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.”

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Pfiz­er's big block­buster Xel­janz flunks its post-mar­ket­ing safe­ty study, re­new­ing harsh ques­tions for JAK class

When the FDA approved Pfizer’s JAK inhibitor Xeljanz for rheumatoid arthritis in 2012, they slapped on a black box warning for a laundry list of adverse events and required the New York drugmaker to run a long-term safety study.

That study has since become a consistent headache for Pfizer and their blockbuster molecule. Last year, Pfizer dropped the entire high dose cohort after an independent monitoring board found more patients died in that group than in the low dose arm or a control arm of patients who received one of two TNF inhibitors, Enbrel or Humira.

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Steve Harr (L) and Hans Bishop

One of the most am­bi­tious start­up teams in biotech just out­lined plans for a $400M IPO and a val­u­a­tion of about $4B

The executive team at Sana Biotechnology has sketched out more details about the full scope of its ambitions as the new unicorn to watch. They amended their S-1 today to include a price range of $20 to $23 a share — which puts them in reach of pulling in around $400 million on the high end with a market value starting right around $4 billion.

That’s not bad for a preclinical biotech with no drugs yet in human studies, but it squares with its ambitions to remake the cell therapy field with a slate of in-house platforms. The biotech raised $705 million — primarily from ARCH (44 million shares) and Flagship (34.2 million shares) — to get to this stage.

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Top gene ther­a­py deals, M&A pacts in 2020 high­light an­oth­er big year in one of the hottest fields in bio­phar­ma

Chris Dokomajilar at DealForma has been crunching the numbers on gene therapy deals over the last 2 years and came away with a few key observations.

Both the upfront cash and deal totals last year backed off a bit from the record high hit in 2019, but the totals are still running well ahead of anything we’ve seen in the years prior to 2019/2020.
2020 R&D partnerships came in at 23 deals, with $1.1 billion in disclosed upfront cash and equity and more than $8.5 billion in total deal value. Looking at 2019-2020 M&A, Dokomajilar found: 9 Acquisitions, with over $11.1 billion in disclosed upfront cash and equity and more than $13.4 billion in total M&A value.

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Bob Nelsen (Michael Kovac/Getty Images)

ARCH an­nounces largest fund yet, rais­ing $1.85B to back men­tal health, cell and gene edit­ing ap­proach­es

Nearly a year ago, as the pandemic encroached and the stock market cratered, Flagship and ARCH Venture announced three mega-funds worth a combined $2.6 billion. They wanted, ARCH’s Bob Nelsen said, to restore confidence “that there was money out there and a lot of it” to invest in biotech.

Since then, the stock market has returned — almost frighteningly so — and Nelsen has kept raising and spending cash. On Thursday, he announced a new fund, worth $1.85 billion. It’s the largest pot yet for a VC famous for its deep pockets.

Lil­ly at­tempts to re­vive an old idea for tack­ling pain, li­cens­ing PhI pro­gram from Japan’s Asahi Ka­sei Phar­ma

Eli Lilly is fronting some new cash in a space they’re quite familiar with.

The company is partnering with Japan’s Asahi Kasei Pharma on an experimental drug for chronic pain, acquiring the rights for the P2X7 receptor antagonist program dubbed AK1780. Lilly will shell out a pretty penny for the program, promising up to $410 million total should each milestone payment come to pass.

Asahi Kasei will receive an upfront sum of $20 million for the candidate. In addition, Lilly is on the hook for up to $210 million in development and regulatory milestones and another potential $180 million in sales milestones. Asahi Kasei can also obtain royalties ranging from the mid-single to low-double digits should an approved product come out of the deal.

Ther­mo Fish­er plat­form seeks to ex­pe­dite donor cell cul­ti­va­tion for al­lo­gene­ic cell ther­a­pies

One of the world’s leading CDMOs has launched a new technology it says will expedite a quickly-growing sect of biotech drug development: off-the-shelf, allogeneic cell therapies.

It’s been nearly a decade since the FDA approved the first use of the method that uses healthy donor cells to create a master cell bank, which is then used for specific therapies — a cord blood allogeneic treatment called Hemacord. In the years since, the use of allogeneic cells has taken off in research circles, most notably in the use of T cell therapies to target solid tumor cancers.

Take­da earns win for its TKI in­hibitor in tiny lung can­cer group — but GI side ef­fects could be an ear­ly red flag

Japanese drugmaker Takeda has made a big push in recent years to build a hand in oncology, particularly in the next-gen cancer space. One of those candidates, tyrosine kinase inhibitor (TKI) mobocertinib, recently earned the FDA’s interest in a small section of untreated lung cancer patients, but will severe GI side effects be a roadblock?

Takeda’s oral mobocertinib posted clinically significant objective response rates in a Phase I/II adaptive trial drugging metastatic non-small cell lung cancer patients with EGFR exon 20 gene mutations who had previously undergone platinum-based chemotherapy, according to data presented Thursday at the virtual World Conference on Lung Cancer.

Covid-19 roundup: EU and As­traZeneca trade blows over slow­downs; Un­usu­al unions pop up to test an­ti­bod­ies, vac­cines

After coming under fire for manufacturing delays last week, AstraZeneca’s feud with the European Union has spilled into the open.

The bloc accused the pharma giant on Wednesday of pulling out of a meeting to discuss cuts to its vaccine supplies, the AP reported. AstraZeneca denied the reports, saying it still planned on attending the discussion.

Early Wednesday, an EU Commission spokeswoman said that “the representative of AstraZeneca had announced this morning, had informed us this morning that their participation is not confirmed, is not happening.” But an AstraZeneca spokesperson later called the reports “not accurate.”

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