Kripa Devarakonda

Kripa Devarakonda questioned whether Scholar Rock plans to wait for the Catalent reinspection and OAI resolution before submitting the BLA. She also asked if Novo Nordisk could request an inspection and if they have engaged outside consultants to assist with the remediation process.

Answer

Akshay Vaishnaw, President of R&D, stated that the base case is to wait for the reinspection and OAI resolution before BLA submission, but acknowledged the dynamic situation and readiness to act if guided by the FDA. David Hallal, Chairman and Chief Executive Officer, confirmed Novo Nordisk is signaling readiness for an unannounced reinspection and is working with outside experts on the remediation plan.

Kripa Devarakonda inquired whether Scholar Rock's BLA resubmission plan involves waiting for the Catalent Bloomington facility's OAI classification to be resolved via reinspection. She also asked if Novo Nordisk could proactively request an inspection and if they have engaged any outside consultants to assist with the remediation process.

Answer

President of R&D Akshay Vaishnaw stated that the base case is to resubmit after the reinspection, but acknowledged the dynamic situation and readiness to file efficiently if the FDA supports an earlier submission. CEO David Hallal added that Novo Nordisk has signaled readiness for reinspection by year-end, implying they are effectively requesting it, and confirmed that Novo is working with outside experts on the remediation plan.

Kripa Devarakonda from Truist Securities asked about the timeline for knowing if a CDMO re-inspection is needed and the typical resolution timelines for such issues.

Answer

CEO David Hallal stated it is difficult to predict FDA actions but emphasized the company's focus is on submitting a "robust response" to the observations, which could influence the need for a re-inspection. He reiterated that the positive late-cycle meeting held after the observations were issued provides confidence that the FDA is working toward the September 22 PDUFA date.

An analyst on behalf of Kripa Devarakonda asked for more specifics on the future development path for the muscle preservation program, particularly regarding functional strength endpoints like grip strength or stair climb that could be used for regulatory review.

Answer

Executive Jay Backstrom explained that after establishing the right dose for SRK-439, they envision studies with enriched patient subgroups to demonstrate impact on endpoints like HbA1c, which is a clear regulatory path. For functional measures, he stated they are not committed to any specific test yet and plan to engage the FDA on meaningful functional endpoints after the IND is open, using early clinical data to inform the design of a registration program.

Speaking for Kripa Devarakonda, an analyst asked about the likelihood of apitegromab receiving a broad label that includes patients irrespective of their ambulatory status. They also questioned if there were any notable differences in how the FDA and European regulators view SMA that could impact final labeling decisions.

Answer

Executive Jay Backstrom responded that the FDA typically views SMA as a single disease continuum and has granted broad labels to other therapies without restrictions on ambulatory status. He anticipates a similar broad labeling approach for apitegromab. He also noted that European regulators seem to share this view, which is reinforced by the drug's PRIME designation. While labeling is negotiated late in the approval process, he expressed strong confidence in their position for a broad label based on the strength of the SAPPHIRE data.

Kripa Devarakonda from Truist Securities asked about the CDK2 partnership with Gilead, seeking details on the degrader's differentiation and development strategy, and also asked if the Q4 data would show the rapidity of itch relief.

Answer

CEO Nello Mainolfi explained the CDK2 program was partnered to align with Kymera's immunology focus. He highlighted its differentiation through high specificity over CDK1 and brain penetrance. CMO Jared Gollob and Nello Mainolfi confirmed the Q4 data will show the kinetics of itch relief, with measurements at days 7, 14, 21, and 28.

Kripa Devarakonda of Truist Securities inquired about the specifics of the ABCL635 Phase I trial, including patient numbers, and asked whether renewed advocacy for Hormone Replacement Therapy (HRT) could impact the market opportunity.

Answer

CEO Carl Hansen detailed the trial design, stating the SAD/MAD phases would enroll about 56-60 participants, while the proof-of-concept phase will enroll up to 80 postmenopausal women. He reiterated that the NKTR class targets a population where HRT is contraindicated or not preferred, representing a substantial and distinct market opportunity.

Anna, on for Kripa Devarakonda, asked what percentage of Parkinson's disease patients might be eligible for an LRRK2-targeting drug like ARV-102. She also questioned how potential neoadjuvant Vepdeq data could inform its use in earlier treatment settings.

Answer

CMO Noah Berkowitz explained that while LRRK2 mutations are present in a subset of familial and idiopathic Parkinson's patients, pathway perturbations are much broader, potentially implicating 30% or more of patients. The company is actively exploring patient selection strategies. Regarding Vepdeq, he reiterated that while there is every reason to believe it could be effective in earlier lines, the decision was made with Pfizer not to pursue those studies at this time, reinforcing the strategy to renegotiate or find a new partner.

Kripa Devarakonda asked about patient enrollment for the ARV-393 (BCL6) trial, its potential fit in the treatment landscape, and any concerns regarding FDA review timelines.

Answer

CMO Noah Berkowitz reported that after a slow start, enrollment for the ARV-393 Phase 1 trial is now steady with a backlog of patients. He sees potential for the drug as both a monotherapy and in combination, particularly with bispecifics. CEO John Houston added that Arvinas has not experienced any FDA-related delays but continues to monitor the situation.

Kripa Devarakonda from Truist Securities inquired about the drivers of Jakafi's growth in polycythemia vera (PV) and plans for life cycle management. She also asked for expectations for Monjuvy in follicular lymphoma (FL).

Answer

EVP & Head of U.S. Oncology Mohamed Issa identified PV as Jakafi's biggest growth driver due to being the least penetrated indication, with a focus on educating about earlier use. For Monjuvy in FL, he expects a gradual ramp due to the disease's indolent nature but sees potential for it to become a new standard of care and a significant incremental growth driver.

Nicole on for Kripa Devarakonda of Truist Securities asked when to expect data for the bimagrumab combination and whether the goal is incremental weight loss or improved body composition.

Answer

Executive Daniel Skovronsky stated that to show a benefit, the combination would likely need to demonstrate either incremental weight loss or a tangible functional benefit from lean mass preservation. He said it is a 'safe assumption' that data from the completed Phase II study will be shared this year.

Kripa Devarakonda of Truist Securities asked how the label updates for Camzyos could impact its peak sales opportunity and also relayed a concern about Cobenfy's pharmacy availability.

Answer

CCO Adam Lenkowsky explained that the Camzyos label update should increase capacity at Centers of Excellence, supporting continued growth, which will be further boosted by upcoming ODYSSEY data. He also stated that the reported pharmacy issue with Cobenfy was likely a one-off, as the company is not aware of broad availability problems. CMO Samit Hirawat directed analysts to a recent publication for details on the ODYSSEY trial design.

Alex Xenakis, on behalf of Kripa Devarakonda, asked if economic pressures are causing more partners to develop molecules in-house and whether this trend could impact OmniAb's business model.

Answer

CEO Matt Foehr expressed confidence in the company's current business model, stating he sees no change. He emphasized that partners specifically seek out OmniAb for its advanced discovery technologies to generate novel antibodies, a core function they are not moving away from. Foehr reiterated that the business model is purposely designed to be resilient through various industry cycles.

Kripa Devrakonda from Truist asked how Sanofi's separate collaboration on a STAT6 degrader might impact Chimera's IRAC4 partnership, and also asked if the KT474 dose expansion was discussed with the FDA.

Answer

CEO Nello Manalfi stated that Sanofi's interest in STAT6 validates the target's importance and does not expect it to impact their strong, independent IRAC4 collaboration. Regarding the FDA, he could not comment on specific regulatory discussions, noting that Sanofi, as the trial sponsor, is responsible for that communication.