Colleen Kusy

Colleen Kusy asked about the expected details in the SOL1 topline data, specifically if six-month BCVA would be included, and what data points would provide confidence for the read-through to SOLAR.

Answer

Dr. Pravin Dugel (Executive Chairman, President and CEO) stated that the company has not yet guided on specific SOL1 topline data details, including six-month BCVA. He emphasized SOL1's goal is a superiority label, while SOLAR aims for clinical relevance, assuring that SOL1's secondary and exploratory analyses would provide confidence for SOLAR's success.

Colleen Kusy from Robert W. Baird & Co. questioned if the FDA was consulted on the SOLAR rescue criteria change, confirmed the previous criteria included a 10-letter loss rule, and asked about the impact on the primary endpoint and trial timeline.

Answer

Pravin Dugel, Executive Chairman, President & CEO, confirmed the change was a purely strategic decision and not discussed with the FDA, as it has no bearing on the primary endpoint. He also affirmed that the change does not delay the SOLAR trial's timeline, highlighting the study's robust design which includes a lengthy de-risking phase for patient selection.

Colleen Kusy from Robert W. Baird & Co. asked for more detail on the higher baseline Total Symptom Score (TSS) in the pivotal myelofibrosis study and its potential impact, and also questioned the rationale behind the observed lower rates of grade 3/4 anemia with the combination therapy.

Answer

Chief Medical Officer Reshma Rangwala explained that historical data suggests a higher baseline TSS is more likely to result in a meaningful clinical outcome, making the current trend in the SENTRY trial encouraging. Regarding anemia, she attributed the lower rates to selinexor's disease-modifying properties, citing prior data showing reductions in key cytokines involved in anemia and preliminary evidence of improved bone marrow health, which could allow for the repopulation of red blood cell precursors.

Colleen Kusy of Robert W. Baird & Co. asked about the investments being made to penetrate the community oncology setting and their expected impact on sales, potential data readouts from partners for Loktorzi label expansion, and the enrollment status of the randomized Phase II trial for casdozoketog.

Answer

EVP of Commercial Sameer Goregaoker detailed a multi-pronged strategy for the community setting, expecting peak sales in 3-4 years. CEO Dennis Lanfear and CSO Theresa Lavallee clarified that their strategy involves supplying Loktorzi for partner trials, not out-licensing, with a potential update from STORM Therapeutics next year. Chief Medical Officer Rosh Dias confirmed that enrollment for the casdozoketog HCC trial is progressing well and is on track for data in H1 2026.

Colleen Kusy of Robert W. Baird & Co. asked if the EMA feedback on the melanoma application would affect the regulatory path for non-small cell lung cancer in the U.S. and questioned what specific problems the new specialty pharmacy channel solves for treatment centers.

Answer

Interim CEO Frederick Vogt and Chief Regulatory Officer Raj Puri asserted the EMA feedback has no impact on the U.S. regulatory path for NSCLC. CCO Dan Kirby explained the specialty pharmacy channel solves a financial hurdle for hospitals, particularly medium-sized community centers, whose finance departments may be unwilling to purchase high-cost therapies directly.

Colleen Kusy of Baird asked for clarification on whether the out-of-spec rate specifically increased, if this was linked to the annual maintenance, and if the one-month manufacturing disruption will be a recurring annual event.

Answer

Chief Operating Officer Igor Bilinsky attributed the transient Q1 issue to new ATC and surgeon learning curves on patient selection and tumor procurement, not the maintenance. He confirmed the maintenance is an annual event but stated its impact will be reduced in future years once the iCTC facility build-out is complete.

Colleen Kusy asked if there was a slowdown in patient infusions during the latter half of Q4 and whether any holiday-related seasonality was observed.

Answer

Interim CEO and President Frederick Vogt confirmed there was no holiday-related seasonality. He did note that a planned manufacturing maintenance period occurred, which is a normal event in cell therapy that can cause minor peaks and valleys in launch curves, similar to what has been seen with CAR-T products.

Colleen Kusy asked about the FDA's perspective on rescue criteria, how rescued patients are evaluated in the Phase 3 data, and for any additional color on the feedback received from the FDA regarding the DME program.

Answer

CEO Dr. Jay Duker explained that the FDA allows companies to define their own rescue criteria, and EyePoint's are based on Phase 2 data. He clarified that rescue injections are not considered treatment failures and will be handled with sensitivity analyses. Regarding DME, he described the end-of-Phase 2 meeting with the FDA as positive and said more details on the pivotal plan will be shared after receiving the official written minutes.

Colleen Kusy from Robert W. Baird & Co. asked about the FDA's view on rescue criteria and how rescued patients are evaluated, and also requested any available color on the feedback from the FDA regarding the DME program.

Answer

CEO Jay Duker explained that the FDA allows companies to set their own rescue criteria and that supplemental injections are not considered treatment failures but are handled via sensitivity analyses. Regarding DME, he reported a 'very pleased' outcome from discussions with the agency and confirmed plans to enroll the first patient in the pivotal DME program in 2026, with more details to be shared after receiving written minutes from the FDA.

Colleen Kusy asked if EyePoint expects differences in patient types or results between U.S. and ex-U.S. sites, and also inquired about the FDA's non-inferiority margin for DME trials compared to wet AMD.

Answer

Chief Medical Officer Ramiro Ribeiro stated that he does not expect significant differences between sites, as wet AMD trials are globally mature and the study was designed to accommodate a global population. He also noted that while subject to FDA discussion, the non-inferiority margin for DME has historically been 4 letters, versus 4.5 for wet AMD.

The analyst asked about the design of the wet AMD Phase III trial, specifically seeking clarification on the role of the low-dose arm. She questioned whether the higher dose needs to outperform the lower dose or if the second arm is primarily for masking purposes.

Answer

The executive, Jay Duker, explained that from the FDA's perspective, the second dose arm serves to improve study masking, and there is no requirement for one dose to be superior to the other. From the company's perspective, including two doses is strategic because the Phase II trial showed no dose-response. This allows them to test a dose around 2mg and a potentially lower dose, with the trial powered for the lower dose to also demonstrate non-inferiority to the control.

Colleen Kusy from Robert W. Baird & Co. asked for an update on the disruption in third-party co-pay assistance and its expected resolution. She also inquired about expectations for the VAIL long-term extension study data and how it might differentiate Empaveli in C3G and iCMPGN.

Answer

Co-Founder and CEO Dr. Cedric Francois stated that Apellis supports the patient assistance foundations but operates independently of their funding dynamics. Chief Medical Officer Dr. Caroline Baumal highlighted that Empaveli's differentiation comes from achieving a 'trifecta' of outcomes (proteinuria reduction, eGFR stabilization, C3 deposit clearance), which continued into the VAIL extension study, exciting physicians.

Colleen Kusy from Robert W. Baird & Co. asked for an update on the third-party co-pay assistance disruption and about expectations for the VAIL long-term extension study data in differentiating Empaveli for C3G and iCMPGN.

Answer

CEO Dr. Cedric Francois commented that Apellis supports the patient assistance foundations. CMO Dr. Caroline Baumal added that Empaveli's 'trifecta' of positive outcomes (proteinuria reduction, eGFR stabilization, C3 deposit clearance) continued into the VAIL extension study, which is exciting physicians and helps differentiate the product.

Colleen Kusy from Robert W. Baird & Co. inquired about any updates on the disruption in third-party co-pay assistance programs and asked about expectations for the VAIL long-term extension study data in differentiating Empaveli for C3G and iCMPGN.

Answer

Co-Founder and CEO Cedric Francois stated that Apellis supports the patient assistance foundations. Chief Medical Officer Dr. Caroline Baumal highlighted that Empaveli's differentiation comes from achieving a 'trifecta' of outcomes (proteinuria reduction, eGFR stabilization, C3 deposit clearance), and noted these positive effects continued in the VAIL extension study, exciting physicians.

Colleen Kusy of Robert W. Baird & Co. asked for an update on the third-party co-pay assistance disruption and about expectations for the VAIL long-term extension study data in differentiating Empaveli for C3G and iCMPGN.

Answer

CEO Dr. Cedric Francois stated Apellis supports patient foundations but is independent of their funding. CMO Dr. Caroline Baumal highlighted that the VAIL extension data confirms Empaveli's 'trifecta' of benefits (proteinuria, eGFR, C3 clearance), which continues to differentiate the product and resonate with physicians.

Colleen Kusy of Baird asked for the percentage of the market where SYFOVRE has preferred positioning and about the similarity between U.S. and European regulatory reviews for C3G/IC-MPGN.

Answer

EVP of Commercial David Acheson noted significant preferred access with plans like Aetna but stated it's hard to give a specific market percentage. CEO Dr. Cedric Francois added that Apellis and its partner Sobi are in 'lockstep' on the global deployment of EMPAVELI for nephrology, highlighting the large opportunity where they will only compete for about 20% of the 5,000 U.S. patients.

An associate for Colleen Kusy asked about the upcoming XMT-2056 pharmacodynamic (PD) data update, inquiring what the company needs to demonstrate, the potential number of patients, and if early efficacy data might be included.

Answer

An executive responded that the primary objective of the update is to share PD data demonstrating selective activation of the STING pathway in HER2-positive tumors. The company declined to provide further details on patient numbers, follow-up, or the potential inclusion of efficacy data at this time.

Asked about any seasonality observed in Q4, the typical adoption speed of dermatologists and GIs compared to other specialists, and any notable differences in the patient types they see.

Answer

The company noted that Q1 typically faces financial headwinds like insurance reverifications and ordering dynamics, which are factored into the annual guidance. Dermatologists often see patients with skin involvement (cutaneous mastocytosis), while GIs see patients with IBS-like symptoms. Engaging these specialties is expected to grow the market and increase treatment rates.

Colleen Kusy of Robert W. Baird & Co. asked for an update on the prostate cancer patients from the ORIC-101 trial who were still on treatment at the last update, clarification on why PFS was similar in selected vs. unselected groups, and about learnings applicable to the ORIC-944 program.

Answer

CEO Jacob Chacko confirmed the remaining prostate cancer patients had all progressed, with times on treatment ranging from 3.7 to 9.2 months. He explained the PFS similarity was due to a subgroup with unknown GR status (due to lack of tumor cells in biopsy) having a longer PFS, which skewed the unselected population data. For ORIC-944, he stated it's a different target (EED) with single-agent potential, but learnings from ORIC-101's translational work on tumor heterogeneity will be applied.