#ES­MO18: No­var­tis claims a key win in breast can­cer with PI3K drug alpelis­ib

MU­NICH — PI3K in­hibitors have had their prob­lems over the years, fail­ing some stud­ies, prov­ing to be dis­ap­point­ing in terms of dura­bil­i­ty in oth­ers. But No­var­tis $NVS be­lieves it may have come up with part of the an­swer with its lat­est da­ta on BYL719 (alpelis­ib) in com­bi­na­tion with ful­ves­trant.

Their al­pha-spe­cif­ic drug for PIK3CA mu­tat­ed HR+/HER2- ad­vanced breast can­cer, BYL719 plus ful­ves­trant near­ly dou­bled the pro­gres­sion-free sur­vival times for pa­tients, hit­ting 11 months com­pared to 5.7 months for a con­trol arm treat­ed on­ly with ful­ves­trant.

Fab­rice An­dré, the re­search di­rec­tor and head of IN­SERM Unit U981, called the re­sults the da­ta the “most en­cour­ag­ing” re­sults seen to date in this pa­tient group.

Rough­ly 40% of HR+ ad­vanced breast can­cer pa­tients have a PIK3CA mu­ta­tion, mak­ing this a sig­nif­i­cant piece of the over­all mar­ket.

The da­ta were re­leased over the week­end at ES­MO18.

An­dré says this drug is a stand­out, per­haps be­cause it is al­pha spe­cif­ic. Due to low­er tox­i­c­i­ty, the ma­jor­i­ty of the pa­tients can re­main on ther­a­py longer, he adds, which would ex­plain their hope for achiev­ing more durable re­spons­es than what’s been seen in oth­er PI3K pro­grams.

No­var­tis is now look­ing to fol­low up with talks at key reg­u­la­to­ry groups about an OK.

No­var­tis has had its is­sues with PI3K be­fore. The Swiss com­pa­ny hand­ed world­wide com­mer­cial rights to the late-stage bu­parlis­ib to one of Chi­na’s new up­start biotechs — rais­ing some thorny is­sues about how ques­tion­able drug as­sets may be passed to new play­ers for a com­mer­cial roll­out.

And Roche de­cid­ed to scrap its con­tender taselis­ib last June af­ter in­ves­ti­ga­tors re­port­ed a slight, 2-month pro­gres­sion-free sur­vival ad­van­tage for the drug — along with a sketchy safe­ty pro­file com­mon to the class — com­bined with ful­ves­trant hor­mone ther­a­py in a Phase III study of metasta­t­ic breast can­cer. 

No­var­tis, though, be­lieves it has grounds for an ap­proval, based large­ly on the speci­fici­ty of the drug.

“We are ex­cit­ed about the mean­ing­ful re­sults seen in SO­LAR-1 and about the pos­si­bil­i­ty to reimag­ine what po­ten­tial treat­ment op­tions could look like for pa­tients liv­ing with PIK3CA mu­tat­ed HR+/HER2- ad­vanced breast can­cer – some of who were pre­vi­ous­ly treat­ed with a CDK4/6 in­hibitor,” said Samit Hi­rawat, the head of No­var­tis On­col­o­gy Glob­al Drug De­vel­op­ment. “We are ac­tive­ly en­gag­ing in dis­cus­sions on these re­sults with health au­thor­i­ties world­wide.”

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.