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).

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.

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.