3 for 3 in PhI­II: GW Phar­ma rac­ing to the FDA af­ter bag­ging an­oth­er late-stage epilep­sy suc­cess

Ex­tend­ing its win­ning streak, GW Phar­ma­ceu­ti­cals says the sec­ond Phase III for Epid­i­olex in treat­ing Lennox-Gas­taut syn­drome — a rare form of epilep­sy — has al­so come through with solid­ly pos­i­tive ef­fi­ca­cy da­ta. And the UK biotech is now plan­ning to hus­tle ahead with a new drug ap­pli­ca­tion at the FDA in the com­ing months.

Or­rin Devin­sky, New York Uni­ver­si­ty Lan­gone Med­ical Cen­ter

The da­ta from this sec­ond study on the com­pa­ny’s lead cannabi­noid ther­a­py were even bet­ter than the first, with a me­di­an 42% drop in month­ly seizures, com­pared to 17% in the place­bo group for a 20 mg dose. The 10 mg dose was al­so a suc­cess, with a 37% drop in the seizure rate.

Shares of GW Phar­ma $GW­PH shot up 15% on the lat­est suc­cess, which may well leave the com­pa­ny poised for a mar­ket launch.

Back in March the com­pa­ny ex­cit­ed in­vestors with re­sults for the first of four late-stage stud­ies on Epid­i­olex, with the drug re­duc­ing con­vul­sions in chil­dren with treat­ment-re­sis­tant Dravet syn­drome by a me­di­an av­er­age of 39% com­pared to on­ly 13% in the place­bo arm. That da­ta helped in­spire block­buster-sized peak sales es­ti­mates for this drug.

GW Phar­ma had a pre-NDA meet­ing with the FDA last sum­mer, af­ter it racked up the pos­i­tive Phase III study for Dravet syn­drome. Now it plans to hus­tle along an NDA for both Dravet and LGS at the same time, rather than wait for an­oth­er round of late-stage da­ta on Dravet syn­drome

Stat­ed Or­rin Devin­sky, M.D., of New York Uni­ver­si­ty Lan­gone Med­ical Cen­ter’s Com­pre­hen­sive Epilep­sy Cen­ter and prin­ci­pal in­ves­ti­ga­tor in the tri­al:

“The pos­i­tive out­come in this sec­ond tri­al of Epid­i­olex in pa­tients with Lennox-Gas­taut syn­drome demon­strates the ef­fec­tive­ness of this prod­uct in this par­tic­u­lar­ly dif­fi­cult to treat, child­hood-on­set epilep­sy. The da­ta from the Epid­i­olex Dravet and LGS stud­ies of­fers the prospect of an FDA-ap­proved CBD med­i­cine that shows both clin­i­cal­ly mean­ing­ful seizure re­duc­tion and a con­sis­tent safe­ty and tol­er­a­bil­i­ty pro­file. I be­lieve Epid­i­olex has the po­ten­tial to be­come an im­por­tant new op­tion with­in the field of treat­ment-re­sis­tant epilep­sy.”

One pa­tient on 10mg/kg Epid­i­olex dis­con­tin­ued treat­ment due to an ad­verse event, in­ves­ti­ga­tors said, com­pared with six pa­tients on 20mg/kg and one pa­tient on place­bo.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.

Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

Endpoints Premium

Premium subscription required

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

Rami Elghandour, Arcellx CEO

Up­dat­ed: Gilead, Ar­cel­lx team up on an­ti-BC­MA CAR-T as biotech touts a 100% re­sponse rate at #ASH22

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,300+ biopharma pros reading Endpoints daily — and it's free.

David Light, Valisure CEO

Val­isure in the hot seat: New Form 483 over a 2021 in­spec­tion as CEO fires back

The notorious drug testing company Valisure, which has made a name for itself by forcing FDA’s hand with some of its safety-related uncoverings, received a letter this week after the FDA uncovered violations at its Connecticut-based testing lab in 2021.

The letter, which was sent on Dec. 5, stated that the FDA is “concerned” that Valisure was not aware of  drug supply chain security requirements.

Mark Cuban (Jed Jacobsohn/AP Images)

Mov­ing to the em­ploy­er side of health­care, Mark Cuban's Cost Plus Drugs part­ners with a PBM

From “Shark Tank” to direct-to-consumer generic drugs, Mark Cuban has made another inroad in the ongoing battle over prescription drug prices. His cost-plus-15% generic drug company, frequently undercutting many competitors, now has its sights set on the employer healthcare market.

Cost Plus Drugs, which originally pledged to cut out PBMs, has now partnered with the PBM EmsanaRx, majority owned by the Purchaser Business Group on Health, to launch a supplemental drug discount program designed specifically for self-funded employers, the company announced Thursday.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,300+ biopharma pros reading Endpoints daily — and it's free.

Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.