Zaf­gen stakes out a PhI beach­head for its next weight drug as it fights for a come­back

Last sum­mer Zaf­gen $ZFGN ex­pe­ri­enced one of the worst biotech dis­as­ters the in­dus­try wit­nessed for all of last year — the kind of night­mare that CEOs dread. Af­ter the FDA put their lead drug be­lo­ranib on clin­i­cal hold in late 2015 — link­ing it to the death of a pa­tient from a pul­monary em­bolism linked with the ther­a­py — the biotech had lit­tle choice but to kill the pro­gram in Ju­ly and slash its staff af­ter reg­u­la­tors spelled out what they would have to do to qual­i­fy for a mar­ket­ing OK.

Tom Hugh­es

But with its share price dec­i­mat­ed, Zaf­gen still didn’t throw in the tow­el. And now in an ef­fort to make a come­back, Zaf­gen’s team has re­leased Phase I da­ta that shows its sole clin­i­cal drug hope, ZGN-1061, a fol­lowup MetAP2 drug, was able to knock about a pound a week off of obese and over­weight pa­tients for four weeks, with­out any signs of throm­bot­ic risk.

Zaf­gen shares surged 15% on the news Thurs­day evening, in post-mar­ket trad­ing.

Break­ing down the da­ta, pa­tients treat­ed with ZGN-1061 for four weeks lost sig­nif­i­cant­ly more weight rel­a­tive to place­bo-treat­ed pa­tients, drop­ping 4.6 lbs, 2.2 lbs and 3.8 lbs for 0.2 mg, 0.6 mg, and 1.8 mg dos­es, re­spec­tive­ly, com­pared to a 0.51 lb loss for the place­bo arm. And that’s what they were hop­ing to see as they po­si­tion them­selves for a run at obe­si­ty and Type 2 di­a­betes, two of the most dif­fi­cult mass mar­ket con­di­tions any biotech ever tack­led.

That’s not the kind of sud­den and mas­sive weight loss that be­lo­ranib could trig­ger in the first few weeks, CEO Tom Hugh­es con­cedes, but cu­mu­la­tive­ly it’s sig­nif­i­cant­ly bet­ter than the dis­ap­point­ing trio of weight drugs that hit a few years ago. This drug, he em­pha­sized, is on the right track for a Phase I, bless­ed­ly free of any sur­pris­es for the re­searchers in­volved.

“We’ve been work­ing in this area for quite some time,” Hugh­es told me ear­li­er Thurs­day. That gives the team at Zaf­gen a chance to care­ful­ly eval­u­ate the phar­ma­co­ki­net­ic qual­i­ties of  ZGN-1061, tar­get en­gage­ment and the dose ef­fect, con­firm­ing the “good be­hav­ior” of the drug in an ad­mit­ted­ly small num­ber of sub­jects.

Hugh­es was def­i­nite­ly up­beat about the re­sults, but al­so per­haps a lit­tle more sub­dued than in past dis­cus­sions about be­lo­ranib as that drug head­ed through the clin­ic be­fore im­plod­ing in Phase III.

“I don’t know if you can tell by our tone,” he said at one point, “but we are re­al­ly, re­al­ly pleased with the re­sult….It’s good to have a well-be­haved mol­e­cule.”

And how.

Phase I is a rel­a­tive­ly small step in the obe­si­ty/di­a­betes R&D world, with ma­jor de­mands that ul­ti­mate­ly have to be met on safe­ty and ef­fi­ca­cy that have be­dev­iled oth­er de­vel­op­ers over the years and forced in­vestors to di­rect their cash else­where.

Phase II, slat­ed to get un­der­way lat­er this year, will re­cruit about 120 pa­tients with Type 2 di­a­betes, says CMO Den­nis Kim, prob­a­bly look­ing at he­mo­glo­bin A1c lev­els — a clas­sic mea­sure of ef­fi­ca­cy — as the pri­ma­ry end­point. The com­pa­ny still prob­a­bly hasn’t es­tab­lished the low­est most ef­fec­tive dose of the drug, which they should be able to shine a light on as mid-stage da­ta comes through.

Lat­er on, adds Hugh­es, it’s pos­si­ble that Zaf­gen could take on a part­ner as it an­gles for a Phase III. And cer­tain­ly on­ly a ma­jor play­er could ex­pect to mar­ket a drug like this if it even­tu­al­ly wins an OK. But if things con­tin­ue to go the biotech’s way this time, Hugh­es feels that the cap­i­tal will be there for what’s need­ed.

Now it’s time to con­cen­trate on step 2 in their re­hab pro­gram.


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.

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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.”

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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.

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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.

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.

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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.

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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.