What Cellectis (CLLS) Said on Its Q3 Earnings Call

Key Points

• Cellectis expects its cash runway to extend into the second half of 2028 after restructuring, which includes cutting up to 70% of pre-transition headcount and ending two cell therapy programs.
• The company is shifting toward in vivo gene editing, with preliminary HEAL-101 trial data expected in the second half of 2027 and HEAL-201 data in the first half of 2028. Both programs have shown promising preclinical results, which do not establish how they will perform in patients.
• Nine-month revenue and other income fell to $19.9 million from $67.4 million a year earlier, while R&D expenses rose to $108.8 million, partly due to transformation-related costs.

Cellectis NASDAQ: CLLS said its cash runway is expected to extend into the second half of 2028 once its planned restructuring is fully implemented, giving the gene editing company time to reach anticipated initial clinical readouts for its two lead programs.

The company expects preliminary data from an investigator-initiated trial in China of HEAL-101, its candidate for severe hypertriglyceridemia, in the second half of 2027. Preliminary clinical data for HEAL-201, which targets severe hypercholesterolemia, are expected in the first half of 2028.

The timelines come as Cellectis shifts its focus to in vivo gene editing and exits its lasme-cel and eti-cel cell therapy programs. Chief Financial Officer and Chief Business Officer Arthur Stril said enrollment in those programs has stopped, site closeouts are underway and treated patients will continue to be monitored under the trial protocols.

HEAL-101 Trial Plans and Preclinical Data

Chief Executive Officer André Choulika said Cellectis is developing HEAL-101 as a single-injection treatment intended to provide a lasting reduction in triglycerides. The candidate uses a TALE-based editor to change a base in the APOC3 gene, creating a stop signal that prevents production of the APOC3 protein.

Chief Medical Officer Adrian Kilcoyne said a single intravenous dose produced mean APOC3 editing of approximately 65%, reaching as high as 70%, in a humanized liver mouse model. In a separate mouse model of hypertriglyceridemia, the treatment reduced APOC3 levels by 67% and triglycerides by 76%, he said. The results are preclinical and do not establish how the treatment will perform in patients.

Editing was detected in the liver, with no significant editing found in the other tissues analyzed, Kilcoyne said. He added that mouse and nonhuman primate data on liver toxicity had been reassuring.

During the question-and-answer session, Kilcoyne said Cellectis had selected trial sites in China but had not received agreement to disclose them. He said the company expects to begin dose escalation in the first quarter of 2027 and anticipates that the starting dose will show some efficacy, though it may not produce an optimal effect. If patients are recruited in the first quarter, the company could begin to assess roughly nine months of durability by year-end, he said.

Asked what degree of triglyceride reduction would support broader development, Kilcoyne said the company expects efficacy at least as good as current treatments. He also emphasized the proportion of patients reaching a triglyceride level below 500, rather than percentage reduction alone.

HEAL-201 and Market Strategy

HEAL-201 is designed to silence the PCSK9 gene without changing the DNA sequence. Choulika said the candidate reduced mean plasma PCSK9 levels by more than 90% in a humanized liver mouse model. In human liver cell lines, it achieved mean on-target epigenetic editing above 90%, with no off-site changes in gene or protein expression detected, he said.

Kilcoyne said Cellectis had not yet disclosed the LDL cholesterol reductions observed alongside PCSK9 suppression and expected to release those data in June. The company plans to advance HEAL-201 into a Phase 1 trial.

Kilcoyne said Cellectis chose APOC3 for HEAL-101 after market research indicated it was preferable to ANGPTL3 for specifically lowering triglycerides. He estimated the target population at about 1.1 million people across the United States and five major European markets, including the United Kingdom.

Restructuring and Nine-Month Results

Cellectis expects to reduce global headcount by up to 70% from its pre-transition level during 2026 and 2027, subject to completion of the applicable French Works Council consultation and compliance with French and U.S. labor laws, Stril said. Future spending is expected to focus on the gene editing pipeline and one-time restructuring costs.

Cash, cash equivalents, restricted cash and fixed-term deposits totaled $149 million at Sept. 30, down from $211 million at the end of 2025. The anticipated runway into the second half of 2028 depends on the realignment being fully implemented as planned.

For the nine months ended Sept. 30, consolidated revenues and other income fell to $19.9 million from $67.4 million a year earlier. Stril attributed the decline primarily to the level of work performed under research plans for the company’s AstraZeneca collaboration.

Research and development expenses rose to $108.8 million from $69.1 million, including $34.9 million in impacts from the strategic transformation. Those impacts included a $26.6 million impairment loss, $6.2 million in termination benefits and $2.1 million in costs associated with closing out the two cell therapy programs. Selling, general and administrative expenses rose to $17.5 million from $15 million.

Cellectis reported a consolidated net financial gain of $5.2 million for the nine-month period, compared with a net financial loss of $25.6 million a year earlier. Stril attributed the change in part to fair-value movements on warrants issued to the European Investment Bank and lower foreign-exchange losses.

About Cellectis (NASDAQ:CLLS)

Cellectis is a biotechnology company focused on developing gene-edited cell and gene therapies, with an emphasis on treatments for cancer. Founded in France in 1999, the company operates in France and the United States. Its research centers on TALEN gene-editing technology, which it uses to modify cells for potential therapeutic applications. Cellectis is developing allogeneic CAR T-cell therapies made from donor T cells, an approach intended to create treatments that can be prepared for use by multiple patients rather than made individually for each patient.

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