News brief­ing: No­var­tis' Zol­gens­ma hits block­buster sta­tus, trig­ger­ing $80M mile­stone; Caris rais­es $310M for ge­nom­ic pro­fil­ing ex­pan­sion

No­var­tis re­port­ed that Zol­gens­ma earned $291 mil­lion in Q3, which puts the SMA gene ther­a­py square­ly in the block­buster cat­e­go­ry. That, in turn, is pay­ing ben­e­fits to Re­genxbio, which pro­vid­ed the NAV AAV9 vec­tor used by Zol­gens­ma.

The biotech $RGNX re­port­ed Tues­day morn­ing that it is get­ting an $80 mil­lion mile­stone pay­ment from No­var­tis, which now has earned more than $1 bil­lion cu­mu­la­tive­ly from the new gene ther­a­py fran­chise. And with large per­cent­ages of new­borns now be­ing screened for the rare, lethal ail­ment, the fran­chise can con­tin­ue to grow.

Re­genxbio has a trove of gene ther­a­py tech which has been used at a va­ri­ety of gene ther­a­py shops around the world. Their orig­i­nal SMA deal was with AveX­is, which No­var­tis bought out.

‘Ex­cit­ing,’ ‘ex­tra­or­di­nary’ ‘leader’ Caris rais­es $310M for ge­nom­ic pro­fil­ing work

No one at Caris Life Sci­ences is un­der­selling the work they do.

The words “ex­tra­or­di­nary” and “unique” come in­to play fre­quent­ly for the Dal­las-based ge­nom­ic pro­fil­ing com­pa­ny, which has ma­jor league com­pe­ti­tion at places like Foun­da­tion Med­i­cine, now part of di­ag­nos­tics pow­er­house Roche. And “leader” seems to be pep­pered in just about every­where.

“The growth prospects are re­al­ly quite ex­tra­or­di­nary,” says vice chair­man Bri­an Brille, “and we’re a leader in this area.”

They are al­so quite pleased that they raised a pack­et of mon­ey for the com­pa­ny, which Brille says now em­ploys around 1,000 staffers, with plans to add more.

$310 mil­lion, to be ex­act. From a group of “lead­ing” in­vestors drawn to the AI-pow­ered ma­chine be­ing built at Caris — which has been busi­ly sign­ing up a host of can­cer cen­ters for their ser­vices.

There’s $235 mil­lion in eq­ui­ty from High­land Cap­i­tal Man­age­ment and Coat­ue, with T. Rowe Price As­so­ci­ates, Or­biMed, Mil­len­ni­um Man­age­ment, Neu­berg­er Berman, Clear­Bridge In­vest­ments, First Light As­set Man­age­ment and “oth­er undis­closed in­vestors.” And there’s $75 mil­lion in debt from Sixth Street com­ing in as an ex­ten­sion to the $150 mil­lion in struc­tured debt fi­nanc­ing Sixth Street in­vest­ed in 2 years ago.

“As tu­mor pro­fil­ing be­comes stan­dard prac­tice, it’s im­por­tant that we con­tin­ue to grow rapid­ly as we main­tain our lead­er­ship po­si­tion,” said CEO David Hal­bert.

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

In fi­nal days at Mer­ck, Roger Perl­mut­ter bets big on a lit­tle-known Covid-19 treat­ment

Roger Perlmutter is spending his last days at Merck, well, spending.

Two weeks after snapping up the antibody-drug conjugate biotech VelosBio for $2.75 billion, Merck announced today that it had purchased OncoImmune and its experimental Covid-19 drug for $425 million. The drug, known as CD24Fc, appeared to reduce the risk of respiratory failure or death in severe Covid-19 patients by 50% in a 203-person Phase III trial, OncoImmune said in September.

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Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

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John Maraganore, Alnylam CEO (Scott Eisen/Bloomberg via Getty Images)

Al­ny­lam gets the green light from the FDA for drug #3 — and CEO John Maraganore is ready to roll

Score another early win at the FDA for Alnylam.

The FDA put out word today that the agency has approved its third drug, lumasiran, for primary hyperoxaluria type 1, better known as PH1. The news comes just 4 days after the European Commission took the lead in offering a green light.

An ultra rare genetic condition, Alnylam CEO John Maraganore says there are only some 1,000 to 1,700 patients in the US and Europe at any particular point. The patients, mostly kids, suffer from an overproduction of oxalate in the liver that spurs the development of kidney stones, right through to end stage kidney disease.

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Bob Nelsen (Photo by Michael Kovac/Getty Images)

Bob Nelsen rais­es $800M and re­cruits a star-stud­ded board to build the 'Fox­con­n' of biotech

Bob Nelsen spent his pandemic spring in his Seattle home, talking on the phone with Luciana Borio, the scientist who used to run pandemic preparedness on the National Security Council, and fuming with her about the dire state of American manufacturing.

Companies were rushing to develop vaccines and antibodies for the new virus, but even if they succeeded, there was no immediate supply chain or infrastructure to mass-produce them in a way that could make a dent in the outbreak.

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News brief­ing: Ab­b­Vie part­ner Teneo­bio ex­pands tech li­cense with CAR-T play­er Po­sei­da; Ar­genx buys PRV from Bay­er for $98M

Teneobio may be best known for its pact with AbbVie and Gilead, but before its big break the bispecific player had licensed its antibodies for a different use: as binders in CAR-T therapies being developed by Poseida.

Now, the biotechs are expanding their partnership, with Poseida exercising four options to deploy Teneobio’s heavy chain only domain antibodies commercially.

The commercial licensing fees remained under wraps, but Teneobio is eligible for $250 million in milestones for these CAR-Ts against undisclosed targets.

Carl Hansen, AbCellera CEO (University of British Columbia)

From a pair of Air Jor­dans to a $200M-plus IPO, Carl Hansen is craft­ing an overnight R&D for­tune fu­eled by Covid-19

Back in the summer of 2019, Carl Hansen left his post as a professor at the University of British Columbia to go full time as the CEO at a low-profile antibody shop he had founded called AbCellera.

As biotech CEOs go, even after a fundraise Hansen wasn’t paid a whole heck of a lot. He ended up earning right at $250,000 for the year. His compensation package included a loan — which he later paid back — and a pair of Air Jordan tennis shoes. His newly-hired CFO, Andrew Booth, got a sweeter pay packet than that — which included his own pair of Air Jordans.

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Leonard Schleifer, Regeneron CEO (Andrew Harnik/AP)

Trail­ing Eli Lil­ly by 12 days, Re­gen­eron gets the FDA OK for their Covid-19 an­ti­body cock­tail

A month and a half after becoming the experimental treatment of choice for a newly diagnosed president, Regeneron’s antibody cocktail has received emergency use authorization from the FDA. It will be used to treat non-hospitalized Covid-19 patients who are at high-risk of progressing.

Although the Rgeneron drug is not the first antibody treatment authorized by the FDA, the news comes as a significant milestone for a company and a treatment scientists have watched closely since the outbreak began.

Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

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