Hunt­ing a full OK for Baven­cio in front­line blad­der can­cer, Pfiz­er, Mer­ck KGaA re­ceive VIP treat­ment at FDA

The JAVELIN Blad­der 100 study was one of the rare bright spots in Baven­cio’s check­ered de­vel­op­ment record. Now Pfiz­er and Mer­ck KGaA get to bur­nish that with a break­through ther­a­py des­ig­na­tion for first-line main­te­nance treat­ment of lo­cal­ly ad­vanced or metasta­t­ic urothe­lial car­ci­no­ma.

The part­ners have sub­mit­ted their sBLA, which is now be­ing re­viewed on a re­al-time ba­sis. Un­der that pi­lot pro­gram — which be­gan in late 2018 — reg­u­la­tors could ac­cess da­ta pri­or to the of­fi­cial ap­pli­ca­tion fil­ing.

Pfiz­er and Mer­ck KGaA would have ap­plied for the RTOR at the be­gin­ning of this year, when it re­leased topline re­sults of a Phase III study with Baven­cio. As a main­te­nance ther­a­py, the PD-L1 drug hit the mark on over­all sur­vival for both the to­tal pa­tient group and a sub­set with PD-L1 pos­i­tive tu­mors.

The drug was al­ready sanc­tioned, un­der ac­cel­er­at­ed ap­proval, for use in pa­tients fol­low­ing dis­ease pro­gres­sion — an OK ce­ment­ed by tu­mor re­sponse and du­ra­tion of re­sponse back in 2017.

“For the past 30 years, chemother­a­py has been the first-line stan­dard of care for pa­tients with ad­vanced urothe­lial car­ci­no­ma. While this is an ef­fec­tive short-term op­tion for many pa­tients, most will ul­ti­mate­ly ex­pe­ri­ence dis­ease pro­gres­sion, un­der­scor­ing a need for ad­di­tion­al treat­ment op­tions,” said Pet­ros Gri­vas, a prin­ci­pal in­ves­ti­ga­tor in the JAVELIN Blad­der 100 tri­al.

Chris Boshoff

In the tri­al, Baven­cio was added to the reg­i­men for pa­tients whose dis­ease had not pro­gressed dur­ing or fol­low­ing plat­inum-con­tain­ing chemo. The OS re­sults could be prac­tice-chang­ing, Gri­vas added.

Chris Boshoff, Pfiz­er’s chief de­vel­op­ment of­fi­cer in on­col­o­gy, high­light­ed that Baven­cio is the first im­munother­a­py to demon­strate an OS ben­e­fit in first-line, lo­cal­ly ad­vanced or metasta­t­ic urothe­lial car­ci­no­ma.

As­traZeneca pro­vid­ed the lat­est il­lus­tra­tion on just how tough it can be to beat this ad­vanced can­cer when Imfinzi flopped an open la­bel piv­otal study last month. The ther­a­py — which al­so blocks PD-L1 — nabbed ac­cel­er­at­ed ap­proval in 2017 but failed to help pa­tients live longer com­pared to stan­dard-of-care chemother­a­py. The drug­mak­er may shift their fo­cus to the sec­ond-line set­ting, it in­di­cat­ed to End­points News.

Biotech and Big Phar­ma: A blue­print for a suc­cess­ful part­ner­ship

Strategic partnerships have long been an important contributor to how drugs are discovered and developed. For decades, big pharma companies have been forming alliances with biotech innovators to increase R&D productivity, expand geographical reach and better manage late-stage commercialization costs.

Noël Brown, Managing Director and Head of Biotechnology Investment Banking, and Greg Wiederrecht, Ph.D., Managing Director in the Global Healthcare Investment Banking Group at RBC Capital Markets, are no strangers to the importance of these tie-ups. Noël has over 20 years of investment banking experience in the industry. Before moving to the banking world in 2015, Greg was the Vice President and Head of External Scientific Affairs (ESA) at Merck, where he was responsible for the scientific assessment of strategic partnership opportunities worldwide.

No­var­tis' sec­ond at­tempt to repli­cate a stun­ning can­cer re­sult falls flat

Novartis’ hopes of turning one of the most surprising trial data points of the last decade into a lung cancer drug has taken another setback.

The Swiss pharma announced Monday that its IL-1 inhibitor canakinumab did not significantly extend the lives or slow the disease progression of patients with previously untreated locally advanced or metastatic non-small cell lung cancer when compared to standard of-care alone.

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How Chi­na turned the ta­bles on bio­phar­ma's glob­al deal­mak­ing

Fenlai Tan still gets chills thinking about the darkest day of his life.

Three out of eight lung cancer patients who received a tyrosine kinase inhibitor developed by his company, Betta Pharma, died in the span of a month. Tan, the chief medical officer, was summoned to Peking Union Medical College Hospital, where the head of the clinical trial department told him that the trial investigators would be conducting an autopsy to see if the patients had died of the disease — they were all very sick by the time they enrolled — or of interstitial lung disease, a deadly side effect tied to the TKI class that’s been reported in Japan.

No­var­tis dumps AveX­is pro­gram for Rett syn­drome af­ter fail­ing re­peat round of pre­clin­i­cal test­ing

Say goodbye to AVXS-201.

The Rett syndrome gene therapy drug made by AveXis — the biotech that was bought, kept separate, then renamed and finally absorbed by Novartis into its R&D division — has been dropped by the biopharma.

In Novartis’ third quarter financial report, the pharma had found that preclinical data did not support development of the gene therapy into IND-enabling trials and beyond. The announcement comes a year after Novartis told the Rett Society how excited it was by the drug — and its potential benefits and uses.

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FDA is much worse than its reg­u­la­to­ry peers at proac­tive­ly dis­clos­ing da­ta, re­searchers find

The European Medicines Agency and Health Canada continue to outpace the FDA when it comes to proactively releasing data on drugs and biologics the agency has reviewed, leading to further questions of why the American agency can’t be more transparent.

In a study published recently in the Journal of Law, Medicine, & Ethics, Yale and other academic lawyers and researchers found that between 2016 and April 2021, the EMA proactively released data for 123 unique medical products, while Health Canada proactively released data for 73 unique medical products between 2019 and April 2021. What’s more, the EMA and Health Canada didn’t proactively release the same data on the same drugs. In stark contrast, the FDA in 2018 only proactively disclosed data supporting one drug that was approved that year.

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As pres­sure to share tech­nol­o­gy mounts, BioN­Tech se­lects Rwan­da for lat­est vac­cine site

BioNTech’s first mRNA-based vaccine site in Africa will call Rwanda home, and construction is set to start in mid-2022, the company announced Tuesday at a public health forum.

The German company signed a memorandum of understanding, after a meeting between Rwanda’s Minister of Health, Daniel Ngamije, Senegal’s Minister of Foreign Affairs Aïssata Tall Sall, and senior BioNTech officials. Construction plans have been finalized, and assets have been ordered. The agreement will help bring end-to-end manufacturing to Africa, and as many as several hundred million doses of vaccines per year, though initial production will be more modest.

Robert Spurr, President Salix Pharmaceuticals

Bausch Health’s Sal­ix pi­lots study to shine light on chron­ic liv­er dis­ease and push back on stereo­types

October is both breast cancer awareness and liver disease awareness month. While there’s no doubt which condition draws more attention during the month, Salix wants to change that.

Salix, Bausch Health’s gastroenterology arm, piloted its first chronic liver disease report and physician survey with results out this week aimed at raising awareness and dispelling stereotypes.

While 4.5 million people have chronic liver disease or cirrhosis – which is even more than 3.8 million women diagnosed with breast cancer – the research found chronic liver disease “has not received the attention or level of effort needed for adequate prevention, diagnosis, and standardization of its management.”

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Sean Ianchulev, Eyenovia CEO and CMO

Re­cent court de­ci­sion push­es FDA to re­ject and re­clas­si­fy drug-de­vice com­bo, crush­ing shares

Back in April, the FDA lost a crucial court case in which its broad discretion of regulating medical products that might satisfy the legal definitions of either “drug” and/or “medical device” was sharply curtailed.

In addition to the appeals court ruling that Genus Medical Technologies’ contrast agent barium sulfate (aka Vanilla SilQ) should not be considered a drug, as the FDA had initially ruled, but as a medical device, the agency also was forced to spell out which drugs would transition to devices as a result of the ruling.

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Katie Fanning, Mozart Therapeutics CEO

Mozart Ther­a­peu­tics makes its of­fi­cial de­but, jump­ing in­to the hot Treg R&D field with some big-name in­vestors back­ing it

Treg cells have been getting more and more attention recently among autoimmune specialists. There’s been Jeff Bluestone’s Sonoma, the $157 million launch of GentiBio this summer and Egle Therapeutics — which launched just last week — to name a few.

Now, there’s a new Treg player jumping in that wants to distinguish itself in the market: Mozart Therapeutics. Today, the biotech is emerging from stealth in its official debut with a $55 million Series A — with a bunch of A-list Big Pharma names on board a syndicate led by ARCH.