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▎WuXi AppTec Content Team Editor Tomorrow, the U.
S.
FDA will convene an external committee of experts to discuss whether the clinical trial data of Amylyx's investigational therapy AMX0035 in patients with amyotrophic lateral sclerosis (ALS) support its regulatory approval
.
This investigational therapy has received extensive attention from the industry and the ALS patient community because there are currently only two FDA-approved therapies in the ALS disease area, and they do not significantly alter the course of the disease
.
For this 100% fatal disease, patients urgently need innovative treatment options
.
Speaking of ALS, readers may have heard of the "Ice Bucket Challenge," a fundraiser that once swept social media and brought attention to ALS (aka ALS)
.
Famous physicist Professor Hawking once fought bravely against ALS for a long time
.
The motor neurons in the brain and spine of people with the disease continue to die, resulting in muscle weakness and paralysis, ranging from the inability to walk to the inability to speak, swallow, and breathe
.
People with ALS live an average of only four years after diagnosis
.
Although ALS was discovered more than 150 years ago, its pathogenesis has not been fully elucidated.
Gene mutations are associated with about 10% of patients, and environmental factors are also believed to play an important role
.
This adds to the challenge of developing targeted therapies because the pathogenesis is unknown
.
▲ Joshua Cohen (right) and Justin Clay (left) (Photo source: Amylyx's official website) Amylyx's AMX0035 originated from two college students, Joshua Cohen and Justin Clay Mr.
Klee's reflections in his university dormitory
.
Their thinking is that, although scientists have not been able to identify the root cause of nerve cell death due to the complexity of the nervous system, since nerve cell death is associated with the clinical progression of the disease, can they find a way to delay nerve cell death? death process? Thus was born AMX0035, which contains two ingredients - sodium phenylbutrate and taurorsodiol
.
In neurodegenerative diseases, mitochondria and endoplasmic reticulum are often abnormal in nerve cells
.
Mitochondria are the "energy centers" of cells, while the endoplasmic reticulum is the "factory" for the production of various proteins.
Disorders of these two types of organelles can lead to protein folding errors, abnormal energy metabolism and other problems, resulting in the death of nerve cells
.
Amylyx's vision is to use these two drugs to improve the health of mitochondria and endoplasmic reticulum in cells, thereby delaying the death of nerve cells
.
They have the advantage of being FDA-approved for other diseases and therefore have a proven safety profile
.
When Amylyx first started, the two co-founders were only 21 and 22 years old
.
In a phase 2 clinical trial, patients treated with AMX0035 experienced significantly less reductions in mean scores after 24 weeks compared with placebo, using the ALSFRS-R score, a composite measure of daily functioning in patients with ALS
.
The study has been published in the New England Journal of Medicine
.
"Even a two-point change in score can be a significant change in a patient's daily life," said study leader Dr.
Sabrina Paganoni of Massachusetts General Hospital.
"The two-point difference means that The difference between being able to eat successfully and needing to use a feeding tube, or the difference between being able to walk and needing to use a wheelchair
.
In addition, AMX0035 reduced the risk of death in patients by 44% in a long-term follow-up study
.
Amylyx’s press release states that this is the first randomized controlled clinical trial to simultaneously provide benefit in prolonging patient life and improving patient motor function.
Amylyx is a promising ALS therapy
.
At the end of last year, its new drug application was granted priority review status by the US FDA
.
It is worth mentioning that Amylyx is already conducting a confirmatory phase 3 clinical trial, which is planned to be tested in 600 ALS patients.
The effect of AMX0035 is expected to be obtained in 2024.
In
addition
to AMX0035, according to incomplete statistics, there are more than 100 innovative treatments in the ALS R&D pipeline that have entered the clinical development stage
.
Many
innovative treatments are expected to reach development milestones in 2022 , including Biohaven Pharmaceuticals' myeloperoxidase (MPO) inhibitor verdiperstat, which reduces microglia activation and reduces neuroinflammation in ALS patients
.
CNMAU-8, a gold nanocrystal suspension developed by Clene Nanomedicine , it has neuroprotective and remyelination-promoting effects
.
Prilenia Therapeutics' S1R agonist pridopidine is also a dopamine D2 receptor antagonist
.
It has effects such as enhancing neuroprotection and neuroplasticity
.
These therapies are expected to gain 2/3 this year
In
addition, Wave Life Sciences’ WVE-004 is an antisense oligonucleotide (ASO) therapy for the treatment of C9orf72-related ALS
.
Eledon Pharmaceuticals' monoclonal antibody AT-1501 blocks CD40 ligand-mediated signaling, potentially improving muscle function and slowing disease progression
.
These treatments are expected to receive early clinical trial results this year
.
WuXi PharmaTech related reading: When we talk about ALS, it's not just the ice bucket challenge and Hawking: drug development and financing in 2021 , plays an important role in the discovery and validation of new targets
.
For example, in July last year, Verge Genomics and Eli Lilly reached a research and development cooperation to develop innovative treatments for ALS
.
Using AI "unbiased" algorithms, the company's technology platform can provide insights related to disease causal mechanisms in patient populations segmented by genetic characteristics to discover therapeutic targets for complex diseases
.
Image source: 123RF Recently, the FDA published a review document for AMX0035 on its official website.
The document pointed out some deficiencies in the phase 2 clinical trial data of AMX0035, and also stated that the FDA has approval for serious diseases with significant unmet medical needs.
'Regulatory flexibility' for innovative therapies
.
At tomorrow's expert committee meeting, participating experts will not only hear from Amylyx representatives, FDA reviewers, but also from ALS patients
.
Regardless of the outcome, the field of ALS has received more attention and R&D investment in recent years.
We look forward to the advancement of science and technology and the help of capital to bring breakthrough therapies to patients as soon as possible, and to thaw the "gradually frozen people"
.
Reference: [1] 'I'm going to prove you wrong': How a DC power couple used an ALS diagnosis to create a political juggernaut.
Retrieved March 28, 2022, from https:// /01/11/brian-wallach-sandra-abrevaya-als-advocacy/[2] Scientists take key step toward unraveling the genetic roots of ALS in a pair of studies.
Retrieved March 28, 2022, from https://www.
statnews.
com/2022/02/23/scientists-take-key-step-toward-unraveling-the-genetic-roots-of-als-in-a-pair-of-studies/[3] UPDATED: Amylyx - and Hopeful ALS Community – Face Another Moment of Truth.
Retrieved March 28, 2022, from https:// [4] FDA review is critical of data on Amylyx ALS drug, but cites 'regulatory flexibility'.
Retrieved March 28, 2022, from https:// An ALS therapy sets up a crucial test of the FDA's independence.
Retrieved March 28, 2022, from https:// Freezing Man, More Than Ice Bucket Challenge and Hawking: Drug Development and Financing in 2021.
Retrieved March 28, 2022, from https://mp.
weixin.
qq.
com/s?__biz=MzIzMTU0NTM5MA==&mid=2247509785&idx=1&sn= f49bbbd713cb7701831612cba928d33b & chksm = e8a06858dfd7e14efb1ff9b317cd892f9ca230ed6cd9bd2beb4749ea052ccc813f1b6f64bd6a # rd [7] An ALS therapy sets up a crucial test of the FDA's independence.
Retrieved March 28, 2022, from https:// therapy-tees-up-test-fda-independence/Disclaimer: WuXi AppTec content team focuses on introducing global biomedical health research progresscom/2022/03/22/amylyx-als-disease-therapy-tees-up-test-fda-independence/Disclaimer: WuXi AppTec content team focuses on introducing global biomedical health research progress
.
This article is for information exchange purposes only, and the views expressed in this article do not represent WuXi AppTec's position, nor do they represent WuXi AppTec's support or opposition to the views expressed in this article
.
This article is also not a treatment plan recommendation
.
For guidance on treatment options, please visit a regular hospital
.
S.
FDA will convene an external committee of experts to discuss whether the clinical trial data of Amylyx's investigational therapy AMX0035 in patients with amyotrophic lateral sclerosis (ALS) support its regulatory approval
.
This investigational therapy has received extensive attention from the industry and the ALS patient community because there are currently only two FDA-approved therapies in the ALS disease area, and they do not significantly alter the course of the disease
.
For this 100% fatal disease, patients urgently need innovative treatment options
.
Speaking of ALS, readers may have heard of the "Ice Bucket Challenge," a fundraiser that once swept social media and brought attention to ALS (aka ALS)
.
Famous physicist Professor Hawking once fought bravely against ALS for a long time
.
The motor neurons in the brain and spine of people with the disease continue to die, resulting in muscle weakness and paralysis, ranging from the inability to walk to the inability to speak, swallow, and breathe
.
People with ALS live an average of only four years after diagnosis
.
Although ALS was discovered more than 150 years ago, its pathogenesis has not been fully elucidated.
Gene mutations are associated with about 10% of patients, and environmental factors are also believed to play an important role
.
This adds to the challenge of developing targeted therapies because the pathogenesis is unknown
.
▲ Joshua Cohen (right) and Justin Clay (left) (Photo source: Amylyx's official website) Amylyx's AMX0035 originated from two college students, Joshua Cohen and Justin Clay Mr.
Klee's reflections in his university dormitory
.
Their thinking is that, although scientists have not been able to identify the root cause of nerve cell death due to the complexity of the nervous system, since nerve cell death is associated with the clinical progression of the disease, can they find a way to delay nerve cell death? death process? Thus was born AMX0035, which contains two ingredients - sodium phenylbutrate and taurorsodiol
.
In neurodegenerative diseases, mitochondria and endoplasmic reticulum are often abnormal in nerve cells
.
Mitochondria are the "energy centers" of cells, while the endoplasmic reticulum is the "factory" for the production of various proteins.
Disorders of these two types of organelles can lead to protein folding errors, abnormal energy metabolism and other problems, resulting in the death of nerve cells
.
Amylyx's vision is to use these two drugs to improve the health of mitochondria and endoplasmic reticulum in cells, thereby delaying the death of nerve cells
.
They have the advantage of being FDA-approved for other diseases and therefore have a proven safety profile
.
When Amylyx first started, the two co-founders were only 21 and 22 years old
.
In a phase 2 clinical trial, patients treated with AMX0035 experienced significantly less reductions in mean scores after 24 weeks compared with placebo, using the ALSFRS-R score, a composite measure of daily functioning in patients with ALS
.
The study has been published in the New England Journal of Medicine
.
"Even a two-point change in score can be a significant change in a patient's daily life," said study leader Dr.
Sabrina Paganoni of Massachusetts General Hospital.
"The two-point difference means that The difference between being able to eat successfully and needing to use a feeding tube, or the difference between being able to walk and needing to use a wheelchair
.
In addition, AMX0035 reduced the risk of death in patients by 44% in a long-term follow-up study
.
Amylyx’s press release states that this is the first randomized controlled clinical trial to simultaneously provide benefit in prolonging patient life and improving patient motor function.
Amylyx is a promising ALS therapy
.
At the end of last year, its new drug application was granted priority review status by the US FDA
.
It is worth mentioning that Amylyx is already conducting a confirmatory phase 3 clinical trial, which is planned to be tested in 600 ALS patients.
The effect of AMX0035 is expected to be obtained in 2024.
In
addition
to AMX0035, according to incomplete statistics, there are more than 100 innovative treatments in the ALS R&D pipeline that have entered the clinical development stage
.
Many
innovative treatments are expected to reach development milestones in 2022 , including Biohaven Pharmaceuticals' myeloperoxidase (MPO) inhibitor verdiperstat, which reduces microglia activation and reduces neuroinflammation in ALS patients
.
CNMAU-8, a gold nanocrystal suspension developed by Clene Nanomedicine , it has neuroprotective and remyelination-promoting effects
.
Prilenia Therapeutics' S1R agonist pridopidine is also a dopamine D2 receptor antagonist
.
It has effects such as enhancing neuroprotection and neuroplasticity
.
These therapies are expected to gain 2/3 this year
In
addition, Wave Life Sciences’ WVE-004 is an antisense oligonucleotide (ASO) therapy for the treatment of C9orf72-related ALS
.
Eledon Pharmaceuticals' monoclonal antibody AT-1501 blocks CD40 ligand-mediated signaling, potentially improving muscle function and slowing disease progression
.
These treatments are expected to receive early clinical trial results this year
.
WuXi PharmaTech related reading: When we talk about ALS, it's not just the ice bucket challenge and Hawking: drug development and financing in 2021 , plays an important role in the discovery and validation of new targets
.
For example, in July last year, Verge Genomics and Eli Lilly reached a research and development cooperation to develop innovative treatments for ALS
.
Using AI "unbiased" algorithms, the company's technology platform can provide insights related to disease causal mechanisms in patient populations segmented by genetic characteristics to discover therapeutic targets for complex diseases
.
Image source: 123RF Recently, the FDA published a review document for AMX0035 on its official website.
The document pointed out some deficiencies in the phase 2 clinical trial data of AMX0035, and also stated that the FDA has approval for serious diseases with significant unmet medical needs.
'Regulatory flexibility' for innovative therapies
.
At tomorrow's expert committee meeting, participating experts will not only hear from Amylyx representatives, FDA reviewers, but also from ALS patients
.
Regardless of the outcome, the field of ALS has received more attention and R&D investment in recent years.
We look forward to the advancement of science and technology and the help of capital to bring breakthrough therapies to patients as soon as possible, and to thaw the "gradually frozen people"
.
Reference: [1] 'I'm going to prove you wrong': How a DC power couple used an ALS diagnosis to create a political juggernaut.
Retrieved March 28, 2022, from https:// /01/11/brian-wallach-sandra-abrevaya-als-advocacy/[2] Scientists take key step toward unraveling the genetic roots of ALS in a pair of studies.
Retrieved March 28, 2022, from https://www.
statnews.
com/2022/02/23/scientists-take-key-step-toward-unraveling-the-genetic-roots-of-als-in-a-pair-of-studies/[3] UPDATED: Amylyx - and Hopeful ALS Community – Face Another Moment of Truth.
Retrieved March 28, 2022, from https:// [4] FDA review is critical of data on Amylyx ALS drug, but cites 'regulatory flexibility'.
Retrieved March 28, 2022, from https:// An ALS therapy sets up a crucial test of the FDA's independence.
Retrieved March 28, 2022, from https:// Freezing Man, More Than Ice Bucket Challenge and Hawking: Drug Development and Financing in 2021.
Retrieved March 28, 2022, from https://mp.
weixin.
qq.
com/s?__biz=MzIzMTU0NTM5MA==&mid=2247509785&idx=1&sn= f49bbbd713cb7701831612cba928d33b & chksm = e8a06858dfd7e14efb1ff9b317cd892f9ca230ed6cd9bd2beb4749ea052ccc813f1b6f64bd6a # rd [7] An ALS therapy sets up a crucial test of the FDA's independence.
Retrieved March 28, 2022, from https:// therapy-tees-up-test-fda-independence/Disclaimer: WuXi AppTec content team focuses on introducing global biomedical health research progresscom/2022/03/22/amylyx-als-disease-therapy-tees-up-test-fda-independence/Disclaimer: WuXi AppTec content team focuses on introducing global biomedical health research progress
.
This article is for information exchange purposes only, and the views expressed in this article do not represent WuXi AppTec's position, nor do they represent WuXi AppTec's support or opposition to the views expressed in this article
.
This article is also not a treatment plan recommendation
.
For guidance on treatment options, please visit a regular hospital
.
