No­var­tis nabs pri­or­i­ty re­view for a block­buster-to-be aimed at NSCLC niche — de­light­ing In­cyte ex­ecs

In­cyte fi­nal­ly has some good news to share about one drug it’s dis­cov­ered that’s not Jakafi — al­beit one that doesn’t be­long to it any­more.

FDA reg­u­la­tors have ac­cept­ed cap­ma­tinib, an or­phan and “break­through” MET in­hibitor, for pri­or­i­ty re­view. The hon­or tech­ni­cal­ly goes to No­var­tis, which li­censed world­wide de­vel­op­ment and com­mer­cial­iza­tion rights to the drug back in 2009.

But that didn’t stop In­cyte from brag­ging about it in a re­lease, re­mind­ing in­vestors that if every­thing goes well, it’s el­i­gi­ble for $500 mil­lion-plus in mile­stones as well as roy­al­ties up to 14% of glob­al sales.

Shav­ing four months off the re­view time­line — as­sum­ing there aren’t hic­cups — helps en­sure that No­var­tis can score an ap­proval with­in this year. The phar­ma gi­ant had des­ig­nat­ed cap­ma­tinib (for­mer­ly INC280) as one of four ma­jor launch­es of 2020, pre­dict­ing a block­buster fu­ture for a drug ini­tial­ly aimed at a sub­type of non-small cell lung can­cer.

NSCLC pa­tients who har­bor a MET ex­on-14 skip­ping mu­ta­tion cur­rent­ly have no ap­proved tar­get­ed ther­a­py. A No­var­tis-spon­sored Phase II tri­al showed that cap­ma­tinib in­duced an over­all re­sponse rate of 67.9% among treat­ment-naïve pa­tients (du­ra­tion of re­sponse 11.14 months) and 40.6% among pre­vi­ous­ly treat­ed ones (DOR 9.72 months).

Those 97 pa­tients had EGFR wild­type, ALK-neg­a­tive tu­mors, and took 400 mg cap­ma­tinib tablets twice a day.

“ME­Tex14 mu­ta­tions oc­cur in 3-4% of new­ly di­ag­nosed ad­vanced NSCLC cas­es and is a rec­og­nized onco­genic dri­ver,” No­var­tis not­ed in a state­ment.

Mer­ck KGaA and Pfiz­er al­so an­gling for this un­tapped niche; both have been grant­ed break­through ther­a­py des­ig­na­tions, though theirs on­ly cov­er sec­ond-line ther­a­py fol­low­ing plat­inum-based chemo. While tepo­tinib from the Ger­man drug­mak­er was en­gi­neered as a se­lec­tive MET in­hibitor, the drug Pfiz­er has put for­ward is Xalko­ri, orig­i­nal­ly tar­get­ed at ALK and ROS1 mu­ta­tions.

Mean­while, in Chi­na, Chi-Med is al­so test­ing As­traZeneca’s MET in­hibitor, savoli­tinib, in a Phase II reg­is­tra­tional tri­al among pa­tients who have pro­gressed fol­low­ing pri­or sys­temic ther­a­py or are un­able to re­ceive chemo.

A com­pan­ion di­ag­nos­tic will like­ly be key to the adop­tion of any of these treat­ments. No­var­tis has teamed up with Foun­da­tion Med­i­cine — now a Roche sub­sidiary — to in­clude tu­mor tis­sue and liq­uid biop­sy di­ag­nos­tics on its flag­ship prod­ucts.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

Endpoints Premium

Premium subscription required

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

David Light, Valisure CEO

Val­isure in the hot seat: New Form 483 over a 2021 in­spec­tion as CEO fires back

The notorious drug testing company Valisure, which has made a name for itself by forcing FDA’s hand with some of its safety-related uncoverings, received a letter this week after the FDA uncovered violations at its Connecticut-based testing lab in 2021.

The letter, which was sent on Dec. 5, stated that the FDA is “concerned” that Valisure was not aware of  drug supply chain security requirements.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,300+ biopharma pros reading Endpoints daily — and it's free.

FDA re­view­ers head back to White Oak in 2023, with lead­er­ship look­ing to ap­pease a new Con­gress

Republicans have taken a stand against the pandemic era habit of lax work-from-home schedules. Now that they’ve wrestled control of the House majority, the FDA’s leadership is playing ball, sending many of the agency’s more than 18,000 employees back to their desks early next year.

Whether this exodus back to White Oak in Silver Spring, MD (many staff will still be allowed to work from home for multiple days per week) will mean more defections to industry and elsewhere remains to be seen.

Bags of shred­ded docs: In­di­an drug­mak­er Lupin hand­ed a Form 483 by FDA in­spec­tors

The generics manufacturer Lupin has been given another Form 483 from the FDA this year.

US regulators inspected Lupin’s pharmaceutical manufacturing site in the town of Mandideep, India from Nov. 14 through Nov. 23, with the 14-page report marking 16 observations.

The inspection report stated that the site did not have the appropriate controls over its computer systems to ensure that changes in “master production” or records are only done by authorized personnel, along with written procedures not being established to conduct annual reviews of records associated with drug batches.

Bro­ken promis­es? FDA needs more pow­er to re­move drugs from mar­ket­place, JA­MA analy­sis finds

The FDA is struggling to remove drugs from the marketplace that don’t show effectiveness in late stage trials, new JAMA analyses found, thanks to the persistent tension between speed and confidence in early clinical data.

Congress, regulated industry and patients have urged the FDA to shorten the amount of time that the market has to wait for drugs to become available that may help severe and prevalent diseases – and the FDA has listened, offering up a quick accelerated approval pathway that’s frequently used by new cancer drugs.