Aca­dia adopts a new PhI­II game plan for 'break­through' med, dumps Alzheimer's PhII

Aca­dia $ACAD has de­cid­ed to change the game for its drug Nu­plazid (pi­ma­vanserin) go­ing fur­ther in­to Phase III, af­ter the FDA hand­ed out its break­through drug des­ig­na­tion for the ther­a­py.

Steve Davis, Aca­dia

Ap­proved al­ready for Parkin­son’s dis­ease psy­chosis, the biotech had been set­ting the stage for a piv­otal tri­al in Alzheimer’s dis­ease psy­chosis for their 5-HT2A ther­a­py. But in an­nounc­ing the launch of their HAR­MO­NY study, ex­ecs say now that they will re­cruit a mix of pa­tients with Alzheimer’s dis­ease, de­men­tia with Lewy bod­ies, Parkin­son’s dis­ease de­men­tia, vas­cu­lar de­men­tia and fron­totem­po­ral de­men­tia.

The move comes as reg­u­la­tors pro­vid­ed the BTD ti­tle to Nu­plazid as a treat­ment for dis­ease psy­chosis, an en­dorse­ment for eas­i­er ac­cess at the FDA that should help the biotech push through the next round of late-stage work.

At the same time, they’re scrap­ping a Phase II Alzheimer’s study af­ter post­ing some­what con­tro­ver­sial mid-stage da­ta with mixed re­sults. The drug hit at week 6 but failed to on the psy­chosis score at week 12.

The biotech says it de­cid­ed to stop the study be­cause it over­lapped with their Phase III sites. Per­haps not co­in­ci­den­tal­ly, it re­moves any chance of neg­a­tive da­ta squir­rel­ing their work in Phase III.

Shares at Aca­dia surged about 6% this morn­ing.

Leerink’s Paul Mat­teis added up the news and con­clud­ed that it pro­vid­ed a mixed pic­ture for the com­pa­ny at this stage.

On the plus side, FDA feed­back award­ing break­through des­ig­na­tion and al­low­ing for a sin­gle piv­otal study is en­cour­ag­ing, es­pe­cial­ly with re­spect to how the agency views pi­ma­vanserin safe­ty. More­over, if the study suc­ceeds, the ad­dress­able pop­u­la­tion could be very large, ren­der­ing how we cur­rent­ly mod­el the ADP rev­enue opp­ty con­ser­v­a­tive. How­ev­er, ACAD’s “bas­ket” ap­proach to this new ph3 may in­tro­duce ad­di­tion­al chal­lenges that are sim­ply dif­fi­cult to hand­i­cap: while the tri­al works to en­rich the place­bo-con­trolled por­tion for pi­ma­vanserin re­spon­ders, we see the in­clu­sion of such a het­ero­ge­neous pop­u­la­tion in a sin­gle piv­otal as a risky strat­e­gy.

“We are pleased the FDA has agreed to an ef­fi­cient de­vel­op­ment path for pi­ma­vanserin in this broad in­di­ca­tion and grant­ed Break­through Ther­a­py Des­ig­na­tion in recog­ni­tion of this se­ri­ous un­met need,” said Aca­dia R&D chief Serge Stankovic.

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; 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.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'


Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Rahul Singhvi, Resilience CEO

A Bob Nelsen start­up turns to Har­vard to help sharp­en its tech, in­spir­ing first spin­out

One of Bob Nelsen’s latest projects is headed to Harvard.

Resilience, a company started with the goal of establishing itself as a “one-stop-shop” for companies looking to scale manufacturing, including for hard-to-develop cell and gene therapies, is less than a year old. Friday, it announced a five-year R&D deal with Harvard University that includes $30 million to develop biologics, including vaccines, nucleic acids and cell and gene therapies.

Susan Galbraith speaking at Endpoints News' virtual EUBIO21 summit

Imfinzi/treme­li­mum­ab com­bo scores As­traZeneca an­oth­er OS win — this time in liv­er can­cer

Is the tide turning on AstraZeneca’s battered PD-L1/CTLA4 combo?

A single priming dose of the experimental tremelimumab, followed by Imfinzi every four weeks, beat Nexavar (sorafenib) in helping a group of liver cancer patients live longer in a Phase III study, the company reported, meeting the primary endpoint.

Specifically, the two drugs extended overall survival for patients with unresectable hepatocellular carcinoma who had not received prior systemic therapy and were not eligible for localized treatment.

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FDA+ roundup: Marks on Wood­cock­'s tenure as act­ing com­mis­sion­er; FDA lead­ers of­fer per­spec­tive on bar­ri­ers to di­ver­si­ty in re­search

CBER director Peter Marks praised Janet Woodcock’s work as acting FDA commissioner, and while noting that Biden needs to nominate someone to fill the role permanently by Nov. 16, he said he has “no idea” when that actually might occur.

“Dr. Woodcock has been at the agency for over three decades and she, during that time, has proven herself to be a remarkably capable manager,” Marks said at the Alliance for Regenerative Medicine’s meeting Tuesday. “And she’s been managing as if she’s commissioner, unlike some previous acting [commissioners] who are afraid to actually do things. She doesn’t appear to be afraid to do things. I have not felt any different now from when we had a commissioner in place,” he added.

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