Just two months ago, Con­cert Phar­ma­ceu­ti­cals of­floaded most of its CNS pipeline to tiny Ter­ran Bio­sciences as a make-or-break da­ta read­out was well on its way.

Now, with to­day’s read­out from Con­cert, it looks like the biotech’s sole can­di­date pulled it off — and that the biotech could very well be be­fore the FDA next year.

The Lex­ing­ton, MA biotech an­nounced topline da­ta Mon­day morn­ing that in­di­cat­ed the pri­ma­ry end­point, plus all “key sec­ondary end­points,” were met. The topline da­ta came from THRIVE-AA1, a Phase III tri­al with more than 700 par­tic­i­pants en­rolled and one of Con­cert’s two Phase III tri­als in­ves­ti­gat­ing drug can­di­date CTP-543.

The drug, a Janus ki­nase in­hibitor that blocks JAK1 and JA­KA2, is be­ing in­ves­ti­gat­ed in pa­tients with mod­er­ate to se­vere forms of au­toim­mune dis­or­der alope­cia area­ta, which re­sults in patchy or com­plete scalp hair loss.

In­vestors were hap­py with the news, with $CNCE shoot­ing up over 11% Mon­day morn­ing.

The pri­ma­ry end­point, ac­cord­ing to Con­cert, was look­ing at the per­cent­age of pa­tients achiev­ing an ab­solute SALT, or Sever­i­ty of Alope­cia Tool score, of 20 or less af­ter 24 weeks of treat­ment. A SALT score di­rect­ly cor­re­lates to per­cent­age of hair loss across four parts of the scalp — a score of 100 rep­re­sent­ing to­tal scalp hair loss, and a score of 0 mean­ing no scalp hair loss. Pa­tients en­rolled in the tri­al were re­quired to have at least 50 per­cent scalp hair loss due to alope­cia area­ta, as mea­sured by SALT. And ac­cord­ing to Con­cert, the av­er­age start­ing SALT score across all pa­tients was ap­prox­i­mate­ly 85.9.

Now on­to the da­ta: 41.5% of pa­tients in the 12 mg arm and 29.6% of those in the 8 mg group reached a SALT score of 20 and un­der by the end of 24 weeks, com­pared to 0.8% in the place­bo group — achiev­ing the pri­ma­ry end­point and clock­ing in a p-val­ue of un­der 0.0001.

Key sec­ondary end­points that were met in­clud­ed the per­cent­age of re­spon­dents on a Hair Sat­is­fac­tion Pa­tient Re­port­ed Out­come (PRO) scale at Week 24 and the per­cent­age of pa­tients achiev­ing the ab­solute SALT scores of 20 or less at each of Weeks 20, 16, 12 and 8. While Con­cert did not give specifics as to those end­points, the biotech said in a state­ment that “all key sec­ondary end­points were met with sta­tis­ti­cal sig­nif­i­cance in both dose groups.”

As comes with the ter­ri­to­ry with JAK in­hibitors, safe­ty has been a long-stand­ing con­cern for the drug class. Con­cert said that the safe­ty pro­file of CTP-543, for which the biotech got break­through ther­a­py des­ig­na­tion back in 2020, was con­sis­tent with pre­vi­ous stud­ies, in­clud­ing side ef­fects such as in­creased cre­a­tine ki­nase lev­els and up­per res­pi­ra­to­ry in­fec­tion. The biotech added that two pa­tients de­vel­oped shin­gles, and SAEs were ob­served in nine pa­tients, four of which were in the place­bo group. One pa­tient, in the 8 mg twice-dai­ly group, had two events “that were as­sessed as pos­si­bly re­lat­ed to treat­ment.”

As for what’s next, Con­cert said in a state­ment it plans to sub­mit the full re­sults for pub­li­ca­tion and even­tu­al pre­sen­ta­tion. A spokesper­son told End­points News that pa­tients were giv­en the op­por­tu­ni­ty to en­roll in an open-la­bel ex­ten­sion tri­al — and so far, 90% of “el­i­gi­ble pa­tients” opt­ed in. The planned read­out for that tri­al is some­time next year.

In the mean­time, Con­cert is wait­ing on da­ta re­sults from THRIVE-AA2, a sec­ond Phase III tri­al with 500 pa­tients en­rolled — which are ex­pect­ed some­time in Q3. And if those da­ta come back pos­i­tive, Con­cert added that it will use the da­ta from those two tri­als to file an NDA with the FDA by the end of H1 next year.

That said, Con­cert is now in a bet­ter po­si­tion against Big Phar­ma ri­vals Pfiz­er and Eli Lil­ly, each with their own JAK in­hibitor — the al­ready-ap­proved Olu­mi­ant in Eli Lil­ly’s case and drug can­di­date ritlecitinib from Pfiz­er — and all look­ing to in­tro­duce the first ap­proved treat­ment for alope­cia area­ta. Aside from ri­vals, Con­cert has an­oth­er bat­tle it’s fight­ing — a patent dis­pute against In­cyte, which Con­cert ap­pealed back in 2019 af­ter In­cyte won a court chal­lenge against Con­cert. CTP-543 is a deuter­at­ed form of In­cyte’s Jakafi.

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.”