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.

Mi­no­ryx and Sper­o­genix ink an ex­clu­sive li­cense agree­ment to de­vel­op and com­mer­cial­ize lerigli­ta­zone in Chi­na

September 23, 2020 – Hong Kong, Beijing, Shanghai (China) and Mataró, Barcelona (Spain)  

Minoryx will receive an upfront and milestone payments of up to $78 million, as well as double digit royalties on annual net sales 

Sperogenix will receive exclusive rights to develop and commercialize leriglitazone for the treatment of X-linked adrenoleukodystrophy (X-ALD), a rare life-threatening neurological condition

FDA chief Stephen Hahn on Capitol Hill earlier this week (Getty Images)

As FDA buck­les un­der the strain of a pan­dem­ic work­load, Trump again ac­cus­es the agency of a po­lit­i­cal hit job

Peter Marks appeared before a virtual SVB Leerink audience yesterday and said that his staff at FDA’s CBER is on the verge of working around the clock. Manufacturing inspections, policy work and sponsor communications have all been pushed down the to-do list so that they can be responsive to Covid-related interactions. And the agency’s objective right now? “To save as many lives as we can,” Marks said, likening the mortality on the current outbreak as equivalent to “a nuclear bomb on a small city.”

Daniel O'Day, Gilead CEO (Kevin Dietsch/UPI/Bloomberg via Getty Images)

Play-by-play of Gilead­'s $21B Im­munomedics buy­out de­tails a fren­zied push — and mints a new biotech bil­lion­aire

Immunomedics had not really been looking for a buyout when the year began. Excited by its BLA for Trodelvy, submitted to the FDA in late 2019, executive chairman Behzad Aghazadeh started off looking for potential licensing deals and zeroed in on four potential partners, including Gilead, following January’s JP Morgan Healthcare Conference in San Francisco. Such talks advanced throughout the year, with discussions advancing to the second round in mid-August.

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The win­dow is wide open as four more biotechs join the go-go IPO class of 2020

It’s another day of hauling cash in the biopharma world as four more IPOs priced Friday and a fifth filed its initial paperwork.

The biggest offering comes from PMV Pharma, an oncology biotech focusing on p53 mutations, which raised $211.8 million after pricing shares at $18 apiece. Prelude Therapeutics, developing PRMT5 inhibitors for rare cancers, was next with a $158 million raise, pricing shares at $19 each. Graybug Vision raised $90 million after pricing at $16 per share for its wet AMD candidates, and breast cancer biotech Greenwich Lifesciences brought up the rear with a small, $7 million raise after pricing shares at $5.75.

J&J of­fers PhI/IIa da­ta show­ing its sin­gle-dose vac­cine can stir up suf­fi­cient im­mune re­sponse

Days after J&J dosed the first participants of its Phase III ENSEMBLE trial, the pharma giant has detailed the early-stage data that gave them confidence in a single-dose regimen.

Testing two dose levels either as a single dose or in a two-dose schedule spaced by 56 days in, the scientists from Janssen, the J&J subsidiary developing its vaccine, reported that the low dose induced a similar immune response as the high dose. The interim Phase I/IIa results were posted in a preprint on medRxiv.

President Donald Trump reacts after signing an executive order following his remarks on his healthcare policies yesterday in Charlotte, North Carolina (Getty Images)

Op-ed: Will phar­ma re­al­ly pay for Trump’s lat­est law­less promise to 33 mil­lion Medicare ben­e­fi­cia­ries? Not like­ly

Sitting atop the executive branch, President Donald Trump is the ultimate authority at the FDA. He can fast track any vaccine to approval himself. If it came to that, of course.

What he can’t do is unilaterally order the legislative branch to loosen the Treasury’s coffers for $6.6 billion. Nor can he command pharmaceutical companies to pay for $200 vouchers sent to 33 million Medicare beneficiaries for prescription drugs before the election.

Pal­la­dio bags $20 mil­lion Se­ries B to top­ple a prob­lem­at­ic kid­ney dis­ease drug

Palladio Biosciences just took one step further in its quest to topple Otsuka’s Samsca with its own — it hopes safer — autosomal dominant polycystic kidney disease (ADPKD) drug.

The Pennsylvania-based biotech announced a $20 million Series B on Friday, which will fund a 10-person Phase III trial of its vasopressin V2 receptor agonist, lixivaptan. CEO Alex Martin expects to read out data in the first half of next year, then launch straight into a larger pivotal Phase III study with about 1,200 participants.

New York governor Andrew Cuomo (AP Images)

An­drew Cuo­mo says New York will un­der­take its own vac­cine re­view process, and wouldn’t rec­om­mend trust­ing the fed­er­al gov­ern­ment

The concerns keep mounting over President Donald Trump’s politicization of the FDA and other federal agencies guiding the development of a safe and effective vaccine. And today, the telegenic New York governor Andrew Cuomo appeared to introduce even more politics into the matter — latest in an ongoing series of incidents that have cast the proudly independent FDA in starkly political terms.

During his daily press conference Cuomo said that the state will review any coronavirus vaccines approved by the federal government, citing a lack of trust in the Trump administration. The announcement comes one day after Trump accused the FDA of making an “extremely political” move in proposing stricter vaccine guidance.

PhII Alzheimer's fail­ure deals new blow to Roche, AC Im­mune — but the tau hy­poth­e­sis is far from dead

The leading anti-tau antibody has failed its first Phase II testing, casting a shadow on a popular target (just trailing amyloid beta) for Alzheimer’s disease.

Roche and AC Immune are quick to acknowledge disappointment in the topline readout, which suggested that semorinemab did not reduce cognitive decline among patients with early Alzheimer’s disease, who are either just starting to have symptoms or have mild manifestations.

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