Rudy Li

Rudy Li inquired about the timeline for initiating and completing the Phase 3 trial for remlifanserin in Alzheimer's disease psychosis (ADP), and sought details on specific concerns regarding the EU opinion for DAYBUE and the re-examination strategy.

Answer

Elizabeth Thompson, EVP and Head of Research and Development, explained that the remlifanserin program is a seamless master protocol, allowing Phase 3 enrollment to begin later in 2026 after Phase 2 enrollment concludes. Regarding DAYBUE's EU opinion, she anticipated concerns around endpoint relevance, clinical meaningfulness, therapy duration, and mechanism of action, noting a re-examination process could take approximately 120 days with a new CHMP opinion expected around Q2 2026.

Rudy Li inquired about the timeline for initiating and completing the remlifanserin phase III trial in ADP, and sought details on specific concerns regarding the EU opinion for DAYBUE and Acadia's re-examination strategy.

Answer

Elizabeth Thompson, EVP and Head of Research and Development, explained that the remlifanserin program uses a master protocol allowing for a seamless operational transition from phase II to phase III enrollment later this year. For DAYBUE EU, she anticipated concerns regarding endpoint relevance, clinical meaningfulness, duration of therapy, and mechanism of action, and confirmed plans for a re-examination process with a new rapporteur.

Rudy Li asked if Alkermes has discussed key factors for a phase III trial in idiopathic hypersomnia (IH) with the FDA, and if the trial could start earlier or if it needs to await Vibrance-3 data. He also inquired about the market dynamics of NT1, NT2, and IH.

Answer

Richard Pops, CEO of Alkermes, stated that for IH, they will await phase II data from the Vibrance study, then hold an end-of-phase II meeting with the FDA to map out the phase III program. Regarding market dynamics, he noted the IH opportunity is significant but potentially underestimated, with narcolepsy having clearer patient numbers.

Rudy Li asked if Alkermes has discussed key factors for a Phase 3 trial of alixorexton in idiopathic hypersomnia (IH) with the FDA, inquiring if the trial could start earlier or if it depends on Vibrance-3 data, and about market dynamics for NT1, NT2, and IH.

Answer

Richard Pops, CEO, confirmed that for IH, Alkermes will follow the same process as for narcolepsy: obtain Phase 2 data from the Vibrance study, hold a formal end-of-Phase 2 meeting with the FDA, and then map out the Phase 3 program. He noted that the IH market, while currently underestimated, represents a significant opportunity following the narcolepsy market.

Rudy Li from Chardan Capital Markets questioned the higher incidence of CSF protein elevations in the OLE study and asked about any specific concerns, especially for the patients classified as having treatment-emergent adverse events.

Answer

CMO Barry Ticho clarified that elevated CSF protein is a known class effect for intrathecally administered oligonucleotides and is a laboratory finding without any associated clinical manifestations in the 81 patients dosed. He emphasized that the long-term safety profile over four years remains favorable and does not present a concern for the Phase 3 study.

Rudy Li of Chardan Capital Markets asked about the impact of background therapies on pivotal trial enrollment criteria and what additional data would be needed to support vormatrogene's use in combination with other sodium channel blockers.

Answer

President & CEO Marcio Souza stated that the RADIANT study enrolled a real-world sample of aggressively treated patients, including over 30% on cenobamate, and demonstrated a best-in-class safety profile despite this. He expressed no concern about combinations and sees no need to change enrollment criteria, other than providing clearer instructions to physicians on reducing background ASM doses to improve tolerability.

Rudy Li from Chardan Capital Markets inquired about the FDA's data requirements beyond the primary endpoint for a GAD NDA filing and asked about the company's broader pipeline expansion strategy, including potential collaborations.

Answer

CEO & Board Director Robert Barrow stated that the primary endpoints from the VOYAGE and PANORAMA studies are expected to be the key components for a potential NDA filing. Regarding pipeline expansion, he affirmed the team is working on more than just MM120 and is always focused on opportunities to drive long-term value, suggesting more updates will come as time progresses.

Rudy Li of Chardan Capital Markets inquired about the rate-limiting steps for an NDA filing in GAD beyond the primary endpoint data and asked for the company's strategy on expanding its pipeline through potential collaborations.

Answer

CEO Robert Barrow clarified that the 12-week primary endpoint data from the two pivotal GAD studies are the key components expected for a potential NDA filing. Regarding the pipeline, he expressed excitement about internal programs beyond MM120 and the team's capabilities, advising investors to 'stay tuned' for future updates on expansion.

Rudy Li inquired about MindMed's view on psychedelic market dynamics, referencing Spravato's growth, and asked about commercial planning for the second half of the year, including leveraging Spravato's infrastructure.

Answer

CCO Matthew Wiley noted the large unmet need in GAD and MDD, stating that commercial positioning work will be completed in H2 2025. He views Spravato as an interesting surrogate for reimbursement and capacity but sees a distinct opportunity for MM-120. CEO Robert Barrow added that MM-120's data could support a more expansive opportunity than Spravato.

Rudy Li from Chardan asked about the strategic importance of the MDD indication for MM120's competitiveness and when to expect updates on the timing and design of the second MDD study.

Answer

CEO Robert Barrow emphasized that pursuing a broad label covering both GAD and MDD is a key strategic differentiator, allowing them to serve a massive patient population and setting them apart from competitors focused on treatment-resistant depression. He did not provide a specific timeline for updates on the second MDD study, stating it will be informed by ongoing progress.

Asked about the size of the treated AL amyloidosis patient population, the impact of daratumumab's approval on the market, and the rationale for the PRX012 expansion cohort.

Answer

The company quotes a diagnosed and treated population of 16,000 patients in the U.S., which has increased from 15,000 since daratumumab's approval. The PRX012 expansion cohort was an opportunistic way to enroll more patients who were screening for the APOE homozygous 'B' cohorts but were not carriers, allowing for a larger dataset to better characterize the drug.

Rudy Li of Chardan asked about the number of actively treated AL amyloidosis patients, market changes since daratumumab's approval, and the rationale for the 200mg expansion cohort in the PRX012 ASCENT-2 trial.

Answer

COO Brandon Smith confirmed the 16,000 U.S. patients cited are diagnosed and actively treated, a number that has increased from ~15,000 since daratumumab's approval. Chief Development Officer Chad Swanson explained the PRX012 expansion cohort was an opportunistic way to enroll more patients who screened for but were not eligible for the APOE homozygous cohorts, allowing for a larger dataset to better characterize the drug's profile.

Rudy Li of Chardan asked about the number of actively treated AL amyloidosis patients, changes in market dynamics since daratumumab's approval, and the rationale for the 200mg expansion cohort in the PRX012 ASCENT-2 trial.

Answer

COO Brandon Smith stated that the quoted 16,000 U.S. patients are diagnosed and treated, up from 15,000 since daratumumab's 2021 approval. Chief Development Officer Chad Swanson explained the PRX012 expansion cohort was an opportunistic way to utilize patients who screened for but were not eligible for the rarer APOE homozygous cohorts, thereby building a larger dataset to better characterize the drug.

Rudy Li inquired about the number of AL amyloidosis patients actively treated with standard-of-care and any market dynamic changes since daratumumab's approval. He also asked for the rationale behind the 200mg expansion cohort for PRX012 in the ASCENT-2 trial.

Answer

Chief Operating Officer Brandon Smith confirmed their quoted figure of 16,000 U.S. patients are diagnosed and treated, an increase from 15,000 since daratumumab's approval. Chief Development Officer Chad Swanson explained the PRX012 expansion cohort was an opportunistic way to utilize patients screened for the hard-to-enroll APOE homozygous 'B' cohorts, allowing them to build a larger dataset to better characterize the drug.