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.”

Im­ple­ment­ing re­silience in the clin­i­cal tri­al sup­ply chain

Since January 2020, the clinical trials ecosystem has quickly evolved to manage roadblocks impeding clinical trial integrity, and patient care and safety amid a global pandemic. Closed borders, reduced air traffic and delayed or canceled flights disrupted global distribution, revealing how flexible logistics and supply chains can secure the timely delivery of clinical drug products and therapies to sites and patients.

Vas Narasimhan, Novartis CEO (Jason Alden/Bloomberg via Getty Images)

Vas Narasimhan's 'Wild Card' drugs: No­var­tis CEO high­lights po­ten­tial jack­pots, as well as late-stage stars, in R&D pre­sen­ta­tion

Novartis is always one of the industry’s biggest R&D spenders. As they often do toward the end of each year, company execs are highlighting the drugs they expect will most likely be winners in 2021.

And they’re also dreaming about some potential big-time lottery tickets.

As part of its annual investor presentation Tuesday, where the company allows investors and analysts to virtually schmooze with the bigwigs, Novartis CEO Vas Narasimhan will outline what he thinks are the pharma’s “Wild Cards.” The slate of five experimental drugs are those that Novartis hopes can be high-risk, high-reward entrants into the market over the next half-decade or so, and cover a wide range of indications.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

Pascal Soriot (AP Images)

UP­DAT­ED: As­traZeneca, Ox­ford on the de­fen­sive as skep­tics dis­miss 70% av­er­age ef­fi­ca­cy for Covid-19 vac­cine

On the third straight Monday that the world wakes up to positive vaccine news, AstraZeneca and Oxford are declaring a new Phase III milestone in the fight against the pandemic. Not everyone is convinced they will play a big part, though.

With an average efficacy of 70%, the headline number struck analysts as less impressive than the 95% and 94.5% protection that Pfizer/BioNTech and Moderna have boasted in the past two weeks, respectively. But the British partners say they have several other bright spots going for their candidate. One of the two dosing regimens tested in Phase III showed a better profile, bringing efficacy up to 90%; the adenovirus vector-based vaccine requires minimal refrigeration, which may mean easier distribution; and AstraZeneca has pledged to sell it at a fraction of the price that the other two vaccine developers are charging.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,100+ biopharma pros reading Endpoints daily — and it's free.

Bahija Jallal (file photo)

TCR pi­o­neer Im­muno­core scores a first with a land­mark PhI­II snap­shot on over­all sur­vival for a rare melanoma

Bahija Jallal’s crew at TCR pioneer Immunocore says they have nailed down a promising set of pivotal data for their lead drug in a frontline setting for a solid tumor. And they are framing this early interim readout as the convincing snapshot they need to prove that their platform can deliver on a string of breakthrough therapies now in the clinic or planned for it.

In advance of the Monday announcement, Jallal and R&D chief David Berman took some time to walk me through the first round of Phase III data for their lead TCR designed to treat rare, frontline cases of metastatic uveal melanoma that come with a grim set of survival expectations.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Gen­mab ax­es an ADC de­vel­op­ment pro­gram af­ter the da­ta fail to im­press

Genmab $GMAB has opted to ax one of its antibody-drug conjugates after watching it flop in the clinic.

The Danish biotech reported Tuesday that it decided to kill their program for enapotamab vedotin after the data gathered from expansion cohorts failed to measure up. According to the company:

While enapotamab vedotin has shown some evidence of clinical activity, this was not optimized by different dose schedules and/or predictive biomarkers. Accordingly, the data from the expansion cohorts did not meet Genmab’s stringent criteria for proof-of-concept.

Michelle Longmire, Medable CEO (Jeff Rumans)

Med­able gets $91M for vir­tu­al clin­i­cal tri­als, bring­ing to­tal raise to $136M

As biotechs look to get clinical studies back on track amid the pandemic, Medable returned to the venture well for the second time this year, bagging a $91 million Series C to build out its virtual trial platform.

The software provider recently launched three new apps for decentralizing clinical trials, and saw a 500% revenue spike this year. And it isn’t alone. Back in August, Science 37 secured a $40 million round for its virtual trial tech, with support from Novartis, Sanofi Ventures and Amgen. Patients and researchers are taking a liking to the online approach, suggesting regulators could allow it to become a new normal even after the pandemic is over.

Feng Tian, Ambrx CEO (Ambrx)

Af­ter 5 qui­et years, a for­mer Scripps spin­out rais­es $200M and an­nounces plans to try again at an IPO

The first time San Diego biotech Ambrx tried to go public in 2014, they failed and the company’s board switched to a radically different strategy: They sold themselves for an undisclosed amount to a syndicate of Chinese investors and pharma companies.

Now, after 5 quiet years, that syndicate has raised a mountain of cash and indicated they’ll soon make another bid to go public.

Earlier this month, Ambrx raised $200 million in what they billed as a crossover round financed by Fidelity, BlackRock, Cormorant Asset Management, HBM Healthcare Investments, Invus, Adage Capital Partners and Suvretta Capital Management. It’s the largest amount they’ve ever raised and, according to Crunchbase figures, more than doubles the total amount of VC capital collected since their launch 17 years ago.

The ad­u­canum­ab co­nun­drum: The PhI­II failed a clear reg­u­la­to­ry stan­dard, but no one is cer­tain what that means any­more at the FDA

Eighteen days ago, virtually all of the outside experts on an FDA adcomm got together to mug the agency’s Billy Dunn and the Biogen team when they presented their upbeat assessment on aducanumab. But here we are, more than 2 weeks later, and the ongoing debate over that Alzheimer’s drug’s fate continues unabated.

Instead of simply ruling out any chance of an approval, the logical conclusion based on what we heard during that session, a series of questionable approvals that preceded the controversy over the agency’s recent EUA decisions has come back to haunt the FDA, where the power of precedent is leaving an opening some experts believe can still be exploited by the big biotech.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Pur­due Phar­ma pleads guilty in fed­er­al Oxy­Con­tin probe, for­mal­ly rec­og­niz­ing it played a part in the opi­oid cri­sis

Purdue Pharma, the producer of the prescription painkiller OxyContin, admitted Tuesday that, yes, it did contribute to America’s opioid epidemic.

The drugmaker formally pleaded guilty to three criminal charges, the AP reported, including getting in the way of the DEA’s efforts to combat the crisis, failing to prevent the painkillers from ending up on the black market and encouraging doctors to write more painkiller prescriptions through two methods: paying them in a speakers program and directing a medical records company to send them certain patient information. Purdue’s plea deal calls for $8.3 billion in criminal fines and penalties, but the company is only liable for a fraction of that total — $225 million.