Azafaros Secures $150M Series B Funding to Advance Rare Disease Therapies

Azafaros, a Dutch biotech company, has successfully raised €132 million ($148 million) in a Series B financing round to further develop treatments for rare lysosomal storage disorders. The funding, led by Jeito Capital and Forbion Growth, will primarily support the advancement of the company's lead asset, nizubaglustat, into late-stage clinical trials.

Nizubaglustat: Targeting Rare Neurometabolic Disorders

Nizubaglustat, an oral small molecule drug, is being developed for two rare neurometabolic conditions: Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses. Both disorders are characterized by enzyme deficiencies that lead to the abnormal accumulation of toxic substances in the body.

Azafaros plans to initiate two Phase III clinical trials for nizubaglustat later this year. The drug is designed with a dual mode of action and has the potential to offer disease-modifying effects, although specific details about its mechanism of action have not been disclosed.

Rare Disease Landscape: Challenges and Opportunities

The rare disease space has faced recent setbacks, including Rallybio's decision to discontinue development of its monoclonal antibody RLYB212 for fetal and neonatal alloimmune thrombocytopenia, resulting in a 40% workforce reduction. Additionally, Sarepta Therapeutics experienced a significant stock decline following a patient death in its Duchenne muscular dystrophy gene therapy trial.

However, the sector has also seen positive developments. Glycomine recently closed a $115 million Series C financing round to support Phase IIb trials of GLM101 for phosphomannomutase-2 congenital disorder of glycosylation. Moreover, Novartis acquired Regulus Therapeutics and its investigational oligonucleotide farabursen, being tested for autosomal dominant polycystic kidney disease, in a deal worth up to $1.7 billion.

Pipeline Expansion and Future Prospects

In addition to funding the late-stage development of nizubaglustat, Azafaros intends to use the Series B proceeds to expand its pipeline into other indications. This strategic move aims to strengthen the company's position in the rare disease market and potentially address unmet medical needs in various lysosomal storage disorders.