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.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP)

Can a smart­phone app de­tect Covid? Pfiz­er throws down $116M to find out

What can a cough say about a patient’s illness? Quite a bit, according to ResApp Health — and Pfizer’s listening.

The pharma giant is shelling out about $116 million ($179 million AUD) to scoop up the University of Queensland spinout and its smartphone technology that promises to diagnose Covid and other respiratory illnesses based on cough and breathing sounds, the university announced last week.

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Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

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Rob Etherington, Clene CEO

Star­tup's gold nanocrys­tal ALS drug flops a PhII tri­al, a re­minder of the dis­ease's ob­sta­cles de­spite Amy­lyx OK

Despite the FDA approving an ALS drug for the first time in five years last week, the disease continues to fluster researchers, and another biotech is feeling the pain of a mid-stage failure.

Clene Nanomedicine reported early Monday that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. And in a press release, the company noted for the first time that it’s speaking with “potential strategic partners” about the program — language that typically indicates a biotech is preparing to sell off an asset.

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Marc Dunoyer, Alexion CEO (AstraZeneca via YouTube)

Up­dat­ed: As­traZeneca nabs a small rare dis­ease gene ther­a­py play­er for 667% pre­mi­um

AstraZeneca is kicking off the fourth quarter with a little M&A Monday for a gene editing player recently overcoming a second clinical hold to its only program in human studies.

The Big Pharma and its subsidiary Alexion are buying out little LogicBio for $2.07 per share. That’s good for a massive 667% premium over its Friday closing price, when it headed into the weekend at 27 cents and just weeks after Nasdaq said LogicBio would have to delist, which has been put on hold as the biotech requests a hearing. It’s one of two biotech deals to commence October, alongside the news of Incyte buying a vitiligo-focused biotech.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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An­oth­er warn­ing let­ter for Lupin as FDA iden­ti­fies de­fi­cien­cies at In­dia-based site

With few new details of what needs fixing, Lupin disclosed last week that the FDA recently sent a warning letter to its Tarapur, India-based site.

After an inspection from March 22 to April 4, Lupin disclosed in an April stock filing that it received a Form 483 with four observations, but it didn’t offer any details on the observations.

Similar to comments made in April, the company said last week it does not believe the FDA slap will disrupt its drug supplies or the existing revenues from operations of this facility.

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