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.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.