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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 distilled prediction

Teacher imitation

Not calibrated prevalence, not ground truth. Human validation pending. Learned from the 10,348 direct Codex labels and 10,348 direct Gemma labels. Candidate is the union of thresholded teacher heads; consensus is their intersection. These outputs are machine_predicted_unvalidated and are not human labels or direct frontier model labels.

metaresearch head score (Codex)0.001
metaresearch head score (Gemma)0.000
Version: codex-gemma-dda1882f352aValidation status: machine_predicted_unvalidated
Candidate categoriesMeta-epidemiology (narrow), Scholarly communication, Insufficient payload (model declined to judge)
Consensus categoriesInsufficient payload (model declined to judge)
DomainCandidate signal: none · Consensus signal: none
Study designCandidate signal: Not applicable · Consensus signal: Not applicable
GenreCandidate signal: Empirical · Consensus signal: Empirical
Teacher disagreement score0.437
Threshold uncertainty score1.000

Codex and Gemma teacher scores by category

CategoryCodexGemma
Metaresearch0.0010.000
Meta-epidemiology (narrow)0.0010.001
Meta-epidemiology (broad)0.0010.001
Bibliometrics0.0000.001
Science and technology studies0.0000.000
Scholarly communication0.0020.002
Open science0.0010.001
Research integrity0.0010.001
Insufficient payload (model declined to judge)0.0030.017

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; both teacher heads agree on what is shown here.

Study designNot applicable
Domainnot available
GenreEmpirical

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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Same venueC&EN Global EnterpriseSame topicAmyotrophic Lateral Sclerosis ResearchFrench-language works237,207