THE001, Novel Sarcoma Treatment, Earns FDA Orphan Drug Designation
THE001, a thermosensitive doxorubicin liposome for soft tissue sarcomas, received FDA orphan drug designation, offering development benefits.
- The FDA has granted orphan drug designation (ODD) to THE001 (DPPG2-TSL-DOX) for the treatment of soft tissue sarcomas.
US FDA
- THE001 was previously granted ODD by the European Medicines Agency (EMA).
- THE001 is a thermosensitive liposomal formulation of the chemotherapy agent doxorubicin.
THE001, a thermosensitive liposomal formulation of doxorubicin, has been granted FDA ODD for the treatment of soft tissue sarcomas.1
The ODD program supports the development of drugs for rare diseases. With this designation, Thermosome, the sponsor, is eligible for tax credits for clinical trials, exemption from user fees, and a potential 7 years of market exclusivity following approval.
“We see the US orphan drug designation as a strong regulatory validation of the potential of our innovative approach in soft tissue sarcoma,” said Pascal Schweizer, MD, co-founder, chief executive officer, and chief financial officer of Thermosome, in a press release. “This recognition, based on preclinical and early clinical data from our phase 1 study [NCT05858710], marks an important milestone and is a further step into the US 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.”
The EMA also previously granted THE001 ODD for this indication, complementing this new designation.
About THE001
Sarcoma cells: Developing in connective tissues like bones and muscles, these spindle-shaped cells are known for their aggressiveness and rapid spread: © BEST - stock.adobe.com
THE001 utilizes a unique formulation of doxorubicin combined with deep regional radiofrequency hyperthermia in to target doxorubicin-sensitive tumors like soft tissue sarcomas.2 This delivers an up to 15-fold higher local drug concentration in the tumor with the intention to overcome drug resistance.1
The agent is being investigated in a phase 1 clinical trial. Patients at 2 sites in Germany with locally advanced unresectable or metastatic soft tissue sarcomas were enrolled and treated with 1 of 3 dose levels of THE001.3Currently, 2 dose levels have been completed—20 mg/m2 and 40 mg/m2—and both were well tolerated and deemed safe by an independent data safety monitoring board.1
The study’s primary end point is the maximum tolerated dose, and secondary end points include adverse events (AEs), serious AEs, laboratory abnormalities, electrocardiogram abnormalities, echocardiogram abnormalities, renal toxicity, and pharmacokinetics.3
To be eligible for enrollment, patients must have progressive disease not suitable for surgery, received pretreatment with an anthracycline, adequate laboratory values, and an ECOG performance status of 0 to 2. Those with a history of another primary malignancy, uncontrolled intercurrent illness, or serious cardiac condition were not eligible for trial participation.
Early efficacy data was presented at the 2024 Connective Tissue Oncology Society Annual Meeting and showed encouraging signs of clinical activity in 2 of 4 efficacy-evaluable patients treated at dose levels 1 and 2. One patient was still on treatment after 12 cycles.4
THE001 is also being evaluated in other disease settings, including bladder cancer.2
