Mov­ing on from a tri­al dis­as­ter, Juno re­ports pos­i­tive da­ta for JCAR017 -- and a death

Look­ing to dis­tance it­self from a tri­al dis­as­ter that claimed 5 lives last year, Juno Ther­a­peu­tics $JUNO to­day turned to a batch of pos­i­tive ear­ly-stage non-Hodgkin lym­phoma da­ta on its new lead CAR-T ther­a­py, JCAR017.

In­ves­ti­ga­tors for the biotech say that they have gath­ered da­ta that demon­strate its promise, out­lin­ing a com­bined over­all re­sponse rate of 86% and a com­plete re­sponse rate of 59% — 26 out of 44 pa­tients.

At the three-month mark, the ORR was 66% with a 50% com­plete re­sponse rate. As of the May 4 da­ta cut­off lev­el, 37 out of the 38 re­spond­ing pa­tients were still alive.

The safe­ty pro­file, though, in­clud­ed the death of one 82-year-old pa­tient suf­fer­ing from dif­fuse alve­o­lar dam­age which was linked to the drug and the flu/cy con­di­tion­ing reg­i­men that is used to pre­pare pa­tients for a bet­ter re­sponse to ther­a­py.

Al­so, while on­ly one pa­tient suf­fered from se­vere cy­tokine re­lease syn­drome, which was not fa­tal, there were 8 cas­es — 18% of the to­tal — of neu­ro­tox­i­c­i­ty.

Juno was forced to scrap its lead, JCAR015, af­ter it tried and failed to make it safe enough for pa­tients. Five pa­tients in that study died from cere­bral ede­ma, two af­ter the study was briefly sus­pend­ed by a clin­i­cal hold put on by the FDA af­ter the first three deaths. That dis­as­ter forced Juno well be­hind the two lead­ers in the field: No­var­tis and Kite, which are both post­ing fresh da­ta this week.

Sunil Agar­w­al

“To­day’s up­date of da­ta from the TRAN­SCEND tri­al shows con­tin­ued com­pelling re­sults in pa­tients with a wide range of ag­gres­sive NHL,” said Sunil Agar­w­al, Juno’s pres­i­dent of re­search and de­vel­op­ment. “We are en­cour­aged by the high rates of durable re­spons­es and the ear­ly sur­vival da­ta in these pa­tients. We are al­so en­cour­aged by the ear­ly safe­ty da­ta—a ma­jor­i­ty of pa­tients treat­ed ex­pe­ri­enced no cy­tokine re­lease syn­drome or neu­ro­tox­i­c­i­ty of any grade, which sug­gests the po­ten­tial for out­pa­tient ad­min­is­tra­tion.”

MedTech clinical trials require a unique regulatory and study design approach and so engaging a highly experienced CRO to ensure compliance and accurate data across all stages is critical to development milestones.

In­no­v­a­tive MedTech De­mands Spe­cial­ist Clin­i­cal Tri­al Reg­u­la­to­ry Af­fairs and De­sign

Avance Clinical is the Australian CRO for international biotechs providing world-class clinical research services with FDA-accepted data across all phases. With Avance Clinical, biotech companies can leverage Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend. The CRO has been delivering clinical drug development services for international biotechs for FDA and EMA regulatory approval for the past 24 years. The company has been recognized for the past two consecutive years with the prestigious Frost & Sullivan CRO Best Practices Award and a finalist in Informa Pharma’s Best CRO award for 2022.

Uğur Şahin, BioNTech CEO (Kay Nietfeld/picture-alliance/dpa/AP Images)

De­spite falling Covid-19 sales, BioN­Tech main­tains '22 sales guid­ance

While Pfizer raked in almost $28 billion last quarter, its Covid-19 vaccine partner BioNTech reported a rise in total dose orders but a drop in sales.

The German biotech reported over $3.2 billion in revenue in Q2 on Monday, down from more than $6.7 billion in Q1, in part due to falling Covid sales. While management said last quarter that they anticipated a Covid sales drop — CEO Uğur Şahin said at the time that “the pandemic situation is still very much uncertain” — Q2 sales still missed consensus by 14%.

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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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David Reese, Amgen R&D chief

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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FDA commissioner Rob Califf (Tom Williams/CQ Roll Call via AP Images)

With drug pric­ing al­most done, Con­gress looks to wrap up FDA user fee leg­is­la­tion

The Senate won’t return from its summer recess until Sept. 6, but when it does, it officially has 18 business days to finalize the reauthorization of the FDA user fee programs for the next 5 years, or else thousands of drug and biologics reviewers will be laid off and PDUFA dates will vanish in the interim.

FDA commissioner Rob Califf recently sent agency staff a memo explaining how, “Our latest estimates are that we have carryover for PDUFA [Prescription Drug User Fee Act], the user fee funding program that will run out of funding first, to cover only about 5 weeks into the next fiscal year.”

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, just two weeks after submitting a supplemental BLA. 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.

Steve Paul, Karuna Therapeutics CEO (Third Rock)

Karuna's schiz­o­phre­nia drug pass­es a close­ly-watched PhI­II test, will head to FDA in mid-2023

An investigational pill that combines a former Eli Lilly CNS compound with an overactive bladder drug was better than placebo at reducing a scale of symptoms experienced by patients with schizophrenia in a Phase III trial.

Karuna Therapeutics’ drug passed the primary goal in EMERGENT-2, the Boston biotech said early Monday morning, alongside quarterly earnings. The study is the first of Karuna’s four Phase III clinical trials to read out in schizophrenia and will provide the backbone to the biotech’s first drug approval application, slated for mid-2023.

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GSK and IQVIA launch plat­form of US vac­ci­na­tion da­ta, show­ing drop in adult rates

Throughout the Covid-19 pandemic, the issue of vaccine uptake has been a point of contention, but a new platform from GSK and IQVIA is hoping to shed more light on vaccine data, via new transparency and general awareness.

The two companies have launched Vaccine Track, a platform intended to be used by public health officials, medical professionals and others to strengthen data transparency and display vaccination trends. According to the companies, the platform is intended to aid in increasing vaccine rates and will provide data on trends to assist public health efforts.

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Ab­b­Vie sur­veys emo­tion­al im­pact of chron­ic leukemia con­di­tion, finds 'roller coast­er' of emo­tions

Rare diseases often have more than just physical effects on patients — especially when it comes to chronic conditions. In the case of the rare slow-growing blood cancer chronic lymphocytic leukemia (CLL), AbbVie wanted to try to assess the mental and emotional toll on patients.

So it surveyed more than 300 CLL patients, caregivers and physicians. While each group differed in how they felt — caregivers overwhelmingly (81%) felt positive about their role, for instance — patients described a “roller coaster” of emotions traversing diagnosis to treatment to remission and even relapse for some.