Knight Therapeutics Announces Regulatory Approval of NIKTIMVO® in Brazil

MONTREAL, Oct. 06, 2026 (GLOBE NEWSWIRE) -- Knight Therapeutics Inc. ("Knight") (TSX: GUD), a pan-American (ex-US) pharmaceutical company, announced today it has received expedited regulatory approval under ANVISA’s rare disease pathway for NIKTIMVO^® (axatilimab), for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients 6 years and older weighing at least 40 kg.¹

“For patients living with chronic graft-versus-host disease who have exhausted two prior lines of therapy, access to a new treatment option can be life-changing. NIKTIMVO^® offers a first-in-class mechanism that targets the underlying drivers of this debilitating condition, and we are proud to bring it to patients in Brazil. This approval reflects the strength of our partnership with Incyte and our shared commitment to ensuring that patients in Brazil have access to innovative therapies that can make a meaningful difference in their lives,” said Samira Sakhia, President and CEO of Knight Therapeutics.

In September 2021, Knight and Incyte (NASDAQ: INCY) had entered into an exclusive supply and distribution agreement for MINJUVI^® (tafasitamab) and PEMAZYRE^® (pemigatinib) in Latin America. In August 2025, Knight expanded its existing relationship with Incyte, to add exclusive rights to distribute ZYNYZ^® (retifanlimab) and NIKTIMVO^® (axatilimab) in Latin America. Under the terms of the amended agreement, Incyte is responsible for the development, manufacture and supply of such products to Knight, and Knight is responsible for seeking the necessary regulatory approvals and commercializing such products in Latin America.

About NIKTIMVO^®

NIKTIMVO^® (axatilimab-csfr) is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody approved for use in the U.S. for the treatment of chronic GVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs).²

In September 2021, Syndax Pharmaceuticals, Inc. and Incyte entered into an exclusive worldwide co-development and co-commercialization license agreement for axatilimab in chronic GVHD and any future indications.

Axatilimab is being studied in frontline combination trials in chronic GVHD – a Phase 2 combination trial with ruxolitinib (NCT06388564) and a Phase 3 combination trial with steroids (NCT06585774) are underway. Axatilimab is also being studied in an ongoing Phase 2 trial in patients with idiopathic pulmonary fibrosis (NCT06132256).

NIKTIMVO^®, MINJUVI^®, PEMAZYRE^® and ZYNYZ^® are trademarks of Incyte. All other trademarks are the property of their respective owners.

About cGVHD

Chronic GVHD is a serious complication of allogeneic stem cell transplantation in which the donor’s immune cells attack the recipient’s tissues, often occurring during the first-year post-transplant.³ Chronic GVHD involves features of autoimmunity and immunodeficiency, multiple organs (such as the skin, liver, lungs, and gastrointestinal tract), irreversible fibrotic manifestations, and systemic toxicities complicated by the use of immunosuppressants which all contribute to devastating health consequences such as mortality, morbidity, and decreased quality of life in patients.⁴ There are approximately 1400 – 1800 reported allogeneic transplants in Brazil every year.⁵ Chronic GVHD is the most common late complication after allogeneic hematopoietic cell transplantation affecting 30%-70% of recipients, globally.⁴ Based on a recent analysis by the Brazilian National hematopoietic stem cell transplantation (HSCT) Registry, in collaboration with Brazilian Society of Cellular Therapy and Bone Marrow Transplantation (SBTMO), Center for International Blood and Marrow Transplant Research (CIBMTR), and Hematopoietic Cell Transplantation Brazilian Registry (HCTBR), the 2-year cumulative incidence cGVHD was determined to be 29.5%.⁶

About Knight Therapeutics Inc.

Knight Therapeutics Inc., headquartered in Montreal, Canada, is a pharmaceutical company focused on acquiring, in-licensing and commercializing pharmaceutical products for Canada and Latin America. Knight’s Latin American subsidiaries operate under United Medical, Biotoscana Farma and Laboratorio LKM. Knight Therapeutics Inc.'s shares trade on the TSX under the symbol GUD. For more information about Knight Therapeutics Inc., please visit the company's website at www.knighttx.com or www.sedarplus.ca.

Forward-Looking Statements for Knight

This document contains forward-looking statements for Knight Therapeutics Inc. and its subsidiaries. These forward-looking statements, by their nature, necessarily involve risks and uncertainties that could cause actual results to differ materially from those contemplated by the forward-looking statements. Knight Therapeutics Inc. considers the assumptions on which these forward-looking statements are based to be reasonable at the time they were prepared but cautions the reader that these assumptions regarding future events, many of which are beyond the control of Knight Therapeutics Inc. and its subsidiaries, may ultimately prove to be incorrect. Factors and risks which could cause actual results to differ materially from current expectations are discussed in Knight Therapeutics Inc.’s Annual Report and in Knight Therapeutics Inc.’s Annual Information Form for the year ended December 31, 2025, as filed on www.sedarplus.ca. Knight Therapeutics Inc. disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information or future events, except as required by law.

References

1. ANVISA, Prescribing Information. Approved on October 5, 2026.
2. Incyte Corporation. NIKTIMVO (axatilimab-csfr) injection, for intravenous use: Highlights of prescribing information / full prescribing information. Revised August 2024. Accessed June 10, 2026. https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/761411s000lbl.pdf
3. Jagasia M, Giglia J, Chinratanalab W, et al. Incidence and outcome of chronic graft-versus-host disease using National Institutes of Health consensus criteria. Biol Blood Marrow Transplant. 2007;13(10):1207-1215. doi:10.1016/j.bbmt.2007.07.001
4. Kitko CL, Arora M, DeFilipp Z, et al. Axatilimab for Chronic Graft-Versus-Host Disease After Failure of at Least Two Prior Systemic Therapies: Results of a Phase I/II Study. J Clin Oncol. 2023;41(10):1864-1875. doi:10.1200/JCO.22.00958
5. Associação Brasileira de Transplante de Órgãos. (2024). Registro Brasileiro de Transplantes (RBT): Dados numéricos da doação de órgãos e transplantes realizados por estado e instituição no período: janeiro/setembro 2024 (Ano XXV, nº 3) [Report]. Retrieved May 26, 2026, from https://site.abto.org.br/wp-content/uploads/2024/11/RBT2024-3t-abto-populacao.pdf
6. Simione, Anderson Joao, et al. “Hematopoietic Cell Transplantation in Brazil: A National Benchmarking Study Focused on the Foundation for the Accreditation of Cellular Therapy (FACT) Performance Indicators.” Journal of Bone Marrow Transplantation and Cellular Therapy (JBMTCT), vol. 6, no. 1, 2025, pp. 1–13. doi: 10.46765/2675-374X.2025V6N1P270.

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