Most Orphan drug companies acquired at Phase 3 or latter
Posted: Sun Dec 03, 2017 5:32 pm
The below article observes that companies developing orphan drugs are often not bought-out until Phase 3 or after, in contrast to non-orphan drug companies that are often purchased at phase 2. This article concerns privately financed orphan drug companies but logic dictates this conclusion would also apply to public orphan drug companies since the reasons for the late buyout would be the same. In other words it is not unusual that JNJ has yet to make a buyout offer to GERN. Note also the last paragraph:
20 February 2014
Most Orphan drug companies acquired at Phase 3 or latter:
“Companies without an ODN on their lead compound had a median of eight years from founding to buyout—the same time to acquisition for companies with an orphan focus. However, the majority of non-orphan biotech companies gets acquired at phase 2, whereas orphan drug companies tend to get acquired at a later stage (Fig. 2b); more than 62% of the companies with an ODN on their lead were acquired at phase 3 or later compared with less than 33% of non-orphan–based companies…
Therefore, although our data show that orphan drug development is indeed faster, it does not mean a shorter time to acquisition. This might be caused by acquirers needing more validation before betting on a compound with a perceived limited market potential and possible difficulties related to patient recruitment. In addition, buyers may need more clinical data to embrace a new method or platform, especially as phase 2 trials for rare diseases often involve a limited number of patients…
Orphan drugs may be acquired & developed for broader indications:
Often, after acquiring an ODN, a pharmaceutical company will develop that same drug for a broader indication. Orphan-designated drugs such as erythropoietin and rituximab initially benefited from the ODA incentives and subsequently became blockbusters through label extension. Pharmaceutical companies—Novartis in particular—have now fully adopted this approach4. This holds especially true in cancer, in which the same molecular pathways often are deregulated in both rare and common cancers…”
http://www.nature.com/bioent/2014/14020 ... 014.1.html
20 February 2014
Most Orphan drug companies acquired at Phase 3 or latter:
“Companies without an ODN on their lead compound had a median of eight years from founding to buyout—the same time to acquisition for companies with an orphan focus. However, the majority of non-orphan biotech companies gets acquired at phase 2, whereas orphan drug companies tend to get acquired at a later stage (Fig. 2b); more than 62% of the companies with an ODN on their lead were acquired at phase 3 or later compared with less than 33% of non-orphan–based companies…
Therefore, although our data show that orphan drug development is indeed faster, it does not mean a shorter time to acquisition. This might be caused by acquirers needing more validation before betting on a compound with a perceived limited market potential and possible difficulties related to patient recruitment. In addition, buyers may need more clinical data to embrace a new method or platform, especially as phase 2 trials for rare diseases often involve a limited number of patients…
Orphan drugs may be acquired & developed for broader indications:
Often, after acquiring an ODN, a pharmaceutical company will develop that same drug for a broader indication. Orphan-designated drugs such as erythropoietin and rituximab initially benefited from the ODA incentives and subsequently became blockbusters through label extension. Pharmaceutical companies—Novartis in particular—have now fully adopted this approach4. This holds especially true in cancer, in which the same molecular pathways often are deregulated in both rare and common cancers…”
http://www.nature.com/bioent/2014/14020 ... 014.1.html