Minoryx and Neuraxpharm Partner to Provide a New Therapy for Rare CNS Disease Patients in Europe

Rare Daily Staff

Barcelona-based Minoryx Therapeutics and European specialty pharmaceutical Neuraxpharm Group have entered into a license agreement for the European rights to leriglitazone, currently under European Medicines Agency review for the orphan indication X-linked Adrenoleukodystrophy.

Under the agreement, Minoryx granted Neuraxpharm exclusive European rights to its lead candidate leriglitazone, a novel, brain penetrant and selective PPAR gamma agonist. Neuraxpharm has exclusive rights to commercialize leriglitazone in Europe and will join with Minoryx to continue the further development of leriglitazone. Minoryx retains full rights to leriglitazone in the United States and the rest of world, excluding China where it is already partnered.

“Minoryx selected Neuraxpharm specifically for its expertise in the European central nervous system marketplace and its substantial experience and capabilities in successfully commercializing CNS drugs in Europe,” said Marc Martinell, CEO at Minoryx. “Minoryx will continue the development and regulatory preparations for the United States and is currently in discussions with the U.S. Food and Drug Administration to define the next steps for the U.S. approval path.”

As part of the agreement, Neuraxpharm has agreed to pay Minoryx a significant double-digit upfront payment in addition to milestone payments and development funding that total up to $262 million (€258 million) in the aggregate. Minoryx will also receive tiered double-digit royalties.

Minoryx and Neuraxpharm will collaborate on concluding the ongoing European regulatory process to obtain approval for leriglitazone for the treatment of adult male patients with X-ALD. If approved, leriglitazone will be the first approved treatment for this population. Minoryx and Neuraxpharm are further committed to jointly continue the development of leriglitazone for additional X-ALD patient populations and other orphan indications.

X-ALD (X-linked adrenoleukodystrophy) is a rare neurodegenerative disease with a global incidence of approximately 6 to 8 in 100,000 live births mostly in males. However, its chronic form also affects women even though they develop symptoms later in life. X-ALD patients reaching adulthood develop adrenomyeloneuropathy characterized by progressive weakness in the hips and legs, as well as progressive deterioration of balance and sensory function, and development of incontinence. This form progresses chronically with onset of symptoms in adulthood and has a poor prognosis.

Recent literature indicates that up to 60 percent of adult male X-ALD patients will also develop cALD (cerebral ALD), which typically affects boys with an age of onset between 4-8 years. cALD is characterized by aggressive brain inflammation, and if untreated, patients progress quickly with severe neurological impairment, often leading to permanent disability and death within 2-4 years.

There is currently no pharmacological treatment available for adults with X-ALD. In childhood, hematopoietic stem cell transplantation (HSCT) or ex vivo gene therapy can arrest the disease, however, it is an aggressive procedure and only available for a portion of patients. In adults, experience in HSCT is very limited and the intervention is often not recommended.

Leriglitazone is a novel, brain penetrant and selective PPAR gamma agonist under development for treatment of X-ALD and other orphan CNS diseases. Leriglitazone has been granted orphan drug status from the EMA and the FDA as well as Fast Track and Rare Pediatric Disease designations from the FDA for the treatment of X-ALD.

Photo: Marc Martinell, CEO at Minoryx