Ken Song, RayzeBio CEO

With fresh $160M, Rayze­Bio plots sprint through the clin­ic for lead ra­dio­phar­ma­ceu­ti­cal pro­gram — build­ing on No­var­tis' suc­cess

Ever since its launch in 2020, Rayze­Bio has talked at length about build­ing a plat­form com­pa­ny to ful­fill the po­ten­tial that ra­dio­phar­ma­ceu­ti­cals hold — with sev­en ac­tive pro­grams to boast. But it’s stayed mum about what those pro­grams ac­tu­al­ly are, in­stead stick­ing to up­dates about new fund­ing, hires or part­ner­ships.

That’s by de­sign, ac­cord­ing to CEO Ken Song. The for­mer Ven­rock VC and se­r­i­al biotech en­tre­pre­neur want­ed to make sure that he had a re­al busi­ness case be­fore be­com­ing more vis­i­ble.

“Two years ago, when we were writ­ten up, it re­al­ly was just a cou­ple of us with no lab, no of­fice, and just a nice-look­ing Pow­er­Point. And that sort of paint­ed the vi­sion and the plans of what we want­ed to do,” Song told End­points News. “We’ve now reached a point where the or­ga­ni­za­tion is at a place where we do feel that there’s quite a good prospect for the com­pa­ny long term.”

He was ex­ag­ger­at­ing in a sense — by the time Rayze­Bio un­veiled its $45 mil­lion Se­ries A, co-led by Ver­sant and ven­Bio, it al­ready had a set of tar­gets in mind, a deal with Pep­tiDream to search for pep­tides and a sup­ply arrange­ment for Ac­tini­um-225.

But now, with more than 200 in vi­vo stud­ies un­der its belt, Rayze­Bio is fi­nal­ly tak­ing the wraps off its lead pro­gram as well as a $160 mil­lion Se­ries D de­signed to fund the drug’s dash through the clin­ic all the way to Phase III.

While Rayze­Bio set out to dis­cov­er new pep­tide binders that will guide can­cer-killing ra­dioiso­topes to tu­mor cells, the biotech ul­ti­mate­ly set­tled on a tried-and-true binder for its lead drug, RYZ101.

“I would say at con­cep­tu­al lev­el, ra­dio­phar­ma­ceu­ti­cals seem pret­ty straight­for­ward and easy. You take a binder, a link­er, a ra­dioac­tive iso­tope, in the­o­ry you should have your drug,” Song said.

I would say what we came to ful­ly ap­pre­ci­ate is that the dis­cov­ery process, de­spite the con­cept be­ing rel­a­tive­ly straight­for­ward and sim­ple, the ac­tu­al dis­cov­ery of find­ing those binders that can have all the right prop­er­ties to de­liv­er the ra­dioac­tive iso­tope is in­cred­i­bly com­plex and chal­leng­ing. And hence, you know, high­light­ing the fact that we have com­plet­ed over 200 in vi­vo stud­ies. We re­al­ly feel that we need­ed to gen­er­ate that amount of ex­pe­ri­ence and da­ta to re­al­ly un­der­stand the ba­sic rules and roadmap in how to dis­cov­er nov­el binders against tar­gets of in­ter­est to de­liv­er a ra­dioac­tive iso­tope.

No­var­tis us­es the same pep­tide, dotatate, for its ap­proved ra­di­oli­gand ther­a­py Lu­tathera to treat neu­roen­docrine tu­mors, ex­cept that it de­ploys a be­ta iso­tope rather than an al­pha one. De­spite ini­tial dra­mat­ic clin­i­cal ben­e­fit, though, Song not­ed that pa­tients even­tu­al­ly progress and they have no fur­ther op­tions.

That’s where he hopes RYZ101 can come in. By link­ing dotatate with an al­pha iso­tope dubbed Ac­tini­um-225, which he says is 400 to 500 times more pow­er­ful than be­ta iso­topes, Rayze­Bio hopes it can prove it­self first in neu­roen­docrine tu­mors be­fore mov­ing to a sec­ond test in small cell lung can­cer.

Thanks to ex­ist­ing clin­i­cal da­ta on dotatate-based ra­dio­phar­ma­ceu­ti­cals, some from aca­d­e­m­ic stud­ies, Rayze­Bio has de­signed its first tri­al such that if the Phase I por­tion looks good on safe­ty, tol­er­a­bil­i­ty and phar­ma­co­ki­net­ics, it can move straight in­to Phase III. Phase I da­ta are ex­pect­ed next year.

The cash in­fu­sion, which brings the to­tal haul to $418 mil­lion, will al­so sup­port ini­tial stud­ies of sub­se­quent pro­grams as well as the build­out of an in-house man­u­fac­tur­ing fa­cil­i­ty for com­mer­cial-scale pro­duc­tion.

While Rayze­Bio cur­rent­ly works with a con­tract man­u­fac­tur­er to make its drug ma­te­r­i­al, Song not­ed the num­ber of com­pa­nies that can do it is “ex­treme­ly lim­it­ed.” Ex­ist­ing ra­dio­phar­ma­ceu­ti­cals have a shelf life of about three to four days as the ra­dioiso­topes de­cay, and even though Rayze­Bio stretched that out to five days, the just-in-time na­ture of the process still adds ex­tra stress.

“We thought that this was a piece that we want­ed to have more con­trol and over­sight over,” he said.

Viking Glob­al In­vestors, Sofinno­va In­vest­ments and Welling­ton Man­age­ment co-led the round, joined by ex­ist­ing back­ers as well as new in­vestors Al­ly Bridge Group, Sands Cap­i­tal, Lau­ri­on Cap­i­tal Man­age­ment and Soleus Cap­i­tal. An undis­closed glob­al in­vestor al­so jumped in.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

#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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 151,400+ biopharma pros reading Endpoints daily — and it's free.