World Health Organization Director-General Tedros Adhanom Ghebreyesus attends a press conference following an emergency committee meeting over Ebola epidemic in Democratic Republic of Congo at the WHO headquarters in Geneva on October 18, 2019. (via Getty Images)

As Con­go Ebo­la out­break fes­ters, FDA ap­proves first vac­cine to com­bat dead­ly virus

As an Ebo­la out­break rages on in the De­mo­c­ra­t­ic Re­pub­lic of Con­go — the FDA has ap­proved the first vac­cine to pro­tect against the dead­ly he­m­or­rhag­ic virus, months ear­li­er than ex­pect­ed. Man­u­fac­tured by Mer­ck the de­vel­op­ment of the vac­cine, chris­tened Erve­bo, be­gan dur­ing the West African out­break that oc­curred be­tween 2014 and 2016, which killed more than 11,000.

The ap­proval for the ge­net­i­cal­ly en­gi­neered at­ten­u­at­ed live vac­cine comes about five weeks af­ter its Eu­ro­pean en­dorse­ment. Mer­ck ex­pects to launch the vac­cine in the third quar­ter of 2020.

“We have not yet es­tab­lished a price for ERVE­BO. Mer­ck has com­mit­ted to mak­ing the vac­cine avail­able to Gavi (the Vac­cine Al­liance)-el­i­gi­ble coun­tries at the low­est pos­si­ble ac­cess price,” a com­pa­ny spokesper­son told End­points News.

Erve­bo has been test­ed in rough­ly 16,000 in­di­vid­u­als aged 18 and above in tri­als con­duct­ed in Africa, Eu­rope, and the Unit­ed States, and da­ta show the vac­cine is ef­fec­tive against the Zaire Ebo­la virus that cir­cu­lat­ed in West Africa in 2014-2016, as well as the cur­rent out­break in DRC.

In the Guinea tri­al, 3,537 con­tacts, and con­tacts of con­tacts, of in­di­vid­u­als with lab­o­ra­to­ry-con­firmed Ebo­la virus dis­ease re­ceived ei­ther “im­me­di­ate” or 21-day “de­layed” vac­ci­na­tion with Erve­bo. Some 2,108 in­di­vid­u­als were in the “im­me­di­ate” vac­ci­na­tion arm and 1,429 were in the “de­layed” vac­ci­na­tion group, and the re­sults show that Erve­bo was found to be 100% ef­fec­tive in pre­vent­ing Ebo­la cas­es with symp­tom on­set greater than 10 days af­ter vac­ci­na­tion. No Ebo­la cas­es with symp­tom on­set greater than 10 days af­ter vac­ci­na­tion were ob­served in the im­me­di­ate clus­ter group, com­pared with 10 such Ebo­la cas­es in the 21-day de­layed clus­ter group.

An­ti­body re­spons­es to Erve­bo were as­sessed in 477 in­di­vid­u­als in Liberia, rough­ly 500 in Sier­ra Leone and about 900 in­di­vid­u­als in Cana­daSpain, and the Unit­ed States — across ge­og­ra­phy, the an­ti­body re­spons­es were sim­i­lar.

Ebo­la is thought to have in­fil­trat­ed the hu­man pop­u­la­tion through close con­tact with the blood, se­cre­tions, or­gans or oth­er bod­i­ly flu­ids of in­fect­ed an­i­mals such as fruit bats, chim­panzees, go­ril­las, mon­keys, for­est an­te­lope or por­cu­pines. Even­tu­al­ly, it spread be­tween hu­mans through di­rect con­tact, via the blood or bod­i­ly flu­ids of an in­fect­ed per­son.

The cur­rent out­break in the De­mo­c­ra­t­ic Re­pub­lic of Con­go has shown case fa­tal­i­ty rates of more than 65%, the WHO has es­ti­mat­ed. Al­though there has been a re­cent spike in in­fec­tions, the cur­rent in­fec­tion rates are well be­low the 120 cas­es a week re­port­ed dur­ing the peak of the out­break in late April. On Thurs­day, the WHO said it had record­ed the first Ebo­la re­lapse in the cur­rent epi­dem­ic.

Ear­li­er this year the WHO de­clared the out­break — which so far has in­fect­ed more than 3,350 peo­ple — a pub­lic health emer­gency of in­ter­na­tion­al con­cern. As of De­cem­ber 17, over a quar­ter of all con­firmed Ebo­la cas­es in the DRC have been in chil­dren aged less than 18 years. A Mer­ck spokesper­son said the com­pa­ny has stud­ies un­der­way test­ing Erve­bo (same for­mu­la­tion, same dose) in chil­dren.

The vac­cine was ini­tial­ly en­gi­neered by sci­en­tists from the Pub­lic Health Agency of Cana­da’s Na­tion­al Mi­cro­bi­ol­o­gy Lab­o­ra­to­ry and sub­se­quent­ly li­censed to a unit of NewLink Ge­net­ics Corp. In 2014, as the virus wreaked hav­oc in West Africa, Mer­ck ac­quired the tech­nol­o­gy from NewLink (Bat­tered by a se­ries of set­backs, Newlink in Oc­to­ber be­came the ve­hi­cle for a re­verse merg­er).

In re­sponse to re­quests from the WHO, since May 2018, the large US drug­mak­er has do­nat­ed 275,000 dos­es of the vac­cine to com­bat the out­break in the DRC. With the FDA ap­proval, Mer­ck has al­so qual­i­fied for a trop­i­cal dis­ease pri­or­i­ty re­view vouch­er (PRV) — these vouch­ers, used to has­ten drug re­views, have been sold in the past for mil­lions, rang­ing from $95 mil­lion to even $350 mil­lion.

The rel­a­tive­ly few Ebo­la cas­es in the Unit­ed States have been due to in­ter­na­tion­al trav­el­ers or health care work­ers who have con­tract­ed the virus af­ter treat­ing Ebo­la pa­tients.

“While the risk of Ebo­la virus dis­ease in the U.S. re­mains low, the U.S. gov­ern­ment re­mains deeply com­mit­ted to fight­ing dev­as­tat­ing Ebo­la out­breaks in Africa, in­clud­ing the cur­rent out­break in the De­mo­c­ra­t­ic Re­pub­lic of the Con­go,” said An­na Abram, FDA Deputy Com­mis­sion­er for Pol­i­cy, Leg­is­la­tion, and In­ter­na­tion­al Af­fairs, in a state­ment.

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 VOYAGER 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.