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New medicines #47 - April 3, 2021 - April 9, 2021
A weekly overview of the development of new medicines.
- Acadia Pharmaceuticals announced that the FDA issued a Complete Response Letter (CRL) for its supplemental New Drug Application (sNDA) for Nuplazid (pimavanserin) for the treatment of hallucinations and delusions associated with dementia-related psychosis (DRP):
The CRL cited a lack of statistical significance in some of the subgroups of dementia, and insufficient numbers of patients with certain less common dementia subtypes as lack of substantial evidence of effectiveness to support approval
The company noted that the FDA had agreed to the trial design and the FDA did not raise any concerns regarding the agreed upon study design, including the issues raised in the CRL
- Celcuity announced initial data from its phase 1b open label trial evaluating gedatolisib and Ibrance (Palbociclib) together with Letrozole or Fulvestrant, in ER+/HER2- advanced or metastatic breast cancer patients:
In the trial, 53 of the 88 evaluable patients (60%) exhibited an objective response
- FibroGen released a disclosure regarding its anemia treatment, roxadustat, in chronic kidney disease patients:
The company noted that as it was preparing for its upcoming Advisory Committee meeting for its New Drug Application (NDA), it became aware it submitted altered data (post-hoc changes) in its NDA filing relating to MACE (Major Adverse Cardiovascular Events) safety data
The company now notes that it cannot conclude that roxadustat reduces the risk of (or is superior to) MACE+ in dialysis, and MACE and MACE+ in incident dialysis compared to epoetin-alfa
- Provention Bio announced that the FDA has identified deficiencies with its Biologic License Application (BLA) for teplizumab for the delay or prevention of clinical Type 1 diabetes:
Provention noted the FDA evaluated data from its pharmacokinetic/pharmacodynamic (PK/PD) bridging study evaluating two drugs, one drug substance it intends to use for commercialization, and drug product from historic drug substance used for its pivotal Phase 2 trial
The FDA noted the PK profiles of the two drug products were not comparable and that additional data would be required
The Advisory Committee meeting to review the BLA scheduled for May 27, 2021 will still be held
Source: BPC (reformatted), SY, Bioworld