The orphan drug designation follows the generation of a preclinical rodent data package in which a survival advantage was shown for this nanoform-enabled medicine candidate.
The hydrogel nanoformulation developed by Nanoform enabled a 200-fold increase in drug load compared to bulk and a 5-fold increase in drug load compared to nanomilling, which did not achieve the results needed. Hence Nanoform’s proprietary technology and nanoformulation expertise will enable TargTex’s drug candidate TTX101 to move towards clinic.
TargTex is currently raising funds to take this innovative treatment to clinic and is planning a phase 1/2a clinical trial in recurrent glioblastoma (GBM) patients across the US and EU, in which nanoformed TTX101 is applied as adjunct to surgery after tumour excision. The Orphan Drug Designation is an important milestone for TargTex. Orphan Drug Designation is granted by the FDA to a drug or biological product that is developed to prevent, diagnose, or treat a rare disease or condition that affects less than 200,000 people in the United States. The designation qualifies sponsors for among other incentives 7 years of US market exclusivity after approval.
João Seixas, CEO of TargTex, said “We are profoundly grateful to Nanoform, its technology and partnership. The FDA Orphan Drug Designation highlights the significant need of an efficacious therapy for this patient population as well as the potential of nanoforned TTX101 to benefit such patients. Our pre-clinical data showed that nanoformed TTX101 can potentially be used as monotherapy as well as a synergistic agent in combination with other drugs such as temozolomide, to treat GBM and other aggressive central nervous system tumours. We believe that delivering our drug locally at the time of surgery will bring a major benefit to patients. The drug shows a highly promising profile, with an ideal diffusion profile in the brain, without significant local or systemic toxicity and enabling tumour eradication in preclinical models in rodents.”
Christian Jones, CCO of Nanoform, commented “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.”
TargTex (www.targtex.com) is a European biotech company developing a new nanoformed hydrogel-based localized therapy for the treatment of brain tumours, with the lead candidate TTX101 for glioblastoma (GBM). GBM is the most aggressive and most common brain tumour in adults, with an average life expectancy of 16 months. About 3 in 100,000 people are diagnosed with this disease each year and there is as of yet no efficacious treatment for this deadly disease.
