• Designation will ac­cel­er­ate oNKord®'s mar­ket access for MM
• Together with Clinical Trial Approval for a pivotal phase I-IIa trial in AML, this is another im­por­tant step for­ward in making Glyco­stem's allo­geneic Natural Killer cell-based treat­ment accessible for can­cer patients

Oss, The Netherlands (Press Release) – Glyco­stem Thera­peutics, a lead­ing clin­i­cal-stage com­pany focused on the de­vel­op­ment of thera­peutic off-the-shelf Natural Killer (NK) cells, an­nounces it has re­ceived the FDA's Orphan Drug Desig­na­tion (ODD) for treat­ment of mul­ti­ple myeloma (MM) patients with its inves­ti­ga­tional prod­uct oNKord®. The desig­na­tion will pro­vide Glyco­stem with cer­tain incentives, like eligibility for 7 years of mar­ket exclusivity and clear FDA guidance on spe­cif­ic as­pects of de­vel­op­ment for rare dis­eases. These pave an ac­cel­er­ated path to­wards mar­ket access and treat­ment of patients suffer­ing from this rel­a­tively rare form of can­cer.

oNKord® is Glyco­stem's first-generation off-the-shelf Natural Killer (NK) cel­lu­lar im­mu­no­ther­apy prod­uct. Over the com­ing months, AML patients will re­ceive this form of treat­ment as part of a phase I-IIa (pivotal) trial in AML. A phase II trial for MM patients is ex­pected to start in 2021. This makes Glyco­stem one of the frontrunners in this promising field of cel­lu­lar im­mu­no­ther­apy.

"Since 2012 we have been pio­neers in the field of devel­op­ing and manu­fac­tur­ing off-the-shelf Natural Killer cell ther­apy prod­ucts for can­cer treat­ment. In 2020 we're entering a new and ex­cit­ing phase," says Troels Jordansen, CEO of Glyco­stem. "It is great to ex­peri­ence that after re­ceiv­ing FDA and EMA ODD desig­na­tion for AML, the FDA has also granted us this desig­na­tion for MM. This allows us to ac­cel­er­ate oNKord®'s access to the US mar­ket and our ultimate ambition: curing can­cer."

Multiple Myeloma (MM)

MM is the sec­ond most common blood can­cer, accounting for 15% of blood can­cers, and 2% of all can­cers. In the US alone it affects more than 130,000 patients; approx­i­mately 32,000 Americans are diag­nosed with MM each year. MM oc­curs in in­fec­tion-fighting plasma cells (a type of white blood cell) found in the bone mar­row. These can­cer­ous cells multiply, pro­duce an ab­nor­mal pro­tein and push out other healthy blood cells from the bone mar­row.

Orphan Drug Desig­na­tion

The FDA grants orphan drug desig­na­tion to drugs and bio­log­ics for the prevention, diag­nosis, or treat­ment of dis­eases or con­di­tions affecting fewer than 200,000 persons in the US. The desig­na­tion allows manu­fac­turers to qualify for var­i­ous incentives, in­clud­ing exemption of FDA appli­ca­tion fees), tax credits for qualified clin­i­cal trials and be eli­gible for 7 years of mar­ket exclusivity after regu­la­tory ap­prov­al.

About Glyco­stem

Netherlands-based Glyco­stem Thera­peutics BV, a clin­i­cal stage bio­tech com­pany, de­vel­ops allo­geneic cel­lu­lar im­mu­no­ther­apy to treat sev­er­al types of can­cer. By har­nessing the power of stem cell-derived Natural Killer (NK) cells, Glyco­stem's prod­ucts are a safe alter­na­tive to CAR-T-cells. Glyco­stem's lead prod­uct, oNKord®, is manu­fac­tured from allo­geneic raw ma­teri­al and is avail­able off-the shelf. Thanks to its nine pat­ent families, longstanding tech­ni­cal ex­per­tise and resources, as well as 'Orphan Drug Desig­na­tion', Glyco­stem has secured a leadership position in the global NK-cell mar­ket.

oNKord® is pro­duced in a closed sys­tem in Glyco­stem's state-of-the-art and GMP (Good Manu­fac­tur­ing Prac­tice) li­censed pro­duc­tion facility in the Netherlands, from which it can be distributed globally. The pro­duc­tion tech­nology in­cludes ex vivo gen­er­a­tion of high num­bers of NK-cells with a high degree of purity for clin­i­cal appli­ca­tions. oNKord® suc­cess­fully passed phase I clin­i­cal trial (elderly and frail AML – Acute Myeloid Leukemia – patients), providing solid safety data and strong in­di­ca­tion of clin­i­cal ac­tiv­ity, in­clud­ing re­sponse on MRD (Minimal Residual Disease). Results in­di­cate that oNKord® may be safely in­fused in AML patients.

Glycostem is fur­ther­more devel­op­ing a range of CAR-NK prod­ucts in-house and in cooperation with global part­ners.

Source: Glyco­stem Thera­peutics.