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.

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,300+ biopharma pros reading Endpoints daily — and it's free.

BREAK­ING: Math­ai Mam­men makes an abrupt ex­it as head of the big R&D group at J&J

In an after-the-bell shocker, J&J announced Monday evening that Mathai Mammen has abruptly exited J&J as head of its top-10 R&D group.

Recruited from Merck 5 years ago, where the soft spoken Mammen was being groomed as the successor to Roger Perlmutter, he had been one of the top-paid R&D chiefs in biopharma. His group spent $12 billion last year on drug development, putting it in the top 5 in the industry.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,300+ biopharma pros reading Endpoints daily — and it's free.

No­vavax shares shred­ded as Covid vac­cine sales fall more than 90% in Q2

Months after Novavax celebrated its first profitable quarter as a commercial company, the Gaithersburg, MD-based company is back in the red.

Sales for Novavax’s Covid-19 vaccine slipped to $55 million last quarter, down from $586 million in Q1, CEO Stanley Erck revealed on Monday after market close. The company’s stock $NVAX plummeted more than 32% in after-hours trading.

Upon kicking off the call with analysts and investors, Erck addressed the elephant in the room:

Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, less than two weeks after its supplemental BLA was accepted. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.

Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 147,300+ biopharma pros reading Endpoints daily — and it's free.

FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Sen­ate Dems cling to a sim­ple ma­jor­i­ty to pass some of the biggest drug pric­ing re­forms ever

The Pharmaceutical Research and Manufacturers of America — and their fleet of drug industry lobbyists on Capitol Hill — are known for never losing.

Whenever a big drug pricing bill comes up, an army of the industry group’s lobbyists descend onto the Hill and either smash it outright or dismantle it piece by piece.

But for perhaps the largest drug pricing reforms ever enacted, after more than a decade of Congress trying and failing to allow Medicare to negotiate prescription drug prices, those same lobbyists and their biopharma clients were dealt a stunning blow on Sunday afternoon.

FDA's vac­cine ad­comm to re­view first fe­cal trans­plant to treat C. dif­fi­cile in­fec­tions

Back in 2018, Swiss drugmaker Ferring Pharmaceuticals made a big bet on Minnesota-based Rebiotix, buying up the company for its experimental poop-based drug implant to treat an infection caused by C. difficile, a potentially dangerous bacteria, in a new way.

Four years later, Ferring’s fecal microbiota transplant, dubbed RBX2660 or Rebyota, will face the FDA’s adcomm of outside vaccine experts on Sept. 22, debating whether the agency should license the transplant as a treatment for adults following antibiotic treatment for recurrent C. difficile infection.