Tiago Fauth

Tiago Fauth of Wells Fargo asked about the potential implications of a positive readout from United Therapeutics' TETON study, specifically regarding orphan drug exclusivity and any impact on Yutrepia or the L-606 development program.

Answer

CEO Roger Jeffs responded that if the TETON study is successful, United Therapeutics would gain orphan drug protection for a period. He stated that for Liquidia, this would reinforce the strategy to develop L-606 for that indication, positioning it to enter the market after the orphan exclusivity period expires.

Tiago Fauth of Wells Fargo asked for qualitative commentary on the consistency of effect for WVE-006 (AATD) across patients in both the single-dose and multi-dose cohorts, given the well-understood PK/PD of GalNAc conjugates.

Answer

President & CEO Paul Bolno responded that consistency was observed in preclinical models and in the initial proof-of-mechanism data. He expressed high encouragement based on these models and the early clinical translation, stating that the upcoming comprehensive data from the full single and multi-dose cohorts will provide a clear ability to assess this consistency, especially given the substantial exposure from seven doses.

Tiago Fauth noted that channel checks suggest very low discontinuation rates and asked for insights into what might be driving any discontinuations. He also inquired about the company's view on long-term competition from biologics.

Answer

CEO David Zaccardelli responded that it's too early to have clarity on true discontinuation reasons, as patient refill timing can be sporadic. He reiterated Ohtuvayre's favorable tolerability. Regarding biologics, he stated they are not viewed as competitive but rather as potentially complementary, addressing inflammation through different mechanisms. He emphasized that Ohtuvayre's profile includes bronchodilation, which biologics lack, and sees no reason the therapies couldn't be used in conjunction.

Tiago Fauth of Wells Fargo asked for insights into reasons for patient discontinuations and questioned how management views the long-term competitive landscape, particularly with the emergence of biologics for COPD.

Answer

CEO David Zaccardelli explained it is too early for clear data on discontinuations, noting the drug's favorable tolerability. He positioned biologics as complementary rather than competitive, emphasizing Ohtuvayre's unique dual mechanism providing both bronchodilation and anti-inflammatory effects, which biologics lack. He sees no pharmacological reason the treatments could not be used in conjunction.

Tiago Fauth asked about the average duration of therapy for Ohtuvayre, noting low discontinuation rates in channel checks and questioning if there is potential upside to the standard assumption of six months.

Answer

Executive David Zaccardelli acknowledged that the company's model uses a benchmark of around six refills per year, consistent with other COPD drugs. However, he stated that Verona Pharma's specialty pharmacy distribution network, which provides strong patient support, could lead to better persistence. While noting it is still early, he said the company is encouraged by seeing early patients receive over five refills and believes there is potential upside to the six-refill assumption.

Tiago Fauth asked for a breakdown of the $1 billion revenue potential for sepiapterin in PKU, specifically regarding the mix of converting existing patients versus expanding the market to untreated individuals.

Answer

CEO Dr. Matthew Klein responded that the key driver for therapy adoption is diet liberalization. He emphasized that sepiapterin's data package supports its ability to address all patient segments, including those on current therapy, those who have failed therapy, and therapy-naive patients, thereby enabling both market conversion and expansion.