No­var­tis' late-stage block­buster push gets ma­jor boost with an­oth­er 'break­through' for MET ther­a­py

No­var­tis has just re­ceived a ma­jor boost in its race with Pfiz­er to de­vel­op a ther­a­py for a no­to­ri­ous­ly treat­ment-re­sis­tant can­cer and get one of its pro­ject­ed 2020 block­buster drugs to mar­ket.

The FDA has ex­pand­ed No­var­tis’ “break­through ther­a­py” des­ig­na­tion for cap­ma­tinib (INC280) to treat a par­tic­u­lar­ly ag­gres­sive form of non-small cell lung can­cer, MET ex­on14 skip­ping-mu­tat­ed NSCLC. Pfiz­er achieved break­through ther­a­py des­ig­na­tion to treat the same dis­ease with Xalko­ri (crizo­tinib), which is al­ready in use to treat less-re­sis­tant forms of NSCLC, in March of 2018. No known treat­ment is cur­rent­ly ap­proved for the ail­ment.

No­var­tis’ des­ig­na­tion, how­ev­er, is now broad­er than Pfiz­er’s, cov­er­ing both treat­ment-naive pa­tients and those al­ready treat­ed with plat­inum-cell chemother­a­py. Pfiz­er’s was on­ly for those al­ready treat­ed with plat­inum-lev­el chemother­a­py, as of their 2018 an­nounce­ment. Cap­ma­tinib has al­so at­tained or­phan drug sta­tus.

Orig­i­nal­ly dis­cov­ered by In­cyte and li­censed to No­var­tis in 2009, cap­ma­tinib is an oral­ly bioavail­able in­hibitor that works by bind­ing to the c-Met cells that are al­tered and dri­ve tu­mor growth in a small num­ber – around 5%, per the NIH – of NSCLC can­cer pa­tients. It is one of sev­er­al in­hibitors that has gained trac­tion in re­cent years as a vi­able treat­ment for this ther­a­py-re­sis­tant form of lung can­cer, in­clud­ing Mi­rati Ther­a­peu­tics’s gle­sa­tinib and Mer­ck KGaA’s tepo­tinib.

No­var­tis CEO Vas Narasimhan in­clud­ed the ther­a­py as one of sev­en po­ten­tial block­busters the com­pa­ny was hop­ing to bring on to the mar­ket in 2020. More broad­ly, the com­pa­ny has talked up its gene and cell ther­a­pies, pro­ject­ing this week that they will ac­count for 15-20% of its sales in the midterm, or near­ly $50 bil­lion per year – up from the pen­nies they cur­rent­ly ac­count for.

The rise has al­ready be­gun with the blood can­cer ther­a­py Kym­ri­ah and the con­gen­i­tal blind­ness treat­ment Lux­tur­na, both of which run in­to the hun­dreds of thou­sands of dol­lars in price per pa­tient.

Al­ready this year, one of its pro­ject­ed block­busters, MS drug Arz­er­ra, cleared two tri­als to put it on pace for a 2020 launch, seiz­ing head­lines from the flop of its star car­diac drug En­tresto.

If No­var­tis launch­es cap­ma­tinib, In­cyte stands to earn over half a bil­lion dol­lars in mile­stone pay­ments and roy­al­ties.

Break­through sta­tus came af­ter da­ta from No­var­tis’ in­ter­na­tion­al, 97-per­son GEOM­E­TRY mono-1 study showed what the com­pa­ny called “promis­ing” re­sults. Da­ta showed a 68% re­sponse rate from treat­ment-naive pa­tients and 40.6% from those pre­vi­ous­ly treat­ed. Sev­en of 13 pa­tients al­so saw in­tracra­nial ac­tiv­i­ty, in­clud­ing in­stances of com­plete brain le­sion heal­ing.

Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

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Covid-19 roundup: No­vavax inks vac­cine deals with Japan and In­dia; As­traZeneca near­ing agree­ments with Japan and Brazil

Following the release this week of promising early data for their Covid-19 vaccine candidate, Novavax has announced collaborations to supply it to two countries — Japan and India.

The Maryland-based biotech announced a deal Friday morning with Takeda to develop and manufacture up to 250 million doses per year of its adjuvanted vaccine. And late Thursday afternoon, Novavax entered into an agreement with the Serum Institute of India to provide up to 1 billion doses to India and low- and middle-income countries.

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In sur­pris­ing set­back, com­bo of Roche’s Tecen­triq and chemo fails to help pa­tients with triple-neg­a­tive breast can­cer

Roche broke ground last year when they secured the first FDA approval for a checkpoint therapy in triple-negative breast cancer, a notoriously difficult-to-treat indication that has been passed over by the wave of targeted therapies.

Now, though, doctors are puzzling over why a combination of drugs meant to make that therapy more potent instead appeared to make it less effective.

Roche said Thursday that in a Phase III trial, combining their PD-1/L1 checkpoint therapy Tecentriq with the chemotherapy paclitaxel, did not significantly improve progression-free survival for patients with locally advanced or metastatic triple-negative breast cancer over giving those patients chemotherapy alone. In fact, patients on the Tecentriq-chemo arm had lower overall survival than patients on chemo, although the drugmaker cautioned that the trial was not powered for that endpoint and the data were immature.

President Trump (AP Images)

FDA takes the lead on defin­ing es­sen­tial un­der Trump's 'Buy Amer­i­can' ex­ec­u­tive or­der — as in­dus­try warns of sup­ply chain dis­rup­tion

President Donald Trump has signed an executive order detailing how the federal government should help on-shore drug manufacturing — and the FDA will play a central role.

The agency now has three months to draw up the list of “essential medicines, medical countermeasures, and their critical inputs” that the US must have available at all times. Various departments and agencies are then directed to buy these drugs and their ingredients from American manufacturers.

Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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Douglas Fambrough, Dicerna CEO (Boehringer Ingelheim via YouTube)

Roche-backed Dicer­na push­es in­to the pack rac­ing to­ward the block­buster hep B goal line, armed with PhI da­ta

Dicerna has lined up a set of proof-of-concept data from a small cohort of hepatitis B patients in a match-up against some heavyweight rivals which got out in front of this race. And right in the front row you’ll find a team from Roche, which paid $200 million in cash and offered another $1.5 billion in milestones to partner with Dicerna $DRNA on their RNAi program for hep B.

Right now it’s looking competitive, with lots of big challenges ahead.

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Lund­beck sounds taps on an­oth­er CNS drug, re­treat­ing from a mine field still oc­cu­pied by a Mer­ck team

Lundbeck has snipped another clinical-stage branch of its CNS research, dumping a schizophrenia program after determining that their therapy would have no positive influence on the disease.

Designed originally as a 240-patient study, researchers set out in early 2019 to see if a homegrown drug dubbed Lu AF11167 could make it through a proof-of-concept study. The drug is a PDE10Ai inhibitor, targeting an enzyme which it said at the time offered a new pathway to retuning the body’s neurotransmitter dopamine. The big idea was that by hitting their target, the drug would modulate “dopamine D1 and D2 receptor-mediated intraneuronal signaling without binding to these receptors,” influencing negative symptoms of schizophrenia.

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Ab­b­Vie aban­dons a pi­o­neer­ing CRISPR R&D al­liance with Ed­i­tas as Brent Saun­der­s' deal is cast out

A little more than 3 years ago Allergan paid $90 million in a cash upfront to partner with gene editing player Editas on a CRISPR alliance focused on the eye. The lead program centered on LCA10, a rare, inherited retinal degenerative disease that appears in childhood and leads to blindness.

Allergan then went to AbbVie $ABBV in a buyout, and the pharma giant has no interest in moving forward on the gene editing front. The company punted it all back to Editas Thursday, with the biotech $EDIT noting in a statement after the market closed Thursday that it is regaining all rights for its ocular medicines, including EDIT-101.