Tiny In­nate re­ports a PhI­Ib dis­as­ter, bat­ter­ing shares — and a NY con­gress­man

Reps. Chris Collins, R-NY, left, and Robert Pit­tenger, R-NC, leave a meet­ing of the House Re­pub­li­can Con­fer­ence in the Capi­tol on June 7, 2017. CQ Roll Call

Just about every­thing the lit­tle Aus­tralian biotech In­nate Im­munother­a­peu­tics tracked in its Phase IIb study of its ex­per­i­men­tal drug for mul­ti­ple scle­ro­sis points to a dis­as­ter. The da­ta on all 93 pa­tients in the study demon­strate a clear fail­ure of the drug to de­liv­er an im­prove­ment for pa­tients on any of “mul­ti­ple” end­points. The dropout rate in the drug arm was high. And the rate of se­ri­ous ad­verse events was high­er in the drug arm than in the con­trol group.

Rep. Chris Collins (R-NY)

In­nate (ASX Code: IIL) is go­ing back over the da­ta to see if the per pro­to­col re­sults on just the pa­tients who had com­plet­ed a siz­able por­tion of the study did any bet­ter. But in­ves­ti­ga­tors aren’t try­ing to en­cour­age any­one to be­lieve in MIS416, de­liv­ered by IV once a week for a year. They say there is no rea­son to ex­pect a change in out­comes.

All that is bad enough on its own. But even so, you may nev­er have heard about any of this but for one fact: A promi­nent US con­gress­man bet the ranch on this com­pa­ny’s stock — and ev­i­dent­ly lost vir­tu­al­ly all of it. In­nate’s stock al­most dis­ap­peared af­ter in­vestors got a look at the da­ta, falling to a few pen­nies a share.

Bloomberg re­porters, who have been cov­er­ing this sto­ry, did the math and con­clud­ed that New York Re­pub­li­can Chris Collins, In­nate’s biggest share­hold­er and a long­time be­liev­er, lost $16.7 mil­lion af­ter tak­ing the fli­er. And ev­i­dent­ly a num­ber of oth­er Re­pub­li­cans, in­clud­ing HHS Sec­re­tary Tom Price, al­so lost mon­ey as well.

HHS Sec­re­tary Tom Price

The con­nec­tions be­tween law­mak­ers in charge of health­care pol­i­cy and In­nate have now re­port­ed­ly in­spired an ethics in­ves­ti­ga­tion in­to the role Collins played in at­tract­ing in­vestors to the com­pa­ny. And in a sto­ry this morn­ing, The Buf­fa­lo News re­port­ed that the FDA had just grant­ed the biotech an IND to launch tri­als in the US, void­ing the con­gress­man’s claim that he had no con­flicts be­cause the biotech nev­er had busi­ness in front of US reg­u­la­tors.

The biggest les­son from all this, though, may be that any­one who bets a large por­tion of their wealth on a biotech long shot like this — based in Syd­ney — doesn’t know much about in­vest­ing, biotech or health­care. Collins, for his part, doesn’t agree. “For those that in­vest­ed in In­nate, in­clud­ing me, we all were so­phis­ti­cat­ed in­vestors who were aware of the in­her­ent risk,” Collins says in a pre­pared state­ment. He adds: “For every suc­cess­ful drug, there are count­less num­bers that fail. That’s how to­day’s sys­tem works.”

Si­mon Wilkin­son, In­nate Im­munother­a­peu­tics’ chief ex­ec­u­tive said: “These re­sults are a shock and def­i­nite­ly not what we were ex­pect­ing based on our pre­vi­ous clin­i­cal ex­pe­ri­ence with MIS416 and the re­port­ing of treat­ment ben­e­fits we have re­ceived from many com­pas­sion­ate use pa­tients over an ex­ten­sive 8-year pe­ri­od. These da­ta will be as dis­tress­ing to them as they will be for all the stake­hold­ers who were re­ly­ing on the out­come of this study.”

Wilkin­son can count his biggest in­vestor and biggest los­er as one of the most bad­ly shocked of all.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Pfiz­er's big block­buster Xel­janz flunks its post-mar­ket­ing safe­ty study, re­new­ing harsh ques­tions for JAK class

When the FDA approved Pfizer’s JAK inhibitor Xeljanz for rheumatoid arthritis in 2012, they slapped on a black box warning for a laundry list of adverse events and required the New York drugmaker to run a long-term safety study.

That study has since become a consistent headache for Pfizer and their blockbuster molecule. Last year, Pfizer dropped the entire high dose cohort after an independent monitoring board found more patients died in that group than in the low dose arm or a control arm of patients who received one of two TNF inhibitors, Enbrel or Humira.

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Covid-19 roundup: EU and As­traZeneca trade blows over slow­downs; Un­usu­al unions pop up to test an­ti­bod­ies, vac­cines

After coming under fire for manufacturing delays last week, AstraZeneca’s feud with the European Union has spilled into the open.

The bloc accused the pharma giant on Wednesday of pulling out of a meeting to discuss cuts to its vaccine supplies, the AP reported. AstraZeneca denied the reports, saying it still planned on attending the discussion.

Early Wednesday, an EU Commission spokeswoman said that “the representative of AstraZeneca had announced this morning, had informed us this morning that their participation is not confirmed, is not happening.” But an AstraZeneca spokesperson later called the reports “not accurate.”

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Adeno-associated virus-1 illustration; the use of AAVs resurrected the gene therapy field, but companies are now testing the limits of a 20-year-old technology (File photo, Shutterstock)

Af­ter 3 deaths rock the field, gene ther­a­py re­searchers con­tem­plate AAV's fu­ture

Nicole Paulk was scrolling through her phone in bed early one morning in June when an email from a colleague jolted her awake. It was an article: Two patients in an Audentes gene therapy trial had died, grinding the study to a halt.

Paulk, who runs a gene therapy lab at the University of California, San Francisco, had planned to spend the day listening to talks at the American Association for Cancer Research annual meeting, which was taking place that week. Instead, she skipped the conference, canceled every work call on her calendar and began phoning colleagues across academia and industry, trying to figure out what happened and why. All the while, a single name hung in the back of her head.

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Pascal Soriot, AP

As­traZeneca CEO Pas­cal So­ri­ot sev­ers an un­usu­al board con­nec­tion, steer­ing clear of con­flicts while re­tain­ing im­por­tant al­liances

CSL Behring chief Paul Perreault scored an unusual coup last summer when he added AstraZeneca CEO Pascal Soriot to the board, via Zoom. It’s rare, to say the least, to see a Big Pharma CEO take any board post in an industry where interests can simultaneously connect and collide on multiple levels of operations.

The tie set the stage for an important manufacturing connection. The Australian pharma giant agreed to supply the country with 10s of millions of AstraZeneca’s Covid-19 vaccine, once it passes regulatory muster.

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Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Anthony Fauci, NIAID director (AP Images)

As new Covid-19 task force gets un­der­way, threat looms of vac­cine, mon­o­clon­al an­ti­body-re­sis­tant vari­ants

Hours before President Biden’s Covid-19 team gave their first virtual press conference, the famed AIDS researcher David Ho delivered concerning news in a new pre-print: SARS-CoV-2 B.1.351, the variant that emerged in South Africa, is “markedly more resistant” to antibodies from convalescent plasma and vaccinated individuals.

The news for several monoclonal antibodies, including Eli Lilly’s bamlanivimab, was even worse: Their ability to neutralize was “completely or markedly abolished,” Ho wrote. Lilly’s antibody cocktail, which was just shown to dramatically reduce the risk of hospitalizations or death, also became far less potent.

Florian Brand (L) and Srinivas Rao (ATAI)

Psy­che­del­ic biotech ATAI hopes to ex­pand port­fo­lio through re­search part­ner­ship with Mass Gen­er­al

Psychedelics have made a comeback for mental health research, with companies like startup biotech ATAI Life Sciences raising millions and earning the backing of prominent investors like Peter Thiel, but there’s a hole at the heart of the resurgence: Researchers still don’t fully understand how they work.

A new partnership between ATAI and world-renowned Mass General Hospital hopes to change that.

Bomb squad called to As­traZeneca vac­cine plant; Lu­men nabs CARB-X award for low-cost an­tidiar­rheal

A plant located in Wrexham, Wales that is packing the Oxford/AstraZeneca Covid-19 vaccine into vials was surrounded by a bomb squad after officials called police to report a suspicious package.

The alert caused a partial evacuation of the plant, the BBC was among those to report Wednesday. The owner of the plant, British drugmaker Wockhardt UK, said it was cooperating with local authorities and that there were no reports of any injuries.