All we ask is that you sign the petition http://www.als.org/petition - you can sign if you are in the UK, USA or anywhere, but your support is needed to speed up the FDA approval of the promising new drugs #AMX0035 which has been shown to slow the progress of #MND #ALS
November 24, 2024 at 1:13 AM
All we ask is that you sign the petition http://www.als.org/petition - you can sign if you are in the UK, USA or anywhere, but your support is needed to speed up the FDA approval of the promising new drugs #AMX0035 which has been shown to slow the progress of #MND #ALS
Day 1 of John Zimmer’s 100 miles over 6 days challenge, walking with gallery dogs Frida & Diego the entire length of @SouthDownsWayNT to raise awareness of @mndassoc @MNDWestSxSouth & #ALS, raising awareness of new theraputic drug #amx0035 - See next tweet for how you can help.
November 24, 2024 at 1:06 AM
All we ask is that you sign the petition http://als.org/petition - you can sign if you are in the UK, USA or anywhere, but your support is needed to speed up the FDA approval of the promising new drugs #AMX0035 which has been shown to slow the progress of #MND #ALS
November 24, 2024 at 1:06 AM
All we ask is that you sign the petition http://als.org/petition - you can sign if you are in the UK, USA or anywhere, but your support is needed to speed up the FDA approval of the promising new drugs #AMX0035 which has been shown to slow the progress of #MND #ALS
Day 2 & another 20 miles in John Zimmer’s 100 miles over 6 days challenge, walking with gallery dogs Frida & Diego the entire length of @SouthDownsWayNT to raise awareness of @mndassoc @MNDWestSxSouth & #ALS & of new theraputic drug #amx0035 - See next tweet for how you can help.
November 24, 2024 at 12:59 AM
All we ask is that you sign the petition http://als.org/petition - you can sign if you are in the UK, USA or anywhere, but your support is needed to speed up the FDA approval of the promising new drugs #AMX0035 which has been shown to slow the progress of #MND #ALS
The ALS Association Action Center
ALS advocates lead the fight to defeat ALS. You can lead ...
als.org
November 24, 2024 at 12:59 AM
All we ask is that you sign the petition http://als.org/petition - you can sign if you are in the UK, USA or anywhere, but your support is needed to speed up the FDA approval of the promising new drugs #AMX0035 which has been shown to slow the progress of #MND #ALS
Day 3: Cocking to Arundel in John Zimmer’s 100 miles over 6 days challenge, walking with gallery dogs Frida & Diego the entire length of @SouthDownsWayNT to raise awareness of @mndassoc @MNDWestSxSouth & #ALS & of new theraputic drug #amx0035 - See next tweet for how you can help
November 24, 2024 at 12:53 AM
Amylyx Pharmaceuticals announced that it is ending its phase 2b trial for #PSP after test group results turned out similarly to the placebo group. We’re grateful for Amylyx’s work furthering our understanding of PSP’s biology.
Learn more: https://bit.ly/43taApW
Learn more: https://bit.ly/43taApW
November 18, 2025 at 7:46 PM
Amylyx Pharmaceuticals announced that it is ending its phase 2b trial for #PSP after test group results turned out similarly to the placebo group. We’re grateful for Amylyx’s work furthering our understanding of PSP’s biology.
Learn more: https://bit.ly/43taApW
Learn more: https://bit.ly/43taApW
Feed: "Clinical Trials Arena"
By: Robert Barrie on Wednesday, August 27, 2025
By: Robert Barrie on Wednesday, August 27, 2025
Amylyx culls rare neurological disorder programme after trial failure
AMX0035 failed to show a difference to placebo in the Phase IIb ORION trial.
www.clinicaltrialsarena.com
August 28, 2025 at 12:02 AM
Feed: "Clinical Trials Arena"
By: Robert Barrie on Wednesday, August 27, 2025
By: Robert Barrie on Wednesday, August 27, 2025
5. There is a recent published article on Blue Sky that picks up the NfL and continues the general dialog on how it can be used for Setting up Phase 2 drug trials.
6. After reading this article, the loathing I had for Amylyx's AMX0035 has now gone through the roof.
(2)
6. After reading this article, the loathing I had for Amylyx's AMX0035 has now gone through the roof.
(2)
December 14, 2024 at 2:39 PM
5. There is a recent published article on Blue Sky that picks up the NfL and continues the general dialog on how it can be used for Setting up Phase 2 drug trials.
6. After reading this article, the loathing I had for Amylyx's AMX0035 has now gone through the roof.
(2)
6. After reading this article, the loathing I had for Amylyx's AMX0035 has now gone through the roof.
(2)
Breaking News: ( NASDAQ: #AMLX ) Amylyx Pharmaceuticals Announces Positive Topline Results from Phase 2 HELIOS Clinical Trial Demonstrating Sustained Improvements with AMX0035 in People Living with Wolfram Syndrome
#StockMarket #News
#StockMarket #News
October 17, 2024 at 4:55 PM
News; ( NASDAQ: #AMLX ) Amylyx Pharmaceuticals to Host Virtual Webcast to Discuss Topline Results from Phase 2 HELIOS Study of AMX0035 in Wolfram Syndrome on October 17, 2024
#StockMarket #News
#StockMarket #News
October 15, 2024 at 3:45 PM
Amylyx halted the ORION trial in #PSP: no benefit from AMX0035 (sodium phenylbutyrate + taurursodiol).
The question: a false negative trial (inadequate drug) or a true negative (protein aggregation + mitochondrial dysfunction not driving PSP)?
investors.amylyx.com/news-release...
The question: a false negative trial (inadequate drug) or a true negative (protein aggregation + mitochondrial dysfunction not driving PSP)?
investors.amylyx.com/news-release...
Amylyx Pharmaceuticals to Discontinue ORION Program of AMX0035 for Progressive Supranuclear Palsy (PSP) | Amylyx Pharmaceuticals, Inc.
- AMX0035 did not show differences compared to placebo on primary or secondary outcomes at Week 24 - AMX0035 continued to be generally well-tolerated CAMBRIDGE, Mass. --(BUSINESS WIRE)--Aug. 27, 2025-...
investors.amylyx.com
August 27, 2025 at 8:25 PM
Amylyx halted the ORION trial in #PSP: no benefit from AMX0035 (sodium phenylbutyrate + taurursodiol).
The question: a false negative trial (inadequate drug) or a true negative (protein aggregation + mitochondrial dysfunction not driving PSP)?
investors.amylyx.com/news-release...
The question: a false negative trial (inadequate drug) or a true negative (protein aggregation + mitochondrial dysfunction not driving PSP)?
investors.amylyx.com/news-release...
#AMLX Amylyx Pharmaceuticals Announces Peer-Reviewed Publication of Phase 2 Open-Label HELIOS Trial Data for AMX0035 in The Journal of Clinical Investigation
https://www.stocktitan.net/news/AMLX/amylyx-pharmaceuticals-announces-peer-reviewed-publication-of-phase-99zolrlza7hr.html
https://www.stocktitan.net/news/AMLX/amylyx-pharmaceuticals-announces-peer-reviewed-publication-of-phase-99zolrlza7hr.html
Amylyx Pharmaceuticals Announces Peer-Reviewed Publication of Phase 2 Open-Label HELIOS Trial Data for AMX0035 in The Journal of Clinical Investigation
Amylyx Pharmaceuticals (Nasdaq: AMLX) reported peer-reviewed Phase 2 HELIOS data for AMX0035 in adults with Wolfram syndrome, now published in The Journal of Clinical Investigation.At Weeks 24 and 48, results showed improved pancreatic beta cell function, better glycemic control, vision stabilization trends, and generally mild-to-moderate adverse events. The company is working with the FDA on a Phase 3 trial.
www.stocktitan.net
May 15, 2026 at 4:12 PM
#AMLX Amylyx Pharmaceuticals Announces Peer-Reviewed Publication of Phase 2 Open-Label HELIOS Trial Data for AMX0035 in The Journal of Clinical Investigation
https://www.stocktitan.net/news/AMLX/amylyx-pharmaceuticals-announces-peer-reviewed-publication-of-phase-99zolrlza7hr.html
https://www.stocktitan.net/news/AMLX/amylyx-pharmaceuticals-announces-peer-reviewed-publication-of-phase-99zolrlza7hr.html
#AMLX Amylyx Pharmaceuticals to Discontinue ORION Program of AMX0035 for Progressive Supranuclear Palsy (PSP)
https://www.stocktitan.net/news/AMLX/amylyx-pharmaceuticals-to-discontinue-orion-program-of-amx0035-for-hfq2i93ycdjo.html
https://www.stocktitan.net/news/AMLX/amylyx-pharmaceuticals-to-discontinue-orion-program-of-amx0035-for-hfq2i93ycdjo.html
Clinical Trial Setback: Amylyx's AMX0035 Shows No Benefit in Progressive Supranuclear Palsy Study
Amylyx ends ORION program after AMX0035 shows no difference vs placebo in PSP treatment at 24 weeks. Company shifts focus to Phase 3 LUCIDITY trial and Wolfram syndrome development.
www.stocktitan.net
August 27, 2025 at 11:00 AM
#AMLX Amylyx Pharmaceuticals to Discontinue ORION Program of AMX0035 for Progressive Supranuclear Palsy (PSP)
https://www.stocktitan.net/news/AMLX/amylyx-pharmaceuticals-to-discontinue-orion-program-of-amx0035-for-hfq2i93ycdjo.html
https://www.stocktitan.net/news/AMLX/amylyx-pharmaceuticals-to-discontinue-orion-program-of-amx0035-for-hfq2i93ycdjo.html
#AMLX Amylyx Pharmaceuticals Announces Positive Long-Term Results from Phase 2 HELIOS Clinical Trial of AMX0035 in People with Wolfram Syndrome
https://www.stocktitan.net/news/AMLX/amylyx-pharmaceuticals-announces-positive-long-term-results-from-88v7q19h620x.html
https://www.stocktitan.net/news/AMLX/amylyx-pharmaceuticals-announces-positive-long-term-results-from-88v7q19h620x.html
Amylyx Pharmaceuticals Announces Positive Long-Term Results from Phase 2 HELIOS Clinical Trial of AMX0035 in People with Wolfram Syndrome
Amylyx Pharmaceuticals (NASDAQ: AMLX) has reported positive 48-week results from its Phase 2 HELIOS clinical trial of AMX0035 in treating Wolfram syndrome. The trial demonstrated sustained improvements or stabilization across multiple disease measures, including pancreatic function, glycemic control, and vision.Key findings at Week 48 include a mean increase of +34.5 min*ng/mL in C-peptide response, improved glycemic control with HbA1c reduction of -0.40%, and increased time in target glucose range by +9.6%. The treatment was generally well-tolerated, with only mild to moderate adverse events and no serious treatment-related issues. All participants reported stability or improvement in their symptoms according to both patient and clinician assessments.The company plans to use these results and FDA discussions to design a Phase 3 trial. AMX0035 has received Orphan Drug Designation from both FDA and European Commission for Wolfram syndrome treatment.
www.stocktitan.net
May 12, 2025 at 11:00 AM
#AMLX Amylyx Pharmaceuticals Announces Positive Long-Term Results from Phase 2 HELIOS Clinical Trial of AMX0035 in People with Wolfram Syndrome
https://www.stocktitan.net/news/AMLX/amylyx-pharmaceuticals-announces-positive-long-term-results-from-88v7q19h620x.html
https://www.stocktitan.net/news/AMLX/amylyx-pharmaceuticals-announces-positive-long-term-results-from-88v7q19h620x.html
Amylyx are discontinuing their Orion trial of AMX0035 for Progressive Supranuclear Palsy (PSP). After AMX0035 did not show differences compared to the placebo on primary and secondary outcomes at week 24, it was decided the trial should be discontinued. ow.ly/j9AB50WNE4I
September 2, 2025 at 3:10 PM
Amylyx are discontinuing their Orion trial of AMX0035 for Progressive Supranuclear Palsy (PSP). After AMX0035 did not show differences compared to the placebo on primary and secondary outcomes at week 24, it was decided the trial should be discontinued. ow.ly/j9AB50WNE4I
We can no longer trust that other countries mirroring NIH will lead to best practice if this man gets the chair.
I am so tired of fighting for "my" diseases and disorders, two of which are just about to get their first medications out.🤯
#Galactosemia
#Govorestat
#WolframSyndrome
#ALS
#AMX0035
I am so tired of fighting for "my" diseases and disorders, two of which are just about to get their first medications out.🤯
#Galactosemia
#Govorestat
#WolframSyndrome
#ALS
#AMX0035
Hey look, my article is in the Ottawa Citizen!
"RFK Jr. is a danger to health care in the U.S. — and Canada"
ottawacitizen.com/opinion/deon...
"RFK Jr. is a danger to health care in the U.S. — and Canada"
ottawacitizen.com/opinion/deon...
Deonandan: RFK Jr. is a danger to health care in the U.S. — and Canada
RFK's appointment to head health services in the U.S. under Trump normalizes the anti-evidence pseudoscience that abounds today.
ottawacitizen.com
November 20, 2024 at 8:09 PM
We can no longer trust that other countries mirroring NIH will lead to best practice if this man gets the chair.
I am so tired of fighting for "my" diseases and disorders, two of which are just about to get their first medications out.🤯
#Galactosemia
#Govorestat
#WolframSyndrome
#ALS
#AMX0035
I am so tired of fighting for "my" diseases and disorders, two of which are just about to get their first medications out.🤯
#Galactosemia
#Govorestat
#WolframSyndrome
#ALS
#AMX0035
Måtte google litt.
Ser at AMX0035 har blitt godkjent i EU nå, håper du får den snart.
❤️
Ser at AMX0035 har blitt godkjent i EU nå, håper du får den snart.
❤️
November 10, 2024 at 6:21 PM
Måtte google litt.
Ser at AMX0035 har blitt godkjent i EU nå, håper du får den snart.
❤️
Ser at AMX0035 har blitt godkjent i EU nå, håper du får den snart.
❤️
Vi får håpe de får litt fart på utviklingen av AMX0035, og at den blir godkjent av #Beslutningsforum
translationalsciencebenefits.wustl.edu/case-study/a...
translationalsciencebenefits.wustl.edu/case-study/a...
An Update on New Therapies for Wolfram Syndrome – Translational Science Benefits ModelArrow Down
translationalsciencebenefits.wustl.edu
September 24, 2024 at 5:50 PM
Vi får håpe de får litt fart på utviklingen av AMX0035, og at den blir godkjent av #Beslutningsforum
translationalsciencebenefits.wustl.edu/case-study/a...
translationalsciencebenefits.wustl.edu/case-study/a...
Amylyx held Q1 earnings call yesterday. No new data. No new pipeline. Just the same pivot to neurodegenerative disease they announced after AMX0035 failed. The only surprise? They still have cash.
May 29, 2026 at 11:30 PM
Amylyx held Q1 earnings call yesterday. No new data. No new pipeline. Just the same pivot to neurodegenerative disease they announced after AMX0035 failed. The only surprise? They still have cash.
Signal or noise? > Amylyx's old ALS drug fails in another neuro disease, focus
turns to GLP-1 >> Comment below! #strategy #competitiveintelligence #marketing #pharmaceutical #pharma #productmarketing #healthcare #biotech
turns to GLP-1 >> Comment below! #strategy #competitiveintelligence #marketing #pharmaceutical #pharma #productmarketing #healthcare #biotech
Amylyx's old ALS drug fails in another neuro disease, focus
turns to GLP-1
For all intents and purposes, Amylyx is now a GLP-1 biotech. The company's now-withdrawn ALS drug, branded as Relyvrio and codenamed AMX0035, failed a Phase 2b study for a separate rare neurological disease called progressive ...
dlvr.it
August 27, 2025 at 3:51 PM
Signal or noise? > Amylyx's old ALS drug fails in another neuro disease, focus
turns to GLP-1 >> Comment below! #strategy #competitiveintelligence #marketing #pharmaceutical #pharma #productmarketing #healthcare #biotech
turns to GLP-1 >> Comment below! #strategy #competitiveintelligence #marketing #pharmaceutical #pharma #productmarketing #healthcare #biotech
Thoughts on this? >> AMX0035 Continues Promise in Wolfram Syndrome, FDA Clears
Testing for CAP-002, Gene Therapy AAV-GAD Gains RMAT Designation -
Neurology live >> Comment below! #strategy #competitiveintelligence #marketing #pharmaceutical #pharma #biotech #healthcare #competitivemarketing
Testing for CAP-002, Gene Therapy AAV-GAD Gains RMAT Designation -
Neurology live >> Comment below! #strategy #competitiveintelligence #marketing #pharmaceutical #pharma #biotech #healthcare #competitivemarketing
AMX0035 Continues Promise in Wolfram Syndrome, FDA Clears
Testing for CAP-002, Gene Therapy AAV-GAD Gains RMAT Designation -
Neurology live
AMX0035 Continues Promise in Wolfram Syndrome, FDA Clears Testing for CAP-002, Gene Therapy AAV-GAD Gains RMAT Designation Neurology live
dlvr.it
May 17, 2025 at 12:00 PM
Thoughts on this? >> AMX0035 Continues Promise in Wolfram Syndrome, FDA Clears
Testing for CAP-002, Gene Therapy AAV-GAD Gains RMAT Designation -
Neurology live >> Comment below! #strategy #competitiveintelligence #marketing #pharmaceutical #pharma #biotech #healthcare #competitivemarketing
Testing for CAP-002, Gene Therapy AAV-GAD Gains RMAT Designation -
Neurology live >> Comment below! #strategy #competitiveintelligence #marketing #pharmaceutical #pharma #biotech #healthcare #competitivemarketing