Next Generation Gene Therapeutics (NGGT) Announces Positive New Data on NGGT002 for the Treatment of Phenylketonuria (PKU)

NGGT has initiated Phase I/II clinical trials in the U.S. and China to evaluate the safety and efficacy of NGGT002 for the treatment of Phenylketonuria

WALNUT CREEK, Calif., Nov. 11, 2024 /PRNewswire/ -- NGGT Inc., ("Next Generation Gene Therapeutics" or "NGGT"), a clinical-stage biotechnology company pioneering the next wave of gene therapy innovation for genetic rare diseases, today announced positive new data on NGGT002, a clinical-stage gene therapy candidate for the treatment of Phenylketonuria (PKU).

"We are pleased with the results from our investigator-initiated trial, as they show great promise for NGGT002 to serve as a safe and effective treatment for PKU," said Yiting Liu, Ph.D., VP of Translational Research at NGGT. "With our initial 40-week span of positive results, we are optimistic that our Phase I/II clinical trials will further elucidate NGGT002's potential to treat this rare and debilitating disease in a lasting manner."

"Given the difficulties associated with dietary restrictions for PKU and the potential benefits to patients from effective and durable reductions of phenylalanine levels, we view these results with great excitement," said Lixin Jiang, M.D., Ph.D., CEO and Co-Founder of NGGT. "With the impressive safety and efficacy shown by NGGT002 in the investigator-initiated trial, we are enthusiastic about the upcoming results from our ongoing clinical trials in the United States and China."

The data, presented at the National PKU Alliance biennial conference in July, highlight the results from an investigator-initiated trial (IIT) conducted in China and demonstrate the safety and efficacy of NGGT002 for the treatment of PKU. PKU is a rare genetic disorder caused by a mutation in the phenylalanine hydroxylase (PAH) gene that causes an elevated blood level of phenylalanine and results in progressive intellectual and behavioral impairments, skin rashes, movement disorders and seizures. Five out of six patients receiving a high dose of NGGT002 in the study demonstrated a level of plasma phenylalanine within the normal range after three weeks of drug administration. The first patient dosed with NGGT002 in the IIT trial showed a persistent plasma phenylalanine level within the normal range 40 weeks after the drug was dosed. Phase I/II clinical trials in the U.S. (NCT06332807) and China (NCT06061614) have been initiated to evaluate the safety and efficacy of NGGT002 in adult patients with PKU. First patients in both trials will be dosed based on the effective dose which has been proved in the IIT study.

About NGGT002 Gene Therapy Clinical Study in Adult Classic PKU
The U.S.-based clinical trial (NCT06332807) is a Phase I/II, open-label, multiple-center, dose escalation and cohort expansion study to evaluate the safety and efficacy of NGGT002, an rAAV-based vector carrying a functional copy of the human PAH gene, in adult subjects with classic PKU. Participants in two dose cohorts will receive a single administration of NGGT002 and will be followed for efficacy for 1 year and safety and 5 years. Further information regarding this Phase I/II study, including eligibility and exclusion criteria, can be found on clinicaltrials.gov or by calling 801-261-2000.

About NGGT Inc.
NGGT is a clinical-stage biotechnology company developing novel gene therapies for the treatment of retinal, metabolic and neurodegenerative diseases. NGGT is currently advancing an extensive therapeutic development pipeline with multiple clinical and pre-clinical programs built around several proprietary AAV (Adeno-Associated Virus) products: NGGT001, a Phase I/II-ready gene therapy for Bietti's Crystalline Dystrophy; NGGT002, a Phase I/II-ready gene therapy for PKU; NGGT006, a gene therapy for familial hypercholesterolemia, currently undergoing Investigator-Initiated Trials; and NGGT007, a gene therapy for Wet-AMD. NGGT is headquartered in Walnut Creek, California. For more information, please visit www.nggtbio.com, contact [email protected] and follow us on X and LinkedIn. 

Corporate Contact:
NGGT, Inc.
2910 Camino Diablo, Suite 120
Walnut Creek, CA 94597
[email protected]

Media Contact:
Russo Partners
Ignacio Guerrero-Ros, Ph.D.
+1 (646) 942-5604
[email protected]

SOURCE NGGT Inc.