I thought I’d do a thread on a concrete example of using ML to discover a drug candidate about to enter Phase 2a for a stroke disorder:
@RecursionPharma’s $RXRX REC-994
It’s in this Circulation paper from 2015 and illustrates the seed that became $RXRX “phenomics” platform with Chris Gibson (CEO, co-founder) and his former academic mentor Dean Li (board & co-founder) as first/last authors
3. Q4: Possibly communication of PK modelling results – best outcome would be straightforward PK comparability (AUC[0-inf] 80-120%) – otherwise question is if FDA can be convinced by PD data showing similarity between DPs regarding efficacy-linked immunological markers
4. Possibly (Q1/2022?) communication around another Type A meeting prior to re-submitting BLA to double-check all issues are addressed
Oct 4, 2021 • 11 tweets • 3 min read
Update #ProventionBio#Provention $PRVB #teplizumab. For a summary of AdCom etc. see thread below. I am not involved with the company but have other conflicts of interest. All is based on public information and personal opinion.
Recap: Company has Breakthrough Therapy Designation and Priority Review, an AdCom vote 10-7 in favour for “delay of Type 1 diabetes #T1D”, and got a CRL stating CMC problems and failure to demonstrate PK/PD comparability between the studied Lilly-produced
Upcoming questions cont’d:
(4) If risk/benefit is acceptable, which target population is deemed suitable by the FDA? There were proponents of 3 options in EMDAC: (a) Stage 1 + 2, (b) Stage 2, or (c) Stage 2 + family Hx of T1D (= inclusion criteria of TN-10).
Aug 14, 2021 • 27 tweets • 6 min read
Thread on #ProventionBio#Provention $PRVB and their flagship #antiCD3 monoclonal antibody #teplizumab under #FDA consideration for the “delay of clinical #Type1Diabetes#T1D in at-risk individuals”. I have no connection to the company – but I do have COIs. No recommendations
or claims of accuracy, just a summary of the status quo and personal opinion. Assume I’m biased. All public information. Feel free to correct me. For references & links, contact me. Some pictures are copies from public PRVB presentations/FDA briefing docs.
