FDA spurns Minerva's schizophrenia drug in RTF, sending share price into tailspin – Endpoints News

The writ­ing was on the wall.
The FDA spelled out its qualms with Min­er­va Neu­ro­sciences’ schiz­o­phre­nia drug can­di­date rolu­peri­done in 2020. Nev­er­the­less, the biotech pushed for­ward and had meet­ings with the reg­u­la­tor to at­tempt to as­suage con­cerns and get its drug to mar­ket.
So the biotech went ahead and, in Au­gust, asked the drug reg­u­la­tor to ap­prove the treat­ment for neg­a­tive symp­toms in peo­ple with schiz­o­phre­nia.
With­in eight weeks, the FDA flashed a red light and sent the Burling­ton, MA, biotech a refuse-to-file let­ter.
In­vestors im­me­di­ate­ly threw in the tow­el. Shares $NERV nose­dived 67% be­fore the open­ing bell Mon­day, as Min­er­va’s stock price has slid pre­cip­i­tous­ly since the De­cem­ber 2020 news about the FDA’s ap­pre­hen­sive re­ac­tion to the clin­i­cal pack­age the biotech had put to­geth­er.
Once again, the biotech wants to hash it out with the FDA again in a Type A meet­ing, which can be re­quest­ed as per the RFL. CEO Re­my Luthringer said the com­pa­ny is “dis­ap­point­ed” in the FDA’s de­ci­sion, es­pe­cial­ly giv­en the lack of treat­ment op­tions for peo­ple with the so-called neg­a­tive symp­toms, which in­clude a dwin­dling of mo­ti­va­tion and in­ter­est in life and its var­i­ous ac­tiv­i­ties, sleep be­hav­ior changes, among oth­er changes to dai­ly life.
Back in 2020, the FDA ex­pect­ed a lot more out of Min­er­va. The reg­u­la­tor want­ed an­oth­er well-con­trolled Phase III. The agency al­so list­ed off un­easi­ness about the Phase III fail­ure at both dos­es and hav­ing a tri­al that was con­duct­ed en­tire­ly out­side the US.
When fil­ing the drug in Au­gust, Luthringer em­pha­sized the so­cial bur­den that neg­a­tive symp­toms can have and the pauci­ty of op­tions avail­able to these pa­tients.
“While pos­i­tive symp­toms of schiz­o­phre­nia are gen­er­al­ly well man­aged with an­tipsy­chotics, neg­a­tive symp­toms are of­ten the main bur­den of ill­ness and can im­pact the pa­tients’ qual­i­ty of life as a re­sult of dis­abil­i­ties caused by im­paired vo­ca­tion­al and so­cial skills,” the CEO said in a state­ment at the time.
Mul­ti­ple bio­phar­mas have run in­to hur­dles over the years at­tempt­ing to de­vel­op new treat­ments for schiz­o­phre­nia, leav­ing pa­tients with few op­tions, if any. But some pos­i­tive steps have come in re­cent months with a Phase III da­ta read­out that sent Karuna Ther­a­peu­tics and an ear­li­er-stage com­peti­tor on mas­sive fi­nanc­ing hauls. Roy­al­ty Phar­ma al­so dished out cap­i­tal last week to help Mer­ck get through a Phase II.
In one study of Min­er­va’s drug, the 64mg dose of rolu­peri­done was sta­tis­ti­cal­ly sig­nif­i­cant at re­duc­ing neg­a­tive symp­toms, with a p-val­ue of 0.0036. But the drug missed the mark in the oth­er tri­al, com­ing in at a p-val­ue of 0.064.
The com­pa­ny and reg­u­la­tor met in March to dis­cuss the mar­ket­ing path. Af­ter the con­fab, Min­er­va said it pro­vid­ed ad­di­tion­al da­ta to FDA con­cern­ing “po­ten­tial im­pact of rolu­peri­done ad­min­is­tra­tion on the ef­fi­ca­cy and safe­ty of an­tipsy­chot­ic drugs,” as well as com­pa­ra­bil­i­ty of US and non-US pa­tients be­cause the FDA was con­cerned whether the ex-US re­sults could be ap­plied to pa­tients state­side.
But it didn’t ap­pear to be enough for the agency. Rolu­peri­done is the on­ly Min­er­va drug in the clin­ic, and the biotech hasn’t up­dat­ed its pub­lic pipeline since March 2021. The com­pa­ny wants to work in an­oth­er dif­fi­cult R&D field, Parkin­son’s dis­ease, but that as­set has yet to en­ter the clin­ic.
The com­pa­ny al­so pre­vi­ous­ly worked on a drug for ma­jor de­pres­sive dis­or­der with in­som­nia symp­toms, but opt­ed out of its agree­ment with John­son & John­son’s Janssen in ear­ly 2021. The Big Phar­ma is test­ing the drug, sel­torex­ant, in a Phase II study in pa­tients with “prob­a­ble Alzheimer’s Dis­ease with clin­i­cal­ly sig­nif­i­cant ag­i­ta­tion/ag­gres­sion,” a Phase III in adults with MDD with in­som­nia symp­toms and oth­er ear­li­er-stage tri­als. Janssen has tout­ed block­buster po­ten­tial. Roy­al­ty Phar­ma ac­quired Min­er­va’s roy­al­ty in­ter­est for $60 mil­lion up­front and an­oth­er $95 mil­lion on the line.
Key takeaways:
The data supporting regulatory approval alone are often insufficient for demonstrating the added benefit of a new therapy
Planning in advance to continuously deliver data that illustrate value and post-launch, to not only the regulator, but payers, healthcare professionals, and patients results in more clinically meaningful benefits
By working cross-functionally, biopharmaceutical teams can uncover evidence gaps and better shape registration trials to ensure the needs of as many stakeholders as possible are met
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