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.

The top 100 bio­phar­ma VCs, Bob Brad­way places $2B bet in can­cer, gene edit­ing pi­o­neer's new big idea, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Before diving in, we had some news to share: Endpoints is launching a premium weekly report focusing on all things regulatory. Coverage will be led by our new senior editor, Zachary Brennan, who joins us from POLITICO. Arsalan Arif has more details in his Publisher’s Note.

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Robert Bradway (Photographer: Scott Eisen/Bloomberg via Getty Images)

UP­DAT­ED: Am­gen snaps up can­cer drug play­er Five Prime, adding PhI­II-ready FGFR2b drug in $2B M&A play

Amgen is making a long-awaited move on the M&A side, buying South San Francisco-based Five Prime $FPRX for close to $2 billion and adding a slate of new cancer drugs to the pipeline.

Amgen is paying $38 a share, putting the deal value at $1.9 billion. The stock closed at $21.26 last night, giving investors a 78% premium.

The jewel in the crown of this deal is bemarituzumab, which Amgen describes as a first-in-class, Phase III-ready anti-FGFR2b antibody. Amgen was drawn to the bargaining table by Five Prime’s mid-stage data on gastric cancer, satisfied by PFS and OS data helping to validate FGFR2b as a target. Amgen researchers will now expand on the R&D program in other epithelial cancers, including lung, breast, ovarian and other cancers.

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David Liu (Casey Atkins Photography courtesy Broad Institute)

David Liu has a new big idea: pro­teome edit­ing. It could one day shred tau, RAS and some of the worst dis­ease-caus­ing pro­teins

Before David Liu became famous for inventing new forms of gene editing, he was known around academia in part for a more obscure innovation: a Rube Goldberg-esque system that uses bacteria-infecting viruses to take one protein and turn it into another.

Since 2011, Liu’s lab has used the system, called PACE, to dream up fantastical new proteins: DNA base editors far more powerful than the original; more versatile forms of the gene editor Cas9; insecticides that kill insecticide-resistant bugs; enzymes that slide synthetic amino acids into living organisms. But they struggled throughout to master one of the most common and powerful proteins in the biological world: proteases, a set of Swiss army knife enzymes that cut, cleave or shred other proteins in everything from viruses to humans.

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The 2021 top 100 bio­phar­ma in­vestors: As the pan­dem­ic hit and IPOs boomed, VCs swung in­to ac­tion like nev­er be­fore

The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights.

Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates.

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Eli Lil­ly claims a TKO in its long-run­ning ti­tle fight with No­vo Nordisk for the block­buster di­a­betes mar­ket — but there’s a hitch

Eli Lilly isn’t just gunning for a better diabetes drug in tirzepatide. They want to cut ahead of Novo Nordisk’s blockbuster rival Ozempic (semaglutide) on the obesity front as well. But a newly-claimed win in a head-to-head Phase III showdown over reducing A1C while shedding pounds — complete with clear evidence of superiority over the approved rival — could prove a tough sell right now.

Let’s start with the latest data from Lilly.

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Hal Barron, Endpoints UKBIO19

GSK, Vir's hopes for a Covid-19 an­ti­body fall flat in NIH 'mas­ter pro­to­col' with no ben­e­fit in hos­pi­tal­ized pa­tients

GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan.

The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday.

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Eli Lil­ly claims suc­cess in a new JAK in­di­ca­tion: hair loss

Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one.

Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms.

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Bruce Cozadd, Jazz CEO (Jazz Pharmaceuticals)

Jazz CEO Bruce Cozadd cam­paigned for 6 months to buy GW Phar­ma. A 90% pre­mi­um sealed the deal — along with $17.6M in ‘re­ten­tion’ in­cen­tives

Jazz CEO Bruce Cozadd didn’t beat around the bush.

In his first video meeting with GW Pharma chief Justin Gover last July 8, he offered to pay $172 a share to get the company, which had beaten the odds in getting its remarkable cannabinoid drug Epidiolex across the regulatory finish line for epilepsy. GW’s stock closed at $129 that day.

Cozadd had already done his homework on the financing to make sure he could swing it the way he wanted. He just needed to do some due diligence before making the non-binding bid firm.

UP­DAT­ED: Not 3 weeks af­ter tak­ing Hu­ma­cyte pub­lic, Ra­jiv Shuk­la launch­es an­oth­er blank check com­pa­ny

One of biotech’s earliest SPAC investors is back with another blank-check company, less than a month after his last effort announced its intent to merge.

Rajiv Shukla is intending to take a third lucky winner public with Alpha Healthcare Acquisition III, filing to go public Thursday with a $150 million raise penciled in. The move comes just a couple of weeks after Shukla’s second SPAC said it would jump to Nasdaq in tandem with Laura Niklason’s Humacyte in a $255 million new investment.

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