Fast biotech: Gilead hands $200M to com­pu­ta­tion­al wiz Nim­bus as it speeds in­to PhII NASH tri­al

Nim­bus CEO Don Nichol­son

When Nim­bus Ther­a­peu­tics CEO Don Nichol­son struc­tured his $1.2 bil­lion li­cens­ing deal with Gilead $GILD ear­li­er this year, he built it with the Big Biotech’s rep for fo­cus, sci­ence and clin­i­cal speed in mind.

It paid off. Fast.

To­day, Cam­bridge, MA-based Nim­bus is re­veal­ing that it grabbed a $200 mil­lion mile­stone from Gilead for its ear­ly-stage NASH drug, just 6 months af­ter nab­bing a $400 mil­lion up­front pay­ment. The pay­out now gives them $600 mil­lion this year, half of that $1.2 bil­lion deal to­tal that Gilead signed off on.

“It’s a great drug for a com­pa­ny that re­al­ly needs a pipeline,” Nichol­son tells me about his new part­ners at Gilead. “Once they sink their teeth in­to some­thing, they go af­ter it ham­mer and tong.”

Nichol­son says the pay­out is a di­rect ben­e­fit of his de­ci­sion to over­look an­oth­er of­fer for more mon­ey — no, he’s not say­ing which com­pa­ny was left at the deal ta­ble — to fa­vor a part­ner that demon­strat­ed with hep C that it will move heav­en and earth to push rapid­ly through the clin­ic. What he al­so can’t say is ex­act­ly what the mile­stone pay­ment is for, though it seems rea­son­able to as­sume on my end that it might have some­thing to do with the quick ramp up for a mid-stage clin­i­cal pro­gram.

Work­ing with a com­pu­ta­tion­al chem­istry plat­form that has at­tract­ed the back­ing of Mi­crosoft mogul Bill Gates, Nim­bus built a pro­gram for a drug that tar­gets an en­zyme called Acetyl-CoA car­boxy­lase, or ACC, in­volved in the cre­ation of en­doge­nous fat­ty acids and the reg­u­la­tion of be­ta-ox­i­da­tion, de­scribed as the process by which fat­ty acids are bro­ken down at a cel­lu­lar lev­el.

Tack­ling ACC, if it works as planned, should pre­vent the buildup of lipids in the liv­er and break down the ones al­ready present, re­duc­ing the fi­bro­sis that is char­ac­ter­is­tic of the fat­ty liv­er dis­ease. For Gilead, it’s al­so a can­di­date for the kind of com­bi­na­tion ap­proach­es that they fa­vor in look­ing to com­mand a mar­ket like NASH.

Now with $600 mil­lion in hand from the sale of a sub­sidiary, all hands on deck at Nim­bus have switched to work­ing on pre­clin­i­cal ef­forts, with lead ef­forts un­der­way for Tyk2 and STING.

The Gilead deal came along at a per­fect time, adds the CEO. In late 2015, Nim­bus was work­ing on an S-1. Then the IPO mar­ket chilled over — par­tic­u­lar­ly for ear­ly-stage, high-risk biotechs. Now he has the cash to re­ward in­vestors as well as em­ploy­ees while stock­pil­ing enough cash to pay for years of run­way.

Says Nichol­son: “It gave us an aw­ful lot of fi­nan­cial op­tions.”

And the biotech can hang on to the S-1 for a bet­ter mo­ment on the mar­ket.

In cer­tain cir­cles among the scribes that cov­er biotech, biobucks have a faint scent of the dis­rep­utable. The cash awards of­fered for de­vel­op­ment and com­mer­cial mile­stones can be writ­ten off as un­at­tain­able stacks of Mo­nop­oly mon­ey, of­ten use­ful on­ly in the realm of fan­ta­sy — Lep­rechaun gold found in the pot at the end of every rain­bow.

But Nim­bus Ther­a­peu­tics of­fers us an ex­am­ple of how wrong that view can be.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

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Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

Rami Elghandour, Arcellx CEO

Up­dat­ed: Gilead, Ar­cel­lx team up on an­ti-BC­MA CAR-T as biotech touts a 100% re­sponse rate at #ASH22

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

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Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

David Light, Valisure CEO

Val­isure in the hot seat: New Form 483 over a 2021 in­spec­tion as CEO fires back

The notorious drug testing company Valisure, which has made a name for itself by forcing FDA’s hand with some of its safety-related uncoverings, received a letter this week after the FDA uncovered violations at its Connecticut-based testing lab in 2021.

The letter, which was sent on Dec. 5, stated that the FDA is “concerned” that Valisure was not aware of  drug supply chain security requirements.

Mark Cuban (Jed Jacobsohn/AP Images)

Mov­ing to the em­ploy­er side of health­care, Mark Cuban's Cost Plus Drugs part­ners with a PBM

From “Shark Tank” to direct-to-consumer generic drugs, Mark Cuban has made another inroad in the ongoing battle over prescription drug prices. His cost-plus-15% generic drug company, frequently undercutting many competitors, now has its sights set on the employer healthcare market.

Cost Plus Drugs, which originally pledged to cut out PBMs, has now partnered with the PBM EmsanaRx, majority owned by the Purchaser Business Group on Health, to launch a supplemental drug discount program designed specifically for self-funded employers, the company announced Thursday.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

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Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.