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).

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

Bio­gen hit by ALS set­back with PhI­II fail­ure for tofersen — but fol­lows a fa­mil­iar strat­e­gy high­light­ing the pos­i­tive

Patients and analysts waiting to hear Sunday how Biogen’s SOD1-ALS drug tofersen fared in Phase III didn’t have to wait long for the top-line result they were all waiting for. The drug failed the primary endpoint on significantly improving the functional and neurologic decline of patients over 28 weeks as well as the extension period for continued observation.

In fact, there was very little difference in response.

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UP­DAT­ED: Boehringer nabs FDA's first in­ter­change­abil­i­ty des­ig­na­tion for its Hu­mi­ra com­peti­tor — but will it mat­ter?

The FDA late Friday awarded Boehringer Ingelheim the first interchangeability designation for its Humira biosimilar Cyltezo, meaning that when it launches in July 2023, pharmacists will be able to automatically substitute the Boehringer’s version for AbbVie’s mega-blockbuster without a doctor’s input.

The designation will likely give Boehringer, which first won approval for Cyltezo in 2017, the leg up on a crowded field of Humira competitors.

Reshma Kewalramani, Vertex CEO (YouTube)

Ver­tex gets much-need­ed win with ‘ex­tra­or­di­nary’ first pa­tient re­sults on po­ten­tial di­a­betes cure

Vertex said Monday that the first patient dosed with its cell therapy for type 1 diabetes saw their need for insulin injections vanish almost entirely, a key early step in the decades-long effort to develop a curative treatment for the chronic disease.

The patient, who had suffered five potentially life-threatening hypoglycemic — or low blood sugar — episodes in the year before the therapy, was injected with synthetic insulin-producing cells. After 90 days, the patient’s new cells produced insulin steadily and ramped up their insulin production after a meal like normal cells do, as measured by a standard biomarker for insulin production.

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No­var­tis de­vel­op­ment chief John Tsai: 'We go deep in the new plat­form­s'

During our recent European Biopharma Summit, I talked with Novartis development chief John Tsai about his experiences over the 3-plus years he’s been at the pharma giant. You can read the transcript below or listen to the exchange in the link above.

John Carroll: I followed your career for quite some time. You’ve had more than 20 years in big pharma R&D and you’ve obviously seen quite a lot. I really was curious about what it was like for you three and a half years ago when you took over as R&D chief at Novartis. Obviously a big move, a lot of changes. You went to work for the former R&D chief of Novartis, Vas Narasimhan, who had his own track record there. So what was the biggest adjustment when you went into this position?

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Susan Galbraith, Executive VP, Oncology R&D, AstraZeneca

As­traZeneca on­col­o­gy R&D chief Su­san Gal­braith: 'Y­ou're go­ing to need or­thog­o­nal com­bi­na­tion­s'


Earlier in the week we broadcast our 4th annual European Biopharma Summit with a great lineup of top execs. One of the one-on-one conversations I set up was with Susan Galbraith, the oncology research chief at AstraZeneca. In a wide-ranging discussion, Galbraith reviewed the cancer drug pipeline and key trends influencing development work at the pharma giant. You can watch the video, above, or stick with the script below. — JC

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Two drug­mak­ers hit with PDU­FA date de­lays from FDA amid back­log of in­spec­tions

As the FDA is weighed down with more and more pandemic responsibilities, the agency is beginning to miss PDUFA dates with more frequency too. Two different companies on Monday said they received notices that the FDA has not completed their drug reviews on time.

The review of an NDA for Avadel Pharmaceuticals’ candidate treatment for narcolepsy is not coming this month, the company said, and the review of UCB’s BLA for bimekizumab, used to treat moderate to severe plaque psoriasis, will miss its target date as well.

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Terrie Curran, Phathom CEO (Credit: Arcutis)

Phath­om's old Take­da drug bests Pre­vacid in a PhI­II GI tri­al. Next stop? The FDA

There’s no time for rest in biopharma — at least not at Phathom Pharmaceuticals. Just over a month after submitting two NDAs for its lead acid-fighter vonoprazan, the biotech is already lining up a third, and collecting an extra $50 million to push things along.

Vonoprazan met its primary non-inferiority endpoints in a Phase III study comparing it to standard-of-care Prevacid in a type of gastroesophageal reflux disease (GERD) called erosive esophagitis (EE). It also proved superior to the popular heartburn drug by multiple measures, including healing rate and maintenance of healing.

Thomas Lingelbach, Valneva CEO

Small biotech says its Covid-19 vac­cine spurs more an­ti­bod­ies than As­traZeneca’s. Will sup­ply deals come now?

In a first, a small runner-up vaccine developer says its own Covid-19 jab has induced “superior neutralizing antibody titer levels” over AstraZeneca’s AZD1222 when pitted head-to-head in a Phase III trial.

That and non-inferiority in seroconversion rate were the co-primary endpoints of the trial, which recruited 4,012 adult volunteers across the UK.

But on the exploratory endpoint of Covid-19 case counts, Valneva notes that both treatment groups saw a similar number of infections.