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.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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Sulagna Bhattacharya, Nanoscope Therapeutics CEO

Nanoscope’s eye dis­ease gene ther­a­py shows mixed re­sults in PhII

Dallas-based biotech Nanoscope Therapeutics unveiled Phase II results on its gene therapy for a rare eye disease Thursday morning.

In the RESTORE trial, 18 patients with retinitis pigmentosa got a gene therapy called MCO-010 while nine got placebo. On a vision test called the MLYMT, the treatment group had a one-point greater change over one year in their score compared to the placebo group, the primary endpoint of the study. However, the 95% confidence interval was 0.0 to 3.0, meaning the result was not statistically significant. The p-value was not provided.

Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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No longer ‘dead or just hi­ber­nat­ing,’ drug­mak­ers re­turn to heart med­i­cines

In 2015, now-FDA Commissioner Robert Califf joined industry, academic and regulatory representatives in Washington to discuss why more drugs weren’t in development for cardiovascular diseases, the leading US cause of death and once a mainstay of pharmaceutical industry blockbusters.

The group pointed to many reasons. Clinical trials could take years and testing was expensive. Wide availability of generic drugs made the commercial prospects uncertain. Their paper title summed up the mood: “Cardiovascular Drug Development: Is it Dead or Just Hibernating?”

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Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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