Pipeline landscape

Helminth drug and target pipeline

A working map of approved, clinical, repurposed and discovery-stage opportunities for parasitic worm diseases.

Helminthix is not presenting these as Helminthix-owned assets. This page is a landscape view: the medicines, targets, developers and gaps that matter if the field is going to move from a handful of ageing drug classes toward a deeper, resistance-aware medicine cabinet.

Petri dish in a laboratory
Programme / asset Disease focus Target / mechanism Phase 1 Phase 2 Phase 3 Approved / access Developer or steward Evidence links
Arpraziquantelpaediatric praziquantel formulation Schistosomiasis in preschool children Praziquantel-sensitive flatworm calcium signalling; TRPMPZQ-linked biology Late-stage / access Pediatric Praziquantel Consortium led by Merck KGaA, Darmstadt, Germany, with Astellas, Swiss TPH, Lygature and partners
Moxidectinrepurposed macrocyclic lactone Onchocerciasis Glutamate-gated chloride channel biology; long-acting microfilarial suppression Approved / implementation Medicines Development for Global Health, WHO/TDR and endemic-country partners
Moxidectinrepurposing / dose-ranging Strongyloides stercoralisStrongyloidiasis; STH species-aware efficacy gaps Strongyloides requires distinct ivermectin/moxidectin-centred strategies; standard benzimidazole deworming is insufficient. Resistance-aware monitoring needed; veterinary nematodes are the warning system. Phase 2a / repurposing Clinical repurposing investigators and endemic-country partners
Emodepsideveterinary-origin nematocide Onchocerciasis and filarial disease concepts Latrophilin / SLO-1 / BK-channel-associated nematode signalling Clinical development DNDi and Bayer
Oxantel / oxantel combinationsTrichuris gap Trichuris trichiuraSoil-transmitted helminths; species-aware efficacy gaps Nicotinic acetylcholine receptor biology; whipworm-focused treatment gap Clinical / translational Clinical and translational STH research community
Albendazole-ivermectin and moxidectin-albendazoleimproved combination regimens Trichuris trichiura, hookworm, AscarisSoil-transmitted helminths; species-aware efficacy gaps Beta-tubulin plus macrocyclic-lactone treatment biology; improved species-aware regimens. Resistance-aware monitoring needed; veterinary nematodes are the warning system. Clinical / translational Clinical and translational STH research community
Emodepsidenew-mechanism nematode pharmacology Soil-transmitted helminths and nematode drug-development conceptsSpecies-aware efficacy gaps Latrophilin / SLO-1 / BK-channel-associated nematode signalling; new mechanism beyond beta-tubulin and macrocyclic-lactone backbones Clinical / translational Clinical and translational STH research community
Oxfendazolerepurposed benzimidazole River blindness, soil-transmitted helminths and tissue-stage cestode concepts β-tubulin / microtubule disruption; improved benzimidazole exposure, but still β-tubulin class; useful but not a new mechanism. Resistance-aware monitoring needed; veterinary nematodes are the warning system. Phase 2 / repurposing DNDi and clinical / academic partners
Oxfendazoleactive isomer evidence; flubendazole comparator biology Onchocerca, Litomosoides and cestode conceptsOxfendazole repurposing Beta-tubulin / benzimidazole pharmacology, improved exposure and macrofilaricidal activity; useful but not a new mechanism Phase 1 PK and safety Preclinical / repurposing Veterinary benzimidazole repurposing groups and clinical pharmacology partners
FlubentylosinTylAMac / ABBV-4083 Onchocerciasis Anti-Wolbachia antibiotic approach Terminated after Phase 2 DNDi, AbbVie and A-WOL / Liverpool School of Tropical Medicine partners
Doxycyclineexisting antibiotic regimen Filarial diseases Wolbachia depletion Repurposed / guideline use Generic medicine; anti-Wolbachia clinical research community
TribendimidineChina-origin anthelmintic Soil-transmitted helminths and trematode exploration Nicotinic acetylcholine receptor signalling Approved regionally Chinese development and manufacturing ecosystem; academic tropical-medicine collaborators
Albendazole / mebendazolecurrent backbone Soil-transmitted helminths, lymphatic filariasis combinations, selected cestode infectionsSpecies-aware efficacy gaps: Trichuris, hookworm and Strongyloides β-tubulin / microtubule disruption. Resistance-aware monitoring needed; veterinary nematodes are the warning system. Approved / backbone Generic supply, donation programmes and global-control partners
Emodepside and ivermectinIDA / IA combinations and related treatment strategies Onchocerca, lymphatic filariasis parasites and Litomosoides sigmodontisFilarial disease Macrofilaricidal treatment biology, SLO-1 / latrophilin pharmacology, paediatric safety and immune context Phase 1 evidence Clinical-stage strategy DNDi, filarial clinical researchers and endemic-country programme partners
Praziquantelcurrent schistosomiasis and cestode backbone Schistosomiasis, taeniasis and other flatworm infections Flatworm calcium signalling; TRPMPZQ-linked biology Approved / backbone Generic manufacturers, Merck KGaA donation programme and public-health partners
Niclosamiderepurposing scaffold; discovery scaffold Intestinal cestodes and broader screening concepts Mitochondrial / energy metabolism disruption Generic medicine; academic and translational repositioning studies

Discovery evidence

Gene targets and early discovery programmes

For helminths, some of the most important opportunities are not company assets yet. This table keeps the species, target biology, molecule or treatment concept, and paper evidence together so each row reads like a possible development lane.

Programme / asset Disease focus Target / mechanism Pipeline relevance Evidence links
Afatinibapproved ErbB-family cancer drug Echinococcus multilocularisAlveolar echinococcosis EmER1 EGF receptor and parasite neuregulin-like ligand EmNRG in germinative-cell biology. Brehm-linked Echinococcus stem-cell biology mechanistically connects an oncology drug class to a parasite germinative-cell vulnerability.
BI 2536human PLK1 oncology inhibitor Echinococcus multilocularisLarval cestode disease EmPlk1, a polo-like kinase required for germinative-cell proliferation. University of Würzburg / Brehm group work with named species, named kinase, named inhibitor and parasite-growth readout.
SGI-1776 and CX-6258mammalian PIM kinase inhibitors Echinococcus multilocularisLarval cestode disease Parasite PIM kinase orthologues and growth / viability pathways. Anti-Echinococcus chemotherapy work linking parasite genomics, cancer-kinase pharmacology and existing chemical matter.
Oxamniquinestructure-guided analogues Schistosoma mansoniSchistosomiasis SmSULT, the parasite sulfotransferase that activates oxamniquine. Explains species specificity and resistance, giving medicinal chemistry a route to redesign an older class.
cbp1 RNAitarget-validation knockdown Schistosoma mansoniAdult schistosome survival biology RNAi knockdown of cbp1 reduced transcript levels and was associated with tissue degeneration, increased stem-cell proliferation and parasite death in vivo. Specific genetic loss-of-function evidence that cbp1-linked chromatin / transcriptional regulation is a survival vulnerability.
SmNAGAL depletionRNAi and CRISPR/Cas9 target validation Schistosoma mansoniMovement and egg-production phenotypes RNAi and CRISPR/Cas9 depletion of SmNAGAL impaired coordinated adult-worm movement and disrupted egg production / egg morphology. Links a named glycosidase target to measurable viability-adjacent phenotypes that can support assay design and target triage.
SmVAL6 knockdowntegument-barrier target validation Schistosoma mansoniTegument and host-interface biology RNAi knockdown of SmVAL6 increased surface membrane permeability and disrupted tegumental barrier function. Makes a named tegument-associated protein experimentally actionable for barrier-integrity assays and parasite vulnerability work.
Alpha-mannosidase inhibitionlive-worm glycan remodelling Schistosoma mansoniAdult worm glycan biology Chemical inhibition of alpha-mannosidases in live adult worms remodelled parasite N-glycans. Specific target-class perturbation in living parasites; useful as a glycan-pathway entry point, though not yet a named drug programme.
SmFHII selective inhibitionclass II fumarase structural target work Schistosoma mansoniEnergy metabolism enzyme target Schistosome class II fumarase, SmFHII, was structurally and biochemically characterized, including ligand screening and comparison with the human enzyme for selective-inhibitor design. Provides target-structure and selectivity rationale for a named metabolic enzyme, but still needs parasite-killing translation.
G-quadruplex ligandsnucleic-acid structure targeting Schistosoma mansoniLarval and adult parasite assays Predicted G-quadruplex motifs were validated in S. mansoni; G4-binding small molecules were then tested against newly transformed schistosomula and adult worms. Connects a defined nucleic-acid structural feature to live-parasite compound activity, while target specificity still needs refinement.
BET-like bromodomain ligandsSmLSD1 modulators and text-mined / ChEMBL chemical matter Schistosoma mansoniEpigenetic and cheminformatics-led discovery SmBRD3, SmLSD1 and curated anti-schistosomal compound knowledge. Turns scattered compound evidence into reusable small-molecule and target hypotheses.
Quinoxalines and triazinesN-acyl homoserine lactones, biarylalkyl carboxylic acids, natural molluscicide and tiruchanduramine analogues Schistosoma mansoniAnti-schistosomal chemical matter Pleiotropic parasite phenotypes, cercarial development, glycosidase inhibition and scaffold exploration. Early small-molecule evidence for anti-schistosomal optimization and phenotypic triage.
Modified hederagenin derivativesabietane diterpenoid analogues Fasciola hepaticaFascioliasis and flukes Fluke viability and ex vivo flukicide phenotypes. Early chemical matter for foodborne trematodes and veterinary-human overlap.
Corallopyronin A and quinazolinesAN11251 boron-pleuromutilin and substituted thiadiazoles / benzimidazole-benzoxaborole hybrids Filarial nematodes with WolbachiaAnti-Wolbachia strategy Wolbachia depletion and bacterial RNA polymerase / antibiotic-sensitive endosymbiont biology. Macrofilaricide concept built around shorter, safer alternatives to long-course antibiotic anti-Wolbachia regimens.
Diagnostic TPPs and trial simulatorsantifilarial trial design frameworks Helminth infections and filarial programmesTranslation, trials and access Diagnostics, clinical-trial simulation and discovery sustainability. Keeps translational infrastructure visible beside target and molecule evidence.

Additional resources

Useful context papers that support target discovery, tissue interpretation or field framing, but are not single target-or-compound programme rows.

Helminthix role

From target lists to investable programmes

Our work is to make the missing middle legible: matching AI biopharma and biotech platforms with parasite experts, high-throughput screening, viability assays, medicinal chemistry, funders and endemic-region partners.

  • Target discovery: conserved, parasite-selective, druggable vulnerabilities.
  • Functional evidence: motility, viability, development, fecundity, washout persistence and stage-specific phenotypes.
  • Repurposing acceleration: known-safe compounds can sometimes move faster when exposure, formulation and indication risks are tractable.
  • Resistance readiness: escape risk, veterinary lessons and combination logic should be considered before deployment.

Help improve this landscape

This is a living page. If you know of a programme, target, molecule, clinical trial or correction that should be included, please contact us.