Lakefront Biotherapeutics: 18 of 20 Evaluable Responders in ITP with Gamgertamig

Response Rates Rising with Dose, from 67% to 100% in ITP

Of the 33 patients treated, 22 had immune thrombocytopenia (ITP) and 11 had autoimmune hemolytic anemia (AIHA). All received a short subcutaneous administration, over three weeks at most, divided into three dose cohorts. Median disease duration was 8.4 years in ITP and 3.7 years in AIHA.

In ITP, 18 of the 20 evaluable patients responded, with platelet counts of at least 30 × 10⁹/L and at least a doubling compared with baseline. The response rate rises from 2 of 3 (67%) at the low dose to 4 of 5 (80%) at the intermediate dose and 12 of 12 (100%) at the high dose.

In addition, 17 of 18 responders (94%) normalized their platelet count. In AIHA, the evaluable patients, all drawn from the intermediate and high dose cohorts, all responded (6 of 6 and 3 of 3), with a hemoglobin increase of at least 2 g/dL.

The median time to response was 11 days in ITP and 22 days in AIHA. Eric Hedrick, Chief Medical Officer, recalled that the product's potential had been identified before the acquisition of Ouro by Gilead, "mainly on the basis of results from investigator-initiated trials."

The poster data come from trials sponsored by Lakefront and its Chinese partner Keymed. Gamgertamig, an investigational BCMAxCD3 bispecific antibody licensed from Keymed, which holds the rights in Greater China, has Orphan Drug and Fast Track designations from the FDA for certain autoimmune diseases. It is expected to enter registrational studies in 2027.

Some Loss of Response and No Neurotoxicity Syndrome Observed

These results remain preliminary: 20 of the 22 patients with ITP and 9 of the 11 patients with AIHA were evaluable for response. Median follow-up was 141 days in the first group (ranging from 29 to 253 days) and 85 days in the second (ranging from 57 to 198 days).

At the last follow-up, 15 of the 18 ITP responders (83%) and 8 of the 9 AIHA responders (89%) maintained their response, meaning that three ITP patients and one AIHA patient no longer maintained it.

Regarding tolerability, grade 1 cytokine release syndrome was observed in 6 of 33 patients (18%), with no grade 2 or higher cases and no ICANS-type neurotoxicity. Infections affected 10 of 33 patients (30%), mainly grade 1 or 2, all successfully managed according to the company.

The poster will be presented on December 13, 2026 by Phil Choi, with Eric Hedrick indicating he wishes to share "updated data." An investor conference call will follow on December 14 at 8:00 a.m. Eastern Time.