Hit by an ex­pect­ed EMA re­jec­tion, Puma plans to ap­peal neg­a­tive opin­ion on ner­a­tinib

Ex­act­ly a month af­ter warn­ing Puma Biotech­nol­o­gy $PBYI of a neg­a­tive trend vote re­gard­ing ner­a­tinib, Eu­ro­pean reg­u­la­tors have come down with a for­mal neg­a­tive opin­ion for the can­cer drug.

Alan Auer­bach

In a brief note, Puma told in­vestors that the CHMP has rec­om­mend­ed the re­fusal of the mar­ket­ing au­tho­riza­tion ap­pli­ca­tion for ner­a­tinib as a treat­ment of ear­ly-stage HER2-pos­i­tive breast can­cer. Giv­en that the Eu­ro­pean Com­mis­sion usu­al­ly fol­lows EMA rec­om­men­da­tions, a red light is like­ly to fol­low.

All hope is not lost, though, said the LA-based biotech:

The CHMP has not­ed that Puma may re­quest a re-ex­am­i­na­tion of the opin­ion and that a let­ter of in­tent to seek re-ex­am­i­na­tion should be sub­mit­ted with­in 15 days of ac­knowl­edge­ment of re­ceipt of the fi­nal opin­ion pack­age. Puma plans to sub­mit this re­quest in ac­cor­dance with this time­line.

We saw the road­block com­ing. The EMA has raised con­cerns over the clin­i­cal rel­e­vance of the da­ta com­ing out of Puma’s PhII and PhI­II stud­ies in ex­tend­ed ad­ju­vant ear­ly-stage HER2-pos­i­tive breast can­cer. Com­bined with the ev­i­dence of of se­vere di­ar­rhea linked to the drug, that led to a neg­a­tive ben­e­fit risk as­sess­ment.

Puma won the all-im­por­tant US ap­proval, though, af­ter agency ex­perts large­ly gave the biotech a pass on safe­ty is­sues, pre­fer­ring to give physi­cians and pa­tients an­oth­er drug to con­sid­er.

Puma re­cent­ly out-li­censed the drug — mar­ket­ed as Ner­l­ynx in the US — to Is­rael’s Medi­son Phar­ma and Chi­na’s CAN­bridge Life Sci­ences. It’s al­so test­ing ner­a­tinib in a bas­ket of tri­als in hopes of swift­ly ex­pand­ing its in­di­ca­tions in the US.

Puma’s stock slid al­most 2% in pre-mar­ket trad­ing.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

Mer­ck helps bankroll new part­ner Themis' game plan to fin­ish the chikun­gun­ya race and be­gin on­colyt­ic virus quest

As Themis gears up for a Phase III trial of its chikungunya vaccine, the Vienna-based biotech has closed out €40 million ($44 million) to foot the clinical and manufacturing bills.

Its heavyweight partners at Merck — which signed a pact around a mysterious “blockbuster indication” last month — jumped into the Series D, led by new investors Farallon Capital and Hadean Ventures. Adjuvant Capital also joined, as did current investors Global Health Investment Fund, aws Gruenderfonds, Omnes Capital, Ventech and Wellington Partners Life Sciences.