Developing metabolite

replacement therapies

for effective treatments in inborn errors of metabolism disorders

Latest News

Press Release

TM3 Therapeutics Appoints Peter Van Vlasselaer as Chairman of the Board

Current ARMO BioSciences CEO Brings Profound Drug Development, Operations & Strategy Expertise to TM3  …
Press Release

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

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

About Us

TM3 Therapeutics’ purpose is to develop effective treatments for Inborn Errors in Metabolism (IEM), a group of rare but severe metabolic and often neurological disorders that primarily affect children.

Our development programs are specifically targeted to help compensate impaired cellular metabolic pathways, whose malfunction leads to impaired quality of life, progressive loss of function and significantly reduced life expectancy.

We were established in 2017 based on an extremely promising scientific discovery that could effectively treat patients with Pantothenate Kinase-Associated Neurodegeneration (PKAN). Our genesis follows the completion of a European Union-funded research consortium called Treat Iron-Related Childhood-Onset Neurodegeneration (TIRCON). It was set-up to improve the diagnosis and treatment of Neurodegeneration with Brain Iron Accumulation (NBIA) disorders, a subcategory of IEM’s.

We are backed by a consortium of top-tier international investors including Sofinnova Partners and INKEF Capital.

Our Focus

TM3’s focus is to develop new therapeutics to treat rare diseases with significant unmet medical need.

About NBIA

Neurodegeneration with Brain Iron Accumulation (NBIA) is a group of rare, inherited movement disorders, which result in high levels of iron in the brain. Symptoms usually begin in childhood and relentlessly progresses to cause severe disability, eventually leading to premature death.  As a first indication from the NBIA group of diseases, TM3 is pursuing a drug development program against Pantothenate Kinase-Associated Neurodegeneration (PKAN).

About PKAN

Pantothenate Kinase-Associated Neurodegeneration (PKAN) is an extremely rare hereditary disorder caused by mutations in the PANK2 gene and accounts for 50-70% of cases of NBIA. The protein, PANK2, is an enzyme that catalyzes a key step in the synthesis of coenzyme A (CoA) from vitamin B5 (pantothenate). Patients whose cells lack a functional PANK2 protein can suffer from either classic PKAN, manifested by early onset, rapid loss of function and shortened life expectancy, or atypical PKAN, characterized by Parkinson disease-like symptoms in later childhood and adult life.

Our lead compound, TM-1803 has shown very promising efficacy in patient-derived cell lines and mouse models, and has been granted an Orphan Drug Designation by the European Medicines Agency. It is currently in advanced pre-clinical stage of development.

Our Team

TM3 Therapeutics is supported by a team of experts committed to bringing innovative therapies to patients and working together with leading academic researchers, patients’ families and the medical professionals serving them. The company is backed by leading European VC firms, Sofinnova Partners and INKEF Capital.

Enej Kuscer, PhD

CEO / Managing director

Angelina Sekirnik, PhD

Senior Scientist

Jessica Sipkens, PhD

Head of DMPK

Charles Simons, MBA, BSc

Head of CMC

Peter Van Vlasselaer, PhD

Chairman of BOD

Henrijette Richter, PhD

Member of BOD

Lucas De Breed, MBA, PhD

Member of BOD


September 12, 2017

Acetyl-4'-phosphopantetheine is stable in serum and prevents phenotypes induced by pantothenate kinase deficiency

Scientific Reports 7(1):11260

August 31, 2015

Extracellular 4′-phosphopantetheine is a source for intracellular coenzyme A synthesis

Nature Chemical Biology 11(10):784-92

Contact Us

TM3 Therapeutics

Galileiweg 8
2333 BD Leiden
The Netherlands

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