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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Common performs onstage, December 2020 (Getty Images)

Com­mon, Jamie Foxx among celebs stand­ing up for clin­i­cal tri­als in star-stud­ded can­cer group's pan­dem­ic push

Healthcare screenings and clinical trial enrollment were battered by the pandemic. But the well-known celebrity-backed Stand Up To Cancer non-profit, along with pharma and advocacy partners, has been working to reverse that and make up lost ground, by stepping up awareness campaigns.

Twelve campaigns launched in 2020 and another five in 2021 amplify the need for cancer screening and care, especially for underserved communities. While pharma companies have long been donors to the cancer research group, Covid brought new support — and increased awareness efforts.

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News. 

Vas Narasimhan, Novartis CEO (Thibault Camus/Pool via AP Images)

With gener­ic com­pe­ti­tion heat­ing up, Vas Narasimhan out­lines No­var­tis' growth plans at R&D day

Thursday marks Novartis’ annual R&D day, and with it comes CEO Vas Narasimhan’s attempt to spotlight the company’s pipeline strategy and emerging stars.

The biggest question entering Thursday’s presentation dealt with how the big biopharma will make up revenues from upcoming generic competition — Novartis says within the next five years, generics will eat away roughly $9 billion in sales. To offset this, Narasimhan outlined a strategy for 4% growth or higher until 2026, focusing on six key medicines he believes will see multibillion dollar profits during this time.

UP­DAT­ED: Am­gen halts some drug dis­counts for safe­ty net hos­pi­tals as SCO­TUS takes on 340B case

Amgen will soon be the 10th biopharma company to pull back on offering drug discounts to contract pharmacies of safety-net hospitals under a federal program. Like its peers, Amgen argues that the growth of these contract pharmacies has ballooned in recent years and needs to be reigned in.

Beginning Jan. 3, 2022, Amgen’s policy will only allow 340B covered hospitals to designate a single pharmacy location, with the exception of federal grantees and contract pharmacies wholly owned by a 340B hospital, or that have common ownership with a health system.