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Record W4392928088 · doi:10.1021/cen-10208-buscon3

An FDA-approved ALS drug doesn’t work. Now what?

2024· article· de· W4392928088 on OpenAlexaboutno aff
Rowan Walrath

Bibliographic record

VenueC&EN Global Enterprise · 2024
Typearticle
Languagede
FieldMedicine
TopicAmyotrophic Lateral Sclerosis Research
Canadian institutionsnot available
Fundersnot available
KeywordsDrugApproved drugDrug approvalMedicineBusinessPharmacology

Abstract

fetched live from OpenAlex

A Phase 3 trial wasn’t technically a condition for approval of Amylyx Pharmaceuticals’ Relyvrio, a drug for amyotrophic lateral sclerosis (ALS) that was greenlit in the US and Canada in 2022. The Food and Drug Administration and Health Canada both approved the drug based on a successful Phase 2 study. But there was pressure for a confirmatory trial, both from patient advocacy groups and from regulators. Billy Dunn, then the director of the FDA’s neuroscience office, went so far as to ask the company’s cofounders during a public meeting in 2022 if they’d be willing to pull their drug from the market, should it fail in a 664-person Phase 3 study that was already underway. The cofounders—Justin Klee and Joshua Cohen, who are also Amylyx’s co-CEOs—agreed. “If the drug’s not helping patients, then why would we want to be giving it to them?” Klee said at the time. But that’s

Fetched live from OpenAlex and de-inverted. Abstracts are not stored in this database: the inverted indexes are 8.6 GB of the frame’s 9.3 GB of text, and the host has 13 GB free.

How this classification was reachedexpand

Full frame machine prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. The Gemma side is a direct model label for every work in the frame, read from the title-only record. The Codex side is a classifier learned from the 10,348 direct Codex labels and calibrated to design-weighted sample rates; fields without enough sample support carry no Codex call. Candidate is the union of the two sides; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels.

metaresearch head score (Codex)0.009
metaresearch head score (Gemma)0.023
Version: metacan-v3-hybrid-931329e0061cValidation status: machine_predicted_unvalidated
Candidate categoriesnone
Consensus categoriesnone
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Not applicable · Consensus signal: Not applicable
GenreCandidate signal: Commentary · Consensus signal: Commentary
Teacher disagreement score0.031
Threshold uncertainty score0.103

Distilled classifier scores by category (both heads)

CategoryCodexGemma
Metaresearch0.0090.023
Meta-epidemiology (narrow)0.0010.000
Meta-epidemiology (broad)0.0010.001
Bibliometrics0.0010.001
Science and technology studies0.0030.004
Scholarly communication0.0060.008
Open science0.0010.002
Research integrity0.0110.013
Insufficient payload (model declined to judge)0.0310.017

Machine scores (provisional)

The two teacher heads of the student model, read on this work. A score orders the frame for review; it never asserts a category, and the validation status ships verbatim with every row.

Baseline scores from an immature model (maturity gate not passed, 7 training rounds). Scores rank; they never assert a category.

Opus teacher head0.012
GPT teacher head0.319
Teacher spread0.307 · how far apart the two teachers sit on this one work
Validation statusscore_only:v0-immature-baseline · verbatim from the scoring run: score_only means the number may rank works, and no category label ships from it

Classification

machine, unvalidated

Machine predicted; a candidate call from one source (direct Gemma or distilled Codex), not a consensus.

The models applied no category: nothing in the taxonomy fit this work.
Study designNot applicable
Domainnot available
GenreCommentary

How this classification was reached, model by model and score by score, is at the end of the page under "How this classification was reached".

Quick stats

Citations0
Published2024
Admission routes1
Has abstractyes

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