Pfiz­er racks up its third can­cer drug OK in 2 months, but is it enough to re­place reg­u­lar price hikes?

Pfiz­er has notched an­oth­er win for the late-stage drug pipeline, scor­ing an ac­cel­er­at­ed FDA ap­proval for lor­la­tinib, now head­ed for the mar­ket as Lor­bre­na. 

The drug is an ALK/ROS1 TKI for metasta­t­ic non-small cell lung can­cer — the lat­est in a line of these drugs — now ap­proved as a sec­ond-line treat­ment for pa­tients who have failed crizo­tinib and at least one oth­er ALK in­hibitor. And you can be sure that Pfiz­er didn’t pass on the chance of boast­ing about its third can­cer drug OK in the past 2 months, fol­low­ing ap­provals for its PARP ta­la­zoparib and da­comi­tinib.

There’s a lot about this drug that il­lus­trates the cur­rent state of can­cer drug de­vel­op­ment. Pfiz­er picked up an ac­cel­er­at­ed OK on pos­i­tive Phase I/II da­ta that will need to be borne out by piv­otal re­sults. Reg­u­la­tors were hap­py to pro­vide a fast green light for a drug that fits in the treat­ment spec­trum in the wake of a fail­ure on alec­tinib, briga­tinib or cer­i­tinib. 

The key da­ta points from their Phase II post­ed a year ago:

In ALK-pos­i­tive treat­ment-naïve pa­tients, the over­all re­sponse rate was 90%. The in­tracra­nial ORR (IC-ORR) was 75%.

For ALK-pos­i­tive pa­tients pre­vi­ous­ly treat­ed with Xalko­ri (crizo­tinib) with or with­out chemother­a­py: ORR was 69%, the IC-ORR was 68%.

In ALK-pos­i­tive pa­tients pre­vi­ous­ly treat­ed with a non-crizo­tinib ALK in­hibitor with or with­out chemother­a­py: ORR was 33%.

ALK-pos­i­tive pre­vi­ous­ly treat­ed with two or three pri­or ALK in­hibitors with or with­out chemother­a­py: ORR was 39%.

ROS1-pos­i­tive re­gard­less of pri­or treat­ment: ORR was 36%.

ALK-pos­i­tive lung can­cer cov­ers about a 5% slice of the over­all mar­ket, lim­it­ing the up­side for Pfiz­er. But with the change of the guard un­der­way at Pfiz­er’s C-suite, the phar­ma gi­ant in­sists it can re­ly on its late-stage pipeline to de­liv­er the added rev­enue it needs to please Wall Street. And that fol­lowed their de­ci­sion to back off price hikes for H2.

Or is that just win­dow dress­ing for the gi­ant phar­ma op­er­a­tor known for an ag­gres­sive M&A strat­e­gy to beef up its port­fo­lio? 

“I ex­pect our ap­proach by the end of year will be, what I would char­ac­ter­ize as busi­ness as nor­mal,” CEO Ian Read told an­a­lysts about their 2019 pric­ing strat­e­gy. And that has been wide­ly in­ter­pret­ed as mean­ing more price hikes ahead.

Pfiz­er field­ed the first TKI for ALK-pos­i­tive cas­es, Xalko­ri, 7 years ago. But it’s been a big tar­get for ri­vals. No­var­tis’ Zyka­dia picked up an FDA ap­proval to chal­lenge Xalko­ri in front­line ALK-pos­i­tive lung can­cer in mid-2017. And Roche’s Ale­cen­sa came along soon af­ter.

The game plan now is to fight back for its share of the fran­chise, with the FDA’s break­through drug des­ig­na­tion to help back up their cam­paign. And Pfiz­er wants to move as quick­ly as pos­si­ble in­to front­line use for lor­la­tinib, ex­pand­ing its po­ten­tial.

“The last decade has wit­nessed dra­mat­ic im­prove­ments in the treat­ment of metasta­t­ic ALK-pos­i­tive non-small cell lung can­cer due to ear­li­er gen­er­a­tion ALK bio­mark­er-dri­ven ther­a­pies. Yet al­most all pa­tients still re­lapse due to drug re­sis­tance, with a large pro­por­tion of pa­tients de­vel­op­ing new or wors­en­ing brain metas­tases,” said Al­ice Shaw, a pro­fes­sor at Har­vard Med­ical School and di­rec­tor of the Cen­ter for Tho­racic Can­cers at Mass­a­chu­setts Gen­er­al Hos­pi­tal.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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Covid-19 roundup: J&J be­gins piv­otal Phase III tri­al for vac­cine; Con­tro­ver­sial hu­man chal­lenge tri­als to be­gin in Lon­don — re­port

Johnson & Johnson announced it’s beginning a pivotal Phase III trial for its Covid-19 candidate, JNJ-78436735 — the first single-dose vaccine in this stage.

The Phase III trial, dubbed ENSEMBLE, will enroll 60,000 patients worldwide, making it the largest Phase III study of a Covid-19 vaccine to date. J&J said the candidate achieved positive interim results in a Phase I/IIa study, which will be published “imminently.” There’s a possibility that the first batches will be ready for potential emergency use in early 2021, according to the biotech.

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Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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Samit Hirawat (Bristol Myers Squibb)

Af­ter bruis­ing re­jec­tion, blue­bird and Bris­tol My­ers Squibb land ide-cel pri­or­i­ty re­view. But will it mat­ter for the CVR?

With the clock all but up, the FDA accepted and handed priority review to Bristol Myers Squibb and bluebird bio’s BCMA CAR-T, keeping a narrow window open for Celgene investors to still cash in on the $9 CVR from the $63 billion Celgene merger.

The acceptance comes five months after the two companies weres slammed with a surprise refuse-to-file that threatened to foreclose the CVR entirely. Today’s acceptance sets the FDA decision date for March 27, 2021 – or precisely 4 days before the CVR deadline of March 31. Given the breakthrough designation and strong pivotal data — 81.5% response rate, 35.2% complete response rate — priority review was largely expected.

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