Steve Davis, Acadia Pharmaceuticals CEO

Aca­dia hits an­oth­er Nu­plazid road­block as the FDA doles out CRL and rec­om­mends an­oth­er tri­al

De­spite win­ning an ap­proval back in 2016, Aca­dia’s Nu­plazid has not had the eas­i­est of paths in its jour­ney to ex­pand in­to new ar­eas. And late Thurs­day night, the com­pa­ny put out a press re­lease say­ing it re­ceived more bad news from the FDA.

Reg­u­la­tors re­ject­ed Aca­dia’s ap­pli­ca­tion to ex­pand Nu­plazid cov­er­age in­to Alzheimer’s-re­lat­ed psy­chosis, the biotech said, turn­ing away its sup­ple­men­tal NDA with a CRL. The re­jec­tion let­ter in­di­cat­ed the FDA al­so rec­om­mend­ed that Aca­dia con­duct an ad­di­tion­al tri­al in ADP, Aca­dia said.

Ac­cord­ing to Aca­dia, while the FDA stat­ed its study did demon­strate a sta­tis­ti­cal­ly sig­nif­i­cant treat­ment ef­fect on its pri­ma­ry end­point, lim­i­ta­tions re­main re­gard­ing its ef­fec­tive­ness.

The FDA added that al­though there was pos­i­tive treat­ment ef­fect of the drug on de­men­tia-re­lat­ed psy­chosis in Aca­dia’s HAR­MO­NY study, it ap­peared to be dri­ven pri­mar­i­ly by pa­tients with Parkin­son’s dis­ease de­men­tia — a con­di­tion the FDA stat­ed in­clud­ed Nu­plazid’s ap­proval for pa­tients with Parkin­son’s dis­ease psy­chosis.

“We are dis­ap­point­ed with this out­come. The treat­ment of Alzheimer’s dis­ease psy­chosis con­tin­ues to be an area of high un­met need, for which there is no ap­proved ther­a­py. We want to ex­press our grat­i­tude to all of the pa­tients, their fam­i­lies and in­ves­ti­ga­tors who have par­tic­i­pat­ed in our clin­i­cal tri­als,” Aca­dia CEO Steve Davis said in a state­ment.

The com­pa­ny did not re­spond to in­quiries from End­points News. Aca­dia shares $ACAD are down 35% since Jan­u­ary but re­mained flat in Fri­day morn­ing trad­ing.

Thurs­day’s news is just the lat­est in a string of is­sues that Nu­plazid has faced. In 2018, the drug first came un­der fire af­ter a re­port ques­tioned its safe­ty of the drug, but the FDA even­tu­al­ly gave an all-clear for its safe­ty.

Amidst the push to have the drug treat de­men­tia-re­lat­ed psy­chosis and with the FDA be­ing at­ten­tive af­ter the Aduhelm ap­proval, the drug has not been win­ning fa­vor with the FDA.

Af­ter a CRL in 2021, due to a “lack of sub­stan­tial ev­i­dence of ef­fec­tive­ness” in a Phase III tri­al, the com­pa­ny re­turned to the FDA on­ly to re­ceive more bad news. In June, a pan­el of the FDA’s out­side ex­perts vot­ed 9-3 stat­ing that the drug did not ap­pear to be ef­fec­tive at treat­ing pa­tients with Alzheimer’s-re­lat­ed psy­chosis.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,800+ biopharma pros reading Endpoints daily — and it's free.

EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

FDA's out­side ex­perts vote in fa­vor of Fer­ring's fe­cal trans­plant for C. dif­fi­cile, set­ting the stage for Seres

FDA’s outside advisors voted in favor of Ferring Pharmaceuticals’ RBX2660, an experimental poop-based drug implant that the company says would be the first microbiota-based live biotherapeutic to receive an FDA green light.

That was a point repeatedly discussed during the Vaccines and Related Biological Products Advisory Committee, or VRBPAC, meeting Thursday when evaluating Ferring’s fecal microbiota transplant, or FMT, for reducing the recurrence of Clostridioides difficile infection in adults who have received antibiotics. Multiple members brought up the need for a regulated product amid a landscape of unregulated FMTs already happening in clinical care.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,800+ biopharma pros reading Endpoints daily — and it's free.

Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Klick Health gath­ers biotech and phar­ma lu­mi­nar­ies to dis­cuss in­dus­try in­no­va­tions, in­vest­ments and fu­ture

At Klick Health’s first Ideas Exchange conference with biotech and pharma industry insiders since before the pandemic began, it was no surprise many conversations included Covid topics. Yet while vaccines and treatments were discussed, so too were the effects on drug development, federal responses, health inequities — and what to do now and next.

George Yancopoulos, chief scientist and cofounder of Regeneron, opened the conference responding to a question from Acorda CEO Ron Cohen about the spotlight on the industry during Covid and some of the “flak” biopharma has taken in the past.

Vas Narasimhan, Novartis CEO (Simon Dawson/Bloomberg via Getty Images)

No­var­tis un­veils 'US-first' strat­e­gy ahead of San­doz spin­off

Weeks after announcing the spinoff of generics arm Sandoz, Vas Narasimhan paints a picture of the new, slimmer Novartis — with a “US-first mindset,” he said at an investor event on Thursday.

The CEO unveiled ambitious plans to become a top-five player in the US by 2027 at Novartis’ “Meet the Management” event in Basel, Switzerland, which means ramping up clinical trials in the states and “building capability and talent, among other things.” The company’s also shooting for a top-three ranking in China.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,800+ biopharma pros reading Endpoints daily — and it's free.

Pfiz­er sacks phar­ma com­pe­ti­tion in ear­ly NFL TV ad­ver­tis­ing sea­son

If pharma advertising had a fantasy football league, Pfizer would be crushing the competition. A dive into the National Football League’s TV commercial buys across early season games by iSpot shows a hefty lead with its Covid-19 Comirnaty vaccine ads.

More than 175 million impressions with $9.5 million in media spending put Pfizer in the top spot with a 65% share of voice across NFL pharma spending, according to the real-time TV ad tracker. In a distant second place is Bristol Myers Squibb’s Opdivo with 44 million impressions, $5.2 million in spending and a 16% share, followed by BMS’ Zeposia with 31 million impressions, $3.3 million in media buys and an 11% share.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 150,800+ biopharma pros reading Endpoints daily — and it's free.