Things were look­ing good for Mus­tang Bio’s MB-107 gene ther­a­py pro­gram. By ear­ly Sep­tem­ber, it had nabbed both rare pe­di­atric dis­ease and or­phan drug des­ig­na­tions, adding to the re­gen­er­a­tive med­i­cine ad­vanced ther­a­py des­ig­na­tion it al­ready scored at the FDA. It all bod­ed well for the piv­otal tri­al they were look­ing to start in new­ly di­ag­nosed in­fants with X-linked se­vere com­bined im­mun­od­e­fi­cien­cy.

There was just a prob­lem: The FDA had put a clin­i­cal hold on that tri­al in June.

Six months af­ter ex­ecs first alert­ed in­vestors to the hold, Mus­tang said they’ve fi­nal­ly been cleared to pro­ceed with dos­ing.

Reg­u­la­tors lift­ed the hold on the piv­otal Phase II tri­al on Jan. 28 af­ter re­view­ing a com­pre­hen­sive pack­age on chem­istry, man­u­fac­tur­ing and con­trols (CMC) — which was what raised con­cerns in the first place — sub­mit­ted in late De­cem­ber, Mus­tang added.

Manuel Litch­man

“The clin­i­cal out­comes ob­served in XS­CID pa­tients in the on­go­ing Phase 1/2 clin­i­cal tri­als con­tin­ue to be en­cour­ag­ing,” CEO Manuel Litch­man said in a state­ment. “It is es­pe­cial­ly grat­i­fy­ing to see the con­sis­tent safe­ty and ef­fi­ca­cy of our lentivi­ral vec­tor over the course of more than eight years since the first pa­tient was treat­ed at NI­AID in 2012.”

Mus­tang plans to start the tri­al in 2021, en­rolling 10 pa­tients whose da­ta would be com­piled with those from the 15 al­ready treat­ed in the cur­rent study. It’s pre­vi­ous­ly not­ed that in­ves­ti­ga­tors were con­tin­u­ing to ac­crue par­tic­pants while wait­ing for the hold to lift.

Af­ter treat­ment with MB-107, a one-time ther­a­py de­liv­er­ing the IL2RG gene in a lentivi­ral vec­tor, the 25 pa­tients will be com­pared with 25 “matched his­tor­i­cal con­trol pa­tients who have un­der­gone hematopoi­et­ic stem cell trans­plan­ta­tion” — the cur­rent treat­ment of choice that, while the­o­ret­i­cal­ly cu­ra­tive, can still leave pa­tients vul­ner­a­ble to in­fec­tions.

The pro­gram now be­ing de­vel­oped by Mus­tang came from St. Jude Chil­dren’s Re­search Hos­pi­tal, but the ap­proach has been test­ed for al­most two decades ei­ther as an al­ter­na­tive or fol­lowup to HSCT.

A sec­ond pro­gram, dubbed MB-207, is tar­get­ing that pop­u­la­tion who’s re­ceived trans­plants.

The piv­otal tri­al will be com­par­ing event-free sur­vival be­tween the two groups. Al­though it’s start­ing lat­er than orig­i­nal­ly ex­pect­ed, the com­pa­ny says it still an­tic­i­pates topline da­ta in the sec­ond half of 2022.

So­cial: Manuel Litch­man, PR Newswire

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

S&P Global has taken a look at the dominant forces shaping the pharma market and come to the conclusion that there will be more downgrades than upgrades in 2021 — the 8th straight year of steady decline.

But it’s not all bad news. Some things are looking up, and there’s still plenty of money to be made in an industry that enjoys a 30% to 40% profit margin, once you factor in steep R&D expenses.

Neither Janet Woodcock nor Joshua Sharfstein is likely to be nominated as the permanent FDA commissioner, Steve Usdin at BioCentury reports.

The White House is looking for alternatives to Woodcock, the acting chief and longtime CDER director, after opposition from several Democratic senators who are calling on others to block her nomination if her name is put forth, according to Usdin. Sharfstein, the former principal deputy FDA commissioner and current Johns Hopkins professor, is out of the running altogether.