New York, NY (Press Release) – Cellectis (Euronext Growth: ALCLS - Nasdaq: CLLS), a bio­pharma­ceu­tical com­pany focused on devel­op­ing immuno­therapies based on gene-edited allo­geneic CAR T-cells (UCART), to­day an­nounced that the MELANI-01 trial has been placed on clin­i­cal hold by the U.S. Food and Drug Admin­istra­tion (FDA).

This clin­i­cal hold which im­pacts one of the three Cellectis prod­uct can­di­dates cur­rently in clin­i­cal stud­ies, was ini­ti­ated fol­low­ing the sub­mission of a safety report re­gard­ing one patient en­rolled in the MELANI-01 study at dose level two (DL2), with re­lapsed and re­frac­tory mul­ti­ple myeloma. This patient, who had been treated un­suc­cess­fully, prior to en­roll­ment with nu­mer­ous lines of prior ther­apy, in­clud­ing au­tol­o­gous CAR T-cells, ex­peri­enced a fatal treat­ment-emergent adverse event of cardiac arrest. Clinical evaluation of the case remains on­go­ing and addi­tional details as to the im­medi­ate and under­lying causes of this event are being collected.

Of note, prior to the clin­i­cal hold being issued by the FDA, Cellectis had decided to ex­pand en­roll­ment at DL1, which may be the appro­pri­ate dose for fur­ther evaluation in the ex­pan­sion portion of the trial and po­ten­tially the rec­om­mended Phase 2 dose based on an assess­ment of the pre­lim­i­nary clin­i­cal and trans­la­tional data. The Com­pany had begun executing up­dates to the clin­i­cal protocol to reflect this as well as to monitor and mitigate for addi­tional po­ten­tial risks given its novel mech­a­nism of action.

“We share the FDA’s com­mitment to patient safety and are work­ing col­lab­o­ratively with the agency and the in­ves­ti­ga­tors to re­solve this clin­i­cal hold,” said Carrie Brownstein, MD, Chief Medical Officer, Cellectis. “The safety of patients en­rolled in our clin­i­cal trials is our utmost priority and we at Cellectis remain com­mit­ted to safely resuming the clin­i­cal de­vel­op­ment of UCART prod­uct can­di­date targeting CS1 for patients with mul­ti­ple myeloma and unmet med­i­cal need.”

Cellectis is work­ing closely with the FDA to address the agency’s re­quests in­clud­ing changes to the MELANI-01 clin­i­cal protocol de­signed to en­hance patient safety, and ex­pect to submit re­quested in­for­ma­tion in­clud­ing an amended protocol in due course.

Patient en­roll­ment is on­go­ing in our two other pro­pri­e­tary Phase 1 dose escalation trials: AMELI-01 eval­u­ating UCART123 in re­lapsed and re­frac­tory acute myeloid leukemia and BALLI-01 eval­u­ating UCART22 in re­lapsed and re­frac­tory B-cell acute lymphoblastic leukemia.

About MELANI-01

MELANI-01 is a Phase 1 open-label First-In-Human dose escalation clin­i­cal study eval­u­ating UCARTCS1A prod­uct can­di­date for the treat­ment of patients with re­lapsed or re­frac­tory mul­ti­ple myeloma (MM). UCARTCS1A is an allo­geneic, off-the-shelf, gene-edited T-cell prod­uct can­di­date de­signed for the treat­ment of CS1/SLAMF7-expressing hema­to­logic malig­nan­cies. CS1 (SLAMF7) is highly ex­pressed on MM tumor cells.

About Multiple Myeloma (MM)

Multiple myeloma is a cancer that affects a type of white blood cells called plasma cells that are spe­cial­ized mature B-cells, which secrete anti­bodies to combat in­fec­tions. Multiple myeloma is char­ac­ter­ized by the un­con­trolled pro­lif­er­a­tion of neoplastic plasma cells in the bone mar­row, where they over­crowd healthy blood cells. Although MM is a chronic dis­ease and an exact cause has not yet been identified, re­searchers have made sig­nif­i­cant progress over the years in managing the dis­ease through better under­stand­ing MM’s pathophysiology. The progress in finding a cure needs to be con­tinued as The American Cancer Society esti­mates that 32,110 new cases of MM will be diag­nosed, and 12,960 deaths are ex­pected to oc­cur in 2019 in the U.S. alone.

About Cellectis

Cellectis is devel­op­ing the first of its kind allo­geneic ap­proach for CAR-T immuno­therapies in on­col­ogy, pioneering the concept of off-the-shelf and ready-to-use gene-edited CAR T-cells to treat cancer patients. As a clin­i­cal-stage bio­pharma­ceu­tical com­pany with over 20 years of ex­per­tise in gene edit­ing, Cellectis is devel­op­ing life-changing prod­uct can­di­dates uti­liz­ing TALEN®, its gene edit­ing tech­nology, and PulseAgile, its pioneering electroporation sys­tem to har­ness the power of the im­mune sys­tem in order to target and eradicate cancer cells.

As part of its com­mitment to a cure, Cellectis remains ded­i­cated to its goal of providing life-saving UCART prod­uct can­di­dates to address unmet needs for mul­ti­ple cancers in­clud­ing acute myeloid leukemia (AML), B-cell acute lymphoblastic leukemia (B-ALL) and mul­ti­ple myeloma (MM).

Cellectis headquarters are in Paris, France, with addi­tional locations in New York, New York and Raleigh, North Carolina. Cellectis is listed on the Nasdaq Global Market (ticker: CLLS) and on Euro­next Growth (ticker: ALCLS). For more in­for­ma­tion, visit www.cellectis.com.

Follow Cellectis on social media: @cellectis, LinkedIn and YouTube.

TALEN® is a registered trademark owned by Cellectis.

Disclaimer

This press re­lease con­tains “forward-looking” state­ments within the meaning of appli­cable se­cu­ri­ties laws, in­clud­ing the Private Se­cu­ri­ties Lit­i­ga­tion Reform Act of 1995. These for­ward-looking state­ments in­clude state­ments re­gard­ing Cellectis’ re­sponses to the FDA re­quests, the timing of clin­i­cal protocol amendments and the resolution of the FDA clin­i­cal hold. These for­ward-looking state­ments are based on man­age­ment’s cur­rent ex­pec­ta­tions and assump­tions and on in­for­ma­tion cur­rently avail­able to man­age­ment. Forward-looking state­ments in­volve known and unknown risks, un­cer­tainties and other factors (including, without lim­i­ta­tion, the re­­sults of the on­go­ing clin­i­cal evaluation) that may cause our actual re­­sults, per­for­mance or achieve­ments to be ma­teri­ally dif­fer­en­t from any future re­­sults, per­for­mance or achieve­ments ex­pressed or im­plied by the for­ward-looking state­ments. Fur­ther in­for­ma­tion on the risk factors that may affect com­pany business and fi­nan­cial per­for­mance is in­cluded in Cellectis’ Annual Report on Form 20-F and the fi­nan­cial report (including the man­age­ment report) for the year ended De­cem­ber 31, 2019 and sub­se­quent filings Cellectis makes with the Se­cu­ri­ties Ex­change Com­mis­sion from time to time. Except as re­quired by law, we assume no obli­ga­tion to up­date these for­ward-looking state­ments pub­licly, or to up­date the reasons why actual re­­sults could differ ma­teri­ally from those antic­i­pated in the for­ward-looking state­ments, even if new in­for­ma­tion be­comes avail­able in the future.

Source: Cellectis.