PYC Reports $49M Cash, Key FDA Designations, and Clinical Milestones in Q4 2024

• Phase 1/2 clinical data presented for Retinitis Pigmentosa type 11 showing patient improvement
• FDA grants Orphan Drug and Rare Pediatric Disease designations for RP11 candidate
• First dosing completed in Autosomal Dominant Optic Atrophy clinical trial
• Pre-clinical studies completed and regulatory submission made for Polycystic Kidney Disease program
• Clinical candidate nominated for Phelan-McDermid Syndrome following successful pre-clinical results

Clinical Progress Across Four Genetic Disease Programs

PYC Therapeutics, a clinical-stage biotech focused on RNA therapies for genetic diseases, has delivered a robust update for Q4 2024, highlighting material advancements across its pipeline. The company is developing precision medicines targeting monogenic disorders with high unmet need and limited treatment options.

In Retinitis Pigmentosa type 11 (RP11), a rare childhood blinding disease, PYC presented encouraging Phase 1/2 data at an international conference, demonstrating patient improvements on registrational endpoints. This progress underpins plans to initiate a registrational trial in the second half of 2025, pending FDA alignment. The RP11 candidate has also secured Orphan Drug and Rare Pediatric Disease designations from the FDA, which provide regulatory and commercial advantages including accelerated review pathways.

For Autosomal Dominant Optic Atrophy (ADOA), PYC achieved a significant milestone by dosing the first patient in a Phase 1/2 study with PYC-001, the first investigational drug candidate addressing the disease’s underlying cause to reach this stage. The company expects safety data in the first half of 2025, followed by early efficacy results later in the year.

Advancing Kidney and Neurodevelopmental Programs

In the Polycystic Kidney Disease (PKD) program, PYC completed pre-clinical studies and submitted a regulatory application to commence human trials, anticipated to start in the first half of 2025. PKD is a prevalent, life-threatening genetic disorder, and PYC’s candidate holds promise as a disease-modifying therapy, a critical unmet need in nephrology.

The Phelan-McDermid Syndrome (PMS) program, targeting a severe neurodevelopmental disorder, reached a key pre-clinical milestone with nomination of a clinical candidate. PYC plans to initiate Investigational New Drug (IND)-enabling studies in 2025, aiming for clinical trials in 2026. The company draws confidence from similarities in chemistry, administration, and target tissues with other successful RNA therapeutics.

Financial Position and Outlook

Financially, PYC ended the quarter with $49.3 million in cash, bolstered by a $17.3 million R&D tax rebate. Operating cash flow was positive for the quarter, reflecting ongoing clinical and discovery activities. The company’s four-year operational roadmap remains on track, with multiple regulatory and clinical milestones slated for 2025 and beyond.

Overall, PYC Therapeutics is positioning itself for a transformative year ahead, with its RNA-based precision medicines advancing steadily through clinical development and regulatory engagement. The company’s focus on rare genetic diseases with high unmet needs aligns well with emerging trends in biotechnology and personalized medicine.

Bottom Line?

PYC’s Q4 momentum sets a promising stage for pivotal trials and regulatory milestones in 2025, but clinical and regulatory risks remain key watchpoints.

Questions in the middle?

• Will PYC’s RP11 registrational trial design align smoothly with FDA expectations in upcoming Q2 discussions?
• How will early safety and efficacy data from the ADOA trial influence investor confidence and development timelines?
• What are the potential regulatory hurdles for initiating first-in-human trials in the PKD and PMS programs?