‍Minoryx Therapeutics, a drug development company specialized in the discovery of new drugs for orphan diseases, today announces it has completed a Series A funding round of €19.4M ($21.7M). Ysios Capital, a Spanish investment fund, led the round. The fundraising was also supported by a substantial syndicate of new investors including Kurma Partners, Roche Venture Fund, Idinvest Partners and Chiesi Ventures. Existing investors Caixa Capital Risc, the venture capital division of ‘la Caixa’ and HealthEquity also participated in the round.

With this fundraising, the company also welcomes three new board members: Laia Crespo from Ysios Capital, Thierry Laugel from Kurma Partners and Monique Schiersing from Roche Venture Fund.

“We are delighted to have closed such an important fundraising round. The investment syndicate includes complementary parties such as international investors specialized in orphan drugs and corporate investors from big pharma as well as specialty pharma,” said Marc Martinell, CEO and co-founder of Minoryx Therapeutics. “As a new player in the field of rare diseases, we would like to thank the investors that have joined us in this important project and the former investors for their renewed trust.”

“Minoryx Therapeutics is a very promising company in the field of rare diseases. Its unique approach convinced us of the value of its activities,” said Laia Crespo, PhD, investment director at Ysios Capital. “We are very confident in its X-ALD candidate and we look forward to the next stages of development for this product.”

The funds raised will be used to complete the team, especially in R&D, to move lead project MIN-102 to clinical validation and to further develop a pipeline of non-competitive pharmacological chaperones identified through the company’s innovative proprietary platform, SEE-Tx.

About X-ALD

X-linked adrenoleukodystrophy (X-ALD) is the most prevalent peroxisomal disorder. It is caused by mutations on the ABCD1 gene, which codes for a membrane transporter protein. The disease is characterized by the accumulation of very long chain fatty acids (VLCFA) leading to a neurodegenerative disorder that is chronically debilitating and life-threatening, where the most affected tissues are the myelin in the central nervous system (CNS) and the adrenal cortex. The CNS related effects lead to two main phenotypes: adrenomyeloneuropathy, (AMN) characterized by progressive motor dysfunction, and cerebral ALD (cALD), characterized by severe neuroinflammation leading to early death. X-ALD is a rare disease that occurs all over the world. Its estimated incidence is 1:17,000 newborns. Although it primarily affects males, heterozygous women also develop the disease later in life. There is no pharmacological treatment available on the market. The only available alternative for cALD patients is bone marrow transplantation. Such an approach does not prevent the development of the AMN form, for which there are no therapies available.

About MIN-102

MIN-102, Minoryx’s candidate for X-ALD, is in the preclinical stage. It is a differentiated PPAR gamma agonist with a superior profile for CNS related diseases with excellent in-vivo efficacy, a comprehensive development plan and early involvement from key opinion leaders. PPAR gamma agonists have shown strong potential in animal models related to the various phenotypes associated with X-ALD. Minoryx’s candidate is the only product in development for potential use across all the main phenotypes. PPAR gamma agonists also showed efficacy in multiple models of neurodegenerative diseases, meaning that MIN-102 also offers a significant potential for indication expansion.

About SEE-Tx

This dedicated platform relies on proprietary technology and know-how for the identification of a new class of pharmacological chaperones that are non-competitive with the natural substrate and with optimized drug-like properties; offering greater potential than previous generations. The most advanced program related to the platform targets GM1 Gangliosidosis and Morquio B. The company is also moving forward with a pipeline of several other Inborn Errors of Metabolism including Lysosomal storage diseases.

About Minoryx Therapeutics

Minoryx is a drug development company specializing in the discovery of new drugs for orphan diseases. The company targets Inborn Errors of Metabolism; a group of rare diseases of genetic origin with a high unmet medical need. The company’s leading program is a differentiated PPAR gamma agonist (MIN-102) that has multiple CNS indications. Minoryx harnesses its unique mechanism of action for potential use in X-ALD, a genetic disease characterized by progressive neurologic deterioration with no available pharmacological treatment. Minoryx is also working on a new class of compounds: non-competitive pharmacological chaperones, identified through its innovative proprietary platform – SEE-Tx.

The Minoryx team is made up of a group of drug discovery and development experts with several decades of experience in biotech and pharma. The company is backed by a syndicate of experienced investors and has support from a network of other organizations. Minoryx was founded in 2011 and previous to this current round it raised €5M ($5.6M).