Sloan School of Management, MIT, Cambridge, USA.
Laboratory for Financial Engineering, MIT, Cambridge, USA.
Orphanet J Rare Dis. 2023 Sep 12;18(1):287. doi: 10.1186/s13023-023-02753-y.
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.
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.
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.
我们认为早期生物技术公司在制定可持续融资策略和商业模式方面面临两个关键挑战：开发药物化合物的估值模型，以及选择合适的运营模式和公司结构。我们以 Unravel Biosciences 为例——一家通过现有药物的脱靶机制识别新型药物靶点，然后开发优化的新分子的治疗平台公司——贯穿全文，并探讨了针对罕见遗传疾病的药物重定位的具体情况。
第一个挑战包括对潜在的罕见病重定位药物化合物进行现实的财务估值，在这种情况下，目标是雷特综合征。更一般地，我们开发了一个框架来评估早期开发中具有成对相关的罕见病化合物组合，并量化其风险状况。我们估计单个化合物的负回报概率为[公式：见正文]，8 种药物组合的概率为[公式：见正文]。单个化合物项目以亏损出售的概率从[公式：见正文]（第 3 阶段）下降到 8 种药物组合的[公式：见正文]（第 3 阶段）。对于第二个挑战，我们发现运营模式和公司结构的选择对早期生物技术创业公司至关重要，并通过三个具体示例说明了这一点。
重新利用现有化合物具有重要优势，可以帮助早期生物技术创业公司更好地将其业务和融资问题与科学和医学目标保持一致，进入大型制药公司尚未涉足的领域，并加速其药物开发平台的验证。
