No­var­tis drums up EU launch plans as CHMP gives thumbs up to Spark's gene ther­a­py Lux­tur­na

Nine months af­ter the FDA gave its land­mark OK for Spark Ther­a­peu­tics’ trail­blaz­ing gene ther­a­py Lux­tur­na, Eu­ro­pean reg­u­la­tors have sig­naled that they are ready to green­light the vi­sion loss treat­ment, too.

Paul Hud­son

The team at Spark $ONCE isn’t the on­ly ones cel­e­brat­ing the pos­i­tive opin­ion from the Com­mit­tee for Med­i­c­i­nal Prod­ucts for Hu­man Use: No­var­tis, which grabbed ex-US rights to Lux­tur­na ear­li­er this year in a $170 mil­lion deal, will be com­mer­cial­iz­ing the ther­a­py once it’s ap­proved in Eu­rope.

“To­day’s pos­i­tive CHMP opin­ion rep­re­sents a sig­nif­i­cant step in our jour­ney to­ward ad­vanc­ing po­ten­tial­ly life-chang­ing cell and gene ther­a­pies in oph­thal­mol­o­gy,” said Paul Hud­son, CEO of No­var­tis Phar­ma­ceu­ti­cals, in a state­ment. “We look for­ward to work­ing with Spark Ther­a­peu­tics and the EMA to es­tab­lish ac­cess and reimag­ine care for peo­ple in the EU who face the threat of to­tal blind­ness from this in­her­it­ed reti­nal dis­ease.”

Jef­frey Mar­raz­zo

While (un­like in the US) Lux­tur­na won’t be the first gene ther­a­py ap­proved in the EU — Glax­o­SmithK­line and uniQure beat them to it with their ther­a­pies for “bub­ble boy syn­drome” and fa­mil­ial lipopro­tein li­pase de­fi­cien­cy — No­var­tis is hop­ing it would be the first one with a re­al com­mer­cial prospect.

Price would be key here, es­pe­cial­ly as the phar­ma gi­ant faces with a group of sin­gle pay­er op­er­a­tions with con­sid­er­able lever­age over pric­ing.

Some an­a­lysts have al­so ex­pressed con­cerns about the small mar­ket: Lux­tur­na is de­signed as a one-time treat­ment for a rare form of vi­sion loss (which can even­tu­al­ly re­sult in reti­nal blind­ness) caused by mu­ta­tions in the RPE65 mu­ta­tions, which af­fect 1 in 200,000 peo­ple.

In the US, Spark CEO Jeff Mar­raz­zo has painstak­ing­ly laid out a pric­ing mod­el in hopes of con­vinc­ing pay­ers to cov­er the $850,000 pro­ce­dure — $425,000 per eye — for a tiny group of un­der 2,000 po­ten­tial pa­tients, with few­er than 20 new cas­es per year. The biotech booked $6.7 mil­lion in net sales for the first six months of the year, but it’s still too ear­ly to tell how that’s work­ing.

The pos­i­tive CHMP opin­ion is based on a da­ta pack­age that in­clud­ed a to­tal of 43 pa­tients with in­her­it­ed reti­nal dis­ease caused by RPE65 mu­ta­tions. A mar­ket­ing au­tho­riza­tion from the Eu­ro­pean Com­mis­sion is ex­pect­ed to fol­low with­in two months.

Sean Parker, AP

Sean Park­er helps cre­ate a CRISPRed cell ther­a­py 2.0 play — and he’s got a high-pro­file set of lead­ers on the team

You can rack up one more high-profile debut effort in the wave of activity forming around cell therapy 2.0. It’s another appealing Bay Area group that’s attracted some of the top hands in the business to a multi-year effort to create a breakthrough. And they have $85 million in hand to make that first big step to the clinic.

Today it’s Ken Drazan and the team at South San Francisco-based ArsenalBio that are coming from behind the curtain for a public bow, backed by billionaire Sean Parker and a collection of investors that includes Beth Seidenberg’s new venture investment operation based in LA.
Drazan — a J&J Innovation vet with a long record of entrepreneurial endeavors — exited the stage in 2018 when his last mission ended as he stepped aside as president of Grail. It wasn’t long, though, before he was helping out with a business plan for ArsenalBio that revolved around the work of a large group of interconnected scientists supported by the Parker Institute for Cancer Immunology.

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Alice Shaw, Lung Cancer Foundation of America

Top ALK ex­pert and can­cer drug re­searcher Al­ice Shaw bids adieu to acad­e­mia, hel­lo to No­var­tis

Jay Bradner has recruited a marquee oncology drug researcher into the ranks of the Novartis Institutes for BioMedical Research. Alice Shaw is jumping from prestigious posts intertwined through Mass General, Harvard and Dana-Farber to take the lead of NIBR’s translational clinical oncology group.

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Flu Virus (Source: CDC)

FDA ex­pands Xofluza ap­proval as Roche strug­gles to catch loom­ing flu mar­ket

As a potentially powerful flu season looms, so does a big test for Roche and its new flu drug, Xofluza. The Swiss giant just got a small boost in advance of that test as the FDA expanded Xofluza’s indication to include patients at high risk of developing flu-related complications.

Xofluza (baloxavir marboxil) was approved last October in the US, the first landmark flu drug approval in 20 years and a much-needed green light for a company that had watched its leading flu drug Tamiflu get eaten alive by generics. Like its predecessor, the pill offered a reduction in flu symptoms but not a cure.

EMA backs sev­en ther­a­pies, in­clud­ing Mer­ck­'s Ebo­la vac­cine

The first-ever Ebola vaccine is on the precipice of approval after the European Medicine’s Agency (EMA) backed the Merck product in this week’s roster of recommendations.

The drugmaker $MRK began developing the vaccine, christened Ervebo, during the West African outbreak that occurred between 2014 and 2016, killing more than 11,000.

The current outbreak in the Democratic Republic of Congo (DRC) has shown case fatality rates of approximately 67%, the agency estimated. Earlier this year, the WHO declared the outbreak — which so far has infected more than 3,000 people — a public health emergency of international concern.

Ronald Herb­st fol­lows Med­Im­mune ex­o­dus to Pyx­is CSO post; Jeff God­dard to suc­ceed CEO of AIT Bio­science

→ The outflow of top execs from MedImmune continues to fill the leadership ranks of smaller biotechs. The latest to take off is Ronald Herbst, the head of oncology research, who’s assuming the CSO post at Pyxis Oncology.  

Herbst was part of the old MedImmune organization AstraZeneca CEO Pascal Soriot restructured earlier this year, reorganizing the company and eliminating the storied subsidiary as a separate organization.

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Mer­ck is tak­ing the ax to its US op­er­a­tions, cut­ting 500 jobs in its lat­est re­or­ga­ni­za­tion

Merck is cutting 500 jobs in its US sales and headquarters commercial teams in its latest effort to find new ways to streamline the operation.

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Mi­rati preps its first look at their KRAS G12C con­tender, and they have to clear a high bar for suc­cess

If you’re a big KRAS G12C fan, mark your calendars for October 28 at 4:20 pm EDT.

That’s when Mirati $MRTX will unveil its first peek at the early clinical data available on MRTX849 in presentations at the AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics in Boston.

Mirati has been experiencing the full effect of a rival’s initial success at targeting the G12C pocket found on KRAS, offering the biotech some support on the concept they’re after — and biotech fans a race to the top. Amgen made a big splash with its first positive snapshot on lung cancer, but deflated sky-high expectations as it proved harder to find similar benefits in other types of cancers.

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The FDA will hus­tle up an ex­pe­dit­ed re­view for As­traZeneca’s next shot at a block­buster can­cer drug fran­chise

AstraZeneca paid a hefty price to partner with Daiichi Sankyo on their experimental antibody drug conjugate for HER2 positive breast cancer. And they’ve been rewarded with a fast ride through the FDA, with a straight shot at creating another blockbuster oncology franchise.

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Med­ical an­i­ma­tion: Mak­ing it eas­i­er for the site and the pa­tient to un­der­stand

Medical animation has in recent years become an increasingly important tool for conveying niche information to a varied audience, particularly to those audiences without expertise in the specialist area. Science programmes today, for example, have moved from the piece-to-camera of the university professor explaining how a complex disease mechanism works, to actually showing the viewer first-hand what it might look like to shrink ourselves down to the size of an ant’s foot, and travel inside the human body to witness these processes in action. Effectively communicating a complex disease pathophysiology, or the novel mechanism of action of a new drug, can be complex. This is especially difficult when the audience domain knowledge is limited or non-existent. Medical animation can help with this communication challenge in several ways.
Improved accessibility to visualisation
Visualisation is a core component of our ability to understand a concept. Ask 10 people to visualise an apple, and each will come up with a slightly different image, some apples smaller than others, some more round, some with bites taken. Acceptable, you say, we can move on to the next part of the story. Now ask 10 people to visualise how HIV’s capsid protein gets arranged into the hexamers and pentamers that form the viral capsid that holds HIV’s genetic material. This request may pose a challenge even to someone with some virology knowledge, and it is that inability to effectively visualise what is going on that holds us back from fully understanding the rest of the story. So how does medical animation help us to overcome this visualisation challenge?