China’s investigator-initiated trials, once celebrated for accelerating the country’s biotech boom, are now facing serious international scrutiny over transparency failures.
The controversy began after several previously undisclosed deaths were uncovered within weeks, starting with two young children enrolled in trials for rare genetic conditions.
China’s investigator-initiated trials, known as IITs, allow hospitals and academics to launch early-stage human trials under a regulatory pathway with significantly less oversight than traditional drug approval routes.
Experts consulted by Fierce Pharma raised concerns about what the patient deaths and poor public disclosure could mean for the long-term credibility of the IIT system.
A third death linked to China’s popular but opaque trial pathway has since emerged, this time connected to a CAR-T therapy, deepening questions about the regulatory framework.
Meanwhile, AstraZeneca (AZ) and Daiichi Sankyo’s antibody-drug conjugate Enhertu delivered notable phase 3 results in non-small cell lung cancer patients, beating Keytruda in first-line HER2-mutant nonsquamous NSCLC.
The Huchmed-partnered Orpathys-Tagrisso combination also outperformed platinum-based chemotherapy in Tagrisso-pretreated EGFR-mutated disease, adding another win to AstraZeneca’s lung cancer portfolio.
However, those victories were partially overshadowed by a phase 3 failure from investigational bispecific volrustomig, which did not outperform Keytruda and chemotherapy in first-line NSCLC patients with PD-L1 expression below 50%.
Biokin Pharma’s first-in-class bispecific antibody-drug conjugate iza-bren, which targets both EGFR and HER3, met its primary endpoint in an interim phase 3 analysis, demonstrating significant progression-free survival benefits.
That Chinese trial success strengthens the outlook for a global study launching this autumn, which is being led by global partner Bristol Myers Squibb (BMY).
In dermatology, Tanabe Pharma has secured $435 million in upfront and near-term milestone payments through a licensing agreement with Leo Pharma covering the rare skin disease treatment dersimelagon.
Leo Pharma receives global rights to dersimelagon, a therapy targeting the rare genetic dermatology conditions erythropoietic protoporphyria and X-linked protoporphyria, with an FDA approval decision now pending.
Tanabe had already announced a phase 3 win for dersimelagon across both indications earlier in 2026, making the Leo deal a timely move ahead of a potential US regulatory green light.
Sandoz has also moved to expand its biosimilars presence in Asia, entering a collaboration with Shanghai Henlius Biotechnology and paying $100.5 million upfront for rights to three early-stage biosimilar assets.
The deal, which could ultimately cover as many as 10 biosimilars, carries total potential payments of up to $322 million should all development milestones be reached, reflecting sustained investor appetite for the biosimilars sector.