At­las, No­vo-backed biotech reels in ex-Nim­bus CEO Don Nichol­son as ex­ec chair­man, hooks $50M to con­quer ane­mia

Bri­an Mac­Don­ald’s orig­i­nal for­ay in­to treat­ing iron dys­reg­u­la­tion by in­ject­ing hep­cidin, a small pep­tide hor­mone key for iron home­osta­sis, didn’t quite get off the ground. This time, the for­mer GSK ex­ec­u­tive is tak­ing a fresh ap­proach — by reg­u­lat­ing hep­cidin ex­pres­sion with­in the body.

His new com­pa­ny, called Disc Med­i­cine, was seed­ed by At­las Ven­ture in 2017. On Tues­day, it emerged with $50 mil­lion in se­ries A fund­ing, led by No­vo Hold­ings A/S and the par­tic­i­pa­tion of Ac­cess Biotech­nol­o­gy and found­ing in­vestor At­las Ven­ture. The Cam­bridge, Mass­a­chu­setts-based com­pa­ny has al­so lured the for­mer CEO of Nim­bus Ther­a­peu­tics, Don Nichol­son, as its ex­ec­u­tive chair­man.

Hep­cidin, or he­pat­ic bac­te­ri­ci­dal pro­tein, was ini­tial­ly iden­ti­fied as a uri­nary an­timi­cro­bial pep­tide rich in cys­teine. It is pro­duced in the liv­er — and now un­der­stood to con­trol the ab­sorp­tion of iron from the di­et and the trans­fer of iron from cel­lu­lar stores for in­fu­sion in­to he­mo­glo­bin.

“It is to iron, what in­sulin is to glu­cose,” Mac­Don­ald told End­points News.

Di­min­ished hep­cidin lev­els trig­ger an iron over­load, while high hep­cidin starves the body of iron and lim­its its abil­i­ty to make red blood cells. Chron­ic hep­cidin dys­reg­u­la­tion is im­pli­cat­ed in con­di­tions as­so­ci­at­ed with im­paired red blood cell pro­duc­tion, such as myelodys­plas­tic syn­dromes, tha­lassemia, and ane­mia.

Disc Med­i­cine, which in­tends to ad­dress both iron over­load and iron de­fi­cien­cy, has two pro­grams in its ar­se­nal. The first is an oral in­hibitor en­gi­neered to in­hib­it ma­trip­tase-2, a key sup­pres­sor of he­pat­ic hep­cidin ex­pres­sion, to treat iron load­ing ane­mias. It is in the lead op­ti­miza­tion stage, and the com­pa­ny plans to have a de­vel­op­ment can­di­date in place in the next twelve months.

The sec­ond pro­gram, which Disc in-li­censed from Ab­b­Vie, is a mon­o­clon­al an­ti­body that tar­gets he­mo­ju­velin — a reg­u­la­tor of hep­cidin pro­duc­tion — to sup­press hep­cidin ex­pres­sion and tack­le ane­mia that is as­so­ci­at­ed with a range of chron­ic in­flam­ma­to­ry and hema­to­log­ic dis­eases.  It is in pre­clin­i­cal de­vel­op­ment.

If all goes well, Mac­Don­ald an­tic­i­pates both pro­grams should be in the clin­ic by 2021.

Back in 2011, Mac­Don­ald co-found­ed Mer­ganser Biotech to fo­cus sole­ly on treat­ing dis­or­ders as­so­ci­at­ed with iron over­load, by in­ject­ing hep­cidin mimet­ic pep­tides. But that ef­fort flailed in Phase I, he said.

Oth­er drug de­vel­op­ers are still bet­ting the ap­proach will work, in­clud­ing Pro­tag­o­nist Ther­a­peu­tics. The Newark, Cal­i­for­nia-based com­pa­ny’s lead ex­per­i­men­tal drug, PTG-300, is a hep­cidin mimet­ic pep­tide in mid-stage de­vel­op­ment for use in be­ta-tha­lassemia.

For Mac­Don­ald, the Mer­ganser fail­ure came with perks — he re­al­ized that an oral small mol­e­cule ap­proach is more pa­tient-friend­ly and that it was “prob­a­bly safer to ad­min­is­ter some­thing that changes the body’s pro­duc­tion hep­cidin in rather than hav­ing to ad­min­is­ter an­oth­er dose of hep­cidin.”

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.

Su­per-se­cre­tive an­ti-ag­ing biotech Cal­i­co tees up the first vis­i­ble clin­i­cal tri­al of an ex­per­i­men­tal drug. And it’s for can­cer?

Over the past 7 years, Calico has been so much more than your average, run-of-the-mill secretive biotech players. It’s a riddle, wrapped in a mystery, inside an enigma, to repurpose an old Winston Churchill line dating from the time he confronted the Iron Curtain surrounding Stalin’s thoughts.

Launched by industry legend Art Levinson of Genentech fame, with the infinitely deep pockets of Google for support, one of the few big headlines the anti-aging biotech has sparked focused on a major alliance with AbbVie — a giant outfit that conversely likes to show off its drug prospects whenever it can. Together, they’ve been focused on diseases that limit life span — quite an arc of ailments.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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Derek Chalmers, Cara Ther

Cara lines up a $440M deal for US rights to its late-stage drug for se­vere itch, with $150M cash on the ta­ble

With plans afoot to file an NDA for what could be its first approved drug, Cara Therapeutics is pivoting its focus to commercialization. And Swiss company Vifor Pharma is willing to surrender up to $440 million to market the candidate in the US.

Cara $CARA CEO Derek Chalmers said an NDA submission is coming this quarter for their intravenous drug Korsuva in chronic kidney disease-associated pruritus (CKD-aP), a severe itching condition. The Stamford, CT-based biotech read out positive topline data from a Phase III pivotal study back in April, and announced plans to approach EMA regulators shortly after filing with the FDA.

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Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Gilead feels the heat as close al­ly Gala­pa­gos re­ports a big set­back on one of their top ex­per­i­men­tal drugs

The bad news keeps stacking up at Galapagos — which quite likely just lost control of a billion-dollar deal — and by extension their close partners at Gilead.

The biotech $GLPG reported after the bell Thursday that GLPG1972, one of their top development programs, flat failed a mid-stage study for osteoarthritis, flunking the primary and all secondary endpoints.

Testing 3 different doses of their drug, which relies on ADAMTS-5 inhibition, investigators concluded that none of them triggered a statistically significant response — as measured by cartilage thickness.

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Covid-19 roundup: Pars­ing Bourla, a top an­a­lyst sees im­proved chances for Pfiz­er vac­cine; Fau­ci: No sur­prise that Trump was hit by Covid-19

With a medley of adverse events hobbling the late-stage development of vaccines and drugs, Pfizer CEO Albert Bourla’s latest — extended — timeline for the mRNA approach they’re working on with BioNTech is giving some top analysts added confidence that the pharma giant can come up with the regulatory goods next month.

Parsing Bourla’s language in his comments last week, SVB Leerink’s Geoffrey Porges notes that Bourla’s decision to say they “may” be able to nail down the positive efficacy of their vaccine in a matter of days — a big change from his earlier certainty — may also indicate a delay on that to early November.

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CAR-plus: Irish biotech re­cruits Kite alum Chris Now­ers to prep dual-tar­get­ing NK cell ther­a­py for the clin­ic

Soon after Chris Nowers left Cell Medica — freshly rebranded Kuur Therapeutics — in February, the Kite Pharma alum was introduced to another cell therapy player.

The basic idea of building an off-the-shelf allogeneic platform with a CAR-NK approach was familiar to him, riding on the same wave as Takeda, J&J-backed Fate, Nkarta and others. But then there was something else that stood out: a membrane-bound TNF related apoptosis inducing ligand variant, or TRAIL variant, that’s also engineered onto the NK cell for a dual-targeted attack.

Mer­ck touts new da­ta for Keytru­da com­bos in NSCLC at North Amer­i­can con­fer­ence

Merck marched out new data from two studies on Friday to back king Keytruda — the drug that made the Big Pharma $11.1 billion last year — in advanced non-small cell lung cancer (NSCLC).

At the IASLC 2020 North America Conference on Lung Cancer, Merck read out long-term data from Cohort G of its Keynote-021 study, which assessed Keytruda in combination with chemotherapy. It also touted results from a Phase I/II study testing Keytruda and quavonlimab, its anti-CTLA-4 therapy, as a first-line therapy.

IN8bio CEO William Ho (IN8bio)

Bring­ing their ge­net­i­cal­ly mod­i­fied gam­ma delta T cells to Nas­daq, IN8bio files for $86M IPO

The biotech IPO parade continues marching forward as 2020 turns toward the fourth quarter.

IN8bio, a New York-based company focused on genetically modified gamma delta T cell therapies, filed to go public Friday seeking an $86 million raise. The company has two clinical-stage candidates being studied in glioblastoma and leukemia, respectively.

By any stretch of the imagination, 2020 has already been a huge year for biotech, and nowhere does it appear more obvious than the vast amounts of companies hitting the public market.