The 2nd PhI­II schiz­o­phre­nia study for In­tra-Cel­lu­lar drug is a dis­as­ter, shares shred­ded

A year af­ter turn­ing up some mixed schiz­o­phre­nia da­ta from their first late-stage study of their lead drug for schiz­o­phre­nia, In­tra-Cel­lu­lar Ther­a­peu­tics to­day said that the sec­ond late-stage tri­al was a near com­plete flop.

As in many sim­i­lar stud­ies, In­tra-Cel­lu­lar $IT­CI says their psy­chi­atric drug was done in by a high place­bo re­sponse. And like many oth­ers, it is vow­ing to re­main com­mit­ted to the de­vel­op­ment pro­gram.

In­vestors had oth­er ideas on that score. The New York-based biotech’s stock went in­to a melt­down once trad­ing re­sumed, plung­ing 68% and eras­ing the li­on’s share of its $1.8 bil­lion mar­ket cap.

The first time out in Phase III, In­tra-Cel­lu­lar in­ves­ti­ga­tors said that the high dose proved more ef­fec­tive than a place­bo in im­prov­ing symp­toms of schiz­o­phre­nia. This time, though, nei­ther dose of the 5-HT2A re­cep­tor an­tag­o­nist did bet­ter. But the con­trol drug risperi­done did do bet­ter than the place­bo, mak­ing it all the more dif­fi­cult for com­pa­ny ex­ecs to ex­plain away the flop.

In­tra-Cel­lu­lar’s drug ac­tu­al­ly did just a tad worse than place­bo, and sig­nif­i­cant­ly worse than the risperi­done con­trol arm. “ITI-007 60 mg and 20 mg demon­strat­ed a change from base­line on the PANSS to­tal score of -14.6 points and -15.0 points re­spec­tive­ly ver­sus a -15.1 point change in place­bo,” the com­pa­ny re­port­ed. “Risperi­done, the ac­tive con­trol, demon­strat­ed a change from base­line on the PANSS to­tal score of -20.5 points.”

Re­searchers fin­gered a few key tri­al sites for the un­usu­al­ly high place­bo re­sponse.

The biotech swift­ly tried to shift fo­cus to an im­proved safe­ty pro­file, adding that they have the mon­ey in hand need­ed to con­tin­ue work on the drug. And they added that they still be­lieve that the full set of da­ta from a mid-stage study and the two Phase III tri­als proved that the drug is ef­fec­tive.

“Based on the strength of the clin­i­cal da­ta gen­er­at­ed in this pro­gram to date, in­clud­ing two pos­i­tive stud­ies, sup­port­ive ev­i­dence from Study ‘302 and a con­sis­tent, well-tol­er­at­ed and place­bo-like safe­ty pro­file across all stud­ies, we con­tin­ue to be­lieve ITI-007 will be an im­por­tant treat­ment for pa­tients suf­fer­ing from schiz­o­phre­nia. We re­main com­mit­ted to the de­vel­op­ment of ITI-007 for the treat­ment of schiz­o­phre­nia, bipo­lar de­pres­sion, ag­i­ta­tion as­so­ci­at­ed with de­men­tia, in­clud­ing Alzheimer’s dis­ease and oth­er neu­ropsy­chi­atric in­di­ca­tions,” said Dr. Sharon Mates, Chair­man and CEO of IT­CI.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

As of now, the competition for the top job appears to have narrowed down to 2 people: The longtime CDER chief Janet Woodcock and Joshua Sharfstein, the former principal deputy at the FDA under Peggy Hamburg. Both were appointed by Barack Obama.

Dan Skovronsky, Eli Lilly CSO (Lilly via Facebook)

Eli Lil­ly tees up dis­cov­ery pact worth more than $1.6B with Merus for T cell-fo­cused bis­pe­cif­ic an­ti­bod­ies

Under science chief Dan Skovronsky, Eli Lilly has taken some big swings at next-gen therapies, including trying to find the next big thing in oncology. Now, after one early failure in the field, Lilly is going back to the bispecific antibody well with a new deal with a Dutch biotech.

Lilly will pay $40 million upfront with an additional $20 million equity stake in Merus NV to identify and develop three bispecific antibodies looking to engage the CD3 antigen on T cells and redirect immune cells, the Indianapolis pharma giant said Tuesday.

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Andrew Allen (Gritstone)

As coro­n­avirus vari­ants trig­ger new alarms, the NIH is putting an un­der-the-radar ‘next-gen’ vac­cine in­to PhI

Over the past year, the world has been transfixed by the development of new vaccines to fight SARS-CoV-2. In a frenzy of activity, the new mRNA approach has delivered pioneering emergency approvals in record time. And with some setbacks, the more traditional big players are coming along with added jabs as the most affluent nations in the world begin to vaccinate large portions of their populations.

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The IPO queue adds 5 more biotechs hop­ing to ring in 2021 by blitz­ing Nas­daq

Following a record year for IPOs — in terms of both proceeds and count — there’s already a long lineup of biotechs ready to jump onto Nasdaq in the new year. The companies are likely looking for much higher raises than they initially projected on their S-1s. Now it’s time to see if investors are still hungry for another round of biotech stocks.

Sana helped set the pace early on, as its founders look to divvy up a fortune from their IPO. And late last week 5 more biotechs crowded in, looking to pick up the pace where 2020 left off. Here they are:

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News brief­ing: Beam bags a $260M pri­vate place­ment; mi­Ra­gen re­brands to Virid­i­an Ther­a­peu­tics

Agios vet John Evans has demonstrated how to raise big money for a little biotech.

The Beam Therapeutics CEO — and ARCH partner — has pieced together a $260 million private placement from a group of backers that includes Perceptive Advisors, Farallon Capital, Casdin Capital, Redmile Group and Cormorant Asset Management. And there are 3 main goals they’ll pursue with it: clinical development, strategic partnerships and general corporate purposes.

As­traZeneca keeps the ball rolling on Dai­ichi-part­nered En­her­tu, pick­ing up 2nd in­di­ca­tion in gas­tric can­cer

AstraZeneca’s big gamble on Daiichi Sankyo’s antibody-drug conjugate Enhertu has already paid off with a big approval in breast cancer more than a year ago. But the partners have big plans for their blockbuster in the making, and a new nod in gastric cancer will raise their spirits even higher.

The FDA on Friday approved Enhertu to treat locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma in patients who have previously undergone at least one round of treatment with a Herceptin-based regimen, AstraZeneca said in a release.

Ox­ford gets £100M to seize a 'break­through mo­men­t' in fight­ing su­per­bugs

Close to 70 years after Oxford scientists purified penicillin and confirmed its effect as an antibacterial drug, the university is establishing a new research institute at the forefront of combating antimicrobial resistance.

The Ineos Oxford Institute for AMR Research will initially be powered by a $136 million (£100 million) donation from Ineos, the UK-based chemicals giant founded by billionaire Jim Ratcliffe, that also plays a hand in manufacturing medical and pharma products.