In a stun­ner, FDA im­me­di­ate­ly drops its hold on Juno’s lead CAR-T

So much for bu­reau­cra­cy. In what may well be the short­est high-pro­file FDA clin­i­cal hold in the agency’s his­to­ry, Juno re­port­ed to­day that the FDA has lift­ed the halt on its lead CAR-T pro­gram, sat­is­fied that it found the cause for three pa­tient deaths.

Juno’s bat­tered shares $JUNO im­me­di­ate­ly rock­et­ed back up 28%.

Juno con­found­ed in­vestors last week when it re­port­ed the deaths and the sur­pris­ing hold, cit­ing the re­cent ad­di­tion of the chemother­a­py agent flu­dara­bine to its pre­con­di­tion­ing of pa­tients as a like­ly cul­prit. Some ex­perts, who had used the drug in oth­er stud­ies to help clear the path for a fol­lowup ther­a­py, pub­licly ques­tioned the ra­tio­nale. But the agency had no prob­lem pro­vid­ing a quick green light to re­sume the piv­otal study.

The Phase II ROCK­ET study will now con­tin­ue in adult pa­tients with re­lapsed or re­frac­to­ry B-cell acute lym­phoblas­tic leukemia (r/r ALL), us­ing on­ly cy­clophos­phamide in pre-con­di­tion­ing pa­tients for JCAR015.

Not im­me­di­ate­ly clear is whether Juno will be able to gain an ap­proval in 2017, as it had hoped be­fore the hold, putting it back in a race with ri­val Kite.

CAR-T ther­a­pies like this take cells from pa­tients and reengi­neer them with chimeric anti­gen re­cep­tors so they can be rein­ject­ed in­to pa­tients, mul­ti­ply and swarm can­cer cells. Ear­ly stud­ies have im­pressed in­ves­ti­ga­tors with their po­ten­tial for blood can­cers. But Juno roiled the field just days ago with news that a new kind of neu­ro­tox­i­c­i­ty had killed sev­er­al can­cer pa­tients in a study.

Some in­ves­ti­ga­tors, though, note that flu­darib­ine has been used in many oth­er stud­ies, with­out the kind of tox­i­c­i­ty Juno’s ther­a­py trig­gered. And be­cause it was used to help amp up the ef­fect of the drug, some won­der if Juno’s lead ther­a­py may not mea­sure up to Kite’s, which now ap­pears to be in the lead for the first, pi­o­neer­ing ap­proval.

Brad Lon­car, who man­ages an in­dex of the top 30 im­munother­a­py com­pa­nies on the mar­ket, not­ed that Juno CEO Hans Bish­op in­di­cat­ed that he would up­date in­vestors on the time­line for JCAR015 in Au­gust. Kite, mean­while, is push­ing full steam ahead on their piv­otal study. And No­var­tis in­di­cat­ed back in late May that it is on track to file an ap­proval for CTL019 in ear­ly 2017.

 

From left to right: Lilian Kim, Associate Director Business Development; John Moller, CEO; Yooni Kim, Executive Director, Asia Operations; Michelle Park, Director South Korea Operations.

Novotech CRO sees 26% growth in Asia tri­al ac­tiv­i­ty from biotechs, but still plen­ty of ca­pac­i­ty

As the Asia-Pacific clinical trials sector continues to grow rapidly, Novotech the Asia-Pacific-based CRO is seeing biotech clinical activity up by 26%. But says there is still plenty of capacity in the region that features advanced medical facilities, supportive regulatory environments, and more than 2.3 billion people, largely treatment naïve, living in urban areas.

China, South Korea and Australia have the most studies registered as recruiting or about to recruit according to ClinicalTrials.Gov.

Cell ther­a­py start­up rais­es $16 mil­lion to fund its quest for the Holy Grail in re­gen­er­a­tive med­i­cine

In 2006, Shinya Yamanaka shook stem cell research with his discovery that mature cells can be converted into stem cells, relieving a longstanding political-ethical blockage and throwing open medical research on everything from curbing eye degeneration to organ printing.

But that process still has pitfalls, including in risk and scalability, and some researchers are exploring another way first hinted at years ago: new technology to convert mature cells directly into other mature cells without the complex and time-consuming process of first making them into stem cells.

Pfizer, South San Francisco — Jeff Rumans for Endpoints News

UP­DAT­ED: Pfiz­er takes aim at a flag­ship fran­chise at Sanofi and Re­gen­eron — and scores a few di­rect hits

Count Pfizer in as a top player in the blockbuster game of JAK1 inhibitors.

Over the weekend the pharma giant posted some stellar Phase III efficacy data for their heavyweight contender abrocitinib in atopic dermatitis (eczema) that lines up ahead of a booming Dupixent (dupilumab), a blockbuster in the portfolios of Regeneron and Sanofi. And they put some real distance ahead of Eli Lilly’s trailing Olumiant, which made a delayed initial arrival on the market for rheumatoid arthritis after the FDA hobbled it with some additional hurdles on safety concerns.

JADE-MONO-1 scores well for Pfizer, teeing up what will be an intensely followed breakdown of the JADE MONO-2 data, which the pharma giant recently top-lined as “similar” to the first Phase III when tested against a placebo — a control group that has been easily outclassed by all the drugs in this market niche.

As of now, Pfizer looks to be equipped to run into the review stage — advantaged by a breakthrough therapy designation that is intended to speed up the regulatory process.

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Eye­ing $86M, Galera leads a pack of three mod­est biotech IPOs push­ing past high pro­file stum­bles

Exactly one year after kicking off a pivotal Phase III study for its lead drug — a companion for cancer patients receiving radiotherapy — Galera is looking to the Nasdaq for some new cash to complete the clinical work and fuel its commercial drive.

CEO Mel Sorensen has penciled in an $86 million ask, which was filed on the same day as liver disease company 89bio and rare disease diagnostics shop Centogene. The trio marks the first batch of IPO filings in the wake of two highly anticipated but ultimately disappointing public debuts by BioNTech and Vir, signaling dwindling biotech fervor on Wall Street. 89bio and Centogene are seeking $70 million and $69 million, respectively.

Pelosi drug pric­ing bill promis­es sav­ings, but could gag R&D — CBO analy­sis

The Democrats’ drug pricing bill — unveiled by Speaker Nancy Pelosi last month — could save Medicare spending by $345 billion over a seven year period, a new analysis suggests. But the venomous climate of impeachment proceedings and the intensifying discord between the Democrat-controlled House and Republican-majority Senate portends the bill will unlikely ever become law.

Technically, both sides of the aisle agree drug prices in the United States need some lowering. The Democrats’ bill, H.R.3 – Lower Drug Costs Now Act of 2019, is engineered to empower the HHS to negotiate prices for the 125 most expensive prescription drugs without at least two competitors — the Trump administration has already backed such a measure for the Veterans Association. Under the bill, prices for this category of medicines are not intended to exceed 120% of the average price in certain other countries (Australia, Canada, France, Germany and the United Kingdom), akin to a proposal floated by Trump earlier this year, which suggested prices be pegged against what other nations were paying as part of an “international pricing index”.

First place fin­ish: Eli Lil­ly just moved to fran­chise leader with their sec­ond mi­graine drug OK in 1 year

In a rare twist for Eli Lilly’s historically slow-moving R&D group, the pharma giant has seized bragging rights to a first-in-class new drug approval. And all signs point to an aggressive marketing followup as they look to outclass some major franchise rivals hobbled by internal dissension.

The FDA came through with an OK for lasmiditan on Friday evening, branding it as Reyvow and lining it up — once a substance classification comes through from the DEA — for a major market release. The oral drug binds to 5-HT1F receptors and is designed to stop an acute migraine after it starts. That makes it a complementary therapy to their CGRP drug Emgality, which has a statistically significant impact on preventing attacks.

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Hal Barron and Rick Klausner (GSK, Lyell)

Ex­clu­sive: GSK’s Hal Bar­ron al­lies with Rick Klaus­ner’s $600M cell ther­a­py start­up, look­ing to break new ground blitz­ing sol­id tu­mors

LONDON — Chances are, you’ve heard little or nothing about Rick Klausner’s startup Lyell. But that ends now.

Klausner, the former head of the National Cancer Institute, former executive director for global health at the Gates Foundation, co-founder at Juno and one of the leaders in the booming cell therapy field, has brought together one of the most prominent teams of scientists tackling cell therapy 2.0 — highlighted by a quest to bridge a daunting tech gap that separates some profound advances in blood cancers with solid tumors. And today he’s officially adding Hal Barron and GlaxoSmithKline as a major league collaborator which is pitching in a large portion of the $600 million he’s raised in the past year to make that vision a reality.

“We’ve being staying stealth,” Klausner tells me, then adding with a chuckle: “and going back to stealth after this.”

“Cell therapy has a lot of challenges,” notes Barron, the R&D chief at GSK, ticking off the resistance put up by solid tumors to cell therapies, the vein-to-vein time involved in taking immune cells out of patients, engineering them to attack cancer cells, and getting them back in, and more. “Over the years Rick and I talked about how it would be wonderful to take that on as a mission.”

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Court green-lights Clo­vis case af­ter de­tail­ing ev­i­dence the board ‘ig­nored red flags’ on false safe­ty and ef­fi­ca­cy da­ta

Clovis investors have cleared a major hurdle in their long-running case against the board of directors, with a Delaware court making a rare finding that they had a strong enough case against the board to proceed with the action.

In a detailed ruling at the beginning of the month that’s been getting careful scrutiny at firms specializing in biotech and corporate governance, the Delaware Court of Chancery found that the attorneys for the investors had made a careful case that the board — a collection of experts that includes high-profile biotech entrepreneurs, a Harvard professor and well-known investigator as well as Clovis CEO Patrick Mahaffy — repeatedly ignored obvious warnings that Mahaffy’s executive crew was touting inflated, unconfirmed data for their big drug Roci. Serious safety issues were also reportedly overlooked while the company continued a fundraising campaign that brought in more than a half-billion dollars. And that leaves the board open to claims related to their role in the fiasco.

The bottom line:

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Bill Gates backs Gink­go Biowork­s' $350M raise to fu­el the buzzy syn­thet­ic bi­ol­o­gy 'rev­o­lu­tion'

If you want to understand Ginkgo Bioworks, the name should suffice: Bioworks, a spin off “ironworks,” that old industrial linchpin devoted to leveraging scale as a wellspring for vast new industries capable of remaking society. Ginkgo wants to be the ironworks for the revolution it’s heralded with as much fanfare as they can, playing off of one of the buzziest technologies in biotech.

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