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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive”  pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Ex­elix­is pulls a sur­prise win in thy­roid can­cer just days ahead of fi­nal Cabome­tyx read­out

Exelixis added a thyroid cancer indication to its super-seller Cabometyx’s label on Friday — months before the FDA was expected to make a decision, and days before the company was set to unveil the final data at #ESMO21.

At a median follow-up of 10.1 months, differentiated thyroid cancer patients treated with Cabometyx (cabozantinib) lived a median of 11 months without their disease worsening, compared to just 1.9 months for patients given a placebo, Exelixis said on Monday.

Volker Wagner (L) and Jeff Legos

As Bay­er, No­var­tis stack up their ra­dio­phar­ma­ceu­ti­cal da­ta at #ES­MO21, a key de­bate takes shape

Ten years ago, a small Norwegian biotech by the name of Algeta showed up at ESMO — then the European Multidisciplinary Cancer Conference 2011 — and declared that its Bayer-partnered targeted radionuclide therapy, radium-223 chloride, boosted the overall survival of castration-resistant prostate cancer patients with symptomatic bone metastases.

In a Phase III study dubbed ALSYMPCA, patients who were treated with radium-223 chloride lived a median of 14 months compared to 11.2 months. The FDA would stamp an approval on it based on those data two years later, after Bayer snapped up Algeta and christened the drug Xofigo.

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Mi­rati tri­umphs again in KRAS-mu­tat­ed lung can­cer with a close­ly watched FDA fil­ing now in the cards

After a busy weekend at #ESMO21, which included a big readout for its KRAS drug adagrasib in colon cancer, Mirati Therapeutics is ready to keep the pressure on competitor Amgen with lung cancer data that will undergird an upcoming filing.

In topline results from a Phase II cohort of its KRYSTAL-1 study, adagrasib posted a response rate of 43% in second-line-or-later patients with metastatic non-small cell lung cancer containing a KRAS-G12C mutation, Mirati said Monday.

Dave Lennon, former president of Novartis Gene Therapies

Zol­gens­ma patent spat brews be­tween No­var­tis and Re­genxbio as top No­var­tis gene ther­a­py ex­ec de­parts

Regenxbio, a small licensor of gene therapy viral vectors spun out from the University of Pennsylvania, is now finding itself in the middle of some major league patent fights.

In addition to a patent suit with Sarepta Therapeutics from last September, Novartis, is now trying to push its smaller partner out of the way. The Swiss biopharma licensed Regenxbio’s AAV9 vector for its $2.1 million spinal muscular atrophy therapy Zolgensma.

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Ex-My­lan em­ploy­ee pleads guilty to in­sid­er trad­ing, il­le­gal­ly deal­ing on FDA ap­provals, earn­ings and Up­john merg­er

A former Mylan IT executive pleaded guilty Friday to an insider trading scheme where he bought and sold stock options on another executive’s advice.

Prosecutors secured the plea from Dayakar Mallu, Mylan’s former VP of global operations information technology, after uncovering the plan. Mallu collaborated with an unnamed “senior manager,” the SEC said, to trade options ahead of Mylan public announcements regarding FDA approvals, revenue reports and its merger with the Pfizer generics subsidiary Upjohn. The two subsequently shared profits.

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As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

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