Wing Yip

Wing Yip of H.C. Wainwright, asking on behalf of Ed Arce, questioned if DURECT would consider a smaller, more rigorous Phase IIb study to confirm larsucosterol's efficacy and also asked about opportunities for non-dilutive funding from ex-U.S. regional partnerships.

Answer

Executive James Brown explained that the planned Phase III trial is already designed to be very tightly controlled, making another Phase IIb study less efficient and more costly than proceeding directly to Phase III. Executive Norman Sussman added that the previous trial's two active arms provided robust data, supporting a direct move to Phase III. Regarding ex-U.S. funding, Brown stated that while the company's sole focus is on the AH program, they would consider a regional study with an ex-U.S. partner.

On behalf of Ed Arce, Wing Yip asked about the remaining preparations needed for the larsucosterol Phase III trial besides funding, and requested an estimated cost for the study.

Answer

James Brown (Executive) stated that funding is the primary remaining gate, with preparatory work like site contracts and CRO selection already completed. Timothy Papp (Chief Financial Officer) estimated the external trial costs to be in the $20-$25 million range, with an additional corporate burn rate of $3-$4 million per quarter once the trial is fully underway.

Wing Yip, on for Ed Arce, asked how the ARCT-032 cystic fibrosis (CF) therapy fits into the competitive landscape and its addressable market size, and also inquired about the efficacy bar for success in the upcoming ARCT-810 interim data.

Answer

President and CEO Joseph Payne positioned ARCT-032 as an ideal therapy for the 15-18% of CF patients who do not respond to modulators, representing a significant market opportunity. For the ARCT-810 OTC program, he explained that success will be measured by a range of biomarkers beyond just ammonia, including other amino acids, with a full biomarker strategy to be detailed later in the year.

Wing Yip inquired about Acurx's predictive model for C. difficile recurrence, its international regulatory filing initiatives, and the potential funding options for the upcoming Phase III pivotal trials.

Answer

David Luci, President and CEO, explained that the predictive model is a patent-pending diagnostic tool that uses stool sample measurements to predict reinfection risk early in treatment. He detailed the regulatory strategy, starting with the European Medicines Agency (EMA) followed by the U.K., Canada, and Japan. For funding, Luci highlighted a preference for non-dilutive sources, including government partnerships, territorial licensing deals, and the potential passage of the PASTEUR Act.

Asked for clarification on the upcoming AASLD data, the next steps in the Pfizer litigation after the Markman hearing, and whether the cash runway guidance includes ATM proceeds.

Answer

The company clarified the AASLD data will be for the IM-PROVE II Group C cohort. For the litigation, a court schedule will follow the Markman hearing results. They confirmed the cash runway guidance does not assume any future financing from the ATM program.

Wing Yip of H.C. Wainwright & Co. asked if the upcoming IM-PROVE II expansion data would be presented at a major conference, if new interim data from IM-PROVE I and II would be released before year-end, and how the operational streamlining would affect other programs.

Answer

Interim President and CEO Michael McElhaugh stated they hope to present at a conference but cannot commit yet. He confirmed more interim data is coming, with timing to be determined. He clarified the streamlining focuses resources on advancing imdusiran and will not negatively impact other clinical programs, as the goal is to fund the Phase 2b trial with existing cash.

Wing Yip, on behalf of Ed Arce, asked if the upcoming IM-PROVE II expansion cohort data would be presented at a major medical conference, if other interim data from IM-PROVE I and II would be released before year-end, and how the operational streamlining would affect other programs.

Answer

Interim President and CEO Mike McElhaugh stated they hope to present the IM-PROVE II data at a conference but cannot commit yet. He confirmed more interim data from IM-PROVE I and II is planned for release, with timing to be determined. He also clarified the streamlining focuses resources on imdusiran's late-stage development without impacting other clinical programs.

Wing Yip, on for Ed Arce, asked about the significance of the 12-week primary efficacy period in the Angelman Phase 3 study and inquired about the next development steps for setrusumab (UX143) in 2025.

Answer

CMO Eric Crombez explained the Angelman study duration was chosen to allow sufficient time for skills to develop and be detected, based on Phase 1/2 data. CEO Emil Kakkis added that 2025 for setrusumab is focused on the interim analyses of the Orbit study and data from the Cosmic study, with the goal of filing a BLA next year, pending the timing of the final data readout.

Wing Yip from H.C. Wainwright & Co. requested a breakdown of clinical trial sites or patient enrollment between the Northern and Southern Hemispheres for the RSVPEDs and RSVHR studies. He also asked about the overlap of RSV and flu seasons and if RSVHR enrollment would complete in the Southern Hemisphere.

Answer

Dr. Jay Luly, President and CEO, and Dr. Scott Rottinghaus, Chief Medical Officer, confirmed active enrollment in both hemispheres but noted the Northern Hemisphere has a significantly larger site footprint. Dr. Luly stated his expectation is that the RSVHR study will likely need to continue enrollment into the next Northern Hemisphere season to be completed. He also commented that RSV seasonality is beginning to normalize after pandemic-related disruptions.