Ver­sant-backed Gotham Ther­a­peu­tics launch­es with $54M round and a new way to con­nect on mR­NA

New York has added an­oth­er biotech start­up to its grow­ing ros­ter for the Big Ap­ple hub.

Samie Jaf­frey

The com­pa­ny is Gotham Ther­a­peu­tics, which now has a $54 mil­lion launch round to back its work on a plat­form built on the in­sights gleaned by Samie Jaf­frey, an in­ves­ti­ga­tor at the Weill Med­ical Col­lege of Cor­nell Uni­ver­si­ty who has spe­cial­ized in post-tran­scrip­tion­al mR­NA mod­i­fi­ca­tions.


Lee Babiss

The goal at Gotham is to cre­ate new drugs that can drug key pro­teins which mod­u­late mes­sen­ger RNA, with po­ten­tial­ly a big im­pact on a slate of dis­eases. Can­cer is an ear­ly fo­cus at the ear­ly-stage com­pa­ny, along with au­toim­mune dis­eases as well as a run at neu­ro­sciences, but CEO Lee Babiss is re­luc­tant to go in­to much de­tail about the specifics of their work.

That’s al­so some­thing you can ex­pect to see in a start­up, where no one likes to get in­to the weeds on drug pro­grams, or de­vel­op­ment time­lines. But the sci­ence de­serves a close look.

“While aca­d­e­m­ic re­search and the phar­ma­ceu­ti­cal in­dus­try fo­cused ini­tial­ly on mod­i­fi­ca­tions of DNA, a grow­ing body of ev­i­dence in­di­cates that mR­NA mod­i­fi­ca­tions help de­ter­mine to which de­gree genes are trans­lat­ed in­to pro­teins. RNA mod­i­fi­ca­tions and their as­so­ci­at­ed pro­tein com­plex­es there­fore rep­re­sent an un­tapped fron­tier that could yield new ther­a­peu­tic ap­proach­es,” says Jaf­frey.

In Babiss the ven­ture back­ers found some­one with a deep lev­el of ex­pe­ri­ence in R&D. He is the for­mer pres­i­dent of phar­ma re­search at Roche and was al­so the CSO at PPD, a big CRO.

That will help. Gotham is keep­ing the staffing lev­els low, with 6 full timers on the pay­roll. That might ex­pand a lit­tle, but Babiss wants much of the heavy lift­ing to be done by con­tract work­ers, keep­ing the team at Gotham — which in­cludes a sub in Mu­nich — at a vir­tu­al lev­el.

Seed in­vestor Ver­sant Ven­tures led the round, along with For­bion and GSK’s SR One. The syn­di­cate al­so in­cludes Cel­gene and Alexan­dria Ven­ture In­vest­ments.

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

Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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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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IPOs abound in the time of coro­n­avirus, as For­ma Ther­a­peu­tics pen­cils in $150M Nas­daq de­but

The IPO engine is thriving, never mind the rampage of the coronavirus crisis on R&D timelines.

On Friday, along with synthetic lethality-focused biotech Repare Therapeutics, another Bristol Myers partner Forma Therapeutics also unveiled its plans to vault on to the Nasdaq — penciling in a target of $150 million.

The Watertown, Massachusetts-based company — which poached senior Genentech executive Frank Lee to take over the reins last year after more than a decade under founder Steve Tregay — raised a plump $100 million late last year, while shepherding its sickle cell disease (SCD) drug through an early-stage trial.

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.

Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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Covid-19 roundup: Did in­sid­ers cash in on pos­i­tive news re­port about Gilead be­fore pub­li­ca­tion?

A series of bullish trades on Gilead options just before the release of a favorable news story is raising questions among regulatory experts, Reuters reported.

On April 16, just hours before STAT published anecdotes from a Chicago hospital that served as one of the clinical sites to test Gilead’s remdesivir in Covid-19 patients, the California-based company’s shares were trading at around $75. Four large blocks of options were purchased for about $1.5 million each, betting that the stock would rise beyond that to as much as $87.5 by mid-August.

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Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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