Modis Therapeutics

Modis Therapeutics, established in 2016 by Peter Barber and Daniel Di Pietro, operated as a biopharmaceutical firm centered on creating treatments for rare genetic disorders with significant unmet medical needs. The company's primary focus was on Thymidine Kinase 2 deficiency (TK2d), a severe and often fatal inherited mitochondrial DNA depletion disorder that predominantly affects children. TK2d stems from mutations in the TK2 gene, leading to mitochondrial dysfunction and insufficient energy production in cells, which causes severe muscle weakness.

The company's core asset was MT1621, an investigational oral substrate enhancement therapy. This therapy, consisting of a combination of deoxynucleosides, aimed to address the fundamental pathophysiology of TK2d by providing the building blocks for mitochondrial DNA, thereby improving cellular function. The development of MT1621 was rooted in a collaboration with academic experts in mitochondrial biology, including research from Columbia University Irving Medical Center and the Vall d'Hebron Research Institute in Barcelona. This research demonstrated that the therapy could prolong survival in animal models and showed encouraging results in initial human clinical studies. As a result of its potential, MT1621 received Breakthrough Therapy designation from the U.S. Food and Drug Administration (FDA) and PRIME designation from the European Medicines Agency (EMA).

In October 2018, Modis Therapeutics secured $30 million in a Series A financing round co-led by OrbiMed and F-Prime Capital Partners, with participation from founding investor Aceras Life Sciences and Osage University Partners. This funding was instrumental in advancing the clinical development of MT1621. On August 26, 2019, Zogenix, a global pharmaceutical company also focused on rare diseases, announced its acquisition of Modis Therapeutics. The deal involved an upfront payment of $250 million, comprised of $175 million in cash and $75 million in Zogenix stock, with potential future milestone payments of up to $150 million tied to regulatory approvals in the U.S. and Europe, plus a 5% royalty on future net sales of MT1621. The acquisition integrated Modis and its promising lead candidate, MT1621, into Zogenix's pipeline, aiming to expedite the therapy's development and global availability for patients with TK2d. Keywords: Modis Therapeutics, rare genetic diseases, biopharmaceutical, Thymidine Kinase 2 deficiency, TK2d, mitochondrial disease, MT1621, substrate enhancement therapy, Zogenix acquisition, orphan drugs, deoxynucleoside therapy, F-Prime Capital, OrbiMed, mitochondrial DNA depletion, neuromuscular disorder, clinical development, rare pediatric disease, Columbia University, Vall d'Hebron Research Institute, Joshua Grass