Josh Cohen, Justin Klee

Armed with pos­i­tive ALS da­ta, Amy­lyx scores $30M in fresh fund­ing to com­plete Alzheimer's PhII

Four years af­ter an­nounc­ing them­selves to the biotech world with a new idea for drug­ging neu­rode­gen­er­a­tion, back­ing by the late Hen­ri Ter­meer and $5 mil­lion from Morn­ing­side Ven­ture, the young en­tre­pre­neurs at Amy­lyx are back for round 2.

Morn­ing­side con­tin­ued to lead the $30 mil­lion Se­ries B, with par­tic­i­pa­tion from Ter­meer’s wid­ow, Be­lin­da, and oth­er un­named in­vestors. Hav­ing cel­e­brat­ed a topline Phase II win for its lead pro­gram in amy­otroph­ic lat­er­al scle­ro­sis, Amy­lyx ex­pects the cash to fund talks with reg­u­la­tors as well as a sep­a­rate tri­al for the same drug in Alzheimer’s — for which they had just fin­ished en­rolling.

Jef­frey Trig­ilio

Aside from the CEN­TAUR tri­al, the theme at the biotech for the past year has been hir­ing. In­clud­ing the co-founders — CEO Josh Co­hen and pres­i­dent Justin Klee — and long­time CSO Kent Leslie, the team now con­sists of 15 full-time em­ploy­ees.

“Up un­til about a year ago, it was three peo­ple for about six years,” Jeff Trig­ilio, who joined as CFO in Feb­ru­ary, told End­points News.

Al­so new to the C-suite are bio­phar­ma vets like CMO Patrick Yerami­an and chief com­mer­cial of­fi­cer Mar­garet Olinger, who helped launch Soliris for Alex­ion. Tom Holmes was re­cruit­ed from Bio­gen to head up the glob­al sup­ply chain.

Patrick Yerami­an

Un­der Yerami­an, the clin­i­cal team has gen­er­at­ed da­ta show­ing that AMX0035 slowed ALS dis­ease pro­gres­sion by a sta­tis­ti­cal­ly sig­nif­i­cant mar­gin from place­bo.

“With these re­sults, Amy­lyx now has a re­spon­si­bil­i­ty to move ahead as ef­fi­cient­ly as pos­si­ble, as peo­ple liv­ing with ALS don’t have time to wait,” Klee said when the com­pa­ny re­leased the re­sults in De­cem­ber.

The drug has since been grant­ed or­phan sta­tus by the EMA. Stay tuned for the full da­ta and next steps, Trig­ilio said.

Mar­garet Olinger

As for the Alzheimer’s study, PE­GA­SUS, 96 of the pre­vi­ous­ly planned 100 pa­tients have been dosed and topline re­sults are ex­pect­ed in the first quar­ter of next year.

Here’s CSO Leslie for a re­fresh­er on how AMX-0035, which start­ed out as a re­search project at Brown, tar­gets neu­rode­gen­er­a­tion by go­ing af­ter neu­ronal apop­to­sis:

Many pre­vi­ous ap­proach­es have fo­cused on tar­get­ing the pathog­no­mon­ic fea­tures of Alzheimer’s Dis­ease, amy­loid and/or tau. We de­signed a neu­ro-pro­tec­tive in­ves­ti­ga­tion­al med­ica­tion, AMX0035, to tar­get key path­ways in the mi­to­chon­dria and en­do­plas­mic retic­u­lum which are thought to dri­ve cel­lu­lar death. Amy­lyx’s ap­proach is ir­re­spec­tive of amy­loid or tau, tar­get­ing neu­ronal dam­age path­ways that may be down­stream of these species but al­so which may arise due to oth­er cel­lu­lar or en­vi­ron­men­tal fac­tors.

Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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Ver­sant de­buts Ridge­line's start­up #4, armed with $30M and al­ter­na­tive TCR cell ther­a­pies for sol­id tu­mors

For all the iterations and advances in TCR therapies for cancer, any experimental treatments involving T cell receptors share one trait: By definition, they only recognize antigens presented as peptides on the major histocompatibility complex (MHC) on cells.

Versant reckons it’s time to expand the arsenal. With $30 million in initial funding, its Ridgeline Discovery Engine in Switzerland has been working on a non-peptidic approach that it says has tumor-agnostic potential, especially in solid tumors. They’ve named it Matterhorn, after a Swiss mountain as they did with the three other companies that have emerged from the Basel-based incubator.

UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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CF Foun­da­tion, Long­wood team on new in­cu­ba­tor for com­pa­nies with cut­ting-edge CF treat­ments

Nine months after launching a $500 million hunt for a cure for cystic fibrosis, the Cystic Fibrosis Foundation said it will use a portion of those funds to do something it has never done before: help launch new companies.

The CF Foundation, whose venture philanthropy efforts helped fund Vertex’s line of powerful CF drugs, is teaming with Longwood Fund to create a CF incubator. The incubator will identify new companies with platforms or technologies that can be applied in the rare genetic condition. The partners can then finance early development in exchange for a commitment from the companies to focus on applications in cystic fibrosis.

Kiersten Stead, John Hamer (DCVC Bio)

Deep tech, round 2: DCVC Bio bags $350M fund to chase the tip of the life sci­ence spear

It took one trip from San Francisco to Vancouver for Kiersten Stead and her DCVC Bio crew to feel confident about throwing their weight — and cash — behind AbCellera.

CEO Carl Hansen’s academic background and the potential of the platform, which combined machine vision and robotics with microfluidics, were promising. But the site visit sealed the Series A deal, where DCVC was the lead and only investor.

J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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Covid-19 roundup: BAR­DA qui­et­ly pulls plug on IL-6 drugs; BioN­Tech and Fo­s­un be­gin mR­NA tri­al

IL-6 inhibitors showed some early promise in potentially treating Covid-19 patients, but recent trial flops have dashed hopes. Now it appears BARDA has officially pulled the plug.

The HHS office’s website has quietly updated to reflect that it is “no longer supporting product development” for Covid-19 in both Actemra (tocilizumab) and Kevzara (sarilumab), dealing a blow to the Roche and Regeneron/Sanofi drugs. This all but assures that IL-6 drugs repurposed to treat Covid-19 are essentially dead in the water.

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Igor Splawski (CureVac)

Cure­Vac nabs a top No­var­tis sci­en­tist for CSO slot as mR­NA vac­cines seize the spot­light

One of the key players in the race to develop a new mRNA vaccine to fight Covid-19 has reshuffled the top spots in the executive suite. And they’re bringing in a Novartis vet out of Harvard to spearhead their work on mRNA.

CureVac, which just filed for an IPO that’s still taking shape, has formally handed Franz-Werner Haas the CEO title, after giving it to him on an interim basis. And the still rather stealthy German biotech largely owned by billionaire Dietmar Hopp has recruited Igor Splawski as its chief scientific officer.

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Tony Coles, Cerevel Therapeutics CEO

Adding $445M, Tony Coles and his big Pfiz­er neu­ro spin­out hitch a ride to Wall Street on Per­cep­tive’s SPAC

Two years ago, after Pfizer abruptly shut down its entire neuroscience division, Bain Capital bet $350 million that those assets were still worth something and packaged them into a new biotech: Cerevel Therapeutics. A year later, they got seasoned executive Tony Coles, who had recently jumped back into the C-suite of another neuroscience startup, to run the company.

Now Coles is steering Cerevel public, in what he says is the largest ever transaction of its kind. Cerevel has agreed to merge with Perceptive Advisors’ specialty acquisition company ARYA II. Between the roughly $125 million Perceptive raised through ARYA and an additional investment of $320 million Bain Capital, Perceptive and — yes, really — Pfizer, among others, Cerevel will now move forward with an added $445 million in its coffers.

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