Priyanka Grover

Priyanka Grover of JPMorgan Chase & Co. inquired about the specific levers Day One Biopharmaceuticals will focus on to achieve the second-half revenue growth implied by its new 2025 guidance.

Answer

CEO Jeremy Bender identified two critical variables for meeting the 2025 revenue guidance: continued adoption in the form of new patient starts and patient persistence on Ogenda therapy. He noted that after a full year on the market, the trends for these variables have become more predictable, providing confidence in the provided range.

Priyanka Grover of JPMorgan Chase & Co., on behalf of Anupam Rama, inquired about the observed duration of OJEMDA therapy in the market and physicians' long-term treatment plans for the drug.

Answer

Chief Commercial Officer Lauren Merendino responded that while it is still early to comment definitively on duration, the company is observing a very high percentage of patients remaining on therapy month-over-month. She noted these trends are consistent with the results seen in the pivotal FIREFLY-1 study.

Priyanka Grover of JPMorgan Chase & Co. asked about the key clinical assessments that resonate with physicians and patients when considering a switch from Nexviazyme to Pombility and OpFolda, and whether these factors differ between the U.S. and ex-U.S. markets.

Answer

Chief Development Officer Dr. Jeff Castelli explained that physicians primarily look for stability of declining function or improvements in biomarkers, muscle strength, and six-minute walk tests, along with overall quality of life. He noted these criteria are similar for switches from both Lumizyme and Nexviazyme. President and CEO Bradley Campbell added that there are no significant differences in these considerations between U.S. and international physicians.

Priyanka Grover of JPMorgan Chase & Co. inquired about the prescribing patterns for Revuforj, asking if repeat prescribers are concentrated in centers of excellence or more broadly distributed.

Answer

Chief Commercial Officer Steven Closter explained that while top-tier academic centers represent a significant portion of the business, prescribing is broadly distributed. He noted that 44% of Tier 1 and Tier 2 accounts had ordered by the end of March, with about 80% of those ordering multiple times. He highlighted that usage is also strong outside these top tiers, including at smaller academic centers and community practices, indicating a growing and widespread user base.

Priyanka Grover of JPMorgan Chase & Co. inquired about the process and expected timelines for incorporating Revumenib into NCCN guidelines following its potential approvals for KMT2A and NPM1 indications.

Answer

CEO Michael Metzger outlined the strategy, which involves securing FDA approval, publishing the clinical data, and then submitting to the NCCN panels. He highlighted that this process can be rapid, referencing the experience with axatilimab, which was added to guidelines within two to three weeks of its approval and contemporaneous data publication.

Priyanka Grover, on for Anupam Rama at JPMorgan, asked if the R&D Day planned for the second half of the year would feature new data from the Arrowhead collaboration or other pipeline programs.

Answer

Chief Scientific Officer Dr. Louise Rodino-Klapac explained that the timing of the Arrowhead data release will determine if it aligns with the R&D Day. She emphasized that the event's primary purpose is to provide a detailed overview of Sarepta's deep pipeline across multiple platforms, which has not been shared in detail before. The specific timing of data presentations will be shared closer to the date.