PAA 0.00% 21.0¢ pharmaust limited

Ann: Monepantel Selected for Entry into HEALEY ALS Platform Trial, page-52

  1. 211 Posts.
    lightbulb Created with Sketch. 27
    So the first one, Zilucoplan did not need a phase 1, I think, as it was already safe for use in Myasthenia gravis patients. From the Healy website....

    "Zilucoplan, a new drug candidate developed by UCB (formerly Ra Pharmaceuticals), binds complement component 5 (C5) and inhibits tissue damage caused by pathological complement activation. Clinical data in a Phase 2 trial of zilucoplan in another neuromuscular condition generalized myasthenia gravis, demonstrated rapid, clinically meaningful and statistically significant improvements, as well as a favorable safety and tolerability profile. Zilucoplan has been designed for convenient “in-home” use by patients and is self-administered as a small volume, subcutaneous administration."


    For Verdiperstat:

    Between 2008 and 2013, AstraZeneca completed multiple Phase 1 studies of AZD3241/verdiperstat, and a Phase 2 safety and tolerability study in people with Parkinson’s disease (PD). In these trials, verdiperstat was reported to dose-proportionally reduce MPO activity in plasma (Tong et al., 2018).

    In July 2020, a Phase 2/3 trial began testing verdiperstat in ALS. This study is part of the Healey ALS platform trial, a multicenter clinical collaboration testing four different interventions in parallel against a common placebo group. As with all interventions in the Healey trial, verdiperstat was selected by an independent review board, based on evidence for the role of oxidative damage and microglia activation in ALS, including an observed elevation of TSPO-PET signals in ALS patients (Zürcher et al., 2015; Ratai et al., 2018). One hundred and sixty ALS patients were to receive 600 mg verdiperstat twice daily for 24 weeks. The primary outcome was disease progression on the ALS Functional Rating scale, with secondary outcomes spanning breathing function, muscle strength, and survival. The trial ended in April 2022, with a final enrollment of 167. On September 29, 2022, the company announced that verdiperstat had failed to show any difference from placebo on the primary or key secondary measures of disease progression (press release). Verdiperstat has been removed from the company pipeline.

    So again, it does not look like there were any phase 1 studies on ALS







 
watchlist Created with Sketch. Add PAA (ASX) to my watchlist
(20min delay)
Last
21.0¢
Change
0.000(0.00%)
Mkt cap ! $93.45M
Open High Low Value Volume
21.0¢ 21.5¢ 20.5¢ $106.7K 516.1K

Buyers (Bids)

No. Vol. Price($)
2 24033 20.5¢
 

Sellers (Offers)

Price($) Vol. No.
21.0¢ 55077 2
View Market Depth
Last trade - 15.22pm 22/07/2024 (20 minute delay) ?
PAA (ASX) Chart
arrow-down-2 Created with Sketch. arrow-down-2 Created with Sketch.