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.

Tar­get­ing a Po­ten­tial Vul­ner­a­bil­i­ty of Cer­tain Can­cers with DNA Dam­age Re­sponse

Every individual’s DNA is unique, and because of this, every patient responds differently to disease and treatment. It is astonishing how four tiny building blocks of our DNA – A, T, C, G – dictate our health, disease, and how we age.

The tricky thing about DNA is that it is constantly exposed to damage by sources such as ultraviolet light, certain chemicals, toxins, and even natural biochemical processes inside our cells.¹ If ignored, DNA damage will accumulate in replicating cells, giving rise to mutations that can lead to premature aging, cancer, and other diseases.

Roivant par­lays a $450M chunk of eq­ui­ty in biotech buy­out, grab­bing a com­pu­ta­tion­al group to dri­ve dis­cov­ery work

New Roivant CEO Matt Gline has crafted an all-equity upfront deal to buy out a Boston-based biotech that has been toiling for several years now at building a supercomputing-based computational platform to design new drugs. And he’s adding it to the Erector set of science operations that are being built up to support their network of biotech subsidiaries with an eye to growing the pipeline in a play to create a new kind of pharma company.

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Fol­low biotechs go­ing pub­lic with the End­points News IPO Track­er

The Endpoints News team is continuing to track IPO filings for 2021, and we’ve designed a new tracker page for the effort.

Check it out here: Biopharma IPOs 2021 from Endpoints News

You’ll be able to find all the biotechs that have filed and priced so far this year, sortable by quarter and listed by newest first. As of the time of publishing on Feb. 25, there have already been 16 biotechs debuting on Nasdaq so far this year, with an additional four having filed their S-1 paperwork.

Ken Frazier, Merck CEO (Bess Adler/Bloomberg via Getty Images)

UP­DAT­ED: Mer­ck takes a swing at the IL-2 puz­zle­box with a $1.85B play for buzzy Pan­dion and its au­toim­mune hope­fuls

When Roger Perlmutter bid farewell to Merck late last year, the drugmaker perhaps best known now for sales giant Keytruda signaled its intent to take a swing at early-stage novelty with the appointment of discovery head Dean Li. Now, Merck is signing a decent-sized check to bring an IL-2 moonshot into the fold.

Merck will shell out roughly $1.85 billion for Pandion Pharmaceuticals, a biotech hoping to gin up regulatory T cells (Tregs) to treat a range of autoimmune disorders, the drugmaker said Thursday.

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Per­cep­tive's fourth — yes, fourth — SPAC jumps to Nas­daq as the blank check tree con­tin­ues to ripen

The biotech SPAC boom has gone almost hand-in-hand with the industry’s IPO gold rush, and this week saw more blank check companies hop aboard the train.

Leading the way is Perceptive Advisors’ fourth SPAC, appropriately named Arya Sciences Acquisition IV, which priced Friday morning after raising $130 million. And on top of that, new Ziopharm executive chair James Huang is launching his own SPAC with MSD Partners and Panacea Venture, filing S-1 paperwork Thursday with plans to raise $200 million.

CEO Fred Aslan (Artiva)

NK cell ther­a­py play­er Arti­va makes some more noise, pulling in $120M Se­ries B less than a month af­ter Mer­ck deal

Not even one month after Big Pharma took notice of Artiva when Merck signed a collaboration worth nearly $2 billion in milestones, the off-the-shelf NK cell biotech already has its next big fundraise.

Artiva returns from the venture well Friday with a $120 million Series B round, money they will use to get their first program into the clinic and to file INDs for another two candidates. The raise marks the latest development in a rapidly expanding footprint for Artiva, which, in addition to the Merck deal last month, has now raised almost $200 million since its Series A last June.

With dust set­tled on ac­tivist at­tack, Lau­rence Coop­er leaves Zio­pharm to a new board

Laurence Cooper has done his part.

In the five years since he left a tenured position at Houston’s MD Anderson Cancer Center to become CEO of Boston-based Ziopharm, he’s steered the small-cap immunotherapy player through patient deaths in trials, clinical holds, short attacks and, most recently, an activist attack on the board.

So when the company has “fantastic news” like an IND clearance for a TCR T cell therapy program, he’s ready to pass on the baton.

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Doug Ingram (file photo)

Why not? Sarep­ta’s third Duchenne MD drug sails to ac­cel­er­at­ed ap­proval

Sarepta may be running into some trouble with its next-gen gene therapy approach to Duchenne muscular dystrophy. But when it comes to antisense oligonucleotides, the well-trodden regulatory path is still leading straight to an accelerated approval for casimersen, now christened Amondys 45.

We just have to wait until 2024 to find out if it works.

Amondys 45’s approval was unceremonious, compared to its two older siblings. There was no controversy within the FDA over approving a drug based on a biomarker rather than clinical benefit, setting up a powerful precedent that still haunts acting FDA commissioner Janet Woodcock as biotech insiders weighed her potential permanent appointment; no drama like the FDA issuing a stunning rejection only to reverse its decision and hand out an OK four months later, which got more complicated after the scathing complete response letter was published; no anxious tea leaf reading or heated arguments from drug developers and patient advocates who were tired of having corticosteroids as their loved ones’ only (sometimes expensive) option.

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Steve Cutler, Icon CEO (Icon)

In the biggest CRO takeover in years, Icon doles out $12B for PRA Health Sci­ences to fo­cus on de­cen­tral­ized clin­i­cal work

Contract research M&A had a healthy run in recent years before recently petering out. But with the market ripe for a big buyout and the Covid-19 pandemic emphasizing the importance of decentralized trials, Wednesday saw a tectonic shift in the CRO world.

Icon, the Dublin-based CRO, will acquire PRA Health Sciences for $12 billion in a move that will shake up the highest rungs of a fragmented market. The merger would combine the 5th- and 6th-largest CROs by 2020 revenue, according to Icon, and the merger will set the newco up to be the second-largest global CRO behind only IQVIA.

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