The next logical step is that the Drug Supply Agreement with Otsuka (Astex) progresses to a Collaboration and licensing arrangement.
We know that the Extramedullary AML and MDS Phase 2 trial currently underway is Race's pathway to approval. Also, Race have advised that approval off the back of Phase 2 data is a possible avenue, without needing to undertake a costly Phase 3 trial.
Link:
Investors – Race Oncology Recent Progress update, where is this all headed?...
A couple of observations:
Phase 2 readouts may be available in coming months, as per
@wombat777 speculation on the timeline could be from October 2022 -> April 2023, which doesn't seem unreasonable but subject to recruitment.
Given the trial is open label, readouts should identify whether Zantrene works as a single agent, synergy in EMD AML & MDS with ASTX727.
Based on recent mouse models and in-human historical AML and EMD AML results, the program in my view is derisked. It is just the ASTX727 and Zantrene combo that needs to be firmed up in-human data, which I am not aware of historical data (stand to be corrected).
Why would Otsuka take up Zantrene for EMD AML & MDS. A number of reasons:
1. Sales of circa $250m USD that they want to protect using improved SoC and extent patent thicket.
https://pink.pharmaintelligence.informa.com/PS003003/MGISuperGen-Estimate-Dacogen-Peak-Sales-Of-250-Mil-Action-Date-Is-Sept-12. Otsuka's focus on treatments for patients who can not tolerate high intensity chemo, and complementing Phase 3 program currently underway.
Astex announces that the phase 3 ASCERTAIN – acute myeloid leukemia (AML) clinical study of oral hypomethylating agent decitabine and cedazuridine fixed-dose combination (ASTX727 or DEC-C) met the trial’s primary endpoint – Astex3. Risk reward benefit, if structured as upfront payments with ongoing milestone adds value to Race and Otsuka without taking significant risk from both sides. Everyone's a winner.
4. Currently no approved SoC treatment for EMD AML, despite 25% of AML presenting with EMD AML. Furthermore, I understand that Zantrene has better penetration that ASTX727 and also boosts / synergizes due to upregulation of FTO from ASTX727 treatments.
Otsuka could choose not to extend their hand beyond the drug supply agreement but this seems unlikely except for a failure in the Phase 2 trial. Race's style is to fail in the lab rather than in the clinic and also we have backing of 1,500+ patients and 60 previous trials. Also, two French ladies cured from AML using Zantrene in the past!
Race / Otsuka would in my view have what they need to make a decision on how to move forward.
Link:
Investor-Presentation-November-2021.pdf (raceoncology.com)Assuming a modest deal, 20% upfront 80% milestone, x5 peak sales of $300m USD not JPY would be multiples of current market cap - even if risk adjusted.
That's only AML which is just the toe in the water for Zantrene with FTO and Cardio Protect programs being blockbusters in their own right.
No expert, so interesting to see what eventuates, but upcoming readouts should see what direction the EMD AML program is likely to take this financial year.
(20min delay)