No­var­tis heads to the fin­ish line with CDK 4/6 block­buster con­tender ri­bo­ci­clib

In the fi­nal stretch in its jour­ney to reg­u­la­tors, No­var­tis post­ed a pos­i­tive snap­shot of piv­otal Phase III da­ta on its CDK 4/6 con­tender ri­bo­ci­clib (LEE011), set­ting up a like­ly show­down with Pfiz­er in treat­ing breast can­cer.

The head­line fig­ure from ES­MO is that ri­bo­ci­clib com­bined with letro­zole re­duced the risk of death or pro­gres­sion by 44% among first-line pa­tients com­pared to letro­zole alone, of­fer­ing fur­ther en­cour­age­ment to the phar­ma gi­ant that it’s right on track to gain­ing an ap­proval for this drug next year.

A me­di­an pro­gres­sion-free sur­vival ben­e­fit could not be es­tab­lished, but the trend is clear. Their study, pub­lished in the New Eng­land Jour­nal of Med­i­cine, con­clud­ed:

Af­ter 12 months, the pro­gres­sion-free sur­vival rate was 72.8% (95% con­fi­dence in­ter­val [CI], 67.3 to 77.6) in the ri­bo­ci­clib group and 60.9% (95% CI, 55.1 to 66.2) in the place­bo group; af­ter 18 months, the pro­gres­sion-free sur­vival rate was 63.0% (95% CI, 54.6 to 70.3) and 42.2% (95% CI, 34.8 to 49.5), re­spec­tive­ly.

Pfiz­er was able to land an ac­cel­er­at­ed ap­proval for pal­bo­ci­clib (sold as Ibrance) in ear­ly 2015 with ear­ly da­ta demon­strat­ing a 20.2-month PFS rate for Ibrance plus letro­zole com­pared to about 10.2 months for par­tic­i­pants re­ceiv­ing on­ly letro­zole.

No­var­tis — which end­ed their study ear­ly on pos­i­tive da­ta — has sev­er­al things go­ing in its fa­vor now. The FDA has ex­pe­ri­ence with CDK 4/6 and al­so grant­ed ri­bo­ci­clib a break­through drug des­ig­na­tion, flag­ging the dis­tinct pos­si­bil­i­ty of a fast OK. Bar­ring a nasty sur­prise, that would put No­var­tis in line to chal­lenge Pfiz­er for a block­buster pop­u­la­tion. And it would like­ly get them out in­to a mar­ket they know well ahead of Eli Lil­ly, which is bull­ish about its chances with the ri­val CDK 4/6 drug abe­maci­clib.

Said prin­ci­pal in­ves­ti­ga­tor Gabriel N. Hor­to­bagyi:

“The MONALEESA-2 re­sults show the com­bi­na­tion of LEE011 plus letro­zole rep­re­sents a sig­nif­i­cant step for­ward in the man­age­ment of HR+ metasta­t­ic breast can­cer and, if ap­proved, would be a ma­jor ad­di­tion to the treat­ment op­tions these pa­tients have. Women liv­ing with metasta­t­ic breast can­cer will be on treat­ment for the rest of their lives, so it is crit­i­cal to find treat­ment op­tions that ef­fec­tive­ly de­lay pro­gres­sion.”

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.

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.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

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.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,800+ biopharma pros reading Endpoints daily — and it's free.

Ted White, Verrica CEO

Ver­ri­ca hits an­oth­er bump in the road with CMO re­lat­ed let­ter from FDA

The FDA has rejected Verrica’s new drug application for VP-102 again, with the company pinning the CRL on problems at a CMO that it was partnered with, the company announced Monday.

The FDA didn’t raise issues that directly relate to the manufacturing of VP-102, the company said, but raised “general quality issues” at the CMO’s facility. There were also no clinical concerns, it said, or need to collect more data.

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.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,800+ biopharma pros reading Endpoints daily — and it's free.

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.

Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 117,800+ biopharma pros reading Endpoints daily — and it's free.