Appie Ananas

just my own opinions

[Appie Ananas @AppieAnanas]

The key takeaway isn't just Rett syndrome—it's that Axiomer continues to show potential across liver, CNS and rare disease programs, reinforcing the platform's versatility.

Still early, but another encouraging proof point for ADAR-mediated RNA editing. #biotech #RNAediting $prqr

[Appie Ananas @AppieAnanas]

New preclinical data from $PRQR further strengthen the Axiomer platform story.

AX-2402 restored MeCP2 protein levels in Rett syndrome neurons and demonstrated sustained functional improvement in a severe Rett mouse model.

[Appie Ananas @AppieAnanas]

@hannesokj Don’t know, only Lily knows.

[Appie Ananas @AppieAnanas]

Following the updated corporate presentation, a few things stood out to me about $PRQR.

[Appie Ananas @AppieAnanas]

@hannesokj If Lilly were to want to acquire it fully now it would pay for a great deal of uncertainty.

If Lilly waits until mid-2027, there will likely be:

AX-0811 data
IDUA data
PNPLA3 data
preparation for phase 2

That provides a more complete picture, but possibly also a higher price

[Appie Ananas @AppieAnanas]

If Lilly concludes that the combination of clinically validated RNA editing + AI-driven EON design + a broad pipeline is strategically unique, the risk may arise that waiting becomes more expensive than acting now. $PRQR

[Appie Ananas @AppieAnanas]

7/ Whether that ultimately leads to a larger collaboration or even an acquisition remains to be seen.

But yesterday's messaging is clearly focused on building long-term platform value rather than celebrating a single clinical result.

[Appie Ananas @AppieAnanas]

10/ The market currently seems focused on dilution and near-term financing.
But after listening to the update, I think the more important question is:
"Did ProQR just clinically validate an RNA editing platform?"
If yes, today's valuation may eventually look very different.

[Appie Ananas @AppieAnanas]

1/ ProQR ($PRQR) is down ~22% after announcing the first clinical validation of its Axiomer RNA editing platform.
After reading the full presentation and Q&A, I think the market may be missing some important details.

[Appie Ananas @AppieAnanas]

9/ Timeline from here:
• AX-0811 Phase 1 target engagement data: YE 2026
• Initial biliary atresia patient data: H1 2027
• AX-0422 and AX-2911 data: H1 2027
• Phase 2 interim analysis: Mid-2028
Multiple catalysts ahead.