NEA-backed Night­star Ther­a­peu­tics sets sights on an $86M IPO to fund PhI­II gene ther­a­py study

David Fel­lows

Two months af­ter bank­ing a $45 mil­lion crossover round from some top biotech in­vestors, Ox­ford gene ther­a­py spin­out Night­star Ther­a­peu­tics has pen­ciled in an $86 mil­lion IPO to push its lead drug through Phase III test­ing.

Night­star is a reti­nal gene ther­a­py com­pa­ny— of­fi­cial­ly set up as a new sub­sidiary to Night­StaRx — ad­vanc­ing new ther­a­pies aimed at dis­eases that cause blind­ing. First up is NSR-REP1, de­signed to treat choroi­deremia by cor­rect­ing mu­ta­tions in the CHM gene. Ac­cord­ing to the F-1, the biotech has da­ta on 32 pa­tients show­ing their gene ther­a­py ei­ther main­tained or im­proved vi­su­al acu­ity in 90% of the pa­tients in the study. The Phase III pro­gram, set to start in ear­ly 2018, will re­cruit 140 pa­tients and di­vide it in­to high- and low- and no-dose groups, while run­ning a par­al­lel nat­ur­al his­to­ry study to de­tail ex­pect­ed rates of de­cline.

Their next clin­i­cal-stage pro­gram is fo­cused on XL­RP, which “ac­counts for ap­prox­i­mate­ly 15% of all cas­es of re­tini­tis pig­men­tosa, an in­her­it­ed X-linked re­ces­sive reti­nal dis­ease char­ac­ter­ized by a lack of pro­tein trans­port that leads to a loss of pho­tore­cep­tors.” That’s in a Phase I/II study, and there’s a pre­clin­i­cal ther­a­py in view as well.

David Mott, NEA

As of mid-2017, the biotech re­ports that it had burned through about $40 mil­lion, rel­a­tive­ly mod­est for a three-year-old com­pa­ny look­ing to kick off a Phase III study and an IPO.

The com­pa­ny is led by an ex­pe­ri­enced group of bio­phar­ma vets. David Fel­lows, who held se­nior-lev­el mar­ket­ing po­si­tions at J&J and Al­ler­gan, is CEO. CMO Aniz Girach came from Throm­bo­Gen­ics and CSO Gre­go­ry Robin­son once helped Shire on sci­en­tif­ic li­cens­ing for rare dis­eases.

NEA (rep­re­sent­ed by a pro­lif­ic David Mott), Syn­cona, Welling­ton Man­age­ment are the prin­ci­pal share­hold­ers, though the com­pa­ny didn’t ac­tu­al­ly spell out the shares they own in the F1, as re­quired.

The com­pa­ny plans to list as $NITE.

They’ll be head­ed in­to a good-though-not-great mar­ket for biotech IPOs. We’ve seen a string of new of­fer­ings over the last few months, but it doesn’t come close to the 2014 boom, when gene ther­a­py IPOs were gold­en.

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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Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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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.

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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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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