Af­ter some care­ful think­ing, Cel­gene strikes a $295M dis­cov­ery deal on neu­rode­gen­er­a­tion

Bob Hug­in, Cel­gene

A year ago, Cel­gene’s Bob Hug­in told a room­ful of re­porters at JP Mor­gan — which in­clud­ed me — that af­ter some care­ful con­tem­pla­tion he had con­clud­ed that any com­pa­ny that in­tend­ed to have a big im­pact on health in 10 years would have to be in­volved in de­vel­op­ing new drugs for neu­rode­gen­er­a­tion.

To­day, the big biotech $CELG, known for its dizzy­ing string of col­lab­o­ra­tions aimed at main­tain­ing its dom­i­nant role in mul­ti­ple myelo­ma and blood can­cers, said it would pay Evotec $45 mil­lion up­front and up to $250 mil­lion in mile­stones to get start­ed on a dis­cov­ery pro­gram aimed at a wide range of neu­rode­gen­er­a­tive con­di­tions.

The deal cov­ers 5 years and brings Evotec’s iP­SC plat­form in­to play. As part of the col­lab­o­ra­tion, Cel­gene added that it may al­so choose to screen com­pounds from its CELMoD li­brary us­ing Evotec’s iP­SC plat­form to eval­u­ate ac­tiv­i­ty in mod­els of neu­rode­gen­er­a­tive dis­eases.

The move by Cel­gene to launch in­to neu­rode­gen­er­a­tive dis­eases comes af­ter a long line­up of phar­ma gi­ants like As­traZeneca and Glax­o­SmithK­line ei­ther ex­it­ed the field or ex­e­cut­ed a ma­jor re­treat. At the same time, new com­pa­nies like De­nali have been jump­ing in­to the game, pur­su­ing new ge­net­ic in­sights in­to these dis­eases even as a big play­er like Bio­gen makes re­al progress in search­ing for a drug that can slow or stop Alzheimer’s, which re­mains the Holy Grail of neu­rode­gen­er­a­tive R&D ef­forts. Cel­gene’s ar­rival now sends a new sig­nal about a brew­ing turn­around.

The clin­i­cal record in this field, though, would test the per­se­ver­ance of any­one in­volved in drug dis­cov­ery and de­vel­op­ment. The last 10 years has been lit­tered with re­peat fail­ures, capped by Eli Lil­ly’s dev­as­tat­ing fail­ure on solanezum­ab, its third straight set­back on the same ther­a­py.

Cel­gene be­lieves it can beat those odds, and plans to be a play­er when the win­nings are be­ing count­ed by Hug­in’s dead­line, in 2026.

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.