By Fineline Cube 
Anhui Anke Biotechnology (Group) Co., Ltd. (SHE: 300009) announced that the China National Medical Products Administration (NMPA) has accepted the clinical trial application for an additional indication of its self-developed PA3-17 injection for pediatric and adolescent patients with relapsed/refractory T-cell acute lymphoblastic leukemia/lymphoblastic lymphoma (R/R T-ALL/LBL).
Regulatory Milestone & Drug Profile
| Attribute | Details |
|---|---|
| Company | Anhui Anke Biotechnology (Group) Co., Ltd. (300009.SZ) |
| Product | PA3-17 injection (CD7-targeted CAR-T cell therapy) |
| Developer | PersonGen Biotherapeutics (Anke Biotech holds a stake) |
| Application Type | Clinical trial application for new pediatric indication |
| New Indication | Pediatric and adolescent R/R T-ALL/LBL |
| Current Status | Pivotal Phase 2 trial ongoing for adult R/R T-ALL/LBL |
| Designation | Breakthrough Therapy Designation (CDE, Aug 2025) |
| Innovation | First-in-class autologous CD7 CAR-T cell therapy |
Technology & Mechanism of Action
PA3-17 is a CD7-targeted autologous CAR-T cell therapy:
- Target: CD7 antigen expressed on T-cell malignancies
- Approach: Engineered patient T-cells to recognize and eliminate CD7-positive tumor cells
- First-in-Class: First CD7 CAR-T therapy to receive Breakthrough Therapy Designation in China
- Manufacturing: Autologous process requiring patient-specific cell collection and engineering
Development Timeline & Strategic Expansion
| Milestone | Date | Scope |
|---|---|---|
| Breakthrough Therapy Designation | Aug 2025 | Adult R/R T-ALL/LBL |
| Adult Phase 2 Trial | Ongoing | R/R T-ALL/LBL pivotal study |
| Pediatric Application Acceptance | 08 Dec 2025 | Expands to patients aged <18 years |
| Strategic Goal | 2026 | Double addressable patient population |
Patient Population Expansion:
- Adults: ~1,500 new R/R T-ALL/LBL cases annually in China
- Pediatric/Adolescent: Additional ~800 cases annually
- Total Addressable Market: ~2,300 patients/year with potential CAR-T eligibility
Market Context & Competitive Landscape
| Competitive Asset | Company | Target | Status | Differentiation |
|---|---|---|---|---|
| PA3-17 | Anke Biotech/PersonGen | CD7 | Phase 2 (adult), IND (pediatric) | First CD7 CAR-T |
| GC012F | Gracell Bio | CD19/BCMA | Phase 1/2 (various) | Dual-target, but not CD7 |
| C-CAR039 | JW Therapeutics | CD19 | Approved (DLBCL) | Not for T-ALL |
Market Opportunity:
- T-ALL/LBL Treatment Gap: Limited options after relapse; 5-year survival <20% for R/R patients
- CAR‑T Efficacy: Early data shows >80% CR rate in heavily pre-treated adults
- Pediatric Unmet Need: No approved CAR-T therapies for pediatric T-cell malignancies globally
- Commercial Potential: Analysts project ¥1.5-2.0 billion peak sales (≈ US$210-280 million) if approved across both indications
Strategic Outlook & Next Steps
- Clinical Path: Pediatric trial design will mirror adult protocol with age-adjusted dosing
- Regulatory Timeline: NMPA review expected to take 6-9 months; Phase 1 pediatric initiation Q3 2026
- Manufacturing: Leverages PersonGen’s established autologous CAR-T production facility
- Global Ambition: Data may support future FDA Rare Pediatric Disease designation application
Forward‑Looking Statements
This brief contains forward‑looking statements regarding PA3-17 development timelines, clinical outcomes, and market potential. Actual results may differ due to clinical, regulatory, and competitive uncertainties.-Fineline Info & Tech