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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Jacob Van Naarden (Eli Lilly)

Ex­clu­sives: Eli Lil­ly out to crash the megablock­buster PD-(L)1 par­ty with 'dis­rup­tive' pric­ing; re­veals can­cer biotech buy­out

It’s taken 7 years, but Eli Lilly is promising to finally start hammering the small and affluent PD-(L)1 club with a “disruptive” pricing strategy for their checkpoint therapy allied with China’s Innovent.

Lilly in-licensed global rights to sintilimab a year ago, building on the China alliance they have with Innovent. That cost the pharma giant $200 million in cash upfront, which they plan to capitalize on now with a long-awaited plan to bust up the high-price market in lung cancer and other cancers that have created a market worth tens of billions of dollars.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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Joshua Liang, Clover Biopharmaceuticals CEO

With world still in sore need of dos­es, Clover says its Covid-19 vac­cine is 67% ef­fec­tive in Phase III

With concerns about the Delta variant rising and much of the world still in desperate need of vaccine doses, a Chinese biotech announced Wednesday that a new shot has shown positive results in a large trial against Covid-19, including new variants.

Clover Biopharmaceuticals announced Wednesday that its vaccine candidate showed 79% efficacy against the Delta variant in a Phase II/III trial dubbed Spectra, and 67% effective against Covid-19 overall.

Jean Bennett (Brent N. Clarke/Invision/AP Images)

Lux­tur­na in­ven­tor Jean Ben­nett starts a new gene ther­a­py com­pa­ny to tack­le rare dis­eases left be­hind by phar­ma, VCs

A few years ago Jean Bennett found herself in a surprising place for a woman who invented the first gene therapy ever approved in the United States: No one, it seemed, wanted her work.

Bennett, who designed and co-developed Luxturna, approved in 2018 for a rare form of blindness, had kept building new gene therapies for eye diseases at her University of Pennsylvania lab. But although the results in animals looked promising, pharma companies and investors kept turning down the pedigreed ophthalmology professor.

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.