GeneScience’s GenSci155 Wins NMPA Clinical Trial Approval – Long‑Acting IGF‑1 Analog Targets Bronchopulmonary Dysplasia and Rare Growth Disorder

Changchun High & New Technology Industries (Group) Inc. (SHE: 000661) announced that its subsidiary, GeneScience Pharmaceuticals Co., Ltd., has received clinical trial approval from China’s National Medical Products Administration (NMPA) for GenSci155 Injection, a long‑acting fatty acid‑conjugated recombinant human IGF‑1 analog, for the prevention of bronchopulmonary dysplasia (BPD) in preterm infants and the treatment of primary insulin‑like growth factor‑1 deficiency (PIGFD) — two indications with no approved therapies or significant unmet needs globally.

Regulatory Milestone

Drug Profile & Mechanism of Action

• Molecule: GenSci155, a long‑acting fatty acid‑conjugated recombinant human insulin‑like growth factor‑1 (IGF‑1) analog, independently developed by GeneScience Pharmaceuticals.
• Platform: Built on GeneScience’s proprietary Duratide™ long‑acting sustained‑release peptide platform.
• Mechanism: Fatty acid chain modification enables reversible binding to serum albumin, extending the drug’s in‑vivo half‑life and providing sustained pharmacological activity with reduced side effects (notably lower hypoglycemia risk).
• Dosing Innovation: Preclinical data suggest potential for once‑every‑2‑to‑3‑day dosing, a substantial improvement over the current standard of twice‑daily injections for IGF‑1 replacement therapy.
• Dual Administration Design: GenSci155 is innovatively engineered for both intravenous (IV) infusion and subcutaneous (SC) injection, enabling indication‑specific clinical benefit:
  • BPD: Early IV infusion aligns with existing NICU venous access, ensuring stable drug exposure during the critical pulmonary developmental window; later switch to SC injection reduces invasive procedures and catheter‑related complications.
  • PIGFD: Long‑acting SC administration avoids the large pharmacokinetic fluctuations associated with short‑acting IGF‑1, improving safety and potential height outcomes.

Indication 1: Bronchopulmonary Dysplasia (BPD)

• Disease: BPD is the most common pulmonary complication in preterm infants, with incidence rising sharply as gestational age decreases. It causes persistent impact on respiratory function and long‑term health.
• Epidemiology: Approximately 25,000 high‑risk neonates (gestational age <28 weeks) are born annually in each of China and the United States.
• Unmet Need: No drug is currently approved globally for the prevention of BPD in preterm infants.
• Prior Approach Limitations: Short‑acting IGF‑1 (OHB‑607, OPKO Health) showed early clinical promise but required continuous IV infusion, limiting clinical feasibility and resulting in insufficient drug exposure that compromised efficacy.

Indication 2: Primary IGF‑1 Deficiency (PIGFD)

• Disease: PIGFD is a rare genetic growth disorder caused by hereditary defects in the growth hormone–IGF‑1 (GH‑IGF‑1) axis, characterized by impaired height, significantly low IGF‑1 levels, and normal or elevated growth hormone secretion. It spans a continuous disease spectrum from mild to severe (sPIGFD).
• Epidemiology: A global rare disease with an estimated ~1,000 patients in the United States; EU data indicate an overall prevalence of <2 per 10,000.
• Unmet Need: Traditional growth hormone therapy is ineffective for PIGFD. Globally, only short‑acting recombinant human IGF‑1 (mecasermin, administered twice daily by SC injection) is approved for severe patients.
• Current Therapy Limitations:
  • Large blood concentration fluctuations impair pharmacokinetic profiles
  • Fluctuating levels limit dose escalation, constraining potential height improvement
  • High incidence of hypoglycemia
  • High injection frequency undermines long‑term treatment adherence

Clinical Development Status

• Current Approval (October 2026): NMPA clinical trial clearance for GenSci155 (SC injection) in BPD prevention and PIGFD treatment.
• Prior Approvals: GenSci155 (IV injection) has previously received NMPA clinical trial approval for BPD prevention and acute ischemic stroke (AIS).
• Development Stage: Clinical trial initiation; specific trial design parameters not yet disclosed.

Market Impact & Outlook

• BPD – A Blue‑Ocean Opportunity: With no approved pharmacotherapy globally for BPD prevention, GenSci155 enters a whitespace market affecting ~50,000 high‑risk neonates annually across China and the U.S. alone. A viable long‑acting IGF‑1 with flexible IV‑to‑SC administration could become a practice‑changing therapy in neonatal intensive care.
• PIGFD – Addressing Fundamental Pharmacokinetic Flaws: GenSci155’s long‑acting profile directly addresses the core limitations of twice‑daily mecasermin — smoothing concentration peaks (reducing hypoglycemia), enabling dose optimization (improving height outcomes), and cutting injection frequency (boosting adherence). This positions GenSci155 as a potential best‑in‑class replacement for the current standard of care.
• Duratide™ Platform Value: The Duratide™ sustained‑release technology is a reusable platform asset with potential application across GeneScience’s broader peptide and protein pipeline, amplifying the strategic significance of GenSci155 beyond a single product.
• Pipeline Depth: With clinical programs now spanning BPD (IV + SC), AIS (IV), and PIGFD (SC), GenSci155 is emerging as a multi‑indication asset with both rare disease and high‑volume neonatal applications.
• Commercial Upside for GeneScience: As a Class 1 biological product with full proprietary IP, GenSci155 strengthens GeneScience’s position in pediatric endocrinology and neonatology — complementary to its established growth hormone franchise.

Forward‑Looking Statements
This brief contains forward‑looking statements regarding regulatory timelines, clinical development plans, and commercial expectations for GenSci155. Actual results may differ materially due to risks including clinical trial outcomes, dose‑finding results, regulatory review timelines across multiple indications, manufacturing scale‑up, competitive developments, and the inherent uncertainties of biological drug development. Investors are advised to exercise caution.-Fineline Info & Tech