It was wonderful to attend my first PAA investor briefing in Sydney today and met some lovely folks from HC. I think there were about 20 attendees and the session was quite open and lively.
Mgmt: MT showed how he has been strategic right from applying for the HEALEY platform, the benefits of it to a small company like us, the global focus it brings etc.,. So needless to say that he is extremely pleased that this has now materialised.
He also explained how they are planning ahead such as the patents situation as mentioned below, funding, onboarding key personnel/ consultants in all capacities.
Patents- method of use for neuro degenerative diseases, not just for ALS. This patent is very valuable given MPL molecule patent licensed it from Elanco. In discussions with Elanco for their MPL patent.
SPP is going well (I am downplaying the comments!)
Katie McFarlane - in-depth connection to US BP. Has done commercial assessment before.
Chronic conditions- 5-10 year treatments, not just one-off; long term revenues at about $100k per annum with about 300k patients worldwide.
Healey platform:
Will know more about HEALEY trial cost in the next few months.
24 week interim analysis in Healey- potential accelerated approval.
5 of the Healey has been completed and reported on. Only 1 (Denali, Sanofi) was successful, only marginally.
All participants on Healey have different MOAs- we do autophagy via mTOR1 pathway. So that is the unique aspect.
UCB got acquired after being on boarded into Healey for $2Bn after than phase 1 (implying that a partnership/ licensing/ M&A could be on the cards anytime).
For FDA accelerated approval we need to have started phase 3 study- that’s why the adaptive part is important (phase 2 for 24 weeks+ same patients likely will continue to phase 3 for 24 weeks). FDA accelerated approval request will be made after meeting phase 2 results if the endpoints and FDA expectations (substantial clinical benefit) are met.
Interim readouts by 2025 end. FDA accelerated approval by mid-2026
I may have missed or misunderstood somethings. I am sure others attending would correct me.
(20min delay)