Below summary from Otter for the presentation:

PYC Investor Call

Transcript

Rohan Hockings outlined PYC Therapeutics' 2025 objectives, focusing on three clinical-stage assets. The company aims to demonstrate the translational ability of its preclinical work into life-changing patient impact. Key milestones include the RP11 program's Phase 1/2 extension to 24 months, FDA meetings in June, and the PKD program's Phase 1 study initiation. PYC raised $145 million and has a $200-$250 million cash runway. The company emphasized its high propensity for success in single-gene disorders, with a five-fold higher success rate in clinical development. The RP11 program's early data shows promising vision improvements, and the PKD program aims to assess safety, tolerability, and efficacy in both healthy volunteers and patients.

Action Items

• [ ] Conduct end-of-phase 2 meeting with FDA for the RP11 program in the first week of June 2025 to align on the design of the registrational study.
• [ ] Complete dosing of the first cohort of healthy volunteers in the PKD phase 1a study within April 2025.
• [ ] Present updates on the RP11 clinical data at the Foundation Fighting Blindness conference and the Association for Research Technology Conference in May 2025.
• [ ] Extend the RP11 phase 1/2 open-label extension study to a 24-month follow-up window to assess the durability of the improvement in vision.
• [ ] Transition the PKD phase 1a single ascending dose studies into an open-label extension and run a concurrent multiple ascending dose study to generate a larger pool of patient data.
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Outline

Introduction and Housekeeping Matters

• Rohan Hockings begins the meeting by thanking the team at PYC for their hard work and outlining the objectives for the day.
• The call is being recorded, and forward-looking statements are outlined, noting that actual results may differ materially.
• The first objective is to introduce the company to newcomers, and the second is to frame the vision for 2025 and the operational roadmap.
• The focus will be on the three clinical-stage assets and how data from the retinitis pigmentosa type 11 program will create commercial opportunities.

Overview of PYC Therapeutics

• Rohan Hockings explains that PYC is a drug discovery and development company focused on genetic medicines for patients with severe unmet needs.
• The company specializes in making drugs for patients with monogenic disorders, addressing the root cause of the disease.
• PYC has a high propensity for success in clinical development of drugs targeting single gene disorders, with a five times higher success rate.
• The company validates its human genetic medicines in human mini-organs before entering clinical development.

Impact of PYC's Work on Patients

• Rohan Hockings describes the profound impact of PYC's work on patients, using the example of polycystic kidneys removed from a patient due to end-stage renal failure.
• The company aims to stop disease progression and potentially reverse some disease processes in patients with sufficient tissue plasticity.
• PYC's focus is on high-impact medicines that can have a profound impact on the quality of life of patients with severe unmet needs.
• The company has progressed three of its four programs through clinical-stage development, with a high velocity path to market.

Financial and Clinical Development Updates

• Rohan Hockings provides an update on the company's financial status, mentioning a recent rights issue to raise $145 million and a cash runway of $200-$250 million.
• The company is well-capitalized with funding for 24 months of the forward part of the roadmap.
• Rohan discusses the importance of the upcoming clinical data from the RP 11 program, which is expected to demonstrate excellent clinical efficacy.
• The company is preparing for a meeting with the FDA in June to align on the design of the registrational study for the RP 11 program.

Operational Roadmap and Clinical Milestones

• Rohan Hockings outlines the operational roadmap for the next 12-18 months, focusing on the RP 11 and PKD programs.
• The RP 11 program will extend its open-label extension study to a 24-month follow-up to assess the duration of improvement in vision.
• The PKD program will transition into clinical development with the first cohort of healthy volunteers expected to be dosed in April.
• The company will evaluate safety, tolerability, and efficacy data in both healthy volunteers and PKD patients, with a focus on urinary biomarkers and kidney volume.

Business Development and Licensing Opportunities

• Rohan Hockings discusses the potential for business development and licensing deals, emphasizing the importance of strategic alignment, strong scientific hypotheses, and differentiated clinical data.
• The company is looking to extract substantial value from its assets through high-quality data that de-risks the proposition for counterparties.
• The focus is on creating clinical data packs that support the establishment of standard of care and market uptake.
• Rohan highlights the importance of demonstrating excellent clinical efficacy data to attract potential partners and investors.

Q&A Session and Closing Remarks

• Rohan Hockings addresses questions from the audience, discussing the company's plans for market recognition, FDA discussions, and competitive pressures.
• The company is actively engaging with institutional investors and maintaining strong levels of engagement with the investment community.
• Rohan emphasizes the importance of delivering clinical milestones on time to drive value recognition and support the company's growth.
• The meeting concludes with Rohan expressing excitement for the upcoming clinical data and the potential impact on the company's future.