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.”

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Covid-19 vac­cine boost­ers earn big thumbs up, but Mod­er­na draws ire over world sup­ply; What's next for Mer­ck’s Covid pill?; The C-suite view on biotech; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

You may remember that at the beginning of this year, Endpoints News set a goal to go broader and deeper. We are still working towards that, and are excited to share that Beth Snyder Bulik will be joining us on Monday to cover all things pharma marketing. You can sign up for her weekly Endpoints MarketingRx newsletter in your reader profile.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'


Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Amit Etkin, Alto Neuroscience CEO (Alto via Vimeo)

A star Stan­ford pro­fes­sor leaves his lab for a start­up out to re­make psy­chi­a­try

About five years ago, Amit Etkin had a breakthrough.

The Stanford neurologist, a soft-spoken demi-prodigy who became a professor while still a resident, had been obsessed for a decade with how to better define psychiatric disorders. Drugs for depression or bipolar disorder didn’t work for many patients with the conditions, and he suspected the reason was how traditional diagnoses didn’t actually get at the heart of what was going on in a patient’s brain.

Roche's Tecen­triq cross­es the fin­ish line first in ad­ju­vant lung can­cer, po­ten­tial­ly kick­ing off gold rush

While falling behind the biggest PD-(L)1 drugs in terms of sales, Roche has looked to carve out a space for its Tecentriq with a growing expertise in lung cancer. The drug will now take an early lead in the sought-after adjuvant setting — but competitors are on the way.

The FDA on Friday approved Tecentriq as an adjuvant therapy for patients with Stage II-IIIA non small cell lung cancer with PD-(L)1 scores greater than or equal to 1, making it the first drug of its kind approved in an early setting that covers around 40% of all NSCLC patients.

Susan Galbraith speaking at Endpoints News' virtual EUBIO21 summit

Imfinzi/treme­li­mum­ab com­bo scores As­traZeneca an­oth­er OS win — this time in liv­er can­cer

Is the tide turning on AstraZeneca’s battered PD-L1/CTLA4 combo?

A single priming dose of the experimental tremelimumab, followed by Imfinzi every four weeks, beat Nexavar (sorafenib) in helping a group of liver cancer patients live longer in a Phase III study, the company reported, meeting the primary endpoint.

Specifically, the two drugs extended overall survival for patients with unresectable hepatocellular carcinoma who had not received prior systemic therapy and were not eligible for localized treatment.

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FDA ad­comm votes unan­i­mous­ly in sup­port of a J&J Covid-19 boost­er two months af­ter one-dose shot

The FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) on Friday voted 19-0 in favor of authorizing a second shot of J&J’s Covid-19 vaccine to follow at least two months after the initial dose.

Regulators don’t have to follow VRBPAC’s recommendation, but they almost always do. Considering that the CDC’s advisory committee has already been set to review the expanded EUA, VRBPAC’s recommendation is likely to be adopted.

FDA+ roundup: Marks on Wood­cock­'s tenure as act­ing com­mis­sion­er; FDA lead­ers of­fer per­spec­tive on bar­ri­ers to di­ver­si­ty in re­search

CBER director Peter Marks praised Janet Woodcock’s work as acting FDA commissioner, and while noting that Biden needs to nominate someone to fill the role permanently by Nov. 16, he said he has “no idea” when that actually might occur.

“Dr. Woodcock has been at the agency for over three decades and she, during that time, has proven herself to be a remarkably capable manager,” Marks said at the Alliance for Regenerative Medicine’s meeting Tuesday. “And she’s been managing as if she’s commissioner, unlike some previous acting [commissioners] who are afraid to actually do things. She doesn’t appear to be afraid to do things. I have not felt any different now from when we had a commissioner in place,” he added.

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