Jim Wilson (WuXi Global Forum at JPM20)

Roiled by se­nior-lev­el ex­its, Jim Wil­son’s Pas­sage Bio re­trench­es, chop­ping back on staff as it ex­tends cash run­way

A lit­tle more than 3 years af­ter Penn’s Jim Wil­son and the late Tachi Ya­ma­da her­ald­ed the launch of their gene ther­a­py up­start Pas­sage Bio with a bevy of mar­quee VCs div­ing in, the biotech is hun­ker­ing down for a re­or­ga­ni­za­tion. And it comes with the ex­it of its R&D chief just days af­ter one of the in­vestors on its board quit af­ter reg­is­ter­ing a sharp dis­agree­ment over their plans.

Pas­sage put out word to­day that it’s chop­ping 13% of its work­force and cir­cling the wag­ons around 3 key pro­grams — for GM1 gan­gliosi­do­sis, Krabbe dis­ease and fron­totem­po­ral de­men­tia — which will give it enough cash on hand to get to Q2 2024. The state­ment didn’t say just how many staffers are be­ing cut, but Pas­sage — close­ly aligned with Wil­son’s Gene Ther­a­py Pro­gram at Penn, which has pro­vid­ed rights to a port­fo­lio of prod­ucts — not­ed in its last 10-K that it had 133 full time em­ploy­ees, in ad­di­tion to in­de­pen­dent con­trac­tors.

Eliseo Sali­nas

That ex­tend­ed cash run­way is re­quired now as Pas­sage’s stock price has been scut­tled by the gen­er­al biotech rout trig­gered by a gen­er­al­ist ex­it out of risky plays like drug de­vel­op­ment — a dar­ling of the mar­ket dur­ing the pan­dem­ic. Pas­sage’s stock price has fall­en 86% in the past 12 months, leav­ing it with a mar­ket cap of $152 mil­lion, not enough to in­spire much con­fi­dence.

The re­or­ga­ni­za­tion comes with the de­par­ture of R&D chief Eliseo Sali­nas on March 18. Sali­nas has an ex­ten­sive back­ground in biotech, with stints stretch­ing back to CSO work at Shire and a se­nior post at Wyeth, ac­cord­ing to his LinkedIn page.

Al­so gone is board mem­ber Liam Rat­cliffe, a ven­ture part­ner at Pas­sage backer New Leaf Ven­ture. And Rat­cliffe made it clear that he was not hap­py with the biotech’s cur­rent di­rec­tion. In an SEC fil­ing, Pas­sage not­ed:

On March 7, 2022, Liam Rat­cliffe, MD, Ph.D., no­ti­fied Pas­sage Bio, Inc. (the “Com­pa­ny”) of his de­ci­sion to re­sign from the Com­pa­ny’s Board of Di­rec­tors (the “Board”), in­clud­ing from the Nom­i­nat­ing and Gov­er­nance Com­mit­tee of the Board, ef­fec­tive im­me­di­ate­ly. Dr. Rat­cliffe’s de­ci­sion to re­sign was due to a dis­agree­ment with the Com­pa­ny re­gard­ing its busi­ness plans and op­er­a­tions.

That didn’t stop the re­struc­tur­ing.

Bruce Gold­smith

“We have made the strate­gic de­ci­sion to fo­cus our or­ga­ni­za­tion­al pri­or­i­ties, re­duce our op­er­at­ing ex­pens­es and in­vest in the high­est val­ue de­vel­op­ment ac­tiv­i­ties,” not­ed CEO Bruce Gold­smith in a pre­pared state­ment.

Back in 2019, the emer­gence of Pas­sage Bio was part of the over­all re­nais­sance of gene ther­a­py R&D, com­ing in the wake of the first US ap­proval and at a time dozens of dis­eases were be­ing tar­get­ed with once-and-done can­di­dates. But the re­nais­sance, fol­low­ing a slow re­cu­per­a­tion in the wake of Jesse Gelsinger’s death in a tri­al in 1999, quick­ly ran in­to a whole new set of safe­ty is­sues, in­clud­ing the deaths of sev­er­al pa­tients in clin­i­cal tri­als.

Those re­newed hopes led to a $216 mil­lion IPO for Pas­sage 2 years ago. And they brought in Or­biMed Ad­vi­sors, Fra­zier Health­care Part­ners, Ver­sant Ven­tures, New Leaf Ven­ture Part­ners, Vi­vo Cap­i­tal and Lil­ly Asia Ven­tures as foun­da­tion in­vestors.

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

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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.

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.

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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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.

Peter Hecht, Cyclerion CEO

Cy­cle­ri­on board quick­ly nix­es CEO Pe­ter Hecht's un­ortho­dox pitch for low cash re­serves

It’s been less than two months since Cyclerion laid out a new R&D strategy around its lead drug in mitochondrial diseases, one that triggered the company to lay off close to half of its employees and explore licensing deals for the rest of the pipeline. But CEO Peter Hecht apparently has other plans in mind.

Hecht, who led Ironwood for close to 20 years before spinning out Cyclerion, disclosed in an SEC filing Monday that a “newly-formed private company” that he “may have or may acquire an interest” submitted a proposal to Cyclerion the day prior to purchase Cyclerion’s CNS assets, including CY6463 and CY3018 — the top two programs listed in the pipeline.

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Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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