Metabolic Diseases

Treatments for inborn errors of metabolism, lysosomal storage disorders, and other metabolic conditions.

Companies

Pipeline Drugs

1021

Key People

1155

Metabolic Diseases Pipeline (1021 drugs)

Commercial: 141Pre-clinical: 134Preclinical: 123Phase 2: 60Phase 1: 59Phase 3: 55Approved: 46Discovery: 41Development: 28Phase 1/2: 24Phase III: 21Research: 17Not Specified: 12Phase I: 10Phase II: 9Marketed: 9Phase 2b: 8Discovery/Preclinical: 8Phase I/II: 8Validated: 7Various: 6Pre-clinical Tool: 5Unknown: 5Marketed (Global): 5Phase 2/3: 4IND-Enabling: 4Clinical: 4Phase 2-4: 4IND Approved: 3Platform/Tool Development: 3Feasibility: 3Discovery/Pre-clinical: 3Phase 1b/2a: 3Service Platform: 3Discovery/Lead Optimization: 3Clinical POC: 3Phase II/III: 3Human Clinical: 3Approved/Marketed: 2Approved (Tentative): 2Market: 2Pre-clinical/Development: 2Phase 1b: 2Various (Typically Phase 2-4): 2Commercial Expansion: 2Exploratory: 2Phase 3 (planned): 2Pre-clinical/Phase 1: 2Not Disclosed: 2Development/Validation: 2Research/Pre-clinical: 2Pre-approval: 2Commercial Launch: 2Phases I-III & Commercial: 2Phase 2 (Planned): 2Phase 3 Ready: 2Phase IIb/III: 1Preclinical/Research: 1Marketed / NDA: 1ANDA / Marketed: 1Research / Pre-clinical: 1Application Submitted / BE Stage: 1IND Filed: 1Phase 3 / BLA: 1Pre-clinical/Clinical: 1Phase 4: 1Not Specified (Likely Phase 3/NDA): 1Commercial Tool: 1Service Offering: 1Various (Typically Phase 3): 1R&D: 1Approved/Commercial: 1Research Use Only: 1Approved (CE Mark): 1Development/Commercial: 1Commercial (Iterative Development): 1Regulatory: 1Development Stage (Pre-clinical): 1Development Stage: 1Phase 2a: 1IND-Prep: 1Optimization: 1Phase 2/Phase 3: 1Investigational Device: 1Prototyping: 1Phase 2B: 1Commercial Deployment: 1Commercial Enhancement: 1Clinical Validation: 1Phase 2 (planned): 1Clinical-stage (phase unspecified): 1Registry: 1Post-Market: 1Phase 1 / Bioequivalence: 1IND-enabling: 1Lead Optimization: 1Pre-clinical (integrated into Lilly pipeline): 1Co-development: 1Phase 1-ready: 1Proof-of-Principle: 1NA (Service for all trial phases): 1Phase 1/Phase 2: 1Pre-clinical/Clinical Study: 1Proof-of-Concept/Phase 2: 1Discovery to Pre-clinical Candidate: 1Research/Discovery: 1Preclinical/Clinical: 1Phase 1/2a: 1Orphan Designated: 1Proof-of-Concept (Human data reported): 1Pre-clinical or early clinical (inferred from license): 1Research-Use Only: 1Phase IIb: 1Pre-clinical/Research: 1Discovery/Development: 1Commercial/Development: 1Commercial (Support Program): 1Research Tool: 1Preclinical/Co-development: 1Regulatory Review: 1Pre‑launch: 1IND Accepted: 1NDA: 1IND: 1IDE/comparative study: 1Development & Commercial: 1Approved/Phase III: 1Discovery through Commercial: 1Preclinical through Clinical: 1Preclinical/IND-enabling: 1Application Submitted: 1BE Studies / Scale-up: 1Preclinical / Early Development: 1Formulation Development: 1Phase 3 (Preparatory): 1NDA Submitted: 1Phase III / NDA: 1Phase II / Phase III: 1Phase I / Phase II: 1ANDA Submitted: 1Phase not specified (Class 1 New Drug): 1Not specified (API/Formulation): 1Preclinical / Early Clinical: 1Partnered / Discovery: 1Discovery to Clinical: 1Preclinical Research: 1Preclinical to Commercial: 1Phase 1/2b: 1Not specified (likely clinical): 1Not Applicable: 1Not Specified (Likely Clinical): 1Phase 3 (Planned): 1Preclinical/Development: 1Pilot-Scale Validation: 1

Drug	Company	Indication	Phase	Status
Cell-in-a-Box® + Ifosfamide	PharmaCyte Biotech	Locally Advanced Pancreatic Cancer (LAPC)	Phase 2b	On Hold
Cell-in-a-Box® Insulin-Producing Cells	PharmaCyte Biotech	Type 1 & Insulin-dependent Type 2 Diabetes	Preclinical	On Hold
Piclidenoson (CF101)	Can Fite Biopharma	Plaque Psoriasis	Phase III	Active
Namodenoson (CF102)	Can Fite Biopharma	Hepatocellular Carcinoma	Phase IIb/III	Active
CF602	Can Fite Biopharma	Erectile Dysfunction	Preclinical	Active
Luna Overnight Automated Insulin Delivery System	Luna Diabetes	Glycemic control in diabetes (for Multiple Daily Injection users)	Pre-clinical	Active
DVI-001	Divamics	Inflammatory Bowel Disease	Preclinical	Approaching PCC selection
DVI-002	Divamics	Inflammatory Bowel Disease	Preclinical	Lead optimization
DVI-003	Divamics	Diabetes and Obesity	Preclinical	Early-stage optimization
DVI-004	Divamics	Metabolic Disease	Preclinical	Recently disclosed
REQORSA + Tagrisso	Genprex	EGFRm NSCLC post-TKI	Phase 1/2	Active (Acclaim-1)
REQORSA + Tecentriq	Genprex	Extensive-Stage SCLC	Phase 1/2	Active (Acclaim-3)
REQORSA	Genprex	NSCLC	Phase 1/2	Status Unclear (ONC-001)
GPX-002	Genprex	Type 1 & Type 2 Diabetes	Preclinical	Active
NPRL2 Gene Therapy	Genprex	Solid Tumors (e.g., KRAS/STK11 NSCLC)	Preclinical/Research	Active
TCM Product Portfolio	China SXT Pharmaceuticals	Cardiovascular, Digestive, Metabolic Disorders	Marketed / NDA	Commercial
Generic Drug Portfolio	China SXT Pharmaceuticals	CNS, Cardiovascular, Anti-infective	ANDA / Marketed	Commercial
New TCM Formulations	China SXT Pharmaceuticals	Not Specified	Research / Pre-clinical	Development
New Generic Approvals	China SXT Pharmaceuticals	Various	Application Submitted / BE Stage	Regulatory Review
Batoclimab (HBM9161)	Harbour BioMed	Myasthenia Gravis	Phase 3	Out-Licensed
HBM9378 (SKB378/WIN378)	Harbour BioMed	Asthma, COPD	Phase 1	Active (Greater China)
HBM7575 (SKB575)	Harbour BioMed	Atopic Dermatitis	IND Filed	Active (Global)
HBM2001	Harbour BioMed	Inflammatory Bowel Disease (IBD)	Discovery	Active (Global)
J9003	Harbour BioMed	IBD	Discovery	Active (Global)
R1065	Harbour BioMed	IgA Nephropathy (IgAN)	Discovery	Active (Global)
OX40L Family	Harbour BioMed	Dermatosis	Discovery	Active (Global)
HBM7020	Harbour BioMed	Autoimmune Diseases	Discovery	Out-Licensed
R2006	Harbour BioMed	Autoimmune Diseases	Discovery	Active (Global)
R7027	Harbour BioMed	Autoimmune Diseases	Discovery	Active (Global)
Porustobart (HBM4003)	Harbour BioMed	Melanoma, Colorectal Cancer, Hepatocellular Carcinoma, Neuroendocrine Neoplasm	Phase 2/3	Active (Greater China)
HBM1020	Harbour BioMed	Solid Tumors	Phase 1	Active (Global)
HBM7022	Harbour BioMed	Solid Tumors	Phase 1	Out-Licensed
HBM7008	Harbour BioMed	NSCLC	Phase 1	Active (Global)
HBM9027	Harbour BioMed	Pancreatic Cancer	Phase 1	Active (Global)
HBM7004	Harbour BioMed	NSCLC	Discovery	Active (Global)
HBM9033	Harbour BioMed	Solid Tumors	Discovery	Out-Licensed
LET003	Harbour BioMed	Obesity	Discovery	Active (Global)
LET001	Harbour BioMed	Obesity	Discovery	Active (Global)
eRapa	Biodexa Pharmaceuticals	Familial Adenomatous Polyposis (FAP)	Phase 3	Active - Enrollment
MTX240 (OPB-171775)	Biodexa Pharmaceuticals	TKI-Resistant Gastrointestinal Stromal Tumors (GIST)	Phase 1/2	Licensed Asset
Tolimidone	Biodexa Pharmaceuticals	Type 2 Diabetes	Phase 2	Acquired Asset
Chidamide (Tucidinostat)	Chipscreen Biosciences	Lymphoma (and other cancers)	Approved/Marketed	Commercial; Included in NRDL
CS08399	Chipscreen Biosciences	MTAP-Deficient Tumors	IND Approved	Clinical development imminent
Second Marketed Product	Chipscreen Biosciences	Not specified on homepage	Approved/Marketed	Commercial
NGM120	NGM Biopharmaceuticals	Hyperemesis Gravidarum	Phase 2	Active
NGM831 (MK-3655)	NGM Biopharmaceuticals	Metabolic Dysfunction-Associated Steatohepatitis (MASH)	Phase 2b	Active (Merck-led)
NGM621	NGM Biopharmaceuticals	Geographic Atrophy (GA)	Phase 2	Active (Merck-led)
NGM438	NGM Biopharmaceuticals	Genetic Lipodystrophy	Preclinical	Active
Nemonoxacin Malate Capsules	Zhejiang Medicine	Bacterial Infections	Commercial	Marketed
Dupixent (dupilumab)	Regeneron	Chronic Obstructive Pulmonary Disease (COPD)	Phase 3	Active