Notes from today's AGM VP-001 in RP11 Program Meeting with FDA...

Notes from today's AGM

VP-001 in RP11 Program

Meeting with FDA in late Q1 or early Q2 25 to discuss pivotal trial design including agreement on endpoints.

Aiming to commence Phase 3 trial mid-next year. Will be a global trial.

There won’t be progressive reporting of efficacy data from the Phase 3 trial but, as patients currently in SAD and MAD trials will be able to continue in extension studies lasting 12 months or more, it will be possible to report ongoing results for these patients.

Feedback from Principal Investigators that multiple patients are reporting functional improvements.

Growing interest in trial participation.

Will be applying for Rare Paediatric Disease designation for this asset which can possibly lead to a saleable Priority Review Voucher (Neuren’s has just sold for US$150m), if the drug is approved.

(My note: If PYC commences a 2 year pivotal trial mid-next year and completes in mid-2027 it will have taken just 4 years to complete the full clinical program.  In the world of drug development, this would represent amazingly fast progress. By way of comparison, Percheron’s antisense drug program in DMD commenced clinical trials in mid-2018 and is likely to have full Phase 2b trial results by mid-next year – almost 7 years later. It is then up to the FDA as to whether it will accept this data for accelerated review. As another example,Neuren took 10 years to complete its clinical trial program for trofinetide in Rett syndrome.)


PYC-001 in ADOA Program

Potential for early efficacy data from mid-next year.


PYC-001 in Glaucoma

Company has conducted a mouse model study of PYC-001 in glaucoma. Data needs to be built out and will be shared next year.

Possibility of entering Phase 2 in 2026.


PYC-003 in ADPKD Program

This program has already attracted a great deal of interest from potential partners. Company wants to advance the program through to at least proof-of-concept to maximise value prior to partnering. Said that company does not have the resources to take a drug like this through to market.

(My note: This is a condition with a very high patient population – approx. 12.5 m sufferers globally. Expect any Phase 3 trial to be very large; e.g. the Phase 3 trial for Tolvaptan recruited 1450 patients globally. Hence, if Phase 2 data is favourable, it would make sense for PYC to partner this asset prior to Phase 3.)

PYC needs to first prove in an NHP study that it can deliver therapeutic doses of the drug into the liver.

Potential partners also want evidence that the drug is trafficked to those cells in the liver in which cysts are formed.


PYC-002 in Phelan McDermid syndrome

Data and drug candidate selection to be announced late next month with IND-enabling studies to occur next year.

Already have “a beautiful data set”.

Stated that there is a lot of interest in CNS assets even at very early stage.

Intention to also apply for Rare Paediatric Disease designation for this asset.


AI Driven Drug Discovery Collaboration with Google Cloud

12 month term expires at end of this year. Likely extension.


Near-term events

Regulatory submission to commence ADPKD clinical program expected next month.

Nomination of candidate drug for Phelan McDermid syndrome (along with release of preclinical data) to occur late next month.