Af­ter get­ting crushed by bad da­ta, Zyner­ba says its cannabis-based drug worked on Frag­ile X symp­toms

Less than two months since a pair of mid-stage set­backs crushed its stock price, re­searchers at Zyner­ba $ZYNE say they had bet­ter re­sults from a small Phase II study that test­ed their cannabis-based ther­a­py on symp­toms of Frag­ile X syn­drome. And the news quick­ly sparked a big ral­ly for its bat­tered stock.

Ar­man­do Anido

Of­ten com­pared to GW Phar­ma­ceu­ti­cals, which has had some strik­ing suc­cess­es in the field, Zyner­ba set out to see what im­pact their cannabid­i­ol ther­a­py would have tamp­ing down on a range of com­mon symp­toms for Frag­ile X, in­clud­ing anx­i­ety and de­pres­sion. And with 20 pa­tients in the sin­gle-arm study, the in­ves­ti­ga­tors say they hit their pri­ma­ry with a 46% drop in their ADAMS score, track­ing Anx­i­ety, De­pres­sion, and Mood Scale.

Frag­ile X is an autism spec­trum dis­or­der, one tough dis­ease to treat at the source. It’s sig­nif­i­cant that Zyner­ba didn’t go af­ter the dis­ease it­self, but rather symp­toms that can make life mis­er­able for the pa­tients and their fam­i­lies. That’s an eas­i­er tar­get, es­pe­cial­ly when you have a small, un­con­trolled study to use — which is far from ide­al.

Now, the biotech plans to steer this drug in­to a piv­otal Phase II/III, news that re­vived their share price — which im­me­di­ate­ly soared 90% this morn­ing in pre-mar­ket trad­ing.

Zyner­ba was forced to con­cede last month that its drug failed two back-to-back mid-stage stud­ies for epilep­sy as well as os­teoarthri­tis, with their cannabi­noid un­able to sig­nif­i­cant­ly re­duce knee pain.

“The clin­i­cal­ly mean­ing­ful im­prove­ments in Frag­ile X symp­toms and the ex­cel­lent tol­er­a­bil­i­ty seen in the FAB-C tri­al are com­pelling,” ex­claimed Zyner­ba CEO Ar­man­do Anido in a state­ment. “These da­ta will al­low us to dis­cuss the path­way to ap­proval in a meet­ing with the FDA, which we ex­pect to take place dur­ing the first half of 2018.”

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotechs that fit that general description.

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Gilead re­leas­es an­oth­er round of murky remde­sivir re­sults

A month after the NIH declared the first trial on remdesivir in Covid-19 a success, Gilead is out with new results on their antiviral. But although the study met one of its primary endpoints, the data are likely to only add to a growing debate over how effective the drug actually is.

In a Phase III trial, patients given a 5-day dose of remdesivir were 65% more likely to show “clinical improvement” compared to an arm given standard-of-care. The trial, though, gave little indication for whether the drug had an impact on key endpoints such as survival or time-to-recovery. And in a surprising twist, a 10-day dosing arm of remdesivir didn’t lead to a statistically significant improvement over standard of care.

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Novus Ther­a­peu­tics plunges deep in­to pen­ny stock ter­ri­to­ry af­ter failed ear tri­al

After a more than 15-year run, a California-based biotech is exploring options, including a sale, after its lead experimental therapy failed an exploratory mid-stage study in patients with middle ear infections characterized by a build-up of fluid behind the eardrum.

The company, initially called Tokai Pharmaceuticals but which subsequently changed its name to Novus Therapeutics in 2017, saw its shares more than halve on Monday after the drug — OP0201— did not pass muster as an adjunct therapy to oral antibiotics in infants and children aged 6 to 24 months with acute otitis media (OM).

Fangliang Zhang (Imaginechina via AP Images)

The big mon­ey: Poised to make drug R&D his­to­ry, a Chi­na biotech un­veils uni­corn rac­ing am­bi­tions in a bid to raise $350M-plus on Nas­daq

Almost exactly three years after Shanghai-based Legend came out of nowhere to steal the show at ASCO with jaw-dropping data on their BCMA-targeted CAR-T for multiple myeloma, the little player with Big Pharma connections is taking a giant step toward making it big on Wall Street. And this time they want to seal the deal on a global rep after staking out a unicorn valuation in what’s turned out to be a bull market for biotech IPOs — in the middle of a pandemic.

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Len Schleifer (left) and George Yancopoulos, Regeneron (Vimeo)

Eyes on he­mo­phil­ia prize, Re­gen­eron adds a $100M wa­ger on joint de­vel­op­ment cam­paign with In­tel­lia

When George Yancopoulos first signed up Intellia to be its CRISPR/Cas9 partner on gene editing projects 4 years ago, the upstart smartly ramped up its IPO at the same time. Today, Regeneron $REGN is coming back in, adding $100 million in an upfront fee and equity to significantly boot up a whole roster of new development projects.

And they’re highlighting some clinical hemophilia research plans in the process.

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Jean-Jacques Bienaimé, BioMarin chairman and CEO

Bio­Marin holds the line on bleeds with 4-year val­rox up­date on he­mo­phil­ia A — but what's this about an­oth­er de­cline in Fac­tor 8 lev­els?

BioMarin has posted some top-line results for their 4-year followup on the most advanced gene therapy for hemophilia A — extending its streak on keeping a handful of patients free of bleeds and off Factor VIII therapy, but likely stirring fresh worries over a continued drop in Factor VIII levels.

We just don’t know how big a drop.

We’ll see more data when the results are presented at the World Federation of Hemophilia in a couple of weeks. But in a statement out Sunday night, BioMarin $BMRN reported that none of the patients required Factor VIII treatment, adding:

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As­traZeneca trum­pets the 'mo­men­tous' da­ta they found for Tagris­so in an ad­ju­vant set­ting for NSCLC — but many of the ex­perts aren’t cheer­ing along

AstraZeneca is rolling out the big guns this evening to provide a salute to their ADAURA data on Tagrisso at ASCO.

Cancer R&D chief José Baselga calls the disease-free survival data for their drug in an adjuvant setting of early stage, epidermal growth factor receptor-mutated NSCLC patients following surgery “momentous.” Roy Herbst, the principal investigator out of Yale, calls it “transformative.”

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Stephen Hahn, FDA commissioner (AP Images)

Stephen Hahn: FDA will make some changes amid Covid-19 per­ma­nent

The FDA will look to permanently implement some of the processes and policies adopted in its response to the Covid-19 pandemic, FDA commissioner Stephen Hahn said during a virtual briefing hosted by the Alliance for a Stronger FDA on Monday.

Hahn’s statements during the briefing closely mirrored statements he made in a pre-recorded video FDA uploaded to YouTube on Friday.

“As this pandemic has evolved, it was clear to all of us that some FDA processes needed to be adjusted to accommodate the urgency of the pandemic and I think the entire FDA team has now seen first-hand that we need to take a critical look at some of our processes and policies,” Hahn said.