What are the projected milestones and timeline for FDA approval and commercialization following this trial result?
Projected FDA & Commercialisation Timeline
Milestones after the RestorAATionâ2 update
| Milestone | Anticipated Timing | Rationale |
|---|---|---|
| Completion of the RestorAATionâ2 doseâescalation cohort (final safety and durability readâout) | Q4âŻ2024 â the trial is already reporting durable serum AAT levels after repeat 200âŻmg dosing; the next step is the prespecified safety followâup and the expansion into higherâdose cohorts. | |
| Submission of a BLA (Biologics License Application) to the FDA | Q2âŻ2025 â assuming the full doseârange data and the planned 6âmonth durability analysis meet the preâdefined efficacy thresholds, the company will have a complete data package for a âstandâaloneâ biologicâlicence filing. | |
| FDA review (standard 8âmonth cycle) + potential advisory committee | LateâŻ2025 â earlyâŻ2026 â a standard BLA review takes roughly 8âŻmonths; because WVEâ006 addresses an orphan indication (α1âantitrypsin deficiency) and the data are likely to be clearâcut, regulatory timing should follow the typical âpriorityâ path for rare diseases. | |
| Potential FDA approval (conditional or full) | MidâŻ2026 â if the agency accepts the submission without major queries, a conditional âaccelerated approvalâ could be granted by Q2âŻ2026, with a full approval by Q4âŻ2026 after confirmation of the durability and safety endpoints. | |
| Commercial launch (US & EU) | H2âŻ2026 â H1âŻ2027 â once the BLA is cleared, Wave will need ~3â6âŻmonths to scale up cGMP manufacturing, finalize reimbursement (e.g., US payer coverage, EU orphanâdrug incentives), and initiate sales/distribution. The pipelineâs focus on preventing both liver and lung disease gives it a differentiated positioning that can be leveraged for early market uptake. |
Trading Implications
- Shortâterm catalyst: The immediate market reaction to the durability readâout has already lifted the ticker (+~6% on the day of the release). Expect a priceâconsolidation phase over the next 4â6âŻweeks as investors priceâin the Q4âŻ2024 trialâcompletion milestone.
- Midâterm catalyst: A BLA filing announcement in earlyâ2025 will likely act as the next major catalyst, sparking a breakout if the submission underscores confidence in meeting the FDAâs efficacy thresholds.
- Risk: The timeline assumes no unexpected safety signals at higher doses or manufacturing setbacks; any adverse data could push the BLA filing back to lateâ2025 or beyond, which would keep the stock under pressure.
Actionable Takeâaway: Hold or lightly add on the stock ahead of the Q4âŻ2024 data finalisation, but consider a partial profitâtake/stopâloss around $6â7 (current levels) ahead of the Q2âŻ2025 BLA filing. If the filing proceeds without major FDA queries, a positionâbuilding phase from Q2âŻ2025 to midâ2026 aligns with a potential âapprovalâlinked upsideâ as the product moves toward commercialization.