The end of the press release describes the commercialization goals:
Based on the strength and durability of the clinical effect observed after a single infusion, NextCell’s primary objective is to bring ProTrans to patients as a single-infusion treatment, where the Company’s data is most extensive, and to pursue market approval on this basis.
In Finnish: Although the repeated dose seems to work, Nextcell aims to enter the market through a single dose first, as it is regulatorily easier and cheaper. Repeated treatments offer growth opportunities for later stages.
Following market approval, NextCell intends to extend its label with the addition of paediatric patients. The Company will also explore the possibility to further optimise the treatment regimen by evaluating repeated ProTrans infusions as a strategy to maximise and prolong the therapeutic effect in selected patient populations.
But wait a minute! Did Nextcell just radically change its strategy?
Previously, Phase 3 was intended to include a wide age distribution, which is why the “young” results must be awaited before Phase 3. According to the CEO’s Q&A section, this was also the EMA’s wish.
Now there is talk that “After obtaining market approval, NextCell intends to extend the market approval to also cover pediatric patients.”
Isn’t this in conflict with the previous statement? I think there is a good explanation for this: market approval is being sought first in Hong Kong or China, where it has been proven that conditional market approval could be obtained already with Phase 2 results.
However, the problem in Hong Kong is that you cannot apply for the first market approval there. Hong Kong does not have its own drug regulatory authority capable of evaluating a permit application, which is why they use the +1 mechanism. But according to information I just received, this is changing: the Hong Kong government has set a goal for “Primary Evaluation” capability in 2026. The goal is most likely to be an agile counterpart to Western bureaucracy that speeds up access to innovative drugs. (And honestly, the goal is surely to steal Western intellectual property rights).
‘The Chief Executive’s 2025 Policy Address’ announced that the Government will set up the Hong Kong Centre for Medical Products Regulation (CMPR) in 2026, and implement the ‘primary evaluation’ mechanism for new drug registration in phases starting from that year, enabling Hong Kong to independently assess and approve the safety and efficacy of medical products based on clinical data’
Recently, Nextcell even visited the Hong Kong embassy. Could it be that Nextcell is one of the first pilots for Hong Kong’s newly established CMPR?
If true: an interesting question is, could it be possible that Nextcell does not need to separately demonstrate the drug’s efficacy in the local population? And could Nextcell receive “orphan drug” status from the Hong Kong authority, which would allow it to apply for market approval with Phase 2 evidence, as was done in the case of the drug Fucaso? In that case, the first market approval could be very close indeed.