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

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Marianne De Backer (L) and Jeff Hatfield

Bay­er nabs star biotech Vi­vid­ion with a $2B buy­out and an ‘arms-length’ pact, pulling a part­ner out of the IPO con­ga line

Vividion is canceling that IPO it filed. Instead of following the industry-wide migration to Nasdaq, the biotech that has captured considerable attention for its still-preclinical work finding cryptic pockets to bind to on proteins is going to work for Bayer now.

The pharma giant is putting out word today that it has bought out Vividion for $1.5 billion in cash and another half-billion dollars in milestones.

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Tadataka Yamada (Photographer: Kiyoshi Ota/Bloomberg via Getty Images)

Sci­ence pi­o­neer, phar­ma re­search chief, glob­al health ad­vo­cate and biotech en­tre­pre­neur Tadata­ka ‘Tachi’ Ya­ma­da has died

Tadataka Yamada, a towering physician-scientist who made his name in academia before transforming drug development at GlaxoSmithKline and developing vaccines for malaria and meningitis at the Gates Foundation, died unexpectedly of natural causes at his home in Seattle Wednesday morning.

He was 76. Frazier Healthcare Partners’ David Socks confirmed his death.

Known widely by the mononym “Tachi,” Yamada had a globetrotting career and arrived in industry relatively late in life. A 2004 Independent article noted GSK had asked Yamada to stay on beyond his approaching 60th birthday, the company’s usual retirement age. Yamada would continue working for the next 17 years, steering the Gates Foundation’s global health division for 6 years, funding Jim Wilson’s gene therapy work when few would touch it, launching Takeda Vaccines and co-founding a series of high-profile biotechs.

Covid-19 roundup: Pfiz­er im­pos­es vac­cine man­date for US work­ers; WHO calls for mora­to­ri­um on boost­ers, while some coun­tries make plans any­way — re­port

As the US struggles to keep pace with the fast-spreading Delta variant, big companies like Walmart and Disney are imposing vaccine mandates for some workers. It may come as no surprise that Pfizer — the Big Pharma behind the US’ first authorized Covid-19 vaccine — is joining them.

Pfizer will start requiring all US employees and contractors to get vaccinated, or participate in weekly Covid-19 testing, spokesperson Pamela Eisele told Reuters. Workers outside the US are strongly urged to get a vaccine if they can, according to the report. And those with medical conditions or religious objections can seek accommodations.

Tien Lee, Aardvark Therapeutics CEO

Emerg­ing from stealth mode, Aard­vark rounds up enough cash to put its lead drug through Prad­er-Willi PhII

When Aardvark Therapeutics CEO Tien Lee started his work on the biotech’s lead candidate, appetite suppression was the goal for the small molecule.  Soon after, his team started to see added benefits with lower blood glucose levels and anti-inflammatory activity. On the tail end of that, the company has emerged from stealth mode and announced today that they’ve raised enough cash in the B round to cover mid-stage development work.

Josh Hoffman, outgoing Zymergen CEO (Zymergen)

UP­DAT­ED: Syn­bio uni­corn Zymer­gen jet­ti­sons found­ing CEO, cuts guid­ance as cus­tomers re­port lead prod­uct does­n't work

Zymergen, just months off a $500 million IPO that put the synthetic bio firm in rarified air, has now ejected its founding CEO and downgraded its revenue forecasts after customers reported its lead film product doesn’t work as advertised, the company said Tuesday afternoon.

CEO Josh Hoffman will leave his role and sacrifice his board seat immediately in favor of Jay Flatley, the former CEO of Illumina who will take the lead role on an interim basis as the company conducts a search for its next leader.

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UK re-in­ves­ti­gates Pfiz­er's eye-pop­ping price goug­ing on an epilep­sy drug

When a drugmaker raises the price of a drug in the US by more than 2,000% overnight, and without any particular reason for that increase, nothing typically happens to the company. No fines, no court orders, just business as usual.

Martin Shkreli’s decades-old anti-parasitic drug Daraprim was the perfect example — massive price spike on an old drug, lots of media attention, public outcry, congressional committees dragging his former company through multiple hearings, and at the end of it? Nothing happened to the price or the company (until generic competition came).

Thomas Lingelbach, Valneva CEO

A small vac­cine de­vel­op­er fa­vored by the UK gov­ern­ment in Covid-19 touts a PhI­II first in chikun­gun­ya

Before Valneva garnered the favor of the UK government as a potential supplier of Covid-19 vaccines, the French biotech prided itself on being the first company to bring a chikungunya vaccine into Phase III.

It now has positive pivotal results to back up the breakthrough therapy designation the FDA granted just weeks ago.

There are currently no approved jabs to prevent chikungunya virus infection despite decades of R&D efforts, a fact that underscores just how arduous traditional vaccine development can be, particularly for neglected tropical disease. In a absence of a major commercial market, the US government and NGOs such as CEPI have deployed various grants and incentives to spur on a small crew of academics and industry players, with Merck, via its acquisition of Themis, claiming a spot in that race.

Zymergen co-founders Zach Serber, Josh Hoffman, and Jed Dean (Zymergen via website)

Zymer­gen's sud­den im­plo­sion shocked biotech. A lin­ger­ing loan could make things even worse

As former synbio unicorn Zymergen picks up the pieces from its spectacular implosion Tuesday, an outstanding loan from Perceptive Advisors — the only blue-chip biotech crossover investor to touch Zymergen’s fundraising efforts — could make the situation worse, according to public documents.

In December 2019, more than a year before Zymergen filed for what would eventually become a $500 million IPO, the “biofacturing” firm signed a $100 million credit facility with Perceptive to help supplement the nearly $700 million the company had raised across four VC rounds.