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.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.

Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

Steffen Schuster, ITM CEO

Ra­dio­phar­ma re­mains hot as Ger­many's ITM rais­es $109M to ad­vance neu­roen­docrine can­cer pro­gram

The world of radiopharmaceuticals has been heating up over the last few years, and Thursday saw another company focused on the field pull in a new nine-figure raise.

Germany’s ITM, or Isotopen Technologien München, scored a $109 million round of loan financing to push forward its precision oncology pipeline and fund late-stage development for its lead program. As part of the agreement, the loan will convert to shares in the event of future financial or corporate transactions, ITM said.