Pi­lot project pro­vides a snap­shot of ef­fec­tive­ness of Pfiz­er’s PARP con­tender ta­la­zoparib

Jen­nifer Lit­ton, MD An­der­son Can­cer Cen­ter

You can ex­pect some no­table re­search up­dates on the slate of late-stage PARP in­hibitors at this week­end’s an­nu­al Eu­ro­pean So­ci­ety of Med­ical On­col­o­gy meet­ing. And an ear­ly teas­er on what’s to come at ES­MO should help whet every­one’s ap­petite for more.

Jen­nifer Lit­ton, the prin­ci­pal in­ves­ti­ga­tor in the Phase III ta­la­zoparib study and an in­ves­ti­ga­tor at MD An­der­son Can­cer Cen­ter, set up a small pi­lot study that used the PARP in­hibitor on a group of 13 breast can­cer pa­tients with BR­CA mu­ta­tions.

These pa­tients were giv­en the drug ahead of pre-sur­gi­cal chemo, and Lit­ton says her group tracked an av­er­age 78% tu­mor shrink­age among the women in the study. The full range ex­tend­ed from 30% to 98%.

“Ac­knowl­edg­ing that this is a small study, I can’t think of any sys­temic ther­a­py that gives re­sults this con­sis­tent­ly strong in on­ly two months,” Lit­ton said. A few weeks ago, Lit­ton start­ed an ex­ten­sion of the pi­lot, en­rolling the first 6 of 20 pa­tients who can shed fur­ther light on the drug’s use in treat­ing breast can­cer. And her group is us­ing back­ing from MD An­der­son’s Moon Shots pro­gram for the work, look­ing to quick­ly ad­vance new ther­a­pies for can­cer.

“If this study pro­duces sim­i­lar­ly strong re­sults, the next step would be to di­rect­ly com­pare ta­la­zoparib to chemother­a­py in the pre-sur­gi­cal, cu­ra­tive set­ting,” Lit­ton added, “We might be able to de­lay or re­place chemother­a­py if we can get sim­i­lar ef­fi­ca­cy with less tox­i­c­i­ty from treat­ment.”

That’s an in­trigu­ing piece of the puz­zle for Pfiz­er $PFE, which agreed to pay $14 bil­lion for Medi­va­tion to get its hands on Xtan­di as well as the late-stage PARP drug. These drugs work by block­ing a DNA re­pair path­way and there has been a con­sid­er­able amount of re­search demon­strat­ing their ef­fec­tive­ness as well as a rel­a­tive­ly good safe­ty pro­file.

The big show this week­end, though, will be re­served for Tesaro $TSRO and Clo­vis On­col­o­gy $CLVS, which both have close­ly-watched PARPs lin­ing up for po­ten­tial ap­provals. For Clo­vis, ru­ca­parib presents its first re­al op­por­tu­ni­ty to gain a cru­cial­ly im­por­tant FDA ap­proval. And Tesaro has been fu­el­ing high hopes with its own late-stage da­ta set. Share prices on both com­pa­nies have been soar­ing over the last few months.

We’ll keep you post­ed on the re­sults.

An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Can an FDA hus­tle up on trastuzum­ab be far be­hind?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Mer­ck Serono’s Se­nior Vice Pres­i­dent and Glob­al Head of On­col­o­gy
EL­LIOTT LEVY — Am­gen’s Se­nior Vice Pres­i­dent of Glob­al De­vel­op­ment
CHRIS BOSHOFF — Pfiz­er On­col­o­gy’s Chief De­vel­op­ment Of­fi­cer

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Ver­sant-backed Chi­nook gets a $65M launch round for its dis­cov­ery quest in a resur­gent kid­ney field

Versant is once again stepping off the beaten track in biotech to see if they can blaze a trail of their own in a field that has looked too thorny to many investors for years.

The venture group and their partners at Apple Tree are bringing their latest creation out of stealth mode today. Born in Versant’s Inception Sciences’ Chinook Therapeutics is betting that its preclinical take on kidney disease can get an early lead among the companies starting up in the field.

Sir An­drew Dil­lon, NICE's first — and on­ly — chief ex­ec­u­tive to step down next year

Using a laptop borrowed from his former employer, South London’s St George’s Hospital, Sir Andrew Dillon set about establishing NICE — launched by the then health secretary Frank Dobson — in 1999.  On Thursday, the UK cost-effectiveness watchdog said its first and only chief executive — Dillon — is stepping down in March 2020.

Back in the day, decisions about which drugs and interventions were funded by the National Health Service (NHS) were made at the local level, but this ‘postcode prescribing’ system was fraught with skewed healthcare deployment making the structure unsustainable. A national system was deemed necessary — and NICE was formed to bridge that gap.

Eight weeks be­tween each HIV treat­ment? GSK notch­es PhI­II win as it chas­es OK for long-act­ing reg­i­men

GSK has cleared another test in its grand plan to topple Gilead’s HIV dominance by offering alternative treatments that consist of fewer drugs and last longer. A year after scoring positive Phase III data on a four-week course of cabotegravir and rilpivirine, its ViiV subsidiary now says that an eight-week regimen seem to work just as well.

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Finch grabs a $53M round de­signed to take their ‘break­through’ mi­cro­bio­me treat­ment through a po­ten­tial­ly piv­otal tri­al

With a breakthrough designation in one hand and a fresh $53 million in venture backing in the other, Somerville, MA-based Finch Therapeutics is taking a shot at a one-trial pathway to a possible FDA OK for their new treatment for preventing recurrent C. difficile infections.

The funding brings their total raise for the microbiome company to $130 million, CEO Mark Smith tells me — enough money to pave a runway past the FDA approval they’ve sketched into the most optimistic version for their near-term future. 

Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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