Modis Therapeutics, Inc., a company developing medicines for patients with rare genetic diseases, announced today a $30M Series A investment led by F-Prime Capital Partners and OrbiMed. Joshua Grass, former F-Prime Entrepreneur-in-Residence and head of Corporate Development at BioMarin Pharmaceutical, has joined as President and Chief Executive Officer. F-Prime's Ben Auspitz and OrbiMed's Rishi Guptahave joined the board, with Mr. Auspitz as Chairman. F-Prime and OrbiMed have each invested in multiple successful rare disease companies, including Ultragenyx, Orchard Therapeutics, Enobia Pharma, True North Therapeutics, Dimension Therapeutics, and others. Founding investor Aceras Life Sciences, LLC and new investor Osage University Partners also participated in the financing.

Modis is advancing its lead experimental therapy, MT1621, as a treatment for thymidine kinase 2 deficiency (TK2d), a rare, genetically defined mitochondrial DNA (mtDNA) depletion disorder. TK2d results from inherited mutations in the nuclear TK2 gene and leads to severe muscle weakness and premature death. TK2d symptom onset varies in age from infants to adults and there are currently no approved treatments. The development of MT1621 is based on research from the lab of Dr. Michio Hirano, the chief of the Division of Neuromuscular Medicine at Columbia University Irving Medical Center, the lab of Dr. Ramon Martí, the head of the Neuromuscular and Mitochondrial Pathology Research Group at Vall d'Hebron Research Institute in Barcelona, and other academic collaborators. Modis has exclusively licensed worldwide rights to data and intellectual property related to a broad range of mtDNA depletion disorders from Columbia University Irving Medical Center and Vall d'Hebron Research Institute.

Dr. Hirano's lab was able to first show that a combination of nucleosides, the active ingredients in MT1621, addresses the direct cause of mitochondrial dysfunction and prolongs survival in a genetic animal model of TK2 deficiency. Dr. Hirano, Dr. Martí and other collaborators in Spain translated this work to the treatment of TK2d patients with encouraging results. Modis is planning additional clinical studies and will be meeting with the FDA and EMA to discuss the registration path for MT1621 with the goal of enabling broad global access for patients with TK2d. Modis has orphan drug designation for MT1621 in both the United States and Europe and has rare pediatric disease designation in the US.

Beyond MT1621, Modis is developing other molecules and therapeutic modalities to treat TK2 deficiency and other mitochondrial diseases with defined genetic causes.

"This is a compelling orphan drug development opportunity for a potentially high impact therapeutic for a very serious disease that affects both children and adults. We are fortunate to have encouraging clinical data, committed physician-scientist collaborators, and two recently published natural history studies of TK2d to help interpret the effect of treatment," said Joshua Grass, President and Chief Executive Officer of Modis Therapeutics.

"The increasing use of advanced DNA sequencing technology to diagnose patients with mitochondrial disease at the genetic level creates new opportunities to target therapeutics to specific causes of disease with large treatment benefits."

About Modis Therapeutics
Modis Therapeutics, Inc. is a biopharmaceutical company focused on developing disease-modifying therapies for rare genetic diseases with high unmet medical need. It was formed in 2016 through a collaboration with academic experts in mitochondrial biology. The company's lead program (MT1621) is in clinical development for thymidine kinase 2 deficiency (TK2d), an inherited mitochondrial disease. Modis Therapeutics is headquartered in Oakland, CA, with offices in New York City. For more information please visit www.modistx.com.

About TK2 Deficiency
Thymidine kinase 2 deficiency (TK2d) is a genetic disorder that results in mitochondrial dysfunction, leading to inadequate energy production in cells. TK2d may present at all ages and causes progressive and severe muscle weakness, respiratory insufficiency, and is often fatal. There are currently no approved therapies for this disease. To learn more, please visit www.tk2d.com.

About F-Prime Capital Partners
F-Prime Capital Partners is a global venture capital firm investing in life sciences, healthcare, and technology. Since 1969, F-Prime has worked closely with entrepreneurs and academics to create innovative solutions to some of the world's most significant challenges in healthcare and technology.

About OrbiMed
OrbiMed is a leading investment firm dedicated exclusively to the healthcare sector, with over $14 billion in assets under management. OrbiMed invests globally across the spectrum of healthcare companies, from venture capital start-ups to large multinational companies utilizing a range of private equity funds, public equity funds, royalty/debt funds and other investment vehicles. OrbiMed maintains its headquarters in New York City, with additional offices in San Francisco, Shanghai, Mumbai and Herzliya. OrbiMed seeks to be a capital provider of choice, with the flexibility to provide equity and debt capital solutions that are tailored to the unique needs of our portfolio companies. The firm's global team of over 80 professionals brings the resources and experience required to be an exceptional long-term partner in building world-class healthcare companies.

About Aceras Life Sciences
Aceras Life Sciences is a healthcare focused investment firm that specializes in funding the development of novel medical innovations and creating new biotechnology companies through collaborations with academic researchers and companies. Aceras brings together strong industry expertise and dedicated capital to offer partnership opportunities to translate early stage discoveries into innovative therapeutic products.

About Osage University Partners

Osage University Partners (OUP) is a venture capital firm focused on investing in startups that are commercializing pioneering university technologies. OUP partners with top research universities to invest in their most innovative startups, and OUP shares its investment profit with its partner institutions. The firm invests in software, hardware, and life science companies at all stages of company development. OUP has partnered with over 90 universities, including 39 of the top 50 U.S. institutions by research expenditures, and has invested in over 80 of their spinouts. OUP is part of a family of investment funds within Osage Partners, which is based in Philadelphia, PA and manages in excess of $500 million.