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.

Scoop: Boehringer qui­et­ly shut­ters a PhII for one of its top drugs — now un­der re­view

Boehringer Ingelheim has quietly shut down a small Phase II study for one of its lead drugs.

The private pharma player confirmed to Endpoints News that it had shuttered a study testing spesolimab as a therapy for Crohn’s patients suffering from bowel obstructions.

A spokesperson for the company tells Endpoints:

Taking into consideration the current therapeutic landscape and ongoing clinical development programs, Boehringer Ingelheim decided to discontinue our program in Crohn’s disease. It is important to note that this decision is not based on any safety findings in the clinical trials.

Endpoints Premium

Premium subscription required

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

Alex­ion puts €65M for­ward to strength­en its po­si­tion on the Emer­ald Isle

Ireland has been on a roll in 2022, with several large pharma companies announcing multimillion-euro projects. Now AstraZeneca’s rare disease outfit Alexion is looking to get in on the action.

Alexion on Friday announced a €65 million ($68.8 million) investment in new and enhanced capabilities across two sites in the country, including at College Park in the Dublin suburb of Blanchardstown and the Monksland Industrial Park in the central Irish town of Athlone, according to the Industrial Development Agency of Ireland.

Deborah Dunsire, Lundbeck CEO

Af­ter a 5-year re­peat PhI­II so­journ, Lund­beck and Ot­su­ka say they're fi­nal­ly ready to pur­sue OK to use Rex­ul­ti against Alzheimer's ag­i­ta­tion

Five years after Lundbeck and their longtime collaborators at Otsuka turned up a mixed set of Phase III data for Rexulti as a treatment for Alzheimer’s dementia-related agitation, they’ve come through with a new pivotal trial success they believe will finally put them on the road to an approval at the FDA. And if they’re right, some analysts believe they’re a short step away from adding more than $500 million in annual sales for the drug, already approved in depression and schizophrenia.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Fed­er­al judge de­nies Bris­tol My­er­s' at­tempt to avoid Cel­gene share­hold­er law­suit

Some Celgene shareholders aren’t happy with how Bristol Myers Squibb’s takeover went down.

On Friday, a New York federal judge ruled that they have a case against the pharma giant, denying a request to dismiss allegations that it purposely slow-rolled Breyanzi’s approval to avoid paying out $6.4 billion in contingent value rights (CVR).

When Bristol Myers put down $74 billion to scoop up Celgene back in 2019, liso-cel — the CAR-T lymphoma treatment now marketed as Breyanzi — was supposedly one of the centerpieces of the deal. After going back and forth on negotiations for about six months, BMS put $6.4 billion into a CVR agreement that required an FDA approval for Zeposia, Breyanzi and Abecma, each by an established date.

Chris Anzalone, Arrowhead CEO

Take­da, Ar­row­head spot­light da­ta from small tri­al show­ing RNAi works in a rare liv­er con­di­tion

Almost two years after Takeda wagered $300 million cash to partner with Arrowhead on an RNAi therapy for a rare disease, the companies are spelling out Phase II data that they believe put them one step closer to their big dreams.

In a small, open label study involving only 16 patients who had liver disease associated with alpha-1 antitrypsin deficiency (AATD), Arrowhead’s candidate — fazirsiran, previously ARO-AAT — spurred substantial reductions in accumulated mutant AAT protein in the liver, a hallmark of the condition. Investigators also tracked improvements in symptoms, with seven out of 12 who received the high, 200 mg dose seeing regression of liver fibrosis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

Am­gen takes next step with its Chi­na am­bi­tions, out-li­cens­ing drugs to Fo­s­un Phar­ma

In a bid to increase its market share in China, Amgen has agreed to a partnership with a Shanghai biotech — a collaboration and out-licensing agreement for two of its drugs.

Amgen and Fosun Pharma announced a deal Monday in a bid to increase Amgen’s presence in the country. The stated goal so far is to commercialize Amgen’s blockbuster psoriasis drug Otezla alongside Parsabiv, a drug for secondary hyperparathyroidism in adults with chronic kidney disease and on a specific type of dialysis.

As court case looms, Bris­tol My­ers touts la­bel ex­pan­sion for Breyanzi

As Bristol Myers Squibb braces for a court battle over a costly delay — at least for Celgene shareholders — for its CAR-T lymphoma treatment Breyanzi, the pharma giant is touting a label expansion in the second-line setting.

Breyanzi, also known as liso-cel, snagged a win on Friday in adults with large B-cell lymphoma (LBCL) who: don’t respond to chemotherapy, or relapse within 12 months; don’t respond or relapse after 12 months; or are not eligible for hematopoietic stem cell transplant after chemo due to their age or comorbidities.

State bat­tles over mifepri­s­tone ac­cess could tie the FDA to any post-Roe cross­roads

As more than a dozen states are now readying so-called “trigger” laws to kick into effect immediate abortion bans following the overturning of Roe v. Wade on Friday, these laws, in the works for more than a decade in some states, will likely kick off even more legal battles as states seek to restrict the use of prescription drug-based abortions.

Since Friday’s SCOTUS opinion to overturn Americans’ constitutional right to an abortion after almost 50 years, reproductive rights lawyers at Planned Parenthood and other organizations have already challenged these trigger laws in Utah and Louisiana. According to the Guttmacher Institute, other states with trigger laws that could take effect include Arkansas, Idaho, Kentucky, Mississippi, Missouri, North Dakota, Oklahoma, South Dakota, Tennessee, Texas, and Wyoming.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 144,400+ biopharma pros reading Endpoints daily — and it's free.

A Mer­ck part­ner is sucked in­to the fi­nan­cial quag­mire as key lender calls in a note

Another biotech standing on shaky financial legs has fallen victim to the bears.

Merck partner 4D Pharma has reported that a key lender, Oxford Finance, shoved the UK company into administration after calling in a $14 million loan they couldn’t immediately make good on. Trading in their stock was halted with a market cap that had fallen to a mere £30 million.

“Despite the very difficult prevailing market conditions,” 4D reported on Friday, the biotech had been making progress on finding some new financing and turned to Oxford with an alternative late on Thursday and then again Friday morning.