Core Viewpoint - Goldman Sachs raised the 12-month target price for Agios Pharmaceuticals (AGIO.US) from $25 to $28, indicating a potential upside of approximately 14%, while maintaining a "Neutral" rating. The analysis focuses on the market prospects and risk assessment of its core drug, Aqvesme (mitapivat), following FDA approval [1]. Group 1: Drug Approval and Market Potential - Aqvesme is the first and only drug approved for treating adult transfusion-dependent (TD) and non-transfusion-dependent (NTD) alpha or beta thalassemia, based on significant efficacy data from Phase 3 ENERGIZE-T and ENERGIZE trials [1][2]. - In the ENERGIZE trial, 42.3% of patients in the Aqvesme group achieved a hemoglobin increase of ≥1 g/dL, compared to only 1.6% in the placebo group (p<0.0001) [2]. - In the ENERGIZE-T trial, 10% of patients in the Aqvesme group became transfusion-independent within 48 weeks, while only 1% in the placebo group achieved this [2]. Group 2: Sales Forecast and Market Size - Goldman Sachs increased the probability of Aqvesme's market launch for thalassemia from 90% to 100%, setting the annual U.S. price at $425,000, higher than the $335,000 for Pyrukynd, which is used for pyruvate kinase deficiency [2]. - The management has identified approximately 4,000 "easiest" patients to target initially: 2,000 in the TD population and 2,000 with significant symptoms or low hemoglobin in the NTD group. The total addressable population in the U.S. is estimated at around 6,000, with NTD patients making up two-thirds [3]. - Goldman Sachs forecasts Aqvesme sales of $69 million for FY2026, with a gradual increase in prescriptions and revenue expected to align by late 2026 or early 2027, reaching a global peak of approximately $600 million by 2033 [3]. Group 3: Financial Projections and Valuation - Goldman Sachs adjusted revenue forecasts for 2025-2027 to $36.5 million, $47 million, and $146 million, respectively, while maintaining a trend of narrowing losses, projecting a loss of $6.25 per share in 2027, about $1 less than previous estimates [4]. - The target price of $28 is derived using a 100% risk-adjusted DCF model, with a WACC of 17% and a perpetual growth rate of 3%. This valuation includes the successful commercialization of existing assets [4]. - The company is still in a net loss position, and if commercialization speeds are below expectations, financing needs may arise again [4]. Group 4: Strategic Considerations - Goldman Sachs believes Agios will realize initial value from Aqvesme's U.S. launch in the short term, with medium-term focus on the alignment of prescriptions and revenue, and long-term potential hinging on the success of expanding indications for sickle cell disease [5]. - The target price corresponds to a revenue multiple of about ten times the 2027 revenue forecast, which, while not cheap, is considered reasonable within the narrative of rare disease blockbuster products [5]. - Investors are advised to closely monitor the prescription curve and liver safety signals starting in Q1 of next year, as alignment in these areas will be crucial for maintaining interest from event-driven funds [5].

Summary of KalVista Pharmaceuticals Conference Call Company Overview - **Company**: KalVista Pharmaceuticals (NasdaqGM: KALV) - **Industry**: Pharmaceuticals, specifically focusing on treatments for Hereditary Angioedema (HAE) - **Product**: EKTERLY, the first and only oral therapy for HAE attacks, launched on July 7, 2025 after receiving approval on July 3, 2025 [2][3] Key Points Launch and Market Uptake - EKTERLY has achieved a conversion rate of approximately 10%-12% of patients within the first four months of launch, indicating strong market uptake [3] - Revenues exceeded expectations for the initial commercial quarter, reflecting high patient demand [3][4] Revenue Insights - The revenue reported includes a stocking element typical for high-priced therapies, but a large portion is attributed to actual patient sales [4] - The company is still assessing the breakdown between new starts and refills due to limited experience [4][5] Payer Coverage and Market Dynamics - Payer coverage is progressing as anticipated, with smaller formularies approving policies faster than larger ones [6][7] - Most prescriptions in the initial quarter were medical exceptions due to the lack of established policies [7] - Prior authorization is required for prescriptions, with some policies necessitating a trial of generic Icatibant before EKTERLY [8] Patient Demographics - A significant portion of early adopters are patients with high attack rates (two or more attacks per month), validating the company's hypothesis about this patient population [10][11] - These high-attack patients are expected to have higher refill rates due to their increased need for on-demand therapy [11][12] Future Growth and Market Potential - The market for EKTERLY is estimated to be around 8,000 to 9,000 patients, with a potential shift towards oral therapies over time [17][18] - The company anticipates that the majority of patients will eventually switch to oral therapies, which could lead to sustained growth [18][19] Competitive Landscape - The company is aware of potential competition from other oral therapies but believes EKTERLY's strong launch and patient satisfaction will position it well in the market [21][22] - The company expects to maintain a competitive edge due to its extensive data set and high patient satisfaction [22] International Expansion - KalVista has launched EKTERLY in Germany and plans to expand into the UK and other European markets, with a focus on maintaining a cost-effective operational model [29][30] - Pricing power in Europe is limited, with prices expected to be around 20%-25% of US prices [31] Long-term Strategy - The company aims to grow into a leading rare disease commercial entity, considering additional high-quality assets for future development [28] - KalVista has established a strong commercial and regulatory framework, with five global regulatory approvals as of the conference date [26][27] Clinical Development - The company has conducted the largest clinical trial program in HAE, with plans to file an NDA for a pediatric indication in Q3 of the following year [35] Additional Insights - The company emphasizes the importance of maintaining operational efficiency and managing administrative challenges related to prior authorizations [20] - The focus remains on building a robust company rather than seeking acquisition opportunities [28]