GW Phar­ma de­tails an im­pres­sive PhI­II case for a cannabis-based drug for se­vere epilep­sy as it preps an NDA

Or­rin Devin­sky, NYU Lan­gone Med­ical Cen­ter

GW Phar­ma­ceu­ti­cals lined up a straight shot at a key ap­proval for Epid­i­olex to­day, pub­lish­ing its im­pres­sive Phase III da­ta for a se­vere form of child­hood epilep­sy which in­spired a block­buster fore­cast for the cannabis-based ther­a­py.

GW an­nounced last spring that Epid­i­olex — a liq­uid ther­a­py us­ing pu­ri­fied cannaba­di­ol — trig­gered a mean re­duc­tion in con­vul­sive seizures of 39% among treat­ment-re­sis­tant pa­tients with Dravet syn­drome, com­pared to on­ly 13% among the place­bo group. A re­port pub­lished in The New Eng­land Jour­nal of Med­i­cine adds to­day that 43% of the drug group had a mean drop of 50% or more — com­pared to 27% in the place­bo group. And 5% stopped hav­ing seizures, with none in the place­bo arm do­ing as well.

GW Phar­ma $GW­PH now plans to file for an FDA ap­proval some­time in the com­ing weeks, with an­a­lysts pro­ject­ing peak sales over a bil­lion dol­lars. GW has had suc­cess­ful tri­als for both Dravet syn­drome as well as an­oth­er rare form of epilep­sy called Lennox-Gas­taut syn­drome, with plans to file for ap­provals on both.

GW’s stock jumped 4.4% on the pub­li­ca­tion of the da­ta, which helps ver­i­fy their re­sults.

We al­so got a much bet­ter look at the safe­ty da­ta in the NE­JM ar­ti­cle. A to­tal of 93% of the pa­tients tak­ing the drug ex­pe­ri­enced a side ef­fect, though most were mild or mod­er­ate. AEs were reg­is­tered for som­no­lence, di­ar­rhea, de­creased ap­petite, fa­tigue, vom­it­ing, pyrex­ia, lethar­gy, con­vul­sion and up­per res­pi­ra­to­ry tract in­fec­tions. Ten pa­tients in the drug arm dropped out of the study, com­pared to 3 pa­tients on place­bo.

Most se­ri­ous­ly, 12 pa­tients ex­pe­ri­enced a spike in liv­er en­zymes — a clas­sic red flag on tox­i­c­i­ty — and 4 dropped out of the study. Of the oth­er 9 who stayed, all saw liv­er en­zyme lev­els re­turn to nor­mal.

Leerink’s Paul Mat­teis, though, sound­ed a note of con­cern about two is­sues that could trip up GW Phar­ma. In a note to in­vestors, he wrote:

From our con­ver­sa­tions with in­vestors we’ve heard two bear points on Epid­i­olex ahead of an NDA fil­ing: (1) that clobazam may have been a ma­jor con­trib­u­tor to Epid­i­olex’s ef­fi­ca­cy [and that this could ul­ti­mate­ly prove to be im­por­tant to or prob­lem­at­ic for FDA]; and (2) that liv­er en­zyme el­e­va­tions on Epid­i­olex could in part hin­der broad use, pos­si­bly via un­fa­vor­able la­bel­ing lan­guage. We don’t re­al­ly un­der­stand the lat­ter con­cern since val­proate – an an­ti-epilep­tic with a black box warn­ing for he­pa­to­tox­i­c­i­ty and more se­ri­ous liv­er en­zyme is­sues than ob­served in GW’s ph3 pro­gram – is broad­ly used in the re­frac­to­ry epilep­sy set­ting. How­ev­er, re­gard­ing the clobazam drug-drug in­ter­ac­tion, seizure re­duc­tion sub-group analy­ses re­main of in­ter­est. GW has sug­gest­ed that these analy­ses could be pre­sent­ed be­fore a po­ten­tial FDA ad­vi­so­ry com­mit­tee but spe­cif­ic guid­ance has not been com­mu­ni­cat­ed.

“Dravet syn­drome is one of the most dif­fi­cult types of epilep­sy to treat and many of the chil­dren in this study were ex­pe­ri­enc­ing dozens, even hun­dreds, of seizures per month de­spite tak­ing mul­ti­ple con­cur­rent an­ti-epilep­tic med­ica­tions,” said Or­rin Devin­sky, the lead au­thor from the NYU Lan­gone Med­ical Cen­ter’s Com­pre­hen­sive Epilep­sy Cen­ter. “These re­sults sug­gest that Epid­i­olex can pro­vide clin­i­cal­ly mean­ing­ful ben­e­fits and I look for­ward to the prospect of an ap­pro­pri­ate­ly stan­dard­ized and test­ed phar­ma­ceu­ti­cal for­mu­la­tion of cannabid­i­ol avail­able as a treat­ment op­tion for these pa­tients.”

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,200+ biopharma pros reading Endpoints daily — and it's free.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

FDA de­lays de­ci­sion on No­var­tis’ po­ten­tial block­buster MS drug, wip­ing away pri­or­i­ty re­view

So much for a speedy review.

In February, Novartis announced that an application for their much-touted multiple sclerosis drug ofatumumab had been accepted and, with the drug company cashing in on one of their priority review vouchers, the agency was due for a decision by June.

But with June less than 48 hours old, Novartis announced the agency has extended their review, pushing back the timeline for approval or rejection to September. The Swiss pharma filed the application in December, meaning their new schedule will be nearly in line with the standard 10-month window period had they not used the priority voucher.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,200+ biopharma pros reading Endpoints daily — and it's free.

Credit: AP Images

Covid-19 roundup: BAR­DA sup­ports Op­er­a­tion Warp Speed with big $628M con­tract to ser­vice Amer­i­ca's vac­cine pro­duc­tion needs

Another BARDA contract designed to service America’s Covid-19 vaccine needs has been deployed.

The White House-led initiative designed to bankroll development to bring a vaccine to the American public by this fall — Operation Warp Speed — has via BARDA handed a meaty contract to the maker of an FDA-licensed anthrax vaccine to open up its manufacturing apparatus to shore up production of Covid-19 vaccines.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,200+ biopharma pros reading Endpoints daily — and it's free.

Ken Frazier, AP Images

Why Mer­ck wait­ed, and what they now bring to the Covid-19 fight

Nicholas Kartsonis had been running clinical infectious disease research at Merck for almost 2 years when, in mid-January, he got a new assignment: searching the pharma giant’s vast libraries for something that could treat the novel coronavirus.

The outbreak was barely two weeks old when Kartsonis and a few dozen others got to work, first in small teams and then in a larger task force that sucked in more and more parts of the sprawling company as Covid-19 infected more and more of the globe. By late February, the group began formally searching for vaccine and antiviral candidates to license. Still, while other companies jumped out to announce their programs and, eventually and sometimes controversially, early glimpses at human data, Merck remained silent. They made only a brief announcement about a data collection partnership in April and mentioned vaguely a vaccine and antiviral search in their April 28 earnings call.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 82,200+ biopharma pros reading Endpoints daily — and it's free.

A low-pro­file biotech bests Re­gen­eron in high-pro­file patent suit

For nearly a decade now, the low-profile Cambridge biotech Kymab has been battling in US, UK, Japanese and Australian courts with the biotech behemoth Regeneron.

Regeneron has turned itself into a $70 billion company off of a platform of transgenically humanized mice they can use to make antibodies for anything from Ebola to colorectal cancer. The technology took decades and billions to build, 20 years from the company’s founding to the first approved drug. And the company guards and touts it zealously, breaking their production process down into various branded components — Velocimmune, Velocigene, Velocimouse and four other Velocis — and sometimes suing would-be copycats. In 2014, most notably, they sued two Pfizer-backed entities for patent infringement.