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.”

CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means.

Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society.

One of Europe’s most high-profile biopharma investors is getting $540 million to invest in new crossover deals for late-stage companies.

The Paris-based VC says the fresh Sofinnova Crossover Fund raise positions them as the “largest crossover investor in Europe dedicated to late-stage biopharma and medtech investments.”

They got a leg up in France after winning a special “Tibi” designation from the French government, giving them access to a pool of €6 billion that helped them gain an edge with institutional investors. Since they were founded close to 50 years ago, the venture group has backed more than 500 companies and currently has more than €2 billion under management.

Seattle-based Presage Biosciences, which approaches drug development through its microdosing platform, has some new partnerships and cash to come with them.

Presage closed a $13 million financing round Tuesday, aiming to expand its network of clinical trial sites and advance development of its microdosing injection devices. They also closed partnership deals with Merck and Maverick Therapeutics.

The financing included $7 million from new investors, including the LabCorp Venture Fund, Bristol Myers Squibb, and InHarv Partners. An additional $6 million convertible note from Takeda Ventures will convert to equity.