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.

 

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End­points 20(+2) un­der 40, 2023; Bio­phar­ma's high­est-paid CEOs; N-of-1 CRISPR sto­ry goes on af­ter tragedy; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

We will be off Monday in observance of Memorial Day — and when we get back, it will be a straight march to ASCO, BIO and more. Enjoy the (long) weekend!

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Douglas Love, Annexon CEO

An­nex­on’s GA drug miss­es on pri­ma­ry goal but win on vi­su­al acu­ity will be fo­cus of planned late-stage tri­al

Annexon’s complement inhibitor didn’t prove better than sham at reducing lesion growth in a leading cause of blindness, but the biotech still plans to move forward on the back of secondary endpoints showing visual acuity preservation, which will “certainly” be the primary goal in a late-stage trial to be discussed shortly with the FDA, CEO Douglas Love told Endpoints News. 

The California biotech’s ANX007 was not statistically significant compared to pooled sham, the comparator, at 12 months in patients with geographic atrophy, per a Wednesday presentation. In every-month dosing, the GA lesion area changed about 6.2% from baseline (p=0.526) and 1.3% (p=0.896) in the every-other-month group. In a March note, Jefferies analyst Suji Jeong said a reduction of 20% to 30% would be “encouraging.”

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FDA ap­proves Lex­i­con’s heart-fail­ure drug af­ter de­feat in di­a­betes

The FDA on Friday approved Lexicon’s heart failure drug sotagliflozin following a string of setbacks for the pharma company, including an FDA rejection in diabetes and the loss of a development deal with Sanofi.

The dual SGLT1 and SGLT2 inhibitor will be marketed as Inpefa and is a once-daily tablet. It’s been approved to reduce the risk of cardiovascular death and heart failure-related hospitalization or urgent visits in adults with heart failure or type 2 diabetes mellitus, chronic kidney disease, and other cardiovascular risk factors. The label spans the range of left ventricular ejection fraction, including preserved ejection fraction and reduced ejection fraction, as well as patients with or without diabetes, Lexicon said Friday.

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Eu­ro­pean Com­mis­sion to re­ceive few­er Pfiz­er-BioN­Tech vac­cine dos­es un­der amend­ed con­tract

The European Commission has made a few changes to its vaccine contract with Pfizer and BioNTech, reducing the dose volume while extending the delivery timeline to cope with “evolving public health needs.”

The Commission previously struck a contract in May 2021 for 900 million doses, with the option to purchase another 900 million. Of those, 450 million were expected to be delivered in 2023, though an amendment now calls for fewer doses. While neither the Commission nor Pfizer and BioNTech have revealed an exact amount, an unnamed source told Reuters that the amendment reduces the remaining expected doses by about a third.

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EMA rec­om­mends re­vok­ing au­tho­riza­tion of No­var­tis' sick­le cell drug

The European Medicines Agency’s committee for medicinal products for human use (CHMP) on Friday recommended revoking the marketing authorization for Novartis’ treatment for a painful complication related to sickle cell, after a recent study did not confirm its clinical benefit.

CHMP’s review looked at results of the STAND study, finding that Adakveo (crizanlizumab) did not reduce the number of painful crises leading to a healthcare visit, and patients treated with Adakveo had slightly more painful crises on average, with a subsequent healthcare visit, over the first year of treatment, compared with those on placebo.

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Rich Horgan (R) with his late brother, Terry

Rich Hor­gan spear­head­ed a gene ther­a­py for his broth­er. The tri­al end­ed in tragedy, but the work con­tin­ues for more pa­tients

Rich Horgan’s quest to create a custom gene therapy for his brother, Terry, ended in tragedy. But Horgan doesn’t believe it’s the end of the story.

Terry, a 27-year-old patient with Duchenne muscular dystrophy, died last October just eight days after receiving the therapy in a clinical trial in which he was the only participant. The case raised questions about the safety of certain gene therapies and what would happen to other drug programs under a nonprofit that Horgan created, called Cure Rare Disease.

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Bio­phar­ma's 20 high­est-paid CEOs of 2022, each bring­ing in $20M+ pay­days

Even in a down year for much of the biopharma market, 20 CEOs brought in pay packages valued at more than $20 million, an Endpoints News analysis found.

Endpoints collected data on more than 350 CEO compensation packages, covering a wide range of pharma, biotech, and life sciences companies. All told, the 20 largest earners made over $725 million in 2022 — an average package of $36.4 million. Three brought in paydays over $50 million, and one CEO broke the $100 million mark.

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The 20(+2) un­der 40: Your guide to the next gen­er­a­tion of biotech lead­ers

This year’s list of 20 biotech leaders under the age of 40 includes a huge range of ambitions. Some of our honorees are planning to create the next big drug giant. Others are pushing the bounds of AI. One is working to revolutionize TB testing. All are compelling talents who are still young in age, but already far along in achievement.

And, as in years past, we went over. The 20 are actually 22 because of two double profiles that reflect how important teamwork is in the industry. As one of our honorees, Joe Illingworth of DJS Antibodies, told me in our interview, “It takes a village to raise a biotech.”

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