EIP Phar­ma flies in­to a high-risk PhI­Ib Alzheimer’s study fu­eled with $20.5M from bil­lion­aire Len Blavat­nik’s Ac­cess In­dus­tries

John Alam

Un­de­terred by the string of fail­ures plagu­ing Alzheimer’s drug de­vel­op­ment, a young start­up based in Cam­bridge, MA has tapped an un­con­ven­tion­al source for $20.5 mil­lion to ad­vance a re­pur­posed drug in the clin­ic.

The new funds — com­ing from bil­lion­aire Len Blavat­nik’s Ac­cess In­dus­tries — will pow­er EIP Phar­ma’s Phase IIb study of ne­flamapi­mod, a p38 ki­nase in­hibitor in-li­censed from Ver­tex Phar­ma. Named RE­VERSE-SD, the high-risk tri­al will mea­sure im­prove­ment in episod­ic mem­o­ry as a pri­ma­ry end­point and have sev­er­al sec­ondary end­points in­clud­ing Clin­i­cal De­men­tia Rat­ing Scale Sum-of-Box­es, Wech­sler Mem­o­ry Scale, and spinal flu­id bio­mark­ers of dis­ease pro­gres­sion.

The fo­cus here is on cog­ni­tion — a gold stan­dard cheered on by the FDA in a set of guid­ance doc­u­ments re­leased in Feb­ru­ary. They came just in time for EIP Phar­ma, which launched RE­VERSE-SD in March.

“That’s the first time they’ve said that be­cause they rec­og­nize that it’s re­al­ly hard in the ear­ly stages of Alzheimer’s and in mild Alzheimer’s dis­ease to show an ef­fect on, for ex­am­ple, rates of go­ing in­to a nurs­ing home, be­cause that hap­pens lat­er on,” CEO John Alam tells me. “What mat­ters to pa­tients — when they first get di­ag­nosed — is the mem­o­ry func­tion.”

By tar­get­ing dys­func­tion­al neu­rons and the synaps­es that con­nect them, Alam be­lieves, their drug can di­rect­ly ad­dress mem­o­ry deficits. As such, their study will be “re­al­ly well aligned with the new FDA guid­ance doc­u­ment in terms of how we de­fine ear­ly Alzheimer’s dis­ease and how we’re mea­sur­ing drug ef­fect,” says Alam, a for­mer Ver­tex CMO who head­ed Sanofi’s ag­ing unit be­fore mov­ing on to start EIP Phar­ma.

Len Blavat­nik

By the time EIP was found­ed in 2014, Ver­tex was al­ready well in­to mid-stage de­vel­op­ment with ne­flamapi­mod, al­beit in non-CNS dis­or­ders. Hav­ing in­her­it­ed full sets of an­i­mal test­ing, Phase I and Phase II clin­i­cal da­ta — along­side an es­tab­lished man­u­fac­tur­ing process — the biotech went straight to Phase IIa, which yield­ed pos­i­tive re­sults ear­li­er this year.

That’s ad­vanced com­pared to the new­com­ers work­ing on nov­el path­ways in Alzheimer’s — a high pro­file start­up, De­nali Ther­a­peu­tics, has on­ly re­cent­ly be­gan Phase I test­ing; Rodin Ther­a­peu­tics, which is work­ing on synap­tic re­silience, has yet to en­ter the clin­ic — and Alam was quick to point it out.

“The things that are in Phase III to­day, the idea for them and con­cep­tion work­ing on them start­ed 10 or more years ago,” he says. “What we’re work­ing on, and the mech­a­nism we’re work­ing on, is tru­ly the state of the art sci­ence of our un­der­stand­ing of [how] ac­tu­al­ly mem­o­ry deficits form.”

Ground­work laid by Ver­tex had al­so al­lowed EIP Phar­ma to be cap­i­tal ef­fi­cient pri­or to this point, en­list­ing a small group of 10 in­di­vid­ual in­vestors to back their ear­li­er study. And it’s that pri­vate net­work that con­nect­ed Alam’s team to Ac­cess In­dus­tries, the first in­sti­tu­tion­al backer.

While Ac­cess had not been known for mak­ing a lot of life sci­ence in­vest­ments, Blavat­nik, the ec­cen­tric bil­lion­aire founder of Ac­cess, had backed biotechs through Clal Bio­sciences and dished out bio­med­ical sci­ence grants at top in­sti­tu­tions around the world. He was, how­ev­er, not di­rect­ly in­volved in the round.

Ac­cess could not be reached for com­ment. Ac­cord­ing to Alam, the two par­ties bond­ed over a vi­sion for the long term — a work­ing drug for Alzheimer’s pa­tients, not a quick ex­it.

Down the line, EIP Phar­ma’s ap­proach to synap­tic dys­func­tion could touch “more or less every CNS dis­or­der,” from Parkin­sons and Hunt­ing­ton’s dis­ease to autism and stroke re­cov­ery mech­a­nisms. But for now, they will con­cen­trate on chas­ing the suc­cess that’s elud­ed one big play­er af­ter an­oth­er.

“We have a re­al­ly good shot,” Alam says.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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Robert Califf (Pablo Martinez Monsivais, AP Images, File)

As buzz on Califf FDA nom heats up, in­dus­try and agency in­sid­ers of­fer a strong nod for the ‘per­fect’ choice

For once in this long, dramatic road to finding a new FDA commissioner, there’s been some continuity. Both CNN and Politico reported this weekend that Rob Califf met with President Biden to discuss the permanent commish role, following earlier news broken by the Washington Post that all signs point to Califf.

Although there may be a few Democrats who continue to grandstand about the dangers of COI (Califf has worked for Verily, sits on the board of Centessa Pharmaceuticals, and has other ties to industry research), with the pandemic ongoing and the need for some kind of continuity at FDA mounting, Califf is likely to meet the same fate as when he first won Senate confirmation in 2016, by a vote of 89-4 — Bernie Sanders and 6 others didn’t vote.

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AstraZeneca CEO Pascal Soriot (Raphael Lafargue/Abaca/Sipa USA)

A com­bo of As­traZeneca's Imfinzi and chemo wins where oth­ers have failed in piv­otal bil­iary tract test

Looking to run with the big dogs in the PD-(L)1 class, AstraZeneca’s Imfinzi has a tall hill to climb to compete in an increasingly bustling market. An aggressive combo strategy for the drug has paid off so far, and now AstraZeneca is adding another notch to its belt.

A combo of Imfinzi (durvalumab) and chemotherapy significantly extended the lives of first-line patients with advanced biliary tract cancer over chemo alone, according to topline results from the Phase III TOPAZ-1 study revealed Monday.

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Sean Ianchulev, Eyenovia CEO and CMO

Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

Back in April, the FDA lost a crucial court case in which its broad discretion of regulating medical products that might satisfy the legal definitions of either “drug” and/or “medical device” was sharply curtailed.

In addition to the appeals court ruling that Genus Medical Technologies’ contrast agent barium sulfate (aka Vanilla SilQ) should not be considered a drug, as the FDA had initially ruled, but as a medical device, the agency also was forced to spell out which drugs would transition to devices as a result of the ruling.

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Peter Greenleaf, Aurinia CEO

Af­ter pass­ing on Ac­celeron, Bris­tol My­ers eyes bolt-on ac­qui­si­tion of au­toim­mune spe­cial­ist — re­port

Bristol Myers Squibb is looking to beef up its autoimmune portfolio by scooping up Aurinia Pharmaceuticals, Bloomberg reported.

The recent overtures to Aurinia, relayed by anonymous insiders, came just as Bristol Myers turned down buyout talks with partners at Acceleron — which Merck ultimately struck a deal to acquire for $11.5 billion. Bristol Myers has reportedly decided to cash out on its minority stake, likely bagging $1.3 billion in the process, while keeping the royalty deals on two of Acceleron’s blood disorder drugs.

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So — that pig-to-hu­man trans­plant; Po­ten­tial di­a­betes cure reach­es pa­tient; Ac­cused MIT sci­en­tist lash­es back; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We’re incredibly excited to welcome Beth Bulik, seasoned pharma marketing reporter, to the team. You can find much of her work in our new Marketing channel — and in her weekly newsletter, Endpoints PharmaRx, which will launch in early November. Add it to your subscriptions here.

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NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

No, sci­en­tists are not any clos­er to pig-to-hu­man trans­plants than they were last week

Steve Holtzman was awoken by a 1 a.m. call from a doctor at Duke University asking if he could put some pigs on a plane and fly them from Ohio to North Carolina that day. A motorcyclist had gotten into a horrific crash, the doctor explained. He believed the pigs’ livers, sutured onto the patient’s skin like an external filter, might be able to tide the young man over until a donor liver became available.

UP­DAT­ED: Agenus calls out FDA for play­ing fa­vorites with Mer­ck, pulls cer­vi­cal can­cer BLA at agen­cy's re­quest

While criticizing the FDA for what may be some favoritism towards Merck, Agenus on Friday officially pulled its accelerated BLA for its anti-PD-1 inhibitor balstilimab as a potential second-line treatment for cervical cancer because of the recent full approval for Merck’s Keytruda in the same indication.

The company said the BLA, which was due for an FDA decision by Dec. 16, was withdrawn “when the window for accelerated approval of balstilimab closed,” thanks to the conversion of Keytruda’s accelerated approval to a full approval four months prior to its PDUFA date.

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