Aim for the brain: RA Cap­i­tal bets on Col­orado biotech to re­for­mu­late epilep­sy drug for re­frac­to­ry pa­tients

A decades-old an­ti-epilep­tic drug, linked to de­for­mi­ties in un­born chil­dren, is be­ing re­for­mu­lat­ed for in­jec­tion straight in­to the brain of pa­tients whose epilep­sy has per­sist­ed de­spite ther­a­py.

On Tues­day, the com­pa­ny be­hind the ef­fort — Au­ro­ra, Col­orado-based Cere­bral Ther­a­peu­tics — scored $35 mil­lion in Se­ries B fi­nanc­ing led by RA Cap­i­tal Man­age­ment to fund a Phase IIb tri­al of its drug-de­vice com­bo.

The prod­uct is an im­plant­ed, re­fill­able catheter and pump sys­tem en­gi­neered to de­liv­er a dose of the re­for­mu­lat­ed ver­sion of val­proic acid, called CT-010, di­rect­ly in­to the cere­brospinal flu­id by by­pass­ing the blood-brain bar­ri­er. This form of ad­min­is­tra­tion is aimed at elim­i­nat­ing the bur­den of dai­ly pills and lim­it­ing the body’s ex­po­sure to the drug.

Da­ta from a proof-of-con­cept Phase Ib/IIa tri­al in pa­tients with epilep­sy who have not de­rived ad­e­quate ben­e­fit from oral med­ica­tions ex­pe­ri­enced a 50% to 90% re­duc­tion in seizure fre­quen­cy and sever­i­ty fol­low­ing im­plan­ta­tion with the Cere­bral drug-de­vice com­bo, notes the com­pa­ny’s web­site.

Val­proic acid and its de­riv­a­tives such as sodi­um val­proate were ap­proved in the 1960s and 1970s for use in epilep­sy and bipo­lar dis­or­der. In the Unit­ed States, Ab­b­Vie (for­mer­ly Ab­bott Lab­o­ra­to­ries) man­u­fac­tures the drug as De­pakote, De­pakine and De­pa­con. In Eu­rope, it is sold as De­pakine by Sanofi. Ev­i­dence that val­proate was as­so­ci­at­ed with neu­rode­vel­op­men­tal risks emerged in the lat­ter half of the 20th cen­tu­ry.

The lat­est fund­ing for Cere­bral in­clud­ed the par­tic­i­pa­tion of new in­vestor Per­cep­tive Ad­vi­sors, as well as ex­ist­ing in­vestors Vi­vo Cap­i­tal and Gran­ite Point Cap­i­tal Man­age­ment. The round was led by RA Cap­i­tal, which is typ­i­cal­ly hun­gry for build­ing biotechs but fa­vor the clas­sic “val­ue in­flec­tion point” such as an IPO a year or so post their bet. The com­pa­ny has pre­vi­ous­ly raised $11 mil­lion in Se­ries A fi­nanc­ing.

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.

Charlie Silver (Mission Bio)

'We want to be every­where.' Mis­sion Bio rais­es $70M be­hind re­sis­tance-hunt­ing se­quenc­ing plat­form

Charlie Silver wants to look really, really closely at a lot of your cells. And he just got a lot of money to do so.

Silver’s startup, Mission Bio, raised $70 million in a Series C round Thursday led by Novo Holdings. The money, which brings Mission Bio to $120 million raised since its 2012 founding, will be used to advance the single-cell sequencing platform they built to detect early response or resistance to new cancer therapies.

Franz-Werner Haas, CureVac CEO

UP­DAT­ED: On the heels of a snap $1B raise, Cure­Vac out­lines plans to seek emer­gency OK for their Covid-19 vac­cine in a mat­ter of months

CureVac jumped onto Nasdaq Thursday night, landing with an extra $213 million after pricing a batch of shares at $16 a pop. Add in an extra $118 million share purchase by founder Dietmar Hopp — who owns a controlling interest — and another $640 million from deals and the German biotech has raised a cool billion dollars in the space of just 3 weeks.

The company’s stock $CVAC will now start trading this morning, with analysts eager to find out whether the go-go atmosphere on Wall Street will swell the biotech’s share price.

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Martin Shkreli (Shutterstock)

Mar­tin Shkre­li con­tin­ued to or­ches­trate an­ti-com­pet­i­tive schemes for Dara­prim be­hind bars — FTC

Martin Shkreli didn’t just blog, read up on drug development news and run his biotech business with a contraband cell phone in prison. According to the FTC, he was also coordinating the anticompetitive scheme to shield Daraprim — the drug at the center of a price-gouging controversy that earned him the “Pharma Bro” nickname — from generic rivals.

Back in January the FTC, together with New York’s attorney general, launched a federal lawsuit against Shkreli, who’s now serving a 7-year sentence for defrauding investors in his hedge fund, alleging that he effectively created a drug monopoly. While Shkreli’s notorious move to raise the per tablet price of Daraprim from $17.50 to $750 was perfectly legal, the tactics he allegedly deployed to box out competitors weren’t.

NIH director Francis Collins at a Senate Appropriations subcommittee hearing for Operation Warp Speed (Graeme Jennings/Pool via AP Images)

Covid-19 roundup: 'No­vem­ber or De­cem­ber' Collins' best bet on a vac­cine OK; First plas­ma tri­al sug­gests mor­tal­i­ty re­duc­tion

Count NIH director Francis Collins out for any wager that the FDA would authorize a Covid-19 vaccine in October.

The discussion came up during a call with reporters because some states and local governments have been told by the CDC to have vaccination plans ready to go by Oct. 1. Pharma execs, most notably from Pfizer and BioNTech, have raised hopes about a licensure during that month; President Donald Trump last week sounded an optimistic note about having a vaccine on the market “right around” Election Day on Nov. 3 — or possibly before.

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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.