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.

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.

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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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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Scott Gottlieb, AP Images

Scott Got­tlieb has a new board po­si­tion to add to the re­sume — and this one is fo­cused on a fa­vorite sub­ject

Scott Gottlieb has another position to add to his lengthy roster of boards and advisory roles in the wake of his departure from the helm of the FDA.

He’ll be joining the advisory board of FasterCures, a think tank which former junk bond king Michael Milken set up to help drive more drugs to the market, looking to accelerate drug R&D. That’s a subject close to the heart of Gottlieb, who blazed a trail at the FDA focused on hustling up the process. That helped endear him to the industry, making him one of the most popular commissioners in FDA history.

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Karyopharm lines up $150 mil­lion cash in­jec­tion to back con­tro­ver­sial drug launch

Karyopharm has entered into a royalty agreement worth up to $150 million to back the launch of their multiple myeloma drug — recently approved by the FDA over the objections of a majority of the agency’s outside experts.

The deal with HealthCare Royalty Partners, worth $75 million now and $75 million once certain regulatory and commercial milestones have been reached, will fund the commercialization of Karyopharm’s oral SINE compound Xpovio (selinexor) for patients with multiple myeloma who have already had at least four prior therapies. The money will help Karyopharm as it markets its newly approved drug and pushes through clinical trials testing the drug on refractory multiple myeloma patients with one to three therapies and patients with treatment-resistant diffuse large B-cell lymphoma. It will give Karyopharm a cushion through mid-2021.

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CTLA-4/PD-(L)1 combinations have been one of the El Dorados of oncology, its promise forever behind that next hill but apparently unattainable after a series of pivotal clinical failures. But researchers at New York’s Memorial Sloan Kettering Cancer Center and the Technical University of Munich think they may know how to fix what’s wrong and boost the drive to next-gen cancer combos.

In a preclinical animal research program, researchers found that within a cell, checkpoints rely on a specific molecule — RNA-sensing molecule RIG-I — to work. If that sounds familiar, it’s because it has already been identified as a target for boosting immune responses and was subject to at least one Phase I/II trial. Pfizer in December allied itself with Kineta with $15 million upfront and $505 million in potential milestones to develop RIG-I immunotherapies, and three years ago Merck purchased German upstart Rigontec for $137 million upfront and over $400 million in potential milestones for the same purpose.

Pur­due Phar­ma files for bank­rupt­cy as first step in $10B opi­oid set­tle­ment

It’s settled. Purdue Pharma has filed for bankruptcy as part of a deal that would see the OxyContin maker hand over $10 billion in cash and other contributions to mitigate the opioid crisis — without acknowledging any wrongdoing in the protracted epidemic that’s resulted in hundreds of thousands of deaths.

The announcement came two weeks after news of a proposed settlement surfaced and largely confirm what’s already been reported.

Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

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