Ver­tex wa­gers $70M on Kymera and its new fas­ci­na­tion with pro­tein degra­da­tion

One of Ver­tex’s key promis­es to in­vestors and pa­tients has been that it will de­ploy its suc­cess in cys­tic fi­bro­sis — which fi­nal­ly turned the 30-year-old biotech in­to a prof­itable busi­ness three years ago — to fund more R&D projects with in­no­v­a­tive tech­nolo­gies. It has pro­pelled them to dive in­to gene edit­ing, team up with CRISPR Ther­a­peu­tics to test what could be a pi­o­neer­ing treat­ment for sick­le cell dis­ease.

With a new col­lab­o­ra­tion with At­las-in­cu­bat­ed Kymera Ther­a­peu­tics, Ver­tex $VRTX is putting its foot down in the bur­geon­ing pro­tein degra­da­tion field.

Ver­tex is in­fus­ing $70 mil­lion — part cash, part eq­ui­ty — to bankroll a 4-year re­search odyssey at Kymera to gen­er­ate clin­i­cal can­di­dates against up to six tar­gets for “se­ri­ous dis­eases.” A de­ci­sion to li­cense would kick off ad­di­tion­al mile­stone pay­ments rack­ing up to more than $1 bil­lion.

While both part­ners de­clined to dis­cuss the tar­gets, Ver­tex spokesper­son Heather Nichols said they will be con­sis­tent with its fo­cus on “val­i­dat­ed tar­gets that ad­dress causal hu­man bi­ol­o­gy.” Nel­lo Main­olfi, CSO and co-founder of Kymera, told me they bond­ed over a shared ea­ger­ness to iden­ti­fy the right re­spon­der pa­tient pop­u­la­tions and tar­gets.

Nel­lo Main­olfi

Hot on a trail blazed by the likes of C4 Ther­a­peu­tics and Arv­inas, Kymera boasts of a plat­form built on a com­plex ap­proach us­ing in­for­mat­ics-dri­ven tar­get iden­ti­fi­ca­tion cou­pled to pre­dic­tive mod­el­ing, new degra­da­tion tools as well as nov­el E3 lig­as­es and lig­ands. It promis­es to de­liv­er a more so­phis­ti­cat­ed way to tag dis­ease-caus­ing pro­teins for dis­pos­al — one that has gar­nered the in­ter­est of Glax­o­SmithK­line.

“The beau­ty about tar­get­ed pro­tein degra­da­tion is you [com­bine] the flex­i­bil­i­ty of small mol­e­cule, pow­er of ge­net­ic knock­down, and the pre­ci­sion of ge­net­ic ma­nip­u­la­tion in one sin­gle tech­nol­o­gy,” Main­olfi said.

Lau­rent Au­doly

Its in-house pipeline so far has fo­cused on on­col­o­gy and in­flam­ma­tion/im­munol­o­gy. This new part­ner­ship will help broad­en the plat­form’s ap­pli­ca­tion be­yond can­cer, CEO Lau­rent Au­doly said, a tes­ta­ment to the dis­ease ag­nos­tic po­ten­tial of Kymera’s tech as well as their am­bi­tion to grow in­to a ful­ly in­te­grat­ed shop.

Mean­while, Ver­tex’s un­der­tak­ings out­side of CF have spanned pain, blood dis­eases and a ge­net­ic dis­or­der dubbed al­pha-1 an­tit­rypsin de­fi­cien­cy (which caus­es lung and liv­er dam­age).

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; 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.

Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.

Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,600+ biopharma pros reading Endpoints daily — and it's free.