Sebastian Nijman (file photo)

Roche looks to ge­net­ic mod­i­fiers for new drug tar­gets, team­ing up with Dutch biotech in $375M deal

Roche is gam­bling on a new way of dis­cov­er­ing drug tar­gets and, ul­ti­mate­ly, promis­ing to in­fuse more than $375 mil­lion in­to a small biotech if all goes well.

A spin­out of the Nether­lands Can­cer In­sti­tute and Ox­ford Uni­ver­si­ty, Scenic Biotech set out to pi­o­neer a field that’s gain­ing some trac­tion among top VCs in the US: to har­ness the nat­ur­al pro­tect­ing pow­ers of ge­net­ic mod­i­fiers — spe­cif­ic genes that sup­press a dis­ease phe­no­type.

Os­car Ize­boud

Af­ter each get­ting a PhD from the pres­ti­gious Dutch re­search cen­ter and spend­ing time in the Boston/Cam­bridge biotech hub, Thi­jn Brum­melkamp and Se­bas­t­ian Ni­j­man built their func­tion­al ge­nomics plat­form and named it Cell-Seq. The tech al­lows sci­en­tists to sift through all 20,000 genes in hap­loid cells (which have on­ly one set of chro­mo­somes), iden­ti­fy the genes that mod­i­fy a giv­en dis­ease, and cre­ate a li­brary of maps pin­point­ing them.

“There have been oth­er mod­i­fiers dis­cov­ered but these have most­ly been dis­cov­ered by serendip­i­ty,” Ni­j­man, who’s al­so the CSO, told End­points News. “What we are now do­ing is do­ing this sys­tem­at­i­cal­ly.”

Os­car Ize­boud, a for­mer banker who was re­cruit­ed to be CEO this Ju­ly, put it this way: “It’s a very fast way to find a nee­dle in a haystack.”

Once they find it, the strat­e­gy is to then find gene prod­ucts — pro­teins — that can be tar­get­ed with small mol­e­cule drugs or an­ti­bod­ies. In most cas­es they would be en­zymes, such as QPCTL, which Scenic be­lieves is a drug­gable mod­i­fi­er of the CD47 check­point.

The oth­er in­ter­nal ef­forts have main­ly fo­cused on rare in­her­it­ed meta­bol­ic dis­or­ders, where Ni­j­man and Ize­boud be­lieve the 20-peo­ple-and-grow­ing team can han­dle the clin­i­cal work.

Their new part­ners at Genen­tech are in­ter­est­ed in an area that Scenic has not gone in­to. In ad­di­tion to the undis­closed up­front, the deal cov­ers tar­get se­lec­tion fees as well as mile­stones and roy­al­ties.

Thi­jn Brum­melkamp

Scenic, though, isn’t the on­ly start­up claim­ing to have the nec­es­sary tools to un­pack ge­net­ic mod­i­fiers — an idea that’s cen­tral to the mys­ti­fy­ing no­tion that peo­ple born in­to the same fam­i­ly, with the same genes, don’t al­ways have the same dis­ease.

San Fran­cis­co-based Maze Ther­a­peu­tics launched last year with $191 mil­lion from a syn­di­cate led by ARCH and Third Rock, boast­ing a sim­i­lar ge­net­ics plat­form de­signed to un­earth new tar­gets. Nes­san Birm­ing­ham’s Triplet Ther­a­peu­tics, mean­while, is fo­cused on the DNA lev­el, lever­ag­ing an­ti­sense oligonu­cleotides to hit the DNA dam­age re­sponse path­way.

In com­par­i­son Scenic’s be­gin­ning was much more hum­ble, rais­ing €6.5 mil­lion from Eu­ro­pean firms Bio­Gen­er­a­tion Ven­tures and INKEF Cap­i­tal, with some sup­port from the uni­ver­si­ty-af­fil­i­at­ed Ox­ford Sci­ences In­no­va­tion and lat­er the Dutch gov­ern­ment.

But it’s not stop­ping Ni­j­man from dream­ing big.

Aside from the big Roche col­lab­o­ra­tion, he al­so has in his cor­ner sea­soned drug such as GSK vet Chas Boun­tra and cell ther­a­py ex­pert Ton Schu­mach­er, who’s just em­barked on his own new ven­ture with Arie Bellde­grun and David Chang. If any­thing, the buzz that ri­vals are gen­er­at­ing is wel­come.

“Peo­ple are start­ing to re­al­ize and wake up to the fact that this is now pos­si­ble,” Ni­j­man said. “We can now go af­ter this type of drug tar­get.”

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.

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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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.

Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

Here's how Bris­tol My­er­s' CEO Gio­van­ni Caforio com­plet­ed a $13B buy­out: He moved fast, upped the bid quick­ly and de­mand­ed every­one to keep up

Bristol Myers Squibb CEO Giovanni Caforio does not waste time. He also likes everyone around him to keep up.

Anyone reading over the insider account filed with the SEC of the back-and-forth over his $13 billion buyout of MyoKardia $MYOK could reach only one conclusion: The CEO who had willingly crafted a $74 billion Celgene acquisition had found something else he liked — and he was willing to pay a nice premium to get it.

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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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