CEO Khurem Farooq (Gyroscope)

Gy­ro­scope posts ear­ly win with gene ther­a­py for AMD, giv­ing hope for its im­mune com­ple­ment sys­tem tar­get

Gene ther­a­pies for blind­ness are noth­ing new af­ter Roche’s Spark unit hooked an FDA ap­proval for Lux­tur­na back in 2017. But a UK biotech hop­ing to turn its gene ther­a­py against an over­ac­tive im­mune com­ple­ment sys­tem tied to vi­sion loss is now sport­ing da­ta that could help change the game in AMD.

In a Phase I/II dose es­ca­la­tion tri­al, pa­tients who re­spond­ed to Gy­ro­scope’s gene ther­a­py GT005 saw a 146% in­crease in their vit­re­ous Com­ple­ment Fac­tor I lev­els as well as a de­crease in down­stream bio­mark­ers tied to ge­o­graph­ic at­ro­phy for pa­tients with dry AMD, ac­cord­ing to da­ta un­veiled Fri­day at the vir­tu­al An­gio­gen­e­sis, Ex­u­da­tion, and De­gen­er­a­tion meet­ing.

At an in­ter­im check-in, nine out of 10 pa­tients across four co­hort groups saw in­creased CFI lev­els af­ter re­ceiv­ing the ther­a­py, and eight of those nine main­tained el­e­vat­ed lev­els of CFI af­ter six months. The ear­li­est pa­tient dosed in the open-la­bel study was still show­ing el­e­vat­ed lev­els at 84 weeks, Gy­ro­scope said.

Gy­ro­scope be­lieves rais­ing CFI lev­els can damp­en an over­ac­tive im­mune com­ple­ment sys­tem, which has been con­nect­ed to wors­en­ing at­ro­phy in AMD pa­tients, ac­cord­ing to a re­lease.

GT005 al­so spurred low­er lev­els of pro­teins as­so­ci­at­ed with an over­ac­tive com­ple­ment sys­tem. Pa­tients saw an av­er­age de­crease of 41% in Ba pro­tein lev­els com­pared to base­line be­tween weeks 24 and 56, and an av­er­age de­crease of 42% C3 break­down pro­tein lev­els com­pared to base­line dur­ing the same pe­ri­od.

Na­dia Wa­heed

Ac­cord­ing to Na­dia Wa­heed, Gy­ro­scope’s CMO, see­ing big cuts in bio­mark­ers tied to the com­ple­ment sys­tem could help sup­port the mech­a­nism of ac­tion be­hind GT005 and add weight to a piv­otal tri­al down the road.

“Con­sis­tent­ly we’re hit­ting the tar­gets we want to hit in the down­stream am­pli­fi­ca­tion loop,” she said. “Every­thing so far looks like it’s point­ing in the right di­rec­tion.”

The ther­a­py’s safe­ty pro­file was most­ly clean, al­though Gy­ro­scope did re­port one po­ten­tial­ly treat­ment-re­lat­ed side ef­fect — a case of choroidal neo­vas­cu­lar­iza­tion, a ma­jor cause of vi­sion loss. Wa­heed said an ear­ly in­ves­ti­ga­tion in­di­cat­ed the event was prob­a­bly not re­lat­ed to the ther­a­py and was more like­ly a re­sult of the pa­tient’s wors­en­ing con­di­tion. Choroidal neo­vas­cu­lar­iza­tions are com­mon in pa­tients treat­ed with an­ti-VEGF ther­a­pies, Wa­heed said, due in part to the struc­ture of the mol­e­cule used in treat­ment.

“We don’t think we should see an in­crease (in those events over time), but of course we fol­low it very close­ly,” she said.

In terms of next steps, Wa­heed said the Phase I/II FO­CUS tri­al would fin­ish en­roll­ment by the end of 2021. Mean­while, the com­pa­ny is al­so en­rolling two Phase II ef­fi­ca­cy tri­als for GT005 and Wa­heed said her team is “on track” to get those filled out.

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.

Rob Etherington, Clene CEO

Star­tup's gold nanocrys­tal ALS drug flops a PhII tri­al, a re­minder of the dis­ease's ob­sta­cles de­spite Amy­lyx OK

Despite the FDA approving an ALS drug for the first time in five years last week, the disease continues to fluster researchers, and another biotech is feeling the pain of a mid-stage failure.

Clene Nanomedicine reported early Monday that its ALS program, which uses gold nanocrystals to try to catalyze intracellular reactions, did not achieve its Phase II primary or secondary endpoints. And in a press release, the company noted for the first time that it’s speaking with “potential strategic partners” about the program — language that typically indicates a biotech is preparing to sell off an asset.

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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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Amy West, Novo Nordisk head of US digital innovation and transformation (Illustration: Assistant Editor Kathy Wong for Endpoints News)

Q&A: No­vo Nordisk dig­i­tal in­no­va­tion chief Amy West dis­cuss­es phar­ma pain points and a health­care 'easy but­ton’

Amy West joined Novo Nordisk more than a decade ago to oversee marketing strategies and campaigns for its US diabetes portfolio. However, her career path shifted into digital, and she hasn’t looked back. West went from leading Novo’s first digital health strategy in the US to now heading up digital innovation and transformation.

She’s currently leading the charge at Novo Nordisk to not only go beyond the pill with digital marketing and health tech, but also test, pilot and develop groundbreaking new strategies needed in today’s consumerized healthcare world.

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Astel­las, Pan­th­er­na add or­gan to mR­NA tie-up; Rock­et launch­es sale of six fig­ures worth of stock

Astellas and Pantherna have expanded their November 2021 pact surrounding the latter’s mRNA platform to include a new target organ, the duo announced Tuesday morning, though they did not specify what that target is.

German biotech Pantherna is home to two platform technologies — one that designs mRNAs for non-vaccine therapies and another that designs LNPs. Astellas and Pantherna’s deal appears to mainly revolve around the first platform, which Astellas said it is using to research direct reprogramming, or turning cells from one kind into another without an intermediate stem cell phase.