PYC Therapeutics管线进展及人体数据发布筹备投资者会议纪要

发布时间：2026-09-04 一、AI总结内容 一、核心要点 1. PYC Therapeutics主营基于RNA技术的精准药物，聚焦4种严重未满足需求的疾病，计划将4种候选药物全部推进至临床开发，第四种将于2027年进入临床。 2. 公司近期调整高管团队，任命首席财务官、公司法务官已到位，首席临床开发官、高级临床开发人员将于2026年9月底入职，未来将扩充美国团队，新增监管事务主管、眼科项目负责人岗位。 3. 核心管线进展： - Polycystic Kidney Disease（多囊肾病）项目：处于1b期临床试验，24例患者中已招募13例，计划2026年11月完成前2个队列各12例患者招募，2027年6、9、12月将公布单剂量、多剂量及12个月总肾体积（TKV）数据，该数据将用于支持注册试验设计，美国FDA已认可TKV作为加速批准的替代终点。 - PYC002项目：针对多囊肾病的新药研究申请（IND）相关研究正在进行，与FDA沟通后，原计划的单剂量研究调整为直接开展重复剂量研究，预计2027年中提交IND，不会影响整体开发进度。- 两项眼科项目处于1/2期试验阶段，其中ADOA项目在2026年9月初公布的数据显示安全性及早期疗效信号良好，视觉 acuity改善显著，相关数据将用于支撑注册试验设计。二、风险与关注 1. 研发风险：包括临床试验招募进度、尿PC1蛋白检测分析难度大尚未公布数据、眼科项目需满足FDA15字母视力改善的高获批标准、多囊肾病项目需应对同类药物（如Regulus）的竞争。 2. 临床开发复杂性：多囊肾病注册试验需同时考虑总肾体积（TKV）和肾小球滤过率（EGFR）两个终点，需与FDA沟通试验设计细节。 3. 行业竞争：RNA疗法领域竞争加剧，其他公司（如Regulus、Nevitas）在同类疾病管线推进更快，PYC需快速推进数据发布以确立差异化优势。 三、后续关键节点 1. 2026年10月公布多囊肾病单剂量研究的安全性数据及初步疗效信号，11月召开年度股东大会。 2. 2027年重点公布多囊肾病多剂量研究的6、9、12个月数据，以及眼科项目的关键疗效数据，用于支持注册试验决策。 3. 2026年底前完成多囊肾病1b期前两个队列招募，2027年中提交PYC002的IND申请。 二、音频原文（转写） Good morning everyone and welcome to the p y c therapeutics third quarter investor webinar my name is rowan hockings andi'm the managing director at p y c i will be your host for this morning's call but before you begin i would like to readthe following safe harper statementreminding yo u that today's discussion will contain forward looking statements that involve risks and uncertainties these risks and uncertainties are outlined in our filings with the australian securitiesexchange as such actual results may differ materially from what we will discuss on today's callwe disclaim any obligation or intention to update these statements in the future and also like to remind you that today's call isbeing recordedbefore we begin i would like to also thank the py c team there are many people within the team who are putting in an exceptional effort to maintain the organizational cadence that allows me tostand up and have the opportunity to present toyou meaningful progress across all four of thecompany's pipeline programs and i am very grateful to those who areleaning in to maintain that organizational momentum.There's really only one objective for today and that's to give you as much transparency as possible into upcoming human data readouts i have split out a particular deepdive on the polycistic kidney disease program because that program is in a particularly interesting eighteen month window as we look to transition that drug candidate intoa series of milestones that are coming over the next eighteen monthsthat i would really like to engage with you on to day to try and give you the benefit of the thinking that we've done in relation to what is coming what to expect and whyprior to doing that iwill give a brief introduction to the company for those who are new to the story and i will alsodescribe the changes that are being made at theorganizational level to prepare the company forthis transition that sits in front of us to multiple assets into late stage clinical developmentat the end we'll open up for q and a and give you a chance to go deeper on topics of particularinterest to you.So i see designs and develops precision medicines for patients who have severeunmet needs and is our raisond'etre we exist for patients like sierra who have a mutation in onecopy of one gene in the bodyparticular approachthat we have taken to these diseases is to design an r n a therapy that does nothing to the effect of allele you are made up of twenty thousandgenes and have two copies of each of those genes but rather engages with the one remaining goodcopy of the gene at the r n a level in a regulatory region to stabilise that transand tell thatgene that copy of the gene to make two units ofprotein where it otherwise would have made one.These are what we call disease modifying drugs that address a disease at its root cause and therefore hold the greatest potential for impactingthe phenotype or the way that that disease is manifesting in patience so these are the types ofdrugs that if we or any of our family members had any one of the four indications that we're looking at we would want to have available.We haveused that strategy coupled with the platform tofacilitate delivery of the rna therapies acknowledging that delivery is the primary challenge for this modality to create a pipeline of four investigational drug candidates we have taken fourof those drug candidates into clinical development at different stages as you see on the screenin frontw and we will bring the fourth candidateinto the clinic next year.You can see here thatwe are now at what they call the turning of thecards for those who have been on the journey we've described previously we're going to go intoa lot of depth at the human efficacy data that constitutes clinical proof of concept accompaniedby the all important safety data that marks thetransition between the phase one two studies and the registrational trials that followthe ambition of the organization in the near term is nowto p