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.

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Olivier Brandicourt (AP Images)

Ex-Sanofi chief Olivi­er Brandi­court, cur­rent Black­stone ad­vi­sor, jumps on Al­ny­lam board

Former Sanofi chief Olivier Brandicourt, who departed his post with an unexpected early retirement last year, has made his move — as most C-suite executives inevitably do — to become a director on the board of a biopharma company.

RNAi player Alnylam is Brandicourt’s destination. Meanwhile, the Cambridge, Massachusetts-based drugmaker — which pioneered the first approval in the field — also disclosed the retirement of Alnylam co-founder Dr. Paul Schimmel from its board.

Jim Wilson's gene ther­a­py start­up Pas­sage Bio bucks mar­ket sen­ti­ments, rais­ing up­sized $216M IPO

A coronavirus fear-induced bloodbath on the Nasdaq has not stopped Passage Bio from making a public debut — and an exuberant one.

By pricing an upsized offering at $18, the top of the range, the gene therapy biotech bagged $216 million from its IPO, 72% more than it’s originally penciled in.

The proceeds likely reflected confidence in Jim Wilson, who gathered all the tools he’s built over decades of gene therapy research to assemble the startup and teamed up with Frazier and OrbiMed to hone its focus on rare, monogenic disorders of the central nervous system. Just before the IPO, Deerfield partner Bruce Goldsmith took over from OrbiMed’s Stephen Squinto as CEO.

Dan O'Day (AP Images)

UP­DAT­ED: A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisition, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

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Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

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Spark los­es an­oth­er top ex­ec in the wake of $4.3B takeover by Roche — re­port

Days after bidding farewell to co-founder Kathy High, Spark Therapeutics — now operating under Roche — has one more opening on its C-suite.

Kathy Reape

Kathy Reape, who joined the Philadelphia-based biotech in 2016 as head of clinical R&D and became chief medical officer in 2018, is reportedly set to leave.

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Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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'The head­lines are the head­lines, but': Bio­Marin talks up po­ten­tial sav­ings as he­mo­phil­ia gene ther­a­py launch looms

BioMarin execs are still staying tight-lipped about their pricing plans for what is poised to be the world’s first hemophilia gene therapy. But as the company enters the final regulatory stretch and approaches a potential launch this summer, they are also dropping more hints to get investors ready.

First thing to know: They really, really don’t expect an advisory committee to be convened for valrox, which is under priority review, to pop up before its PDUFA date on August 21.

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