drug is a recombinant (lab-made) Factor IX and was originally given FDA approval in 1997 as an on-demand treatment to manage spontaneous bleeds and bleeding episodes during surgeries for adults and children with hemophilia B. Last year, the FDA expanded the use of BeneFIX to include routine prophylaxis, however, in February, the FDA rescinded its approval of prophylactic use. The agency explained that they had erred in their initial approval; the usage of BeneFIX as a preventative treatment in pediatric patients overlaps with the Orphan Drug exclusivity granted to Baxalta’s Rixubis in 2013. The Orphan Drug Act provides special status to products developed to treat medical conditions which, because they are so rare, would not be profitable to produce

BDANENY Board of Directors and Staff President……………………….. Bruce Weiner Treasurer…………………………. Cindi Keeler Board Member…………………. Bill Brownell Board Member………………….. Dave Huskie

Board Member………... Christina Mastrianni Board Member………. Juliemar Abreu Boria Executive Director………………. Beth Mahar Operations Manager……... Patricia Bengraff Administrative Assistant…….. Melissa Deeb