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.”

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

CEO Khurem Farooq (Gyroscope)

Hours be­fore ex­pect­ed de­but, Gy­ro­scope post­pones its IPO as 2 oth­er biotechs hold the line on their march to Nas­daq

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

In a surprising turn of events, UK-based Gyroscope Therapeutics has postponed its IPO mere hours before it was set to debut on Nasdaq.

Working on a gene therapy for wet AMD, Gyroscope was all set and ready to go public earlier this week, setting terms for a $142 million raise with a price range of $20 to $22. But in the wee hours of Friday morning, the company put out a press release saying they would delay their debut “in light of market conditions,” CEO Khurem Farooq said in a statement.

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Angela Merkel (AP Photo/Michael Sohn)

Covid-19 roundup: Pfiz­er sub­mits vac­cine for full ap­proval; Merkel op­pos­es Biden pro­pos­al to sus­pend IP for vac­cines

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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In­cyte ponies up $12M to set­tle char­i­ty foun­da­tion kick­back claims; US ex­er­cis­es op­tion for more dos­es of mon­key­pox vac­cine

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.

David Coman, Science 37

Amid vir­tu­al tri­al craze, Sci­ence 37 earns uni­corn sta­tus and a trip to Nas­daq on the back of SPAC deal

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As the Covid-19 pandemic made conventional trials impossible for some drugmakers, more and more companies moved to decentralize their clinical studies, accelerating business for tech developers like Science 37. Leveraging that boost, the company is on the verge of a SPAC merger, landing unicorn status and its very own stock ticker.