UP­DAT­ED: Amid the 'su­per­bug' cri­sis, a Big Phar­ma-backed fund scores a mea­ger raise for an­tibi­otics R&D. Is $140M near­ly enough?

Years af­ter Big Phar­ma aban­doned the an­tibi­otics space, a small syn­di­cate has raised $140 mil­lion to ad­dress the loom­ing threat of an­timi­cro­bial re­sis­tance — but is it too lit­tle, too late?

Back in Ju­ly, more than 20 biotechs — in­clud­ing heavy­weight play­ers Pfiz­er, Eli Lil­ly, No­vo Nordisk, Boehringer In­gel­heim, Bay­er Phar­ma­ceu­ti­cals, Mer­ck KGaA and the Amer­i­can Mer­ck — band­ed to­geth­er to help launch the AMR Ac­tion Fund. With an ini­tial $1 bil­lion, the fund set out to bring two to four new an­tibi­otics to pa­tients by 2030.

Je­re­my Far­rar

On Thurs­day, the fund closed on an­oth­er $140 mil­lion raise from the Boehringer In­gel­heim Foun­da­tion, the Eu­ro­pean In­vest­ment Bank and the Well­come Trust — a fig­ure that pales in com­par­i­son to the tens of bil­lions of VC dol­lars spent in on­col­o­gy over the last decade.

“Sys­temic mar­ket fail­ure has left an­tibi­ot­ic in­no­va­tion starved of fi­nanc­ing, mean­ing po­ten­tial­ly life­sav­ing prod­ucts are un­able to make it to the pa­tients who need them,” Je­re­my Far­rar, di­rec­tor of the Well­come Trust, said in a state­ment, adding that the fund is on­ly “part of the so­lu­tion.”

“The AMR Ac­tion Fund is buy­ing time for the an­tibi­ot­ic pipeline. It is now up to gov­ern­ments to use this time wise­ly and take de­ci­sive ac­tion to fix the mar­ket,” Far­rar added.

It’s es­ti­mat­ed that the rise of su­per­bugs takes 700,000 lives every year, ac­cord­ing to the AMR Fund, which was launched with the help of the WHO and the In­ter­na­tion­al Fed­er­a­tion of Phar­ma­ceu­ti­cal Man­u­fac­tur­ers & As­so­ci­a­tions. By 2050, ex­perts guess an­timi­cro­bial re­sis­tance could lead to as many as 10 mil­lion deaths per year.

But de­spite the ris­ing threat, Big Phar­ma has re­treat­ed from the risky field, fraught with cheap gener­ics and poor fi­nan­cial re­turns. Many an­tibi­otics fail in de­vel­op­ment, while oth­ers “with­er on the vine” due to a lack of avail­able fund­ing. And the ones that do get ap­proved are of­ten used spar­ing­ly to pre­serve ef­fec­tive­ness and slow the de­vel­op­ment of fur­ther re­sis­tance.

No­var­tis — one of the many biotechs chip­ping in­to the AMR Fund — joined the pa­rade of big phar­mas ex­it­ing the field two years ago when it culled its an­tibac­te­r­i­al and an­tivi­ral re­search ef­forts. As­traZeneca, Sanofi and Al­ler­gan had al­ready aban­doned their own pro­grams, which, fol­lowed by a slew of bank­rupt­cies, left few oth­ers in pur­suit of a so­lu­tion to an­timi­cro­bial re­sis­tance.

“Nev­er has the threat of an­timi­cro­bial re­sis­tance been more im­me­di­ate and the need for so­lu­tions more ur­gent,” Tedros Ad­hanom Ghe­breye­sus, WHO di­rec­tor-gen­er­al, said in a Jan­u­ary 2020 state­ment.

The AMR Fund isn’t the on­ly ini­tia­tive look­ing to spur new de­vel­op­ment in the space. CARB-X launched in 2016, with the goal of pump­ing up to $480 mil­lion in­to ear­ly projects through 2022. Since its launch, CARB-X has an­nounced 78 awards worth more than $284.4 mil­lion, not count­ing po­ten­tial mile­stones.

“We need in­no­v­a­tive so­lu­tions to avert the loom­ing health cri­sis posed by AMR, which threat­ens to make even com­mon med­ical pro­ce­dures po­ten­tial­ly dead­ly,” said Christoph Boehringer, chair­man of the Boehringer In­gel­heim Foun­da­tion which con­tributed $50 mil­lion to the cause.

Isaac Ston­er, CEO at Oc­ta­gon Ther­a­peu­tics, said the fundrais­ing ef­forts are “re­al­ly in­ter­est­ing, but I have un­for­tu­nate­ly be­come a pes­simist.” He added:

This seems like a sim­i­lar mech­a­nism to the No­vo RE­PAIR fund and CARB-X. It’s a nice idea, but still a very small amount of mon­ey and doesn’t fix the ac­tu­al prob­lem. As they de­scribe them­selves, this is “buy­ing time” for re­al ac­tion. As COVID vac­cine de­vel­op­ment has shown, if gov­ern­ments sign pur­chase con­tracts around large vol­umes of these prod­ucts at guar­an­teed price points, that can re­al­ly help to stim­u­late de­vel­op­ment. But these push in­cen­tives are a bridge to nowhere, as long as the mar­ket op­por­tu­ni­ty is cou­pled to uti­liza­tion.

Ankit Ma­hade­via, founder and CEO of Spero Ther­a­peu­tics, said it’s “help­ful to see the com­mit­ment to find­ing bet­ter so­lu­tions for in­fec­tions.”

“Our re­spon­si­bil­i­ty as biotechs is to find sci­en­tif­i­cal­ly in­no­v­a­tive med­i­cines that meet true un­met needs in sus­tain­able mar­kets and the fund can help us in that mis­sion,” he con­tin­ued.

Com­par­ing the threat to the nov­el coro­n­avirus, which was “prac­ti­cal­ly un­known” un­til last year, the AMR Fund said an­timi­cro­bial re­sis­tance is “a threat we know, and we must take col­lec­tive ac­tion now.”

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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George Church (courtesy EnPlusOne BioSciences)

George Church, Wyss sci­en­tists and North­pond chal­lenge con­ven­tion­al RNA man­u­fac­tur­ing with new biotech

RNA medicine has been at the forefront for the past few years, with the first RNA silencing therapy approved in 2018, and mRNA Covid vaccines following after. But flying under the radar has been the process of actually making RNA for these treatments.

That’s what Daniel Wiegand and Jonathan Rittichier have been working on in George Church’s lab for the past six years.

Friday morning, they unveiled EnPlusOne Biosciences, a biotech built on their RNA synthesis platform. Wiegand will serve as the Watertown, MA-based biotech’s CEO, and Rittichier will be CSO. And no different from his other startups, Church will be acting as scientific advisor. Its fourth co-founder, Dan Ahlstedt, joined through a Harvard Business School program, and will be COO.

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Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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