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.”

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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Ed­i­tas and Cel­gene sub Juno are tack­ling hottest im­munother­a­py cells

As the first CRISPR-edited cancer patients watch their treatments unfold, one of the first CRISPR companies is rejigging a major oncology deal.

Editas Medicine is amending its long-running collaboration with Celgene and their subsidiary Juno Therapeutics. The new deal will expand the focus of their work to cover a subset of immune cells that have become an increasingly hot target for immunotherapy: gamma-delta cells.

Prakash Raman. Flagship

Flag­ship woos No­var­tis top deal­mak­er Prakash Ra­man in move to get the BD ball rolling ear­ly

Flagship Pioneering likes to be ahead of its times — so far ahead, perhaps, that it is often challenging to find partners for their startups while the scientific scaffolding is underway. But Prakash Raman is here to change that.

Raman, who most recently headed up business development at the Novartis Institutes for BioMedical Research, became Flagship’s first chief business development officer two weeks ago. By acting as a “central resource” for the 100 companies in the venture fund’s portfolio, he hopes to help entrepreneurs and management teams strategize about dealmaking to capture value beyond the near-term validation of their platform technologies, Raman told Endpoints News.

FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.
The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.
The insider take at the agency includes a note on two new safety signals seen in the big cardio outcomes study of the omega-3 fatty acid drug that shocked many analysts with a solid set of efficacy data. There’s a key concern over whether the use of mineral oil in the placebo skewed LDL levels in such a way that tilted the data in Amarin’s favor.
The FDA overview was written by John Sharretts, the acting deputy director in the Division of Metabolism and Endocrinology Products. 
On the safety side, the internal review focused on a 3.1% versus 2.1% rate of adjudicated events of atrial fibrillation or atrial flutter requiring hospitalization. But they also say a-fib shouldn’t confound the benefit-safety of the drug — given the improvement on MACE — or prevent its use. And then there was also a higher rate of bleeding events in the drug arm.

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Bill Haney, Skyhawk

Cel­gene ex­ecs shell out $92M cash for a pair of R&D deals that will fit per­fect­ly in their new home at Bris­tol-My­ers

With Bristol-Myers Squibb’s Celgene buyout all but complete, the BD teams are working in perfect synchrony now. The Celgene side is going back to Skyhawk, a darling of the crowd that set out to drug RNA, and they’re adding a suite of new programs that mesh perfectly with the new regime in charge.

Celgene is shelling out $80 million in a cash upfront to add oncology, immuno-oncology and autoimmune diseases to the initial roundup of neurological targets mapped early in Skyhawk’s existence.

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Reata's bar­dox­olone of­fers promise in pa­tients with rare kid­ney dis­or­der

After surprising Wall Street with positive data on its drug, omaveloxolone, in patients suffering from a notoriously hard-to-treat degenerative neuromuscular disorder last month, Reata Pharma on Monday unveiled pivotal results from a trial testing another drug, bardoxolone, in patients with a rare, genetic form of chronic kidney disease for which there exist no approved therapies.

Bardoxolone, like Reata’s other lead drug — omaveloxolone — is a small molecule engineered to bind to a gene called Keap1 to enhance the activity of the protein Nrf2 in order to defuse inflammation.

FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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Am­gen ax­es 149 of its staff in Cam­bridge of­fice; Evotec, Mil­li­pore­Sig­ma en­ter re­search pact

→ Amgen has submitted a Worker Adjustment and Retraining Act (WARN) — a warning of impending mass layoffs 60 days in advance of the date — to the state of Massachusetts in the wake of the company’s exodus from the neurosciences R&D sector. David Reese, the company’s R&D chief, said at the time that the company is cutting ties in the field to focus on other undisclosed areas. In its WARN notice, the Cambridge-based company stated that 149 of its employees would be affected — among the total 180 being let go. The terminations will take effect on December 31, 2019.