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.

Sen. Ron Wyden (D-OR) (Francis Chung/E&E News/Politico via AP Images)

In­fla­tion re­bates in­com­ing: Wyden calls on CMS to move quick­ly as No­var­tis CEO pledges re­ver­sal

Senate Finance Chair Ron Wyden (D-OR) this week sent a letter to the head of the Centers for Medicare & Medicaid Services seeking an update on how and when new inflation-linked rebates will take effect for drugs that see major price spikes.

The newly signed Inflation Reduction Act requires manufacturers to pay a rebate to Medicare when they increase drug prices faster than the rate of inflation.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Ma­gen­ta halts stem cell work and may sell it­self fol­low­ing pa­tient death, clin­i­cal hold

Magenta Therapeutics said it is halting work on its stem cell transplant drug pipeline and may sell itself, a week after the company reported the death of a patient in an early stage trial of its antibody-drug conjugate.

The Cambridge, MA-based company said it will conduct a “review of strategic alternatives,” and that could include an “acquisition, merger, business combination, or other transaction.”

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

How to use ex­ter­nal con­trols: FDA spells out think­ing in new draft guid­ance

The use of real-world evidence to inform the FDA’s decision-making continues apace, with the agency releasing new draft guidance yesterday on how sponsors can compare outcomes of trial participants receiving a test treatment with outcomes in a group of people external to the trial.

The practice of externally controlled trials is common, particularly in oncology or other difficult areas where it’s not ethical or feasible to use internal controls.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

The Big Phar­ma axe: Mer­ck cuts chikun­gun­ya vax, Bris­tol My­ers drops Cy­tomX-part­nered pro­gram, and more

As fourth quarter earnings come in, Big Pharmas are disclosing changes to their pipelines during their investor calls, and sometimes more quietly in presentation appendices.

Merck dropped its chikungunya vaccine candidate, which completed a Phase II study. Merck acquired the vaccine through its purchase of Themis Bioscience in 2020. In developing a vaccine for chikungunya, a mosquito-borne virus, Valneva is the frontrunner, as it submitted its vaccine to the FDA at the end of December.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Goldfinch Bio CEO Tony Johnson (L) and Karuna Therapeutics CEO Bill Meury

Karuna li­cens­es Goldfinch as­sets to com­pete with Boehringer In­gel­heim in neu­ro­science

Karuna Therapeutics is looking to compete with Boehringer Ingelheim on depression and anxiety with a new license to Goldfinch Bio’s assets, starting with $15 million to the shuttered biotech.

Karuna steps into an arena already being tested by Boehringer in multiple Phase II studies — the two are targeting transient receptor potential canonical 4 and 5, or TRPC4/5, which is thought to have a role in neuroscience indications. Goldfinch’s asset went through a Phase II in kidney diseases, but Karuna’s sights are set on mood and anxiety disorders for now.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Teresa Graham, incoming Roche Pharmaceuticals CEO

In­com­ing Roche CEO builds out his top team, tap­ping Genen­tech vet to lead phar­ma di­vi­sion

Roche announced another leadership shuffle Thursday morning – the head of global product strategy, Teresa Graham, will take over as CEO of Roche Pharmaceuticals in March while the company’s corporate executive committee will make a spot for Levi Garraway, CMO and executive VP of global product development.

Thomas Schinecker will take over the top spot as Roche group CEO in March, leaving his spot as head of diagnostics.

Roche's headquarters in Basel, Switzerland (Kyle LaHucik for Endpoints News)

Roche ditch­es fi­nal PhI­II for can­cer hope­ful, re­ports set­back for key drug in $1.4B buy­out

Over the past few years, Roche has released news about its AKT inhibitor ipatasertib in drips — most of them negative. The drug yielded mixed data in a key prostate cancer trial, Phase III flops in triple-negative breast cancer forced the pharma giant to pull the plug there, and in mid-2022 Roche trimmed two more early-stage indications in prostate cancer after completing the trials.

Now, the last piece of the program is gone.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.

Trodelvy notch­es a win in most com­mon form of breast can­cer

Following a promise last year to go “big and fast in breast cancer,” Gilead has secured a win for Trodelvy in the most common form.

The drug was approved to treat HR-positive, HER2-negative breast cancer patients who’ve already received endocrine-based therapy and at least two other systemic therapies for metastatic cancer, Gilead announced on Friday.

Trodelvy won its first indication in metastatic triple-negative breast cancer back in 2020, and has since added urothelial cancer to the list. HR-positive HER2-negative breast cancer accounts for roughly 70% of new breast cancer cases worldwide per year, according to senior VP of oncology clinical development Bill Grossman, and many patients develop resistance to endocrine-based therapies or worsen on chemotherapy.

Sanofi scraps PhI­II tri­al for Prin­cip­ia drug af­ter re­view­ing com­pe­ti­tion

Months after the FDA placed Phase III trials of Sanofi’s BTK inhibitor on hold, the company is winding down one of the studies.

Sanofi reported in its Q4 earnings that the URSA study “was discontinued after careful evaluation of the emerging competitive treatment landscape in” myasthenia gravis, a rare disease that causes muscle weakness.

The Phase III, placebo-controlled trial was testing tolebrutinib in patients with the moderate-to-severe form of the disease. It started in late 2021, according to records on clinicaltrials.gov, and was originally designed to recruit 154 participants who were receiving the standard of care.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 158,500+ biopharma pros reading Endpoints daily — and it's free.