Jean-Jacques Bienaimé. BioMarin via YouTube

Bil­lions on the line: Bio­Marin bus­tles past en­thused ri­val, plan­ning to launch world’s first he­mo­phil­ia A gene ther­a­py

Bio­Marin isn’t go­ing to let a ri­val’s suc­cess over the week­end get in the way of mak­ing a bee-line to the FDA with an im­per­fect, but pre­sentable, gene ther­a­py for he­mo­phil­ia A.

On Mon­day the biotech $BM­RN re­port­ed that its talks with the FDA and the EMA con­vinced ex­ecs to make a leap for an ac­cel­er­at­ed ap­proval with da­ta on just a hand­ful of pa­tients. And they’re hand­ing over Phase I/II and in­ter­im Phase III re­sults in Q4 to make their case.

That de­ci­sion was made de­spite a con­sid­er­able back­lash against the lat­est snap­shot of the da­ta, with wors­en­ing ef­fi­ca­cy over time and a low­er ini­tial re­sponse in their piv­otal tri­al — but ex­cel­lent bleed rates, so far. Bio­Marin says that with a break­through drug des­ig­na­tion in the USA and Eu­rope’s prime sta­tus, they have good rea­son to be­lieve they are on track to field the world’s first gene ther­a­py.

In­vestors ap­pear some­what skep­ti­cal though, with Bio­Marin shares down 2.4% in Mon­day af­ter­noon trad­ing.

Here’s the bet they’re mak­ing, out­lined in their lat­est state­ment:

Fac­tor VI­II lev­els sus­tained over a three-year pe­ri­od were suf­fi­cient to achieve strik­ing he­mo­sta­t­ic ef­fi­ca­cy. Fac­tor VI­II ex­pres­sion has en­tered a plateau phase where the rate of de­cline has sub­stan­tial­ly slowed, which could be in­dica­tive of durable, long-term ex­pres­sion.

Leerink’s Joseph Schwartz of­fers a bull­ish as­sess­ment of Bio­Marin’s gam­ble:

Based on BM­RN’s Ph.3 da­ta, the com­pa­ny met their pre-spec­i­fied cri­te­ria for ac­cel­er­at­ed ap­proval – 8 pts achiev­ing mean fac­tor VI­II/FVI­II lev­els above 40 IU/dL. With on­ly 17 pts (out of 20 pts) eval­u­at­ed so far, it is pos­si­ble that the re­main­ing 3 could pro­vide more con­fi­dence in Val­rox al­though it is not re­quired for ac­cel­er­at­ed ap­proval since BM­RN has al­ready met the bar. The com­pa­ny is fo­cused on get­ting Val­rox to mar­ket as soon as pos­si­ble by sub­mit­ting their fil­ing in 4Q19 based on their Ph.3 in­ter­im analy­sis and aims to com­plete GEN­Er8-1 (6E13 vg/kg) en­roll­ment in ear­ly fall 2019. As­sum­ing BM­RN could in­crease and op­ti­mize ef­fi­ca­cy — re­call, chro­mogenic mea­sures were high­er in Ph.1/2 at weeks 23-26 than Ph.3 (68 IU/dL mean/57 IU/dL me­di­an in Ph.1/2 vs 36 IU/dL mean/33 IU/dL me­di­an Ph.3) — we be­lieve that po­ten­tial stud­ies BM­RN may con­duct lat­er could fur­ther boost sales and in­crease Val­rox’s at­trac­tive­ness among he­mo­phil­ia A pts, es­pe­cial­ly as com­pet­ing pro­grams (i.e., SG­MO/PFE) ap­pear to be de­liv­er­ing com­pet­i­tive ef­fi­ca­cy.

Spark Ther­a­peu­tics $ONCE is wide­ly viewed as the run­ner up for now, while Roche strug­gles to com­plete its $4.3 bil­lion buy­out as soon as pos­si­ble. But Sang­amo/Pfiz­er gar­nered some care­ful sec­ond looks a few days ago as they post­ed bet­ter ef­fi­ca­cy and a fast re­sponse among 4 evalu­able pa­tients. That team al­so out­lined plans to make a leap for­ward on an ac­cel­er­at­ed path­way as they sought to catch up with the two fron­trun­ners.

Any stum­ble at this point could prove a set­back, but if they stay on track Sang­amo and Pfiz­er may set up a sit­u­a­tion where pa­tients can choose an im­per­fect gene ther­a­py or wait for a bet­ter one. And Bio­Marin has out­lined plans to charge in the range of $2 mil­lion to $3 mil­lion per treat­ment — with the ca­pa­bil­i­ty of earn­ing $10 bil­lion to $15 bil­lion a year — adding stick­er shock for pay­ers to the list of chal­lenges.

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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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.

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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Covid-19 roundup: Left out no longer, No­vavax se­cures largest Warp Speed deal yet: $1.6B

It looks like Novavax won’t be left out of Operation Warp Speed after all.

A month after the Gaithersburg, MD biotech saw its shares tumble when it was left off the first reported list of finalists for the White House’s Covid-19 vaccine accelerator, HHS and the Department of Defense have announced a $1.6 billion deal to scale up their Covid-19 candidate. It is the largest deal HHS has announced yet, eclipsing the $1.2 billion deal the administration reached with AstraZeneca in May.

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Cel­lec­tis slammed af­ter pa­tient dies and FDA slaps a hold on their tri­al for an off-the-shelf CAR-T for mul­ti­ple myelo­ma

Cellectis was slammed after the market close on Monday as the biotech reported that the FDA demanded it hit the brakes on their MELANI-01 trial for their off-the-shelf cell therapy UCARTCS1A after one of the patients in the study died of treatment-related cardiac arrest.

The multiple myeloma patient had previously been treated unsuccessfully with various therapies, noted the biotech, and had been given dose level two (DL2) of their allogeneic CAR-T.

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Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)

A lit­tle biotech slaps back at a 'crim­i­nal' short at­tack, vow­ing to pur­sue a pros­e­cu­tion of their case

As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.

But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.

Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.

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Covid-19 roundup: Teamed up with NIH, Re­gen­eron launch­es PhI­II pre­ven­tion tri­al for an­ti­body cock­tail

As Regeneron moves its antibody cocktail into Phase II/III trials testing REGN-COV2 as a treatment for both hospitalized and non-hospitalized patients with Covid-19, the biotech is also starting a Phase III in the prevention setting.

The National Institute of Allergy and Infectious Diseases — which orchestrated the large, randomized study for remdesivir that produced positive results — will jointly run the study.

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Douglas Love, Annexon CEO (Annexon)

IPO bound? Ac­tu­al­ly, An­nex­on was al­ready prepped and primed to toss its S-1 to Wall Street as in­vestors ral­lied

The Wall Street IPO shuffle generally calls for a little distance between the crossover ante and the Wall Street double, but with the window on the street wide open and biotech sizzling hot, who’s waiting?

The crew at Annexon didn’t leave anyone in suspense for long about their IPO plans. A day after the Bay Area biotech with clinical plans to target neurodegeneration quietly unveiled a $100 million raise, they were back with an S-1 outlining a pitch to double that — or more.

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