DNDi’s current filaria portfolio includes:

Research

One project in the research phase:

• Macro-filaricide 3: Following a drug repurposing strategy, completion of screening of compounds against Onchocerca gutturosa and Onchocerca lienalis identified several hits and leads. Screened compounds have been provided by a number of pharmaceutical companies and belong to well-characterized chemical series which have been extensively optimized for other indications. Although the project was quite successful, none of the identified hits had the right profile to become a drug candidate for filarial diseases. Further lead optimization has been undertaken in collaboration with industrial partners. These efforts will continue throughout 2017, with the aim of delivering a pre-clinical candidate for filarial diseases.

Translation

Two projects in the translation phase:

• Emodepside¹: Originating from the Japanese pharmaceutical company Astellas, emodepside has been developed and is currently commercialized by Bayer Animal Health as an anti-helmintic veterinary drug for cats and dogs. DNDi has a collaboration agreement with Bayer to jointly develop emodepside for the treatment of onchocerciasis. Emodepside entered into healthy volunteer Phase I studies in 2016. The Single Ascending Dose Study will be completed in 2017 while the protocols for the Multiple Dose Study will be initiated. The design of two Phase Ib studies to be performed in patients in Africa is also under finalization.

• ABBV-4083 (TylaMac): ABBV-4083 is a derivative of Tylosin, a veterinary antibiotic which targets the worm-symbiont Wolbachia The compound is currently in full pre-clinical development for the treatment of filarial diseases. ABBV-4083 is orally available, induces a robust anti-Wolbachia effect in several in vivo models, demonstrates clear superiority over doxycycline, and is effective after a shorter dosing regimen. Preliminary safety and toxicology profiling of this compound suggests a favourable safety profile. Upon completion of enabling toxicology studies, expected in 2017, and the development of an oral formulation, a Phase I study in healthy human volunteers will be performed in 2017. The aim will be to assess the safety, tolerability and the pharmacokinetics of ABBV-4083.