The next Nim­bus? Head of bil­lion-dol­lar drug pro­gram launch­es HotSpot with $45M A round

Re­mem­ber that bil­lion-dol­lar drug idea that got lit­tle Nim­bus on Gilead’s radar back in 2016? Well, the folks be­hind that drug are build­ing a whole plat­form on a sim­i­lar con­cept. And they’re not just tack­ling NASH, but a whole line­up of pre­vi­ous­ly un­drug­gable tar­gets.

The ven­ture, called HotSpot Ther­a­peu­tics, came out Tues­day with news that it’s be­ing backed by Nim­bus’ lead in­vestor — At­las Ven­ture — in a $45 mil­lion Se­ries A round co-led by Sofinno­va.

HotSpot was launched last year by Geral­dine Har­ri­man, the for­mer Nim­bus ex­ec­u­tive who led the com­pa­ny’s NASH pro­gram — the very one that earned the Boston biotech a $1.2 bil­lion part­ner­ship with Gilead. That drug, at the time called NDI-01097, used a way (rel­a­tive­ly new to phar­ma) to ma­nip­u­late pro­teins out­side the ac­tive site, go­ing in­stead af­ter some­thing called “reg­u­la­to­ry hotspots.” These, I’m told, are al­losteric sites that the body us­es to reg­u­late pro­tein func­tion. Har­ri­man says tar­get­ing these hotspots could be a boon for drug de­vel­op­ment, which is why she’s co-found­ed an en­tire­ly new ven­ture on the con­cept.

“The ac­tive sites of pro­teins tend to look a lot like each oth­er, so get­ting mol­e­cules that can be se­lec­tive is a hur­dle,” she said. “It takes a lot of ef­fort and some­times the prob­lem isn’t solved. Reg­u­la­to­ry hotspots, on oth­er hand, are how na­ture con­trols the pro­teins — turn­ing things on and off. And reg­u­la­to­ry hotspots are unique to the pro­tein.”

Bruce Booth

Short­ly af­ter the Gilead deal, Har­ri­man, to­geth­er with an­oth­er ex-Nim­bus ex­ec Jonathan Mon­tagu, formed HotSpot to har­ness what they know about reg­u­la­to­ry hotspots to build a whole slew of drug pro­grams. Mon­tagu is serv­ing as HotSpot’s CEO, while Har­ri­man is CSO.

With its plat­form, the com­pa­ny says it’s iden­ti­fied reg­u­la­to­ry hotspots in over 100 pro­teins span­ning sev­er­al path­ways and dis­eases. Mon­tagu says they’re hom­ing in on im­munol­o­gy, im­muno-on­col­o­gy, and NASH.

Mon­tagu says HotSpot has five pro­grams in the pipeline, al­though they’re mum on de­tails. We do know they’re tar­get­ing PKC-theta, which plays a role in au­toim­mune dis­eases, and S6 ki­nase, a meta­bol­ic en­zyme in­volved in reg­u­lat­ing he­pat­ic in­sulin sen­si­tiv­i­ty and mi­to­chon­dr­i­al func­tion.

Ce­ment­ing its ties to Nim­bus, HotSpot al­so sports the same chair­man: At­las Ven­ture part­ner Bruce Booth.

“HotSpot’s el­e­gant and sys­tem­at­ic ap­proach to al­lostery, fo­cused unique­ly on reg­u­la­to­ry hotspots, sets it apart from every­thing else we have seen in the field,” Booth said in a state­ment. “From the out­set, we knew that reg­u­la­to­ry hotspots were crit­i­cal to pro­tein func­tion and now we see vivid­ly that the known foot­print of the nat­ur­al pro­tein lig­and ac­cel­er­ates our chem­istry ef­forts. HotSpot is chang­ing the way al­losteric drug dis­cov­ery is con­duct­ed in a pro­found way and At­las is very ex­cit­ed about the launch of this com­pa­ny.”

Im­age: Jonathan Man­tagu and Geral­dine Har­ri­man. HOTSPOT

Inside FDA HQ (File photo)

The FDA just ap­proved the third Duchenne MD drug. And reg­u­la­tors still don’t know if any of them work

Last year Sarepta hit center stage with the FDA’s controversial reversal of its CRL for the company’s second Duchenne muscular dystrophy drug — after the biotech was ambushed by agency insiders ready to reject a second pitch based on the same disease biomarker used for the first approval for eteplirsen, without actual data on the efficacy of the drug.

On Wednesday the FDA approved the third Duchenne MD drug, based on the same biomarker. And regulators were ready to act yet again despite the lack of efficacy data.

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Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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Cal­lid­i­tas bets up to $102M on a biotech buy­out, snag­ging a once-failed PBC drug

After spending years developing its oral formulation of the corticosteroid budesonide, Sweden’s Calliditas now has its sights set on the primary biliary cholangitis field.

The company will buy out France-based Genkyotex, and it’s willing to bet up to €87 million ($102 million) that Genkyotex’s failed Phase II drug, GKT831, will do better in late-stage trials.

Under the current agreement, Calliditas $CALT will initially pay €20.3 million in cash for 62.7% of Genkyotex (or €2.80 a piece for 7,236,515 shares) in early October, then circle back for the rest of Genkyotex’s shares under the same terms. If nothing changes, the whole buyout will cost Calliditas €32.3 million, plus up to  €55 million in contingent rights.

Qi­a­gen in­vestors spurn Ther­mo Fish­er’s takeover of­fer, de­rail­ing a $12B+ deal

Thermo Fisher Scientific had announced an $11.5 billion takeover of Dutch diagnostics company Qiagen back in March, but the deal apparently did not sit well with Qiagen investors.

After getting hammered by critics who contended that Qiagen $QGEN was worth a lot more than what Thermo Fisher wanted to spend, investors turned thumbs down on the offer — derailing the buyout even after Thermo Fisher increased its offer to $12.6 billion in July. Qiagen’s share price has been boosted considerably by Covid-19 as demand for its testing kits surged.

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Xuefeng Yu in Hong Kong, 2019 (Imaginechina via AP Images)

CanSi­no reaps $748M wind­fall from Shang­hai IPO — as it warns Covid-19 vac­cine won't be a huge mon­ey mak­er

CanSino began the year with a clear goal to secure a secondary listing on Shanghai’s STAR market. Then something more urgent came along: As a rising vaccine developer on a mission to bring global standard immunizations to China, it heeded the call to make a vaccine to protect against a virus that would paralyze the whole world.

Xuefeng Yu and his team managed to keep doing both.

More than a month after CanSino’s Covid-19 vaccine candidate is authorized for military use in China, the Hong Kong-listed company has made a roaring debut in Shanghai. It fetched $748 million (RMB$5.2 billion) by floating 24.8 million shares, and soared 88% on its first trading day.

James Wilson, WuXi Global Forum at JPM20

FDA puts up a red light for Pas­sage Bio’s first gene ther­a­py pro­gram, de­lay­ing a pro­gram from James Wilson's group at Penn

Gene therapy pioneer James Wilson spearheaded animal studies demonstrating the potential of new treatments injected directly into the brain, looking to jumpstart a once-and-done fix for an extraordinarily rare disease called GM1 gangliosidosis in infants. His team at the University of Pennsylvania published their work on monkeys and handed it over to Passage Bio, a Wilson-inspired startup building a pipeline of gene therapies — with an IND for PBGM01 to lead the way.

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A lab technician works during research on coronavirus at Johnson & Johnson subsidiary Janssen Pharmaceutical in Beerse, Belgium, Wednesday, June 17, 2020. (Virginia Mayo/AP Images)

UP­DAT­ED: End­points News ranks all 28 play­ers in the Covid-19 vac­cine race. Here's how it stacks up to­day

(This piece was last updated on August 13. Endpoints News will continue to track the latest developments through the FDA’s marketing decisions.)

The 28 players now in or close to the clinical race to get a Covid-19 vaccine over the finish line are angling for a piece of a multibillion-dollar market. And being first — or among the leaders — will play a big role in determining just how big a piece.

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Bayer's Marianne De Backer with Endpoints founder John Carroll, Endpoints@JPM20 (Jeff Rumans for Endpoints News)

UP­DAT­ED: Hunt­ing a block­buster, Bay­er forges an $875M-plus M&A deal to ac­quire women’s health biotech

Bayer has dropped $425 million in cash on its latest women’s health bet, bringing a UK biotech and its non-hormonal menopause treatment into the fold.

KaNDy Therapeutics had its roots in GlaxoSmithKline, which spun out several neuroscience drugs into NeRRe Therapeutics back in 2012. Five years later the team created a new biotech to focus solely on NT-814 — which they considered “one of the few true innovations in women’s health in more than two decades.”

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