Proven­tion Bio roller­coast­er con­tin­ues with gen­er­al­ly pos­i­tive re­marks from FDA ahead of Thurs­day’s ad­comm

Ear­ly last month, Proven­tion Bio’s shares cratered af­ter dis­clos­ing that the FDA found the phar­ma­co­ki­net­ic pro­files of its po­ten­tial type 1 di­a­betes drug, ac­quired from Eli Lil­ly, dif­fered when man­u­fac­tured by Lil­ly ver­sus Proven­tion.

At the time, the FDA said its con­cerns meant that it was not ready to start post-mar­ket­ing and la­bel dis­cus­sions with the com­pa­ny.

But now, two days ahead of the FDA’s En­docrino­log­ic and Meta­bol­ic Drugs Ad­vi­so­ry Com­mit­tee meet­ing to dis­cuss the drug, known as teplizum­ab, the FDA sound­ed a rel­a­tive­ly pos­i­tive tone on safe­ty and ef­fi­ca­cy. The com­pa­ny’s stock was up about 30% ear­ly Tues­day.

While not­ing some miss­ing da­ta from the com­pa­ny’s tri­als, the FDA said Tues­day that “be­cause the amount of miss­ing da­ta was small, sen­si­tiv­i­ty analy­ses per­formed us­ing dif­fer­ent miss­ing da­ta han­dling ap­proach­es show that the ob­served ef­fi­ca­cy by the pri­ma­ry analy­sis method is suf­fi­cient­ly ro­bust to with­stand con­ser­v­a­tive ap­proach­es in han­dling miss­ing da­ta.”

The ques­tion of what con­sti­tutes sub­stan­tial ev­i­dence of ef­fec­tive­ness will be top of mind for the com­mit­tee to ad­dress, FDA notes, ex­plain­ing how Proven­tion sub­mit­ted a rel­a­tive­ly small, sin­gle place­bo-con­trolled tri­al with the pri­ma­ry end­point of de­lay of clin­i­cal type 1 di­a­betes, as well as ad­di­tion­al da­ta, which FDA said can be used as “con­fir­ma­to­ry ev­i­dence.”

On safe­ty, the brief­ing doc­u­ments did not re­veal any glar­ing red flags, al­though the agency not­ed that “slight­ly more than 10% of pa­tients” in the tri­al “were not able to re­ceive the full course of teplizum­ab sec­ondary to meet­ing pro­to­col-spec­i­fied with­draw­al cri­te­ria.”

The com­mit­tee on Thurs­day will be tasked with not on­ly dis­cussing the clin­i­cal mean­ing­ful­ness of the ob­served me­di­an 2-year de­lay of on­set of type 1 di­a­betes demon­strat­ed in the com­pa­ny’s main study, but vot­ing on an up or down ap­proval ques­tion ahead of the FDA’s de­ci­sion, which is due by Ju­ly 2.

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In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

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Mathai Mammen (Rob Tannenbaum, Endpoints News at BIO 2018)

Math­ai Mam­men makes an abrupt ex­it as head of the big R&D group at J&J

In an after-the-bell shocker, J&J announced Monday evening that Mathai Mammen has abruptly exited J&J as head of its top-10 R&D group.

Recruited from Merck five years ago, where the soft-spoken Mammen was being groomed as the successor to Roger Perlmutter, he had been one of the top-paid R&D chiefs in biopharma. His group spent $12 billion last year on drug development, putting it in the top 5 in the industry.

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Illustration: Kim Ryu for Endpoints News

Why non-opi­oid pain drugs keep fail­ing — and what's next for the field

In 1938, Rita Levi-Montalcini was forced to move her lab into her bedroom in Turin, as Mussolini’s facist government expelled Jewish people from studying or working in schools in Italy. Levi-Montalcini, then just a few years out of medical school and using sewing needles as scalpels in her makeshift lab, would soon discover nerve growth factor, or NGF, in chicken embryos.

Her discoveries formed the basis of our understanding of the peripheral nervous system and how cells talk to each other, and Levi-Montalcini went on to win the Nobel Prize in 1986. Much later, NGF was hailed as a promising target for new pain therapies, with some analysts quoting an $11 billion market. However, the latest anti-NGF candidate, Pfizer and Eli Lilly’s tanezumab, was rejected by the FDA last year because of a side effect that dissolved bone in some of its patients.

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Ted Love, Global Blood Therapeutics CEO

Up­dat­ed: Pfiz­er scoops up Glob­al Blood Ther­a­peu­tics and its sick­le cell ther­a­pies for $5.4B

Pfizer is dropping $5.4 billion to acquire Global Blood Therapeutics.

Just ahead of the weekend, word got out that Pfizer was close to clinching a $5 billion buyout — albeit with other potential buyers still at the table. The pharma giant, flush with cash from Covid-19 vaccine sales, apparently got out on top.

The deal immediately swells Pfizer’s previously tiny sickle cell disease portfolio from just a Phase I program to one with an approved drug, Oxbryta, plus a whole pipeline that, if all approved, the company believes could make for a $3 billion franchise at peak.

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HHS Secretary Xavier Becerra (Patrick Semansky/AP Images)

US weighs new route of ad­min­is­tra­tion for mon­key­pox vac­cine as cas­es climb — re­port

Less than a week after HHS Secretary Xavier Becerra declared monkeypox a national health emergency, reports have emerged that the US plans to extend its vaccine supply by opting for a different route of administration.

Officials are expected to call for intradermal injection of Bavarian Nordic’s Jynneos vaccine — the only shot approved specifically for monkeypox in the US — as opposed to subcutaneous injection, unnamed sources told both the New York Times and Washington Post on Tuesday.

'Messy at best': Is the US re­peat­ing the same Covid mis­steps with mon­key­pox mes­sag­ing?

When Kyle Planck first suspected he might have monkeypox in late June, he went to the CDC website and found six photos of different types of lesions. And that was about it for general public information.

Planck, who is a sixth-year PhD pharmacology researcher at Weill Cornell, kept looking though and found a separate part of the CDC website meant for healthcare professionals. There he found a medical slide deck with more pictures, professional journal articles and more details about symptoms and diagnosis.

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Andy Jassy, Amazon CEO (Isaac Brekken/AP Images for NFL, File)

Up­dat­ed: FDA slaps Ama­zon with a warn­ing let­ter for sell­ing OTC mole re­moval prod­ucts

The FDA’s Center for Drug Evaluation and Research on Tuesday released a warning letter sent last week to Amazon CEO Andy Jassy in Seattle for selling mole removal products over-the-counter, or, as the FDA explains, “introducing, delivering, or causing the introduction or delivery into interstate commerce of products that are unapproved new drugs.”

“There are no over-the-counter (OTC) drugs that can be legally sold for mole or skin tag removal, and FDA has safety concerns about drugs marketed OTC directly to consumers for these uses,” the agency said in its Aug. 4 warning.

US to stop sup­ply­ing Lil­ly's mAb for Covid-19 this month as com­mer­cial mar­ket awaits

Federal officials said yesterday that shipments of Eli Lilly’s bebtelovimab — one of the final two remaining mAb treatments for Covid-19 — would halt later this month, setting up a commercial market where the government no longer pays for the doses and hospitals and other clinics will have to purchase supplies.

According to ASPR, the arm of HHS that ships Covid-19 drugs, states have ordered 627,536 bebtelovimab courses, and 383,515 courses have been administered as of July 31. The US has paid Lilly a total of about $1.27 billion for all of the courses so far, amounting to about $2,100 per course to start and then receiving a discounted $1,833 ASP for the later part of the deal. According to the Wall Street Journal, Lilly’s list price for bebtelovimab is $2,100 per dose.

Pascal Soriot, AstraZeneca CEO (David Zorrakino/Europa Press via AP Images)

As­traZeneca and Dai­ichi Sankyo sprint to mar­ket af­ter FDA clears En­her­tu in just two weeks

Regulators didn’t keep AstraZeneca and Daiichi Sankyo waiting long at all for their latest Enhertu approval.

The partners pulled a win on Friday in HER2-low breast cancer patients who’ve already failed on chemotherapy, less than two weeks after its supplemental BLA was accepted. While this isn’t the FDA’s fastest approval — Bristol Myers Squibb won an OK for its blockbuster checkpoint inhibitor Opdivo in just five days back in March — it comes well ahead of Enhertu’s original Q4 PDUFA date.