Phase 1 trial in re­lapsed and re­frac­tory mul­ti­ple myeloma con­tinues to en­roll patients

Gaithersburg, MD (Press Release) – Arcellx, a clin­i­cal-stage bio­pharma­ceu­tical com­pany devoted to devel­op­ing immune cell ther­a­pies for cancer and other dis­eases, to­day an­nounced that the U.S. Food and Drug Admin­istra­tion (FDA) has granted Orphan Drug Desig­na­tion to CART-ddBCMA, its engi­neered T cell ther­apy uti­liz­ing a novel binding domain, for the treat­ment of patients with mul­ti­ple myeloma. Arcellx con­tinues to en­roll patients in its Phase 1 trial of CART-ddBCMA ther­apy for the treat­ment of re­lapsed and re­frac­tory mul­ti­ple myeloma, the first in a series of clin­i­cal trials planned for the stepwise devel­op­ment of the Arcellx ARC-T + sparX cell ther­apy plat­form. This trial marks the first-in-human trial of the Arcellx binding domain, which the Com­pany plans to eval­u­ate in future ARC-T + sparX trials.

“We are pleased to be one of the first sites to test this new tech­nology in the clinic,” said Matthew Frigault, M.D., Assis­tant Director of the Cellular Therapy Service at Massachusetts General Hospital Cancer Center and Instructor at Harvard Medical School. “The novel deimmunized binding domain uti­lized in this trial may be poten­tially less immunogenic than the single chain variable fragment (scFv) or camelid binders used in con­ven­tional CAR T-cell ther­a­pies.”

“Orphan Desig­na­tion recog­nizes the unmet need of pop­u­la­tions with rare dis­eases like mul­ti­ple myeloma, par­tic­u­larly for those patients who have ex­hausted other thera­peutic op­tions,” stated Amy B. Fix, M.S., M.B.A., Senior Vice Pres­i­dent of Regulatory Affairs of Arcellx. “Receiving both Orphan and Fast Track Desig­na­tions fur­ther val­i­dates the poten­tial of Arcellx tech­nology and our novel binding domain. We hope to provide patients with a new ther­apy that may sig­nif­i­cantly im­prove treat­ment re­sponse for this debilitating dis­ease.”

The FDA’s Office of Orphan Drug Products grants orphan status to drugs in­tended to treat rare dis­eases affecting fewer than 200,000 people in the U.S. The desig­na­tion provides cer­tain benefits to drug de­vel­opers, in­clud­ing tax credits for clin­i­cal trial ex­pen­di­tures, waived user fees for mar­ket­ing appli­ca­tions, and eligibility for seven years of mar­ket­ing exclusivity.

About the Arcellx ddBCMA T Cell Therapy Phase 1 Trial

The open label Phase 1 trial is eval­u­ating an engi­neered T cell ther­apy that uses the Com­pany’s novel syn­thet­ic binding domain in the treat­ment of patients with re­lapsed and re­frac­tory mul­ti­ple myeloma. In the trial, a patient’s T cells are engi­neered to express a re­cep­tor targeting the B-cell maturation an­ti­gen (BCMA) on the tumor cell surface using the novel binding domain. The binding domain, which is a deimmunized syn­thet­ic pro­tein, is a key component of the Arcellx ARC-T + sparX cell ther­apy plat­form. The Arcellx ddBCMA cell ther­apy has been granted Fast Track Desig­na­tion and Orphan Drug Desig­na­tion by the U.S. Food and Drug Admin­istra­tion (FDA). The trial is cur­rently en­rolling patients. Additional in­for­ma­tion about the trial can be found at https://www.clinicaltrials.gov/ct2/show/NCT04155749.

About ARC-T + sparX Technology

Arcellx has devel­oped a pro­pri­e­tary plat­form in which ARC-T (Antigen Receptor Complex T cells) are con­trolled by the admin­istra­tion of a tumor-targeting pro­tein called a sparX (Soluble Protein Antigen-Receptor X-linker). A library of deimmunized sparX pro­teins that recog­nize dif­fer­en­t cell surface an­ti­gens are functional as monovalent, bivalent, or bispecific con­structs, and could poten­tially be admin­istered simultaneously or sequentially to address the in­her­ent heterogeneity of dis­eases such as cancer. The ARC-T cells can be readily silenced, activated and reprogrammed by sparX, allow­ing dose con­trol to minimize toxicities and mul­ti­ple an­ti­gen targeting to im­prove ef­fi­cacy and address relapse. The ARC-T + sparX thera­peutic plat­form is de­signed to poten­tially provide en­hanced ef­fi­cacy, safety, patient accessibility and efficiency of manu­fac­tur­ing rel­a­tive­ to existing cell ther­a­pies.

About Multiple Myeloma

Multiple myeloma is a blood cancer that may cause can­cer­ous tumors in bone and soft tissue. It is char­ac­ter­ized by ab­nor­mal plasma cells called myeloma cells. Plasma cells de­vel­op from B lym­pho­cytes (B cells), a type of white blood cell that is made in the bone mar­row. When myeloma cells collect in mul­ti­ple bones, the dis­ease is called mul­ti­ple myeloma. The National Cancer Institute esti­mates that in the United States, there were 32,110 new cases of myeloma in 2019 and 131,392 people living with the dis­ease in 2016.¹ The current standard of care in­cludes chemo­ther­apy, targeted ther­apy, high-dose chemo­ther­apy with stem cell trans­plant, bio­logic ther­apy, radi­a­tion ther­apy and surgery.

About Arcellx, Inc.

Arcellx is a clin­i­cal-stage bio­pharma­ceu­tical com­pany devoted to providing patients with superior immune cell ther­a­pies through scientific inno­va­tion and ac­cel­er­ated devel­op­ment of next-gener­a­tion tech­nology. Arcellx initially is devel­op­ing the ARC-T cell ther­a­pies for cancer in­di­ca­tions, and in the future, broader in­di­ca­tions, in­clud­ing auto­immune dis­ease. More in­for­ma­tion can be found at www.arcellx.com.

Reference

1. National Cancer Institute. Surveillance, Epidemiology, and End Results (SEER) Program. Cancer Stat Facts: Myeloma. Available at https://seer.cancer.gov/statfacts/html/mulmy.html. Accessed March 12, 2020.

Source: Arcellx.