On November 29, 2022, people all around the world are coming together to tap into the power of human connection and strengthen communities and change our world. Will you be one of them? 

Amyloidosis Foundation will be participating in Giving Tuesday and we need your help! 

By joining the Giving Tuesday movement, you’re proving that in times of uncertainty, generosity can bring the whole world together. 

Here is how you can get ready to give:

1. Mark your calendar 
2. Give. On November 29th, go to our donation link and give.
3. Spread the word. Encourage your friends and family to join you in creating a real impact on November 30 by sharing what our mission means to you and why you support our organization. Make sure to use the hashtag #GivingTuesday and tag us so we can share!

Let’s rally together to build stronger communities. 

The Amyloidosis Foundation is a non-profit organization that supports research through our annual grant program. Our mission is to raise awareness of the disease within the medical community and the public, in addition to providing education, information, and support for patients and their families. 

In 2021, we raised over $15,000. This year our goal is $20,000 and we need your help!

Those who are interested in joining Amyloidosis Foundation’s #GivingTuesday initiative can click here.

Read our latest newsletter here

September 16, 2022 at 7:15 AM EDT

• Interim data from the cardiomyopathy arm of the Phase 1 study of NTLA-2001 showed deep and sustained mean serum transthyretin (TTR) reductions of 93% and 92% at 0.7 mg/kg and 1.0 mg/kg doses, respectively, at day 28
• NTLA-2001 was generally well-tolerated at both dose levels
• Intellia to discuss data at investor event today, Friday, September 16, at 8:00 a.m. ET

CAMBRIDGE, Mass. and TARRYTOWN, N.Y., Sept. 16, 2022 (GLOBE NEWSWIRE) — Intellia Therapeutics, Inc. (NASDAQ:NTLA) and Regeneron Pharmaceuticals, Inc. (NASDAQ:REGN) today announced positive interim results from an ongoing Phase 1 clinical trial of NTLA-2001, an investigational, in vivo CRISPR/Cas9 genome editing therapy in development as a single-dose treatment for transthyretin (ATTR) amyloidosis. The interim data include 12 adult patients with ATTR amyloidosis with cardiomyopathy (ATTR-CM) with New York Heart Association (NYHA) Class I – III heart failure. Single doses of 0.7 mg/kg and 1.0 mg/kg of NTLA-2001 were administered via intravenous infusion, and the change from baseline in serum transthyretin (TTR) protein concentration was measured for each patient.

Read the full article here

Read our latest newsletter here

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:

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

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)

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-L_Rx, 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 ATTR^2,4-6.

Read the Full Article Here

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:

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/