Rare Daily Staff

Allievex has completed a series A financing of an undisclosed size and obtained an exclusive global license to BioMarin Pharmaceutical’s mid-stage experimental enzyme replacement therapy tralesinidase alfa to treat Sanfilippo syndrome type B, a rare, pediatric neurodegenerative disease.

Sanfilippo syndrome type B, or mucopolysaccharidosis IIIB (MPS IIIB), is a lysosomal storage disease that is caused by deficiency in the enzyme alpha-N-acetyglucosaminidase (NAGLU), one of the four enzymes required for heparan sulfate degradation. The first symptoms generally appear between the ages of two and six years and consist of behavior disorders, intellectual deterioration, sleep disorders and, in some cases, mild dysmorphism. With time neurological involvement becomes more prominent with progressive loss of motor milestones and communication problems. The prognosis is poor with death occurring in most cases in the late teens or early 20s.

Tralesinidase alfa is an investigational enzyme replacement therapy using a novel fusion of recombinant human alpha-N-acetyglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 for the treatment of Sanfilippo syndrome type B or MPS IIIB. It is designed to restore NAGLU activity in the brain, and is delivered directly to the fluid surrounding the brain by an intracerebroventricular infusion. Tralesinidase alfa has been granted Orphan Drug designation by the European Commission and the U.S. Food and Drug Administration.

Allievex was founded by Pappas Capital, which co-led the financing with Novo Holdings. Kyle Rasbach, managing partner at Pappas Ventures and Tiba Aynechi, partner at Novo Holdings were appointed to its board of directors.

“Our mission at Allievex is to develop novel therapies for children with rare pediatric neurodegenerative diseases, and we are pleased to begin executing on this mission with tralesinidase alfa,” said Thomas Mathers, founder, president and CEO at Allievex and a partner at Pappas Ventures.

The financing will support registrational phase 2 clinical studies of tralesinidase alfa, its lead product candidate. Under its licensing agreement with BioMarin, the development program will continue uninterrupted with Allievex assuming all financial obligations for development and commercialization. BioMarin will provide certain manufacturing services in support of a regulatory filing for marketing approval.

The tralesinidase alfa development program consists of four independent and complementary multicenter, international studies: an observational study of the progression of Sanfilippo syndrome type B over time and has been completed; an ongoing phase 2 treatment study consisting primarily of eligible patients rolling over from the observational study; a treatment extension study designed to study longer term treatment effects of tralesinidase alfa; and a natural history study of the progression of Sanfilippo syndrome type B over time in children 0-18 years of age with all levels of cognitive function.

Under the terms of their agreement, Allievex will manage all clinical studies in the tralesinidase alfa program, which will continue uninterrupted. All study participants in the treatment studies will continue to receive tralesinidase alfa to evaluate its safety and efficacy as a potential treatment. BioMarin will provide necessary technical support and guidance throughout the transfer process to support a smooth transition by the end of the year.

Photo: Thomas Mathers, founder, president and CEO at Allievex

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