Aca­dia shares slide — again — af­ter FDA con­firms it has launched a safe­ty re­view of Nu­plazid

Aca­dia’s suf­fer­ing shares $ACAD took an­oth­er hit on Wednes­day, plung­ing 22% af­ter the FDA con­firmed to­day that reg­u­la­tors have be­gun a re­view of its an­ti-psy­chosis drug Nu­plazid — fol­low­ing CNN’s re­port ques­tion­ing the safe­ty of the drug.

A ’break­through’ drug ush­ered in­to the mar­ket to treat Parkin­son’s dis­ease psy­chosis, CNN re­cent­ly high­light­ed a non­prof­it’s warn­ing on 244 deaths in­volv­ing pa­tients on the drug in less than a year af­ter its launch. Aca­dia re­spond­ed by say­ing there’s no rea­son to be­lieve the drug was di­rect­ly re­spon­si­ble for any of the deaths CNN spot­light­ed in its sto­ry. And the FDA not­ed at the time that there is no rea­son to be­lieve there is any safe­ty is­sue that isn’t al­ready cov­ered by the la­bel. 

In a re­cent ex­change with law­mak­ers, though, FDA com­mis­sion­er Scott Got­tlieb — who took the helm well af­ter the drug was ap­proved — said he would “take an­oth­er look” at Nu­plazid, CNN re­port­ed.

A spokesper­son for the FDA con­firmed to me that the drug is un­der re­view, say­ing:

The FDA is con­duct­ing an eval­u­a­tion of avail­able in­for­ma­tion about Nu­plazid. This re­view has been go­ing on for sev­er­al weeks. We have noth­ing more to share at this time.

The at­ten­tion on safe­ty is­sues has pushed a steep drop in Aca­dia shares, which have lost more than half their val­ue over the last 6 months.

Aca­dia won over an ex­pert pan­el two years ago, de­spite a crit­i­cal re­view by FDA in­sid­ers that clear­ly red-flagged their safe­ty con­cerns, not­ing a dis­tinct in­crease in the num­ber of deaths as well as ad­verse events among the pa­tients tak­ing the drug com­pared to the con­trol arm of the study — even if there was no ob­vi­ous clue what was trig­ger­ing those events. The agency’s ap­proval came with a black box warn­ing on the risks, not un­com­mon at the FDA.

Steve Davis, Aca­dia CEO

Aca­dia had set out to prove that the drug could al­so be used for Alzheimer’s dis­ease psy­chosis, claim­ing a win in a clin­i­cal tri­al on the da­ta. The re­sults, though, quick­ly drew scruti­ny from an­a­lysts and one Alzheimer’s ex­pert, Lon Schnei­der, who not­ed in a sub­se­quent re­view of the da­ta that in­ves­ti­ga­tors had on­ly tracked a tem­po­rary wors­en­ing in the place­bo arm be­fore claim­ing suc­cess. The biotech lat­er switched strate­gies, though, chang­ing to a mix of dis­eases as­so­ci­at­ed with psy­chosis.

In­ves­ti­ga­tors last fall de­scribed their new ap­proach for the piv­otal study, re­cruit­ing a mix of pa­tients with Alzheimer’s dis­ease, de­men­tia with Lewy bod­ies, Parkin­son’s dis­ease de­men­tia, vas­cu­lar de­men­tia and fron­totem­po­ral de­men­tia.

The added scruti­ny of Nu­plazid comes as the FDA has been work­ing on ac­cel­er­at­ing the speed of drug de­vel­op­ment and agency re­views. Any is­sues with one of its ‘break­through’ drugs could con­ceiv­ably slow that process down.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Who’s spend­ing and who’s cut­ting from Big Phar­ma’s $127B R&D bud­get? Here are the top 15 play­ers

A couple of the Big 15 biopharma companies in R&D hit the gas on research spending last year. Merck and Sanofi still have lots to prove in the pipeline, and they’re willing to gamble large sums to make a better future for themselves.

Doing nothing would be infinitely worse.

But collectively, the top players rang up a modest 2.4% increase in spending in 2022, which didn’t cover inflationary pressures. And that set the tone for an extraordinarily cautious year for the industry — even as it laid out about $127 billion to advance new drugs or up the ante on approved therapies.

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Jeff Bluestone (R), Sonoma Biotherapeutics CEO

Jef­frey Blue­stone brings his start­up haul to $400M+, join­ing forces with Re­gen­eron on cell ther­a­pies

These days, when Jeffrey Bluestone gets together with his contemporaries in science, the conversation often turns to retirement plans.

But a little more than three years ago, Bluestone reached a momentous turning point in his career, exiting a prestigious post at UCSF, where he had spent decades in the scientific pursuit of new therapies. And it had nothing to do with retirement anytime in the near future.

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Sally Susman, Pfizer EVP and chief corporate affairs officer

Q&A: Pfiz­er cor­po­rate com­mu­ni­ca­tions chief Sal­ly Sus­man dis­cuss­es book craft­ed in pan­dem­ic and per­son­al lessons

From the political arena to the finance and beauty industries to pharmaceuticals, Pfizer’s Sally Susman has broken barriers, stereotypes and conventions. And now the chief communicator is “Breaking Through,” the title of her first book about effective and innovative communications launching today. The full official title is “Breaking Through: Communicating to Open Minds, Move Hearts, and Change the World.”

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Gun­ning for 2023 ap­proval, GSK de­tails PhI­II da­ta for Jem­per­li in front­line en­dome­tri­al can­cer

GSK has a new slate of data to offer on its PD-1 inhibitor, Jemperli — data that the pharma giant hopes will cement one of the four drug approvals it’s expecting this year.

While Jemperli (dostarlimab) is already approved for a subset of patients with second-line endometrial cancer, GSK set out in the Phase III RUBY trial to test it as an earlier line of treatment while also enrolling a broader group of patients. In an interim analysis, Jemperli was shown to extend progression-free survival for both the subset and the overall trial population when added to chemotherapy.

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Es­pe­ri­on sues Dai­ichi Sankyo, de­mand­ing pay­ment of $300M mile­stone for car­dio drug

Esperion is suing its business partner Daiichi Sankyo, saying the Japanese drugmaker is improperly refusing to pay a $300 million milestone that the biotech company will be owed after reporting positive data from a large trial of its cardiovascular drug Nexletol.

The 2019 deal between the companies had Daiichi Sankyo pay $150 million upfront plus another $150 million after the first sales of the drug. But another major payout was tied to an outcomes study reported this month, known as CLEAR. Esperion, in its suit against Daiichi, argues that the drug’s more than 20% reduction of heart attack risk is enough to trigger a $300 million payout from Daiichi once it’s added to the drug’s label.

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Eli Lil­ly to in­crease in­vest­ment to $1B in­to new Irish man­u­fac­tur­ing fa­cil­i­ty — re­port

The US pharma giant Eli Lilly will be increasing its financial commitment to a manufacturing site in Ireland.

According to a release from Ireland’s Industrial Development Agency (IDA) on Monday, Lilly will be investing another $500 million in its manufacturing facility in Limerick, Ireland — bringing the total investment into the facility to approximately $1 billion.

In January of last year, Lilly announced it was placing a $446 million investment into the site to expand active pharmaceutical ingredient and monoclonal antibody production.

Uğur Şahin, BioNTech CEO (Andreas Arnold/picture-alliance/dpa/AP Images)

BioN­Tech read­ies for plunge in Covid sales, will boost mR­NA and on­col­o­gy pipelines

BioNTech is estimating €5 billion (nearly $5.4 billion) in Covid-19 vaccine sales this year, a marked drop from €17.1 billion ($18.5 billion) in 2022 — and way off analysts’ expectations of around €8 billion ($8.6 billion).

In BioNTech’s year-end earnings call on Monday, it reported a total of €17.3 billion ($18.7 billion) in 2022 revenue, almost all from vaccine sales, which include those via its Pfizer deal and direct sales.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Van­da wins court case against FDA over dis­clo­sure of CRL de­tails for sleep drug

DC District Court Judge Christopher Cooper today granted Vanda Pharma’s request to require the FDA to disclose more info on the complete response letter for its sleep disorder drug Hetlioz.

The melatonin receptor agonist is approved by the FDA to treat non-24-hour sleep-wake disorder, a circadian rhythm disorder. But in 2018 Vanda filed a supplemental application to market Hetlioz as a treatment for jet lag, which the FDA rejected in August 2019, with few details on what Vanda needed to correct course, according to the company.