J&J wins snap ap­proval of apa­lu­tamide, the first drug OK'd for non­metasta­t­ic prostate can­cer

Richard Padzur

The FDA has giv­en J&J a big boost to­day, cut­ting its pri­or­i­ty re­view for apa­lu­tamide down to a quick once over and a snap ap­proval months ahead of its PDU­FA date for non­metasta­t­ic prostate can­cer.

The drug will now be sold as Er­leade, hand­i­ly beat­ing a ri­val sup­ple­men­tal ap­pli­ca­tion from Pfiz­er on its can­cer drug Xtan­di. This ap­proval came through just two months af­ter the agency of­fered to give it a pri­or­i­ty re­view, un­der­scor­ing how fast reg­u­la­tors are will­ing to act.

Eval­u­ate Phar­ma has pegged 2022 sales at $1.6 bil­lion for apa­lu­tamide, flag­ging some of the zeal that an­a­lysts have for this drug. A spokesper­son for the com­pa­ny says that the “whole­sale ac­qui­si­tion cost (WAC) of a 30-day sup­ply of ER­LEA­DA is $10,920.00 per bot­tle of 120 tablets (60 mg tablets), which is in line with oth­er oral on­col­o­gy med­i­cines.”

The ap­proval couldn’t come too soon. J&J is fac­ing the near-term loss of patent pro­tec­tion on Zyti­ga, which is sold for metasta­t­ic prostate can­cer.

“This ap­proval is the first to use the end­point of metas­ta­sis-free sur­vival, mea­sur­ing the length of time that tu­mors did not spread to oth­er parts of the body or that death oc­curred af­ter start­ing treat­ment,” said Richard Paz­dur, di­rec­tor of the FDA’s On­col­o­gy Cen­ter of Ex­cel­lence. “In the tri­al sup­port­ing ap­proval, Er­lea­da had a ro­bust ef­fect on this end­point. This demon­strates the agency’s com­mit­ment to us­ing nov­el end­points to ex­pe­dite im­por­tant ther­a­pies to the Amer­i­can pub­lic.”

J&J spent $650 mil­lion in cash and of­fered an­oth­er $350 mil­lion in mile­stones to bag Rich Hey­man’s Aragon in 2013, just so it could have this drug.

Just days ago in­ves­ti­ga­tors spelled out some im­pres­sive da­ta. Their piv­otal tri­al hit a me­di­an MFS rate of 40.5 months vs 16.2 months in the place­bo group. “Sec­ondary end­points (TTM, PFS, and Sym­Prog) were all sig­nif­i­cant­ly im­proved.

On the oth­er hand you have Pfiz­er, which post­ed an im­pres­sive 21.9-month im­prove­ment  in metas­ta­sis-free sur­vival for prostate can­cer — 36.6 months vs 14.7 months [P < .0001] — for Xtan­di as well as time to first use of new an­ti­neo­plas­tic ther­a­py (39.6 mo vs 17.7 mo [P < .0001]) and time to PSA pro­gres­sion (37.2 mo vs 3.9 mo).

J&J can now get a head start in the field, look­ing to score some block­buster rev­enue.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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Maureen Hillenmeyer, Hexagon Bio CEO

Hexa­gon Bio rais­es $61M to con­tin­ue ef­forts to turn fun­gi in­to drugs

A year after raising a $47 million launch round, the fungi-loving drug hunters at Hexagon Bio have more than doubled their coffers.

Hexagon announced today that it raised another $61 million for its efforts to design cancer and infectious disease drugs based on insights mined from the DNA in millions of species of fungi. The new financing brings Hexagon’s committed funding to over $108 million.

Blue­bird sends blood dis­or­der drug to FDA for ap­proval; CG On­col­o­gy en­ters col­lab­o­ra­tion with Roche for Tecen­triq

Bluebird bio announced it completed the rolling submission of its BLA to the FDA for betibeglogene autotemcel gene therapy.

The therapy, designed for patients with beta-thalassemia who require regular red blood cell transfusions, was previously granted breakthrough therapy designation for treating transfusion-dependent beta-thalassemia (TDT). If approved, beti-cel will be the first hematopoietic stem cell ex-vivo gene therapy for patients in the US.