Belite Bio, BioArctic, and Recordati Report Q4 2025 Financial Results

Belite Bio announced its preliminary, unaudited financial results for the fourth quarter and full-year ended December 31, 2025, and provided a business update. Following positive topline results from the pivotal, global Phase 3 DRAGON trial of tinlarebant in adolescents with Stargardt disease type 1 (STGD1), the Company is on track to submit a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in Q2 2026. The Company completed a $402 million underwritten public offering of American Depositary Shares.

Tinlarebant is an oral, potent, once-daily, retinol binding protein 4 (RBP4) antagonist that decreases RBP4 levels in the blood and reduces vitamin A (retinol) delivery to the eye without disrupting systemic retinol delivery to other tissues. Tinlarebant has been granted Breakthrough Therapy, Fast Track, and Rare Pediatric Disease Designations in the U.S.; Orphan Drug Designation in the U.S., Europe, and Japan; and Sakigake (Pioneer Drug) Designation in Japan for the treatment of STGD1.

The DRAGON Trial was a completed, 24-month, 104 subjects, aged 12 to 20 years old, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in adolescent STGD1 patients. The trial met its primary efficacy endpoint, demonstrating a statistically significant and clinically meaningful 35.7% reduction in the growth rate of macular lesions, measured as definitely decreased autofluorescence (DDAF) by fundus autofluorescence imaging, compared with placebo. The trial achieved statistical significance when applying the pre-specified analysis (p-value = 0.0033). A post-hoc analysis demonstrated that the treatment effect remained (35.4%) consistent with a p-value < 0.0001 when considering the progressive nature typically seen in STGD1.

Belite Bio completed enrollment in the Phase 2/3 DRAGON II trial in STGD1. Targeted enrollment of 60 subjects was reached in January 2026. 72 subjects were enrolled as of February 27, 2026. The trial design and inclusion of Japanese patients are intended to facilitate a future NDA in Japan. The primary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability will also be assessed.

The Company's PHOENIX Trial is an ongoing, 24-month, randomized (2:1, active: placebo), double-masked, placebo-controlled, global, multi-center, pivotal Phase 3 trial in geographic atrophy (GA) patients. The trial completed enrollment with 530 subjects. The primary efficacy endpoint is the growth rate of atrophic lesions; safety and tolerability will also be assessed.

BioArctic reported record financial results for 2025, with an operating profit of more than SEK 1.2 billion. Net revenues for the fourth quarter 2025 amounted to SEK 184.0 M (101.2), of which SEK 127.0 M (96.7) in royalties for Leqembi and SEK 51.1 M (-) from the agreement with Novartis. Operating profit amounted to SEK 33.2 M (-53.5). Cash and cash equivalents and short-term investments at the end of the period amounted to SEK 2,190.4 M (778.9). The Board of Directors proposed a dividend of 2.00 SEK per share to be paid for the financial year 2025.

In the fourth quarter, royalty revenues grew by 31 percent compared to the same quarter the year before, despite significant negative currency impact. Leqembi is now approved in more than 50 markets and sales continue to grow every quarter. In the US, Japan and China, the number of patients on treatment is steadily increasing.

Leqembi Iqlik was launched for weekly maintenance dosing in October 2025, marking the first time the therapy can easily be administered at home via an autoinjector. The FDA is reviewing Leqembi Iqlik for initial treatment under a priority review process, with a decision expected by the end of May. Leqembi Iqlik sBLA for subcutaneous initiation dose was granted Priority Review by the US FDA. In China and Japan, the regulatory authorities are conducting similar reviews, with China also running a priority process. BLA for subcutaneous initiation treatment with Leqembi was accepted and designated for priority review in China.

Leqembi was approved in Brazil and Canada during the fourth quarter 2025. Leqembi was approved for IV maintenance treatment in the United Kingdom. The first Nordic patient was treated with Leqembi at private clinic in Finland. New Leqembi data presented at CTAD 2025 suggested potential to delay disease progression by up to 8.3 years with continuous treatment.

Recordati reported strong preliminary full-year 2025 results. The Group delivered on its financial targets despite a challenging macroenvironment, including increased FX headwinds. There is excellent momentum in Rare Diseases, which continues to be a key driver of growth and value creation for the Group.

On April 15, 2025, the U.S. Food and Drug Administration (FDA) approved the supplemental new drug application (sNDA) for Isturisa (osilodrostat) for the treatment of endogenous hypercortisolemia in adults with Cushing's syndrome for whom surgery is not an option or has not been curative. This was an expansion of the previous indication for the treatment of patients with Cushing's disease, which is a sub-type of Cushing's syndrome. The Isturisa indication expansion was supported by the extensive Isturisa clinical development program, which included over 350 patients. During the second quarter of 2025, Isturisa was granted regulatory approval in both Canada and Russia. A Phase IV study to assess the efficacy and safety of osilodrostat in adults with mild hypercortisolemia and uncontrolled hypertension (HTN) due to Cushing's syndrome is expected to start in 2026.

On July 28, 2025, the European Commission issued a positive decision and granted marketing authorization, under exceptional circumstances, for Maapliv, a solution of amino acids intended for the treatment of maple syrup urine disease (MSUD) presenting with an acute decompensation episode in patients from birth who are not eligible for an oral and enteral branched-chain amino acids (BCAA)-free formulation.

Recordati completed enrollment of the pasireotide Phase 2 trial for the treatment of post-bariatric hypoglycemia in August 2025. Top-line results are expected in the second quarter of 2026. Following the meeting with the U.S. Food and Drug Administration (FDA) in early September, a potential U.S. biologics license application (BLA) pathway was established with the FDA for Qarziba requiring an additional set of clinical data from the ongoing BEACON-2 investigator-sponsored trial. Results of the interim analysis are expected in the first half of 2028 and are expected to form the basis, together with existing clinical data, for a potential regulatory filing.

On January 5, 2026, the UK Medicines and Healthcare products Regulatory Agency (MHRA) granted marketing authorization for Eligard for the treatment of hormone dependent advanced prostate cancer and for the treatment of high-risk localized and locally advanced hormone dependent prostate cancer in combination with radiotherapy.

On June 24, 2025, Recordati announced a licensing and supply agreement with Amarin to commercialize the marketed cardiovascular medicine, Vazkepa (icosapent ethyl) across 59 countries, focused in Europe. Vazkepa is indicated to reduce the risk of cardiovascular events in statin-treated adult patients at high cardiovascular risk with elevated triglycerides and either established cardiovascular disease or diabetes with at least one other cardiovascular risk factor. Vazkepa is expected to achieve over € 40 million in revenues in 2027 and to be EBITDA positive from 2026. Under the terms of the agreement, Recordati paid Amarin an upfront cash payment of USD 25 million.