Corinne Jenkins

Corinne Jenkins requested details on the development of the subcutaneous BRIUMVI product, including the planned administration method (auto-injector or prefilled syringe) and the remaining work required on the device side for approval.

Answer

Chairman and CEO Michael Weiss explained that the pivotal trial will use a syringe from a vial to establish bioequivalence, followed by a bridging study to an auto-injector. He clarified that the company plans to use a standard, well-established auto-injector, and does not anticipate major technical challenges. The development work is factored into the existing timeline, which targets a BLA filing in 2027 and a potential launch in 2028.

Corinne Jenkins of Goldman Sachs inquired about the proportion of MS patients treated in the hospital setting and why it's a strong growth area, and asked if new patient growth could accelerate in 2025.

Answer

Chief Commercialization Officer Adam Waldman estimated that 60-65% of MS patients are treated in the hospital setting, making it a key growth segment. CEO Michael Weiss stated that accelerating new patient growth in 2025 is possible as the user base reaches a critical mass, and the company is investing in its commercial team and marketing to fuel this potential acceleration.

Corinne Jenkins of Goldman Sachs asked for clarification on whether the 'fully funded' status of the pociredir program applies only to the current high-risk patient group or if it encompasses a potentially broader patient population.

Answer

Executive Alexander Sapir reiterated the strategy to use data from the current high-risk cohort to negotiate with the FDA for a broader patient population. CFO Alan Musso clarified that the company's financial forecasting and cash runway guidance into 2027 already account for the complete, multi-stage development plan anticipated for pociredir.

Corinne Jenkins inquired about the specifics of the FDA's requirements for validating the reachable workspace (RSA) endpoint for losmapimod, and also asked what clinical and commercial activities are covered by the company's cash runway guidance into 2027.

Answer

Executive Iain Fraser explained that since RSA is a new endpoint, the FDA has not set a pre-established numerical criterion, and Fulcrum's work aims to define a 'meaningful score difference' for individual patients. CFO Alan Musso confirmed the cash runway guidance assumes a 'full success scenario,' funding losmapimod's development, filing, and commercialization, the ongoing pociredir trial and a follow-up trial, and preclinical work.

Corinne Jenkins of Goldman Sachs requested more detail on what 'steady growth' implies, whether it refers to absolute patient numbers or growth rate, and asked about the current mix of second-line versus third-line patients starting therapy.

Answer

EVP & CCO Jim Ziegler clarified that Geron expects growth across all patient segments in both academic and community settings. He noted the initial focus is on increasing the breadth of prescribing accounts, with depth expected to follow. He confirmed uptake is being seen across all lines of therapy, including first-line ESA-ineligible patients.

Corinne Jenkins asked about the expected revenue contribution from Europe in 2025 and requested an update on patient adherence and compliance rates for IMCIVREE in the Bardet-Biedl syndrome (BBS) population.

Answer

Chief Financial Officer Hunter Smith indicated that while international revenue is expected to grow, its pace relative to the larger U.S. base will be variable. Chairman, CEO and President David Meeker and EVP, Head of North America Jennifer Chien noted the discontinuation rate is near 30%. They are focused on improving adherence through better education on efficacy timelines and maintaining engagement with patients who have discontinued, some of whom have returned to therapy.

Corinne Jenkins asked about the clinical measurement of fatigue for the pitolisant HD study and requested more detail on the patents extending its exclusivity into the 2040s.

Answer

Chief Medical and Scientific Officer Dr. Kumar Budur noted that fatigue is prevalent in 60-70% of narcolepsy patients and that Harmony is developing a specific measurement instrument for the trial. CEO Dr. Jeffrey Dayno explained the long-dated patents for pitolisant HD are based on its unique formulation, gastro-resistant coating, and improved pharmacokinetic profile, which will be supported by clinical data.

Corinne Jenkins of Goldman Sachs asked for clarification on how AL002's mechanism can be related to amyloid clearance without requiring significant centiloid reduction for clinical effect. She also inquired about which ABC platform activities are covered by the current cash runway guidance.

Answer

Dr. Gary Romano, CMO, and Dr. Arnon Rosenthal, CEO, elaborated that AL002's mechanism is broad, restoring overall microglial health which supports synaptic repair and myelination, independent of amyloid removal. They noted microglia can also contain amyloid plaques, providing benefit without full clearance. Dr. Marc Grasso, CFO, added that the cash runway through 2026 is expected to advance ABC platform programs through IND filings.

Asked about the greatest near-term growth opportunities for OGSIVEO given new patient visibility, and for mirdametinib, the initial adoption targets and how its launch trajectory might compare to OGSIVEO's.

Answer

For OGSIVEO, the opportunity is to continue driving depth of prescribing in Centers of Excellence and both breadth and depth in the community setting. For mirdametinib, the opportunity is considered at least as large as desmoid tumors, targeting the sizable adult population (first-in-class) and pediatric population (best-in-class), rather than just 'low-hanging fruit'.

On behalf of Corinne Jenkins, an analyst asked if Aclaris is sufficiently funded to complete the ATI-2138 proof-of-concept study and where the company identifies the primary unmet need in the atopic dermatitis market.

Answer

Interim CEO Neal Walker confirmed that the company is fully funded for the 'exceedingly cost-efficient' study, supported by a robust balance sheet and low expected cash burn. Regarding the unmet need, Walker stated that the atopic dermatitis market is still in its early stages, with significant room for efficacy improvement. He noted that even successful biologics like dupilumab leave many patients with suboptimal responses, and Aclaris aims to fill this gap with ATI-2138's strong dual-inhibition mechanism.

Speaking on behalf of Corinne Jenkins, an analyst asked if Aclaris is sufficiently funded to complete the proof-of-concept study and where the company identifies the greatest unmet need in atopic dermatitis.

Answer

Interim CEO Neal Walker affirmed that the company is "certainly funded" to complete the cost-efficient study, citing a robust balance sheet and a low expected burn rate. On the unmet need, Walker described the atopic dermatitis market as being in its early stages with significant room for improvement in efficacy, noting that even successful biologics leave many patients with suboptimal responses and that there is "a lot of headroom" for more effective treatments.

Corinne Jenkins asked if management had conducted a blinded review of the ACT-AD study data and if any insights could be shared. She also requested an update on the enrollment pace for the LIFT-AD study since its expansion and whether the company would announce the completion of enrollment.

Answer

Chief Medical Officer Hans Moebius stated he could not comment on any blinded data findings as the trial is ongoing. President & CEO Mark Litton confirmed they are still targeting the end of Q3 to complete enrollment for the LIFT-AD study and will issue an announcement when that milestone is achieved.