Aveo takes a big step for­ward as come­back plan de­liv­ers EU OK for ti­vo

Michael Bai­ley, Aveo

Four years af­ter Aveo On­col­o­gy $AVEO was hand­ed a hu­mil­i­at­ing re­jec­tion for its can­cer drug tivozanib, the Cam­bridge, MA-based biotech has turned an im­por­tant cor­ner in its dri­ve to re­ha­bil­i­tate it­self and turn the com­pa­ny around. The EU has sanc­tioned its lead drug tivozanib for mar­ket­ing, a de­ci­sion which trig­gers a $4 mil­lion bonus from its Eu­ro­pean part­ner with a big step to­ward an­oth­er $12 mil­lion laid out for re­im­burse­ment and reg­u­la­to­ry mile­stones.

EU­SA will roll out the drug, now ap­proved as a front­line ther­a­py for ad­vanced re­nal cell car­ci­no­ma. EU­SA com­mit­ted to a to­tal of $394 mil­lion in mile­stones when it struck the deal.

Aveo shares surged 9% this morn­ing.

Back in the spring of 2016 the new man­age­ment at Aveo agreed to pay $4 mil­lion to set­tle SEC charges that the biotech had mis­led in­vestors on ti­vo, fail­ing to di­vulge the crit­i­cal fact that reg­u­la­tors were de­mand­ing a sec­ond con­fir­ma­to­ry Phase III ahead of an ap­proval. Sep­a­rate from that deal are the fates of three for­mer top ex­ecs at the com­pa­ny — CEO Tu­an Ha-Ngoc, CFO David John­ston, and CMO William Slichen­my­er — in­volved in po­si­tion­ing the com­pa­ny as they al­leged­ly con­tin­ued to keep in­vestors in the dark as they raised fresh funds.

FDA on­col­o­gy chief Richard Paz­dur led a bru­tal slap­down of Aveo when he took to the floor dur­ing the pan­el re­view. He ar­gued ve­he­ment­ly against tivozanib, say­ing that a vote in fa­vor of a drug that had pro­duced da­ta of an in­creased risk of death would set a dan­ger­ous prece­dent. In the end, the pan­el vot­ed 13 to one against an ap­proval and the FDA soon for­mal­ly said no.

Now Aveo wants a do-over, and will like­ly get it.

“The Eu­ro­pean Com­mis­sion’s de­ci­sion is the first reg­u­la­to­ry ap­proval of tivozanib glob­al­ly, and a tremen­dous ac­com­plish­ment for Aveo and its part­ner, EU­SA Phar­ma. We are very pleased that tivozanib is now avail­able to pa­tients in Eu­rope,” said Michael Bai­ley, pres­i­dent and chief ex­ec­u­tive of­fi­cer of Aveo. “We al­so con­tin­ue to make progress on the next two pil­lars in our tivozanib strat­e­gy: U.S. reg­is­tra­tion, dri­ven by the piv­otal Phase III TI­VO-3 tri­al, which is ex­pect­ed to read out in the first quar­ter of 2018; and im­munother­a­py com­bi­na­tion tri­als, start­ing with the TiNi­vo tri­al, our Op­di­vo com­bi­na­tion tri­al.”

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.
Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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In­vestors could emerge from Neil Wood­ford de­ba­cle with £1B loss, in­ter­nal analy­sis re­veals

When Link Fund Solutions announced that it is closing Woodford Equity Income Fund permanently and kicking out Neil Woodford, it was implied that investors probably won’t get back everything they entrusted to the fund manager. But nobody knew just how much they would lose.

An internal analysis commissioned by Link suggested that the collective loss could amount to £1 billion — out of a fund last valued at £3.1 billion — Citywire has revealed.

GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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FDA Vas­cepa re­view spot­lights new safe­ty sig­nals, pos­si­ble min­er­al oil spoil­er as Amarin hunts a block­buster ap­proval

An in-house FDA review of Amarin’s Vascepa raises a set of hurdles the biotech will have to clear if the biotech expects to get the long-awaited FDA approval that could set it on a path to superstar status. But it appears that Amarin has survived another potential setback without introducing a major new threat to its prospects.

The stakes don’t get much higher, with analysts saying a win this week for Amarin could lead to billions in new sales — provided the agency stamps it with an OK. And investors liked what they say in the FDA review this morning, bumping the stock $AMRN 17%.

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FDA puts Sol­id Bio’s lead gene ther­a­py pro­gram on hold — again — af­ter an­oth­er pa­tient is hurt by SGT-001

Solid Biosciences continues to be plagued by safety issues.

Close to 18 months after the gene therapy biotech was able to quickly shed an FDA hold on their lead Duchenne muscular dystrophy program for SGT-001, regulators have stepped back in to force another halt after another patient was hit hard by a set of serious adverse events remarkably similar to the first set.

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FDA slaps a hold on an AML tri­al as Mark­er scraps a fail­ing ovar­i­an can­cer pro­gram, sink­ing shares

The FDA has placed a hold on a Phase II AML trial from the small immuno-oncology biotech Marker Therapeutics. Marker disclosed the issue two weeks after responding to FDA concerns, adding it to the Q3 release Tuesday. The company also announced it was scrapping a Phase II ovarian cancer program it determined was unlikely to succeed.

The agency’s concern centers around two reagents used in manufacturing for their trial for acute myeloid leukemia patients who have received a stem cell transplant. The reagents are from third parties and not present in the final product, Marker said.

Eli Lil­ly-backed biotech grabs $100M to dis­patch an­ti­body-oligonu­cleotide con­ju­gates af­ter mus­cu­lar dy­s­tro­phy

Hold up your hand. Make a fist. Now open it. And again.

If you can do it fully and with ease, then the proteins in your hand are likely working properly. If you can’t then they may not be. In people with myotonic muscular dystrophy, something more atomic is going on.

In those folks, the problem is RNA. Certain base pairs repeat far beyond normal, up to 11,000 superfluous letters in some cases. The extended strands form “clumps.” Proteins misform and can’t function properly. They often allow one movement but not the reverse, a condition called myotonia that gives the dystrophy its name.