Goldman Sachs Group, Inc. has raised the target price of Agios (AGIO.US) to 28 dollars, bullish on the prospects of the new drug Aqvesme for Mediterranean anemia.

Goldman Sachs Group, Inc. raises Agios Pharmaceuticals' (AGIO.US) 12-month target price from $25 to $28, corresponding to a potential increase of approximately 14%, maintaining a "neutral" rating. The analysis by Goldman Sachs Group, Inc. focuses on the market prospects and risk assessment of Agios Pharmaceuticals' core drug Aqvesme (mitapivat) following FDA approval. Agios Pharmaceuticals, as a biopharmaceutical company focused on innovative therapies, has become the first and only drug approved to treat adult transfusion-dependent (TD) and non-transfusion-dependent (NTD) - or -thalassemia. This approval is based on significant efficacy data from the Ph3 ENERGIZE-T and ENERGIZE trials - in NTD patients, 42.3% of patients in the Aqvesme treatment group showed a significant increase in hemoglobin concentration compared to 1.6% in the placebo group; in TD patients, 10% achieved transfusion independence within 48 weeks, compared to 1% in the placebo group. Specifically, the FDA approval is based on two Phase III registration trials: ENERGIZE (NTD) and ENERGIZE-T (TD). In the ENERGIZE trial, 42.3% of patients in the Aqvesme group achieved the primary endpoint of hemoglobin 1 g/dL out of 194 subjects, while only 1.6% in the placebo group (p <0.0001); secondary endpoints such as fatigue scores and average hemoglobin concentrations were also met. In ENERGIZE-T, 10% of patients in the Aqvesme group achieved transfusion independence within 48 weeks, compared to 1% in the placebo group, with a significant reduction in transfusion burden. Because of the solid data, Goldman Sachs Group, Inc. has increased the likelihood of Aqvesme's market entry for thalassemia from 90% to 100%, and has also raised the model's US annual pricing to $425,000, higher than the company's already marketed molecule (Pyrukynd, used for pyruvate kinase deficiency) at $335,000. However, aside from efficacy, liver damage signals have prompted regulatory caution. Five Aqvesme patients in Phase III experienced suspected hepatocellular injury, with two requiring hospitalization; all events occurred within the first six months of treatment, with liver parameters recovering after discontinuation. As a result, the FDA has required the initiation of a REMS program: liver function must be checked before medication, with testing every four weeks for the first 24 weeks, followed by clinical monitoring according to indications; patients, prescribing physicians, and pharmacies must all complete certification training. Agios expects to begin commercial distribution by the end of January 2026, one step behind Pyrukynd without REMS. However, the company believes that procedural obstacles will not significantly slow penetration. How big is the market exactly? Management has initially targeted approximately 4,000 "easiest" patients: 2,000 with TD and an additional 2,000 with significant symptoms, low hemoglobin, or prominent fatigue in the NTD patient population. In the long term, the total accessible patient population in the US is estimated to be around 6,000, with two-thirds being NTD, forming a "seven-three" structure alongside TD. Based on this model, Goldman Sachs Group, Inc. predicts Aqvesme sales of $69 million in the 2026 fiscal year (compared to the Visible Alpha consensus of $38 million), and anticipates prescription and revenue to ramp up in sync from 2027 onwards, reaching a global peak of around $600 million by 2033, almost in line with the Wall Street consensus of $588 million. It is worth noting that the company has indicated that the first few quarters may show a characteristic of "prescriptions leading revenue": there is a 10-12 week lag between filling out initiation forms and actual dosing, meaning that it may not be until the end of 2026 or early 2027 that the two curves align. On the cash flow front, Goldman Sachs Group, Inc. has adjusted revenue forecasts for 2025-2027 to $36.5 million, $47 million, and $146 million respectively, while maintaining a pace of narrowing losses, projecting a loss of $6.25 per share in 2027, about $1 less than the old model. Valuation is based on a risk-adjusted 100% DCF with a WACC of 17% and perpetual growth rate of 3%, yielding a target price of $28. Including pipeline premiums, Goldman Sachs Group, Inc. has factored in scenarios where Pyrukynd is 100% successful for pyruvate kinase deficiency, 70% successful for sickle cell disease, and Aqvesme is 100% successful for thalassemia; in other words, $28 incorporates a full discounting of visible commercial assets. The risks and upside potential are equally clear. Upside catalysts include: successful label expansions for sickle cell disease, clinical setbacks for competitors leading to market share redistribution, faster-than-expected market entry and patient penetration; downside risks include poor pipeline readouts, escalated liver toxicity, competition taking the lead, pressure from payers to lower prices, intellectual property or production bottlenecks. Goldman Sachs Group, Inc. explicitly notes that the company is still in a net loss position, and if the commercialization pace falls short of expectations, financing needs may resurface. In conclusion, Goldman Sachs Group, Inc. believes that Agios will realize its first wave of value in the short term through the US launch of Aqvesme, with the mid-term focusing on when the prescription-revenue mismatch converges, and the long-term success depending on whether Aqvesme can capture additional indications for sickle cell disease. The $28 target price corresponds to a market-to-sales ratio of approximately ten times the 2027 revenue forecast, not a cheap valuation but within reason for a "one-hit wonder" narrative in rare diseases. Investors should closely monitor the prescription curve and liver safety signals that will be initiated in the first quarter of next year; as long as these two factors do not diverge, Agios' small-cap biotech stock will continue to attract event-driven funds.