FDA Approves Kidney Drug Despite No Functional Benefit
This week's Biotech Scorecard newsletter: $TVTX Travere Therapeutics' drug for a rare kidney disease doesn’t improve kidney function. The FDA approved it anyway. https://t.co/63hDsPfOcT
GLP‑1 Drugs Don’t Disproportionately Reduce Functional Muscle, DXA Misleads
1/2) New research suggests GLP-1 weight loss drugs probably do NOT cause disproportionate loss of functional lean muscle. The confusion has arisen from the fact that DXA scans capture and pool a lot of different organs and tissues into the category...
Roche Initiates New Elevidys Trial Targeting EU Approval
Roche to launch another Elevidys trial, with eyes on European approval https://t.co/eAANNiihpt via @DrewQJoseph $SRPT
Patient‑Driven Platform Accelerates Multiple Myeloma Research
.@HealthtreeMM Hub: A Patient-Derived, Patient-Driven Clinical Cancer Information Platform Used to Overcome Hurdles & Accelerate Research in Multiple Myeloma [3/10/22] @NSweeneyPhd @AhlstromJenny @FaithEDavies1 @mtmdphd @JCOCCI_ASCO https://t.co/WOwLb9Eozv @myelomacrowd #mmsm https://t.co/6HieAzjSES
New Trial Compares Dara‑Bor‑Dex vs Cy‑Bor‑Dex in Myeloma‑Induced Kidney
EAA241 - Ph 2 RCT Dara-Bor-Dex vs Cy-Bor-Dex in the treatment of Newly Diagnosed Multiple Myeloma with Light Chain Cast Nephropathy (LCCN) [Study activated 8/11/25] @keruakous https://t.co/1NgvVZ3fTA #NCT07085728 #mmsm @eaonc https://t.co/KJfqVVcwEr
Daratumumab Boosts Revlimid Efficacy in Smoldering Myeloma
#EAonc EAA173 - Daratumumab to Enhance Therapeutic Effectiveness of Revlimid in Smoldering Myeloma (DETER-SMM) - PI: @nsc_natalie https://t.co/VtBMJUjI5X Activated: Apr 30, 2019 #mmsm @eaonc #NCT03937635 @VincentRK @mweissmdphd https://t.co/SKPUQiSaU4
Phase 3 Trial Tests Daratum
.@SWOG S2213 Ph3 RCT Dara-VC Induction Followed by ASCT or Dara-VCD Consolidation & Daratumumab Maintenance in Pts w/ Newly Diagnosed AL Amyloidosis [Activated: 12/1/23] https://t.co/OizUfJCc2c #mmsm #bmtsm https://t.co/Hq9VlRCTCI
Four Candidates Target Ultra‑Rare Polish PUS3 Syndrome
“We have four drug candidates for patients with an ultra-rare disease with Polish origins, the PUS3 syndrome; one of these candidates will be used in practice,” Lisowski said. https://t.co/GTJbPQSdXQ
When to Stop Myeloma Treatment: MRD Insights
MT @hhashmi87 #IMS25 Great Meet The Expert talk by @bdermanmd on MRD in Myeloma Risk vs benefit Who can stop treatment Active surveillance post discontinuation #mmMRD #mmsm https://t.co/FIDPrE7QRb
MRD‑negative Patients May Stop Myeloma Maintenance Therapy
Discontinuation of maintenance therapy in multiple myeloma guided by multimodal measurable residual disease negativity (MRD2STOP) - @bdermanmd et al. @ajjakubowiak #ASCO24 Abstract 106 https://t.co/FBTY7SnCxK #NCT04108624 #mmsm #mmMRD
ODAC Approves MRD as Early Endpoint for Myeloma Trials
A Historic Turning Point: ODAC Unanimously Votes [4/12/24] in Favor of MRD Testing as an Early Endpoint in Myeloma Clinical Trials to Support Accelerated Approvals of New Treatments [Apr 18, 2024] @IMFmyeloma https://t.co/eDOgIrpVeR #mmMRD #mmsm #ctsm @FDAOncology https://t.co/V4wrNgvTrG
Clinicians Show Mixed Adoption of Myeloma MRD Testing
Clinician attitudes and practices toward measurable residual disease in multiple myeloma [Jun 7, 2020] @bdermanmd @jagoda_jasielec @ajjakubowiak Brit J Haematol https://t.co/8zhxYfYwZQ #mmsm #mmMRD https://t.co/mPtRCjnB9f
Vaccines Vanishing Threatens Global Health Catastrophe
The Horrors That Could Lie Ahead if Vaccines Vanish — @propublica good article on the work of @NathanLo3579 @Stanford my former mentee and amazing colleague https://t.co/n9oUgkvOZ7
Glioblastoma's Immune Microenvironment Blocks Checkpoint Therapy
The glioblastoma tumor immune microenvironment (TIME) is an immunosuppressive barrier to therapy that encumbers glioblastoma responses to immune checkpoint inhibition (ICI). https://t.co/rgIKZ8N0Q4
Synthetic Biology Localizes Drug Production, Ending Fragile Supply Chains
Most pharmaceutical APIs still travel thousands of miles through fragile global supply chains before they reach a patient. COVID and the Russia-Ukraine war showed exactly what that fragility costs. Synthetic biology offers a different model: program cells to manufacture the molecules in...
