PTC Therapeutics (PTCT) Update / Briefing Transcript

Summary of PTC Therapeutics (PTCT) Update / Briefing July 28, 2025 Company Overview - Company: PTC Therapeutics - Product: SUFIANCE (cepiaterin), a once-daily oral therapy for phenylketonuria (PKU) patients Key Points FDA Approval and Product Details - SUFIANCE received FDA approval for all disease subtypes and ages from one month and older, marking a pivotal moment for PTC and the PKU community [3][4] - The therapy demonstrated robust phenylalanine level reductions, allowing PKU patients to liberalize their restrictive diets [4][5] - There are approximately 17,000 PKU patients in the U.S., with over 300 new diagnoses annually through newborn screening [5][6] Market Opportunity - Despite two existing therapies, a significant unmet need remains, as most patients are not on any therapy [6] - The revenue opportunity for SUFIANCE in the U.S. is projected to exceed $1 billion due to its strong clinical data and ability to address all key patient segments [6][19] - PTC has established customer-facing teams experienced in commercializing rare disease therapies [7][9] Launch Strategy - The commercial launch is set for August, with a focus on engaging over 1,200 potential prescribers at 104 PKU Centers of Excellence [10][11] - Initial targeting will focus on patients who have failed existing therapies, accounting for approximately 40% of the PKU population [12][56] - PTC plans to utilize digital tools and social media to promote SUFIANCE and engage with the PKU community [12][13] Pricing and Access - The wholesale acquisition cost of SUFIANCE is $41,000 per month, based on an average patient weight of 45 kg [18] - PTC aims to ensure low barriers for access, with expectations of minimal prior authorization requirements from payers [16][19] - All commercially insured PKU patients can expect a $0 copay per month upon enrolling in the PTC care program [15][81] Patient Engagement and Support - The PTC Cares team will provide comprehensive support, including insurance verification and copay assistance [14][81] - PTC is committed to ensuring that all eligible patients receive access to SUFIANCE [14][19] Clinical Data and Efficacy - Clinical trials showed that 97% of patients could liberalize their diet, with significant reductions in phenylalanine levels [61][87] - The therapy is expected to be the new standard of care for PKU patients, with a focus on both pediatric and adult populations [20][56] Future Outlook - PTC anticipates contributions from international markets, including Europe and Japan, as part of its global launch strategy [36][94] - The company is prepared to track and share launch metrics, including patient uptake and payer dynamics, starting from the third quarter earnings call [19][70] Additional Insights - There is a strong emphasis on the importance of diet management alongside therapy, with healthcare providers expected to guide patients in diet liberalization [61][89] - The company has engaged with payers to ensure a favorable pricing strategy that reflects the therapy's value and efficacy [67][69] - PTC is focused on addressing the needs of both treatment-naive patients and those who have previously failed therapies, ensuring a comprehensive approach to patient care [38][56]