More re­search is need­ed to jus­ti­fy use of 'med­i­c­i­nal' cannabis: UK agen­cies

A con­cert­ed cam­paign to un­lock the use of cannabis to treat se­vere child­hood epilep­sy prompt­ed the British au­thor­i­ties last year to sanc­tion its use in cer­tain pa­tients, when oth­er med­i­cines have failed, af­ter con­sul­ta­tion with a spe­cial­ist doc­tor. On Thurs­day, NHS Eng­land and cost-ef­fec­tive­ness watch­dog NICE un­veiled their pre­lim­i­nary rec­om­men­da­tions on the adop­tion of cannabis-de­rived med­i­cines for a va­ri­ety of con­di­tions. In short, they want more re­search to jus­ti­fy the med­ical use of cannabis.

NHS Eng­land is­sued a re­view that un­der­scored a pauci­ty of long-term safe­ty and ef­fi­ca­cy of med­i­c­i­nal cannabis, ac­knowl­edg­ing that clin­i­cians have found that some pa­tients with se­vere treat­ment-re­sis­tant epilep­sy do re­spond to cannabis-based ther­a­py and that ob­ser­va­tion­al da­ta sug­gest cannabis ther­a­py can di­min­ish seizure fre­quen­cy, re­sult­ing in few­er trips to emer­gency rooms and a bet­ter qual­i­ty of life.

While med­i­c­i­nal cannabis was be­ing con­sid­ered on a case-by-case ba­sis since last No­vem­ber, the lack of ran­dom­ized con­trol tri­al (RCT) da­ta is a ma­jor hur­dle to pre­scrib­ing, in ad­di­tion to the cost of ac­quir­ing the prod­uct, which is not read­i­ly at­tain­able on the NHS or by NHS Trusts.

Con­se­quent­ly, the NHS rec­om­mend­ed two clin­i­cal tri­als be set up.

In par­al­lel, NICE rec­om­mend­ed fur­ther re­search in­to the use of cannabis-based prod­ucts in in­di­ca­tions such as fi­bromyal­gia, chron­ic pain, nau­sea and vom­it­ing and spas­tic­i­ty.

The agency al­so high­light­ed that it was un­able to make a rec­om­men­da­tion for the use of cannabis-de­rived med­i­cines in se­vere treat­ment-re­sis­tant epilep­sy, cit­ing a lack of clear ev­i­dence of ben­e­fit. (GW Phar­ma’s FDA-ap­proved cannabis-de­rived drug for epilep­sy, Epid­i­olex, is un­der Eu­ro­pean re­view.)

Si­mon Wig­glesworth Epilep­sy Ac­tion

“For those af­fect­ed by se­vere epilep­sies, hope is of­ten in short sup­ply. We recog­nise how dis­ap­point­ing the draft rec­om­men­da­tions will be for many. Hav­ing al­ready wait­ed al­most a year for this guid­ance, the prospect of wait­ing many years more for tri­als to be com­plet­ed is un­ac­cept­able,” Si­mon Wig­glesworth, deputy chief ex­ec­u­tive at UK-based Epilep­sy Ac­tion, told End­points News.

On the ba­sis of a lack of cost-ef­fec­tive­ness, NICE al­so ad­vo­cat­ed against the use of GW Phar­ma’s $GW­PH ap­proved cannabis-de­rived spray Sativex in pa­tients with spas­tic­i­ty. In ad­di­tion, NICE said that that oth­er cannabis-based med­i­c­i­nal prod­ucts should not be of­fered to treat spas­tic­i­ty un­less as part of a clin­i­cal tri­al. NICE did, how­ev­er, en­dorse the use of syn­thet­ic cannabis treat­ment nabilone, as an add-on treat­ment for adults with chemother­a­py-in­duced nau­sea and vom­it­ing that haven’t re­spond­ed to con­ven­tion­al med­i­cine.

Paul Chrisp NICE

“We recog­nise that some peo­ple will be dis­ap­point­ed…How­ev­er, we were con­cerned when we be­gan de­vel­op­ing this guid­ance that a ro­bust ev­i­dence base for these most­ly un­li­censed prod­ucts was prob­a­bly lack­ing,” Paul Chrisp, di­rec­tor of the Cen­tre for Guide­lines at NICE, said in a state­ment. “Hav­ing now con­sid­ered all the avail­able ev­i­dence it’s there­fore not sur­pris­ing that the com­mit­tee has not been able to make many pos­i­tive rec­om­men­da­tions about their use.”

While the UK is slow­ly build­ing the ev­i­dence case to eval­u­ate the po­ten­tial med­i­c­i­nal prop­er­ties of cannabis — in the Unit­ed States, re­searchers have found that gain­ing ac­cess to cannabis for med­ical re­search has been a frus­trat­ing, up­hill bat­tle. A ma­jor­i­ty of US states have sanc­tioned the use of cannabis in a med­ical and/or recre­ation­al ca­pac­i­ty — but at the fed­er­al lev­el, it re­mains a sched­ule 1 sub­stance con­sid­ered to have no med­i­c­i­nal val­ue.

So­cial im­age: Shut­ter­stock

Regeneron CEO Leonard Schleifer speaks at a meeting with President Donald Trump, members of the Coronavirus Task Force, and pharmaceutical executives in the Cabinet Room of the White House (AP Photo/Andrew Harnik)

OWS shifts spot­light to drugs to fight Covid-19, hand­ing Re­gen­eron $450M to be­gin large scale man­u­fac­tur­ing in the US

The US government is on a spending spree. And after committing billions to vaccines defense operations are now doling out more of the big bucks through Operation Warp Speed to back a rapid flip of a drug into the market to stop Covid-19 from ravaging patients — possibly inside of 2 months.

The beneficiary this morning is Regeneron, the big biotech engaged in a frenzied race to develop an antibody cocktail called REGN-COV2 that just started a late-stage program to prove its worth in fighting the virus. BARDA and the Department of Defense are awarding Regeneron a $450 million contract to cover bulk delivery of the cocktail starting as early as late summer, with money added for fill/finish and storage activities.

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Cel­lec­tis slammed af­ter pa­tient dies and FDA slaps a hold on their tri­al for an off-the-shelf CAR-T for mul­ti­ple myelo­ma

Cellectis was slammed after the market close on Monday as the biotech reported that the FDA demanded it hit the brakes on their MELANI-01 trial for their off-the-shelf cell therapy UCARTCS1A after one of the patients in the study died of treatment-related cardiac arrest.

The multiple myeloma patient had previously been treated unsuccessfully with various therapies, noted the biotech, and had been given dose level two (DL2) of their allogeneic CAR-T.

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Noubar Afeyan, Flagship CEO and Tessera chairman (Victor Boyko/Getty Images)

Flag­ship ex­ecs take a les­son from na­ture to mas­ter ‘gene writ­ing,’ launch­ing a star-stud­ded biotech with big am­bi­tions to cure dis­ease

Flagship Pioneering has opened up its deep pockets to fund a biotech upstart out to revolutionize the whole gene therapy/gene editing field — before gene editing has even made it to the market. And they’ve surrounded themselves with some marquee scientists and execs who have crowded around to help shepherd the technology ahead.

The lead player here is Flagship general partner Geoff von Maltzahn, an MIT-trained synthetic biologist who set out in 2018 to do CRISPR — a widely used gene editing tool — and other rival technologies one or two better. Von Maltzahn has been working with Sana co-founder Jake Rubens, another synthetic biology player out of MIT who he describes as his “superstar,” who’s taken the CSO role.

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Covid-19 roundup: Squab­bles with gov­ern­ment de­lay Mod­er­na’s PhI­II — re­ports; No­vavax se­cures largest Warp Speed deal yet: $1.6B

A much-anticipated Phase III trial for Moderna’s Covid-19 vaccine is being held up as the company delayed submitting trial protocols and sparred with government scientists on how to run the study and even what the benchmark for success should be, Reuters reported.

Moderna, the first US company to put their vaccine into human testing, was supposed to enter a 30,000-person study this month in partnership with the NIH to determine whether it can prevent infection. STAT reported last week that the trial was facing delays over the protocol, but that a July start was still possible. Neither the NIH nor Moderna ever disclosed a specific date the trial should start, but Reuters reported that the agency had hoped to begin on July 10.

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Donald and Melania Trump watch the smoke of fireworks from the South Lawn of the White House on July 4, 2020 (via Getty)

Which drug de­vel­op­ers of­fer Trump a quick, game-chang­ing ‘so­lu­tion’ as the pan­dem­ic roars back? Eli Lil­ly and Ab­Cellera look to break out of the pack

We are unleashing our nation’s scientific brilliance and will likely have a therapeutic and/or vaccine solution long before the end of the year.

— Donald Trump, July 4

Next week administration officials plan to promote a new study they say shows promising results on therapeutics, the officials said. They wouldn’t describe the study in any further detail because, they said, its disclosure would be “market-moving.”

— NBC News, July 3

Something’s cooking. And it’s not just July 4 leftovers involving stale buns and uneaten hot dogs.

Over the long weekend observers picked up signs that the focus in the Trump administration may swiftly shift from the bright spotlight on vaccines being promised this fall, around the time of the election, to include drugs that could possibly keep patients out of the hospital and take the political sting out of the soaring Covid-19 numbers causing embarrassment in states that swiftly reopened — as Trump cheered along.

So far, Gilead has been the chief beneficiary of the drive on drugs, swiftly offering enough early data to get remdesivir an emergency authorization and into the hands of the US government. But their drug, while helpful in cutting stays, is known for a limited, modest effect. And that won’t tamp down on the hurricane of criticism that’s been tearing at the White House, and buffeting the president’s most stalwart core defenders as the economy suffers.

We’ve had positive early-stage vaccine data, most recently from Pfizer and BioNTech, playing catchup on an mRNA race led by Moderna — where every little sign of potential trouble is magnified into a lethal threat, just as every advance excites a frenzy of support. But that race still has months to play out, with more Phase I data due ahead of the mid-stage numbers looming ahead. A vaccine may not be available in large enough quantities until well into 2021, which is still wildly ambitious.

So what about a drug solution?

Trump’s initial support for a panacea focused on hydroxychloroquine. But that fizzled in the face of data underscoring its ineffectiveness — killing trials that aren’t likely to be restarted because of a recent population-based study offering some support. And there are a number of existing drugs being repurposed to see how they help hospitalized patients.

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Shoshanna Shendelman, Applied Therapeutics CEO (Applied Therapeutics)

A lit­tle biotech slaps back at a 'crim­i­nal' short at­tack, vow­ing to pur­sue a pros­e­cu­tion of their case

As short attacks go, Biotech Research Partners’ assault on Applied Therapeutics’ “cherry picked” data and a variety of so-called red flags didn’t cause a whole lot of damage. Ahead of the July 4 holiday, its shares $APLT were dinged and showed signs of quick recovery.

But that didn’t stop an incendiary response, as the biotech swung into action bright and early Monday morning.

Applied Therapeutics accused the authors of the short report of manipulating graphs and figures, misrepresenting data and included factual misrepresentations — all of which added up, in their view, to fraud.

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FDA lifts par­tial hold on ADC, clear­ing way for a 2021 read­out and two po­ten­tial near-term ap­provals

In throwing $267 million and a multi-billion dollar valuation at ADC Therapeutics for their May IPO, investors were betting that the partial hold the FDA had just placed on their second lead drug would prove immaterial. Time, it appears, has proven them right.

The FDA has lifted the partial hold, ADC said this morning. The move clears the way for an anticipated 2021 readout from their second pivotal study, a Phase II trial testing their experimental antibody drug conjugate camidanlumab tesirine, or Cami, in Hodgkin’s lymphoma.

Bill Haney, Dragonfly CEO (Dave Pedley/Getty Images for SXSW)

A boom­ing Drag­on­fly is tak­ing its TriN­KETs to Copen­hagen as the lat­est Bris­tol My­ers pact spurs ex­pan­sion plans — out­side the US

Bristol Myers Squibb is making a habit out of collaborating with the crew at Dragonfly, adding their 3rd deal in a series that now will take them into newly charted R&D territory. And the fast-growing team at the Cambridge-based biotech is adding a facility in Copenhagen for its next growth spurt, where the government is making it easy to recruit scientists internationally as the U.S. throttles back.

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Covid-19 roundup: Teamed up with NIH, Re­gen­eron launch­es PhI­II pre­ven­tion tri­al for an­ti­body cock­tail

As Regeneron moves its antibody cocktail into Phase II/III trials testing REGN-COV2 as a treatment for both hospitalized and non-hospitalized patients with Covid-19, the biotech is also starting a Phase III in the prevention setting.

The National Institute of Allergy and Infectious Diseases — which orchestrated the large, randomized study for remdesivir that produced positive results — will jointly run the study.

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