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SOM1311

Phenylketonuria (PKU)

PreclinicalActive

Key Facts

Indication

Phenylketonuria (PKU)

Phase

Preclinical

Status

Active

Company

SOM Biotech

About SOM Biotech

SOM Biotech is a Spanish biotech leveraging its proprietary AI platform, SOMaiPRO®, to accelerate drug discovery for rare CNS conditions with high unmet need. The platform has demonstrated strong clinical predictivity, with two identified compounds achieving positive Phase 2a proof-of-concept results. The company's lead program, SOM3355 for Huntington's disease, is poised to enter Phase 3, positioning SOM Biotech as a late-stage clinical development entity. Its strategy combines AI-driven discovery with deep clinical expertise to build a targeted pipeline in valuable, underserved neurological and psychiatric markets.

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Therapeutic Areas

Neurology Psychiatry

Other Phenylketonuria (PKU) Drugs

Drug	Company	Phase	Status
SYNB1934	Synlogic	Phase 3	Completed; Did Not Meet Primary Endpoint
PKU Treatment	Cycle Pharmaceuticals	Commercial	Active
NB-20X	Nerai Biosciences	Pre-clinical	Active
NGGT002	NGGT	Phase 1/2 (inferred)	Active
Repinatrabit	Otsuka	Open-Label Extension	Active
PER303	Perseo Pharma	Preclinical	Active
Engineered Native Bacteria Platform	Endure Biotherapeutics	Pre-clinical	Active
JNT-517	JCR Pharmaceuticals	Phase 1	Active
PALYNZIQ (pegvaliase-pqpz)	BioMarin Pharmaceutical	Approved/Marketed	Active (Label expansion)
PTC923 (sepiapterin)	PTC Therapeutics	Phase 3	Active