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.

Forge Bi­o­log­ics’ cGMP Com­pli­ant and Com­mer­cial­ly Vi­able Be­spoke Affin­i­ty Chro­matog­ra­phy Plat­form

Forge Biologics has developed a bespoke affinity chromatography platform approach that factors in unique vector combinations to streamline development timelines and assist our clients in efficiently entering the clinic. By leveraging our experience with natural and novel serotypes and transgene conformations, we are able to accelerate affinity chromatography development by nearly 3-fold. Many downstream purification models are serotype-dependent, demanding unique and time-consuming development strategies for each AAV gene therapy product1. With the increasing demand to propel AAV gene therapies to market, platform purification methods that support commercial-scale manufacturing of high-quality vectors with excellent safety and efficacy profiles are essential.

Mathai Mammen, FogPharma's next CEO

Math­ai Mam­men hands in J&J's R&D keys to lead Greg Ver­dine’s Fog­Phar­ma 

In the early 1990s, Mathai Mammen was a teaching assistant in Greg Verdine’s Science B46 course at Harvard. In June, the former R&D head at Johnson & Johnson will succeed Verdine as CEO, president and chair of FogPharma, the same month the seven-year-old biotech kickstarts its first clinical trial.

After leading R&D at one of the largest drugmakers in the world, taking the company through more than half a dozen drug approvals in the past few years, not to mention a Covid-19 vaccine race, Mammen departed J&J last month and will take the helm of a Cambridge, MA biotech attempting to go after what Verdine calls the “true emperor of all oncogenes” — beta-catenin.

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Sen­ate Fi­nance Com­mit­tee lobs more bi­par­ti­san pres­sure on­to PBMs

Congress is honing in on how it wants to overhaul the rules of the road for pharmacy benefit managers, with a Senate Finance Committee hearing Thursday serving as the latest example of the Hill’s readiness to make changes to how pharma middlemen operate.

While pledging to ensure patients and pharmacies “don’t get a raw deal,” Finance Committee Chair Ron Wyden (D-OR) laid out the beginning of what looks like a major bipartisan effort — moves the PBM industry is likely to challenge vigorously.

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Nicklas Westerholm, Egetis Therapeutics CEO

Ac­qui­si­tion talks on­go­ing for Swedish rare dis­ease biotech Egetis, shares up al­most 40%

Shares of the Sweden-based rare disease biotech Egetis Therapeutics skyrocketed on Thursday afternoon as the company said it’s engaged in “ongoing discussion” with external parties regarding a “potential acquisition.”

Egetis confirmed rumors with a statement on Thursday while noting that there is no certainty that a takeover offer will be made.

Nonetheless, the possibility of an acquisition has shot up Egetis’ share price. By the afternoon on Thursday, its stock price was {$EGTX.ST} up over 38%. An Egetis spokesperson told Endpoints News in an email that it has no further comments.

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Feng Zhang (Susan Walsh/AP Images)

In search of new way to de­liv­er gene ed­i­tors, CRISPR pi­o­neer turns to mol­e­c­u­lar sy­ringes

Bug bacteria are ruthless.

Some soil bacteria have evolved tiny, but deadly injection systems that attach to insect cells, perforate them and release toxins inside — killing a bug in just a few days’ time. Scientists, on the other hand, want to leverage that system to deliver medicines.

In a paper published Wednesday in Nature, MIT CRISPR researcher Feng Zhang and his lab describe how they engineered these syringes made by bacteria to deliver potential therapies like toxins that kill cancer cells and gene editors. With the help of an AI program, they developed syringes that can load proteins of their choice and selectively target human cells.

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J&J bows out of RSV vac­cine race, end­ing PhI­II study and ced­ing to Pfiz­er, GSK

Johnson & Johnson announced Wednesday morning it is ending development of its adult RSV vaccine that was in the middle of a 27,200-patient trial, giving up a big slice of what’s expected to be the next multibillion-dollar pharma market.

The decision came down to the shifting RSV “competitive landscape,” a company spokesperson tells Endpoints News, adding the “breadth of options” was much different than when J&J first started its pivotal study. The spokesperson declined to comment on the Phase III data, saying only the shot is undergoing an “ongoing assessment.”

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No longer ‘dead or just hi­ber­nat­ing,’ drug­mak­ers re­turn to heart med­i­cines

In 2015, now-FDA Commissioner Robert Califf joined industry, academic and regulatory representatives in Washington to discuss why more drugs weren’t in development for cardiovascular diseases, the leading US cause of death and once a mainstay of pharmaceutical industry blockbusters.

The group pointed to many reasons. Clinical trials could take years and testing was expensive. Wide availability of generic drugs made the commercial prospects uncertain. Their paper title summed up the mood: “Cardiovascular Drug Development: Is it Dead or Just Hibernating?”

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Lu­pus drug de­vel­op­ment mar­ket heat­ing up, while FDA links with ad­vo­ca­cy group to fur­ther ac­cel­er­ate re­search

The long-underserved systemic lupus erythematosus (SLE) market is suddenly buzzing with treatment possibilities. Less than two years after AstraZeneca’s approval for Saphnelo — the first new SLE drug in a decade and joining just one other approved in GSK’s Benlysta – the pipeline of potential drugs numbers in the dozens.

Although most are very early stage — Spherix Global Insights estimates five in Phase II/III — the pharma R&D enthusiasm is catching on among doctors, patients and advocacy groups. On Wednesday, the Lupus Research Alliance and the FDA formed a novel private-public partnership called Lupus Accelerating Breakthroughs Consortium (Lupus ABC) to help advance lupus clinical trial success.

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Af­ter safe­ty re­view, EMA mir­rors FDA with up­dat­ed rec­om­men­da­tions for JAK in­hibitors

The EMA released updated recommendations today for the use of JAK inhibitors (JAKi) after reviewing data from several clinical trials that showed increased incidents of issues in certain patients who have rheumatoid arthritis and other risk factors.

The EMA noted malignancy, major adverse cardiovascular events (MACE), serious infections, venous thromboembolism (VTE) and mortality in some patients.