Kym­ri­ah wins sec­ond FDA OK, boost­ing hope for No­var­tis' low CAR-T sales

Af­ter dis­ap­point­ing in­vestors this year with slug­gish sales, the prospects for No­var­tis’ CAR-T ther­a­py Kym­ri­ah may be im­prov­ing. The FDA has hand­ed the phar­ma gi­ant its sec­ond ap­proval for the drug, the com­pa­ny said Tues­day af­ter­noon.

The drug, which was first ap­proved last Au­gust for pa­tients un­der 25 with B-cell pre­cur­sor acute lym­phoblas­tic leukemia, is now OK’ed to treat large B-cell lym­phoma. The can­cer must be re­lapsed or re­frac­to­ry, and the pa­tient must have gone through two or more lines of sys­temic ther­a­py first.

Kym­ri­ah — the first CAR-T ever to get ap­proval — is now al­so the first CAR-T to get ap­proval for two dis­tinct in­di­ca­tions in non-Hodgkin lym­phoma (NHL) and B-cell ALL.

Liz Bar­rett

“To­day’s FDA ap­proval of Kym­ri­ah pro­vides an­oth­er op­por­tu­ni­ty for No­var­tis to build on its lead­er­ship in CAR-T de­vel­op­ment, de­liv­er­ing a po­ten­tial­ly trans­for­ma­tive ther­a­py with durable and sus­tained re­sponse rates and a well-char­ac­ter­ized safe­ty pro­file to help pa­tients in dire need of new treat­ment op­tions,” said Liz Bar­rett, CEO of No­var­tis On­col­o­gy, in a state­ment. “We look for­ward to lever­ag­ing all of our learn­ings and new ca­pa­bil­i­ties from the ini­tial launch of Kym­ri­ah in pe­di­atric and young adult B-cell ALL for this larg­er group of pa­tients.”

Kym­ri­ah was a bit of a dis­ap­point­ment to $NVS in­vestors when Q1 re­sults came out. In the first quar­ter of 2018, the treat­ment brought in $12 mil­lion, which is rough­ly four times be­low the sales need­ed to meet an­a­lyst ex­pec­ta­tions of around $159 mil­lion for the year.

The cost of Kym­ri­ah ($475,000) could be part of the prob­lem, but there’s al­so the small pa­tient group to con­sid­er. Its first in­di­ca­tion is a rare can­cer that af­fects on­ly 3,100 new pa­tients each year, and the drug on­ly tar­gets rough­ly 30% of those pa­tients who do not re­spond to stan­dard ther­a­py.

The new ap­proval will add to that pa­tient pool and give in­vestors rea­son to hope for bet­ter sales fig­ures down the road.

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.

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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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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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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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.

As­traZeneca sets stage for mar­ket­ing ap­pli­ca­tion with promis­ing piv­otal lu­pus drug da­ta

After fumbling in its first late-stage lupus study, AstraZeneca disclosed that a second pivotal trial testing its experimental drug, anifrolumab, had met the main goal, in August. Earlier this week, the British drugmaker broke out the numbers from its successful study.

Last year, anifrolumab failed to meet the main goal of diminishing disease activity in the 460-patient TULIP I study, a 52-week trial that tested two doses of the drug versus a placebo. But in the 373-patient TULIP II study, the higher dose (300 mg) was compared to patients given a placebo — patients in both arms were on baseline standard care.

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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