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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Paul Hudson, Sanofi CEO (Cyril Marcilhacy/Bloomberg via Getty Images)

Sanofi snaps up new vac­cine can­di­date and de­vis­es mR­NA game plan around it — but not for what you think

Paul Hudson has spotlighted vaccines, immunology and dermatology as some of the top R&D focuses at Sanofi. His latest deal brings all of them together.

The French pharma giant isn’t sharing any financial details about the buyout of Origimm, a low-profile, private Austrian biotech whose technology promises to identify antigens causing skin disease and build vaccines against them. Their lead candidate targets acne vulgaris.

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As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

Mod­er­na los­es lat­est bat­tle in key vac­cine de­liv­ery patent fight as fed­er­al ap­peal falls flat

The US Court of Appeals for the Federal Circuit on Wednesday rejected Moderna’s attempt to overturn key patents related to the delivery vehicle for its Covid-19 vaccine after the biotech sought to preempt a potentially risky infringement lawsuit.

For years, Moderna has been battling a tiny Pennsylvania biotech known as Arbutus over patents for a technology required to deliver its mRNA drugs and vaccines, known as lipid nanoparticles or LNP. Moderna is concerned there’s a substantial risk that Arbutus will assert the ’069 patent in an infringement suit targeting Moderna’s Covid-19 vaccine, particularly as Arbutus has boasted of its patent protection and refused to grant a covenant not to sue Moderna.

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Lan Huang, BeyondSpring CEO

Months af­ter shock­ing in­vestors with lung can­cer win, Be­yond­Spring's lead drug hits road­block at the FDA

BeyondSpring shocked investors in early August after its once-marginal lead drug suddenly showed a lot of promise in a common form of lung cancer. With hopes high, the FDA has now slammed the door on that drug in another indication — does that spell bad news for BeyondSpring’s Cinderella story?

The FDA issued BeyondSpring a complete response letter for its plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia, effectively shutting down the drug’s immediate chances at a marketing approval, the biotech said Wednesday.

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