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.

Months after Novavax celebrated its first profitable quarter as a commercial company, the Gaithersburg, MD-based company is back in the red.

Sales for Novavax’s Covid-19 vaccine slipped to $55 million last quarter, down from $586 million in Q1, CEO Stanley Erck revealed on Monday after market close. The company’s stock $NVAX plummeted more than 32% in after-hours trading.

Upon kicking off the call with analysts and investors, Erck addressed the elephant in the room:

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, less than two weeks after its supplemental BLA was accepted. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.