Farzin Haque

Farzin Haque inquired about the underlying assumptions for Aurinia's 2026 LUPKYNIS sales guidance, noting its perceived conservatism given the Q4 run rate, and asked for insights into early 2026 performance. Haque also sought clarification on the termination of the VOCAL pediatric study, specifically regarding DSMB recommendations and any FDA involvement.

Answer

Peter Greenleaf, President and CEO, explained that the commercial strategy for LUPKYNIS remains consistent, focusing on efficacy data, AURORA extension study, biopsy data, and leveraging ACR/EULAR guidelines to promote earlier diagnosis and aggressive treatment of lupus nephritis. He declined to provide specific Q1 2026 steer, referring to Q1 2025 trends as a historical predictor. Dr. Greg Keenan, Chief Medical Officer, clarified that the VOCAL study was terminated due to recruitment difficulties, and Aurinia plans to negotiate with the FDA regarding pediatric commitments, noting the small burden and business opportunity of the disease in pediatric patients.

Farzin Haque from Jefferies inquired about the underlying assumptions for Aurinia's 2026 guidance, particularly regarding the commercial outlook and early 2026 performance. Haque also asked for clarification on the termination of the VOCAL and VOCAL extension pediatric studies, specifically whether the DSMB or FDA initiated the stop.

Answer

President and CEO Peter Greenleaf explained that the commercial strategy for LUPKYNIS continues to leverage strong efficacy data, expanded AURORA trial data, and ACR/EULAR guidelines promoting earlier diagnosis and aggressive treatment of proteinuria. He noted that the first two months of 2026 were consistent with historical trends, referring to Q1 2025 for comparison. Chief Medical Officer Dr. Greg Keenan clarified that the VOCAL study was terminated due to recruitment difficulties, and Aurinia plans to negotiate with the FDA regarding pediatric commitments, noting the small burden and business opportunity in pediatric lupus nephritis.

A representative for Farzin Haque asked if the 2025 revenue guidance of $240M-$250M is conservative given the Q4 run rate, and questioned the company's strategy regarding LUPKYNIS patent exclusivity in light of a recent ANDA filing.

Answer

President and CEO Peter Greenleaf stated the guidance is not conservative but is based on historical financial results from five years on the market. He reaffirmed the company's robust patent portfolio, which could provide protection until 2037, and stated Aurinia's full intention to vigorously defend its intellectual property against any ANDA filings.

Farzin Haque, on behalf of Maury Raycroft, inquired about Arrowhead's perspective on an upcoming competitor data release for severe hypertriglyceridemia (SHTG) and how it might compare to their own plozasiran studies.

Answer

CEO Christopher Anzalone stated that it's difficult to compare drugs across different studies and that Arrowhead is focused on its own data, where they expect plozasiran to be a best-in-class triglyceride reducer. Interim Chief Medical Scientist Bruce Given added that while the competitor's patient population appears similar, the ultimate impact of their data remains to be seen.

Representing Maury Raycroft at Jefferies, Farzin Haque questioned the latest assumptions on the Phase 3 event accrual rate for Nexi and whether this influenced the decision to expand the MAGNITUDE study.

Answer

President and CEO John Leonard explained that while specific event rates are not disclosed, their calculations are informed by competitor data, other databases, and their own maturing Phase 1 data. He noted that the deep and sustained TTR reduction seen with Nexi is expected to result in a lower event rate, and the study expansion is intended to ensure a more robust outcome while staying within original timelines.

Farzin Haque from Jefferies requested more perspective on the dynamics and breakdown of Q2 growth, specifically contributions from stocking, new patients, and persistence rates, and how this might look for the rest of the year.

Answer

Peter Heerma, Chief Commercial Officer, stated that there was no stocking and the growth was performance-driven. President & CEO Dr. Eric Dube added that persistence rates remain very high and that all lead indicators are strong. He noted the dynamic of growth from new patients on top of a growing base of continuing patients is precisely what they expect to be sustainable.

Representing Maury Raycroft of Jefferies, Farzin Haque asked for more granular timing on the cetrusumab data disclosure, details on the Angelman Phase 3 patient baseline profile versus Phase 1/2, and whether more durability data from the Phase 1/2 cohort could be expected this year.

Answer

Emil Kakkis, Founder, President & CEO, indicated the cetrusumab data would be available 'around the end of the year,' declining to be more specific to ensure a high-quality study closeout. He confirmed the Angelman patient profiles are identical between phases and stated there are no current plans to release more Phase 1/2 data as the team is focused on the Phase 3 program.

Farzin Haque asked about the expectations for the split doses of R289 being explored and whether regulatory feedback is required before advancing to the recommended Phase 2 dose. He also followed up on the TAVALISSE patent litigation, asking if future ANDA filers could potentially enter the market earlier.

Answer

Chief Medical Officer Lisa Rojkjaer addressed the R289 question, explaining the switch to twice-daily dosing aims for tonic suppression of inflammation and confirmed that Rigel will seek FDA input, consistent with Project Optimus, before selecting doses for the expansion phase. President and CEO Raul Rodriguez answered the patent question, stating that while a second ANDA filer is technically possible, the existing settlement with Annora creates a disincentive, and Rigel has no notice of any other filers.

Farzin Haque questioned what Rigel considers the bar for success for the R289 program in lower-risk MDS to advance it, and whether the new GAVRETO safety signal regarding infections is expected to be a rate-limiting factor for its use.

Answer

Chief Medical Officer Lisa Rojkjaer explained that for R289, the study includes heavily pre-treated patients, making a direct efficacy comparison difficult, but the team is encouraged by the safety and efficacy seen so far. Regarding GAVRETO, she noted the infection risk is not new information and is manageable for oncologists. Chief Commercial Officer David Santos added that proactive communication to over 10,000 providers yielded no concerned feedback, and they do not see it impacting GAVRETO's opportunity.

Farzin Haque of Jefferies inquired about the specifics of the FDA's feedback on the two outstanding CMC items, the sterility and identity assays, and whether new experiments were required. He also asked for confirmation on the status of the retroviral replication assay and for more granularity on the BLA resubmission timing.

Answer

CEO Vishwas Seshadri clarified that the FDA's feedback on the rapid sterility test pertained to the statistical approach for validation, not a request for a new experimental method. He noted alignment on the identity assay approach and confirmed the retroviral replication assay is now considered resolved. Seshadri reiterated that the company remains on track for a BLA resubmission in the second half of 2024 but declined to provide a more specific timeline.