Voy­ager's shares sink af­ter ex­ecs say FDA has re­versed course on an ac­cel­er­at­ed fil­ing for its gene ther­a­py

The FDA ap­pears to have lost its ap­petite for an ac­cel­er­at­ed mar­ket­ing ap­pli­ca­tion that could pave the way to an ear­ly OK for Voy­ager’s lead gene ther­a­py. The com­pa­ny an­nounced af­ter the mar­ket closed Wednes­day that the agency’s view has shift­ed dra­mat­i­cal­ly. 

Af­ter tan­ta­liz­ing in­vestors in Ju­ly with its claim that FDA of­fi­cials said in a Type C ex­change that they were open to a fil­ing based on the safe­ty and ef­fi­ca­cy seen in a Phase II study for VY-AADC in Parkin­son’s dis­ease, the new word is that reg­u­la­tors are now view­ing the mid-stage study as a nec­es­sary pre­lim­i­nary step to a piv­otal tri­al. In an ad­den­dum that ar­rived in late Oc­to­ber, the biotech said, the FDA now says that “al­though the da­ta from the Phase II tri­al may sup­port the safe­ty and ef­fi­ca­cy of VY-AADC and could be con­sid­ered in the BLA re­view, the FDA cur­rent­ly con­sid­ers the Phase II tri­al as an ear­ly phase ex­plorato­ry study.”

Voy­ager’s stock $VY­GR took a 12% dive in af­ter-mar­ket trad­ing.

But it gets worse when you plumb the com­pa­ny’s SEC fil­ing on its lat­est 10K. The biotech says it de­cid­ed to skip a for­mal sit-down with reg­u­la­tors in a Type C meet­ing af­ter they got the ini­tial writ­ten re­spons­es on the time­line from the FDA. But along with the note on their cur­rent view re­gard­ing Phase II’s ex­plorato­ry sta­tus, the com­pa­ny adds:

Con­sis­tent with this state­ment, the FDA fur­ther in­formed us that it has sig­nif­i­cant con­cerns re­gard­ing the abil­i­ty of the Phase 2 tri­al by it­self to pro­vide sub­stan­tial ev­i­dence of safe­ty and ef­fec­tive­ness and that the FDA gen­er­al­ly re­quires at least two ad­e­quate and well-con­trolled clin­i­cal tri­als to pro­vide sub­stan­tial ev­i­dence of ef­fec­tive­ness for sub­mis­sion of a mar­ket­ing ap­pli­ca­tion

Their gene ther­a­py is de­signed to dis­patch the AADC gene di­rect­ly in­to neu­rons of the puta­men where dopamine re­cep­tors are lo­cat­ed, en­abling the neu­rons to ex­press the AADC en­zyme and con­vert lev­odopa in­to dopamine to bet­ter con­trol symp­toms of the dis­ease.

In an up­date on their Phase Ib tri­al, the biotech sep­a­rate­ly not­ed that they picked their Phase II dose af­ter see­ing re­sults for the two high­est dose co­horts in their study, which in­clud­ed a small group of 10 pa­tients.

This com­bined analy­sis demon­strat­ed an in­crease from base­line in good ON time of 2.4 hours per day at 12 months, the time­point for the pri­ma­ry end­point in the Phase 2 tri­al, and 2.6 hours per day at 18 months, the lat­est time­point mea­sured for both co­horts. Of the com­bined ten pa­tients in Co­horts 2 and 3, sev­en pa­tients would be el­i­gi­ble for the Phase 2 tri­al based on lim­its in sever­i­ty of dysk­i­ne­sia and min­i­mum OFF time at base­line. For these sev­en pa­tients, the Phase 2 tri­al rel­e­vant group, the im­prove­ments in good ON time were 2.8 hours at 12 months and 2.5 hours at 18 months. These re­sults were achieved with clin­i­cal­ly mean­ing­ful and sus­tained re­duc­tions in dai­ly oral lev­odopa and re­lat­ed med­ica­tions.

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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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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Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

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.

Den­mark's Gen­mab hits the jack­pot with $500M+ US IPO as small­er biotechs rake in a com­bined $147M

Danish drugmaker Genmab A/S is off to the races with perhaps one of the biggest biotech public listings in decades, having reaped over $500 million on the Nasdaq, as it positions itself as a bonafide player in antibody-based cancer therapies.

The company, which has long served as J&J’s $JNJ key partner on the blockbuster multiple myeloma therapy Darzalex, has asserted it has been looking to launch its own proprietary product — one it owns at least half of — by 2025.

FDA over­rides ad­comm opin­ions a fifth of the time, study finds — but why?

For drugmakers, FDA advisory panels are often an apprehended barometer of regulators’ final decisions. While the experts’ endorsement or criticism often translate directly to final outcomes, the FDA sometimes stun observers by diverging from recommendations.

A new paper out of Milbank Quarterly put a number on that trend by analyzing 376 voting meetings and subsequent actions from 2008 through 2015, confirming the general impression that regulators tend to agree with the adcomms most of the time — with discordances in only 22% of the cases.

UP­DAT­ED: With loom­ing ‘apoc­a­lypse of drug re­sis­tance,’ Mer­ck’s com­bi­na­tion an­tibi­ot­ic scores FDA ap­proval on two fronts

Merck — one of the last large biopharmaceuticals companies in the beleaguered field of antibiotic drug development — on Wednesday said the FDA had sanctioned the approval of its combination antibacterial for the treatment of complicated urinary tract and intra-abdominal infections.

To curb the rise of drug-resistant bacteria and maintain the efficacy of the therapy, Recarbrio (and other antibacterials) — the drug must be used to treat or prevent infections that are proven or strongly suspected to be caused by susceptible gram-negative bacteria, Merck $MRK said.

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