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.”

Biotech Half­time Re­port: Af­ter a bumpy year, is biotech ready to re­bound?

The biotech sector has come down firmly from the highs of February as negative sentiment takes hold. The sector had a major boost of optimism from the success of the COVID-19 vaccines, making investors keenly aware of the potential of biopharma R&D engines. But from early this year, clinical trial, regulatory and access setbacks have reminded investors of the sector’s inherent risks.

RBC Capital Markets recently surveyed investors to take the temperature of the market, a mix of specialists/generalists and long-only/ long-short investment strategies. Heading into the second half of the year, investors mostly see the sector as undervalued (49%), a large change from the first half of the year when only 20% rated it as undervalued. Around 41% of investors now believe that biotech will underperform the S&P500 in the second half of 2021. Despite that view, 54% plan to maintain their position in the market and 41% still plan to increase their holdings.

David Lockhart, ReCode Therapeutics CEO

Pfiz­er throws its weight be­hind LNP play­er eye­ing mR­NA treat­ments for CF, PCD

David Lockhart did not see the meteoric rise of messenger RNA and lipid nanoparticles coming.

Thanks to the worldwide fight against Covid-19, mRNA — the genetic code that can be engineered to turn the body into a mini protein factory — and LNPs, those tiny bubbles of fat carrying those instructions, have found their way into hundreds of millions of people. Within the biotech world, pioneers like Alnylam and Intellia have demonstrated just how versatile LNPs can be as a delivery vehicle for anything from siRNA to CRISPR/Cas9.

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How to col­lect and sub­mit RWD to win ap­proval for a new drug in­di­ca­tion: FDA spells it out in a long-await­ed guid­ance

Real-world data is messy. There can be differences in the standards used to collect different types of data, differences in terminologies and curation strategies, and even in the way data is exchanged.

While acknowledging this somewhat controlled chaos, the FDA is now explaining how biopharma companies can submit study data derived from real-world data (RWD) sources in applicable regulatory submissions, including new drug indications.

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David Livingston (Credit: Michael Sazel for CeMM)

Renowned Dana-Far­ber sci­en­tist, men­tor and bio­phar­ma ad­vi­sor David Liv­ingston has died

David Livingston, the Dana-Farber/Harvard Med scientist who helped shine a light on some of the key molecular drivers of breast and ovarian cancer, died unexpectedly last Sunday.

One of the senior leaders at Dana-Farber during his nearly half century of work there, Livingston was credited with shedding light on the genes that regulate cell growth, with insights into inherited BRCA1 and BRCA2 mutations that helped lay the scientific foundation for targeted therapies and earlier detection that have transformed the field.

Leen Kawas (L) has resigned as CEO of Athira and will be replaced by COO Mark Litton

Ex­clu­sive: Athi­ra CEO Leen Kawas re­signs af­ter in­ves­ti­ga­tion finds she ma­nip­u­lat­ed da­ta

Leen Kawas, CEO and founder of the Alzheimer’s upstart Athira Pharma, has resigned after an internal investigation found she altered images in her doctoral thesis and four other papers that were foundational to establishing the company.

Mark Litton, the company’s COO since June 2019 and a longtime biotech executive, has been named full-time CEO. Kawas, meanwhile, will no longer have ties to the company except for owning a few hundred thousand shares.

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Ep­i­darex, Sofinno­va dou­ble down on a par­al­lel take on 3rd-gen CAR-T — aim­ing straight at ovar­i­an can­cer

When John Maher treated the first head and neck cancer patient at Guy’s Hospital in London with his pan-ErbB CAR-T back in 2015, he was among a small club of researchers convinced they had an answer to the challenges that had kept those engineered T cells — wildly successful in hematological cancers — either too dangerous or out of reach for patients with solid tumors.

The field has blossomed since then, with a proliferation of technologies that promise to address any number of challenges identified as unique to solid tumors. And Maher himself has rethought his approach and come up with a new CAR-T platform to generate the next slate of candidates.

Sen. Richard Durbin (D-IL, foreground) and Sen. Richard Blumenthal (D-CT) (Patrick Semansky/AP Images)

Sen­a­tors back FDA's plan to re­quire manda­to­ry pre­scriber ed­u­ca­tion for opi­oids

Three Senate Democrats are backing an FDA plan to require mandatory prescriber education for opioids as overdose deaths have risen sharply over the past decade, with almost 97,000 American opioid-related overdose deaths in the past year alone.

While acknowledging a decline in overall opioid analgesic dispensing in recent years, the FDA said it’s reconsidering the need for mandatory prescriber training through a REMS given the current situation with overdoses, and is seeking input on the aspects of the opioid crisis that mandatory training could potentially mitigate.

Suresh Katta, Saama CEO (via YouTube)

As AI con­tin­ues to en­tice Big Phar­ma, a Car­lyle-led drug­mak­er syn­di­cate shells out $430M for cloud com­put­ing play­er

The AI revolution permeating Big Pharma took a big financial step forward Wednesday, with VCs and major drugmakers coming together to acquire a cloud-focused company.

Led by the Carlyle Group, the investors will put up $430 million for a majority stake in Saama, a company that collects patient data to help speed along the drug development process. The investment arms of Pfizer, Merck, Amgen and McKesson all participated in the financing, in addition to other prominent life sciences VCs like Northpond.

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Raju Mohan, Ventyx Biosciences CEO

Ven­tyx sprints to Wall Street less than a year af­ter emerg­ing from stealth

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It took seven months from exiting “quiet mode” for Ventyx Biosciences to land its very own stock ticker, raising $165 million in venture funds along the way.

Now, after pricing a massive $151.5 million IPO, the Encinitas, CA-based biotech is gunning for Phase II.

Ventyx priced close to 9.5 million shares at $16 apiece on Wednesday, the midpoint of its $15 to $17 range. CEO Raju Mohan filed the S-1 papers at the end of September, just over a week after unveiling a $114 million Series B round. He penciled in the standard figure of $100 million at first, likely knowing that in the last year, it’s been common for biotechs to raise much more than those initial estimates.