TriAct Therapeutics’ Novel TrxR1 Inhibitor Receives FDA Rare Pediatric Disease Designation for Treatment of Pediatric Diffuse High Grade Gliomas
San Francisco, CA, September 28, 2022 --(PR.com)-- TriAct Therapeutics, a private, late clinical-stage oncology therapeutics company, announced today that the U.S. Food and Drug Administration (FDA) has granted rare pediatric disease designation (RPDD) to its novel, proprietary TrxR1 inhibitor (iniparib), for the treatment of patients with pediatric diffuse high grade gliomas (pdHGG).
“Receiving this rare pediatric disease designation, on top of the FDA’s earlier orphan drug designation (ODD), is another important milestone in our effort to bring this promising new therapy to both pediatric and adult patients suffering from malignant gliomas,” said Tom White, co-founder and Chief Executive Officer of TriAct. “Our initial pediatric focus is on DIPG/DMG where only 10% of patients survive for 2 years after diagnosis and there is an urgent need to develop promising new therapies such as Iniparib.”
The RPDD and ODD designations confer benefits to therapy developers that are designed to speed the review and potential approval of treatments for rare diseases and orphan drugs.
Rare pediatric disease designation is granted to promising investigational therapies for rare disorders that mainly affect children. Recipients of this designation qualify for a priority review voucher if the therapy is ultimately approved, in addition to meeting other conditions. The voucher may be used to request priority review of a future application to the FDA, or it may be transferred or sold.
Orphan drug designation is given to investigational therapies designed to treat diseases that affect fewer than 200,000 people in the U.S. Such status provides benefits that notably include a seven-year period of market exclusivity in the U.S. upon treatment approval. An additional half year US market exclusivity is added if the therapy’s initial approval is for a rare pediatric disease.
About iniparib
Iniparib, their lead program, is a small molecule, brain penetrant inhibitor of TrxR1 being developed to treat an extensive list of TrxR1 over expressing tumors, including pediatric high grade gliomas, newly diagnosed glioblastoma and triple negative breast cancer with CNS metastases. Over expression of TrxR1 has been shown in several published studies to be prognostic for poor or worse survival in a wide range of tumors. Preclinical, mechanistic studies have shown iniparib to be an effective inhibitor of TrxR1 leading to tumor cell death by ROS accumulation and the resultant cellular oxidative stress as well as ASK1 pathway activation. Seven completed clinical trials in TrxR1 overexpressing tumor types met their primary efficacy endpoints, including iniparib’s Phase 2 Study in newly diagnosed glioblastoma. Approximately 2,900 patients have been dosed to date, with iniparib being well tolerated. Based in part on these promising results, TriAct is designing pivotal trials in TrxR1 overexpressing cancers intended to support submission of one or more NDAs in the U.S. and MAAs in Europe.
About Pediatric Diffuse High Grade Glioma
Pediatric-type diffuse high grade gliomas (pdHGG) are a group of rare, rapidly progressing tumors, affecting approximately 2,500 patients annually in the United States (US). Median age of the DIPG/DMG sub-group of pdHGG patients is 6-9 years and 3-10 years respectively. There are no FDA approved therapies currently and surgical resection is generally not an option due to tumor location. Standard of care is typically radiation alone. TrxR1 expression is 5-10x greater in pdHGG tissue and CNF vs health tissue and CNF. TrxR1 overexpression has been shown in published studies to desensitize tumor cells to radiation therapy. Currently, survival for DIPG/DMG patients is poor, with 10% or less surviving 2 years from diagnosis.
About Glioblastoma
Glioblastoma (GBM) tumors are the most common and aggressive form of brain cancer, affecting approximately 15,000 patients annually in the United States (US). TrxR1 expression is 5x or greater in GBM tissue and CNF vs health tissue and CNF. Annual incidence of GBM in the US is approximately 3 per 100,000 population. The standard of care since 2005 in the US comprises surgical resection of the tumor followed by adjuvant radiotherapy combined with temozolomide (TMZ), and subsequent maintenance TMZ. Currently, survival for GBM patients is poor, with approximately 75% of patients dying within 2 years of diagnosis and 95% dying within 5 years
About TriAct Therapeutics
TriAct Therapeutics is a private, late clinical-stage cancer therapeutics company developing novel, small molecule drugs for patients who have no effective therapies available to extend and enhance their lives. TriAct’s in-licensed portfolio includes two clinical stage small molecules with distinct mechanisms of action.
Contact:
Tom White
President & CEO
415-602-7497
tom.white@triactinc.com
“Receiving this rare pediatric disease designation, on top of the FDA’s earlier orphan drug designation (ODD), is another important milestone in our effort to bring this promising new therapy to both pediatric and adult patients suffering from malignant gliomas,” said Tom White, co-founder and Chief Executive Officer of TriAct. “Our initial pediatric focus is on DIPG/DMG where only 10% of patients survive for 2 years after diagnosis and there is an urgent need to develop promising new therapies such as Iniparib.”
The RPDD and ODD designations confer benefits to therapy developers that are designed to speed the review and potential approval of treatments for rare diseases and orphan drugs.
Rare pediatric disease designation is granted to promising investigational therapies for rare disorders that mainly affect children. Recipients of this designation qualify for a priority review voucher if the therapy is ultimately approved, in addition to meeting other conditions. The voucher may be used to request priority review of a future application to the FDA, or it may be transferred or sold.
Orphan drug designation is given to investigational therapies designed to treat diseases that affect fewer than 200,000 people in the U.S. Such status provides benefits that notably include a seven-year period of market exclusivity in the U.S. upon treatment approval. An additional half year US market exclusivity is added if the therapy’s initial approval is for a rare pediatric disease.
About iniparib
Iniparib, their lead program, is a small molecule, brain penetrant inhibitor of TrxR1 being developed to treat an extensive list of TrxR1 over expressing tumors, including pediatric high grade gliomas, newly diagnosed glioblastoma and triple negative breast cancer with CNS metastases. Over expression of TrxR1 has been shown in several published studies to be prognostic for poor or worse survival in a wide range of tumors. Preclinical, mechanistic studies have shown iniparib to be an effective inhibitor of TrxR1 leading to tumor cell death by ROS accumulation and the resultant cellular oxidative stress as well as ASK1 pathway activation. Seven completed clinical trials in TrxR1 overexpressing tumor types met their primary efficacy endpoints, including iniparib’s Phase 2 Study in newly diagnosed glioblastoma. Approximately 2,900 patients have been dosed to date, with iniparib being well tolerated. Based in part on these promising results, TriAct is designing pivotal trials in TrxR1 overexpressing cancers intended to support submission of one or more NDAs in the U.S. and MAAs in Europe.
About Pediatric Diffuse High Grade Glioma
Pediatric-type diffuse high grade gliomas (pdHGG) are a group of rare, rapidly progressing tumors, affecting approximately 2,500 patients annually in the United States (US). Median age of the DIPG/DMG sub-group of pdHGG patients is 6-9 years and 3-10 years respectively. There are no FDA approved therapies currently and surgical resection is generally not an option due to tumor location. Standard of care is typically radiation alone. TrxR1 expression is 5-10x greater in pdHGG tissue and CNF vs health tissue and CNF. TrxR1 overexpression has been shown in published studies to desensitize tumor cells to radiation therapy. Currently, survival for DIPG/DMG patients is poor, with 10% or less surviving 2 years from diagnosis.
About Glioblastoma
Glioblastoma (GBM) tumors are the most common and aggressive form of brain cancer, affecting approximately 15,000 patients annually in the United States (US). TrxR1 expression is 5x or greater in GBM tissue and CNF vs health tissue and CNF. Annual incidence of GBM in the US is approximately 3 per 100,000 population. The standard of care since 2005 in the US comprises surgical resection of the tumor followed by adjuvant radiotherapy combined with temozolomide (TMZ), and subsequent maintenance TMZ. Currently, survival for GBM patients is poor, with approximately 75% of patients dying within 2 years of diagnosis and 95% dying within 5 years
About TriAct Therapeutics
TriAct Therapeutics is a private, late clinical-stage cancer therapeutics company developing novel, small molecule drugs for patients who have no effective therapies available to extend and enhance their lives. TriAct’s in-licensed portfolio includes two clinical stage small molecules with distinct mechanisms of action.
Contact:
Tom White
President & CEO
415-602-7497
tom.white@triactinc.com
Contact
TriAct Therapeutics
Tom White
415-602-7497
Tom White
415-602-7497
Contact
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