Yun Zhong

Yun Zhong from Wedbush Securities asked about the potential launch timing for TransCon CNP following approval and whether a monotherapy arm is still being considered for the hypochondroplasia trial.

Answer

President & CEO Jan Møller Mikkelsen stated that launch plans for TransCon CNP would be detailed on a call following regulatory approval. For hypochondroplasia, he reiterated the belief that combination therapy is the most robust treatment for the heterogeneous patient population, but did not rule out including a monotherapy arm in the trial design pending further regulatory discussions.

Yun Zhong of Wedbush Securities sought confirmation that the Phase 2 nintedanib trial is not powered for efficacy, asked if the goal is to select one dosing regimen for Phase 3, and inquired about the conditions for drawing additional capital from the Blackstone loan agreement.

Answer

CEO Michael Castagna confirmed the Phase 2 trial is not powered for efficacy but is designed to inform the Phase 3 trial, with the goal of selecting a single dosing regimen. CFO Chris Prentiss explained that the initial $125 million of the Blackstone facility is committed and can be drawn at the company's discretion without being tied to specific clinical or sales milestones, as it is intended for general growth initiatives.

Yun Zhong of Wedbush Securities asked to what the strong NRx growth for Afrezza could be attributed and whether the launch strategy for the pediatric indication would mirror the adult strategy.

Answer

Executive Michael Castagna attributed the NRx growth to a combination of new INHALE-3 data and strong sales force execution. He stated the pediatric launch strategy will be 'very different' from the adult launch, focusing on children's hospitals and academic centers with a comprehensive plan to be shared later.

Yun Zhong of Wedbush Securities inquired about feedback from adult subjects in the INHALE-3 study and details of the upcoming interim analysis for the clofazimine trial (MNKD-101), including potential study size adjustments.

Answer

Executive Michael Castagna reported that about 50% of patients in the INHALE-3 study opted to remain on Afrezza, with outcomes often linked to physician experience. For MNKD-101, he explained the interim analysis will occur after 100 patients reach their endpoint, and enrollment will continue during the analysis to preemptively meet any potential increase in trial size, which could go from 180 up to 300 patients.

Yun Zhong of Wedbush Securities questioned what Wave considers a desirable Inhibin E knockdown level for WVE-007 and if dose escalation would continue past cohort three. He also asked if recent FDA updates would affect the company's strategy for its DMD program.

Answer

President & CEO Paul Bolno responded that the 240 mg dose (cohort two) was modeled to replicate effects seen in their DIO mouse model and that the company is not stopping there, with the 400 mg cohort already dosing and the potential to go higher if needed. Regarding the DMD program, Bolno noted their program is with CDER and that while they are monitoring the situation at the FDA, there is nothing to suggest their current plan for an NDA submission in 2026 is changing.

Yun Zhong from BTIG asked if the GM1 dose escalation is contingent on data from Cohort 4, whether the higher dose will be explored in both early and late-onset patients, and if the trial will now specifically recruit patients with shorter developmental delays.

Answer

CEO Will Chou confirmed the dose escalation is proceeding independently of Cohort 4 results and will include both early and late infantile patients. Chief Medical Officer Mark Forman added that the revised inclusion criteria aim to enroll more patients at earlier stages of the disease, who inherently have shorter developmental delays, to optimize the risk-benefit profile.

Yun Zhong from BTIG asked for clarification on the GM1 dose escalation, questioning if it is dependent on data from Cohort 4, whether both early and late-onset patients will be treated at the higher dose, and if the company will now specifically recruit patients with shorter developmental delays.

Answer

CEO Will Chou confirmed the dose escalation is proceeding regardless of Cohort 4 results and will include both early and late infantile patients. CMO Mark Forman added that the revised inclusion criteria are designed to shift focus to earlier stages of the disease and enroll more patients with shorter developmental delays to optimize the benefit-risk profile.

Yun Zhong of BTIG asked for a conclusion on the cause of the hydrocephalus safety finding in the Krabbe program and whether this risk extends to GM1 or FTD. He also asked about the expected functional measures in the upcoming December GM1 data update.

Answer

Chief Medical Officer Mark Forman stated that the hydrocephalus case was deemed "possibly related" to the Krabbe treatment but causality is unknown, and this risk is not expected in FTD or GM1. Both he and CEO William Chou confirmed the December GM1 update will include safety, biomarker data (beta-gal activity), and clinical assessments via the Bayley and Vineland scales.

Yun Zhong from BTIG asked for confirmation on the lead indication for ImmTOR-IL-2, noting PBC was not mentioned in the press release. He also inquired if Astellas showed interest in ImmTOR in addition to Xork and confirmed if both platforms are capsid-agnostic.

Answer

President and CEO Carsten Brunn clarified that while PBC is still being considered, they are broadening their focus to include other autoimmune liver diseases like autoimmune hepatitis, with more guidance to come post-DISSOLVE. He confirmed the Astellas deal is an exclusive license for Xork in their Pompe program and stated that both ImmTOR and Xork are believed to be agnostic to the AAV capsid subtype used.

Yun Zhong from BTIG sought confirmation on whether PBC is still the likely first indication for the ImmTOR-IL-2 program. He also asked about the Astellas collaboration, inquiring if Astellas showed interest in ImmTOR and confirming that both ImmTOR and Xork are agnostic to the AAV capsid used.

Answer

President and CEO Carsten Brunn confirmed that while PBC is still part of the mix, the company is broadening its focus to other autoimmune liver diseases like autoimmune hepatitis. He clarified that Astellas has an exclusive license for Xork in its Pompe program and affirmed the belief that both ImmTOR and Xork are capsid-agnostic based on current data.

Yun Zhong of BTIG questioned what Selecta aims to achieve with its proprietary engineered IL-2 compared to existing candidates and asked about the ownership rights and development plans for the IgA protease candidate once selected.

Answer

President and CEO Carsten Brunn and CSO Takashi Kishimoto explained that the key differentiation for their IL-2 approach is combining it with ImmTOR for an antigen-specific effect. Brunn confirmed that Selecta will have 100% ownership of the selected IgA protease and will drive its development, with the initial plan being to use ImmTOR alone, similar to the SEL-212 program.

Yun Zhong from Janney Montgomery Scott LLC asked if the upcoming pancreatic cancer data readout at ASCO would include updated information from all three study groups. He also inquired about patient enrollment numbers since the last data update in July 2019.

Answer

President and CEO Peter Hoang deferred the question on patient enrollment until the ASCO abstracts are released later in the week. He also clarified that the poster presentation is determined by the Baylor investigators and is expected to focus on the trial arm where patients receive therapy in conjunction with the standard of care.

Yun Zhong from Janney asked whether the IND submission package included clinical data from the investigator-sponsored studies conducted at Baylor College of Medicine.

Answer

President and CEO Peter Hoang confirmed that data from the Baylor studies was included in the IND submission. He characterized the FDA's subsequent questions about reagents as a standard part of the transition from an academic process to a more rigorous, commercial-grade manufacturing process suitable for a pivotal trial, noting the agency is aware of the therapy's safety profile.