Milan, Italy (Press Release) – MolMed S.p.A. (MLMD.MI), a bio­technology com­pany focused on research, devel­op­ment, pro­duc­tion and clin­i­cal val­i­da­tion of gene and cell ther­a­pies for the treat­ment of cancer and rare diseases, announces to have obtained the authori­za­tion from AIFA to start phase I-II first in man clin­i­cal trials with its own CAR-T CD44v6 for the treat­ment of patients with acute myeloid leukemia (AML) and multiple myeloma (MM). The authori­za­tion from AIFA follows the positive tech­ni­cal opinion expressed by the Italian National Institute of Health – ISS (Istituto Superiore di Sanità) on March 12th, 2019.

The clin­i­cal multi­center phase I-II in­ves­ti­ga­tion is part of the European project EURE CART Horizon 2020, coordinated and sponsored by MolMed. The trials foresee the par­tic­i­pa­tion of 5 clin­i­cal centers: two in Italy (San Raffaele Hospital in Milan, clin­i­cal trials coordination center, and Bambino Gesù Children's Hospital in Rome) and three clin­i­cal centers in other European countries, Spain, Germany and Czech Republic.

The study consists of two phases: a first phase involving adult patients with AML and MM, aimed at identifying the Maximum Tolerated Dose (MTD) among the dose levels foreseen by the clin­i­cal protocol, and a second phase, which will in­clude also pediatric patients, with the pri­mary objective to eval­u­ate the thera­peutic activity of CAR-T cells in each pathology in a larger number of patients.

CD44v6 is in fact an an­ti­gen that has never been used as target in a CAR before, and expressed not only by some hema­to­logical tumors such as myeloma and leukemia, but also from several solid tumors, in­­clud­ing some big killers, such as pancreatic, head and neck adenocarcinomas and many others.

CAR-T CD44v6 is also char­ac­ter­ized by its con­struct which in­cludes MolMed’s pro­pri­e­tary suicide gene, aimed at in­creas­ing and extending the safety profile of the prod­uct.

Riccardo Palmisano, MolMed’s CEO commented: "The authori­za­tion to start the clin­i­cal study with CAR-T CD44v6 rep­re­sents a main mile­stone for our Company, and exploits all the effort made by our researchers over the past few years to develop an inno­va­tive pipe­line in the promising field of CAR ther­a­pies. As in its tradition, MolMed wanted to con­firm its pioneer­ing ap­proach also in this area, devel­op­ing a com­pletely original CAR-T: while the only two CARTs cur­rently authorized for trade in the USA and Europe and most of those now undergoing clin­i­cal trials, use specific CARs for CD19 an­ti­gen, limiting their indi­ca­tions to patients with hema­to­logic diseases of the line lym­pho­cyte B, our CAR-T CD44v6 has a com­pletely dif­fer­en­t target, expressed in both hema­to­logical and solid tumors, as well as a higher promise of safety thanks to the presence of the suicide gene. We are confident that, thanks to the com­bi­na­tion of our skills and continuous commitment to research, devel­op­ment and pro­duc­tion of inno­va­tive gene and cellular ther­a­pies, the results of this first phase of the clin­i­cal study could con­firm our ex­pec­ta­tions, and those of clinicians and patients, bringing a safe and effective solu­tion to unmet medical needs”.

About MolMed

MolMed S.p.A. is a clin­i­cal stage bio­tech com­pany focused on research, devel­op­ment, manu­fac­tur­ing and clin­i­cal val­i­da­tion of inno­va­tive ther­a­pies. MolMed's prod­uct portfolio in­cludes pro­pri­e­tary anti-tumor ther­a­pies in both clin­i­cal and pre­clin­i­cal devel­op­ment: Zalmoxis® (TK) is a cell ther­apy based on donor T cells genetically engi­neered to enable bone marrow trans­plants from partially compatible donors for patients with high-risk hema­to­logical malig­nan­cies, eliminating post-transplant immuno­sup­pres­sion prophylaxis and inducing a rapid immune reconstitution. Zalmoxis®, that received orphan drug desig­na­tion and is cur­rently in Phase III in a high-risk pop­u­la­tion of acute leukemia patients, but has already obtained a Conditional Marketing Authorization by the European Com­mis­sion in the second half of 2016 as well as reim­burse­ment con­di­tions in Italy and in Germany at the beginning of 2018. Still focusing on this cell & gene tech­nology, the Company is devel­op­ing a new thera­peutic plat­form based on Chimeric Antigen Receptor (CAR), both au­tol­o­gous and allo­geneic; the most ad­vanced prod­uct, CAR-T CD44v6, which in March 2019 received the authori­za­tion to start human clin­i­cal trials in onco-hematologic indi­ca­tions (AML and MM), fol­low­ing an extensive pre-clinical phase; the prod­uct is poten­tially effective also in several epithelial solid tumors. With regards to allo­geneic CARs, MolMed is devel­op­ing a pipe­line based on NK (Natural Killer) cells, fol­low­ing a research agree­ment signed in 2018 with Glycostem. MolMed is also the first com­pany in Europe to have obtained the GMP manu­fac­tur­ing authori­za­tion for cell & gene ther­a­pies for its pro­pri­e­tary prod­ucts (Zalmoxis®) as well as for third parties and/or in part­ner­ship (Strimvelis, an Orchard gene ther­apy for the ADA-SCID). With reference to GMP devel­op­ment and manu­fac­tur­ing activities for third parties, MolMed signed numerous part­ner­ship agree­ments with leading European and US com­pa­nies. In the field of inno­va­tive onco­logical ther­a­pies MolMed pipe­line also in­cludes NGR-hTFN, a thera­peutic agent for the treat­ment of solid tumors. MolMed, founded in 1996 as an academic spin-off of the San Raffaele Scientific Institute, is listed on the main market (MTA) of the Milan stock exchange man­aged by Borsa Italiana since March 2008. MolMed is headquartered and based in Milan, at the San Raffaele Biotechnology Department (DIBIT) and has an operating unit at OpenZone in Bresso.

Source: MolMed.