Seth Lederman, Tonix Pharmaceuticals CEO

Tonix will test its mon­key­pox vac­cine in Kenya next year, and no eyes on ac­cel­er­at­ed path at FDA

As mon­key­pox spreads across dozens of coun­tries, one of the few biotechs pub­licly work­ing on a new vac­cine for the virus is gear­ing up to en­ter the clin­ic in the first half of next year.

The study of Tonix Phar­ma­ceu­ti­cals’ live virus vac­cine will oc­cur at Kenya Med­ical Re­search In­sti­tute, or KEM­RI, in the East African coun­try, the part­ners said Thurs­day. While Kenya has no re­port­ed cas­es of the virus that leads to painful le­sions, the coun­try is close to the De­mo­c­ra­t­ic Re­pub­lic of the Con­go, which had 163 cas­es as of Ju­ly 27, ac­cord­ing to a CDC track­er.

Sim­i­lar to the ini­tial roll­out of Covid-19 jabs, the US and Eu­rope have con­sumed much of the vac­cine sup­ply. Africa is en­tire­ly de­void of vac­cines for the virus, the con­ti­nent’s CDC di­rec­tor said Thurs­day. Vac­cines are cru­cial in pub­lic health. Africa is home to the on­ly deaths thus far of the virus that in re­cent days was de­clared a pub­lic health emer­gency by the World Health Or­ga­ni­za­tion. The Biden ad­min­is­tra­tion is re­port­ed­ly on track to give the virus a sim­i­lar des­ig­na­tion.

“Be­cause of the need in Africa, we think that the de­vel­op­ment there will go faster, but it is our in­ten­tion to file an IND and do stud­ies in the Unit­ed States,” Tonix CEO Seth Le­d­er­man told End­points News. “The view of the Unit­ed States right now is that we do have two ap­proved vac­cines.”

“It’s hard for me to imag­ine an ac­cel­er­at­ed path­way in the Unit­ed States, and that’s why I think that this part­ner­ship with KEM­RI and do­ing stud­ies in Kenya is good for both par­ties,” the CEO said. “Kenya has po­ten­tial to get a vac­cine that they don’t re­al­ly have right now, and we have an op­por­tu­ni­ty to get da­ta that we can bring back to the FDA to make a more com­pelling case that we may have a safe­ty ad­van­tage for a live virus vac­cine.”

Stud­ies of the vac­cine could lead to an­oth­er tool in the ar­se­nal against the virus, with two vac­cines al­ready in use in the US — Bavar­i­an Nordic’s Jyn­neos and Emer­gent BioSo­lu­tions’ ACAM2000, bought from Sanofi a few years ago. Re­port­ed cas­es tal­lied up to more than 20,600 as of Ju­ly 27.

The Phase I study in Kenya will be­gin in the first half of next year, with KEM­RI lead­ing the study and Tonix sup­port­ing reg­u­la­to­ry fil­ings and oth­er lo­gis­tics. If mon­key­pox cas­es arise in Kenya, then a Phase II clin­i­cal tri­al might serve as a barom­e­ter of ef­fi­ca­cy, Le­d­er­man said.

With two vac­cines al­ready on the mar­ket — al­beit in lim­it­ed sup­plies to the point where hun­dreds of peo­ple have wait­ed for hours in lines at clin­ics, bars and pub­lic saunas in cities like Chica­go — the New Jer­sey biotech is un­like­ly to test the vac­cine state­side in the near fu­ture, Le­d­er­man said.

Tonix has been work­ing on mon­key­pox and small­pox for close to a decade, Le­d­er­man said. The com­pa­ny’s vac­cine, dubbed TNX-801, is traced back to a re­search col­lab­o­ra­tion with the De­part­ment of Cell Bi­ol­o­gy at the Uni­ver­si­ty of Al­ber­ta.

In a pre­vi­ous so-called chal­lenge study, Tonix gave the vac­cine to eight non-hu­man pri­mates and then de­liv­ered a lethal dose of mon­key­pox in­to their lungs via the in­tra­tra­cheal route. All eight were pro­tect­ed and did not de­vel­op le­sions, Le­d­er­man said.

For years, the biotech has been at­tempt­ing to treat fi­bromyal­gia and is cur­rent­ly in a Phase III tri­al for the con­di­tion, as well as near­ing mid-stage stud­ies in PTSD, de­pres­sion and long Covid.

Ear­li­er this week, the biotech said it will hand back the rights to an in­hibitor of the virus be­hind Covid-19. Le­d­er­man said “the biotech eq­ui­ty cap­i­tal mar­kets are chal­leng­ing” and the com­pa­ny is fo­cused on ad­vanc­ing its oth­er pro­grams.

But Tonix is not out of Covid, with a Phase II study in long Covid slat­ed to start this quar­ter and a col­lab­o­ra­tion with Co­lum­bia Uni­ver­si­ty, which is tak­ing an­ti­bod­ies from pa­tients who have re­cov­ered from the pan­dem­ic dis­ease, that might spur re­search in­to a new Covid-19 an­ti­body, Le­d­er­man said.

“We are go­ing to be very ac­tive­ly pur­su­ing an­ti­bod­ies to new vari­ants as they come up,” the CEO said, not­ing it could be­come a “niche mar­ket where you’re de­vel­op­ing these an­ti­bod­ies, they’re go­ing to have a rel­a­tive­ly short lifes­pan in terms of treat­ment, but there are peo­ple for whom these are life­sav­ing, so that’s the an­gle to the mar­ket that we’re go­ing af­ter.”

UP­DAT­ED: In a fresh dis­ap­point­ment, Am­gen spot­lights a ma­jor safe­ty is­sue with KRAS com­bo

Amgen had hoped that its latest study matching its landmark KRAS G12C drug Lumakras with checkpoint inhibitors would open up its treatment horizons and expand its commercial potential. Instead, the combo spurred safety issues that blunted efficacy and forced the pharma giant to alter course on its treatment strategy, once again disappointing analysts who have been tracking the drug’s faltering sales and limited therapeutic reach.

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Ad­dress­ing the ‘Ca­pac­i­ty Crunch’ with a Scal­able Plat­form Process Ap­proach

The field of gene therapy has been diligently moving forward over the past several decades to bring potentially life-saving treatments to patients with genetic diseases. In addition to two approved adeno-associated viral (AAV) gene therapies, there are more than 250 AAV gene therapies in various clinical trial stages.1 AAV vectors remain the most frequently used vector for delivering therapeutic transgenes to target tissues due to their demonstrated and lasting clinical efficacy and extensive safety track record. As AAV therapies advance through clinical trials and into commercialization, many biotech companies are turning to contract development and manufacturing organizations (CDMOs) to prepare their programs for late-stage clinical and commercial scale manufacturing. Given the scope and scale of the manufacturing needs that will accompany regulatory approvals for these assets, CDMOs continue to expand their capacity to meet the needs of increasing prevalent patient populations. However, despite rapid growth, projected gene therapy manufacturing demands still outpace the collective capacity of the CDMO industry.

A $5B Pfiz­er buy­out? Am­gen, Gilead head­line M&A Thurs­day; Al­ny­lam's AT­TR sweep; An­drew Lo's rare dis­ease quest; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

One of the cool things about adding EndpointsPharma to the daily roster is that my colleagues can now dedicate time to tracking quarterly updates and tuning into calls with Big Pharma companies. Check out their dispatch from the Q2 earnings below.

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Bob Bradway, Amgen CEO (Justin Kase Conder/AP Images for Amgen)

UP­DAT­ED: Am­gen chief Brad­way nabs a rare dis­ease play­er in $4B buy­out as the M&A tem­po ac­cel­er­ates

Amgen CEO Bob Bradway is bellying up to the M&A table today, scooping up the newly anointed commercial biotech ChemoCentryx $CCXI and its recently approved rare disease drug for $3.7 billion out of the cash stockpile. The deal comes in at $52 a share — a hefty increase over the $24.11 close yesterday.

Bradway and the Amgen team get a drug called Tavneos (avacopan) in the deal, a complement factor C5a inhibitor OK’d to treat anti-neutrophil cytoplasmic autoantibody (ANCA)-vasculitis, an autoimmune disease which can be lethal.

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Albert Bourla, Pfizer CEO (Laurent Gillieron/Keystone via AP)

Break­ing: Pfiz­er in hot pur­suit of a $5B buy­out of Glob­al Blood Ther­a­peu­tics — re­port

Pfizer CEO Albert Bourla has vowed to leave no stone unturned in the search for new biotech deals, and the BD team is not letting him down.

The Wall Street Journal reported today that Pfizer is in the final stages of acquiring Global Blood Therapeutics for $5 billion. According to the Journal report, though, Pfizer is not the only buyer at the deal table and while the pharma giant may be close to clinching it, there are no guarantees it will continue.

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George Yancopoulos, Regeneron president and CSO (Brendan McDermid/Reuters/Alamy)

George Yan­copou­los says he's on the trail of the holy grail: ‘This could rep­re­sent the next break­through for im­munother­a­py’

Two of the most outspoken — and successful — drug developers in biotech say they’ve collected early-stage clinical data that are pointing them down the trail to the holy grail in cancer immunotherapy R&D.

While analysts largely busied themselves today with chronicling the ongoing success of Regeneron’s two big cash cows — Dupixent and Eylea — chief scientist George Yancopoulos and CEO Len Schleifer used the Q2 call to spotlight their early success with a combination of the “homegrown” PSMAxCD28 costimulatory bispecific antibody REGN5678 in combination with their PD-1 checkpoint Libtayo. The presentation comes just weeks after Regeneron completed a deal to gather all rights to the PD-1 that had been in Sanofi’s hands. And the two top execs are unstinting in their praise of the potential of a whole set of costimulatory pipeline projects which they say may finally deliver the long-awaited next-level approach to broadening the immunotherapy field of drugs.

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(AP Photo/Richard Vogel, File)

US de­clares mon­key­pox a na­tion­al health emer­gency, as new drug­mak­ers con­sid­er en­ter­ing vac­cine race

Rising monkeypox cases have put the US on high alert as it announces a national health emergency, which grants the government more power in its response.

The news comes as Bavarian Nordic continues to fill orders for its Jynneos vaccine and other companies – including Moderna – consider jumping into the vaccine race. Meanwhile, the New York Times reports that the US has allowed around 20 million doses of smallpox vaccine in its stockpile to expire.

Al Sandrock, Voyager Therapeutics CEO

Al San­drock prunes his post-Bio­gen voy­age with sim­i­lar fo­cus on ALS

Al Sandrock is narrowing the focus of Voyager Therapeutics, concentrating on CNS diseases that were the hallmark of his time leading R&D at Biogen, including an emphasis on a familial form of ALS for which his former employer is getting a speedy review at the FDA.

Less than six months into his journey as CEO at Voyager, Sandrock is focusing the preclinical pipeline on Alzheimer’s disease, GBA1 Parkinson’s disease and SOD1 amyotrophic lateral sclerosis, the rare form of ALS for which the FDA will decide whether to approve Biogen’s tofersen by Jan. 25, 2023.

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Pfiz­er ter­mi­nates PhI­II study of rare car­dio­vas­cu­lar drug picked up in $11.4B Ar­ray ac­qui­si­tion

While Pfizer’s $11.4 billion acquisition of Array BioPharma in the summer of 2019 was mainly focused on oncology, namely Braftovi and Mektovi, there were a few non-cancer assets, including a Phase III drug being tested in a rare cardiovascular disease.

The late-stage trial is now being axed, alongside any further development of the oral small molecule, the pharma giant disclosed after the closing bell on Wednesday. Based on an interim futility analysis of the global Phase III REALM-DCM trial, Pfizer determined a path forward was not in its best interest. Pfizer no longer expected the study would meet its primary goal.

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