Khurem Farooq, Gyroscope

With an­oth­er $148M in the bank, Gy­ro­scope Ther­a­peu­tics looks to hus­tle its AMD gene ther­a­py through mid-stage tests

A month and a half lat­er, Gy­ro­scope Ther­a­peu­tics’ Phase I/II win for its dry-AMD gene ther­a­py is al­ready pay­ing off. On Fri­day, the biotech took the wraps off a $148 mil­lion Se­ries C round to ad­vance the can­di­date through two Phase II tri­als in pa­tients with a de­bil­i­tat­ing eye dis­or­der that caus­es grad­ual and per­ma­nent blind­ness.

Syn­cona — which found­ed Gy­ro­scope with Cam­bridge En­ter­prise — con­tributed $42.3 mil­lion to the Se­ries C round, and now main­tains a 54% stake in the Lon­don-based biotech.

Spark’s cost­ly Lux­tur­na made waves when it be­came the first gene ther­a­py ap­proved for in­her­it­ed blind­ness back in 2017. Since then, oth­er gene ther­a­pies for rare eye dis­or­ders have ad­vanced in de­vel­op­ment — but none have been ap­proved for dry, age-re­lat­ed mac­u­lar de­gen­er­a­tion (AMD), a lead­ing cause of blind­ness in peo­ple over 50 years old. About 85% to 90% of peo­ple with AMD have the dry type, ac­cord­ing to Gy­ro­scope.

Gy­ro­scope’s lead can­di­date, GT005, is cur­rent­ly in two Phase IIs for pa­tients with ge­o­graph­ic at­ro­phy, an ir­re­versible de­gen­er­a­tion of reti­nal cells caused by dry-AMD. The can­di­date is in­ject­ed un­der the reti­na to in­crease pro­duc­tion of the Com­ple­ment Fac­tor I pro­tein, which Gy­ro­scope be­lieves could damp­en an over­ac­tive com­ple­ment sys­tem that’s been tied to wors­en­ing at­ro­phy in AMD pa­tients.

“We re­cent­ly an­nounced en­cour­ag­ing Phase I/II clin­i­cal tri­al da­ta with our lead in­ves­ti­ga­tion­al gene ther­a­py, GT005, that give us con­fi­dence in its po­ten­tial as a treat­ment for ge­o­graph­ic at­ro­phy and are con­tin­u­ing to ad­vance our Phase II clin­i­cal pro­gramme,” CEO Khurem Fa­rooq said in a state­ment.

Fa­rooq, a Genen­tech vet, over­saw com­mer­cial ac­tiv­i­ties for the wet-AMD drug Lu­cen­tis and pre-launch work for Roche’s lam­pal­izum­ab, a dry-AMD can­di­date that was dropped af­ter fail­ing two Phase III tri­als in 2017.

In a Phase I/II dose-es­ca­la­tion tri­al, pa­tients who re­spond­ed to 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 re­leased in mid-Feb­ru­ary.

Na­dia Wa­heed

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, Gy­ro­scope said.

“Con­sis­tent­ly we’re hit­ting the tar­gets we want to hit in the down­stream am­pli­fi­ca­tion loop,” CMO Na­dia Wa­heed told End­points News last month. “Every­thing so far looks like it’s point­ing in the right di­rec­tion.”

The Se­ries C funds will al­so be used to ad­vance Gy­ro­scope’s ear­ly-stage pipeline and de­liv­ery tech, ac­cord­ing to a state­ment. The round was led by For­bion’s Growth Op­por­tu­ni­ties Fund, with a hand from Sofinno­va In­vest­ments, funds and ac­counts ad­vised by T. Rowe Price As­so­ci­ates, Tetragon Fi­nan­cial Group Lim­it­ed, an undis­closed health­care-fo­cused fund, Fo­s­un Phar­ma and Cam­bridge In­no­va­tion Cap­i­tal.

Wouter Jous­tra and Ma­ha Katabi, gen­er­al part­ners at For­bion and Sofinno­va, re­spec­tive­ly, are join­ing the board. And di­rec­tor Do­minic Schmidt is on his way out.

“With the ex­pan­sion of the Board it is the right time for Do­minic (Schmidt) to step down from his po­si­tion as Di­rec­tor. We thank Do­minic for his key role in the launch and growth of Gy­ro­scope and his sup­port of the Com­pa­ny,” chief in­vest­ment of­fi­cer Chris Hol­lowood wrote in a state­ment.

Last month, Wa­heed told us the Phase I/II FO­CUS tri­al would fin­ish en­roll­ment by the end of 2021, and that the team was “on track” to fill out en­roll­ment in the two Phase II ef­fi­ca­cy tri­als.

A cor­rec­tion has been made to re­flect that Lux­tur­na is the on­ly oc­u­lar gene ther­a­py cur­rent­ly ap­proved.

What Will it Take to Re­al­ize the Promise and Po­ten­tial of Im­mune Cell Ther­a­pies?

What does it take to get to the finish line with a new cancer therapy – fast? With approvals in place and hundreds of immune cell therapy candidates in the pipeline, the global industry is poised to create a fundamental shift in cancer treatments towards precision medicine. At the same time, unique challenges associated with cell and process complexity present manufacturing bottlenecks that delay speed to market and heighten cost of goods sold (COGS) — these hurdles must be overcome to make precision treatments an option for every cancer patient. This series of articles highlights some of the key manufacturing challenges associated with the production of cell-based cancer therapies as well as the solutions needed to transcend them. Automation, process knowledge, scalability, and assured supply of high-quality starting material and reagents are all critical to realizing the full potential of CAR-based therapies and sustaining the momentum achieved in recent years. The articles will highlight leading-edge technologies that incorporate these features to integrate across workflows, accelerate timelines and reduce COGS – along with how these approaches are enabling the biopharmaceutical industry to cross the finish line faster with new treatment options for patients in need.

The biggest ques­tions fac­ing gene ther­a­py, the XLMTM com­mu­ni­ty, and Astel­las af­ter fourth pa­tient death

After three patients died last year in an Astellas gene therapy trial, the company halted the study and began figuring out how to safely get the program back on track. They would, executives eventually explained, cut the dose by more than half and institute a battery of other measures to try to prevent the same thing from happening again.

Then tragically, Astellas announced this week that the first patient to receive the new regimen had died, just weeks after administration.

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Lat­est news: It’s a no on uni­ver­sal boost­ers; Pa­tient death stuns gene ther­a­py field; In­side Tril­li­um’s $2.3B turn­around; 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.

Next week is shaping up to be a busy one, as our editor-in-chief John Carroll and managing editor Kyle Blankenship lead back-to-back discussions with a great group of experts to discuss the weekend news and trends. John will be spending 30 minutes with Jake Van Naarden, the CEO of Lilly Oncology, and Kyle has a brilliant panel lined up: Harvard’s Cigall Kadoch, Susan Galbraith, the new head of cancer R&D at AstraZeneca, Roy Baynes at Merck, and James Christensen at Mirati. Don’t miss out on the action — sign up here.

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President Biden and Pfizer CEO Albert Bourla (Patrick Semansky/AP Images)

Chaot­ic ad­comm sees Pfiz­er/BioN­Tech boost­ers re­ject­ed for gen­er­al pop­u­la­tion, but rec­om­mend­ed for old­er and high-risk pop­u­la­tions

With just days before President Joe Biden’s Covid-19 booster rollout is set to go into effect, an FDA advisory committee appeared on the verge of not recommending boosters for anyone in the US before a last-minute change of wording laid the groundwork for older adults to have access to a third dose.

The FDA’s adcomm on Vaccines and Related Biological Products (VRBPAC) roundly rejected Pfizer/BioNTech booster shots for all individuals older than 16 by a 16-2 vote Friday afternoon. Soon after, however, the agency posed committee members a new question limiting booster use to the 65-and-older population and individuals at high risk of disease due to occupational exposure or comorbidities.

As­traZeneca, Dai­ichi Sanky­o's ADC En­her­tu blows away Roche's Kad­cy­la in sec­ond-line ad­vanced breast can­cer

AstraZeneca and Japanese drugmaker Daiichi Sankyo think they’ve struck gold with their next-gen ADC drug Enhertu, which has shown some striking data in late-stage breast cancer trials and early solid tumor tests. Getting into earlier patients is now the goal, starting with Enhertu’s complete walkover of a Roche drug in second-line breast cancer revealed Saturday.

Enhertu cut the risk of disease progression or death by a whopping 72% (p=<0.0001) compared with Roche’s ADC Kadcyla in second-line unresectable and/or metastatic HER2-positive breast cancer patients who had previously undergone treatment with a Herceptin-chemo combo, according to interim data from the Phase III DESTINY-Breast03 head-to-head study presented at this weekend’s #ESMO21.

Merck Research Laboratories CMO Roy Baynes

Mer­ck­'s Keytru­da un­corks full da­ta on lat­est ad­ju­vant win — this time in melanoma — adding bricks to ear­ly can­cer wall

In recent months, the battle for PD-(L)1 dominance has spilled over into early cancer with Merck’s Keytruda and Bristol Myers Squibb’s Opdivo all alone on the front lines. Keytruda now has another shell in its bandolier, and it could spell a quick approval.

Keytruda cut the risk of relapse or death by 35% over placebo (p=0.00658) in high-risk, stage 2 melanoma patients who had previously undergone surgery to remove their tumors, according to full data from the Phase III KEYNOTE-716 presented Saturday at #ESMO21.

Mer­ck flesh­es out Keytru­da win in first-line cer­vi­cal can­cer, adding more fire­pow­er to its ear­ly can­cer push

Merck has worked hard to bring its I/O blockbuster Keytruda into earlier and earlier lines of therapy, and now the wonder drug appears poised to make a quick entry into early advanced cervical cancer.

A combination of Keytruda and chemotherapy with or without Roche’s Avastin cut the risk of death by 33% over chemo with or without Avastin (p=<0.001) in first-line patients with persistent, recurrent or metastatic cervical cancer, according to full data from the Phase III KEYNOTE-826 study presented Saturday at #ESMO21.

Skin tu­mors in mice force Pro­tag­o­nist to halt lead pro­gram, crush­ing stock

Protagonist Therapeutics just can’t catch a break.

Six months after the Newark, CA-based biotech unveiled grand plans to launch its lead candidate for blood disorders into a Phase III trial, the FDA has slapped the program with a clinical hold. The halt — which applies to all trials involving the candidate, rusfertide — comes after skin tumors were discovered in mice treated with the drug, according to Protagonist.

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Chi­nese biotech Ever­est signs $550M+ li­cens­ing deal for BTK in­hibitors on heels of Covid-19 pact

Everest Medicines is on a roll with two licensing deals in one week.

The Shanghai-based biotech has paid Sinovent and SinoMab $12 million upfront for the rights to a BTK inhibitor for renal diseases, the company announced Thursday. The deal comes just days after Everest came away with rights to a Covid-19 vaccine in China, Taiwan, Singapore, Thailand and Indonesia.

Everest will pay Sinovent and SinoMab up to $549 million in milestone payments and royalties. The agreement includes tech transfer of Sinovent and SinoMab’s manufacturing process for the candidate, named XNW1011.