First Sanfilippo Syndrome Type A Patient Receives Sobi’s Investigational SOBI003

Swedish Orphan Biovitrum AB’s (Sobi) has dosed the first patient in its Phase 1/2 clinical trial evaluating SOBI003 to treat Sanfilippo syndrome type A.

The SOBI003-001 study (NCT03423186), which is still recruiting participants, intends to assess the therapy’s safety, tolerability and effectiveness when delivered to Sanfilippo A children, 1 to 6 years old, who have not received prior treatment.

Nine children will be enrolled from two clinical sites in the U.S. — the Childrens’s Hospital and Research Center, Oakland, California (recruitment active) and the University of North Carolina Hospitals — and from Gazi University Hospital, Turkey.

Participants will be divided into three groups, each receiving escalating doses of SOBI003, starting at 3 mg/kg and delivered directly into the blood (intravenously), once a week for 24 weeks.

“Since there is currently no treatment available for MPS IIIA [mucopolysaccharidosis type IIIA], the initiation of this study is an important first step towards finding a potential treatment for this debilitating disease,” Milan Zdravkovic, MD, said in a press release. Zdravkovic is chief medical officer and head of research and development at Sobi.

“I am very pleased that we were able to enroll the first patient in this important study. We look forward to learning more about how SOBI003 may potentially be able to help patients in the future as we enroll more patients into the study,” said Paul Harmatz, of University of California San Francisco Benioff Children’s Hospital in Oakland, California.

Sanfilippo syndrome type A is caused by a deficiency in the enzyme heparan N-sulfatase, which is responsible for the breakdown of heparan sulfate — a specific type of large sugar molecule called  glycosaminoglycans (GAGs).

This leads to the toxic accumulation of heparan sulfate within cells and tissues, causing cellular damage in multiple organs.

SOBI003 is a chemically modified human recombinant sulfamidase that can reduce the levels of heparan sulfate in affected tissues. This enzyme replacement therapy is taken up by cells and transported into the area where GAGs accumulate.

In October 2016, SOBI003 received orphan drug designation by the European Commission for Sanfilippo syndrome type A, and by the U.S. Food and Drug Administration (FDA) in 2017.

The FDA accepted Sobi’s investigational new drug application (IND) for SOBI003 in January 2018.

SOBI003 also was granted fast track status by the FDA, which is expected to accelerate its clinical development and regulatory review.