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.”

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In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Gold for adults, sil­ver for in­fants: Pfiz­er's Pre­vnar 2.0 head­ed to FDA months af­ter Mer­ck­'s green light

Pfizer was first to the finish line for the next-gen pneumococcal vaccine in adults, but Merck beat its rival with a jab for children in June.

Now, two months after Merck’s 15-valent Vaxneuvance won the FDA stamp of approval for kids, Pfizer is out with some late-stage data on its 20-valent shot for infants.

Known as Prevnar 20 for adults, Pfizer’s 20vPnC will head to the FDA by the end of this year for an approval request in infants, the Big Pharma said Friday morning. Discussions with the FDA will occur first and more late-stage pediatric trials are expected to read out soon, informing the regulatory pathway in other countries and regions.

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Senate Finance Committee Chair Ron Wyden (D-OR) (Francis Chung/E&E News/POLITICO via AP Images)

Sen­ate Fi­nance Chair con­tin­ues his in­ves­ti­ga­tion in­to phar­ma tax­es with re­quests for Am­gen

Amgen is the latest pharma company to appear on the radar of Senate Finance Committee Chair Ron Wyden (D-OR), who is investigating the way pharma companies are using subsidiaries in low- or zero-tax countries to lower their tax bills.

Like its peers Merck, AbbVie and Bristol Myers Squibb, Wyden notes how Amgen uses its Puerto Rico operations to consistently pay tax rates that are substantially lower than the U.S. corporate tax rate of 21%, with an effective tax rate of 10.7% in 2020 and 12.1% in 2021.

FDA ap­proves sec­ond in­di­ca­tion for As­traZeneca and Dai­ichi's En­her­tu in less than a week

AstraZeneca and Daiichi Sankyo’s antibody-drug conjugate Enhertu scored its second approval in less than a week, this time for a subset of lung cancer patients.

Enhertu received accelerated approval on Thursday to treat adults with unresectable or metastatic non-small cell lung cancer (NSCLC) whose tumors have activating HER2 (ERBB2) mutations, and who have already received a prior systemic therapy.

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J&J to re­move talc prod­ucts from shelves world­wide, re­plac­ing with corn­starch-based port­fo­lio

After controversially spinning out its talc liabilities and filing for bankruptcy in an attempt to settle 38,000 lawsuits, Johnson & Johnson is now changing up the formula for its baby powder products.

J&J is beginning the transition to an all cornstarch-based baby powder portfolio, the pharma giant announced on Thursday — just months after a federal judge ruled in favor of its “Texas two-step” bankruptcy to settle allegations that its talc products contained asbestos and caused cancer. An appeals court has since agreed to revisit that case.

CSL is gathering its four business units under a unified brand identity strategy (Credit: CSL company site)

CSL brings Se­qirus, Vi­for un­der par­ent um­brel­la brand in iden­ti­ty re­vamp

CSL is gathering its brands under the family name umbrella, renaming its vaccine and newly acquired nephrology specialty businesses with the parent initials.

CSL Seqirus and CSL Vifor join CSL Plasma and CSL Behring as the four now uniformly branded business units of the global biopharma. The Seqirus vaccine division was formed in 2015 with the combination of bioCSL and its purchase of Novartis’ flu vaccine business. CSL picked up Vifor Pharma late last year in an $11.7 billion deal for the nephrology, iron deficiency and cardio-renal drug developer.

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Pharma brands are trying to figure out new ways to better reach patients and doctors, but also measure results. (Credit: Shutterstock)

Do phar­ma TV and so­cial ads work? Phar­ma mar­ket­ing agen­cies adopt­ing new tech so­lu­tions to find out

It’s a timeworn advertising question — is my ad campaign working? In pharma, that can be an especially difficult question to answer in part because of privacy regulations, but also because the brands spend a lot of money on TV commercials where viewers can’t directly click on an ad.

Healthcare marketing services companies like Lasso and CMI Media Group are trying to change that with new measurement methods and partnerships that aim to get closer to patients’ and physicians’ actions.

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Corey McCann, Pear Therapeutics CEO

Pear Ther­a­peu­tics touts Q2 growth while scal­ing back full-year goals and chop­ping 9% of staff

Pear Therapeutics set some ambitious goals back in March, predicting a five-fold boost in revenue and a surge in new prescriptions for its digital therapeutics. Now the company is scaling back those estimates and chopping 9% of its workforce — an all-too-common occurrence in biotech lately.

CEO Corey McCann unveiled Pear’s Q2 numbers on Thursday, touting a 20% quarter-over-quarter revenue growth totaling $3.3 million. That’s more than double what the company made in Q2 2021, and McCann thinks the team could see a nearly four-fold jump in revenue this year, falling in the range of $14 million to $16 million.

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Seagen interim CEO Roger Dansey and Daiichi Sankyo CEO Sunao Manabe

Paving the way for Mer­ck­'s buy­out, Seagen los­es ar­bi­tra­tion dis­pute with Dai­ichi over ADC tech

As Seagen awaits a final buyout offer from Merck that could be in the territory of $40 billion, Seagen revealed Friday afternoon that it lost an arbitration dispute with Daiichi Sankyo relating to the companies’ 2008 collaboration around the use of antibody-drug conjugate (ADC) technology.

But that loss likely won’t matter much when it comes to Merck’s deal.

After breaking off its pact with Daiichi in mid-2015, the two companies battled over “linker” tech — a chemical bridge between an ADC’s antibody component and the cytotoxic payload — that Seagen claims Daiichi would improve upon and implement in its current generation of ADCs.

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