FDA Advisory Board Meets Again to Discuss Amylyx's Controversial Amylyx Lateral Sclerosis Drug Albrioza (AMX0035)

The FDA Advisory Board met again to discuss Amylyx's controversial amyotrophic lateral sclerosis (ALS) drug, Albrioza (AMX0035

The FDA Advisory Board met again to discuss Amylyx's controversial amyotrophic lateral sclerosis (ALS) drug, Albrioza (AMX0035

Earlier, at its March meeting, the FDA's Advisory Committee on Drugs for the Outer and Central Nervous Systems voted 6:4 to express doubts about the persuasiveness of the Phase II data — including the small size of the trial, missing data, the appropriateness of statistical methods for analyzing primary endpoints, and randomization protocols

Earlier, at its March meeting, the FDA's Advisory Committee on Drugs for the Outer and Central Nervous Systems voted 6:4 to express doubts about the persuasiveness of the Phase II data — including the small size of the trial, missing data, the appropriateness of statistical methods for analyzing primary endpoints, and randomization protocols

These ultimately point to the conclusion that the Phase II results, while statistically significant, are not sufficient to establish that AMX0035 can improve disease progression

These ultimately point to the conclusion that the Phase II results, while statistically significant, are not sufficient to establish that AMX0035 can improve disease progression

Amylyx pinned a slight hope for discussion at the second meeting, after the FDA released a briefing heralding what appeared to be a failure — Amylyx's new analysis still doesn't have "enough independence or convincing" to support the effectiveness of

Amylyx pinned a slight hope for discussion at the second meeting, after the FDA released a briefing heralding what appeared to be a failure — Amylyx's new analysis still doesn't have "enough independence or convincing" to support the effectiveness of

However, the results of the meeting, released sept.

However, the results of the meeting, released sept.

Even more unusual, Billy Dunn, director of neuroscience at CDER, and Amylyx's co-CEO both promised that AMX0035 would be withdrawn from the market if AMX0035 failed the ongoing Phase III trial

Even more unusual, Billy Dunn, director of neuroscience at CDER, and Amylyx's co-CEO both promised that AMX0035 would be withdrawn from the market if AMX0035 failed the ongoing Phase III trial

In this update, Amylyx submitted a new analysis of AMX0035 survival data, as well as biomarker data from phase II studies conducted in Alzheimer's disease, to show the effect

In this update, Amylyx submitted a new analysis of AMX0035 survival data, as well as biomarker data from phase II studies conducted in Alzheimer's disease, to show the effect

One of the new analyses used participants' response rates to assess treatment effectiveness

One of the new analyses used participants' response rates to assess treatment effectiveness

Amylyx also showcased the AMX0035 survival prediction algorithm, which was created
based on natural history data from more than 10,000 ALS patients from 12 specialized centers in Europe.
However, the FDA rejected the data because the patients in the database did not participate in clinical trials and there were many hidden dangers in the reliability of the analysis, such as external control or lack of pre-specified protocols
.

Amylyx also showcased the AMX0035 survival prediction algorithm, which was created
based on natural history data from more than 10,000 ALS patients from 12 specialized centers in Europe.
However, the FDA rejected the data because the patients in the database did not participate in clinical trials and there were many hidden dangers in the reliability of the analysis, such as external control or lack of pre-specified protocols
.

In addition, Amylyx has also tried to use placebo crossover design to estimate the survival benefit of
treatment.
The FDA labeled it not as new data, but as a new method of analysis of the same survival data in the original submission, concerning that Amylyx "relies heavily on unverified hypotheses
.
" Amylyx does not have best practices for implementing analytics, including considering other methods for sensitivity analysis
.

In addition, Amylyx has also tried to use placebo crossover design to estimate the survival benefit of
treatment.
The FDA labeled it not as new data, but as a new method of analysis of the same survival data in the original submission, concerning that Amylyx "relies heavily on unverified hypotheses
.
" Amylyx does not have best practices for implementing analytics, including considering other methods for sensitivity analysis
.

As for the biomarker results of the AD study, the FDA notes that the correlation with ALS is unclear — "even if they prove beneficial to AD, it is unclear whether they can be generalized to ALS.
"

As for the biomarker results of the AD study, the FDA notes that the correlation with ALS is unclear — "even if they prove beneficial to AD, it is unclear whether they can be generalized to ALS.
"

Adcomm's attitude has undergone an unusual shift, perhaps due to the strong demand
for treatment from the ALS patient population.

Adcomm's attitude has undergone an unusual shift, perhaps due to the strong demand
for treatment from the ALS patient population.

According to Amylyx, the Phase III study of AMX0035 won't yield results until 2024, and time is of the essence
for als patients with ALS who have only 3-5 years to live.
For example, the Als Association of the United States has previously said that AMX0035 has been shown to be relatively safe and can be administered orally, and for the "terminal disease" of ALS, patients prefer to do something rather than just wait
.

According to Amylyx, the Phase III study of AMX0035 won't yield results until 2024, and time is of the essence
for als patients with ALS who have only 3-5 years to live.
For example, the Als Association of the United States has previously said that AMX0035 has been shown to be relatively safe and can be administered orally, and for the "terminal disease" of ALS, patients prefer to do something rather than just wait
.

It is worth noting that based on the results of the Phase II trial, AMX0035 was conditionally approved for marketing
in Canada in June this year.
However, the FDA must be cautious, and the AD drug Aduhelm, which was partially forced by public opinion, is a lesson for the past
.

It is worth noting that based on the results of the Phase II trial, AMX0035 was conditionally approved for marketing
in Canada in June this year.
However, the FDA must be cautious, and the AD drug Aduhelm, which was partially forced by public opinion, is a lesson for the past
.

The PDUFA date for AMX0035 is September 29, will the FDA follow Adcomm's advice as usual? We can wait and see
.

The PDUFA date for AMX0035 is September 29, will the FDA follow Adcomm's advice as usual? We can wait and see
.