Ken Song (RayzeBio)

No­var­tis bet $6B on the idea — now Ver­sant, ven­Bio have $45M to birth a plat­form play for ra­dio­phar­ma­ceu­ti­cals

Back in 2017, in the wake of back-to-back set­backs and a re­or­ga­ni­za­tion, a strug­gling lit­tle biotech named En­do­cyte com­plet­ed a $12 mil­lion li­cens­ing deal that would ul­ti­mate­ly put it on a path to­ward a $2.1 bil­lion buy­out.

As it turned out, Ver­sant Ven­tures was al­so eye­ing that very same drug out of Ger­many: PS­MA-617, a ra­di­oli­gand ther­a­peu­tic for cas­tra­tion-re­sis­tant prostate can­cer.

Jer­el Davis

“We ac­tu­al­ly put a term sheet out on that as­set be­cause we just thought the ef­fi­ca­cy da­ta were so pro­found there,” man­ag­ing di­rec­tor Jer­el Davis told End­points News, re­fer­ring to a drug that reg­is­tered a pro­gres­sion-free sur­vival rate of 7.6 months among PS­MA-pos­i­tive pa­tients, with one co­hort hit­ting an over­all sur­vival rate of 13.5 months.

Af­ter three years of scout­ing the (small) space for op­por­tu­ni­ties Ver­sant reck­ons a fresh start is the way to go. The biotech fledg­ling, Rayze­Bio, is de­but­ing to­day with $45 mil­lion in Se­ries A mon­ey, with ven­Bio as a co-leader and Sam­sara Bio­Cap­i­tal al­so chim­ing in.

With No­var­tis now claim­ing two of the most ad­vanced pro­grams in the field — both PS­MA-617 and FDA-ap­proved Lu­tathera, from a $3.9 bil­lion ac­qui­si­tion of Ad­vanced Ac­cel­er­a­tor Ap­pli­ca­tions — ra­dio­phar­ma­ceu­ti­cals has in­trigued quite a few biotech ob­servers, not­ed CEO Ken Song. But se­cur­ing a steady sup­ply of ther­a­peu­tic ra­dioiso­topes, es­pe­cial­ly the more po­tent al­pha iso­topes, has been a chal­lenge for any­one look­ing to mount an ear­ly-stage ef­fort.

While the tar­get­ed de­liv­ery ap­proach be­hind ra­dio­phar­ma­ceu­ti­cals is sim­i­lar to an­ti­body-drug con­ju­gates, ra­dioiso­topes such as Ac­tini­um-225, which Rayze­Bio is de­ploy­ing, are “many fold more po­tent, prob­a­bly 100 or 500 times more po­tent” than cy­to­tox­ic pay­loads in AD­Cs, Davis added.

“What has hap­pened over the last sev­er­al years is that a mul­ti­tude of groups in both acad­e­mia and in­dus­try have de­vised al­ter­nate ways to gen­er­ate Ac­tini­um-225 where they’ve ac­tu­al­ly pro­vid­ed da­ta to show that this can be pro­duced and scaled up to suf­fi­cient quan­ti­ties,” he said. “That’s on­ly come to light over the last year, year and a half.”

Deb­o­rah Charych

That opened up a white space that Song, who was look­ing to “take a con­sid­er­able amount of time off” and turned down dozens of oth­er of­fers as he stepped down from the helm at Metacrine, couldn’t pass up.

The founders, led by Nek­tar vet and for­mer Third Rock en­tre­pre­neur Deb­o­rah Charych, were in­tent on cre­at­ing a pur­pose-built op­er­a­tion that would go broad­er and deep­er than cur­rent play­ers, said Aaron Roys­ton, man­ag­ing part­ner of ven­Bio.

In­stead of re­pur­pos­ing ex­ist­ing mol­e­cules to tar­get can­cer anti­gens, for in­stance, Rayze­Bio has part­nered up with Japan’s Pep­tiDream to dis­cov­er new pep­tides against a range of val­i­dat­ed sol­id tu­mor tar­gets. The pep­tides will then be ra­di­o­la­beled with Ac­tini­um-225 — in-house for pre­clin­i­cal work and by con­tract man­u­fac­tur­ers in clin­i­cal stud­ies through com­mer­cial­iza­tion.

“We’re re­al­ly ask­ing the pep­tides to do ex­act­ly what pep­tides do well,” Roys­ton said. They can pen­e­trate tu­mors with their small sizes, are spe­cif­ic to tar­gets, and have rel­a­tive­ly short half life — prop­er­ties that peo­ple usu­al­ly don’t want in a drug. Yet “with ra­dio­phar­ma I think it’s a per­fect ap­pli­ca­tion.”

Aaron Roys­ton

In line with a vi­sion to be the first ra­dio­phar­ma plat­form play, the biotech has put to­geth­er 7 ac­tive pro­grams, with the goal to have at least one de­vel­op­ment can­di­date by the sec­ond half of 2021. The first clin­i­cal tri­als should hap­pen no more than one year there­after.

No­tably, Rayze­Bio will be able to use part of its drugs to iden­ti­fy the pa­tients it wants to re­cruit in­to tri­als by tak­ing the same binders but swap­ping out the ther­a­peu­tic iso­tope for an imag­ing agent. It spells a tar­get­ed yet broad­ly ap­plic­a­ble ap­proach that Song be­lieves would at­tract and ac­com­mo­date a large num­ber of play­ers.

“Some­one needs to be part of the ear­ly pack,” Song said about Rayze­Bio’s team, which con­sists of 10 full timers and about a dozen con­sul­tants and ad­vi­sors. “Our goal ob­vi­ous­ly is to al­ways be ahead of every­one be­cause we’re get­ting a head start by fo­cus­ing on it now. But that is up to us to en­sure that we main­tain that ad­van­tage.”

UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Michael Shpigelmacher

Khosla joins bet on un­con­ven­tion­al start­up look­ing to send drug de­liv­er­ing ro­bots in­to the brain

When Michael Shpigelmacher started the project, he knew he’d have to fund it himself. Every other effort of its kind was academic, rejected as too risky by investors.

Shpigelmacher, a robotics geek and entrepeneur who had drifted into consulting for pharma, wanted to build the real-life equivalent of technology from the 1960s film Fantastic Voyage, the one where a submarine crew is shrunk to “about the size of a microbe” and sent on a mission to repair a scientist’s brain. He scanned the literature, found the lab that was working on the most advanced project — at the Max Planck Institute in Germany, it turned out — and started funding them with money from his own account, along with some seed cash from friends and family.

Paul Sekhri

The next big biotech su­per­star? Paul Sekhri has some thoughts on that

It occasionally occurs to Paul Sekhri that if they pull this off, his company will be on the front page of the New York Times and a lead story in just about every major news outlet on the planet. He tries not to dwell on it, though.

“I just want to be laser-focused on getting to that point,” Sekhri says, before acknowledging, “Yes, it absolutely crossed my mind.”

Sekhri, a longtime biopharma executive with tenures at Sanofi and Novartis, is now entering year three as CEO of eGenesis, the biotech that George Church protégé Luhan Yang founded to genetically alter pigs so that they can be used for organ transplants. He led them through one megaround and has just closed another, raising $125 million from 17 different investors to push the first-ever (humanized) pig to human transplants into the clinic.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

With stars aligned and cash in re­serve, Bob Nelsen's Re­silience plans a makeover at 2 new fa­cil­i­ty ad­di­tions to its drug man­u­fac­tur­ing up­start

Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape.

Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling.

The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs.

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Or­biMed, bio­phar­ma's biggest in­vestor, clos­es $3.5B in three new pri­vate funds

One of the world’s leading biopharma investors has pulled in its next rounds of cash, with the funds planned to go to dozens of companies around the world.

OrbiMed raised $3.5 billion across three private investment funds, it announced Monday, as it continues building on its long track record in healthcare and biopharma. All in all, the firm expects to invest in at least 60 companies across the US, Asia and Europe.

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Af­ter bail­ing on Covid-19 vac­cines, Mer­ck will team up with J&J to pro­duce its shot as part of un­usu­al Big Phar­ma pact

Merck took a big gamble when it opted to jump into the Covid-19 vaccine race late, and made an equally momentous decision to back out in late January. Now, looking to chip in on the effort, Merck reportedly agreed to team up with one of the companies that has already crossed the finish line.

President Joe Biden on Tuesday is expected to announce a partnership between drugmakers Merck and Johnson & Johnson to jointly produce J&J’s recombinant protein Covid-19 vaccine that received the FDA’s emergency use authorization Saturday, the Washington Post reported.

Ab­b­Vie tees up a biotech buy­out af­ter siz­ing up their Parkin­son's drug spun out of Ke­van Shokat's lab

AbbVie has teed up a small but intriguing biotech buyout after looking over the preclinical work it’s been doing in Parkinson’s disease.

The company is called Mitokinin, a Bay Area biotech spun out of the lab of UCSF’s Kevan Shokat, whose scientific explorations have formed the academic basis of a slew of startups in the biotech hub. One of Shokat’s PhD students in the lab, Nicholas Hertz, co-founded Mitokinin using their lab work on PINK1 suggesting that amping up its activity could play an important role in regulating the mitochondrial dysfunction contributing to Parkinson’s disease pathogenesis and progression.

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Fi­bro­Gen shares skid low­er as a sur­prise ad­comm rais­es risks on roxa OK

FibroGen will likely have to delay its US rollout for roxadustat once again.

In an unexpected move, the FDA is convening its Cardiovascular and Renal Drugs Advisory Committee to review the NDA in an advisory committee meeting. The date is yet to be confirmed.

Just a few weeks ago, SVB Leerink analyst Geoffrey Porges predicted that the roxa approval could come ahead of the PDUFA date on March 20 — effusive despite already being let down once by the FDA’s extension of its review back in December. AstraZeneca, which is partnered with FibroGen on the chronic kidney disease-related anemia drug, disclosed regulators had requested further clarifying analyses of clinical data.

In­tro­duc­ing End­pointsF­DA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.