Michelle Longmire, Medable CEO (Photo Credit: Jeff Rumans)

Med­able trots its way to uni­corn sta­tus amid vir­tu­al tri­al craze. Is a pub­lic de­but next?

As it be­comes ap­par­ent that de­cen­tral­ized clin­i­cal tri­als will like­ly be a part of drug­mak­ers’ strate­gies long af­ter the pan­dem­ic is over, a tech de­vel­op­er is gal­lop­ing its way to uni­corn sta­tus with a $304 mil­lion megaround.

Med­able un­veiled a hefty Se­ries D round on Tues­day — its fourth in the last year and a half — led by Black­stone Growth, Tiger Glob­al and GSR Ven­tures. The new cash brings the Pa­lo Al­to-based com­pa­ny’s to­tal raise to $521 mil­lion, with a whop­ping $2.1 bil­lion val­u­a­tion.

The team will use these funds to tack­le long­stand­ing “bar­ri­ers to drug de­vel­op­ment,” in­clud­ing a lack of di­ver­si­ty in clin­i­cal tri­als, the time it takes to con­duct a large study, and a lack of aware­ness for tri­als them­selves.

San­skri­ti Thakur

“We are not mak­ing med­i­cine for every­body,” chief growth of­fi­cer San­skri­ti Thakur told End­points News. “The re­al­i­ty is on­ly 2 to 3% of the pop­u­la­tion who are ap­pro­pri­ate for sci­en­tif­ic re­search ever get ac­cess to it.”

The num­bers are even more stark for com­mu­ni­ties of col­or. Of the 53 drugs ap­proved this past year, Black pa­tients rep­re­sent­ed about 8% of par­tic­i­pants in the tri­als reg­u­la­tors based their de­ci­sions on (and for which da­ta on race was col­lect­ed), de­spite rep­re­sent­ing about 13% of the US pop­u­la­tion. Lat­inX par­tic­i­pants rep­re­sent­ed just un­der 13% of stud­ies.

Med­able be­lieves its plat­form can be used to re­cruit more di­verse pa­tient pop­u­la­tions faster — and re­tain them. It’s the type of tech­nol­o­gy that al­lows vol­un­teers to pick up a phone to par­tic­i­pate in a tri­al, rather than vis­it­ing a clin­ic, which may be far away, or op­er­at­ing un­der re­stric­tions amid the pan­dem­ic.

“De­cen­tral­ized tri­al tech­nolo­gies have been crit­i­cal to drug de­vel­op­ment dur­ing the pan­dem­ic, pro­vid­ing glob­al re­mote ac­cess and sup­port­ing COVID vac­cine and ther­a­peu­tic re­search at record speed,” CEO and co-founder Michelle Long­mire said in a state­ment. “Pa­tients need the life sci­ences in­dus­try to con­tin­ue in­no­vat­ing at this pace.

Since launch­ing in 2012 and then se­cur­ing its first cus­tomer in 2016, Med­able has de­ployed its soft­ware in more than 150 de­cen­tral­ized and hy­brid tri­als in 60 coun­tries. As ex­pect­ed, the busi­ness saw a big boost amid the pan­dem­ic, an­nounc­ing last year that its rev­enue had soared more than 500%.

Now that it’s had the time to look at the da­ta from those tri­als, Med­able claims its plat­form helped achieve 200% faster en­roll­ment rates, 90% re­ten­tion rates, and 50% cost re­duc­tions.

Last year, the com­pa­ny launched three new ap­pli­ca­tions: Tele­Vis­it, which con­nects pa­tients with site co­or­di­na­tors and in­ves­ti­ga­tors; Tele­Con­sent, which al­lows pa­tients to vir­tu­al­ly con­sent and re-con­sent to clin­i­cal tri­als; and Tele­COA, which com­bines elec­tron­ic Clin­i­cal Out­come As­sess­ments (eCOAs) with Tele­Vis­its. It al­so struck part­ner­ships with Data­vant, AliveCor and MRN.

When asked if an IPO is on the hori­zon, Thakur re­spond­ed: “We will fund our­selves in or­der to achieve our mis­sion. And I think that we have to re­al­ly con­sid­er what the right struc­ture is for us.”

Med­able is far from alone in this space. Sci­ence 37 took its first stroll down Wall Street just last month when it com­plet­ed a $235 mil­lion SPAC merg­er with LifeSci Ac­qui­si­tion II Corp. Parex­el, a CRO with vir­tu­al tri­al abil­i­ties, was snapped up by Gold­man Sachs’ pri­vate eq­ui­ty branch and the EQT IX fund for $8.5 bil­lion ear­li­er this year. And those are just a few of the play­ers bet­ting that the fu­ture of clin­i­cal tri­als is vir­tu­al.

“It’s im­por­tant that we rec­og­nize that the whole in­dus­try is at a de­ci­sion point,” Thakur said.

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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