R&D MODEL & PORTFOLIO
2015 KEY FINANCIAL PERFORMANCE INDICATORS
EUR 32.7 million: moving toward a more dynamic R&D portfolio while maintaining a robust Kinetoplastids disease pipeline
R&D EXPENDITURE BY DISEASE (2014-2015)
HAT Leishmaniasis Chagas Filaria
8% 2% 2%
Overall R&D expenditures (EUR 32.7 M) increased by 18% (EUR 5.1 M) compared to 2014. Percentage breakdown highlights of 2015 R&D expenditures per disease (screening and lead optimization expenditures are split and allocated towards disease expenditures) • Kinetoplastids diseases remain at the heart of the portfolio with 77% of the expenses: • Portfolio expansion: The three new disease and exploratory areas represent 23%, compared to 17% in 2014. Their increase (+EUR 2.3 M, +55%) is the most signiﬁcant of the DNDi portfolio.
Human African trypanosomiasis (HAT)
With a total of EUR 8.7 M, HAT represents the most substantial R&D expenditure (31%). Investments increased due to the growth in clinical activities for fexinidazole (+ EUR 1.6 M), with the Phase IIb/III clinical study and the two additional cohorts (for stage 1 & early stage 2 and for children) with 10 operational sites in the DRC plus the preparation of three new clinical trial sites. The SCYX-7158 project completed Phase I, with Phase II/III now being prepared (writing synopsis, getting scientiﬁc advice from EMA, meeting with CARSAC (Cameroon) to present and evaluate the protocol, and ﬁnally submission to the Ethics Committee in the DRC). More details on page 25, “Development”.
Project expenditures increased by 66% (+EUR 1.2 M). Screening work is ongoing and increased (+EUR 0.2 M) as well as the preclinical activities (+EUR 0.3 M), with two new candidates (oxfendazole and TylAMac). The ﬂubendazole project was however closed in early 2015 (- EUR 0.8 M). The main increase is related to the Phase I for emodepside (+ EUR 1.5 M).
Project expenditure increased by 108% (+EUR 1.1 M). The implementation study for a “4-in-1” product (+EUR 0.3 M) is developing with three countries and 9 sites involved in the project, and 49 patients recruited in January 2016. The clinical ‘superbooster’ study (ritonavir for super boosting LPV/r) in South Africa (+ EUR 0.1 M) is ongoing with the purchase of equipment (+EUR 0.7 M) for the formulation development of the “4-in-1” with Cipla Ltd as an industrial partner to enable the production of treatments for the implementation study.
Overall expenditures remained stable between 2014 and 2015 at EUR 8.4 M. Some projects are entering into the portfolio or progressing, such as the preclinical work for VL with DNDI-0690 (+EUR 0.4 M), the CPG for CL project (+EUR 0.1 M), the preparation of clinical trial study for PKDL treatment (+EUR 0.1 M), the combination fexinidazole/miltefosine project, and the completion of the recruitment of 30 patients for the Miltefosine Allometric Study (+EUR 0.4 M). Other projects are completed, such as the preclinical package for VL-2098 (-EUR 0.7 M) and the VL Combo study in Asia (-EUR 0.2 M), or progressing in a different phase, like the VL India implementation (-EUR 0.2 M).
Handover to MMV is complete (- EUR 0.5 M), however the ASAQ technology transfer was still ongoing in 2015.
Projects remained stable in 2015 (- EUR 0.2 M), accounting for a total of EUR 4.8 M (17%) of R&D expenditure. The screening and lead optimization work toward Chagas disease increased by EUR 0.7 M. The Chagas access projects (+EUR 0.2 M) are developing activities in Latin America and North America. The completion of the study of fexinidazole for Chagas and the closure of the E1224 project incurred a decrease of EUR 0.7 M. The biomarker project was put on hold due to safety issues at the end of 2015, which entailed a budget decrease of EUR 0.4 M.
Dynamic portfolio - Exploratory and feasibility studies