CD38-Targeting Molecule To Be Evaluated In Multiple Patient Populations

New Haven, CT (Press Release) – Kleo Pharma­ceu­ticals, a targeted immuno­therapy com­pany devel­op­ing fully syn­thet­ic bispecific ther­a­pies to redirect, en­hance or re­place anti­bodies, an­nounced to­day that its lead com­pound, KP1237, has re­ceived Orphan Drug desig­na­tion from the U.S. Food and Drug Admin­istra­tion (FDA) in mul­ti­ple myeloma. KP1237 is an anti­body-redirecting mol­e­cule (ARM) that targets CD38, a clin­i­cally val­i­dated target for mul­ti­ple myeloma.

“We are pleased to re­ceive this Orphan Drug desig­na­tion for KP1237, which is ad­vanc­ing to Phase 1 clin­i­cal testing in the fourth quarter of 2020,” said Doug Manion, MD, CEO of Kleo Pharma­ceu­ticals. “Based on compelling pre­clin­i­cal data, we will eval­u­ate KP1237 in po­ten­tially three dif­fer­en­t clin­i­cal pro­grams where KP1237 can have the greatest im­pact in patients whose op­tions may be severely lim­ited.”

Orphan Drug desig­na­tion qualifies KP1237 for a num­ber of de­vel­op­ment incentives, in­clud­ing tax credits for cer­tain clin­i­cal costs, a waiver of the new drug appli­ca­tion fee and a designated period of mar­ket exclusivity fol­low­ing ap­prov­al.

Kleo is eval­u­ating KP1237 both as a sys­temic ther­apy for Dar­za­lex®-relapsed/refractory mul­ti­ple myeloma patients, and in com­bi­na­tion with au­tol­o­gous (the patient’s own) nat­u­ral killer (NK) cells in newly diag­nosed mul­ti­ple myeloma patients who are min­i­mum residual dis­ease-positive (MRD+) post-autologous stem cell trans­plant. Clinical trials to sup­port both these pro­grams are planned to begin in the fourth quarter of 2020. A third clin­i­cal pro­gram in mul­ti­ple myeloma using KP1237 in com­bi­na­tion with allo­geneic (off-the-shelf) NK cells is also under de­vel­op­ment.

In addi­tion to mul­ti­ple myeloma, Kleo has an active COVID-19 pro­gram, whose first clin­i­cal can­di­date is ex­pected to enter clin­i­cal testing in early 2021.

About Anti­body-Redirecting Molecules (ARMs)

Kleo’s bispecific, anti­body-redirecting mol­e­cules (ARMs) are engi­neered with mod­u­lar components that are readily in­ter­change­able, giving the plat­form tre­men­dous flexibility and rapid de­vel­op­ment times. ARMs are smaller than bio­logic ther­a­pies, which allows for en­hanced tumor / tissue pen­e­tra­tion, re­duced immuno­gen­icity, admin­istra­tion of higher dose levels with im­proved safety profiles, and the po­ten­tial for oral bio­avail­abil­ity. The ARM plat­form is also being used to redirect NK cell-based ther­a­pies to dis­ease targets without chi­meric engi­neer­ing.

About Multiple Myeloma

Multiple myeloma is a type of blood can­cer that affects plasma cells. In mul­ti­ple myeloma, malignant plasma cells ac­cu­mu­late in bone mar­row, the soft, spongy tissue at the center of a person’s bones, and crowd out nor­mal plasma cells that help fight in­fec­tion. The American Cancer Society esti­mates that approx­i­mately 32,270 new cases will be diag­nosed, and 12,830 deaths will oc­cur in 2020 from mul­ti­ple myeloma.

About Kleo Pharma­ceu­ticals, Inc.

Kleo Pharma­ceu­ticals is a targeted immuno­therapy com­pany that de­vel­ops fully syn­thet­ic bispecific ther­a­pies to redirect, en­hance or re­place anti­bodies. The com­pany was founded on the ground­break­ing re­search of its sci­en­tif­ic founder Dr. David Spiegel at Yale Uni­ver­sity. Kleo’s syn­thet­ic immuno­therapy plat­form uses two chemistry-based ap­proaches – anti­body-redirecting mol­e­cule (ARM) and multitargeted anti­body ther­apy en­hancer (MATE) - that help redirect and stim­u­late key components of the im­mune sys­tem to eradicate can­cer cells and virulent path­o­gens. Kleo’s com­pounds are smaller and more versatile than bio­logic ther­a­pies, which allows for en­hanced tumor/tissue pen­e­tra­tion, re­duced immuno­gen­icity, admin­istra­tion of higher dose levels with im­proved safety profiles, more ef­fi­cient pro­duc­tion and the po­ten­tial for oral bio­avail­abil­ity. They can be op­ti­mized against specified bio­logical targets or com­bined with existing cell- or anti­body-based ther­a­pies.

Kleo’s lead pro­gram, KP1237, is entering Phase 1 clin­i­cal stud­ies in 2020 in mul­ti­ple myeloma in mul­ti­ple settings. The com­pany’s sec­ond pro­gram targeting COVID-19, a human globulin mimic (HGM), is funded with $5 mil­lion grant from the Bill & Melinda Gates Foundation and ex­pected to begin clin­i­cal testing in early 2021, with addi­tional COVID-19 treat­ments in de­vel­op­ment. Kleo in­vestors in­clude Biohaven Pharma­ceu­tical Holding Com­pany (NYSE:BHVN) and PeptiDream Inc. (Nikkei:PPTDF). For more in­for­ma­tion, visit www.kleopharmaceuticals.com.

Source: Kleo Pharma­ceu­ticals.