Tessa Romero

Tessa Romero sought clarity on Scholar Rock's BLA submission strategy for 2026, specifically if Catalent will remain the primary fill-finish facility and if the SBLA for the additional facility will follow potential BLA approval. She also inquired about the status of the EMA review concerning manufacturing-related items.

Answer

David Hallal, Chairman and Chief Executive Officer, confirmed the plan to resubmit the BLA with Catalent as the primary filler, given the constructive Type A meeting and Novo's progress, with the second facility added via an SBLA later in 2026. Akshay Vaishnaw, President of R&D, stated that the EMA review is proceeding as expected, with a decision anticipated mid-next year, and that U.S. BLA efforts are aligned to support the MAA.

Tessa Romero sought clarification on Scholar Rock's BLA submission strategy, specifically whether the 2026 BLA for apitegromab will designate Catalent as the primary fill-finish facility, with an SBLA for an additional facility later in 2026. She also asked for an update on the EMA review process concerning manufacturing-related items.

Answer

CEO David Hallal confirmed that the plan is to resubmit the BLA with Catalent as the primary filler, given Novo Nordisk's progress and the constructive FDA meeting, with the second filler to be added via an SBLA later. President of R&D Akshay Vaishnaw stated that the EMA review is proceeding as expected, with a decision anticipated by mid-next year, and that U.S. BLA efforts are aligned to support the MAA, acknowledging the interdependency regarding manufacturing status.

Tessa Romero from J.P. Morgan questioned the next steps following the CDMO observations, the likelihood of a timely and positive review outcome, and what an ideal label for apitigramab would entail.

Answer

CEO David Hallal outlined the plan to work with CDMOs on robust responses to the FDA. President of R&D Akshay Vaishnaw stated the ideal label would cover patients aged two and older, based on consistent SAFIRE trial data across age groups. Hallal added that patient advocacy groups like Cure SMA have advocated for such broad labels.

Tessa Romero sought confirmation on whether the mid-cycle review meeting with the FDA for apitegromab has occurred and asked about any high-level discussions regarding the potential label or the need for an Advisory Committee meeting.

Answer

CEO David Hallal and President of R&D Akshay Vaishnaw both reiterated that the regulatory process is on track. Dr. Vaishnaw described interactions with the FDA as 'extremely constructive' but declined to provide specific details on meetings or labeling discussions, while reaffirming confidence in the September 22 PDUFA date.

Tessa Romero inquired about Scholar Rock's framework for exploring additional neuromuscular indications for apitegromab, such as DMD. She asked how the company plans to allocate capital for these potential new studies while ensuring sufficient investment in the SMA launch and what the timeline for starting new trials might be.

Answer

Executive Jay Backstrom explained that the company feels obligated to explore adjacent indications like DMD and FSHD following apitegromab's success in SMA. He stated the clinical team has the capacity to take on at least one additional study and emphasized the goal is to build a multi-billion dollar neuromuscular franchise, starting with the potential $2 billion-plus opportunity in SMA alone.

Tessa Romero from JPMorgan Chase & Co. requested more specific quantitative details on the patient numbers within the three key target populations for the initial PYRUKYND launch in thalassemia and asked about the frequency of their provider visits.

Answer

CCO Tsveta Milanova stated the initial launch will target approximately 65% of the 6,000 diagnosed U.S. adults. She identified three segments: transfusion-dependent patients (seen frequently, e.g., monthly), non-transfusion-dependent patients with comorbidities, and NTD patients with debilitating fatigue. Specific breakdowns were not provided, but she noted these groups are actively engaged with the healthcare system.

Tessa Romero of JPMorgan Chase & Co. asked for confirmation that the potential risk of hepatocellular injury would remain in the 'warnings and precautions' section of the Pyrokine label and inquired about the timing for presenting open-label extension data for the thalassemia population.

Answer

CMO Dr. Sarah Gheuens stated that the regulatory review is ongoing and the final label details will be available at the PDUFA date. She also mentioned that while the open-label extension data will be published, the team's current priority is the ongoing regulatory reviews.

Tessa Romero from JPMorgan Chase & Co. sought confirmation on whether the FDA mid-cycle meeting for PYRUKYND in thalassemia has occurred, asked about any high-level labeling discussions, and questioned the potential for a REMS requirement.

Answer

CMO Dr. Sarah Gheuens explained that the FDA review is a continuous process of interaction rather than one with formally defined meetings like the mid-cycle review. She stated that engagement is collaborative and ongoing, and it is currently too early for labeling negotiations, which occur later in the review cycle. While ultimate certainty on a REMS comes only with approval, she conveyed that the process is progressing normally and they are pleased with where they stand.

Tessa Romero of JPMorgan Chase & Co. asked about the expected evolution and cadence of SG&A and R&D expenses through 2026 and for an update on the anticipated timing of regulatory approvals in the EU, Saudi Arabia, and the UAE.

Answer

CFO Cecilia Jones indicated that both SG&A and R&D expenses are expected to grow year-over-year to support upcoming launches and pipeline advancement, though she did not quantify the growth. For ex-U.S. approvals, CMO Dr. Sarah Gheuens noted that timelines are less certain than the U.S. PDUFA date, but progress is on track. CCO Tsveta Milanova added that a time lag between approval and commercialization is expected in these regions due to pricing and reimbursement processes.

Tessa Romero from JPMorgan Chase & Co. asked if physicians appreciate the lack of pharmacogenomic and drug-drug interaction (DDI) liability with aficamten compared to Camzyos, and whether pharmacy-level monitoring for concomitant medications would be required.

Answer

EVP of R&D Fady Malik stated that physicians in their trials recognize aficamten's different DDI profile, which doesn't involve CYP2C19, and that any potential interactions are rare and manageable without a formal monitoring program. EVP & CCO Andrew Callos clarified that the company does not anticipate pharmacy-level monitoring being part of a potential REMS program for aficamten.

Tessa Romero of JPMorgan Chase & Co. asked about the ideal label for aficamtan in obstructive HCM and for specifics on the updated REMS program that was submitted to the FDA.

Answer

President and CEO Robert Blum stated that while he could not share specifics on the ongoing FDA review, an ideal label would reflect aficamtan's differentiated properties. Andrew Callos, EVP & Chief Commercial Officer, added that market research indicates a differentiated label and REMS would support strong uptake and expansion into community cardiology.

Tessa Romero of JPMorgan Chase & Co. asked if the FDA had indicated a REMS was unnecessary, why the lack of a REMS in the NDA was not disclosed, and when the FDA requested it.

Answer

Robert I. Blum, President and CEO, stated that the decision not to submit a REMS was based on their interpretation of FDA feedback from three meetings. He explained the lack of disclosure was a strategic decision to maintain competitive advantage and was not unconventional. The FDA's request for the REMS was recent, which then prompted the company's public communications.

Tessa Romero from JPMorgan Chase & Co. inquired about the company's confidence in a positive outcome for the Phase III MAPLE-HCM trial and the magnitude of effect needed for statistical significance.

Answer

Fady Malik, EVP of R&D, stated that the trial is highly anticipated by investigators and has enrolled the target patient population. He noted the trial is conservatively powered to detect a delta-Peak VO2 of 1.5 to 2.0 mL/kg/min, a range consistent with previous positive data, expressing confidence in the study's design and hypothesis.

Tessa Romero from J.P. Morgan asked for specifics on the EXTOL-two trial, including the timing for final patient randomization, the geographic split of patient recruitment, the number of clinical sites used, and whether there has been any interest in compassionate use for Ezetucalner.

Answer

President and CEO Ian Mortimer explained that the last patients would be randomized following an eight-week baseline period. Chief Medical Officer Christopher Kenney added that while final numbers are pending, the geographic split and site strategy are expected to be similar to the successful EXTOL study, though with more sites to accommodate a larger trial. Kenney also confirmed constant interest in compassionate use, but stated no program is currently active.

Tessa Romero inquired about the number of patients remaining to be recruited for the X-TOLE2 study, the rationale for adding new trial sites, and how the screen failure rate compares to the previous X-TOLE study.

Answer

President and CEO Ian Mortimer noted that recruitment is nearing completion and will be finished in the next few months, attributing some variability to normal trial ebbs and flows. He clarified that the few late-additions of trial sites are not significant. Chief Medical Officer Dr. Chris Kenney added that the screen failure rate is very similar to the Phase II study due to nearly identical inclusion/exclusion criteria.

An analyst on behalf of Tess Romero asked about the synergies between Neumora's neuroscience pipeline and the new focus on obesity, the rationale for entering the indication, and for clarification on what R&D initiatives are included in the current cash runway guidance.

Answer

Joshua Pinto, President, addressed the questions. He stated that obesity fits squarely within Neumora's mandate to tackle large population health disorders driven by central mechanisms, leveraging the team's expertise in developing brain-penetrant molecules. Regarding the cash runway, he confirmed the balance of $217 million provides runway into 2027 and fully funds all critical programs, including NMRA-215, through their currently guided clinical milestones. This funding covers the six potential clinical catalysts expected over the next 18 months.

Tessa Romero asked for an update on NMRA-266, which has been on clinical hold for nearly a year, and questioned if the preclinical convulsions observed were specific to that particular candidate.

Answer

President Joshua Pinto stated that while work on the NMRA-266 clinical hold continues, the company is advancing its follow-on M4 PAM franchise and expects to move a new compound into the clinic by mid-2025. He confirmed it would be logical to de-risk these follow-on compounds for the convulsion issue before clinical progression and that a full update on the M4 franchise would be provided at that time.

Tessa Romero inquired about the planned approach for the ACP101 top-line data release, asking about the level of detail to be provided and the significance of secondary endpoints beyond the primary HQCT.

Answer

EVP, Head of Research & Development, Elizabeth Thompson, stated that the top-line release will focus on the primary endpoint and a high-level overview of safety and tolerability. She noted that secondary endpoints include clinician assessments and a responder analysis for the HQCT. Dr. Thompson also mentioned that a key focus during the trial has been robustly monitoring assessment performance and variability to ensure data consistency across sites.

Tessa Romero of JPMorgan Chase & Co. asked for guidance on the expected sequential growth from Q1 to Q2 for both NUPLAZID and DAYBUE, and the key drivers to consider for financial modeling.

Answer

An executive, likely CCO Thomas Garner, explained that DAYBUE's growth will be driven by an expanded field force and improved patient persistency, while NUPLAZID's momentum will be sustained by direct-to-consumer campaigns. CFO Mark Schneyer added that for both brands, volume will be the primary growth driver for the remainder of the year, with DAYBUE also benefiting from gradual price increases.

Tessa Romero asked for clarification on the Q1 2025 outlook for DAYBUE, questioning if a decline in active patients was expected, and inquired about year-end patient targets and long-term penetration goals.

Answer

CFO Mark Schneyer clarified that the patient base is expected to remain stable, with the anticipated Q1 revenue decline driven by seasonal pull-forward and net price changes, not fewer patients. CCO Tom Garner added that with 70% of the Rett population yet to try DAYBUE, a key growth strategy is penetrating community settings, which treat 65% of patients but currently have only 25% penetration.

An analyst on behalf of Tess Romero of JPMorgan Chase & Co. asked for clarification on the statement that the Daybue launch has reached a 'steady state of new patient flow,' inquiring if this means steady net adds and what the strategy is to accelerate new patient starts.

Answer

CEO Catherine Owen Adams explained that after a strong initial launch burst, new patient additions have plateaued, which is common for rare disease launches. To re-accelerate growth, the strategy focuses on three areas: 1) better communicating the drug's efficacy by translating clinical scales into real-world benefits, 2) improving management of the early patient journey to enhance persistence, and 3) increasing penetration outside of Centers of Excellence (COEs), where 70% of patients are treated.

Tessa Romero inquired about the overall trial conduct for EyePoint's two pivotal wet AMD studies, LUGANO and LUCIA, and the key strategies being employed to mitigate risks.

Answer

CMO Dr. Ramiro Ribeiro and CEO Dr. Jay Duker responded. Dr. Ribeiro highlighted the company's experience from prior studies, close collaboration with clinical sites to ensure protocol adherence, and diligent safety tracking. Dr. Duker added that the non-inferiority trial design is familiar to physicians and that the current patient dropout rate is very low at less than 2%, which helps de-risk the program.

Tessa Romero of J.P. Morgan inquired about the overall conduct of the two pivotal wet AMD studies and EyePoint's key areas of focus for mitigating risks now that enrollment is complete.

Answer

CMO Ramiro Ribeiro stated that the focus is now on study conduct, leveraging experience from prior trials. He highlighted close collaboration with clinical sites to prevent protocol deviations and diligent safety tracking. CEO Jay Duker added that the non-inferiority trial design is familiar to physicians and the patient dropout rate is very low at less than 2%, which helps de-risk the studies.

Tessa Romero asked for the reasons behind the rapid enrollment in the Phase III wet AMD trials and inquired about the company's primary focus for mitigating clinical trial execution risks.

Answer

Chief Medical Officer Dr. Ramiro Ribeiro attributed the strong enrollment to the patient-centric trial design and the robust Phase I/II data package, which gives investigators confidence. He stated that the team's primary focus for execution is on ensuring pristine data integrity to support an efficient NDA submission.

Tessa Romero from JPMorgan Chase & Co. asked for the primary reasons behind the rapid enrollment in the wet AMD trials and inquired about the company's main focus for mitigating clinical trial execution risks.

Answer

Chief Medical Officer Dr. Ramiro Ribeiro, as directed by CEO Dr. Jay Duker, attributed the strong enrollment to the robust Phase I and II data giving investigators confidence, and a patient-centric, easy-to-follow trial design. Dr. Ribeiro stated the team is focused on ensuring data integrity across all areas to prepare for an efficient NDA submission.

Tessa Romero inquired about the clinical site activation for EyePoint's wet AMD pivotal program, asking for the current number of active sites for the LUGANO and LUCIA trials and the target for ex-U.S. locations.

Answer

Chief Medical Officer Ramiro Ribeiro stated that most U.S. sites are activated, with approximately 60 active sites per study. He added that the company plans to activate between 60 and 80 ex-U.S. sites per study later in the year, noting that enrollment numbers are progressing very well.

Tessa Romero of JPMorgan asked for clarification on the Krabbe disease trial, specifically whether the IDMC had given a go-ahead for continued dosing, how the inclusion/exclusion criteria would change, and if it's possible to identify patients at higher risk for hydrocephalus. She also questioned the feasibility of reporting data on all four patients in the expanded cohort by year-end.

Answer

President and CEO Bruce Goldsmith confirmed that the IDMC reviewed the data and recommended continuing with trial recruitment. He clarified that the clinical trial protocol and informed consent forms must first be revised to incorporate the IDMC's recommendations, which include enhanced screening based on baseline imaging to identify at-risk patients. Regarding the year-end data readout, Goldsmith reiterated the commitment to report initial safety and biomarker data but noted that the number of patients included in that update would depend on the pace of recruitment throughout the year.

Tessa Romero of JPMorgan Chase & Co. asked if the company had previously discussed the IHS-4 score with regulators and requested a comparison of IFX-1's observed improvements on draining fistulas with published data for Humira.

Answer

Niels Riedemann, an executive, clarified that they had not yet discussed the IHS-4 score with regulators but noted it was developed and partially validated by the HS foundation and key opinion leaders (KOLs) to have low variability and correlate well with patient improvement. Regarding Humira, he stated that to his knowledge, the original Pioneer trial publications did not focus on specific lesion counts or draining fistulas, though some non-comparable mean numbers may have been submitted to U.K. authorities.