Anixa Biosciences, Inc. (NASDAQ: ANIX) provided an update on the progress and patient outcomes within its ongoing Phase 1 clinical trial investigating liraltagene autoleucel (lira-cel), a novel CAR-T cell therapy designed to combat ovarian cancer. This disclosure coincides with recent regulatory approval of a protocol amendment enabling a substantial increase in the administered dose of the therapy.
The investigational treatment lira-cel is engineered to target the follicle-stimulating hormone receptor (FSHR), a cell surface protein selectively expressed on ovarian tissue cells, tumor-associated blood vessels, and certain cancerous cells, while notably absent from healthy tissue. This targeted approach aims to minimize off-target effects while maximizing therapeutic impact.
The Phase 1 clinical study enrolls adult female patients diagnosed with recurrent ovarian cancer who have exhausted standard-of-care chemotherapy options and exhibit disease progression following administration of two or more prior lines of therapy. The primary intent of the trial is to assess safety, dosing tolerability, and preliminary efficacy signals.
As of the latest data cut, a total of twelve patients have undergone treatment at four escalating dose levels within this study. Remarkably, seven of these individuals have exceeded their anticipated median survival duration, which is typically estimated at approximately three to four months for patients of similar disease severity and treatment history.
Detailed survival outcomes highlight one patient who has survived for 28 months post-treatment. In addition, three patients have demonstrated survival durations surpassing one year, with recorded periods of 17, 15, and 14 months respectively. Further, three other patients have remained alive for 11, 8, and 8 months post-therapy.
Among this subset, the patients with survival lengths of 15, 14, and 8 months continue to be alive at present, alongside a more recently treated participant who is also currently living. These data points contribute to an encouraging trend in extended survival following treatment with lira-cel.
Critically, the trial has not reported any dose-limiting toxicities (DLTs) thus far. In response, Anixa’s clinical partner, Moffitt Cancer Center, obtained necessary Institutional Review Board (IRB) clearance to amend the study protocol, allowing for a marked increase in dose levels. If the escalated dosage is tolerated without DLTs, the principal investigator may authorize further dose escalation in line with trial design.
The safety profile demonstrated to date is thought to be enhanced by the method of drug delivery. Unlike traditional CAR-T therapies administered intravenously, lira-cel is delivered directly into the peritoneal cavity (intra-peritoneally). This localized administration likely reduces systemic toxicity while concentrating therapeutic effects on tumor sites within the abdominal compartment.
Under the amended clinical protocol, dosing may be increased from an initial range between 1×10⁵ to 1×10⁷ CAR-positive cells per kilogram of patient body weight, up to as high as 1×10⁹ cells per kilogram. This represents a two-orders-of-magnitude increase in administered cell numbers.
The upcoming patient cohort under the revised dosing regimen will receive 1×10⁷ cells/kg following pre-treatment lymphodepletion using cyclophosphamide and fludarabine. Lymphodepletion serves to reduce native immune cell populations that could compete with or hinder the expansion and persistence of the infused CAR-T cells.
While the application of lymphodepletion in CAR-T therapies is established in hematologic malignancies, its utility and impact in the context of solid tumors such as ovarian cancer remain investigational. The current trial is positioned to evaluate this within its study design.
Market reaction to these developments has been moderately positive, with Anixa Biosciences shares trading slightly higher by 1.69% at $3.01 in premarket activity, reflecting investor interest in the evolving therapeutic potential indicated by these clinical updates.