Minoryx Therapeutics SL, of Barcelona, completed recruitment early for its FRAMES phase II trial of MIN-102 (leriglitazone), a peroxisome proliferator-activated receptor gamma agonist, to treat Friedreich's ataxia.

"The advantage of recruiting fast is that it saves time as it allows reaching faster the results of the study," Marc Martinell, Minoryx's CEO, told BioWorld. "Obviously for a small biotech that has limited funding, even if it has a very good VC syndicate behind, saving time is very important and also has a positive impact on the budget required to reach results."

The recruitment process took a scant four and a half months to complete, well ahead of schedule. The first patient was dosed with MIN-102 at the end of April in a hospital in Madrid.

"In rare diseases, recruitment is always a challenge, so speedy recruitments are always remarkable," Martinell added.

The multicenter, randomized, double-blind, placebo-controlled FRAMES trial takes place with 39 participants ages 12 to 60 in Belgium, France, Germany and Spain. Participants will orally take 15 mg/ml of leriglitazone or a placebo once daily for a year to study the effect of MIN-102 on biochemical, imaging, neurophysical and clinical markers on those patients with Friedreich's ataxia, a rare genetic disease whose symptoms include impaired walking ability and speech, plus a compromised ability to speak. The culprit is FXN, a protein necessary for healthy mitochondria function. There is no cure for the orphan disease, which affects about one in every 40,000 people, and has a general onset between ages 5 and 18. The disease is fatal, mainly due to cardiac failure. It is the most common inherited ataxia across Europe, the Middle East, the Indian subcontinent and north Africa.

The primary outcome measure is the change from baseline in the spinal cord area cervical segment C2-C3, measured through imaging of the spinal cord, within a 48-week time frame. The secondary objectives include safety and tolerability, any effect on additional clinical measures, such as patient-reported outcomes, functional disability scores and exploratory biomarkers. The study continues for a year with a report on the data expected by the end of next year.

MIN-102 is designed to penetrate the brain to meet the target receptor inside the central nervous system. In animal models, it modulates pathways leading to mitochondrial dysfunction, oxidative stress, neuroinflammation, demyelination and axonal degeneration. It could be used to treat other CNS conditions, including X-linked adrenoleukodystrophy.

The first U.S. patient in Minoryx's ongoing phase II/III trial in adrenomyeloneuropathy (AMN) with MIN-102 was dosed last September at Massachusetts General Hospital. The trial enrolled adult male patients affected by AMN, the most frequent phenotype of X-linked adrenoleukodystrophy (X-ALD). The primary outcome is to evaluate the efficacy of MIN-102 on the progression of AMN, as determined by a motor function test. The trial aims to enroll more than 100 patients, and the results are expected at the end of 2020.

Other clinical trials addressing Friedreich's ataxia have used interventions including interferon gamma-1b, methylprednisolone, rosuvastatin, nicotinamide, resveratrol and omaveloxolone.

There are others in this race. Earlier this year, Stridebio Inc. landed a collaboration and license agreement with Takeda Pharmaceutical Co. Ltd. to develop in vivo adeno-associated virus-based therapies for three targets, including Friedreich's ataxia. Stridebio is eligible to receive about $680 million in future development and commercial milestones from Takeda, plus royalties on global net sales of commercial products. Stridebio is eligible to receive about $30 million in up-front and near-term preclinical milestones. (See BioWorld, April 3, 2019.)

Also, in late January, Neurocrine Biosciences Inc. agreed to pay Voyager Therapeutics Inc. $165 million up front and up to $1.7 billion in milestone payments for rights to develop and commercialize four experimental AAV-based gene therapies, one for Friedreich's ataxia and another for Parkinson's disease. (See BioWorld, Jan. 30, 2019.)

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