J&J's block­buster hope­ful erdafi­tinib gets 'break­through' moniker

Janssen has snagged the FDA’s break­through sta­tus on its sol­id tu­mor drug can­di­date erdafi­tinib, putting J&J one step clos­er to its lofty goal of bring­ing nu­mer­ous new block­buster drugs to mar­ket.

The drug, an FGFR in­hibitor, got the des­ig­na­tion for its po­ten­tial to treat pa­tients with metasta­t­ic urothe­lial can­cer. Urothe­lial can­cer is the sixth most com­mon type of can­cer in the US, most fre­quent­ly show­ing up in the blad­der.

Pe­ter Lebowitz

“For pa­tients di­ag­nosed with urothe­lial can­cer, out­comes are un­for­tu­nate­ly dis­heart­en­ing due to the ag­gres­sive­ness of the dis­ease,” said Pe­ter Lebowitz, Janssen’s glob­al on­col­o­gy head, in a state­ment. “Through the con­tin­ued de­vel­op­ment of erdafi­tinib, and work­ing close­ly with the FDA, we look for­ward to bring­ing a po­ten­tial new treat­ment op­tion to pa­tients.”

The break­through ther­a­py des­ig­na­tion is based on da­ta from a Phase II tri­al. The da­ta, pre­sent­ed at AS­CO, show an over­all re­sponse rate of 42% in 59 pa­tients with re­lapsed/re­frac­to­ry metasta­t­ic urothe­lial can­cer whose tu­mors har­bored ac­tion­able FGFR mu­ta­tions.

Erdafi­tinib is one of 11 block­buster hope­fuls in J&J’s pipeline — a list the phar­ma gi­ant tout­ed last sum­mer, each with a peak sales promise of $1 bil­lion-plus. The top drugs in­clude two new im­munol­o­gy drugs al­ready filed for ap­proval and well known to in­vestors: guselkum­ab for pso­ri­a­sis and sirukum­ab for rheuma­toid arthri­tis. The re­main­ing ther­a­pies cov­er a range of fo­cus­es, but with a no­table con­cen­tra­tion in on­col­o­gy.

J&J needs them all to keep up with an am­bi­tious growth fore­cast for its phar­ma di­vi­sion rev­enue.

J&J gets a fresh OK for es­ke­t­a­mine, but is it re­al­ly the game-chang­er for de­pres­sion Trump keeps tweet­ing about?

Backed by an enthusiastic set of tweets from President Trump and a landmark OK for depression, J&J scooped up a new approval from the FDA for Spravato today. But this latest advance will likely bring fresh scrutiny to a drug that’s spurred some serious questions about the data, as well as the price.

First, the approval.

Regulators stamped their OK on the use of Spravato — developed as esketamine, a nasal spray version of the party drug Special K or ketamine — for patients suffering from major depressive disorder with acute suicidal ideation or behavior.

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FDA hands Mor­phoSys and In­cyte a quick OK on their po­ten­tial block­buster CAR-T al­ter­na­tive

Nearly three years after okaying the CAR-Ts Yescarta and Kymriah, the FDA has approved a new CD19 therapy.

MorphoSys’ Monjuvi, or tafasitamab-cxix, was cleared Friday for use in refractory diffuse large B-cell lymphoma (DBLCL). The approval sets up both MorphoSys and their commercial partner Incyte to compete with Gilead and Novartis in the ultra-competitive indication, where similar trial results and far easier delivery could allow them to cut a fair share of the market.

CymaBay flash­es pos­i­tive re­sults from the tri­al they have to re­launch

Two weeks after the FDA lifted its clinical hold on their lead drug, CymaBay said it showed positive results in an aborted Phase III trial.

The drug, a small molecule known as seladelpar, had been in development for three different liver conditions before an independent review of a NASH study last year showed that it might actually be damaging patient’s liver cells. The FDA slapped a clinical hold across all three trials, only lifting it last month when an FDA review determined that the drug hadn’t caused liver damage.

So Covid-19 leader BioN­Tech has a can­cer vac­cine in de­vel­op­ment? Yes, and Re­gen­eron just jumped in for the PhII com­bo study

Before the coronavirus global emergency stole the R&D show in biopharma, the leaders in the race to develop new mRNA therapies had a big interest in determining if their tech could be used to create an effective cancer vaccine after all the first-gen tries had failed to impress. So perhaps it’s not surprising that an early cut of the data at frontrunner BioNTech went largely unnoticed.

Unless you were at Regeneron.

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Sanofi un­der for­mal in­ves­ti­ga­tion for De­pakine al­le­ga­tions; Beam li­cens­es CAR-T tech from Ox­ford Bio­med­ica

Sanofi is facing a formal investigation on manslaughter charges, due to accusations that its epilepsy drug Depakine caused birth malfunctions and slow neurological development when taken during pregnancy.

The French pharma was formally charged in February, years after evidence surfaced that the drug, sodium valproate, posed neurodevelopmental risks. Sodium valproate first hit the market in 1967 for the treatment of epilepsy and bipolar disorder, and is currently prescribed in more than 100 countries.

Rich Heyman (ARCH)

Rich Hey­man joins PMV Phar­ma, a p53 biotech, as it adds $70 mil­lion in Se­ries D

Less than a year after pulling in an impressive $62 million Series C round, PMV Pharma is back at it again.

The Cranbury, NJ-based biotech announced Monday an additional $70 million in Series D financing as it seeks to develop cancer therapies targeting p53 mutations. Additionally, PMV also introduced longtime biotech entrepreneur Rich Heyman as chairman of the board of directors.

“This financing provides PMV Pharma with the resources to expand our pipeline and to potentially advance multiple p53 therapies into the clinic,” said PMV president and CEO David Mack in a statement.

Days af­ter seal­ing Sanofi pact, Kymera beats a path to the Nas­daq with $100M IPO pitch

Back in March, when Kymera Therapeutics closed $102 million in Series C funding led by Biotechnology Value Fund and Redmile Group, CEO Nello Mainolfi noted the protein degradation player was “at the cusp of transitioning” into a fully integrated R&D company. Five months and a major Sanofi pact later, he’s back asking for another little push to get there.

Kymera has penciled in $100 million in its first IPO pitch — although given the public market’s seemingly insatiable appetite for biotechs these days the final figure is anyone’s guess.

Covid-19 roundup: Eli Lil­ly retro­fits RVs for first-of-its-kind an­ti­body tri­al with NIH; Am­gen, Ab­b­Vie, Take­da team on a drug

Eli Lilly and the NIH are about to start a first-of-its-kind trial that researchers and developers have talked about for months as a way of providing temporary immunity to the most at-risk populations.

Lilly announced this morning that it will start a 2,400-person trial with the National Institute for Allergy and Infectious Diseases to test whether its experimental Covid-19 neutralizing antibody can prevent people in nursing homes and assisted living facilities from developing the disease. The idea, known as passive immunity, is that rather than waiting on a vaccine to induce people to develop antibodies, doctors can give them lab-grown antibodies. Ideally, those antibodies will either attack the new SARS-CoV-2 infection, if the patient has recently been exposed, or persist in the blood for several weeks and prevent infection or disease for that period.

Im­mu­nic's lead MS drug hits pri­ma­ry and key sec­ondary end­points in PhII, but ques­tions re­main

Just a week after its lead program began enrolling patients in a study to treat Covid-19, Immunic Therapeutics is making more waves.

This time, the biotech is providing a glimpse at topline data from a Phase II trial studying the efficacy of vidofludimus calcium, or IMU-838, in relapsing-remitting multiple sclerosis patients. Taken orally, the candidate met its primary endpoint in reducing the cumulative number of combined unique active MRI lesions after 24 weeks for a 45 mg dose compared to a placebo, as well as a key secondary endpoint in such reductions for the 30 mg dose.