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.”

Once fu­ri­ous over No­var­tis’ da­ta ma­nip­u­la­tion scan­dal, the FDA now says it’s noth­ing they need to take ac­tion on

Back in the BP era — Before Pandemic — the FDA ripped Novartis for its decision to keep the agency in the dark about manipulated data used in its application for Zolgensma while its marketing application for the gene therapy was under review.

Civil and criminal sanctions were being discussed, the agency noted in a rare broadside at one of the world’s largest pharma companies. Notable lawmakers cheered the angry regulators on, urging the FDA to make an example of Novartis, which fielded Zolgensma at $2.1 million — the current record for a one-off therapy.

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Covid-19 roundup: GSK, Am­gen tai­lor R&D work to fit the coro­n­avirus age; Doud­na's ge­nomics crew launch­es di­ag­nos­tic lab

You can add Amgen and GSK to the list of deep-pocket drug R&D players who are tailoring their pipeline work to fit a new age of coronavirus.

Following in the footsteps of a lineup of big players like Eli Lilly — which has suspended patient recruitment for drug studies — Amgen and GSK have opted to take a more tailored approach. Amgen is intent on circling the wagons around key studies that are already fully enrolled, and GSK has the red light on new studies while the pandemic plays out.

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In a stun­ning set­back, Amarin los­es big patent fight over Vas­cepa IP. And its high-fly­ing stock crash­es to earth

Amarin’s shares $AMRN were blitzed Monday evening, losing billions in value as reports spread that the company had lost its high-profile effort to keep its Vascepa patents protected from generic drugmakers.

Amarin had been fighting to keep key patents under lock and key — and away from generic rivals — for another 10 years, but District Court Judge Miranda Du in Las Vegas ruled against the biotech. She ruled that:
(A)ll the Asserted Claims are invalid as obvious under 35 U.S.C.§ 103. Thus, the Court finds in favor of Defendants on Plaintiff’s remaining infringementclaim, and in their favor on their counterclaims asserting the invalidity of the AssertedClaims under 35 U.S.C. § 103.

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Covid-19 roundup: J&J, BAR­DA set ear­ly 2021 fin­ish line for $1B vac­cine race; FDA al­lows emer­gency drug use, ahead of piv­otal da­ta

J&J has zeroed in on a Covid-19 vaccine candidate that it hopes to begin testing in humans by September this year — with the extraordinary goal of getting it ready for emergency use in early 2021. And together with BARDA, it’s committing $1 billion to make it happen.

That kind of accelerated timeline would fall on the fast side of NIAID director Anthony Fauci’s well-publicized prediction that it would be another 12 to 18 months before a vaccine can be available for public use. A Phase I trial of Moderna’s mRNA vaccine began two weeks ago, and both the biotech and fellow mRNA player CureVac have discussed similar, if not even faster, timelines for emergency use among healthcare workers.

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As­traZeneca says its block­buster Farx­i­ga proved to be a game-chang­er in CKD — wrap­ping PhI­II ear­ly

If the FDA can still hold up its end of the bargain, AstraZeneca is already on a short path to scooping up a cutting-edge win with a likely approval for their SGLT2 drug Farxiga in cutting the risk of heart failure. Now the pharma giant says it can point to solid evidence that the drug — initially restricted to diabetes — also works for chronic kidney disease, potentially adding a blockbuster indication for the franchise.

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It is 'kind of a proven tech­nol­o­gy': Hep B vac­cine mak­er joins glob­al hunt for coro­n­avirus vac­cine

Using lab-grown proteins that are engineered to mimic the architecture of viruses to induce an immune response, VBI Vaccines is joining the hunt for a coronavirus vaccine — harnessing technology that has initially been proved safe in early trials as a prophylactic for cytomegalovirus (CMV) infection.

Unlike the raft of the companies in the Covid-19 vaccine race — including Moderna, CureVac and J&J — VBI is taking a pan-coronavirus approach, by developing a vaccine that will encompass Covid-19, severe acute respiratory syndrome (SARS), and Middle East respiratory syndrome (MERS).

Can a pair of top AveX­is alum­ni steer a new gene ther­a­py up­start to R&D glo­ry? 3 VCs bet $60M on it

VCs love few things more than a proven executive team when it comes to launching a new company. And now a group of A-listers has turned to a pair of top execs out of AveXis to steer the latest gene therapy player into the clinic.

The biotech is Waltham, MA-based Affinia and the two execs are Sean Nolan and Rick Modi — the former CEO and CBO respectively of AveXis, the gene therapy pioneer that fetched $8.7 billion in a sale to Novartis. Nolan has now taken the chairman’s role at Affinia while Modi moves up to the CEO post at the company.

Un­de­terred by a pan­dem­ic, Gilde Health­care rais­es their largest fund yet

When Pieter van der Meer started raising the capital for Gilde Healthcare’s fifth fund in the waning months of 2019, he had his eyes on a different chain of events that could change the healthcare system and perhaps even play to his firm’s advantage: The US presidential election.

Since raising their third fund in 2011, the 34-year-old Dutch firm had focused on value-based care. They chose late-stage biotechs that came up with new devices and delivery systems for de-risked established compounds, and when they chose preclinical biotechs, they spoke with potential pharma partners, payers and regulators to ask where and at what prices the drug made sense. As the Democratic primary became a contest over how to lower healthcare costs, it looked like a strategy that could pay off.

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Gilead CEO Dan O'­Day of­fers a de­tailed ex­pla­na­tion on remde­sivir ac­cess — re­as­sur­ing an­a­lysts that Covid-19 da­ta are com­ing fast

After coming under heavy fire from consumer groups ready to pummel them for grabbing the FDA’s orphan status for remdesivir — reserved to encourage the development of rare disease therapies — Gilead CEO Daniel O’Day had some explaining to do about the company’s approach to providing access to this drug to patients suffering from Covid-19. And he set aside time over the weekend to patiently explain how they are making their potential pandemic drug available in a new program — one he feels can better be used to address a growing pack of infected patients desperately seeking remdesivir under compassionate use provisions.

In addition to trying to reassure patients that they will once again have an avenue to pursue access, O’Day also reassured some analysts who had been fretting that China’s quick comeback from the coronavirus outbreak could derail its ultra-fast schedule for testing the drug in patients. The data are still expected in a few weeks, he says in the letter, putting the readout in April.

O’Day emphasizes that Gilead intends to pursue a pricing approach that will make this drug widely available — if it proves effective and safe. But no one is quite sure just what the longterm value would be, given the work being done on a variety of vaccines that may be rolled out as early as this fall — at least to the most heavily threatened groups.

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