Once fo­cused on hard-to-treat liv­er dis­eases, Mi­NA Ther­a­peu­tics is join­ing forces with Servi­er to en­gage its small ac­ti­vat­ing RNA tech­nol­o­gy on an­oth­er dif­fi­cult front: neu­rol­o­gy.

Mi­NA and Servi­er an­nounced a new re­search al­liance Thurs­day cen­tered around neu­ro­log­i­cal dis­or­ders. While the part­ners are keep­ing qui­et about their tar­gets for now, Mi­NA CEO Robert Habib vague­ly re­vealed that the first one was nom­i­nat­ed by Servi­er, which has an op­tion over it. Mi­NA stands to re­ceive up to $266.3 mil­lion (€220 mil­lion) in an up­front pay­ment and mile­stones on that tar­get alone, though they de­clined to break those num­bers down any fur­ther.

In ad­di­tion, Servi­er has ex­clu­siv­i­ty over two more tar­gets, Habib said. Un­der the terms of the deal, Mi­NA will iden­ti­fy po­ten­tial can­di­dates and Servi­er will take the lead on pre­clin­i­cal and clin­i­cal de­vel­op­ment.

“This is sort of our first step in CNS with Servi­er, but it has the po­ten­tial to ex­pand quite sig­nif­i­cant­ly across many tar­gets,” Habib told End­points News.

Mi­NA was co-found­ed in 2008 by Habib’s fa­ther, the promi­nent Im­pe­r­i­al Col­lege Lon­don pro­fes­sor Nagy Habib. Robert left in­vest­ment bank­ing about sev­en years ago to take the helm at his fa­ther’s com­pa­ny, set­ting out on a mis­sion to help treat the “un­drug­gable” — start­ing with liv­er can­cer.

saR­NA is sim­i­lar to RNA in­ter­fer­ence, in that they both re­ly on short strands of RNA and a pro­tein called arg­onaute-2. Ex­cept in­stead of si­lenc­ing genes, saR­NA am­pli­fies.

“We have en­zymes with­in our bod­ies that are re­spon­si­ble for gene reg­u­la­tion. And what we do is we de­sign small RNA se­quences that di­rect those en­zymes to spe­cif­ic re­gions of a gene, which in do­ing so can trig­ger the gene to in­crease tran­scrip­tion, to re­lease more mes­sen­ger RNA and more pro­tein,” Habib ex­plained.

The Lon­don-based biotech’s lead pro­gram goes af­ter CEB­PA — a gene that pro­vides in­struc­tions to make a type of tran­scrip­tion fac­tor pro­tein — in the hopes of re­duc­ing im­mune sup­pres­sion caused by im­ma­ture myeloid cells to im­prove the ef­fi­ca­cy of can­cer ther­a­pies. Its can­di­date, MTL-CEB­PA, is be­ing test­ed as a com­bi­na­tion ther­a­py with Bay­er’s Nex­avar in he­pa­to­cel­lu­lar car­ci­no­ma and Mer­ck’s Keytru­da in ad­vanced sol­id tu­mors. Sev­er­al months ago, Mi­NA land­ed a near­ly $30 mil­lion Se­ries A round to push those pro­grams for­ward.

Habib ex­pects to wrap up the Phase I dose es­ca­la­tion por­tion of the Keytru­da study around the end of this quar­ter, and launch in­to dose ex­pan­sion in Q2. The first da­ta snap­shot could come as ear­ly as the end of this year, he said.

The Servi­er deal al­lows Mi­NA to branch out from liv­er dis­eases and ex­plore new ter­ri­to­ry “ripe for our tech­nol­o­gy,” Habib said. It’s one of sev­er­al col­lab­o­ra­tions that Mi­NA has inked over the last few years, in­clud­ing one with As­traZeneca last Jan­u­ary that gave the phar­ma ne­go­ti­at­ing rights to a li­cens­ing agree­ment af­ter a se­ries of pre­clin­i­cal stud­ies.

Pe­ter Bains

In 2017, So­sei put down about $45 mil­lion for 25.6% of Mi­NA’s eq­ui­ty. Pe­ter Bains, So­sei’s CEO at the time, laid out a $534 mil­lion plan to ac­quire Mi­NA, and thus ex­pand So­sei’s glob­al reach. That same year, Mi­NA signed an up-to $371.9 mil­lion deal with Boehringer In­gel­heim for three liv­er fi­bro­sis tar­gets, in­clud­ing at least one NASH drug. But a year and a half lat­er, So­sei passed on the op­tion, keep­ing its stake.

“The pow­er of saR­NA tech­nol­o­gy is that it is a chem­i­cal drug that works by a tran­sient dose-de­pen­dent mech­a­nism, very much like a chem­i­cal drug. But be­cause it tar­gets gene tran­scrip­tion and can in­crease gene tran­scrip­tion, it can hit tar­gets that are not drug­gable by con­ven­tion­al med­i­cine,” Habib said.

“This is re­al­ly ex­tend­ing the broad­er suc­cess that we’re see­ing in RNA more wide­ly,” CBO Pe­ter Bains said, ref­er­enc­ing com­pa­nies like Al­ny­lam and Mod­er­na. “So this is… an­oth­er modal­i­ty for RNA in­ter­ven­tion.”

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After years as the top spending pharma TV advertiser, AbbVie’s Humira brand finally downshifted earlier this year, ceding much of its marketing budget to up-and-coming sibling meds Skyrizi and Rinvoq. However, now Humira is back on TV with ads for another condition — Hidradenitis suppurativa (HS).

The chronic and painful skin condition results in lumps and abscesses caused by inflammation or infection of sweat glands, most often in the armpits or groin. Humira was first approved to treat HS in 2015 and remains the only FDA-approved drug for the condition. Two TV ads both note more than 30,000 people with HS have been prescribed Humira.