Rob de Ree. Biogeneration Ventures

Fish oil pill for NASH? Dutch biotech scores $40M in­jec­tion to make a mark in the bustling field

If Amarin’s fish oil pill is any­thing to go by, Dutch drug de­vel­op­er North­Sea Ther­a­peu­tics is in for a ride in its quest to mar­ket its own omega 3 drug.

North­Sea’s com­pound, icos­abu­tate, was in-li­censed from Nor­way’s Prono­va Bio­Phar­ma — the de­vel­op­er of the block­buster omega 3 car­dio­vas­cu­lar drug Oma­cor that was swal­lowed by Ger­man chem­i­cals gi­ant BASF in 2013.

Akin to Amarin’s pill, icos­abu­tate was orig­i­nal­ly test­ed as a treat­ment for hy­per­triglyc­eridemia. North­Sea is fo­cused on test­ing it for NASH, a fat­ty liv­er dis­ease, which has lured an army of drug de­vel­op­ers but is a field lit­tered with fail­ure.

On Wednes­day, North­Sea se­cured $40 mil­lion in Se­ries B fi­nanc­ing, led by ven­Bio Part­ners, for its shot at the lu­cra­tive mar­ket that so far has no ap­proved drugs, but is ex­pect­ed to eclipse $20 bil­lion by 2025.

The funds will be used to com­plete an on­go­ing Phase IIb NASH study, which is ex­pect­ed to be com­plet­ed by 2021, as well as the de­vel­op­ment of two oth­er omega 3 com­pounds for use in dys­lip­i­daemia and an or­phan liv­er con­di­tion called PNALD (par­enter­al nu­tri­tion as­so­ci­at­ed liv­er dis­ease) re­spec­tive­ly.

Non-al­co­holic steato­hep­ati­tis, or NASH, is char­ac­ter­ized by a buildup of ex­cess fat in the liv­er that in­duces chron­ic in­flam­ma­tion and even­tu­al­ly cul­mi­nates in scar­ring that can lead to cir­rho­sis, liv­er fail­ure, can­cer and/or death. Dubbed the silent dis­ease, it is hard to di­ag­nose in the ear­ly stages, mak­ing it dif­fi­cult to es­ti­mate its preva­lence, but stud­ies show that it af­flicts up to 12% of the adult pop­u­la­tion in de­vel­oped coun­tries.

The first wave of NASH drug de­vel­op­ers have strug­gled with ef­fi­ca­cy as well as safe­ty. Com­pa­nies big and small have crashed and burned. Ma­jor con­tender In­ter­cept Phar­ma­ceu­ti­cals’ drug obeti­cholic acid, the on­ly ther­a­py so far with es­tab­lished an­tifi­brot­ic ac­tiv­i­ty, is plagued by tol­er­a­bil­i­ty lim­i­ta­tions. Gilead has lit­tle to show for its ef­forts, and France’s Gen­fit is ex­pect­ed to come out with its Phase III in­ter­im re­sults in the next few months.

Gen­fit has un­der­scored its drug’s abil­i­ty to low­er car­dio­vas­cu­lar risk — the lead­ing cause of mor­tal­i­ty in NASH pa­tients are CV events. North­Sea chief Rob de Ree is al­so gun­ning to prove his com­pound has a ben­e­fi­cial im­pact on the heart, giv­en its pre­vi­ous test­ing in triglyc­erides.

“We have shown in over 200 pa­tients in the lipid study that the (drug’s) safe­ty has an ex­cel­lent pro­file as well…hav­ing shown that we can re­duce triglyc­erides; that we al­so can re­duce cho­les­terol in this pa­tient pop­u­la­tion is very im­por­tant — most of these (NASH) pa­tients in stage II or III don’t die of liv­er dis­ease, but still die of car­dio­vas­cu­lar dis­ease,” de Ree said in an in­ter­view with End­points News. “We be­lieve that we have quite a com­plete pro­file and strong­ly dif­fer­en­ti­ate from the first wave of com­pounds.”

It is not nec­es­sar­i­ly a far fetched goal. Amarin’s Vas­cepa, known chem­i­cal­ly as icos­apent eth­yl, is an omega-3 fat­ty acid de­rived from fish oil that was orig­i­nal­ly en­dorsed by the US reg­u­la­tor as a treat­ment for el­e­vat­ed triglyc­erides. How­ev­er, last month the FDA sanc­tioned its use in the block­buster in­di­ca­tion of re­duc­ing car­dio­vas­cu­lar risk in pa­tients al­ready on statins fol­low­ing a land­mark tri­al — RE­DUCE-IT — which showed the pill trig­gered a 25% re­duc­tion in the risk for the first oc­cur­rence of a ma­jor car­dio event, and a 26% re­duc­tion in a com­pos­ite end­point of car­dio­vas­cu­lar death, non­fa­tal heart at­tack, and non­fa­tal stroke.

The lat­est in­jec­tion for North­Sea in­clud­ed the par­tic­i­pa­tion of new in­vestors Sofinno­va In­vest­ments, and ex­ist­ing in­vestors, For­bion, No­vo Seeds, New Sci­ence Ven­tures and Bio­Gen­er­a­tion Ven­tures. The com­pa­ny raised €25 mil­lion in se­ries A fund­ing back in De­cem­ber 2017.

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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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 is going from being one of the quietest players in the race to develop a new vaccine to fight the worst public health crisis in a century to a challenger for the multibillion-dollar market that awaits the first vaccines to make it over the finish line. Typically low-key at a time of brash comments and incredibly ambitious development timelines from the leaders, CureVac now is jumping straight into the spotlight.

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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.

US gov­ern­ment re­port­ed­ly be­gins prepar­ing for Covid-19 chal­lenge tri­als. Are they eth­i­cal?

Controversial human challenge trials for potential Covid-19 vaccines reportedly have a new booster — the US government.

Scientists working for the government have begun manufacturing a strain of the novel coronavirus that could be used in such studies, Reuters reported Friday morning. The trials would enroll healthy volunteers to be vaccinated and then intentionally infected with a weakened coronavirus.

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Trevor Martin (Mammoth)

Eye­ing in-vi­vo edit­ing, Mam­moth li­cens­es Jen­nifer Doud­na’s new CRISPR en­zyme

Last month, Jennifer Doudna revealed in Science a new, “hyper-compact” CRISPR enzyme that was half the size of traditional CRISPR enzymes and could, she suspected, offer a new, more versatile tool for gene editing.

Now, the University of California-Berkeley has licensed that enzyme, known as Casφ, exclusively to a biotech startup she and two former students set up three years ago: Mammoth Biosciences. It’s the second new CRISPR protein Mammoth has licensed from Doudna’s lab, after they licensed Cas14 in 2019.

Clockwise from left: Canaccord Genuity principal Michelle Gilson, Canaccord Genuity CSO Brian Mueller and BioMarin CSO Hank Fuchs (Canaccord Genuity webcast)

Bio­Marin CSO diss­es ri­vals for the he­mo­phil­ia A gene ther­a­py crown: Way be­hind, fac­ing big re­cruit­ment chal­lenges and at best a .6 on the gen-one scale

The leader in the race to a hemophilia A gene therapy does not like to be compared unfavorably to the competition. And when their top execs do the comparing, don’t look for any modesty — BioMarin, they say, owns the lead.

As Factor VIII expression wanes over time, quite a few analysts have raised questions about the kind of future BioMarin’s gene therapy — a supposed once-and-done treatment — faces if it stops working. But just 7 days away from their PDUFA date, with high odds of success, the top execs clearly feel that they are way out front, while promising their rivals will discover there’s a tough slog ahead trying to pursue trials where large numbers of patients are ineligible for new therapies.

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Sanofi vet Kather­ine Bowdish named CEO of PIC Ther­a­peu­tics; As the world Terns: Liv­er dis­ease biotech makes ex­ec­u­tive changes

PIC Therapeutics hasn’t raised much money, yet. But the fledgling biotech has attracted a high-profile player to the helm.

The Boston-based biotech has handed the reins to Katherine Bowdish as its president and CEO. Bowdish will also join the board of directors of PIC. Bowdish joins from Sanofi where she served as VP and head of R&D strategy, as well as helping launch and lead Sanofi Sunrise, a venture investment and partnering vehicle at Sanofi. Before that, Bowdish held several exec roles at Permeon Biologics, Anaphore, Alexion Pharmaceuticals and Prolifaron (acquired by Alexion).

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.

Stéphane Bancel speaks to President Donald Trump at the White House meeting on March 2 (AP Images)

UP­DAT­ED: Mod­er­na of­fers steep dis­count in US sup­ply deal — but still takes the crown with close to $2.5B in vac­cine con­tracts

The US pre-order for Moderna’s Covid-19 vaccine is in.

Operation Warp Speed is reserving $1.525 billion for 100 million doses of Moderna’s Phase III mRNA candidate, rounding out to about $15 per dose — including $300 million in incentive payments for timely delivery. Given that Moderna has a two-dose regimen, it’s good for vaccinating 50 million people. The US government also has the option to purchase another 400 million doses for a total of $6.6 billion, or $16.5 per dose.

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