Se­quenc­ing up­start rais­es $273M ahead of IPO; Ac­er­a­gen ac­quires high­ly-an­tic­i­pat­ed Far­ber dis­ease treat­ment

Uni­ver­si­ty of Ox­ford spin­out Ox­ford Nanopore has raised a mon­ster round for its “scal­able” DNA and RNA se­quenc­ing tech ahead of a re­port­ed IPO fil­ing in Lon­don.

Once caught up in the Wood­ford de­ba­cle, the se­quenc­ing group has raised £195 mil­lion — $273 mil­lion — with the help of a ros­ter of new in­vestors. Temasek, Welling­ton Man­age­ment, M&G In­vest­ments and Nikon pitched in £125 mil­lion of that while ex­ist­ing in­vestors ponied up the rest.

Reuters re­port­ed re­cent­ly that the se­quenc­ing out­fit has signed up bankers for a sprint to the Lon­don ex­change in the sec­ond half of the year.

IP Group — a backer which it­self com­mit­ted £18.7 mil­lion to the round — re­ports that the com­pa­ny now has a val­u­a­tion of about $3.4 bil­lion, with its stake worth £359 mil­lion. — John Car­roll

Ac­er­a­gen ac­quires high­ly-an­tic­i­pat­ed Far­ber dis­ease treat­ment

Ac­er­a­gen is ac­quir­ing a com­pa­ny’s lead en­zyme re­place­ment ther­a­py can­di­date for Far­ber dis­ease, and en­ter­ing a $35 mil­lion part­ner­ship to fund the de­vel­op­ment pro­gram in­to a po­ten­tial reg­is­tra­tional study.

The com­pa­ny an­nounced the ac­qui­si­tion of En­zy­vant’s en­zyme re­place­ment ther­a­py RVT-801 for re­com­bi­nant hu­man acid ce­rami­dase de­fi­cien­cy that presents as Far­ber dis­ease. Ac­er­a­gen will al­so en­ter a fi­nanc­ing agree­ment with No­vaQue­st Cap­i­tal Man­age­ment to fund the drug’s de­vel­op­ment.

En­zy­vant will re­ceive up to $226 mil­lion, as well as tiered roy­al­ties on net sales, the com­pa­ny said in a re­lease Mon­day. In 2019, the agent was grant­ed rare pe­di­atric dis­ease and fast track des­ig­na­tions from the FDA.

There is no known cure for Far­ber’s dis­ease, which builds up harm­ful lev­els of lipids in the joints, tis­sues and cen­tral ner­vous sys­tem. It is caused by mu­ta­tions in the ASAH1 gene and caus­es a de­fi­cien­cy of acid ce­rami­dase, a nat­ur­al lyso­so­mal en­zyme. Pre­clin­i­cal de­vel­op­ment has been com­plet­ed, in­clud­ing in vi­vo proof of con­cept in a mouse mod­el of Far­ber’s dis­ease.

With the ac­qui­si­tion comes sev­er­al com­plet­ed tox­i­col­o­gy stud­ies, a quan­ti­ta­tive pa­tient re­search study and a nat­ur­al his­to­ry study in pa­tients with Far­ber’s dis­ease that high­lights the pro­gres­sion and im­pact of the con­di­tion, CEO John Tay­lor said in a state­ment.

“The Ac­er­a­gen team is de­light­ed to an­nounce the strate­gic trans­ac­tion with En­zy­vant and the re­la­tion­ship we have es­tab­lished with No­vaQue­st to con­tin­ue the de­vel­op­ment of ACG-801. This pro­gram is based on the foun­da­tion­al work of Dr. Ed Schuch­man at the Ic­ahn School of Med­i­cine at Mount Sinai, es­tab­lish­ing the po­ten­tial to ad­dress the un­der­ly­ing pathol­o­gy of Far­ber dis­ease, a ge­net­ic de­fi­cien­cy of acid ce­rami­dase,” Tay­lor said.

— Josh Sul­li­van

How Pa­tients with Epilep­sy Ben­e­fit from Re­al-World Da­ta

Amanda Shields, Principal Data Scientist, Scientific Data Steward

Keith Wenzel, Senior Business Operations Director

Andy Wilson, Scientific Lead

Real-world data (RWD) has the potential to transform the drug development industry’s efforts to predict and treat seizures for patients with epilepsy. Anticipating or controlling an impending seizure can significantly increase quality of life for patients with epilepsy. However, because RWD is secondary data originally collected for other purposes, the challenge is selecting, harmonizing, and analyzing the data from multiple sources in a way that helps support patients.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

UP­DAT­ED: Gink­go Bioworks re­sizes the de­f­i­n­i­tion of go­ing big in biotech, rais­ing $2.5B in a record SPAC deal that weighs in with a whop­ping $15B-plus val­u­a­tion

Ginkgo Bioworks execs always thought big. But today should redefine just how big an upstart biotech player can dream.

In the largest SPAC deal to clear the hurdles to Nasdaq, the biotech that envisioned everything from remaking synthetic meat to a whole new approach to developing drugs has joined forces with one of the biggest disruptors in biotech to slam the Richter scale on dealmaking.

Soon after becoming the darling of the VC crew and clearing the bar on a $4 billion valuation, Ginkgo — a synthetic biotech player out to reprogram cells with industrial efficiency — has now struck a deal to go public in the latest leviathan SPAC that sets its pre-money valuation at $15 billion. In one swift vault, Ginkgo will combine with Harry Sloan’s Soaring Eagle Acquisition Corp. and leap into the public markets.

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FDA un­veils six ICH guide­lines ahead of meet­ing with Health Cana­da

A sign that the FDA’s non-Covid-related processes are beginning to normalize: The release of six guidelines from the International Council of Harmonisation.

Years in development, the ICH documents offer an international perspective on drug development, with these latest guidelines covering everything from recommendations to support the classification of drug substances, featured in the M9 guidance, to standards for nonclinical safety studies for pediatric medicines in the S11 guideline.

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Sanofi, Glax­o­SmithK­line, Boehringer ac­cused of play­ing games, de­stroy­ing emails re­lat­ed to law­suit over con­t­a­m­i­nat­ed Zan­tac

A recent court filing raises new questions about how major pharma companies like Sanofi, GlaxoSmithKline, and Boehringer Ingelheim have dealt with a lawsuit related to recalls of certain over-the-counter heartburn drugs due to the presence of a potentially cancer-causing substance found in them.

More than 70,000 people who took Sanofi’s Zantac and other heartburn drugs containing ranitidine, which have been recalled over the past two years, have sued the manufacturers, including generic drugmakers, and other retailers and distributors as part of a consolidated suit before US District Court Judge Robin Rosenberg in Florida.

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Sanofi, Cel­lec­tis team up to pair Lem­tra­da with al­lo­gene­ic CAR-Ts; Bris­tol My­ers joins Evotec's dri­ve to ad­vance UK sci­ence

Sanofi is partnering up with Cellectis on their allogeneic CAR-Ts.

Cellectis struck a deal to use the French pharma’s drug alemtuzumab (Lemtrada, an MS agent) as part of its lymphodepletion regimen — used to sideline host immune cells and improve CAR-T cell expansion and persistence.

The Sanofi drug is already used in some clinical trials, and now the pharma giant is on the hook for clinical studies as they engage in negotiations over a commercial pact. — John Carroll

Chris Garabedian (Xontogeny)

Per­cep­tive Ad­vi­sors, Xon­toge­ny bring the band back and then some with a $515M sec­ond fund sniff­ing out lead com­pounds

When Perceptive Advisors and startup accelerator Xontogeny initially teamed up on an early-stage VC round in 2019, the partners hoped to prove their investments could be a force multiplier for early-stage companies. Now, with that proof of concept behind them, the pair have closed a second VC round worth more than double the money.

Dubbed PXV Fund II and headed by Xontogeny CEO and former Sarepta head Chris Garabedian, the $515 million fund will target 10 to 12 early-stage preclinical companies with Series A rounds in the $20 million to $40 million range with opportunities for Series B follow-ups. The oversubscribed fund is bringing the band back with initial investors from PXVI as well as new investors that include “top-tier” asset managers, endowments, foundations, family offices, and individual investors.

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A clos­er look at the FDA’s more than 700 pan­dem­ic-re­lat­ed record re­quests to re­place on­site in­spec­tions

As the pandemic constrained the FDA’s ability to travel for onsite manufacturing inspections, the agency increasingly turned to requesting records to fill the gap, even for hundreds of US-based facilities.

FDA explains in its guidance on manufacturing inspections during the pandemic that the agency can request records (not to be confused with the FDA’s remote interactive evaluations) directly from facilities “in advance of or in lieu of” certain onsite inspections. Companies are legally required to fulfill those requests because a denial may be considered limiting an inspection, which could lead to the FDA deeming a drug made at that site to be adulterated.

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Stephen Squinto, Gennao Bio CEO (Gennao)

Alex­ion co-founder Stephen Squin­to is back in the game as CEO, this time for a small gene ther­a­py play­er

With his name already behind a rare disease success story in Alexion, Stephen Squinto was looking for a great story to drive him to jump back into the biotech game. He found that in a fledging non-viral gene therapy company, and now he’s got a few backers on board as well.

On Tuesday, Gennao Bio launched with a $40 million Series A co-led by OrbiMed and Logos Capital with participation by Surveyor Capital. The biotech, which is looking to use its cell-penetrating antibody platform to deliver nucleic acid “payloads” during into the nucleus, had to rush for its initial series — and had a name change along the way.

Alvotech takes Ab­b­Vie to court over al­leged patent 'mine­field' sur­round­ing megablock­buster Hu­mi­ra

AbbVie has so far been successful in shooing away competition to its megablockbuster Humira, deploying a number of patents and settlements to keep biosimilars off the US market until 2023. But one Icelandic drugmaker doesn’t want to wait — and on Tuesday, it filed a lawsuit challenging what it called a patent “minefield.”

Alvotech has accused AbbVie of trying to “overwhelm” and “intimidate” it with “an outrageous number of patents of dubious validity,” according to court documents. The company is currently seeking approval for its Humira copycat AVT02, which AbbVie says would infringe upon 62 patents.