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.

A small, ob­scure biotech just won big with their IPO. In this mar­ket. Are you kid­ding me?

How could a small, largely unknown biotech that emerged from stealth mode just months ago with early-stage cancer programs jump onto Wall Street in the middle of a Force 5 financial hurricane and sail through with a $165 million IPO?

And what does that mean for the rest of the industry waiting to see just how much damage global lockdowns will wreak on clinical development?

The biotech is a company called Zentalis. The crew there nabbed an $85 million crossover round late last year — notably waiting 5 years before waving the numbers around to attract attention, according to my read of a FierceBiotech story. Perceptive joined in, but the syndicate was not in general the kind of marquee affair that gets tongues wagging.

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Bob Nelsen at the Milken Institute Global Conference on April 29, 2019 in Beverly Hills, California. (Photo by Michael Kovac/Getty Images)

ARCH chief Bob Nelsen has $1.5B to prove 2 sim­ple points: ‘We’re in the most in­no­v­a­tive time ever’ and in­vestors are stay­ing

ARCH co-founder and managing director Bob Nelsen has a well known yen for the home run swing, betting big on potentially transformative meds and tech and the biotech teams he helps bring together. He thrives and bleeds on the cutting edge. And now Nelsen and the ARCH group have debuted 2 big funds to prove that this is the time for the best of biotech to shine — deadly pandemic be damned.

Two new funds, ARCH Venture Fund X and ARCH Venture Fund X Overage, gathered a combined $1.46 billion. And that’s a record. ARCH Venture Fund IX and ARCH Venture Fund IX Overage closed in 2016 with a combined $1.1 billion. ARCH Venture Fund VIII and ARCH Venture Fund VIII Overage closed in 2014 with a combined $560 million.

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In­vest­ing in the time of coro­n­avirus: the good, the bad and the hope­ful, as biotech VC firms close funds worth $3B

Apart from disrupting biopharma R&D and regulatory timelines, the coronavirus pandemic has inevitably ravaged financial markets and eroded investor risk appetite. Investing in the time of coronavirus feels reckless, but if biotech venture funds are any indication, the time is ripe.

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GSK vac­cine chief heads for AIDS vac­cine ini­tia­tive; Pfiz­er en­lists Sue Desmond-Hell­mann to its board of di­rec­tors

→ Rip Ballou, who until very recently led vaccine research and development at GlaxoSmithKline, is joining the International AIDS Vaccine Initiative (IAVI) to lead its USAID-funded ADVANCE program. The program uses a network of researchers and institutions in Africa to help develop a vaccine for HIV. Ballou had worked at GSK since 2010 and has led global vaccine R&D since 2015. Prior to that he held posts at the Bill & Melinda Gates Foundation, a different post at GSK, Medimmune, and Walter Reed Army Institute of Research.  IAVI is led by Mark Feinberg, the former CSO of Merck Vaccines. 

Ahead of US IPO, Leg­end Biotech adds $150M, top-tier in­vestors to back CAR-T pipeline

Last month Nanjing Legend Biotech revealed that it sees, and was quietly planning for, a future as a public company in the US, separate but still tied to its former parent, Chinese CRO GenScript. It’s evidently a vision that enticed investors, drawing marquee names for a pre-IPO round.

The Series A fetched a whopping $150.5 million from Hudson Bay Capital Management, Lilly Asia Ventures, Vivo Capital, RA Capital Management and JJDC, the venture arm of J&J. The pharma giant has helped fund Legend’s CAR-T work with the $350 million upfront payment it handed over to partner on the lead BCMA program.

Covid-19 roundup: Mod­er­na CEO Stéphane Ban­cel be­comes a bil­lion­aire; No­var­tis, In­cyte pitch Jakafi PhI­II to tack­le se­vere cas­es

You can now add Moderna CEO Stéphane Bancel to the list of biotech billionaires.

Since investors began to understand the full scope of the coronavirus outbreak in late February, cash has flushed out of much of the stock market and into the handful of companies leading the fight for drugs and vaccines. That’s meant hundreds of millions for Moderna, the Flagship-backed company that emerged early as the world’s first best hope for a Covid-19 vaccine.

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J&J gives pi­o­neer­ing stem cell biotech its first Big Phar­ma deal, part­ner­ing on iP­SC CAR-T and CAR-NK

Late last summer, one of the earliest stem cell therapy companies got two government decisions in the span of three weeks: The USPTO granted them a patent for iPSC-derived CAR-T cells, and then the FDA cleared them for their first-in-human CAR-NK trial.

Yesterday, the two technologies landed them an up-to $3.1 billion deal.

Fate Therapeutics and J&J announced a global collaboration that will pay Fate $100 million upfront and a trove of potential milestones to develop multiple CAR-T and CAR-NK therapies. It’s the first Big Pharma partnership Fate has announced in their 13-year existence and the largest, although at least one longtime follower thought they could have landed more.

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FDA re­ports short­age of se­da­tion drug used for putting Covid-19 pa­tients on ven­ti­la­tors

The FDA on Thursday updated its list of drugs in shortage to include the sedation drug midazolam, which along with other sedatives is being used to treat COVID-19 patients requiring mechanical ventilation.

The updated listings for five manufacturers note an increased demand for midazolam and may just be the beginning of what’s to come for other sedation drugs too.

Michael Ganio, senior director of pharmacy practice and quality at the American Society for Health-System Pharmacists, told Focus via email: “We have multiple reports of increases in purchases and utilization of sedatives like midazolam, fentanyl, and propofol to treat COVID-19 patients who require mechanical ventilation.”

Drug dis­cov­ery in the age of coro­n­avirus

Developing new drugs is incredibly hard. That’s why, despite superhuman efforts from the industry, we’re still looking at 12-18 months minimum before we can realistically hope for a vaccine for Covid-19, and probably months before there’s a proven viable drug treatment.

But our increasing ability to begin to industrialize the drug discovery and development process through an engineering approach means that we have more hope for speeding up this process than ever before — and not just to defeat coronavirus, but to benefit the development of all new medicines in the future.