Press Release

TM3 Therapeutics Debuts with Publication of In Vivo Proof of Concept Data for Lead Compound in Treating Pantothenate Kinase-Associated Neurodegeneration

By September 13, 2017 No Comments

TM-1803/P-PantSAc shows potential in reducing severe outcomes of the disease; study published in Scientific Reports


LEIDEN, The Netherlands, September 13, 2017 – TM3 Therapeutics, a biopharmaceutical company focused on developing metabolite replacement therapies for rare, genetic neurological diseases, today announced the publication of novel preclinical data on its lead compound, TM-1803 (referred in the publication as P-PantSAc), in mice and in other models of Pantothenate Kinase-Associated Neurodegeneration (PKAN). The lead candidate was shown to have serum-stability, low toxicity, and efficacy in mitigating the phenotypes associated with the disease. The paper titled, “Acetyl-4’-phosphopantetheine is stable in serum and prevents phenotypes induced by pantothenate kinase deficiency” can be accessed in the online edition of the journal, Scientific Reports published yesterday, September 12, 2017.

“TM3 Therapeutics was established in 2017 to further the research and development started through the European Union-funded TIRCON consortium, and we are excited to move our program forward and toward the clinic. Our compound has already been given Orphan Drug Designation by the European Medicines Agency,” said Enej Kuscer, PhD, Chief Executive Officer at TM3 Therapeutics. “We strongly believe in the potential of our drug candidate as an effective treatment option for patients with PKAN.”

Ody C.M. Sibon, PhD, Professor in the Department of Cell Biology, University Medical Center Groningen, and corresponding author of the paper added: “Our study validates the ability of TM-1803 to serve as a replacement metabolite in the pathway for coenzyme A synthesis and can thus effectively bypass the deficiency caused by impairment of pantothenate kinases.”

Susan J. Hayflick, MD, Chair of the Department of Molecular & Medical Genetics, Oregon Health & Science University, who originally discovered the PANK2 gene and showed that mutations cause PKAN, commented: “This exciting data and TM3 Therapeutics’ commitment to developing this program offer hope for a promising drug candidate to help PKAN patients, who have no effective treatment options.”

PKAN is an extremely rare hereditary disorder caused by mutations in the PANK2 gene and accounts for 50-70% of cases of “Neurodegeneration with Brain Iron Accumulation” (NBIA), a genetically heterogeneous group of disorders. The protein, PANK2, is one of four functional human PANK enzymes that catalyze a key step in the synthesis of coenzyme A (CoA) from vitamin B5 (pantothenate). CoA is an essential cellular metabolite that is vital for hundreds of biological processes including cell growth and cell death. Patients whose cells lack a functional pantothenate kinase 2 protein can suffer from either classic PKAN, manifested by early onset, rapid loss of function and death by early adulthood, or atypical PKAN, characterized by Parkinson disease-like symptoms in later childhood and adult life.

Valeria Tiranti, PhD, Associate Professor, Unit of Molecular Neurogenetics, IRCCS Foundation Neurological Institute “Carlo Besta”, Milano stated: “We are especially encouraged that these results reinforce the potential effectiveness of TM-1803 for PKAN by not only preventing CoA depletion but completely reverting a very severe phenotype induced in a mouse model of CoA deprivation. These results have been made possible also thanks to AISNAF, the Italian advocacy association made up of families and patients, that provided the funds necessary to support the work of my collaborator Ivano Di Meo, first author of the paper.”

TM3 Therapeutics was established to further the research program started through the European FP7 consortium TIRCON, which included a network of leading clinicians, academic researchers and industrial contributors with the purpose of finding new treatments for PKAN. TM3 Therapeutics’ lead compound, TM-1803 is the continuation of the initial research conducted in the scope of TIRCON by ACIES BIO d.o.o., a Slovenian biotechnology firm and the University Medical Center Groningen.

About TM3 Therapeutics B.V.

TM3 Therapeutics is focused on novel treatments for neurodegenerative and metabolic diseases by developing unique metabolite replacement therapies. Our goal is to improve survival outcomes and quality of life of patients with rare, genetic neurological diseases that have limited or no treatment options. Our clinical development program specifically targets the coenzyme A biosynthetic pathway, whose malfunction leads to impaired quality of life, progressive loss of function and significantly reduced life expectancy. Our vision is to work together with key opinion leaders, researchers and clinicians to expedite the development of new therapeutics to treat diseases with significant unmet medical need. Based in Leiden, The Netherlands, the company was founded in 2017.


TM3 Therapeutics B.V.
Enej Kuscer, Ph.D., CEO
Tel: +386 40 414 818


MacDougall Biomedical Communications
Jacob Verghese, Ph.D.
Tel: +49 173 364 1607