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.

2019 Trin­i­ty Drug In­dex Eval­u­ates Ac­tu­al Com­mer­cial Per­for­mance of Nov­el Drugs Ap­proved in 2016

Fewer Approvals, but Neurology Rivals Oncology and Sees Major Innovations

This report, the fourth in our Trinity Drug Index series, outlines key themes and emerging trends in the industry as we progress towards a new world of targeted and innovative products. It provides a comprehensive evaluation of the performance of novel drugs approved by the FDA in 2016, scoring each on its commercial performance, therapeutic value, and R&D investment (Table 1: Drug ranking – Ratings on a 1-5 scale).

UP­DAT­ED: FDA’s golodirsen CRL: Sarep­ta’s Duchenne drugs are dan­ger­ous to pa­tients, of­fer­ing on­ly a small ben­e­fit. And where's that con­fir­ma­to­ry tri­al?

Back last summer, Sarepta CEO Doug Ingram told Duchenne MD families and investors that the FDA’s shock rejection of their second Duchenne MD drug golodirsen was due to some concerns regulators raised about the risk of infection and the possibility of kidney toxicity. But when pressed to release the letter for all to see, he declined, according to a report from BioPharmaDive, saying that kind of move “might not look like we’re being as respectful as we’d like to be.”

He went on to assure everyone that he hadn’t misrepresented the CRL.

But Ingram’s public remarks didn’t include everything in the letter, which — following the FDA’s surprise about-face and unexplained approval — has now been posted on the FDA’s website and broadly circulated on Twitter early Wednesday.

The CRL raises plenty of fresh questions about why the FDA abruptly decided to reverse itself and hand out an OK for a drug a senior regulator at the FDA believed — 5 months ago, when he wrote the letter — is dangerous to patients. It also puts the spotlight back on Sarepta $SRPT, which failed to launch a confirmatory study of eteplirsen, which was only approved after a heated internal controversy at the FDA. Ellis Unger, director of CDER’s Office of Drug Evaluation I, notes that study could have clarified quite a lot about the benefit and risks associated with their drugs — which can cost as much as a million dollars per patient per year, depending on weight.

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How to cap­i­talise on a lean launch

For start-up biotechnology companies and resource stretched pharmaceutical organisations, launching a novel product can be challenging. Lean teams can make setting a launch strategy and achieving your commercial goals seem like a colossal undertaking, but can these barriers be transformed into opportunities that work to your brand’s advantage?
We spoke to Managing Consultant Frances Hendry to find out how Blue Latitude Health partnered with a fledgling subsidiary of a pharmaceutical organisation to launch an innovative product in a
complex market.
What does the launch environment look like for this product?
FH: We started working on the product at Phase II and now we’re going into Phase III trials. There is a significant unmet need in this disease area, and everyone is excited about the launch. However, the organisation is still evolving and the team is quite small – naturally this causes a little turbulence.

Wuhan virus out­break trig­gers in­evitable small-biotech ral­ly

Every few years, a public health crisis (think Ebola, Zika) spurred by a rogue pathogen triggers a small-biotech rally, as drugmakers emerge from the woodwork with ambitious plans to treat the mounting outbreak. In most cases, that enthusiasm never quite delivers.

Things are no different, as the coronavirus outbreak in Wuhan, China takes hold. There have been close to 300 confirmed human infections in China, and at least four deaths. Coronaviruses are a large family of viruses, which include MERS and SARS. On Tuesday, the CDC reported the virus was detected in a US traveler returning from Wuhan.

Brex­it fears, Wood­ford woes over­shad­owed UK biotech and cut 2019 fi­nanc­ing by al­most half

The venture tide might have subsided, the IPO window may be closing and certain listed biotechs may be having a tough time amid Neil Woodford’s well-publicized demised, but there’s still plenty to celebrate in the UK BioIndustry Association’s eyes.

Overall investment in UK biotech last year fell from the record-breaking £2.2 billion levels of 2018 to £1.3 billion — including £679 million in venture capital, a meager £64 million in IPOs plus £596 million when you add up all public financings, according to a new report from the BIA.

Blue­print Med­i­cines po­ten­tial­ly de­lays Ay­vak­it de­ci­sion; Con­trol beats treat­ment in mesothe­lioma tri­al

→ Blueprint Medicines filed an amendment to its application to get the gastrointestinal stromal tumor (GIST) drug Ayvakit approved in fourth-line GIST, the company disclosed in the prospectus for a new $325 million public offering.  Blueprint got a big accelerated OK on the drug this month in a particular mutation, but because the FDA decided to split their review in two, they didn’t hear on fourth-line GIST. They were supposed to hear before February 14, but this amendment could push that date back by 3 months. Blueprint wrote that the amendment is designed to allow the company to comply with the FDA’s request for data from the Phase III Voyage trial before they give a judgment.

Io­n­is, Akcea boost­ed by a pos­i­tive PhII for their No­var­tis castoff car­dio drug — and they plan to push ahead in­to piv­otals

Late last year Novartis abandoned a cardio drug from Ionis’ spinoff Akcea just after the pharma giant snapped up inclisiran, going the RNAi way in guarding against heart disease in the $9.7 billion Medco buyout.

Now the pharma goliath — which is headed down the PCSK9 road with a drug it believes can be used in a mass population — can get a clearer picture of just what they gave up.

Akcea $AKCA and the mother company $IONS put out a statement early Wednesday saying that their Phase II study of AKCEA-APOCIII-LR delivered solid efficacy data, with the high dose clearly outperforming placebo in significantly reducing triglycerides as a means to cutting the risk of cardiovascular disease. In addition, investigators concluded that the drug slashed apoC-III, very low-density lipoprotein and remnant cholesterol while boosting “good” HDL levels.

Hal Barron and Emma Walmsley, GSK

GSK’s ‘break­through’ BC­MA can­cer drug gets a pri­or­i­ty re­view — and a big win for the on­col­o­gy R&D team

After largely whiffing the past 2 years on the pharma R&D front, GlaxoSmithKline research chief Hal Barron has seized boasting rights to a key win that puts them back in the cancer drug development game.

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Who are the young bio­phar­ma lead­ers shap­ing the in­dus­try? Nom­i­nate them for End­points' spe­cial re­port

Update: Nominations open through end of day, Monday, January 27

Two years ago, when we did our first Endpoints 20-under-40, we profiled a set of up-and-comers who promised to help reshape the industry as we know it. Now we’re back and once again looking for the top 20 biopharma professionals under the age of 40. We’ll be profiling folks who have accomplished a lot at a young age but seem on the verge of accomplishing so much more.