Lit­tle Alder bags pos­i­tive PhI­II mi­graine da­ta, but gi­ant ri­vals are rac­ing ahead in a crowd­ed field

Lit­tle Alder Bio­Phar­ma­ceu­ti­cals $AL­DR has racked up a slate of pos­i­tive late-stage da­ta for its Phase III study of a new CGRP mi­graine drug, but it will like­ly have trou­ble stand­ing out from some of the gi­ant play­ers that are al­ready out front in the race to reg­u­la­tors.

Randy Schatz­man

Dubbed PROMISE 1, in­ves­ti­ga­tors es­tab­lished a base­line av­er­age of 8.6 month­ly mi­graines in its group of fre­quent episod­ic mi­graine suf­fer­ers. Alder’s quar­ter­ly-dosed 300 mg and 100 mg cut that rate by 4.3 and 3.9 days. But the place­bo arm al­so ex­pe­ri­enced a hefty 3.2 day im­prove­ment, leav­ing the biotech with a sta­tis­ti­cal­ly sig­nif­i­cant but un­re­mark­able hit on the pri­ma­ry end­point.

In­vestors didn’t re­spond well to the da­ta, ei­ther. The shares dropped a painful 24% in pre-mar­ket trad­ing.

The Both­ell, WA-based biotech did flag some high­lights, though, in­clud­ing a 100% re­sponse among 20% of the pa­tients, there was a sig­nif­i­cant drop ear­ly on in the eptinezum­ab arm for mi­graines and a third achieved a 75% re­duc­tion in mi­graines in weeks 4 through 12. There was not, though, a sig­nif­i­cant drop in the 75% re­duc­tion group over­all for the 100 mg dose.

Alder’s big promise is that its quar­ter­ly IV in­fu­sion ther­a­py can beat or match oth­er drugs which are more fre­quent­ly dosed, mak­ing it an eas­i­er al­ter­na­tive pre­ferred by pa­tients. And it has plans for self-ad­min­is­tra­tion that could al­so po­si­tion the biotech against ri­vals, notes Leerink’s Paul Mat­teis. He adds:

The “high rate of “su­per re­spon­ders” (75% and 100% re­duc­tions)…looks very com­pet­i­tive com­pared to phase III re­sults for oth­er an­ti-CGRPs. While the ap­proval of the IV – pend­ing da­ta from PROMISE2 – is most­ly de­risked, the re­sults raise the ques­tion of what AL­DR will do with its self-ad­min­is­tra­tion as the high­er 300mg dose ap­pears bet­ter than 100mg. One of the most im­pres­sive dat­a­points was the speed-of-on­set: >50% of eptinezum­ab-treat­ed pa­tients had no mi­graines on day two of the study ver­sus 37% on place­bo; this was sta­tis­ti­cal­ly sig­nif­i­cant.

But this is a field where a full line­up of ma­jor league drug de­vel­op­ers has been cheer­ing a se­ries of achieve­ments. Te­va just days ago lined up a 1.5-day ad­van­tage for fre­manezum­ab, putting it in a mix of re­sults post­ed with an im­prove­ment for these drugs that tends to hov­er around the 2-day mark. And it has re­sults for month­ly and quar­ter­ly dos­ing.

Re­searchers are al­ways quick to protest any tri­al com­par­isons that aren’t head-to-head, and pa­tients pop­u­la­tions and dos­ing aren’t an even match in the stud­ies. But pay­ers will al­so be ex­pect­ed to con­sid­er Eli Lil­ly’s 2-day ad­van­tage, or a Phase III out­come for Am­gen and No­var­tis that was quite sim­i­lar to Alder’s for 70 mg erenum­ab, the ther­a­py that is the fur­thest out front. Al­ler­gan al­so has high hopes for its oral ther­a­py in-li­censed from Mer­ck for $250 mil­lion up­front.

Alder al­so has a ways to go in com­plet­ing its Phase III pro­gram.

“These pos­i­tive re­sults, con­sis­tent with pre­vi­ous­ly re­port­ed eptinezum­ab stud­ies, sup­port the unique clin­i­cal pro­file of eptinezum­ab as a po­ten­tial first-of-its-kind in­fu­sion ther­a­py to pre­vent mi­graines,” says Randy Schatz­man. “En­roll­ment is on track for PROMISE 2, our sec­ond piv­otal Phase III study that fo­cus­es on chron­ic mi­graine, and we re­main on track to sub­mit our BLA with the U.S. Food and Drug Ad­min­is­tra­tion (FDA) in the sec­ond half of 2018.”

What’s clear is that no mat­ter how this race ul­ti­mate­ly pans out, mi­graine suf­fer­ers will soon have plen­ty to pick from for a new stan­dard of care in the field. And Alder re­mains a key play­er among the de­vel­op­ers out to make a ma­jor dif­fer­ence for pa­tients.

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.

Endpoints News

Basic subscription required

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

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).

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.

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.

Endpoints News

Basic subscription required

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