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.

UP­DAT­ED: Bio­gen shares spike as ex­ecs com­plete a de­layed pitch for their con­tro­ver­sial Alzheimer's drug — the next move be­longs to the FDA

Biogen is stepping out onto the high wire today, reporting that the team working on the controversial Alzheimer’s drug aducanumab has now completed their submission to the FDA. And they want the agency to bless it with a priority review that would cut the agency’s decision-making time to a mere 6 months.

The news drove a 10% spike in Biogen’s stock $BIIB ahead of the bell.

Part of that spike can be attributed to a relief rally. Biogen execs rattled backers and a host of analysts earlier in the year when they unexpectedly delayed their filing to the third quarter. That delay provoked all manner of speculation after CEO Michel Vounatsos and R&D chief Al Sandrock failed to persuade influential observers that the pandemic and other factors had slowed the timeline for filing. Actually making the pitch at least satisfies skeptics that the FDA was not likely pushing back as Biogen was pushing in. From the start, Biogen execs claimed that they were doing everything in cooperation with the FDA, saying that regulators had signaled their interest in reviewing the submission.

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Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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FDA bars the door — for now — against Mer­ck’s star can­cer drug af­ter Roche beat them to the punch

Merck has been handed a rare setback at the FDA.

After filing for the accelerated approval of a combination of their star PD-1 drug Keytruda with Eisai’s Lenvima as a first-line treatment for unresectable hepatocellular carcinoma, the FDA nixed the move, handing out a CRL because Roche beat them to the punch on the same indication by a matter of weeks.

According to Merck:

Ahead of the Prescription Drug User Fee Act action dates of Merck’s and Eisai’s applications, another combination therapy was approved based on a randomized, controlled trial that demonstrated overall survival. Consequently, the CRL stated that Merck’s and Eisai’s applications do not provide evidence that Keytruda in combination with Lenvima represents a meaningful advantage over available therapies for the treatment of unresectable or metastatic HCC with no prior systemic therapy for advanced disease. Since the applications for KEYNOTE-524/Study 116 no longer meet the criteria for accelerated approval, both companies plan to work with the FDA to take appropriate next steps, which include conducting a well-controlled clinical trial that demonstrates substantial evidence of effectiveness and the clinical benefit of the combination.

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Covid-19 roundup: EU backs Os­i­vax's pur­suit of a uni­ver­sal vac­cine to fight Covid-19

The race to find a vaccine for the novel coronavirus continues to heat up, as the European Commission’s pilot R&D arm taps French biotech Osivax to head up its unique approach to the research.

Aiming to develop a universal jab for the flu and Covid-19, Osivax secured around $20 million in “blended financing” from the European Innovation Council. About $3 million comes from a Covid-19 “accelerator grant” and will go toward completing Osivax’s signature flu vaccine, dubbed OVX836 and currently in Phase IIa. The rest will be included as part of Osivax’s Series B funding, which aims to launch the Phase IIb portion of the study.

Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

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Trump and Navar­ro press again for hy­drox­y­chloro­quine. Can the FDA stay in­de­pen­dent?

Tuesday morning, economist and Trump advisor Peter Navarro walked onto the White House driveway and promptly brought a political cloud back onto the FDA.

Speaking to a White House pool reporter, Navarro said that four Detroit doctors were, based on a single disputed study, filing for the FDA to again issue an emergency authorization for hydroxychloroquine, the anti-malarial pill that President Trump hyped for months as a Covid-19 treatment over the objections of his own scientists. Then, while avoiding directly calling for the FDA to OK the drug, blasted the agency. He said its decision to pull an earlier authorization “was based on bad science” and “had a tremendously negative effect” on doctors and patients.

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Adrian Gottschalk, Foghorn CEO

Mer­ck dan­gles up to $425 mil­lion to team with Flag­ship’s Foghorn Ther­a­peu­tics on drug­ging the shape of DNA

Two years after it first emerged from stealth mode, Flagship’s Foghorn Therapeutics has nabbed its first Big Pharma partner as Merck signs on to the biotech’s vision of drugging the very shape of DNA.

The deal, worth up to $425 million but with the upfront cash undisclosed, comes as Foghorn nears a pivot to a clinical stage biotech. The Cambridge-based company has added nearly 60 staffers from the 25 it had when it first emerged out of Flagship and, CEO Adrian Gottschalk said, they have finally refined the screening technology at the heart of the company, with plans to file their first IND towards the end of the year.

John Reed, Sanofi R&D chief (Endpoints News)

John Reed brings NK cells in­to Sanofi's CD38 ri­val­ry with J&J — and of­fers thumbs up for Kiadis' new fo­cus

Sanofi doesn’t just want to be a challenger to J&J’s dominant Darzalex multiple myeloma franchise. It’s looking to pioneer a new approach by pairing its own — newly approved — anti-CD38 drug with an NK cell therapy it’s just picked up.

The French pharma giant has teed up $19.7 million (€17.5 million) upfront and close to a billion dollars (€857.5 million) in milestones for a license to Kiadis Pharma’s preclinical K-NK004 program, which consists of NK cells that have been genetically engineered not to express CD38.

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David Hallal

AlloVir tests how much an an­tivi­ral biotech can reap in a pan­dem­ic stock mar­ket

The pandemic stock market has proven fruitful for virtually any type of biotech. Now a 7-year-old cell therapy startup will see how much it can yield for a company that specializes in fighting viruses.

AlloVir, a company that until 2019 largely lived off grant money, has filed for a $100 million IPO to back its line of off-the-shelf, virus-fighting T cells. Although in normal circumstances, $100 million could be a solid return for a biotech that got its first major round of funding only last year, we’ll have to wait to see how much the company ultimately earns. As Covid-19 has sent investor money scurrying to almost anyone in drug development, every single biotech to go public this year has prized above their midpoint or upsized their offering, according to Renaissance Capital, sometimes dramatically so.