Yahoo Finance PTC Therapeutics Inc (PTCT) Q1 2024 Earnings Call Transcript Highlights: Strong Revenue Growth ...
Total Revenue: $210 million in Q1 2024.
DMD Franchise Revenue: $161 million in Q1 2024.
Translarna Revenue: $104 million in Q1 2024.
Emflaza Revenue: $57 million in Q1 2024.
Evrysdi Royalty Revenue: $31 million in Q1 2024 from Roche.
Non-GAAP R&D Expense: $107 million in Q1 2024, down from $180 million in Q1 2023.
Non-GAAP SG&A Expense: $64 million in Q1 2024, down from $73 million in Q1 2023.
Cash and Equivalents: $885 million as of March 31, 2024.
Release Date: April 25, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Q & A Highlights
Q: On PKU, is there a specific market segment where you'll see the uptake being the strongest? A: Matthew Klein, Independent Director at PTC Therapeutics, emphasized the strong data set from APHENITY and the long-term extension, which positions the company well to address all key market segments, seeing this as a $1 billion-plus opportunity. Kylie O'Keefe, Chief Commercial Officer, added that the near-term focus post-launch would be on therapy-naive patients, those who have classical PKU, high unmet medical need, previously failed on Kuvan, and those poorly controlled.
Q: What work has been done around Europe as well as Japan and Brazil as you think about those filings and upcoming visits? A: Kylie O'Keefe mentioned that consistent work has been done across all different regions, with existing commercial infrastructure in place and teams ready to execute upon launch. They are engaging with treatment centers of excellence and getting to know the patient communities.
Q: Regarding the pending European Commission decision for Translarna, do you have any visibility as to when the EC might come through with this decision? A: Eric Pauwels, Chief Business Officer, noted that typically it would be 67 days following the CHMP opinion that the European Commission would adopt that opinion. However, the commission is meeting this week to discuss the matter, and it remains business as usual while Translarna is authorized.
Q: For the ALS trial, what kind of genetic biomarker associated with ferroptosis in ALS patients? Do you expect more prominent treatment outcome in subgroup for ALS patients? A: Matthew Klein explained that ferroptosis is a pathway linked to neurodegenerative diseases, including ALS, and is not related to any specific genetic abnormality. It's a common response pathway in ALS, and the trial with utreloxastat includes both genetic and idiopathic ALS patients.
Q: Can you provide any additional color on how much the EU contributed to Translarna revenue this quarter? A: Kylie O'Keefe stated that the contribution from European revenue for Translarna is consistent with the first quarter of last year, representing around 45% of total revenue. The team has executed well to ensure patients have access to Translarna while it remains on the market.
Q: Regarding sepiapterin in PKU, can you help us understand how sepiapterin will be helpful in classic PKU patient population? A: Eric Pauwels clarified that classical PKU patients have a documented phenylalanine level of greater than 1,200 micromolar per liter at some point, and a very small number have homozygous null mutation. Sepiapterin, being a precursor of BH4 and a chaperone culture, stabilizes the enzyme, which is why significant phenylalanine reduction is observed in classical PKU patients.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
This article first appeared on GuruFocus.
