Things are progressing.
Herantis reports that “Phase 1b biomarker data shows a clear biological response to HER-096 in patients with Parkinson’s disease.”
Therefore, mechanistic evidence of the drug’s effect on the disease is being obtained, which will be important for future marketing authorizations. However, Herantis also states that the data will be submitted to scientific journals, which means the significance of the results now obtained will remain for the scientific community to assess through peer review. Those publications take several months.
If one aims for accelerated approval (AA), for example via a randomized Phase 2 trial, there should be strong evidence that a specific biomarker predicts clinical benefit, unless clinical benefit—i.e., symptom relief—has not yet been observed due to the slow-progressing nature of the disease. It may be that Herantis is only now building the scientific case for this and may be able to prove through various phases that this is indeed the case.
Although diseases and mechanisms cannot be fully compared, among neurological diseases, AA has been granted for tofersen in ALS based on a biomarker and similarly in Alzheimer’s based on plaque reduction; this has caused some controversy, and this AA pathway is currently uncertain.
From a partnering perspective, the data now obtained is encouraging, but we will see what it is sufficient for. Would biomarker evidence be enough for FDA/EMA Fast Track status after peer review, if the evidence proves to be strong?