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Partially active disease is incomplete clinical remission, defined as incomplete clinical response with CRP or SAA levels improved by ≥ 50% over Baseline, but still elevated. Incomplete clinical response is defined as IGA score specific at the time of consultation of ≤ 3 and > 50% improvement over Baseline and IGA general assessment improvement by at least one category (e.g., moderate to mild) - Number of participants with active disease at Week 12 for participants receiving single daily dose of IZD174 and participants receiving twice daily dose of IZD174 Active disease is no clinical remission, defined as no clinical response with CRP or SAA elevated. No clinical response is defined as no improvement or worsening of the clinical symptoms and IGA general assessment unchanged or worsened. - Duration of clinical remission Time from onset of remission until loss of complete clinical remission. Loss of Complete clinical remission is defined as: IGA score specific at the time of consultation > 3 and IGA general assessment worse than minimal with CRP or SAA > ULN or IGA score specific at the time of consultation > 3 and IGA general assessment worse than minimal or CRP or SAA > ULN (with no evidence of other cause of CRP/SAA rise such as infection or trauma) - Time to flare The time from stopping of study drug until flare. For the purpose of this endpoint, flare is defined as loss of complete clinical remission with CRP or SAA levels to be rising on 2 successive samples at the stopping of study drug. |
| DFV890 (IFM-2427), Novartis P2, FCAS, NCT04868968 |
NLRP3 inhibitor (Marianthi) (PFR-4231-100)
Executive summary
| LGE (202102) | PE (Dec, 2021) | CN (202210) ☐ CN | PDE: 3/21 Philip von Rosenstiel, Anne heatherington | CS (202409) RSLT | CDE (PRC) Dec2023→ 202402? → 202507 | IND (202508 ← 202410) | pH? (202509 / 202411) | ||
|---|---|---|---|---|---|---|---|---|---|
|
Nlrp3 □ NLRP3 Timeline toward CS_202209.pptx □ NLRP3 Inhibitor Global Team_2022.pptx 0928: TMQB September 2022 1005: AQB1 1025: RSLT 15 min: hypothesis/target validation/pharmacology 10 min: chemistry/safety 10 min: BM strategy □ Meeting 24-25October2022 중점: andy, ann heatherington | |||||||||
| In vitro | 다음 둘 필요 i) human iPSC microglia ii) microglia from PD patients | 202501-03 | |||||||
| TR06802042 (5c) | acieved | 202406 | CS 에 넣겠지 | ||||||
| In vitro | TR06851074 (5c) | ||||||||
| In vivo | 안 넣기로, | Tox | |||||||
The molecular weight of the homologous cold compound is : MW: 370.39 (Da이겠지.)
| Feb | Mar | Apr | May (early) | June (Late) | July | Aug | Sep | Oct | Nov | Dec | 1 | 2 | 3 | 4 | 5 | ||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| TR06693098 | NonGLP 14 d tox result | ||||||||||||||||
| CVB | 14C label compound | CVB completion | w1 result (<10mg/day & negative?) | PQR 9/21 (1.098 & 6602.5c, 3. BM) | CN | PDE 3/21 | |||||||||||
| TR06698660 | AMES (mid) | CN ☐ CN-TR660 | 14C-C-synthesis | Dog CV result? | |||||||||||||
| 2w rat tox start | FTO: Tuojie | (6/30) 2w rat tox draft result | micronization / micronization | AAV Rat start | AAV Rat start-TH | ||||||||||||
| CVB synthesis | Dog SAD | ||||||||||||||||
| 2nd atuka | TH, BM, NeuN start | aSyn result ongoing | |||||||||||||||
| Bz-ATP Rat | Small BzATP study (5 sham, 5 BzATP) to collect CSF for assay feasibility test. 남는건 proteina, ASC spec, Cosomil CN | ongoing | |||||||||||||||
| series 5c TR06798980 |
AV2/5 (WT) Sy Rat injection at 5-ge, mpling at 7- age. 1. AAV2/5-EV + vehicle 2. AAV2/5-hα-Syn + vehicle 3. AAV2/5-hα-Syn + 1 mg/kg TR06798980 4. AAV2/5-hα-Syn + 3mg/kg TR06798980 5. AAV2/5-α-Syn + 10mg/kg TR06798980 Fischer (F344) 4-5 mo. of age 18 rats per group bilateral injection of AAV-human alpha-SYN in the SNpc | ||||||||||||||||
| API delivery | Start → | CSF/Plasma collected | WB result CSF/Plasma Results | Histology Result: TH & HuC, CSF/Plasma Results | |||||||||||||
| Katy start assay (NFL 3 µL), Casp1 activity (16 µL), total Casp1 ELISA (10 µL), IL-18 (25 µL) | |||||||||||||||||
| RAT formulation pk study | Rat SAD tox (start Oct2) | Rat SAD tox report. Rat 2w... | |||||||||||||||
Uncertain Spans
| location | transcription | uncertainty |
|---|---|---|
executive summary header columns | the column headers LGE (202102) / PE (Dec, 2021) / CN (202210) ☐ CN / PDE: 3/21 / CS (202409) RSLT / CDE (PRC) Dec2023→ 202402? → 202507 / IND (202508 ← 202410) / pH? (202509 / 202411) are reconstructed from the partial text visible across body_r01_c01.jpg and body_r01_c02.jpg; column placement and field-vs-content boundaries are partly clipped. | low confidence on column header / field placement. |
executive summary / TR06802042 row / acieved cell | reads acieved (likely a typo for achieved); preserved verbatim. | source typography preserved. |
Pipeline CAPS Gantt grid / month resolution | the column header strip uses Feb/Mar/Apr/May (early)/June (Late)/July/Aug/Sep/Oct/Nov/Dec then numeric 1 / 2 / 3 / 4 / 5 columns at the right; per-cell column placement is collapsed into reading order rather than month-by-month placement. | column placement marked uncertain; row content captured but month alignment is approximate. |
Pipeline CAPS / TR06693098 sub-row label | the third sub-row label reads Sep PQR 9/21 (1.098 & 6602.5c, 3. BM); the 1.098 & 6602.5c, 3. BM token cluster is partly clipped and may be an internal compound code list. | low confidence on sub-token boundaries. |
TR06798980 row drug series numbering | reads 5. AAV2/5-α-Syn + 10mg/kg TR06798980; the leading α-Syn lacks the prefix h present in rows 2-4 (hα-Syn); preserved verbatim. | low confidence on whether the prefix omission is intentional. |