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.

 

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Bris­tol-My­ers is clean­ing up the post-Cel­gene merg­er pipeline, and they’re sweep­ing out an ex­per­i­men­tal check­point in the process

Back during the lead up to the $74 billion buyout of Celgene, the big biotech’s leadership did a little housecleaning with a major pact it had forged with Jounce. Out went the $2.6 billion deal and a collaboration on ICOS and PD-1.

Celgene, though, also added a $530 million deal — $50 million up front — to get the worldwide rights to JTX-8064, a drug that targets the LILRB2 receptor on macrophages.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.