Gilmore O'Neill, incoming Editas Medicine CEO

With Ed­i­tas' third CEO in as many years, ex­pect a new CMO and new de­vel­op­ment part­ners

Sarep­ta Ther­a­peu­tics CMO Gilmore O’Neill will take over as CEO and pres­i­dent of CRISPR biotech Ed­i­tas Med­i­cine on June 1.

But al­ready in the hours since be­ing an­nounced, O’Neill has been hear­ing from his net­work on who could po­ten­tial­ly be the new CMO at the gene edit­ing com­pa­ny he’s about to helm. Lisa Michaels was fired from the med­ical chief post in ear­ly Feb­ru­ary, with­out pub­lic ex­pla­na­tion, as part of a re­build­ing of the C-suite.

Hir­ing a new CMO will be one of O’Neill’s top pri­or­i­ties when he takes over the C-suite posts from ex­ec­u­tive chair Jim Mullen, who was in the post for the past 14 months, the two said in a joint in­ter­view with End­points News. The pair know each oth­er well and will have few kinks to work out in the tran­si­tion pe­ri­od be­cause of their pri­or time to­geth­er at Bio­gen in the aughts, where Mullen was CEO and O’Neill held var­i­ous VP and SVP posts.

“The com­pa­ny hasn’t been as crisp on ex­e­cu­tion as it just needs to be, should be, has to be,” Mullen said of the lead­er­ship tran­si­tion. Ed­i­tas’ shares $ED­IT were down about 5.5% Thurs­day morn­ing. As ex­ec­u­tive chair, he’ll spend more time on “in­vestor de­vel­op­ment, think­ing about some pol­i­cy de­vel­op­ment, look­ing over the hori­zon at the days when we may start to com­mer­cial­ize prod­ucts.”

Jim Mullen

Busi­ness de­vel­op­ment and cap­i­tal rais­ing will be ad­di­tion­al fo­cus­es on “day one,” O’Neill said. His pre­de­ces­sor said Ed­i­tas has “not been as ac­tive or as pro­duc­tive on the busi­ness de­vel­op­ment front” as it could have been. The biotech will look to shore up ex­per­tise via part­ner­ing on its en­gi­neered NK cell pro­grams be­cause of the space’s “uber com­pet­i­tive” and ex­pen­sive na­ture, Mullen said. In­bound in­ter­est is al­so flow­ing in from drug de­vel­op­ers look­ing to pair up their ther­a­peu­tics with Ed­i­tas’ edit­ing ma­chin­ery and tech, he added.

O’Neill was al­so look­ing for a new part­ner over the past few months. His job search was laser-fo­cused on CEO posts.

“Ed­i­tas jumped off the page at me just as an enor­mous­ly ex­cit­ing op­por­tu­ni­ty. Why? The tech­nol­o­gy. It has un­lim­it­ed, re­al­ly, po­ten­tial and then the com­pa­ny it­self there [is] dif­fer­en­ti­at­ed core ex­per­tise in the com­pa­ny around nu­cle­as­es en­hance­ments, around guide RNA chem­istry and de­sign, as well as ex­pe­ri­ences with dif­fer­ent de­liv­ery method­olo­gies,” O’Neill said.

He in­her­its a nine-year-old biotech found­ed with the po­ten­tial to be a pi­o­neer in the CRISPR field. The com­pa­ny was born out of the sci­ence from CRISPR and gene edit­ing lead­ers Feng Zhang, George Church, J. Kei­th Joung and David Liu.

But the com­pa­ny has had on­ly a few clin­i­cal tri­al da­ta re­leas­es since en­ter­ing hu­man stud­ies. The com­pa­ny pre­sent­ed some ini­tial da­ta on ED­IT-101 for Leber con­gen­i­tal amau­ro­sis, a rare ge­net­ic vi­sion-loss dis­or­der, in Oc­to­ber 2021. And this week, the biotech said it be­came the first to test a CRISPR gene edit­ing med in vi­vo in a pe­di­atric pa­tient with ED­IT-101. Ed­i­tas be­gan test­ing ED­IT-101 in Ju­ly 2019 with part­ner Al­ler­gan, but Ab­b­Vie ditched the deal in Au­gust 2020.

Al­so this year, Ed­i­tas will be­gin test­ing its sec­ond pro­gram, ED­IT-301, in pa­tients with sick­le cell dis­ease and in an­oth­er study in pa­tients with trans­fu­sion-de­pen­dent be­ta tha­lassemia.

O’Neill has been CMO and EVP of R&D at Sarep­ta since June 2018, where he’s con­tributed to the Cam­bridge, MA biotech’s gene ther­a­pies for Duchenne mus­cu­lar dy­s­tro­phy, Amondys and Vyondys. Ed­i­tas al­so tout­ed O’Neill’s work on Bio­gen’s spinal mus­cu­lar at­ro­phy drug Spin­raza and MS ther­a­pies Ple­gridy and Tec­fidera.

He’s the third new CEO since 2019. Mullen took over in Feb­ru­ary 2021, when Cyn­thia Collins de­part­ed fol­low­ing a two-year stint af­ter Ka­trine Bosley’s ex­it in Jan­u­ary 2019. Mul­ti­ple oth­er sci­ence and med­ical chiefs have packed up their bags in re­cent years, too.

“The first thing to start with on long-term vi­a­bil­i­ty is to have some­body that’s younger, which prob­a­bly doesn’t nec­es­sar­i­ly in­clude me,” Mullen jok­ing­ly said.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

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