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Webinar: “Progress in Amyloidosis: 2022”

Webinar: “Progress in Amyloidosis: 2022”

July 2022 Webinar Event

View the recorded webinar below

View Webinar Here

Dr. Zonder will describe recent advances in the management of AL amyloidosis; review approved therapies for ATTR amyloidosis and how they work, and discuss new classes of agents designed to help remove existing amyloid deposits.

Jeffrey Zonder, MD, is a Professor in the Departments of Oncology at the Barbara Ann Karmanos Cancer Institute (KCI) and Wayne State University School of Medicine. He is the Leader of the KCI Multiple Myeloma and Amyloidosis Multidisciplinary Team and Co-Leader of the Molecular Therapeutics Program. He is a member of the Steering Committee of the Multiple Myeloma Research Consortium and a medical advisor for the Amyloidosis Support Groups Networks. Dr. Zonder is a member of the International Myeloma Working Group, the International Myeloma Society, and the International Society of Amyloidosis.  He is a member of the Southwest Oncology Group’s (SWOG) Barlogie-Salmon Myeloma Committee. Dr. Zonder has authored or co-authored numerous original research papers, review articles, book chapters, and research abstracts on myeloma and amyloidosis.

He completed a fellowship in hematology-oncology at Wayne State University and his medical residency at the University of Rochester, New York.  He received his medical degree from Wayne State University School of Medicine.

Sponsored by:


Intellia and Regeneron Present Updated Interim Data from Phase 1 Study of CRISPR-based NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis Demonstrating that Deep Serum TTR Reductions Remained Durable After a Single Dose

Intellia and Regeneron Present Updated Interim Data from Phase 1 Study of CRISPR-based NTLA-2001 for the Treatment of Transthyretin (ATTR) Amyloidosis Demonstrating that Deep Serum TTR Reductions Remained Durable After a Single Dose

June 24, 2022 7:30 AM EDT

CAMBRIDGE, Mass. and TARRYTOWN, N.Y., June 24, 2022 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) today announced additional positive interim data from an ongoing Phase 1 study of their lead investigational in vivo genome editing candidate, NTLA-2001, which is being developed as a single-dose treatment for transthyretin (ATTR) amyloidosis. The data were presented in an oral presentation at the European Association for the Study of the Liver (EASL) International Liver Congress™ 2022, taking place June 22 – 26 in London.

Read article here


AFFIRM-AL Study Protocol Amendment

AFFIRM-AL Study Protocol Amendment

June 23, 2022

Prothena has recently implemented an amendment for the AFFIRM-AL Phase 3 study of birtamimab in Mayo Stage IV AL (light chain) amyloidosis which is currently recruiting patients. Birtamimab is an investigational monoclonal antibody designed to target and clear the abnormal protein (amyloid) that can accumulate and cause organ failure in patients with AL amyloidosis. 

The amendment has expanded the number of sites globally from 80 to 120 in over 20 countries and will also allow the use of daratumumab as part of standard of care treatment if initiated at randomization to align with emerging changes to standard of care.

About AFFIRM-AL:

  • The AFFIRM-AL Study is a global, randomized, phase 3, efficacy and safety study that will compare birtamimab plus standard of care to placebo plus standard of care by assessing time to all-cause mortality.
  • The goal of this clinical research study is to evaluate an investigational drug called birtamimab to find out if it works and if it is safe when given with chemotherapy to people with AL amyloidosis who are in Mayo Stage IV.  The study will enroll approximately 150 patients.
  • Patients who may be eligible are 18 years of age or older, newly diagnosed with Mayo Stage IV AL amyloidosis, and have not yet received any medical treatment for AL amyloidosis.
  • Patients will receive birtamimab or placebo by intravenous infusion monthly and will also receive concurrent standard of care therapy consisting of a first line regimen that contains a drug called bortezomib.

For more information about the AFFIRM-AL study, please visit the AFFIRM-AL website (https://affirm-al.com) or Clinicaltrials.gov (https://clinicaltrials.gov/ct2/show/NCT04973137)


Eplontersen met co-primary and secondary endpoints in interim analysis of the NEURO-TTRansform Phase III trial for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)

Eplontersen met co-primary and secondary endpoints in interim analysis of the NEURO-TTRansform Phase III trial for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN)

PUBLISHED 21 June 2022

New Drug Application filing anticipated based on positive data from interim analysis

Positive high-level results from the NEURO-TTRansform Phase III trial in patients with hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN) showed AstraZeneca and Ionis’ eplontersen met its co-primary endpoints in a planned interim analysis at 35 weeks. In the trial, eplontersen reached a statistically significant and clinically meaningful change from baseline for its co-primary endpoint of percent change in serum transthyretin (TTR) concentration, reducing TTR protein production. Eplontersen also reached its co-primary endpoint of change from baseline in the modified Neuropathy Impairment Score +7 (mNIS+7), a measure of neuropathic disease progression1, versus external placebo group.

High-level results showed the trial also met its secondary endpoint of change from baseline in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) showing treatment with eplontersen significantly improved patient-reported quality of life versus external placebo group. In the trial, eplontersen demonstrated a favorable safety and tolerability profile with no specific concerns.

ATTRv-PN is a debilitating disease that leads to peripheral nerve damage with motor disability within five years of diagnosis and, without treatment, is generally fatal within a decade3. Eplontersen, formerly known as IONIS-TTR-LRx, is a ligand-conjugated antisense (LICA) investigational medicine designed to reduce the production of TTR protein at its source to treat both hereditary and non-hereditary forms of ATTR2,4-6.

Read the Full Article Here


Webinar: “Progress in Amyloidosis: 2022”

Webinar: “Progress in Amyloidosis: 2022”

July 2022 Live Webinar Event

Please join us for our webinar event presented by Dr. Jeffrey Zonder

on

July 13, 2022, at 2:30 pm ET

Register Here

Dr. Zonder will describe recent advances in the management of AL amyloidosis; review approved therapies for ATTR amyloidosis and how they work, and discuss new classes of agents designed to help remove existing amyloid deposits.

Jeffrey Zonder, MD, is a Professor in the Departments of Oncology at the Barbara Ann Karmanos Cancer Institute (KCI) and Wayne State University School of Medicine. He is the Leader of the KCI Multiple Myeloma and Amyloidosis Multidisciplinary Team and Co-Leader of the Molecular Therapeutics Program. He is a member of the Steering Committee of the Multiple Myeloma Research Consortium and a medical advisor for the Amyloidosis Support Groups Networks. Dr. Zonder is a member of the International Myeloma Working Group, the International Myeloma Society, and the International Society of Amyloidosis.  He is a member of the Southwest Oncology Group’s (SWOG) Barlogie-Salmon Myeloma Committee. Dr. Zonder has authored or co-authored numerous original research papers, review articles, book chapters, and research abstracts on myeloma and amyloidosis.

He completed a fellowship in hematology-oncology at Wayne State University and his medical residency at the University of Rochester, New York.  He received his medical degree from Wayne State University School of Medicine.

Sponsored by:


Alnylam Announces FDA Approval of AMVUTTRA™ (vutrisiran), an RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

Alnylam Announces FDA Approval of AMVUTTRA™ (vutrisiran), an RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

Jun 13, 2022

CAMBRIDGE, Mass.–(BUSINESS WIRE)–Jun. 13, 2022– Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, today announced that the U.S. Food and Drug Administration (FDA) approved AMVUTTRA™ (vutrisiran), an RNAi therapeutic administered via subcutaneous injection once every three months (quarterly) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. hATTR amyloidosis is a rare, inherited, rapidly progressive, and fatal disease with debilitating polyneuropathy manifestations, for which there are few treatment options. The FDA approval is based on positive 9-month results from the HELIOS-A Phase 3 study, where AMVUTTRA significantly improved the signs and symptoms of polyneuropathy, with more than 50 percent of patients experiencing halting or reversal of their disease manifestations.

View the full release here: https://www.businesswire.com/news/home/20220603005487/en/


As we look toward the future

As we look toward the future

In May of 2012, my father received a diagnosis that no one saw coming: Heavy Chain Amyloidosis. He had been sick for years, decades even. He had atherosclerotic plaques throughout his heart and carotid arteries and suffered GI symptoms that would cause severe bleeding and nausea. I remember my father being sick for most of my life, but despite his failing systems, he fought every minute to work hard to provide for his family and to spend more time with those he loved. 

Before he started chemotherapy, my dad had his kidney biopsy sample sent to Mayo Clinic. Our lives changed forever in July 2012 when we found out that my dad had an ultra-rare form of hereditary Amyloidosis called Fibrinogen A Alpha Chain Amyloidosis. Fifty-percent chance of passing the gene to his three daughters. Chemotherapy was not an option for treatment, and Dad was told he was not a candidate for an organ transplant. Instead, he would go through the years of his diagnosis doing supportive therapies. Peritoneal dialysis. Albumin infusions. Arthrectomies. Stents. Cardiac catheterizations. Hemodialysis. We balanced our lives with caregiving, considering our own future health, and trying to live normal lives–something my dad wanted for my mom, my sisters, and myself. We continued this cadence until my father passed away on November 26, 2018, of a heart attack. He was a huge presence in our family, and he continues to be dearly missed by so many.

My dad always denied that his disease was hereditary. The thought of passing down a rare genetic mutation was devastating for my dad, so he somehow managed to interpret his results in a way that made them “inconclusive.” We all dared not to argue with this, as we knew that believing the truth would devastate him to a point where he would not want to live. He ended up enjoying time with family and friends between dialysis and infusions. He and my mother would form close relationships with their treatment teams. They became family, and I credit them all for giving our dad six more years of life after being diagnosed with a disease that no one understood.

I would wait until 2021 to begin the process of genetic testing for the FGA gene, the gene that causes Fibrinogen A Alpha Chain Amyloidosis (AFib). July 2021, eleven years after my father found out he had the gene, I found out I tested positive for the mutation as well. Almost simultaneously, my aunt (my dad’s sister) had a transient ischemic attack and started going into kidney failure. She, too, tested positive for the FGA gene. I have since been evaluated at Boston Medical Center’s Amyloidosis Center, and I am healthy for now with no sign of the disease. 

As I look to the future for myself and my son, I see so many possibilities for improved treatment and cures for genetic diseases. I am encouraged by the research and drugs being produced for ATTR and gene therapy. My family and I are actively working on a fundraiser for the Amyloidosis Foundation for November 26, 2022, in memory of an incredible husband, father, brother, cousin, and friend: Glenn Dale Gallaway. We will continue to advocate, spread awareness, and support those who feel like they are not understood because of the rarity of their disease. Just because we are rare, does not mean that we are alone.

Please learn more about our family story on Instagram, Facebook, or Twitter @FibrinogenAF   

Written by:  Mandy Gallaway Lacey, daughter of Glenn Dale Gallaway (1948-2018)



3rd Annual AF Virtual Run/Walk/Roll

3rd Annual AF Virtual Run/Walk/Roll

** EXTENDED ** Now through August 31st!!

WHAT IS A VIRTUAL RUN?

A virtual run is a run that can be run (walked or rolled) from any location you choose. You can run, jog, walk or bike on the road, on the trail, on the treadmill, at the gym, or on the track. You get to run your own race, at your own pace, and time it yourself. 

Sign Up for the “Run for Your Life” Virtual Run

Registration for the Virtual Run is currently open.

  • 5K Run – $25.00
  • 10K Run – $35.00

Register Here

Or call the office to register at 248-922-9610.

Once you have signed up for the run, either a 5K or 10K, then decide on the course you want to do, or you can do it on the treadmill. You time yourself. You can even get your friends and/or your kids to run with you too. The run should be completed between May 1, 2022, and July 31, 2022.

Don’t forget to share the run with your friends and family!

Take Photos and Share Your Run with Us

Once you have completed your run, send us some photos to share with [email protected]

We would love to hear about your run! Join our Amyloidosis Facebook page and Instagram page. #AFVirtualRun #AFVirtualRun2022 #VirtualRun2022

Join Us