Ann: PharmAust Presentation, page-82

ALS treatment pipeline, worth a read highlighted some key points below:

I have considered that the high rate of ALS treatment failures is due to the sporadic nature of ALS. Therefore, for a treatment to succeed it would need to be selectivity without off-target side effects. We know Monepantel has a high safety profile. Also the most recent HEALY platform regimens are both focused on TDP43 inhibition. Take this as a sign that the specialists agree this is an area of focus.

This bodes well for Monepantel, as an activator of Autophagy (natural cleansing process for protein aggregation), mTor inhibition (anti-inflammatory) and its downstream target (preventing TDP-43 aggregation along with other proteins linked to ALS).

Also, as highlighted by Pharmaust and many here, Monepantel has the potential to translate to other neurodegenerative disorders. Preclinicals inbound - Watch this Space!



Selective ALS Targets

Lots of downstream targets but TDP43, mtor and autophagy closer to the top of the list - interesting how does this sit with Monepantal?






READ ME - ALS PIPELINE!

Source: 14728214.2024.2333420 (tandfonline.com)

Healy Platform Trials (status)



Source:


1Study Drugs in the HEALEY ALS Platform Trial (massgeneral.org)2Zilucoplan Arm in HEALEY Trial for ALS Stopped Early for Lack of Benefit (alsnewstoday.com)3Biohaven Provides Update From Pivotal Phase 2/3 Trial with Verdiperstat in Amyotrophic Lateral Sclerosis (Healy ALS Platform Trial) (prnewswire.com)4Healey & AMG Center announces top line results in ALS platform trial with CMN-Au8 (massgeneral.org)5Phase 3 clinical trial of pridopidine in ALS patients likely this year (alsnewstoday.com)