Pfiz­er wins a “break­through” ti­tle for ALK/ROS1 con­tender lor­la­tinib af­ter hus­tling in­to PhI­II

Mace Rothen­berg, Pfiz­er

Last June at AS­CO, Pfiz­er re­searchers were hap­py to dis­cuss the pos­i­tive hu­man da­ta they had plumbed for lor­la­tinib (PF-06463922), their “next-gen­er­a­tion” ALK/ROS1 ty­ro­sine ki­nase in­hibitor. Hus­tling it in­to Phase II, Pfiz­er saw this pro­gram as a key part of its on­col­o­gy sto­ry, which has played a ma­jor role in win­ning new re­spect for the phar­ma gi­ant’s pipeline ef­forts.

Now the FDA is en­dors­ing that sen­ti­ment, giv­ing Pfiz­er its break­through drug des­ig­na­tion for this drug — now in a piv­otal tri­al — which could help fur­ther ac­cel­er­ate it along the path­way to a po­ten­tial ap­proval.

The BTD was pro­vid­ed for their work with ALK-pos­i­tive metasta­t­ic non-small cell lung can­cer, a huge fo­cus in the clin­ic right now. Pfiz­er re­cent­ly launched a Phase III sec­ond-line study dubbed CROWN to pro­vide the piv­otal da­ta that would be need­ed for a mar­ket­ing ap­proval.

Pfiz­er has been pour­ing mon­ey in­to its can­cer R&D work, pick­ing up a PARP drug in its $14 bil­lion Medi­va­tion buy­out and re­cent­ly win­ning an ap­proval for the check­point in­hibitor avelum­ab, part­nered with Mer­ck KGaA in a record $850 mil­lion up­front deal.

The FDA’s on­col­o­gy di­vi­sion, mean­while, has em­braced the BTD pro­gram in the last few years, rapid­ly ac­cel­er­at­ing re­views and ap­provals for the lat­est wave of can­cer ther­a­pies to hit the mar­ket.

“This reg­u­la­to­ry des­ig­na­tion rec­og­nizes the po­ten­tial for lor­la­tinib to pro­vide an im­por­tant treat­ment op­tion for pa­tients with ALK-pos­i­tive NSCLC whose can­cers have pro­gressed de­spite treat­ment. Pfiz­er’s rapid de­vel­op­ment of lor­la­tinib re­flects a com­mit­ment to de­vel­op­ing bio­mark­er-dri­ven ther­a­pies to meet the evolv­ing needs of pa­tients,” said Mace Rothen­berg, chief de­vel­op­ment of­fi­cer for on­col­o­gy Pfiz­er. “We look for­ward to work­ing with the FDA to ac­cel­er­ate the de­vel­op­ment of this ther­a­py.”

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

UP­DAT­ED: Bris­tol My­ers Squibb com­mits $300 mil­lion to com­bat racial dis­par­i­ties, but de­clines to re­lease own de­mo­graph­ic da­ta

After the police killing of George Floyd, a flurry of pharma and biotech companies, executives and investors jumped out to make statements, either expressing support for Black Lives Matter and the protests or condemning systemic racism.

Now, a Big Pharma company is publicly putting some teeth behind those statements. This morning, Bristol Myers Squibb announced they would spend $300 million on a broad effort to reduce racial health disparities, and diversify both their clinical trials and their own executive team and workforce.

Martin Shkreli (AP Images)

Mar­tin Shkre­li's in­fa­mous Dara­prim falls off top 20 most ex­pen­sive drugs list

Martin Shkreli incited a national uproar five years ago when he raised the price of Daraprim by a factor of 56 essentially overnight from $13.50 to $750 per pill. Now that the “Pharma Bro’s” high-priced project has received a generic, it no longer places among the most expensive drugs in the world.

GoodRx is back with the latest update of the top 20 most expensive drugs and Daraprim’s exclusion marks the biggest change. The drug had previously ranked seventh on the list’s last iteration, which came in February before the world went into quarantine. Another of Shkreli’s former companies, Retrophin, saw its Chenodal drug place in the top 10 again.

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