Spar­ingVi­sion rais­es $52M to kick off long jour­ney for a next-gen gene ther­a­py that goes much, much broad­er than Lux­tur­na

Un­til Spark Ther­a­peu­tics’ pi­o­neer­ing gene ther­a­py, Lux­tur­na, came along, pa­tients with re­tini­tis pig­men­tosa had few treat­ment op­tions. Even af­ter it was ap­proved, though, the ma­jor­i­ty were left with the ex­act same op­tions.

Stephane Bois­sel

Be­cause it’s tar­get­ing mu­ta­tions in a spe­cif­ic gene known as RPE65, Lux­tur­na can on­ly ad­dress 2 to 3% of the en­tire RP pop­u­la­tion, Stephane Bois­sel told End­points News.

Bois­sel is the new­ly-mint­ed CEO of Spar­ingVi­sion, a French biotech co-found­ed by José-Alain Sa­hel and Thier­ry Léveil­lard at the In­sti­tut de la Vi­sion. They have their sight set on a new kind of AAV con­struct — a next-gen­er­a­tion gene ther­a­py if you will — that can treat all pa­tients of RP in­de­pen­dent of ge­net­ic mu­ta­tions.

And they have $52 mil­lion-plus (€44.5 mil­lion) to re­al­ize that vi­sion steadi­ly over the next four or five years, with plans to com­plete both first- and sec­ond-in-hu­man tri­als.

What the found­ing sci­en­tists pin­point­ed, in­stead of a gene, was a neu­rotroph­ic fac­tor se­cret­ed by rod pho­tore­cep­tors that ap­pears to pro­tect the cones, which ex­plains why RP pa­tients see their cones die even in the ab­sence of ge­net­ic ab­nor­mal­i­ties. By wrap­ping Rd­CVF, or rod-de­rived cone vi­a­bil­i­ty fac­tor, and an ox­ida­tive stress re­duc­ing en­zyme in­to a vi­ral vec­tor, they came up with SPVN06 in hopes of re­plac­ing one key func­tion that RP pa­tients lose along­side their rods.

Owen Smith

It won’t re­verse the dis­ease, but Spar­ingVi­sion is hop­ing it can slow or stop its pro­gres­sion.

The ap­proach paves a path that reach­es all 2 mil­lion RP pa­tients — a kind of “medi­um rare” in­di­ca­tion and broad­er ap­pli­ca­tion of gene ther­a­py fa­vored by 4BIO Cap­i­tal, said di­rec­tor of in­vest­ment Owen Smith. 4BIO co-led the round with UPMC En­ter­pris­es, the ven­ture arm of Pitts­burgh-based health sys­tem UPMC, where Sa­hel chairs the De­part­ment of Oph­thal­mol­o­gy.

“One of the al­ter­na­tives is de­vel­op­ing a Lux­tur­na in every sin­gle geno­type, and we haven’t even ful­ly char­ac­ter­ized the dis­ease on a ge­net­ic lev­el,” he said.

Cur­rent­ly fo­cused on a sin­gle as­set, Spar­ingVi­sion now op­er­ates vir­tu­al­ly with­out labs or man­u­fac­tur­ing — every­thing is out­sourced and will be for a while — with all 12 of its staffers cur­rent­ly in France. Hav­ing re­cent­ly moved to Paris from the Bay Area, where he was work­ing on cor­po­rate strat­e­gy for Sang­amo fol­low­ing the sale of Tx­Cell two years ago, Bois­sel al­so plans to build a US-based team to start prepar­ing for that sec­ond-in-hu­man ef­fi­ca­cy tri­al.

“It’s eas­i­er to find tal­ents who have done it in the US than in Eu­rope, and I want­ed peo­ple who have done it,” he said. “So our CMO, our CTO, our head of trans­la­tion­al sci­ence will be based in the US.”

He en­vi­sions re­cruit­ing a lean team of very se­nior peo­ple, build­ing out to 20 or 25 as­sum­ing Spar­ingVi­sion sticks on­ly to SPVN06. With that pro­gram alone there’s po­ten­tial to go in­to dry AMD, where the neu­ro­pro­tec­tive mech­a­nism could be rel­e­vant. There is al­so a pos­si­bil­i­ty of bring­ing in new prod­ucts or tech­nolo­gies that would fit un­der ge­nom­ic med­i­cines for the eye, from gene re­place­ment to edit­ing to reg­u­la­tion. But it’s ear­ly days, and Bois­sel said the syn­di­cate — which al­so fea­tures Jeito Cap­i­tal, Ys­ios Cap­i­tal, Bpifrance and Foun­da­tion Fight­ing Blind­ness — isn’t ex­pect­ing a quick flip to the mar­ket.

It’s tak­en the com­pa­ny four years to get to where they are. The first clin­i­cal tri­al should be­gin next year in Eu­rope, while the ef­fi­ca­cy tri­al is planned for 2023.

“That is a very unique set­ting in a world where things are very of­ten go­ing too fast,” he said.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Paul Hudson, Sanofi CEO (ROMUALD MEIGNEUX/Sipa via AP Images)

Sanofi and Am­gen are bring­ing cash to cov­er the ta­ble stakes for the Hori­zon M&A game

With the market cap on Horizon Therapeutics $HZNP pushed up to the $23 billion mark today, one of the Big Pharmas in the hunt for a major league buyout deal signaled it’s playing the M&A game with cash.

Paris-based Sanofi, where CEO Paul Hudson has been largely focused on some risky biotech acquisitions to win some respect for its future pipeline prospects, issued a statement early Friday — complying with rule 2.12 of the Irish takeover rules — making clear that while the certainty or size of an offer can’t be determined, any offer “will be solely in cash.” And Amgen CEO Robert Bradway came right in behind him, filing a statement on the London Stock Exchange overnight that any offer they may make will “likely” be in cash as well.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls

Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Uğur Şahin, BioNTech CEO (ddp images/Sipa USA/Sipa via AP Images)

BioN­Tech bets on dif­fi­cult STING field via small mol­e­cule pact with a Pol­ish biotech

BioNTech is beefing up its relatively thin small molecule pipeline by adding weight to a clinically difficult corner of oncology R&D: STING agonists. To do so, BioNTech is teaming up with a 15-year-old Polish biotech and doling out €40 million, about $41.5 million, to start.

The deal is broken into two parts: First, BioNTech obtains an exclusive global license to develop and market Ryvu Therapeutics’ STING agonist portfolio as small molecules, whether alone or in combination with other agents.

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Lynn Baxter, Viiv Healthcare's head of North America

Vi­iV dri­ves new cor­po­rate coali­tion in­clud­ing Uber, Tin­der and Wal­mart, aimed at end­ing HIV

ViiV Healthcare is pulling together an eclectic coalition of consumer businesses in a new White House-endorsed effort to end HIV by the end of the decade.

The new US Business Action to End HIV includes pharma and health companies — Gilead Sciences, CVS Health and Walgreens — but extends to a wide range of consumer companies that includes Tinder, Uber and Walmart.

ViiV is the catalyst for the group, plunking down more than half a million dollars in seed money and taking on ringmaster duties for launch today on World AIDS Day, but co-creator Health Action Alliance will organize joint activities going forward. ViiV and the alliance want and expect more companies to not only join the effort, but also pitch in funding.

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Pfiz­er will in­vest $1.2B+ in Irish man­u­fac­tur­ing site, adding 500 em­ploy­ees

Covid-19 trailblazer Pfizer has confirmed its commitment to a large expansion project on the Emerald Isle.

The New York-based company announced on Thursday that it will make a €1.2 billion ($1.26 billion) capital investment into its manufacturing site at Grange Castle in Dublin.

The expansion of the site marks Pfizer’s largest expansion investment in Ireland to date. The expansion includes the construction of a new facility on the premises as well as adding in more laboratory space and will ultimately double the capacity for “biological drug substance manufacturing” in the oncology and rare disease space as well as inflammation, immunology and internal medicines.

In­tel­lia and Iver­ic sell stocks to raise mon­ey, each net­ting $300M

Wednesday afternoon, Gene editing company Intellia and eye disease company Iveric Bio announced that they had each raised $300 million by selling off some of their stocks. The two biotechs are the latest to raise money via public stock offerings, an increasingly popular tactic used by public companies as the industry falls back from its pandemic boom.

Intellia’s raise comes a few weeks after it posted an update on its hereditary angioedema program that uses CRISPR/Cas9 to directly edit the gene that makes the protein responsible for the attacks that occur with the disease. In that interim cut, Intellia showed that patients dosed with its one-time therapy became attack free (at least thus far) after an observation period of 16 weeks, with the longest patient remaining attack free for 10 months.

Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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