Hori­zon takes the leap in­to man­u­fac­tur­ing, buy­ing out an Irish plant with around 40 em­ploy­ees in tow

Hori­zon Ther­a­peu­tics, the biotech known for scoop­ing up or­phan drugs, has made its first for­ay in­to man­u­fac­tur­ing with the pur­chase of a fa­cil­i­ty in Wa­ter­ford, Ire­land.

Eir­Gen Phar­ma has sold its site to Hori­zon for $65 mil­lion and will trans­fer 40 em­ploy­ees to the com­pa­ny. The site is lo­cat­ed in an In­dus­tri­al De­vel­op­ment Agency Ire­land busi­ness park and will in­clude a fill­ing line and a freeze dry­er that can be used for Hori­zon’s com­mer­cial med­i­cines Te­pez­za, Up­liz­na and Krys­texxa. It will al­so be used for drug de­vel­op­ment, the com­pa­ny said in a re­lease.

Hori­zon is col­lab­o­rat­ing with Irish reg­u­la­tors and the FDA to be­come li­censed for fill-fin­ish man­u­fac­tur­ing and ex­pects the first med­i­cine will be ap­proved for re­lease in two years. The com­pa­ny will hire an­oth­er 50 staff mem­bers by the end of 2022, the com­pa­ny said.

“Adding to our strong net­work of con­tract man­u­fac­tur­ing or­ga­ni­za­tions, this fa­cil­i­ty will al­so play an im­por­tant role in our glob­al ex­pan­sion,” CEO Tim Wal­bert said in a press re­lease. “We look for­ward to lever­ag­ing our ex­ist­ing tech­ni­cal op­er­a­tions ex­per­tise in Ire­land, along with the ex­per­tise of the Eir­Gen em­ploy­ees, to build a ro­bust and ef­fec­tive man­u­fac­tur­ing op­er­a­tion that will en­able us to meet the un­met needs of peo­ple im­pact­ed by rare dis­eases around the world.”

Just a week ago, Hori­zon inked a lic­ns­ing deal with Ar­row­head Phar­ma­ceu­ti­cals’ ARO-XDH, in­ves­ti­ga­tion­al RNAi ther­a­py for un­con­trolled gout. That drug will be used to tar­get pa­tients who fail to re­spond to oral urate low­er­ing ther­a­pies, the com­pa­ny said. The com­pa­ny has made a name for it­self tak­ing or­phan drugs tossed aside and get­ting them ap­proved.

Te­pez­za treats thy­roid eye dis­ease, while Up­liz­na is a treat­ment for De­vic dis­ease, a dis­or­der of the brain and spinal cord caused by in­flam­ma­tion of the op­tic nerve and spinal cord.

Hori­zon has agreed to ac­quire the fa­cil­i­ty for $65 mil­lion in up­front cash pay­ment, and the deal will close in Q3 of 2021. There is ad­di­tion­al land next door for fur­ther de­vel­op­ment and ex­pan­sion, should the com­pa­ny de­cide to do so in the fu­ture. Krys­texxa is an IV treat­ment that low­ers uric acid lev­els and helps pre­vent per­ma­nent joint dam­age.

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.

Tien Lee, Aardvark Therapeutics

Emerg­ing from stealth mode, Aard­vark triples its tar­get num­ber in its Se­ries B

When Aardvark Therapeutics CEO Tien Lee started his work on its lead candidate, appetite suppression was the goal for the small molecule.  Soon after, his team started to see benefits to lowering blood glucose and showing anti-inflammatory activity. On the tail end of that, the company has emerged from stealth mode and announced that has tripled its financing goal in its Series B.

Aardvark has secured $29 million in Series B financing. The money will be used to complete three Phase II trials of its lead compound ARD-101, a small molecule bitter taste receptor pan-agonist.  The funds will also be used to advance additional formulations for the candidate.

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.

Am­gen adds new NC plant to the list as part of $1B man­u­fac­tur­ing ex­pan­sion plans state­side

What can $1 billion buy? If you’re Amgen, it’s good for two manufacturing facilities in the US.

The California-based drug giant will invest close to $550 million in a drug substance plant in Holly Springs, NC, adding itself to an ever-growing list of biotech companies that have decided to call North Carolina home, and marking its second drug manufacturing announcement in a little more than a month.