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.

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

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San Diego cou­ple charged with steal­ing trade se­crets, open­ing Chi­nese biotech as DOJ crack­down con­tin­ues

A San Diego couple has been charged with stealing trade secrets from a US hospital and opening a business based off those secrets in China as the controversial industry-wide crackdown on alleged corporate espionage continues. On the same day, the Department of Justice announced they had arrested Beijing representative Zhongsan Liu for allegedly trying to obtain research visas for government recruiters.

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Deborah Dunsire. Lundbeck

UP­DAT­ED: Deb­o­rah Dun­sire is pay­ing $2B for a chance to leap di­rect­ly in­to a block­buster show­down with a few of the world's biggest phar­ma gi­ants

A year after taking the reins as CEO of Lundbeck, Deborah Dunsire is making a bold bid to beef up the Danish biotech’s portfolio of drugs in what will likely be a direct leap into an intense rivalry with a group of giants now carving up a growing market for new migraine drugs.

Bright and early European time Monday morning the company announced that it will pay up to about $2 billion to buy Alder, a little biotech that is far along the path in developing a quarterly IV formulation of a CGRP drug aimed at cutting back the number of crippling migraines patients experience each month. In a followup call, Dunsire also noted that the company will likely need 200 to 250 reps for this marketing task on both sides of the Atlantic. And analysts were quick to note that the dealmaking at Lundbeck isn’t done, with another $2 billion to $3 billion available for more deals to beef up the pipeline.

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Tower Bridge in London [Shutterstock]

#UK­BIO19: Join GSK’s Hal Bar­ron and a group of top biotech ex­ecs for our 2nd an­nu­al biotech sum­mit in Lon­don

Over the past 10 years I’ve made a point of getting to know the Golden Triangle and the special role the UK biopharma industry plays there in drug development. The concentration of world class research institutes, some of the most accomplished scientists I’ve ever seen at work and a rising tide of global investment cash leaves an impression that there’s much, much more to come as biotech hubs are birthed and nurtured.

UP­DAT­ED: Bio­gen pulls the plug on prized IPF drug from $562M+ Stromedix buy­out

One of Biogen’s attempts to branch out has flopped as the biotech scraps a mid-stage program for idiopathic pulmonary fibrosis.

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Warts for the win: Aclar­is' lead drug clears piv­otal study

Aclaris Therapeutics has found a way to get rid of the warts and all.

The company — which earlier this month decided to focus on its arsenal of kinase inhibitors — on Monday unveiled positive data from a pivotal study testing its lead experimental drug for use in common warts.

The drug, A-101, was tested in a 502-patient study called THWART-2 — patients enrolled had one to six warts before qualifying for the trial. Patients either self-administered A-101 topical solution or a vehicle twice a week over a two-month period. A higher proportion of patients on the drug (a potent hydrogen peroxide topical solution) saw their warts disappear at day 60, versus the vehicle (p<0.0001) — meeting the main goal of the study.  Each secondary endpoint also emerged in favor of A-101, the company said.

Charles Nichols, LSU School of Medicine

Could psy­che­delics tack­le the obe­si­ty cri­sis? A long­time re­searcher in the field says his lat­est mouse study sug­gests po­ten­tial

Psychedelics have experienced a renaissance in recent years amid a torrent of preclinical and clinical research suggesting it might provide a path to treat mood disorders conventional remedies have only scraped at. Now a preclinical trial from a young biotech suggests at least one psychedelic compound has effects beyond the mind, and — if you believe the still very, very early hype — could provide the first single remedy for some of the main complications of obesity.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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Ac­celeron drops a de­vel­op­ment pro­gram as #2 drug fails to spark func­tion­al ben­e­fits in pa­tients with a rare neu­ro­mus­cu­lar ail­ment

Acceleron is scrapping a muscular dystrophy development program underway for its number 2 drug in the pipeline after pouring over some failed mid-stage secondary data.

Gone is the ACE-083 project in patients with facioscapulohumeral muscular dystrophy. Their drug hit the primary endpoint on building muscle but flopped on key secondaries for functional improvements in patients, which execs felt was vital to the drug’s success.