Drugs / Simufilam
Simufilam
also known as
PTI-125 · C-0105 · C0105M · Filora · PTI-125 DIHYDROCHLORIDE MONOHYDRATE · PTI-125 hydrochloride · PTI-910 · PTI-910 DIHYDROCHLORIDE MONOHYDRATE · +2 more
Small molecule targets FLNA via activation
Regulatory milestones approvals, filings & regulatory actions · 1 recorded
| Milestone | Jurisdiction | Brand | Indication | Date | Sentence it was read from |
|---|---|---|---|---|---|
| Clinical hold | US (FDA) | — | epilepsy, early-onset | — | “requests for additional information from the U.S. Food and Drug Administration (FDA) concerning the full clinical hold on our investigational new drug application (IND) for...” filanatx.com ↗ “full clinical hold on our investigational new drug application (IND) for simufilam in TSC-related epilepsy” filanatx.com ↗ |
Trials 9
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 12 trials | ||||||
| Phase 1 | NCT06390410 | Jun → Dec 2024 | liver disorder | Cassava Sciences, Inc. | Completed | No outcome recorded |
| Phase 1 | NCT06195319 | Feb → Mar 2024 | — | Cassava Sciences, Inc. | Completed | No outcome recorded |
| Phase 24 trials | ||||||
| Phase 2 | NCT05352763 | May 2022 → Dec 2024 | Alzheimer disease | Cassava Sciences, Inc. | Terminated | No outcome recorded Stop: Business |
| Phase 2 | NCT04388254 | Mar 2020 → Nov 2023 | Alzheimer disease | Cassava Sciences, Inc. | Completed | No outcome recorded |
| Phase 2 | NCT04079803 | Sep 2019 → Mar 2020 | Alzheimer disease | Cassava Sciences, Inc. | Completed | Mixed |
| Phase 2 | NCT03748706 | Mar → May 2019 | Alzheimer disease | Pain Therapeutics | Completed | No outcome recorded |
| Phase 33 trials · 2 missed | ||||||
| Phase 3 | NCT05575076 | Nov 2022 → Jan 2025 | Alzheimer disease | Cassava Sciences, Inc. | Terminated | No outcome recorded Stop: Business |
| Phase 3 | NCT05026177 | Nov 2021 → Dec 2024 | Alzheimer disease | Cassava Sciences, Inc. | Terminated | Missed primary Stop: Business |
| Phase 3 | NCT04994483 | Nov 2021 → Oct 2024 | Alzheimer disease | Cassava Sciences, Inc. | Completed | Missed primary |
| Comparator or background therapy1 trial | ||||||
| Phase 1 | NCT03784300 Comparator | Aug → Oct 2017 | early-onset autosomal dominant Alzheimer disease | Pain Therapeutics | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 5
| Date | Issuer | Release |
|---|---|---|
| 2026-04-15 | Pain Therapeutics | Results Filana Therapeutics Announces Publication in Epilepsia of Preclinical Simufilam Data filanatx.com ↗
today announced publication in Epilepsia of preclinical data showing that simufilam attenuated seizure progression in a well-accepted mouse model of severe TSC-related epilepsy 1 . |
| 2026-01-13 | Pain Therapeutics | Results Cassava Announces Publication of Peer-Reviewed Phase 3 Results for Simufilam in Alzheimer’s Disease in the Journal of Prevention of Alzheimer’s Disease filanatx.com ↗
While, as previously disclosed, the two studies did not meet their pre-specified co-primary, secondary, or exploratory biomarker endpoints, exploratory post-hoc analysis of the studies offers informative insights. |
| 2025-08-04 | Pain Therapeutics | Results Cassava Sciences Reports Positive Preclinical Study Evaluating Simufilam for TSC-Related Epilepsy filanatx.com ↗
today reported positive preclinical results of a study evaluating simufilam in a well-accepted mouse model of tuberous sclerosis complex (TSC)-related epilepsy. |
| 2025-06-30 | Pain Therapeutics | Results Cassava Presents Promising Preclinical Simufilam Data at TSC Alliance Meeting filanatx.com ↗
Treatment with simufilam alleviated neuronal abnormalities and reduced seizure frequency by 60% compared to vehicle in a mouse model of focal onset seizures. |
| 2025-03-25 | Pain Therapeutics | Results Cassava Sciences Reports Topline Phase 3 REFOCUS-ALZ Data filanatx.com ↗
Topline data indicate that REFOCUS-ALZ did not meet each of the prespecified co-primary, secondary and exploratory biomarker endpoints. |
All press releases naming this drug 5 releases
| Date | Issuer | Release |
|---|---|---|
| 2026-04-15 | Pain Therapeutics | Filana Therapeutics Announces Publication in Epilepsia of Preclinical Simufilam Data filanatx.com ↗ |
| 2026-01-13 | Pain Therapeutics | Cassava Announces Publication of Peer-Reviewed Phase 3 Results for Simufilam in Alzheimer’s Disease in the Journal of Prevention of Alzheimer’s Disease filanatx.com ↗ |
| 2025-08-04 | Pain Therapeutics | Cassava Sciences Reports Positive Preclinical Study Evaluating Simufilam for TSC-Related Epilepsy filanatx.com ↗ |
| 2025-06-30 | Pain Therapeutics | Cassava Presents Promising Preclinical Simufilam Data at TSC Alliance Meeting filanatx.com ↗ |
| 2025-03-25 | Pain Therapeutics | Cassava Sciences Reports Topline Phase 3 REFOCUS-ALZ Data filanatx.com ↗ |
Evidence & citations 15 cited values
Every value below carries the sentence it was read from. 11 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | Simufilam | “Simufilam (PTI-125) 100 mg oral tablets administered twice daily (BID)” NCT03748706 ↗ |
| Known as | C-0105 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650230 ↗ |
| Known as | C0105M | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650230 ↗ |
| Known as | Filora | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650230 ↗ |
| Known as | PTI-125 | “Simufilam (PTI-125) 100 mg oral tablets administered twice daily (BID)” NCT03748706 ↗ |
| Known as | PTI-125 DIHYDROCHLORIDE MONOHYDRATE | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650230 ↗ |
| Known as | PTI-125 hydrochloride | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650230 ↗ |
| Known as | PTI-910 | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650230 ↗ |
| Known as | PTI-910 DIHYDROCHLORIDE MONOHYDRATE | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650230 ↗ |
| Known as | PTI-910 hydrochloride | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650230 ↗ |
| Known as | SIMUFILAM HYDROCHLORIDE | ChEMBL registry synonym — accepted as the source's own label CHEMBL4650230 ↗ |
| Action | Activate | “Filamin-A positive modulator” CHEMBL4650230 ↗ |
| Modality | Small molecule | — CHEMBL4650230 ↗ |
| Route | Oral | “100 mg oral tablets administered twice daily (BID)” NCT03748706 ↗ |
| Target | FLNA | “Filamin-A positive modulator” CHEMBL4650230 ↗ |