Con­fo Ther­a­peu­tics rais­es €30 mil­lion; Waylivra re­ceives ap­proval from EU; end­point met for Mer­ck­'s Bel­som­ra in PhI­II tri­al

Cedric Ververken

→ In an in­vest­ment led by Bio­Gen­er­a­tion Ven­tures and co-led by Welling­ton Part­ners, with Fund+ and Per­cep­tive Ad­vi­sors step­ping in, Con­fo Ther­a­peu­tics an­nounced to­day that it has raised €30 mil­lion ($33.4 mil­lion) in Se­ries A fi­nanc­ing. The com­pa­ny said that the funds will be al­lo­cat­ed to use to­wards ac­cel­er­at­ing its Con­fo­Body-en­abled drug dis­cov­ery ac­tiv­i­ties and to de­vel­op its pipeline of GPCR mod­u­lat­ing com­pounds to pro­duce drug can­di­dates for clin­i­cal tri­als.

“We ap­pre­ci­ate the strong and con­tin­ued sup­port from our ex­ist­ing in­vestors, who have en­abled the Com­pa­ny to progress to this point. We are proud to wel­come BGV, Welling­ton Part­ners, Fund+ and Per­cep­tive Ad­vi­sors as new in­vestors, com­plet­ing a high-qual­i­ty in­ter­na­tion­al syn­di­cate. We look for­ward to boost­ing the com­pa­ny’s drug dis­cov­ery en­gine and ad­vanc­ing our port­fo­lio of GPCR-mod­u­lat­ing com­pounds,” com­ment­ed CEO Cedric Ververken.

Mer­ck $MRK an­nounced to­day that its drug Bel­som­ra (su­vorex­ant) C-IV met the pri­ma­ry ef­fi­ca­cy end­point in its Phase III tri­al for the treat­ment of in­som­nia in peo­ple with mild-to-mod­er­ate Alzheimer’s dis­ease de­men­tia. 

The com­pa­ny said that “this is the first ded­i­cat­ed Phase III polysomnog­ra­phy study of an in­som­nia med­ica­tion in peo­ple with mild-to-mod­er­ate Alzheimer’s dis­ease de­men­tia, and in the tri­al, Bel­som­ra met its pri­ma­ry and sec­ondary ef­fi­ca­cy end­points. For the pri­ma­ry end­point, 4-weeks of treat­ment with Bel­som­ra im­proved mean to­tal sleep time (TST) by 28.2 min­utes ver­sus place­bo (n=135 vs. n=139, re­spec­tive­ly; [95% CI:11.1,45.2], p<0.005). This cor­re­spond­ed to a mean in­crease from base­line of 73.4 min­utes with Bel­som­ra [95% CI: 61.3, 85.5] and a mean in­crease from base­line of 45.2 min­utes with place­bo [95% CI: 33.3, 57.2]. Ad­verse events were re­port­ed in 22.5% of pa­tients re­ceiv­ing Bel­som­ra com­pared to 16.1% of those re­ceiv­ing place­bo.”

MIT Solve an­nounced to­day the launch of Solve In­no­va­tion Fund — a phil­an­thropic ven­ture fund that will raise $30 mil­lion over time from donors con­tribut­ing through tax-de­ductible gifts to MIT, to cre­ate debt and eq­ui­ty in­vest­ments for ear­ly-stage en­tre­pre­neurs look­ing to solve glob­al chal­lenges. The com­pa­ny said that the fund will be set up as a donor-ad­vised fund (DAF), in­de­pen­dent to and ex­ter­nal to MIT, with MIT Solve act­ing as a donor-ad­vi­sor.

Noubar Afeyan

MIT grad­u­ate, founder and CEO of Flag­ship Pi­o­neer­ing Noubar Afeyan is com­mit­ting up to $3 mil­lion to the fund.

“Solve’s mis­sion is to tack­le glob­al chal­lenges by help­ing ear­ly-stage in­no­va­tors from all around the world con­nect with each oth­er, tap the strength of MIT’s in­no­va­tion ecosys­tem and, cru­cial­ly, gain the re­sources to trans­form their ideas in­to im­pact­ful so­lu­tions. The Solve In­no­va­tion Fund is an in­spir­ing step to pro­vid­ing Solver teams with the cap­i­tal to de­liv­er their so­lu­tions at scale,” said MIT pres­i­dent L. Rafael Reif.

→ The Eu­ro­pean Union has ap­proved Akcea and Io­n­is’ drug, Waylivra (volane­sors­en) — a ther­a­py used to­wards Fa­mil­ial Chy­lomi­crone­mia Syn­drome (FCS). The com­pa­ny said that the ther­a­py will be used as an ad­junct di­et in adult pa­tients with ge­net­i­cal­ly con­firmed FCS and at high risk for pan­cre­ati­tis, who’ve had an in­ad­e­quate re­sponse to di­et and triglyc­eride-low­er­ing ther­a­py.

Paula Soteropou­los

“Waylivra is the on­ly ap­proved treat­ment for peo­ple with FCS and is a ma­jor mile­stone for the glob­al FCS com­mu­ni­ty. This al­so marks Akcea’s sec­ond drug ap­proval in the last year,” said Paula Soteropou­los, CEO of Akcea Ther­a­peu­tics. “We plan to launch Waylivra in Ger­many this year fol­lowed by ad­di­tion­al Eu­ro­pean coun­tries in 2020. Our team is ready to de­liv­er this treat­ment op­tion to pa­tients with FCS.”

→ On Tues­day, Den­ver vot­ers will de­cide whether to de­crim­i­nal­ize pos­ses­sion of psilo­cy­bin, a hal­lu­cino­genic drug used in “mag­ic mush­rooms,” re­port­ed Reuters. Den­ver will be the first US city to stop such pros­e­cu­tions and if passed, in­di­vid­u­als at least 21 years old who use or pos­sess the drug will not re­ceive crim­i­nal penal­ties. 

The drug will re­main il­le­gal un­der both Col­orado and fed­er­al law and has been deemed by the US Drug En­force­ment Ad­min­is­tra­tion (DEA) to have a high po­ten­tial for abuse and cur­rent­ly has no ac­cept­ed med­ical use.

A New Fron­tier: The In­ner Ear

What happens when a successful biotech venture capitalist is unexpectedly diagnosed with a chronic, life-disrupting vertigo disorder? Innovation in neurotology.

That venture capitalist was Jay Lichter, Ph.D., and after learning there was no FDA-approved drug treatment for his condition, Ménière’s disease, he decided to create a company to bring drug development to neurotology. Otonomy was founded in 2008 and is dedicated to finding new drug treatments for the hugely underserved community living with balance and hearing disorders. Helping patients like Jay has been the driving force behind Otonomy, a company heading into a transformative 2020 with three clinical trial readouts: Phase 3 in Ménière’s disease, Phase 2 in tinnitus, and Phase 1/2 in hearing loss. These catalysts, together with others in the field, highlight the emerging opportunity in neurotology.
Otonomy is leading the way in neurotology
Neurotology, or the treatment of inner ear neurological disorders, is a large and untapped market for drug developers: one in eight individuals in the U.S. have moderate-to-severe hearing loss, tinnitus or vertigo disorders such as Ménière’s disease.1 With no FDA-approved drug treatments available for these conditions, the burden on patients—including social anxiety, lower quality of life, reduced work productivity, and higher rates of depression—can be significant.2, 3, 4

Joe Jimenez, Getty

Ex-No­var­tis CEO Joe Jimenez is tak­ing an­oth­er crack at open­ing a new chap­ter in his ca­reer — and that in­cludes a new board seat and a $250M start­up

Joe Jimenez is back.

The ex-CEO of Novartis has taken a board seat on Century Therapeutics, the Versant and Bayer-backed startup focused on coming up with a brand new twist on cell therapies for cancer — a field where Jimenez made his mark backing the first personalized CAR-T approved for use.

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Can we make the an­tibi­ot­ic mar­ket great again?

The standard for-profit model in drug development is straightforward. Spend millions, even billions, to develop a medicine from scratch. The return on investment (and ideally a tidy profit) comes via volume and/or price, depending on the disease. But the string of big pharma exits and slew of biotech bankruptcies indicate that the model is sorely flawed when it comes to antibiotics.

The industry players contributing to the arsenal of antimicrobials are fast dwindling, and the pipeline for new antibiotics is embarrassingly sparse, the WHO has warned. Drugmakers are enticed by greener pastures, compared to the long, arduous and expensive path to antibiotic approval that offers little financial gain as treatments are typically priced cheaply, and often lose potency over time as microbes grow resistant to them.

The FTC and New York state ac­cuse Mar­tin Shkre­li of run­ning a drug mo­nop­oly. They plan to squash it — and per­ma­nent­ly ex­ile him

Pharma bro Martin Shkreli was jailed, publicly pilloried and forced to confront some lawmakers in Washington riled by his move to take an old generic and move the price from $17.50 per pill to $750. But through 4 years of controversy and public revulsion, his company never backed away from the price — left uncontrolled by a laissez faire federal policy on a drug’s cost.

Now the FTC and the state of New York plan to pry his fingers off the drug once and for all and open it up to some cheap competition. And their lawsuit is asking that Shkreli — with several years left on his prison sentence — be banned permanently from the pharma industry.

Amber Saltzman (Ohana)

Flag­ship's first ven­ture of 2020 is out, and it's all about sperm

A couple years ago, Amber Salzman got a call as she was returning East full-time after a two-year stint running a gene therapy company in California.

It was from someone at Flagship Pioneering, the deep-pocketed biotech venture firm. They had a new company with a new way of thinking about sperm. It had been incubating for over a year, and now they wanted her to run it.

“It exactly fit,” Salzman told Endpoints News. “I just thought I had to do something.”

Pfiz­er ax­es 6 ear­ly to late-stage can­cer stud­ies from the pipeline — with one oth­er cut for sick­le cell dis­ease

Pfizer trimmed a group of 3 R&D programs using their PD-L1 Bavencio — partnered with Merck KGaA — in their latest pipeline cull.

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UP­DAT­ED: In­cyte scores much need­ed PhI­II suc­cess — and of course it’s de­liv­ered by rux­oli­tinib

Incyte’s efforts to breathe a second life into ruxolitinib — its JAK inhibitor sold in pill form as Jakafi — has been greeted with clear, if preliminary and unsurprising, Phase III success.

Topline data from the TRuE-AD2 cements ruxolitinib’s foundational importance for Incyte, and gives analysts hope that there might yet be room for growth in a pipeline that’s suffered multiple R&D setbacks.

Stephen Hahn, AP

The FDA un­veils a new reg­u­la­to­ry frame­work to speed along gene ther­a­pies, re­ward­ing the lead­ing play­ers

Bioregnum Opinion Column by John Carroll

The emphasis at the FDA over the past 5 years or so has been on assisting drug developers as much as they can to speed up regulatory reviews and push more drugs into the market. And they are now crafting a final set of regulations aimed at flagging through a whole new generation of gene therapies in clinical testing at a rapid clip.

In a set of 6 prospective guidances posted on the FDA web site Tuesday morning, FDA commissioner Stephen Hahn committed the agency to staying flexible in handing out designations that are critical to gaining early approvals for drugs that claim to be once-and-done but don’t have anything close to the data needed to prove it.

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Mike Bloomberg (AP IMAGES)

Mike Bloomberg joins a grow­ing cho­rus of De­mo­c­ra­t­ic pres­i­den­tial can­di­dates threat­en­ing to go af­ter drug patents

As the mayor of New York City, Mike Bloomberg had a few modest ideas about lowering prescription drug prices in the Big Apple that gained little traction. Now on the campaign trail on a faint hope of clinching the Democratic presidential nomination, the billionaire has some bigger plans — including one that would alter the patent system central to the biopharma business.

In a barebones drug pricing plan posted on Monday, Bloomberg came out blasting President Donald Trump for failing to deliver his promise to lower drug prices, and then making misleading claims about them. The price of over 3,000 drugs still increased at a rate five times higher than inflation in the first six months of 2019, he wrote.