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.”

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (AP Images)

As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

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The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Vivek Ramaswamy (Jeff Rumans/JPM 2020)

Urovan­t's lead drug dis­ap­points in mid-stage study as first big FDA de­ci­sion looms

Just as Urovant gets ready for its first big FDA decision on vibegron, the drug has flopped in what would’ve been a follow-on indication.

In a Phase IIa trial involving women with abdominal pain due to irritable bowel syndrome, vibegron failed to meet the bar on improving “average worst abdominal pain” over 12 weeks, compared to placebo, among IBS-D patients.

There were actually slightly more responders in the placebo group than in the drug arm, with only 40.9% of those randomized to vigebron achieving at least a 30% decrease in “worst abdominal pain” in the past 24 hours. The trial enrolled 222 women but only 189 completed the study.

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The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.

Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.

Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications.

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