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.”

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

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A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.

David Loew (Ipsen)

Ipsen snags an ap­proved can­cer drug in $247M M&A deal as an­oth­er bat­tered biotech sells cheap

You can add Paris-based Ipsen to the list of discount buyers patrolling the penny stock pack for a cheap M&A deal.

The French biotech, which has had plenty of its own problems to grapple with, has swooped in to buy Epizyme $EPZM for $247 million in cash and a CVR with milestones attached to it. Epizyme shareholders, who had to suffer through a painfully soft launch of their EZH2a inhibitor cancer drug Tazverik, will get $1.45 per share along with a $1 CVR tied to achieving $250 million in sales from the drug over four consecutive quarters as well as an OK for second-line follicular lymphoma by Jan. 1, 2028.

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Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

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Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

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Members of the G7 from left to right: Prime Minister of Italy Mario Draghi, European Commission President Ursula von der Leyen, President Joe Biden, German Chancellor Olaf Scholz, British Prime Minister Boris Johnson, Canadian Prime Minister Justin Trudeau, Prime Minister of Japan Fumio Kishida, French President Emmanuel Macron and European Council President Charles Michel (AP Photo/Susan Walsh)

Biden and G7 na­tions of­fer funds for vac­cine and med­ical prod­uct man­u­fac­tur­ing project in Sene­gal

Amidst recently broader vaccine manufacturing initiatives from the EU and European companies, the G7 summit in the mountains of Bavaria has brought about some positive news for closing vaccine and medical product manufacturing gaps around the globe.

According to a statement from the White House, the G7 leaders have formally launched the partnership for global infrastructure, PGII. The effort will aim to mobilize hundreds of billions of dollars to deliver infrastructure projects in several sectors including the medical and pharmaceutical manufacturing space.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

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