Rivus Pharmaceuticals CEO Allen Cunningham (L) and CSO Shaharyar Khan

Weight loss play­er gets at­ten­tion of RA Cap­i­tal, walks away with $132M to keep pur­su­ing big tar­gets

A biotech out  to cor­ner a new mar­ket in the di­a­betes and weight loss space has record­ed its sec­ond, and so far largest, raise yet.

Char­lottesville, VA-based Rivus Phar­ma­ceu­ti­cals made the an­nounce­ment Thurs­day that it has closed its Se­ries B at $132 mil­lion. Led by RA Cap­i­tal, the round will push the biotech fur­ther along in clin­i­cal de­vel­op­ment of its lead can­di­date, HU6. Per Rivus, the lead can­di­date is a type of small mol­e­cule called a “con­trolled meta­bol­ic ac­cel­er­a­tor,” a term the biotech coined to de­scribe a drug de­signed to tar­get obe­si­ty — by in­creas­ing the num­ber of calo­ries a pa­tient burns.

As CEO Allen Cun­ning­ham and CSO Sha­har­yar Khan tell End­points News, Rivus is look­ing at in­creas­ing en­er­gy ex­pen­di­ture phar­ma­co­log­i­cal­ly to re­duce obe­si­ty, in­stead of fo­cus­ing more on re­duc­ing caloric in­take.

“You’ve got en­er­gy com­ing in, caloric in­take, and then you’ve got en­er­gy go­ing out — caloric ex­pen­di­ture. And most of these ap­proach­es that are see­ing this, the obe­si­ty ther­a­peu­tics are tar­get­ing that front end, the caloric in­take side of the equa­tion,” Khan said.

So far, the biotech has al­ready cleared its lead can­di­date in obe­si­ty through Phase IIa, read­ing out ear­li­er this year and show­ing that the drug helped pa­tients lose a to­tal of six pounds of fat over the course of two months.

And as part of the raise, Rivus is con­tin­u­ing a sec­ond Phase IIa study that the biotech re­cent­ly start­ed in obese par­tic­i­pants with heart fail­ure with pre­served ejec­tion frac­tion (HF­pEF), as well as start­ing a Phase IIb in obe­si­ty — a 12-month-long study that is slat­ed to start some­time next year and have a sub­set of pa­tients with type II di­a­betes.

On top of those in­di­ca­tions, the biotech has al­so been look­ing at se­vere hy­per­triglyc­eridemia (SHTG) and NASH, which has been a prover­bial mine­field with­out many pos­i­tive read­outs over the years.

The Rivus ex­ecs said that they’re now ful­ly fund­ed through 2024 as the biotech plans to run two clin­i­cal tri­als si­mul­ta­ne­ous­ly. And while head­count is still small, a few hires are planned, the ex­ecs not­ed.

The CSO added that the key mech­a­nism they’re try­ing to lever­age is mi­to­chon­dr­i­al un­cou­pling, a nat­ur­al process in the body where cel­lu­lar en­er­gy trans­port mech­a­nisms are di­vert­ed to be used out­side their nor­mal func­tion of syn­the­siz­ing adeno­sine triphos­phate, or ATP. Nor­mal­ly, mi­to­chon­dria take sub­strates, fats and sug­ars and gen­er­ate en­er­gy as a byprod­uct. If that process gets slowed down, the body has to burn more calo­ries to get its en­er­gy be­fore it can re­turn to en­er­gy bal­ance.

Rivus says its lead can­di­date al­so is fat-se­lec­tive, forc­ing the body to burn fat in­stead of oth­er mol­e­cules to sur­vive. Part of the perks be­hind go­ing on­ly af­ter fat, Cun­ning­ham says, is that skele­tal mass re­mains in­tact.

“Af­ter a caloric deficit, the first thing you gain back is fat. And once you lose that skele­tal mus­cle mass, it’s hard to get it back,” Cun­ning­ham added.

As for next fi­nanc­ing plans, con­sid­er­ing RA Cap­i­tal’s pre­vi­ous habit of back­ing com­pa­nies in prepa­ra­tion for an IPO, ex­ecs de­clined to an­swer — but not­ed that as part of the fi­nanc­ing, they will be look­ing more in­to new in­di­ca­tions and bring­ing a new CMA can­di­date in­to its pipeline.

“We are look­ing at where the sci­ence is high­ly sup­port­ive, whether it’s in ar­eas like can­cer or neu­rode­gen­er­a­tion, or even ag­ing,” Khan not­ed.

The biotech got its start in March 2019, se­cur­ing $35 mil­lion via a Se­ries A round — but re­mained in stealth mode un­til it emerged just over two years lat­er.

Oth­er in­vestors that tagged along on to­day’s Se­ries B in­clud­ed new in­vestors Bain Cap­i­tal Life Sci­ences and BB Biotech, plus pre­vi­ous back­ers Lon­gi­tude Cap­i­tal, Medicxi and Rx­Cap­i­tal.

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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#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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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.

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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