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.”

Janet Woodcock (Greg Nash/Pool via AP Images)

'I re­al­ly don’t look back': Janet Wood­cock on her tran­si­tion away from drugs

Janet Woodcock may have one of the most historically long and drug-intense tenures in FDA history, but her new role is outside of all things pharma and the once-acting FDA commissioner isn’t looking back.

“No I really don’t look back,” Woodcock told Endpoints News via email on Monday morning. “Yes I will be transitioning. Longer discussion on infrastructure needed.”

Co­pay coupons gone wrong, again: Pfiz­er pays al­most $300K to set­tle com­plaints in four states

Pfizer has agreed to pay $290,000 to settle allegations of questionable copay coupon practices in Arizona, Colorado, Kansas, and Vermont from 2014 to 2018.

While the company has not admitted any wrongdoing as part of the settlement, Pfizer has agreed to issue restitution checks to about 5,000 consumers.

A Pfizer spokesperson said the company has “enhanced its co-pay coupons to alleviate the concerns raised by states and agreed to a $30,000 payment to each.”

Delaware court rules against Gilead and Astel­las in years-long patent case

A judge in Delaware has ruled against Astellas Pharma and Gilead in a long-running patent case over Pfizer-onwed Hospira’s generic version of Lexiscan.

The case kicked off in 2018, after Hospira submitted an Abbreviated New Drug Application (ANDA) for approval to market a generic version of Gilead’s Lexiscan. The drug is used in myocardial perfusion imaging (MPI), a type of nuclear stress test.

Taye Diggs (courtesy Idorsia)

Idor­sia inks an­oth­er celebri­ty en­dors­er deal with ac­tor and dad Taye Dig­gs as Qu­viviq in­som­nia am­bas­sador

Idorsia’s latest Quviviq insomnia campaign details the relatable dad story of a well-known celebrity — actor and Broadway star Taye Diggs.

Diggs stopped sleeping well after the birth of his son, now more than 10 years ago. Switching mom-and-dad nightly shifts to take care of a baby interrupted his sleep patterns and led to insomnia.

“When you’re lucky enough to be living out your dream and doing what you want, but because of something as simple as a lack of sleep, you’re unable to do that, it felt absolutely — it was treacherous,” he says in an interview-style video on the Quviviq website.

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Belén Garijo, Merck KGaA CEO (Kevin Wolf/AP Images for EMD Serono)

Mer­ck KGaA pumps €440M in­to ex­pand­ing and con­struct­ing Irish man­u­fac­tur­ing fa­cil­i­ties

The area of Ireland famous for Blarney Castle and its cliffsides along the Atlantic Ocean is seeing Merck KGaA expand its commitment there.

The German drug manufacturer is expanding its membrane and filtration manufacturing capabilities in Ireland. The company will invest approximately €440 million ($470 million) to increase membrane manufacturing capacity in Carrigtwohill, Ireland, and build a new manufacturing facility at Blarney Business Park, in County Cork, Ireland.

Rep. Katie Porter (D-CA) (Michael Brochstein/Sipa USA/Sipa via AP Images)

House Dems to Sen­ate lead­er­ship: Quick­ly move a rec­on­cil­i­a­tion bill with drug price ne­go­ti­a­tion re­forms

Twenty House Democrats, including Reps. Katie Porter of California and Susan Wild of Pennsylvania, are calling on Senate leaders to move quickly with a reconciliation bill (meaning they only need a simple majority for passage) with prescription drug pricing reforms, and to include adding new authority for Medicare to negotiate drug prices.

They also called on the Senate to specifically follow suit with the House passage of a $35 per month insulin cap (as Senate Majority Leader Chuck Schumer’s deadline for a vote on that provision has come and gone), and to cap Medicare Part D costs at $2,000 per year for seniors.

An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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Phillip Gomez, SIGA CEO

UP­DAT­ED: On the back of SIGA Tech­nolo­gies' win with the FDA, the mon­key­pox virus sees the com­pa­ny spring­ing to fur­ther ac­tion

As the cases of monkeypox now sit at well over 100 worldwide and have spread to multiple continents, the orders for any type of vaccine against monkeypox are seeing nations and medical bodies looking to get their hands on anything and everything. And now SIGA Technologies seems to be getting in on the action.

According to Euronews, SIGA Technologies, a pharmaceutical company that is focused on providing medical countermeasures to biological and chemical attacks, is now in talks with several European authorities looking to stockpile its antiviral that can counter monkeypox. The drug known as tecovirimat or Tpoxx was approved by the FDA in 2018 as a vaccine for smallpox but was approved by the European Medicines Agency to also act against monkeypox, cowpox and complications from immunization with vaccinia.

Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

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