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.”

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Vas Narasimhan (Photographer: Jason Alden/Bloomberg via Getty Images)

No­var­tis de­tails plans to axe 8,000 staffers as Narasimhan be­gins sec­ond phase of a glob­al re­org

We now know the number of jobs coming under the axe at Novartis, and it isn’t small.

The pharma giant is confirming a report from Swiss newspaper Tages-Anzeiger that it is chopping 8,000 jobs out of its 108,000 global staffers. A large segment will hit right at company headquarters in Basel, as CEO Vas Narasimhan axes some 1,400 of a little more than 11,000  jobs in Switzerland.

The first phase of the work is almost done, the company says in a statement to Endpoints News. Now it’s on to phase two. In the statement, Novartis says:

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Lina Gugucheva, NewAmsterdam Pharma CBO

Phar­ma group bets up to $1B-plus on the PhI­II res­ur­rec­tion of a once dead-and-buried LDL drug

Close to 5 years after then-Amgen R&D chief Sean Harper tamped the last spade of dirt on the last broadly focused CETP cholesterol drug — burying their $300 million upfront and the few remaining hopes for the class with it — the therapy has been fully resurrected. And today, the NewAmsterdam Pharma crew that did the Lazarus treatment on obicetrapib is taking another big step on the comeback trail with a €1 billion-plus regional licensing deal, complete with close to $150 million in upfront cash.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

How pre­pared is bio­phar­ma for the cy­ber dooms­day?

One of the largest cyberattacks in history happened on a Friday, Eric Perakslis distinctly remembers.

Perakslis, who was head of Takeda’s R&D Data Sciences Institute and visiting faculty at Harvard Medical School at the time, had spent that morning completing a review on cybersecurity for the British Medical Journal. Moments after he turned it in, he heard back from the editor: “Have you heard what’s going on right now?”

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Scoop: Roche scraps one of two schiz­o­phre­nia PhII tri­als af­ter fail­ing the pri­ma­ry end­point

Roche has terminated one of two Phase II trials testing its drug ralmitaront in patients with schizophrenia, the Big Pharma confirmed to Endpoints News.

The study was terminated last month, according to a June 22 update to the registry on clinicaltrials.gov. Begun in September 2020, the trial was looking at ralmitaront in patients with acute schizophrenia. The trial enrolled 286 patients out of an originally planned 308.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

(AP Photo/Gemunu Amarasinghe)

Some phar­ma com­pa­nies promise to cov­er abor­tion-re­lat­ed trav­el costs — while oth­ers won't go that far yet

As the US Department of Health and Human Services promises to support the millions of women who would now need to cross state lines to receive a legal abortion, a handful of pharma companies have said they will pick up employees’ travel expenses.

GSK, Sanofi, Johnson & Johnson, BeiGene, Alnylam and Gilead have all committed to covering abortion-related travel expenses just four days after the Supreme Court overturned Roe v. Wade and revoked women’s constitutional right to an abortion.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Aurobindo Pharma co-founders P. V. Ram Prasad Reddy (L) and K. Nityananda Reddy

Au­robindo Phar­ma re­ceives warn­ing let­ter from In­di­a's SEC fol­low­ing more FDA ques­tion marks

Indian-based generics manufacturer Aurobindo Pharma has been in the crosshairs of the FDA for several years now, but the company is also attracting attention from regulators within the subcontinent.

According to the Indian business news site Business Standard, a warning letter was sent to the company from the Securities Exchange Board of India, or SEBI.

The letter is related to disclosures made by the company on an ongoing FDA audit of the company’s Unit-1 API facility in Hyderabad, India as well as observations made by the US regulator between 2019 and 2022.

David Veitch, Basilea CEO

Basilea toss­es an on­col­o­gy drug back to Mer­ck, dou­bling down on an­tibi­otics in re­vamp

Swiss biotech Basilea Pharmaceutica has shed one of its last pipeline ties with oncology as it makes a move to concentrate on antibiotics in a race to profitability next year. And they’ve followed up on that with news of a Phase III success they believe can help expand prospects for a key franchise player.

The company put out word on Monday that the execs are handing rights to the FGFR inhibitor derazantinib back to Merck by the end of this year as it shuts down clinical work on the tumor checkpoint controller lisavanbulin. They’re still working on partnering that out along with their TTK/PLK1-inhibitor BAL0891 and preclinical oncology assets.

New Charles River Laboratories High Quality (HQ) Plasmid DNA Centre of Excellence at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. (Charles River)

Charles Riv­er Lab­o­ra­to­ries to start cell and gene ther­a­py man­u­fac­tur­ing at UK site in Sep­tem­ber

While Massachusetts-based Charles River Laboratories has been on an acquisition spree, they are not against planting their flag. The latest move by the company sees them crossing the pond to establish a manufacturing site in the UK.

The company on Tuesday opened its cell and gene therapy manufacturing center at Bruntwood SciTech’s Alderley Park in Cheshire, United Kingdom. The expansion follows Charles River’s acquisition of Cognate BioServices and Cobra Biologics in 2021 for $875 million. Cognate is a plasmid DNA, viral vector and cell therapy CDMO.