Company: PTHS
Filing Date: 2025-03-27
Form Type: 10-K
Source: 0001753926-25-000503
Chunk: 686

Company: Pelthos Therapeutics Inc.
Filing Date: 2025-03-27
Form: 10-K
Item: Item 2
Chunk 686
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 through which the work will be conducted. The location of the POC has not been determined at this time, with availability
of facilities and patient population, costs, tax credits, centers of excellence in the respective fields (EM or iSFN) are all
factors in the ultimate determination of the location.

In
parallel with the dose escalation study, we expect to run a pilot efficacy study on approximately ten EM patients. In this study,
we will induce EM flares, determine baseline pain, and then dose escalate CC8464, after which, we will attempt to induce flares.
The primary endpoint will be the amount of pain experienced, and the secondary endpoint is a determination if CC8464 reduces the
frequency of EM flares.

We
are currently working on the development of the Phase 2a POC plan and expect to launch the Phase 2a POC study following the dose
escalation study and EM pilot study, to assess the potential efficacy of CC8464 in iSFN patients. Both of iSFN and EM are orphan
indications for which we plan to apply for orphan drug designations. The orphan indication may decrease the scope of the ultimate
development program that is necessary for approval and is associated with a marketing exclusivity period from the FDA along with
some tax advantages.

Though
the Phase 2a POC study design has not yet been completed, the study will take approximately twelve months after it is initiated.
The primary endpoint will be the amount of pain experienced from iSFN with secondary endpoints including other measurements like
pain relief and neuropathy scores. The final design may change based on feedback from regulatory authorities or information learned
during the dose escalation trial.

The
potential population for EM in the United States is estimated to be between 5,000 and 50,000 patients and the potential population
for ISFN in the United States is estimated to be between 20,000 and 80,000 patients. In both instances, we expect patients would
potentially take our drug for the remainder of their lives, and given the lack of good therapeutic alternatives, we expect to
have a robust, ongoing, and durable market.

The
Phase 2a results will have significance beyond EM and iSFN and provide important insights about NaV1.7 as a potential target to
find novel pain medications as an alternative to opioids, the continuing primary standard of care in analgesics. We believe that
positive results from the Phase 2a study could