ProQR Therapeutics N.V. Ordinary SharesPRQR
Recorded
ProQR Therapeutics N.V. Ordinary Shares 12th Annual Cantor Fitzgerald Global Healthcare Conference
Review the key takeaways and the transcript of this earnings call.
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Key takeaways
- ProQR announced that their second RNA editing drug, AX811, has entered the clinic, following positive target engagement data for AX810 announced in June 2023.
- They have four RNA editing programs expected to enter the clinic in the near term, with five clinical trials expected to read out over the next 12 months.
- AX810 showed dose-dependent editing with 10 to 15 percent editing at the two lowest doses, while AX811 is projected to achieve between 40 and 65 percent editing based on modeling.
- AX810 and AX811 target NTCP to modulate bile acid levels, aiming to treat cholestatic diseases such as biliary atresia by increasing bile acid excretion and reducing liver damage.
- The company plans to host an investor event at the end of September 2023 to discuss AX811 clinical study expectations and review AX810 data.
- AX422, targeting NPS1 (Hürrler syndrome), will enter the clinic in the next few months with initial data expected in the first half of 2024.
- AX2911, targeting PNPLA3 for lean MASH, will enter an investigator-initiated trial (IIT) with first data expected in the first half of 2024.
- ProQR plans a phase two study in biliary atresia starting mid-2024, potentially pivotal depending on regulatory discussions, with an interim readout expected in mid-2028.
- The company differentiates its RNA editing approach from small molecule NTCP inhibitors, highlighting potential safety advantages and focusing on conjugated bile acids as key disease drivers.
- For the NPS1 program, ProQR plans systemic subcutaneous dosing and intra-articular administration to treat both systemic and CNS aspects of the disease, aiming to reduce treatment burden compared to current enzyme replacement therapies.
- The RET program is staged behind the NPS1 program to leverage learnings and de-risk clinical development.
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