Minoryx Therapeutics, a drug development company specialized in the discovery of new drugs for orphan diseases, announced the appointment of Dr. Khalid Islam as chairman of the board of directors. Dr. Islam has 27 years experience in the pharmaceutical and biotechnology industries.

Dr. Islam previously served as chairman and CEO of Gentium S.p.A. (a Nasdaq-listed company; 2009-2014) where he led the transition of the loss-making company to cash-flow positive and profitable. Under his leadership Defitelio (defibrotide) was granted marketing authorization in the EU and the company’s value increased from $25 million to $1 billion in a cash merger with Jazz Pharmaceuticals, plc.

“2016 is an important year for Minoryx as we progress our lead candidate MIN-102 into clinical trials and further develop the SEE-Tx platform,” said Dr. Islam, “I am excited to work with the Minoryx team and board to address the high unmet medical need in orphan diseases for patients who currently have no or few therapeutic options.”

The appointment follows the €19.4 million series A fundraising that closed in October 2015. Dr. Khalid Islam succeeds Dr. Nigel Ten Fleming, who has been instrumental in the company’s success so far.

“We are very pleased to welcome Dr. Islam; his extensive experience in managing innovative drug discovery and development companies will be key to the future success of Minoryx,” said Dr. Marc Martinell, CEO and co-founder of Minoryx. “On behalf of the board and company, I would also like to thank Dr. Nigel Ten Fleming for his outstanding contribution to Minoryx Therapeutics’ development in recent years.”

Following the new appointment, Minoryx Therapeutics will move its lead candidate, MIN-102, into clinical stage. MIN-102 targets the most prevalent peroxisomal disorder, X-linked adrenoleukodystrophy (X-ALD). MIN-102 is a differentiated PPAR gamma agonist with a superior profile for central nervous system related diseases and excellent in-vivo efficacy.

Minoryx also plans to further develop SEE-Tx, a dedicated platform that relies on proprietary technology and know-how for the identification of a new class of pharmacological chaperones.

Dr. Islam is currently the chairman of Fennec, North Carolina and a member of the board of directors at Karolinska Development (KDEV.ST; Stockholm), Molmed (MLM.MI; Milan), and OxThera (Stockholm). He has previously been a member of the board of directors at Arpida AG (Basel and DC), Rheoscience AS (Copenhagen), PCovery AS (Copenhagen), Adenium AS (Copenhagen) and C10 Pharma AS (Oslo).

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 a bone marrow transplant. Such an approach does not prevent the development of the AMN form, for which there are no therapies available.

MIN-102, Minoryx’ 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’ 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.

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 company is moving forward with a pipeline of several Inborn Errors of Metabolism (IEM) including Lysosomal Storage Diseases (LSD).