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.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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'Chang­ing the whole game of drug dis­cov­ery': Leg­endary R&D vet Roger Perl­mut­ter leaps back in­to work as a biotech CEO

Roger Perlmutter needs no introduction to anyone remotely involved in biopharma. As the R&D chief first at Amgen and then Merck, he’s built a stellar reputation and a prolific career steering new drugs toward the market for everything from cancer to infectious diseases.

But for years, he’s also held a less known title: science partner at The Column Group, where he’s regularly consulted about the various ideas the VCs had for new startups.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Gold­man Sachs jumps aboard Bain-backed 503(b) com­pound­ing phar­ma­cy with a $275M debt loan to sup­ply hos­pi­tals

Long the bane of the FDA’s existence, compounding pharmacies have seen a minor resurgence in the past year as short-term saviors for hospital drug shortages. Now, a 503(b) company specializing in hospital meds has earned a big backer to keep expanding its 200-drug strong portfolio.

Goldman Sachs and Owl Rock Capital Partners have doled out a $275 million debt loan to QuVa Pharma, a 503(b)-certified outsourcing facility providing compounded drugs to hospitals, the company said Thursday.