Lenzumestrocel (Neuronata-R)

CorestemChemon is a South Korean biotech listed on the KOSDAQ exchange. Neuronata-R is its main product. Its share price fell by about 30% when the Phase 3 results were announced in December 2024. In December 2025 it said it had raised KRW 26.1 billion to support commercialisation and its US plans.

Doctors collect a sample of the patient's own bone marrow, grow the stem cells in a lab, and inject them into the spinal fluid. The cells are thought to release substances that calm harmful inflammation and support motor neurons rather than replace them. In the Phase 3 trial, treated patients had lower levels of NfL, a marker of nerve damage, and of MCP-1, an inflammation marker.

South Korea gave Neuronata-R conditional approval in July 2014, and more than 400 patients have been treated there. On May 26, 2026 the Korean regulator (MFDS) kept the approval, on condition that a confirmatory trial is run. It is not approved in the USA or Europe, where it has orphan drug designation. At an FDA meeting in October 2026, the FDA called a new placebo-controlled Phase 3b trial reasonable, but asked for more manufacturing data.

ALSUMMIT compared two or five injections with placebo in 123 people in Korea. It missed its main goal, a combined score of function and survival (p=0.118 at 12 months). In a post-hoc group of slower-progressing patients, the company reports higher ALSFRS-R scores at 12 months (31.2 vs 26.4) and better breathing on five injections. Because this group was chosen after the results were known, it needs to be confirmed in a new trial. The earlier Phase 2 trial (64 people) that supported the Korean approval reported 3.4 points less decline at six months, but the comparison group had no sham procedure and survival did not differ.

This is the most favourable result reported. See all key studies for the full picture.

ALSUMMIT enrolled adults aged 25 to 75 who had been diagnosed within the past two years, at five Korean hospitals. The planned US Phase 3b trial would focus on sporadic ALS and use a blood NfL level of 75 pg/mL as a cut-off, aiming to pick people the company thinks are most likely to respond.

In July 2026 CorestemChemon said it now plans to file a US approval application (BLA) next year, after earlier targets of late 2025 and then 2026 passed. After the October 2026 FDA meeting, the company still needs to supply more manufacturing data and support for its NfL cut-off, and the faster accelerated-approval route has not been agreed.

Production has been paused since 2025 while manufacturing moves to a new plant in Osong. The company says full production and treatment will resume early in 2027, and that patients from other countries can then travel to Korea for treatment. No trial is currently recruiting.

• ALSUMMIT (Phase 3) (NCT04745299) completed · South Korea