William Wood

William Wood inquired about the specific GLP-1 receptor agonist (tirzepatide vs. semaglutide) to be used in Veru's Phase 2b Plateau trial, how different agents might affect achieving the 5% weight loss target, and the FDA's flexibility regarding functional endpoints as primary outcomes in Phase 3. He also asked for clarification on the target patient population (older than 65) for the Plateau study, considering FDA guidance, Medicare reimbursement, and patient need.

Answer

Dr. Mitchell Steiner, Chairman, CEO, and President of Veru Inc., clarified that the company will select either tirzepatide or semaglutide for the Phase 2b Plateau study to minimize variability, with tirzepatide currently a placeholder. He explained that the Phase 2b is designed as a 'mini Phase 3' to inform the optimal primary endpoint, whether incremental weight loss or physical function, leveraging FDA's flexible guidance. Dr. Steiner further detailed that targeting patients over 65 with BMI greater than or equal to 35 is strategic for demonstrating significant physical function benefits and de-risking the program, allowing for potential expansion to all patient populations with pre-specified subsets for functional endpoints in Phase 3.

William Wood inquired about the rationale behind targeting an older patient population (BMI >35, age >65) in the Phase 2b PLATEAU study, an increase from the >60 age group in the QUALITY study, and whether this shift was influenced by FDA guidance, Medicare reimbursement dynamics, or focusing on the most in-need patients.

Answer

Dr. Mitchell Steiner, Chairman, CEO, and President, clarified that targeting patients over 65 in PLATEAU is strategic. He explained that this population is most informative for physical function benefits and helps de-risk the program by identifying the most in-need patients, which aligns with FDA guidance for pre-specified subsets for functional endpoints.

William Wood of B. Riley Securities inquired about the specifics of the proposed Phase III trial design, including its structure with dechallenge and rescue arms, and which endpoints would be prioritized. He also asked about the expected timing and venue for the presentation of full data from the Phase 2b study.

Answer

Chief Scientific Officer, Gary Barnette, detailed the innovative Phase III design, which aims to replicate successful short-term Phase II endpoints (lean mass, physical function) while also assessing longer-term outcomes like bone density and incremental weight loss. The study will uniquely feature a 'dechallenge' arm (withdrawing Inovasarm) and a 'rescue' arm (adding Inovasarm to the placebo group) to robustly demonstrate efficacy. Chairman, President & CEO, Mitchell Steiner, stated that the company is targeting Obesity Week in the fall to present more comprehensive data.

Asked for more color on the blinded safety data, especially regarding liver tests, and inquired about the company's view on the two potential regulatory pathways (functional vs. metabolic improvements) and what metabolic data to expect from the Phase IIb study.

Answer

Regarding safety, the company expects to see mild, self-limiting ALT increases, which is considered 'adaptation tolerance' and not drug-induced liver injury, consistent with prior studies and what is seen with GLP-1s. For the regulatory path, the company is focused on functional improvement (via Stair Climb Test) as a primary endpoint because GLP-1s can worsen function, making enobosarm's benefit clear and clinically meaningful. This path is well-aligned with FDA guidance. Metabolic data, including HbA1c and LDL, will be reported from the Phase IIb study.

William Wood of B. Riley Securities inquired about the patient rollover rate into the enobosarm extension trial, reasons for discontinuation, and whether the Q2 2025 timeline for the maintenance data readout represents a delay.

Answer

Chief Scientific Officer Dr. K. Barnette reported a consistent 13% dropout rate, primarily due to GI side effects from the GLP-1 therapy itself, which was expected. He clarified that the safety data remains blinded. Dr. Barnette also stated there are "relatively no changes" to the Q2 timeline, explaining that the timing is fixed by the 12-week study duration and subsequent data processing.

William Wood of B. Riley Securities, Inc. sought more detail on the expected content of the January topline data release, specifically what metrics beyond lean mass would be shared. He also asked if the upcoming Obesity Week presentation would feature new, previously unseen data.

Answer

Dr. Mitchell Steiner, Chairman, CEO, and President, confirmed the January topline results will include the primary endpoint of lean body mass and data on total fat mass to demonstrate dose response. He indicated other data might be reserved for presentation at scientific meetings. He also affirmed that the Obesity Week abstract will present a new analysis of data from previous studies, not repeated information.

William Wood of B.Riley Securities sought clarification on the patient numbers for the upcoming six-month REVEAL-one data, whether DEXA scans would be included, and the specific timing for REJUVA-one's first patient dosing and data release.

Answer

Harith Rajagopalan, Co-Founder, CEO & Director, responded that the majority of the 22 patients in REVEAL-one will have six-month data available for the Q4 update. He clarified that DEXA scans are not part of this open-label study due to a lack of historical comparators. For Rejuva, he confirmed that both the first patient dosing and the release of preliminary data are expected to occur in 2026.

William Wood of B. Riley Securities asked about future data readouts for REVEAL-1, specifically if they would include biomarkers and body composition analysis. He also questioned if weight regain dynamics might differ between patients who used semaglutide versus tirzepatide, and inquired about discussions with state payers regarding potential coverage for Revita.

Answer

CEO Harith Rajagopalan confirmed that biomarker data, including leptin, is being collected and will be shared later in the year. He clarified that the company's analysis suggests weight regain is more dependent on the time since drug discontinuation than the specific GLP-1 agent used. Regarding payers, he noted that Fractyl has had productive initial conversations and that the data from the REMAIN-1 study will be key to articulating a clear value proposition to them.

William Wood of B. Riley Securities asked how closely the Rejuva-002 candidate's design mimics Rejuva-001 and whether the first candidate derisks the second. He also inquired about plans for European expansion beyond the current focus on Germany.

Answer

Executive Harith Rajagopalan confirmed that Rejuva-001 significantly derisks Rejuva-002, as they share the same delivery catheter, AAV9 vector, and insulin promoter. Regarding European expansion, he stated that while Germany presents a unique near-term opportunity, the company is actively preparing for a broader global launch in other key geographies to coincide with the eventual U.S. launch.

William Wood of B.Riley Securities asked how competitors gaining FDA acceptance for biopsy-free, NIT-based trials could create a more capital-efficient Phase 3 path for pemvidutide. He also questioned what Merck's upcoming efinopegutide data could imply for Altimmune's 48-week data and Phase 3 confidence.

Answer

Dr. M. Scott Harris and Dr. Vipin Garg agreed that recent regulatory developments create a 'real opportunity' for a more innovative trial design, which they will discuss with the FDA. Regarding Merck, Dr. Harris noted that Altimmune's strong 24-week data was unprecedented for an incretin and that their 48-week NIT data will be used to model and support the drug's efficacy.

William Wood of B. Riley Securities inquired about Lineage's manufacturing differentiation compared to peers, its scale-up potential for OpRegen, and the potential impact of tariffs on its Israeli facility.

Answer

CEO Brian Culley asserted that Lineage's GMP dual banking system, capable of producing millions of doses from one cell line, is a highly differentiated capability that peers have not demonstrated. CFO Jill Howe added that the company does not expect tariff impacts and mitigates supply risk by purchasing materials far in advance.

William Wood of B. Riley Securities sought clarification on the clinical timeline for the OPC1 DOSED study and inquired about the company's confidence in achieving adequate cell coverage for the ANP1 hearing loss program based on learnings from OpRegen.

Answer

CEO Brian Culley detailed the OPC1 timeline, expecting final data submission to the FDA within weeks and a subsequent agency review in Q1 2025, allowing for site activation to proceed in parallel. For the ANP1 program, he expressed encouragement from preclinical data showing unexpected cell migration within the cochlea, which could be beneficial for achieving therapeutic coverage in a complex anatomy.

William Wood inquired about the level of detail expected for the upcoming CALLIPER trial readout in progressive MS, including data on subpopulations and key endpoints, and also asked about the timeline for the investigator-led post-COVID trial and its potential implications for the ENSURE program.

Answer

CEO Dr. Daniel Vitt confirmed that the April 2025 CALLIPER readout will be comprehensive, including data on all sub-forms, clinical endpoints like confirmed disability worsening, and biomarkers such as NfL and GFAP. Regarding the post-COVID trial, he explained that as an investigator-sponsored study, Immunic cannot guide on timing but noted its scientific value for understanding fatigue. He added that post-COVID patient data from ENSURE or CALLIPER might be analyzed retrospectively.

William Wood of H.C. Wainwright & Co. inquired about the level of detail expected in the April 2025 CALLIPER trial readout for progressive MS, including data across subpopulations and key endpoints. He also asked for the potential data timeline of the investigator-led post-COVID trial and how its findings on fatigue might inform the larger ENSURE and CALLIPER trials.

Answer

CEO Dr. Daniel Vitt confirmed that the CALLIPER readout will be comprehensive, providing detailed data on general and sub-populations, including clinical endpoints like confirmed disability worsening, biomarkers (NfL, GFAP), and brain atrophy. Regarding the post-COVID trial, he explained that as it is investigator-sponsored, Immunic cannot provide guidance on timing but noted its scientific importance. He added that they may analyze post-COVID syndrome patients within the ENSURE and CALLIPER trials at a later date.

William Wood inquired about the level of detail expected in the April 2025 top-line data readout for the Phase II CALLIPER trial in progressive MS, specifically regarding subpopulation data and key endpoints. He also asked for the data timeline of the investigator-led post-COVID trial and how its fatigue findings might inform the ENSURE and CALLIPER trials.

Answer

CEO Dr. Daniel Vitt confirmed that the CALLIPER readout will be comprehensive, including detailed data on general and sub-form populations, confirmed disability worsening, biomarkers like NfL and GFAP, and brain atrophy. Regarding the post-COVID trial, he explained that as an investigator-sponsored study, Immunic cannot provide timelines but noted the scientific value in understanding fatigue and EBV's role. He added that post-COVID syndrome is not a predefined sub-analysis for the ENSURE or CALLIPER trials but could be explored later.

Asked about the timing of ENSURE baseline data release, expectations for new data at the upcoming ACTRIMS forum, the significance of blocking EBV reactivation, and how vidofludimus's MOA positions it against other MS therapies like anti-CD20s and CAR-Ts.

Answer

ENSURE baseline data will likely be released at unblinding in 2026. Upcoming ACTRIMS presentations will not contain new data but will focus on the EBV mechanism and the CALLIPER trial's NfL endpoint. Vidofludimus is differentiated by its unique Nurr1 neuroprotective mechanism, which targets non-inflammatory progression, in addition to its anti-inflammatory DHODH effect, addressing both key aspects of MS pathology.

William Wood from B. Riley Securities inquired about the procedural next steps following the HAP trial data, asking if results would be immediately submitted to the FDA or bundled into a larger end-of-Phase II meeting.

Answer

CEO Jennifer Good and CDO Dr. James Cassella explained that the clinical study report (CSR) will be submitted to the FDA per standard timing. The results will be discussed in the end-of-Phase II meeting for the IPF trial. They emphasized the HAP study does not gate ongoing clinical programs and is primarily part of the larger NDA package for potential approval.

William Wood from TD Cowen asked about the competitive landscape in proteomics, specifically how Seer views the evolution of the affinity-based market and whether growing overall awareness is a tailwind. He also questioned the outlook for gross margins exiting 2024 and into 2025, particularly if increased instrument placements could cause a dip.

Answer

CEO Omid Farokhzad stated that the proteomics market is in its infancy and that unbiased approaches like Seer's Proteograph are becoming more accessible, complementing rather than just competing with targeted affinity-based methods. He believes unbiased discovery will drive demand for targeted validation, creating a flywheel effect. CFO & President David Horn addressed margins, confirming they are on track to be around 50% for the full year 2024. He deferred providing specific 2025 guidance until the year-end call but acknowledged the quarterly variability based on product mix.