Ther­a­pix shares rise on ear­ly da­ta of its re­pur­posed cannabi­noid in Tourette syn­drome

An Is­raeli biotech de­vel­op­ing cannabi­noid-based drugs has found pre­lim­i­nary ev­i­dence that its lead prod­uct can re­duce symp­toms in adults suf­fer­ing from Tourette syn­drome.

In a sin­gle-arm, open-la­bel Phase IIa tri­al de­signed pri­mar­i­ly to con­firm safe­ty, tol­er­a­bil­i­ty and fea­si­bil­i­ty, six of 16 TS pa­tients ex­pe­ri­enced a re­sponse to Ther­a­pix’s treat­ment, as de­fined by a re­duc­tion in the Yale Glob­al Tic Sever­i­ty Scale To­tal Tic Score of greater than 25%, af­ter 12 weeks. The in­ves­ti­ga­tors from Yale al­so re­port­ed a mean dif­fer­ence of 7.9 in score (from 38.4 to 30.5). That trans­lates to an av­er­age tic re­duc­tion of 21% — re­fer­ring to the re­peat­ed in­vol­un­tary move­ments and sounds that char­ac­ter­izes the dis­ease — though the lack of a con­trol arm makes it dif­fi­cult to ac­cess just how sig­nif­i­cant it is.

Ac­cord­ing to Michael Bloch, the prin­ci­pal in­ves­ti­ga­tor, the re­sults are en­cour­ag­ing giv­en that these are pa­tients who have not re­spond­ed to pre­vi­ous treat­ments (like an­tipsy­chot­ic med­ica­tions and be­hav­ioral ther­a­py) or ex­per­i­men­tal ther­a­pies (like top­i­ra­mate or deep brain stim­u­la­tion).

Ther­a­pix shares $TR­PX shot up 32% in pre­mar­ket trad­ing.

As­ch­er Shmule­witz

The drug, named THX-110, is a com­bi­na­tion of dron­abi­nol (a syn­thet­ic cannabi­noid) and palmi­toylethanolamide. The com­pa­ny now plans to take it in­to a ran­dom­ized, dou­ble-blind, place­bo con­trolled study to eval­u­ate its ef­fi­ca­cy, in ad­di­tion to a 24-week ex­ten­sion phase of the tri­al read out to­day. Twelve of the study sub­jects have agreed to take part in the ex­ten­sion.

“We be­lieve that the ef­fi­ca­cy demon­strat­ed in this study fur­ther re­in­force the po­ten­tial role of cannabi­noids in the field of CNS and move­ment dis­or­ders and sug­gests that Ther­a­pix Bio­sciences may have a pro­pri­etary cannabi­noid prod­uct can­di­date in the treat­ment of Tourette syn­drome,” said As­ch­er Shmule­witz, Ther­a­pix’s chair­man and in­ter­im CEO, in a state­ment.

THX-110 is al­so be­ing stud­ied for ob­struc­tive sleep ap­nea and for the treat­ment of pain.

5AM Ven­tures: Fu­el­ing the Next Gen­er­a­tion of In­no­va­tors

By RBC Capital Markets
With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures

Key Points

Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities.
Unlocking the potential of the microbiome could be the missing link to better disease diagnosis.
Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies.
Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom.

Biohaven CEO Vlad Coric (Photo Credit: Andrew Venditti)

Pssst: That big Bio­haven Alzheimer's study? It was a bust. Even the sub­group analy­sis ex­ecs tout­ed was a flop

You know it’s bad when a biopharma player plucks out a subgroup analysis for a positive take — even though it was way off the statistical mark for success, like everything else.

So it was for Biohaven $BHVN on MLK Monday, as the biotech reported on the holiday that their Phase II/III Alzheimer’s study for troriluzole flunked both co-primary endpoints as well as a key biomarker analysis.

The drug — a revised version of the ALS drug riluzole designed to regulate glutamate — did not “statistically differentiate” from placebo on the Alzheimer’s Disease Assessment Scale-Cognitive Subscale 11 (ADAS-cog) and the Clinical Dementia Rating Scale Sum of Boxes (CDR-SB).  The “hippocampal volume” assessment by MRI also failed to distinguish itself from placebo for all patients fitting the mild-to-moderate disease profile they had established for the study.

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Janet Woodcock and Joshua Sharfstein (AP, Images)

Poll: Should Joshua Sharf­stein or Janet Wood­cock lead the FDA from here?

It’s time for a new FDA commissioner to come on board, a rite of passage for Joe Biden’s administration that should help seal the new president’s rep on seeking out the experts to lead the government over the next 4 years.

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Dan Skovronsky, Eli Lilly CSO (Lilly via Facebook)

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Under science chief Dan Skovronsky, Eli Lilly has taken some big swings at next-gen therapies, including trying to find the next big thing in oncology. Now, after one early failure in the field, Lilly is going back to the bispecific antibody well with a new deal with a Dutch biotech.

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Andrew Allen (Gritstone)

As coro­n­avirus vari­ants trig­ger new alarms, the NIH is putting an un­der-the-radar ‘next-gen’ vac­cine in­to PhI

Over the past year, the world has been transfixed by the development of new vaccines to fight SARS-CoV-2. In a frenzy of activity, the new mRNA approach has delivered pioneering emergency approvals in record time. And with some setbacks, the more traditional big players are coming along with added jabs as the most affluent nations in the world begin to vaccinate large portions of their populations.

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The IPO queue adds 5 more biotechs hop­ing to ring in 2021 by blitz­ing Nas­daq

Following a record year for IPOs — in terms of both proceeds and count — there’s already a long lineup of biotechs ready to jump onto Nasdaq in the new year. The companies are likely looking for much higher raises than they initially projected on their S-1s. Now it’s time to see if investors are still hungry for another round of biotech stocks.

Sana helped set the pace early on, as its founders look to divvy up a fortune from their IPO. And late last week 5 more biotechs crowded in, looking to pick up the pace where 2020 left off. Here they are:

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News brief­ing: Beam bags a $260M pri­vate place­ment; mi­Ra­gen re­brands to Virid­i­an Ther­a­peu­tics

Agios vet John Evans has demonstrated how to raise big money for a little biotech.

The Beam Therapeutics CEO — and ARCH partner — has pieced together a $260 million private placement from a group of backers that includes Perceptive Advisors, Farallon Capital, Casdin Capital, Redmile Group and Cormorant Asset Management. And there are 3 main goals they’ll pursue with it: clinical development, strategic partnerships and general corporate purposes.

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The FDA on Friday approved Enhertu to treat locally advanced or metastatic HER2-positive gastric or gastroesophageal junction adenocarcinoma in patients who have previously undergone at least one round of treatment with a Herceptin-based regimen, AstraZeneca said in a release.

Ox­ford gets £100M to seize a 'break­through mo­men­t' in fight­ing su­per­bugs

Close to 70 years after Oxford scientists purified penicillin and confirmed its effect as an antibacterial drug, the university is establishing a new research institute at the forefront of combating antimicrobial resistance.

The Ineos Oxford Institute for AMR Research will initially be powered by a $136 million (£100 million) donation from Ineos, the UK-based chemicals giant founded by billionaire Jim Ratcliffe, that also plays a hand in manufacturing medical and pharma products.