Goldman Sachs raised the target price of Agios (AGIO.US) to $28, optimistic about the launch prospects of Aqvesme, a new drug for anemia in China

The Zhitong Finance App learned that Goldman Sachs raised the 12-month target price of Agios Pharmaceuticals (AGIO.US) from $25 to $28, corresponding to a potential increase of about 14%, maintaining a “neutral” rating. The bank's in-depth analysis of Agios Pharmaceuticals focused on market prospects and risk assessments after its core drug, Aqvesme (mitapivat), was approved by the FDA.

As a biopharmaceutical company focusing on innovative therapies, Agios Pharmaceuticals became the first and only drug approved to treat adult transfusion-dependent (TD) and non-transfusion-dependent (NTD) α- or beta-thalassemia anaemia. The approval was based on significant efficacy data from Ph3 ENERGIZE-T and ENERGIZE trials — among non-transfusion-dependent patients, 42.3% of patients in the Aqvesme treatment group had a significant increase in hemoglobin concentration, compared with only 1.6% in the placebo group; transfusion-dependent Of the patients, 10% achieved independent blood transfusion within 48 weeks, and only 1% in the placebo group.

Specifically, FDA approval is based on two Phase III registered clinical trials: ENERGIZE (NTD) and ENERGIZE-T (TD). In the ENERGIZE trial, 42.3% of patients in the Aqvesme group reached the main end point of hemoglobin ≥1 g/dL, while placebo was only 1.6% (p<0.0001); secondary indicators such as fatigue score and average hemoglobin concentration also met the standards. In terms of ENERGIZE-T, 10% of patients in the Aqvesme group got rid of blood transfusions within 48 weeks, and only 1% in the placebo group. The drop in blood transfusion load was also significant. Precisely because of solid data, Goldman Sachs directly adjusted Aqvesme's marketing probability of thalassemia from 90% to 100%, and simultaneously set the US annual price of 425,000 US dollars in the model, which is a bit higher than the 335,000 US dollars that the company has already listed the same molecule (Pyrukynd, for pyrukynd, for pyruvate kinase deficiency).

However, in addition to curative effects, signs of liver damage make it difficult to relax supervision. Five Aqvesme patients in phase III showed suspected hepatocyte damage. Of these, 2 were hospitalized. All events occurred within the first six months of drug use, and liver indicators recovered after discontinuation of the drug. Therefore, the FDA requires the launch of the REMS program: liver function must be checked before use, reviewed every 4 weeks for the first 24 weeks, and followed according to clinical indications thereafter; patients, prescribers, and pharmacies are all required to complete certification training. Agios is expected to be officially launched at the end of January 2026, one step later than Pyrukynd without REMS, but the company believes that procedural barriers will not significantly slow penetration.

How big is the market? Management first targeted about 4,000 “easiest” patients: 2,000 TD patients, plus 2,000 NTD patients with obvious symptoms, low hemoglobin, or severe fatigue. In the long run, the US can reach a total of about 6,000 people, of which NTD accounts for two-thirds, and TD has an “inverted 37” structure. Based on this model, Goldman Sachs predicts Aqvesme's sales of 69 million US dollars in fiscal year 2026 (Visible Alpha consensus is 38 million US dollars), and believes that prescriptions and revenue will rise at the same time from 2027, reaching a global peak of about 600 million US dollars in 2033, which almost coincides with Wall Street's consensus estimate of 588 million US dollars. Notably, the company revealed that the first few quarters will show the characteristics of “prescriptions ahead of revenue”: there is a 10-12 week lag from filling out the initiation form to actual administration, so the two curves will not coincide until the end of 2026 or the beginning of 2027.

In terms of cash flow, Goldman Sachs adjusted its 2025-2027 revenue forecast to 36.5 million, 47 million, and 146 million US dollars in sequence, while maintaining a narrowing pace of losses. It is expected to lose 6.25 US dollars per share in 2027, which is about 1 US dollar less than the old model. The valuation used 100% risk-adjusted DCF, WACC 17%, and a sustainable growth rate of 3% to arrive at a target price of $28. If the pipeline premium is included, Goldman Sachs's scenario already includes Pyrukynd's 100% success in pyrukynd's pyrukynd's pyruvate kinase deficiency, 70% success in sickle cell disease, and Aqvesme's 100% success in thalassemia; in other words, $28 has fully discounted “visible” commercial assets.

The risk is as clear as the room for upward flexibility. Upward catalysts include: successful subsequent expansion of the sickle cell disease label; share redistribution from competitor clinical setbacks; faster than expected pace of market launch and patient penetration; downward risks include poor pipeline readings, escalating hepatotoxicity, competitive preemption, payer pressure cuts, intellectual property rights, or production bottlenecks. Goldman Sachs specifically warned that the company is still in a state of net loss. If commercialization falls short of expectations, financing needs may return.

Taken together, Goldman Sachs believes that Agios relied on Aqvesme's US listing to achieve the first wave of value in the short term. In the medium term, it depends on when the prescription-revenue mismatch will subside; in the long run, it depends on whether the sickle cell disease indications can go back to the next level. The target price of 28 US dollars corresponds to the 2027 revenue forecast market sales rate. The valuation is not cheap, but it is also reasonable to put it in the “single product heavy” narrative of rare diseases. What investors need to keep an eye on is the prescription curve and liver safety signals that will be launched in the first quarter of next year. As long as the two do not diverge in direction, Agios's small-cap biotech stock will continue to attract event-driven capital.