Alain Baron (L), Escient CEO and Marcus Boehm, Escient CSO

Thanks to a $77.5M Se­ries B, Escient moves in­to the clin­ic with the first of its sen­so­ry re­cep­tor can­di­dates

A lit­tle over two years af­ter emerg­ing from stealth, Escient Phar­ma­ceu­ti­cals is back Mon­day with a new fundrais­ing round and their first clin­i­cal tri­al.

The San Diego-based biotech an­nounced a $77.5 mil­lion Se­ries B and a Phase I/Ib study for their EP547 pro­gram, which is aim­ing to treat the neu­ro­log­i­cal “itch” that can man­i­fest when bile and waste prod­ucts are backed up in the liv­er and kid­neys, re­spec­tive­ly. This would be the first in what CEO Alain Baron hopes is a long line of Mas-re­lat­ed G pro­tein-cou­pled re­cep­tor can­di­dates.

“We should have an IND on av­er­age once every year and a half or so,” Baron said. “So it al­lows us to de­vel­op the val­ue of some of these ear­ly tar­gets and cre­ate op­tion­al­i­ty for us to move the com­pa­ny for­ward in a num­ber of ways, and of course we’ll be da­ta-dri­ven.”

Sanofi’s VC arm and Cowen Health­care In­vest­ments led the round and were joined by new in­vestors Red­mile and Per­cep­tive. All pre­vi­ous in­vestors, in­clud­ing The Col­umn Group, 5AM Ven­tures and Os­age Uni­ver­si­ty Part­ners, pitched in again.

MRG­PRs, a fam­i­ly of GPCRs, have been the fo­cus of Escient since its found­ing, stem­ming from re­search by Johns Hop­kins neu­rol­o­gist and Escient sci­en­tif­ic founder Xinzhong Dong. With­in the MRG­PR um­brel­la are eight sen­so­ry re­cep­tor tar­gets, four of which have been de-or­phaned in the last six years, Baron said, while the re­main­ing four are still be­ing de­cod­ed. Escient ex­pects the tar­gets to serve a wide range of ther­a­peu­tic us­es and has spent the last two years try­ing to un­der­stand every­thing about this fam­i­ly while prep­ping their clin­i­cal pro­grams.

“Each re­cep­tor can be uti­lized with an an­tag­o­nist to treat more than one dis­or­der, so if you think about that, it’s pret­ty daunt­ing if we were to dis­cov­er util­i­ties for all eight,” Baron said. “We could have eight times two, eight times three in­di­ca­tions. We’re go­ing to be very dis­ci­plined in how we do this.”

Escient CSO Mar­cus Boehm, pre­vi­ous­ly the co-founder of the biotech Re­cep­tos that sold to Cel­gene for $7.2 bil­lion in 2015, added that MRG­PRs “each rec­og­nize a unique ag­o­nist, so they clear­ly have a func­tion where they rec­og­nize some­thing in the en­vi­ron­ment, or en­doge­nous­ly, that is unique from one an­oth­er.”

EP547 cen­ters around MRG­PRX4, an itch re­cep­tor trig­gered in cholesta­sis and ure­mia. Though the con­di­tions don’t cause an itch in the typ­i­cal sense, pa­tients’ brains per­ceive the sen­sa­tion of bile leak­age as an itch — caus­ing de­bil­i­tat­ing and even “em­bar­rass­ing” dis­com­fort in some cas­es, Baron said.

From the pa­tient’s per­spec­tive, an even­tu­al ther­a­py would like­ly be a once-a-day pill to an­tag­o­nize the re­cep­tors. Should the drug prove ef­fec­tive, Baron an­tic­i­pates the treat­ment to func­tion sim­i­lar­ly to an an­ti­his­t­a­mine, dra­mat­i­cal­ly re­duc­ing the itch sen­sa­tion on the first ad­min­is­tra­tion.

“It im­proves the qual­i­ty of life for these pa­tients that have re­al­ly un­bear­able itch,” Baron said. “Think about be­ing stung by 100 mos­qui­toes every day and you get a sense of the dis­tur­bance in your life that you would ex­pe­ri­ence.”

The Phase I/Ib tri­al has al­ready be­gun en­rolling, and topline da­ta are ex­pect­ed some­time in the first half of next year. Escient plans to ex­am­ine mul­ti­ple dos­es of the can­di­date in a group of about 100 in­di­vid­u­als di­vid­ed in­to sev­er­al co­horts in a ran­dom­ized, dou­ble-blind, place­bo-con­trolled set­ting.

While that work goes on, the biotech has a sec­ond pro­gram in the works tar­get­ing MRG­PRX2, a mast-cell based re­cep­tor, which could prove as a workaround in dis­eases that are not re­spon­sive to con­ven­tion­al mast-cell sta­bi­liz­ing drugs. And with enough run­way to take them past the lead pro­gram’s Phase II and the lat­ter’s Phase Ib, Baron is ex­cit­ed for what’s to come in this fam­i­ly of tar­gets.

“MRP­GRs are very in­ter­est­ing in that each is amenable to prob­a­bly more than one in­di­ca­tion,” Baron said. “So what we’re try­ing to do is pros­e­cute these re­cep­tors, and ba­si­cal­ly we do so ag­nos­tic to the ther­a­peu­tic area…whether it’s asth­ma, whether it’s der­ma­tol­ogy, whether it’s CNS, whether it’s GI, we’ll pur­sue it.”

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

So what hap­pened with No­var­tis' gene ther­a­py group? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Rafaèle Tordjman (Jeito Capital)

Con­ti­nu­ity and di­ver­si­ty: Rafaèle Tord­j­man's women-led VC firm tops out first fund at $630M

For a first-time fund, Jeito Capital talks a lot about continuity.

Rafaèle Tordjman had spotlighted that concept ever since she started building the firm in 2018, promising to go the extra mile(s) with biotech entrepreneurs while pushing them to reach patients faster.

Coincidentally, the lack of continuity was one of the sore spots listed in a report about the European healthcare sector published that same year by the European Investment Bank — whose fund is one of the LPs, alongside the American pension fund Teacher Retirement System of Texas and Singapore’s Temasek, to help Jeito close its first fund at $630 million (€534 million). As previously reported, Sanofi had chimed in €50 million, marking its first investment in a French life sciences fund.

Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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