PhI­II de­tails boost Acor­da as ex­ecs rush to FDA with the clock tick­ing on a time bomb

Ron Co­hen, Acor­da

Shares of Acor­da got a boost af­ter the mar­ket closed on Mon­day as in­vestors got a chance to look over the de­tailed Phase III da­ta for its in­haled lev­odopa drug CVT-301. And while we al­ready knew that the top-line da­ta on the pri­ma­ry end­point for Parkin­son’s dis­ease were pos­i­tive, it seemed to help that this late-stage ther­a­py ap­pears on track to ar­rive at the FDA lat­er this month as com­pa­ny ex­ecs look to re­place its big mon­ey­mak­er, now poised to lose patent pro­tec­tion.

Fail­ure, or even the hint of a set­back here, would be cat­a­stroph­ic. So the p=0.009 val­ue for a sta­tis­ti­cal­ly sig­nif­i­cant im­prove­ment in mo­tor func­tion among pa­tients ex­pe­ri­enc­ing OFF times was re­as­sur­ing. The switch from OFF to ON— mark­ing pe­ri­ods of the day when the drug is work­ing— and stay­ing ON at 60 min­utes, a key sec­ondary end­point, was al­so pos­i­tive. But the third step in the sec­ondary re­view ran in­to trou­ble, with no sig­nif­i­cant change to the Uni­fied Parkin­son’s Dis­ease Rat­ing Scale — III at 20 min­utes.

An­a­lysts al­so flagged some wor­ries about the lack of sig­nif­i­cant im­prove­ment in to­tal OFF time, which could crimp its roll­out, pro­vid­ed reg­u­la­tors of­fer a green light in the first place.

Acor­da’s shares jumped 10% af­ter in post-mar­ket trad­ing.

Paul Mat­teis at Leerink of­fered a quick thumbs up:

We be­lieve the full re­sult – con­veyed at the Move­ment Dis­or­ders So­ci­ety An­nu­al Meet­ing – sup­ports our fair­ly high, 90% odds of FDA ap­proval. With­in the full da­ta we are en­cour­aged with what looks to be an ac­cept­able safe­ty pro­file as well as a low, place­bo-like dropout rate. Sec­ondary end­points al­so most­ly trend or are clear­ly in fa­vor of drug – in­clud­ing the pa­tient glob­al im­pres­sion of change – though the lack of dif­fer­ence (p-val­ue of 0.98) on to­tal OFF time may emerge as a sub­ject of dis­cus­sion as in­vestors de­bate the size of the com­mer­cial op­por­tu­ni­ty. We mod­el ~$278MM in CVT-301 sales in 2022E, and we ex­pect the prod­uct to be ap­proved in the mid­dle of next year.

Acor­da shift­ed to sur­vival mode in ear­ly April af­ter a US dis­trict court tossed four key patents on its flag­ship drug Ampyra, leav­ing it with one to stand on in­to next year. The com­pa­ny chopped 20% of its staff as it scram­bles to re­struc­ture while gam­bling that it can field new drugs in short or­der.

But Acor­da CEO Ron Co­hen left his mar­ket­ing team in place, gam­bling that an on-time ap­proval would al­low for a quick hand­off of CVT-301 as they scram­ble to pre­vent a plunge in rev­enue. Ampyra pro­vides the bulk of the com­pa­ny’s mon­ey.

Even with high odds of an ap­proval, though, biotech is known for any num­ber of sna­fus that can af­flict the progress of a drug. And this time there’s no room for er­ror as they stay fo­cused on the high wire act.

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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