Exactly, imagine...well, there is no need for IMU to imagine they currently have data across many different cancer types at varying dosage levels. Gathering a HUGE amount of valuable data on how CF33 works in humans, its limitations, and what it's doing exceptionally well.
As
@Outlander2 puts it, the 'sandbox’ type trial is to determine where CF33 shines brightest within the shortest timeframe. From there, expansion cohorts for any cancer and delivery type that show efficacy. The playbook they are executing (expansion cohorts) is literally the FDA's own playbook.
Expansion Cohorts: Use in First-in-Human Clinical Trials to Expedite Development of Oncology Drugs and Biologics Guidance for Industry CF33 has been safe up to Cohort 6 thus far; however, needs more efficacy data to compare to SOC.
That's what the expansion cohort is all about. Within the context of bile duct cancer, it fits the bill for a rare unmet need, given that the current SOC is woeful. Thus, showing even modest efficacy will be significant, justifying a smaller, focused cohort. Coupled with fast-track, this expansion cohort need not finish before a hypothetical registration trail could commence or be put forward to the FDA.
Thi
s is the way I see it possibly playing out, assuming no issues with enrolment and efficacy is where it needs to be:- First Patient Dosed in Expansion Cohort: Month 0
- Initial Data Collection: Months 0-4
- Preliminary Data Analysis and Interim Analysis: Months 4-7
- Engagement with FDA (Type B Meeting): Months 7-9
- Breakthrough Therapy Designation Request and Decision: Months 9-10
- Planning and Finalising Registrational Trial Design: Months 10-14
- Initiation of Registrational Trial: Months 14-16
These are ambitious timelines, but there is precedent (Keytruda, Cretostimogene Grenadenorepvec (an OV), Tecentriq). With Fast track, excellent safety and surrogate endpoints, I believe they are achievable.
There is still much to be confirmed in the clinic, but if CF33 does what we all hope, we'll know within the next 12 months if we have something commercially viable.
Patiently waiting...
(20min delay)