Armed with $42.5M, Gem­i­ni em­braces the Goldilocks strat­e­gy in bring­ing pre­ci­sion med to AMD

For most biotech star­tups, the big chal­lenge lies in de­vel­op­ing one or two new drugs that promise to do a bet­ter job of tar­get­ing a par­tic­u­lar dis­ease.

But Gem­i­ni Ther­a­peu­tics — the lat­est in a long line­up of Cam­bridge, MA biotechs to roll out this year — isn’t like most up­starts.

James McLaugh­lin

CEO James McLaugh­lin has set out to divvy up the large pop­u­la­tion of pa­tients at high risk of dry age-re­lat­ed mac­u­lar de­gen­er­a­tion — AMD — in­to small, ge­net­i­cal­ly de­fined buck­ets of pa­tients, and then find the right drug most like­ly to help them. And that’s big enough to even­tu­al­ly in­volve a whole pipeline of ther­a­peu­tics.

“There are ge­net­ic vari­ants that in­crease the risk of dis­ease by 20 times or more. In­ter­est­ing tar­gets that were nev­er pur­sued; that is what got the pro­gram start­ed. Could we de­vel­op ther­a­peu­tics that were fea­si­ble and tractable for the eye?” says McLaugh­lin, the for­mer op­er­a­tions chief at gene ther­a­py pi­o­neer Voy­ager.

That process be­gan in stealth two and a half years ago, says McLaugh­lin, fol­lowed by the first seed fund­ing in 2016 as Gem­i­ni start­ed work­ing on the R&D side of the busi­ness. Now McLaugh­lin has a $42.5 mil­lion launch round co-led by At­las Ven­ture, Light­stone Ven­tures and Or­biMed. And the grow­ing 7-mem­ber team at Gem­i­ni will be start­ing out with three ba­sic tech­nolo­gies: re­com­bi­nant pro­teins, mon­o­clon­al an­ti­bod­ies and gene ther­a­py.

Find the right pop­u­la­tion first, he says, and you can de­ter­mine the right ther­a­peu­tic ap­proach sec­ond.

“It’s def­i­nite­ly am­bi­tious,” says the CEO, re­quir­ing dif­fer­ent teams to break down the tech and the pre­ci­sion med­i­cine they’re pur­su­ing. So there’s a team of gene ther­a­py ex­perts, a team on tar­get bi­ol­o­gy and a team on reti­nal dis­ease and AMD. “A lot of the con­ver­sa­tion is bridg­ing the gap be­tween the dis­ci­plines,” he adds, with ad­vis­ers and founders from around the globe.

The Uni­ver­si­ty of Ed­in­burgh’s Paul Bar­low, Andy Her­bert and Tufts Uni­ver­si­ty’s Jo­han­na Sed­don are the sci­en­tif­ic founders.

Gem­i­ni’s sci­en­tif­ic ad­vi­so­ry board in­cludes:

  • Reti­nal dis­ease and AMD ex­perts Alan Wright from the Uni­ver­si­ty of Ed­in­burgh, Dean Bok from UCLA and Bruce Jaf­fee.
  • Tar­get bi­ol­o­gy ex­perts in­clude John Atkin­son from Wash­ing­ton Uni­ver­si­ty in St. Louis, Michael Ehrmann from the Uni­ver­si­ty of Es­sen-Duis­berg, Kevin March­bank and Claire Har­ris from New­cas­tle Uni­ver­si­ty, and Si­mon Clark from the Uni­ver­si­ty of Man­ches­ter.
  • Gene ther­a­py ex­perts in­clude Shan­non Boye from Uni­ver­si­ty of Flori­da, Ar­avind Asokan from the Uni­ver­si­ty of North Car­oli­na, and Chris Mueller and Clau­dio Pun­zo from the Uni­ver­si­ty of Mass­a­chu­setts Med­ical School.

At this stage, Gem­i­ni has iden­ti­fied hun­dreds of ge­net­ic mu­ta­tions that play a role in the eye dis­ease. They’re start­ing out with the “Goldilocks zone,” small groups of 1% to 2% or so of the pa­tients like­ly to ben­e­fit the most from one of their tar­get­ed ap­proach­es. The big­ger groups can wait un­til lat­er, af­ter the biotech has shown what it can do with pa­tient pop­u­la­tions that re­sem­ble those found in rare dis­eases, where small tri­als can tell you a lot.

The re­cent up­beat re­view of Spark’s ground­break­ing gene ther­a­py for the eye fo­cused con­sid­er­able at­ten­tion on just how at­trac­tive the eye is as a drug tar­get. It’s large­ly con­tained, al­low­ing in­ves­ti­ga­tors to take a high­ly fo­cused ap­proach that avoids sys­temic threats. And it can be more straight­for­ward in as­sess­ing a drug’s im­pact.

For now, it’s Gem­i­ni’s en­tire world.

A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

Endpoints News writer Nicole DeFeudis has posted a snapshot of all the companies, universities and hospital-based groups now racing through the clinic, ranking them according to their place in the pipeline as well as the latest remarks available on timelines. And we’ll keep this lineup updated right through the end of the year, as the checkered flags start to fall, possibly as early as October.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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Giovanni Caforio, Bristol Myers Squibb CEO (Christopher Goodney/Bloomberg via Getty Images)

UP­DAT­ED: Bris­tol My­ers Squibb com­mits $300 mil­lion to com­bat racial dis­par­i­ties, but de­clines to re­lease own de­mo­graph­ic da­ta

After the police killing of George Floyd, a flurry of pharma and biotech companies, executives and investors jumped out to make statements, either expressing support for Black Lives Matter and the protests or condemning systemic racism.

Now, a Big Pharma company is publicly putting some teeth behind those statements. This morning, Bristol Myers Squibb announced they would spend $300 million on a broad effort to reduce racial health disparities, and diversify both their clinical trials and their own executive team and workforce.

Martin Shkreli (AP Images)

Mar­tin Shkre­li's in­fa­mous Dara­prim falls off top 20 most ex­pen­sive drugs list

Martin Shkreli incited a national uproar five years ago when he raised the price of Daraprim by a factor of 56 essentially overnight from $13.50 to $750 per pill. Now that the “Pharma Bro’s” high-priced project has received a generic, it no longer places among the most expensive drugs in the world.

GoodRx is back with the latest update of the top 20 most expensive drugs and Daraprim’s exclusion marks the biggest change. The drug had previously ranked seventh on the list’s last iteration, which came in February before the world went into quarantine. Another of Shkreli’s former companies, Retrophin, saw its Chenodal drug place in the top 10 again.

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Ian Nicholson (F2G)

No­vo backs 'break­through' an­ti­fun­gal play­er's $60M round — with a fo­cus on rare mold in­fec­tions

The plight of antibiotics developers has been well documented: chronically underfunded research, daunting scientific challenges, and little commercial upside even for the ones that make it to the market. But in an adjacent corner of the antimicrobial space, an antifungal player is out to paint a very different picture.

F2G, a UK-Austria hybrid, has raised $60.8 million for its final push toward the clinic. Clearing the test could pave the way for its drug to be the first new antifungal agent in 20 years.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

VBL an­nounces pos­i­tive PhI­II da­ta for ovar­i­an can­cer drug; Gene­Cen­tric part­ners with Janssen on blad­der can­cer

VBL Therapeutics announced that it is on track in a Phase III trial of its drug VB-111 (ofranergene obadenovec) to treat platinum-resistant ovarian cancer in combination with paclitaxel.

After conducting its second interim analysis, the independent Data Safety Monitoring Committee (DSMC) recommended that Israel-based VBL continue full steam ahead. The committee looked at unblinded overall survival rate, which is the primary endpoint of the study, dubbed OVAL. The first analysis evaluated CA-125 response.