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What do Mass Gen­er­al, the NCI and Mayo Clin­ic have in com­mon? They rou­tine­ly blow off an FDA rule on post­ing tri­al re­sults

Two years ago, an FDA rule went into effect requiring all clinical trials to report their results on clinicaltrials.gov within a year of completion. It was meant to guard against a publication bias that can leave negative results unpublished and skew the literature and, by extension, our understanding of how and if drugs work.

A team at Ben Goldacre’s Evidence-Based Medicines Data Lab at the University of Oxford just examined how well trial organizers are complying with those rules, and the results, at least for some organizations, are damning.

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Hal Barron and Emma Walmsley, GSK

GSK’s ‘break­through’ BC­MA can­cer drug gets a pri­or­i­ty re­view — and a big win for the on­col­o­gy R&D team

After largely whiffing the past 2 years on the pharma R&D front, GlaxoSmithKline research chief Hal Barron has seized boasting rights to a key win that puts them back in the cancer drug development game.

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John Oyler, Endpoints

BeiGene lines up its first shot at crack­ing the megablock­buster PD-1 mar­ket for lung can­cer. But can they over­come un­der­dog sta­tus?

BeiGene took another big step towards challenging Merck, Bristol-Myers Squibb, AstraZeneca and some other Big Pharma heavyweights for a share of the lucrative lung cancer market for the PD-(L)1s racking up billions in annual revenue.

The China-based biotech $BGNE run by CEO John Oyler posted positive top-line progression-free survival results for their pivotal Chinese study on their PD-1 antibody tislelizumab combined with chemo for squamous non-small cell lung cancer in frontline cases. Squamous NSCLC accounts for about 30% of the overall lung cancer market.

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Sci­en­tists find un­ex­pect­ed an­ti-can­cer ac­tiv­i­ty in range of non-on­col­o­gy drugs — study

As the second leading cause of mortality globally, the lucrative field of cancer treatment has elicited a frenzy of drug development and billions in venture funding. But a new study suggests that cancer-killing compounds may be lurking in the existing arsenal of non-oncology medicines.

By analyzing thousands of FDA-approved drugs and compounds that have been proven safe in clinical trials, scientists at the Broad Institute of MIT and Harvard and Dana-Farber Cancer Institute found nearly 50 compounds — including drugs for diabetes, inflammation, alcoholism and even a treatment for arthritis in dogs — with previously undetected anti-cancer activity.

As­traZeneca nabs pri­or­i­ty re­view for Lyn­parza in prostate as PARP in­hibitors ex­pand to more can­cers

Lynparza keeps rolling.

Three weeks after the FDA cleared the AstraZeneca drug as a first-line treatment of a particular form of advanced pancreatic cancer, the agency has granted priority review for the drug as a second-line treatment of a particular form of advanced prostate cancer. A PDUFA date is set for the second quarter of 2020.

The news is another notch in the belt for an R&D program and an oncology pipeline that was struggling just three years ago. Lynparza, a PARP inhibitor, and Imfinzi, AstraZeneca’s CTLA4 immunotherapy, will each finish 2019 with over $1 billion sales, and they only continue to expand their potential market. Last week, a Lynparza combo got priority review for a form of advanced ovarian cancer and yesterday an Imfinzi combo earned orphan designation for liver cancer.

Rev­o­lu­tion Med shoots for $100M+ IPO — and di­vulges some se­crets about that Warp Dri­ve buy­out

Biotech investors who like to wager on the race to the front of the KRAS market now have a new team to consider.

Revolution Medicines, which extended its reach on RAS with a deal to acquire Warp Drive Bio about 18 months ago, filed their S-1 in search of $100 million-plus. And they gave up a few secrets in the process.

The main clinical claim to fame that Revolution has centered on is the SHP2 inhibitor RMC-4630, partnered with Sanofi back in the summer of 2018 — just after John Reed was named the incoming R&D chief. We already knew that the pharma giant handed over $50 million in cash plus a commitment of hundreds of millions more to align itself with Revolution as it makes a fresh foray into oncology. Now we know that Sanofi is also footing 80% of Revolution’s R&D bill on the program, while setting up a smorgasbord of $235 million in development milestones and $285 million in commercial bonuses.

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Nick Leschly at Endpoints News' panel at the 2020 JP Morgan Healthcare Conference. Credit: Jeff Rumans

At #JPM20, two CEOs, two rad­i­cal­ly dif­fer­ent ther­a­pies, and a fight to chase down sick­le cell

SAN FRANCISCO – Few CEOs tell a story better than bluebird’s Nick Leschly.

He cuts a Jeff Bezos figure on stage at the Colonial Room, the JP Morgan presentation hall for A-list biotechs: lean and bald, fast-talking and vest-wearing. He explains in simple language, apologizing when he has to brush on the data. It helps that he has a good story to tell.

“We treated them one time,” Leschly tells a packed crowd, gesturing to the slide behind him. “Look what happened.”

The slide shows 9 horizontal bars studded with diamonds. Each bar, he explained, represented a sickle cell patient, and each diamond represented a severe medical event, such as a pain crisis. The diamonds stud one side – before the therapy – and vanish on the other, afterward.

“A 99% reduction in these events — this is a functional cure for sickle cell disease,” Leschly says. “This is unprecedented data.”

Upstairs and an hour later, Ted Love stands before a narrow conference room in his suit and polka-dot tie. Love, the CEO of Global Blood Therapeutics, is a 60-year-old physician. His voice trails off at the end of sentences, and the story he tells is less compelling. There are no cured patients.

“This is the first drug that addresses the root cause of sickle cell disease,” Love says, speaking in front of a slide showing a white pill bottle for GBT’s new drug Oxbryta. “Right in the label, it says that this drug inhibits polymerization.”

In the 60 years after scientists discovered the cause of sickle cell, almost no treatments emerged, even as the condition debilitated hundreds of thousands of Americans, most of them black or Hispanic. But the last few years have seen a resurgence of interest as new technologies have made the disease seem newly beatable.

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No­vo Nordisk's block­buster Ozem­pic wins FDA la­bel boost with CV in­di­ca­tion

Novo Nordisk’s blockbuster weekly injectable formulation of semaglutide — Ozempic — has secured an even more formidable label. Apart from its ability to stimulate insulin production in patients with diabetes, the FDA has now also endorsed its ability to reduce the risk of major adverse cardiovascular events.

Patients with diabetes are often afflicted with other comorbidities, such as obesity, CV disease, and kidney problems. Diabetes drugmakers have been clamoring for a broader market share by differentiating their drugs on the basis of therapeutic impact on these intersecting indications — but the major, most lucrative battleground is the heart.

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Hung ju­ry vote leaves FDA with a dif­fi­cult de­ci­sion on Durec­t's non-opi­oid painkiller

A split advisory panel vote on Durect’s non-opioid drug to manage post-surgical pain is only going to make the FDA’s task harder.

After FDA staff reviewers issued an internal review that underscored their unease with the side-effect profile of the long-acting anesthetic posimir — on Thursday a panel of independent experts could not reach a consensus on whether to back the therapy.

Six panelists endorsed approval, while the remaining six dissented. The therapy is a formulation of the anesthetic bupivacaine and is designed to manage pain up to three days following surgery after being administered directly into the surgical incision.

JJ Bienaimé, BioMarin via YouTube

Bio­Marin ex­ecs dis­cuss some eye-pop­ping num­bers for the price of val­rox — but haven't we heard this be­fore?

Robert Baffi BioMarin

SAN FRANCISCO — BioMarin got some considerable attention from the Wall Street Journal today with its revelation that CEO JJ Bienaimé has been thinking about charging $2 million to $3 million for the company’s new gene therapy for hemophilia A, valrox, which is the lead drug in the race for a pioneering FDA approval.

It’s an important discussion, and we’re glad to see it play out in the gray lady of Street speak, which has a big reach. But close observers will note that this isn’t a new number. Bienaime and his executive team discussed that very same range back in February during their Q4 call, which we reported on. Maybe others buzzed about it as well.

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