Al­bireo re­veals $385K price tag for new­ly ap­proved pru­ri­tis drug; Jen­nifer Doud­na's Cari­bou up­sizes IPO

Al­bireo scored a big break­through when it won ap­proval by the FDA and EMA this week for Byl­vay (ode­vix­i­bat), the first drug ap­proved to treat pru­ri­tus in pa­tients suf­fer­ing from all forms of pro­gres­sive fa­mil­ial in­tra­hep­at­ic cholesta­sis. And they wast­ed no time in rolling out a jaw-drop­ping price. In a call with an­a­lysts on Wednes­day, com­pa­ny ex­ecs out­lined a weight-based ap­proach they say will cre­ate an av­er­age per-pa­tient price of $385,000.

The com­pa­ny ex­pect­ed this ap­proval and had set up a full com­mer­cial­iza­tion plan, with re­im­burse­ment as­sis­tance and a plan to do every­thing they can to make sure pay­ers cov­er the bulk of the cost.

Peak sales pro­jec­tions have run a full gamut of num­bers. And even though their price is high, top sales may not amount to big num­bers. Cowen put peak US sales in 2026 at $163 mil­lion. — John Car­roll

Cari­bou up­sizes IPO ahead of of­fer­ing

Ahead of an an­tic­i­pat­ed IPO ei­ther lat­er Thurs­day or Fri­day, the Jen­nifer Doud­na-backed Cari­bou Bio­sciences is say­ing they’re get­ting more in­ter­est than ex­pect­ed.

Cari­bou up­sized its IPO of­fer­ing by 26% ahead of the pric­ing, per Re­nais­sance Cap­i­tal, pin­ning the raise es­ti­mate at $255 mil­lion. The range is ex­pect­ed to fall be­tween $14 to $16 per share, and Cari­bou will of­fer 3.5 mil­lion more shares to keep up with de­mand.

The biotech is us­ing its CRISPR-based DNA-RNA hy­brid plat­form to cre­ate chRD­NA ther­a­pies, pro­nounced “chardon­nay” like the wine. Cari­bou has an off-the-shelf CAR-T tar­get­ing CD-19 mak­ing its way through a Phase I study in B-cell non-Hodgkin lym­phoma.

Cari­bou filed for an IPO at the be­gin­ning of Ju­ly af­ter net­ting a $115 mil­lion Se­ries C round back in March. — Max Gel­man

Pro­found­Bio re­cruits Synaf­fix for ADC ef­forts

In mak­ing any an­ti­body-drug con­ju­gate, se­lect­ing the right link­er-pay­load is crit­i­cal, Pro­found­Bio CEO Bait­eng Zhao says — which is why he’s promis­ing up to $246 mil­lion for Synaf­fix’s ADC plat­form tech.

Pe­ter van de Sande

The com­pa­nies inked a li­cens­ing deal on Thurs­day for Synaf­fix’s Gly­co­Con­nect and Hy­dra­Space tech­nolo­gies. Com­ing out of Rad­boud Uni­ver­si­ty, Gly­co­Con­nect re­lies on gly­cans as an an­chor­ing point in an­ti­bod­ies, en­zy­mat­i­cal­ly re­mov­ing them to cre­ate space for the pay­load. Hy­dra­Space, on the oth­er hand, is Synaf­fix’s way of ex­tend­ing AD­Cs’ half lives.

While Pro­found­Bio will be re­spon­si­ble for the re­search, de­vel­op­ment, man­u­fac­tur­ing and com­mer­cial­iza­tion of its ADC prod­ucts, Synaf­fix will fo­cus on the man­u­fac­tur­ing of com­po­nents specif­i­cal­ly re­lat­ed to its plat­forms.

“The Pro­found­Bio team brings mul­ti­ple decades of valu­able ADC de­vel­op­ment ex­pe­ri­ence from Seat­tle Ge­net­ics (now Seagen) and MSD. in­to our col­lab­o­ra­tion,” Synaf­fix CEO Pe­ter van de Sande said in a state­ment. — Nicole De­Feud­is

No­var­tis reshuf­fles its wild cards; Tough sell for Bio­gen? Googling pro­teins; Ken Fra­zier's new gig; 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.

If you enjoy the People section in this report, you may also want to check out Peer Review, my colleagues Alex Hoffman and Kathy Wong’s comprehensive compilation of comings and goings in biopharma.

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Demis Hassabis, DeepMind CEO (Qianlong/Imaginechina via AP Images)

Google's Deep­Mind opens its pro­tein data­base to sci­ence — po­ten­tial­ly crack­ing drug R&D wide open

Nearly a year ago, Google’s AI outfit DeepMind announced they had cracked one of the oldest problems in biology: predicting a protein’s structure from its sequence alone. Now they’ve turned that software on nearly every human protein and hundreds of thousands of additional proteins from organisms important to medical research, such as fruit flies, mice and malaria parasite.

The new database of roughly 350,000 protein sequences and structures represents a potentially monumental achievement for the life sciences, one that could hasten new biological insights and the development of new drugs. DeepMind said it will be free and accessible to all researchers and companies.

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In­side Bio­gen's scram­ble to sell Aduhelm: Pro­ject 'Javelin' and pres­sure to ID as many pa­tients as pos­si­ble

In anticipation of Aduhelm’s approval for Alzheimer’s in June, Biogen employees were directed to identify and guarantee treatment centers would administer the drug through a program called “Javelin,” a senior Biogen employee told Endpoints News.

The program identified about 800 centers for use, he said, and Biogen now pays for the use of bioassays to identify beta amyloid in potential patients having undergone a lumbar puncture procedure, the employee said — and one center preparing to administer the drug confirmed its participation in the bioassay program.

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Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

No­var­tis dis­cards one of its ‘wild card’ drugs af­ter it flops in key study. But it takes one more for the hand

Always remember just how risky it is to gamble big on small studies.

A little more than 4 years ago, Novartis reportedly put up a package worth up to $1 billion for the dry eye drug ECF843 after a small biotech called Lubris put it through its paces in a tiny study of 40 moderate to severe patients, tracking some statistically significant markers of efficacy.

By last fall, the program had risen up to become one of CEO Vas Narasimhan’s top “wild card” programs in line for a potential breakthrough year in 2021. These drugs were all considered high-risk, high-reward efforts. And in this case, risk won.

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Mod­er­na es­tab­lish­es pub­lic health-fo­cused char­i­ty; FDA ap­proves As­traZeneca di­a­betes drug for pe­di­atric use

To help promote public health and healthcare in underserved areas of the world, Moderna will establish a charity with a $50 million endowment.

The Cambridge, MA-based company announced the board of directors’ approval Thursday. The foundation will focus on “charitable, scientific and educational endeavors” with an emphasis on promoting public health and the access to healthcare, the press release said. The foundation will start operations once its status as a 501(c)(3) is approved.

EMA re­jects FDA-ap­proved Parkin­son's drug, signs off on Mod­er­na vac­cine use in ado­les­cents ahead of FDA

The European Medicines Agency on Friday rejected Kyowa Kirin’s Parkinson’s disease drug Nouryant (istradefylline), which the US FDA approved in 2019 under the brand name Nourianz.

EMA said it considered that the results of the clinical studies used to support the application “were inconsistent and did not satisfactorily show that Nouryant was effective at reducing the ‘off’ time. Only four out of the eight studies showed a reduction in ‘off’ time, and the effect did not increase with an increased dose of Nouryant.”

6 top drug­mak­ers of­fer per­spec­tives on FDA's new co­vari­ates in RCTs guid­ance

Back in May, the FDA revised and expanded a 2019 draft guidance that spells out how to adjust for covariates in the statistical analysis of randomized controlled trials.

Building on the ICH’s E9 guideline on the statistical principles for clinical trials, the 3-page draft was transformed into an 8-page draft, with more detailed recommendations on linear and nonlinear models to analyze the efficacy endpoints in RCTs.

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Al Sandrock, Biogen R&D chief (Biogen via YouTube)

Bio­gen has a shaky end to H1 with a $542M write-off adding to its woes — but an­a­lysts see big rev­enue ahead for Aduhelm

All eyes at Biogen’s Q2 earnings call Thursday were on Aduhelm, but investors also got a glimpse of what Biogen would have faced had the FDA not opted to approve their controversial Alzheimer’s drug.

That glimpse, revealing a combination of declining sales, growing competition and failed medicines, underscores the stakes of the big biotech’s Aduhelm efforts, as execs punch back at the criticism they’ve engendered in the political and medical world and vigorously pushes its sales staff to roll out the drug as fast as possible.

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Mol­e­c­u­lar Di­ag­nos­tics Can Trans­form Can­cer Care. Let’s Make It Hap­pen.

Like so many people around the world, my life has been profoundly shaped by cancer. Those personal experiences, along with a deep love of clinical laboratory science and a passion to apply the power of genomics in medicine, motivated me to launch a company that would improve cancer care through better diagnostics. Thirteen years later, I am proud that we are delivering more accurate information at multiple points along the patient journey, with a focus on eight of the 10 cancers that are most commonly diagnosed in the United States.