Azafaros will use the Series B haul to push lead asset nizubaglustat into late-stage studies for Niemann-Pick disease Type C and GM1/GM2 gangliosidoses later this year.
Azafaros has brought home €132 million ($148 million) in series B capital to advance a pipeline of therapies against rare lysosomal storage disorders.
The money will help the Dutch biotech run two Phase III programs for the investigational small molecule drug nizubaglustat, which is being tested for Niemann-Pick disease Type C (NPC) and GM1/GM2 gangliosidoses. The company expects to launch both late-stage trials later this year, according to a Tuesday release.
Both of Azafaros’ disease targets are rare neurometabolic conditions characterized by enzyme deficiencies, leading to the abnormal accumulation of toxic substances. NPC, for instance, involves the build-up of lipids in various tissues throughout the body, including the brain and liver. Symptoms of NPC vary from patient to patient, with some experiencing difficulties swallowing and walking. In some cases, NPC can be fatal within months of birth.
GM1 and GM2 gangliosidoses involve the build-up of specific types of glycolipids—fatty molecules with a sugar moiety attached. These conditions affect the peripheral and central nervous system and, when left unchecked, can turn fatal. Common symptoms include progressive neurodegeneration, cognitive decline and impaired motor function.
Designed to be taken orally, nizubaglustat has a “dual mode of action” and “the potential to offer disease-modifying” effects, according to Azafaros’ website, though the biotech did not reveal what the drug’s specific target is.
Aside from funding the late-stage development of nizubaglustat, Azafaros will also use the funds from Tuesday’s series B to expand its pipeline into other indications. The financing round was led by Jeito Capital and Forbion Growth.
Azafaros’ series B round is welcome news to the broader rare disease space, which in recent weeks has been hit with financial troubles and clinical setbacks. In April, for instance, Rallybio shelved its monoclonal antibody RLYB212, which was being developed for the rare bleeding disorder fetal and neonatal alloimmune thrombocytopenia, after a disappointing Phase II showing. Last week, Rallybio cut off 40% of its workforce. Meanwhile, in March, Sarepta Therapeutics took a 22% stock hit after a patient taking its Duchenne muscular dystrophy gene therapy Elevidys died.
Not all is bleak, however. In mid-April, California’s Glycomine closed a $115 million Series C financing round, which will help bankroll Phase IIb assessments for its lead asset GLM101 in phosphomannomutase-2 congenital disorder of glycosylation. A couple of weeks later, Novartis acquired Regulus Therapeutics and its investigational oligonucleotide farabursen, being tested for autosomal dominant polycystic kidney disease, for a potential $1.7 billion.
