Marino Garcia, Dianthus CEO

5AM, Avid­i­ty, Fi­deli­ty lead $100M in­fu­sion in­to next-gen, tar­get­ed com­ple­ment ap­proach

When the FDA first ap­proved Alex­ion’s Soliris — then eculizum­ab — in 2007, ex­perts cel­e­brat­ed how the first-of-its-kind drug trans­formed the way physi­cians could treat a rare dis­ease called parox­ys­mal noc­tur­nal he­mo­glo­bin­uria.

As Soliris racked up more land­mark OKs, the C5 in­hibitor al­so be­came a wild­ly suc­cess­ful fran­chise for the com­pa­ny, spurring block­buster sales that led to a fol­low-on drug and a $39 bil­lion buy­out by As­traZeneca while in­spir­ing new play­ers to jump in­to the com­ple­ment space.

Di­anthus Ther­a­peu­tics be­lieves it can make an even big­ger leap than what’s been pro­posed to date — and the start­up is tak­ing the wraps off a $100 mil­lion Se­ries A as it ex­plains the mis­sion.

“This field is ripe for pa­tient cen­tric in­no­va­tion and im­prove­ments,” CEO Mari­no Gar­cia told End­points News.

Sim­rat Rand­hawa

With ba­sic un­der­stand­ing of how dys­reg­u­la­tion of the com­ple­ment path­way trig­gered the com­ple­ment cas­cade and au­toim­mune dis­or­ders, the first-gen­er­a­tion prod­ucts “hit where they could,” CMO Sim­rat Rand­hawa said. The orig­i­nal C5 in­hibitors proved high­ly ef­fec­tive in tam­ing down the prob­lem­at­ic im­mune re­ac­tion, but they al­so shut down a lot else, in­clud­ing the im­mune re­ac­tions one needs to fend off for­eign at­tacks. As a re­sult, the drug la­bels came with boxed warn­ings about life-threat­en­ing bac­te­r­i­al in­fec­tions.

As the sci­ence evolves — and it’s still evolv­ing — sci­en­tists have un­earthed new ways of ap­proach­ing this part of the in­nate im­mune sys­tem.

For in­stance, Di­anthus’s lead com­pound is de­signed to se­lec­tive­ly hit the ac­tive forms of C1s in or­der to on­ly tar­get the clas­si­cal com­ple­ment path­way — one of three path­ways that trig­ger the com­ple­ment cas­cade.

“What we’re try­ing to pro­vide is pret­ty much the most tar­get­ed ap­proach to a com­ple­ment in­hibitor that is avail­able,” Rand­hawa said.

Oth­er than po­ten­tial­ly be­ing safer, the biotech hopes that the se­lec­tiv­i­ty will al­so mean that it will need to de­liv­er a lot less an­ti­body than the first-gen­er­a­tion com­ple­ment in­hibitors, lend­ing it­self to sub­cu­ta­neous self-ad­min­is­tra­tion.

Gar­cia not­ed that Di­anthus worked with a part­ner to ex­plore half-life ex­ten­sion tech­nolo­gies that made the an­ti­bod­ies last even longer.

With the team of 15 scat­tered across the US — the in­com­ing head of qual­i­ty will be work­ing out of Ohio, while the head of clin­i­cal op­er­a­tions re­sides in Seat­tle and the head of tech ops re­ports from the Philadel­phia area — the com­pa­ny hopes to be in the clin­ic by the end of the year.

Lon­nie Moul­der’s Tel­lus BioVen­tures pro­vid­ed the ini­tial fund­ing along­side Fair­mount, while Ven­rock helped with the seed. 5AM Ven­tures, Avid­i­ty Part­ners and Fi­deli­ty led the round, with par­tic­i­pa­tion from Wed­bush Health­care Part­ners.

Al­though Di­anthus has no wet labs, it hopes to lever­age its net­work of tech­ni­cal ex­perts in be­com­ing a next-gen leader in com­ple­ment ther­a­pies — an am­bi­tion hint­ed in its name, which com­bines the Greek words for god (“dios”) and flower (“an­thus”).

“The whole idea was that the an­thus flower was cre­at­ed by Zeus to sort of sur­pass the beau­ty and the scent of the lily,” Gar­cia said. “And I mean, it’s a great sym­bol for the com­pa­ny, be­cause es­sen­tial­ly, that’s what we’re try­ing to do.”

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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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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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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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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