Vas Narasimhan, Novartis CEO (Patrick Straub/Keystone via AP)

No­var­tis, BeiGene lay out front­line PhI­II da­ta for PD-1 drug as sec­ond-line PDU­FA date looms

As the FDA pre­pares to de­cide on No­var­tis and BeiGene’s PD-1 drug tislelizum­ab in the sec­ond-line set­ting, the pair is al­ready prepar­ing for its po­ten­tial use as pa­tients’ first treat­ment op­tion.

The two com­pa­nies an­nounced at an ES­MO con­gress on gas­troin­testi­nal can­cer Thurs­day that first-line pa­tients on the an­ti-PD-1 an­ti­body plus chemother­a­py re­port­ed a me­di­an over­all sur­vival of 17.2 months ver­sus 10.6 months for pa­tients on chemo plus place­bo. This meets the study’s pri­ma­ry end­point of over­all sur­vival. The risk of death in pa­tients on the PD-1 drug was al­so 34% low­er than pa­tients on place­bo, clock­ing in a p-val­ue of un­der 0.0001.

The Phase III study was in­ves­ti­gat­ing tislelizum­ab as a front­line ther­a­py for adult pa­tients with un­re­sectable, lo­cal­ly ad­vanced or metasta­t­ic esophageal squa­mous cell car­ci­no­ma (ES­CC), re­gard­less of a pa­tient’s PD-L1 sta­tus. These da­ta were re­port­ed less than two weeks be­fore tislelizum­ab’s PDU­FA date as a sec­ond-line treat­ment, which the FDA has set for Ju­ly 12.

In pa­tients with a PD-L1 score equal or above 10% (go­ing for one of the sec­ondary end­points in the study), tislelizum­ab plus chemother­a­py showed a me­di­an over­all sur­vival of 16.6 months, giv­ing a 66% ad­van­tage over pa­tients in the chemo plus place­bo co­hort, which saw a me­di­an OS of 10.0 months. Risk of death in the first group was re­duced by 38%, with a p-val­ue of p=0.002.

On the flip slide, in pa­tients with PD-L1 score less than 10% as part of an ex­plorato­ry analy­sis, the me­di­an OS for tislelizum­ab and chemother­a­py was slight­ly high­er than in the 10%-plus PD-L1 pa­tient group, re­port­ing 16.7 months ver­sus 10.4 months in the con­trol group.

Tislelizum­ab plus chemother­a­py al­so sig­nif­i­cant­ly im­proved pro­gres­sion-free sur­vival and ob­jec­tive re­sponse rate, each with p-val­ues be­low 0.0001.

On safe­ty, No­var­tis said that treat­ment-re­lat­ed ad­verse events were sim­i­lar across both arms. Those events in­clud­ed ane­mia (68% in the tislelizum­ab group vs 61% in chemo on­ly), de­creased neu­trophils (78% vs 80%), de­creased white blood cell count (55% vs 65%), de­creased ap­petite (39% vs 38%), nau­sea (37% vs 42%) and pe­riph­er­al sen­so­ry neu­ropa­thy (26% vs 21%).

The drug can­di­date was al­so ac­cept­ed for re­view by the EMA back in April, in­clud­ing in in­di­ca­tions for mul­ti­ple non-small cell lung can­cer on top of ES­CC.

No­var­tis paid BeiGene $650 mil­lion in cash up­front ear­ly last year to in-li­cense tislelizum­ab and take it abroad, as BeiGene’s PD-1 in­hibitor had al­ready been ap­proved in Chi­na for eight on­col­o­gy in­di­ca­tions so far. At the time the deal was inked, $1.55 bil­lion in fu­ture reg­u­la­to­ry/sales mile­stones and roy­al­ty rights were on the ta­ble.

So far, those mar­kets in­clude Japan, Eu­rope and North Amer­i­ca, com­pris­ing more than 30 coun­tries so far. And with the FDA’s demon­strat­ed re­luc­tance ear­li­er this year on grant­i­ng drug ap­provals based sole­ly on Chi­nese da­ta, BeiGene and No­var­tis’ da­ta con­trast with a sim­i­lar drug from Eli Lil­ly, as the pair con­duct­ed re­search at more than 160 tri­al sites, in­clud­ing in Chi­na, the US, Aus­tralia, Italy and Spain.

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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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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.

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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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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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.”

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.

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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