Clene Nanomed­i­cine, re­search­ing the use of gold atoms to slow ALS pro­gres­sion, nets $42.5M Se­ries D

A bio­phar­ma that us­es gold to de­vel­op treat­ments for neu­rode­gen­er­a­tive dis­eases just got a lit­tle bit rich­er.

Rob Ether­ing­ton

Clene Nanomed­i­cine pulled in $42.5 mil­lion in a Se­ries D fi­nanc­ing round Wednes­day, mon­ey which will go to­ward ad­vanc­ing its lead pro­gram through a Phase III plat­form tri­al in ALS and sup­port Phase II tri­als in MS, Parkin­son’s dis­ease and ALS. CEO Rob Ether­ing­ton said that by the end of 2021, Clene will know whether or not the can­di­date, called CNM-Au8, will prove ef­fec­tive.

“It will take us to the end of all these clin­i­cal end­points,” Ether­ing­ton told End­points News. “The ex­cit­ing thing for us is that one as­set could po­ten­tial­ly be in­di­cat­ed to im­prove neu­ro­log­i­cal func­tion in MS, as well as ALS, and [though] Parkin­son’s is the slow­er pro­gram, this mon­ey is go­ing to help us launch more com­plete­ly that pro­gram.”

CNM-Au8 is a liq­uid sus­pen­sion of gold nanocrys­tals that cat­alyze in­tra­cel­lu­lar bi­o­log­i­cal re­ac­tions. Such cat­alyza­tion can lead to im­prove­ment in nerve cell sur­vival, func­tion, and com­mu­ni­ca­tion. Chem­i­cal­ly, the “clean sur­faces” of the nanocrys­tals help nor­mal­ize ATP pro­duc­tion in cells, which is lack­ing in se­ri­ous neu­ro­log­i­cal dis­eases like ALS, CMO Robert Glanz­man said.

“We’re pro­vid­ing bioen­er­gy sup­port to cells,” Glanz­man said. “There’s a rea­son why we tend to get neu­rode­gen­er­a­tive dis­eases as we get old­er, and that is be­cause as we age, there’s a lin­ear loss of bioen­er­getic ca­pac­i­ty with­in neu­rons … and what we’re do­ing is ac­tu­al­ly pro­vid­ing these neu­rons and oth­er cells with free en­er­gy, es­sen­tial­ly.”

In terms of vis­i­ble symp­toms, Glanz­man added that pa­tients tak­ing CNM-Au8 will see bet­ter strength, mus­cle mass and be able to speak, breathe and swal­low more eas­i­ly over a longer pe­ri­od of time.

Clene’s Phase III study comes as it was se­lect­ed to par­tic­i­pate in the first-ever plat­form tri­al for ALS, which en­rolled its first pa­tients ear­li­er this month. The tri­al com­pares three sep­a­rate treat­ments for the dis­ease, with UCB’s zilu­coplan and Bio­haven’s verdiper­stat join­ing CNM-Au8 at Har­vard-backed Mass­a­chu­setts Gen­er­al Hos­pi­tal in test­ing 480 to­tal pa­tients.

Though de­layed from a March start due to the Covid-19 pan­dem­ic, the plat­form tri­al aims to ex­pe­dite the de­vel­op­ment of ther­a­pies for a dis­ease that ad­vances rapid­ly and that has few ef­fec­tive treat­ment op­tions. On­ly rilu­zole, al­so known as Ri­lutek and OK’ed in 1995, shows any mea­sur­able ef­fect on ALS pa­tients, Ether­ing­ton said.

“Rilu­zole, which func­tion­al­ly is re­al­ly the on­ly drug that most peo­ple with ALS use, was orig­i­nal­ly ap­proved to de­lay the need for tra­cheostomies to en­cour­age breath­ing for an ex­tra cou­ple months,” Ether­ing­ton said. “But it has a very mod­est ef­fect gen­er­al­ly. It is the stan­dard of care, how­ev­er, be­cause it’s the on­ly re­al­ly marked­ly rel­e­vant drug that’s been ap­proved for ALS in this coun­try. There’s a few oth­ers but most of them do very lit­tle.”

Clene has two oth­er pro­grams in the pipeline, though nei­ther have reached the clin­ic just yet. The first is a top­i­cal gel con­tain­ing sil­ver and zinc ions, with re­searchers look­ing at burn treat­ment, ac­cel­er­at­ed wound-heal­ing and as an an­ti-in­fec­tive. There’s al­so a gold-plat­inum ther­a­peu­tic be­ing stud­ied for use in on­col­o­gy, which is still in the ini­tial in vit­ro stage.

The bot­tom line for Clene though is that find­ing a treat­ment op­tion for the ex­treme­ly dif­fi­cult ALS in­di­ca­tion be­comes clos­er to re­al­i­ty, with a po­ten­tial­ly huge im­pact on the field.

“The way you and I move and can grasp things and can talk, all this fine mo­tor move­ment we take for grant­ed,” Ether­ing­ton said. “An ALS pa­tient los­es these and this is ex­act­ly what we are study­ing.”

How one start­up fore­told the neu­ro­science re­nais­sance af­ter '50 years of shit­show'

In the past couple of years, something curious has happened: Pharma and VC dollars started gushing into neuroscience research.

Biogen’s controversial new Alzheimer’s drug Aduhelm has been approved on the basis of removing amyloid plaque from the brain, but the new neuro-focused pharma and biotechs have much loftier aims. Significantly curbing or even curing the most notorious disorders would prove the Holy Grail for a complex system that has tied the world’s best drug developers in knots for decades.

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Ryan Watts, Denali CEO

De­nali slips as a snap­shot of ear­ly da­ta rais­es some trou­bling ques­tions on its pi­o­neer­ing blood-brain bar­ri­er neu­ro work

Denali Therapeutics had drummed up considerable hype for their blood-brain barrier technology since launching over six years ago, hype that’s only intensified in the last 14 months following the publications of a pair of papers last spring and proof of concept data earlier this year. On Sunday, the South San Francisco-based biotech gave the biopharma world the next look at in-human data for its lead candidate in Hunter syndrome.

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Art Levinson (Calico)

Google-backed Cal­i­co dou­bles down on an­ti-ag­ing R&D pact with Ab­b­Vie as part­ners ante up $1B, start to de­tail drug tar­gets

Seven years after striking up a major R&D alliance, AbbVie and Google-backed anti-aging specialist Calico are doubling down on their work with a joint, $1 billion commitment to continuing their work together. And they’re also beginning to offer some details on where this project is taking them in the clinic.

According to their statement, each of the two players is putting up $500 million more to keep the labs humming.

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Ugur Sahin, BioNTech CEO (Bernd von Jutrczenka/dpa via AP Images)

BioN­Tech is spear­head­ing an mR­NA vac­cine de­vel­op­ment pro­gram for malar­ia, with a tech trans­fer planned for Africa

Flush with the success of its mRNA Covid-19 vaccine, BioNTech is now gearing up for one of the biggest challenges in vaccine development — which comes without potential profit.

The German mRNA pioneer says it plans to work on a jab for malaria, then transfer the tech to the African continent, where it will work with partners on developing the manufacturing ops needed to make this and other vaccines.

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Why is On­col­o­gy Drug De­vel­op­ment Re­search Late to the Dig­i­tal Bio­mark­ers Game?

During the recent Annual ASCO Meeting, thousands of cancer researchers and clinicians from across the globe joined together virtually to present and discuss the latest findings and breakthroughs in cancer research and care. There were more than 5000+ scientific abstracts presented during this event, yet only a handful involved the use of motion-tracking wearables to collect digital measures relating to activity, sleep, mobility, functional status, and/or quality of life. Although these results were a bit disappointing, they should come as no surprise to those of us in the wearable technology field.

Andrea Pfeifer, AC Immune CEO (AC Immune)

Look­ing to repli­cate Covid-19 suc­cess in neu­ro, BioN­Tech back­ers bet on AC Im­mune and its new­ly-ac­quired Parkin­son's vac­cine

The German billionaires behind BioNTech have found a new vaccine project to back.

Through their family office Athos Service, twin brothers Thomas and Andreas Strüngmann are leading a $25 million private placement into Switzerland’s AC Immune — which concurrently announced that it’s shelling out $58.7 million worth of stock to acquire Affiris’ portfolio of therapies targeting alpha-synuclein, including a vaccine candidate, for Parkinson’s disease.

Rajiv Shukla, Constellation Alpha Holdings

Can­del gets busy IPO week mov­ing with down­sized raise as Ra­jiv Shuk­la's third SPAC goes pub­lic

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

In a week that’s expected to see several biotechs price their IPOs, Candel Therapeutics got things kicked off Tuesday with a muted opener.

The company helmed by former GlaxoSmithKline vet Paul Peter Tak made its way to Nasdaq thanks to a $72 million raise, which was downsized by about 15% than originally anticipated, according to Renaissance Capital. Candel priced at $8 per share after initially seeking to launch in the $13 to $15 range.

UP­DAT­ED: Pan­el of neu­ro­science ex­perts lays out the com­pli­ca­tions with us­ing Bio­gen's new Alzheimer's drug

Treatment of early Alzheimer’s patients with Biogen’s new drug Aduhelm should closely resemble how the drug was studied in its pivotal clinical trials, according to new recommendations from a panel of neuroscience experts led by UNLV’s Jeffrey Cummings.

“Those considering aducanumab therapy should understand that the expected benefit is slowing of cognitive and functional decline; improvement of the current clinical state is not anticipated,” they wrote Tuesday in The Journal of Prevention of Alzheimer’s Disease, noting that some of their recommendations are more specific or more restrictive than the information provided in the FDA’s prescribing information.

Christophe Weber, Takeda CEO (Kyodo via AP Images)

Take­da flesh­es out CNS pact with pep­tide drug­mak­er, set­ting aside $3.5B in fu­ture mile­stones

One of a suite of drugmakers looking to reinvest in the neuroscience space, Takeda has been aggressive in signing on new partners to help build up its pipeline in that space. But sometimes the best partner is the one you already have.

Takeda will set aside $3.5 billion in future milestones and an undisclosed upfront payment to build out its drug discovery deal with Japanese peptide conjugate maker PeptiDream, adding neurodegeneration to the partnership’s list of CNS targets, the companies said Tuesday.

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