Science

Uncovering a novel compound

petri dish
Combining our expertise in fungal molecular biology and biochemistry, we identify and develop novel antifungal compounds. Our team discovered the orotomides, a class of small-molecule inhibitors that selectively target fungal dihydroorotate dehydrogenase (DHODH).1 DHODH is a key enzyme involved in the synthesis of DNA, RNA, and proteins.2 Inhibition of DHODH initiates a cascade of multiple cellular effects, ultimately leading to fungal cell death. This novel MOA may kill the fungus, while many other antifungals simply stop it from growing.3

A scientific breakthrough

Our lead candidate is the first antifungal to be granted orphan drug status from the European Medicines Agency, as well as orphan drug status and qualified infectious disease product (QIDP) designation from the FDA. Additionally, the FDA granted the candidate two Breakthrough Therapy Designations (BTD), a process designed to speed up the development and review of drugs for which preliminary clinical evidence indicates that they may demonstrate substantial improvement over currently available therapies.

BTD products are offered all Fast Track Designation features, as well as additional guidance and organisational commitment from the FDA. This designation means that we have a chance to deliver a novel class of therapeutics to patients who need other treatment options.

Our pipeline

Lead candidate formulation
Disease area
Formulation & preclinical
Phase 1
Phase 2
Phase 3
Commercial rights
Oral
Invasive aspergillosis, Coccidioidomycosis, and Other rare molds
Formulation & preclinical
Phase 1
Phase 2
Phase 3
Breakthrough Therapy Designation*
f2g & Shionogi Logos
Oral
Invasive aspergillosis
Formulation & preclinical
Phase 1
Phase 2
Phase 3
f2g & Shionogi Logos
Oral
Coccidioidomycosis
Formulation & preclinical
Phase 1
Phase 2
Phase 3
f2g logo
Pediatric oral
Invasive aspergillosis, Coccidioidomycosis, and Other rare molds
Formulation & preclinical
Phase 1
Phase 2
Phase 3
f2g & Shionogi Logos
Other
IV formulation
Inhaled formulation
Formulation & preclinical
Phase 1
Phase 2
Phase 3
f2g logo

We also are leveraging our expertise in fungal molecular biology and biochemistry and our deep understanding of the orotomide class of antifungal agents that we discovered to identify and develop  additional novel antifungal compounds.
*First antifungal agent to receive Breakthrough Therapy designation.
†Commercial rights partner exclusive to Europe and Asia.

Our focus

Invasive aspergillosis
Every year, more than 250,000 people worldwide contract invasive aspergillosis (IA), a life-threatening infection caused by several Aspergillus mold species.4 Aspergillus is an opportunistic pathogen, meaning that it causes infections in immunocompromised individuals but is normally harmless to healthy populations.5 Aspergillus spores are inhaled from the environment and can cause serious respiratory infections, resulting in pneumonia-like symptoms that may worsen.6 Critically ill and immunocompromised patients are at high risk for contracting severe and possibly fatal infections.7
Learn more about invasive aspergillosis:

NORD
Aspergillus.org (UK)
Aspergillosis Trust
Fungal Infection Trust

Rare, highly resistant mold infections

These infections are caused by a less common but highly resistant group of molds that includes the Lomentospora, Scedosporium, Scopulariopsis, and Rasamsonia species. These molds exhibit a pathophysiology similar to Aspergillus, presenting a significant challenge to both the diagnosis and treatment of patients and often cause life-threatening infections in immunocompromised patients.

Learn more about rare, highly resistant mold species:

American Society for Microbiology

Coccidioidomycosis (Valley fever)
Coccidioidomycosis is an infection caused by inhaling the Coccidioides fungus that lives in the soil of southwestern America, parts of Mexico, and Central and South America.8 The fungus is endemic, meaning that it is always present but limited to a specific geographical area. However, it shows the potential to spread to other regions due to climate change, increased travel, and other factors.9 Both healthy and immunocompromised individuals can contract this serious infection. In some cases, the infection can spread from the lungs and cause serious extrapulmonary infections, including to the CNS.10
Learn more about coccidioidomycosis (Valley fever):

Center for Disease Control and Prevention
California Department of Public Heath

Projected Expansion of Coccidioidomycosis Endemic Regions9
2007
map cocci region 2007
2035
2065
2095
map cocci region 2095
For more information, contact medinfo email address
Reach out for more information
References
  1. Hope WW, McEntee L, Livermore J, et al. Pharmacodynamics of the orotomides against Aspergillus fumigatus: new opportunities for treatment of multidrug-resistant fungal disease. mBio. 2017;8(4):e01157-17. doi:10.1128/mBio.01157-17
  2. White RM, Cech J, Ratanasirintrawoot S, et al. DHODH modulates transcriptional elongation in the neural crest and melanoma. Nature. 2011;471(7339):518-522. doi:10.1038/nature09882
  3. Mourad A, Perfect JR. Tolerability profile of the current antifungal armoury. J Antimicrob Chemother. 2018;73(suppl_1):i26-i32. doi:10.1093/jac/dkx446
  4. Bongomin F, Gago S, Oladele RO, Denning DW. Global and multi-national prevalence of fungal diseases-estimate precision. J Fungi (Basel). 2017;3(4):57. doi:10.3390/jof3040057
  5. Martínez JL. Short-sighted evolution of bacterial opportunistic pathogens with an environmental origin. Front Microbiol. 2014;5:239. doi:10.3389/fmicb.2014.00239
  6. Cadena J, Thompson GR III, Patterson TF. Aspergillosis: epidemiology, diagnosis, and treatment. Infect Dis Clin North Am. 2021;35(2):415-434. doi:10.1016/j.idc.2021.03.008
  7. WHO fungal priority pathogens list to guide research, development and public health action. Geneva: World Health Organization; 2022. Licence: CC BY-NC-SA 3.0 IGO.
  8. About valley fever (coccidioidomycosis). Centers for Disease Control and Prevention. https://www.cdc.gov/fungal/diseases/coccidioidomycosis/definition.html. Published November 12, 2020. Accessed January 30, 2023.
  9. Gorris ME, Treseder KK, Zender CS, Randerson JT. Expansion of coccidioidomycosis endemic regions in the United States in response to climate change. Geohealth. 2019;3(10):308-327. doi:10.1029/2019GH000209
  10. Jackson NR, Blair JE, Ampel NM. Central nervous system infections due to coccidioidomycosis. J Fungi (Basel). 2019;5(3):54. doi:10.3390/jof5030054
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