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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

No­vo Nordisk re­mains un­der UK scruti­ny as MHRA con­ducts its own re­view in 'in­cred­i­bly rare' case

The UK’s Medicines and Healthcare products Regulatory Agency is now reviewing Novo Nordisk’s marketing violation that resulted in its loss of UK trade group membership last week. Novo Nordisk was suspended on Thursday from the Association of the British Pharmaceutical Industry (ABPI) for two years after an investigation by its regulatory arm found the pharma broke its conduct rules.

MHRA said on Tuesday that its review of the Prescription Medicines Code of Practice Authority (PMCPA) investigation is standard practice. An MHRA spokesperson emphasized in an email to Endpoints News that the situation with Novo Nordisk is “incredibly rare” while also noting ABPI took “swift and proportionate action.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,200+ biopharma pros reading Endpoints daily — and it's free.

Andy Plump, Takeda R&D chief (Jeff Rumans for Endpoints News)

What kind of PhI­Ib da­ta is worth $4B cash? Take­da’s Andy Plump has some thoughts on that

A few months back, when Takeda caused jaws to drop with its eye-watering $4 billion cash upfront for a mid-stage TYK2 drug from Nimbus, it had already taken a deep dive on the solid Phase IIb data Nimbus had assembled from its dose-ranging study in psoriasis.

Now, it’s rolling that data out, eager to demonstrate what inspired the global biopharma to go long in a neighboring, but new, disease arena for the pipeline. And the most avid students of the numbers will likely be at Bristol Myers Squibb, who will have a multi-year head start on pioneering the TYK2 space with Sotyktu (deucravacitinib) as Takeda makes its lunge for best-in-class status.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,200+ biopharma pros reading Endpoints daily — and it's free.

FDA warns Proc­ter & Gam­ble over NyQuil la­bel's in­gre­di­ent list­ings

The FDA on Tuesday released a warning letter sent earlier this month to the Mason, OH-based site of Procter & Gamble Manufactura, raising questions about the list of ingredients on the label and in the electronic filing.

The warning says that for P&G’s over-the-counter Vicks Nyquil Severe Hot Remedy Cold and Flu Plus Congestion, there’s a “mismatched” list of active ingredients between the labeling and the electronic listing file. The listing file for the active ingredients did not match the active ingredients in the electronic file.

FTC says patent bat­tle over Parkin­son's drug could have 'sig­nif­i­cant im­pli­ca­tion­s' for pa­tients

The Federal Trade Commission has gotten involved in a patent feud over Supernus’ Parkinson’s drug Apokyn, a case the agency said may have ‘‘significant implications” for patients who rely on the drug.

Sage Chemical won the first generic approval for its Apokyn formulation (also known as apomorphine hydrochloride injection) back in 2022. The non-ergoline dopamine agonist is approved to treat Parkinson’s symptoms during “off episodes,” such as difficulty moving, tremors and intense cramping. However, regulators specified that the approval pertained to the generic drug cartridges only, not the injector pen required for administration.

Mar­ket­ingRx roundup: What could a US Tik­Tok ban mean for phar­ma? Pfiz­er, Lil­ly lead phar­ma March Mad­ness ad­ver­tis­ers

Just as pharma marketers finally make moves into TikTok, the threat of a US ban on the social media channel is now looming. Already banned on federal employee phones by an initial Congressional act, more bills and maybe bans are on the way. With rare bipartisan agreement, lawmakers have introduced legislation that would give the US president the power to ban TikTok (although not mentioned by name) and other foreign-owned technology platforms that represent a security threat to the US.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,200+ biopharma pros reading Endpoints daily — and it's free.

Chat­G­PT with phar­ma da­ta de­buts for med­ical meet­ings, be­gin­ning with AACR

What do you get when you combine ChatGPT generative AI technology with specific pharma and clinical datasets? A time-saving tool that can answer questions about medical conference abstracts and clinical findings in seconds in one new application from ZoomRx called FermaGPT.

ZoomRx is debuting a public version of its generative AI product specifically for medical conferences beginning this week for the upcoming American Association for Cancer Research (AACR) annual meeting that runs April 14-19.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,200+ biopharma pros reading Endpoints daily — and it's free.

Vipin Garg, Altimmune CEO

Al­tim­mune’s shares halved af­ter in­ter­im look at PhII weight loss drug da­ta

Altimmune’s attempt to catch up to Novo Nordisk and Eli Lilly’s GLP-1 drugs hit an investor snag Tuesday after the biotech shared interim Phase II weight loss data.

The Maryland biotech’s pemvidutide is a GLP-1/glucagon dual receptor agonist meant to activate GLP-1 receptors to squash appetite and glucagon to ramp up energy use. The 2.4 mg dose showed a placebo-adjusted weight loss of 9.7% at week 24 of 48, which Jefferies analysts said would be comparable to Novo Nordisk’s semaglutide (Wegovy) and Eli Lilly’s tirzepatide (Mounjaro).

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 163,200+ biopharma pros reading Endpoints daily — and it's free.