FDA launch­es crim­i­nal probe in­to unau­tho­rized her­pes vac­cine R&D backed by Pe­ter Thiel

The Food and Drug Ad­min­is­tra­tion has launched a crim­i­nal in­ves­ti­ga­tion in­to re­search by a South­ern Illi­nois Uni­ver­si­ty pro­fes­sor who in­ject­ed peo­ple with his unau­tho­rized her­pes vac­cine, Kaiser Health News has learned.

SIU pro­fes­sor William Hal­ford, who died in June, in­ject­ed par­tic­i­pants with his ex­per­i­men­tal her­pes vac­cine in St. Kitts and Nevis in 2016 and in Illi­nois ho­tel rooms in 2013 with­out safe­ty over­sight that is rou­tine­ly per­formed by the FDA or an in­sti­tu­tion­al re­view board.

Ac­cord­ing to four peo­ple with knowl­edge about the in­quiry, the FDA’s Of­fice of Crim­i­nal In­ves­ti­ga­tions is look­ing in­to whether any­one from SIU or Hal­ford’s for­mer com­pa­ny, Ra­tio­nal Vac­cines, vi­o­lat­ed FDA reg­u­la­tions by help­ing Hal­ford con­duct unau­tho­rized re­search. The probe is al­so look­ing at any­one else out­side the com­pa­ny or uni­ver­si­ty who might have been com­plic­it, ac­cord­ing to the sources who asked not to be iden­ti­fied be­cause of the sen­si­tiv­i­ty of the mat­ter.

The FDA rarely pros­e­cutes re­search vi­o­la­tions, usu­al­ly choos­ing to ad­min­is­tra­tive­ly sanc­tion or ban re­searchers or com­pa­nies from fu­ture clin­i­cal tri­als, le­gal ex­perts said. Even so, the agency is em­pow­ered to pur­sue as a crime the unau­tho­rized de­vel­op­ment of vac­cines and drugs — and some­times goes af­ter such cas­es to send a mes­sage.

In this case, hu­man-sub­ject vi­o­la­tions would be deemed es­pe­cial­ly se­ri­ous giv­en Hal­ford was not a med­ical doc­tor and had in­ject­ed peo­ple with his ex­per­i­men­tal vac­cine with­out any rou­tine over­sight, ex­perts said.

“Since the re­search ap­pears to be an ef­fort to to­tal­ly evade FDA over­sight and is egre­gious, it makes sense the FDA would in­ves­ti­gate it as a crim­i­nal mat­ter,” said Pa­tri­cia Zettler, a for­mer FDA lawyer who was told of the crim­i­nal in­ves­ti­ga­tion by KHN. “There is a de­ter­rent ef­fect for oth­ers who might con­sid­er this a very brazen way to get out of hu­man sub­ject and FDA re­quire­ments.”

The FDA de­clined to com­ment. Ra­tio­nal Vac­cines did not re­spond to re­quests for com­ment. An SIU spokes­woman said, with­out elab­o­ra­tion, “The gov­ern­ment is in­ves­ti­gat­ing and we are co­op­er­at­ing.”

Any re­sult­ing crim­i­nal pros­e­cu­tion from the in­ves­ti­ga­tion could have po­lit­i­cal ram­i­fi­ca­tions.

Ra­tio­nal Vac­cines was co-found­ed with Hol­ly­wood film­mak­er Agustín Fer­nán­dez III and the com­pa­ny re­ceived mil­lions of dol­lars in pri­vate in­vest­ment from in­vestors af­ter the Caribbean tri­al, in­clud­ing from bil­lion­aire Pe­ter Thiel.

Thiel, who for months has re­fused to re­spond to ques­tions from KHN, con­tributed to Pres­i­dent Don­ald Trump’s cam­paign and is a high-pro­file crit­ic of the FDA. Thiel is part of a larg­er lib­er­tar­i­an move­ment to roll back FDA reg­u­la­tions to speed up med­ical in­no­va­tion.

The sources fa­mil­iar with the in­quiry said the FDA’s Of­fice of Crim­i­nal In­ves­ti­ga­tions, which has dozens of of­fices across the coun­try, be­gan to ag­gres­sive­ly pur­sue the case weeks ago.

The in­ves­ti­ga­tors have in­ter­viewed wit­ness­es across the coun­try, ask­ing them to iden­ti­fy Hal­ford’s as­so­ciates, and have de­scribed his ac­tions as pos­si­ble vi­o­la­tions of hu­man-sub­ject guide­lines and of FDA reg­u­la­tions, the sources told KHN.

The in­ves­ti­ga­tors al­so have ex­pressed in­ter­est in whether Hal­ford’s for­mer as­so­ciates at the uni­ver­si­ty or oth­er re­searchers and med­ical pro­fes­sion­als out­side the uni­ver­si­ty might have helped or known about his con­duct, the sources said. They al­so have raised ques­tions about the com­pa­ny’s knowl­edge of the vi­o­la­tions.

Ra­tio­nal Vac­cines helped over­see the Caribbean tri­al, but the 2013 ho­tel in­jec­tions took place be­fore the com­pa­ny was formed.

Un­der a Supreme Court rul­ing, a cor­po­rate of­fi­cial may be pros­e­cut­ed for a crim­i­nal mis­de­meanor of­fense un­der the Fed­er­al Food, Drug and Cos­met­ic Act even with­out proof that the of­fi­cial act­ed with in­tent or ac­tu­al knowl­edge of the of­fense.

Ini­tial­ly, uni­ver­si­ty of­fi­cials and Ra­tio­nal Vac­cines pub­licly de­fend­ed Hal­ford’s re­search. Ra­tio­nal Vac­cines has said it con­sid­ered the 2016 tri­al a suc­cess — though it is un­clear what da­ta it used to sup­port that claim.

Af­ter KHN’s in­ves­ti­ga­tion re­vealed that Hal­ford in­ject­ed peo­ple in the Unit­ed States, not just in the Caribbean, Ra­tio­nal Vac­cines took down its web­site, al­though it had vowed to con­tin­ue re­search.

SIU, a state uni­ver­si­ty with a med­ical school in Spring­field, IL, ini­tial­ly said it bore no re­spon­si­bil­i­ty for the ex­per­i­ments be­cause Hal­ford con­duct­ed the re­search in­de­pen­dent­ly and over­seas.

Af­ter Kaiser Health News raised ques­tions about Hal­ford’s prac­tices, the De­part­ment of Health and Hu­man Ser­vices asked the uni­ver­si­ty to de­ter­mine whether his ac­tiv­i­ties vi­o­lat­ed the in­sti­tu­tion’s pledge to HHS to fol­low hu­man-sub­ject safe­ty pro­to­cols for all re­search. SIU’s med­ical school re­ceives about $9 mil­lion a year in fed­er­al re­search dol­lars.

SIU has since ac­knowl­edged that Hal­ford’s con­duct vi­o­lat­ed uni­ver­si­ty rules and US laws. Uni­ver­si­ty of­fi­cials have de­nied know­ing about his mis­con­duct, an as­ser­tion that FDA in­ves­ti­ga­tors are still prob­ing, the sources said.

Hal­ford’s ac­tions al­ready raised un­usu­al le­gal ques­tions be­cause the FDA would not or­di­nar­i­ly have ju­ris­dic­tion over clin­i­cal tri­als when they oc­cur over­seas and the re­searchers have not sought FDA ap­proval.

It’s al­so un­clear where Hal­ford man­u­fac­tured the vac­cine.

If it was man­u­fac­tured in the Unit­ed States, the FDA like­ly has ju­ris­dic­tion, said Zettler, a law pro­fes­sor at Geor­gia State Uni­ver­si­ty.

The OCI of­ten goes af­ter such cas­es of con­t­a­m­i­nat­ed food, coun­ter­feit or off-la­bel phar­ma­ceu­ti­cals. The of­fice was cre­at­ed in the wake of a 1988 scan­dal in which phar­ma­ceu­ti­cal ex­ec­u­tives bribed FDA of­fi­cials in ex­change for speed­ing up gener­ic drug ap­provals.

While rare, the OCI oc­ca­sion­al­ly pur­sues re­search abus­es as a crime. A Glax­o­SmithK­line re­searcher, for in­stance, plead­ed guilty in 2010 to charges re­lat­ed to her fab­ri­ca­tion of da­ta in a study of chil­dren tak­ing the an­ti­de­pres­sant Pax­il. Glax­o­SmithK­line lat­er agreed to plead guilty and to pay $3 bil­lion to re­solve its crim­i­nal and civ­il li­a­bil­i­ty in the case.


By Marisa Tay­lor. Orig­i­nal­ly post­ed at Kaiser Health News, a na­tion­al health pol­i­cy news ser­vice that is part of the non­par­ti­san Hen­ry J Kaiser Fam­i­ly Foun­da­tion.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,600+ biopharma pros reading Endpoints daily — and it's free.

It's not per­fect, but it's a good start: FDA pan­elists large­ly en­dorse Aim­mune's peanut al­ler­gy ther­a­py

Two days after a fairly benign review from FDA staff, an independent panel of experts largely endorsed the efficacy and safety of Aimmune’s peanut allergy therapy, laying the groundwork for approval with a risk evaluation and mitigation strategy (REMS).

Traditionally, peanut allergies are managed by avoidance, but the threat of accidental exposure cannot be nullified. Some allergists have devised a way to dose patients off-label with peanut protein derived from supermarket products to wean them off their allergies. But the idea behind Aimmune’s product was to standardize the peanut protein, and track the process of desensitization — so when accidental exposure in the real world invariably occurs, patients are less likely to experience a life-threatening allergic reaction.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,600+ biopharma pros reading Endpoints daily — and it's free.

Lisa M. DeAngelis, MSKCC

MSK picks brain can­cer ex­pert Lisa DeAn­ge­lis as its next CMO — fol­low­ing José Basel­ga’s con­tro­ver­sial ex­it

It’s official. Memorial Sloan Kettering has picked a brain cancer expert as its new physician-in-chief and CMO, replacing José Baselga, who left under a cloud after being singled out by The New York Times and ProPublica for failing to properly air his lucrative industry ties.

His replacement, who now will be in charge of MSK’s cutting-edge research work as well as the cancer care delivered by hundreds of practitioners, is Lisa M. DeAngelis. DeAngelis had been chair of the neurology department and co-founder of MSK’s brain tumor center and was moved in to the acting CMO role in the wake of Baselga’s departure.

Penn team adapts CAR-T tech, reengi­neer­ing mouse cells to treat car­diac fi­bro­sis

After establishing itself as one of the pioneer research centers in the world for CAR-T cancer therapies, creating new attack vehicles to eradicate cancer cells, a team at Penn Medicine has begun the tricky transition of using the basic technology for heart repair work.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,600+ biopharma pros reading Endpoints daily — and it's free.

Tal Zaks. Moderna

The mR­NA uni­corn Mod­er­na has more ear­ly-stage hu­man da­ta it wants to show off — reach­ing new peaks in prov­ing the po­ten­tial

The whole messenger RNA field has attracted billions of dollars in public and private investor cash gambled on the prospect of getting in on the ground floor. And this morning Boston-based Moderna, one of the leaders in the field, wants to show off a few more of the cards it has to play to prove to you that they’re really in the game.

The whole hand, of course, has yet to be dealt. And there’s no telling who gets to walk with a share of the pot. But any cards on display at this point — especially after being accused of keeping its deck under lock and key — will attract plenty of attention from some very wary, and wired, observers.

“In terms of the complexity and unmet need,” says Tal Zaks, the chief medical officer, “this is peak for what we’ve accomplished.”

Moderna has two Phase I studies it wants to talk about now.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,600+ biopharma pros reading Endpoints daily — and it's free.

Sanofi takes a $260M hit to ex­tri­cate it­self from a dis­as­trous al­liance with Lex­i­con

Sanofi spent $300 million in cash to get into a $1.7 billion alliance with Lexicon on their SGLT1/2 diabetes drug sotagliflozin. And now that the drug has been spurned by the FDA after burning through a program that provided mixed late-stage data and a late shot at a last-place finish, the French pharma giant is forking over another $260 million to get out of the deal.

Sanofi’s unhappiness was already apparent when the company — now under new CEO Paul Hudson — posted a statement back in July that they were dropping the deal. But it wasn’t that simple. 

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,600+ biopharma pros reading Endpoints daily — and it's free.

Rit­ter bombs fi­nal PhI­II for sole lac­tose in­tol­er­ance drug — shares plum­met

More than two years ago Ritter Pharmaceuticals managed to find enough silver lining in its Phase IIb/III study — after missing the top-line mark — to propel its lactose intolerance toward a confirmatory trial. But as it turned out, the enthusiasm only set the biotech and its investors up to be sorely disappointed.

This time around there’s little left to salvage. Not only did RP-G28 fail to beat placebo in reducing lactose intolerance symptoms, patients in the treatment group actually averaged a smaller improvement. On a composite score measuring symptoms like abdominal pain, cramping, bloating and gas, patients given the drug had a mean reduction of 3.159 while the placebo cohort saw a 3.420 drop on average (one-sided p-value = 0.0106).

Ear­ly snap­shot of Ad­verum's eye gene ther­a­py sparks con­cern about vi­sion loss

An early-stage update on Adverum Biotechnologies’ intravitreal gene therapy has triggered investor concern, after patients with wet age-related macular degeneration (AMD) saw their vision deteriorate, despite signs that the treatment is improving retinal anatomy.

Adverum, on Wednesday, unveiled 24-week data from the OPTIC trial of its experimental therapy, ADVM-022, in six patients who have been administered with one dose of the therapy. On average, patients in the trial had severe disease with an average of 6.2 anti-VEGF injections in the eight months prior to screening and an average annualized injection frequency of 9.3 injections.

Alex Ar­faei trades his an­a­lyst's post for a new role as biotech VC; Sanofi vet heads to Vi­for

Too often, Alex Arfaei arrived too late. 

An analyst at BMO Capital Markets, he’d meet with biotech or pharmaceutical heads for their IPO or secondary funding and his brain, trained on a biology degree and six years at Merck and Endo, would spring with questions: Why this biomarker? Why this design? Why not this endpoint? Not that he could do anything about it. These execs were coming for clinical money; their decisions had been made and finalized long ago.