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.”

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

President Donald Trump (via AP Images)

Signs of an 'Oc­to­ber Vac­cine Sur­prise' alarm ca­reer sci­en­tists

President Donald Trump, who seems intent on announcing a COVID-19 vaccine before Election Day, could legally authorize a vaccine over the objections of experts, officials at the FDA and even vaccine manufacturers, who have pledged not to release any vaccine unless it’s proved safe and effective.

In podcasts, public forums, social media and medical journals, a growing number of prominent health leaders say they fear that Trump — who has repeatedly signaled his desire for the swift approval of a vaccine and his displeasure with perceived delays at the FDA — will take matters into his own hands, running roughshod over the usual regulatory process.

#ES­MO20: Push­ing in­to front­line, Mer­ck and Bris­tol My­ers duke it out with new slate of GI can­cer da­ta

Having worked in parallel for years to move their respective PD-1 inhibitors up to the first-line treatment of gastrointestinal cancers, Merck and Bristol Myers Squibb finally have the data at ESMO for a showdown.

Comparing KEYNOTE-590 and CheckMate-649, of course, comes with the usual caveats. But a side-by-side look at the overall survival numbers also offer some perspective on a new frontier for the reigning checkpoint rivals, both of whom are claiming to have achieved a first.

Jonathan Rigby, Immune Regulation group CEO

Im­mune Reg­u­la­tion, tak­ing two clin­i­cal pro­grams to 're­set' the im­mune sys­tem, nets $53M+ Se­ries B

A little under two years after a company rebranding, Immune Regulation is taking an even bigger step toward advancing its goals.

Formerly known as Peptinnovate, the British biotech announced a $53.4 million Series B early Monday morning, helping to further advance two clinical programs in rheumatoid arthritis and asthma. Though those are the two initial indications the company is focusing on, CEO Jonathan Rigby told Endpoints News he hopes the candidates can be applied to a broad swath of autoimmune disorders.

#ES­MO20: Bris­tol My­ers marks Op­di­vo's sec­ond ad­ju­vant win — eye­ing a stan­dard of care gap

Moving into earlier and earlier treatment lines, Bristol Myers Squibb is reporting that adjuvant treatment with Opdivo has doubled the time that esophageal or gastroesophageal junction cancer patients stay free of disease.

With the CheckMate-577 data at ESMO, CMO Samit Hirawat said, the company believes it can change the treatment paradigm.

While a quarter to 30% of patients typically achieve a complete response following chemoradiation therapy and surgery, the rest do not, said Ronan Kelly of Baylor University Medical Center. The recurrence rate is also high within the first year, Hirawat added.

Is­raeli biotech rais­es $57M to go where cur­rent BRAF in­hibitors can't, with back­ing from No­var­tis, SR One

For the blockbuster potential of Novartis’ Tafinlar and Pfizer’s Braftovi, all the BRAF inhibitors on the market so far only target V600 mutations — which accounts for roughly 50% of patients.

Israeli biotech Novellus now has $57 million to develop a drug that they say can help the other 50% who have everything else.

The Series C will fund a Phase II trial for PLX-8394, a “paradox breaker” that could block RAF without activating MAPK signaling. In a Phase I trial, a patient with a BRAF fusion saw their tumor go away after taking the drug, allowing Novellus to hit the ground running.

Clay Siegall (Life Science Washington via YouTube)

#ES­MO20: Seat­tle Ge­net­ics eyes 4th ap­proval with new da­ta in a crowd­ed field

Does Seattle Genetics have another approval on its hands?

The last 12 months, not so great for the world, has been great for Seattle Genetics. The company landed two separate FDA approvals, signed a $4.5 billion deal with Merck and watched antibody-drug conjugates — the technology they spent years developing to broad industry skepticism — emerge suddenly as one of the most popular approaches in oncology. And on Monday at ESMO, the company and their partners at Genmab unveiled the data behind the ADC it hopes will provide its next major FDA approval.

Israel Lowy (Regeneron)

#ES­MO20: 'As good as any PD-1 out there': Re­gen­eron flash­es PD-(L)1 lung can­cer da­ta to ri­val Mer­ck

Regeneron entered the PD-(L)1 game late, so they devised a two-pronged strategy to catch up with Big Pharma rivals: They would push it into cancers where PD-1s had yet been tested, and they would prove that it’s as powerful in the big indications as any other on the market.

They cleared a hurdle on the first goal Friday, showing a 31% response in patients with the rare skin cancer basal cell carcinoma. And with the data they’re rolling out Monday, Regeneron cancer chief Israel Lowy is ready to declare success on the second.

Eli Lilly CSO Dan Skovronsky (file photo)

UP­DAT­ED: #ES­MO20: Eli Lil­ly shows off the da­ta for its Verzenio suc­cess. Was it worth $18 bil­lion?

The press release alone, devoid of any number except for the size of the trial, added nearly $20 billion to Eli Lilly’s market cap back in June. Now investors and oncologists will get to see if the data live up to the hype.

On Sunday at ESMO, Eli Lilly announced the full results for its Phase III MonarchE trial of Verzenio, showing that across over 5,000 women who had had HR+, HER2- breast cancer, the drug reduced the odds of recurrence by 25%. That meant 7.8% of the patients on the drug arm saw their cancers return within 2 years, compared with 11.3% on the placebo arm.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 90,400+ biopharma pros reading Endpoints daily — and it's free.