Thermosome Receives U.S. Orphan Drug Designation for Lead Compound THE001, Expands Patent Estate

May 12, 2025 at 04:00 AM EDT

ODD granted for the treatment of soft tissue sarcomas (STS)
Thermosome adds 4 patents to its growing patent estate

Munich, Germany – May 12, 2025 – Thermosome, a drug development company focused on targeted tumor therapies, today announced that its lead compound, THE001, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of soft tissue sarcomas (STS). This designation complements the ODD already granted by the European Medicines Agency (EMA) and validates the clinical potential of THE001.

The FDA's ODD program supports the development of drugs for rare diseases and provides benefits such as tax credits for qualified clinical trials, exemption from user fees, and seven years of market exclusivity following approval. In its decision, the FDA recognized that Thermosome's innovative doxorubicin-encapsulating thermosensitive liposomes may be clinically superior to currently approved non-liposomal forms of doxorubicin due to its potential for increased efficacy.

“We see the U.S. Orphan Drug Designation as a strong regulatory validation of the potential of our innovative approach in soft tissue sarcoma,” said Dr. Pascal Schweizer, co-founder and CEO/CFO of Thermosome. “This recognition, based on preclinical and early clinical data from our Phase 1 study, marks an important milestone and is a further step into the U.S. market – the world’s most important market for patent-protected drugs. In parallel, we are evaluating strategic partnerships to advance THE001 and fully realize its therapeutic potential.”

Separately, Thermosome has significantly expanded its intellectual property portfolio with four new PCT patent applications recently published. These patents strengthen the protection of THE001 and the Company’s technology platform:

PCT/EP2024/076239: Covers the synthetic phospholipid excipient DPPG₂, which is incorporated into Thermosome’s lead candidate THE001, potentially providing composition of matter protection by claiming a single stereoisomer form.
PCT/EP2024/076242: Describes a novel lipid excipient toolbox for independent adjustment of liposomal surface charge, offering enhanced control over protein corona and liposomal fate.
PCT/EP2024/076287: Protects new lipid excipients for lipid-based nanoparticles (LNPs) designed for RNA/DNA delivery.
PCT/EP2024/076319: Covers a new LNP morphology with improved dispersion stability for the delivery of small molecules and genetic material.

These patent applications bring Thermosome’s total number of patent families to seven. Key families include the composition of matter patent for DPPG₂, which is fully granted in all 33 countries where it was nationalized, the protection of the specific liposomal formulation of THE001 and the dosing regimen of THE001 in combination with hyperthermia. The patent landscape is regularly evaluated by external patent attorneys, with no freedom-to-operate concerns for Thermosome identified to date.

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About Thermosome

Thermosome is a clinical-stage drug development company focused on targeted tumor therapy combined with immune stimulation for improved cancer therapy. At its core is a novel, proprietary tumor targeting approach that allows for significantly increased local drug concentration and improved tumor penetration to achieve improved clinical treatment efficacy.

The first clinical indication for its lead drug candidate THE001 is soft tissue sarcoma, where the Company aims to improve the current standard of care (free doxorubicin). Thermosome’s approach enables targeted tumor treatment independent of specific molecular targets and covers patient populations across all chemo-sensitive tumor subtypes. More information: www.thermosome.com

About THE001

Thermosome’s clinical-stage lead drug candidate THE001 is a thermosensitive liposomal formulation of the chemotherapeutic drug doxorubicin (DPPG₂-TSL-DOX). It has a different mode of action than conventional liposomes. Thermosome’s technology enables intravascular drug release initiated by a mild heat trigger using clinically established hyperthermia devices. This results in up to 15-fold higher local drug concentrations in the tumor and aims to improve clinical treatment efficacy by creating a local boost at the desired site of action. These high local concentrations, which also reach less well perfused areas, are intended to overcome drug resistance. This effect cannot be achieved by administration of conventional doxorubicin due to systemic toxicity. Thermosome intends to further enhance treatment efficacy through an additive immune response induced by regional hyperthermia. THE001 has potential for further development in other anthracycline-sensitive solid tumors, such as breast, bladder, and ovarian cancer. THE001 has been granted Orphan Drug Designation for STS in Europe and the United States.

About the Phase I Study

The Phase I, open-label, interventional dose-escalation trial enrolling patients with locally advanced unresectable or metastatic STS (NCT05858710) is being conducted at two German clinical sites testing THE001 at various different dose levels in initially up to 6 cycles every 3 weeks with option to extend to up to 12 cycles in participants with at least disease control. Both completed dose level (20 mg/m² and 40 mg/m²) were well tolerated and declared safe by the independent data safety monitoring board (DSMB). Primary endpoints of the study are the safety and tolerability of THE001 and the determination of the maximum tolerated dose. A secondary objective is the evaluation of anti-tumor activity. Initial clinical data were presented at the CTOS 2024 Annual Meeting (link).

About Soft Tissue Sarcomas (STS)

STS is an atypical tumor with a patient population that includes many young patients. Locally advanced STS (LA-STS) are large invasive tumors that are difficult or impossible to resect. Neoadjuvant therapy is used to shrink these tumors preoperatively to allow tumor surgery with curative intent. Free doxorubicin in combination with ifosfamide or dacarbazine has been the gold standard for neoadjuvant therapy of all chemo-sensitive LA-STS for several decades. Guidelines also recommend combining DOX-based therapy with regional hyperthermia. However, with response rates of less than 30%, there is a significant unmet need for improved treatment options.

Soft tissue sarcomas occur in more than 50 different subtypes that do not share a common driver mutation, making biologic targeting more difficult than physically controlled targeting with the most active agent.

Company Contact
Thermosome GmbH
Am Klopferspitz 19
82152 Planegg/Martinsried (Germany)
Phone: +49 89 7167760 31
media@thermosome.com

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Thermosome Receives U.S. Orphan Drug Designation for Lead Compound THE001, Expands Patent Estate

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