Allievex Completes Series A Financing to Advance Novel Therapy for Rare Pediatric Neurodegenerative Disease

VCBeat (WeChat ID: vcbeat) learned from foreign media that on October 23, 2019, Allievex Corporation (Allievex), a clinical-stage biotechnology company, announced the completion of its Series A financing round led by Pappas Capital and Novo Holdings.

Upon the closing of this financing round, Dr. Kyle Rasbach, Executive Partner at Pappas Ventures, and Dr. Tiba Aynechi, Partner at Novo Holdings, will join the board of directors of Allievex.

Allievex, founded by Pappas Capital, is a clinical-stage biotechnology company developing novel therapies for pediatric neurodegenerative diseases. The company’s lead candidate, traesinidase alfa, is an investigational enzyme replacement therapy that combines recombinant human alpha-N-acetylglucosaminidase (NAGLU) with a peptide derived from insulin-like growth factor 2 (IGF2) for the treatment of Sanfilippo syndrome type B, also known as mucopolysaccharidosis IIIB (MPS IIIB), in children. The drug has been designated as an orphan drug by both the European Commission and the U.S. FDA.

Sanfilippo Syndrome Type B is a disorder caused by NAGLU deficiency. NAGLU is one of the four enzymes required for the degradation of heparan sulfate (HS). Sanfilippo Syndrome Type B commonly affects children aged 2 to 6 years, with symptoms including behavioral disturbances, intellectual decline, and sleep disorders; in some cases, mild dysmorphic features are also present. Patients have a poor prognosis and a shortened life expectancy.

The proceeds from this round of financing will be used to support Phase 2 clinical studies of Allievex’s lead candidate, tralesinidase alfa. It is reported that Allievex has obtained an exclusive global license from BioMarin Pharmaceutical Inc. Under the agreement, Allievex will assume all financial obligations related to the development and commercialization of tralesinidase alfa, while BioMarin will be responsible for providing certain manufacturing services to support marketing authorization filings.

The open-label program for tradosinidase alfa comprises four independent and complementary multicenter international studies. BMN 250-901, an observational study of Sanfilippo syndrome type B, has been completed. BMN 250-201 is an ongoing Phase 2 therapeutic study, with its cohort consisting of eligible patients from the BMN 250-901 observational study. BMN 250-202 is a treatment extension study designed to evaluate the long-term therapeutic effects of tradosinidase alfa. BMN 250-902 is a natural history study investigating the progression of Sanfilippo syndrome type B in children aged 0–18 years across various cognitive levels.

“Allievex’s mission is to develop novel therapies for children with rare pediatric neurodegenerative diseases,” said Thomas P. Mathers, founder and CEO of Allievex.

Dr. Rasbach added, “Pappas Capital’s strategy is to build innovative life sciences companies, and our partnership with Allievex is a prime example of this strategy in action. Allievex has successfully completed its first round of financing, and Thomas P. Mathers has assembled an experienced biotechnology team. We believe that Allievex holds tremendous potential to transform the lives of children with rare diseases. We look forward to supporting Allievex over the long term as it drives its business forward.”

Dr. Aynechi stated, “As parents, we can imagine the immense suffering endured by children with MPS IIIB and their families. Novo’s significant investment in Allievex has enabled the continued development of tradesinidase alfa. We have full confidence in the Allievex team and look forward to advancing a portfolio of rare disease programs.”

Pappas Capital, founded in 1994 and headquartered in North Carolina, USA, is a venture capital firm specializing in investments in innovative life sciences companies. The firm primarily invests in enterprises in the United States and Canada operating in the fields of biotechnology, biopharmaceuticals, drug delivery, and medical devices. To date, Pappas Capital has raised over $540 million.

Novo Holdings, established in 1999 and headquartered in the Capital Region of Denmark, is wholly owned by the Novo Nordisk Foundation and primarily invests in the life sciences sector.

(Compiled by He Li)