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.

Com­mu­ni­cat­ing the val­ue of pre­ci­sion med­i­cine

By Natasha Cowan, Content Marketing Manager at Blue Latitude Health.
Many stakeholders are confused by novel precision medicines, including patients and healthcare professionals. So, how can industry help them to navigate this complexity?

Precision medicine represents a new paradigm in healthcare. It embodies the shift from treating many patients with the same therapy, to having the tools to identify the best treatment for every patient.

(Image: Associated Press)

Amarin emerges from an ex­pert pan­el re­view with a clear en­dorse­ment for Vas­cepa and high odds of suc­cess when the FDA weighs in for­mal­ly

Several FDA experts who gathered Thursday to consider the landmark approval of Vascepa to reduce cardio events in an at-risk population voiced their unease about various aspects of the efficacy and safety data, or ultimately the population it should be used to treat. But the overwhelming belief that the data pointed to the drug’s benefit and clearly outweighed risks carried the day for Amarin.

The panel voted unanimously (16 to 0) to support the company’s positive data presentation — backing an OK for expanding the label to include reducing cardio risk. The vote points Amarin $AMRN down a short path to a formal decision by the FDA, with the odds heavily in its favor. Chances are the rest of the questions about the future of this drug will be hashed out in the label’s small print.

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What does $62B buy you these days? A lot, says Take­da ex­ecs as the phar­ma play­er promis­es a block­buster R&D fu­ture

First comes the $62 billion buyout. Then comes the asset auction and reorganization to pay down debt. Now comes the detailed pledge of a bigger, brighter future in drug development.

That’s where Takeda finds itself on R&D day today, about 11 months after closing on their Shire acquisition. R&D chief Andy Plump is joining CEO Christophe Weber and other top members of the team to outline a new set of priorities in the greatly expanded pipeline at Takeda, which has jumped into the top ranks of the world’s pharma giants in the wake of the Shire deal.

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BeiGene CEO John Oyler at an Endpoints event in Shanghai, October 2018 (Credit: Endpoints News/PharmCube)

UP­DAT­ED: Chi­na's BeiGene scores first-ever FDA ap­proval — but can they carve up J&J's block­buster fran­chise?

Weeks after Amgen took a $2.7 billion stake in BeiGene, the Beijing-based biotech has secured its first-ever FDA approval for zanubrutinib, a BTK inhibitor, months ahead of schedule.

BeiGene’s drug, branded as Brukinsa, has secured accelerated approval for adult patients with mantle cell lymphoma (MCL) — a typically aggressive, rare, form of blood cancer — who have received at least one prior therapy.

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Spe­cial re­port: Twen­ty ex­tra­or­di­nary women in bio­phar­ma R&D who worked their way to the top

What differentiates a woman leader in biopharma R&D from a man?

Not much, except there are fewer of them in senior posts. Data suggest women are not more risk-averse, family-oriented or less confident than their male counterparts — indeed the differences between the two sexes are negligible. But a glance at the top R&D positions in Big Pharma leaves little doubt that upward migration in the executive ranks of biopharma R&D is tough.

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GSK's asth­ma bi­o­log­ic Nu­cala scores in rare blood dis­or­der study

GlaxoSmithKline’s asthma drug Nucala, which received a resounding FDA rejection for use in chronic obstructive pulmonary disease (COPD) last year, has shown promise in a rare blood disorder.

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Mer­ck buys a fledg­ling neu­rode­gen­er­a­tive biotech spawned by an old GSK dis­cov­ery al­liance. What’s up with that?

Avalon Ventures chief Jay Lichter has a well-known yen for drug development programs picked up in academia. And what he found in Haoxing Xu’s lab at the University of Michigan pricked his interest enough to launch one of his umbrella biotechs in San Diego.

Xu’s work laid the foundation for Avalon to launch Calporta, which has been working on finding small molecule agonists of TRPML1 (transient receptor potential cation channel, mucolipin subfamily, member 1) for lysosomal storage disorders. And that pathway, they believe, points to new approaches on major market neurodegenerative diseases like Parkinson’s, ALS and Alzheimer’s.

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Carson Block. Muddy Waters via YouTube

Shorts ga­lore: Mud­dy Wa­ters sees slide for Pep­tiDream, tweets con­cerns about Fi­bro­Gen's new da­ta

The short seller Muddy Waters is taking aim at Japan’s most profitable biotech, projecting a slide for a company that has skyrocketed over the last four years. Meanwhile, the firm tweeted out an analysis accusing FibroGen of manipulating data to obscure safety concerns in their latest reveal, although some investors seem satisfied by the biotech’s explanation.

Muddy Waters shorted PeptiDream, a Japanese biotech-for-hire that leveraged its peptide library into partnerships with some of the world’s largest pharmaceutical companies, a 50% profit margin and $6 billion valuation. The firm noted that despite its esteem, PeptiDream has failed to bring a drug to market 13 years after its 2006 launch (although this is not especially rare for biotech).

Pin­cer move­ment: Cal­i­for­nia biotech gets $35M to suf­fo­cate can­cer in co­or­di­nat­ed at­tack

Having served in Afghanistan, the navy veteran leading California-based EpicentRx wants to leave no patient behind with his arsenal of anti-cancer drugs. On Thursday, the company was given a $35 million boost to further its mission.

The injection of funds will be used to shepherd its late-stage CD47 drug, RRx-001, to the FDA for marketing, and its oncolytic virus program into the clinic.

RRx-001, engineered as an agent that makes tumor cells more sensitive to therapy, is in a Phase III trial in combination with chemotherapy for use in third-line and beyond small cell lung cancer (SCLC). The drug has been granted orphan drug designation from FDA for SCLC, neuroendocrine cancer and glioblastoma.