Healx’s Post

Attralus announces enrollment of first patient in Phase 3 trial for the pan-amyloid diagnostic 124I-evuzamitide (AT-01) for cardiac amyloidosis. ✳ Read the press release – https://lnkd.in/eWykiPEW. ✳ Phase 3 – REVEAL study for iodine 124I-evuzamitide (AT-01) in patients with suspected cardiac amyloidosis has begun enrollment by Brigham and Women's Hospital, Boston MA, in conjunction with Attralus. ✳ Attralus is developing 124I-evuzamitide as the first non-invasive pan-amyloid PET imaging agent specifically designed for the diagnosis and management of patients with all types of systemic amyloidosis. ✳ 124I-evuzamitide has received Breakthrough Therapy Designation from the Food and Drug Administration (FDA) for PET imaging in patients with suspected or known cardiac amyloidosis. ✳ 124I-evuzamitide has been granted orphan drug designations as a diagnostic for the management of ATTR and AL amyloidosis by both the US FDA and the European Commission. #amyloidosis #rarediseases

Davelyn Eaves Hood, MD, MBA, BCMAS, and I share a common goal: to make a meaningful impact on patients in need, one at a time, and to enhance access to innovative medicine. This article encapsulates our experiences in this field—her expertise in the rare diseases therapies, and my background in expanded access. Our goal is to provide guidance for all the life science professionals considering the establishment of compassionate use programs for rare disease drugs, but struggeling with numerous questions. Thank you to Clinical Leader for the opportunity to publish this article and share our knowledge! WEP Clinical #witheverypatient #expandedaccess

I'm pleased to share that we received our 2nd FDA Breakthrough Therapy Designation (BTD) this year for one of our innovative, investigational biologic therapies. The designation, for the treatment of patients with moderate-to-severe Sjögren’s disease (SjD), represents an important milestone for this systemic autoantibody-driven disease that it is believed to impact some three million people in the U.S. where, unfortunately, there are no FDA-approved advanced therapies. Read more about this milestone here: https://bit.ly/4fHGiTM At Johnson & Johnson Innovative Medicine, we never rest in our commitment to change treatment paradigms and transform patients’ lives. #MyCompany #JNJImmunology #SjögrensDisease #Innovation #PatientDriven

I'm happy to share that I recently sat down with Pharma Times to discuss how UCB is prioritizing patient perspectives in rare disease. The integration of patient voices is not just beneficial – it is essential. Our work in myasthenia gravis shows how important it is to understand and learn from those we serve.   Read the full article here https://lnkd.in/dj-xhh4R #ucb #pharmatime #myastheniagravis #gMG #rarediseases

AL CAR-T CLINICAL TRIAL UPDATE ... Immix Biopharma Announces Positive U.S. Clinical Data From First Four Patients in NEXICART-2 U.S. Trial of sterically-optimized CAR-T NXC-201 in relapsed/refractory Light Chain (AL) Amyloidosis. All four patients treated with NXC-201 normalized their disease markers within 30 days of dosing, of which, two are already classified as complete responders (CR), and the remaining two are bone marrow MRD negative (10-6); all patients remain in response as of the data cutoff of Nov 14, 2024 Bone marrow MRD negativity predicts future CR; company believes remaining two patients could be confirmed as CRs in the coming weeks and months https://lnkd.in/de3WS4US

A very insightful interview with Dr. Mazen Noureddin, MD, MHSc, discusses the use of non-invasive testing in liver disease treatment monitoring and the role of MRI Proton Density Fat Fraction (MRI PDFF). https://lnkd.in/gcsd779R "𝗠𝗥𝗜-𝗣𝗗𝗙𝗙 𝘄𝗮𝘀 𝘁𝗵𝗲 𝗯𝗲𝘀𝘁 𝗽𝗿𝗲𝗱𝗶𝗰𝘁𝗼𝗿 𝗼𝗳 𝗿𝗲𝘀𝗽𝗼𝗻𝘀𝗲. In general, PDFF by itself could be sufficient; otherwise, you might want to use two noninvasive tests to confirm that patients are improving." Original Healio post: https://lnkd.in/gEWACfAq At Livivos, Inc., we are dedicated to delivering PDFF directly to the point of care. Learn more about how the #LiverScope is set to revolutionize liver disease diagnosis and treatment monitoring at www.livivos.com. #liverdisease #Rezdiffra #resmetirom Madrigal Pharmaceuticals #fattyliver #PDFF #MRIPDFF #pointofcare #liver #NIT #MASH #NASH #MASLD

Today, we announced the U.S. FDA has cleared our Investigational New Drug Application for CLN-978 in systemic lupus erythematosus (#SLE).  CLN-978 is the first development stage CD19 T cell engager to receive IND Clearance in #autoimmune diseases. CLN-978 is a novel bispecific T cell engager that targets CD19, offering a highly differentiated approach to deliver the potency of T cell redirecting therapy with off-the-shelf access and convenient dosing through subcutaneous administration.  We are advancing CLN-978 in our global Phase 1 clinical trial for patients living with SLE.   Learn more: https://bit.ly/3zX2F8P

Don't miss our keynote presentation by Dr. Daniel O'Connor. He will discuss new perspectives on how the regulatory system can be used to advocate for patients and individualized therapeutics in the rare disease ecosystem. Register today! https://lnkd.in/gzaxRX8q #N1CAnnualMeeting #IndividualizedMedicine #GlobalHealthPerspective #RareDiseases #PersonalizedMedicine #RegulatorySystem

There are over 10,000 rare diseases impacting small patient populations, but 95% of these lack any FDA-approved treatment, says the National Organization for Rare Disorders. For patients and caregivers navigating the complex diagnosis, treatment and care landscape, self-organized communities can be a lifeline. Our experts at Avalere Health investigated how these groups can be best supported and when biopharmaceutical companies should partner – and should step back. Download the whitepaper now: https://bit.ly/3SRVGDA #AvalereHealth #RareDisease #PatientCommunities

There are over 10,000 rare diseases impacting small patient populations, but 95% of these lack any FDA-approved treatment, says the National Organization for Rare Disorders. For patients and caregivers navigating the complex diagnosis, treatment and care landscape, self-organized communities can be a lifeline. Our experts at Avalere Health investigated how these groups can be best supported and when biopharmaceutical companies should partner – and should step back. Download the whitepaper now: https://bit.ly/3SRVGDA #AvalereHealth #RareDisease #PatientCommunities