Hi OutofboundsThere reallyis no short answer, but it lies in the...

Hi Outofbounds

There reallyis no short answer, but it lies in the AA and new BTD processes that the FDAnow uses for ‘critical need’ treatments (eg. cancer ,etc).

To meet thesubstantial need for oncology therapeutics, the FDA’s Oncology Center of Excellence launched a pilot program for oncology therapeutics in 2018, the Real-Time Oncology Review (RTOR). The goal of the RTOR program is to review applications for approval efficiently “to ensure that safe and effective treatments areavailable to patients as early as possible”. Applications made under this program must meet certain criteria regarding the clinical trial design and development program, such as evidence of prominent advantages over othertherapeutics, straightforward study designs, and easily interpretable endpoints.

The AA andBTD processes are outlined below. IMU may well become a beneficiary of either or both.

Acceleratedapproval (AA) targets candidates that are similar to those awarded BTD interms of qualifying characteristics, such as aiming to treat serious conditions and providing improvements over current therapy. Most importantly, however, AA allows drugs to be approved using surrogate endpoints that are reasonably expected to predict clinical benefits. For example, for some cancer drugs,progression free survival (PFS) may be an effective surrogate endpoint that canbe assessed instead of overall survival (OS), which may take much more time to determine, thus delaying the approval of a novel therapy for patients who have the potential to benefit. (in other words, some of IMU’s virus treatments may only need to complete Phase 2 prior to approval. This really speeds up the process). There is, however, reasonable room for error inextrapolating PFS findings to OS. Consequently, confirmatory trials such asphase 3 double-blind controlled trials are necessary to prove further the efficacy and safety of drugs that have been awarded accelerated approval. These trials must be carried out within a certain time window, after which, the drug may be withdrawn from the market if not determined to be acceptable.

Breakthrough therapy designation (BTD) is granted for drugs aimed at treating serious orlife-threatening diseases for which there is promising preliminary clinicalevidence that shows superiority over current therapies. BTD does not guarantee future positive results in trials or successful approval, given that this designation is based only on preliminary data. As additional data are acquired, a BTD drug may no longer show superiority over current drugs and thedesignation may be rescinded to conserve agency resources. Preliminary clinical data are expected to be provided with the investigational new drug(IND) application or no later than the phase 2 meeting, and these data must demonstrate the superiority of the new drug by direct comparison with the current therapy or with valid historical data. Drugs granted BTD receive greater scrutiny and support from the regulatory agency through rolling review,organizational commitment, and guidance on efficient drug development.

So, you cansee how certain treatments are able to avoid (initially at least) the huge and time-consuming Phase 3 trails and apply for approval after Phase 2. I believe IMU has several treatments that could easily fall into this situation, ieCHECKvacc, VAXINIA, onCARlytics and even HERvaxx which has ticked off Phase 2and is now in combo trials.

Hope this clarifies my comments a bit for you and, as always, I am happy to take counter arguments.

As always, my opinion only so please do your own research.