Sezary Syndrome

Lund, Sweden – April 29, 2025 – BioInvent International AB (“BioInvent”) (Nasdaq Stockholm: BINV), a biotech company focused on the discovery and development of novel and first-in-class immune-modulatory antibodies for cancer immunotherapy, today announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to BI-1808, a first-in-class anti-TNFR2 antibody, for the treatment of adults with relapsed or refractory mycosis fungoides and Sézary syndrome, subtypes of cutaneous T-cell lymphoma (CTCL). Fast Track Designation (FTD) is intended to facilitate the development and review of drugs that treat serious conditions. “Receiving FDA’s Fast Track Designation following the recent Orphan Drug Designation underscores the potential of this novel immunomodulatory agent and reflects the urgent need for new, safe and durable treatment options for patients with CTCL", said Martin Welschof, Chief Executive Officer of BioInvent. “It’s very encouraging that the FDA confirms that the presented BI-1808 data meet expectations to address this important unmet medical need”. To date, BI-1808 demonstrated early clinical efficacy in heavily pretreated patients with an excellent safety and tolerability profile. We are committed to continue advancing the development of BI-1808 and look forward to providing an update from the ongoing Phase 2a by mid-2025.” CTCL is a rare and aggressive form of non-Hodgkin’s lymphoma that originates in T-lymphocytes residing in the skin, with the most prevalent and advanced subtypes mycosis fungoides and Sézary syndrome. It typically manifests with persistent skin lesions, itching, and potential systemic complications, significantly impacting patients’ quality of life and life expectancy. Each year, approximately 3,000 new cases are diagnosed in the United States with limited effective treatment options available.1 The FDA’s Fast Track Designation is designed to facilitate the development and expedite the review of drugs that treat serious conditions and address important unmet medical needs. The aim is to accelerate the development of urgently needed new drugs for patients. Drugs granted FTD are eligible for more frequent meetings with the FDA to discuss the drug development plan and ensure the collection of appropriate data needed to support approval, as well as eligibility for Accelerated Approval, Priority Review and Rolling Review if relevant criteria are met.2 About Phase 1/2a studyDuring the first part of the Phase 1/2a study (NCT04752826) the safety, tolerability, and potential signs of efficacy of BI-1808 as a single agent (Part A) and in combination with the anti-PD-1 therapy pembrolizumab (Part B) are evaluated in patients with advanced solid tumors and T-cell lymphoma. The efficacy of BI-1808 as single agent is currently explored in the Phase 2a part of the trial in a larger sample of patients. Expansion cohorts include ovarian cancer, all tumor types and T-cell lymphomas (including CTCL). The dose escalation in Phase 1 Part B has been completed and the Phase 2a dose expansion study is ongoing and include ovarian cancer, all tumor types and T-cell lymphoma (including CTCL). Results from the single agent CTCL cohort disclosed in the autumn of 2024 showed three patients with partial response (PR) and one with stable disease (SD) out of four evaluable patients. All these patients had previously deteriorated after standard treatment. These data corroborate the antitumoral activity observed with BI-1808 as single agent in the treatment of solid tumors disclosed earlier in 2024, showing one complete response (CR), one PR and nine patients with SD, presented at the American Society of Clinical Oncology conference (ASCO) in June 2024. Additional single agent data are expected by mid-2025. About BioInventBioInvent International AB (Nasdaq Stockholm: BINV) is a clinical-stage biotech company that discovers and develops novel and first-in-class immuno-modulatory antibodies for cancer therapy, with currently five drug candidates in six ongoing clinical programs in Phase 1/2 trials for the treatment of hematological cancer and solid tumors. The Company's validated, proprietary F.I.R.S.T™ technology platform identifies both targets and the antibodies that bind to them, generating many promising new immune-modulatory candidates to fuel the Company's own clinical development pipeline and providing licensing and partnering opportunities. The Company generates revenues from research collaborations and license agreements with multiple top-tier pharmaceutical companies, as well as from producing antibodies for third parties in the Company's fully integrated manufacturing unit. More information is available at www.bioinvent.com. 1. https://www.clfoundation.org/cutaneous-t-cell-lymphoma 2. https://www.fda.gov/patients/fast-track-breakthrough-therapy-accelerated-approval-priority-review/fast-track For further information, please contact:Cecilia Hofvander, VP Investor RelationsPhone: +46 (0)46 286 85 50Email: cecilia.hofvander@bioinvent.comBioInvent International AB (publ)Co. Reg. No.: 556537-7263Visiting address: Ideongatan 1Mailing address: 223 70 LUNDPhone: +46 (0)46 286 85 50www.bioinvent.comThe press release contains statements about the future, consisting of subjective assumptions and forecasts for future scenarios. Predictions for the future only apply as the date they are made and are, by their very nature, in the same way as research and development work in the biotech segment, associated with risk and uncertainty. With this in mind, the actual outcome may deviate significantly from the scenarios described in this press release.

February 17, 2025 -- Innate Pharma SA (Euronext Paris: IPH; Nasdaq: IPHA) (“Innate” or the “Company”) today announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to lacutamab, an anti-KIR3DL2 cytotoxicity-inducing antibody, for the treatment of adult patients with relapsed or refractory (r/r) Sézary syndrome (SS) after at least 2 prior systemic therapies including mogamulizumab. The BTD is granted based on Phase 1 study results as well as results from the Phase 2 TELLOMAK study, where lacutamab demonstrated encouraging efficacy and a favorable safety profile in heavily pretreated, post-mogamulizumab patients with advanced Sézary syndrome. “There is a high unmet medical need for patients with Sézary syndrome. In this aggressive and rare form of cutaneous T-cell lymphoma, patients in advanced disease often experience very poor quality of life and are in strong need of new, targeted treatment options,” commented Sonia Quaratino, MD, Chief Medical Officer of Innate Pharma. “The Breakthrough Therapy Designation underscores lacutamab’s potential to transform the patient’s care by achieving clinically meaningful efficacy and favorable safety profile compared to available therapies. This is an important step in Innate’s strategy for lacutamab. We are excited to work with the U.S. FDA to accelerate the development of this therapy.” A Breakthrough Therapy Designation by the FDA is intended to accelerate the development and regulatory review in the U.S. of drugs that are intended to treat a serious condition and that have shown encouraging early clinical results, which may demonstrate substantial improvement on a clinically significant endpoint over available medicines. Lacutamab previously received a Fast Track designation by the FDA in 2019 for the treatment of adult patients with relapsed or refractory Sézary syndrome who have received at least two prior systemic therapies as well as a PRIME designation by European Medicines Agency in 2020. Innate continues to align with the regulatory agencies around the confirmatory Phase 3 trial in CTCL and is actively seeking for a partner Cutaneous T-Cell Lymphoma (CTCL) is a heterogeneous group of non-Hodgkin’s lymphomas which arise primarily in the skin and are characterized by the presence of malignant clonal mature T-cells. CTCL accounts for approximately 4% of all non-Hodgkin’s lymphomas and has a median age at diagnosis of 55-65 years. Mycosis fungoides, and Sézary syndrome, its leukemic variant, are the most common CTCL subtypes. The overall 5-year survival rate, which depends in part on disease subtype, is approximately 10% for Sézary syndrome. There are approximately 6,000 new CTCL cases in Europe and the United States per year. Sézary syndrome is the leukemic variant of CTCL. Patients often experience very poor quality of life with severe and debilitating pruritus (chronic itchy skin). Despite recent advancements, Sézary syndrome is associated with a high relapse rate with currently available therapies. Lacutamab is a first-in-class anti-KIR3DL2 humanized cytotoxicity-inducing antibody that is currently in clinical trials for treatment of cutaneous T-cell lymphoma (CTCL), an orphan disease, and peripheral T cell lymphoma (PTCL). Rare cutaneous lymphomas of T lymphocytes have a poor prognosis with few efficacious and safe therapeutic options at advanced stages. KIR3DL2 is an inhibitory receptor of the KIR family, expressed by approximately 65% of patients across all CTCL subtypes and expressed by up 90% of patients with certain aggressive CTCL subtypes, in particular Sézary syndrome. It is expressed by up to 50% of patients with mycosis fungoides and peripheral T-cell lymphoma (PTCL). It has a restricted expression on normal tissues. Lacutamab is granted European Medicines Agency (EMA) PRIME designation and US Food and Drug Administration (FDA) granted Fast Track designation and Breakthrough Therapy Designation for the treatment of patients with relapsed or refractory Sézary syndrome who have received at least two prior systemic therapies. Lacutamab is granted orphan drug status in the European Union and in the United States for the treatment of CTCL. TELLOMAK (NCT03902184) is a global, open-label, multi-cohort Phase 2 clinical trial in patients with Sézary syndrome and mycosis fungoides (MF) in the United States and Europe. Specifically: The trial is fully enrolled. The primary endpoint of the trial is objective global response rate. Key secondary endpoints are progression-free survival, duration of response, overall survival, quality of life, pharmacokinetics and immunogenicity and adverse events. Source: Innate Pharma SA Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.

Innate has secured FDA’s fast track designation and PRIME designation from the European Medicines Agency for lacutamab. Credit: Stock-Asso/Shutterstock. Innate Pharma has secured US Food and Drug Administration (FDA) breakthrough therapy designation for lacutamab to treat adults with relapsed or refractory Sézary syndrome. Lacutamab is a first-in-class antibody designed to target killer cell immunoglobulin-like receptor 3DL2 (KIR3DL2) and induce cytotoxicity. It is being tested in clinical trials for cutaneous T-cell lymphoma (CTCL) and peripheral T-cell lymphoma. The FDA’s designation follows positive results from the Phase I study and the Phase II TELLOMAK trial, which showed lacutamab’s efficacy and safety in patients who had previously undergone at least two systemic therapies, including mogamulizumab. TELLOMAK is a global Phase II clinical trial for patients with Sézary syndrome and mycosis fungoides in the US and Europe. It is an open-label, multi-cohort study. The trial’s primary endpoint is an objective global response rate, with key secondary endpoints including progression-free survival, overall survival, duration of response and quality of life. Innate Pharma previously received fast track designation from the FDA in 2019 and PRIME designation from the European Medicines Agency in 2020 for lacutamab. The company is currently aligning with regulatory agencies for a confirmatory Phase III trial in CTCL and is seeking a partner. Lacutamab also holds orphan drug status in both the European Union (EU) and the US for the treatment of CTCL. Innate Pharma chief medical officer Sonia Quaratino stated: “There is a high unmet medical need for patients with Sézary syndrome. “In this aggressive and rare form of cutaneous T-cell lymphoma, patients in advanced disease often experience very poor quality of life and are in strong need of new, targeted treatment options.” In March 2024, Innate dropped plans to assess lacutamab as a monotherapy in KIR3DL2-expressing refractory/relapsing peripheral T-cell lymphoma following disappointing data from a Phase Ib trial.