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.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA commissioner Stephen Hahn at the White House (AP Images)

Un­der fire, FDA to is­sue stricter guid­ance for Covid-19 vac­cine EUA this week — re­port

The FDA has been insisting for months that a Covid-19 vaccine had to be at least 50% effective – a measure of transparency meant to shore public trust in the agency and in a vaccine that had been brought forward at record speed and record political pressure. But now, with concerns of a Trump-driven authorization arriving before the election, the agency may be raising the bar.

The FDA is set to release new guidance that would raise safety and efficacy requirements for a vaccine EUA above earlier guidance and above the criteria used for convalescent plasma or hydroxychloroquine, The Washington Post reported. Experts say this significantly lowers the odds of an approval before the election on November 3, which Trump has promised despite vocal concerns from public health officials, and could help shore up public trust in the agency and any eventual vaccine.

Secretary of health and human services Alex Azar speaking in the Rose Garden at the White House (Photo: AFP)

Trump’s HHS claims ab­solute au­thor­i­ty over the FDA, clear­ing path to a vac­cine EUA

The top career staff at the FDA has vowed not to let politics overrule science when looking at vaccine data this fall. But Alex Azar, who happens to be their boss’s boss, apparently won’t even give them a chance to stand in the way.

In a new memorandum issued Tuesday last week, the HHS chief stripped the FDA and other health agencies under his purview of their rule making ability, asserting all such power “is reserved to the Secretary.” Sheila Kaplan of the New York Times first obtained and reported the details of the September 15 bulletin.

Vas Narasimhan (AP Images)

UP­DAT­ED: Still held down by clin­i­cal hold, No­var­tis' Zol­gens­ma falls fur­ther be­hind Bio­gen and Roche as FDA asks for a new piv­otal study

Last October, the FDA slowed down Novartis’ quest to extend its gene therapy to older spinal muscular atrophy patients by slapping a partial hold on intrathecal administration. Almost a year later, the hold is still there, and regulators are adding another hurdle required for regulatory submission: a new pivotal confirmatory study.

The new requirement — which departs significantly from Novartis’ prior expectations — will likely stretch the path to registration beyond 2021, when analysts were expecting a BLA submission. That could mean more time for Biogen to reap Spinraza revenues and Roche to ramp up sales of Evrysdi in the absence of a rival.

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Scoop: ARCH’s Bob Nelsen is back­ing an mR­NA up­start that promis­es to up­end the en­tire man­u­fac­tur­ing side of the glob­al busi­ness

For the past 2 years, serial entrepreneur Igor Khandros relied on a small network of friends and close insiders to supply the first millions he needed to fund a secretive project to master a new approach to manufacturing mRNA therapies.

Right now, he says, he has a working “GMP-in-a-box” prototype for a new company he’s building — after launching 3 public companies — which plans to spread this contained, precise manufacturing tech around the world with a set of partners. He’s raised $60 million, recruited some prominent experts. And not coincidentally, he’s going semi-public with this just as a small group of pioneers appears to be on the threshold of ushering in the world’s first mRNA vaccines to fight a worldwide pandemic.

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Isaac Veinbergs, Libra CEO

With $29M in Se­ries A, Boehringer-backed Li­bra looks to tack­le neu­rode­gen­er­a­tion through cel­lu­lar clean­ing

Can the natural process by which cells clean out toxic proteins be harnessed to create potential treatments for neurodegenerative disorders?

That’s the question Libra Therapeutics will be trying to answer, as the new biotech officially launched Wednesday morning with $29 million in Series A financing. The company has three preclinical programs at the ready, with its lead candidate targeting ALS and frontotemporal dementia. But CEO Isaac Veinbergs said he hopes to develop therapies for a wide range of diseases, including Parkinson’s, Alzheimer’s and Huntington’s.

Patrick Enright, Longitude co-founder (Longitude)

As its biotechs hit the pan­dem­ic ex­it, Lon­gi­tude rais­es $585M for new neu­ro, can­cer, ag­ing and or­phan-fo­cused fund

The years have been kind to Longitude Capital. This year, too.

A 2006 spinout of Pequot Capital, its founders started their new firm just four years before the parent company would go under amid insider trading allegations. Their first life sciences fund raised $325 million amid the financial crisis, they added a second for $385 million and then in, 2016, a third for $525 million. In the last few months, the pandemic biotech IPO boom netted several high-value exits from those funds, as Checkmate, Vaxcyte, Inozyme and Poseida all went public.

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Gene Wang, Immetas co-founder and CEO (file photo)

Im­metas Ther­a­peu­tics nabs $11M Se­ries A to nar­row their bis­pe­cif­ic work tar­get­ing in­flam­ma­tion in age-re­lat­ed dis­eases

How does a biotech celebrate its two-year anniversary? For Immetas Therapeutics, it’s with an $11 million Series A round and a game plan to fight age-related disease.

Co-founders Gene Wang and David Sinclair came together years ago around the idea that inflammation is the ultimate process driving age-related illnesses, including cancer. The duo launched Immetas in 2018 and packed the staff with industry experts. Wang, who says he’s always had an entrepreneurial spirit, has held lead roles at Novartis, GSK, Bristol Myers Squibb and Merck. He’s worked on blockbuster drugs like Humira, Gardasil, Varubi and Zolinza. And now, he’s channeling that spirit as CEO.

News brief­ing: Tiny Vac­cinex's drug flops in PhII Hunt­ing­ton's tri­al, stock craters; Siol­ta nabs $30M Se­ries B to de­vel­op mi­cro­bio­me drug

Siolta Therapeutics, a microbiome company targeting allergic diseases, raked in a $30 million Series B to develop its lead candidate, STMC-103H. The drug, which has been FDA fast-tracked, is headed for proof-of-concept trials, according to the company. Its various indications include allergic asthma, food allergies, atopic dermatitis, allergic rhinitis, and allergy prevention.

The news comes just after the California-based biotech added a prominent biopharma veteran as an advisor: 20-year Gilead CEO John Martin. The biotech also gained Richard Shames as CMO, who came by way of Protagonist Therapeutics.