Mus­tang Bio is in fa­mil­iar ter­ri­to­ry, but that isn’t a place it nec­es­sar­i­ly wants to be.

The FDA has placed a hold on Mus­tang Bio’s piv­i­tol tri­al for its gene ther­a­py to treat pa­tients with bub­ble boy dis­ease, cit­ing is­sues sur­round­ing chem­istry, man­u­fac­tur­ing and con­trols clear­ance. It’s the sec­ond hold due to CMC is­sues the com­pa­ny has re­ceived in rough­ly 18 months.

An in­ves­ti­ga­tion­al new drug ap­pli­ca­tion was sub­mit­ted in De­cem­ber 2021. If grant­ed an IND, a Phase II study will then as­sess safe­ty, tol­er­a­bil­i­ty and ef­fi­ca­cy of MB-207. If ap­proved by the FDA, the ther­a­py would one day be el­i­gi­ble for a rare pe­di­atric dis­ease vouch­er.

The ther­a­py, known as MB-207, has been pre­vi­ous­ly grant­ed or­phan drug and rare pe­di­atric dis­ease des­ig­na­tions. The lentivi­ral ther­a­py is for those pa­tients who have al­ready been treat­ed with a hematopoi­et­ic stem cell trans­plan­ta­tion, and need more treat­ment.

Bub­ble boy dis­ease is of­fi­cial­ly known as X-linked se­vere com­bined im­mun­od­e­fi­cien­cy. It’s a rare ge­net­ic dis­or­der that oc­curs in 1 per 225,000 births, and is char­ac­ter­ized by the lack of func­tion of key im­mune cells. That leaves chil­dren with a se­vere­ly com­pro­mised im­mune sys­tem, and can lead to the ear­ly death of chil­dren. Many who are af­fect­ed by the dis­ease are un­able to es­tab­lish T cell im­mu­ni­ty.

Mus­tang was pre­vi­ous­ly is­sued a hold for MB-107, which is cur­rent­ly in Phase I/II tri­als at St Jude, UCSF Be­nioff Chil­dren’s Hos­pi­tal in San Fran­cis­co and Seat­tle Chil­dren’s Hos­pi­tal in new­ly di­ag­nosed in­fants un­der the age of two. In June 2021, the progress of MB-107 was de­layed, but about a year ago, the hold was cleared up, and reg­u­la­tors al­lowed the Phase II tri­al to pro­ceed af­ter re­ceiv­ing a com­pre­hen­sive pack­age on CMC that was sub­mit­ted in De­cem­ber 2020.

That tri­al en­rolled 10 pa­tients, and com­bined them with an­oth­er 15 al­ready treat­ed. Then, the 25 pa­tients will be com­pared with an­oth­er 25 pa­tients who “matched his­tor­i­cal con­trol pa­tients who have un­der­gone hematopoi­et­ic stem cell trans­plan­ta­tion.” Re­sults are ex­pect­ed from that in the sec­ond half of this year.

In a state­ment, CEO Manuel Litch­man said:

In light of our pos­i­tive ex­pe­ri­ence man­ag­ing the pri­or MB-107 CMC hold, and our abil­i­ty to se­cure FDA clear­ance to pro­ceed with that pro­gram, we be­lieve that our CMC team is well po­si­tioned to ad­dress the agency’s con­cerns around MB-207 once ad­di­tion­al clar­i­fi­ca­tion of the hold be­comes avail­able. Fur­ther­more, we re­main ful­ly com­mit­ted to the suc­cess of the piv­otal Phase II MB-207 clin­i­cal tri­al for chil­dren with XS­CID who have pre­vi­ous­ly re­ceived HSCT and re­quire re-treat­ment.

The news comes a lit­tle more than a month af­ter the com­pa­ny joined the NAS­DAQ. The stock hov­ered around the usu­al dol­lar-and-change mark Tues­day, as it sat around $1.17 per share around 11 a.m.

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