Shionogi & Jordan’s Guardian Angels Launch First Neurodevelopment Trial

Shionogi and Jordan’s Guardian Angels have announced a collaboration to initiate the first clinical trial for PPP2 syndrome type R5D (Houge-Janssens syndrome 1, HJS1), also known as Jordan’s Syndrome.

Jordan’s Syndrome causes global developmental delays, seizures, vision impairments, muscle weakness, attention issues, and other challenges.
 
Symptoms vary among individuals, and no specific treatments currently exist. Although only about 350 people worldwide have been diagnosed, experts believe the actual number is higher. 

Diagnosis requires genetic testing, which may be difficult to access.

The US Food and Drug Administration (FDA) has granted zatolmilast a Rare Paediatric Disease Designation, a status for serious conditions primarily affecting children under 18 with fewer than 200,000 cases in the US.

The Phase 2 randomised, double-blind, placebo-controlled trial will assess the safety and tolerability of zatolmilast (BPN14770), a selective PDE4D inhibitor, in individuals with Jordan’s Syndrome. 

The study will also gather preliminary efficacy data, pharmacokinetic insights, and biomarker information. Zatolmilast was originally discovered by Tetra Therapeutics, acquired by Shionogi in 2020, and is also being studied for Fragile X syndrome in an advanced Phase 2b/3 trial in the US.

Jordan’s Guardian Angels, the only advocacy group dedicated to Jordan’s Syndrome, has spent eight years raising awareness and funding research. 

The study is supported by ten research and academic institutions, including Columbia University, Seattle Children’s Hospital, and Boston Children’s Hospital, with financial backing from the state of California.

The trial is expected to conclude in late 2026. More details are available at ClinicalTrials.gov (ID: NCT06717438).

Shionogi is also conducting late-stage clinical trials of zatolmilast for Fragile X syndrome (FXS). A prior Phase 2 study involving 30 adult males with FXS demonstrated safety and showed improvements in cognitive function, particularly in language skills. 

The most common side effects were vomiting and upper respiratory tract infections, with similar rates in both active and placebo groups. The EXPERIENCE trial programme continues to investigate zatolmilast’s impact on cognition and daily functioning in FXS.

Zatolmilast remains an investigational drug, with its safety and efficacy not yet confirmed. Regulatory approval is not guaranteed.

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