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.”

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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Stephen Hahn, FDA commissioner (AP Images)

As FDA sets the stage for the first Covid-19 vac­cine EUAs, some big play­ers are ask­ing for a tweak of the guide­lines

Setting the stage for an extraordinary one-day meeting of the Vaccines and Related Biological Products Advisory Committee this Thursday, the FDA has cleared 2 experts of financial conflicts to help beef up the committee. And regulators went on to specify the safety, efficacy and CMC input they’re looking for on EUAs, before they move on to the full BLA approval process.

All of this has already been spelled out to the developers. But the devil is in the details, and it’s clear from the first round of posted responses that some of the top players — including J&J and Pfizer — would like some adjustments and added feedback. And on Thursday, the experts can offer their own thoughts on shaping the first OKs.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Lit­tle Zosano takes an­oth­er beat­ing as the FDA slaps down their ap­pli­ca­tion for a mi­graine patch

Zosano $ZSAN has officially come up short in its bid to develop a migraine patch.

The FDA rejected the company’s application to repurpose the triptan zolmitriptan in a new delivery system as Qtrypta, Zosano said Wednesday morning, issuing a CRL for the microneedle patch. Regulators cited inconsistent exposure levels across multiple clinical trials as the main reason for the thumbs down.

Investors did not take too kindly to the news, with Zosano shares plunking down around 25%. The company is requesting a Type A meeting to “provide clarity on the next steps for the program,” CEO Steven Lo said in a statement.

Glax­o­SmithK­line's vac­cines group aims for a first as it kicks off PhI­II RSV stud­ies

One of GlaxoSmithKline’s big projects at its global vaccine R&D center in Rockville, MD is set to enter Phase III after passing early-stage tests with flying colors.

Eyeing the wide-open respiratory syncytial virus (RSV) space, GSK is pushing two different vaccine candidates: GSK3888550A is designed to confer protection to infants via maternal immunization, while GSK3844766A is meant for the elderly.

Pur­due Phar­ma signs guilty plea, preps $8B+ set­tle­ment on Oxy con­tro­ver­sy — re­port; Flag­ship brings in a comms chief

Purdue Pharma may soon be signing off on a guilty plea and an $8 million-plus settlement to wrap up its controversial role distributing OxyContin.

The AP has the breaking story this morning.

Purdue filed for bankruptcy last year, along with Insys and followed by Mallinckrodt, as it navigated its way through a blizzard of litigation surrounding Oxy, which triggered an epidemic of abuse around the country.

Pfizer CEO Albert Bourla (Drew Angerer/Getty Images)

Pfiz­er is on the verge of claim­ing a multi­bil­lion-dol­lar first-mover ad­van­tage with their Covid-19 vac­cine — an­a­lyst

From the beginning, Pfizer CEO Albert Bourla eschewed government funding for his Covid-19 vaccine work with BioNTech, willing to take all the $2 billion-plus risk of a lightning-fast development campaign in exchange for all the rewards that could fall its way with success. And now that the pharma giant has seized a solid lead in the race to the market, those rewards loom large.

SVB Leerink’s Geoff Porges has been running the numbers on Pfizer’s vaccine, the mRNA BNT162b2 program that the German biotech partnered on. And he sees a $3.5 billion peak in windfall revenue next year alone. Even after the pandemic is brought to heel, though, Porges sees a continuing blockbuster role for this vaccine as people around the world look to guard against a new, thoroughly endemic virus that will pose a permanent threat.

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CEO Grace Colón (InCarda)

Look­ing to re­pur­pose an old drug to treat ir­reg­u­lar heart­beats, In­Car­da rais­es $30M in first Se­ries C close

A little less than two years after completing its $42 million Series B round, InCarda has returned to the venture well.

The San Francisco-based biotech announced the first portion of its Series C on Wednesday, pulling in $30 million in new funding. Most of the money will give enough runway for InCarda’s InRhythm program, an inhaled therapeutic aiming to treat sudden episodes of irregular heartbeats, through its Phase II trials and prepare it for Phase III.

UP­DAT­ED: CRISPR Ther­a­peu­tics gets a snap­shot of off-the-shelf CAR-T suc­cess in B-cell ma­lig­nan­cies — marred by the death of a pa­tient

Just days after scientific founder Emmanuelle Charpentier shared the Nobel prize for her work on CRISPR/Cas9, CRISPR Therapeutics $CRSP is showing off a snapshot of success in their early-stage study for an off-the-shelf CAR-T approach to CD19+ B cell malignancies — a snapshot marred by the death of a patient who had been given a high dose of the treatment.

Using their gene editing tech, researchers for CRISPR engineered cells from healthy donors into an attack vehicle aimed at cancer, something that has been achieved with great success using patients’ own cells — the autologous approach. But autologous CAR-T is hampered by the more complex vein-to-vein requirement that delays treatment, and now CRISPR Therapeutics along with other players like Allogene are determined to replace the pioneers with CAR-T 2.0.

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