Join­ing forces with Sky­hawk, C4 Ther­a­peu­tics, Bio­gen buys more shots on goal on SMA, Alzheimer's

These days at Bio­gen, the spot­light tends to fall on one of two pro­grams: Its com­mer­cial SMA drug Spin­raza, and the ex­per­i­men­tal Alzheimer’s ther­a­py ad­u­canum­ab — and its deal­mak­ing team knows it.

Michael Ehlers

In twin deals an­nounced on Fri­day, Bio­gen $BI­IB is ty­ing up with Sky­hawk Ther­a­peu­tics to ex­plore small mol­e­cule RNA splic­ing mod­i­fiers for spinal mus­cu­lar at­ro­phy and sign­ing on C4 Ther­a­peu­tics to re­search the ap­pli­ca­tion of pro­tein degra­da­tion tech in Alzheimer’s — among oth­er ear­ly-stage projects in neu­rol­o­gy.

The fi­nan­cial terms dis­closed so far add up to $489 mil­lion be­tween the two pacts.

Bill Haney’s Sky­hawk has the more de­lin­eat­ed deal: $74 mil­lion up­front for re­search ser­vices and in­tel­lec­tu­al prop­er­ty rights on pre­clin­i­cal can­di­dates to treat SMA, mul­ti­ple scle­ro­sis and ad­di­tion­al neu­ro­log­i­cal dis­or­ders. Any ther­a­pies re­sult­ing from the col­lab­o­ra­tion are up for grabs for Bio­gen, which will be re­spon­si­ble for de­vel­op­ment and com­mer­cial­iza­tion.

Kath­leen Mc­Carthy

Sky­hawk’s work is in­spired by an ex­pe­ri­enced group led by co-founder and CSO Kath­leen Mc­Carthy, who worked at Roche on the SMA drug RG7916 — now in piv­otal tri­als af­ter scor­ing pos­i­tive ear­ly re­sults — with a stint at the Spinal Mus­cu­lar At­ro­phy Foun­da­tion, where she had worked on a small mol­e­cule ther­a­peu­tic tar­get­ing mR­NA-pro­tein in­ter­ac­tions for SMA.

Both Roche and No­var­tis are now hot on the trail of Bio­gen’s Spin­raza, the pi­o­neer­ing ther­a­py in the field that’s al­so one of the world’s most ex­pen­sive drugs.

“The SMA piece of the Bio­gen col­lab­o­ra­tion is es­pe­cial­ly in­ter­est­ing as: (1) com­pe­ti­tion in the space has in­creased and (2) an oral small mol­e­cule drug from Roche has re­cent­ly shown very promis­ing ev­i­dence of ac­tiv­i­ty, val­i­dat­ing the vi­a­bil­i­ty of the oral splic­ing mod­u­la­tion ap­proach,” Stifel an­a­lysts wrote in a note.

C4, mean­while, will help Bio­gen iden­ti­fy tar­gets in Alzheimer’s, Parkin­son’s and oth­er dis­eases, and pro­vide the tech to tag these dis­ease-caus­ing pro­teins for de­struc­tion by the cell’s in­nate degra­da­tion mech­a­nism, ac­cord­ing to Michael Ehlers, EVP of R&D at Bio­gen. The whole col­lab­o­ra­tion is worth $415 mil­lion and though there’s no men­tion of the up­front, Bio­gen says it ex­pects to record an R&D ex­pense of $15 to $25 mil­lion in Q4 2018.

Found­ed by Jay Brad­ner be­fore he took the reins of the No­var­tis In­sti­tutes for Bio­Med­ical Re­search, C4 has on deck a col­lab­o­ra­tion with stealthy an­ti-ag­ing start­up Cal­i­co and an­oth­er on­col­o­gy pact with Roche, which got a $900 mil­lion ex­pan­sion Fri­day morn­ing.

 

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Covid-19 man­u­fac­tur­ing roundup: Mary­land looks to grow biotech ca­pac­i­ty with $400M check; Rus­sia lands sec­ond Sput­nik V part­ner this week

A Maryland real estate project has added three new biotech-focused manufacturing and research buildings to an office park to keep up with demand created by the pandemic, the Washington Business Journal reported.

The Milestone Business Park — located off of I-270 in Germantown, MD — will see the new buildings and a total of 532,000 square feet as the campus rebrands to Milestone Innovation Park.

Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.