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.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Steven James, Pionyr Immunotherapeutics CEO

Gilead pass­es on ful­ly ac­quir­ing Pi­o­nyr, as eyes now turn to Tizona, a fel­low sum­mer 2020 buy­out op­tion

Gilead and Pionyr Immunotherapeutics, a biotech trying to follow up on the first generation of checkpoint inhibitors, have “mutually agreed” on a rewrite to their 2020 terms, with Gilead deciding not to buy out the company.

The California biopharma waived its option to acquire the remaining 50.1% of Pionyr, which would have triggered a $315 million upfront payment and up to $1.15 billion down the road. Had Gilead waited to decide, the drugmaker would have had a potential payment to make in the near term under their agreement, a spokesperson said in an email to Endpoints News.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

Eu­ro­pean doc­tors di­al up dig­i­tal com­mu­ni­ca­tion with phar­mas, but still lean to­ward in-per­son med meet­ings, study finds

As in-person sales rep access declines in the big five European countries, a corresponding uptick in virtual rep access is happening. It’s not surprising, but it does run counter to pharma companies’ assessment – along with long-held sales rep sway in Europe – that in-person access hadn’t changed.

CMI Media Group and Medscape’s recent study reports that 75% of physicians in the EU5 countries of Spain, Germany, Italy, France and the UK already limit engagements with pharma sales reps, and 25% of those surveyed plan to decrease time with reps.

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