The US Food and Drug Administration has accepted Arovella Therapeutics Ltd (ASX:ALA, FRA:E4NA)'s investigational new drug (IND) application for ALA-101, a step that allows the company to move into first-in-human clinical testing.
The market welcomed the news, with the company over 11% higher just before market close today.
The acceptance is a key regulatory and operational milestone and enables Arovella to begin a Phase 1 trial of ALA-101 in patients with CD19-positive non-Hodgkin’s lymphoma (NHL) and leukaemia.
Key points
- FDA accepts IND application for ALA-101
- Acceptance enables first-in-human Phase 1 trial and supports potential US trial sites
- IND package expected to provide a framework for future CAR-iNKT programs, including ALA-105
“The team has done an amazing job capturing all of the experimental data and related technical information required for a successful IND submission," Dr Michael Baker, managing director and CEO of Arovella Therapeutics, said.
Regulatory framework and validation
The IND provides the regulatory framework for Arovella's planned Phase 1 program and also supports the company’s intention to conduct its Phase 1 trial in Australia under the Clinical Trial Notification (CTN) scheme rather than the Clinical Trial Application (CTA) pathway.
IND acceptance enables Arovella to open clinical trial sites in the US. It is also regulatory validation of ALA-101’s preclinical package, manufacturing process and clinical development plan, which it considers a de-risking step ahead of dosing patients.
The company expects the IND submission and acceptance process for ALA-101 to be leveraged across its broader pipeline of CAR-iNKT cell therapy programs, including ALA-105, which is being developed for gastric cancer, as well as other solid tumour candidates.
"To have it accepted by the US FDA demonstrates the capability of our team and the company’s selected partners, including our manufacturer, Cell Therapies Pty Ltd. We have initiated the activities required for our Human Research Ethics Committee (HREC) submission and clinical trial site selection and activation. Now that the IND is accepted, we look forward to accelerating these activities and taking ALA-101 into the first-in-human phase 1 clinical trial. We are hopeful that ALA-101 will bring meaningful benefit to patients enrolled in the study who would otherwise have no remaining treatment options”
Lead allogeneic cell therapy product
ALA-101 is Arovella’s lead allogeneic cell therapy product and is derived from invariant natural killer T (iNKT) cells engineered to express a CD19-specific chimeric antigen receptor (CAR). The approach is intended to support an “off-the-shelf” product format, with manufacturing designed to be scalable and cost-efficient compared with patient-specific (autologous) approaches.
The planned Phase 1 study is expected to assess safety and tolerability, along with pharmacokinetics and early signals of anti-tumour activity, in patients with relapsed or refractory CD19-positive NHL and leukaemias.
Arovella also flagged the IND package as relevant to future development work, including additional solid tumour products and potential new technologies targeting neuroblastoma and hepatocellular carcinoma that it said are under negotiation for licence from Baylor College of Medicine.