An­oth­er AD­HD fail­ure ce­ments Ae­vi's pen­ny stock sta­tus

Ae­vi Ge­nom­ic Med­i­cine’s ge­net­ic ap­proach to treat­ing AD­HD was ex­pect­ed to as­cend its lead drug to great heights in the com­mon­ly di­ag­nosed neu­robe­hav­ioral dis­or­der, but this saga has on­ly cul­mi­nat­ed in an­oth­er tri­al fail­ure, forc­ing the re­source-poor com­pa­ny to “re­duce the scope” of its op­er­a­tions to pre­serve cap­i­tal.

Shares of the Philadel­phia-based com­pa­ny $GN­MX were oblit­er­at­ed Wednes­day af­ter­noon, nose­div­ing near­ly 65% af­ter the bell, wad­ing deep­er in­to pen­ny stock ter­ri­to­ry.

The SAGA tri­al re­port­ed in March 2017 cast doubt on the vi­a­bil­i­ty of the drug, AE­VI-001, when it failed to hit sta­tis­ti­cal sig­nif­i­cance against a place­bo on an AD­HD rat­ing scale in ado­les­cent pa­tients with a mu­ta­tion that dis­rupts the mGluR (metabotrop­ic glu­ta­mate re­cep­tor) net­work — an abun­dant fam­i­ly of re­cep­tors in the brain. How­ev­er, an analy­sis of the mid-stage da­ta in the study that en­rolled 101 pa­tients sug­gest­ed the drug did have a pro­nounced im­pact on the symp­toms of inat­ten­tion. En­cour­aged, Ae­vi elect­ed to con­tin­ue eval­u­at­ing the non-stim­u­lant mol­e­cule.

Ac­cord­ing to the com­pa­ny, about 1 in 5 AD­HD pa­tients car­ries such a mu­ta­tion. The Amer­i­can Psy­chi­atric As­so­ci­a­tion es­ti­mates around 5% of Amer­i­can chil­dren have AD­HD, but with in­creas­ing aware­ness, the CDC pegs that num­ber was clos­er to 9.4% in 2016.

Ae­vi took AE­VI-001 in­to an­oth­er mid-stage place­bo-con­trolled study, dubbed AS­CEND, in two types of pa­tients aged 6 to 17.  In one arm of the tri­al, 69 pa­tients with gene mu­ta­tions as­so­ci­at­ed with glu­ta­mater­gic sig­nal­ing were en­rolled, while  for an­oth­er arm of the tri­al, 109 pa­tients with no such mu­ta­tions were en­rolled. Da­ta showed the drug did not in­duce a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in AD­HD symp­toms in ei­ther cat­e­go­ry of pa­tient, but the com­pa­ny has not thrown in the tow­el on the pro­gram just yet.

“Ae­vi Ge­nom­ic Med­i­cine re­mains com­mit­ted to the de­vel­op­ment of AE­VI-001 as a po­ten­tial treat­ment for a sub-pop­u­la­tion of…AD­HD pa­tients with ge­net­ic mu­ta­tions that dis­rupt the mGluR net­work,” the com­pa­ny re­it­er­at­ed in a state­ment on Wednes­day.

How­ev­er, the drug de­vel­op­er is strapped for cash. It an­tic­i­pates re­duc­ing the scope of its op­er­a­tions to pre­serve net work­ing cap­i­tal, which was ap­prox­i­mate­ly $5 mil­lion (unau­dit­ed) as of De­cem­ber 31, 2018, it said.

The com­pa­ny al­so has AE­VI-004, a co-crys­tal ver­sion of AE­VI-001, which is al­so called fa­so­rac­etam. AE­VI-004 is de­signed to im­prove the sta­bil­i­ty of the orig­i­nal AE­VI-001 mol­e­cule, while main­tain­ing sim­i­lar sol­u­bil­i­ty. Mean­while, it is al­so de­vel­op­ing two ad­di­tion­al mol­e­cules: AE­VI-002 for se­vere pe­di­atric-on­set Crohn’s dis­ease and AE­VI-005 for an undis­closed au­toim­mune or­phan dis­ease, both part­nered with Ky­owa Hakko Kirin.

Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,000+ biopharma pros reading Endpoints daily — and it's free.

Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,000+ biopharma pros reading Endpoints daily — and it's free.

Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,000+ biopharma pros reading Endpoints daily — and it's free.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,000+ biopharma pros reading Endpoints daily — and it's free.

On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

Endpoints News

Basic subscription required

Unlock this story instantly and join 55,000+ biopharma pros reading Endpoints daily — and it's free.

Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.