Bio­Marin says PhI­II gene ther­a­py study par­tic­i­pan­t's can­cer is un­re­lat­ed to ther­a­py

As the FDA has de­layed any hope of Bio­Marin launch­ing its he­mo­phil­ia A gene ther­a­py un­til next year at the ear­li­est, the com­pa­ny said in an SEC fil­ing Mon­day that a par­tic­i­pant in one of its Phase III stud­ies for the ther­a­py was di­ag­nosed with a type of can­cer known as B-cell acute lym­phoblas­tic leukemia.

Af­ter sub­mit­ting the safe­ty re­port to FDA, Bio­Marin said it “be­lieves at this time that this can­cer is un­re­lat­ed” to the treat­ment, known as val­oc­toco­gene rox­a­parvovec. None of the gene ther­a­py’s on­go­ing tri­als have been halt­ed as a re­sult, the com­pa­ny said. On the sta­tus of its in­ves­ti­ga­tion in­to this case of B-ALL, Bio­Marin told in­vestors:

The pre­lim­i­nary re­sults of ge­net­ic test­ing per­formed at the site re­vealed that 85% of leukemic cells car­ried a Philadel­phia-like (Ph-like) chro­mo­so­mal translo­ca­tion, which is a well-known dri­ver-mu­ta­tion in leukemic cells. Fur­ther, test­ing on leukemic cells en­riched to 90% pu­ri­ty from pe­riph­er­al blood car­ried neg­li­gi­ble lev­els of BMN 270 vec­tor DNA (less than 1 copy per 500 cells). These neg­li­gi­ble, near back­ground lev­els of BMN 270 in­di­cate that BMN 270 is not clon­al­ly ex­pand­ing in these leukemic cells. Ad­di­tion­al ge­nom­ic analy­ses are un­der­way, which Bio­Marin ex­pects to con­firm the ab­sence of BMN 270 vec­tor in­te­gra­tion events con­tribut­ing to leukemic growth, as well as to pro­vide fur­ther in­sights in­to the un­der­ly­ing ge­net­ic eti­ol­o­gy of this case.

The an­nounce­ment comes as Eu­rope re­cent­ly signed off on the new gene ther­a­py, known com­mer­cial­ly as Roc­ta­vian, based on the re­sults of a Phase III sin­gle-arm, non-ran­dom­ized study of 134 male pa­tients with he­mo­phil­ia A with­out a his­to­ry of fac­tor VI­II in­hibitor and with­out de­tectable pre-ex­ist­ing an­ti­bod­ies to AAV5.

“Two years af­ter the ad­min­is­tra­tion, ef­fi­ca­cy da­ta showed that the ther­a­py sig­nif­i­cant­ly in­creased fac­tor VI­II ac­tiv­i­ty lev­els in the ma­jor­i­ty of pa­tients. Bleed­ing rates were re­duced by 85% and most pa­tients (128) no longer need­ed fac­tor VI­II re­place­ment ther­a­py,” EMA said.

In the US, the FDA is­sued a CRL for the ther­a­py in 2020, seek­ing two years of fol­low-up safe­ty and ef­fi­ca­cy da­ta on all study par­tic­i­pants. Since the CRL, the FDA has grant­ed a re­gen­er­a­tive med­i­cine ad­vanced ther­a­py des­ig­na­tion to val­oc­toco­gene rox­a­parvovec, which us­es an AAV vec­tor to de­liv­er the gene for fac­tor VI­II.

But FDA in May al­so passed along more in­for­ma­tion re­quests, and Bio­Marin said its BLA re­sub­mis­sion at the time was ex­pect­ed by the end of this month, which is a time­line the com­pa­ny said it still ex­pects to meet.

Biotech in­vestors and CEOs see two paths to growth, but are they equal­ly vi­able?

The dynamic in the biotech market has been highly volatile in the last few years, from the high peaks immediately after the COVID vaccine in 2021, to the lowest downturns of the last 20 years in 2022. This uncertainty makes calling the exact timing of the market’s turn something of a fool’s errand, according to Dr. Chen Yu, Founder and Managing Partner of TCG Crossover (TCG X). He speaks with RBC’s Noël Brown, Head of US Biotechnology Investment Banking, about the market’s road ahead and two possible paths for growth.

Dave Marek, Myovant CEO

My­ovant board balks as ma­jor­i­ty own­er Sum­it­o­mo swoops in with a $2.5B deal to buy them out

Three years after Sumitomo scooped up Roivant’s 46% stake in the publicly traded Myovant $MYOV as part of a 5-company, $3 billion deal, they’re coming back for the whole thing.

But these other investors at Myovant want more than what the Japanese pharma company is currently offering to pay at this stage.

Sumitomo is bidding $22.75 a share for the outstanding stock, which now represents 48% of the company after Sumitomo bumped its ownership since the original deal with Roivant. Myovant, however, created a special committee on the board, and they’re shaking their heads over the offer.

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Casey McPherson shows his daughters Rose (left) and Weston around Everlum Bio, a lab that he co-founded to spark a treatment for Rose and others with ultra-rare conditions. (Ilana Panich-Linsman)

Fa­ther starts lab af­ter in­tel­lec­tu­al prop­er­ty is­sues stymie rare dis­ease drug de­vel­op­ment

Under bright lab lights, Casey McPherson holds his 6-year-old daughter, Rose. His free hand directs Rose’s gaze toward a computer screen with potential clues in treating her one-of-a kind genetic condition.

Gray specks on the screen show her cells that scientists reprogrammed with the goal of zeroing in on a custom medicine. McPherson co-founded the lab, Everlum Bio, to spark a treatment for Rose — and others like her. A regarded singer-songwriter, McPherson never imagined going into drug development.

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Vlad Coric, Biohaven CEO

Vlad Coric charts course for new Bio­haven with neu­ro­science push and Big Phar­ma vets on board

What’s Biohaven without its CGRP portfolio? That’s what CEO Vlad Coric is tasked with deciding as he maps out the new Biohaven post-Pfizer takeover.

Pfizer officially scooped up Biohaven’s CGRP assets on Monday, including blockbuster migraine drug Nurtec and the investigational zavegepant, for $11.6 billion. As a result, Coric spun the broader pipeline into an independent company on Tuesday — with the same R&D team behind Nurtec but about 1,000 fewer staffers and a renewed focus on neuroscience and rare disease.

In AstraZeneca's latest campaign, wild eosinophils called Phils personify the acting up often seen in uncontrolled asthma

As­traZeneca de­buts an­noy­ing pur­ple ‘Phil’ crea­tures, per­son­i­fied asth­ma eosinophils ‘be­hav­ing bad­ly’

There are some odd-looking purple creatures lurking around the halls of AstraZenca lately. The “Phil” character cutouts are purple, personified eosinophils with big buggy eyes and wide mouths, and they’re a part of AZ’s newest awareness effort to help people understand eosinophilic asthma.

The “Asthma Behaving Badly” characters aren’t only on the walls at AZ to show the new campaign to employees, however. The “Phils” are also showing up online on the campaign website, and in digital and social ads and posts on Facebook and Instagram.

Kite Phar­ma gets FDA to sign off on new Cal­i­for­nia-based vec­tor man­u­fac­tur­ing fa­cil­i­ty

Kite Pharma just got FDA approval to kick off operations at a new manufacturing campus.

The cancer-focused, CAR-T cell therapy player made the announcement Monday, saying that the federal regulatory agency gave the green light to Kite’s 100,000 square-foot, retroviral vector manufacturing facility in Oceanside, CA.

Kite’s global head of technical operations Chris McDonald tells Endpoints News that the facility has been in the works for about four years, after Kite teamed up with its parent company Gilead. Gilead acquired Kite Pharma for just shy of $12 billion in 2017.

Big Phar­ma heavy­weights seek tweaks to FDA's clin­i­cal out­come as­sess­ment guid­ance

Pfizer, GSK, Janssen, Regeneron, Boehringer Ingelheim and at least a half dozen other companies are calling on the FDA to provide significantly more clarity in its draft guidance from this summer on clinical outcome assessments, which are a type of patient experience.

The draft is the third in a series of four patient-focused drug development guidance documents that the FDA had to create as part of the 21st Century Cures Act, and they describe how stakeholders (patients, caregivers, researchers, medical product developers and others) can collect and submit patient experience data and other relevant information for medical product development and regulatory decision-making.

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Robert Califf, FDA commissioner (via AP Images)

User fees in ac­tion: FDA un­veils new short­ened sup­ple­ment re­view, rare dis­ease pi­lots

Thanks to PDUFA VII, signed into law last Friday by President Joe Biden, the FDA this week unveiled two new industry-friendly pilot programs to advance new rare disease endpoints via additional meetings, and to shorten FDA review times for supplemental apps aimed at unmet medical needs.

The agency this week released eagerly-awaited details behind the shortened pilot, known as the Split Real Time Application Review or STAR pilot program, which will speed up certain FDA reviews of efficacy supplements across all therapeutic areas (thanks to earlier submissions of data), but only for those that propose addressing an unmet medical need.

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Mar­ket­ingRx roundup: No­var­tis re­cruits NFL coach for Leqvio cam­paign; Pfiz­er pro­motes ‘Sci­ence’ merch on so­cial me­dia

Novartis is turning to a winning coach to talk about Leqvio and the struggles of high cholesterol — including his own. Bruce Arians, the retired NFL head coach of the Arizona Cardinals and Super Bowl-winning Tampa Bay Buccaneers, is partnering with the pharma for its “Coaching Cholesterol” digital, social and public relations effort.

In the campaign, Arians talks about the potential for “great comebacks” in football and heart health. Once nicknamed a “quarterback whisperer,” he is now retired from fulltime coaching (although still a front-office consultant for Tampa Bay), and did a round of media interviews for Novartis, including one with People and Forbes.

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