Take­da antes up $44M for rare dis­ease pact, turn­ing to Ox­ford spin­out's ex­o­some tech

More than two weeks in­to Take­da’s new work guide­lines in re­sponse to the Covid-19 cri­sis — or­der­ing staffers to work from home and stay off the road — the BD team has some­thing to cel­e­brate.

An­tonin de Fougerolles

In a boost to the rare dis­ease unit built around Shire’s lega­cy R&D op­er­a­tion af­ter its $62 bil­lion buy­out deal, Take­da has inked a new col­lab­o­ra­tion in pur­suit of ex­o­some de­liv­ery tech­nol­o­gy. Their part­ner of choice is Evox Ther­a­peu­tics, an Ox­ford spin­out.

In­clud­ing up­front, mile­stones and re­search fund­ing, the near-term pay­ment comes in at $44 mil­lion.

Evox stands to earn a to­tal of $882 mil­lion if the Japan­ese phar­ma gi­ant fol­lows through with all five pro­tein re­place­ment and mR­NA ther­a­pies that it has the op­tion to take over. On­ly two tar­gets have been se­lect­ed; the first is a pre­clin­i­cal pro­gram for Nie­mann-Pick dis­ease type C while the sec­ond is undis­closed.

The British biotech is in charge of every­thing un­til IND-en­abling stud­ies, as well as man­u­fac­tur­ing up to the end of Phase I tri­als. But past the pre­clin­i­cal han­dover, the man­u­fac­tur­ing costs will be re­im­bursed.

“[T]he deal sig­nif­i­cant­ly ex­tends our cash run­way in­to late 2022 and al­lows us to ag­gres­sive­ly ex­pand our own pro­pri­etary pipeline of rare dis­ease drugs, in­clud­ing a urea cy­cle dis­or­der pro­gramme we ex­pect to en­ter the clin­ic in 2021,” CEO An­tonin de Fougerolles said in a state­ment.

Hav­ing raised over $55 mil­lion from in­vestors in­clud­ing GV, Red­mile Group and Panacea Health­care Ven­ture, Evox has po­si­tioned it­self among the hand­ful of play­ers that are us­ing tiny ex­o­somes — part of the body’s nat­ur­al vesi­cle couri­er sys­tem — to de­liv­er a ther­a­peu­tic pay­load where need­ed, in­clud­ing tar­gets in the brain. Boehringer In­gel­heim is al­so in for an al­liance.

Mad­hu Natara­jan

For Take­da, this adds a promis­ing plat­form to “en­hance tis­sue de­liv­ery for a va­ri­ety of pay­loads,” said Mad­hu Natara­jan, a Shire vet who’s now head­ing Take­da’s rare dis­ease drug dis­cov­ery unit. “Col­lab­o­rat­ing on the Evox ex­o­some plat­form al­so com­ple­ments our ex­pand­ing ca­pa­bil­i­ties in cell and gene ther­a­pies.”

Though falling well be­hind some of its Big Phar­ma brethren, Take­da is de­vel­op­ing its own AAV-based gene ther­a­pies for he­mo­phil­ia. Out with non-core as­sets that are ei­ther get­ting spun out or sold, in with in­no­v­a­tive ther­a­pies — the new modus operan­di is in full swing.

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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