How CAMP4 maps transcription [CAMP4TX]

Tar­get­ing the BF­Ps of the CNS, Bio­gen li­cens­es map of ge­net­ic neigh­bor­hoods in the brain

Search­ing for new meth­ods of at­tack­ing se­ri­ous cen­tral ner­vous sys­tem dis­eases, Bio­gen has signed a deal that could be run in­to the hun­dreds of mil­lions of dol­lars to es­sen­tial­ly li­cense a tran­scrip­tion­al map of the brain and scan it for new drug tar­gets.

The part­ner­ship, worth $15 mil­lion up­front and far more in mile­stones, is with a new bioin­for­mat­ics start­up called CAMP4 Ther­a­peu­tics.  Found­ed in 2018 year by the White­head In­sti­tute’s Richard Young and Har­vard Med­ical School’s Leonard Zon, CAMP4 takes genes con­sid­ered af­fil­i­at­ed with a dis­ease and maps out the var­i­ous ways cells ex­press those genes and turn them on or off.  They then take that map and – in the bi­o­log­i­cal equiv­a­lent of stretch­ing a war map across a ta­ble in a bunker —  mark up all the dif­fer­ent meth­ods of at­tack.

“The more we know about a gene, it doesn’t mean we can drug that gene,” CEO Josh Man­del-Brehm, who worked at Bio­gen be­fore join­ing CAMP4, told End­points News. “Many genes are not quote-un-quote drug­gable, so then we have an in­ter­est­ing prob­lem: I think I un­der­stand the ge­net­ics of the dis­ease, but how do I drug it?  And that’s where our map solves for this prob­lem.”

Along­side the $15 mil­lion up­front pay­ment, CAMP4 will be el­i­gi­ble for up to $96 mil­lion in mile­stones for each of the ini­tial tar­gets it sup­plies, and up to $173 mil­lion for each sub­se­quent one.

CEO Josh Man­del-Brehm

Bio­gen is not dis­clos­ing what genes or dis­eases they will tar­get, but the col­lab­o­ra­tion will fo­cus on mi­croglial cells. These macrophages are found in the cen­tral ner­vous sys­tem and have long been con­sid­ered a key fac­tor in neu­rode­gen­er­a­tive dis­eases such as Alzheimer’s and Parkin­son’s.

Bio­gen will bring CAMP4 the genes it be­lieves are cen­tral to dis­eases in the cen­tral ner­vous sys­tem, and CAMP4 will in­form them all the ways the gene is be­ing ex­pressed and where Bio­gen might go about turn­ing up or down the ex­pres­sion.

“So you say ‘hey I want to move this par­tic­u­lar gene for this dis­ease, how should I do that? I want to change the ex­pres­sion of it,'” Man­del-Brehm said, de­scrib­ing how they work with part­ners. “We’ll show you the dif­fer­ent ‘nodes,’ if you will: ‘hey you can drug this, you can drug this, you can drug this.’ It’s modal­i­ty ag­nos­tic.”

CAMP4, found­ed out of Po­laris Part­ners, is one of a cou­ple of new biotechs that build on a dis­cov­ery Young had back in 2015. The long­time tran­scrip­tion­al bi­ol­o­gist pub­lished work show­ing that cells mod­i­fy gene ex­pres­sion us­ing a se­ries of “in­su­lat­ed neigh­bor­hoods,” 3-D loops of DNA that con­trol gene ex­pres­sion with­in them. CAMP4 maps those (along with oth­er as­pects of the tran­scrip­tome). Flag­ship Pi­o­neer­ing’s new Omega Ther­a­peu­tics al­so tries to drug them.

Since launch­ing in 2018, CAMP4 has done ex­ten­sive work on map­ping genes for liv­er dis­eases, in­clud­ing NASH, as part of an in-house de­vel­op­ment pro­gram. They’ve al­so part­nered with syn­thet­ic RNAi com­pa­ny Al­ny­lam and are work­ing to ex­pand their maps of cells in the brain, heart, mus­cles, im­mune sys­tem, blood and kid­neys, which they could then li­cense out or use to de­vel­op drugs in-house.

“The way I think about the dis­eases we’re go­ing af­ter is BFP: Big Fuck­ing Prob­lems,” Man­del-Brehm said, cit­ing NASH and oth­er dis­eases that lack ef­fec­tive treat­ments. And “It’s well-val­i­dat­ed and un­der­stood: If you’re drug­ging some­thing that’s tied to the gene, you’re much more like­ly to be suc­cess­ful.”

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

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Pascal Soriot (AP Images)

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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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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Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

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Vivek Ramaswamy (Jeff Rumans/JPM 2020)

Urovan­t's lead drug dis­ap­points in mid-stage study as first big FDA de­ci­sion looms

Just as Urovant gets ready for its first big FDA decision on vibegron, the drug has flopped in what would’ve been a follow-on indication.

In a Phase IIa trial involving women with abdominal pain due to irritable bowel syndrome, vibegron failed to meet the bar on improving “average worst abdominal pain” over 12 weeks, compared to placebo, among IBS-D patients.

There were actually slightly more responders in the placebo group than in the drug arm, with only 40.9% of those randomized to vigebron achieving at least a 30% decrease in “worst abdominal pain” in the past 24 hours. The trial enrolled 222 women but only 189 completed the study.

Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications.

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The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

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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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