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.

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

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Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

A boom­ing Drag­on­fly is tak­ing its TriN­KETs to Copen­hagen as the lat­est Bris­tol My­ers pact spurs ex­pan­sion plans — out­side the US

Bristol Myers Squibb is making a habit out of collaborating with the crew at Dragonfly, adding their 3rd deal in a series that now will take them into newly charted R&D territory. And the fast-growing team at the Cambridge-based biotech is adding a facility in Copenhagen for its next growth spurt, where the government is making it easy to recruit scientists internationally as the U.S. throttles back.

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RA Cap­i­tal dou­bles down on Sid­dhartha Mukher­jee's vi­sion for a new cell en­gi­neer­ing ap­proach, lead­ing Vor's $110M Se­ries B

Vor Biopharma is muscling up.

CEO Robert Ang, who was reluctant to divulge the headcount when discussing his move from Neon Therapeutics to Vor last August, readily offered that the team has grown from 6 to 50 in less than a year. The biotech is moving to a larger office on Cambridge Parkway Drive in weeks, giving it more space to complete the IND-enabling work and manufacturing scale-up — conducted by a CDMO partner — in preparation for clinical trials planned for the first half of 2021.

Covid-19 roundup: Squab­bles with gov­ern­ment de­lay Mod­er­na’s PhI­II — re­ports; No­vavax se­cures largest Warp Speed deal yet: $1.6B

A much-anticipated Phase III trial for Moderna’s Covid-19 vaccine is being held up as the company delayed submitting trial protocols and sparred with government scientists on how to run the study and even what the benchmark for success should be, Reuters reported.

Moderna, the first US company to put their vaccine into human testing, was supposed to enter a 30,000-person study this month in partnership with the NIH to determine whether it can prevent infection. STAT reported last week that the trial was facing delays over the protocol, but that a July start was still possible. Neither the NIH nor Moderna ever disclosed a specific date the trial should start, but Reuters reported that the agency had hoped to begin on July 10.

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Boehringer In­gel­heim ties the knot with Num­ab on new an­ti­bod­ies; Ca­balet­ta inks pact with Ar­ti­san

→ Switzerland’s Numab Therapeutics has added Boehringer Ingelheim to its roster of collaborators. And they will start with two projects aiming at developing new drugs for difficult-to-treat lung and gastrointestinal cancers and patients with geographic atrophy. “Numab’s technology platform fits well with our internal antibody discovery and engineering capabilities and will enhance our efforts to deliver transformative antibody-based therapeutics to patients,” said Paige Mahaney, an SVP at Boehringer Ingelheim.

David Hallal

AlloVir tests how much an an­tivi­ral biotech can reap in a pan­dem­ic stock mar­ket

The pandemic stock market has proven fruitful for virtually any type of biotech. Now a 7-year-old cell therapy startup will see how much it can yield for a company that specializes in fighting viruses.

AlloVir, a company that until 2019 largely lived off grant money, has filed for a $100 million IPO to back its line of off-the-shelf, virus-fighting T cells. Although in normal circumstances, $100 million could be a solid return for a biotech that got its first major round of funding only last year, we’ll have to wait to see how much the company ultimately earns. As Covid-19 has sent investor money scurrying to almost anyone in drug development, every single biotech to go public this year has prized above their midpoint or upsized their offering, according to Renaissance Capital, sometimes dramatically so.

Take­da’s post-merg­er deals con­tin­ue as OTC sub­sidiary is pre­pared for sale

Takeda has been burdened under mountains of debt since acquiring Shire for $62 billion 18 months ago, but one of the company’s biggest moves yet to relieve the stress could be on the horizon.

The Japanese pharma is preparing a bidding war for its $3.7 billion over-the-counter subsidiary, a sale that would dwarf previous spinoffs. Any transaction would be the latest in what’s been a long string of sell-offs, as Takeda marches unflinchingly toward its ultimate goal of shedding $10 billion in assets.