Mer­ck dou­bles down on Sky­hawk's tech to drug RNA, promis­ing $600M per au­toim­mune, meta­bol­ic tar­get

Ear­ly col­lab­o­ra­tions with big-name part­ners lent Sky­hawk Ther­a­peu­tics both the en­dorse­ments and the cap­i­tal to pur­sue a small mol­e­cule plat­form for drug­ging RNA. And they soon want­ed more. When Mer­ck signed on to dis­cov­er can­di­dates against neu­rode­gen­er­a­tion and can­cer last Ju­ly, Bio­gen was just ex­pand­ing the list of neu­ro tar­gets they want to go af­ter.

Now Mer­ck is com­ing back for its own round two, adding au­toim­mune and meta­bol­ic dis­eases to the menu.

Kath­leen Mc­Carthy

As in the orig­i­nal deal, the phar­ma gi­ant isn’t break­ing down the up­front, stick­ing to the $600 mil­lion per pro­gram fig­ure to il­lus­trate their in­ter­est here. Li­cens­es to any IP aris­ing from the col­lab­o­ra­tion goes to Mer­ck, which would take over de­vel­op­ment once it ex­er­cis­es an op­tion.

At the core of Sky­hawk’s plat­form is RNA splic­ing mod­i­fi­ca­tion — a way to coax dys­func­tion­al genes in­to pro­duc­ing func­tion­al pro­teins with­out al­ter­ing them per­ma­nent­ly. Kath­leen Mc­Carthy, its co-founder and CSO, was part of a team that hit on their phe­nom­e­non by re­verse en­gi­neer­ing how ris­diplam, an SMA drug now shep­herd­ed by Roche and PTC Ther­a­peu­tics, worked.

The ap­proach of­fers a han­dle on un­drug­gable tar­gets, said Dean Li, SVP of dis­cov­ery sci­ences and trans­la­tion­al med­i­cine at Mer­ck Re­search Lab­o­ra­to­ries.

Bill Haney

Bill Haney, the an­gel in­vestor and CEO, bet that it could ap­ply broad­ly be­yond rare dis­eases. Mul­ti­ple drug de­vel­op­ers have since chimed in over $300 mil­lion up­front to match the $8 mil­lion seed round he ini­tial­ly put to­geth­er with some un­con­ven­tion­al back­ers.

“Mer­ck has been a won­der­ful part­ner in dis­cov­er­ing nov­el drug can­di­dates for neu­ro­log­i­cal dis­eases and can­cer,” Haney said in a state­ment.

Oth­er biotechs have sprung up in the RNA mod­u­la­tion space, in­clud­ing Ri­bometrix, which counts Mer­ck as an in­vestor, as well as Ar­rakis, where Michael Gilman has stuck to a more mea­sured ap­proach on the BD front.

So Covid-19 leader BioN­Tech has a can­cer vac­cine in de­vel­op­ment? Yes, and Re­gen­eron just jumped in for the PhII com­bo study

Before the coronavirus global emergency stole the R&D show in biopharma, the leaders in the race to develop new mRNA therapies had a big interest in determining if their tech could be used to create an effective cancer vaccine after all the first-gen tries had failed to impress. So perhaps it’s not surprising that an early cut of the data at frontrunner BioNTech went largely unnoticed.

Unless you were at Regeneron.

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Elizabeth Nabel speaks at a news conference, Oct. 7, 2019 (Elise Amendola/AP Images)

Brigham and Wom­en's pres­i­dent Eliz­a­beth Nabel fol­lows Mon­cef Slaoui off Mod­er­na's board

Amid recent scrutiny on how Moderna’s top executives have been cashing out their increasingly valuable shares, the biotech is parting ways with a board member who’s also heading a hospital where its Covid-19 vaccine is being tested.

Elizabeth Nabel — the president of Brigham and Women’s Hospital — has followed in Moncef Slaoui’s footsteps in resigning from Moderna’s board of directors. She took the role in 2015, two years before the Operation Warp Speed leader did; and as with Slaoui and MIT professor Robert Langer, her term was due to expire in 2021.

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Tony Coles, Cerevel Therapeutics CEO

Adding $445M, Tony Coles and his big Pfiz­er neu­ro spin­out hitch a ride to Wall Street on Per­cep­tive’s SPAC

Two years ago, after Pfizer abruptly shut down its entire neuroscience division, Bain Capital bet $350 million that those assets were still worth something and packaged them into a new biotech: Cerevel Therapeutics. A year later, they got seasoned executive Tony Coles, who had recently jumped back into the C-suite of another neuroscience startup, to run the company.

Now Coles is steering Cerevel public, in what he says is the largest ever transaction of its kind. Cerevel has agreed to merge with Perceptive Advisors’ specialty acquisition company ARYA II. Between the roughly $125 million Perceptive raised through ARYA and an additional investment of $320 million Bain Capital, Perceptive and — yes, really — Pfizer, among others, Cerevel will now move forward with an added $445 million in its coffers.

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Sanofi and GSK say they're near a vac­cine deal with EU hours af­ter fi­nal­iz­ing Warp Speed con­tract

On the heels of landing the largest Warp Speed contract to date, Sanofi and GlaxoSmithKline continued to make moves Friday afternoon.

The two companies announced they are in advanced discussions with the EU to supply up to 300 million doses of their Covid-19 vaccine candidate, coming just a few hours after securing their $2.1 billion deal with the US. Should the agreement be finalized, all EU member states will have the option to purchase the vaccine.

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Roche de­clares a PhI­II fail­ure for Covid-19 as the IL-6 re­pur­pos­ing the­o­ry bites the dust

Another big IL-6 drug has failed to move the needle for Covid-19 patients, leaving that particular field of repurposed drug R&D on the ropes for the pandemic.

This morning it was Roche’s turn to outline a Phase III failure for Actemra, adding compelling data that have now all but extinguished the theory that an IL-6 drug could significantly help the most severely afflicted patients. That comes just weeks after Regeneron and Sanofi hit the red light on their trial for Kevzara after getting back-to-back readouts that made Roche’s trial a long shot at best.

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Sandy Macrae, Sangamo CEO

No­var­tis turns to Sang­amo with a $795M-plus deal aimed at us­ing zinc fin­ger tech for the neu­ro pipeline

When Novartis recruited Ricardo Dolmetsch from Stanford to lead its neuroscience group, great emphasis was placed on decoding genomics and the brain circuitry to find new breakthroughs for the beleaguered field. Seven years and a failed Fragile X therapy later, he has a new tool to go after some of these targets his team has unearthed.

In a new collaboration, Novartis is paying Sangamo Therapeutics $75 million upfront to leverage zinc finger protein transcription factors in the regulation of three genes ties to autism — a core focus of Dolmetsch’s academic research days — intellectual disability and other neurodevelopmental disorders. Another $720 million in milestones are on the table, alongside a pledge to reimburse all of Sangamo’s research work.

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Ludwig Hantson, Alexion CEO

UP­DAT­ED: The lead drug in Alex­ion’s $930M buy­out deal last fall just flopped — adding in­jury to an­a­lysts’ M&A in­sults

When Alexion $ALXN put down $930 million in cash last fall to buy Achillion, the biotech’s top execs were particularly proud of 2 clinical-stage assets, with a spotlight on the lead drug danicopan (ACH-4471) in Phase II. That drug, along with a companion therapy in Phase I, fit right in their R&D wheelhouse, noted CEO Ludwig Hantson.

But now the lead drug, redubbed ALXN2040, is being washed out and repositioned after failing 2 Phase II trials for C3 Glomerulopathy.

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Robert Nelsen (Illustration by Emma Kumer for Endpoints News)

Af­ter Big Phar­ma aban­doned in­fec­tious dis­eases, 5 biotech con­trar­i­ans de­cid­ed to go all in. Then Covid-19 changed every­thing

Bob Nelsen had been quietly wondering how to eradicate viruses for years, before one day in 2015, he welcomed a pair of immunologists into the ARCH Venture Partners offices on the 34th floor of Seattle’s Wells Fargo Building.

Louis Picker and Klaus Früh, professors at Oregon Health & Science University, had by then spent 5 years running around the country in search of funding for their startup, TomegaVax, and Früh, at least, was nearing wit’s end. The Gates Foundation was interested but told them they needed other investors. Investors told them to come back with more data, pharmaceutical executives said they’re in the wrong game — too little money to be made fighting infectious disease. Still, a well-connected board member named Bob More landed them a meeting with the coveted venture capitalist, and so, in a narrow conference room overlooking the Puget Sound, Picker prepared to again explain the idea he had spent 15 years on: re-engineering a benign microbe into the first vaccines for HIV and better ones for hepatitis and tuberculosis.

“This lightbulb went on his head,” Picker recalled in a recent interview. “Most of them just didn’t get it. And Bob’s hit.”

By that point, Nelsen was more than just a venture capitalist. Scraggly and greying but no less opinionated at 52, he was mobbed at biotech conferences, having earned a reputation for crass wisdom and uncanny foresight, for making big bets on big ideas that changed medicine. Those ideas included DNA sequencing, which he first cut a check for in the 90s, and leveraging the immune system to tackle cancer. He earned millions making billion-dollar companies.

Yet for years he had harbored an almost singular obsession: “I hate viruses,” he told Forbes in 2016. He told me he was “pissed off” at them. The obsession drove him to his first biotech investment in 1993, for an inhalable flu vaccine approved a decade later and still in use. And it drove him to invest in CAR-T as a potential cure for HIV, years before it proved a wildly effective treatment for some cancers.

Now, listening to Picker talk about T cells and antibodies and the curious biology of cytomegalovirus, Nelsen began wondering if it was time for another bet. Picker’s technology was not only promising, he reasoned, it could be the basis of a company that changed how researchers approached viruses. Instead of trying to come up with an antidote for every pathogen, you could do what cancer researchers had learned to do, and harness the immune system to do the work for you.

This wasn’t a popular opinion at the time. “It’s like the least trendy idea in the world,” Nelsen told me. “People would say, ‘Why the hell are you going into infectious disease?’”

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Roche/Genen­tech dou­bles down on tau, fronting $120M cash to al­ly on a 2-pronged strat­e­gy to fight Alzheimer’s

After butting up against repeated failures on the amyloid beta side of the R&D strategy for fighting Alzheimer’s, Roche is once again turning to the runner-up target that has been getting an increased focus in the hopes of finding something — anything — that can slow the progress of this disease.

The terms of the deal include $120 million upfront for UCB, plus potential development and sales milestones, cost reimbursement and royalties totaling nearly $2 billion. UCB will fund and conduct a proof-of-concept study in Alzheimer’s, and with the results in hand, Genentech, a Roche subsidiary, can either return the rights to UCB or push ahead in a drive to the market.

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