BDANENY Summer 2022 - The Vessel

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

BDA COMMUNITY EVENTS ~ RE-ENGAGING, RE-CONNECTING The first few months of 2022 provided multiple opportunities to get together, have some fun and even raise some funds. In February, we attended the Adirondack Thunder hockey game courtesy of Bayer and Cottrill’s; in April, we learned how to make dumplings at Different Drummer’s Kitchen, courtesy of Novo Nordisk and BioMatrix; Also in April, Sanofi Genzyme treated us to dinner at 677 Prime as part of their One Family program to recognize the world-wide challenges of hemophilia. Our final event in April was an epic Pub Crawl. We visited McAddy’s, Rare Form, Footsie Magoo’s and ended up underground in Franklin Alley. Proceeds from this event support the activities of the BDA. Lastly, we held a Family Game Night at Ted’s Fish Fry on Western Avenue where Bio Marin educated us the basics of gene therapy.

As you will see in the pictures (page 6 and page 7) a good time was had by all. And we want you to join in the fun! Watch our website for upcoming events and plan to join us for the Cornhole Tournament, the Annual Meeting and the UNITE Walk.

In This Issue

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Cornhole Tournament

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UNITE Walk

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Industry Update

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Annual Meeting

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Job Skills Training Grant

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Legislative Recap

SUMMER 2022

From the Executive Director I realize that this may be a tad hypocritical, coming from someone whose favorite activity is “running my mouth,” but this time of year really inspires me to get of off my couch and push those underutilized muscles. Physical activity has long been known to reduce depression and anxiety and is commonly prescribed to prevent or cure negative mental health conditions. Living here in upstate New York, it’s hard to get motivated when the temperature is in the single digits, but now that summer is here, there are no excuses – and the payoff is tremendous!

was born that I figured out how to get exercise in a way that was enjoyable.

It took me a really long time to find a form of physical activity I liked. It didn’t help matters that my Irish-twin sister Kathy has varsity letters in soccer and track. (She got all my dad’s genes.) I could never compete at her level, no matter the sport, so I just took my book and retreated to the corner. It wasn’t until my son

Don’t believe me? Here are 15 ways exercise can benefit you: 1. Increased dopamine: Dopamine is a chemical in the brain that’s associated with feelings of pleasure and happiness and exercise is one of the best ways to increase your brain’s production of dopamine.

For me, the key realization was that I needed to find something that was fun and not competitive. Don’t get me wrong – I will bury you in a trivia game- but that’s my strong suit. Athletic prowess is not my strong suit and any serious competition just highlights my inferiority. That makes me cranky. And less likely to participate. But when I started to take my son skiing, or teaching him to swim, it didn’t matter that I couldn’t ski black diamonds or do a Researchers studying exercise have consistently found that perfect butterfly crawl. I found joy in the process of teachit has a positive impact on mood. It has been proven that ing someone who I knew would surpass my abilities before physical activity stimulates the release of “feel-good” he hit puberty. I was engaging in the activity for a purpose chemicals in the brain, called endorphins. Some researchers other than to win or to be the best. In fact, I have discovargue that exercise acts as a diversion from negative ered that solo activities are better suited for me. If my comthoughts while others argue that exercise improves mood petitive spirit raises its ugly head, I compete for personal by virtue of the personal growth and goal attainment that best, not against another. results from efforts to master a physical skill. Furthermore, Although I hate competing, that doesn’t mean I hate comresearch evidence indicates that the social interaction inpany. In fact, I find that scheduling an activity with a friend volved in certain kinds of exercise (such as team sports) contributes to personal satisfaction and consequently, mood makes me more likely to show up and less likely to hit the snooze button. Eileen and I walk 2 miles every Tuesday enhancement. morning. It’s a great chance to catch up, enjoy some fresh I am not athletic. My eye-hand coordination is only present air and blow off some steam about our families. I’m proud when shopping on Amazon. I have weak ankles and mild to report that there have been only two mornings, where we asthma. My dad was a tremendously gifted athlete who have decided it was too cold to walk and grabbed breakfast played golf against Arnold Palmer and took up skiing in his at the diner instead. Some days it’s tough to get out there – forties; I take after my mom. But I value my health. I want especially in January – but once we’ve finished our walk, I to take care of my body and make it last as long as possifind that I am energized to face the day and have a sense of ble. accomplishment to boot.

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2. Lowers anxiety and depression: Studies have shown that people who are suffering from anxiety and depression benefit from the increased dopamine production that exercise enables.

3. Energy booster: Exercise delivers oxygen and nutrients to your tissues and helps your cardiovascular system work more efficiently, and when your heart and lung health imCont. Pg 10

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Sponsored by:

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Supreme Court Supreme Court Rules Against Cuts to 340B Medicare Funding at Hospitals .

In a unanimous decision, the Supreme Court ruled that the Department of Health and Human Services (HHS) was wrong to cut billions of dollars annually from the federal 340B Drug Pricing Program.

The 340B program helps fund hospitals and clinics that serve vulnerable communities. The safety-net program reimburses the costs of outpatient drugs at a much higher price than hospitals pay; the resultant surplus covers the cost of serving uninsured and underinsured individuals.

As the justices noted in their ruling, HHS erred by not first surveying the hospitals to inquire about costs before making payment cuts. HHS had argued that such surveys weren’t required. The Supreme Court disagreed.

The lawsuit was brought by the American Hospital Association (AHA), which sued after HHS slashed $1.6 billion from the reimbursement program in 2018—a 30% cut.

A federal district court sided with AHA, but an appeals court reversed the decision, sending the case to the Supreme Court.

A joint statement from AHA, the Association of American Medical Colleges and America’s Essential Hospitals read. “340B discounts help hospitals devote more resources to services and programs for vulnerable communities and increase access to prescription drugs for low-income patients.”

The 340B reimbursement program has remained a controversial but vital source of funding for health care services.

Several drugmakers have tried to restrict pharmacies and health care providers from accessing 340B program discounts. A similar scenario regarding the cost of HIV prevention meds is currently unfolding.

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Industry Update BioMarin The regulatory agency that recommends whether new drugs should be approved in Europe has thrown its support behind a gene therapy for the rare bleeding disorder hemophilia A, putting the potentially curative treatment one step closer to market. If approved by the European Commission, the therapy, known as Roctavian, would be the first commercially available genetic medicine for hemophilia A. Developed by BioMarin, a California-based biotechnology company, Roctavian uses an engineered virus to shuttle the gene for making Factor VIII into liver cells, where it can then produce the vital protein. Data released early this year — from the largest ever study of a hemophilia gene therapy — show that patients with severe hemophilia A who were treated with Roctavian experienced few, if any, bleeds over a two-year study period.

While Factor VIII levels spiked in the initial months after Roctavian treatment, the data collected thus far suggests they decline over time. BioMarin executives have maintained that the treatment should still offer at least five years of bleeding control. On a broader level, though, drug regulators have raised concerns about how long gene therapies can remain effective in the body.

BioMarin expects a final verdict from the European Commission sometime between July and the end of September. In the US, the company had planned to resubmit its Roctavian application to the FDA by the end of June, but now expects to file in September due to “additional information” the FDA in May had asked to be included.

Partnering with Believe Ltd. BioMarin is partnering with Believe Limited and nonprofit public health group The Foundation for Art & Healing to create a new documentary about hemophilia. The partners will set up a combination of hybrid virtual and inperson workshops and productions from June to October with the aim to “provide powerful education on the healing and therapeutic power of the arts and self-expression,” according to a statement.

Ten adults with hemophilia across the U.S. will be picked to participate and tell their stories of “the challenges and victories” of living with a bleeding disorder in a documentary film. They will also first learn about the filmmaking process during virtual workshops on topics such as writing, prop selection, set decoration, directing and edit8

ing. Then, the Believe Limited production team will travel to the hometowns of participants to help bring their stories to life on camera.

The documentary film will premiere during an as-yetunspecified fall event.

Documentary and films are becoming more commonplace in pharma marketing: Alnylam, Pfizer/BioNTech and the Merck Foundation have all in the past year turned to this medium, though in different ways, to boost awareness around particular health issues and in April, the Merck Foundation, the philanthropic arm of Merck KGaA, also launched a new TV program in Africa, helmed by the Foundation’s CEO, as a talk show about major public health issues across the continent. .

Sanofi Sanofi and Sobi collaborated on the development and commercialization of efanesoctocog alfa (BIVV001) and announced on June 3 that the drug received Breakthrough Therapy designation from the US Food and Drug Administration (FDA) for treating hemophilia A. The Breakthrough therapy designation serves to expedite the development and review process for certain drugs in the US. Because hemophilia A is a rare, life-threatening bleeding disorder, the therapy was a good candidate. In order to attain the designation though, preliminary clinical evidence demonstrating improvement on significant endpoints needs to be compared with available therapies.

Efanesoctocog alfa is the first factor VIII therapy to be granted Breakthrough Therapy designation by the FDA. The novel, investigational factor VIII therapy was designed to be a once-weekly prophylactic treatment to normalize factor activity levels in patients for the majority of a week.

Data from the XTEND-1 phase 3 study supported the designation. The study showed clinically meaningful prevention of bleeds in patients over a 52-week period. It also demonstrated that prevention provided by efanesoctocog alfa was superior to prior prophylactic factor VIII replacement therapy based on an intra-patient comparison.

In August 2017 the FDA granted efanesoctocog alfa Orphan Drug designation and then Fast Track designation in February 2021. In June 2019 the treatment was granted Orphan Drug designation by the European Commission.

Regulatory submission in the EU will follow availability of

Industry Update cont’d. data from the ongoing XTEND-Kids pediatric study, expected in 2023. Sanofi expects to submit an application to the FDA sometime this year.

Pfizer The Food and Drug Administration has cleared Pfizer to resume dosing patients with an experimental hemophilia gene therapy that was put on hold last year because of the risk of dangerous blood clots, the company announced on May 3. Worries over thromboses were triggered by findings that patients’ levels of clotting factor rose to 150% of normal after an infusion of the gene therapy. The drugmaker, which is developing the gene therapy with Sangamo Therapeutics, said it hopes to begin treating patients again before the end of September.

With the clearance to restart the hemophilia A trial, Pfizer has now two candidates in its gene therapy research pipeline. In April, the company announced the FDA approved resumption of a trial of its experimental treatment for Duchenne muscular dystrophy, which had been stopped because of side effects and the death of a patient.

In restarting the muscular dystrophy trial, Pfizer said patients will now need to stay in a hospital for a week following their infusion to monitor for any dangerous side effects and treat them if necessary.

Takeda Takeda has been working with the molecular engineering company Evozyne to develop new protein sequences that can be incorporated into the next generation of gene therapy for rare diseases. Evozyne will use its advanced computational and machine learning methods to discover novel protein sequences for gene therapies. Once the research is complete, Takeda will have a licensing option to develop and commercialize the sequences through the Japanese pharma’s gene therapy program.

This is not Takeda’s first venture into gene therapy. It signed a $2 billion pact with Code Biotherapeutics for four programs in February; a $1.12 billion deal with Selecta Biosciences covering gene therapies for lysosomal storage disorders; and a research collaboration with Poseida Therapeutics in October 2021 worth a potential $3.6 billion and a $303 million pact with Genevant in August 2021.

ASC Therapeutics.

ASC Therapeutics has joined forces with Charles River to scale the manufacturing of ASC618, its second-generation virus-based gene therapy for hemophilia A. The agreement expands upon their current collaboration, begun in 2019 that focused on achieving Good Manufacturing Practice (GMP)-virus manufacturing and establishing processes for adeno-associated virus (AAV) production and purification. GMPs are standards to ensure batches of a medicine are produced with consistently high quality and one of the requirements for therapies to be considered for market approval.

The deal comes nearly a year after ASC Therapeutics received a green light from the U.S. Food and Drug Administration (FDA) to launch the first-in-human clinical trial of ASC618. The study (NCT04676048) is expected to assess the one-time gene therapy’s safety and preliminary effectiveness in up to 12 adults with moderate to severe hemophilia A. Charles River has expanded its development and manufacturing capacities through a string of recent acquisitions.

By acquiring Cognate BioServices, Cobra Biologics, and Vigene Biosciences, its CDMO platforms now cover cell therapy, viral vectors, and small circular DNA molecule production.

The gene therapy was developed based on work by researchers at Emory University and optimized through a partnership between ASC Therapeutics and Expression Therapeutics. In 2020, the company began collaborating with Vigene Biosciences, now part of Charles River, to support the manufacturing process of its current and future gene therapy clinical programs.

ASC618 received orphan drug designation in both the U.S. and Europe and fast track designations in the U.S. for hemophilia A. These designations are expected to accelerate the therapy’s clinical development and regulatory review.

ASC Therapeutics, a division of Applied StemCell, is also developing potential gene editing therapies for hemophilia A (ASC518) and hemophilia B (ASC519).

Freeline Freeline Therapeutics, a UK-based clinical-stage biotechnology company focused on developing gene therapies for people with inherited diseases, has begun dosing a second 9

Executive Director, cont. prove, you have more energy to tackle daily chores.

4. Stress reducer: Exercise itself is a low-level form of stress, and the more you subject yourself to the “stress” of physical activity, the better your body will adapt to handling other types of stress.

5. Slows cognitive decline: Many experts believe that maintaining physical fitness can help keep blood flowing normally to brain tissue, which can reduce the risk of damage or deterioration.

6. Builds self-confidence: Exercise boosts your self-confidence by seeing your body transforming for the better.

7. Get better sleep: Exercise has been shown to improve sleep quality by helping you fall asleep faster and by decreasing daytime sleepiness. For some people, this reduces the need for sleep medication.

8. Improves creativity: A study in the journal Frontiers in Human Neuroscience shows that regular exercisers do better on tests of creativity than their more sedentary peers.

9. Improves productivity: Exercise gives you more energy and stops that afternoon fatigue slump thereby fighting that dreaded “brain fog” and facilitating a positive momentum.

10. Boost your memory: Many studies have suggested that the parts of the brain that control thinking and memory are larger in volume in people who exercise than in people who don't.

11. Be more relaxed: Exercise reduces stress hormones and stimulates production of endorphins, which together help foster relaxation.

12. Improve your coping skills: The release of endorphins combined with the reduction of stress hormones helps you feel calm and in control of your life, thanks to exercise.

13. Get your addictions under control: Exercise has been investigated as a treatment for reducing the risk of relapse to addictive behavior and has been shown to reduce drug (including alcohol and nicotine) cravings and improve treatment outcomes.

14. Increase your brain power: It’s been shown that getting in some good cardio can help create new brain cells, improving your overall brain performance.

15. Enjoy the great outdoors: Outdoor exercise can help ward off seasonal affective disorder, depression and anxiety because sunshine naturally increases serotonin, a hormone that affects your mood.

So take advantage of the mild temperatures of summer to make some good exercise habits that carry into the cooler months.

Your body, your family, and even your employer will thank you!

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Industry Update, cont. group of hemophilia B patients enrolled in a trial of FLT180a experimental gene therapy treatment. The Phase 1/2 B-LIEVE trial (NCT05164471) is evaluating FLT180a in up to nine men with severe or moderately severe hemophilia B, meaning FIX activity levels of 2% or lower.

The trial launched earlier this year at a single site in California, with the first patient dosed in March.

Patients in a previous Phase 1/2 trial called B-AMAZE (NCT03369444) are being followed to assess the gene therapy’s long-term safety and effectiveness in an ongoing study (NCT03641703) expected to run through December 2035. The company announced last year that factor IX levels within the normal range had been achieved with relatively low doses of FLT180a and maintained for more than three years.

Bayer Bayer has announced that they are discontinuing the recombinant factor VIII product Kogenate® FS. First ap-

proved by the U.S. Food and Drug Administration in 1993, the therapy has carried multiple indications for children and adults with hemophilia A. The company cited the increasing shift in patient usage towards newer extended half-life products as a primary consideration in the decision. The emergence of novel non-factor hemophilia therapies in recent years may also be contributing to this trend away from standard half-life therapies.

The company anticipates a potential depletion of the larger vial sizes of Kogenate® FS during the fall of 2022, with supplies of the smaller vial sizes likely to be available into 2023.

Bayer has set up a hotline (1.833.40.BAYER) to field questions specific to the discontinuation. The line will be available Monday through Friday from 8:30am-8:00pm ET. More information can also be found on a company webpage.

Job Skills Training Grant Program How can you stay ahead of inflation? By getting a raise!

Could you use $1,000 to gain a skill or certification to help you get a job? HFA is offering adults, ages 18-64 the opportunity to apply for a $1,000 training grant to cover costs associated with acquiring technical skills or certification for career advancement. Examples include: project management, dog grooming, customer service, medical billing, web design, phlebotomy, real estate sales, SCRUM methodology, and more.

The applicant must be a person with a bleeding disorder between the ages of 18 to 64. Carriers are eligible. Past recipients of a Job Readiness grant cannot apply. Applicants cannot be HFA board members, HFA staff, or immediate family members of the board or staff. Funds must be used for technical skills courses or certifications.

Applications must include: a brief statement of need; proof of a bleeding disorder; resume; and documentation of course cost. A letter of recommendation and completion of HFA’s Learning Central’s Career Planning course are both optional, but recommended.

The deadline to apply is August 5, 2022. Access the application and further information here.

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State Legislative Update The State Legislature operates on a two-year cycle. Bills that were introduced in2021 were still considered active through the 2022 session, which ended on June 22. Here is a review of the major legislative developments of the cycle and a look towards what we can expect in 2023.

A. 1741-A/S.5299-A Patient Assistance/Accumulator Bill This bill requires New York State Marketplace plans to count third party assistance payments toward a patient’s out of pocket cost obligations.

Status: This bill passed both the Senate and Assembly and will be forwarded to the Governor. She has until the end of the year call it up and to sign it. We are working with a coalition of 70+ groups to advocate for her signature.

A.5854-A/S.3566 Prescription Access Reform This bill bars health plans and Pharmacy Benefit Managers (PBM) from imposing additional terms and conditions on already in-network local pharmacies to preclude them from serving patients.

Status: This bill was signed by Governor Hochul on December 21 and took effect immediately.

A.7129/S.6435-B Prior Authorization Reform This bill creates common sense guidelines for the use of prior authorization measures by health plans and PBMs. The guidelines are designed to allow for prior authorization to be used in a way that doesn’t delay treatment for patients or add unnecessarily to the burden on medical staff.

Status: This bill never advanced to a vote in either house. It would need to be introduced again in January 2023. Several influential members of the Assembly have their own versions of slightly different bills that they believe would achieve similar results. None of these bills advanced during the past session and will need to be reintroduced as well.

New York State Rare Disease Council The Council will be charged with a two-year review of the economic burdens of the treatment of rare diseases, with regard to quality of care, health care coverage, and access to treatment and services. The council will develop recommendations and identify best practices in the treatment of rare diseases.

Status: The Council was established by an amendment to the Public Health Law in 2019. The Department of Health is currently selecting members for the council and anticipates convening the group this year.

Medicaid 1 in 3 New Yorkers receives coverage through Medicaid, including many people with bleeding disorders. While the 2022-23 state budget saw some improvements in Medicaid, including increased eligibility for older and disabled people and modest reimbursement increases for providers, future budgets are likely to be more constrained. Two key Medicaid developments to watch closely are: a. DOH is expected to make health plans re-apply to be Medicaid plans, meaning there may soon be new and fewer Medicaid plans available. No timeline for finalizing this process has been announced. b. Medicaid is working to create uniform formulary and utilization management rules across all managed care and feefor-service programs. No word has been given on what the new rules will look like.

PBM Regulation Last December, Governor Hochul signed a law instituting first-ever state registration and reporting requirements for PBMs operating in New York State. PBMs handle medication benefits for 266 million Americans and are often responsible for limiting access to medications for people with bleeding disorders. PBMs typically claim exemption from state regulation under the federal ERISA law. However, the 2020 Supreme Court Rutledge Decision opened the door to state regulation of ERISA plans, and many states are beginning to impose regulations on PBMs. 12

State Legislative Update, contd.

What Issue Areas Bear Watching?

One-Stop Shopping for Healthcare CVS recently launched a virtual care platform providing access to primary, on-demand, chronic, and mental health care services. CVS already provides direct care services at selected pharmacy locations. In addition, CVS operates one of America’s largest: health plans (Aetna), PBM (Caremark), mail-order pharmacy (CVS Caremark), and local pharmacy chain (CVS Pharmacy). With the new online platform, the CVS conglomerate can control a patient’s healthcare from end-to-end. As well, Wal-Mart continues to ramp up their PBG health plan association and EmsanaRX PBM. As more companies look to save or make money in the healthcare space expect more mega-mergers and creative company design. What effect these developments will have on patient access, quality of care or costs is unknown.

ICER The Institute for Clinical and Economic Research is a private, non-profit think-tank funded by an array of donors which assesses the effectiveness of a medication versus the improvement it brings to a patient’s quality of life and determines an “ideal” price for the medication based on that assessment. ICER recommendations are used by a variety of payers including New York State Medicaid to make coverage and reimbursement decisions. ICER recently announced plans to assess several new bleeding disorders medications including gene therapy, and their recommendations could have a significant impact on patient access to these new medications/therapies.

Telehealth During the pandemic, telehealth was seen as a care delivery option with many advantages. Questions over when and how much to pay for telehealth however are leading many payers and health plans to dial back on telehealth. New York State is examining ways to promote telehealth as an important way to help those who lack access to providers or have difficulty safely travelling to provider locations.

Elected Officials Gottfried Retires Assembly Health Committee Chair Richard Gottfried is retiring after a record 52 years of service in the legislature.

During his long tenure, Assemblyman Gottfried has done more to shape healthcare policy in New York State than any other state legislator. For example, he led creation of the NYS Child Health Plus program, the first CHP program in the nation. A new Assembly Health Chair won’t be announced until at least November.

Redistricting The redrawing of NYS legislative districts is making many state races more competitive this election cycle, leading several incumbent legislators to step aside, or run for different seats. Turnover in the legislature has become more of a norm the past few years. This year, the combination of redistricting and a Congressional mid-term will make that turnover less predictable.

Governor’s Race Governor Hochul is expected to win election to a full term, though her soft approval poll numbers mean she’s likely to pursue a more moderate agenda. The Democrats are expected to retain majority control of the Senate and Assembly, though their majorities will likely be split on moderate/progressive lines. Look for the legislature to continue taking back power they lost under Gov. Cuomo.

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The Coalition for Hemophilia B is now accepting applications for their Men’s Fall Retreat. Learn more about the program and apply to join here.

Contact Us BDANENY Office 333 Broadway Suite 210 Troy, NY 12180 (518) 729-3577 BDANENY.org memberservices@bdaneny.org

BDANENY Board of Directors and Staff President…………………….…………..…Bruce Weiner Vice President…………………….………..Chris Huskie Treasurer………………………………….....Cindi Keeler Secretary……..…….………….…..Christina Mastrianni Board Member…………………..Juliemar Abreu Boria Board Member………..……...…....…Matthew Delaney

Board Member……………………...…....…Dave Huskie Board Member……………..……………..Jessica Smith Executive Director……………..…….….…..Beth Mahar Operations Manager………………....Patricia Bengraff Special Events & Social Media…..…......Melissa Deeb