Robert Habib (file photo)

Once laser-fo­cused on liv­er, Mi­NA takes swing at neu­rol­o­gy with Servier's back­ing

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.”

The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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UP­DAT­ED: Mer­ck pulls Keytru­da in SCLC af­ter ac­cel­er­at­ed nod. Is the FDA get­ting tough on drug­mak­ers that don't hit their marks?

In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders?

Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday.

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Thank you, next: Take­da hands Ovid $196M cash to rein back in Phase III-ready seizure drug, re­viv­ing bat­tered stock

Soticlestat made it.

Takeda is bringing the drug back into its fold more than four years after first entrusting the team at Ovid with the mid-stage clinical work. For all that — generating what they saw as positive Phase II data in Dravet syndrome and Lennox-Gastaut syndrome — the biotech has been rewarded with $196 million in upfront cash, with another $660 million reserved for regulatory and commercial milestones.

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Hal Barron, Endpoints UKBIO19

GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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As Brain­Storm con­tin­ues to tout ‘clear sig­nal’ on ALS drug, the FDA of­fers a rare pub­lic slap­down on the da­ta

A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement.

The FDA statement amounts to a straight slap own, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance.

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In­tro­duc­ing End­points FDA+, our new pre­mi­um week­ly reg­u­la­to­ry news re­port led by Zachary Bren­nan

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.

Eli Lil­ly claims suc­cess in a new JAK in­di­ca­tion: hair loss

Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one.

Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms.

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Antoine Papiernik, Sofinnova managing director (Business Wire)

Sofinno­va Part­ners stays fo­cused on late-stage deals with a new, $540M crossover fund

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.

Presage teams with Mer­ck on its Phase 0 test­ing; Kem­Pharm AD­HD drug wins ap­proval in chil­dren aged 6 and up

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.