Lit­tle Tetra is prep­ping a PhII Alzheimer’s study. In fact, they just got $40M to fund it

Af­ter all the no­to­ri­ous late-stage fail­ures in Alzheimer’s over the past year, you could say it’s con­sid­er­ably hard­er to win peo­ple over to a new mech­a­nism of ac­tion for the mem­o­ry-wast­ing plague.

But Mark Gur­ney isn’t let­ting a lit­tle neg­a­tiv­i­ty stop him now.

The CEO of Tetra Dis­cov­ery Part­ners in Grand Rapids, MI, be­lieves he and his 11-mem­ber team can ac­com­plish what the ma­jor league play­ers with far big­ger op­er­a­tions and a whole lot more mon­ey have failed at. And to­day he has an ex­tra $40 mil­lion in hard cash to help pay for the mid-stage tri­al that’s need­ed to help prove whether it works in pa­tients.

Sh­iono­gi is hand­ing over a very mod­est $5 mil­lion up­front and a more sub­stan­tial $35 mil­lion for an eq­ui­ty stake in Tetra in ex­change for a re­gion­al set of Asian rights to BPN14770, a PDE4D al­losteric in­hibitor for Alzheimer’s and Frag­ile X dis­ease. Aside from their shot at an his­toric break­through against some of the tough­est odds in R&D, Sh­iono­gi — which has a long­stand­ing in­ter­est in neu­ronal R&D — is al­so on the hook for $120 mil­lion in mile­stones plus roy­al­ties.


“This is a non-amy­loid mech­a­nism, not pre­vi­ous­ly ex­plored in hu­mans,” Gur­ney tells me ear­ly on in our con­ver­sa­tion, putting some quick dis­tance be­tween his work and the land­mark fail­ures that have cast doubt on the amy­loid the­o­ry.

The work is based on ob­ser­va­tions of cog­ni­tive re­silience in pa­tients who have clas­sic bio­mark­ers for the dis­ease — amy­loid and tau — with­out demon­strat­ing any symp­toms. 

By se­lec­tive­ly in­hibit­ing PDE4D — which falls un­der a well-known mech­a­nism of ac­tion — Tetra will set out to prove in a loom­ing Phase II that their ap­proach can bol­ster neu­ronal con­nec­tions, pro­tect­ing them from dam­age and im­prov­ing the prospects of ear­ly-stage pa­tients.

Gur­ney be­lieves their drug can im­prove symp­toms of the dis­ease over a 3-month span, but even a sig­nif­i­cant im­prove­ment in the de­cline of pa­tients — or as much as a flatlin­ing on de­te­ri­o­ra­tion — would be greet­ed with con­sid­er­able en­thu­si­asm.

He got here with an aw­ful lot of help from grants and con­tracts with the NIH, which pro­vid­ed the li­on’s share of the $30 mil­lion they’ve need­ed so far. There was al­so $7.3 mil­lion in A-round cash by late 2016 from Apjohn Group, Grand An­gels, Dol­by Fam­i­ly Ven­tures and the Alzheimer’s Drug Dis­cov­ery Foun­da­tion.

Gur­ney was en­gaged ear­ly in the dis­cov­ery of be­ta-sec­re­tase, a field in amy­loid re­search that in­spired huge in­vest­ments and colos­sal fail­ures. He al­so was a se­nior in­ves­ti­ga­tor at de­CODE. Scott Reines, the CMO, has held se­nior posts in neu­ro­sciences R&D at J&J and Mer­ck.

There are PDE4s on the mar­ket as an­ti-in­flam­ma­to­ries, of course, car­ry­ing no­table names like apre­mal­ist. But the broad­band in­hi­bi­tion of PDE4 has al­so been linked with tox­i­c­i­ty. Tetra’s goal was to find a more se­lec­tive ap­proach in neu­ro­sciences — where in­flam­ma­tion it­self is a grow­ing tar­get — while al­so re­serv­ing a sep­a­rate pro­gram (PDE4B)that is specif­i­cal­ly a next-gen ap­proach to mega-block­buster in­flam­ma­to­ry dis­eases like pso­ri­a­sis.

That’s al­so no easy task.

We’ve been here with oth­er new drugs of course, many times; wait­ing it out through a mid-stage or piv­otal study to demon­strate whether a 5HT6 can guard cog­ni­tion — on­ly to watch one pro­gram af­ter an­oth­er go down in flames un­til the tar­get it­self is wiped off the R&D map. Ax­o­vant’s crown­ing fail­ure af­ter re­peat­ed ex­pres­sions of op­ti­mism like­ly put the ki­bosh on that one.

Gur­ney is used to be­ing greet­ed with skep­ti­cism. That goes with the ter­ri­to­ry for any­one work­ing in Alzheimer’s R&D to­day.

Now that he has the deal he need­ed to do the Alzheimer’s study, which will fol­low a crit­i­cal Phase II in Frag­ile X, he can en­dure the head­winds bet­ter. Af­ter­wards, if he’s proved right, there should be no prob­lem find­ing a part­ner for a piv­otal tri­al in Azheimer’s. Frag­ile X is the kind of rare in­di­ca­tion they can go it alone on. 

But the lit­tle team has some very big hur­dles to clear first.

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

Endpoints News writer Nicole DeFeudis has posted a snapshot of all the companies, universities and hospital-based groups now racing through the clinic, ranking them according to their place in the pipeline as well as the latest remarks available on timelines. And we’ll keep this lineup updated right through the end of the year, as the checkered flags start to fall, possibly as early as October.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Eisai moves to 200 Metro Blvd. by late 2021 (ON3)

Ei­sai is cre­at­ing a new US cor­po­rate, R&D HQ in Roche’s old Nut­ley, NJ cam­pus

Eight years after Roche pulled up stakes from Nutley, NJ in a major R&D reorganization, Japan’s Eisai is moving its US corporate and research hub into their old campus.

Now the ON3 property, Eisai — a longtime Biogen partner focused on neurodegenerative disorders like Alzheimer’s — will bring together a staff of up to 1,200 employees. And execs are pitching the move to the New Jersey campus as a cultural game-changer.

Bing Li, Debra Yu and Konstantin Poukalov, LianBio

Per­cep­tive births its first in-house start­up — and it's a Chi­na play

Perceptive Advisors is going to China.

The decision dates back two years, chief investment officer Adam Stone tells Endpoints News, when the firm began to figure out how it can, in hedge fund-speak, strategically increase its exposure to a growing biopharma market poised to be a key geographic area in the next several decades. It was a bit of a blindspot for Perceptive, he admits.

As “globalized scientist-investors, we just couldn’t afford to have that blindspot in place,” he says.

Ugur Sahin, BioNTech CEO

Covid-19 roundup: Pfiz­er-backed BioN­Tech plans to seek FDA OK for a new vac­cine 'as ear­ly as' Oc­to­ber — ahead of the elec­tion

BioNTech execs say they’re on track to get their late-stage data on a Covid-19 vaccine — partnered with Pfizer — into the hands of regulators as early as October.

In their Q2 release Tuesday morning, the biotech reported that investigators could have late-stage data as early as October, and they won’t be wasting any time in hustling that over to the FDA.

“I am incredibly proud of our team, who has worked tirelessly to initiate our BNT162 Phase 2b/3 trial in record time and put us in a position to seek regulatory review as early as October of this year, if our trials are successful,” said Ugur Sahin, BioNTech’s CEO and co-founder.

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DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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