Ax­some cel­e­brates mi­graine PhI­II win, shift­ing eyes to­ward a pair of Q4 sub­mis­sions

One week af­ter re­port­ing a late-stage fail­ure for its lead drug, Ax­some is back with pos­i­tive news: In the 302-per­son Phase III IN­TER­CEPT tri­al, its ex­per­i­men­tal mi­graine drug hit both pri­ma­ry end­points: Stop­ping mi­graine pain en­tire­ly and pre­vent­ing pain from pro­gress­ing be­yond ”mild in­ten­si­ty.”

The tri­al win sets up a big fourth quar­ter for the 8-year-old com­pa­ny and keeps them on track to file for ap­proval for the mi­graine drug, known as AXS-05. And de­spite the late-stage fail­ure, they plan to file for ap­proval on their lead de­pres­sion drug, known as AXS-07, cit­ing sec­ondary end­points and the high place­bo rates that have long cloud­ed de­pres­sion tri­al re­sults.

Her­riot Tabuteau

Ax­some was hop­ing for a win with IN­TER­CEPT, but far from de­pend­ing on it. AXS-07 — a com­bi­na­tion of the gener­ic mi­graine drug riza­trip­tan and a ver­sion of the non-steroidal an­ti-in­flam­ma­to­ry meloxi­cam — al­ready beat place­bo and riza­trip­tan alone in a Phase III tri­al of near­ly 1,600 pa­tients who had re­spond­ed poor­ly to pre­vi­ous mi­graine ther­a­pies in De­cem­ber. Af­ter those re­sults, SVB Leerink upped their odds of FDA ap­proval from 60% to 100%.

The new tri­al was de­signed and launched pri­mar­i­ly to as­sure a wider la­bel — the equiv­a­lent of mov­ing from sec­ond-line to first-line for a can­cer med­i­cine.

“Man­age­ment not­ed that the study is a pure com­mer­cial play to in­form prod­uct po­si­tion­ing, and the da­ta read­outs won’t have any im­pact on the up­com­ing NDA in 2H20,” Leerink’s Mark Good­man wrote in Feb­ru­ary.

If ap­proved, though, the drug will en­ter a new­ly con­gest­ed mar­ket. In the last two years, a se­ries of in­jectable mi­graine pre­ven­tion drugs have changed the out­look for many pa­tients. And in just the last a few months, for the first time in years, new drugs have ap­peared for treat­ing acute pain. Eli Lil­ly’s Reyvow was the first, in Oc­to­ber, fol­lowed by Al­ler­gan’s Ubrelvy in De­cem­ber and Bio­haven’s Nurtec in Feb­ru­ary.

On the pri­ma­ry end­points, AXS-07 stopped all pain for 33% of pa­tients com­pared with 16% in place­bo. And, be­tween 2 and 24 hours af­ter the at­tack, it pre­vent­ed pain from pro­gress­ing be­yond mild in­ten­si­ty in 74% of pa­tients, com­pared with 47% for place­bo.

“With IN­TER­CEPT and the pre­vi­ous­ly com­plet­ed MO­MEN­TUM Phase 3 tri­al in pa­tients with a his­to­ry of in­ad­e­quate re­sponse to pri­or acute treat­ments, AXS-07 has now been eval­u­at­ed in two pos­i­tive well-con­trolled tri­als,” CEO Her­riot Tabuteau said in a state­ment. “These tri­als demon­strate the ef­fi­ca­cy of AXS-07 against po­tent ac­tive and place­bo com­para­tors, across a spec­trum of mi­graine at­tack set­tings, re­gard­less of the tim­ing of mi­graine treat­ment, dis­ease sever­i­ty, or base­line pain in­ten­si­ty.”

As­traZeneca trum­pets the good da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Paul Hudson, Sanofi CEO (Getty Images)

Sanofi CEO Paul Hud­son has $23B burn­ing a hole in his pock­et. And here are some hints on how he plans to spend that

Sanofi has reaped $11.1 billion after selling off a big chunk of its Regeneron stock at $515 a share. And now everyone on the M&A side of the business is focused on how CEO Paul Hudson plans to spend it.

After getting stung in France for some awkward politicking — suggesting the US was in the front of the line for Sanofi’s vaccines given American financial support for their work, versus little help from European powers — Hudson now has the much more popular task of managing a major cash cache to pull off something in the order of a big bolt-on. Or two.

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David Chang, Allogene CEO (Jeff Rumans)

Head­ed to PhII: Al­lo­gene CEO David Chang com­pletes a pos­i­tive ear­ly snap­shot of their off-the-shelf CAR-T pi­o­neer

Allogene CEO David Chang has completed the upbeat first portrait of the biotech’s off-the-shelf CAR-T contender ALLO-501 at virtual ASCO today, keeping all eyes on a drug that will now try to go on to replace the first-wave personalized pioneers he helped create.

The overall response rate outlined in Allogene’s abstract for treatment-resistant patients with non-Hodgkin lymphoma slipped a little from the leadup, but if you narrow the patient profile to treatment-naïve patients — removing the 3 who had previous CAR-T therapy who didn’t respond, leaving 16 — the ORR lands at 75% with a 44% complete response rate. And 9 of the 12 responders remained in response at the data cutoff, offering a glimpse on durability that still has a long way to go before it can be completely nailed down.

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The Avance Clinical leadership team: CEO Yvonne Lungershausen, Sandrien Louwaars - Director Business Development Operations, Gabriel Kremmidiotis - Chief Scientific Officer, Ben Edwards - Chief Strategy Officer

How Aus­tralia De­liv­ers Rapid Start-up and 43.5% Re­bate for Ear­ly Phase On­col­o­gy Tri­als

About Avance Clinical

Avance Clinical is an Australian owned Contract Research Organisation that has been providing high-quality clinical research services to the local and international drug development industry for 20 years. They specialise in working with biotech companies to execute Phase 1 and Phase 2 clinical trials to deliver high-quality outcomes fit for global regulatory standards.

As oncology sponsors look internationally to speed-up trials after unprecedented COVID-19 suspensions and delays, Australia, which has led the world in minimizing the pandemic’s impact, stands out as an attractive destination for early phase trials. This in combination with the streamlined regulatory system and the financial benefits including a very favourable exchange rate and the R & D cash rebate makes Australia the perfect location for accelerating biotech clinical programs.

Ear­ly sur­vival da­ta boost Zio­phar­m's 'con­trolled IL-12' im­munother­a­py for glioblas­toma

An unconventional pairing of a gene therapy and an oral drug that promises to attack recurrent or progressive glioblastoma with controlled release of IL-12 has turned up more promising — if early — overall survival data. On top of boosting its case as a monotherapy, the data can also bode well for a combination with Regeneron’s PD-1 inhibitor, Libtayo.

Both the treatment and its developer, Ziopharm Oncology, have come a long way. The stock price peaked in 2015 but cratered in 2016 following a patient death in a Phase I.

As tislelizum­ab gains trac­tion in Chi­na, BeiGene pulls the cur­tain on NSCLC da­ta sup­port­ing the PD-1 drug

In a world now brimming with checkpoint inhibitors, companies often struggle to make a mark given a raft of therapies have already captured a considerable portion of the vast oncology market.

BeiGene’s tislelizumab was the fourth PD(L)-1 inhibitor to secure approval in China — and as it works on expanding its share the company has put out detailed data on the use of the drug in certain patients with lung cancer.

Ku­ra flash­es pos­i­tive HRAS da­ta on once-failed J&J drug

Troy Wilson was working with J&J on their KRAS inhibitor and periodically thumbing through their publications when he spotted an old drug called tipifarnib that looked promising. So promising, in fact, that the large pharma had run it through over 5,000 patients across 70 trials, hoping they would at some point be able to nail down who were the small slice of patients who responded in some studies.

Af­ter star­ring at ASH last fall, Gilead’s new Forty Sev­en crew col­ors in more promis­ing da­ta for ma­grolimab at AS­CO

We now know the full, early-stage story behind the drug that inspired Gilead CEO Dan O’Day’s recent $5 billion acquisition of Forty Seven.

Following up on their ASCO abstract from a couple of weeks ago, the team at Forty Seven is making their return appearance this week holding clearly promising early-stage data on their lead drug magrolimab as they ponder whether they should roll on a quest to obtain an accelerated approval.

Pfiz­er, Mer­ck KGaA ce­ment Baven­cio blad­der can­cer win with OS da­ta — while carv­ing an­oth­er niche in rare can­cer

Pfizer and Merck KGaA have detailed the Phase III data that inspired FDA regulators to designate Bavencio a “breakthrough” for first-line advanced bladder cancer and offered an early glance at how the PD-L1 can help patients with a rare gynecological cancer — carving out niches in the checkpoint space for itself after being shut out of numerous others.

In JAVELIN Bladder 100, Bavencio led to a 31% reduction in risk of death compared to standard care alone. It also extended median survival by more than seven months — a historic feat in this setting, according to investigators at Queen Mary University of London.