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.”

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.

'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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