Ver­sant-backed, Bris­tol My­ers-stamped Re­pare Ther­a­peu­tics guns for $100M IPO

With a Bris­tol My­ers Squibb en­dorse­ment in tow, Ver­sant-backed can­cer drug de­vel­op­er Re­pare Ther­a­peu­tics has set its sights on a Nas­daq de­but.

On Fri­day, the Mon­tre­al-based com­pa­ny with op­er­a­tions in Cam­bridge, Mass­a­chu­setts that is yet to en­ter the clin­ic, un­veiled plans for a $100 mil­lion IPO, bank­ing on its “syn­thet­ic lethal­i­ty” plat­form.

The ba­sic idea is to tar­get the ge­net­ic ba­sis of tu­mors, a com­mon idea across pre­ci­sion on­col­o­gy med­i­cines. But in­stead of tar­get­ing the per­pe­tra­tor mu­ta­tion di­rect­ly, the com­pound is de­signed to go af­ter the oth­er gene in the gene pair. The ra­tio­nale is based on the decades-old ge­net­ic prin­ci­ple that in­di­cates two mu­ta­tions are lethal on­ly when com­bined to­geth­er.

A sim­i­lar ap­proach was first so­lid­i­fied with the ap­proval of PARP in­hibitors, such as As­traZeneca and Mer­ck’s Lyn­parza, Clo­vis’ Rubra­ca and GSK’s Ze­ju­la. PARP is a pro­tein used by dam­aged cells to ini­ti­ate re­pair, and by thwart­ing it, the class of drugs is en­gi­neered to pre­vent can­cer cells from re­pair­ing them­selves, there­by cat­alyz­ing their de­struc­tion.

At Re­pare, sci­en­tists have em­ployed the use of a CRISPR-based screen­ing tool to hone in on these syn­thet­i­cal­ly lethal gene pairs and then en­gi­neered drugs to hunt them. The Re­pare ap­proach promis­es to be tu­mor-ag­nos­tic — the com­pa­ny’s lead ex­per­i­men­tal prod­uct, RP-3500, is a small mol­e­cule de­signed to block the DNA re­pair pro­tein — atax­ia telang­iec­ta­sia —  as well as an en­zyme that’s ac­ti­vat­ed by DNA repli­ca­tion stress. Clin­i­cal tri­als are an­tic­i­pat­ed to be­gin lat­er this quar­ter, and if all goes well, a Phase I/II study in the third quar­ter.

Re­pare has com­pa­ny in the field, such as its Cam­bridge neigh­bor Tan­go Ther­a­peu­tics that has CRISPR dis­cov­ered five pre­clin­i­cal pro­grams and raised $60 mil­lion in a fresh in­jec­tion of fund­ing in April. Cyteir Ther­a­peu­tics, an­oth­er ri­val, net­ted about $40 mil­lion last Oc­to­ber.

So far, 2016 formed-Re­pare has raised $135.2 mil­lion — its most re­cent cap­i­tal in­jec­tion was an $82.5 mil­lion Se­ries B fi­nanc­ing last Sep­tem­ber. The IPO fil­ing shows the com­pa­ny has pen­ciled in a $100 mil­lion IPO, but of late, the fi­nal fig­ures raised are typ­i­cal­ly far high­er.

Daniel Durocher

Days ago, Bris­tol My­ers bought in­to the dream, fork­ing over $50 mil­lion up­front and $15 mil­lion in an eq­ui­ty in­vest­ment in a col­lab­o­ra­tion with Re­pare. Pre-IPO, Ver­sant is Re­pare’s largest share­hold­er, own­ing rough­ly a 30% stake — oth­er in­vestors in­clude MPM Cap­i­tal (11.8%) and Or­biMed (11.2%).

Frank Sicheri

The ar­chi­tect of Re­pare’s tech­nol­o­gy is com­pa­ny co-founder Daniel Durocher of the Lunen­feld-Tanen­baum Re­search In­sti­tute (the re­search arm of Mount Sinai Hos­pi­tal) who is one of the first sci­en­tists who pi­o­neered the use of CRISPR to screen for syn­thet­i­cal­ly lethal gene pairs. Frank Sicheri, the oth­er co-founder, is al­so from Lunen­feld-Tanen­baum.

A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Su­per-se­cre­tive an­ti-ag­ing biotech Cal­i­co tees up the first vis­i­ble clin­i­cal tri­al of an ex­per­i­men­tal drug. And it’s for can­cer?

Over the past 7 years, Calico has been so much more than your average, run-of-the-mill secretive biotech players. It’s a riddle, wrapped in a mystery, inside an enigma, to repurpose an old Winston Churchill line dating from the time he confronted the Iron Curtain surrounding Stalin’s thoughts.

Launched by industry legend Art Levinson of Genentech fame, with the infinitely deep pockets of Google for support, one of the few big headlines the anti-aging biotech has sparked focused on a major alliance with AbbVie — a giant outfit that conversely likes to show off its drug prospects whenever it can. Together, they’ve been focused on diseases that limit life span — quite an arc of ailments.

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RBC's Bri­an Abra­hams holds a mock ad­comm on Bio­gen's iffy ad­u­canum­ab da­ta — and most of these ex­perts don't see a path to an ap­proval

As catalysts go, few loom larger than the aducanumab adcomm slated for Nov. 6.

With its big franchise under assault, Biogen is betting the ranch that its mixed late-stage Alzheimer’s data can squeak past the experts and regulators and get onto the market. And the topic — after a decade of Alzheimer’s R&D disasters in what still represents the El Dorado of drug markets — remains in the center ring of discussions around late-stage pipeline prospects.

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Af­ter near­ly a year of de­bate, the Covid-19 vac­cine chal­lenge tri­als are of­fi­cial­ly com­ing

After nearly a year of public advocacy and often rancorous ethical debate, human challenge trials for Covid-19 vaccines are getting off the ground in London.

The UK government’s Covid-19 Vaccine Taskforce and the contract research firm Open Orphan announced today £10 million ($13 million) plan to test experimental Covid-19 vaccines in volunteers whintentionally exposed to the novel coronavirus. The studies, which won’t launch until early 2021, come after 9 months of debate over whether such studies were safe and would actually hasten vaccine development, and they follow a long history of researchers using challenge models to study other respiratory viruses, including flu and the coronaviruses that cause the common cold.

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Derek Chalmers, Cara Ther

Cara lines up a $440M deal for US rights to its late-stage drug for se­vere itch, with $150M cash on the ta­ble

With plans afoot to file an NDA for what could be its first approved drug, Cara Therapeutics is pivoting its focus to commercialization. And Swiss company Vifor Pharma is willing to surrender up to $440 million to market the candidate in the US.

Cara $CARA CEO Derek Chalmers said an NDA submission is coming this quarter for their intravenous drug Korsuva in chronic kidney disease-associated pruritus (CKD-aP), a severe itching condition. The Stamford, CT-based biotech read out positive topline data from a Phase III pivotal study back in April, and announced plans to approach EMA regulators shortly after filing with the FDA.

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IN8bio CEO William Ho (IN8bio)

Bring­ing their ge­net­i­cal­ly mod­i­fied gam­ma delta T cells to Nas­daq, IN8bio files for $86M IPO

The biotech IPO parade continues marching forward as 2020 turns toward the fourth quarter.

IN8bio, a New York-based company focused on genetically modified gamma delta T cell therapies, filed to go public Friday seeking an $86 million raise. The company has two clinical-stage candidates being studied in glioblastoma and leukemia, respectively.

By any stretch of the imagination, 2020 has already been a huge year for biotech, and nowhere does it appear more obvious than the vast amounts of companies hitting the public market.

Covid-19 roundup: Pars­ing Bourla, a top an­a­lyst sees im­proved chances for Pfiz­er vac­cine; Fau­ci: No sur­prise that Trump was hit by Covid-19

With a medley of adverse events hobbling the late-stage development of vaccines and drugs, Pfizer CEO Albert Bourla’s latest — extended — timeline for the mRNA approach they’re working on with BioNTech is giving some top analysts added confidence that the pharma giant can come up with the regulatory goods next month.

Parsing Bourla’s language in his comments last week, SVB Leerink’s Geoffrey Porges notes that Bourla’s decision to say they “may” be able to nail down the positive efficacy of their vaccine in a matter of days — a big change from his earlier certainty — may also indicate a delay on that to early November.

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CAR-plus: Irish biotech re­cruits Kite alum Chris Now­ers to prep dual-tar­get­ing NK cell ther­a­py for the clin­ic

Soon after Chris Nowers left Cell Medica — freshly rebranded Kuur Therapeutics — in February, the Kite Pharma alum was introduced to another cell therapy player.

The basic idea of building an off-the-shelf allogeneic platform with a CAR-NK approach was familiar to him, riding on the same wave as Takeda, J&J-backed Fate, Nkarta and others. But then there was something else that stood out: a membrane-bound TNF related apoptosis inducing ligand variant, or TRAIL variant, that’s also engineered onto the NK cell for a dual-targeted attack.

New Dewpoint Therapeutics CEO Ameet Nathwani (Sanofi)

A long-haul biotech with some im­pres­sive back­ers and big goals re­cruits a ma­jor league R&D ex­ec to the helm. What’s next?

A few weeks ago Kite and Allogene founder Arie Belldegrun jumped into the expanded syndicate for a Boston-based biotech called Dewpoint Therapeutics — a Polaris-birthed venture that’s styled itself as a drug development pioneer out to craft a major pipeline.

That round — which also added deep-pocket player ARCH to the list of backers — came up with $77 million for the next step in the long journey toward the clinic, a nice add to the A round that launched the company. Now we hear that Dewpoint has recruited Ameet Nathwani to the executive suite as the new CEO, who’s taking the helm from Polaris managing partner Amir Nashat, who brought the company into existence.

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