Tonix crash­es on PhI­II PTSD fail­ure, send­ing stock to the gut­ter

Af­ter scor­ing two promis­ing FDA des­ig­na­tions on its lead drug, tiny Tonix Phar­ma­ceu­ti­cals is abort­ing its late-stage study in PTSD due to lousy re­sults — near­ly flat-lin­ing the com­pa­ny’s stock Fri­day morn­ing.

The drug, called Ton­mya, ap­par­ent­ly failed to im­prove PTSD symp­toms when com­pared to place­bo, miss­ing its pri­ma­ry end­point af­ter 12 weeks of treat­ment. Ton­mya is an un­der-the-tongue for­mu­la­tion of cy­cloben­za­prine, com­mon­ly used as a mus­cle re­lax­er.

Shares of Tonix $TNXP were halt­ed in pre-mar­ket trad­ing. But once Nas­daq hit the play but­ton, Tonix tanked over 77%.

Seth Le­d­er­man

Al­though the Phase III tri­al crashed and burned, the com­pa­ny still has hopes for a re­con­fig­ured piv­otal study, point­ing to da­ta at 4 weeks that showed some ear­ly promise.

“At week 4, the Ton­mya treat­ed group sep­a­rat­ed from place­bo in CAPS-5 (p = 0.019) and in the Clin­i­cal Glob­al Im­pres­sion – Im­prove­ment (CGI-I) scale (p = 0.015), a key sec­ondary end­point,” the com­pa­ny said in a state­ment. “Al­so, at week 4, sleep qual­i­ty im­proved as mea­sured by both the PROMIS sleep dis­tur­bance scale and the CAPS-5 sleep dis­tur­bance item, sup­port­ing the pro­posed mech­a­nism of ac­tion of Ton­mya.”

Now, it looks like Tonix will try to re­design its tri­al with new goal­posts. The com­pa­ny’s pres­i­dent and CEO Seth Le­d­er­man said they were “en­cour­aged by clin­i­cal im­prove­ment at week 4,” and will use da­ta from this failed study to de­sign the next piv­otal study.

“We plan to meet with the FDA as soon as pos­si­ble to dis­cuss the HON­OR re­sults and our pro­pos­al to con­duct the pri­ma­ry analy­sis at the week-4 time point in the next piv­otal study,” Le­d­er­man said.

He al­so blamed the com­pa­ny’s fail­ure on the dif­fi­cul­ties of their cho­sen in­di­ca­tion. “These re­sults un­der­score the chal­lenges in de­sign­ing and con­duct­ing well-con­trolled clin­i­cal stud­ies in PTSD, es­pe­cial­ly mil­i­tary-re­lat­ed PTSD,” he said.

Ton­mya, al­so known as TNX-102 SL, had re­ceived break­through ther­a­py des­ig­na­tion from the FDA for its ap­pli­ca­tions in PTSD. And just this month, the agency al­so hand­ed over fast track sta­tus for the drug in a dif­fer­ent in­di­ca­tion: ag­i­ta­tion in Alzheimer’s.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

As­traZeneca caps PD-L1/CT­LA-4/chemo com­bo come­back with OS win. Is treme­li­mum­ab fi­nal­ly ready for ap­proval?

AstraZeneca’s closely-watched POSEIDON study continues to be the rare bright spot in its push for an in-house PD-L1/CTLA-4 combo.

Combining Imfinzi and tremelimumab with physicians’ choice of chemotherapy helped patients with stage IV non-small cell lung cancer live longer, the company reported — marking the first time the still-experimental tremelimumab has demonstrated an OS benefit.

For AstraZeneca and CEO Pascal Soriot, the positive readout — which is devoid of numbers — offers much-needed validation for the big bet they made on Imfinzi plus tremelimumab, after the PD-L1/CTLA-4 regimen failed multiple trials in head and neck cancer as well as lung cancer.

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Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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Ron DePinho (file photo)

A 'fly­over' biotech launch­es in Texas with four Ron De­Pin­ho-found­ed com­pa­nies un­der its belt

In his 13 years at Genzyme, Michael Wyzga noticed something about East Coast drugmakers. Execs would often jet from Boston or New York to San Francisco to find more assets, and completely miss the work being done in flyover states, like Texas or Wisconsin.

“If it doesn’t come out of MGH or MIT or Harvard, probably not that interesting,” he said of the mindset.

Now, he and some well-known industry players are looking to change that, and they’ve reeled in just over $38 million to do it.

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Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Ad­comm splits slight­ly in fa­vor of FDA ap­prov­ing Chemo­Cen­tryx’s rare dis­ease drug

The FDA’s Arthritis Advisory Committee on Thursday voted 10 for and 8 against the approval of ChemoCentryx’s $CCXI investigational drug avacopan as a treatment for adults with a rare and serious disease known as anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis.

The vote on whether the FDA should approve the drug was preceded by a split vote of 9 to 9 on whether the efficacy data support approval, and 10 to 8 that the safety profile of avacopan is adequate enough to support approval.

Paul Hastings, Nkarta CEO

With no up­front pay­ment or mile­stones on the line, Nkar­ta and CRISPR join forces on CAR-NK search

Most deals in biotech come with hefty upfront payments attached, and the promise of big biobucks if a program works out. Not this one.

Nkarta has struck what CEO Paul Hastings calls a “real collaboration” with CRISPR Therapeutics to co-develop and commercialize two CAR-NK therapies, in addition to an NK+T program. The duo will split all R&D costs — and any worldwide profits — 50/50, Hastings said.

Brent Saunders (Richard Drew, AP Images)

OcuWho? Star deal­mak­er turned aes­thet­ics czar Brent Saun­ders flips back in­to biotech. But who’s he team­ing up with now?

Brent Saunders went on a tear of headline-blazing deals building Allergan, merging and rearranging a variety of big companies into one before an M&A pact with Pfizer blew up and sent him on a bout of biotech drug deals. That didn’t work so well, so under pressure, he got his buyout at AbbVie — which needed a big franchise like Botox. And it was no big surprise to see him riding the SPAC wave into a recent $1 billion-plus deal that left him in the executive chairman’s seat at an aesthetics outfit — now redubbed The Beauty Health Company — holding a big chunk of the equity.

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Drug pric­ing watch­dog joins the cho­rus of crit­ics on Bio­gen's ad­u­canum­ab: What about charg­ing $2,560 per year?

As if Biogen’s aducanumab isn’t controversial enough, the researchers at drug pricing watchdog ICER have drawn up the contours of a new debate: If the therapy does get approved for Alzheimer’s by June, what price should it command?

Their answer: At most $8,290 per year — and perhaps as little as $2,560.

Even at the top of the range, the proposed price is a fraction of the $50,000 that Wall Street has reportedly come to expect (although RBC analyst Brian Abrahams puts the consensus figure at $11.5K). With critics, including experts on the FDA’s advisory committee, making their fierce opposition to aducanumab’s approval loud and clear, the pricing pressure adds one extra wrinkle Biogen CEO Michel Vounatsos doesn’t need as he orders full-steam preparation for a launch.