Joe Schwartz

Joe Schwartz asked if the relatively linear patient start form (PSF) growth rate is expected to continue and at what point it might taper off. He also inquired about the agreed-upon price for EKTERLY in Germany and its comparison to the U.S. price, as well as plans for other European countries in 2026.

Answer

Ben Palleiko, Chief Executive Officer, noted consistent PSF rates but anticipated a slowdown in Q4 due to holidays and seasonality, expecting the pace to moderate in 2026 as the company deepens its penetration into the patient population. Nicole Sweeney, Chief Commercial Officer, stated that the German price is not disclosed due to ongoing negotiations. Ms. Sweeney also outlined plans for a U.K. launch in H1 2026, following discussions with NICE, and expansion into other larger European countries towards the end of 2026.

Joe Schwartz asked about the expected continuation of EKTERLY's relatively linear PSF growth rate, at what point it might taper off, and for an update on the German launch price and plans for other European countries in 2026.

Answer

Ben Palleiko (CEO) indicated that PSF rates have been consistent but expects some slowing in Q4 2025 due to holiday seasonality, with an eventual tapering in 2026 as the company penetrates deeper into the patient population. Nicole Sweeny (Chief Commercial Officer) stated that the German price is not disclosed due to ongoing negotiations, and outlined plans for a UK launch in H1 2026 and other larger European countries towards the end of 2026.

Joe Schwartz asked about the specific BMN-333 pharmacokinetic (PK) exposure levels that would indicate clinical superiority over VOXZOGO. He also inquired about the minimum annualized growth velocity (AGV) delta over VOXZOGO required to demonstrate clear clinical superiority and drive patient switching.

Answer

Greg Friberg (Chief R&D Officer, BioMarin Pharmaceutical) stated that for BMN-333, they sought at least a 3x increase in free CNP AUC from the phase I PK study, which was achieved in three dose levels. He declined to provide an exact AGV delta but mentioned a clinically meaningful differentiation level has been determined that could translate to improved health and functional outcomes.

Joe Schwartz asked about the specific BMN-333 PK exposure levels that would instill confidence in achieving clinical superiority over Voxzogo. He also inquired about the minimum annualized growth velocity (AGV) delta over Voxzogo required to demonstrate clear clinical superiority, drive patient switching, and reestablish a standard of care.

Answer

Greg Friberg, Chief R&D Officer, stated that BioMarin was looking for at least a 3x increase in AUC for free CNP levels from the phase one PK study, which was achieved in three different dose levels. He declined to provide a specific AGV delta number but emphasized that the company has determined a clinically meaningful differentiation level that is expected to translate into improved health, wellness, and functional outcomes beyond linear growth.

Jenny, on for Joe Schwartz of Leerink Partners, asked about the WVE-006 RNA editing program, seeking to confirm if the 60% M-AAT protein level suggests editing efficiency could still increase, if Wave is at the bottom of the dose curve, and how much higher the dosage could go.

Answer

CEO Paul Bolno confirmed that achieving 60% M-protein with the lowest single dose is a very encouraging surrogate for editing efficiency and that they are at the low end of the dose curve. He expects efficiency to increase with repeat dosing, based on preclinical data. He also stated they have ample room to increase the dose, are already dosing higher in healthy volunteers, can dose higher than inclisiran, and will also explore extending the dosing interval for less frequent administration.

Joe Schwartz from SVB Leerink asked about any notable changes in other blood components like leukocytes and platelets, the timeline for the next data updates from the current trial, how quickly a pivotal trial could begin, and what the design and endpoints of such a trial might look like.

Answer

Dr. Ronald Hoffman referred to appendix slides showing that leukocyte and platelet counts remained steady during treatment. Chief Medical Officer Dr. Samuel Saks explained that the company is currently working with thought leaders to design the pivotal study and will discuss it with the FDA once finalized, with further data updates expected in the second half of the year. President and CEO Dinesh Patel added that enrollment is strong and the company intends to share more data as it is gathered from the expanded study.