Karishma Raghuram

Karishma Raghuram asked about expectations for the non-liver metastases (NLM) subgroup data from Stellar-303, the rationale for enrolling 900 patients compared to Lonsurf BEV's 500, and overall implications.

Answer

Dana Aftab, EVP of Research and Development, clarified that the study initially focused on NLM but evolved to include the ITT population due to the opportunity for earlier results. The ITT population, which includes both liver and non-liver metastases, was the basis for the positive results presented. The NLM subgroup, which progresses more slowly, is expected to have results around mid-2026.

Karishma Raghuram inquired about expectations for the non-liver metastases (NLM) data cut from STELLAR-303, anticipated early next year, and the rationale behind enrolling approximately 900 patients in the study, particularly in comparison to the SUNLIGHT trial's patient count.

Answer

Dana Aftab, Executive Vice President of Research and Development, clarified that the study initially had a single primary endpoint in NLM patients but evolved to include dual primary endpoints for both the ITT and NLM populations. She explained that the ITT population, which includes patients with liver metastases, allowed for earlier results due to faster event accrual. The NLM subgroup, progressing more slowly, is expected to yield results around mid-2026.

Karishma Raghuram, on for Chris Shibutani, asked about the decision to pursue the idiopathic hypersomnia (IH) indication for the orexin program despite potential negative implications from the Inflation Reduction Act (IRA) and the commercial opportunity needed to justify this move.

Answer

CEO Richard Pops explained the decision was multifaceted. He noted the low probability of the IRA remaining unchanged in nine years and the potential for Congress to amend the provision hurting rare disease patients. From a commercial standpoint, the significant size of the IH market (~40,000 patients) and its clinical adjacency to narcolepsy made the expansion compelling, as it significantly grows the addressable market for nine years at the same price point.

Asked for framing on the cervical dystonia opportunity, including product positioning with payers and the impact of the permanent J-code on the revenue ramp.

Answer

Mark Foley described cervical dystonia as a $340 million market where DAXXIFY's long duration addresses a key unmet need. For payers, the product is attractive due to potential cost savings from its pricing and dosing, which has led to strong early commercial coverage (>50% of lives). The launch will be measured as physicians titrate doses over time, but the J-code is in place, and early feedback is very positive, positioning it as a gateway to the broader therapeutics market.