Shrink the pa­tient group, boost the ef­fi­ca­cy: Tonix says a retro look shows promise in se­lect PTSD group

A back­wards glance at tri­al da­ta — and a reshuf­fling of the pa­tients in­volved — has re­vert­ed fail­ure to op­ti­mism for Tonix Phar­ma­ceu­ti­cals. At least that’s what com­pa­ny ex­ecs are root­ing for, ac­cord­ing to an up­date they’ve pro­vid­ed to­day on its PTSD drug Ton­mya.

You might re­mem­ber Ton­mya for the re­cent Phase III tri­al flop that washed Tonix’s stock in­to the gut­ter, sink­ing the share price near­ly 70% over the past four weeks.

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

But now, the NYC-based com­pa­ny says it’s tak­en a deep­er look at the da­ta on that tri­al — along with some old da­ta from a Phase II study — and has de­ter­mined a sub­set of the pa­tients saw bet­ter re­sults than oth­ers. Tonix shrunk the pa­tient group to on­ly in­clude those with PTSD who had suf­fered their trau­ma with­in the past 9 years. These pa­tients — which make up about 50% of the to­tal pa­tient group — saw a sig­nif­i­cant­ly bet­ter re­sponse, with a P val­ue of 0.039.

Seth Le­d­er­man

“The find­ing that treat­ment re­sponse to Ton­mya in P301 de­creas­es as the time since trau­ma gets longer, sug­gests that mil­i­tary ser­vice mem­bers and vet­er­ans with PTSD are tran­si­tion­ing from a Ton­mya-treat­ment re­spon­sive state to a non-re­spon­sive state af­ter ap­prox­i­mate­ly nine years,” said the com­pa­ny’s CEO Seth Le­d­er­man in a state­ment. “These re­sults em­pha­size the ur­gency for ear­ly di­ag­no­sis and treat­ment for PTSD, es­pe­cial­ly for mil­i­tary-re­lat­ed PTSD.”

Un­for­tu­nate­ly for Tonix, it doesn’t look like in­vestors are quite as jazzed about the ret­ro­spec­tive analy­sis. The stock $TNXP is up about 4% from yes­ter­day’s close as of press time, but that’s still down about 68% from its pre-tri­al-re­sults crash last month.

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

The com­pa­ny says its fu­ture clin­i­cal tri­als in PTSD will on­ly look at pa­tients who ex­pe­ri­enced trau­ma with­in the past 9 years.

Qual­i­ty Con­trol in Cell and Gene Ther­a­py – What’s Re­al­ly at Stake?

In early 2021, Bluebird Bio was forced to suspend clinical trials of its gene therapy for sickle cell disease after two patients in the trial developed cancer. As company scientists rushed to assess whether there was any causal link between the therapy and the cancer cases, Bluebird’s stock value plummeted – as did those of multiple other biopharma companies developing similar therapies.

While investigations concluded that the gene therapy was unlikely to have caused cancer, investors and the public may be more skittish regarding the safety of gene and cell therapies after this episode. This recent example highlights how delicate the fields of cell and gene therapy remain today, even as they show great promise.

Sen. Patty Murray (D-WA) (Graeme Sloan/Sipa USA/Sipa via AP Images)

Sen­a­tors to NIH: Do more to pro­tect US bio­med­ical re­search from for­eign in­flu­ence

Although Thursday’s Senate health committee hearing was focused on how foreign countries and adversaries might be trying to steal or negatively influence biomedical research in the US, the only country mentioned by the senators and expert witnesses was China.

Committee chair Patty Murray (D-WA) made clear in her opening remarks that the US cannot “let the few instances of bad actors” overshadow the hard work of the many immigrant researchers in the US, many of which have won Nobel prizes for their work. But she also said, “There is more the NIH can be doing here.”

Covid-19 man­u­fac­tur­ing roundup: Mary­land looks to grow biotech ca­pac­i­ty with $400M check; Rus­sia lands sec­ond Sput­nik V part­ner this week

A Maryland real estate project has added three new biotech-focused manufacturing and research buildings to an office park to keep up with demand created by the pandemic, the Washington Business Journal reported.

The Milestone Business Park — located off of I-270 in Germantown, MD — will see the new buildings and a total of 532,000 square feet as the campus rebrands to Milestone Innovation Park.

Law pro­fes­sors call for FDA to dis­close all safe­ty and ef­fi­ca­cy da­ta for drugs

Back in early 2018 when Scott Gottlieb led the FDA, there was a moment when the agency seemed poised to release redacted complete response letters and other previously undisclosed data. But that initiative never gained steam.

Now, a growing chorus of researchers are finding that a dearth of public data on clinical trials and pharmaceuticals means industry and the FDA cannot be held accountable, two law professors from Yale and New York University write in an article published Wednesday in the California Law Review.

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Novavax CEO Stanley Erck at the White House in 2020 (Andrew Harnik, AP Images)

As fears mount over J&J and As­traZeneca, No­vavax en­ters a shaky spot­light

As concerns rise around the J&J and AstraZeneca vaccines, global attention is increasingly turning to the little, 33-year-old, productless, bankruptcy-flirting biotech that could: Novavax.

In the now 16-month race to develop and deploy Covid-19 vaccines, Novavax has at times seemed like the pandemic’s most unsuspecting frontrunner and at times like an overhyped also-ran. Although they started the pandemic with only enough cash to last 6 months, they leveraged old connections and believers into $2 billion and emerged last summer with data experts said surpassed Pfizer and Moderna. They unveiled plans to quickly scale to 2 billion doses. Then they couldn’t even make enough material to run their US trial and watched four other companies beat them to the finish line.

FDA of­fers scathing re­view of Emer­gent plan­t's san­i­tary con­di­tions, em­ploy­ee train­ing af­ter halt­ing pro­duc­tion

The FDA wrapped up its inspection of Emergent’s troubled vaccine manufacturing plant in Baltimore on Tuesday, after halting production there on Monday. By Wednesday morning, the agency already released a series of scathing observations on the cross contamination, sanitary issues and lack of staff training that caused the contract manufacturer to dispose of millions of AstraZeneca and J&J vaccine doses.

Brad Bolzon (Versant)

Ver­sant pulls the wraps off of near­ly $1B in 3 new funds out to build the next fleet of biotech star­tups. And this new gen­er­a­tion is built for speed

Brad Bolzon has an apology to offer by way of introducing a set of 3 new funds that together pack a $950 million wallop in new biotech creation and growth.

“I want to apologize,” says the Versant chairman and managing partner, laughing a little in the intro, “that we don’t have anything fancy or flashy to tell you about our new fund. Same team, around the same amount of capital, same investment strategy. If it ain’t broke, don’t fix it.”

But then there’s the flip side, where everything has changed. Or at least speeded into a relative blur. Here’s Bolzon:

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Jenny Rooke (Genoa Ventures)

Ear­ly Zymer­gen in­vestor Jen­ny Rooke re­flects on 'chimeras' in biotech, what it takes to spot a $500M gem

When Jenny Rooke first heard of Zymergen back in 2014, she knew she was looking at something different and exciting. The Emeryville, CA biotech held the promise of blending biology and technology to solve a huge unmet need for cost-effective chemicals — of all things — and a stellar founding team to boot.

But back then, West Coast venture capitalists didn’t see in Zymergen the one thing they were looking for in a winning biotech: therapeutic potential. Rooke, however, saw an opportunity and made her bets. Seven years later, that bet is paying off in a big way.

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Saurabh Saha at Endpoints News' #BIO19

On the heels of $250M launch, Centes­sa barges ahead with an IPO to fu­el its 10-in-1 Medicxi pipeline

Francesco De Rubertis made no secret of IPO plans for Centessa, his 10-in-1 legacy play. Barely two months later, the S-1 is in.

The hot-off-the-press filing depicts the same grand vision that the longtime VC touted when he did the rounds in February: Take the asset-centric mindset that he’s been preaching at Medicxi over the years, and roll up a bunch of biotech upstarts, with unrelated risk profiles, into 1 pharma company that can carry on the development at scale.