Af­ter reel­ing back a gene ther­a­py from Sanofi, a North Car­oli­na up­start bags enough mon­ey to do some­thing about it

Af­ter spend­ing some time in Sanofi’s pipeline, a gene ther­a­py for a ge­net­ic eye dis­ease that caus­es blind­ness is now back in the hands of its cre­ators at At­se­na Ther­a­peu­tics. And on Wednes­day, the North Car­oli­na-based start­up land­ed a $55 mil­lion Se­ries A to push it through a Phase I/II tri­al.

At­se­na’s founders, pow­er cou­ple Shan­non and San­ford Boye, did some ear­ly work on the gene ther­a­py at the Uni­ver­si­ty of Flori­da in the ear­ly 2000s, be­fore it was li­censed by Sanofi. The biotech launched this year with $8 mil­lion in fund­ing, then li­censed the can­di­date from Sanofi for an undis­closed amount in Ju­ly.

“It’s kind of like a home­com­ing for them,” At­se­na CEO Patrick Ritschel said of Shan­non and San­ford Boye, who now serve as CSO and chief tech­ni­cal of­fi­cer, re­spec­tive­ly.

Patrick Ritschel

The lead can­di­date is de­signed to treat GUCY2D-as­so­ci­at­ed Leber con­gen­i­tal amau­ro­sis, or LCA1 — a dis­ease that af­fects the reti­na and is a lead­ing cause of blind­ness in chil­dren. The drug us­es an AAV cap­sid to de­liv­er the tar­get GUCY2D gene, the idea be­ing that if you pro­vide a re­place­ment gene, it will en­code the cor­rect form of the pro­tein.

“This is a very good tar­get for gene ther­a­py. It’s mono­genic. It has a well-de­fined sin­gle gene char­ac­ter­is­tic. Im­por­tant­ly, the struc­ture of the reti­na is well-pre­served, so we think that the like­li­hood of suc­cess in gene ther­a­py should al­so be quite high be­cause the cells are there, they’re just not func­tion­al,” Ritschel said.

The Phase I/II is en­rolling 15 par­tic­i­pants to­tal over five co­horts, with com­ple­tion of en­roll­ment ex­pect­ed in late 2021 or ear­ly 2022. Then it’s a 52-week read­out, Ritschel said, with a one-year fol­low-up. The Se­ries A should car­ry the can­di­date to an ef­fi­ca­cy read­out, ac­cord­ing to At­se­na.

In ad­di­tion to sup­port­ing that tri­al, the fi­nanc­ing will al­so be used to build out the team, com­plete man­u­fac­tur­ing de­vel­op­ment for Phase III, and ad­vance the biotech’s pre­clin­i­cal pro­grams to­ward an IND. At­se­na has a pre­clin­i­cal can­di­date for MYO7A-as­so­ci­at­ed Ush­er syn­drome (USH1B) that was al­so de­vel­oped in the Boyes’ labs, and an­oth­er mys­tery can­di­date that it has yet to re­lease de­tails on. And Ritschel is look­ing to add at least an­oth­er 20 staffers by 2021.

Spark Ther­a­peu­tics won ap­proval for their own oph­thalmic gene ther­a­py back in 2017. The drug, Lux­tur­na, treats pa­tients with in­her­it­ed reti­nal dis­ease due to mu­ta­tions in both copies of the RPE65 gene. In some cas­es, a de­fect in that gene re­sults in LCA.

The field gained a Big Phar­ma play­er in Oc­to­ber, when the No­var­tis In­sti­tutes for Bio­Med­ical Re­search bought out Cam­bridge, MA-based start­up Vedere Bio, which Cyrus Moza­yeni built around aca­d­e­m­ic work from Ehud Isacoff and John Flan­nery at Cal-Berke­ley to find a gene ther­a­py ap­proach to pan-geno­typ­ic vi­sion restora­tion in pa­tients with pho­tore­cep­tor-based blind­ness. No­var­tis paid $150 mil­lion, with an­oth­er $130 mil­lion on the ta­ble in mile­stones.

“We’re re­al­ly ex­cit­ed,” Ritschel said. “I think that this is a com­pa­ny that has got a lot of pro­grams, very sol­id sci­ence be­hind it. It’s got great founders, strong sup­port from the Uni­ver­si­ty of Flori­da where a lot of the tech­nol­o­gy orig­i­nat­ed from. And I think we’re look­ing for­ward to re­al­ly build­ing things out.”

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Can strug­gling Iterum turn the cor­ner to an an­tibi­ot­ic suc­cess sto­ry? They will know in six months

More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

The decision, six months from now, will mark a make-or-break moment for a struggling biotech that has just enough cash to keep the lights on until the third quarter.

Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

IPO track­er: 2021 gets start­ed with a flur­ry of new of­fer­ings

A global pandemic couldn’t slow down what turned out to be a record year for biotech IPOs. With the calendar turning toward 2021, the Endpoints News team is prepped to track each new filing this year, and the outcome. We’re off to another hot start at least.

Below, you’ll find the companies that have filed to go public, in addition to those that have already priced. Through the first two business weeks of January, there have already been 9 biotechs that have filed or priced, and the number is only expected to grow. We’ll keep the tracker updated as it does.

Matt Gline (L) and Vivek Ramaswamy

Scoop: Vivek Ra­maswamy is hand­ing the CEO job to a top lieu­tenant at Roivant — but he’s not ex­act­ly leav­ing the biotech scene

Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

He launched his first biotech with an Alzheimer’s drug he picked up cheap, and watched the experiment implode in one of the highest profile pivotal disasters seen in the last decade. But it didn’t slow the 30-something exec down; if anything, he hit the accelerator. Ramaswamy blazed global paths and went on to raise billions to spur the creation of a large lineup of little Vants promising big things at a fast pace. He sold off a section of the Vant brigade to Sumitomo Dainippon for $3 billion. And more recently the relentless dealmaker has been building a computational discovery arm to add an AI-driven approach to kicking up new programs and companies, supplementing the in-licensing drive while pursuing advances that have created more than 700 jobs at Roivant, with $2 billion in reserves.

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Ron Cooper, Albireo CEO

Al­bireo just ad­vanced down to the 10-yard line at the FDA. And Ron Coop­er’s team is get­ting prepped for the next big play

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

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David Southwell (L) and Christoph Westphal

Har­vard spin­out kicks off 2021 with a crossover round and sights set on the clin­ic

Several months after striking an alliance with Novartis, TCR therapy-focused TScan Therapeutics has reeled in a crossover round that should hold it over for the next two years as it eyes a public debut.

The Christoph Westphal portfolio company had been arranging the crossover for the last few months, CEO David Southwell said. Just before Christmas, they nailed down what he called a “really blue-chip” syndicate of four new investors, including BlackRock, RA Capital Management and two undisclosed funds. They closed on the $100 million Series C just over a week ago, and waited until Monday morning to announce it.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

Covid-19 roundup: Mod­er­na dou­bles down on Covid-19 with new boost­er tri­als; Aus­tralia plans do­mes­tic pro­duc­tion of As­traZeneca vac­cine amid dis­tri­b­u­tion lag

As Merck bows out of the global race to develop vaccines for Covid-19, Moderna is doubling down to make sure they can quell new variants that have recently emerged and quickly spread.

The Cambridge, MA-based biotech put out word on Monday that in vivo studies indicate their mRNA vaccine works well enough against two strains first detected in the UK and South Africa. But with a six-fold reduction in neutralizing titers observed against the latter strain, the company is launching a new study of a booster version to make sure it can do the job.

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