Clin­i­cal holds have ham­pered a host of tri­als test­ing treat­ments for Duchenne mus­cu­lar dy­s­tro­phy, but one biotech is get­ting the go-ahead to start its study five and a half months af­ter the ini­tial road­block.

The FDA cleared Dyne Ther­a­peu­tics’ first clin­i­cal tri­al in boys with a cer­tain type of Duchenne mus­cu­lar dy­s­tro­phy, the Mass­a­chu­setts biotech said Tues­day morn­ing, send­ing shares $DYN up near­ly 10% be­fore the open­ing bell. The reg­u­la­tor had re­quest­ed more clin­i­cal and non-clin­i­cal in­for­ma­tion back in Jan­u­ary, but the biotech has been mum on de­tails of what ex­act­ly the agency want­ed to look at be­fore sign­ing off on the Phase I/II tri­al.

In the months since the hold, Dyne has re­peat­ed­ly said it ex­pects to start the study in mid-2022, and the com­pa­ny re­it­er­at­ed that time­line Tues­day with the hold’s lift. The sec­ond half of the year be­gan mere days ago.

Dyne’s can­di­date, dubbed DYNE-251, will be test­ed in 30 to 50 boys aged 4 to 16 with Duchenne who have mu­ta­tions amenable to ex­on 51 skip­ping ther­a­py. Sarep­ta Ther­a­peu­tics mar­kets a drug, Ex­ondys 51, for the spe­cif­ic ex­on, among oth­er treat­ments avail­able for the pro­gres­sive mus­cle weak­ness dis­or­der.

The lift marks a key mo­ment for Dyne as it nears the clin­ic with DYNE-251 and my­oton­ic dy­s­tro­phy type 1 as­set DYNE-101, both slat­ed for mid-2022 tri­al starts. Once DYNE-251 en­ters hu­man stud­ies, Dyne hopes to progress ad­di­tion­al work in oth­er DMD ex­ons, in­clud­ing 53, 45 and 44, as part of its broad­er am­bi­tions to com­pete against oth­er DMD treat­ment mak­ers.

“To­day marks a sig­nif­i­cant step in our jour­ney to build a DMD fran­chise to serve peo­ple across the globe with Duchenne mu­ta­tions amenable to ex­on skip­ping. The clear­ance of our first IND is an im­por­tant achieve­ment for Dyne, and we ap­pre­ci­ate the part­ner­ship with the FDA through­out this process,” Dyne pres­i­dent and CEO Joshua Brumm said in a state­ment.

While there are a few treat­ments on the mar­ket for DMD from the likes of Sarep­ta, PTC Ther­a­peu­tics and an at­tempt from now-de­funct Marathon Phar­ma­ceu­ti­cals at turn­ing a cheap over­seas steroid in­to a steep stick­er price drug, the DMD com­mu­ni­ty has run in­to many hur­dles over the years in get­ting ad­di­tion­al ap­proved ther­a­pies.

Pfiz­er, in its at­tempt to catch up to Sarep­ta, was able to re­sume its Phase III gene ther­a­py tri­al in late April and, at the time, was on track to sub­mit for FDA ap­proval in late 2023. But oth­er bio­phar­mas have run in­to holds and elon­gat­ed paths to ap­proval in the months since.

Just last week, San­thera Phar­ma­ceu­ti­cals said its at­tempt at get­ting a green light for its DMD treat­ment has been pushed back four to six months. Two weeks ago, Sarep­ta’s next-gen Duchenne as­set got hit by a clin­i­cal hold in Part B of a Phase II study. Al­so last month, PTC’s years-long shot at get­ting the FDA to ap­prove its Eu­ro­pean-nod­ded DMD drug Translar­na hit an­oth­er set­back with mixed da­ta from a con­fir­ma­to­ry study.

One biotech, though, has pro­vid­ed some pos­i­tive news to the field in re­cent weeks, with Capri­cor Ther­a­peu­tics say­ing its DMD drug notched the pri­ma­ry end­point in an open-la­bel ex­ten­sion to its Phase II study.

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

GSK has landed a new first from UNICEF — the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”