The FDA opens an in­side track to let No­var­tis race ahead on Zyka­dia as Roche ri­val­ry heats up

Vas­ant Narasimhan, Glob­al Head Drug De­vel­op­ment and Chief Med­ical Of­fi­cer for No­var­tis

No­var­tis just leaped for­ward with its can­cer drug Zyka­dia (cer­i­tinib), gain­ing a pri­or­i­ty re­view from the FDA as a front­line treat­ment for ALK-pos­i­tive non-small cell lung can­cer as well as break­through sta­tus for lung can­cer pa­tients with brain metas­tases. The ad­vances are keep­ing the phar­ma gi­ant close to their fel­low Basel res­i­dents at Roche in their head-to-head ri­val­ry with Ale­cen­sa, which an­a­lysts have blessed as the odds-on win­ner.

Last fall No­var­tis not­ed that Zyka­dia had aced a Phase III study for lung can­cer, hand­i­ly beat­ing out chemother­a­py in hold­ing back ALK-pos­i­tive lung can­cer pro­gres­sion among pa­tients pre­vi­ous­ly treat­ed with Pfiz­er’s Xalko­ri. Roche’s Ale­cen­sa has a break­through drug des­ig­na­tion — a like­ly in­di­ca­tor of a quick OK in this case — for use as a front­line drug in ALK-pos­i­tive lung can­cer.

Ale­cen­sa has jumped to an ear­ly, though small, sales lead as the two Big Phar­ma com­pa­nies duke it out over front­line sta­tus. And Roche land­ed its sec­ond break­through des­ig­na­tion from the FDA last Oc­to­ber af­ter their drug beat out Xalko­ri among Japan­ese pa­tients with an ALK-pos­i­tive form of the dis­ease.

The FDA is like­ly to act quick­ly on both of these drugs, pos­si­bly not even wait­ing out the full six-month re­view for Zyka­dia now that No­var­tis will get a pri­or­i­ty re­view. In No­var­tis’ Phase III Zyka­dia pa­tients had a me­di­an pro­gres­sion-free sur­vival rate of 16.6 months com­pared to 8.1 months for chemo.

There are about 1.8 mil­lion lung can­cer di­ag­noses each year and rough­ly 2% to 7% of cas­es are ALK-pos­i­tive. Ale­cen­sa is man­u­fac­tured by Roche’s big Genen­tech sub­sidiary.

“We are com­mit­ted to ad­vanc­ing our un­der­stand­ing of mu­ta­tion-dri­ven lung can­cer, where there con­tin­ues to be sig­nif­i­cant un­met need,” said Vas Narasimhan, Glob­al Head Drug De­vel­op­ment and Chief Med­ical Of­fi­cer, No­var­tis. “To­day`s Pri­or­i­ty Re­view of Zyka­dia for new­ly di­ag­nosed pa­tients with ALK+ metasta­t­ic NSCLC, in­clud­ing Break­through Ther­a­py des­ig­na­tion for those with brain metas­tases, brings us clos­er to de­liv­er­ing the right treat­ment to the right pa­tient at the right time.”

Take­da swoops in to buy lit­tle biotech part­ner and its celi­ac drug poised to 'change stan­dard of care'

Having spent three years carefully grooming PvP Biologics and its drug for celiac disease, Takeda is happy enough with the proof-of-concept data to buy it all.

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Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Andre Kalil, AP Images

A 9/11-era Om­a­ha fa­cil­i­ty, an old Ebo­la drug, and the ubiq­ui­tous Dr. Fau­ci: In­side the first US nov­el coro­n­avirus tri­al

The first 11 coronavirus patients who arrived in Omaha last week, airlifted across the globe after two weeks quarantined on a cruise ship, showed only minor symptoms or none at all. And then one of them — or one of the couple of Americans who arrived later — got worse. He developed pneumonia, a life-threatening complication for coronavirus patients.

In a biocontainment room at the University of Nebraska Medical Center on Friday, doctors infused him with an experimental Gilead drug once developed for Ebola, called remdesivir. Or they gave him a placebo. For the first time in the US, neither he nor the doctors knew.

The first US novel coronavirus trial was underway and with it, a mad dash for an answer. Sponsored by the NIH, the study marked a critical point in the epidemic. Since the start of the outbreak, the agency had helped lead a global effort to contain the virus. Now, as it spread worldwide and the CDC issued warnings the US could see a major internal outbreak, they were looking at home.

“We don’t have too much time,” Andre Kalil, the trial’s lead investigator, told Endpoints News. “Everything’s moving really fast.”

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Dan O'Day (AP Images)

A name emerges out of the Gilead M&A ru­mor mill, and it’s a can­cer biotech

After months of questions and speculation about when and if Gilead will make a major acquisitions, a name has emerged.

The California-based drugmaker has approached Forty Seven Inc, a cancer biotech, with a takeover offer, Bloomberg News reports. With Forty Seven’s market cap at $2.3 billion, an acquisition would likely be Gilead’s largest since they acquired Kite Pharma for $11.9 billion in 2017.

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Biogen head of R&D Al Sandrock, Sangamo CEO Sandy Macrae

UP­DAT­ED: Bio­gen makes an­oth­er bold Alzheimer’s bet, drop­ping $350M up­front to part­ner with genome-edit­ing fo­cused Sang­amo

While the fate of Biogen’s resurrected Alzheimer’s drug aducanumab remains uncertain, the Cambridge, MA-based drugmaker is joining forces with genome editing company Sangamo Therapeutics to develop therapies for neurological conditions.

Sangamo is set to receive a meaty $350 million upfront in cash and stock and is eligible to receive up to $2.37 billion in milestone payments, in addition to royalties. In return, Biogen gets the rights to two Sangamo preclinical compounds: ST-501 (for use in tauopathies including Alzheimer’s disease) and ST-502 (for synucleinopathies including Parkinson’s disease).

“The partnership represents a lower-cost way to expand its work in neurologic disease,” Credit Suisse’s Evan Seigerman said in a note, referring to Biogen.

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Spark los­es an­oth­er top ex­ec in the wake of $4.3B takeover by Roche — re­port

Days after bidding farewell to co-founder Kathy High, Spark Therapeutics — now operating under Roche — has one more opening on its C-suite.

Kathy Reape

Kathy Reape, who joined the Philadelphia-based biotech in 2016 as head of clinical R&D and became chief medical officer in 2018, is reportedly set to leave.

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Vlad Coric (Photo Credit: Andrew Venditti)

Bio­haven scores CGRP OK for acute mi­graine — can the com­mer­cial team catch up with Al­ler­gan on its de­but?

Seven years after spinning out of Yale, Biohaven has entered the ranks of commercial-stage biotechs.

The FDA handed down an OK for its CGRP drug, rimegepant, as an acute treatment. Dubbed Nurtec, the orally dissolving pill will join Allergan’s (soon to be AbbVie’s) Ubrelvy and Lilly’s Reyvow on the market amid a new wave of migraine therapies reshaping the disease space.

In a pivotal Phase III trial, Nurtec hit the co-primary endpoints on pain freedom and freedom from most bothersome symptoms at two hours post dose, proving superior to placebo.

'The head­lines are the head­lines, but': Bio­Marin talks up po­ten­tial sav­ings as he­mo­phil­ia gene ther­a­py launch looms

BioMarin execs are still staying tight-lipped about their pricing plans for what is poised to be the world’s first hemophilia gene therapy. But as the company enters the final regulatory stretch and approaches a potential launch this summer, they are also dropping more hints to get investors ready.

First thing to know: They really, really don’t expect an advisory committee to be convened for valrox, which is under priority review, to pop up before its PDUFA date on August 21.

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An­oth­er hic­cup for GW Phar­ma's seizure drug ri­val, as Zo­genix dis­clos­es FDA re­view de­lay

Zogenix has had a troubling 2020 so far. Earlier this month, its experimental seizure drug met the main goal in a pivotal study in patients with Lennox-Gastaut Syndrome, but the company saw its shares plummet after the magnitude of the therapy’s effect fell short of Wall Street expectations. On Thursday, the drug developer said that the FDA had extended the review of the drug in patients with Dravet syndrome by three months.