Second orphan medicinal product designation for Neuro-Cells

The Euro­pean Union has grant­ed stem cell biotech Neu­ro­plast an orphan med­i­c­i­nal prod­uct des­ig­na­tion for the applic­a­bil­i­ty of its stem cell tech­nol­o­gy plat­form to fron­totem­po­ral demen­tia (FTD), fol­low­ing a pos­i­tive opin­ion from The Euro­pean Med­i­cines Agency (EMA). With the exist­ing orphan dis­ease des­ig­na­tion (ODD) for trau­mat­ic spinal cord injury (TSCI), Neu­ro-Cells® is now approved for a fast-track devel­op­ment path­way with mar­ket exclu­siv­i­ty for both a trau­ma-induced and a chron­ic degen­er­a­tive cen­tral ner­vous sys­tem dis­or­der. This marks an impor­tant mile­stone in the devel­op­ment roadmap of Neuroplast’s Neu­ro-Cells® plat­form, as a step­ping stone to oth­er chron­ic neu­rode­gen­er­a­tive dis­eases such as Alzheimer’s, ALS and Parkinson’s Dis­ease. The poten­tial width in ther­a­peu­tic applic­a­bil­i­ty of the Neu­ro­plast tech­nol­o­gy gives per­spec­tive to mil­lions of peo­ple suf­fer­ing from neu­rode­gen­er­a­tive dis­eases that cur­rent­ly have no out­look on effec­tive treatment.

One tech­nol­o­gy address­es under­ly­ing mech­a­nisms of mul­ti­ple acute and chron­ic neu­ro­log­i­cal disorders

Sev­er­al con­di­tions of the cen­tral ner­vous sys­tem, even when they seem unre­lat­ed at first and may have dis­tinc­tive caus­es, have sim­i­lar under­ly­ing dis­ease mech­a­nisms in com­mon. These include unpro­grammed cell death boost­ed by inflam­ma­tion. Neu­ro-Cells, an autol­o­gous, bone-mar­row derived Advanced Ther­a­py Med­i­c­i­nal Prod­uct, address­es that dis­ease mech­a­nism by mod­er­at­ing inflam­ma­tion of dam­aged cells in the cen­tral ner­vous sys­tem, to lim­it fur­ther impair­ment. The treat­ment objec­tive in acute dis­or­ders is to lim­it impact of sud­den injury, where the treat­ment objec­tive in chron­ic dis­or­ders is to lim­it pro­gres­sion of the disease.

Neu­ro­plast is already run­ning a fast-track devel­op­ment path­way for trau­mat­ic spinal cord injury (TSCI), with a Phase II clin­i­cal tri­al in progress. This des­ig­na­tion for fron­totem­po­ral demen­tia illus­trates the broad­er applic­a­bil­i­ty of the same tech­nol­o­gy for acute as well as chron­ic neu­rode­gen­er­a­tive dis­or­ders, paving  the way to explore fur­ther applic­a­bil­i­ty to con­di­tions such as ALS, Alzheimer’s dis­ease, trau­mat­ic brain injury, sub­arach­noid stroke and Parkinson’s Disease.

Orphan dis­ease des­ig­na­tion for FTD award­ed based on pre-clin­i­cal evidence

Orphan dis­ease des­ig­na­tions are restrict­ed to prod­ucts for rare con­di­tions for which there are no sat­is­fac­to­ry meth­ods of treat­ment autho­rized. It allows for a faster mar­ket autho­riza­tion path­way and ten-year mar­ket exclusivity.

Fron­totem­po­ral demen­tia (FTD) is a degen­er­a­tive con­di­tion in the brain that affect approx­i­mate­ly 3.8 peo­ple in 10,000 per­sons in the EU. Typ­i­cal sur­vival rate lies between three and four­teen years from symp­tom onset, depen­dent on the FTD vari­ant at play.

For this approval, the Euro­pean Union fol­lowed the pos­i­tive opin­ion from the EMA after the EMA fol­lowed pos­i­tive rec­om­men­da­tions from the Com­mit­tee for Orphan Med­i­c­i­nal Prod­ucts (COMP). COMP part­ly based their con­clu­sions on the avail­abil­i­ty of pre-clin­i­cal evi­dence in mice, that showed decrease in neu­roin­flam­ma­tion mark­ers and res­cue of cog­ni­tive and social behav­ioral deficits. Exam­ples include reduc­tion of anx­i­ety, depres­sive-like behav­ior and abnor­mal social behavior.

Neu­ro­plast CEO Johannes de Munter states:“This des­ig­na­tion for fron­totem­po­ral demen­tia is an impor­tant mile­stone in expand­ing the Neu­ro-Cells devel­op­ment to a wider range of ther­a­peu­tic areas. Using the same tech­nol­o­gy plat­form for trau­mat­ic spinal cord injury and fron­totem­po­ral demen­tia, illus­trates an unusu­al range of acute and chron­ic neu­ro­log­i­cal dis­or­ders that could poten­tial­ly ben­e­fit from this.”

Neu­ro­plast is open to dis­cuss investor oppor­tu­ni­ties to effec­tu­ate the clin­i­cal path­ways to a wider scope of neu­ro­log­i­cal conditions.