Gilmore O’Neill, Editas Medicine CEO

Ed­i­tas halts lead CRISPR pro­gram af­ter ef­fi­ca­cy da­ta un­der­whelm

Ed­i­tas Med­i­cine will put a wrap on its lead clin­i­cal pro­gram af­ter re­port­ing dis­ap­point­ing re­sults from a Phase I/II tri­al.

The biotech, one of the first to try to use CRISPR gene edit­ing tech to de­vel­op new drugs, said Thurs­day morn­ing that on­ly three of 14 pa­tients re­spond­ed to the ther­a­py, known as ED­IT-101. Ex­ecs will try to find a part­ner for the pro­gram, which is de­signed to treat an in­her­it­ed form of blind­ness called CEP290-me­di­at­ed LCA10.

In­vestors ap­peared dis­ap­point­ed with the news, as Ed­i­tas shares $ED­IT fell about 18% in ear­ly Thurs­day trad­ing.

Re­searchers were test­ing ED­IT-101 in both adults and chil­dren at a few dif­fer­ent dosage lev­els. Da­ta from Thurs­day’s up­date came from 14 pa­tients — 12 adults and two kids — 11 of whom re­ceived the ex­per­i­men­tal drug at least six months out. Ad­di­tion­al­ly, Ed­i­tas had at least a year of fol­low-up da­ta for sev­en of the 14.

In an­a­lyz­ing the three re­spon­ders’ re­sults, the biotech found that two pos­sessed the ho­mozy­gous ver­sion of the dis­ease, or two copies of an af­fect­ed gene. These were the on­ly two ho­mozy­gous pa­tients in the study. On­ly one of the oth­er 12 pa­tients, all het­erozy­gous (one gene) in­di­vid­u­als, achieved a “clin­i­cal­ly mean­ing­ful” re­sponse.

Giv­en that ho­mozy­gous LCA10 is much rar­er than its het­erozy­gous coun­ter­part, and it’s the on­ly pop­u­la­tion where a pos­si­ble ef­fect could prove con­clu­sive, Ed­i­tas said a fi­nal prod­uct would not be com­mer­cial­ly vi­able. On­ly about 300 peo­ple in the US suf­fer from ho­mozy­gous dis­ease, so Ed­i­tas de­cid­ed to halt de­vel­op­ment and seek a part­ner.

An­a­lysts pep­pered Ed­i­tas’ ex­ecs on an in­vestor call Thurs­day morn­ing, try­ing to pick out rea­sons if ED­IT-101 might be sal­vage­able and who the biotech might seek for a part­ner, among oth­er top­ics. New CEO Gilmore O’Neill large­ly gave few specifics when it came to busi­ness strat­e­gy, but con­tin­ued to tout Thurs­day’s da­ta as proof the idea be­hind the drug is sound.

One an­a­lyst asked O’Neill whether the da­ta would move Ed­i­tas to­ward con­sid­er­ing lay­offs or a re­verse merg­er, which O’Neill side­stepped while al­so throw­ing cold wa­ter in tout­ing an­oth­er clin­i­cal pro­gram, ED­IT-301. For now, the part­ner­ship path is the on­ly con­crete step Ed­i­tas is will­ing to talk about.

Baisong Mei

The da­ta drop comes af­ter a rocky few years for Ed­i­tas, whose CRISPR ther­a­py was one of the first to be test­ed di­rect­ly in peo­ple. O’Neill is the com­pa­ny’s third chief ex­ec­u­tive in as many years af­ter jump­ing from Sarep­ta in June. Chief med­ical of­fi­cer Baisong Mei al­so joined this year af­ter for­mer CMO Lisa Michaels was abrupt­ly fired in Feb­ru­ary.

Ear­li­er da­ta snap­shots had in­di­cat­ed Ed­i­tas would like­ly need stronger ef­fi­ca­cy re­sults, de­spite the in vi­vo ther­a­py prov­ing safe. In Sep­tem­ber 2021, two of five pa­tients showed any sign of their vi­sion im­prov­ing, both of whom were treat­ed with ED­IT-101’s mid­dle dose.

Some of the com­pa­ny’s pre­vi­ous part­ner­ships al­so did not work out, as Ab­b­Vie scrapped an R&D al­liance — orig­i­nal­ly signed by Al­ler­gan — back in Au­gust 2020. Al­ler­gan paid the biotech $90 mil­lion in 2017 to team up.

Thurs­day’s da­ta al­so boost com­peti­tors In­tel­lia and CRISPR Ther­a­peu­tics, SVB Se­cu­ri­ties an­a­lysts wrote in a note, not­ing Ed­i­tas thus far “has yet to ex­e­cute with the speed nec­es­sary to close the gap” with the oth­er biotechs.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; and more

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As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Isao Teshirogi, Shionogi president and CEO (Kyodo via AP Images)

Sh­ionogi's Covid an­tivi­ral lands first ap­proval in Japan's new emer­gency ap­proval path­way

Japanese regulators on Tuesday signed off on Shionogi’s homegrown antiviral for Covid-19, known as Xocova (ensitrelvir), making it the first approval under Japan’s emergency regulatory approval system.

The emergency approval, following a back-and-forth with regulators since last February, is based on a safety profile with more than 2,000 patients who have accessed the pill, and clinical symptomatic efficacy for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in patients with mild to moderate SARS-CoV-2 infection, regardless of risk factors or vaccination status, and during the Omicron-dominant phase of the pandemic.

Alzheimer’s drug bites the dust; Re­struc­ture, re­struc­ture, re­struc­ture; Land­mark di­a­betes OK; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Being in the news business can give one a warped sense of time — it feels like quite a while since we published some of these stories below. But next Saturday’s Endpoints Weekly will definitely be shorter, as we take off Thursday and Friday for Thanksgiving. We will still have the abbreviated edition in your inbox at the usual time.

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Karen Aiach, Lysogene CEO (RE(ACT) Discovery Institute)

Gene ther­a­py flunks PhII/III study, but for­mer Sarep­ta part­ner sees a path for­ward — if it can find the cash

The development path for Lysogene’s gene therapy for MPS IIIA has been a rocky one. After the FDA slapped a partial clinical hold on a Phase II/III study, a patient already dosed in the trial died, although it was deemed unrelated to treatment. Then earlier this year, Sarepta pulled out of their three-year partnership due to disagreements on who will handle commercial supply.

And now, Lysogene reported the trial has failed its primary endpoint.

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