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.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

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It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. The idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

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Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.

Arde­lyx bags its first FDA OK for IBS, set­ting up a show­down with Al­ler­gan, Iron­wood

In the first of what it hopes will be a couple of major regulatory milestones for its new drug, Ardelyx has bagged an FDA approval to market Ibsrela (tenapanor) for irritable bowel syndrome.

The drug’s first application will be for IBS with constipation (IBS-C), inhibiting sodium-hydrogen exchanger NHE3 in the GI tract in such a way as to increase bowel movements and decrease abdominal pain. This comes on the heels of two successful Phase III trials.

Ver­tex deal for Scot­land — no deal for Eng­land

Cystic fibrosis (CF) drug maker Vertex Pharmaceuticals — which is still locked in negotiation with NHS England to endorse the use of its medicines — has successfully negotiated a deal with Scottish authorities.

A month ago, the Scottish Medicines Consortium spurned two of the company’s medicines — Orkambi and Symkevi — citing uncertainty over their long-term efficacy in relation to their cost.

Tony Kulesa, Brian Baynes. Petri

Star founders, in­ves­ti­ga­tors hud­dle around new Boston ac­cel­er­a­tor spot­light­ing young en­tre­pre­neurs

As a widely recognized biotech hub, Boston is undoubtedly one of the best places to start a new company at the frontier of biology and engineering. With a dense network of incubators, venture capitalists and talent, seasoned company founders can have their pick of partners and models launching their latest startups.

But for young, aspiring entrepreneurs, it’s a very different scene.

Big VC firms might hire you to work on their ideas instead of yours, and accelerators may not offer the kind of deep technical expertise and guidance needed to make it in the field.

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Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.