Financing repurposed drugs for rare diseases: a case study of Unravel Biosciences.
Orphanet J Rare Dis
; 18(1): 287, 2023 09 12.
Article
em En
| MEDLINE
| ID: mdl-37700316
ABSTRACT
BACKGROUND:
We consider two key challenges that early-stage biotechnology firms face in developing a sustainable financing strategy and a sustainable business model developing a valuation model for drug compounds, and choosing an appropriate operating model and corporate structure. We use the specific example of Unravel Biosciences-a therapeutics platform company that identifies novel drug targets through off-target mechanisms of existing drugs and then develops optimized new molecules-throughout the paper and explore a specific scenario of drug repurposing for rare genetic diseases.RESULTS:
The first challenge consists of producing a realistic financial valuation of a potential rare disease repurposed drug compound, in this case targeting Rett syndrome. More generally, we develop a framework to value a portfolio of pairwise correlated rare disease compounds in early-stage development and quantify its risk profile. We estimate the probability of a negative return to be [Formula see text] for a single compound and [Formula see text] for a portfolio of 8 drugs. The probability of selling the project at a loss decreases from [Formula see text] (phase 3) for a single compound to [Formula see text] (phase 3) for the 8-drug portfolio. For the second challenge, we find that the choice of operating model and corporate structure is crucial for early-stage biotech startups and illustrate this point with three concrete examples.CONCLUSIONS:
Repurposing existing compounds offers important advantages that could help early-stage biotech startups better align their business and financing issues with their scientific and medical objectives, enter a space that is not occupied by large pharmaceutical companies, and accelerate the validation of their drug development platform.Palavras-chave
Texto completo:
1
Coleções:
01-internacional
Base de dados:
MEDLINE
Assunto principal:
Comércio
/
Doenças Raras
Tipo de estudo:
Health_economic_evaluation
/
Prognostic_studies
Limite:
Humans
Idioma:
En
Revista:
Orphanet J Rare Dis
Assunto da revista:
MEDICINA
Ano de publicação:
2023
Tipo de documento:
Article
País de afiliação:
Estados Unidos