Release Date: August 01, 2024
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
Positive Points
- Agios Pharmaceuticals Inc (AGIO, Financial) reported positive momentum in Q2 with multiple key milestones, including the Phase 3 ENERGIZE-T study of mitapivat meeting both primary and secondary endpoints in adults with transfusion-dependent alpha or beta-thalassemia.
- The company plans to submit an sNDA for mitapivat in thalassemia by the end of 2024, seeking a label that covers all subtypes of thalassemia.
- Agios Pharmaceuticals Inc (AGIO) announced a significant financial agreement with Royalty Pharma, which will provide an upfront payment of $905 million upon FDA approval of vorasidenib.
- The company reported strong cash reserves, ending the second quarter with approximately $645 million in cash and investments.
- Agios Pharmaceuticals Inc (AGIO) has completed enrollment in the ACTIVATE-kids study and looks forward to top-line readout in 2025, indicating progress in their pediatric studies.
Negative Points
- The Phase 3 ACTIVATE-kids T study of mitapivat did not meet the prespecified statistical criterion for the primary endpoint using Bayesian methodology.
- There were adverse events reported in the mitapivat arm of the ENERGIZE-T study, with 5.8% of patients experiencing an AE that led to discontinuation compared to 1.2% in the placebo arm.
- The company expects more muted growth going forward and quarter-over-quarter variability in PKD revenues as they pivot focus to thalassemia launch preparedness.
- R&D expenses increased by $8.5 million year-over-year, primarily due to costs associated with the TMPRSS6 program in-licensed from siRNA.
- SG&A expenses also saw an increase of $5.1 million compared to the same quarter in 2023, attributed to commercial-related activities in preparation for the potential approval of PYRUKYND in thalassemia.
Q & A Highlights
Q: Can you give us more color on the NewBridge transaction and the timing now that you have breakthrough designation in Saudi Arabia?
A: Tsveta Milanova, Chief Commercial Officer, explained that NewBridge will represent Agios across regulatory approval and commercialization activities in the GCC region. The agreement is a revenue split deal with no upfront payments. The breakthrough designation in Saudi Arabia is significant, and Agios plans to submit for regulatory approval for thalassemia as soon as possible.
Q: How are you viewing M&A now with the potential $1 billion-plus infusion from the vorasidenib PDUFA?
A: Brian Goff, CEO, emphasized that the focus remains on efficient capital use, with top priority on preparing for and delivering upcoming launches in thalassemia and sickle cell disease. While M&A is always a consideration, the company is disciplined and has strong internal opportunities.
Q: Can you discuss the applicability of VOCs as an endpoint in sickle cell disease and its relevance to a broad patient population?
A: Sarah Gheuens, Chief Medical Officer, noted that while not all sickle cell patients experience VOCs, the drug aims to treat all aspects of the disease, including hemolytic anemia and sickle cell pain crises. The clinical trial focuses on patients with two or more VOCs to measure treatment effect, but the drug is expected to benefit a broader population.
Q: Could you provide more details on the statistical assumptions and clinical meaningfulness of the ACTIVATE-kids T study?
A: Sarah Gheuens explained that the study used complex statistical methodology to leverage adult data. While the primary endpoint was not met using low or moderate borrowing, the results were clinically meaningful, with six patients becoming transfusion-free. The safety data was also consistent with previous findings.
Q: What are your expectations for real-world usage patterns of PYRUKYND in thalassemia, especially regarding long-term patient retention?
A: Tsveta Milanova highlighted that initial focus will likely be on transfusion-dependent patients and those with lower hemoglobin levels and fatigue. Over time, the aim is to expand to a broader patient population through education and awareness campaigns.
Q: Are there any changes in sex hormone levels in the mitapivat arm versus the placebo in the ACTIVATE-kids T study?
A: Sarah Gheuens stated that detailed safety data, including sex hormone levels, will be presented at an upcoming medical meeting.
Q: When can we expect to see the Phase 2a data for AG-946, and will it be presented at a medical meeting?
A: Sarah Gheuens confirmed that the Phase 2a data will be presented at an upcoming medical meeting, with the current focus on the Phase 2b study for low-risk MDS.
Q: What additional insights can we expect from the full Phase 3 ENERGIZE-T data presentation later this year?
A: Sarah Gheuens mentioned that the presentation will include detailed analyses of primary and secondary endpoints, as well as safety data. The data will underscore mitapivat's potential as a foundational treatment for all subtypes of thalassemia.
Q: What are the gating factors for the sNDA submission for thalassemia, and should we expect a standard or priority review?
A: Sarah Gheuens indicated that the team is focused on preparing the submission package and is eager to work with regulators. Specific timelines and review types were not disclosed.
Q: How soon could we start seeing revenue from the NewBridge arrangement in the GCC region?
A: Tsveta Milanova explained that the process involves regulatory submission, pricing, and reimbursement agreements, followed by formulary approvals. While specific timelines were not provided, the focus is on expediting patient access.
For the complete transcript of the earnings call, please refer to the full earnings call transcript.
