Sec­ond piv­otal study bodes well for VBI's hep B vac­cine

In­vestors of VBI Vac­cines were unim­pressed last year when the com­pa­ny’s hep B vac­cine fell short in com­par­i­son to GSK’s es­tab­lished En­ger­ix-B in a key late-stage study. On Thurs­day, the Cam­bridge, Mass­a­chu­setts-based com­pa­ny un­veiled da­ta from a sec­ond piv­otal tri­al that ap­pears to ame­lio­rate some of those con­cerns.

VBI’s vac­cine, Sci-B-Vac, has al­ready se­cured ap­proval in Is­rael and ten oth­er coun­tries. It is en­gi­neered to mim­ic all three sur­face anti­gens of the hep B virus to in­duce a po­tent im­mune re­sponse and is be­ing de­vel­oped to work at low­er dos­es than ex­ist­ing hep B vac­cines.

The sec­ond piv­otal study, dubbed CON­STANT, was de­signed to as­sess the safe­ty and im­muno­genic­i­ty of Sci-B-Vac ver­sus En­ger­ix-B, as well as eval­u­ate the lot-to-lot man­u­fac­tur­ing con­sis­ten­cy of Sci-B-Vac.

The main goal of the 2,838-sub­ject study — man­u­fac­tur­ing con­sis­ten­cy — was met. The key sec­ondary end­point, to achieve non-in­fe­ri­or im­muno­genic­i­ty as mea­sured by sero­pro­tec­tion rates (SPR) at day 196, one month af­ter com­ple­tion of the full course of vac­ci­na­tion with ei­ther Sci-B-Vac or En­ger­ix-B, was al­so achieved, the com­pa­ny said.

At day 196, the pooled SPR in sub­jects who re­ceived Sci-B-Vac was 99.3% com­pared to 94.8% for those on En­ger­ix-B – a dif­fer­ence of 4.49% (95% con­fi­dence in­ter­val [2.90%, 6.63%]). Pre­vi­ous­ly, on day 168 (af­ter two vac­ci­na­tions on­ly) sub­jects on Sci-B-Vac were at 90.4%, while those on En­ger­ix-B were at 51.6%.

VBI’s shares $VBIB jumped about 24% to $1.69 in morn­ing trad­ing.

The com­pa­ny plans to use CON­STANT da­ta, along with the re­sults of the PRO­TECT study, to sub­mit mar­ket­ing ap­pli­ca­tions in the Unit­ed States, Cana­da, and Eu­rope, the sec­ond half of this year.

Da­ta from the 1,607-sub­ject PRO­TECT tri­al were an­nounced in June 2019. In the study, Sci-B-Vac failed to show that two dos­es of the drug were equal­ly ef­fec­tive as three dos­es of En­ger­ix-B in the study — a key sec­ondary end­point. How­ev­er, the VBI vac­cine was found to be as pro­tec­tive as En­ger­ix-B at the end of the third vac­ci­na­tion — meet­ing the main goal of the study.

VBI must al­so con­tend with an­oth­er ri­val. Af­ter be­ing linked to a string of in­ex­plic­a­ble safe­ty is­sues, Dy­navax’s hep B vac­cine Hep­lisav-B was fi­nal­ly ap­proved in No­vem­ber 2017 — but de­spite its check­ered path to the fin­ish line, the vac­cine’s ef­fi­ca­cy and con­ve­nience pro­file ap­pear to su­per­sede En­ger­ix-B.

Hep B is trans­mit­ted through con­tact with blood and bod­i­ly flu­ids of an in­fect­ed per­son and is a high­ly in­fec­tious virus, and is 50 to 100 times more in­fec­tious than HIV — even slight laps­es in in­fec­tion con­trol can re­sult in pa­tient-to-pa­tient trans­mis­sion. Since the ear­ly 1990’s the Unit­ed States has im­ple­ment­ed vac­ci­na­tion pro­grams in new­born chil­dren, but those born be­fore re­main sus­cep­ti­ble. CDC da­ta now sug­gests 95% per­cent of new hep B in­fec­tions in the Unit­ed States oc­cur in adults, as there is no stan­dard­ized sys­tem for vac­ci­nat­ing peo­ple af­ter school age, and par­tic­u­lar­ly since spikes in in­jec­tion drug use may be lead­ing to acute out­breaks.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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When ef­fi­ca­cy is bor­der­line: FDA needs to get more con­sis­tent on close-call drug ap­provals, agency-fund­ed re­search finds

In the exceedingly rare instances in which clinical efficacy is the only barrier to a new drug’s approval, new FDA-funded research from FDA and Stanford found that the agency does not have a consistent standard for defining “substantial evidence” when flexible criteria are used for an approval.

The research comes as the FDA is at a crossroads with its expedited-review pathways. The accelerated approval pathway is under fire as the agency recently signed off on a controversial new Alzheimer’s drug, with little precedent to explain its decision. Meanwhile, top officials like Rick Pazdur have called for a major push to simplify and clarify all of the various expedited pathways, which have grown to be must-haves for sponsors of nearly every newly approved drug.

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Ted White, Verrica CEO

Ver­ri­ca hits an­oth­er bump in the road with CMO re­lat­ed let­ter from FDA

The FDA has rejected Verrica’s new drug application for VP-102 again, with the company pinning the CRL on problems at a CMO that it was partnered with, the company announced Monday.

The FDA didn’t raise issues that directly relate to the manufacturing of VP-102, the company said, but raised “general quality issues” at the CMO’s facility. There were also no clinical concerns, it said, or need to collect more data.

Jay Bradner (Jeff Rumans for Endpoints News)

Div­ing deep­er in­to in­her­it­ed reti­nal dis­or­ders, No­var­tis gob­bles up an­oth­er bite-sized op­to­ge­net­ics biotech

Right about a year ago, a Novartis team led by Jay Bradner and Cynthia Grosskreutz at NIBR swooped in to scoop up a Cambridge, MA-based opthalmology gene therapy company called Vedere. Their focus was on a specific market niche: inherited retinal dystrophies that include a wide range of genetic retinal disorders marked by the loss of photoreceptor cells and progressive vision loss.

But that was just the first deal that whet their appetite.

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Take­da snaps up the Japan­ese rights to an old Shire cast-off; Boehringer In­gel­heim ac­quires Abexxa Bi­o­log­ics

A week before the FDA is set to decide on Mirum Pharmaceuticals’ lead liver disease drug — an old Shire cast-off called maralixibat — Takeda is swooping in to secure the rights in Japan.

Maralixibat’s roots trace back to Lumena, which was snapped up by Shire for $260 million-plus back in 2014. While the candidate had failed mid-stage studies at Shire, Mirum believes better trial design and patient selection will deliver the wins it needs. The drug is currently in development for Alagille syndrome (a condition called ALGS in which bile builds up in the liver), progressive familial intrahepatic cholestasis (PFIC, which causes progressive liver disease) and biliary atresia (a blockage in the ducts that carry bile from the liver to the gallbladder).

Vicente Anido (University of West Virginia via YouTube)

Aerie fires CEO af­ter lead pro­gram flop, com­ments about pri­ma­ry end­points be­ing 'not re­quired'

Aerie Pharmaceuticals CEO Vicente Anido has left the company less than a week after trying to chart a Phase III study in the wake of a serious Phase IIb flop.

Anido’s last day at Aerie was Friday, the biotech announced in a news release Tuesday morning, and Benjamin McGraw is taking his place in an interim role. The now former CEO was terminated without cause, according to an SEC filing.

The board has started looking for a full-time chief to take his place.

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