Brian McVeigh, Code Biotherapeutics CEO

Gene ther­a­py biotech look­ing to by­pass vi­ral de­liv­ery wins in­vestor ap­proval, bag­ging Se­ries A

The vi­ral vec­tors that car­ried the first gen­er­a­tion of gene ther­a­pies in­to the clin­ic have proven to be a trou­ble­some lot for a va­ri­ety of rea­sons. And now one of the biotechs brew­ing new ways to cir­cum­vent those ob­sta­cles in gene ther­a­py 2.0 has a ma­jor new fund­ing round to fu­el the work.

Code Bio­ther­a­peu­tics an­nounced Tues­day that it raised $75 mil­lion via a Se­ries A round, cour­tesy of a group of blue-chip in­vestors. CEO Bri­an McVeigh — a GSK vet­er­an who used to run the phar­ma’s BD unit — told End­points News that the funds will last the biotech well in­to 2024. And so far, there are some big plans for that fund­ing, in­clud­ing pipeline and in-house man­u­fac­tur­ing ex­pan­sion, plus pro­gress­ing the com­pa­ny’s two dis­ease pro­grams fur­ther in pre­clin­i­cal in­to IND-en­abling stud­ies.

The biotech said in a state­ment that North­pond Ven­tures led the fi­nanc­ing round, along­side Am­gen Ven­tures, Hat­teras Ven­ture Part­ners and UCB Ven­tures. Pre­vi­ous in­vestors who jumped in on the round in­clud­ed NEA, 4BIO Cap­i­tal and Take­da Ven­tures.

The biotech has been fo­cus­ing on non-vi­ral gene ther­a­py de­liv­ery since the biotech of­fi­cial­ly start­ed in 2020, launch­ing out of stealth with a $10 mil­lion seed round last year in a cur­rent fo­cus on type 1 di­a­betes and Duchenne mus­cu­lar dy­s­tro­phy, or DMD. As McVeigh ex­plained, the main tech­nol­o­gy be­hind Code Bio is a drug de­liv­ery scaf­fold made of syn­thet­ic DNA strands, and has 18 ends of base pairs 3 prime and 5 prime. Com­bined with a tar­get­ing moi­ety, the scaf­fold can be brought to a spe­cif­ic cell type — and bring along a ge­net­ic med­i­cine pay­load to the cell, too.

As McVeigh fur­ther elab­o­rat­ed, go­ing the syn­thet­ic scaf­fold ap­proach would al­low for one key dif­fer­en­tia­tor — re-dos­ing. Cur­rent AAV vec­tors are on­ly good for one dose, as pa­tient’s im­mune sys­tems ei­ther build up an im­mu­ni­ty to the virus or are al­ready nat­u­ral­ly im­mune.

So far, Code Bio has been work­ing on da­ta to show that its tar­get­ing sys­tem works — that the scaf­fold can go in­to mus­cle cells for DMD and al­pha/be­ta cells in the liv­er for type 1 di­a­betes. The next step — at least for the de­liv­ery sys­tem — is to test the ef­fi­ca­cy in in vi­vo mod­els for both dis­eases.

Ad­di­tion­al­ly, Code is mov­ing for­ward with an ap­proach that will al­low for the scaf­fold to take an en­tire copy of the dy­s­trophin gene — the one that is dys­reg­u­lat­ed in pa­tients with DMD and the largest gene — in­to the body. Be­yond that, McVeigh said the biotech is al­so look­ing at a small­er ver­sion called Beck­er’s gene (about two-thirds the length of the dy­s­trophin gene) and its own mi­crody­s­trophin.

While it’s too soon to say which of Code’s op­tions will move for­ward, McVeigh added that it has seen a wan­ing ef­fect in AAV-based mi­crody­s­trophin ap­proach­es. How­ev­er, if on­ly Code Bio’s mi­crody­s­trophin ap­proach makes it through to the fin­ish line and the FDA’s thumbs-up, it would still be able to re-dose pa­tients — and then it may not mat­ter what DMD ap­proach comes through clin­i­cals and reg­u­la­to­ry re­view.

Michael Ehlers

The biotech is one of sev­er­al play­ers to try and get move­ment in the non-vi­ral gene ther­a­py de­liv­ery space, and most of them have sub­stan­tial back­ing of some sort. Gen­er­a­tion Bio closed a nine-fig­ure IPO last June in a bid to push pre­clin­i­cal work for a non-vi­ral gene ther­a­py in liv­er and reti­na dis­eases. Michael Ehlers — the for­mer R&D chief at Bio­gen and ex-CSO, neu­ro­science at Pfiz­er — is part of an­oth­er ef­fort as an ad­vi­sor at Xalud Ther­a­peu­tics.

This new Se­ries A is al­so just a few months af­ter Code Bio scored a part­ner­ship with Take­da — worth up to $2 bil­lion — back in Feb­ru­ary for four un­spec­i­fied rare dis­ease projects. And ac­cord­ing to McVeigh, while it is still very ear­ly in the re­la­tion­ship, it has been great so far. The biotech al­so has two more phar­ma part­ner­ships — but as to who those phar­mas are, that re­mains a mys­tery.

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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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.

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.

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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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.

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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No stranger to gene ther­a­py woes, Astel­las runs in­to an­oth­er safe­ty-re­lat­ed clin­i­cal hold

Astellas Pharma, which has been at the forefront of uncovering the risks associated with gene therapies delivered by adeno-associated viruses, must take another safety alarm head-on.

The FDA has slapped a clinical hold on Astellas’ Phase I/II trial of a gene therapy candidate for late-onset Pompe disease, after investigators flagged a serious case of peripheral sensory neuropathy.

It marks the latest in a streak of setbacks Astellas has encountered since making a splashy entry into the gene therapy space with its $3 billion buyout of Audentes. But the lead program, AT132 for the treatment of X-linked myotubular myopathy (XLMTM), had to be halted more than once after a total of four patients died in the trial — and the scientific community still doesn’t have all the answers of what caused the deaths.

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