Plot­ting clin­i­cal en­try, James Wilson's gene ther­a­py start­up brings in bil­lion­aire for $110M Se­ries B

James Wil­son

The gene ther­a­py biotech that James Wil­son helped found to take some rare dis­ease pro­grams out of Penn all the way to an ap­proval has scored $110 mil­lion in its lat­est fi­nanc­ing.

Pas­sage Bio is get­ting a boost just sev­en months af­ter launch­ing with an­oth­er megaround. Ac­cess Biotech­nol­o­gy — the ther­a­peu­tic-fo­cused in­vest­ment arm of bil­lion­aire Len Blavat­nik’s con­glom­er­ate — is lead­ing the Se­ries B, which al­so in­clud­ed all the mar­quee in­vestors who pro­vid­ed the ini­tial $115 mil­lion: Or­biMed, Fra­zier Health­care Part­ners, Ver­sant Ven­tures, Lily Asia Ven­tures, New Leaf Ven­ture Part­ners and Vi­vo Cap­i­tal. New back­ers in­clude Box­er Cap­i­tal of Tavi­s­tock Group, High­line Cap­i­tal Man­age­ment, Lo­gos Cap­i­tal and Sphera Funds Man­age­ment.

Stephen Squin­to

The com­pa­ny is on track to ini­ti­ate clin­i­cal tri­als for its two lead pro­grams in the first half of 2020 as planned. True to its stat­ed mis­sion of tack­ling mono­genic CNS dis­ease, the ther­a­pies would treat GM1 gan­gliosi­do­sis, a neu­ron-de­stroy­ing dis­or­der most com­mon in in­fants, and fron­totem­po­ral de­men­tia, which over­whelm­ing­ly af­fects the el­der­ly, re­spec­tive­ly.

A third pro­gram in Krabbe dis­ease — in­tend­ed to fix a ge­net­ic de­fect caus­ing a short­age of galac­to­syl­ce­rami­dase, an im­por­tant en­zyme for mak­ing myelin — is ex­pect­ed to fol­low lat­er in the year.

Keep­ing the stream of pro­grams flow­ing, Pas­sage Bio dis­closed that it’s li­censed two ad­di­tion­al in­di­ca­tions from the Uni­ver­si­ty of Penn­syl­va­nia while keep­ing an op­tion to grab sev­en more.

Tachi Ya­ma­da

Once Penn’s Gene Ther­a­py Pro­gram — where Wil­son is di­rec­tor — com­pletes the IND-en­abling pre­clin­i­cal work with the help of the Or­phan Dis­ease Cen­ter, the ba­ton is passed to Pas­sage Bio for all clin­i­cal de­vel­op­ment, reg­u­la­to­ry strat­e­gy and even­tu­al com­mer­cial­iza­tion.

“As we con­tin­ue to work to­ward ad­vanc­ing our three lead pro­grams in­to the clin­ic over the com­ing year, we are fo­cused on our mis­sion to serve pa­tients by of­fer­ing best-in-class, life-trans­form­ing ther­a­pies,” said Stephen Squin­to, the Alex­ion co-founder and Or­biMed part­ner who is run­ning the shop as in­ter­im CEO, in a state­ment.

Liam Rat­cliffe, the new­ly mint­ed head of Ac­cess Biotech­nol­o­gy, is join­ing the board chaired by Tachi Ya­ma­da.

Ya­ma­da, a VC at Fra­zier for­mer­ly of Glax­o­SmithK­line and Take­da, was an old men­tor of Wil­son’s who of­fered cru­cial sup­port in the im­me­di­ate af­ter­math of a clin­i­cal tri­al that led to the death of a teenag­er and sent the gene ther­a­py field in­to a harsh win­ter.

Pi­o­neer­ing Click Chem­istry in Hu­mans

Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Ivan Cheung, Eisai US chairman and CEO

Bio­gen, Ei­sai re­fresh amy­loid hy­poth­e­sis with PhI­II show­ing Alzheimer's med slows cog­ni­tive de­cline

In the first look at Phase III data for lecanemab, Eisai and Biogen’s follow-up Alzheimer’s drug to the embattled Aduhelm launch, results show the drug passed with flying colors on a test looking at memory, problem solving and other dementia metrics.

One of the most-watched Alzheimer’s therapies in the clinic, lecanemab met the study’s primary goal on the CDR-SB — Clinical Dementia Rating-Sum of Boxes — giving the biotech the confidence to ask for full approval in the US, EU and Japan by next March 31. The experimental drug reduced clinical decline on the scale by 27% compared to placebo at 18 months, the companies said Tuesday night Eastern time and Wednesday morning in Japan.

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Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Vlad Coric, Biohaven CEO (Photo Credit: Andrew Venditti)

As Amy­lyx de­ci­sion waits in the wings, Bio­haven’s ALS drug sinks (again) in plat­form tri­al

The FDA’s decision on Amylyx’s ALS drug is set to come out sometime Thursday. In a space with few drugs, any approval would be a major landmark.

But elsewhere in the ALS field, things are a bit more tepid.

Thursday morning, Biohaven announced that its drug verdiperstat failed its arm of an ALS platform trial led by Massachusetts General Hospital. According to a press release, the drug did not meet its primary endpoint — improvement on an ALS functional status test — or any key secondary endpoints at 24 weeks. The trial had enrolled 167 patients, giving them either verdiperstat or placebo twice a day.

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Work taking place in the clean rooms at Vor (Credit: Vor)

Vor Bio opts to keep man­u­fac­tur­ing op­er­a­tions in-house for de­vel­op­ing stem cell, CAR-T ther­a­pies

While it is not uncommon for a biotech to go down the route of having the product manufactured by a contract organization, one small biotech is looking to keep its card close to its chest.

Vor Biopharma has started manufacturing operations at an in-house facility at its HQ in Cambridge, MA after beginning construction last summer.

According to the biotech, the facility aims to develop Vor’s hematopoietic stem cells (eHSCs) and CAR-T therapies for patients with blood cancers. The site will initially manufacture a clinical supply of its candidate VCAR33allo to support its IND, which is slated to be submitted in the first half of next year. It also plans to transfer the production of VOR33 to the facility. Vor is getting to work quickly as engineering runs for VCAR33allo has started this week.

Aim­ing for fourth nod, Sarep­ta files an­oth­er DMD gene ther­a­py to FDA; Ax­some head­ed to­ward mi­graine re­sub­mis­sion

Sarepta Therapeutics has filed the data needed for an FDA accelerated approval, which would be the biotech’s fourth if granted by the agency.

The biotech has yet to complete confirmatory trials for those first three conditional nods. The filing for its fourth Duchenne muscular dystrophy treatment, disclosed Thursday, is not a surprise. Sarepta said in late-July it would do so after releasing positive results for the Roche-partnered gene therapy.

Phillip Gomez, Siga Technologies CEO

Siga nabs $10.7M from the US gov­ern­ment in deal for its mon­key­pox an­tivi­ral

The US government is all set to buy $10.7 million worth of Siga Technologies’ monkeypox oral antiviral, the company announced Thursday.

Of the total doses, $5.1 million worth of oral antivirals called Tpoxx (tecovirimat) will be delivered this year, with the US Department of Defense having the option of buying the rest at a later point.

The new contract follows an earlier one in which the government had purchased $7.4 million worth of Tpoxx from the company.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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