FDA Grants Orphan Drug Designation to TTX101 in Malignant Gliomas

• TTX101 is a new hydrogel-based localized therapy being developed for the treatment of brain tumors.

• Preclinical data has shown the potential of TTX101 used as a monotherapy or in combinations for patients with glioblastoma or other aggressive central nervous system tumors.

• With this orphan drug designation (ODD) TTX101 qualifies sponsors for incentives such as tax credits for certain clinical trials, exemption from user fees, and the potential of 7 years of market exclusivity in the United States following approval.

The FDA has granted an ODD to TTX101, a nanoformed drug candidate, for potential use in patients with malignant gliomas.¹

TTX101 utilizes a hydrogel nanoformulation and enables a 200-fold increase in drug load compared with bulk and a 5-fold increase in drug load compared with nanomilling. Over a period of weeks, the product is designed to release the active substance to cover the wound healing period between surgery and radiotherapy.²

3D rendering of brain cancer: ©SciePro - stock.adobe.com

This ODD for TTX101 follows the generation of preclinical data which showed there to be a survival advantage with this nanoform-enabled medicine candidate.¹

“The orphan drug designation highlights the significant need of an efficacious therapy for this patient population as well as the potential of TTX101 to benefit such patients. Our pre-clinical data showed that TTX101 can potentially be used as monotherapy as well as a synergistic agent in combination with other drugs such as TMZ[temozolomide], to treat [glioblastoma] and other aggressive central nervous system tumors,” said João Seixas, PhD, chief executive officer of TargTex, in a press release.²

In preclinical mouse models, TTX101 given as an intracranial injection successfully led to a reduction in tumor volume.3 There were also no significant changes or evidence of toxicity observed in the histopathology of the liver, kidneys, lungs, spleen, or gastrointestinal tract. Additionally, no evidence of necrosis or other lesions were seen in the brain or at the site of administration.

Currently, a phase 1/2a clinical trial of TTX101 is being planned for patients with recurrent glioblastoma. In the study, the agent will be applied as adjunct to surgery after tumor excision.¹

“TargTex is a highly valued innovative client to Nanoform. We are humbled and proud to work on this devastating disease and look forward to a successful fundraising by TargTex to provide hope for patients in need,” added Christian Jones, chief commercial officer at Nanoform, in a press release.²

REFERENCES:

1. Nanoformed TargTex oncology drug candidate TTX101 receives FDA orphan drug designation. News release. Nanoform. October 19, 2023. Accessed October 26, 2023. https://tinyurl.com/52tcym8h

2. TargTex receives orphan drug designation to TTX101. News release. TargTex. September 22, 2023. Accessed October 26, 2023. https://tinyurl.com/36d4u86t

3. Research & technology. TargTex. Accessed October 26, 2023. https://tinyurl.com/2wft7vyy