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 DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

Angela Merkel (AP Photo/Michael Sohn)

Covid-19 roundup: Pfiz­er sub­mits vac­cine for full ap­proval; Merkel op­pos­es Biden pro­pos­al to sus­pend IP for vac­cines

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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UP­DAT­ED: EMA safe­ty com­mit­tee seeks more in­fo on heart in­flam­ma­tion fol­low­ing Pfiz­er Covid-19 vac­cine

The European Medicines Agency’s safety committee said Friday that it’s aware of cases of inflammation of the heart muscle and inflammation of the membrane around the heart, mainly reported following vaccination with Pfizer’s Covid-19 vaccine, known in Europe as Comirnaty.

“There is no indication that these cases are due to the vaccine,” the EMA’s Pharmacovigilance Risk Assessment Committee said.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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In­cyte ponies up $12M to set­tle char­i­ty foun­da­tion kick­back claims; US ex­er­cis­es op­tion for more dos­es of mon­key­pox vac­cine

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.